Yesterday, 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| Comment!
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
Tue, Feb. 17, 9:19 AM| 3 Comments
Tue, Feb. 17, 9:14 AM
Thu, Jan. 22, 12:12 PM
- Thinly-traded micro cap NanoViricides (NNVC +20.5%) moves up in early trading on higher volume albeit on modest turnover of 230K shares. Before the open, the company announced that it shipped several anti-Ebola nanoviricide drug candidates to an unnamed "hi-security bio-containment facility" for evaluation.
- The company states that it progressed from design to synthesis of the drug candidates in only four months using its rapid design platform.
- Related tickers: (TKMR +0.2%)(BCRX -2.9%)(HEB -3.2%)(INO -2.3%)(SRPT -0.4%)(CMRX -1.4%)(ABIO -1.6%)
Mon, Jan. 19, 5:38 PM
- The FDA grants Fast Track status to privately-held Marathon Pharmaceuticals' corticosteroid MP-104 (deflazacort) 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 New Drug Application (NDA).
- DMD-related tickers: (NASDAQ:SRPT) (NASDAQ:PTCT) (NYSE:PFE) (NASDAQ:RNA) (OTCQB:MRNA) (NASDAQ:ROSG)
Mon, Jan. 12, 1:09 PM
- Sarepta Therapeutics (SRPT -17.9%) heads south on double normal volume after the company reported data from Week 168 from Study 202, a Phase 2b open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). After 3+ years of treatment, all patients were able to complete the 6-minute walk test, but all showed a decline in distance since Week 144. The average decline was almost 20%.
Mon, Jan. 12, 12:46 PM
Dec. 23, 2014, 10:20 AM
- PTC Therapeutics (PTCT -4.4%) commences its rolling New Drug Application (NDA) submission for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The company expects to finalize the application in Q4 2015 following the completion of the confirmatory Phase 3 trial.
- DMD-related tickers: (PFE -2.2%)(SRPT -4.2%)(RNA -0.2%)(OTCQB:MRNA)(ROSG +0.4%)
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