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]Disclosure:
1) Stephen Hytha compiled this article for Streetwise Reports LLC and provides services to Streetwise Reports as an independent contractor. He or members of his household own securities of the following companies mentioned in the article: None. He or members of his household are paid by the following companies mentioned in this article: None.
2) The following companies mentioned in this article are billboard sponsors of Streetwise Reports: None. Click here for important disclosures about sponsor fees.
3) Comments and opinions expressed are those of the specific experts and not of Streetwise Reports or its officers. The information provided above is for informational purposes only and is not a recommendation to buy or sell any security.
4) The article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports' terms of use and full legal disclaimer. This article is not a solicitation for investment. Streetwise Reports does not render general or specific investment advice and the information on Streetwise Reports should not be considered a recommendation to buy or sell any security. Streetwise Reports does not endorse or recommend the business, products, services or securities of any company mentioned on Streetwise Reports.
5) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their immediate families are prohibited from making purchases and/or sales of those securities in the open market or otherwise from the time of the interview or the decision to write an article until three business days after the publication of the interview or article. The foregoing prohibition does not apply to articles that in substance only restate previously published company releases.
6) This article does not constitute medical advice. Officers, employees and contributors to Streetwise Reports are not licensed medical professionals. Readers should always contact their healthcare professionals for medical advice.
