Yesterday evening, developer and marketer of precision genetic medicines for rare diseases Sarepta Therapeutics Inc. (SRPT:NASDAQ), announced that the U.S. Food and Drug Administration (FDA) has approved its second RNA exon-skipping treatment drug VYONDYS 53 (golodirsen). The company explained that "VYONDYS 53 is an antisense oligonucleotide from Sarepta's phosphorodiamidate morpholino oligomer (PMO) platform, indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping."
The firm stated in the report that commercial distribution of VYONDYS 53 in the U.S. will commence immediately; however, the continued approval of VYONDYS 53 may be subject to confirmation of a clinical benefit in its post-marketing confirmatory trial. The ESSENCE trial to support VYONDYS 53's accelerated approval is currently enrolling patients and expected to conclude by 2024.
Sarepta's President and CEO Doug Ingram commented, "Today is monumental for Sarepta and, more importantly, for the DMD community...VYONDYS 53, our second approved exon-skipping RNA therapy for DMD, may treat up to 8% of the DMD community, representing those patients who have a confirmed exon 53 amenable mutation. Along with EXONDYS 51 (eteplirsen), we now offer treatment options for approximately 20% of those with DMD in the United States...In the span of four months, we commenced and completed the formal dispute resolution process culminating in the grant of our appeal, resubmitted our NDA and obtained an approval which is a great benefit to DMD patients awaiting treatment."
Pat Furlong, founding president and CEO of Parent Project Muscular Dystrophy (PPMD), remarked, "With the approval of VYONDYS 53, up to another 8% of Duchenne families will have a therapy to treat this devastating disease...For 25 years, PPMD has been working with researchers, clinicians, industry, and the Duchenne community to find treatments for all people living with Duchenne. And while we need to ensure that these approved therapies are accessible for patients, today we celebrate this approval and thank Sarepta for their continued leadership in the fight to end Duchenne."
In a separate release this morning, the company announced that it has entered into an agreement with funds managed by Pharmakon Advisors, LP that will provide Sarepta with up to $500 million of borrowing capacity in two tranches. The first $250 million tranche will be available shortly after closing in December 2019, and the remaining $250 million tranche available at Sarepta's option by December 31, 2020. The funding of the second tranche is subject to certain unnamed conditions. The credit facility will mature in 48 months from the first tranche closing date and the interest rate for the funding is fixed at 8.5% annually, payable quarterly.
Sarepta Therapeutics, which is based in Cambridge, Mass., states that its mission is to profoundly improve and extend the lives of patients with rare genetic-based diseases. The company claims that "it is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for 6 Limb-girdle muscular dystrophy diseases (LGMD), Charcot-Marie-Tooth (CMT), MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development."
Sarepta started today with a market capitalization of about $7.5 billion with approximately 74.54 million shares outstanding and a fairly large short position of around 19.7%. SRPT shares opened nearly 25% higher today at $125.00 (+$24.53, +24.42%) compared to yesterday's $100.47 closing price. The stock has traded today between $125.50 and $138.79 per share and at present is trading at $137.02 (+$36.55, +36.38%).[NLINSERT]
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