Sarepta Therapeutics CEO Doug Ingram announced Wednesday that he will retire by the end of 2026, citing a “shocking and ironic twist of fate” involving the health of family members.
After a tumultuous decade spent at the front lines of the battle against Duchenne muscular dystrophy, Ingram revealed that two of his close family members were recently diagnosed with myotonic dystrophy (DM1), another form of muscular dystrophy.
The diagnoses hit home just about a year after Sarepta entered a multiprogram partnership with Arrowhead Pharmaceuticals to develop RNA interference against rare genetic diseases of the muscles, central nervous system and the lungs, including DM1.
“Subsequent to that partnership, in a fairly shocking and certainly ironic twist of fate, my personal commitment to muscular dystrophy deepened, as two members of my immediate family have been diagnosed now with myotonic dystrophy, DM1,” Ingram said on an investor call Wednesday. “By the end of 2026, the time will have come for me to spend more time in California focusing on family commitments and addressing the realities of DM1.”
Sarepta has begun a search for a new CEO, and Ingram will stay on until either the end of 2026 or once his replacement is found, according to a securities filing (PDF).
Ingram’s departure comes at a crossroads for Sarepta, as the unwavering conviction that defined his tenure—and the Boston-area biotech itself—has materialized into significant commercial uncertainty as of late.
Following a liver safety signal and an FDA label restriction, sales of Sarepta’s DMD gene therapy Elevidys dropped to $110 million in the fourth quarter, down 16% sequentially from the prior quarter.
The underwhelming Q4 performance and Sarepta’s 2026 revenue guidance of $1.2 billion to $1.4 billion serve as a stark reality check for prior optimistic expectations surrounding Elevidys, signaling that safety concerns and the narrowed indication are taking a real toll.
“The Elevidys weakness in the fourth quarter is not an outlier but was an early signal of a materially weaker Elevidys trajectory in 2026,” William Blair analyst Sami Corwin, Ph.D., wrote in a Thursday note.
The tempered guidance is related to Elevidys more than Sarepta’s exon-skipping portfolio, namely Exondys 51, Vyondys 53 and Amondys 45, Ingram said on Wednesday's call, as he characterized the three phosphorodiamidate morpholino oligomer (PMO) therapies as “very stable.”
In 2025, the three exon-skipping drugs generated $966 million in sales. Assuming their stable performance going forward, Sarepta’s guidance indicates Elevidys sales will drop below the $500 million-per-year floor that the company had previously presented. Leerink Partners analysts are modeling 2026 Elevidys sales of just $387 million, according to a Wednesday note.
After a regulatory tug-of-war that briefly caused a complete halt to Elevidys deliveries, the FDA in November officially limited the label of Elevidys to only allow the one-time gene therapy to treat ambulatory DMD patients who are at least 4 years old. The move came after two non-ambulatory patients died from acute liver failure after receiving Elevidys.
Despite the disappointing 2026 guidance, Ingram suggested that Elevidys’ long-term value remains strong.
“While we are being disciplined in our near-term projections, our confidence in the ultimate demand for Elevidys and the transformational nature of this therapy remains exceptionally strong,” the outgoing CEO said.
His stated confidence is rooted in long-term data, including the three-year update from the phase 3 Embark trial, as well as education initiatives, which Ingram suggested will take time.
“There is another big issue […] of educating and information, and that is—and this I find really troubling—that you have a much better chance of getting infused with Elevidys if you are middle class, English-speaking, well-educated,” Ingram said. “There’s a real delta that exists where people that are not in the middle class, Spanish speakers, other people in a different socioeconomic environment, just may not even have access to any information to be able to make thoughtful decision about their therapy. We are going to address that this year.”
Meanwhile, more concerns are piling onto Sarepta’s business. Even though Sarepta projected a stable exon-skipping business performance, William Blair’s Corwin said her team is “hesitant” to assume this will occur out of concerns that Vyondys and Amondys may lose their marketing authorizations. The nine-year Essence trial recently failed to show a statistically significant difference for the two meds compared to placebo on a key DMD patient mobility metric.
Despite this result, Sarepta plans to meet with the FDA late in the first quarter to discuss the Essence data and the potential conversion of their accelerated approvals to full nods.
During Wednesday’s call, Ingram again pointed to real-world evidence showing the two drugs’ disease-moderating benefits across measures, arguing that Essence is “supportive of the risk-benefit of these therapies.”
“Patients and their physicians see significant value in the PMOs, as represented by year-after-year compliance rates consistently over 90%,” Ingram said. “Indeed, families are fiercely supportive of these therapies.”
Staunch patient support kept Elevidys on the market even though the FDA had planned to enforce a full withdrawal. Patient advocacy also played a key part in the contentious initial approval of Exondys in the fall of 2016, less than a year before Ingram’s migration from Allergan to Sarepta.
Under his tenure, Sarepta powered through the additional introduction of Vyondys, Amondys and Elevidys, holding its ground even as the drugs failed to deliver definitive efficacy data proving their benefit toward slowing DMD progression or as serious safety concerns emerged.
Soon, Sarepta may face more hurdles than the FDA as competition lines up in DMD. Dyne Therapeutics plans to file for accelerated approval of its exon 51 skipper, z-rostudirsen, in the second quarter.