Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2020 Financial Results and Recent Corporate Developments

– Net product sales for the fourth quarter and full-year 2020 of $122.6 million and $455.9 million, respectively, were pre-announced in January 2021 at the J.P. Morgan Healthcare Conference –

– Fourth quarter 2020 net product sales increased approximately 23% over the fourth quarter of 2019; full-year 2020 product revenue increased almost 20% over the prior year –

CAMBRIDGE, Mass., March 01, 2021 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the fourth quarter and full-year 2020.

“In the midst of a challenging pandemic, in 2020 the Sarepta team executed and stayed focused on the patients we serve. We advanced our multi-platform portfolio and achieved a number of important milestones, both in our gene therapy and in our RNA platform, including the submission of our FDA application for the approval of AMONDYS 45™ (casimersen), which resulted in the approval we reported last week. This is our third internally developed RNA therapy approved in the U.S. to treat Duchenne muscular dystrophy and we are now able to offer treatment to the 8% of Duchenne patients with a confirmed exon 45 amenable mutation. By providing a treatment option for patients with an exon 45 amenable mutation, AMONDYS 45 will contribute to the steady growth of our RNA product revenue, which in the fourth quarter of 2020 achieved net product sales of $122.6 million, a 23% increase over the same quarter last year and $455.9 million for full year 2020, a nearly 20% increase over the prior year,” stated Doug Ingram, Sarepta’s president and CEO. “This approval moves us closer to our goal of treating the greatest percentage of the Duchenne community as possible. Our three therapies together can treat nearly 30% of Duchenne patients in the U.S. and with our RNA-PMO technology, or its next generation version, the PPMO, we could build constructs to ultimately treat over 80% of Duchenne mutations.”

Mr. Ingram continued, “In January and at the end of the fourth quarter we reported results from two clinical trials for two of our key value-driving pipeline assets in development for Duchenne: Part 1 of Study 102 for our gene therapy SRP-9001, and Part A of the MOMENTUM study for SRP-5051, our first candidate developed using our next-generation PPMO technology. Although Study 102 did not achieve statistical significance on the primary functional endpoint, the top-line results reinforce our confidence in the potentially transformative benefits of SRP-9001 and generated key insights that will inform the protocol for our upcoming trial, Study 301. Additionally, we plan to report data in the second quarter from Study 103 called ENDEAVOR, our open-label study that is testing commercially representative SRP-9001 material. For SRP-5051 we are pleased with the higher tissue concentration, exon skipping and dystrophin production in the 20 mg/kg dosing group observed at an early 12-week timepoint at an order of magnitude lower cumulative drug exposure compared to our current PMO technology. We are on track to report data from the 30 mg/kg arm of the MOMENTUM trial in the second quarter. Additionally, later this month at the 2021 MDA Clinical and Scientific Conference, we will present the data for SRP-9001-102 Part 1 and new long-term functional data from study SRP-9003-101, our gene therapy in development for limb-girdle muscular dystrophy type 2E.”

Fourth Quarter 2020 and Recent Corporate Developments:

• Received FDA Approval of AMONDYS 45 (casimersen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 45, Sarepta’s third RNA exon-skipping treatment for DMD approved in the U.S.: AMONDYS 45 is an antisense oligonucleotide from Sarepta’s phosphorodiamidate morpholino oligomer (PMO) platform, indicated for the treatment of DMD in patients with a confirmed mutation amenable to exon 45 skipping. This indication is based on a statistically significant increase in dystrophin production in skeletal muscle observed in patients treated with AMONDYS 45, which is reasonably likely to predict clinical benefit for those patients who are exon 45 amenable. Consistent with the accelerated approval pathway, the continued approval of AMONDYS 45 may be contingent on confirmation of a clinical benefit in confirmatory trials. The ESSENCE trial, a placebo-controlled, confirmatory trial to support both the AMONDYS 45 and VYONDYS 53 approvals is ongoing and expected to conclude in 2024.
• Reported top-line results for Part 1 of Study SRP-9001-102 (Study 102) evaluating SRP-9001, Sarepta’s investigational gene transfer therapy for the treatment of Duchenne muscular dystrophy: Study 102 is a double-blind, 1:1 randomized, placebo-controlled clinical trial of SRP-9001 in 41 participants with DMD, ages of 4-7, using clinical process SRP-9001. Primary endpoints are micro-dystrophin expression at 12 weeks and change in NSAA total score at 48 weeks compared to placebo. In Part 1 results from the treatment and placebo groups are compared through 48 weeks following treatment. Data from the Part 1 of the study showed the following:
  • Met the primary biological endpoint of micro-dystrophin protein expression at 12 weeks post-treatment (P