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Prosensa Holding N.V. (RNA)

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  • Wed, Aug. 5, 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%)
    | Wed, Aug. 5, 11:25 AM | 1 Comment
  • Mon, Jul. 27, 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)
    | Mon, Jul. 27, 4:27 PM | 7 Comments
  • Mon, Jul. 6, 8:25 AM
    | Mon, Jul. 6, 8:25 AM | 1 Comment
  • Thu, Jun. 25, 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)
    | Thu, Jun. 25, 4:32 PM | 3 Comments
  • Thu, Jun. 18, 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%)
    | Thu, Jun. 18, 10:48 AM | Comment!
  • Wed, Jun. 17, 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%)
    | Wed, Jun. 17, 10:45 AM | Comment!
  • Mon, Jun. 8, 9:41 AM
    • Thinly traded nano cap Capricor Therapeutics (CAPR +19.4%) is set for an opening gap up in response to its announcement that the FDA has approved its Investigational New Drug (IND) application for the development of Orphan Drug-designated CAP-1002 for the treatment of patients with Duchenne Muscular Dystrophy (DMD)-related cardiomyopathy. The company is currently developing CAP-1002 for the treatment of heart disease.
    • A Phase 1/2 study, called HOPE-DUCHENNE, will enroll up to 24 patients.
    • Due to the improvement in the treatment of respiratory failure, cardiomyopathy is the leading cause of death in DMD patients.
    • CAP-1002 is an "off-the-shelf" cell therapy that will be infused via a catheter into the patients' three coronary arteries in the study.
    • DMD-related tickers: (SRPT +1.3%)(OTCPK:SPHDF)(PTCT -2.3%)(SMMT)(PFE -0.2%)(NASDAQ:RNA) (ROSG)(OTCQB:MRNA)(BMRN +0.9%)
    | Mon, Jun. 8, 9:41 AM | 2 Comments
  • 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)
    | Tue, May 19, 5:40 PM | 11 Comments
  • 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)
    | Mon, Apr. 27, 10:24 AM | 10 Comments
  • 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. 22, 12:56 PM | 1 Comment
  • 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. 19, 5:38 PM | Comment!
  • 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%)
    | Dec. 23, 2014, 10:20 AM | 1 Comment
  • Dec. 17, 2014, 10:10 AM
    • Pfizer (PFE +0.2%) initiates a Phase 2 clinical trial assessing its investigational compound, PF-06252616, in boys with Duchenne Muscular Dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. It occurs in ~1 in 3,500 male births worldwide.
    • The study will evaluate the safety, tolerability and efficacy of PF-06252616 in boys aged 6 to <10 years old diagnosed with DMD regardless of genotype.
    • The FDA designated PF-06252616 an Orphan Drug in July 2012 and Fast Track in November 2012.
    • DMD-related tickers: (SRPT +4.1%)(RNA +0.1%)(PTCT +1.6%)(OTCQB:MRNA +5.8%)(ROSG +0.4%)
    • Previously: Catabasis Pharmaceuticals' CAT-1004 tagged an Orphan Drug for DMD (Nov. 24, 2014)
    | Dec. 17, 2014, 10:10 AM | Comment!
  • Nov. 24, 2014, 12:46 PM
    | Nov. 24, 2014, 12:46 PM | Comment!
  • Nov. 24, 2014, 12:15 PM
    • The FDA designates privately-held Catabasis Pharmaceuticals' investigational NF-kB inhibitor, CAT-1004, an Orphan Drug for the treatment of Duchenne muscular dystrophy (DMD).
    • NF-kB is a key mediator of cellular injury. Evidence suggests that activated NF-kB  contributes to the underlying pathology of DMD.
    • In a Phase 1 trial, CAT-1004 was well-tolerated and demonstrated a significant reduction of activated NF-kB. The company plans to proceed to Phase 2 development in 1H 2015.
    • Among the benefits of Orphan Drug designation is a seven-year period of market exclusivity if approved.
    • DMD-related tickers: (SRPT +7.6%)(RNA +63.3%)(BMRN +1.7%)(PTCT +3.4%)(OTCQB:MRNA -3.3%)(ROSG +0.8%)
    | Nov. 24, 2014, 12:15 PM | Comment!
  • Nov. 24, 2014, 9:12 AM
    | Nov. 24, 2014, 9:12 AM | 1 Comment
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Company Description
Prosensa Holding NV is an biotechnology company engaged in the discovery and development of ribonucleic acid-modulating, or RNA-modulating, therapeutics for the treatment of genetic disorders.
Sector: Healthcare
Industry: Biotechnology
Country: Netherlands