Sarepta Therapeutics is entering 2026 with several potential milestones after a challenging previous year that included patient deaths and changes to its research focus. According to analysts at Jefferies, the Massachusetts-based company is looking toward a series of clinical and regulatory events that could shape its future.
Jefferies highlighted Sarepta’s partnership with Arrowhead Pharmaceuticals on small interfering RNA (siRNA) therapies. The siRNA portfolio includes SRP-1001 for facioscapulohumeral muscular dystrophy (FSHD) and SRP-1003 for myotonic dystrophy type 1 (DM1). Both are expected to deliver initial Phase I/II results in the first quarter of this year.
“These assets could drive a +25 – 50% stock move,” Jefferies stated, noting that each program represents a market opportunity exceeding $1 billion.
The company is also progressing with SRP-1005, another siRNA therapy from Arrowhead, targeting Huntington’s disease. Sarepta filed a clinical trial application earlier this month and plans to begin Phase I studies in the first half of 2026.
In addition to its siRNA pipeline, Jefferies pointed out the performance of Sarepta’s commercial portfolio, particularly its phosphorodiamidate morpholino oligomer (PMO) products. Preliminary earnings released by Sarepta show that PMO sales reached $259.2 million in the fourth quarter of 2025, an increase of 8% from the previous quarter and above consensus expectations. Full-year PMO sales were reported at $965.6 million, remaining largely unchanged compared to the prior year.
However, two PMO therapies—Vyondys 53 and Amondys 45—did not meet their goals in a confirmatory study for Duchenne muscular dystrophy (DMD). In the Phase III ESSENCE trial conducted last November, neither drug significantly improved motor function compared to placebo.
Both Vyondys 53 and Amondys 45 are exon-skipping drugs approved through the FDA’s accelerated pathway; Vyondys received approval in 2019 and Amondys in 2021. Despite failing their confirmatory trial, Sarepta continues efforts to secure full approval for these treatments. A meeting with the FDA is scheduled for February or March. Jefferies estimated that reaching an agreement with regulators could result in about a 25% increase in Sarepta’s stock price.
Last year, Sarepta reported preliminary net product revenues totaling $1.86 billion, including nearly $900 million from Elevidys—a gene therapy for DMD. In 2025, two patient deaths related to Elevidys were reported due to acute liver failure: one occurred in March and another in June. Fourth-quarter sales of Elevidys were $110 million, which was below analyst expectations of $121 million according to Jefferies’ report. Following increased safety concerns last July, Sarepta announced it would shift more focus toward developing siRNA therapies.
