Given investor interest in DM1/FSHD but hesitation on DMD pipeline risk, how does management view a potential separation of these businesses?

CEO Michael Severino emphasized the strong synergy between the DMD and siRNA businesses. He stated that the stable, durable revenue from DMD products funds the advancement of the earlier siRNA pipeline, making them complementary and crucial for the company's future growth. “I think there's tremendous synergy between those aspects of what we do here at Sarepta in the big picture. So we're very committed to Duchenne. We've been in Duchenne for more than a decade now.”

With lowered OpEx guidance, what are the puts and takes around the OpEx run rate, and how do ELEVIDYS commercial efforts and competitive dynamics influence long-term OpEx?

CFO Ryan Wong confirmed comfort with the $800M-$900M OpEx range, stating it allows funding of commercial initiatives and pipeline advancement. He emphasized disciplined capital allocation, focusing on science with the highest probability of success, despite competitive dynamics. “Yes, so we've talked previously around, we're very comfortable in that $800 million and $900 million range in terms of OpEx both being able to fund our commercial initiatives and to advance our pipeline.”

Can management discuss the regulatory strategy for siRNA programs, specifically regarding accelerated vs. full approval and primary endpoints for pivotal studies?

Dr. Louise Rodino-Klapac stated that for both FSHD and DM1, the regulatory pathway allows for either accelerated or traditional approval, depending on the landscape and data. The MAD study readout will inform the selection of primary endpoints for Phase 3 trials, with a data-driven discussion with regulators. “Now, for both FSHD and DM1, in terms of the regulatory pathway, as we've described before, the way we've thought about it and set it up is that we have the ability to apply for both accelerated approval and traditional approval depending on the regulatory framework at that time, the landscape, and the data that's generated.”

SRPT

Sarepta (SRPT) Q2 FY26 earnings call: guidance and Q&A

Earnings call· Jun 2026 (Q2 FY26)· Health Care· Aug 5, 2026

Source: earnings call transcript

Executive summary

Sarepta Q2 FY26 — Strong Financials & Pipeline Progress Amidst ELEVIDYS Adoption Focus

Sarepta Therapeutics reported a financially strong second quarter, marked by operating profitability and increased cash, enabling independent advancement of its pipeline. While ELEVIDYS adoption and exon-skipping competition remain concerns, the company is focused on commercial execution and anticipates significant data readouts from its siRNA programs and Duchenne regulatory decisions. The core strategy emphasizes biology-driven innovation and disciplined capital allocation to drive future growth.

Highlights

Achieved GAAP operating income of $13 million and non-GAAP operating income of $86 million for the quarter.
Increased cash and investments by approximately $197 million during the quarter, reaching $945 million.
PMO franchise demonstrated stable demand and sustained patient/physician confidence, with over 1,800 patients treated worldwide and adherence rates exceeding 90%.
FDA accepted supplemental NDA submissions for AMONDYS 45 and VYONDYS 53 for review, with a target action date of February 28, 2027.
Narrowed FY26 total net product revenue guidance to $1.2 billion to $1.3 billion, with the midpoint being the appropriate reference, consistent with prior expectations.

Concerns

Total net product revenue decreased 34% year-over-year in Q2, primarily driven by lower ELEVIDYS demand.
ELEVIDYS revenue in the second half of 2026 is expected to be modestly lower than the first half, with Q3 trending lower than Q2.
A $39 million litigation contingency charge was included in GAAP results for the quarter.
Acknowledged concerns around ELEVIDYS adoption and competition on the horizon for exon skipping treatments.

Guidance & targets

Category	Target	Timeline	Confidence
Total net product revenue	$1.2B to $1.3B high materiality	FY26	High
Total collaboration and other revenue	$550M to $600M medium materiality	FY26	High
Non-GAAP OpEx	$800M to $850M medium materiality	FY26	High
ELEVIDYS revenue	modestly lower than in the first half high materiality	H2 FY26	Medium
ELEVIDYS revenue	trend lower than Q2 high materiality	Q3 · FY26	Medium
ELEVIDYS revenue contribution from enrollment forms	more meaningfully to revenue in 2027 high materiality	FY27	High

Segment performance

Segment	Revenue	YoY	QoQ	Margin
ELEVIDYS Performance was in line with expectations, with sales remaining relatively steady and quarter-over-quarter growth in enrollment forms signaling increasing demand. Enrollment forms: quarter-over-quarter growth	$98M	—	—	—
PMO franchise Performance continues to reflect stable demand and sustained patient and physician confidence, supported by extensive real-world experience and evidence. Patients treated worldwide: >1,800Adherence rates: >90%	$231M	—	—	—
Total Net Product Revenue Total net product revenue for the second quarter.	$329M	—	—	—

Deals & partnerships

Roche Collaboration and contract manufacturing revenue $40M

Receipt of $40 million from the Roche commercial sale milestone earned in Q1.

Risks & headwinds

ELEVIDYS adoption concerns H2 FY26

ELEVIDYS revenue in H2 FY26 expected to be modestly lower than H1; Q3 FY26 expected to trend lower than Q2.

Mitigation:Expanded commercial footprint, improved patient identification, expanded education for patients and families, strengthening healthcare providers' confidence, record number of HCP interactions.

Competition for exon skipping treatments Likely later in 2027

Not explicitly quantified, but acknowledged as a concern.

Mitigation:Confidence in the durability of the PMO franchise due to long track record, extensive real-world evidence, favorable safety profile, high adherence rates (>90%), and established infrastructure for patient support and reimbursement.

Acute liver injury (ALI) associated with AAV gene therapy Acute post-infusion

Known risk associated with AAV gene therapy as a class effect.

Mitigation:Cohort 8 of the ENDEAVOR study is assessing prophylactic sirolimus treatment to reduce ALI. Interim safety data from the Phase 4 ENDURE study showed zero incidence of ALI in patients treated prophylactically with sirolimus.

Litigation contingency charge Q2 FY26

$39M

Mitigation:Charge to potentially resolve certain outstanding patent claims.

What to watch in Q3 FY26

ELEVIDYS 12-week Cohort 8 data

Q1 2027

Current Enrollment underway, expected completion end of 2026.

Target 12-week data from full cohort, including incidence of ALI and dystrophin expression.

Why it matters

Will determine if prophylactic sirolimus can reduce acute liver injury, potentially expanding ELEVIDYS' label to non-ambulatory patients and impacting its commercial opportunity.

We expect to fully enroll the ENDEAVOR Cohort 8 study by the end of 2026. Based on observations that our study investigators are dosing participants sequentially, we now expect 12-week data from the full cohort in the first quarter of 2027.

Find out whether SRPT delivers this. Inve re-checks your case every morning.

Q&A highlights

What aspects of the pipeline are most exciting, specifically Cohort 8 data for ELEVIDYS and the Arrowhead/siRNA programs?

CEO Michael Severino expressed excitement for Cohort 8's potential to improve ELEVIDYS' benefit-risk in non-ambulatory patients and the tremendous potential of the siRNA programs (FSHD, DM1, Huntington's) due to predictive preclinical models, high knockdown, and effective delivery technology.

“But when I look at the earlier pipeline in the siRNA programs that we're advancing, I believe they have tremendous potential. First of all, what I would say is in this space, pre-clinical models and early clinical data have a very high degree of predictive power.”

asked by Anupam Rama · answered by Michael Severino

2 min read 7 chapters

Detailed narrative

CEO Transition and Strategic Vision

Michael Severino, in his first earnings call as CEO, expressed confidence in Sarepta's position as a leader in Duchenne and rare disease innovation. He highlighted the company's scientific achievements, including exon skipping and ELEVIDYS, and the potential of its siRNA platform.

Severino emphasized the financial strength to independently advance pipeline programs and the importance of upcoming milestones, including Cohort 8 data and siRNA program readouts, to clarify the company's growth trajectory.

Commercial Performance and ELEVIDYS Momentum

Total net product revenue for Q2 FY26 was $329 million, with ELEVIDYS contributing $98 million and the PMO franchise $231 million. ELEVIDYS performance was in line with expectations, showing improving quarter-over-quarter enrollment forms, signaling increasing demand. The company expanded its commercial footprint and deployed sales teams, resulting in a record number of healthcare provider interactions and broader site activity, reinforcing confidence in ELEVIDYS' benefit-risk profile.

Pipeline Progress in siRNA Programs

Sarepta remains on track to announce interim results from its Multi-Ascending Study (MAD) for SRP-1001 (FSHD) and SRP-1003 (DM1) in the second half of 2026. The siRNA platform is differentiated by biology-driven tissue targeting, efficient intracellular delivery, and catalytic siRNA potency.

The company aims to achieve deep DUX4 knockdown in FSHD and DMPK knockdown in DM1, with preclinical models showing high muscle concentration and strong safety profiles, positioning these programs as potential best-in-class therapies.

Duchenne Regulatory Updates and ELEVIDYS Safety

The FDA accepted supplemental NDA submissions for AMONDYS 45 and VYONDYS 53, seeking conversion to traditional approvals with a target action date of February 28, 2027. Enrollment and dosing continue in Cohort 8 of the ENDEAVOR study for ELEVIDYS, assessing prophylactic sirolimus to reduce acute liver injury (ALI) in non-ambulant Duchenne patients.

Interim safety data from the Phase 4 ENDURE study showed zero incidence of ALI in patients treated prophylactically with sirolimus.

Financial Strength and Capital Allocation

Sarepta delivered strong financial performance in Q2, with GAAP and non-GAAP operating profitability. Cash and investments increased by $197 million to $945 million. The company's disciplined cost management led to a 44% decrease in non-GAAP R&D and SG&A expenses year-over-year.

Management reiterated its strong financial position, with the base business generating approximately $400 million in cash over the last 12 months, enabling independent funding of its promising pipeline.

EXONDYS 51 10-Year Anniversary

Sarepta celebrated the 10-year anniversary of EXONDYS 51's U.S. approval on September 19th. This milestone underscores Sarepta's leadership in Duchenne, establishing exon skipping as a foundational treatment approach. The company has built a substantial body of real-world evidence demonstrating benefits across ambulation, pulmonary and cardiac function, and survival, with over 1,800 patients treated worldwide with exon skipping therapies.

Huntington's Disease Program Progress

The CNS Huntington's program is ongoing, with the first patients dosed earlier this year. This program utilizes a transferrin receptor-based approach for blood-brain barrier delivery. Early proof of biology data is expected early next year, focusing on safety and knockdown of Huntington in the CSF, which would validate the platform's potential for CNS indications.

Summary of the company's earnings call; see how call summaries are prepared. Not investment advice.