Sarepta Therapeutics, Inc.NASDAQ
Today, 5:30 PM
- ABB, ABMD, ACOR, AET, ALLE, ALV, ALXN, AMT, APD, ASPS, AUO, AVT, AXTA, BBW, BC, BCOR, BGCP, BMS, BMY, BWA, BX, CBG, CCMP, CELG, CFX, CHH, CL, CLF, CMC, CME, CMS, COLB, COP, COR, CRI, CRR, CRS, CVE, CVI, CVRR, CWT, CYS, DFT, DLX, DOW, DPS, EME, EPD, EQGP, EQM, EQT, EXLS, F, FCFS, FCN, FMS, GCI, GLOP, GNC, GOV, GTLS, HCA, HEES, I, IDA, IDCC, IP, IPGP, IRDM, IVZ, LANC, LAZ, LKQ, LLL, MD, MDP, MDXG, MHO, MJN, MO, MPC, MPLX, MSCI, MTH, MTRN, NEWM, NMR, NOK, NOV, NTCT, ODFL, ORI, PATK, PENN, PF, PJC, POT, PRLB, PTEN, PX, QSII, RDN, RGS, ROCK, RTN, SCG, SEE, SFE, SILC, SIRI, SMP, SQNS, SRPT, STM, SUP, SWK, TCK, TDC, TFX, THRM, TKR, TMO, TPH, TPX, TREE, TROW, TUES, TWTR, TZOO, UAN, UFS, UPS, UTHR, VC, VLP, WCC, WEX, WST, WWE, XEL, XRS, YNDX
Fri, Oct. 21, 7:40 AM
- SCYNEXIS (NASDAQ:SCYX) initiated with Buy rating and $14 (272% upside) price target by H.C. Wainwright.
- Catalent (NYSE:CTLT) initiated with Overweight rating and $29 (19% upside) price target by KeyBanc.
- Coherus Biosciences (NASDAQ:CHRS) initiated with Outperform rating and $40 (32% upside) price target by Baird.
- Amarin (NASDAQ:AMRN) initiated with Buy rating and $5 (58% upside) price target by Citigroup.
- AC Immune (Pending:ACIU) initiated with Outperform rating and $18 (20% upside) price target by Credit Suisse.
- Sarepta Therapeutics (NASDAQ:SRPT) initiated with Outperform rating and $68 (39% upside) price target by Credit Suisse.
- Neurocrine Biosciences (NASDAQ:NBIX) initiated with Buy rating and $62 (36% upside) price target by Needham.
- AbbVie (NYSE:ABBV) initiated with Market Perform rating and $70 (13% upside) price target by Leerink Swann.
- Stryker (NYSE:SYK) initiated with Buy rating and $140 (22% upside) price target by SunTrust Robinson Humphrey.
Mon, Oct. 17, 9:03 AM
- The FDA denies PTC Therapeutics' (NASDAQ:PTCT) appeal of the Refuse to File letter it received in February regarding its New Drug Application (NDA) seeking approval of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). Refuse to File means that the application was not complete enough to allow for review.
- The company intends to escalate its appeal to the next supervisory level in the agency. It believes its application should be given the same opportunity for an advisory committee review that was given to Sarepta Therapeutics' (NASDAQ:SRPT) eteplirsen.
- Translarna is currently available in Europe on an annual renewal basis. In a recent oral explanation meeting with the European Medicines Agency, the Committee for Medicinal Products for Human Use (CHMP) issued a request for supplemental information, including a request categorized as a major objection which the company must adequately address in order to receive a renewal. The major objection pertains to the efficacy and overall risk-benefit profile of Translarna in addition to the design and conduct of a new clinical trial to generate comprehensive data.
- PTC believes that if CHMP issues a positive opinion backing a renewal of the marketing authorization, it will be coupled with the obligation to conduct the new study in nmDMD. An opinion should be released by year end.
- Translarna is a protein restoration therapy designed to enable the formation of a functioning protein in patients with nonsense mutation-caused genetic disorders.
- Shares are off 21% premarket on average volume.
Tue, Oct. 4, 8:58 AM
- Catabasis Pharmaceuticals (NASDAQ:CATB) completes the enrollment of 30 patients in Part B of its Phase 2 clinical trial, MoveDMD, assessing edasalonexent (CAT-1004) for the treatment of Duchenne muscular dystrophy (DMD). The primary endpoints are safety and tolerability over 12 weeks and muscle composition and inflammation as measured by MRI at Week 12. Top-line data should be available in Q1 2017.
- Part A of MoveDMD evaluated the safety, tolerability and pharmacokinetics of three regimens of edasalonexent (33 mg/kg/day, 67 mg/kg/day, 100 mg/kg/day) in 17 ambulatory DMD boys aged four to seven who were steroid-naive or had not received steroids for at least six months prior to the study. 16 of the 17 subjects are participating in Part B, a randomized, placebo-controlled trial assessing the two higher doses of edasalonexent. Part C is an open-label extension during which patients will be dosed for 36 weeks beyond Part B.
- Edasalonexent inhibits an protein activated in DMD called NF-kB which plays a key role in inflammation and fibrosis. The company recently announced a joint research collaboration with Sarepta Therapeutics (NASDAQ:SRPT) to explore a combination approach of exon skipping and NF-kB inhibition for treating DMD.
- Previously: Sarepta and Catabasis Pharma team up to explore a combo drug for Duchenne muscular dystrophy; Catabasis ahead 29% premarket (Sept. 29)
Tue, Oct. 4, 8:02 AM
- Thinly traded micro cap Summit Therapeutics (NASDAQ:SMMT) is up 49% premarket, albeit on turnover of only 600 shares, in response to its announcement that it has entered into an exclusive license and collaboration deal with Sarepta Therapeutics (NASDAQ:SRPT) granting it rights to its utrophin modulator pipeline, including lead product candidate ezutromid, for the treatment of Duchenne muscular dystrophy (DMD). Sarepta's licensed territory is Europe, Turkey and the Commonwealth of Independent States (Russia and eight former members of the Soviet bloc). It also has an option to the rights in Latin America. Summit retains commercialization rights in all other countries.
- Under the terms of the agreement, Summit will receive an upfront payment of $40M, up to $522M in milestones and escalating low-to-high-teens royalties on net sales. Sarepta and Summit will share utrophin modulator-related R&D costs on a 45%/55% basis starting in 2018. If Sarepta exercises its option on Latin America, Summit will be eligible for additional fees, milestones and royalties.
- Unlike Sarepta Therapeutics' eteplirsen, which is restricted to DMD amenable to skipping exon 51 (~13% of DMD cases), ezutromid has the potential to treat all boys and young men with DMD. It modulates utrophin, a protein functionally and structurally similar to dystrophin, a protein that helps keep muscle cells intact and whose absence causes DMD.
- The companies will host a conference call on Monday, October 10 at 12:00 pm ET to discuss the deal.
Thu, Sep. 29, 8:57 AM
- Thinly traded nano cap Catabasis Pharmaceuticals (NASDAQ:CATB) is up 29% premarket on increased volume in response to its announcement of a joint research collaboration with Sarepta Therapeutics (NASDAQ:SRPT) to investigate a combination drug approach for the treatment of Duchenne muscular dystrophy (DMD). Sarepta will contribute its expertise in exon skipping and Catabasis its know-how in oral NF-kB inhibition, specifically edasalonexent (CAT-1004).
- NF-kB, a protein activated in DMD, plays a key role in inflammation and fibrosis.
- Financial terms are not disclosed.
- Sarepta is up 1% premarket.
Wed, Sep. 28, 6:42 PM
- The first patient has been dosed in a Phase 3 clinical trial, ESSENCE, evaluating Sarepta Therapeutics' (NASDAQ:SRPT) SRP-4045 and SRP-4053 for the treatment of patients with Duchenne muscular dystrophy (DMD) amenable to exon 45 and exon 53 skipping.
- The double-blind, placebo-controlled study will randomize 99 subjects to 1:1:1 to receive once-weekly infusions of either 30 mg/kg of SRP-4045, 30 mg/kg of SRP-4053 or placebo for up to 96 weeks. The test period will be followed by an open-label extension phase in which all participants will receive active treatment for up to an additional 96 weeks.
- The primary endpoint is the change from baseline to Week 96 in the 6-minute walk test (6MWT). According to ClinicalTrials.gov, the estimated final data collection date for the primary endpoint is September 2019. The estimated study completion date is June 2021.
- SRP-4045 and SRP-4053, incorporating technology to skip exons 45 and 53, respectively, are designed to address the underlying cause of DMD by restoring the messenger RNA reading frame, thereby enabling the production of a shorter form of the dystrophin protein. Recently approved Exondys 51 (eteplirsen) is targeted to DMD patients amenable to skipping exon 51.
- Shares are up a fraction after hours.
Mon, Sep. 26, 8:10 AM
- The FDA designates Summit Therapeutics plc's (NASDAQ:SMMT) Orphan Drug-tagged ezutromid for Fast Track review for the treatment of Duchenne muscular dystrophy (DMD). Fast Track status provides for more frequent interactions with the FDA review team and a rolling review of the NDA.
- Ezutromid is currently being assessed in a Phase 2 study. Top-line data are expected in H2 2017.
- Unlike Sarepta Therapeutics' (NASDAQ:SRPT) eteplirsen or BioMarin Pharmaceutical's (NASDAQ:BMRN) drisapersen, which are restricted to DMD amenable to skipping exon 51 (~13% of DMD cases), ezutromid has the potential to treat all boys and young men with DMD. It modulates utrophin, a protein functionally and structurally similar to dystrophin, a protein that helps keep muscle cells intact and whose absence causes DMD.
- Previously: Summit moving ahead with DMD candidate, mid-stage study sites expand to the U.S. (April 26)
Sun, Sep. 25, 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.
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.
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, 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, 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.
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, 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).
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.