Tue, May 19, 5:40 PM
Tue, May 19, 5:40 PM
- Based on guidance from the FDA provided in a pre-New Drug Application (NDA) meeting, Sarepta Therapeutics (NASDAQ:SRPT) will begin a rolling NDA submission this week for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD). It will file the non-clinical and CMC (Chemistry, Manufacturing and Controls) components by the end of this week and the final component (clinical data) by mid-year.
- Interim CEO Edward Kaye, M.D., says, "We will initiate a rolling NDA submission to facilitate the regulatory review of the submission. [It] marks a significant milestone for the Duchenne community and we look forward to completing the submission by the middle of the year and to continuing to work with the Agency towards the goal of providing treatments to patients as quickly as possible."
- Eteplirsen is designed to skip exon 51 in the dystrophin gene, an essential protein in muscle fiber function. A mutation in dystrophin causes DMD. Approved therapies will command premium prices so a number of companies are racing to the regulatory finish line.
- SRPT is up 38% after hours on robust volume.
- DMD-related tickers: (OTCPK:SPHDF) (NASDAQ:PTCT) (NASDAQ:CAPR) (NASDAQ:SMMT) (NYSE:PFE) (NASDAQ:RNA) (NASDAQ:ROSG) (OTCQB:MRNA) (NASDAQ:BMRN)
Sun, May 10, 7:06 AM
- The World Health Organization has declared Liberia free of Ebola, marking the end of a national outbreak that infected as many as 400 new victims a week at its peak.
- Liberia has now gone 42 days - twice Ebola's maximum incubation period - since the burial of its last confirmed patient without discovering a new case.
- The disease is still spreading in Sierra Leone and Guinea, though at a slower pace. According to WHO statistics, more than 11,000 people have died from the virus, with about half of them in Liberia.
- Ebola-related stocks: JNJ, GSK, HEB, BCRX, NLNK, NNVC, INO, TKMR, OTCQB:GOVX, SRPT, CMRX, PLX, NSPH, LAKE, APT, VSR, SMED.
Thu, May 7, 12:32 PM
- Sarepta Therapeutics (SRPT +1.4%) upgraded to from Neutral to Outperform with a $20 (34% upside) price target by Baird.
- Eli Lilly (LLY +0.1%) upgraded from Neutral by Citigroup.
- Synageva Biopharma (GEVA +3.6%) downgraded to Neutral with $226 (8% upside) price target by Wedbush.
- Sucampo Pharmaceuticals (SCMP -16.7%) downgraded to Hold with $18 (21% upside) by Cantor Fitzgerald.
- OraSure Technologies (OSUR -14.6%) downgraded to Market Perform by JMP Securities.
- PTC Therapeutics (PTCT -0.1%) downgraded to Neutral with a $63 (15% upside) price target by Roth Capital.
- PerkinElmer (PKI +0.2%) downgraded to Sector Weight by KeyBanc.
- Varian Medical Systems (VAR +0.9%) downgraded to Neutral with a $90 (3% upside) price target by JP Morgan.
- Agios Pharmaceuticals (AGIO +1.8%) downgraded to Hold with a $103 (5% upside) price target by Canaccord.
Thu, May 7, 10:23 AM
Thu, May 7, 7:04 AM
Fri, May 1, 5:36 PM
Mon, Apr. 27, 10:24 AM
- BioMarin Pharmaceuticals (BMRN +3.5%) concludes its rolling New Drug Application (NDA) with the FDA, as provided under Fast Track and Breakthrough Therapy designations, for drisapersen for the treatment of Duchenne muscular dystrophy (DMD), a fatal genetic disorder affecting ~20K children worldwide each year.
- DMD, usually affecting boys, is caused by a defective gene for dystrophin, an essential muscle protein.
- Drisapersen induces the skipping of dystrophin exon 51 which restores the proper dystrophin reading frame in ~13% of DMD patients. The FDA has also tagged it an Orphan Drug for DMD, which provides a seven-year period of market exclusivity for the indication, if approved.
- DMD-related tickers: (OTCPK:SPHDF)(PTCT -2.1%)(SRPT +2.2%)(CAPR -1.2%)(SMMT +0.9%)(PFE +0.4%)(RNA)(ROSG)(OTCQB:MRNA)
Thu, Apr. 23, 10:44 AM
- Adam Feuerstein notes that Santhera (OTCPK:SPHDF) isn't getting much attention from investors, despite the fact that it is the only company with a successful phase III study in treating Duchenne muscular dystrophy.
- High-profile pharma companies pursuing approval for DMD drugs include BioMarin (NASDAQ:BMRN), PTC Therapeutics (NASDAQ:PTCT), and Sarepta (NASDAQ:SRPT).
- On Friday, results from the positive phase III study involving Santhera's DMD drug idebenone will be presented at the American Academy of Neurology's annual meeting. Earlier this week, the study was published in The Lancet.
- If idebenone is approved in the U.S. and Europe for DMD, peak sales could exceed $500M according to RBC Capital analyst Simos Simeonidis. He also believes Santhera is likely to put itself up for sale if idebenone is approved.
- Related: Sarepta's Long Road Ahead (Apr. 6)
- Related: BioMarin: Expect First-Pass Drisapersen Approval (Mar. 16)
- Related: RBC's initiation of coverage report on SPHDF (Jan. 22)
Wed, Apr. 22, 12:56 PM
- The FDA designates Capricor Therapeutics' (CAPR +11.3%) lead product candidate, CAP-1002, an Orphan Drug for the treatment of Duchenne muscular dystrophy, a genetic disorder characterized by progressive muscle degeneration and weakness.
- CAP-1002, currently in Phase 2 development, is an "off-the-shelf" cell therapy that is derived from donor heart tissue and is infused directly into a patient's coronary artery via a catheter. In preclinical models, cardiosphere-derived cells have been shown to promote cardiomyogenesis (formation of new heart muscle) and angiogenesis (formation of new blood vessels) while inhibiting oxidative stress, inflammation and fibrosis.
- Duchenne-related tickers: (SMMT -6.8%)(PTCT +0.2%)(SRPT +0.1%)(BMRN)(PFE +0.1%)(RNA)(ROSG -1.9%)(OTCQB:MRNA +1.4%)
Wed, Apr. 1, 9:13 AM
Tue, Mar. 31, 9:54 PM
- The Board of Directors at Sarepta Therapeutics (NASDAQ:SRPT) appoints Chief Medical Officer Edward Kaye, M.D., as interim CEO. He replaces Christopher Garabedian who resigned effective immediately. Dr. Kaye will serve in a dual capacity until a new CEO is hired.
- Interim Chairman John Hodgman says, "We believe this change will facilitate the company's clinical and regulatory discussions and relationships with the goal of meeting its stated timelines for bringing a potentially disease-modifying treatment [eteplirsen] to patients with DMD as soon as possible."
Thu, Feb. 26, 7:02 AM
Mon, Feb. 23, 2:08 PM
- Oxford, U.K.- based Summit Therapeutics (Pending:SMMT) is set for its IPO of 3.45M American Depositary Shares. At the currently traded price in London, it will raise $40M.
- The biopharmaceutical firm focuses on discovering and developing novel medicines for indications for which there are no existing or only inadequate therapies. Its lead program for Clostridium difficile infection is SMT19969, an orally administered small molecule antibiotic in Phase 2 development. It is designed to selectively target C. diff bacteria without causing collateral damage to the gut flora. It expects to report top-line results in H2.
- Its lead product for Duchenne muscular dystrophy (DMD) is a utrophin modulator, Orphan Drug-designated SMT C1100, also an orally administered small molecule. It maintains the production of utrophin to compensate for the absence of dystrophin in DMD patients which protects healthy muscle function. Top-line results from a Phase 1b trial are expected in Q3.
- Financial Performance (nine months ended October 31, 2014) ($000): Operating Income: 2,566 (+16.0%); Operating Expenses: 17,240 (+89.5%); Net Loss: (13,363) (-118.8%); CF Ops: (12,126) (-126.0%).
- DMD-related tickers: (NASDAQ:PTCT) (NASDAQ:SRPT) (NASDAQ:BMRN)
Fri, Feb. 20, 7:15 AM
- The World Health Organization (WHO) approves the use of a 15-minute point-of-care test developed by Corgenix Medical (OTCQB:CONX) to detect the presence of the Ebola virus. The company developed the assay with funding assistance of the Bill & Melinda Gates Foundation and the Paul G. Allen Family Foundation.
- Procurement and roll-out in Africa will begin in a few weeks pending the completion of administrative procedures with the U.S. FDA.
- Ebola-related tickers: (NYSE:MRK) (NASDAQ:NLNK) (NYSEMKT:IBIO) (NASDAQ:NVAX) (NYSE:GSK) (NYSE:JNJ) (NASDAQ:TKMR) (NYSEMKT:HEB) (NASDAQ:BCRX) (NASDAQ:SRPT) (NASDAQ:CMRX) (NASDAQ:ABIO) (OTCQB:AEMD) (OTCPK:BVNRY) (NASDAQ:INO) (OTCQX:RHHBY)
Tue, Feb. 17, 12:44 PM
SRPT vs. ETF Alternatives
Other News & PR