$SRPT: Disappoints on Trial, Gene Therapy Misses Late-Stage Main Goal скачать в хорошем качестве

$SRPT: Disappoints on Trial, Gene Therapy Misses Late-Stage Main Goal

🛠 What options are still on the table: Despite the setback, Sarepta is not without pathways forward. Here are some options: For Sarepta Leverage biomarker/secondary endpoint data: Use the strong expression/micro-dystrophin data and consistent safety profile in certain populations to secure label expansions or approvals in narrower subpopulations (younger ambulatory patients, perhaps). Join this channel to get access to perks:    / @equity4keeps   Consider Supporting this Channel via PayPal: https://www.paypal.com/donate/?hosted... Refocus on earlier-stage and diversified pipeline: The LGMD2E/R4 program, siRNA assets (FSHD, DM1), etc., might become more central to value creation. Diversification may lessen reliance on this single DMD gene-therapy outcome. Real-world evidence & long-term follow-up: If commercial use or open-label extension studies show durability of benefit, that data can bolster confidence and perhaps support future filings or label expansions. Partnering or reacquisition for specific indications: Sarepta might seek partners (or be a partner target) for specific rare-disease indications, leveraging its commercial infrastructure and manufacturing while off-loading risk. Cost/operational restructuring: If commercial uptake is slower, the company may need to align costs, prioritize programs, and preserve cash. For investors Trim outlook or reset expectations: Given the higher risk profile, investors may revise down growth estimates for Elevidys and reduce the weight of DMD gene therapy in their models. Focus on upside from other programs: Monitor upcoming readouts from LGMD, siRNA, label expansions, etc., which may provide new catalysts. Watch for regulatory clarity: Key upcoming regulatory decisions will influence the risk/reward. Consider takeover potential cautiously: While still possible, a gene-therapy miss reduces some upside bargaining power; any acquisition is likely to include risk-sharing or contingent payments. Risk management: Given increased uncertainty, position sizing, stop-losses, and time horizon matter more. 🎯 What to watch Whether Sarepta obtains FDA/EMA feedback indicating that the EMBARK miss will not block label expansion or regulatory acceptance of their data package. The next major readouts in other programs (LGMD, etc.) — success here could re-ignite the valuation story. Commercial uptake of Elevidys in the approved cohort (ambulatory children) — if real-world uptake and early benefits are favorable, that can salvage some narrative. Any restructuring announcements (cost cuts, strategic partnerships) which signal how management is responding. Acquirer interest — whether companies still consider Sarepta “buyable” despite the setback or if this raises red flags.