|Bid||8.22 x 900|
|Ask||8.65 x 2200|
|Day's Range||8.45 - 9.34|
|52 Week Range||4.32 - 54.54|
|Beta (3Y Monthly)||N/A|
|PE Ratio (TTM)||N/A|
|Forward Dividend & Yield||N/A (N/A)|
|1y Target Est||N/A|
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 firstname.lastname@example.orgTo contact the editor responsible for this story: Beth Williams at email@example.comThis 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.
(Bloomberg) -- Sarepta Inc.’s bizarre plunge last week over an “erroneous” safety report only galvanized the one analyst on Wall Street who isn’t bullish on the stock.Oppenheimer’s Hartaj Singh remains skeptical of the company’s valuation, aspirations and the lack of disclosures, which came to a head after one of its drugs was cited in a government database tracking adverse events. That posting triggered a sell-off in the stock even though Sarepta said the safety signal wasn’t submitted by an employee or the principal investigator leading the drug’s clinical trial.A broader lack of transparency surrounding Sarepta’s gene therapy trials in Duchenne muscular dystrophy has stoked questions from investors, according to Singh. The company has run its trials at one center, while peers have expanded to multiple sites.“People are puzzled with why the data hasn’t been presented” by a person that isn’t associated with the company, said Singh, the only analyst tracked by Bloomberg who doesn’t have a buy rating on the stock. Lack of clarity about the data combined with an escalating market valuation that now tops $9 billion -- 16th-largest in the Nasdaq Biotechnology Index -- suggests Sarepta investors are “pricing in perfection at these levels,” he said.“The study is ongoing and blinded and will remain blinded -- and that is why the data haven’t been presented,” a Sarepta spokeswoman commented by telephone.Last week’s safety posting followed at the heels of Sarepta announcing that it was increasing the number of patients in a closely watched study and delaying the start of a trial that would use commercial scale of its gene therapy.A key for bullish investors had been the drug’s superior safety profile and more proof of clinical benefit versus competitors like heavyweight Pfizer Inc. and small-cap Solid Biosciences Inc. At the same time, Sarepta has only treated “four boys over the last few years,” Singh said. Potential regulatory clearance isn’t likely until late 2021 if the company gets accelerated approval, or could take until 2023 while the company gathers more data, he said. Some other analysts expect sales could start in 2021.“We have initiated and completed dosing 24 patients in the Phase 2 study and are dosing up to 40 total patients while doing the manufacturing work to start the Phase 3 trial with commercial drug in early 2020,” the company’s spokeswoman said. “We’re moving with incredible urgency.”Singh said in a phone interview that gene therapy is “much more complicated and going to take a lot more time than people are expecting, and we’ve said that since April. The gap between Sarepta and Pfizer is probably in months as opposed to the current thinking that it’s in years.”To be sure, manufacturing concerns have long been a bear argument that buy-rated analysts have dismissed as irrelevant. Sarepta said setbacks for Pfizer and Solid Biosciences helped persuade the company to spend more time optimizing its commercial production capabilities. And Wall Street bulls say that will help reduce risks for the program, which is the first of its kind.While focus has been on Sarepta and its gene therapy portfolio, the drugmaker already sells Exondys 51 for patients with DMD, and may add another drug to its arsenal next week with the upcoming FDA decision for Vyondys 53 (formerly golodirsen). That drug would have the potential to treat about 8% of boys with exon 53-skipping DMD. Analysts expect it could bring in $217 million in sales by 2022 if approved, compared to $1.5 billion for the gene therapy and $496 million for Exondys 51, data compiled by Bloomberg show.(Updates with company comments in the fifth and eighth paragraphs.)To contact the reporter on this story: Bailey Lipschultz in New York at firstname.lastname@example.orgTo contact the editors responsible for this story: Catherine Larkin at email@example.com, Steven FrommFor more articles like this, please visit us at bloomberg.com©2019 Bloomberg L.P.
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.
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.
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.
Solid Biosciences said in the earnings release that, among the two patients randomized in the second cohort of the Phase 1/2 study dubbed IGNITE, one was dosed with 2E14 vg/kg of its DMD gene therapy candidate and another was added to the control group. The patient in the treatment arm experienced a transient decline in platelet count, considered a serious adverse event, or SAE, related to the study. The patient also experienced a transient elevation of transaminases as well as a transient increase in bilirubin, higher than two times the upper limit of normal, which was reported as an SAE related to the study, the company said.
Here's a roundup of top developments in the biotech space over the last 24 hours. Scaling The Peaks (Biotech stocks hitting 52-week highs on May 13) BioSig Technologies Inc (NASDAQ: BSGM ) Milestone Pharmaceuticals ...
How do you pick the next stock to invest in? One way would be to spend hours of research browsing through thousands of publicly traded companies. However, an easier way is to look at the stocks that smart money investors are collectively bullish on. Hedge funds and other institutional investors usually invest large amounts of […]
Wave Life Sciences (WVE) announces final safety data from a phase I DMD study evaluating suvodirsen and provides design update for phase II/III stage development of the candidate.
Bain Capital is in the process of raising money for a second life sciences fund, less than two years after it amassed $720 million for its inaugural fund targeting the fast-growing sector. Bain revealed the new fund in a securities filing last week. The filing does not say how much the Boston-based private equity firm is hoping to raise.
Gene therapy continues to be in focus given the recent spate of deals and acquisitions. We highlight four stocks, which have promising gene therapy candidates in their pipeline.
With one drug already being considered by the FDA, Sarepta Therapeutics Inc. is now planning to submit another drug for Duchenne muscular dystrophy to the FDA this year.