Sarepta Therapeutics, Inc.
SRPT evidence brief
What we can confirm
What it means
What to watch
Prepared research question
What matters most for SRPT—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · SRPT
Sarepta Therapeutics is a precision genetic medicine company focused on rare diseases, with four approved products for Duchenne muscular dystrophy and a pipeline including gene therapies and RNA-based candidates.
Key risk: ESSENCE trial failure impact
BioSniper Score
Based on 6 of 6 signals
Market Cap
$2.20B
Cash Runway
—
Lead Asset
amondys 45 (Approved)Next Catalyst
Phase3_Topline · Oct 31, 2026Financial data as of Jun 30, 2026.
Tracking 9 pipeline candidates · 27 clinical trials · 8 catalysts · 3 FDA decisions for Sarepta Therapeutics, Inc..
Sarepta Therapeutics is a precision genetic medicine company focused on rare diseases, with four approved products for Duchenne muscular dystrophy and a pipeline including gene therapies and RNA-based candidates.
- Net product revenue for four approved products was approximately $1.9 billion in 2025 [sec · filed 2026-04-24].
- Cash & equivalents of $581.3M as of 2026-06-30 [sec].
- Total revenue of $401.3M and net loss of -$4.9M for the six months ended 2026-06-30 [sec].
- Convertible debt exchanges in 2025 reduced outstanding 2027 notes from $1.15 billion to $159 million [sec · filed 2026-04-24].
- ELEVIDYS gene therapy commercially launched in Japan by partner Chugai, triggering a $40 million milestone [sec · filed 2026-04-24].
What works
- Four approved Duchenne products generated ~$1.9B in net revenue in 2025, demonstrating commercial traction [sec · filed 2026-04-24].
- ELEVIDYS showed durability and clinical significance in three-year EMBARK trial data [sec · filed 2026-04-24].
- Strategic partnerships (e.g., Chugai in Japan, Catalent for manufacturing) support global expansion and production capacity [sec · filed 2026-04-24][sec_mda · as of 2025-11-06].
- Proprietary CMC and manufacturing capabilities for PMO and gene therapies enable both clinical and commercial supply [sec_mda · as of 2026-03-02].
What to weigh
- ESSENCE confirmatory trial for VYONDYS and AMONDYS failed to meet statistical significance on its primary endpoint, potentially impacting cash flows [sec_mda · as of 2025-11-06].
- ELEVIDYS shipments to non-ambulatory patients in the U.S. remain paused, limiting revenue potential [sec_mda · as of 2026-08-05].
- Net loss of -$4.9M for the six months ended 2026-06-30 and substantial future capital requirements [sec][sec_mda · as of 2026-08-05].
- Restructuring and pipeline reprioritization may not generate intended benefits, adding execution risk [sec_mda · as of 2026-08-05].
From the filings
Quoted directly from source documents.
Net product revenue for four approved products was approximately $1.9 billion in 2025.
“Our net product revenue for our four approved products was approximately $1.9 billion.”
SEC filings · April 24, 2026
ESSENCE trial failed to meet statistical significance on primary endpoint.
“topline results of our ESSENCE trial, a confirmatory trial intended to verify the clinical benefits of our two PMO Products, VYONDYS and AMONDYS, that were announced on November 3, 2025 and failed to meet statistical significance on its primary endpoint”
SEC MD&A · November 6, 2025
Convertible debt exchanges reduced 2027 notes from $1.15 billion to $159 million.
“Our convertible debt exchanges in August and December 2025 reduced the total amount due under our outstanding convertible notes due in 2027 from $1.15 billion to $159 million.”
SEC filings · April 24, 2026
ELEVIDYS shipments to non-ambulatory patients in the U.S. are paused.
“our ability to resume commercial shipments of ELEVIDYS for non-ambulatory patients in the U.S.”
SEC MD&A · August 5, 2026
Catalyst timeline
- 2026-10-31 · Phase3_Topline — Phase 3 Topline: A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of High Dos — Topline results from a Phase 3 trial of high-dose ELEVIDYS will be read out, informing potential label expansion.
- 2028-06-28 · Patent_Expiry · VYONDYS 53 — Patent/Exclusivity Expiry: VYONDYS 53 — Patent/exclusivity expiry for VYONDYS 53 could open the door to generic competition.
- 2028-06-30 · Phase3_Topline — Phase 3 Topline: A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therap — Topline results from a Phase 3 systemic gene transfer therapy trial will be released.
- 2029-11-30 · Phase3_Topline — Phase 3 Topline: A Phase 3 Multinational, Open-label, Systemic Gene Delivery Study to Evaluate the Safety and Efficac — Topline results from an open-label systemic gene delivery study will be available.
- 2030-11-12 · Patent_Expiry · AMONDYS 45 — Patent/Exclusivity Expiry: AMONDYS 45 — Patent/exclusivity expiry for AMONDYS 45 could lead to generic entry.
- 2032-03-30 · Phase3_Topline — Phase 3 Topline: A Seamless Phase 1/3, Multicenter, Single Dose Systemic Gene Transfer Study to Evaluate the Safety, — Topline results from a seamless Phase 1/3 gene transfer study will be reported.
- 2033-10-31 · Phase3_Topline — Phase 3 Topline: A Phase 3, Multinational, Long-term Follow-up Study to Evaluate Safety and Efficacy in Subjects Who — Topline results from a long-term follow-up study will be released.
- 2034-03-14 · Patent_Expiry · EXONDYS 51 — Patent/Exclusivity Expiry: EXONDYS 51 — Patent/exclusivity expiry for EXONDYS 51 could allow generic competition.
Risks & what to watch (4)›
Key risks
- ESSENCE trial failure impact — Confirmatory trial for VYONDYS and AMONDYS failed primary endpoint, potentially reducing cash flows [sec_mda · as of 2025-11-06].
- ELEVIDYS shipping pause — Commercial shipments to non-ambulatory U.S. patients remain paused, limiting revenue [sec_mda · as of 2026-08-05].
- Restructuring execution risk — Restructuring and pipeline reprioritization may not achieve intended benefits [sec_mda · as of 2026-08-05].
- Substantial capital needs — Future expenditures and long-term capital requirements are substantial and may require additional financing [sec_mda · as of 2026-08-05].
What to watch
- Topline results from Phase 3 trial of high-dose ELEVIDYS expected October 31, 2026 [catalyst].
- Patent/exclusivity expiry for VYONDYS 53 on June 28, 2028 [catalyst].
- Topline results from Phase 3 systemic gene transfer therapy trial expected June 30, 2028 [catalyst].
- Regulatory discussions regarding SRP-9003 BLA submission and potential approval [sec_mda · as of 2025-08-06].
Sources: SEC filings · SEC MD&A · Generated August 29, 2026 · How we verify sources →
Run Sarepta Therapeutics, Inc. through your own thesis
Use the pipeline, catalyst, financial, FDA, and market facts on this page as inputs to a protocol you control.
Your protocol can:
- Turn available Sarepta Therapeutics, Inc. facts into screen rules or deeper checks
- Show cited findings, blockers, and unresolved evidence together
- Save the logic and rerun it as the company evidence changes
Evidence & data
Score breakdownBased on 6 of 6 signals · derived from public data
Profitable (positive operating cash flow)
D/E 1.09 · 0% dilution
Lead: Approved
9 candidates
8 upcoming, next in ~62d
0 buys · 1 sell (180d)
Catalysts
Pipeline
Regulatory
Financials
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
341
Total value held
$2.38B
| Holder | Shares | Value |
|---|---|---|
| blackrock, inc. | 14,755,277 | $321.1M |
| vanguard capital management llc | 9,156,470 | $199.2M |
| vanguard portfolio management llc | 6,640,692 | $144.5M |
| state street corp | 6,613,314 | $143.9M |
| aqr capital management llc | 6,433,112 | $138.6M |
| two sigma investments, lp | 4,593,195 | $99.9M |
| first trust advisors lp | 4,135,788 | $90.0M |
| ra capital management, l.p. | 3,902,596 | $84.9M |
| susquehanna international group, llp | 3,818,732 | $83.1M |
| morgan stanley | 2,596,993 | $56.5M |
Showing top 10 of 341 institutional holders of Sarepta Therapeutics, Inc.. Open the full 13F history after sign-in.
Short Interest
as of Apr 30, 2026- % of float
- 28.0%
- Days to cover
- 7.7
- Shares short
- 25,334,475
- Daily short volume
- -
Short percent of float and days-to-cover reflect reported short positioning. Daily short volume is the exchange-reported short share of a single day's trading volume and is noisier. See the most-shorted biotech ranking →
SRPT short interestGovernance
Frequently asked questions
What is the AI investment thesis for Sarepta Therapeutics, Inc.?
Sarepta Therapeutics is a precision genetic medicine company focused on rare diseases, with four approved products for Duchenne muscular dystrophy and a pipeline including gene therapies and RNA-based candidates.
What is Sarepta Therapeutics, Inc.'s lead drug candidate?
Sarepta Therapeutics, Inc.'s most advanced tracked candidate is amondys 45 (Approved) for Duchenne muscular dystrophy in patients with confirmed mutation of the dystrophin gene amenable to exon 45 skipping.
What is Sarepta Therapeutics, Inc.'s next catalyst?
Sarepta Therapeutics, Inc.'s next tracked catalyst is a Phase3_Topline event — "Phase 3 Topline: A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of High Dos" — expected October 31, 2026.
Who is the CEO of Sarepta Therapeutics, Inc.?
Dr. Michael E. Severino M.D. is the chief executive officer of Sarepta Therapeutics, Inc. (SRPT).
Where is Sarepta Therapeutics, Inc. headquartered?
Sarepta Therapeutics, Inc. (SRPT) is headquartered in Cambridge, MA, United States.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.