Sarepta Therapeutics, Inc. (SRPT) - NASDAQ
  • Aug. 25, 2015, 5:33 PM
    • Sarepta (NASDAQ:SRPT) states the FDA has "filed [a] New Drug Application (NDA) for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping." The company adds eteplirsen has been granted Priority Review status.
    • Interim CEO Edward Kaye: "We believe eteplirsen has the potential to make a meaningful impact on the lives of patients amenable to skipping exon 51 and we aim to build on our experience with eteplirsen to work with the FDA to inform and potentially expedite the clinical and regulatory pathway for the follow on exons, with the goal of reaching as many patients amenable to exon skipping as possible."
    • Sarepta is just 4 days removed from receiving Rare Pediatric Disease designation for eteplirsen from the FDA. Shares have risen to $34.10 after hours.
    | Aug. 25, 2015, 5:33 PM | 9 Comments
  • Aug. 21, 2015, 9:04 AM
    • The FDA grants Rare Pediatric Disease designation for Sarepta Therapeutics' (NASDAQ:SRPT) eteplirsen for the treatment of Duchenne muscular dystrophy. Under the designation, the regulatory clearance of eteplirsen will trigger the award of a Rare Pediatric Disease Priority Review Voucher to the company. This provides for Priority Review for a future marketing application after giving the FDA a 90-day notice of its intention to use it, a valuable benefit. The voucher is an asset that can be sold an unlimited number of times. AbbVie just bought one from United Therapeutics for $350M.
    • The FDA granted the same Rare Pediatric Disease designation for BioMarin Pharmaceutical's drisapersen two days ago.
    • Previously: BioMarin's drisapersen tagged a treatment for a rare pediatric disease; its fifth unique FDA designation (Aug. 19)
    • Previously: United Therapeutics sells Priority Review Voucher to AbbVie for $350M (Aug. 19)
    | Aug. 21, 2015, 9:04 AM
  • Aug. 6, 2015, 3:17 PM
    • Sarepta Therapeutics (SRPT +5.8%) is up on average volume which is notable considering that the iShares Nasdaq Biotech Index Fund (IBB -3.8%) is down almost 4% and the Nasdaq nearly 2%.
    • The company completed its rolling NDA submission for lead product candidate eteplirsen for the treatment of Duchenne muscular dystrophy in late June. The FDA has 60 days to confirm its completeness for review which should happen in a few weeks.
    | Aug. 6, 2015, 3:17 PM | 1 Comment
  • Aug. 6, 2015, 10:25 AM
    • Sarepta Therapeutics (SRPT +0.7%) Q2 results: Revenues: $0; R&D Expense: $29.2M (+41.7%); SG&A: $12.9M (+5.7%); Operating Loss: ($42.1M) (-38.9%); Net Loss: ($41.9M) (-23.6%); Loss Per Share: ($1.01) (-18.8%); Quick Assets: $156.9M (-25.4%).
    • No guidance given.
    | Aug. 6, 2015, 10:25 AM | 1 Comment
  • Aug. 6, 2015, 7:10 AM
    • Sarepta Therapeutics (NASDAQ:SRPT): Q2 EPS of -$0.87 beats by $0.34.
    • Revenue of $0 misses by $0.1M.
    | Aug. 6, 2015, 7:10 AM
  • Aug. 5, 2015, 11:25 AM
    • Based on a positive pre-NDA meeting with the FDA, privately-held Marathon Pharmaceuticals begins the New Drug Application process seeking approval for Fast Track- and Orphan Drug-tagged deflazacort (MP-104) for the potential treatment of Duchenne muscular dystrophy (DMD).
    • The company expects to submit its NDA in Q1 2016. If all goes well, marketing clearance could happen in Q1 2017, although it can request that the FDA designate it for Priority Review which would shorten the clock to six months from 10.
    • Marathon also seeks authorization from the FDA to provide deflazacort at no charge as an investigational drug to qualified DMD patients via an expanded access program. The agency stated that it may formally authorize the program. A decision could happen in August or September.
    • Deflazacort is a glucocorticoid with anti-inflammatory and immunosuppressive properties. It is not currently available in the U.S. but is approved for a variety of non-DMD indications ex-U.S.
    • DMD-related tickers: (FGEN +0.5%)(PFE)(SRPT +2.5%) (NASDAQ:RNA) (PTCT +1.5%)(OTCQB:MRNA +1.4%)(CAPR -3.5%)(OTCPK:SPHDF)(BMRN +1%)(SMMT -2.2%)
    | Aug. 5, 2015, 11:25 AM
  • Jul. 27, 2015, 4:27 PM
    • The FDA approves FibroGen's (NASDAQ:FGEN) IND for the study of FG-3019 in Duchenne muscular dystrophy (DMD). The company intends to start a multi-site Phase 2 trial in non-ambulatory DMD patients later this year.
    • FG-3019, one of the company's two lead product candidates, is a fully human monoclonal antibody that inhibits connective tissue growth factor (CTGF), a common mediator of fibrotic disease. CTGF is a protein found in the extracellular matrix that plays a key role in many biological processes including cell adhesion, migration, proliferation and angiogenesis. It is also being investigated for the treatment of ideopathic pulmonary fibrosis and pancreatic cancer.
    • FibroGen will have to hustle despite its non-ambulatory focus. The list of companies working on DMD-targeted therapies is quite long: (NYSE:PFE) (NASDAQ:SRPT) (NASDAQ:RNA) (NASDAQ:PTCT) (OTCQB:MRNA) (NASDAQ:CAPR) (OTCPK:SPHDF) (NASDAQ:BMRN) (NASDAQ:SMMT)
    | Jul. 27, 2015, 4:27 PM | 7 Comments
  • Jul. 6, 2015, 8:25 AM
    | Jul. 6, 2015, 8:25 AM | 1 Comment
  • Jul. 1, 2015, 5:36 PM
    | Jul. 1, 2015, 5:36 PM | 8 Comments
  • Jun. 30, 2015, 5:36 PM
    | Jun. 30, 2015, 5:36 PM | 14 Comments
  • Jun. 30, 2015, 9:19 AM
    | Jun. 30, 2015, 9:19 AM
  • Jun. 29, 2015, 11:30 AM
    • The FDA accepts under Priority Review BioMarin Pharmaceutical's (BMRN -0.5%) New Drug Application (NDA) for drisapersen for the treatment of Duchenne muscular dystrophy (DMD). The PDUFA date is December 27.
    • Nipping at BioMarin's heels is Sarepta Therapeutics (SRPT -8.4%) which completed its rolling NDA for eteplirsen on June 26 with a request for Priority Review.
    • Both drug candidates are Fast Track- and Orphan Drug-designated by the FDA. They have the same mechanism of action: skipping exon 51 in the dystrophin gene, an essential protein in muscle fiber function. A mutation in dystrophin causes DMD.
    | Jun. 29, 2015, 11:30 AM | 2 Comments
  • Jun. 25, 2015, 4:32 PM
    • The European Medicines Agency (EMA) accepts for review BioMarin Pharmaceutical's Marketing Authorization Application (MAA) for drisapersen for the treatment of Duchenne Muscular Dystrophy (DMD). This starts the formal review process. The target date for Day 120 questions is October 22. A potential CHMP (Committee for Medicinal Products for Human Use) decision could happen in H1 2016 followed by a decision by the European Commission 60 days later.
    • Drisapersen is an antisense oligonucleotide drug that induces the skipping of dystrophin exon 51, which restores the proper dystrophin reading frame in this subset of DMD patients (~13%). Approximately 23K boys and young men in Europe live with DMD. About 3K would be candidates for drisapersen therapy.
    • The company completed its rolling New Drug Application (NDA) to the FDA in April.
    • Related tickers: (NASDAQ:SRPT) (NYSE:PFE) (Pending:CATB) (NASDAQ:RNA) (NASDAQ:PTCT) (OTCQB:MRNA) (NASDAQ:CAPR) (OTCPK:SPHDF) (NASDAQ:SMMT)
    | Jun. 25, 2015, 4:32 PM | 3 Comments
  • Jun. 18, 2015, 10:48 AM
    • Interim data from a Phase 1b/2a clinical trial assessing privately-held Akashi Therapeutics' product candidate for Duchenne muscular dystrophy (DMD), HT-100, showed that patients with at least six months of continuous dosing achieved average total muscle strength 22.3% greater that levels predicted by comparable steroid-treated external control. The results were determined by quantitative muscle testing (QMT) of upper and lower extremity muscle groups.
    • The average increase in muscle strength compared to baseline over 18-22 months in the 10 patients in the trial was 11.7%. All study participants are on a stable regimen of corticosteroids.
    • Orphan Drug- and Fast Track-designated HT-100 (delayed-release halofuginone) is an orally available small molecule designed to reduce fibrosis (scarring) and inflammation and promote healthy muscle fiber regeneration in patients with DMD.
    • The trial completion date is January 2016. An open label extension study will run until February 2017.
    • DMD-related tickers: (Pending:CATB) (PFE +0.9%)(SRPT +1%) (NASDAQ:RNA) (PTCT +4.5%)(OTCQB:MRNA +5.5%)(CAPR +4.8%)(OTCPK:SPHDF)(BMRN +11.9%)(SMMT +2.2%)
    | Jun. 18, 2015, 10:48 AM | 1 Comment
  • Jun. 17, 2015, 10:45 AM
    • Pending IPO Catabasis Pharmaceuticals (Pending:CATB) commences a Phase 1/2 clinical trial, called MoveDMD, evaluating its candidate for Duchenne muscular dystrophy (DMD), CAT-1004, a small molecule that inhibits a protein called NF-kB which plays a key role in regulating muscle health. Chronic activation of NF-kB has been reported in DMD as well as other musculoskeletal disorders.
    • MoveDMD will enroll ~18 ambulatory boys ages 4-7 with DMD regardless of the specific dystrophin mutation. The primary endpoint of Part A is safety. Part B's endpoint is the change in muscle inflammation as measured by MRI imaging of the leg muscles.
    • Previously: Catabasis Pharmaceuticals' CAT-1004 tagged an Orphan Drug for DMD (Nov. 24, 2014)
    • DMD-related tickers: (PFE +0.4%)(SRPT +6.3%) (NASDAQ:RNA) (PTCT +3.1%)(OTCQB:MRNA +2.2%)(CAPR +1.7%)(OTCPK:SPHDF)(BMRN +0.6%)(SMMT +0.9%)
    | Jun. 17, 2015, 10:45 AM
  • Jun. 9, 2015, 1:07 PM
    • The FDA issues draft guidance for industry related to approval issues/criteria for therapies to treat patients with Duchenne muscular dystrophy. There does not appear to be anything unexpected in the document that would significantly change its approach or criteria to assessing a New Drug Application (NDA) for the indication or radically change the content in a sponsor's NDA.
    • There are three (or four) DMD candidates that are leading the pack: BioMarin Pharmaceutical's (BMRN -1.8%) drisapersen, Sarepta Therapeuticis' (SRPT +1.7%) eteplirsen and PTC Therapeutics' (PTCT -2.6%) Translarna (ataluren).
    • BioMarin completed its rolling NDA submission in April for Fast Track-, Orphan Drug- and Breakthrough Therapy-designated drisapersen.
    • Sarepta started its rolling NDA submission for Orphan Drug- and Fast Track-designated eteplirsen in May.
    • PTC Therapeutics began its rolling NDA for Orphan Drug- and Fast Track-designated Translarna in December 2014.
    • Another potential player is nano cap Santhera Pharmaceuticals (OTCPK:SPHDF) whose Orphan Drug-designated Raxone/Catena (idebenone) was successful in a Phase 3 study.
    | Jun. 9, 2015, 1:07 PM | 3 Comments
Company Description
Sarepta Therapeutics, Inc. is a biopharmaceutical company, which is engaged in the discovery and development of unique RNA-based therapeutics for the treatment of rare and infectious diseases. The company is primarily focused on rapidly advancing the development of its potentially disease... More
Sector: Healthcare
Industry: Biotechnology
Country: United States