#  @surferbackpack Randall Ching
Randall Ching posts on X about $srpt, fda, $capr, $galt the most. They currently have [---] followers and [---] posts still getting attention that total [-----] engagements in the last [--] hours.
### Engagements: [-----] [#](/creator/twitter::1332900003812052996/interactions)

- [--] Week [------] +299%
- [--] Month [------] +2,064%
- [--] Months [------] -2.50%
- [--] Year [------] +2,728%
### Mentions: [--] [#](/creator/twitter::1332900003812052996/posts_active)

- [--] Week [--] +160%
- [--] Month [--] +329%
- [--] Months [--] -35%
- [--] Year [---] +1,682%
### Followers: [---] [#](/creator/twitter::1332900003812052996/followers)

- [--] Week [---] +14%
- [--] Month [---] +22%
- [--] Months [---] +230%
- [--] Year [---] +1,595%
### CreatorRank: [---------] [#](/creator/twitter::1332900003812052996/influencer_rank)

### Social Influence
**Social category influence**
[stocks](/list/stocks) [finance](/list/finance) [technology brands](/list/technology-brands) [social networks](/list/social-networks) [cryptocurrencies](/list/cryptocurrencies) [ncaa basketball](/list/ncaa-basketball) [travel destinations](/list/travel-destinations)
**Social topic influence**
[$srpt](/topic/$srpt), [fda](/topic/fda) #122, [$capr](/topic/$capr), [$galt](/topic/$galt), [data](/topic/data), [market](/topic/market), [$sprb](/topic/$sprb) #1, [gene](/topic/gene), [in the](/topic/in-the), [protocol](/topic/protocol)
**Top accounts mentioned or mentioned by**
[@jeremyj0916](/creator/undefined) [@brainymarsupial](/creator/undefined) [@lqdabull1](/creator/undefined) [@dashwend](/creator/undefined) [@sanctuarybio](/creator/undefined) [@stocksavvyshay](/creator/undefined) [@golfnut48846224](/creator/undefined) [@sarwanjohn](/creator/undefined) [@hall8jack](/creator/undefined) [@greatsehaj](/creator/undefined) [@stockplaymaker1](/creator/undefined) [@daveconnellysg](/creator/undefined) [@toucantrades](/creator/undefined) [@tariusishere](/creator/undefined) [@westsideguy3](/creator/undefined) [@alohaoasis](/creator/undefined) [@alansharp](/creator/undefined) [@yachmod](/creator/undefined) [@yaniruliel](/creator/undefined) [@stockchumpski](/creator/undefined)
**Top assets mentioned**
[Sarepta Therapeutics, Inc. (SRPT)](/topic/$srpt) [Spruce Biosciences, Inc. (SPRB)](/topic/$sprb) [Akropolis (AKRO)](/topic/$akro) [Catalyst Pharmaceutical Inc. (CPRX)](/topic/$cprx) [Soleno Therapeutics, Inc. Common Stock (SLNO)](/topic/$slno) [D-Wave Quantum Inc. (QBTS)](/topic/$qbts) [Biohaven Pharmaceutical Holding Company Ltd. (BHVN)](/topic/$bhvn) [Sharps Technology, Inc. (STSS)](/topic/$stss)
### Top Social Posts
Top posts by engagements in the last [--] hours
"@DaveConnellySG You watch a Temple Basketball game this season"
[X Link](https://x.com/surferbackpack/status/1766076577060737164) 2024-03-08T12:20Z [--] followers, [----] engagements
"@ToucanTrades @SarwanJohn That was up all day. Its only for the A class which $LUXH is not. Might indicate good cash flow"
[X Link](https://x.com/surferbackpack/status/1831112962062286922) 2024-09-03T23:32Z [--] followers, [---] engagements
"$RGTI $6 puts @ [----] February A reverse of the classic [----] thread: Pitch a publicly traded company which: [--]. Has at least $10 billion of market cap [--]. Will decline 90% from current (not peak) prices First pick to -90% wins ties go to earlier post. I'll pick something tasteful but thematic as a prize. A reverse of the classic [----] thread: Pitch a publicly traded company which: [--]. Has at least $10 billion of market cap [--]. Will decline 90% from current (not peak) prices First pick to -90% wins ties go to earlier post. I'll pick something tasteful but thematic as a prize"
[X Link](https://x.com/surferbackpack/status/1871683248545574969) 2024-12-24T22:23Z [--] followers, [---] engagements
"$STSS ouch. Sneaky warrant conversion in the offering. pray for SC plant pr or 💣"
[X Link](https://x.com/anyuser/status/1886426867588100596) 2025-02-03T14:49Z [---] followers, [---] engagements
"@Tariusishere stop. eps on a r/d stage company is dumb. 280k to 308k shares outstanding from last q. dilution. qc is cool stuff any public company rn is a sham. ibm/msft are where qc is at"
[X Link](https://x.com/surferbackpack/status/1922040118417756294) 2025-05-12T21:24Z [--] followers, [---] engagements
"$SRPT my thoughts part [--]. Not premium so its a thread. Alright everybody panicking on $SRPT heres my take. A second non-ambulatory Duchenne patient just died from acute liver failure after receiving Elevidys"
[X Link](https://x.com/surferbackpack/status/1934486645858042305) 2025-06-16T05:42Z [--] followers, [---] engagements
"$SRPT part [--]. Two deaths both in nonambulatory patients (first a 16yearold second age not disclosed) in three months. Sarepta now pauses Elevidys use in that group and halt its ENVISION confirmatory trial which FDA accelerated approval mandated"
[X Link](https://x.com/surferbackpack/status/1934487512086683661) 2025-06-16T05:45Z [--] followers, [---] engagements
"$SRPT part [--]. They also notified regulators and Roche (their ex-US partner) did the same globally. serious inflection point for the stock and for gene therapy trust overall"
[X Link](https://x.com/surferbackpack/status/1934487639673176479) 2025-06-16T05:46Z [--] followers, [---] engagements
"$SRPT part [--]. The FDAs watching closely especially with Vinay Prasad now heading biologics after Peter Marks stepped down. Prasads been openly critical of Elevidys even before this second death"
[X Link](https://x.com/surferbackpack/status/1934487844946653223) 2025-06-16T05:46Z [--] followers, [---] engagements
"$SRPT part [--]. Sareptas going to propose a revised protocol with added immunosuppression (possibly sirolimus + steroids) but dosing in non-ambulatory patients is paused until the FDA signs off. ambulatory use continues but even there uptake might slow"
[X Link](https://x.com/surferbackpack/status/1934488202401992810) 2025-06-16T05:48Z [--] followers, [---] engagements
"$SRPT part [--]. Families and clinicians get more cautious. ENVISION is paused label updates are coming and the path to broader adoption just got steeper"
[X Link](https://x.com/surferbackpack/status/1934488456241271270) 2025-06-16T05:49Z [--] followers, [---] engagements
"$SRPT part [--]. My concerns. Elevidys did $375M in Q1 alone and a lot of that growth was expected to come from expanding into the non-ambulatory population. Thats now frozen"
[X Link](https://x.com/anyuser/status/1934488637833626058) 2025-06-16T05:50Z [---] followers, [---] engagements
"$SRPT part [--]. If the FDA doesnt buy Sareptas revised safety strategy (theyre looking at sirolimus + steroids) its possible Elevidys loses that label entirely. Worst case thats a massive revenue hole. Best case recovery takes time (6-12 months) and the stigma lingers"
[X Link](https://x.com/surferbackpack/status/1934488983519797407) 2025-06-16T05:51Z [--] followers, [---] engagements
"$SRPT here. - Ingrams tone on Monday = everything - Rodino-Klapac: sirolimus or bust - Prasad (FDA) aint Marks not friendly - ENVISION 303: paused no restart label risk - Roche global pullback - No LGMD backfill near term - Risk premium on gene therapy just widened"
[X Link](https://x.com/surferbackpack/status/1934490327987163570) 2025-06-16T05:56Z [--] followers, [---] engagements
"$SRPT Im long. Positions small not adding yet. Let this bleed out. ENVISION needs FDA signoff. If sirolimus clears and no 3rd death recovery trades back to 40s. But if uptake in ambulatories stalls or Prasad blocks trial restart downside opens hard. [---] month trench"
[X Link](https://x.com/surferbackpack/status/1934490958051393562) 2025-06-16T05:59Z [--] followers, [---] engagements
"$SRPT good night press tomorrow. Dont need to catch knife need to know where floor is. Watching tmrw call Roche and FDA comms. Will average only when market stops reacting emotionally and starts asking about revenue math"
[X Link](https://x.com/anyuser/status/1934491130445582492) 2025-06-16T06:00Z [---] followers, [---] engagements
"$SRPT assuming black box label is coming read about Zolgensma gene therapy for SMA. Dont think the FDA pulls Elevidys now just based on patient need for DMD. One more patient death though - game over Label pulled The risk profile of gene therapies man"
[X Link](https://x.com/anyuser/status/1934541706470502854) 2025-06-16T09:20Z [---] followers, [----] engagements
"$SRPT have to say though that AAV therapies are fascinating just from my bio background. Flooding the body with viral vectors to deliver genes is a phenomenon. Then again the immune response is patient specific non linear. For the sake of these patients pray new protocol works"
[X Link](https://x.com/surferbackpack/status/1934542342750707863) 2025-06-16T09:23Z [--] followers, [---] engagements
"$SRPT a gamma trade now not a beta trade"
[X Link](https://x.com/anyuser/status/1934547097841488152) 2025-06-16T09:42Z [---] followers, [----] engagements
"$SRPT added more at [----]. buying a 1.8b company with full exclusivity in an orphan market for [--]. 100m shares outstanding"
[X Link](https://x.com/surferbackpack/status/1934656471863840846) 2025-06-16T16:57Z [--] followers, [---] engagements
"learning how to use USPTO. Cool stuff with the advanced search. Sort of like a new language with the search queries. Last updated [----] but its neat stuff. Going to try do a patent tracker for maybe quantum related developments Learning about A1 B1 B2"
[X Link](https://x.com/surferbackpack/status/1934884527362756943) 2025-06-17T08:03Z [--] followers, [--] engagements
"@jeremyj0916 Yeah Im definitely considering it. Puts way too expensive/if they delay data release those get IV crushed with no movement anyways. Ill look into shorting "
[X Link](https://x.com/surferbackpack/status/1935442316346671391) 2025-06-18T20:59Z [--] followers, [--] engagements
"$SRPT am I right in saying that capricore is not a threat to sarepta at all If deramiocel is for dmd-associated cardiomyopathy would that not just turn DMD treatment into a combination of ELEVIDYS for baseline therapy and deramiocel"
[X Link](https://x.com/anyuser/status/1935490840702632121) 2025-06-19T00:12Z [---] followers, [---] engagements
"$SRPT Sarepta's PMO business (eteplirsen golodirsen casimersen) seems to not be threatened. NS Pharma's Viltepso is the only other competitor to srpt's pmo and theyre not disrupting much"
[X Link](https://x.com/surferbackpack/status/1935491607404663125) 2025-06-19T00:15Z [--] followers, [---] engagements
"$SRPT then Agamree (the steroid competitor) is more so a steroid innovation that wouldnt disrupt dystrophin producing therapies like ELEVIDYS. Couldnt a combination of Agamree + Deramiocel + ELEVIDYS be the future norm"
[X Link](https://x.com/surferbackpack/status/1935492394511908949) 2025-06-19T00:18Z [--] followers, [---] engagements
"$SRPT even if ELEVIDYS revenue collapses by 80% and core PMO takes a hit of 30% the current share price is basically that ($20 on 06-18-2025). Basically market is saying ELEVIDYS is a [--]. Also pricing in potential lawsuits funding ENVISION brand stigma. Peak pessimism"
[X Link](https://x.com/anyuser/status/1935511966992449905) 2025-06-19T01:36Z [---] followers, [---] engagements
"$SRPT difficulty is this. its not as simple as seeing the 0.25% incidence rate of acute liver failure auto assuming that benefit outweighs risk/harm. FDA standard is 'do no harm'. unpredictable death is a monumental failure. srpt has to convince FDA of revised safety protocol"
[X Link](https://x.com/anyuser/status/1935598822471922147) 2025-06-19T07:21Z [---] followers, [---] engagements
"$SRPT Great article from Christine McSherry captures the more human parts of this situation: her son had DMD and took one of srpt's PMOs. shes hopeful for gene therapy. calls out the lack of FAERS system updates from srpt. management must improve. https://www.statnews.com/2025/06/18/sarepta-duchenne-muscular-dystrophy-elevidys-death-fda-advocacy/ https://www.statnews.com/2025/06/18/sarepta-duchenne-muscular-dystrophy-elevidys-death-fda-advocacy/"
[X Link](https://x.com/surferbackpack/status/1935599750214201509) 2025-06-19T07:25Z [--] followers, [---] engagements
"$SRPT Forward Timeline on Elevidys [--]. Enhanced Safety Protocol Submission 2a. FDA Feedback 2b. Status of ENVISION confirmatory trial 2c. Status of commercial halt on non-ambulatory Wildcards: - another serious adverse event (SAE) - international regulatory decision (EMA)"
[X Link](https://x.com/anyuser/status/1935603577491497161) 2025-06-19T07:40Z [---] followers, [---] engagements
"$SRPT $CAPR Reading on this. Deramiocel vs. Eteplirsen Cell therapy vs. Exon-Skipping therapy Eteplirsen for the 13% of patients with exon-51 mutation showed sig. slow rate of LVEF decline. Deramiocel for mainly older patients Stat sig. improvement in LVEF"
[X Link](https://x.com/surferbackpack/status/1935761624423711053) 2025-06-19T18:08Z [--] followers, [---] engagements
"$SPRT $CAPR In DMD heres my cause of death table: Cardiopulmonary Failure (total) 80% - Cardiac Failure (40-55%): cardiomyopathy(20-40%) sudden cardiac/arrhythmia (10-15%) - Respiratory Failure (25-40%): chronic (14%) acute (24%) Other causes (total) 20%"
[X Link](https://x.com/anyuser/status/1935763591422189759) 2025-06-19T18:16Z [---] followers, [---] engagements
"$SRPT $CAPR with NI-ventilation the share of cardiomyopathy/arrhythmia goes up up up. Cardiomyopathy onset [--] years 50% of DMD show evidence at [--] years. LVEF LGE NT-proBNP CK-MB are the critical targets for DMD intervention. assume Deramiocel for whole DMD pop"
[X Link](https://x.com/surferbackpack/status/1935765249535492220) 2025-06-19T18:22Z [--] followers, [---] engagements
"$SRPT $CAPR Eteplirsen sig. slows LVEF decline (-0.66%/yr Tx vs. -1.38%/yr control) but its main function is dystrophin-restoration and for exon-51 mutation patients. Deramiocel improves LVEF (+1.2% overall +3.0% subgroup) ELEVIDYS failed cardiac MRI measures"
[X Link](https://x.com/surferbackpack/status/1935767133037936711) 2025-06-19T18:30Z [--] followers, [---] engagements
"$SRPT $CAPR Deramiocel sidesteps dystophen-restoration focuses on biggest clinical problem: heart failure. compelling value proposition. if Deramiocel works its added to baseline therapy (ELEVIDYS or Agamree this is the question)"
[X Link](https://x.com/anyuser/status/1935767750393385208) 2025-06-19T18:32Z [---] followers, [----] engagements
"$SRPT My main thought: who has the most acceptable balance of benefit and risk for baseline treatment of DMD - is it ELEVIDYS or Agamree ELEVIDYS - you believe in AAV gene therapy Agamree - you believe in safety profile of new corticosteroid $CPRX the competitor"
[X Link](https://x.com/anyuser/status/1935770547725779298) 2025-06-19T18:43Z [---] followers, [---] engagements
"$SRPT vs. $CPRX [--]. ELEVIDYS wins the "wow factor" for foundational repair. [--]. Agamree wins safety profile clinicians know what to expect with corticosteroid ELEVIDYS acute liver fail scary"
[X Link](https://x.com/surferbackpack/status/1935774584546246885) 2025-06-19T19:00Z [--] followers, [---] engagements
"$SRPT [--]. Trust of the DMD community. The heart wants promise of ELEVIDYS but the head leaning towards Agamree [--]. Practicality: ELEVIDYS is one time major medical event. Agamree is daily oral. srpt becomes the high risk-high reward gamble if safety profile doesnt improve"
[X Link](https://x.com/anyuser/status/1935775322949271811) 2025-06-19T19:02Z [---] followers, [---] engagements
"$SRPT In the end I think SRPT is still the most intriguing company in the space. Agamree is still a steroid with lots of competitors. ELEVIDYS is $3 mill a customer. If Deramiocel works lets say priced at $1mill a customer annually $CAPR is intriguing"
[X Link](https://x.com/anyuser/status/1935810049395638436) 2025-06-19T21:20Z [---] followers, [----] engagements
"$SRPT Tomorrows agenda for me is to look into probability of sirolimus getting the pass from FDA for ELEVIDYS. Also will look deeper into Zolgensma. Also comparing study pop. sizes between $CAPR and $SRPT"
[X Link](https://x.com/anyuser/status/1935934313289363932) 2025-06-20T05:34Z [---] followers, [---] engagements
"$CAPR eteplirsen by $SRPT in [----] had [--] patients and they got accelerated approval now deramiocel seeking full BLA but there is precedent. Unmet patient need is on deramiocel's side. ADCOMM July [--] PDUFA deadline Aug 31"
[X Link](https://x.com/anyuser/status/1935937596640878796) 2025-06-20T05:47Z [---] followers, [---] engagements
"$SRPT interesting week in the DMD space and I am glad I started researching this. Verdun and her deputy gets ousted (need more details) its Vinay's show now running biologics. Vinay supposedly still pro gene therapy for rare disease. Post-marketing safety checks is the discount"
[X Link](https://x.com/anyuser/status/1936118333260382416) 2025-06-20T17:45Z [---] followers, [---] engagements
"@westsideguy3 it seems unclear right now. story is that Verdun had it on the table and was discussing with Prasad to do the ADCOMM as scheduled but now she's out it and reports are that Prasad cancelled. Capricore said they are still planning as if its going to happen but unclear. Caution"
[X Link](https://x.com/surferbackpack/status/1936149628065566804) 2025-06-20T19:50Z [--] followers, [--] engagements
"going on vacation will come back with full brkdwn $SRPT just a few bullets if you explore: rituximab + sirolimus UofF health CMV infection in 1st elevidys death sirolimus good but creates complex risk paradigm Roctavian AAV5: label precedent Hemgenix/Zolgensma: take a look"
[X Link](https://x.com/anyuser/status/1936483279705936243) 2025-06-21T17:56Z [---] followers, [---] engagements
"@jeremyj0916 Also Im going to look into the management team more. I just see a lot of potential in SRPT its a good pipeline. New management and Wall Street regains trust in srpt Also want to keep track of the next quarter holdings report. See what funds dumped with the patient death scare"
[X Link](https://x.com/surferbackpack/status/1938813256950522059) 2025-06-28T04:14Z [--] followers, [---] engagements
"$SRPT LGMD2E/R4 is very rare. [----] Bayesian Analysis: [---] per million people. But complete lack of approved modifying treatment. EMERGENE trial: ambulatory + non-amb Jun 2025-FDA grants Pltform tech desig. to AAVrh74 vector. expedite reviews of AAVrh74 products"
[X Link](https://x.com/anyuser/status/1939599326222950786) 2025-06-30T08:18Z [---] followers, [----] engagements
"$SRPT SRP-9003 program: phase1/2 enroll [--] patients. efficacy was good. safety was avg. transient elev. in g-glutamyl-trnsfase. one serious adverse evnt: acute liver injury needed hospitaliz resolved with standard care. phase [--] enrolled 15-17 patients"
[X Link](https://x.com/anyuser/status/1939600926370840764) 2025-06-30T08:24Z [---] followers, [---] engagements
"$SRPT for bull: AAVrh74 SRP9003 EMERGENCE data tmrw shows good safety no acute liver toxicity. Market already hated this with ELEVIDYS. if good BLA goes through SRPT and FDA at least move forward on something for now"
[X Link](https://x.com/anyuser/status/1939604250562314298) 2025-06-30T08:37Z [---] followers, [----] engagements
"@AlohaOasis @Hall8Jack Yes theyd get sued to oblivion otherwise. Company says they release data and doesnt its self sabotage"
[X Link](https://x.com/surferbackpack/status/1939713036828516400) 2025-06-30T15:50Z [--] followers, [---] engagements
"@Hall8Jack Interesting. Yeah I saw that conference schedule. I just stick to what Sarepta says. Said 1st half of [--] which is quarter end today. I dont necessarily trust anything any biotech says before data release. They have fiduciary duty. I appreciate your comments"
[X Link](https://x.com/surferbackpack/status/1939713613436252457) 2025-06-30T15:52Z [--] followers, [---] engagements
"$SRPT no LGMD data today understandable given everything management is going through. today's pr on inducement grant was not much. standard equity incentivization for new employees. good they still hiring. good employees incentivized to help company. nothing special"
[X Link](https://x.com/surferbackpack/status/1939822005924372564) 2025-06-30T23:03Z [--] followers, [---] engagements
"$srpt nothing changed as of today. dont look at the +7% as legit. all pharma/biotech have the same daily charts. correlation not sector rotation"
[X Link](https://x.com/surferbackpack/status/1940091494612050340) 2025-07-01T16:53Z [--] followers, [---] engagements
"$SRPT LGMD phase1/2 read refresher: https://pmc.ncbi.nlm.nih.gov/articles/PMC10803256/pdf/41591_2023_Article_2730.pdf https://pmc.ncbi.nlm.nih.gov/articles/PMC10803256/pdf/41591_2023_Article_2730.pdf"
[X Link](https://x.com/surferbackpack/status/1941754237517766969) 2025-07-06T07:01Z [--] followers, [---] engagements
"$CAPR well I was busy all week but proud to see that my CAPR short worked. Pretty funny looking at all retail so shocked at the rejection. Who in their right mind was long CAPR if you did you need a serious risk assessment lesson. It was clearly you stay cash or go short"
[X Link](https://x.com/surferbackpack/status/1944136905169522763) 2025-07-12T20:48Z [---] followers, [---] engagements
"@jeremyj0916 Any bios on your radar Been looking at Alnylam Biomarin Arrowhead besides dog pound Sarepta"
[X Link](https://x.com/surferbackpack/status/1946105415730954406) 2025-07-18T07:11Z [---] followers, [---] engagements
"@GreatSehaj nice. remember risk management position sizing"
[X Link](https://x.com/anyuser/status/1946274444013621512) 2025-07-18T18:22Z [---] followers, [--] engagements
"Well this manifested back from [--] weeks ago. FDA press release after market close today $SRPT major risk for srpt is this: AAV-mediated hepatotoxicity is not specific to ELEVIDYS. if safety data bad with SRP-9003 its the AAVrh74 platform whole pipeline is down bad. mstanley: project +1.2bill rev/yr from LGMD by [----]. $SRPT major risk for srpt is this: AAV-mediated hepatotoxicity is not specific to ELEVIDYS. if safety data bad with SRP-9003 its the AAVrh74 platform whole pipeline is down bad. mstanley: project +1.2bill rev/yr from LGMD by 2030"
[X Link](https://x.com/anyuser/status/1946275191790960656) 2025-07-18T18:25Z [---] followers, [---] engagements
"This is the craziest timeline of events: $SRPT We wanted to know how much balls Vinay Prasad had how much balls Sarepta had we got a full on fight. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-statement-elevidys https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-statement-elevidys"
[X Link](https://x.com/anyuser/status/1946359415692820957) 2025-07-19T00:00Z [---] followers, [----] engagements
"$SRPT Zolgensma: liver toxicity - fda addresses using black box label and enhanced monitoring protocol. july 16: fda good faith gives black box label. srpt convenes for non-ambulant risk assessment. july 18: fda pulls plat desig requests halt"
[X Link](https://x.com/anyuser/status/1946362888433877057) 2025-07-19T00:14Z [---] followers, [----] engagements
"$SRPT fda is going to make another press release fighting srpt watch. If this thing escalates srpt goes arbitrary and capricious lawsuit"
[X Link](https://x.com/surferbackpack/status/1946363836493394253) 2025-07-19T00:17Z [--] followers, [---] engagements
"$srpt elevidys competitors: AAV: Regenxbio: phase 1/2 AFFINITY DUCHENNE trial. BLA mid '26. Solid Biosci:sgt-003 phase1/2. Antisense oligonucleotides: - sort of the challenge to srpt PMOs Dyne-251 phase1/2 other: capr deramiocel (lol) topline q3 [--] remmber CRL lol"
[X Link](https://x.com/anyuser/status/1946476296294568283) 2025-07-19T07:44Z [---] followers, [---] engagements
"$SRPT stratification has to matter to the fda. non-amb: generally.pre compromised liver (underlying hepatic steatosis) compromised cardiac output impairs systemic clearance of aav vector lack a physiological reserve. sarepta's refusal is smart"
[X Link](https://x.com/anyuser/status/1946480119075488036) 2025-07-19T07:59Z [---] followers, [---] engagements
"$srpt hey srpt itd be really nice if you could identify the risk factor causing the ALF. tysabri for ms got pulled then reintroduced under strict risk management program. i just dont think its game over for ELEVIDYS"
[X Link](https://x.com/surferbackpack/status/1946484582238601266) 2025-07-19T08:17Z [---] followers, [---] engagements
"$srpt TYSABRI (biogen/elan): feb [--] 2005- withdrawn from market - BIIB [--] to [--] ELN [--] to [--] march [--] [----] - fda advis. comm. recommends return to market. - BIIB 46to [--]. ELN [--] to [--]. srpt situation is a hybrid of BIIB (deep piplin) and ELN (tys was tied to future)"
[X Link](https://x.com/anyuser/status/1946489682369696238) 2025-07-19T08:37Z [---] followers, [---] engagements
"$SRPT they really did it. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-voluntary-pause-elevidys https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-voluntary-pause-elevidys"
[X Link](https://x.com/anyuser/status/1947445504692142144) 2025-07-21T23:56Z [---] followers, [----] engagements
"$SRPT So. the entire FDA Newsroom should just be shutdown because the officials prefer playing telephone with journalists directly instead. Updated: In a follow-up conversation on the safety study the senior official said Sarepta could make a change to the dosing or manufacturing and test it on the next [--] or [--] boys and show the liver-related issues were improved Updated: In a follow-up conversation on the safety study the senior official said Sarepta could make a change to the dosing or manufacturing and test it on the next [--] or [--] boys and show the liver-related issues were improved"
[X Link](https://x.com/anyuser/status/1948465770666885488) 2025-07-24T19:30Z [---] followers, [----] engagements
"Times like this where you think: Twitter is the Bloomberg terminal news feed for retailers. Ahh democratization of education via tech. Good times to live in"
[X Link](https://x.com/anyuser/status/1948467082661953636) 2025-07-24T19:35Z [---] followers, [---] engagements
"$SRPT okay thats one step done. https://www.fda.gov/news-events/press-announcements/fda-recommends-removal-voluntary-hold-elevidys-ambulatory-patients https://www.fda.gov/news-events/press-announcements/fda-recommends-removal-voluntary-hold-elevidys-ambulatory-patients"
[X Link](https://x.com/anyuser/status/1949936056281026636) 2025-07-28T20:52Z [---] followers, [---] engagements
"waiting to see an opportunity for puts on $ATYR and $SLNO and $QBTS and maybe $CAPR not doing anything now. will wait. if nothing im cash"
[X Link](https://x.com/anyuser/status/1956989627283480596) 2025-08-17T08:01Z [---] followers, [---] engagements
"adding more $SRPT this week. Will do another deep dive"
[X Link](https://x.com/surferbackpack/status/1967480816503865517) 2025-09-15T06:49Z [---] followers, [---] engagements
"$GALT look at community boards for GALT. foolish "army" of believers with vague hopes of buyout and approval. Little skepticism delusional optimism. Classic signs of a fraudulent biotech"
[X Link](https://x.com/surferbackpack/status/1969127260272640094) 2025-09-19T19:51Z [---] followers, [--] engagements
"$GALT cash value is about 15-30 cents. This should trade below $1 by the time of EOP2 meeting results next year. I believe FDA will ask for another confirmatory trial. GALT cannot keep p-hacking"
[X Link](https://x.com/surferbackpack/status/1969171288221458484) 2025-09-19T22:46Z [---] followers, [---] engagements
"$SRPT https://fis.fda.gov/sense/app/95239e26-e0be-42d9-a960-9a5f7f1c25ee/sheet/6b5a135f-f451-45be-893d-20aaee34e28e/state/analysis https://fis.fda.gov/sense/app/95239e26-e0be-42d9-a960-9a5f7f1c25ee/sheet/6b5a135f-f451-45be-893d-20aaee34e28e/state/analysis"
[X Link](https://x.com/surferbackpack/status/1971841366365012263) 2025-09-27T07:36Z [---] followers, [---] engagements
"$SRPT up 75% on my position. Reducing size a tad today. Still a substantial part of my portfolio"
[X Link](https://x.com/surferbackpack/status/1973778081639969263) 2025-10-02T15:52Z [---] followers, [---] engagements
"$SRPT Abstract 732LBP: Preliminary analysis of safety tolerability and efficacy of a prophylactic sirolimus protocol for patients receiving ELEVIDYS Vienna WMS Conference Oct 7-11 Probably a press release soon from sarepta"
[X Link](https://x.com/surferbackpack/status/1973861636089000345) 2025-10-02T21:24Z [---] followers, [----] engagements
"$SRPT abstract also for SRP-9003 (LGMD) 734LBP Expression of SGCB and safety following bidridistrogene xeboparvovec treatment in patients with LGMD2E/R4: results from the EMERGENE phase [--] study https://www.wms2025.com/file/90a8e78b-872e-4de1-b1a0-0a78ac8c810c/WMS2025%20Congress%20Full%20Programme%20and%20Appendix%20(13)%20.pdf https://www.wms2025.com/file/90a8e78b-872e-4de1-b1a0-0a78ac8c810c/WMS2025%20Congress%20Full%20Programme%20and%20Appendix%20(13)%20.pdf"
[X Link](https://x.com/surferbackpack/status/1973874217088389209) 2025-10-02T22:14Z [---] followers, [---] engagements
"$SRPT another paper on Sirolimus see chart at bottom of paper. https://www.sciencedirect.com/science/article/pii/S0887899425002620 https://www.sciencedirect.com/science/article/pii/S0887899425002620"
[X Link](https://x.com/surferbackpack/status/1973876582000501145) 2025-10-02T22:23Z [---] followers, [----] engagements
"@jeremyj0916 looking at this $OVID mention wowza"
[X Link](https://x.com/surferbackpack/status/1973889050500325517) 2025-10-02T23:13Z [---] followers, [---] engagements
"$SRPT Financial experiment: elevidys = $375m in Q1 PMO = $237m in Q1. Stress Test: Elevidys = $1.5 bill annual x (1-80%)=$300m. PMO=$948m annual x (1-30%) = $664m. Stress Test Revenue = $964m. Lets say trades at 2x p/s multiple. $1.9b/98mill shares = $19.67"
[X Link](https://x.com/anyuser/status/1935510735960031393) 2025-06-19T01:31Z [---] followers, [---] engagements
"@jeremyj0916 Yeah Im going to look at their financials tomorrow to get a gauge of some things (CFFI is one I want to see over quarters). Think just the science side is probably enough to play this one if it makes sense but Ill be active on here to discuss"
[X Link](https://x.com/anyuser/status/1941367057158897688) 2025-07-05T05:22Z [---] followers, [--] engagements
"$SRPT still holding this puppy. Avg. of $14"
[X Link](https://x.com/anyuser/status/1955746500543438854) 2025-08-13T21:41Z [---] followers, [---] engagements
"$GALT No stat sig. on primary endpoint in ITT pop. Stat sig. finding in Per-Protocol pop (but attrition bias) Post-hoc emphasis on U.S. subgroup (data dredging) Inverted dose-response curve (saying it's target-mediated drug disposition convenient post-hoc rationalization)"
[X Link](https://x.com/surferbackpack/status/1969117143682707650) 2025-09-19T19:11Z [---] followers, [---] engagements
"$GALT belapectin (2mg/kg) more effective than (4mg/kg) unpredictable mechanism off-target effects at higher dose GALT says saturation of galectin-3 binding site at 2mg/kg. Usually saturation leads to plateau of efficacy not reduction"
[X Link](https://x.com/surferbackpack/status/1969118222013112630) 2025-09-19T19:15Z [---] followers, [---] engagements
"$GALT Phase 2b GT-026 study: No stat sig. with 2mg/kg 8mg/kg on HVPG (portal pressure) However stat-sig with pre-specified subgroup with no esophageal varices at baseline 1/2 of study pop. 2mg/kg reduces varices (p0.02). Chased the p-value centered NAVIGATE around this"
[X Link](https://x.com/surferbackpack/status/1969119306068771318) 2025-09-19T19:20Z [---] followers, [--] engagements
"$GALT In NAVIGATE trial design a patient in the placebo who takes GLP-1 agonist for diabetes and whose liver health improves is counted as a treatment failure. Confounding true effect of belapectin with established effects of standard care medication"
[X Link](https://x.com/surferbackpack/status/1969120657901244520) 2025-09-19T19:25Z [---] followers, [---] engagements
"$GALT NAVIGATE primary endpoint in ITT population: 2mg vs. placebo p=0.552 4mg vs. placebo p=0.139 Per-Protocol Pivot (excludes patients w/ incomplete 18month treatment or no end of treatment endoscopy: Reduces patient pool by 20%"
[X Link](https://x.com/surferbackpack/status/1969121422543872026) 2025-09-19T19:28Z [---] followers, [---] engagements
"$GALT In the PP pop. GALT only focuses on one component of original composite endpoint: Incidence of new varices. Finds stat sig. for 2mg/kg dose. PP analyses are considered supportive at best in the data package sent to FDA. Breaks randomization and introduces attrition bias"
[X Link](https://x.com/surferbackpack/status/1969121773070229911) 2025-09-19T19:29Z [---] followers, [---] engagements
"$GALT They then present a post-hoc subgroup analysis of PP patients enrolled only in U.S. 2mg/kg then shows 68% relative reduction in new varices vs. placebo (p0.02). GALT then says this effect may be due to higher rate of GLP-1 in U.S. potential synergistic benefit"
[X Link](https://x.com/surferbackpack/status/1969122483513999591) 2025-09-19T19:32Z [---] followers, [---] engagements
"$GALT To sum things up: GALT is a company of statistical maneuvering to find a positive signal in a dataset from a failed trial. If you analyze enough subgroups you will find a stat sig. result likely due to chance alone"
[X Link](https://x.com/surferbackpack/status/1969122965779366302) 2025-09-19T19:34Z [---] followers, [---] engagements
"$GALT Would be required to conduct a whole new trial for that specific varices reduction hypothesis only in U.S. pop to validate results of NAVIGATE. Clear positive of GALT is that it has a clean safety and tolerability profile. Necessary but insufficient condition for approval"
[X Link](https://x.com/surferbackpack/status/1969123529653141745) 2025-09-19T19:36Z [---] followers, [---] engagements
"$GALT Madrigal Pharmaceutical: Rezdiffra - MAESTRO NASH OUTCOMES trial if successful would expand label into same patient pop. Galectin is targeting. Akero Therapeutics: Efruxifermin - Good phase2b in Phase [--]. Outcompeting GALT like hell"
[X Link](https://x.com/surferbackpack/status/1969124339061493916) 2025-09-19T19:40Z [---] followers, [---] engagements
"$GALT Key dates: November data readout at AASLD. 36-month cohort study of NAVIGATE. Truth of long-term efficacy. First half of 2026- End of Phase [--] meeting with FDA (is data sufficient for BLA or new Phase [--] required)"
[X Link](https://x.com/surferbackpack/status/1969128492550795521) 2025-09-19T19:56Z [---] followers, [---] engagements
"$GALT They are going to be forced to do another trial. EMA Assessment Scenario 3: For a subgroup claim from a failed trial to be considered credible: [--]. Subgroup must have been defined before study began [--]. Strong biological plausibility of difference in subgroups. [--] onwards"
[X Link](https://x.com/surferbackpack/status/1969185118183932232) 2025-09-19T23:41Z [---] followers, [---] engagements
"$GALT Company rests on the 2mg dose in a specific group: MASH cirrhosis patients with clinically sig. portal hypertension and without baseline varices. I wonder what the total addressable market is for this enough for a phase 3"
[X Link](https://x.com/surferbackpack/status/1970996202989838544) 2025-09-24T23:38Z [---] followers, [---] engagements
"$AKRO $GALT $MDGL F-Stages of Fibrosis. Rezdiffra captured F2 and F3 (non-cirrhotic) pop. last year. OUTCOMES trial seeks to expand label into F4. Belapectin is targeting the F4 population (cirrhotic). Race to first in-class F4. But Efruxifermin hmm"
[X Link](https://x.com/surferbackpack/status/1970999743540232362) 2025-09-24T23:52Z [---] followers, [---] engagements
"$AKRO $GALT Efruxifermin- phase 2b f4 pop stat sig. reversal of cirrhosis at [--] weeks (39% for 50mg 15% for placebo) Belapectin-is varices prevention endpoint a clinically superior outcome to potential reversal of cirrhosis I like Efruxifermin a lot more than belapectin"
[X Link](https://x.com/surferbackpack/status/1971003812254187686) 2025-09-25T00:08Z [---] followers, [---] engagements
"$AKRO $GALT $MDGL roche MASH F4 Space: [--]. Efruxifermin-my leader in F4 histological data -cirrhosis reverse [--]. Pegozafermin competitor -cirrhosis reverse 1b2b. Rezdiffra- phase [--] OUTCOMES for F4 - liver stiff. reduction last. Belapectin-mixed signal data. -varices reduction"
[X Link](https://x.com/surferbackpack/status/1971005361281958170) 2025-09-25T00:14Z [---] followers, [---] engagements
"$GALT assuming [--] month data presented AASLD Nov 7-11. See Oct. [--] if abstract is uploaded or Nov. [--] for later abstracts on AASLD (GALT likely press releases anyway). September presentation said FDA feedback on NAVIGATE results by EOY. Planning the trade now"
[X Link](https://x.com/surferbackpack/status/1973843161354293743) 2025-10-02T20:10Z [---] followers, [---] engagements
"@jeremyj0916 you ever look at $ANAB"
[X Link](https://x.com/surferbackpack/status/1973914753480470942) 2025-10-03T00:55Z [---] followers, [---] engagements
"research list. $LRMR like-ish $CAPR short $GALT short $SRPT long $AKRO like-ish"
[X Link](https://x.com/surferbackpack/status/1974187507735802234) 2025-10-03T18:59Z [---] followers, [----] engagements
"$NRXP nrx-101 pdufa data: phase [--] (2023 end): phase2b/3 (2024 end): - still looking for the actual poster (W89: A Randomized Double-Blind Controlled Comparison of NRX-101 to Lurasidone.) https://www.biospace.com/nrx-pharmaceuticals-nasdaq-nrxp-presents-landmark-trial-of-nrx-101-in-suicidal-bipolar-depression-at-the-ascp-annual-meeting-nrx-101-is-the-first-oral-antidepressant-demonstrated-to-reduce-suicidality-in-bipolar https://pubmed.ncbi.nlm.nih.gov/37573534/"
[X Link](https://x.com/surferbackpack/status/1974717161542410455) 2025-10-05T06:03Z [---] followers, [---] engagements
"$AVXL EMA decision eoy $GALT Data at AASLD Nov. 7-11 $CAPR HOPE-3 data eoy $NRXP PDUFA decision eoy"
[X Link](https://x.com/surferbackpack/status/1974730384673034733) 2025-10-05T06:56Z [---] followers, [---] engagements
"i never use 2x levered daily etfs but $SRPU seems interesting. might add some before earning day bell close to lever up then sell next day market open vol drag shouldn't be anything for a overnight trade $SRPT"
[X Link](https://x.com/surferbackpack/status/1981605450538955157) 2025-10-24T06:15Z [---] followers, [---] engagements
"nov. update longs: $SRPT $SPRB $PALI shorts: $AVXL $QBTS research: $BHVN $AVTX $INSM $LRMR $SLNO"
[X Link](https://x.com/surferbackpack/status/1985255539824300102) 2025-11-03T07:59Z [---] followers, [---] engagements
"$SRPT on the earnings call rebought @ [--]. Back to this Q1"
[X Link](https://x.com/surferbackpack/status/1985475377838637235) 2025-11-03T22:33Z [---] followers, [---] engagements
"$SRPT anyone have information on the breakdown of PMO revenue - AMONDYS [--] - VYONDYS [--] - EXONDYS [--] Would think majority of PMO revenue comes from EXONDYS [--] - largest single-skip amenable population"
[X Link](https://x.com/surferbackpack/status/1985571267475591663) 2025-11-04T04:54Z [---] followers, [---] engagements
"$SRPT risk is on. -Consolidated Appropriations Act ('23) '25 FDA Guidance grant speedy withdrawal of accel. appr. products. -'23 Makena by Covis was withdrawn by CDER (indic: pre-term birth adcom voted 14-1 to withdraw) EXONDYS [--] imo is the last hope for PMO"
[X Link](https://x.com/surferbackpack/status/1985572108282585188) 2025-11-04T04:57Z [---] followers, [---] engagements
"$SRPT EXONDYS [--] accel. app. was pushed by Dr. Woodcock CDER Director. -argued surrogate endpoint (microdystrophin release) was sufficient partly political (DMD lobbying/advocacy) -SRPT using real world evidence (RWE) as counter. -RWE has no precedent overturning Random PCT"
[X Link](https://x.com/surferbackpack/status/1985572945918607412) 2025-11-04T05:00Z [---] followers, [---] engagements
"$SRPT if this fails (EXONDYS 51) no doubt all PMOs get pulled from the market: https://clinicaltrials.gov/study/NCT03992430 https://clinicaltrials.gov/study/NCT03992430"
[X Link](https://x.com/surferbackpack/status/1985576301336695292) 2025-11-04T05:14Z [---] followers, [---] engagements
"$SRPT Sum of parts valuation (no PMO): ELEVIDYS only amb pop. - 500m annual EV (2.5p/s)=1.2b Debt: 860m cash - 1.1b debt : (-230)m cash siRNA pipe (ARWR): srpt committed 930m lets put this at 500m 1.25b+500m-235m=1.5b mc ($15/s) this is why i bought today. hope it works"
[X Link](https://x.com/surferbackpack/status/1985591858781962616) 2025-11-04T06:16Z [---] followers, [---] engagements
"$SRPT other risks: RGNX lawsuit - over AAV platform of ELEVIDYS jury starts 11/17/25 - RGNX gets royalties if SPRT loss Solvency- dilution with cash run out (ARWR payments before data readouts for siRNA its dicey"
[X Link](https://x.com/surferbackpack/status/1985593167543222603) 2025-11-04T06:21Z [---] followers, [---] engagements
"$BHVN $8.12 right now buying a little size. Honestly have not really deep dived into this but will do so"
[X Link](https://x.com/surferbackpack/status/1986164893289263275) 2025-11-05T20:13Z [---] followers, [---] engagements
"$SPRB Ikarian 13G today Nov. [--] (date of event: Oct. 31) and Squadron Capital's in this. 132m MC gonna be a fun one"
[X Link](https://x.com/surferbackpack/status/1987040246752813556) 2025-11-08T06:11Z [---] followers, [---] engagements
"ADCs to research: B7-H4: $NXTC and $MRSN HNSCC (Nectin-4 experimental EDB+FN): $CRBP and $PYXS HNSCC fits the rare-disease theme better I like"
[X Link](https://x.com/surferbackpack/status/1987805505855934799) 2025-11-10T08:52Z [---] followers, [---] engagements
"$CAPR Sell Side Analyst: "Break a leg" CEO Linda: "Thanks we'll need it" q3 call is too funny"
[X Link](https://x.com/surferbackpack/status/1988002569940357557) 2025-11-10T21:55Z [---] followers, [---] engagements
"godel watchlist 11/10/25 - $CAPR dec. puts - $GALT looking good to short soon - got out the train too early on $AVXL jan p - $SPRB moose doing its thing - quantum short finally working out - $BHVN and $SRPT both scare me but long for now - learning about ADCs (crbp nxtc pyxs)"
[X Link](https://x.com/surferbackpack/status/1988110037437608376) 2025-11-11T05:02Z [---] followers, [----] engagements
"$LRMR i like this one tricky part besides anyphalaxis issue: precedent in FA: Omaveloxolone -functional clinical endpoint (mFARS) with hist. controls. Nomlabofusp -pharmacodynamic marker (skin FXN conc.) as RLSE correlation to clinical outcomes derived from hist. controls"
[X Link](https://x.com/surferbackpack/status/1988514505156878447) 2025-11-12T07:49Z [---] followers, [---] engagements
"$LRMR better have some good data: boils down to consistency of the correlation between FXN increase and mFARS stabilization given the non-randomized historical control design (FACOMS)"
[X Link](https://x.com/surferbackpack/status/1988516928156021143) 2025-11-12T07:59Z [---] followers, [---] engagements
"$CAPR wrote down my thoughts on the HOPE-3 data here. took both sides of the argument (PUL fragility vs. LVEF efficacy) https://docs.google.com/document/d/1qjZ5l1XJ9DhSmZ4_b9sMvQPkAXmEpiO6AiOuofnvcZY/editusp=sharing https://docs.google.com/document/d/1qjZ5l1XJ9DhSmZ4_b9sMvQPkAXmEpiO6AiOuofnvcZY/editusp=sharing"
[X Link](https://x.com/anyuser/status/1996722302785146948) 2025-12-04T23:24Z [---] followers, 28.9K engagements
"@alan_sharp @yachmod $SRPT vs $SRPU's [--] vs [--] price delta is an arbitrary psychological anchor. the product of SRPU is a synthetic 200% daily return contract (capital structure reset nightly). use it don't own it. binary event - capture levered overnight move without the iv premium of a call"
[X Link](https://x.com/surferbackpack/status/1981609613968470017) 2025-10-24T06:32Z [---] followers, [---] engagements
"January watchlist for me: $SPRB $LRMR $KTTA $SRPT writing a report on $KTTA and $LRMR $LPCN past month was interesting to see"
[X Link](https://x.com/surferbackpack/status/2009159410325831805) 2026-01-08T07:05Z [---] followers, [---] engagements
"@BrainyMarsupial Interesting. Thanks for letting me know about that april presentation. My major concern was if they were starting manufacturing from scratch (like no TA-ERT given from Allievex since they ran out of supply). Obv there is still risk with producing rhNAGLU since its quite complex"
[X Link](https://x.com/surferbackpack/status/2019612557871566849) 2026-02-06T03:22Z [---] followers, [--] engagements
"@BrainyMarsupial You mind sharing your thoughts on the markets pricing on SPRB right now. Near PIPE price seems brutal. I want to attribute the price to beta PRV drama and biotech being a bit of a shitter the past couple months. I mean our calculations make the current price a steal but hmm"
[X Link](https://x.com/surferbackpack/status/2019613130092081348) 2026-02-06T03:24Z [---] followers, [--] engagements
"Have a Other bios im looking: business models i like (not bullish yet) Vertex Pharmaceuticals ($VRTX) Alnylam Pharmaceuticals ($ALNY) BioMarin Pharmaceutical ($BMRN) Krystal Biotech ($KRYS) Rocket Pharmaceuticals ($RCKT) Denali Therapeutics ($DNLI) https://www.barrons.com/articles/sarepta-stock-gene-therapy-treatment-148476abreflink=desktopwebshare_permalink https://www.barrons.com/articles/sarepta-stock-gene-therapy-treatment-148476abreflink=desktopwebshare_permalink"
[X Link](https://x.com/anyuser/status/1941735880613609816) 2025-07-06T05:48Z [---] followers, [---] engagements
"if interested in $SRPT you need to be looking at Alnylam Pharmaceuticals Avidity Biosciences Dyne Therapeutics. siRNA. delivery to liver (LNP is good). delivery to skeletal muscle (AOCs look like gold standard target TfR1 for entry)"
[X Link](https://x.com/surferbackpack/status/1951895466670789055) 2025-08-03T06:38Z [---] followers, [---] engagements
"@yanir_uliel The recent management hires boil down to bringing back individuals from BioMarin who worked during the enzyme replacement therapy time at BioMarin. Spruce management should file BLA this Q1 (assuming no delays)"
[X Link](https://x.com/surferbackpack/status/2021138229404024835) 2026-02-10T08:24Z [---] followers, [---] engagements
"$ALDX For the June [----] resubmission (PDUFA March 16): Proof of primary efficacy lays in study [---] (chamber trial) But full CRS of the Field Trial (failed) must be included in NDA package per standard protocol as argued by FDA office of specialty med"
[X Link](https://x.com/surferbackpack/status/2021453215624306774) 2026-02-11T05:16Z [---] followers, [---] engagements
"@StockChumpski $rgnx crl fda cited issues with use of cns-hs to predict clinical benefit. i assume rgnx's clinical function data was weak/used a [--] year time period. sprb does have [--] year data and uses a different type of cns-hs. Its still a massive signal that the FDA doubted cns-hs"
[X Link](https://x.com/surferbackpack/status/2020977396233667045) 2026-02-09T21:45Z [---] followers, [---] engagements
"@BrainyMarsupial acquisition would be sweet of course im putting probability low given the confirmatory trial. my est. of raise needed for that trial is about 100million. EV rn without PRV is [--] million with PRV that goes to [---] mill. market rn pricing an equity raise likely after bla filed"
[X Link](https://x.com/surferbackpack/status/2019523170924130513) 2026-02-05T21:26Z [---] followers, [---] engagements
"@BrainyMarsupial i have net asset value with PRV (@ $110M) BLA approval and a $75 million raise (1.25M shares at $60) at $89.3 per share. I have NPV at $731M then calculating FV I get $430 per share. So like 350-450 FV"
[X Link](https://x.com/surferbackpack/status/2019567363692363857) 2026-02-06T00:22Z [---] followers, [---] engagements
"@BrainyMarsupial if i was a fund as well. i would wait for the raise to happen so they can size without slippage . spruce could also mess up the raise if they get desperate and do a 100m raise at $40 with warrants. i think spruce's sp has been more affected by beta over the past months"
[X Link](https://x.com/surferbackpack/status/2019569106559856803) 2026-02-06T00:29Z [---] followers, [--] engagements
"$RGNX concerning news. RGNX used CNS-HS D2S6 as surrogate endpoint. RGNX used [--] month data. SPRB is using 5-6 year data. RGNX endpoint timing: [--] week endpoint SPRB endpoint: durable reduction RGNX follow-up [--] years SPRB follow-up [--] years"
[X Link](https://x.com/surferbackpack/status/2020974474288619710) 2026-02-09T21:33Z [---] followers, [----] engagements
"@LQDA_Bull1 Dont think its too irrational. I understand selling off because of the similarities between RGNX and SPRB (nat. History comparison CNS-HS surr. endpoint exploratory endpoints for clinical benefit w/ no p-values)"
[X Link](https://x.com/surferbackpack/status/2021004421120078015) 2026-02-09T23:32Z [---] followers, [---] engagements
"@LQDA_Bull1 Now you can make the argument for Spruce that RGNXs HS-D2S6 is not appropriate since its a specific disaccharide in the middle of the chain and can be broken off by other enzymes. While HS-NRE is more specific as NAGLUs only job is to cook the terminal sugar at NRE"
[X Link](https://x.com/surferbackpack/status/2021005003222331763) 2026-02-09T23:35Z [---] followers, [---] engagements
"@LQDA_Bull1 Youd argue that NAGLU is missing so NRE tips cant be removed. This measuring NRE is a 1-to-1 measurement of the drug doing its job. With HS-D2S6 you can lower fragments without clearing the toxic tips that cause the neurodegeneration"
[X Link](https://x.com/surferbackpack/status/2021005368864977399) 2026-02-09T23:36Z [---] followers, [---] engagements
"@LQDA_Bull1 You can also say that RGNX focused on percentage reduction (80%+ drop in HS-D2S6) so that only shows its less toxic. While Spruce focused on normalization (HS-NRE) so they brought it down to healthy range (Spruce would argue it must predict clinical benefit)"
[X Link](https://x.com/surferbackpack/status/2021005885989171547) 2026-02-09T23:38Z [---] followers, [---] engagements
"$SPRB added. I get $70 when pricing in equity raise after BLA is announced. Appears to be a good price right now. I dont have doubts about the science. Small concerns about manufacturing. Major questions about how much they raise after BLA filiing"
[X Link](https://x.com/surferbackpack/status/2019516314352779765) 2026-02-05T20:59Z [---] followers, [----] engagements
"$SPRB TA-ERT as of my understanding holds the bar for cognitive func. stabilization for an MPS drug"
[X Link](https://x.com/surferbackpack/status/2019676069687959789) 2026-02-06T07:34Z [---] followers, [----] engagements
"$SPRB Superior on specificity compared to Denali's DNL-310. FDA's comments on DNL-310's use of Total CSF HS should be stock-moving dependent on what they say. CSF HS-NRE (nre=non-reducing end specific marker of NAGLU metabolic block)"
[X Link](https://x.com/surferbackpack/status/2019677184470970782) 2026-02-06T07:38Z [---] followers, [----] engagements
"$SPRB So Spruce focuses on grey matter rather than NfL (DNL-310 has great NfL data). Spruce argues stabilized brain volume is a more direct correlate of clinical outcome than noisy pediatric NfL. Interesting. In [----] Tofersen for ALS was approved by FDA using NfL reduction"
[X Link](https://x.com/surferbackpack/status/2019679539916599334) 2026-02-06T07:48Z [---] followers, [----] engagements
"$SPRB wrote down my thoughts on TA-ERT's outlook after today's CRL of RGX-121 $RGNX https://docs.google.com/document/d/1f-g25IKFoW9KKPcqtXA07KGr5r0uaUdTUHicJm1cH84/editusp=sharing https://docs.google.com/document/d/1f-g25IKFoW9KKPcqtXA07KGr5r0uaUdTUHicJm1cH84/editusp=sharing"
[X Link](https://x.com/surferbackpack/status/2021079601783636448) 2026-02-10T04:31Z [---] followers, [----] engagements
"$SPRB If you study Brineura by BioMarin and the hurdles they had to go through for approval you would know those same BioMarin folks made sure TA-ERT was going to be robust enough for it's turn with the FDA gauntlet"
[X Link](https://x.com/surferbackpack/status/2021105463979585631) 2026-02-10T06:14Z [---] followers, [----] engagements
"$SPRB Read BRINEURAs Summary Basis of Approval https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/761052Orig1s000SumR.pdf https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/761052Orig1s000SumR.pdf"
[X Link](https://x.com/surferbackpack/status/2021352911197045223) 2026-02-10T22:37Z [---] followers, [----] engagements
"$ALDX Two CRLs for Reproxalap: Nov [----] April [----] Round 3: PDUFA March [--] -FDA may reach out Feb [--] to discuss labeling if no major deficiencies in latest NDA app. -Jun. [----] FDA agrees ALDX submits chamber trial -Dec.2025 FDA requires failed field trial CSR be submitted"
[X Link](https://x.com/surferbackpack/status/2021452416332464290) 2026-02-11T05:13Z [---] followers, [---] engagements
"$SPRB adding on Monday. $IRON : PPIX had no validation as an endpoint. Weak correlation with clinical outcomes. Focused on reduction of PPIX. $RGNX : [--] year trial data Used HS-D2S6 (noisy). Focused on reduction. $SPRB : [--] year data HS-NRE Focused on Normalization"
[X Link](https://x.com/surferbackpack/status/2022513647109705771) 2026-02-14T03:30Z [---] followers, [----] engagements
"$CAPR wrote down my thoughts on the HOPE-3 data here. took both sides of the argument (PUL fragility vs. LVEF efficacy) https://docs.google.com/document/d/1qjZ5l1XJ9DhSmZ4_b9sMvQPkAXmEpiO6AiOuofnvcZY/editusp=sharing https://docs.google.com/document/d/1qjZ5l1XJ9DhSmZ4_b9sMvQPkAXmEpiO6AiOuofnvcZY/editusp=sharing"
[X Link](https://x.com/anyuser/status/1996722302785146948) 2025-12-04T23:24Z [---] followers, 28.9K engagements
"$SRPT Sarepta is reasonable. They halt to non-ambulatory (the accelerated approval) and refuse to stop the ambulatory (traditional approval). srp-9004 for LGMD is completely different. Vinay leaked this to media before formal communication this is political not scientific"
[X Link](https://x.com/anyuser/status/1946361145033695715) 2025-07-19T00:07Z [---] followers, [----] engagements
"@iam_johnw Leading with the head"
[X Link](https://x.com/anyuser/status/1822085716920218105) 2024-08-10T01:41Z [---] followers, [---] engagements
"The "Prasad Effect" is here. The FDA's CBER is dismantling the risk-benefit calculus for rare disease. The actions against $SRPT - the first major signal. My paper analyzes CBER why precedents like Zolgensma & Tysabri were ignored and what's next. https://docs.google.com/document/d/116VzYufxItdTTdx2CtYXX_kziODNN7nxrfyHCI4sDiA/editusp=sharing https://docs.google.com/document/d/116VzYufxItdTTdx2CtYXX_kziODNN7nxrfyHCI4sDiA/editusp=sharing"
[X Link](https://x.com/anyuser/status/1947207716755349592) 2025-07-21T08:11Z [---] followers, [----] engagements
"$SRPT So. the entire FDA Newsroom should just be shutdown because the officials prefer playing telephone with journalists directly instead. Updated: In a follow-up conversation on the safety study the senior official said Sarepta could make a change to the dosing or manufacturing and test it on the next [--] or [--] boys and show the liver-related issues were improved Updated: In a follow-up conversation on the safety study the senior official said Sarepta could make a change to the dosing or manufacturing and test it on the next [--] or [--] boys and show the liver-related issues were improved"
[X Link](https://x.com/anyuser/status/1948465770666885488) 2025-07-24T19:30Z [---] followers, [----] engagements
"Updated: In a follow-up conversation on the safety study the senior official said Sarepta could make a change to the dosing or manufacturing and test it on the next [--] or [--] boys and show the liver-related issues were improved"
[X Link](https://x.com/anyuser/status/1948460555422855338) 2025-07-24T19:09Z 11.1K followers, 63.2K engagements
"This is the craziest timeline of events: $SRPT We wanted to know how much balls Vinay Prasad had how much balls Sarepta had we got a full on fight. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-statement-elevidys https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-statement-elevidys"
[X Link](https://x.com/anyuser/status/1946359415692820957) 2025-07-19T00:00Z [---] followers, [----] engagements
"$SRPT still holding this puppy. Avg. of $14"
[X Link](https://x.com/anyuser/status/1955746500543438854) 2025-08-13T21:41Z [---] followers, [---] engagements
"$srpt time to take your wifes purse buy shares wait a year then pay her back double"
[X Link](https://x.com/anyuser/status/1946056194323525946) 2025-07-18T03:55Z [---] followers, [----] engagements
"$srpt walking to wifeys purse right now. Plan initiated"
[X Link](https://x.com/anyuser/status/1946263902943433149) 2025-07-18T17:40Z [---] followers, [---] engagements
"cash that. $SRPT @Sanctuary_Bio Yes interesting stuff. I bet Vinay Prasad is fired by EOY. @Sanctuary_Bio Yes interesting stuff. I bet Vinay Prasad is fired by EOY"
[X Link](https://x.com/anyuser/status/1950396901922394285) 2025-07-30T03:23Z [---] followers, [---] engagements
"@Sanctuary_Bio Yes interesting stuff. I bet Vinay Prasad is fired by EOY"
[X Link](https://x.com/anyuser/status/1948414077342220577) 2025-07-24T16:04Z [---] followers, [----] engagements
"$SRPT Zolgensma: liver toxicity - fda addresses using black box label and enhanced monitoring protocol. july 16: fda good faith gives black box label. srpt convenes for non-ambulant risk assessment. july 18: fda pulls plat desig requests halt"
[X Link](https://x.com/anyuser/status/1946362888433877057) 2025-07-19T00:14Z [---] followers, [----] engagements
"$SRPT they really did it. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-voluntary-pause-elevidys https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-voluntary-pause-elevidys"
[X Link](https://x.com/anyuser/status/1947445504692142144) 2025-07-21T23:56Z [---] followers, [----] engagements
"WOW WOW We have been on this. $SRPT This is exactly what diligent research gives you an edge on. Let us see what is next. Will be doing more work tonight the thesis always continues. #Breaking: Vinay Prasad a top official at the Food and Drug Administration has suddenly departed after a series of controversial decisions about a treatment for boys with Duchenne muscular dystrophy. https://t.co/2rIEFT5Cvf #Breaking: Vinay Prasad a top official at the Food and Drug Administration has suddenly departed after a series of controversial decisions about a treatment for boys with Duchenne muscular"
[X Link](https://x.com/anyuser/status/1950383115136061734) 2025-07-30T02:29Z [---] followers, [---] engagements
"#Breaking: Vinay Prasad a top official at the Food and Drug Administration has suddenly departed after a series of controversial decisions about a treatment for boys with Duchenne muscular dystrophy. https://trib.al/vfYBVjn https://trib.al/vfYBVjn"
[X Link](https://x.com/anyuser/status/1950361592329097481) 2025-07-30T01:03Z 160.7K followers, 458.9K engagements
"$SRPT In the end I think SRPT is still the most intriguing company in the space. Agamree is still a steroid with lots of competitors. ELEVIDYS is $3 mill a customer. If Deramiocel works lets say priced at $1mill a customer annually $CAPR is intriguing"
[X Link](https://x.com/anyuser/status/1935810049395638436) 2025-06-19T21:20Z [---] followers, [----] engagements
"@stocksandreales Easy 120% appreciate you"
[X Link](https://x.com/anyuser/status/1882808400456908852) 2025-01-24T15:11Z [---] followers, [---] engagements
"@SarwanJohn Thank you brotha up 80% on the week. Loaded $HYZN and $XPON and a small $VEV for the weekend. See what happens with those"
[X Link](https://x.com/anyuser/status/1821986617588117855) 2024-08-09T19:07Z [---] followers, [----] engagements
"$SRPT for bull: AAVrh74 SRP9003 EMERGENCE data tmrw shows good safety no acute liver toxicity. Market already hated this with ELEVIDYS. if good BLA goes through SRPT and FDA at least move forward on something for now"
[X Link](https://x.com/anyuser/status/1939604250562314298) 2025-06-30T08:37Z [---] followers, [----] engagements
"$SRPT "investigating the death of an 8-year-old boy who received Elevidys". "The death occurred on June [--] 2025." this is not a 3rd elevidys kid right is it one of the two originals or is it a new death"
[X Link](https://x.com/anyuser/status/1948863846183305544) 2025-07-25T21:52Z [---] followers, [----] engagements
"$SRPT CHEAT SHEET. AAVrh74 = the car. ELEVIDYS SRP-9003 = the payload. part of SRPT build on idea: the car is safe. FDA grants platform tech desig of AAVrh74. - market wants to know: if data good car is safe. big win. if data bad is the car (the foundation) to the junkyard"
[X Link](https://x.com/anyuser/status/1939608764203827668) 2025-06-30T08:55Z [---] followers, [----] engagements
"$SRPT $CAPR Deramiocel sidesteps dystophen-restoration focuses on biggest clinical problem: heart failure. compelling value proposition. if Deramiocel works its added to baseline therapy (ELEVIDYS or Agamree this is the question)"
[X Link](https://x.com/anyuser/status/1935767750393385208) 2025-06-19T18:32Z [---] followers, [----] engagements
"$SRPT stratification has to matter to the fda. non-amb: generally.pre compromised liver (underlying hepatic steatosis) compromised cardiac output impairs systemic clearance of aav vector lack a physiological reserve. sarepta's refusal is smart"
[X Link](https://x.com/anyuser/status/1946480119075488036) 2025-07-19T07:59Z [---] followers, [---] engagements
"$SRPT played out exactly wait Monday. $SRPT assuming black box label is coming read about Zolgensma gene therapy for SMA. Dont think the FDA pulls Elevidys now just based on patient need for DMD. One more patient death though - game over Label pulled The risk profile of gene therapies man. $SRPT assuming black box label is coming read about Zolgensma gene therapy for SMA. Dont think the FDA pulls Elevidys now just based on patient need for DMD. One more patient death though - game over Label pulled The risk profile of gene therapies man"
[X Link](https://x.com/anyuser/status/1946836135918850095) 2025-07-20T07:34Z [---] followers, [---] engagements
"$SRPT assuming black box label is coming read about Zolgensma gene therapy for SMA. Dont think the FDA pulls Elevidys now just based on patient need for DMD. One more patient death though - game over Label pulled The risk profile of gene therapies man"
[X Link](https://x.com/anyuser/status/1934541706470502854) 2025-06-16T09:20Z [---] followers, [----] engagements
"$srpt elevidys competitors: AAV: Regenxbio: phase 1/2 AFFINITY DUCHENNE trial. BLA mid '26. Solid Biosci:sgt-003 phase1/2. Antisense oligonucleotides: - sort of the challenge to srpt PMOs Dyne-251 phase1/2 other: capr deramiocel (lol) topline q3 [--] remmber CRL lol"
[X Link](https://x.com/anyuser/status/1946476296294568283) 2025-07-19T07:44Z [---] followers, [---] engagements
"$SRPT July [--] read this pls. bridgebio/mlbio give readout on BBP-418 in LGMD 2i/R9 (oral) spr-9003 by sarepta for LGMD 2E/R4 (aav-gene therapy) AskBio AB-1003 for LGMD 2i/R9 (gene therapy) atamyo therapeutics for LGMD 2C/R5 (gene therapy) - atamyo still early stage"
[X Link](https://x.com/anyuser/status/1939616230098579959) 2025-06-30T09:25Z [---] followers, [---] engagements
"@stockplaymaker1 Its fake"
[X Link](https://x.com/anyuser/status/1825976809650892848) 2024-08-20T19:22Z [---] followers, [----] engagements
"@StockSavvyShay NVDA is not a hardware company 🤣🤣🤣🤣🤣. Holy smokes"
[X Link](https://x.com/anyuser/status/1932068610094383408) 2025-06-09T13:33Z [---] followers, [--] engagements
"$SRPT LGMD2E/R4 is very rare. [----] Bayesian Analysis: [---] per million people. But complete lack of approved modifying treatment. EMERGENE trial: ambulatory + non-amb Jun 2025-FDA grants Pltform tech desig. to AAVrh74 vector. expedite reviews of AAVrh74 products"
[X Link](https://x.com/anyuser/status/1939599326222950786) 2025-06-30T08:18Z [---] followers, [----] engagements
"$SRPT even if ELEVIDYS revenue collapses by 80% and core PMO takes a hit of 30% the current share price is basically that ($20 on 06-18-2025). Basically market is saying ELEVIDYS is a [--]. Also pricing in potential lawsuits funding ENVISION brand stigma. Peak pessimism"
[X Link](https://x.com/anyuser/status/1935511966992449905) 2025-06-19T01:36Z [---] followers, [---] engagements
"Agenda for the weekend for me: $SRPT Elevidys + other pipeline stuff $CAPR hmm $AVXL scam Not sure what else trying to get nice with the biotech. The big $INMB data release on Monday should be a fun watch"
[X Link](https://x.com/anyuser/status/1938805287517045236) 2025-06-28T03:42Z [---] followers, [---] engagements
"$SRPT That anonymous FDA official: I dont know how you can approve a product that maybe shouldnt ever come to market in the first place and is killing people. That is a very tough proposition to keep it on the market the official said. - https://endpoints.news/sarepta-would-have-to-conduct-new-studies-to-get-back-on-market-fda-official-says/ https://endpoints.news/sarepta-would-have-to-conduct-new-studies-to-get-back-on-market-fda-official-says/"
[X Link](https://x.com/anyuser/status/1948416253716058469) 2025-07-24T16:13Z [---] followers, [---] engagements
"Times like this where you think: Twitter is the Bloomberg terminal news feed for retailers. Ahh democratization of education via tech. Good times to live in"
[X Link](https://x.com/anyuser/status/1948467082661953636) 2025-07-24T19:35Z [---] followers, [---] engagements
"@xshakeshake if im srpt ceo hell yes hopefully. Hard to tell if Prasad is strongarming or legitimately is going to go through the process of seeing if elevidys can resume shipments in amb group at least"
[X Link](https://x.com/anyuser/status/1947463339564798287) 2025-07-22T01:06Z [---] followers, [---] engagements
"Well this manifested back from [--] weeks ago. FDA press release after market close today $SRPT major risk for srpt is this: AAV-mediated hepatotoxicity is not specific to ELEVIDYS. if safety data bad with SRP-9003 its the AAVrh74 platform whole pipeline is down bad. mstanley: project +1.2bill rev/yr from LGMD by [----]. $SRPT major risk for srpt is this: AAV-mediated hepatotoxicity is not specific to ELEVIDYS. if safety data bad with SRP-9003 its the AAVrh74 platform whole pipeline is down bad. mstanley: project +1.2bill rev/yr from LGMD by 2030"
[X Link](https://x.com/anyuser/status/1946275191790960656) 2025-07-18T18:25Z [---] followers, [---] engagements
"$SRPT major risk for srpt is this: AAV-mediated hepatotoxicity is not specific to ELEVIDYS. if safety data bad with SRP-9003 its the AAVrh74 platform whole pipeline is down bad. mstanley: project +1.2bill rev/yr from LGMD by 2030"
[X Link](https://x.com/anyuser/status/1939603777721696370) 2025-06-30T08:35Z [---] followers, [---] engagements
"if interested in $SRPT you need to be looking at Alnylam Pharmaceuticals Avidity Biosciences Dyne Therapeutics. siRNA. delivery to liver (LNP is good). delivery to skeletal muscle (AOCs look like gold standard target TfR1 for entry)"
[X Link](https://x.com/surferbackpack/status/1951895466670789055) 2025-08-03T06:38Z [---] followers, [---] engagements
"$SRPT leaves me to think. draw down in stock not only bc of ELEVIDYS deaths. AAVrh74 failure/caution/success is priced in to what extent how much lower can SRPT go if EMERGENCE bad tmrw the pipeline is at stake right here. this is the fear. this is the price"
[X Link](https://x.com/anyuser/status/1939604611511603487) 2025-06-30T08:39Z [---] followers, [---] engagements
"@StockSavvyShay Quantum will scale monte Carlo simulations Quantum will boost drug discovery The kicker 90% of the publicly traded quantum stocks are duds. The bull aint bulling. This guy Shay is an idiot"
[X Link](https://x.com/anyuser/status/1932066300928671928) 2025-06-09T13:24Z [---] followers, [---] engagements
"Have a Other bios im looking: business models i like (not bullish yet) Vertex Pharmaceuticals ($VRTX) Alnylam Pharmaceuticals ($ALNY) BioMarin Pharmaceutical ($BMRN) Krystal Biotech ($KRYS) Rocket Pharmaceuticals ($RCKT) Denali Therapeutics ($DNLI) https://www.barrons.com/articles/sarepta-stock-gene-therapy-treatment-148476abreflink=desktopwebshare_permalink https://www.barrons.com/articles/sarepta-stock-gene-therapy-treatment-148476abreflink=desktopwebshare_permalink"
[X Link](https://x.com/anyuser/status/1941735880613609816) 2025-07-06T05:48Z [---] followers, [---] engagements
"@bradloncar @Roche @IAmBiotech I think Vinay needs to reform his biologics program by Monday"
[X Link](https://x.com/anyuser/status/1946638650395267305) 2025-07-19T18:29Z [---] followers, [----] engagements
"$srpt u might be thinking why even talk about AAV anymore if they pivot to siRNA (siRNA is the long game). AAV (the near term game) is still the money engine that would fund siRNA and [----] debt. and dont forget srp-9003 (aavrh74) later this year for LGMD"
[X Link](https://x.com/anyuser/status/1951906075864674560) 2025-08-03T07:20Z [---] followers, [---] engagements
"$SRPT okay thats one step done. https://www.fda.gov/news-events/press-announcements/fda-recommends-removal-voluntary-hold-elevidys-ambulatory-patients https://www.fda.gov/news-events/press-announcements/fda-recommends-removal-voluntary-hold-elevidys-ambulatory-patients"
[X Link](https://x.com/anyuser/status/1949936056281026636) 2025-07-28T20:52Z [---] followers, [---] engagements
"$srpt pt. [--] srpt competitors: LNPs - non viral gene delivery - this is future future dyne-251: they want bla for accelerated approval early '26. challenge srpt EXONDYS 51"
[X Link](https://x.com/anyuser/status/1946477292882186743) 2025-07-19T07:48Z [---] followers, [---] engagements
"nothing to do with political affiliation all to do with landscape. the class of the useless credentialed is on the rise. always believed to use LLMs as your personal associate/analyst. Work rate goes up 10x if you use it like a project manager. you all know this. A partner at a prominent law firm told me AI is now doing work that used to be done by 1st to 3rd year associates. AI can generate a motion in an hour that might take an associate a week. And the work is better. Someone should tell the folks applying to law school right now. A partner at a prominent law firm told me AI is now doing"
[X Link](https://x.com/anyuser/status/1949338378798837995) 2025-07-27T05:17Z [---] followers, [---] engagements
"A partner at a prominent law firm told me AI is now doing work that used to be done by 1st to 3rd year associates. AI can generate a motion in an hour that might take an associate a week. And the work is better. Someone should tell the folks applying to law school right now"
[X Link](https://x.com/anyuser/status/1949160562350522482) 2025-07-26T17:31Z 1.8M followers, 7.1M engagements
"$SRPT am I right in saying that capricore is not a threat to sarepta at all If deramiocel is for dmd-associated cardiomyopathy would that not just turn DMD treatment into a combination of ELEVIDYS for baseline therapy and deramiocel"
[X Link](https://x.com/anyuser/status/1935490840702632121) 2025-06-19T00:12Z [---] followers, [---] engagements
"@stockplaymaker1 $tnxp beautiful"
[X Link](https://x.com/anyuser/status/1826292095826661510) 2024-08-21T16:15Z [---] followers, [---] engagements
"$srpt TYSABRI (biogen/elan): feb [--] 2005- withdrawn from market - BIIB [--] to [--] ELN [--] to [--] march [--] [----] - fda advis. comm. recommends return to market. - BIIB 46to [--]. ELN [--] to [--]. srpt situation is a hybrid of BIIB (deep piplin) and ELN (tys was tied to future)"
[X Link](https://x.com/anyuser/status/1946489682369696238) 2025-07-19T08:37Z [---] followers, [---] engagements
"waiting to see an opportunity for puts on $ATYR and $SLNO and $QBTS and maybe $CAPR not doing anything now. will wait. if nothing im cash"
[X Link](https://x.com/anyuser/status/1956989627283480596) 2025-08-17T08:01Z [---] followers, [---] engagements
"$STSS ouch. Sneaky warrant conversion in the offering. pray for SC plant pr or 💣"
[X Link](https://x.com/anyuser/status/1886426867588100596) 2025-02-03T14:49Z [---] followers, [---] engagements
"$SRPT SRP-9003 program: phase1/2 enroll [--] patients. efficacy was good. safety was avg. transient elev. in g-glutamyl-trnsfase. one serious adverse evnt: acute liver injury needed hospitaliz resolved with standard care. phase [--] enrolled 15-17 patients"
[X Link](https://x.com/anyuser/status/1939600926370840764) 2025-06-30T08:24Z [---] followers, [---] engagements
"$SRPT Forward Timeline on Elevidys [--]. Enhanced Safety Protocol Submission 2a. FDA Feedback 2b. Status of ENVISION confirmatory trial 2c. Status of commercial halt on non-ambulatory Wildcards: - another serious adverse event (SAE) - international regulatory decision (EMA)"
[X Link](https://x.com/anyuser/status/1935603577491497161) 2025-06-19T07:40Z [---] followers, [---] engagements
Limited data mode. Full metrics available with subscription: lunarcrush.com/pricing
@surferbackpack Randall ChingRandall Ching posts on X about $srpt, fda, $capr, $galt the most. They currently have [---] followers and [---] posts still getting attention that total [-----] engagements in the last [--] hours.
Social category influence stocks finance technology brands social networks cryptocurrencies ncaa basketball travel destinations
Social topic influence $srpt, fda #122, $capr, $galt, data, market, $sprb #1, gene, in the, protocol
Top accounts mentioned or mentioned by @jeremyj0916 @brainymarsupial @lqdabull1 @dashwend @sanctuarybio @stocksavvyshay @golfnut48846224 @sarwanjohn @hall8jack @greatsehaj @stockplaymaker1 @daveconnellysg @toucantrades @tariusishere @westsideguy3 @alohaoasis @alansharp @yachmod @yaniruliel @stockchumpski
Top assets mentioned Sarepta Therapeutics, Inc. (SRPT) Spruce Biosciences, Inc. (SPRB) Akropolis (AKRO) Catalyst Pharmaceutical Inc. (CPRX) Soleno Therapeutics, Inc. Common Stock (SLNO) D-Wave Quantum Inc. (QBTS) Biohaven Pharmaceutical Holding Company Ltd. (BHVN) Sharps Technology, Inc. (STSS)
Top posts by engagements in the last [--] hours
"@DaveConnellySG You watch a Temple Basketball game this season"
X Link 2024-03-08T12:20Z [--] followers, [----] engagements
"@ToucanTrades @SarwanJohn That was up all day. Its only for the A class which $LUXH is not. Might indicate good cash flow"
X Link 2024-09-03T23:32Z [--] followers, [---] engagements
"$RGTI $6 puts @ [----] February A reverse of the classic [----] thread: Pitch a publicly traded company which: [--]. Has at least $10 billion of market cap [--]. Will decline 90% from current (not peak) prices First pick to -90% wins ties go to earlier post. I'll pick something tasteful but thematic as a prize. A reverse of the classic [----] thread: Pitch a publicly traded company which: [--]. Has at least $10 billion of market cap [--]. Will decline 90% from current (not peak) prices First pick to -90% wins ties go to earlier post. I'll pick something tasteful but thematic as a prize"
X Link 2024-12-24T22:23Z [--] followers, [---] engagements
"$STSS ouch. Sneaky warrant conversion in the offering. pray for SC plant pr or 💣"
X Link 2025-02-03T14:49Z [---] followers, [---] engagements
"@Tariusishere stop. eps on a r/d stage company is dumb. 280k to 308k shares outstanding from last q. dilution. qc is cool stuff any public company rn is a sham. ibm/msft are where qc is at"
X Link 2025-05-12T21:24Z [--] followers, [---] engagements
"$SRPT my thoughts part [--]. Not premium so its a thread. Alright everybody panicking on $SRPT heres my take. A second non-ambulatory Duchenne patient just died from acute liver failure after receiving Elevidys"
X Link 2025-06-16T05:42Z [--] followers, [---] engagements
"$SRPT part [--]. Two deaths both in nonambulatory patients (first a 16yearold second age not disclosed) in three months. Sarepta now pauses Elevidys use in that group and halt its ENVISION confirmatory trial which FDA accelerated approval mandated"
X Link 2025-06-16T05:45Z [--] followers, [---] engagements
"$SRPT part [--]. They also notified regulators and Roche (their ex-US partner) did the same globally. serious inflection point for the stock and for gene therapy trust overall"
X Link 2025-06-16T05:46Z [--] followers, [---] engagements
"$SRPT part [--]. The FDAs watching closely especially with Vinay Prasad now heading biologics after Peter Marks stepped down. Prasads been openly critical of Elevidys even before this second death"
X Link 2025-06-16T05:46Z [--] followers, [---] engagements
"$SRPT part [--]. Sareptas going to propose a revised protocol with added immunosuppression (possibly sirolimus + steroids) but dosing in non-ambulatory patients is paused until the FDA signs off. ambulatory use continues but even there uptake might slow"
X Link 2025-06-16T05:48Z [--] followers, [---] engagements
"$SRPT part [--]. Families and clinicians get more cautious. ENVISION is paused label updates are coming and the path to broader adoption just got steeper"
X Link 2025-06-16T05:49Z [--] followers, [---] engagements
"$SRPT part [--]. My concerns. Elevidys did $375M in Q1 alone and a lot of that growth was expected to come from expanding into the non-ambulatory population. Thats now frozen"
X Link 2025-06-16T05:50Z [---] followers, [---] engagements
"$SRPT part [--]. If the FDA doesnt buy Sareptas revised safety strategy (theyre looking at sirolimus + steroids) its possible Elevidys loses that label entirely. Worst case thats a massive revenue hole. Best case recovery takes time (6-12 months) and the stigma lingers"
X Link 2025-06-16T05:51Z [--] followers, [---] engagements
"$SRPT here. - Ingrams tone on Monday = everything - Rodino-Klapac: sirolimus or bust - Prasad (FDA) aint Marks not friendly - ENVISION 303: paused no restart label risk - Roche global pullback - No LGMD backfill near term - Risk premium on gene therapy just widened"
X Link 2025-06-16T05:56Z [--] followers, [---] engagements
"$SRPT Im long. Positions small not adding yet. Let this bleed out. ENVISION needs FDA signoff. If sirolimus clears and no 3rd death recovery trades back to 40s. But if uptake in ambulatories stalls or Prasad blocks trial restart downside opens hard. [---] month trench"
X Link 2025-06-16T05:59Z [--] followers, [---] engagements
"$SRPT good night press tomorrow. Dont need to catch knife need to know where floor is. Watching tmrw call Roche and FDA comms. Will average only when market stops reacting emotionally and starts asking about revenue math"
X Link 2025-06-16T06:00Z [---] followers, [---] engagements
"$SRPT assuming black box label is coming read about Zolgensma gene therapy for SMA. Dont think the FDA pulls Elevidys now just based on patient need for DMD. One more patient death though - game over Label pulled The risk profile of gene therapies man"
X Link 2025-06-16T09:20Z [---] followers, [----] engagements
"$SRPT have to say though that AAV therapies are fascinating just from my bio background. Flooding the body with viral vectors to deliver genes is a phenomenon. Then again the immune response is patient specific non linear. For the sake of these patients pray new protocol works"
X Link 2025-06-16T09:23Z [--] followers, [---] engagements
"$SRPT a gamma trade now not a beta trade"
X Link 2025-06-16T09:42Z [---] followers, [----] engagements
"$SRPT added more at [----]. buying a 1.8b company with full exclusivity in an orphan market for [--]. 100m shares outstanding"
X Link 2025-06-16T16:57Z [--] followers, [---] engagements
"learning how to use USPTO. Cool stuff with the advanced search. Sort of like a new language with the search queries. Last updated [----] but its neat stuff. Going to try do a patent tracker for maybe quantum related developments Learning about A1 B1 B2"
X Link 2025-06-17T08:03Z [--] followers, [--] engagements
"@jeremyj0916 Yeah Im definitely considering it. Puts way too expensive/if they delay data release those get IV crushed with no movement anyways. Ill look into shorting "
X Link 2025-06-18T20:59Z [--] followers, [--] engagements
"$SRPT am I right in saying that capricore is not a threat to sarepta at all If deramiocel is for dmd-associated cardiomyopathy would that not just turn DMD treatment into a combination of ELEVIDYS for baseline therapy and deramiocel"
X Link 2025-06-19T00:12Z [---] followers, [---] engagements
"$SRPT Sarepta's PMO business (eteplirsen golodirsen casimersen) seems to not be threatened. NS Pharma's Viltepso is the only other competitor to srpt's pmo and theyre not disrupting much"
X Link 2025-06-19T00:15Z [--] followers, [---] engagements
"$SRPT then Agamree (the steroid competitor) is more so a steroid innovation that wouldnt disrupt dystrophin producing therapies like ELEVIDYS. Couldnt a combination of Agamree + Deramiocel + ELEVIDYS be the future norm"
X Link 2025-06-19T00:18Z [--] followers, [---] engagements
"$SRPT even if ELEVIDYS revenue collapses by 80% and core PMO takes a hit of 30% the current share price is basically that ($20 on 06-18-2025). Basically market is saying ELEVIDYS is a [--]. Also pricing in potential lawsuits funding ENVISION brand stigma. Peak pessimism"
X Link 2025-06-19T01:36Z [---] followers, [---] engagements
"$SRPT difficulty is this. its not as simple as seeing the 0.25% incidence rate of acute liver failure auto assuming that benefit outweighs risk/harm. FDA standard is 'do no harm'. unpredictable death is a monumental failure. srpt has to convince FDA of revised safety protocol"
X Link 2025-06-19T07:21Z [---] followers, [---] engagements
"$SRPT Great article from Christine McSherry captures the more human parts of this situation: her son had DMD and took one of srpt's PMOs. shes hopeful for gene therapy. calls out the lack of FAERS system updates from srpt. management must improve. https://www.statnews.com/2025/06/18/sarepta-duchenne-muscular-dystrophy-elevidys-death-fda-advocacy/ https://www.statnews.com/2025/06/18/sarepta-duchenne-muscular-dystrophy-elevidys-death-fda-advocacy/"
X Link 2025-06-19T07:25Z [--] followers, [---] engagements
"$SRPT Forward Timeline on Elevidys [--]. Enhanced Safety Protocol Submission 2a. FDA Feedback 2b. Status of ENVISION confirmatory trial 2c. Status of commercial halt on non-ambulatory Wildcards: - another serious adverse event (SAE) - international regulatory decision (EMA)"
X Link 2025-06-19T07:40Z [---] followers, [---] engagements
"$SRPT $CAPR Reading on this. Deramiocel vs. Eteplirsen Cell therapy vs. Exon-Skipping therapy Eteplirsen for the 13% of patients with exon-51 mutation showed sig. slow rate of LVEF decline. Deramiocel for mainly older patients Stat sig. improvement in LVEF"
X Link 2025-06-19T18:08Z [--] followers, [---] engagements
"$SPRT $CAPR In DMD heres my cause of death table: Cardiopulmonary Failure (total) 80% - Cardiac Failure (40-55%): cardiomyopathy(20-40%) sudden cardiac/arrhythmia (10-15%) - Respiratory Failure (25-40%): chronic (14%) acute (24%) Other causes (total) 20%"
X Link 2025-06-19T18:16Z [---] followers, [---] engagements
"$SRPT $CAPR with NI-ventilation the share of cardiomyopathy/arrhythmia goes up up up. Cardiomyopathy onset [--] years 50% of DMD show evidence at [--] years. LVEF LGE NT-proBNP CK-MB are the critical targets for DMD intervention. assume Deramiocel for whole DMD pop"
X Link 2025-06-19T18:22Z [--] followers, [---] engagements
"$SRPT $CAPR Eteplirsen sig. slows LVEF decline (-0.66%/yr Tx vs. -1.38%/yr control) but its main function is dystrophin-restoration and for exon-51 mutation patients. Deramiocel improves LVEF (+1.2% overall +3.0% subgroup) ELEVIDYS failed cardiac MRI measures"
X Link 2025-06-19T18:30Z [--] followers, [---] engagements
"$SRPT $CAPR Deramiocel sidesteps dystophen-restoration focuses on biggest clinical problem: heart failure. compelling value proposition. if Deramiocel works its added to baseline therapy (ELEVIDYS or Agamree this is the question)"
X Link 2025-06-19T18:32Z [---] followers, [----] engagements
"$SRPT My main thought: who has the most acceptable balance of benefit and risk for baseline treatment of DMD - is it ELEVIDYS or Agamree ELEVIDYS - you believe in AAV gene therapy Agamree - you believe in safety profile of new corticosteroid $CPRX the competitor"
X Link 2025-06-19T18:43Z [---] followers, [---] engagements
"$SRPT vs. $CPRX [--]. ELEVIDYS wins the "wow factor" for foundational repair. [--]. Agamree wins safety profile clinicians know what to expect with corticosteroid ELEVIDYS acute liver fail scary"
X Link 2025-06-19T19:00Z [--] followers, [---] engagements
"$SRPT [--]. Trust of the DMD community. The heart wants promise of ELEVIDYS but the head leaning towards Agamree [--]. Practicality: ELEVIDYS is one time major medical event. Agamree is daily oral. srpt becomes the high risk-high reward gamble if safety profile doesnt improve"
X Link 2025-06-19T19:02Z [---] followers, [---] engagements
"$SRPT In the end I think SRPT is still the most intriguing company in the space. Agamree is still a steroid with lots of competitors. ELEVIDYS is $3 mill a customer. If Deramiocel works lets say priced at $1mill a customer annually $CAPR is intriguing"
X Link 2025-06-19T21:20Z [---] followers, [----] engagements
"$SRPT Tomorrows agenda for me is to look into probability of sirolimus getting the pass from FDA for ELEVIDYS. Also will look deeper into Zolgensma. Also comparing study pop. sizes between $CAPR and $SRPT"
X Link 2025-06-20T05:34Z [---] followers, [---] engagements
"$CAPR eteplirsen by $SRPT in [----] had [--] patients and they got accelerated approval now deramiocel seeking full BLA but there is precedent. Unmet patient need is on deramiocel's side. ADCOMM July [--] PDUFA deadline Aug 31"
X Link 2025-06-20T05:47Z [---] followers, [---] engagements
"$SRPT interesting week in the DMD space and I am glad I started researching this. Verdun and her deputy gets ousted (need more details) its Vinay's show now running biologics. Vinay supposedly still pro gene therapy for rare disease. Post-marketing safety checks is the discount"
X Link 2025-06-20T17:45Z [---] followers, [---] engagements
"@westsideguy3 it seems unclear right now. story is that Verdun had it on the table and was discussing with Prasad to do the ADCOMM as scheduled but now she's out it and reports are that Prasad cancelled. Capricore said they are still planning as if its going to happen but unclear. Caution"
X Link 2025-06-20T19:50Z [--] followers, [--] engagements
"going on vacation will come back with full brkdwn $SRPT just a few bullets if you explore: rituximab + sirolimus UofF health CMV infection in 1st elevidys death sirolimus good but creates complex risk paradigm Roctavian AAV5: label precedent Hemgenix/Zolgensma: take a look"
X Link 2025-06-21T17:56Z [---] followers, [---] engagements
"@jeremyj0916 Also Im going to look into the management team more. I just see a lot of potential in SRPT its a good pipeline. New management and Wall Street regains trust in srpt Also want to keep track of the next quarter holdings report. See what funds dumped with the patient death scare"
X Link 2025-06-28T04:14Z [--] followers, [---] engagements
"$SRPT LGMD2E/R4 is very rare. [----] Bayesian Analysis: [---] per million people. But complete lack of approved modifying treatment. EMERGENE trial: ambulatory + non-amb Jun 2025-FDA grants Pltform tech desig. to AAVrh74 vector. expedite reviews of AAVrh74 products"
X Link 2025-06-30T08:18Z [---] followers, [----] engagements
"$SRPT SRP-9003 program: phase1/2 enroll [--] patients. efficacy was good. safety was avg. transient elev. in g-glutamyl-trnsfase. one serious adverse evnt: acute liver injury needed hospitaliz resolved with standard care. phase [--] enrolled 15-17 patients"
X Link 2025-06-30T08:24Z [---] followers, [---] engagements
"$SRPT for bull: AAVrh74 SRP9003 EMERGENCE data tmrw shows good safety no acute liver toxicity. Market already hated this with ELEVIDYS. if good BLA goes through SRPT and FDA at least move forward on something for now"
X Link 2025-06-30T08:37Z [---] followers, [----] engagements
"@AlohaOasis @Hall8Jack Yes theyd get sued to oblivion otherwise. Company says they release data and doesnt its self sabotage"
X Link 2025-06-30T15:50Z [--] followers, [---] engagements
"@Hall8Jack Interesting. Yeah I saw that conference schedule. I just stick to what Sarepta says. Said 1st half of [--] which is quarter end today. I dont necessarily trust anything any biotech says before data release. They have fiduciary duty. I appreciate your comments"
X Link 2025-06-30T15:52Z [--] followers, [---] engagements
"$SRPT no LGMD data today understandable given everything management is going through. today's pr on inducement grant was not much. standard equity incentivization for new employees. good they still hiring. good employees incentivized to help company. nothing special"
X Link 2025-06-30T23:03Z [--] followers, [---] engagements
"$srpt nothing changed as of today. dont look at the +7% as legit. all pharma/biotech have the same daily charts. correlation not sector rotation"
X Link 2025-07-01T16:53Z [--] followers, [---] engagements
"$SRPT LGMD phase1/2 read refresher: https://pmc.ncbi.nlm.nih.gov/articles/PMC10803256/pdf/41591_2023_Article_2730.pdf https://pmc.ncbi.nlm.nih.gov/articles/PMC10803256/pdf/41591_2023_Article_2730.pdf"
X Link 2025-07-06T07:01Z [--] followers, [---] engagements
"$CAPR well I was busy all week but proud to see that my CAPR short worked. Pretty funny looking at all retail so shocked at the rejection. Who in their right mind was long CAPR if you did you need a serious risk assessment lesson. It was clearly you stay cash or go short"
X Link 2025-07-12T20:48Z [---] followers, [---] engagements
"@jeremyj0916 Any bios on your radar Been looking at Alnylam Biomarin Arrowhead besides dog pound Sarepta"
X Link 2025-07-18T07:11Z [---] followers, [---] engagements
"@GreatSehaj nice. remember risk management position sizing"
X Link 2025-07-18T18:22Z [---] followers, [--] engagements
"Well this manifested back from [--] weeks ago. FDA press release after market close today $SRPT major risk for srpt is this: AAV-mediated hepatotoxicity is not specific to ELEVIDYS. if safety data bad with SRP-9003 its the AAVrh74 platform whole pipeline is down bad. mstanley: project +1.2bill rev/yr from LGMD by [----]. $SRPT major risk for srpt is this: AAV-mediated hepatotoxicity is not specific to ELEVIDYS. if safety data bad with SRP-9003 its the AAVrh74 platform whole pipeline is down bad. mstanley: project +1.2bill rev/yr from LGMD by 2030"
X Link 2025-07-18T18:25Z [---] followers, [---] engagements
"This is the craziest timeline of events: $SRPT We wanted to know how much balls Vinay Prasad had how much balls Sarepta had we got a full on fight. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-statement-elevidys https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-statement-elevidys"
X Link 2025-07-19T00:00Z [---] followers, [----] engagements
"$SRPT Zolgensma: liver toxicity - fda addresses using black box label and enhanced monitoring protocol. july 16: fda good faith gives black box label. srpt convenes for non-ambulant risk assessment. july 18: fda pulls plat desig requests halt"
X Link 2025-07-19T00:14Z [---] followers, [----] engagements
"$SRPT fda is going to make another press release fighting srpt watch. If this thing escalates srpt goes arbitrary and capricious lawsuit"
X Link 2025-07-19T00:17Z [--] followers, [---] engagements
"$srpt elevidys competitors: AAV: Regenxbio: phase 1/2 AFFINITY DUCHENNE trial. BLA mid '26. Solid Biosci:sgt-003 phase1/2. Antisense oligonucleotides: - sort of the challenge to srpt PMOs Dyne-251 phase1/2 other: capr deramiocel (lol) topline q3 [--] remmber CRL lol"
X Link 2025-07-19T07:44Z [---] followers, [---] engagements
"$SRPT stratification has to matter to the fda. non-amb: generally.pre compromised liver (underlying hepatic steatosis) compromised cardiac output impairs systemic clearance of aav vector lack a physiological reserve. sarepta's refusal is smart"
X Link 2025-07-19T07:59Z [---] followers, [---] engagements
"$srpt hey srpt itd be really nice if you could identify the risk factor causing the ALF. tysabri for ms got pulled then reintroduced under strict risk management program. i just dont think its game over for ELEVIDYS"
X Link 2025-07-19T08:17Z [---] followers, [---] engagements
"$srpt TYSABRI (biogen/elan): feb [--] 2005- withdrawn from market - BIIB [--] to [--] ELN [--] to [--] march [--] [----] - fda advis. comm. recommends return to market. - BIIB 46to [--]. ELN [--] to [--]. srpt situation is a hybrid of BIIB (deep piplin) and ELN (tys was tied to future)"
X Link 2025-07-19T08:37Z [---] followers, [---] engagements
"$SRPT they really did it. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-voluntary-pause-elevidys https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-voluntary-pause-elevidys"
X Link 2025-07-21T23:56Z [---] followers, [----] engagements
"$SRPT So. the entire FDA Newsroom should just be shutdown because the officials prefer playing telephone with journalists directly instead. Updated: In a follow-up conversation on the safety study the senior official said Sarepta could make a change to the dosing or manufacturing and test it on the next [--] or [--] boys and show the liver-related issues were improved Updated: In a follow-up conversation on the safety study the senior official said Sarepta could make a change to the dosing or manufacturing and test it on the next [--] or [--] boys and show the liver-related issues were improved"
X Link 2025-07-24T19:30Z [---] followers, [----] engagements
"Times like this where you think: Twitter is the Bloomberg terminal news feed for retailers. Ahh democratization of education via tech. Good times to live in"
X Link 2025-07-24T19:35Z [---] followers, [---] engagements
"$SRPT okay thats one step done. https://www.fda.gov/news-events/press-announcements/fda-recommends-removal-voluntary-hold-elevidys-ambulatory-patients https://www.fda.gov/news-events/press-announcements/fda-recommends-removal-voluntary-hold-elevidys-ambulatory-patients"
X Link 2025-07-28T20:52Z [---] followers, [---] engagements
"waiting to see an opportunity for puts on $ATYR and $SLNO and $QBTS and maybe $CAPR not doing anything now. will wait. if nothing im cash"
X Link 2025-08-17T08:01Z [---] followers, [---] engagements
"adding more $SRPT this week. Will do another deep dive"
X Link 2025-09-15T06:49Z [---] followers, [---] engagements
"$GALT look at community boards for GALT. foolish "army" of believers with vague hopes of buyout and approval. Little skepticism delusional optimism. Classic signs of a fraudulent biotech"
X Link 2025-09-19T19:51Z [---] followers, [--] engagements
"$GALT cash value is about 15-30 cents. This should trade below $1 by the time of EOP2 meeting results next year. I believe FDA will ask for another confirmatory trial. GALT cannot keep p-hacking"
X Link 2025-09-19T22:46Z [---] followers, [---] engagements
"$SRPT https://fis.fda.gov/sense/app/95239e26-e0be-42d9-a960-9a5f7f1c25ee/sheet/6b5a135f-f451-45be-893d-20aaee34e28e/state/analysis https://fis.fda.gov/sense/app/95239e26-e0be-42d9-a960-9a5f7f1c25ee/sheet/6b5a135f-f451-45be-893d-20aaee34e28e/state/analysis"
X Link 2025-09-27T07:36Z [---] followers, [---] engagements
"$SRPT up 75% on my position. Reducing size a tad today. Still a substantial part of my portfolio"
X Link 2025-10-02T15:52Z [---] followers, [---] engagements
"$SRPT Abstract 732LBP: Preliminary analysis of safety tolerability and efficacy of a prophylactic sirolimus protocol for patients receiving ELEVIDYS Vienna WMS Conference Oct 7-11 Probably a press release soon from sarepta"
X Link 2025-10-02T21:24Z [---] followers, [----] engagements
"$SRPT abstract also for SRP-9003 (LGMD) 734LBP Expression of SGCB and safety following bidridistrogene xeboparvovec treatment in patients with LGMD2E/R4: results from the EMERGENE phase [--] study https://www.wms2025.com/file/90a8e78b-872e-4de1-b1a0-0a78ac8c810c/WMS2025%20Congress%20Full%20Programme%20and%20Appendix%20(13)%20.pdf https://www.wms2025.com/file/90a8e78b-872e-4de1-b1a0-0a78ac8c810c/WMS2025%20Congress%20Full%20Programme%20and%20Appendix%20(13)%20.pdf"
X Link 2025-10-02T22:14Z [---] followers, [---] engagements
"$SRPT another paper on Sirolimus see chart at bottom of paper. https://www.sciencedirect.com/science/article/pii/S0887899425002620 https://www.sciencedirect.com/science/article/pii/S0887899425002620"
X Link 2025-10-02T22:23Z [---] followers, [----] engagements
"@jeremyj0916 looking at this $OVID mention wowza"
X Link 2025-10-02T23:13Z [---] followers, [---] engagements
"$SRPT Financial experiment: elevidys = $375m in Q1 PMO = $237m in Q1. Stress Test: Elevidys = $1.5 bill annual x (1-80%)=$300m. PMO=$948m annual x (1-30%) = $664m. Stress Test Revenue = $964m. Lets say trades at 2x p/s multiple. $1.9b/98mill shares = $19.67"
X Link 2025-06-19T01:31Z [---] followers, [---] engagements
"@jeremyj0916 Yeah Im going to look at their financials tomorrow to get a gauge of some things (CFFI is one I want to see over quarters). Think just the science side is probably enough to play this one if it makes sense but Ill be active on here to discuss"
X Link 2025-07-05T05:22Z [---] followers, [--] engagements
"$SRPT still holding this puppy. Avg. of $14"
X Link 2025-08-13T21:41Z [---] followers, [---] engagements
"$GALT No stat sig. on primary endpoint in ITT pop. Stat sig. finding in Per-Protocol pop (but attrition bias) Post-hoc emphasis on U.S. subgroup (data dredging) Inverted dose-response curve (saying it's target-mediated drug disposition convenient post-hoc rationalization)"
X Link 2025-09-19T19:11Z [---] followers, [---] engagements
"$GALT belapectin (2mg/kg) more effective than (4mg/kg) unpredictable mechanism off-target effects at higher dose GALT says saturation of galectin-3 binding site at 2mg/kg. Usually saturation leads to plateau of efficacy not reduction"
X Link 2025-09-19T19:15Z [---] followers, [---] engagements
"$GALT Phase 2b GT-026 study: No stat sig. with 2mg/kg 8mg/kg on HVPG (portal pressure) However stat-sig with pre-specified subgroup with no esophageal varices at baseline 1/2 of study pop. 2mg/kg reduces varices (p0.02). Chased the p-value centered NAVIGATE around this"
X Link 2025-09-19T19:20Z [---] followers, [--] engagements
"$GALT In NAVIGATE trial design a patient in the placebo who takes GLP-1 agonist for diabetes and whose liver health improves is counted as a treatment failure. Confounding true effect of belapectin with established effects of standard care medication"
X Link 2025-09-19T19:25Z [---] followers, [---] engagements
"$GALT NAVIGATE primary endpoint in ITT population: 2mg vs. placebo p=0.552 4mg vs. placebo p=0.139 Per-Protocol Pivot (excludes patients w/ incomplete 18month treatment or no end of treatment endoscopy: Reduces patient pool by 20%"
X Link 2025-09-19T19:28Z [---] followers, [---] engagements
"$GALT In the PP pop. GALT only focuses on one component of original composite endpoint: Incidence of new varices. Finds stat sig. for 2mg/kg dose. PP analyses are considered supportive at best in the data package sent to FDA. Breaks randomization and introduces attrition bias"
X Link 2025-09-19T19:29Z [---] followers, [---] engagements
"$GALT They then present a post-hoc subgroup analysis of PP patients enrolled only in U.S. 2mg/kg then shows 68% relative reduction in new varices vs. placebo (p0.02). GALT then says this effect may be due to higher rate of GLP-1 in U.S. potential synergistic benefit"
X Link 2025-09-19T19:32Z [---] followers, [---] engagements
"$GALT To sum things up: GALT is a company of statistical maneuvering to find a positive signal in a dataset from a failed trial. If you analyze enough subgroups you will find a stat sig. result likely due to chance alone"
X Link 2025-09-19T19:34Z [---] followers, [---] engagements
"$GALT Would be required to conduct a whole new trial for that specific varices reduction hypothesis only in U.S. pop to validate results of NAVIGATE. Clear positive of GALT is that it has a clean safety and tolerability profile. Necessary but insufficient condition for approval"
X Link 2025-09-19T19:36Z [---] followers, [---] engagements
"$GALT Madrigal Pharmaceutical: Rezdiffra - MAESTRO NASH OUTCOMES trial if successful would expand label into same patient pop. Galectin is targeting. Akero Therapeutics: Efruxifermin - Good phase2b in Phase [--]. Outcompeting GALT like hell"
X Link 2025-09-19T19:40Z [---] followers, [---] engagements
"$GALT Key dates: November data readout at AASLD. 36-month cohort study of NAVIGATE. Truth of long-term efficacy. First half of 2026- End of Phase [--] meeting with FDA (is data sufficient for BLA or new Phase [--] required)"
X Link 2025-09-19T19:56Z [---] followers, [---] engagements
"$GALT They are going to be forced to do another trial. EMA Assessment Scenario 3: For a subgroup claim from a failed trial to be considered credible: [--]. Subgroup must have been defined before study began [--]. Strong biological plausibility of difference in subgroups. [--] onwards"
X Link 2025-09-19T23:41Z [---] followers, [---] engagements
"$GALT Company rests on the 2mg dose in a specific group: MASH cirrhosis patients with clinically sig. portal hypertension and without baseline varices. I wonder what the total addressable market is for this enough for a phase 3"
X Link 2025-09-24T23:38Z [---] followers, [---] engagements
"$AKRO $GALT $MDGL F-Stages of Fibrosis. Rezdiffra captured F2 and F3 (non-cirrhotic) pop. last year. OUTCOMES trial seeks to expand label into F4. Belapectin is targeting the F4 population (cirrhotic). Race to first in-class F4. But Efruxifermin hmm"
X Link 2025-09-24T23:52Z [---] followers, [---] engagements
"$AKRO $GALT Efruxifermin- phase 2b f4 pop stat sig. reversal of cirrhosis at [--] weeks (39% for 50mg 15% for placebo) Belapectin-is varices prevention endpoint a clinically superior outcome to potential reversal of cirrhosis I like Efruxifermin a lot more than belapectin"
X Link 2025-09-25T00:08Z [---] followers, [---] engagements
"$AKRO $GALT $MDGL roche MASH F4 Space: [--]. Efruxifermin-my leader in F4 histological data -cirrhosis reverse [--]. Pegozafermin competitor -cirrhosis reverse 1b2b. Rezdiffra- phase [--] OUTCOMES for F4 - liver stiff. reduction last. Belapectin-mixed signal data. -varices reduction"
X Link 2025-09-25T00:14Z [---] followers, [---] engagements
"$GALT assuming [--] month data presented AASLD Nov 7-11. See Oct. [--] if abstract is uploaded or Nov. [--] for later abstracts on AASLD (GALT likely press releases anyway). September presentation said FDA feedback on NAVIGATE results by EOY. Planning the trade now"
X Link 2025-10-02T20:10Z [---] followers, [---] engagements
"@jeremyj0916 you ever look at $ANAB"
X Link 2025-10-03T00:55Z [---] followers, [---] engagements
"research list. $LRMR like-ish $CAPR short $GALT short $SRPT long $AKRO like-ish"
X Link 2025-10-03T18:59Z [---] followers, [----] engagements
"$NRXP nrx-101 pdufa data: phase [--] (2023 end): phase2b/3 (2024 end): - still looking for the actual poster (W89: A Randomized Double-Blind Controlled Comparison of NRX-101 to Lurasidone.) https://www.biospace.com/nrx-pharmaceuticals-nasdaq-nrxp-presents-landmark-trial-of-nrx-101-in-suicidal-bipolar-depression-at-the-ascp-annual-meeting-nrx-101-is-the-first-oral-antidepressant-demonstrated-to-reduce-suicidality-in-bipolar https://pubmed.ncbi.nlm.nih.gov/37573534/"
X Link 2025-10-05T06:03Z [---] followers, [---] engagements
"$AVXL EMA decision eoy $GALT Data at AASLD Nov. 7-11 $CAPR HOPE-3 data eoy $NRXP PDUFA decision eoy"
X Link 2025-10-05T06:56Z [---] followers, [---] engagements
"i never use 2x levered daily etfs but $SRPU seems interesting. might add some before earning day bell close to lever up then sell next day market open vol drag shouldn't be anything for a overnight trade $SRPT"
X Link 2025-10-24T06:15Z [---] followers, [---] engagements
"nov. update longs: $SRPT $SPRB $PALI shorts: $AVXL $QBTS research: $BHVN $AVTX $INSM $LRMR $SLNO"
X Link 2025-11-03T07:59Z [---] followers, [---] engagements
"$SRPT on the earnings call rebought @ [--]. Back to this Q1"
X Link 2025-11-03T22:33Z [---] followers, [---] engagements
"$SRPT anyone have information on the breakdown of PMO revenue - AMONDYS [--] - VYONDYS [--] - EXONDYS [--] Would think majority of PMO revenue comes from EXONDYS [--] - largest single-skip amenable population"
X Link 2025-11-04T04:54Z [---] followers, [---] engagements
"$SRPT risk is on. -Consolidated Appropriations Act ('23) '25 FDA Guidance grant speedy withdrawal of accel. appr. products. -'23 Makena by Covis was withdrawn by CDER (indic: pre-term birth adcom voted 14-1 to withdraw) EXONDYS [--] imo is the last hope for PMO"
X Link 2025-11-04T04:57Z [---] followers, [---] engagements
"$SRPT EXONDYS [--] accel. app. was pushed by Dr. Woodcock CDER Director. -argued surrogate endpoint (microdystrophin release) was sufficient partly political (DMD lobbying/advocacy) -SRPT using real world evidence (RWE) as counter. -RWE has no precedent overturning Random PCT"
X Link 2025-11-04T05:00Z [---] followers, [---] engagements
"$SRPT if this fails (EXONDYS 51) no doubt all PMOs get pulled from the market: https://clinicaltrials.gov/study/NCT03992430 https://clinicaltrials.gov/study/NCT03992430"
X Link 2025-11-04T05:14Z [---] followers, [---] engagements
"$SRPT Sum of parts valuation (no PMO): ELEVIDYS only amb pop. - 500m annual EV (2.5p/s)=1.2b Debt: 860m cash - 1.1b debt : (-230)m cash siRNA pipe (ARWR): srpt committed 930m lets put this at 500m 1.25b+500m-235m=1.5b mc ($15/s) this is why i bought today. hope it works"
X Link 2025-11-04T06:16Z [---] followers, [---] engagements
"$SRPT other risks: RGNX lawsuit - over AAV platform of ELEVIDYS jury starts 11/17/25 - RGNX gets royalties if SPRT loss Solvency- dilution with cash run out (ARWR payments before data readouts for siRNA its dicey"
X Link 2025-11-04T06:21Z [---] followers, [---] engagements
"$BHVN $8.12 right now buying a little size. Honestly have not really deep dived into this but will do so"
X Link 2025-11-05T20:13Z [---] followers, [---] engagements
"$SPRB Ikarian 13G today Nov. [--] (date of event: Oct. 31) and Squadron Capital's in this. 132m MC gonna be a fun one"
X Link 2025-11-08T06:11Z [---] followers, [---] engagements
"ADCs to research: B7-H4: $NXTC and $MRSN HNSCC (Nectin-4 experimental EDB+FN): $CRBP and $PYXS HNSCC fits the rare-disease theme better I like"
X Link 2025-11-10T08:52Z [---] followers, [---] engagements
"$CAPR Sell Side Analyst: "Break a leg" CEO Linda: "Thanks we'll need it" q3 call is too funny"
X Link 2025-11-10T21:55Z [---] followers, [---] engagements
"godel watchlist 11/10/25 - $CAPR dec. puts - $GALT looking good to short soon - got out the train too early on $AVXL jan p - $SPRB moose doing its thing - quantum short finally working out - $BHVN and $SRPT both scare me but long for now - learning about ADCs (crbp nxtc pyxs)"
X Link 2025-11-11T05:02Z [---] followers, [----] engagements
"$LRMR i like this one tricky part besides anyphalaxis issue: precedent in FA: Omaveloxolone -functional clinical endpoint (mFARS) with hist. controls. Nomlabofusp -pharmacodynamic marker (skin FXN conc.) as RLSE correlation to clinical outcomes derived from hist. controls"
X Link 2025-11-12T07:49Z [---] followers, [---] engagements
"$LRMR better have some good data: boils down to consistency of the correlation between FXN increase and mFARS stabilization given the non-randomized historical control design (FACOMS)"
X Link 2025-11-12T07:59Z [---] followers, [---] engagements
"$CAPR wrote down my thoughts on the HOPE-3 data here. took both sides of the argument (PUL fragility vs. LVEF efficacy) https://docs.google.com/document/d/1qjZ5l1XJ9DhSmZ4_b9sMvQPkAXmEpiO6AiOuofnvcZY/editusp=sharing https://docs.google.com/document/d/1qjZ5l1XJ9DhSmZ4_b9sMvQPkAXmEpiO6AiOuofnvcZY/editusp=sharing"
X Link 2025-12-04T23:24Z [---] followers, 28.9K engagements
"@alan_sharp @yachmod $SRPT vs $SRPU's [--] vs [--] price delta is an arbitrary psychological anchor. the product of SRPU is a synthetic 200% daily return contract (capital structure reset nightly). use it don't own it. binary event - capture levered overnight move without the iv premium of a call"
X Link 2025-10-24T06:32Z [---] followers, [---] engagements
"January watchlist for me: $SPRB $LRMR $KTTA $SRPT writing a report on $KTTA and $LRMR $LPCN past month was interesting to see"
X Link 2026-01-08T07:05Z [---] followers, [---] engagements
"@BrainyMarsupial Interesting. Thanks for letting me know about that april presentation. My major concern was if they were starting manufacturing from scratch (like no TA-ERT given from Allievex since they ran out of supply). Obv there is still risk with producing rhNAGLU since its quite complex"
X Link 2026-02-06T03:22Z [---] followers, [--] engagements
"@BrainyMarsupial You mind sharing your thoughts on the markets pricing on SPRB right now. Near PIPE price seems brutal. I want to attribute the price to beta PRV drama and biotech being a bit of a shitter the past couple months. I mean our calculations make the current price a steal but hmm"
X Link 2026-02-06T03:24Z [---] followers, [--] engagements
"Have a Other bios im looking: business models i like (not bullish yet) Vertex Pharmaceuticals ($VRTX) Alnylam Pharmaceuticals ($ALNY) BioMarin Pharmaceutical ($BMRN) Krystal Biotech ($KRYS) Rocket Pharmaceuticals ($RCKT) Denali Therapeutics ($DNLI) https://www.barrons.com/articles/sarepta-stock-gene-therapy-treatment-148476abreflink=desktopwebshare_permalink https://www.barrons.com/articles/sarepta-stock-gene-therapy-treatment-148476abreflink=desktopwebshare_permalink"
X Link 2025-07-06T05:48Z [---] followers, [---] engagements
"if interested in $SRPT you need to be looking at Alnylam Pharmaceuticals Avidity Biosciences Dyne Therapeutics. siRNA. delivery to liver (LNP is good). delivery to skeletal muscle (AOCs look like gold standard target TfR1 for entry)"
X Link 2025-08-03T06:38Z [---] followers, [---] engagements
"@yanir_uliel The recent management hires boil down to bringing back individuals from BioMarin who worked during the enzyme replacement therapy time at BioMarin. Spruce management should file BLA this Q1 (assuming no delays)"
X Link 2026-02-10T08:24Z [---] followers, [---] engagements
"$ALDX For the June [----] resubmission (PDUFA March 16): Proof of primary efficacy lays in study [---] (chamber trial) But full CRS of the Field Trial (failed) must be included in NDA package per standard protocol as argued by FDA office of specialty med"
X Link 2026-02-11T05:16Z [---] followers, [---] engagements
"@StockChumpski $rgnx crl fda cited issues with use of cns-hs to predict clinical benefit. i assume rgnx's clinical function data was weak/used a [--] year time period. sprb does have [--] year data and uses a different type of cns-hs. Its still a massive signal that the FDA doubted cns-hs"
X Link 2026-02-09T21:45Z [---] followers, [---] engagements
"@BrainyMarsupial acquisition would be sweet of course im putting probability low given the confirmatory trial. my est. of raise needed for that trial is about 100million. EV rn without PRV is [--] million with PRV that goes to [---] mill. market rn pricing an equity raise likely after bla filed"
X Link 2026-02-05T21:26Z [---] followers, [---] engagements
"@BrainyMarsupial i have net asset value with PRV (@ $110M) BLA approval and a $75 million raise (1.25M shares at $60) at $89.3 per share. I have NPV at $731M then calculating FV I get $430 per share. So like 350-450 FV"
X Link 2026-02-06T00:22Z [---] followers, [---] engagements
"@BrainyMarsupial if i was a fund as well. i would wait for the raise to happen so they can size without slippage . spruce could also mess up the raise if they get desperate and do a 100m raise at $40 with warrants. i think spruce's sp has been more affected by beta over the past months"
X Link 2026-02-06T00:29Z [---] followers, [--] engagements
"$RGNX concerning news. RGNX used CNS-HS D2S6 as surrogate endpoint. RGNX used [--] month data. SPRB is using 5-6 year data. RGNX endpoint timing: [--] week endpoint SPRB endpoint: durable reduction RGNX follow-up [--] years SPRB follow-up [--] years"
X Link 2026-02-09T21:33Z [---] followers, [----] engagements
"@LQDA_Bull1 Dont think its too irrational. I understand selling off because of the similarities between RGNX and SPRB (nat. History comparison CNS-HS surr. endpoint exploratory endpoints for clinical benefit w/ no p-values)"
X Link 2026-02-09T23:32Z [---] followers, [---] engagements
"@LQDA_Bull1 Now you can make the argument for Spruce that RGNXs HS-D2S6 is not appropriate since its a specific disaccharide in the middle of the chain and can be broken off by other enzymes. While HS-NRE is more specific as NAGLUs only job is to cook the terminal sugar at NRE"
X Link 2026-02-09T23:35Z [---] followers, [---] engagements
"@LQDA_Bull1 Youd argue that NAGLU is missing so NRE tips cant be removed. This measuring NRE is a 1-to-1 measurement of the drug doing its job. With HS-D2S6 you can lower fragments without clearing the toxic tips that cause the neurodegeneration"
X Link 2026-02-09T23:36Z [---] followers, [---] engagements
"@LQDA_Bull1 You can also say that RGNX focused on percentage reduction (80%+ drop in HS-D2S6) so that only shows its less toxic. While Spruce focused on normalization (HS-NRE) so they brought it down to healthy range (Spruce would argue it must predict clinical benefit)"
X Link 2026-02-09T23:38Z [---] followers, [---] engagements
"$SPRB added. I get $70 when pricing in equity raise after BLA is announced. Appears to be a good price right now. I dont have doubts about the science. Small concerns about manufacturing. Major questions about how much they raise after BLA filiing"
X Link 2026-02-05T20:59Z [---] followers, [----] engagements
"$SPRB TA-ERT as of my understanding holds the bar for cognitive func. stabilization for an MPS drug"
X Link 2026-02-06T07:34Z [---] followers, [----] engagements
"$SPRB Superior on specificity compared to Denali's DNL-310. FDA's comments on DNL-310's use of Total CSF HS should be stock-moving dependent on what they say. CSF HS-NRE (nre=non-reducing end specific marker of NAGLU metabolic block)"
X Link 2026-02-06T07:38Z [---] followers, [----] engagements
"$SPRB So Spruce focuses on grey matter rather than NfL (DNL-310 has great NfL data). Spruce argues stabilized brain volume is a more direct correlate of clinical outcome than noisy pediatric NfL. Interesting. In [----] Tofersen for ALS was approved by FDA using NfL reduction"
X Link 2026-02-06T07:48Z [---] followers, [----] engagements
"$SPRB wrote down my thoughts on TA-ERT's outlook after today's CRL of RGX-121 $RGNX https://docs.google.com/document/d/1f-g25IKFoW9KKPcqtXA07KGr5r0uaUdTUHicJm1cH84/editusp=sharing https://docs.google.com/document/d/1f-g25IKFoW9KKPcqtXA07KGr5r0uaUdTUHicJm1cH84/editusp=sharing"
X Link 2026-02-10T04:31Z [---] followers, [----] engagements
"$SPRB If you study Brineura by BioMarin and the hurdles they had to go through for approval you would know those same BioMarin folks made sure TA-ERT was going to be robust enough for it's turn with the FDA gauntlet"
X Link 2026-02-10T06:14Z [---] followers, [----] engagements
"$SPRB Read BRINEURAs Summary Basis of Approval https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/761052Orig1s000SumR.pdf https://www.accessdata.fda.gov/drugsatfda_docs/nda/2017/761052Orig1s000SumR.pdf"
X Link 2026-02-10T22:37Z [---] followers, [----] engagements
"$ALDX Two CRLs for Reproxalap: Nov [----] April [----] Round 3: PDUFA March [--] -FDA may reach out Feb [--] to discuss labeling if no major deficiencies in latest NDA app. -Jun. [----] FDA agrees ALDX submits chamber trial -Dec.2025 FDA requires failed field trial CSR be submitted"
X Link 2026-02-11T05:13Z [---] followers, [---] engagements
"$SPRB adding on Monday. $IRON : PPIX had no validation as an endpoint. Weak correlation with clinical outcomes. Focused on reduction of PPIX. $RGNX : [--] year trial data Used HS-D2S6 (noisy). Focused on reduction. $SPRB : [--] year data HS-NRE Focused on Normalization"
X Link 2026-02-14T03:30Z [---] followers, [----] engagements
"$CAPR wrote down my thoughts on the HOPE-3 data here. took both sides of the argument (PUL fragility vs. LVEF efficacy) https://docs.google.com/document/d/1qjZ5l1XJ9DhSmZ4_b9sMvQPkAXmEpiO6AiOuofnvcZY/editusp=sharing https://docs.google.com/document/d/1qjZ5l1XJ9DhSmZ4_b9sMvQPkAXmEpiO6AiOuofnvcZY/editusp=sharing"
X Link 2025-12-04T23:24Z [---] followers, 28.9K engagements
"$SRPT Sarepta is reasonable. They halt to non-ambulatory (the accelerated approval) and refuse to stop the ambulatory (traditional approval). srp-9004 for LGMD is completely different. Vinay leaked this to media before formal communication this is political not scientific"
X Link 2025-07-19T00:07Z [---] followers, [----] engagements
"@iam_johnw Leading with the head"
X Link 2024-08-10T01:41Z [---] followers, [---] engagements
"The "Prasad Effect" is here. The FDA's CBER is dismantling the risk-benefit calculus for rare disease. The actions against $SRPT - the first major signal. My paper analyzes CBER why precedents like Zolgensma & Tysabri were ignored and what's next. https://docs.google.com/document/d/116VzYufxItdTTdx2CtYXX_kziODNN7nxrfyHCI4sDiA/editusp=sharing https://docs.google.com/document/d/116VzYufxItdTTdx2CtYXX_kziODNN7nxrfyHCI4sDiA/editusp=sharing"
X Link 2025-07-21T08:11Z [---] followers, [----] engagements
"$SRPT So. the entire FDA Newsroom should just be shutdown because the officials prefer playing telephone with journalists directly instead. Updated: In a follow-up conversation on the safety study the senior official said Sarepta could make a change to the dosing or manufacturing and test it on the next [--] or [--] boys and show the liver-related issues were improved Updated: In a follow-up conversation on the safety study the senior official said Sarepta could make a change to the dosing or manufacturing and test it on the next [--] or [--] boys and show the liver-related issues were improved"
X Link 2025-07-24T19:30Z [---] followers, [----] engagements
"Updated: In a follow-up conversation on the safety study the senior official said Sarepta could make a change to the dosing or manufacturing and test it on the next [--] or [--] boys and show the liver-related issues were improved"
X Link 2025-07-24T19:09Z 11.1K followers, 63.2K engagements
"This is the craziest timeline of events: $SRPT We wanted to know how much balls Vinay Prasad had how much balls Sarepta had we got a full on fight. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-statement-elevidys https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-provides-statement-elevidys"
X Link 2025-07-19T00:00Z [---] followers, [----] engagements
"$SRPT still holding this puppy. Avg. of $14"
X Link 2025-08-13T21:41Z [---] followers, [---] engagements
"$srpt time to take your wifes purse buy shares wait a year then pay her back double"
X Link 2025-07-18T03:55Z [---] followers, [----] engagements
"$srpt walking to wifeys purse right now. Plan initiated"
X Link 2025-07-18T17:40Z [---] followers, [---] engagements
"cash that. $SRPT @Sanctuary_Bio Yes interesting stuff. I bet Vinay Prasad is fired by EOY. @Sanctuary_Bio Yes interesting stuff. I bet Vinay Prasad is fired by EOY"
X Link 2025-07-30T03:23Z [---] followers, [---] engagements
"@Sanctuary_Bio Yes interesting stuff. I bet Vinay Prasad is fired by EOY"
X Link 2025-07-24T16:04Z [---] followers, [----] engagements
"$SRPT Zolgensma: liver toxicity - fda addresses using black box label and enhanced monitoring protocol. july 16: fda good faith gives black box label. srpt convenes for non-ambulant risk assessment. july 18: fda pulls plat desig requests halt"
X Link 2025-07-19T00:14Z [---] followers, [----] engagements
"$SRPT they really did it. https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-voluntary-pause-elevidys https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-voluntary-pause-elevidys"
X Link 2025-07-21T23:56Z [---] followers, [----] engagements
"WOW WOW We have been on this. $SRPT This is exactly what diligent research gives you an edge on. Let us see what is next. Will be doing more work tonight the thesis always continues. #Breaking: Vinay Prasad a top official at the Food and Drug Administration has suddenly departed after a series of controversial decisions about a treatment for boys with Duchenne muscular dystrophy. https://t.co/2rIEFT5Cvf #Breaking: Vinay Prasad a top official at the Food and Drug Administration has suddenly departed after a series of controversial decisions about a treatment for boys with Duchenne muscular"
X Link 2025-07-30T02:29Z [---] followers, [---] engagements
"#Breaking: Vinay Prasad a top official at the Food and Drug Administration has suddenly departed after a series of controversial decisions about a treatment for boys with Duchenne muscular dystrophy. https://trib.al/vfYBVjn https://trib.al/vfYBVjn"
X Link 2025-07-30T01:03Z 160.7K followers, 458.9K engagements
"$SRPT In the end I think SRPT is still the most intriguing company in the space. Agamree is still a steroid with lots of competitors. ELEVIDYS is $3 mill a customer. If Deramiocel works lets say priced at $1mill a customer annually $CAPR is intriguing"
X Link 2025-06-19T21:20Z [---] followers, [----] engagements
"@stocksandreales Easy 120% appreciate you"
X Link 2025-01-24T15:11Z [---] followers, [---] engagements
"@SarwanJohn Thank you brotha up 80% on the week. Loaded $HYZN and $XPON and a small $VEV for the weekend. See what happens with those"
X Link 2024-08-09T19:07Z [---] followers, [----] engagements
"$SRPT for bull: AAVrh74 SRP9003 EMERGENCE data tmrw shows good safety no acute liver toxicity. Market already hated this with ELEVIDYS. if good BLA goes through SRPT and FDA at least move forward on something for now"
X Link 2025-06-30T08:37Z [---] followers, [----] engagements
"$SRPT "investigating the death of an 8-year-old boy who received Elevidys". "The death occurred on June [--] 2025." this is not a 3rd elevidys kid right is it one of the two originals or is it a new death"
X Link 2025-07-25T21:52Z [---] followers, [----] engagements
"$SRPT CHEAT SHEET. AAVrh74 = the car. ELEVIDYS SRP-9003 = the payload. part of SRPT build on idea: the car is safe. FDA grants platform tech desig of AAVrh74. - market wants to know: if data good car is safe. big win. if data bad is the car (the foundation) to the junkyard"
X Link 2025-06-30T08:55Z [---] followers, [----] engagements
"$SRPT $CAPR Deramiocel sidesteps dystophen-restoration focuses on biggest clinical problem: heart failure. compelling value proposition. if Deramiocel works its added to baseline therapy (ELEVIDYS or Agamree this is the question)"
X Link 2025-06-19T18:32Z [---] followers, [----] engagements
"$SRPT stratification has to matter to the fda. non-amb: generally.pre compromised liver (underlying hepatic steatosis) compromised cardiac output impairs systemic clearance of aav vector lack a physiological reserve. sarepta's refusal is smart"
X Link 2025-07-19T07:59Z [---] followers, [---] engagements
"$SRPT played out exactly wait Monday. $SRPT assuming black box label is coming read about Zolgensma gene therapy for SMA. Dont think the FDA pulls Elevidys now just based on patient need for DMD. One more patient death though - game over Label pulled The risk profile of gene therapies man. $SRPT assuming black box label is coming read about Zolgensma gene therapy for SMA. Dont think the FDA pulls Elevidys now just based on patient need for DMD. One more patient death though - game over Label pulled The risk profile of gene therapies man"
X Link 2025-07-20T07:34Z [---] followers, [---] engagements
"$SRPT assuming black box label is coming read about Zolgensma gene therapy for SMA. Dont think the FDA pulls Elevidys now just based on patient need for DMD. One more patient death though - game over Label pulled The risk profile of gene therapies man"
X Link 2025-06-16T09:20Z [---] followers, [----] engagements
"$srpt elevidys competitors: AAV: Regenxbio: phase 1/2 AFFINITY DUCHENNE trial. BLA mid '26. Solid Biosci:sgt-003 phase1/2. Antisense oligonucleotides: - sort of the challenge to srpt PMOs Dyne-251 phase1/2 other: capr deramiocel (lol) topline q3 [--] remmber CRL lol"
X Link 2025-07-19T07:44Z [---] followers, [---] engagements
"$SRPT July [--] read this pls. bridgebio/mlbio give readout on BBP-418 in LGMD 2i/R9 (oral) spr-9003 by sarepta for LGMD 2E/R4 (aav-gene therapy) AskBio AB-1003 for LGMD 2i/R9 (gene therapy) atamyo therapeutics for LGMD 2C/R5 (gene therapy) - atamyo still early stage"
X Link 2025-06-30T09:25Z [---] followers, [---] engagements
"@stockplaymaker1 Its fake"
X Link 2024-08-20T19:22Z [---] followers, [----] engagements
"@StockSavvyShay NVDA is not a hardware company 🤣🤣🤣🤣🤣. Holy smokes"
X Link 2025-06-09T13:33Z [---] followers, [--] engagements
"$SRPT LGMD2E/R4 is very rare. [----] Bayesian Analysis: [---] per million people. But complete lack of approved modifying treatment. EMERGENE trial: ambulatory + non-amb Jun 2025-FDA grants Pltform tech desig. to AAVrh74 vector. expedite reviews of AAVrh74 products"
X Link 2025-06-30T08:18Z [---] followers, [----] engagements
"$SRPT even if ELEVIDYS revenue collapses by 80% and core PMO takes a hit of 30% the current share price is basically that ($20 on 06-18-2025). Basically market is saying ELEVIDYS is a [--]. Also pricing in potential lawsuits funding ENVISION brand stigma. Peak pessimism"
X Link 2025-06-19T01:36Z [---] followers, [---] engagements
"Agenda for the weekend for me: $SRPT Elevidys + other pipeline stuff $CAPR hmm $AVXL scam Not sure what else trying to get nice with the biotech. The big $INMB data release on Monday should be a fun watch"
X Link 2025-06-28T03:42Z [---] followers, [---] engagements
"$SRPT That anonymous FDA official: I dont know how you can approve a product that maybe shouldnt ever come to market in the first place and is killing people. That is a very tough proposition to keep it on the market the official said. - https://endpoints.news/sarepta-would-have-to-conduct-new-studies-to-get-back-on-market-fda-official-says/ https://endpoints.news/sarepta-would-have-to-conduct-new-studies-to-get-back-on-market-fda-official-says/"
X Link 2025-07-24T16:13Z [---] followers, [---] engagements
"Times like this where you think: Twitter is the Bloomberg terminal news feed for retailers. Ahh democratization of education via tech. Good times to live in"
X Link 2025-07-24T19:35Z [---] followers, [---] engagements
"@xshakeshake if im srpt ceo hell yes hopefully. Hard to tell if Prasad is strongarming or legitimately is going to go through the process of seeing if elevidys can resume shipments in amb group at least"
X Link 2025-07-22T01:06Z [---] followers, [---] engagements
"Well this manifested back from [--] weeks ago. FDA press release after market close today $SRPT major risk for srpt is this: AAV-mediated hepatotoxicity is not specific to ELEVIDYS. if safety data bad with SRP-9003 its the AAVrh74 platform whole pipeline is down bad. mstanley: project +1.2bill rev/yr from LGMD by [----]. $SRPT major risk for srpt is this: AAV-mediated hepatotoxicity is not specific to ELEVIDYS. if safety data bad with SRP-9003 its the AAVrh74 platform whole pipeline is down bad. mstanley: project +1.2bill rev/yr from LGMD by 2030"
X Link 2025-07-18T18:25Z [---] followers, [---] engagements
"$SRPT major risk for srpt is this: AAV-mediated hepatotoxicity is not specific to ELEVIDYS. if safety data bad with SRP-9003 its the AAVrh74 platform whole pipeline is down bad. mstanley: project +1.2bill rev/yr from LGMD by 2030"
X Link 2025-06-30T08:35Z [---] followers, [---] engagements
"if interested in $SRPT you need to be looking at Alnylam Pharmaceuticals Avidity Biosciences Dyne Therapeutics. siRNA. delivery to liver (LNP is good). delivery to skeletal muscle (AOCs look like gold standard target TfR1 for entry)"
X Link 2025-08-03T06:38Z [---] followers, [---] engagements
"$SRPT leaves me to think. draw down in stock not only bc of ELEVIDYS deaths. AAVrh74 failure/caution/success is priced in to what extent how much lower can SRPT go if EMERGENCE bad tmrw the pipeline is at stake right here. this is the fear. this is the price"
X Link 2025-06-30T08:39Z [---] followers, [---] engagements
"@StockSavvyShay Quantum will scale monte Carlo simulations Quantum will boost drug discovery The kicker 90% of the publicly traded quantum stocks are duds. The bull aint bulling. This guy Shay is an idiot"
X Link 2025-06-09T13:24Z [---] followers, [---] engagements
"Have a Other bios im looking: business models i like (not bullish yet) Vertex Pharmaceuticals ($VRTX) Alnylam Pharmaceuticals ($ALNY) BioMarin Pharmaceutical ($BMRN) Krystal Biotech ($KRYS) Rocket Pharmaceuticals ($RCKT) Denali Therapeutics ($DNLI) https://www.barrons.com/articles/sarepta-stock-gene-therapy-treatment-148476abreflink=desktopwebshare_permalink https://www.barrons.com/articles/sarepta-stock-gene-therapy-treatment-148476abreflink=desktopwebshare_permalink"
X Link 2025-07-06T05:48Z [---] followers, [---] engagements
"@bradloncar @Roche @IAmBiotech I think Vinay needs to reform his biologics program by Monday"
X Link 2025-07-19T18:29Z [---] followers, [----] engagements
"$srpt u might be thinking why even talk about AAV anymore if they pivot to siRNA (siRNA is the long game). AAV (the near term game) is still the money engine that would fund siRNA and [----] debt. and dont forget srp-9003 (aavrh74) later this year for LGMD"
X Link 2025-08-03T07:20Z [---] followers, [---] engagements
"$SRPT okay thats one step done. https://www.fda.gov/news-events/press-announcements/fda-recommends-removal-voluntary-hold-elevidys-ambulatory-patients https://www.fda.gov/news-events/press-announcements/fda-recommends-removal-voluntary-hold-elevidys-ambulatory-patients"
X Link 2025-07-28T20:52Z [---] followers, [---] engagements
"$srpt pt. [--] srpt competitors: LNPs - non viral gene delivery - this is future future dyne-251: they want bla for accelerated approval early '26. challenge srpt EXONDYS 51"
X Link 2025-07-19T07:48Z [---] followers, [---] engagements
"nothing to do with political affiliation all to do with landscape. the class of the useless credentialed is on the rise. always believed to use LLMs as your personal associate/analyst. Work rate goes up 10x if you use it like a project manager. you all know this. A partner at a prominent law firm told me AI is now doing work that used to be done by 1st to 3rd year associates. AI can generate a motion in an hour that might take an associate a week. And the work is better. Someone should tell the folks applying to law school right now. A partner at a prominent law firm told me AI is now doing"
X Link 2025-07-27T05:17Z [---] followers, [---] engagements
"A partner at a prominent law firm told me AI is now doing work that used to be done by 1st to 3rd year associates. AI can generate a motion in an hour that might take an associate a week. And the work is better. Someone should tell the folks applying to law school right now"
X Link 2025-07-26T17:31Z 1.8M followers, 7.1M engagements
"$SRPT am I right in saying that capricore is not a threat to sarepta at all If deramiocel is for dmd-associated cardiomyopathy would that not just turn DMD treatment into a combination of ELEVIDYS for baseline therapy and deramiocel"
X Link 2025-06-19T00:12Z [---] followers, [---] engagements
"@stockplaymaker1 $tnxp beautiful"
X Link 2024-08-21T16:15Z [---] followers, [---] engagements
"$srpt TYSABRI (biogen/elan): feb [--] 2005- withdrawn from market - BIIB [--] to [--] ELN [--] to [--] march [--] [----] - fda advis. comm. recommends return to market. - BIIB 46to [--]. ELN [--] to [--]. srpt situation is a hybrid of BIIB (deep piplin) and ELN (tys was tied to future)"
X Link 2025-07-19T08:37Z [---] followers, [---] engagements
"waiting to see an opportunity for puts on $ATYR and $SLNO and $QBTS and maybe $CAPR not doing anything now. will wait. if nothing im cash"
X Link 2025-08-17T08:01Z [---] followers, [---] engagements
"$STSS ouch. Sneaky warrant conversion in the offering. pray for SC plant pr or 💣"
X Link 2025-02-03T14:49Z [---] followers, [---] engagements
"$SRPT SRP-9003 program: phase1/2 enroll [--] patients. efficacy was good. safety was avg. transient elev. in g-glutamyl-trnsfase. one serious adverse evnt: acute liver injury needed hospitaliz resolved with standard care. phase [--] enrolled 15-17 patients"
X Link 2025-06-30T08:24Z [---] followers, [---] engagements
"$SRPT Forward Timeline on Elevidys [--]. Enhanced Safety Protocol Submission 2a. FDA Feedback 2b. Status of ENVISION confirmatory trial 2c. Status of commercial halt on non-ambulatory Wildcards: - another serious adverse event (SAE) - international regulatory decision (EMA)"
X Link 2025-06-19T07:40Z [---] followers, [---] engagements
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