|Bid||11.62 x 800|
|Ask||11.63 x 1200|
|Day's Range||10.45 - 12.18|
|52 Week Range||4.32 - 53.19|
|Beta (3Y Monthly)||N/A|
|PE Ratio (TTM)||N/A|
|Earnings Date||Aug 14, 2019|
|Forward Dividend & Yield||N/A (N/A)|
|1y Target Est||11.00|
Unlike commercial-stage companies that have product revenues, drug startups largely rely on private equity funding, stock sales and debt financing to fund their operations. And those operations can be pricey.
Following the near 100% rally in shares of Solid Biosciences Inc (NASDAQ: SLDB ) in August, one Citigroup analyst said the stock price is diverging from worsening fundamentals. The Analyst Joel Beatty ...
Shares of the gene therapy biotech specialist slid Thursday morning as an analyst at Citi Research argued that it was time to sell the stock.
But analysts say the rejection could have a ripple effect far outside of the walls of Sarepta's Cambridge headquarters.
(Bloomberg Opinion) -- For a small biotechnology firm, Sarepta Therapeutics Inc. has made a lot of waves at the Food and Drug Administration. The 2016 approval of its first drug, Exondys 51 – which targets the deadly muscle-wasting disease Duchenne Muscular Dystrophy (DMD) – caused a schism at the agency. A highly organized and vocal group of parents and patients saw hope for boys otherwise resigned to a short and challenging life, helping it gain green-light status, while an agency detractor called the drug an “elegant placebo.” Sarepta’s second drug-approval attempt didn’t go so well. On Monday, the FDA rejected the company’s follow-up drug Vyondys 53, intended for another subset of DMD patients. According to the surprised company, the agency’s principal concerns were with infections related to ports used to infuse the drug, and kidney toxicity observed in pre-clinical models but not seen in the actual trial.If those are the only problems, Vyondys could be back on track in relatively short order. But we don’t know if that’s all there is to it or whether there’s a simple fix because Sarepta didn’t share the FDA’s full rejection letter. The truth is, Sarepta may have a longer, rougher path to further FDA approvals than it previously thought, and its struggles could have a bearing on other drug developers in similar situations.Vyondys likely won’t be approved until at least next year now. That’s not a deal-breaker; the drug was expected to contribute only a modest portion of Sarepta’s future sales. But analysts think the rejection could be about more than a few infections, which may be the reason the company’s shares plunged as much as 20% in early trading Tuesday. Royal Bank of Canada analyst Brian Abrahams suggested in a research note Tuesday that this decision may represent a backlash to Exondys’s approval. It also could be a signal that the agency is upping its safety bar in some cases. There may be something to that. Both Exondys and Vyondys appear able to produce tiny amounts of the protein that boys with DMD lack. It isn’t clear, based on Sarepta’s small trials, that this leads to a real-world benefit. Reliable confirmatory data may not arrive for years.Advocates argue that the FDA should be biased toward approval, when there are no good options even if the data is somewhat scant. Exondys tested the limits of that argument, and it can cost as much as $1 million per year. Potential safety issues for such expensive therapies can make the push for approval harder to support.In the case of Vyondys, the FDA appears to have dug pretty deep for a problem. But if Exondys was an exception and the agency continues to apply a higher standard through the inevitable criticism, another similar medicine of Sarepta’s in development called casamirsen could be in trouble as well. Investors also have to decide if they need to worry about the company’s crucial next generation of drugs, which are gene therapies. Unlike Exondys, which needs to be dosed for years, these medicines could have a long-term impact after just one treatment. Sarepta has produced promising early results for its lead DMD gene therapy, and analysts expect it to generate a billion dollars in sales in 2021. There’s no direct relationship between Vyondys and the gene therapies; the latter work in an entirely different way and don’t have the same kind of efficacy questions. But they are still risky bets that rely on small trials and may require some degree of FDA flexibility.The agency might be in an especially cautious mood in regards to gene therapies after a damaging data fracas recently related to a highly regarded drug made by Novartis AG. Any slowdown for Sarepta would give competitors including Pfizer Inc., Solid Biosciences Inc., and Audentes Therapeutics Inc. opportunities to catch up. At least a chunk of Sarepta’s multi-billion market value likely comes from the perception that it has the magic touch at a more flexible FDA. A previous strength may be turning into a weakness.To contact the author of this story: Max Nisen at email@example.comTo contact the editor responsible for this story: Beth Williams at firstname.lastname@example.orgThis column does not necessarily reflect the opinion of the editorial board or Bloomberg LP and its owners.Max Nisen is a Bloomberg Opinion columnist covering biotech, pharma and health care. He previously wrote about management and corporate strategy for Quartz and Business Insider.For more articles like this, please visit us at bloomberg.com/opinion©2019 Bloomberg L.P.
The CRL to golodirsen NDA comes as a surprise for Sarepta (SRPT) and investors as no issues were raised by the FDA during the review period that could have lead to non-approval of the candidate.
Solid Biosciences (SLDB) amends its phase I/II gene therapy study protocol to expedite the development of its lead candidate, SGT-001, as potential treatment for DMD.
– Additional patient dosed at 2E14 vg/kg in second cohort of IGNITE DMD trial – – Study protocol amended to expedite clinical execution of SGT-001 – – Recent $60M.
Sarepta (SRPT) reports wider year-over-year loss. However, revenues increase year over year. The company plans expansion of gene therapy program into other indications including multiple sclerosis.
SVB Leerink acted as the exclusive placement agent to the Company in connection with the private placement. In this private placement, the Company is selling 10,607,525 shares of common stock at a price of $4.65 per share, representing a premium to the share price as of market close on July 25, 2019.
Sarepta's (SRPT) shares surge after safety issues are observed in Pfizer's early-stage clinical study related to its gene therapy for DMD.
Pfizer (PFE) presents early data from a study on its investigational gene therapy for DMD. However, the study highlighted some serious side effects in two out of the six participants.
Hedge funds and other investment firms that we track manage billions of dollars of their wealthy clients' money, and needless to say, they are painstakingly thorough when analyzing where to invest this money, as their own wealth also depends on it. Regardless of the various methods used by elite investors like David Tepper and David […]
Sarepta Therapeutics stock rocketed in high volume Friday after Pfizer said its gene therapy test is on pause following serious side effects in two boys with Duchenne muscular dystrophy.
Bain Capital has unveiled an approximately $1.1 billion life sciences fund, its second targeting the fast-growing industry, a spokesperson for the private equity giant said Wednesday. As a private equity firm, it generally makes later-stage investments than many of the venture capital firms that target the biotech sector.
Biotech giant Vertex Pharmaceuticals announced plans Thursday to acquire a Cambridge-based preclinical gene editing startup, giving a run for the money to fellow Cambridge drugmaker Sarepta Therapeutics and others developers of drugs for Duchenne muscular dystrophy.
NEW YORK, May 16, 2019 -- Attorney Advertising -- Bronstein, Gewirtz & Grossman, LLC is investigating potential claims on behalf of purchasers of Solid Biosciences Inc..
An analyst suggested investors look at Sarepta Therapeutics' array of gene therapies on Tuesday after rival Solid Biosciences reported its key gene therapy caused side effects in a patient.
(Bloomberg Opinion) -- It’s easy to get excited about gene therapies, a group of new medicines that have the potential to cure diseases in one treatment. The saga of Solid Biosciences Inc. is a reminder that tweaking genes isn’t a trivial task. These therapies are full of promise, but they’re not entirely there yet.
The gene therapy developer appears to be struggling to find a safe and effective dose of its experimental Duchenne muscular dystrophy treatment.