Thu, Jul. 7, 11:10 AM
Thu, Jul. 7, 9:16 AM
Thu, Jul. 7, 8:28 AM
Wed, Jun. 22, 7:21 AM
- Thinly traded nano cap Pluristem Therapeutics (NASDAQ:PSTI) is up 11% premarket on light volume in response to its announcement of preclinical data that suggest its PLX-PAD cells may be effective in treating Duchenne muscular dystrophy.
- In a mouse model study, PLX-PAD cells reduced an enzyme called creatine phosphokinase (CPK), a marker of muscle degeneration or injury, by ~50% compared to placebo. In addition, analyses of tissue samples showed reduced levels of inflammation and necrosis (death of most of the cells in an organ or tissue) and evidence of regeneration of muscle tissue.
- PLX cells are mesenchymal-like adherent stromal cells derived from full term human placentas that may be administered without the need for HLA matching. According to the company, PLX-PAD (peripheral artery disease) cells have demonstrated the ability to stimulate the growth of new blood vessels in preclinical models inducing muscle tissue regeneration and improving muscle function.
- PLX-PAD is currently under development for the treatment of critical limb ischemia, intermittent claudication (leg cramps caused by the obstruction of arteries), muscle injury, pulmonary arterial hypertension and preeclampsia.
- DMD-related tickers: (NASDAQ:SRPT)(NASDAQ:BMRN)(NASDAQ:SMMT)(NASDAQ:PTCT)(NYSE:PFE)(OTCQB:MRNA)
Fri, Jun. 17, 9:14 AM
Fri, Jun. 17, 8:52 AM
Tue, Jun. 14, 9:47 AM
- Privately held Northbrook, IL-based Marathon Pharmaceuticals submits a New Drug Application (NDA) to the FDA seeking approval of lead product candidate deflazacort for the treatment of Duchenne muscular dystrophy (DMD).
- According to the company, one of its pivotal studies showed treatment with deflazacort improved muscle strength compared to placebo at week 12.
- Deflazacort is a steroid called a glucocorticoid. It is a derivative of prednisone with less side effects.
- DMD-related tickers: (SRPT +0.2%)(BMRN +2.4%)(SMMT)(PTCT +3.5%)(PFE +0.2%)(OTCQB:MRNA)
Tue, May 31, 4:49 PM
- Prompted by a negative Ad Comm vote in the U.S. and a looming negative vote in Europe, BioMarin Pharmaceuticals (NASDAQ:BMRN) terminates development of Kyndrisa (drisapersen) for the treatment of Duchenne muscular dystrophy (DMD) patients amenable to skipping exon 51. The company is also terminating development of first-generation follow-on products BMN 044, BMN 045 and BMN 053, all currently in Phase 2.
- Despite the setback, the company will continue to explore the development of next-generation oligonucleotides for the treatment of DMD.
- BioMarin still expects to be non-GAAP break even or better in 2017.
- Shares are down a fraction after hours on increased volume.
Thu, May 26, 5:09 PM
- The FDA approves CSL Behring's AFSTYLA [Antihemophilic Factor (Recombinant) Single Chain], its long-acting recombinant factor VIII single-chain therapy for the treatment of adults and children with hemophilia A.
- AFSTYLA is indicated for routine prophylaxis to reduce the frequency of bleeding episodes with dosing of two-to-three times per week, on-demand treatment and control of bleeding episodes and the perioperative management of bleeding.
- Market launch will commence this summer.
- Hemophilia A-related tickers: (NYSE:BXLT)(OTCPK:BAYRY)(NASDAQ:BMRN)(NYSE:OPK)(NYSE:SNY)(NASDAQ:ALNY)(NASDAQ:BIIB)(NASDAQ:SGMO)(OTCQX:RHHBY)(NASDAQ:CBIO)
Thu, Apr. 28, 4:06 PM
Wed, Apr. 27, 5:35 PM
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Tue, Apr. 26, 9:19 AM
- UK-based Summit Therapeutics (NASDAQ:SMMT) has a bit of good news for Duchenne muscular dystrophy (DMD) investors who are about to bloodied today if they are holding shares in Sarepta Therapeutics (NASDAQ:SRPT). The FDA has cleared its Investigational New Drug (IND) application for DMD candidate ezutromid (formerly SMT C1100) which clears the way to recruit U.S. trial sites in its Phase 2 proof-of-concept study called PhaseOut DMD. The 48-week open-label study, initiated in the UK in January, will enroll up to 40 boys, ages five to ten, with DMD. The primary endpoint is the change from baseline in MRI parameters related to fat infiltration and inflammation in leg muscles. According to the company, top-line data should be available in H2.
- Unlike Sarepta's 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 Therapeutics cleared to start mid-stage study of DMD candidate in UK (Jan. 21)
Wed, Apr. 20, 9:15 AM
Fri, Apr. 8, 5:01 AM
- Celgene (NASDAQ:CELG): Target $144. Implied upside 33%. "We believe that CELG looks cheap relative to its growth after reviewing current and pipeline products. We see more upside on base business products than consensus and guidance. We like Celgene’s pipeline that consists of internal and external candidates that leads us to believe it is best in class."
- Gilead (NASDAQ:GILD): Target $116. Implied upside 19%. Concerns over the patent cliff and lack of revenue sustainability have contracted the 2016 P/E multiple to 8x. Fears are overdone and shares trade at a steep discount.
- Biomarin (NASDAQ:BMRN): Target $109. Implied upside 22%. Upcoming catalysts could lead to outperformance over the next few months. Management sounds upbeat. "If we see positive data in these events we think stock could be worth $126-134/share. Big surprise factor would be an EU DMD approval (not in our valuation) worth $47/share."
- Now read Gilead: My Impatience Grows »
Wed, Mar. 30, 11:52 AM
- Sarepta Therapeutics (SRPT +0.8%) bucks biotech's down day on average volume. On CNBC, hedge fund manage Joe Edelman re-emphasized his confidence that DMD candidate eteplirsen will be approved by the FDA.
- The Ad Comm review is scheduled for April 25 followed by the PDUFA date on May 26.
- This is a superb example of the prominence (risk) of a binary event for biotech investors. It will be either feast or famine. BioMarin Pharmaceutical (BMRN -0.4%) longs felt the sting of rejection a few months ago with its DMD candidate drisapersen.
- Previously: FDA rejects BioMarin's drisapersen for Duchenne muscular dystrophy (Jan. 14)
- Previously: Ad Comm fails to back BioMarin's drisapersen for DMD (Nov. 24, 2015)
Thu, Mar. 24, 10:37 AM
- The European Commission designates BioMarin Pharmaceutical's (BMRN +1.7%) BMN 270 an Orphan Drug for the treatment of hemophilia A, an inherited bleeding disorder caused by a deficiency in clotting factor VIII.
- BMN 270 is a gene therapy designed to restore factor VIII plasma levels. It uses a vector called AAV (adeno-associated virus) to deliver the functional (corrected copy) gene to cells. It is currently in Phase 1/2 development. According to clinicaltrials.gov, the current study's estimated completion date is June 2021.
- Among the benefits of Orphan Drug status in the EU is an 10-year period of market exclusivity for the indication, if approved.
BioMarin Pharmaceutical, Inc. develops and commercializes innovative pharmaceuticals for serious diseases and medical conditions. The company operates its business through one segment, the biopharmaceutical development and commercialization segment. Its company's product portfolio is comprised... More
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