[GUEST ACCESS MODE: Data is scrambled or limited to provide examples. Make requests using your API key to unlock full data. Check https://lunarcrush.ai/auth for authentication information.]  zipjet [@zipjet](/creator/twitter/zipjet) on x 2670 followers Created: 2025-07-21 12:49:35 UTC $SRPT an AI recap of the 7/18 management presentation: Analysis of Sarepta Therapeutics LGMD Program Update Call This call was convened to address investor concerns and questions arising from the disclosure of a fatal adverse event in a clinical trial for SRP-9004, an investigational gene therapy for LGMD2D, and its potential implications for Sarepta's broader gene therapy programs, particularly Elevidys. The company aimed to clarify its disclosure strategy, the rationale behind discontinuing certain LGMD programs, and the impact on Elevidys' safety profile and commercialization strategy. The business is performing in a way that has forced prioritization of the siRNA platform over the gene therapy portfolio due to risk-adjusted NPVs and the need to manage liabilities conservatively. The decision to discontinue certain LGMD programs was based on these financial considerations, not directly on safety concerns, although safety is always a factor in NPV calculations. The company is focusing on SRP-9003 for LGMD2E, which is in a later stage of development, while seeking partnerships or out-licensing opportunities for the other LGMD programs. The company is trying to balance transparency with the standard practice of presenting complete study results at medical meetings. Key Financial Highlights Prioritization of siRNA platform over gene therapy due to risk-adjusted NPVs. Discontinuation of certain LGMD programs to manage liabilities. Focus on SRP-9003 for LGMD2E while seeking partnerships for other LGMD programs. Elevidys sales are still expected to meet a $XXX million floor. Key Takeaways Disclosure Strategy: Sarepta's management defended its decision not to disclose the fatal adverse event earlier, stating it was not material to the strategic update provided on Wednesday and that the standard practice is to present complete study results at medical meetings. Elevidys Safety Signal: The company reiterated that the fatal event in the LGMD2D trial does not change the known safety signal for Elevidys, which involves a rare risk of acute liver injury (ALI) leading to acute liver failure (ALF) in more debilitated, non-ambulatory patients. Prophylactic Immunosuppression: Sarepta has already paused shipments of Elevidys for dosing in non-ambulatory patients and is developing a protocol for using sirolimus as a prophylactic immunosuppressant in this population. FDA Communication: The FDA has been informed of the fatal event and has not changed its position regarding the Elevidys label. AAV Class Risk: Sarepta emphasized that the risk of ALI/ALF is an AAV class-related risk, not specific to Elevidys or its manufacturing process. Physician and Patient Communication: Sarepta aims to provide physicians and patients with balanced and accurate information to make informed risk-based decisions about Elevidys. Platform Designation: The platform designation received from the FDA was specifically for LGMD2E (SRP-9003) and its adherent manufacturing process. No other fatalities: There have been no other fatalities associated with any other gene therapy trials associated with the therapy itself. Guidance Sarepta has paused shipments of Elevidys for use in non-ambulatory patients while they develop a protocol for and obtain data regarding the use of sirolimus as a prophylactic immunosuppression in that patient population. The company hopes to have conversations with the FDA about gathering that data as soon as possible. They are evaluating whether to make any recommendations on sirolimus as they submit the BLA for LGMD2E. The Complete AI Powered Stock Research Platform - XXX engagements  **Related Topics** [fatal](/topic/fatal) [investment](/topic/investment) [sarepta therapeutics](/topic/sarepta-therapeutics) [coins ai](/topic/coins-ai) [$srpt](/topic/$srpt) [sarepta therapeutics inc](/topic/sarepta-therapeutics-inc) [stocks healthcare](/topic/stocks-healthcare) [Post Link](https://x.com/zipjet/status/1947277777100542062)
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zipjet @zipjet on x 2670 followers
Created: 2025-07-21 12:49:35 UTC
$SRPT an AI recap of the 7/18 management presentation:
Analysis of Sarepta Therapeutics LGMD Program Update Call This call was convened to address investor concerns and questions arising from the disclosure of a fatal adverse event in a clinical trial for SRP-9004, an investigational gene therapy for LGMD2D, and its potential implications for Sarepta's broader gene therapy programs, particularly Elevidys. The company aimed to clarify its disclosure strategy, the rationale behind discontinuing certain LGMD programs, and the impact on Elevidys' safety profile and commercialization strategy.
The business is performing in a way that has forced prioritization of the siRNA platform over the gene therapy portfolio due to risk-adjusted NPVs and the need to manage liabilities conservatively. The decision to discontinue certain LGMD programs was based on these financial considerations, not directly on safety concerns, although safety is always a factor in NPV calculations. The company is focusing on SRP-9003 for LGMD2E, which is in a later stage of development, while seeking partnerships or out-licensing opportunities for the other LGMD programs. The company is trying to balance transparency with the standard practice of presenting complete study results at medical meetings.
Key Financial Highlights Prioritization of siRNA platform over gene therapy due to risk-adjusted NPVs. Discontinuation of certain LGMD programs to manage liabilities. Focus on SRP-9003 for LGMD2E while seeking partnerships for other LGMD programs. Elevidys sales are still expected to meet a $XXX million floor. Key Takeaways Disclosure Strategy: Sarepta's management defended its decision not to disclose the fatal adverse event earlier, stating it was not material to the strategic update provided on Wednesday and that the standard practice is to present complete study results at medical meetings. Elevidys Safety Signal: The company reiterated that the fatal event in the LGMD2D trial does not change the known safety signal for Elevidys, which involves a rare risk of acute liver injury (ALI) leading to acute liver failure (ALF) in more debilitated, non-ambulatory patients. Prophylactic Immunosuppression: Sarepta has already paused shipments of Elevidys for dosing in non-ambulatory patients and is developing a protocol for using sirolimus as a prophylactic immunosuppressant in this population. FDA Communication: The FDA has been informed of the fatal event and has not changed its position regarding the Elevidys label. AAV Class Risk: Sarepta emphasized that the risk of ALI/ALF is an AAV class-related risk, not specific to Elevidys or its manufacturing process. Physician and Patient Communication: Sarepta aims to provide physicians and patients with balanced and accurate information to make informed risk-based decisions about Elevidys. Platform Designation: The platform designation received from the FDA was specifically for LGMD2E (SRP-9003) and its adherent manufacturing process. No other fatalities: There have been no other fatalities associated with any other gene therapy trials associated with the therapy itself. Guidance Sarepta has paused shipments of Elevidys for use in non-ambulatory patients while they develop a protocol for and obtain data regarding the use of sirolimus as a prophylactic immunosuppression in that patient population. The company hopes to have conversations with the FDA about gathering that data as soon as possible. They are evaluating whether to make any recommendations on sirolimus as they submit the BLA for LGMD2E.
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