Sarepta Therapeutics, Inc. Q1 FY2026 earnings call

• Business stabilization: Marketed product performance stabilized. - Financial strength: Ended quarter with ~$748 million in cash and investments, positive GAAP and non-GAAP earnings, positive cash flow excluding Arrowhead-related payments. - Pipeline progress: Rapidly advancing siRNA portfolio, including DM1 and FSHD programs with early encouraging data, initiated first patient in Huntington's disease program, progressing clinical trials in SCA2 and idiopathic pulmonary fibrosis. - Alevitis initiatives: Advancing Endeavor Cohort 8 study with serolimus pretreatment, early data from real-world evidence study shows no elevated liver enzymes in pretreated patients. - PMO franchise: Durable in 2026, supported by longstanding physician experience, well-understood safety profile, and robust real-world evidence.

Total net product revenue was $331 million. $229 million from PMO therapies (Exondus, Myondus, Amondus) and $102 million from Alevitis. PMO franchise remains durable with longstanding physician experience, well-understood safety profile, and robust real-world evidence. Alevitis has over 1,300 patients treated, evidence supports disease-modifying impact, and commercial initiatives are underway to drive growth.

Reiterated full-year guidance of $1.2 to $1.4 billion and counseled prudence in raising estimates prematurely. Confident in Alevitis' potential to return to growth but recognizing the long cycle of commercial initiatives.

• Varying risks and uncertainties beyond Sarepta's control that could materially differ from forward-looking statements, adversely affecting business, results of operations, and stock trading. - Risks related to commercial execution of Alevitis, including the time it takes for initiatives to impact sales, and the need for continued education and data-driven conversations to build momentum. - Risks associated with the development and regulatory approval of pipeline programs, including uncertainties in clinical trial outcomes, data readouts, and regulatory decisions.

Q: With Salesforce expansion and ongoing initiatives to help close information gaps, comment on feedback regarding three-year Embark data and which parts of the data specifically have been resonating with physicians.

A: Patrick and others mentioned that the efficacy data and MRI data are moving the needle, with divergence over time from natural history of treated patients tied to MRI data being compelling.

Q: On Salesforce expansion and quarterly dynamics, elaborate on 1Q seasonal dynamics and appropriate run rate for 2Q and beyond for PMO franchise and Alevitis.

A: Patrick and others explained quarter-to-quarter dynamics are noisy, impacted by patient-family dynamics, and there's more variability with a one-time therapy like Alevitis.

Q: On Huntington's program, walk through what's differentiating versus other silencing approaches and what to expect from proof of biology data next year.

A: Luis explained use of TFR ligand in combination with subcutaneous injection allows getting into deep brain regions, high knockdown in deep regions, early days but looking for proof of biology in safety and activity next year.

Q: On reduction of ALI incidence in non-ambulatory cohort 8 data set, expectations and comparison to real-world settings.

A: Luis and others discussed evidence from preclinical data and real-world studies, but need to dose more patients and wait for cohort 8 readout.

Q: On serolimus study and real-world study, talk about new N of 20 Phase 4 enhanced study and its fit with regulatory strategy.

A: James explained the phase four study is looking at serolimus in broader Duchenne population, not part of specific regulatory strategy but supportive.

Q: On guidance, whether more comfortable with middle or high end given green shoots.

A: Doug explained initiatives are long-cycle, making progress but cautioning to be prudent and not changing guidance yet.

Q: On details on patient numbers at dose levels for SIRNA data by year end and functional measures.

A: Louise and James explained data from highest dose cohorts in FSHD and DM1, looking at safety, biomarkers, and early functional outcomes.

Q: On comparing SIRNA data to other products and endpoints, and PMO shift to permanent approval.

A: Louise and others discussed differentiation through alpha-b beta-6 targeting ligand, and ongoing interactions with FDA for PMO approval.

Q: On SRP1001 dose response and serolimus real-world study, comment.

A: Louise explained supralinear increase in muscle concentration and real-world study showing no evidence of liver enzyme elevations.

Q: On Cohort 8 recruitment, DM1 and FSHD functional data, and BD deals.

A: Luis and others discussed Cohort 8 on track, FSHD functional outcomes including safety, PK, PD response, and ongoing search for BD deals.

Q: On Cohort 8 recruitment timelines, DM1 and FSHD functional data, and BD plans.

A: Luis and Louise discussed Cohort 8 on track, FSHD functional outcomes being early, and active search for BD deals.

Q: On Elavides centers offering and cohort 8 regulatory path.

A: Patrick and Luis discussed 75-plus sites offering Elavides, and meeting with FDA to determine regulatory path after cohort 8 data.

Q: On return to growth for Alevitis, assume only access in ambulatory setting or resume non-ambulatory.

A: Doug explained return to growth is currently for ambulatory population, with non-ambulatory depending on cohort 8 success.

Q: On 20% of sites using serolimus and remaining 75% not, reasons.

A: Luis explained education and information flow needed, as Sarepta can't proactively promote serolimus without label.

Q: On DM1 and FSHD half-life and dosing interval implications.

A: Luis and James explained similar half-life due to same peptide, looking at dosing interval in phase three trial.

Q: On Elavitis recent fatality, details.

A: Luis explained fatality was commercially treated patient nearly 14 months post-treatment, deemed unrelated to Alevitis treatment.

Q: On AAV immunity efforts, update.

A: Luis explained preliminary data from apheresis study and study with Hansa indicating potential to reduce titers, but more clinical study needed.