@Prof_Oak_ Professor Oak

Professor Oak posts on X about $xbi, $lly, $regn, $bbio the most. They currently have [------] followers and [---] posts still getting attention that total [-------] engagements in the last [--] hours.

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Social Influence

Social category influence stocks cryptocurrencies finance technology brands currencies countries social networks exchanges celebrities vc firms

Social topic influence $xbi #33, $lly #105, $regn, $bbio #20, $srpt, events, $alny, $nvs, $verv, $bbc

Top accounts mentioned or mentioned by @jacobplieth @endpts @acquiredfm @luxcapital @skathire @andrewedunn @plainyogurt21 @snicobio @statnews @nicoledefeudis @drsam @artofinvest @danahercorp @learnbiotech @nellomainolfi @kymeratx @biotechtv @vervetx @elilillyandco @adeangelisbio

Top assets mentioned Eli Lilly and Company (LLY) Regeneron Pharmaceuticals Inc (REGN) BridgeBio Pharma, Inc. Common Stock (BBIO) Sarepta Therapeutics, Inc. (SRPT) Alnylam Pharmaceuticals, Inc. (ALNY) Novartis AG (NVS) Verve Therapeutics Inc. (VERV) Bristol-Myers Squibb Co (BMY) Synthetify (SNY) CRISPR Therapeutics AG (CRSP) Pfizer, Inc. (PFE) BEAM (BEAM) Johnson & Johnson (JNJ) Ionis Pharmaceuticals, Inc. Common Stock (IONS) Intellia Therapeutics, Inc (NTLA) Vertex Protocol (VRTX) Amgen, Inc. (AMGN) BioMarin Pharmaceutical, Inc. (BMRN) Avidity Biosciences, Inc. (RNA) AstraZeneca PLC (AZN) Biogen Inc (BIIB) Biohaven Pharmaceutical Holding Company Ltd. (BHVN) Gilead Sciences, Inc. (GILD) MoonLake Immunotherapeutics Class A Ordinary Shares (MLTX) Agios Pharmaceuticals, Inc. (AGIO) Prime Medicine, Inc. (PRME) Taysha Gene Therapies, Inc. (TSHA) Arrowhead Research Corporation (ARWR) Dyne Therapeutics, Inc. (DYN) Akouos, Inc. (AKUS) Legend Biotech Corp (LEGN) Crinetics Pharmaceuticals, Inc. (CRNX) GSK plc (GSK) Cullinan Management, Inc. (CGEM) Kymera Therapeutics, Inc. (KYMR) Bluefin (BLUE) Roivant Sciences Ltd. Common Shares (ROIV) Novo-Nordisk (NVO) Apogee Therapeutics, Inc. (APGE) Denali Therapeutics Inc. Common Stock (DNLI) Decibel Therapeutics, Inc. (DBTX) PTC Therapeutics, Inc. (PTCT) Cytokinetics Inc. (CYTK) Liquity BOLD (BOLD) Zenas BioPharma, Inc. (ZBIO) uniQure N.V. (QURE) 4D Molecular Therapeutics Inc. (FDMT) Karuna Therapeutics, Inc. Common Stock (KRTX) Reata Pharmaceuticals, Inc. Class A Common Stock (RETA) Amylyx Pharmaceuticals, Inc. (AMLX) AbbVie Inc (ABBV) Merck & Co., Inc. (MRK) Insmed, Inc. (INSM) Oruka Therapeutics, Inc. Common Stock (ORKA) Viridian Therapeutics, Inc. Common Stock (VRDN) Wave Life Sciences Ltd. Ordinary Shares (WVE)

Top Social Posts

Top posts by engagements in the last [--] hours

"On cue Barrons highlighting the burden of size & illiquidity that ARK is facing. ARK O/S is 10% in [--] cos. This has been reflected in recent buying patterns as $ARKG has been consistent and disproportionately allocating to big pharma $NVS $BMY $RHBBY https://www.barrons.com/articles/ark-etfs-might-be-too-popular-for-their-own-good-51611234009 Fundamentally expect continued mo for bios in [----] - disruptive tech premium steady M&A scientific WsLs. Pullback will be healthy but violent. Retail self-reinforcement; see ARKK/G. Illiquidity will be ARK's challenge in a drawdown - will drag the space"
X Link 2021-01-21T14:12Z 11.1K followers, [--] engagements

"5 years later looks like $BMY $QURE have a real program. Recall this partnership was focused on CV disease - space is poised for momentum following $RCKT Danon data $FDMT Fabry 2H21 Tenaya likely 2H21 IPO and $BMRN / DiNAQOR moving through preclin"
X Link 2021-03-01T12:33Z [----] followers, [--] engagements

"@snicobio This to me is the biggest risk. Id argue there is less biology risk with $DYN. Skepticism is only increased by lack of disclosure around supportive in vivo functional measures like downstream splicing nuclear foci etc. Program is nearing IND but we havent seen any of that"
X Link 2021-03-08T15:38Z [----] followers, [--] engagements

"For the gene therapy buffs on here. Interesting experiment from $SRPT presented this morning at MDA. Compared immunosuppression protocols on anti-AAV titer response as well as potential for plasma exchange to enable re-dosing. Takeaways:"
X Link 2021-03-18T14:55Z [----] followers, [--] engagements

"Casdin is back to the well for SPAC III. $400M base deal size so a Sema4-esque target on the horizon $CMLTU"
X Link 2021-04-06T11:27Z 11.1K followers, [--] engagements

"$IONS ANGPTL3 ALT / AST and liver fat elevations appear most likely ASO/drug-related rather than target-driven No notable liver tox seen in $REGN evinacumab Phase [--] albeit in a slightly different patient population $VERV"
X Link 2021-11-24T12:15Z [----] followers, [--] engagements

"Notable from the $GILD ZUMA-7 ASH plenary presentation: 56% of SOC arm went on to receive 3L CAR-T and these patients were NOT censored in OS analysis. Despite this interim OS analysis still clearly favoring Axi Cel - would have trounced transplant if pts had been censored"
X Link 2021-12-12T19:59Z [----] followers, [--] engagements

"Guess that took care of itself $ALNY Surprised $ALNY holding up so well PM. $BBIO pbo performance has implications for APOLLO-B. In modern paradigm of easy Tc scans/early dx ATTRIBUTE pbo arm may be a better comp than ATTR-ACT Fear was acoramidis raising efficacy bar but pbo improvement can have the same effect Surprised $ALNY holding up so well PM. $BBIO pbo performance has implications for APOLLO-B. In modern paradigm of easy Tc scans/early dx ATTRIBUTE pbo arm may be a better comp than ATTR-ACT Fear was acoramidis raising efficacy bar but pbo improvement can have the same effect"
X Link 2021-12-27T14:38Z [----] followers, [--] engagements

"Looks like $REGN snuck a new $NTLA collaboration target into their #JPM22 presentation - GAA KI + replace approach for Pompe Disease"
X Link 2022-01-17T23:02Z [----] followers, [--] engagements

"Case study in why companies shouldnt press release IND submissions $DYN No communication from $DYN re: DYN-251 IND despite announced filing [--] days ago. $DNLI TREM2 hold this morning. Both leverage TfR1 active transport. $DYN next No communication from $DYN re: DYN-251 IND despite announced filing [--] days ago. $DNLI TREM2 hold this morning. Both leverage TfR1 active transport. $DYN next"
X Link 2022-01-18T13:05Z [----] followers, [--] engagements

"$BBIO blinded mortality analysis supports maintenance of 30M readout vs. extending trial. Supports view that ATTRIBUTE failure driven by conduct vs. BL pt severity Probably a bit of both - incremental pos. for $ALNY but APOLLO-B odds still not much better than a coin toss IMO"
X Link 2022-02-24T13:04Z [----] followers, [--] engagements

"FIH Ribitol data from $BBIO in LGMD2i demonstrated a 70% decline in CK in [--] pts dosed for [--] months with no SAEs. Further detail to be provided during the conference (3/7)"
X Link 2022-03-01T02:46Z 10.8K followers, [--] engagements

"First look at Nippon-Shinyaku's broader exon skipping efforts with FIH data for their Exon [--] skipping ASO. Head-to-head comparison of viltolarsen (NS' exon [--] skipper) vs. VYONDYS have always been tough and to date NS has failed to gain much if any market share vs. $SRPT (4/7)"
X Link 2022-03-01T02:46Z 10.8K followers, [--] engagements

"Gamma deltas stay 🔥 $IMTX $BMY Immatics and Bristol Myers Squibb Expand Strategic Alliance to Develop Gamma Delta Allogeneic Cell Therapy Programs $80M total upfront $700M milestones / program teens royalties"
X Link 2022-06-02T11:05Z [----] followers, [--] engagements

"122% premium and still lower than trading price as recently as September [----] $TPTX $BMY $BMY to buy $TPTX for [--] a share https://t.co/WmbYkOcEUN $BMY to buy $TPTX for [--] a share https://t.co/WmbYkOcEUN"
X Link 2022-06-03T11:08Z [----] followers, [--] engagements

"Alright folks you know what to do $ALNY Anupam might be the best contra in biotech. hate to see $KOD go like this https://t.co/3rXd9d5VCg Anupam might be the best contra in biotech. hate to see $KOD go like this https://t.co/3rXd9d5VCg"
X Link 2022-06-24T10:57Z [----] followers, [--] engagements

"Friendly reminder that 0.2-0.4mg sema achieved 6% weight loss at [--] weeks in its Phase [--] NASH trial It's a long expensive road if pemvidutide's primary claim to differentiation is freedom from dose titration $ALT"
X Link 2022-09-14T11:47Z [----] followers, [--] engagements

"$CRSP $VRTX anticipated completion of rolling BLA for exa cel in 1Q22 - slight delay from YE22 guidance but not as bad as some feared Interestingly avg. pt. follow up likely to be quite a bit shorter at time of submission than what FDA required of $BLUE lentiglobin in SCD"
X Link 2022-09-27T11:41Z [----] followers, [--] engagements

"Feng Zhang @Lux_Capital NewCo coming soon 3/ Last night we also revealedto a few Lux LPs our newest Lux family co soon unstealthed advancing biotech in 🤯historic way Feng Zhang (MIT/Broad) is making many of the most important advances in gene editing therapy diagnostics + deliverymaking programmable medicine real https://t.co/qkmqxv1Od9 3/ Last night we also revealedto a few Lux LPs our newest Lux family co soon unstealthed advancing biotech in 🤯historic way Feng Zhang (MIT/Broad) is making many of the most important advances in gene editing therapy diagnostics + deliverymaking programmable"
X Link 2022-09-30T16:53Z [----] followers, [--] engagements

"Mixed bag for $VYGR. $PFE exercises option for CNS but not cardiac. Cuts total potential deal milestones down to $300M from $600M $VYGR $PFE entered the agreement Oct. 1st [----] and announced the deal Oct. 6th [----]. Should hear about a decision in the next couple days either way. $VYGR $PFE entered the agreement Oct. 1st [----] and announced the deal Oct. 6th [----]. Should hear about a decision in the next couple days either way"
X Link 2022-10-04T11:06Z [----] followers, [--] engagements

"And just like that $AKUS off the board. $LLY to acquire $487M upfront + $123M CVR. 121% premium to 30D VWAP. 25% below [----] IPO price Slight but potentially important difference in age thresholds for $DBTX vs. $AKUS based on language in Otoferlin IND PRs $DBTX: 1st [--] pts 7yrs subsequent pts "as young as 2yrs" AND "infants younger than 2yrs" $AKUS: 1st [--] pts 7yrs subsequent pts "as young as 2yrs" ONLY Slight but potentially important difference in age thresholds for $DBTX vs. $AKUS based on language in Otoferlin IND PRs $DBTX: 1st [--] pts 7yrs subsequent pts "as young as 2yrs" AND "infants"
X Link 2022-10-18T10:57Z [----] followers, [--] engagements

"$IONS pipeline progression in 2022: - Vupanorsen (ANGPTL3) discontinued by $PFE - ION449 (PCSK9) discontinued by $AZN - Fesomersen (FXI) rights returned by $BAYRY And Spinraza ex-growth yet stock is up 37% YTD"
X Link 2022-11-04T11:16Z [----] followers, [--] engagements

"Great visual from @statnews. Of course industry allure has coincided with a decade-long bull market in biotech - will we see mean reversion in a recessionary / capital-constrained future"
X Link 2022-11-10T11:32Z [----] followers, [--] engagements

"Cost of scaling out vs. scaling up in AAV gene therapy exemplified by $SRPT manufacturing line item within R&D expense (i.e. not exon skipper COGS): YTD: $347M 2021: $384M 2020: $366M Total spend of $1.1B since filings began breaking out mfg independent of clinical expenses"
X Link 2022-11-28T21:20Z [----] followers, [--] engagements

"Best case outcome for Tmunity. Lacked a CEO and strategic direction following safety hiccups but still had manufacturing capacity and a set of intg widgets of which $GILD can make use Transaction surely at a fraction of the Series B post - one of a16zs big early investments"
X Link 2022-12-20T13:14Z [----] followers, [----] engagements

"First foray into HSC-tropic LNP from $MRNA disclosed via biorxiv this week. 20% transduction in NHP at undisclosed dose - similar to $NTLA and $BEAM. Broader biodistribution not described but manuscript highlights need for miRNA binding motifs in 3' UTR to endow HSC specificity In vivo HSC delivery remains a key theme. As the next highly vascularized organ BM likely lowest hanging fruit after the liver Work from Weissman lab (123 2147) interrogates LNP decoration with CD117 binding moieties to improve tropism but still early / still high dose (6/11) In vivo HSC delivery remains a key theme."
X Link 2022-12-21T19:04Z [----] followers, [----] engagements

"Clinical / Corporate $ETNB pegozafermin ENLIVEN Topline $ALT pemvi 24W Obesity Data $ALNY ALN-APP FIH Data $ROG TIGIT 2nd OS Interim $IMGN MIRASOL Topline $KRTX EMERGENT-3 Topline $VYGR $NVS Opt-In Decision $DTIL Allo CD19 Update"
X Link 2023-01-23T01:03Z [----] followers, 10.5K engagements

"Aera officially out of stealth with $193M from Arch GV and Lux. Strong $ALNY contingent. Akin Akinc named CEO John as Board Chair Feng Zhang @Lux_Capital NewCo coming soon Feng Zhang @Lux_Capital NewCo coming soon"
X Link 2023-02-16T11:44Z [----] followers, [----] engagements

"$GRPH 50% RIF discontinuing nula-cel development focusing on non-genotoxic conditioning and exploring strategic alternatives"
X Link 2023-02-22T21:07Z [----] followers, [----] engagements

"Three SCD gene editing programs discontinued / deprioritized in the last [--] hours $NTLA $SGMO $GRPH Nature is healing"
X Link 2023-02-23T13:02Z [----] followers, 24.8K engagements

"$ALT 10% weight loss at 24W 27% AE-related discontinuations at 2.4mg despite 4-week titration [--] nausea / vomiting SAE How does this compete with pharma Dont know the oral GLP-1 asset well but valuation not too demanding. $250M pro forma EV with runway through 36-52W Ph2 data in late [----]. $ALT 2x on an EV basis with an injectable that may or may not be differentiated Dont know the oral GLP-1 asset well but valuation not too demanding. $250M pro forma EV with runway through 36-52W Ph2 data in late [----]. $ALT 2x on an EV basis with an injectable that may or may not be differentiated"
X Link 2023-03-21T11:11Z [----] followers, 27.3K engagements

"Why does all this matter (1) This may be the most robust demonstration of activation with a CRISPR-based fusion protein construct to date. Activation could open up an entirely new pool of targets for editing modalities across genetic (e.g. haploinsufficiencies)"
X Link 2023-03-25T20:29Z [----] followers, [----] engagements

"Some Sunday musings on RT-PCR vs. ddPCR for exon skipping quantification in DMD which has been a consistent debate over the years Updates from $PEPG at MDA last week demonstrate the perils of RT-PCR and call into question PGN-EDO51's differentiation vs. $SRPT's SRP-5051 (1/10)"
X Link 2023-03-26T23:17Z [----] followers, 21.2K engagements

"Strong interest from pharma in Treg cell therapy for IBD $REGN / Sonoma today $BMY / Genti last year Stands in contrast to lead programs across CAR-Treg players which are generally focused on organ transplant T1D RA etc"
X Link 2023-03-28T12:10Z [----] followers, [----] engagements

"$CRSP / $VRTX rolling BLA submission guided to complete by end of 1Q23 - first-in-class position now a distinct possibility $BLUE - delays FDA submission of its lovo-cel gene therapy for sickle cell disease. Company is waiting for FDA feedback on full manufacturing data (comparability analysis) expected "within a matter of weeks." $BLUE - delays FDA submission of its lovo-cel gene therapy for sickle cell disease. Company is waiting for FDA feedback on full manufacturing data (comparability analysis) expected "within a matter of weeks.""
X Link 2023-03-29T11:11Z [----] followers, 22.2K engagements

"Remarkable to see $NVS which itself faced significant mfg challenges early in Kymriah's launch stepping in to help $LEGN $JNJ manufacture Carvykti Would love to know the financials; all redacted in 6-K Are there historical analogues from the early days of mAb/biologics mfg Novartis inks deal to help manufacture J&J and Legend's Carvykti @Nicole_DeFeudis - https://t.co/jmfPx9z3zL Novartis inks deal to help manufacture J&J and Legend's Carvykti @Nicole_DeFeudis - https://t.co/jmfPx9z3zL"
X Link 2023-04-14T20:06Z [----] followers, [----] engagements

"Interesting deal on both sides. Great to see $JNJ invest more heavily in CAR-T CBMG was previously Nasdaq listed and taken private in [----] followed by $120M Series A in [----] from a China-centric consortium Is an IPO the next step Running the $LEGN playbook taking the lead on CAR-Ts bit by bit Janssen $JNJ Enters Worldwide Collaboration and License Agreement with Cellular Biomedicine Group to Develop Next Generation CAR-T Therapies 245m$ upfront fro [--] CD20 CAR-Ts (1 CD20 & [--] CD20xCD19) https://t.co/IE6gwplyM7 taking the lead on CAR-Ts bit by bit Janssen $JNJ Enters Worldwide Collaboration and"
X Link 2023-05-02T12:31Z [----] followers, [----] engagements

"Notable resurgence in autologous CAR-T deal volume and magnitude as the viability of first-gen allogeneic approaches has increasingly come into question In the last [--] months: $JNJ / CBMG $GILD / $ACLX $GILD / Tmunity CARGO $200M Series A"
X Link 2023-05-02T12:40Z 10.4K followers, 13.7K engagements

"Short tenure at Laronde. Pablo now joining $INCY as Head of R&D Is the biotech industry so talent-starved that a CEO who just ran a company to zero immediately gets tapped for the chief role at a unicorn Is the biotech industry so talent-starved that a CEO who just ran a company to zero immediately gets tapped for the chief role at a unicorn"
X Link 2023-05-08T12:34Z [----] followers, 11.6K engagements

"One player in the second bucket striking a research collab with $JNJ. Decidedly few details disclosed #1: Delivery (2/2) - Big year for RNPs [--] [---] [---] [----] Ultrasound / Electroporation [--] [--] [---] [----] [----] mAb-Conjugated AAVs $REGN - [--] [---] $RGNX - [---] Kidney Tropism [---] [---] [----] [----] and of course a plethora of abstracts on extra-hepatic LNPs (4/26) #1: Delivery (2/2) - Big year for RNPs [--] [---] [---] [----] Ultrasound / Electroporation [--] [--] [---] [----] [----] mAb-Conjugated AAVs $REGN - [--] [---] $RGNX - [---] Kidney Tropism [---] [---] [----] [----] and of course a plethora of abstracts on extra-hepatic LNPs"
X Link 2023-05-15T20:49Z [----] followers, [----] engagements

"Biotwitter highly recommend Mikes series of newsletters on the corporate governance dynamics at $ILMN / Grail including a credible allegation of undisclosed insider enrichment Unique opportunity to learn from one of the best in corp gov through a case that hits close to home"
X Link 2023-05-25T10:18Z [----] followers, [--] engagements

"$TCRX launching an offering at $2.43 after closing at $4.58 two weeks ago following $AMGN deal Representative of the capital markets sophistication of far too many small cap bio teams"
X Link 2023-06-24T16:13Z [----] followers, [----] engagements

"$BOLT HER2-ISAC left for dead but seems to be a viable drug 29% mono ORR at RP2D responses across multiple tumor types reasonable tolerability Trading at 40% of cash"
X Link 2023-06-28T02:57Z [----] followers, 11.4K engagements

"Upcoming events and expected moves from Cowen. Should be an exciting June to wrap up Q2. Some confirmed events: $MLTX IL-17 Ph2 data $QURE & $PTCT HD data $ACET CD20 gdCAR-T update $FDMT CF functional data High-profile PDUFAs ($BMRN Roctavian $REGN HD Eylea $SRPT SRP-9001)"
X Link 2023-06-01T10:10Z [----] followers, [--] engagements

"Experimenting w/ Blue. Looks like editing the 1st tweet in a thread deletes all subsequent tweets. Other "1H / Mid-23 Events": $STOK 45mg Dravet $ROIV TL1a Maintenance $AMGN TSLP Ph2 CSU $RARE Setrusumab Ph2 $FIXX PKU Ph1/2 $AGIO SCD Go/No-Go $VTYX TKY2 ER Formulation"
X Link 2023-06-01T10:56Z [----] followers, [----] engagements

"$AUTL Obe Cel vs $GILD Tecartus pivotal Adult ALL data H2H: CRi: 71% vs 65% (both 97% MRD-) mDOR: NR (61% @ 9.5M) vs 13.6M Gr3+ CRS: 3% vs 26% Gr3+ ICANS: 7% vs 35% Should be interesting to see how much safety matters commercially vs. entrenched incumbent w/ broader label & multiple products"
X Link 2023-06-02T11:28Z [----] followers, 19.5K engagements

"Nice head-to-head comp vs. Tecartus here including real-world data from ROCCO. Obe Cel durability to be updated at ASH $AUTL $GILD $AUTL Obe Cel vs $GILD Tecartus pivotal Adult ALL data H2H: CRi: 71% vs 65% (both 97% MRD-) mDOR: NR (61% @ 9.5M) vs 13.6M Gr3+ CRS: 3% vs 26% Gr3+ ICANS: 7% vs 35% Should be interesting to see how much safety matters commercially vs. entrenched incumbent w/ broader label & $AUTL Obe Cel vs $GILD Tecartus pivotal Adult ALL data H2H: CRi: 71% vs 65% (both 97% MRD-) mDOR: NR (61% @ 9.5M) vs 13.6M Gr3+ CRS: 3% vs 26% Gr3+ ICANS: 7% vs 35% Should be interesting to see"
X Link 2023-06-02T20:22Z [----] followers, [----] engagements

"Fascinating biology. Spontaneous ATTR-CM regression and cardiac remodeling driven by polyclonal anti-amyloid IgG response. Seen in [--] pts of [----] cases Cool real world proof of mechanism for anti-TTR mAbs $AZN $NVO $PRTA The investigators describe antibodies that bound amyloid in three patients with transthyretin amyloidosis. Spontaneous regression of cardiomyopathy occurred in all three patients. https://t.co/yLYFJcV1OR https://t.co/hdBoLUIeTk The investigators describe antibodies that bound amyloid in three patients with transthyretin amyloidosis. Spontaneous regression of cardiomyopathy"
X Link 2023-06-07T22:29Z 10.2K followers, [----] engagements

"Interesting to see FDA grant $CRSP / $VRTX different review timelines for SCD and B-Thal (reflecting ZYNTEGLO availability) PDUFAs: SCD - 12/8/23 B-Thal - 3/30/24 $CRSP / $VRTX rolling BLA submission guided to complete by end of 1Q23 - first-in-class position now a distinct possibility $CRSP / $VRTX rolling BLA submission guided to complete by end of 1Q23 - first-in-class position now a distinct possibility"
X Link 2023-06-08T23:31Z [----] followers, 14.2K engagements

"$LLY making a big bet on Lp(a). [--] modalities in the pipeline now - small molecule siRNA and gene editing Congrats @skathire $VERV"
X Link 2023-06-15T10:55Z [----] followers, [--] engagements

"$LLY buying $SGTX for less than cash upfront 🤌🏻"
X Link 2023-06-29T10:56Z [----] followers, [----] engagements

"Everyone is excited about the premium & CVR but lets not forget under the terms of $LLY / $SGTX existing T1D collab $SGTX entitled to $410M in milestones and up to double digit royalties Even in the context of success (unlikely IMO) $LLY paying less than they would have"
X Link 2023-06-29T11:37Z [--] followers, [----] engagements

"Clinical/Corporate (1/2) $ARGX CIDP Ph3 $BBIO ATTR-CM Ph3 $NVO GLP-1 SELECT-CVOT $RHBBY TIGIT Ph3 Final OS $CRNX Acromegaly PATHFINDR-1 Ph3 $CBAY PBC Ph3 $SNDX KMT2A Ph2b / cGVHD Ph2 $PLRX PSC Ph2 $AMGN TSLP CSU Ph2b"
X Link 2023-07-05T10:20Z 11.1K followers, [----] engagements

"Regulatory $NTLA ATTR-CM IND / Ph3 Details $INZY ENPP1 Reg Update $KRTX NDA Sub / Acc $FOLD AT-GAA Review Decision $BIIB Leqembi Full Approval PDUFA (7/6) $SAGE / $BIIB Zuranalone PDUFA (8/5) $ISEE Zimura PDUFA (8/19) $SWTX Nirogacestat PDUFA (8/27) $ALNY ATTR-CM AdComm (9/13)"
X Link 2023-07-05T10:20Z [----] followers, [----] engagements

"Early launches to track start forms reimbursement progress etc: $SRPT ELEVIDYS $KRYS VYJUVEK $RETA SKYCLARYS $BMRN ROCTAVIAN $IONS QALSODY $BPMC ISM"
X Link 2023-07-05T10:20Z [----] followers, [----] engagements

"$PFE reconsidering allo CAR-T"
X Link 2023-07-06T12:35Z [----] followers, [--] engagements

"$RGNX getting into the vectorized exon skipping game (IND 1H25) Big bet that exon skipped dystrophin will differentiate from micro-dystrophin clinically which may be true but hard to make the ROI compelling on an exon-by-exon incident population IMO"
X Link 2023-07-11T13:57Z [----] followers, [----] engagements

"$PFE spreading bets in MM $25M CellCentric investment (p300/CBPi) follows last week's $CRBU investment Elrantamab has consistently been highlighted as a top pipeline program for which PFE has laid out peak sales estimates are almost unanimously viewed as overly aggressive"
X Link 2023-07-11T17:20Z [----] followers, [----] engagements

"Surprised to see $ALNY partner Zilebisiran in light of prior comments - long been the core of messaged transition from rare to prevalent w/ wholly-owned assets. $ALNY often comps to Inclisiran yet economics pale in comparison What changed 2H23 KARDIA data should provide context"
X Link 2023-07-24T10:20Z 10.5K followers, 16.6K engagements

"Decision may have been informed by reg disc. and/or underwhelming Ph2 BP reduction Prev guidance ('21): BP reduction sufficient for approval before CVOT (a la LDL for inclisiran) Updated dev plan: CVOT primary Ph3 with readout pre-filing. Significantly extends timelines $ALNY Surprised to see $ALNY partner Zilebisiran in light of prior comments - long been the core of messaged transition from rare to prevalent w/ wholly-owned assets. $ALNY often comps to Inclisiran yet economics pale in comparison What changed 2H23 KARDIA data should provide https://t.co/Gu6uoprest Surprised to see $ALNY"
X Link 2023-07-24T12:08Z [----] followers, [----] engagements

"Perfect example of this in $FIXX. HMI-103 data check a lot of boxes: (1) Phe reductions of 99% & 49%; (2) ability to liberalize diet; (3) No SAEs and LFTs in normal range Yet Co is trading at 1/3rd of cash is cutting 87% of workforce and exploring strategic alternatives Public AAV gene therapy companies dying on the vine but $150M rounds still happening in private land https://t.co/hMZTrjLnJr Public AAV gene therapy companies dying on the vine but $150M rounds still happening in private land https://t.co/hMZTrjLnJr"
X Link 2023-07-27T20:16Z 11.1K followers, 19.8K engagements

"And great to see at least one of these getting rewarded $BIIB to acquire $RETA for $172.50/sh 59% premium to yesterdays close In a post-IRA world encouraging to see early Orphan launches consistently crushing expectations. First $AMLX then $RETA and now $ACAD all meaningfully beating on first quarter sales / launch metrics In a post-IRA world encouraging to see early Orphan launches consistently crushing expectations. First $AMLX then $RETA and now $ACAD all meaningfully beating on first quarter sales / launch metrics"
X Link 2023-07-28T11:11Z [----] followers, 10.6K engagements

"Another exit for Manmeet $BIIB $RETA Manmeet Soni $RETA COO / CFO and President has a pretty remarkable track record: $ALNY CFO (2017-19) - stock 2x 4x since $ARIA CFO (2016-17) - acquired by $TAK $PCYC CFO (2012-15) - acquired by $ABBV Even his original $RETA grants in the money in AH trading Manmeet Soni $RETA COO / CFO and President has a pretty remarkable track record: $ALNY CFO (2017-19) - stock 2x 4x since $ARIA CFO (2016-17) - acquired by $TAK $PCYC CFO (2012-15) - acquired by $ABBV Even his original $RETA grants in the money in AH trading"
X Link 2023-07-28T11:12Z [----] followers, 15.7K engagements

"Busy 2H for the CSU space $AMGN earnings on Thursday - will we get teze data"
X Link 2023-07-29T12:10Z [----] followers, 13.9K engagements

"Now do $BMRN [--] days b/t approval of $SRPT Elevidys and 1st infusion By comparison: -At least [--] months b/t approval of $BLUE Zynteglo (8/17/22) and 1st infusion (sometime b/t Mar [--] and May 9) -At least [--] months b/t approval of $BLUE Skysona (9/17/22) and 1st infusion (b/t Jan and Mar 29) [--] days b/t approval of $SRPT Elevidys and 1st infusion By comparison: -At least [--] months b/t approval of $BLUE Zynteglo (8/17/22) and 1st infusion (sometime b/t Mar [--] and May 9) -At least [--] months b/t approval of $BLUE Skysona (9/17/22) and 1st infusion (b/t Jan and Mar 29)"
X Link 2023-08-02T20:50Z [----] followers, [----] engagements

"$60M Series B extension from Bain and Gordon for Kyverna. CD19 CAR-T for Autoimmune remains hot"
X Link 2023-08-03T10:40Z [----] followers, [----] engagements

"Antisense / siRNA BD on a roll recently $ALNY / $AGIO collaborate on TMPRSS6 siRNA for Polycythemia Vera $IONS / $NVS expand Pelacarsen collaboration to include next-gen Lp(a) candidate"
X Link 2023-08-03T11:09Z [----] followers, 13.9K engagements

"No data but Teze CSU Ph2 noted as complete; data still guided for mid-23. COPD data 1H24 $AMGN"
X Link 2023-08-03T20:12Z [----] followers, [----] engagements

"$CRSP joins $VERV / $LLY in pursuing gene editing for Lp(a). CRISPR's 2nd in vivo candidate - guiding for clinical initiation 1H24"
X Link 2023-08-07T20:35Z [----] followers, [----] engagements

"And $DBTX follows less than a year later. $REGN to acquire for $109M upfront + $213M CVR Was looking forward to seeing Otoferlin data in [----]. Thats probably not the case anymore - hope these programs dont get lost in the bowels of big pharma And just like that $AKUS off the board. $LLY to acquire $487M upfront + $123M CVR. 121% premium to 30D VWAP. 25% below [----] IPO price And just like that $AKUS off the board. $LLY to acquire $487M upfront + $123M CVR. 121% premium to 30D VWAP. 25% below [----] IPO price"
X Link 2023-08-09T11:36Z [----] followers, [----] engagements

"$MRK initiates Phase [--] development program for Oral PCSK9i. 17K patients across [--] studies. Primary completion of HeFH trial expected in [----] $VERV"
X Link 2023-08-25T11:28Z [----] followers, [----] engagements

"$BMY going broad with CD19 CAR-T in Immunology. Lupus in the clinic with data in [----]. Adding cohorts in multiple additional indications (SSc Myositis) to ongoing Phase [--] and initiating a dedicated Phase [--] in MS (first exploration of CAR-T in this setting)"
X Link 2023-09-15T12:31Z [----] followers, [----] engagements

"Great case study on the power of big losses. Despite a 78% () batting avg. a few 200% losses (SGEN IMMU MDGL KITE) were a huge drag on performance of recs which annualized at 15.4% (gross) since [----] narrowly outperforming $NBI but underperforming $SPX We asked on our last show what is the track record of short sellers. Elliot Favus sent us his closed positions. Copied below with permission in multiple tweets. Shared without endorsement or comment https://t.co/Wu2YdyooCj We asked on our last show what is the track record of short sellers. Elliot Favus sent us his closed positions. Copied"
X Link 2023-10-01T13:13Z 11.7K followers, 25.4K engagements

"Add $PNT to the list $LLY to acquire $PNT for $1.4B $LLY capitalizing on momentum and staying active on the BD front $DICE $AKUS Emergence acquisitions $VERV Lp(a) deal Confo Pain deal Now Versanis Bima + incretin combo / co-formulation could be a very interesting way for $LLY to differentiate within obesity injectables $LLY capitalizing on momentum and staying active on the BD front $DICE $AKUS Emergence acquisitions $VERV Lp(a) deal Confo Pain deal Now Versanis Bima + incretin combo / co-formulation could be a very interesting way for $LLY to differentiate within obesity injectables"
X Link 2023-10-03T10:51Z [----] followers, [----] engagements

"$SRRK to Advance Antimyostatin Program to Develop Novel Therapies for Cardiometabolic Disorders Continued momentum in combos targeting lean mass loss with GLP-1s $LLY / Versanis $REGN outspoken at recent conf + internal programs"
X Link 2023-10-11T20:51Z [----] followers, 19.1K engagements

"$BHVN anti-myostatin not far behind Biohaven Presents Preclinical Data Demonstrating Taldefgrobep alfa Reduces Fat and Improves Lean Mass at The Obesity Society's Annual Meeting ObesityWeek Phase [--] obesity study with taldefgrobep planned for 2024"
X Link 2023-10-16T11:07Z [----] followers, [----] engagements

"Getting these out before too many of them happen. My Q4 events of interest Perhaps a few will buck the $XBI's gravitational pull toward zero and perhaps they won't but should be a fun end of the year regardless"
X Link 2023-10-20T00:16Z [----] followers, 18.8K engagements

"Late Clinical $SRPT EMBARK / $PFE Ph3 Interim $SRPT PPMO Pivotal Topline $CYTK Afi HCM Ph3 $ARGX PV / ITP Ph3s $VYTX TYK2 PsO Ph2 $PFE Danu Ph2 $ACRS RA Ph2b $ROIV TYK2/JAK1 Lupus Ph2b $EYPT Wet AMD Ph2 $COGT ISM Ph2 $AMGN TSLP CSU Ph2b $VIGL ALSP Ph2 Interim $CRNX Carcinoid Ph2"
X Link 2023-10-20T00:16Z [----] followers, [----] engagements

"Early Clinical $CLDX PN Ph1b $ALPN Ph1b Basket $BIIB $PRTA SubQ A-Betas $DYN DM1 / DMD Ph1/2 MAD $VERV PCSK9 FIH $SANA Hypoimmune FIH $SGMO TX200 FIH $PASG FTD-GRN FIH $BMRN HAE GTx FIH $PRME NHP Data"
X Link 2023-10-20T00:16Z [----] followers, [----] engagements

"$VERV $BEAM $LLY deal a win-win-win $BEAM economics are not extraordinary but extends runway and defrays opt-in associated spend $VERV gains an expert partner with the resources / infrastructure to optimize dev And of course clear implications for how data will look at AHA"
X Link 2023-10-31T10:32Z [----] followers, 10.2K engagements

"$AMGN TSLP fails in CSU Ph2b. Some suggestion of a signal in xolair-naive patients but thats a tougher path forward Busy 2H for the CSU space $AMGN earnings on Thursday - will we get teze data https://t.co/ykNAmW0Qqj Busy 2H for the CSU space $AMGN earnings on Thursday - will we get teze data https://t.co/ykNAmW0Qqj"
X Link 2023-10-31T12:41Z [----] followers, [----] engagements

"Awesome to see in vivo POC for BE. Congrats to all involved Recommend Sek's summary However data likely below Street expectations which were high after $LLY LDL red. similar to inclisiran. Gr3 ALT at 0.6mg/kg is problematic. Not deal-breakers but margin is so narrow in HeFH"
X Link 2023-11-12T21:20Z [----] followers, 15.4K engagements

"13Fs have many flaws but love seeing the rare wild trade Example: in 3Q EcoR1 sold all of $SRPT a 10% position pre-EMBARK and added $250M of $MRTX a 8% position pre- (some of) PRMT5 fervor / $BMY deal. Enormous moves for a multi-billion dollar fund in an illiquid tape"
X Link 2023-11-14T21:34Z [----] followers, 12.8K engagements

"Nice deal for $RNA. Rare that early-stage collaborations live through an M&A event. Hard to believe that $BMY would double down if the SAE / ongoing hold held any existential read through to the platform"
X Link 2023-11-28T13:08Z [----] followers, [----] engagements

"Nice scoop. Honestly David could have funded this Series A by himself"
X Link 2023-12-05T22:39Z [----] followers, [---] engagements

"Surprising and unsettling to see FTC challenging $SNY license agreement with Maze for their GYS1 inhibitor in Pompe Disease Early stage collabs in areas with TA synergy are critical for both pharma and biotech and most help drugs get to patients faster $XBI"
X Link 2023-12-11T21:23Z [--] followers, 10.4K engagements

"Other thoughts: Equally disappointing that $SNY opted to terminate. Legal battle may be low / neg ROI but decision will only embolden FTC FTC risk & termination contingency will increasingly be built into contracts - more friction & longer cycle times for collabs to get done Surprising and unsettling to see FTC challenging $SNY license agreement with Maze for their GYS1 inhibitor in Pompe Disease Early stage collabs in areas with TA synergy are critical for both pharma and biotech and most help drugs get to patients faster $XBI Surprising and unsettling to see FTC challenging $SNY license"
X Link 2023-12-12T01:09Z [--] followers, [----] engagements

"Provocative slide but I have yet to see a full AE table. EFS likely inclusive of graft failure or death only But how many pts had GvHD With typical haplo as much as 30% acute / 15% chronic. Mgmt of these pts needs to be factored in to overall cost (not to mention pt QOL)"
X Link 2023-12-12T19:27Z [----] followers, [----] engagements

"$VRTX / $EDIT non-exclusive license for Cas9 IP involved in CASGEVY $50M U/F + $50M contingent U/F + $10-40M annual license fee through [----] Important precedent for the broader gene editing space with relevance to $BEAM $PRME Tessera etc. etc"
X Link 2023-12-13T14:35Z [----] followers, 15.8K engagements

"These periods of volatility shift psychology and expectations potentially clearing the way for management / BOD to take strategic interest more seriously Who's next $CYTK hit $27 in October $ARGX hit 52W lows in the $330s $SRPT as low as $67 post-EMBARK"
X Link 2023-12-22T15:13Z [--] followers, [----] engagements

"Business case playing out. Makes a whole lot of sense for $CRL Would be the largest NHP facility in the US by far with capacity to on-shore supply of a meaningful chunk of the 30K+ imported NHPs per year A biotech company bought [---] acres outside of Houston to raise & sell [-----] research monkeys. The monkeys sell for $60k each They were $5k pre-Covid. Residents & PETA are fighting it hard but so is the company. $2.5B isn't chimp change What a wild business. https://t.co/0fb3TtVpe7 A biotech company bought [---] acres outside of Houston to raise & sell [-----] research monkeys. The monkeys sell"
X Link 2024-01-03T02:09Z [----] followers, [----] engagements

"Industry moving quickly on this new work linking GDF15 expression with hyperemesis gravidarum $NGM in discussions with FDA regarding tox package to support clinical investigation of their GFRAL blocking antibody which it had previously investigated in cancer / cachexia Nausea and vomiting in pregnancy linked to hormone from fetus https://t.co/ysQ1ZvMOi4 Nausea and vomiting in pregnancy linked to hormone from fetus https://t.co/ysQ1ZvMOi4"
X Link 2024-01-09T13:10Z [----] followers, [----] engagements

"Clinical / Corporate (2/2): $SPRB Ph3 CAH Ped Data $INZY ABCC6 Ph1/2 Update $RNA AOC1001 DM1 Ph1/2 Update $LRMR 50mg Ph2 FA Data $ROIV Brepo Uveitis Topline Data $TSHA Rett Ph1/2 Data Update $STOK Dravet Data Update $AKRO F2-F3 MASH 96W Data Update $PLRX 12W PSC Data"
X Link 2024-01-16T02:01Z [----] followers, [----] engagements

"Regulatory: $IOVA Lifileucel Melanoma PDUFA (2/24/24) $MIRM LIVMARLI PFIC PDUFA (3/13/24) $MDGL Resmetirom MASH PDUFA (3/14/24) $MRK Sotatercept PDUFA (3/16/24) $REGN Odronextamab DLBCL / FL PDUFA (3/31/24) $SRPT DMD Full Approval Filing Acceptance"
X Link 2024-01-16T02:01Z [----] followers, 10.7K engagements

"Hot hot hot $CGON CG Oncology Inc (CGON) priced20.0mm shares (upsized from 17.0mm) at$19.00($1 Above the $16-$18 range) and will debut on Thursday. #IPO $CGON CG Oncology Inc (CGON) priced20.0mm shares (upsized from 17.0mm) at$19.00($1 Above the $16-$18 range) and will debut on Thursday. #IPO $CGON"
X Link 2024-01-25T00:32Z [----] followers, [----] engagements

"Clinical / Corporate - Late-Stage $ALNY APOLLO-B (Late June / Early July) $INSM Ph3 ASPEN / TPIP PH-ILD Ph2 $AVTE Ph2b AV-101 PAH $ANNX GBS Ph3 Data $SNY Rilzabrutinib ITP Ph3 / Asthma Ph2 Data $AMGN TSLP COPD Ph3 Data $GSK Depemo Asthma Ph3 Data $ARWR APOC3 FCS Pivotal $MRNS Ph3 Interim RSE Data $CRNX ACTH Ph2 Initial Data $MGNX B7-H3 TAMARACK Update (ASCO) $LLY PD-1 Agonist RA Update (EULAR) $MIRM Volixibat PSC / PBC Blinded Interim $ARGX POTS Ph2 Topline"
X Link 2024-04-01T00:42Z [----] followers, [----] engagements

"Clinical / Corporate - Early-Stage $IMCR PRAME Ph1/2 Melanoma Update $RNA Initial AOC [----] FSHD Ph1/2 Data $AZN Ph1 Oral GLP Data $GPCR 12W Obesity Data $GLUE GSPT1 RP2D Data Update $WVE Ph1/2 HD Data $CRBU RP2D / Ph1 Data Update $LYEL ROR1 FIH Data $CGEM MICA/B Dose Escalation Data $ALLK Siglec-6 Ph1 HV Data $ELVN Initial CML POC Data $ARCT OTC / CF mRNA Data $CABA CD19 SLE / IIM Initial Data $KYTX CD19 Update (EULAR) $RARE GSD1a GTx Ph3 WD Ph1/2 Data $SANA T1D Initial Data $PTCT 12M Interim HD Data $EWTX BMD 12M / DMD 3M Data $BPMC CDK2 Combo Data $PSTX Allo MUC1 Update"
X Link 2024-04-01T00:42Z [----] followers, [----] engagements

"Regulatory $LEGN / $JNJ CARVYKTI 2-4L MM PDUFA (4/5) $DAWN Tovo pLGG (4/30) $ASND TransCon PTH (5/14) $ABEO Pz-Cel EB (5/25) $AMGN Tarlatamab (6/12) $SRPT ELEVYDIS Label Expansion (6/21) $ARGX VYVGART Hytrulo CIDP (6/21) $VRNA ensifentrine COPD (6/28) $RCKT KRESLADI LAD-1 (6/30)"
X Link 2024-04-01T00:42Z [----] followers, [----] engagements

"$1B in biotech funding announced this morning alone $XBI $350M Metsera Series $275M $ABIO / $ORKA PIPE $160M Obsidian Series C $132M Alterome Series B $128M Diagonal Series A $63M Neurosterix Series A"
X Link 2024-04-03T12:32Z 10.2K followers, [----] engagements

"Yet another one. $ALPN was a $7 stock a year ago. 9x to $VRTX takeout at $65 Fitting end to another amazing comeback story as $GILD acquires $CBAY $CBAY was a $2 stock in May [----]. 16x to takeout price of $32.50 in [--] years Fitting end to another amazing comeback story as $GILD acquires $CBAY $CBAY was a $2 stock in May [----]. 16x to takeout price of $32.50 in [--] years"
X Link 2024-04-10T20:07Z [----] followers, 13.5K engagements

"Wonderful to see $DAWN's OJEMDA reaching patients. Tovo is a beautiful drug dev story - an asset salvaged by prepared minds who saw opportunity where others didn't acted on it & executed flawlessly. Doesn't hurt that @drsam is one of the nicest guys around. Congrats to all"
X Link 2024-04-24T01:24Z [----] followers, [----] engagements

"Straight into my veins $DHR Our @artofinvest teacher today is Mitch Rales one of the most iconic business builders and philanthropists of our time. Having cofounded @DanaherCorp [--] years ago alongside his brother and best friend Steve the Rales boys have strung together a track record of compounding that https://t.co/d05NnRJoH4 Our @artofinvest teacher today is Mitch Rales one of the most iconic business builders and philanthropists of our time. Having cofounded @DanaherCorp [--] years ago alongside his brother and best friend Steve the Rales boys have strung together a track record of"
X Link 2024-04-29T11:47Z [----] followers, [----] engagements

"Based on continued dialogue with CDER $DNLI believes the division may be open to discussing an accelerated path Better late than never. Plenty of traditional modality RGD drugs that deserve to reach pts quickly; and in some cases like this one are the superior agent Remarkable that FDA seems open to AA pathway for $RGNX in MPSII despite $DNLI biomarker data looking superior and that drug moving into a 96-week 50-patient Phase 2/3 head-to-head vs. Elaprase In tandem with $SRPT shifting tides toward AAV gene therapy at FDA Remarkable that FDA seems open to AA pathway for $RGNX in MPSII"
X Link 2024-05-08T01:44Z 10K followers, 10.8K engagements

"Not sure I've ever seen a Co more willing to hit the ATM than $NTLA $565M raised via ATM since the start of [----] 25% of current market cap 1Q24: $58M 4Q23: $120M 3Q23: $16M 2Q23: $1.5M 1Q23: $1.5M 4Q22: $228M 3Q22: $62M 2Q22: $39M 1Q22: $39M"
X Link 2024-05-09T11:45Z [----] followers, [----] engagements

"Today's $RPRX deal for frexalimab piqued my interest in ImmuNext which is a great example of unconventional value creation in biotech Dartmouth spin-out; still in NH [--] yrs later. As far as I can tell only raised $1M ext. capital. Despite that inked p'ships w/ $SNY $JNJ $RHHBY $LLY over the yrs generating $170M despite all but frex failing. Today's deal with $525M from $RPRX looks like a functional exit $800M on $1M over [--] yrs - not bad"
X Link 2024-05-09T18:04Z [----] followers, [----] engagements

"Welcome signals of generalists getting reacquainted with biotech Leerink 13F analysis shows that generalist mutual funds within Fidelity and T Rowe meaningfully increased smid biotech exposure in 1Q24 $XBI"
X Link 2024-05-17T18:03Z [----] followers, [----] engagements

"China's presence was notable particularly in AAV GTx. Chinese PIs presented [--] clinical datasets spanning IRDs OTOF PKU etc. including some entirely novel targets. Continuation of a known theme but further highlights the relative ease / speed of early IST clin/dev in China"
X Link 2024-05-20T02:13Z [----] followers, [----] engagements

"BBB-penetrant capsids continue to impress. Robust translational data including 1st NHP data w/ tx transgene secreted protein quant. & PK/PD; also some new hot targets (e.g. STXBP1). Several programs hitting the clinic in '25 - like the set-up if target is chosen thoughtfully"
X Link 2024-05-20T02:13Z [----] followers, [----] engagements

"Interesting though not surprising to contrast these data w/ big pharma capsid engineering efforts - w/ the exception of $REGN mAb-AAVs (which are innovative) AAV engineering / directed evolution abstracts from $NVS $BIIB & $RHHBY demonstrate relatively incremental progress"
X Link 2024-05-20T02:13Z [----] followers, [----] engagements

"$REGN to their credit is also innovating in re-dosing. Nice abstract showing BCMAxCD3 + aFcRn could enable AAV re-dosing in mice. Early but such a regimen is far more translationally realistic than the Ig protease / tolerization MOAs that have been explored to date"
X Link 2024-05-20T02:13Z [----] followers, [----] engagements

"For example could driving deep IgA/IgM suppression with BCMAxCD3 induction and maintaining with BAFF/APRIL-targeting agents achieve superior outcomes in IgAN / Glomerulonephritic Disorders as a holistic treatment regimen vs. BAFF/APRIL agents alone (2/5)"
X Link 2024-05-27T12:41Z 10K followers, [---] engagements

"Clinical PK/PD data suggests this is a credible hypothesis $ALPN / $VRTX's Povetacicept has shown B-in-C proteinuria / eGFR data in IgAN attributed to its dual MOA driving deeper auto-ab depletion. Pove achieves 40-60% IgA/M reduction at 1M deepening to 70-80%+ at 6M (3/5)"
X Link 2024-05-27T12:41Z 10K followers, [----] engagements

"In contrast $JNJ's teclistamab (BCMAxCD3) consistently drives IgA/IgM to near-zero in 1-2M. Infections have been an issue for this class but rate/severity increase with duration of therapy suggesting transient treatment may be able to thread the needle on safety (4/5)"
X Link 2024-05-27T12:41Z [----] followers, [----] engagements

"$CRGX pricing a PIPE at an 18.5% discount to Fridays close ($17 vs. $20.86) despite a strong data update recently At that point why not just take the market risk and do a public offering Specialists increasingly obscuring natural price discovery"
X Link 2024-05-28T12:12Z [----] followers, [----] engagements

"Pretty remarkable to see CAR-T / TCEs and immune cell depletion dominating the headlines at EULAR this year. Few interesting tidbits from abstracts (1/5)"
X Link 2024-05-31T11:25Z [----] followers, 15.9K engagements

"First public pres (to my knowledge) from $RHBBY on their CD19 TCE in SLE including NHP data w/ subq / fractionated dosing Ph1 in SLE started recruiting Nov. [----] but only a [--] dose (D1 + D8) maximum study so likely of little consequence regarding an efficacy read (3/5)"
X Link 2024-05-31T11:25Z 11.9K followers, [----] engagements

"Antibody-Oligo conjugates continue to impress in FSHD $RNA data show for the first time convincing measures of DUX4 target engagement in humans $ARWR $DYN not far behind"
X Link 2024-06-12T11:56Z [----] followers, 12.9K engagements

"Tide continues to rise for $DNLI $RARE announces alignment with FDA and intention to file MPS IIIA gtx based on reductions in CSF HS as a surrogate for AA DNL126 MPS IIIA Ph1/2 biomarker data (i.e. CSF HS) by YE24 Based on continued dialogue with CDER $DNLI believes the division may be open to discussing an accelerated path Better late than never. Plenty of traditional modality RGD drugs that deserve to reach pts quickly; and in some cases like this one are the superior agent Based on continued dialogue with CDER $DNLI believes the division may be open to discussing an accelerated path Better"
X Link 2024-06-12T20:24Z 10K followers, [----] engagements

"Clinical (Q3) (2/9) $AMGN Uplinza MG Ph3 $SNY Tolebrutinib MS Ph3 $SGMO / $PFE Hemo A GTx Ph3 $JSPR Ph1b/2a CSU $BIIB Dapi Lupus Ph3 $RNAC BCMA CAR-T Ph2 gMG $VRDN TED Ph3s $LXEO FA Ph1/2 Update $APGE APG777 Ph1 PK $PEPG Ex51 Initial Ph2 $IMCR PRAME Ovarian"
X Link 2024-07-02T00:24Z 11.1K followers, [----] engagements

"Clinical (2H/Q4) (3/9) $FULC FSHD Ph3 $SRRK / $BHVN TGF-B SMA Ph3s $NVS Zolg IT Ph3 $AMGN OX40 AtD Ph3 $SNY / $REGN Dupi CSU/BP Ph3 $SNY rilza IgG4/wAIHA Ph3 $ARGX Myositis Ph2/3 $SNDX NPM1 Ph3 $RHBBY Gazyva LN Ph3 $ALNY ATTR-CM ESC $REGN FXI mAb Ph2 $AMGN '133 Ph2 $NVO CB1 Ph2"
X Link 2024-07-02T00:24Z 11.1K followers, [----] engagements

"Clinical (2H/Q4) (4/9) $WVE AATD RNA Editing FIH $BEAM SCD FIH $TNYA MYBPC3 FIH $LXEO PKP2 FIH $EWTX BMD Ph2 C-Myosin Ph1 $DYN DM1/DMD $RNA Ex44 DMD Ph1/2 $WVE Ex53 DMD Ph1/2 $SLDB uDys DMD FIH $NGNE Rett Ph1/2 $LRMR FA Update $DNLI MPSIIIA FIH $ANAB BTLA AtD Ph2b"
X Link 2024-07-02T00:24Z 11.1K followers, [----] engagements

"Clinical (2H/Q4) (5/9) $CABA gMG/SSc FIH $BMY CD19 SLE FIH $AUTL Obe-cel SLE FIH $SANA CD19 NHL/AID FIH $SANA T1D FIH $TCRX AML Ph1/2 Solid FIH $VOR CD33 CAR FIH Trem Cel Ph1/2 $GILD CD19/20 FIH $IMCR HIV FIH $ASND CNP/HGH Combo $SYRE a4B7 PK FIH $RHBBY CFB ASO GA Ph2"
X Link 2024-07-02T00:24Z 11.1K followers, [----] engagements

"Corporate / Regulatory (6/9) $BMRN R&D Day (Sep.) $LBPH Bexi DEE Reg. Path $RLYB C5 Dev Plan $CRSP New Liver Programs $ADAP afami cel PDUFA (8/4) $ASND PTH PDUFA (8/14) $SNDX Axi GvHD PDUFA (8/28) $SNDX Revu PDUFA (9/26) $AUTL Obe cel ALL PDUFA (11/16) $BBIO Acor PDUFA (11/29)"
X Link 2024-07-02T00:24Z [----] followers, [----] engagements

"1st clinical data for allo CAR-T in AID from Bioray Labs in China Too early for a read on LT durability but initial response resembles auto CAR-T I've speculated that 'immune reset' may obviate the need for durable persistence; these data support that hypothesis (1/6)"
X Link 2024-07-15T18:59Z [----] followers, 37.3K engagements

"Let's run through the details N=3 (1 Myositis [--] SSc) treated with CD19 CAR-T + multiplex KO of TRAC CIITA PD-1 and HLA-A/B though CAR+ / editing were heterogenous across cells All [--] pts. saw CAR expansion with B cell depletion maintained for 2M (2/6)"
X Link 2024-07-15T18:59Z [----] followers, [----] engagements

"Traditional benchmarks built on an aggregated basis suggest a linear step-down in share as entrants increase In reality though a bimodal distribution underlies each 'entrant'. Thus benchmarking to the average is likely to sig. overstate or understate actual share (2/4)"
X Link 2024-07-20T14:14Z [----] followers, [----] engagements

"@learnbiotech Thanks Link in the ALT text of the images but also here: https://www.lek.com/sites/default/files/PDFs/best-class-biopharma.pdf https://www.lek.com/sites/default/files/PDFs/best-class-biopharma.pdf"
X Link 2024-07-20T14:34Z 10K followers, [---] engagements

"Taf is now $PFE's 3rd highest selling drug - $2.5B in 1H24 (+68% YoY) and the consensus view is that they're not going to do everything they can to avoid a [----] patent expiry $BBIO"
X Link 2024-07-30T11:18Z [----] followers, 16.5K engagements

"True changing of the guard w/r/t to R&D priorities at $BIIB Hi-Bio deal & $IONS opt-out were signs but today's termination of $DNLI ATV:A-Beta collab leaves little doubt $BIIB paid $290M to exercise its option in April '23 just over a year ago - how quickly things change"
X Link 2024-08-01T13:15Z 10K followers, [----] engagements

"Synovial sarcoma is a small market and this will be a complex launch $ADAP guides to $400M peak sales across afami cel and lete cel with 70% GM Let's take that at face value. This would imply annual gross profit of $280M (2/6)"
X Link 2024-08-03T13:18Z [----] followers, [----] engagements

"$ASND SKYTROFA Q2 rev down 27% YoY Better hope YORVIPATH works out $ASND SKYTROFA growth decelerated pretty meaningfully in Q4 1Q: [---] - 2Q: [---] (+132%) - 3Q: [----] (+180%) - 4Q: [----] (+39%) Ophthalmology announced as third therapeutic area. Sustained release Lucentis. Yawn $ASND SKYTROFA growth decelerated pretty meaningfully in Q4 1Q: [---] - 2Q: [---] (+132%) - 3Q: [----] (+180%) - 4Q: [----] (+39%) Ophthalmology announced as third therapeutic area. Sustained release Lucentis. Yawn"
X Link 2024-09-03T21:37Z [----] followers, [----] engagements

"VTAMA deal makes sense for $ROIV but $OGN is a surprise Maintaining the dividend is 25% of CFO. Mkt thought a cut was possible as recently as Nov '23 (trading 10% yield) Taking on a high-burn asset in a competitive market with limited synergies seems like a head scratcher"
X Link 2024-09-18T13:02Z [----] followers, [----] engagements

"Interesting graphical depiction of the reversal of Cosentyx growth trends following its HS launch $UCB Bimzelx US launch 1H25 $MLTX Ph3 data mid-25"
X Link 2024-09-22T21:16Z 10.8K followers, 18.2K engagements

"Undoubtedly a big market but price is a big driver of $MLTX's $10-15B estimate Avg. price 2x that of PsO driven by higher dose and more frequent administration Wonder if increased competition will drive price compression in the mid-term"
X Link 2024-09-22T21:16Z [----] followers, [----] engagements

"$MLTX hasn't been an exemplar of transparent communication historically but have to appreciate the objective bar-setting for VELA"
X Link 2024-09-22T21:16Z [----] followers, [----] engagements

"What if its not a late breaker Where does $ANAB go if: - No $LLY PD-1 pump - BTLA at YE disappoints (like others) Friendly reminder that $ANAB has been comping BTLA to rocatinlimab - howd that go"
X Link 2024-09-26T00:30Z [----] followers, 13K engagements

"1st ACR abstract is a detailed depletion analysis of LN biopsies from [--] AID pts (5 CAR-T [--] Rituximab) All [--] showed complete serum B cell depletion Lymph Nodes tell a different story. RTX left residual CD19+/CD20+ cells while CAR-T achieved complete depletion (2/7)"
X Link 2024-10-09T01:29Z [----] followers, [----] engagements

"3rd abstract goes a step further comparing the molecular profile of CAR-T vs. RTX responders Clear differences in global transcriptome w/ CAR-T uniquely suppressing T1 IFN and immune cell trafficking & restoring IL-2 Better Hard to say but definitely different (5/7)"
X Link 2024-10-09T01:29Z 12K followers, [----] engagements

"Ive long believed this should translate to TCEs At the time this was informed largely by $REGN NHP data comparing depletion with CD20xCD3 to RTX Nice to see this observation confirmed by $CGEM (ACR abstract) and $XNCR (R&D Day) in the past few weeks (6/7)"
X Link 2024-10-09T01:29Z 12K followers, [----] engagements

"Wouldn't write off $NTLA in HAE Pbo-adj. response will deepen with F/U & in market binary responder outcomes will be preferred over consistent but incomplete efficacy. SOC is great & improving but 80% odds of one-time functional cure will be compelling for some incident pts"
X Link 2024-10-24T19:06Z 11.7K followers, [----] engagements

"Most Co's in the space especially privates don't have a shot at real drugs but there will be winners $BEAM AATD FIH data in [----] is the next major catalyst for the space - certainly has the commercial / market magnitude to be a big spark as we just saw with $WVE"
X Link 2024-10-24T19:06Z 11.7K followers, [----] engagements

"In the wake of $PFE OXBRYTA withdrawal oral HbF induction MOAs are progressing in SCD $NVS WIZ degrader now in Ph1. Received much fanfare at ASH '23 and in Jul '24 Science pub (not sure why it's in solid tumor) Meanwhile $BMY also moved an HbF activating CELMoD into Ph1"
X Link 2024-11-01T01:13Z [----] followers, [----] engagements

"All early and far from clinical translation but the seeds of a broader theme in my view Hopeful that the ongoing renaissance in lineage-targeted depletion MOAs will lay the foundation for Precision Depletion - excited to watch this space mature in the years to come (8/8)"
X Link 2024-11-11T02:57Z [----] followers, [----] engagements

"Chefs kiss Ron $CERE $ABBV Couple of these payouts are remarkable when considered in the context of CEO tenure Ron Renaud - CEO of $CERE for [---] days David Epstein - CEO of $SGEN for [---] days Nice work if you can get it h/t @AndrewE_Dunn @endpts https://t.co/EqvvqOx2nu Couple of these payouts are remarkable when considered in the context of CEO tenure Ron Renaud - CEO of $CERE for [---] days David Epstein - CEO of $SGEN for [---] days Nice work if you can get it h/t @AndrewE_Dunn @endpts https://t.co/EqvvqOx2nu"
X Link 2024-11-11T14:35Z 10.2K followers, [----] engagements

"$NAMS MACE benefit in excess of that predicted by LDL-C alone bodes well for Lp(a) ASO / siRNA CVOT studies Pelacarsen HORIZON Ph3 data among the biggest catalysts of [----] $NVS $IONS $AMGN $ARWR"
X Link 2024-12-10T13:58Z 10.1K followers, [----] engagements

"China vs US in early drug discovery for known targets / modalities is simply a manifestation of Comparative Advantage. Economics [---]. Solutions Subsidies tariffs moving up the value chain (innovation). Id argue the former two would be destructive for industry writ large Problem for western discovery work is 1) losing expertise - many people good at small molecule chem work or protein engineering work - are not ethnically Western - sorry to say - . why stay at a Pfizer or Mrk and be a grunt for years vs going back "home" as many did in past Problem for western discovery work is 1) losing"
X Link 2024-12-18T13:21Z [----] followers, [----] engagements

"Perhaps the hedge is simply China itself Plenty of rhetoric re: the downfall of US biotech to Chinese competition and I agree the threat is real but is no one willing to reframe the problem and consider the positives There is a scenario in which accepting China's areas of comparative advantage results in more efficient / productive capital allocation within US biotech capital markets over the long-run (granted after a painful reset) $EQRX is an interesting hedge against VC-induced biotech incrementalism and the consequent overcrowding across targets indications and modalities Send tweet $EQRX"
X Link 2025-01-03T13:37Z [----] followers, [---] engagements

"Interim data from Ph1b of imvotamab in RA and SLE show that the depth and consistency of B cell depletion is insufficient to meet our high bar for success $IGMS Specific to human translation of IgM backbone or another datapoint supporting CD19 superiority over CD20 $CGEM"
X Link 2025-01-09T18:29Z [----] followers, [----] engagements

"FPD For $REGN Dupi x Linvo in Severe Allergy - 90% reduction and near-normalization of Serum IgE from 10x ULN and several-fold higher than IgE cut-off for Omalizumab eligibility Updated data in [----] (5/10)"
X Link 2025-01-17T11:01Z 10K followers, 18.8K engagements

"New TransCon Degrader Platform - less interested in the tech as the signal embedded in selecting XLH as the lead $ASND finally pursuing pipeline opps that can provide operating leverage and benefit from organizational synergies rather than Oncology Ophtho etc. (6/10)"
X Link 2025-01-17T11:01Z 10K followers, [----] engagements

"Complement Combinations - $APLS following $REGN with its own mAb + siRNA combination SYFOVRE + APL-3007 GA Ph1/2 starting 2Q25. Pozelimab + Cemdisiran GA Ph3 started 4Q24. C3 vs. C5 redux (7/10)"
X Link 2025-01-17T11:01Z 10K followers, [----] engagements

"Vision [----] at $BBIO - no lack of ambition guiding for the first time to $8B in de-risked peak sales potential Came with the first full reveal of the spun-out Gondola Bio pipeline - few details but some interesting indications / markets represented (8/10)"
X Link 2025-01-17T11:01Z 11.9K followers, [----] engagements

"Executional Excellence at $BGNE - compression of transitional lag times acceleration of enrollment & internalization of ops ownership culminating in industry-best cost per clinical trial Co is severely under-appreciated & among the best pipelines in biopharma IMO (9/10)"
X Link 2025-01-17T11:01Z 11.9K followers, [----] engagements

"Finally Terminal Value Visualized - since [----] $UCB mkt cap +200% relative to EBITDA +75% More interesting though 90% of that mkt cap expansion occurred in [----] attributable largely to a single drug Bimzelx whose success has driven out-year numbers much higher (10/10)"
X Link 2025-01-17T11:01Z 10.8K followers, [----] engagements

"2H25 [--]. $BBIO LGMD2i Ph3 IA [--]. $SRPT / $ARWR DM1+FSHD Ph1 [--]. $AGIO Mitapivat SCD Ph3 [--]. $MLTX SLK HS Ph3 [--]. $CGEM CD19xCD3 SLE Ph1/2 [--]. $BMRN CNP [---] Ph1 PK [--]. $SNY TSLPxIL-13 Asthma Ph2 [--]. $SNY / $INBX AATD Ph2 [--]. $KRRO AATD Ph1/2 FIH [--]. $MBX PTH Ph2 [--]. $AZN Efzim Alfa HPP Ph3 [--]. $REGN C5 Combo MG Ph3 [--]. $REGN DB-OTO Ph1/2 [--]. $REGN Obesity Combo Ph2s [--]. $ETNB Pego SHTG Ph3 [--]. $ARWR INHBE Ph1/2 [--]. $ARGX MuSK CMS Ph1b [--]. $ARGX efgart LN Ph2 [--]. $VRDN [---] IgG HV Ph1 [--]. $ROIV Brepo DM Ph3 [--]. $VTYX NLRP3 Pericarditis Ph2 [--]. $ZBIO Obex IgG4-RD Ph3"
X Link 2025-01-21T11:06Z 10.8K followers, [----] engagements

"2025 To Be Narrowed [--]. $NVS / $IONS Lp(a) Ph3 CVOT [--]. $JNJ TNF+IL-23 IBD Ph2b [--]. $PRME [---] CGD Ph1/2 [--]. $WVE AATD Ph1/2 MAD [--]. $WVE INHBE Ph1/2 [--]. $INCY mCALR MF / ET Ph1 [--]. $INCY JAK2-V617F MF Ph1"
X Link 2025-01-21T11:06Z 10.2K followers, [----] engagements

"LEQVIO poised to cross the $1B threshold in [----] $NVS $ALNY Out-of-pocket market in China is its biggest ex-US contributor - very interesting contrast to NRDL pricing and sales outcomes for many other innovative drugs"
X Link 2025-02-01T15:21Z 10.5K followers, [----] engagements

"Another ADPKD candidate enters the clinic $GSK GSK4771261 - antibody directed toward a "novel kidney target" New Therapies in ADPKD - $VRTX PC1 corrector now in HV Phase [--] $CRNX SST3 Agonist IND in [----] One of the largest still poorly-served rare disease markets - shades of CF (2/10) https://t.co/V86fygP0ns New Therapies in ADPKD - $VRTX PC1 corrector now in HV Phase [--] $CRNX SST3 Agonist IND in [----] One of the largest still poorly-served rare disease markets - shades of CF (2/10) https://t.co/V86fygP0ns"
X Link 2025-02-05T18:23Z 10.3K followers, [----] engagements

"Darkest before dawn May be approaching the trough of disillusionment for gene editing technology: (1) $VERV [---] SAEs (2) $CRSP CASGEVY terrible economics (3) $NTLA HAE degradation / TTR written off (4) Collabs terminated $MGX $DTIL etc. (5) Near-universal restructurings Publics down 50%+ YTD May be approaching the trough of disillusionment for gene editing technology: (1) $VERV [---] SAEs (2) $CRSP CASGEVY terrible economics (3) $NTLA HAE degradation / TTR written off (4) Collabs terminated $MGX $DTIL etc. (5) Near-universal restructurings Publics down 50%+ YTD"
X Link 2025-02-06T19:31Z 10.1K followers, 11.8K engagements

"Clinical (Q2) $RYTM HO Ph3 (Mon) $CYTK MAPLE-HCM ph3 $LLY Orfo T2D Ph3 $MRK oPCKS9 HeFH Ph3 $KYMR STAT6 Ph1 SAD/MAD $NKTR Rezpeg AD Ph2b $VERV [---] FIH $RNA FSHD Update $RZLT cHI Ph3 Interim $SYRE TL1A Ph1 $ASND CNP+HGH Achon Ph2 $SRRK Obesity Ph2 $RLYB FNAIT Ph2 Sentinel Pt $AUPH AUR200 Ph1 SAD $ACET Lupus FIH $BCAX Ficerafusp Update $VIGL ALSP Ph2"
X Link 2025-04-07T00:45Z 10.4K followers, [----] engagements

"Clinical (1H) $NUVL ROS1 Pivotal $RCKT Danon / PKP2 Update $PTCT HD Ph2 Data $SRPT LGMD2E Pivotal $APGE IL-4Ra Asthma Ph1b $CRSP Lp(a) / ANGPTL3 FIH $EWTX DMD Update $VRTX / MRNA CF Ph1/2 MAD $DSGN FECD Ph1 FIH $CRBU CD19 NHL HLA-Matched Cohort Data $KYTX MG Pivotal / Regulatory Update $SNY oTNF PsO Ph2 $SNY TNFa/OX40L bsAb HS Ph2 HS $BHVN IgG/IgA/B1AR Degrader Updates $SION NBD1 HV MAD"
X Link 2025-04-07T00:45Z 10.5K followers, [----] engagements

"Regulatory $ARGX VYVGART PFS sNDA PDUFA (4/10/25) $REGN Dupi CSU PDUFA (4/18/25) $ABEO Pz Cel RDEB PDUFA (4/29/25) $KALV Sebetralstat HAE PDUFA (6/17/25) $NUVB Taletrectinib ROS1 PDUFA (6/23/25)"
X Link 2025-04-07T00:45Z 10.5K followers, [----] engagements

"$SPRB -58% to $10M mkt cap What a mess. How does mgmt / BOD justify licensing an asset w/ BLA filing in 1H26 w/ no concurrent financing when cash-out is YE25 Now facing a full recap to get to approval - hard lesson learned for sloppy strategy and capital markets navet"
X Link 2025-04-15T20:27Z 11.4K followers, [----] engagements

"Interesting Cowen note on historical pharma P/E vs. S&P500 $IBB $DRG $XBI Current discount at levels seen only four other times in history. In all cases pharma relative P/E was higher 1Y and 3Y later"
X Link 2025-04-28T12:55Z 11K followers, 10.8K engagements

"$SNY is crushing $FOLD in Pompe NEXVIAZYME 1Q25 revs $220M (+26% YoY) / $880M annualized vs. PomOp's $21M / $84M (vs. prior $120M FY25 guide) Good case study on the structural advantage endowed by a big pharma sales infrastructure when competing drugs are undifferentiated"
X Link 2025-05-01T20:05Z 10.9K followers, [----] engagements

"#36 [--] [---] - beyond the recent examples of in vivo CAR-T and BE for DMD more indications / modalities in which China beat the US to clinic were on display (2/10) (1) First H2H data for OTOF AAV vs. Cochlear Implant - early but encouraging w/ AAV achieving superior responses on almost all metrics $REGN $LLY (2) Reforgene Medicine - Base-Edited Auto HSCT for Alpha-Thalassemia (3) HuidaGene - AAV-Cas13 RNA Editing for MECP2 Duplication Syndrome"
X Link 2025-05-18T00:12Z 10.7K followers, [----] engagements

"#206 [----] - speaking of nonsense mutations data from tRNA players still look underwhelming after several years of work (5/10) (1) Alltrna shared initial LNP-tRNA data in MMA and PKU restoring 25% and 7% of WT protein levels respectively after a single dose; durability of restoration not provided (2) Tevard shared AAV-tRNA data in DMD achieving 10% of WT dystrophin levels at 2-4e14 vg/kg doses"
X Link 2025-05-18T00:12Z 10.7K followers, [----] engagements

"#643 - interesting work from $SNY analyzing the predictive power of NHP data for CRISPR/Cas9 sgRNA off-target activity in humans. Surprisingly NHP models failed to recapitulate the majority of off-targets observed in the human genome Notable when considering that off-target editing has not (and likely will not due to biopsy burden) been measured in Cas9-treated human patients (6/10)"
X Link 2025-05-18T00:12Z 11.2K followers, [----] engagements

"#83 - fond memories of the $NVS / $AVRO fiasco back in [----]. Unfortunately for $NVS updated data from the Cystinosis Ph1/2 look weak Efficacy degraded meaningfully in more recently dosed patients - Pt. [--] & Pt. [--] (N=6) VCNs dropped below [--] and both restarted cysteamine (7/10)"
X Link 2025-05-18T00:12Z 11.2K followers, [----] engagements

"#256 - yet another modality joining the CD19 AID race with GentiBio unveiling a CD19 CAR-Treg program Murine data look similar to CD19 CAR-T with a potentially favorable cytokine profile - probably not enough to overcome the fields multi-year lead and successfully find a niche (8/10)"
X Link 2025-05-18T00:12Z 10.7K followers, [----] engagements

"#560 - wild multi-modality technology out of $ABBV - AAV-mediated delivery to neurological synapses of an anti-Tau CAR designed to facilitate localized clearance of pathogenic trans-synaptic tau Hard to believe this ever sees patients but too crazy not to highlight (9/10)"
X Link 2025-05-18T00:12Z 10.7K followers, [----] engagements

"#71 [---] [---] [----] - random collection of other interesting / novel programs (10/10) (1) Gene edited autologous CD4+ T cell therapy for Hyper IgM1; in the clinic now led by SR-Tiget (2) BAG3 AAV for protein aggregation disorders beyond DCM; nice work from NCH showing efficacy in a murine model of IBM (3) Anti-IgE BCR CAR-T for depletion of IgE+ B cells; probably not viable but a neat idea from Tel Aviv U (4) AAV-mediated p53 inhibition for TMEM43 mutant ARVC Type 5; interesting biology linking disruptions in nuclear envelope structure with DDR / p53 pathways"
X Link 2025-05-18T00:12Z 10.7K followers, [----] engagements

"Recent work from LEK does a nice job quantifying the me-too crowding dynamic vis-a-vis the industrys aggregate pipeline Key Points: (1) 2% of targets (N=38) were associated with 50+ assets; and collectively these assets represent 25% of the industrys entire R&D pipeline (2) the rate at which novel targets enter industrys pipeline has been cut in half from an average of [---] per year in 2015-20 to [--] per year in 2021-24"
X Link 2025-05-18T23:08Z 10.7K followers, [----] engagements

"Capital market sentiment readings from @endpts biannual Endpoints [---] Survey hitting fresh all time lows by a wide margin $XBI $BBC"
X Link 2025-05-26T11:48Z 10.9K followers, 23.8K engagements

"Almost as aesthetically satisfying as the STAT6 degrader data itself $KYMR Fun biotech trivia ahead of interviewing @NelloMainolfi at @KymeraTx for @BiotechTV today. You will not be able to unsee this. https://t.co/Cie43t4qoe Fun biotech trivia ahead of interviewing @NelloMainolfi at @KymeraTx for @BiotechTV today. You will not be able to unsee this. https://t.co/Cie43t4qoe"
X Link 2025-06-04T17:00Z 10.8K followers, [----] engagements

"Congrats Sek and $VERV team. If any pharma has the infrastructure capabilities and strategic license-to-operate required to bring gene editing to primary care its $LLY. Excited to see if they can pull it off But what will @skathire tweet about now Today we are thrilled to announce that @VerveTx has entered into a definitive agreement to be acquired by @EliLillyandCo From [----] to [----] Verve moved a new concept (a one-dose future for the treatment of atherosclerotic cardiovascular disease) and a new medical technology (in https://t.co/0HCnQMTTY9 Today we are thrilled to announce that @VerveTx"
X Link 2025-06-17T12:41Z 10.8K followers, 10.3K engagements

"Interesting timeline vis-a-vis $BPMC raising SM franchise peak sales substantially to $4B at JPM on 1/12/25 LT Peak Sales guides / raises have been a pretty consistent and reliable signal of strategic discussions / interest $BPMC / $SAN SC TO-T (1/2) 5/24/24: 1st contact on transaction relating to BLU-808 11/22/24: 1st discussion of possible co. transaction 1/13/2025: $124/share 1/24/2025: $129/share + $5 CVR + $4 CVR 1/29/2025: Counter of $135/share + $5 CVR 2/5/2025: Not moving fwd valuation gap $BPMC / $SAN SC TO-T (1/2) 5/24/24: 1st contact on transaction relating to BLU-808 11/22/24: 1st"
X Link 2025-06-17T14:41Z 11.1K followers, [----] engagements

"3Q Clinical / Corp. $APGE IL-13 AD Ph2 (Tomorrow) $MLTX SLK HS Ph3 $FULC SCD Ph1b Co3 $ABVX IBD Ph3 Induction $MBX PTH Ph2 $RARE / $MREO OI Ph3 IA2 $VRDN IgG HV $COGT ISM SUMMIT $UPB TSLP CRSwNP Ph2"
X Link 2025-07-06T22:28Z 11.3K followers, [----] engagements

"3Q Regulatory $REPL Melanoma PDUFA (7/22/25) $APLS C3G IC-MPGN (7/28/25) $PTCT Sepi PKU (7/29/25) $REGN Odro FL (7/30/25) $INSM Brenso Bronchiectasis (8/12/25) $RARE MPSIIIA GTx (8/18/25) $PTCT Vati FA (8/19/25) $IONS Doni HAE (8/21/25) $TVTX IgAN REMS sNDA (8/28/25) $CAPR Deramiocel DMD (8/31/25) $AGIO Mita Thal sNDA (9/7/25) $BIIB / $IONS HD-Spinraza SMA sNDA (9/22/25) $CRNX Paltu Acromegaly (9/25/25) $SRRK Apitegro SMA (9/22/25) $SNY Tolebru MS (9/28/25) $ASND Adult GHD (3Q)"
X Link 2025-07-06T22:28Z 11.7K followers, [----] engagements

"Taf IP durability is a huge swing factor in $PFE & $BBIO DCFs. Despite $PFE's coy demeanor path for Taf IP beyond [----] remains open & tractable 1H26 ruling in the EU Appeal case will foreshadow Taf's fate. IMO POS for $PFE / Taf is underestimated EPO already upheld '441 ('35 Vyndamax polymorph) against gx challengers ('23 ruling). Historically 30% of validity rulings are reversed by appeal. So 70% POS for Taf in EU EU outcome predicts US IPR outcome with 85% accuracy. In most exceptions US upheld while EU did not. So 85% conditional POS for Taf in US Together implied 60% probability that Taf"
X Link 2025-07-22T10:04Z 11.3K followers, 19.8K engagements

"Cool pub describing a mPBPK model for TfR1 mAb CNS exposure built on 10+ NHP datasets Often hear lower TfR affinity = more CNS exposure (vice-versa for muscle) - nice visual of that relationship Striking that max AUC hit at [----] nM K(D) - very very low by mAb standards"
X Link 2025-08-01T20:57Z 11.2K followers, [----] engagements

"Looking good for the lemming VCs who pumped $285M into Latigo's NaV1.8 me-too over the past [--] years $VRTX Vertex Announces Results from Phase [--] Study of VX-993 for the Treatment of Acute Pain Treatment with the selective NaV1.8 pain signal inhibitor VX-993 after bunionectomy surgery did not meet the primary endpoint https://t.co/RCZBGNMmgw $VRTX Vertex Announces Results from Phase [--] Study of VX-993 for the Treatment of Acute Pain Treatment with the selective NaV1.8 pain signal inhibitor VX-993 after bunionectomy surgery did not meet the primary endpoint https://t.co/RCZBGNMmgw"
X Link 2025-08-04T23:20Z 11.2K followers, 10.8K engagements

"Full details: https://www.clinicaltrialsregister.eu/ctr-search/trial/2021-002012-31/results#endPointsSection https://www.clinicaltrialsregister.eu/ctr-search/trial/2021-002012-31/results#endPointsSection"
X Link 2025-08-19T19:27Z 11.3K followers, [----] engagements

"Analysis based on funds raised 2007-13 only time will tell how more recent investments perform Only time Meanwhile In the past 2-3 yrs 7+ TRV Cos shut down - Abata Ambys Cargo Cedilla Faze Goldfinch Rheos - after raising $500M combined GP succession is hard Third Rock tops STATs VC rankings boosted by early biotech investments https://t.co/qRef7wwoKM via @ADeAngelis_bio Third Rock tops STATs VC rankings boosted by early biotech investments https://t.co/qRef7wwoKM via @ADeAngelis_bio"
X Link 2025-08-27T21:03Z 11.4K followers, [----] engagements

"The suspense. How is anyone going to get a wink of sleep this weekend $EDIT"
X Link 2025-08-28T20:25Z 11.4K followers, [----] engagements

"Well-appreciated that in recent years SMid bios have increasingly commercialized independently and those launches have increasingly performed well But how could those trends flow through to $XBI Really interesting work from Schimmer highlighting a potl. inflection in $XBI profitability in [----] and beyond Financial metrics speak for themselves and unlike M&A may be capable of attracting sticky and sustainable generalist flows. It's a contrarian view but the sector needs more $ARGX & $ALNY than $ITCI & $BPMC"
X Link 2025-08-29T00:00Z 11.4K followers, [----] engagements

"$PFE pulling Taf 20mg from the US market by YE25 and you still think the Vynda franchise erosion curve is going to look like this $BBIO $ALNY Taf IP durability is a huge swing factor in $PFE & $BBIO DCFs. Despite $PFE's coy demeanor path for Taf IP beyond [----] remains open & tractable 1H26 ruling in the EU Appeal case will foreshadow Taf's fate. IMO POS for $PFE / Taf is underestimated EPO already upheld '441 ('35 https://t.co/nOMGMm6Av9 Taf IP durability is a huge swing factor in $PFE & $BBIO DCFs. Despite $PFE's coy demeanor path for Taf IP beyond [----] remains open & tractable 1H26 ruling"
X Link 2025-08-29T22:18Z 11.9K followers, [----] engagements

"@BalaBioResearch Thought I was reading a $BMRN short report while skimming this list"
X Link 2025-09-17T20:44Z 12K followers, [---] engagements

"Zolgensma is clearly stalling out. $NVS print at $254M down 13% sequentially. Annualizing at $1B but next leg of growth clearly dependent on IT in Type 2/3. Meanwhile $RHBBY / $PTCT talking about Evrysdi achieving top market share THIS YEAR"
X Link 2021-01-26T14:29Z 12.2K followers, [--] engagements

"But wed never use alemtuzumab allo CAR-T field starting to come around to importance of lymphodepletion $ALLO We are excited to announce an exclusive license agreement with @TizianaLS to explore their anti-CD3 antibody foralumab as a potential lymphodepletion agent with our allogeneic CAR T programs for cancer treatment. Learn more here: https://t.co/KNSHXWURwL https://t.co/X9C2IadlF6 We are excited to announce an exclusive license agreement with @TizianaLS to explore their anti-CD3 antibody foralumab as a potential lymphodepletion agent with our allogeneic CAR T programs for cancer"
X Link 2021-09-02T12:28Z 12.7K followers, [--] engagements

"Initial Pompe gene therapy data from $BOLD / $ALPMY at WORLD. Scope of "efficacy" data limited to VCNs in [--] low dose patients. No disclosure of GAA enzymes levels substrate levels or ERT withdrawal despite 3-6 months of follow up for [--] patients"
X Link 2022-02-08T16:37Z 12.7K followers, [--] engagements

"$ALPMY recognizing $400M+ impairment on $BOLD. Cites delayed approval and restricted label for XLMTM. Discontinuing vectorized exon skipping programs for DMD. PR notes preclinical as the driver but clinical data (via Nationwide) have also shown negligible dystrophin restoration"
X Link 2022-04-22T13:34Z 12.6K followers, [--] engagements

"Astellas' $BOLD acquisition is a gift that keeps on giving. AT845 (Pompe GTx) clinical hold following an SAE of peripheral sensory neuropathy The first case of such in an AAV trial to my knowledge so hard to know if truly drug-related or FDA quick on the trigger $ALPMY"
X Link 2022-06-26T23:51Z 12.6K followers, [--] engagements

"AAV GTx bottom feeding frenzy continues $ALPMY putting $50M into $TSHA for 15% pro forma ownership. Implied 400%+ premium to todays close at $65M market cap Astellas receives exclusive option to license GAN and Rett programs seemingly at to-be-negotiated terms"
X Link 2022-10-24T23:43Z 12.7K followers, [--] engagements

"Former $AVXS A-team stepping in to rescue the subs at $TSHA Sean Nolan former AveXis CEO stepping into CEO role from Chair Sukumar Nagendran former AveXis CMO stepping into President and Head of R&D from BOD seat RA Session to resign but remain on BOD"
X Link 2022-12-16T23:41Z 12.7K followers, [----] engagements

"$ALPMY $BOLD Pompe GTx off clinical hold [--] months to resolve a single Grade [--] SAE that hasnt emerged in other AAV programs Astellas' $BOLD acquisition is a gift that keeps on giving. AT845 (Pompe GTx) clinical hold following an SAE of peripheral sensory neuropathy The first case of such in an AAV trial to my knowledge so hard to know if truly drug-related or FDA quick on the trigger $ALPMY Astellas' $BOLD acquisition is a gift that keeps on giving. AT845 (Pompe GTx) clinical hold following an SAE of peripheral sensory neuropathy The first case of such in an AAV trial to my knowledge so hard"
X Link 2023-01-21T17:48Z 12.7K followers, [----] engagements

"$MYLS upsized at the high end of the range IPOs off to a hot start in 2023"
X Link 2023-02-10T01:01Z 12.1K followers, [----] engagements

"Today's edition of "does this gene therapy actually work" featuring some chart crime from $PASG"
X Link 2023-02-24T12:24Z 12.7K followers, [----] engagements

"Similar fact pattern / timeline as $XLRN. Post-STELLAR data clear that $XLRN undervalued itself Will the same be true for $RXDX More likely than not imo Breaking: Merck in Late-Stage Talks to Acquire Prometheus Biosciences https://t.co/YcTIHqeIJ8 via @LCooperReports @laurenthomas Breaking: Merck in Late-Stage Talks to Acquire Prometheus Biosciences https://t.co/YcTIHqeIJ8 via @LCooperReports @laurenthomas"
X Link 2023-04-16T09:30Z 12K followers, [----] engagements

"#1: Delivery (1/2) - to no ones surprise delivery remains a core theme. I flagged the saturation of novel AAV capsid abstracts last year and [----] is no different but it also includes a broader and more diverse set of technologies than ever before (3/26)"
X Link 2023-05-07T11:37Z 12.6K followers, [----] engagements

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