Press release
Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2019 Financial Results and Recent Corporate Developments
CAMBRIDGE, Mass., Feb. 26, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases,
About this update from Sarepta Therapeutics, Inc.
[{"type":"text","content":"CAMBRIDGE, Mass., Feb. 26, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the three and twelve months ended December 31, 2019.\n “The Sarepta team made great progress in service of our vision to be one of the world’s most meaningful leaders in precision genetic medicine in 2019, and profoundly so in the fourth quarter,” stated Doug Ingram, Sarepta’s President and Chief Executive Officer. “We continued to serve the Duchenne community with EXONDYS 51, achieving revenue of $100 million in the fourth quarter and $381 million for full year 2019, a 26% increase over prior year. With our RNA platform, we also received approval and launched our second RNA therapy, VYONDYS 53, advanced our third RNA therapy, casimersen, to a rolling submission, and continued our multi-ascending dose study for our next-generation RNA technology, PPMO.” Mr. Ingram continued, “We progressed our gene therapy programs such as LGMD2E and MPS IIIA, and significantly advanced our lead gene therapy program, SRP-9001, intended to use micro-dystrophin to treat Duchenne muscular dystrophy. We have now dosed all patients in our blinded, placebo-controlled trial, Study 102, and are preparing to commence Study 301, our next placebo-controlled trial using commercial process material. And importantly, in the fourth quarter we entered into our alliance with Roche, which not only gives us a very strong balance sheet to execute our plans but also, assuming the success of SRP-9001, accelerates our mission to bring a potentially life-enhancing therapy to Duchenne patients around the world.” Fourth Quarter 2019 and Recent Corporate Developments: Received FDA Approval of VYONDYS 53 (golodirsen) Injections for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53: VYONDYS 53 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 53 skipping. This indication is based on a statistically significant increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53, which is reasonably likely to predict clinical benefit for those patients who are exon 53 amenable. ...