Sarepta Therapeutics, Inc.(SRPT)- NASDAQ
  • Today, 1:05 PM
    • Taking a victory lap for his bullish stance on Sarepta Therapeutics (NASDAQ:SRPT), Barron's Andrew Bary says there's still considerable upside even after last week's more than doubling of the stock price. The company now has a market cap of $3B, but biotechs can often be valued at 5-10x annual revenue or more, says Bary, and Exondys 51 has the potential for annual sales of $1B globally.
    • Sarepta is now one of the biotech sector's prime M&A targets, says RBC's Simos Simeonidis. upgrading to Outperform from Market perform while boosting the PT to $83 from $5 (Friday's close was $57.83). Baird's Brian Skorney says a multi-billion dollar franchise lies ahead, and jacks his PT to what could be a Street high of $102 (from $23). Already a bull prior of the FDA decision, Skorney likes the stock even more as the approval removes a major risk.
    • Bary also notes the company took a step to fortify its balance sheet by quickly raising $300M in equity, with big-timers JPMorgan and Goldman Sachs - who previously had little to to with the company - underwriting the deal.
    | Today, 1:05 PM | 11 Comments
  • Fri, Sep. 23, 8:28 AM
    • Clovis Oncology (NASDAQ:CLVS) upgraded to Outperform from Neutral by Credit Suisse. Price target raised to $41 (7% upside) from $19.
    • Sarepta Therapeutics (NASDAQ:SRPT) upgraded to Outperform from Market Perform with a $64 (5% upside) price target by Cowen and Company. Upgraded to Hold from Underperform with a $50 (18% downside risk) price target by Jefferies. Upgraded to Outperform from Market Perform with an $88 (44% upside) price target by William Blair. Upgraded to Neutral from Reduce by SunTrust Banks. Price target raised to $48 (21% downside risk) from $4. Upgraded to Market Perform from Underperform with a $50 (18% downside risk) price target by Leerink Swann. Upgraded to Buy from Neutral by Janney Montgomery Scott. Price target raised to $65 (7% upside) from $30.
    • Molina Healthcare (NYSE:MOH) upgraded to Neutral from Sell by Goldman Sachs. Price target raised to $58 (2% downside risk) from $51.
    • Tobira Therapeutics (NASDAQ:TBRA) downgraded to Hold from Buy by Cantor Fitzgerald. Price target raised to $42 (6% upside) from $19.
    • OraSure Technologies (NASDAQ:OSUR) downgraded to Neutral from Buy by BTIG Research. $8 price target removed.
    | Fri, Sep. 23, 8:28 AM | 3 Comments
  • Thu, Sep. 22, 6:08 PM
    • Sarepta Therapeutics (NASDAQ:SRPT) prices its public offering of 5.02M shares of common stock at $59.75. Underwriters over-allotment is an additional 753,138 shares. Closing date is September 28.
    • Net proceeds will fund clinical development, business development and commercialization activities and general corporate purposes.
    • Today's close was $60.97.
    | Thu, Sep. 22, 6:08 PM | 16 Comments
  • Thu, Sep. 22, 10:45 AM
    • Buoyed in large part by the FDA's approval of Sarepta Therapeutics' (SRPT +4.4%) Exondys 51 for Duchenne muscular dystrophy and Allergan's (AGN +1.9%) over-the-top acquisition of NASH player Tobira Therapeutics (TBRA +0.6%), money flow has turned decidedly bullish for biotechs. On a technical basis, the iShares Nasdaq Biotechnology ETF (IBB +0.6%) is poised for a breakout through 300, a healthy 25% turnaround from the low of ~240 in June.
    • There is a laundry list of leaders in the rally, including bluebird bio (BLUE -0.1%), Clovis Oncology (CLVS +5.4%), Puma Biotechnololgy (PBYI +3.8%), Aerie Pharmaceuticals (AERI -2.1%) and CoLucid Pharmaceuticals (CLCD -3.7%).
    | Thu, Sep. 22, 10:45 AM | 18 Comments
  • Thu, Sep. 22, 8:07 AM
    • FDA Commissioner Robert Califf sent a report to senior FDA officials on September 16, made public on the 19th, calling for a retraction of a 2013 study of Duchenne muscular dystrophy drug eteplirsen published in Annals of Neurology and funded by Sarepta Therapeutics (NASDAQ:SRPT). The study showed a treatment benefit to patients but is considered misleading.
    • In a footnote, Mr. Califf states, "In view of the scientific deficiencies identified in this analysis, I believe it would be appropriate to initiate a dialogue that would lead to a formal correction or retraction (as appropriate) of the published report."
    • The study was conducted at the Nationwide Children's Hospital in Columbus, OH. In 2014, an FDA inspection team visited the institution and the Office of Drug Evaluation director Ellis Unger noted: "We found the analytical procedures to be typical of an academic research center, seemingly appropriate for what was simply an exploratory Phase 1/2 study, but not suitable for an adequate and well-controlled study aimed to serve as the basis for a regulatory action. The procedures and controls that one would expect to see in support of a Phase 3 registrational trial were not in evidence. The immunohistochemistry images were only faintly stained and had to be read by a single technician using an older liquid crystal display computer monitor in a windowed room where lighting was uncontrolled. The technician had to suspend reading around mid-day when brighter light began to fill the room and reading became impossible.
    • FDA chief scientist Luciana Borio added, "I would be remiss if I did not note that the sponsor exhibited serious irresponsibility by playing a role in publishing and promoting selective data during the development of this product. [ ] As determined by the review team, and as acknowledged by Dr. [Janet] Woodcock, the article's scientific findings - with respect to the demonstrated effect of eteplirsen on both surrogate and clinical endpoints - do not withstand proper and objective analyses of the data."
    • Steve Usdin, Washington editor of BioCentury says the study and the FDA's approval of eteplirsen, announced on September 19, are independent events and the agency performed its own analyses of the data.
    • Annals of Neurology editor-in-chief Clifford Saper adds that his organization will engage in a scientific review of the evidence and make appropriate responses. He encourages anyone who has evidence of errors in a paper published in his journal to send the information to him and the article authors.
    | Thu, Sep. 22, 8:07 AM | 23 Comments
  • Wed, Sep. 21, 4:11 PM
    • The company is looking to raise as much as $225M in a common stock sale, with an underwriter greenshoe for up to another 15% of that amount. JPMorgan and Goldman Sachs are running the offering.
    • SRPT +0.5% after hours
    • Update: On September 22, the company announced that it priced its public offering of 5.02M shares of common stock at $59.75. Underwriters over-allotment is an additional 753,138 shares. Closing date is September 28.
    • Net proceeds will fund clinical development, business development and commercialization activities and general corporate purposes.
    • The closing price on 9/22 was $60.97.
    | Wed, Sep. 21, 4:11 PM | 20 Comments
  • Wed, Sep. 21, 8:38 AM
    • The board of Sarepta Therapeutics (NASDAQ:SRPT) appoints Edward Kaye, M.D., as President & CEO effective immediately. He had served on an interim basis since March of last year. Dr. Kaye will continue to serve as Chief Medical Officer as well.
    • Under the terms of his employment contract, he will receive an annual salary of $550K with an annual bonus target of 65% of his base salary ($357.5K).
    | Wed, Sep. 21, 8:38 AM | 7 Comments
  • Tue, Sep. 20, 3:54 PM
    • The good news keeps coming for Sarepta Therapeutics (SRPT +13.2%). Today the USPTO's Patent Trial and Appeals Board (PTAB) sided with Sarepta in its interference proceeding against BioMarin Pharmaceutical (BMRN +0.1%) related to patents covering "exon skipping."
    • A year ago, the PTAB ruled in favor of BioMarin's claims on the use of exon 51 antisense oligonucleotides to treat Duchenne muscular dystrophy (DMD) specified in its U.S. Patent Application No. 14/198,992, a decision Sarepta appealed. BioMarin licensed the patent from Academisch Ziekenhuis Leiden. The ruling included the cancellation of Sarepta's U.S. Patent No. 8,486,907, which Sarepta licensed from the University of Western Australia.
    | Tue, Sep. 20, 3:54 PM
  • Tue, Sep. 20, 9:19 AM
    | Tue, Sep. 20, 9:19 AM | 2 Comments
  • Mon, Sep. 19, 6:55 PM
    • Saying "we tried to be reasonable,", Sarepta Therapeutics (NASDAQ:SRPT) CEO Dr. Edward Kaye announces that the annual price of Exondys 51 (eteplirsen) will be ~$300K, apparently a lower price than some analysts predicted for the rare disease drug.
    • The cost is comparable to Vertex Pharmaceuticals' (NASDAQ:VRTX) cystic fibrosis med Orkambi (ivacaftor/lumacaftor), but less than Alexion Pharmaceuticals' (NASDAQ:ALXN) Soliris (eculizumab), BioMarin Pharmaceutical's (NASDAQ:BMRN) Naglazyme (galsulfase) and Vimizim (elosulfase alfa) and Shire's (NASDAQ:SHPG) Elaprase (idursulfase).
    • CureDuchenne advocacy group rep Debra Miller says, "Considering the cost of manufacturing, delivering and supporting patients (financial assistance programs), not to mention development costs over a decade, I don't think it's too much." Payer reimbursement should commence in no more than 90 days.
    | Mon, Sep. 19, 6:55 PM | 47 Comments
  • Mon, Sep. 19, 5:38 PM
    | Mon, Sep. 19, 5:38 PM
  • Mon, Sep. 19, 12:57 PM
    | Mon, Sep. 19, 12:57 PM | 1 Comment
  • Mon, Sep. 19, 12:41 PM
    • Sell-side analysts, never bashful with their pie-in-sky valuation models, are wasting little time jumping on the Sarepta Therapeutics (SRPT +90.3%) bandwagon. Piper Jaffray, William Blair and Janney Montgomery Scott have all upgraded the stock from Neutral to Buy.
    • Baird takes the top prize by raising its price target to $102 (91% upside) from $23 based, of course, on many heroic assumptions.
    • As always, retail investors should be cautious here.
    | Mon, Sep. 19, 12:41 PM | 11 Comments
  • Mon, Sep. 19, 12:14 PM
    • The stampede into Sarepta Therapeutics (SRPT +87.4%) over the FDA's approval of Duchenne muscular dystrophy (DMD) drug eteplirsen has rippled out beyond PTC Therapeutics (PTCT +26.1%).
    • Thinly traded UK-based Summit Therapeutics (SMMT +16.1%) is up on more than an 11x surge in volume, albeit on turnover of only 53K shares. Its DMD candidate is Phase 2-stage ezutromid (formerly SMT C1100), a modulator of a protein called utrophin which is structurally and functionally similar to dystrophin. The company says it has the potential to treat all young men with DMD, not just those amenable to exon 51 skipping (~13% of DMD cases).
    • PTC's DMD product is protein restoration therapy Translarna (ataluren), currently available in Europe on an annual renewal basis.
    | Mon, Sep. 19, 12:14 PM
  • Mon, Sep. 19, 11:00 AM
    | Mon, Sep. 19, 11:00 AM | 15 Comments
  • Mon, Sep. 19, 10:16 AM
    • Sarepta Therapeutics (SRPT +76%) hits a grand slam on the news that the FDA has approved eteplirsen, branded as Exondys 51, for the treatment of Duchenne muscular dystrophy (DMD).
    • The accelerated approval requires the company to conduct a new clinical trial to prove the drug's clinical benefit, specifically to assess whether Exondys 51 improves motor function in DMD patients amenable to exon 51 skipping. If the trial fails, the agency may withdraw its approval.
    • The approval process has been controversial because the efficacy data supporting the company's New Drug Application (NDA) was sparse, to say the least.
    • Potential competitor PTC Therapeutics (PTCT +16.6%) joins the party based on its DMD candidate Translarna.
    | Mon, Sep. 19, 10:16 AM | 29 Comments