- Total revenues, which consist of net product revenues and collaboration revenues, for the second quarter 2022 totaled $233.5 million
- Net product revenues for the second quarter 2022 totaled $211.2 million, a 49% increase over the same quarter of prior year
- Increased full-year total revenue and net product revenue guidance to a range of $905-$920 million and $825-$840 million, respectively
CAMBRIDGE, Mass., Aug. 02, 2022 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the second quarter 2022.
“Our performance so far this year represents the culmination of years of dedicated patient-centered execution. Over the course of the second quarter, we engaged in an in-depth and cross-departmental engagement with the FDA about the possibility of submitting a biologics license application (“BLA”) for the accelerated approval of our gene therapy, SRP-9001, to treat ambulatory Duchenne patients. Supported by a wealth of safety, biomarker, and functional data, including new analyses presented at the 2022 International Congress on Neuromuscular Diseases in early July, we received the necessary written feedback from the FDA that gives us conviction to submit our BLA in the fall of this year,” said Doug Ingram, president and chief executive officer of Sarepta. “If successful, our BLA would be approved by the middle of 2023 and represents a seminal moment in the treatment of Duchenne muscular dystrophy and an important milestone in the field of gene therapy. At the same time, we have remained dedicated to serving the Duchenne community with our three currently approved RNA-based PMO therapies, reaching total revenues of $233.5 million and net product revenues of $211.2 million in the second quarter, a nearly 50% growth over the second quarter of last year. As a result of our overperformance, we are updating our full-year guidance for net product revenue to a range of between $825 million and $840 million.”
Second Quarter 2022 and Recent Developments:
- Sarepta announces intent to submit an accelerated approval Biologics License Application (BLA) for SRP-9001: At the end of July 2022, following FDA feedback from a thorough and in-depth review, the Company announced that this fall it intends to submit a BLA for SRP-9001, their gene therapy (delandistrogene moxeparvovec) to treat Duchenne muscular dystrophy. SRP-9001 was granted Fast Track designation in July 2020. In addition to Fast Track, SRP-9001 has also been granted Rare Pediatric Disease (RPD) designation in the United States, and Orphan Drug status in the United States, the European Union, Switzerland and Japan.
- Sarepta and partner Roche presented new data for investigational gene therapy SRP-9001 at the 2022 International Congress on Neuromuscular Diseases (ICNMD 2022): In early July 2022, the company shared new functional data across multiple studies from the clinical development program for SRP-9001 (delandistrogene moxeparvovec) for the treatment of Duchenne muscular dystrophy. Key findings included new, one-year functional results from Study SRP-9001-103 (ENDEAVOR), which employs commercially representative SRP-9001 material at the target commercial dose. This is the same material currently being used in the ongoing trial, Study SRP-9001-301 (EMBARK), Sarepta’s global, randomized, double-blind, placebo-controlled clinical trial evaluating SRP-9001 in boys with Duchenne between the ages of 4 to 7 (n=120). Additionally, the Company presented long-term functional results from Study SRP-9001-101 four years after treatment with SRP-9001 along with results from an integrated analysis of Studies 101, 102 and 103 versus propensity weighted external control. These new analyses showed that SRP-9001 treated patients demonstrated statistically significant and clinically meaningful benefit versus propensity-matched external controls and results positively diverge from the natural history of this degenerative disease over time. The safety and tolerability profile of SRP-9001 remains consistent across treated patients.
Results from Study SRP-9001-103, ENDEAVOR (commercially representative material):
- In Cohort 1 (n=20, ages 4 to 7), SRP-9001-treated patients demonstrated a 3.8-point improvement (unadjusted means) and a 3.2-point improvement (least squared means) on the NSAA one-year after treatment when compared to a propensity-score weighted external control (p=0.0001).
- At one year, using unadjusted means, NSAA total scores in the SRP-9001 treated patients improved 4 points from 22.1 to 26.1 and participants in the external control improved 0.2 points from 21.9 to 22.1.
- At one year, using least squared means, NSAA total scores in the SRP-9001 treated patients improved 3.9 points and participants in the external control improved 0.8 points (p
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