BioMarin Pharmaceutical Inc.(BMRN)- NASDAQ
  • 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)
    | Tue, Apr. 26, 9:19 AM | 4 Comments
  • Wed, Apr. 20, 9:15 AM
    | Wed, Apr. 20, 9:15 AM | 8 Comments
  • 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 »
    | Fri, Apr. 8, 5:01 AM | 8 Comments
  • 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)
    | Wed, Mar. 30, 11:52 AM | 6 Comments
  • 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.
    | Thu, Mar. 24, 10:37 AM
  • Mon, Mar. 14, 9:01 AM
    • The FDA has rescheduled its Peripheral and Central Nervous System Drugs Advisory Committee review of Sarepta Therapeutics' (NASDAQ:SRPT) New Drug Application (NDA) for its Duchenne muscular dystrophy (DMD) candidate eteplirsen for April 25. The agency's action date (PDUFA) is May 26.
    • The advisory committee meeting was originally scheduled for January 22 but was canceled due to bad weather.
    • Eteplirsen has Priority Review, Rare Pediatric Disease Designation, Orphan Drug and Fast Track status for the treatment of DMD. The drug's mechanism of action, skipping exon 51, applies to ~13% of the 3,500 - 5,000 boys worldwide with DMD.
    • A positive vote is far from certain. The FDA rejected BioMarin Pharmaceutical's (NASDAQ:BMRN) exon 51-skipping drisapersen in January citing lack of proof of efficacy.
    • Previously: It ain't lookin' too good for Sarepta's eteplirsen at next week's Ad Comm review; shares down 57% (Jan. 15)
    • Previously: FDA rejects BioMarin's drisapersen for Duchenne muscular dystrophy (Jan. 14)
    • Here's an analysis of the data by SA Contributor BosCaptn.
    | Mon, Mar. 14, 9:01 AM | 7 Comments
  • Wed, Mar. 9, 10:35 AM
    • Ultragenyx (RARE -1.5%) initiated with Outperform rating and $85 (44% upside) price target by Baird.
    • BioMarin Pharmaceutical (BMRN -1.6%) initiated with Outperform rating and $110 (33% upside) price target by Baird.
    • Nanosphere (NSPH +1.1%) initiated with Buy rating and $2 (117% upside) price target by Rodman & Renshaw.
    • BioSpecifics Technologies (BSTC -0.9%) initiated with Buy rating and $60 (68% upside) price target by Rodman & Renshaw.
    • Proteostasis Therapeutics (PTI +7.5%) initiated with Buy rating and $15 (67% upside) price target by H.C. Wainwright. Initiated with Outperform rating and $20 (122% upside) price target by RBC Capital. Initiated with Outperform rating and $13 (44% upside) price target by Baird.
    • AveXis (AVXS -0.8%) initiated with Outperform rating and $31 (44% upside) price target by BMO Capital. Initiated with Buy rating and $28 (30% upside) price target by Goldman Sachs.
    • Delcath Systems (DCTH +2.8%) initiated with Buy rating and $1 (233% upside) price target by WallachBeth.
    • Amicus Therapeutics (FOLD -1.8%) upgraded to Buy from Neutral by Janney Capital. Price target raised to $12 (61% upside) from $10.
    • Celldex Therapeutics (CLDX +1.4%) downgraded to Hold from Buy by Jefferies. Price target lowered to $4 (10% upside) from $31.
    • MEDNAX (MD -0.2%) downgraded to Neutral from Positive by Susquehanna. Price target lowered to $70 (8% upside) from $94.
    • Roka Bioscience (ROKA -8.8%) downgraded to Market Perform from Outperform. Price target lowered to $1 (22% upside).
    • Zimmer Biomet Holdings (ZBH +0.1%) downgraded to Hold from Buy by Argus Research.
    | Wed, Mar. 9, 10:35 AM | 5 Comments
  • Thu, Mar. 3, 4:10 PM
    • Results from Phase 1/2 clinical trial, Study 190-201, assessing BioMarin Pharmaceutical's (BMRN +2.4%) cerliponase alfa for the treatment of children with a form of Batten disease, a rare inherited disorder in which the body's cells lose the their ability to dispose of wastes, showed a significant clinical benefit over a 48-week treatment period.
    • The study enrolled 24 subjects with CLN2 disease, a fatal disorder caused by mutations in the CLN2 gene on chromosome 11. CLN2 codes for a lysosomal enzyme called tripeptidyl peptidase (TPP1), a deficiency in which causes proteins and fats to accumulate in neuron and other cells leading to seizures, loss of motor function and learning disabilities. 300 mg of cerliponase alfa was administered every 14 days via intracerebroventricular infusion (infused directly into the fluid surrounding the brain thereby bypassing the blood-brain barrier) for at least 48 weeks.
    • The average rate of clinical decline in cerliponase alfa-treated patients was ~80% less that the expected rate of decline in the untreated population (p<0.0001). 87% of the patients in the treatment group experienced attenuation (lessening) of their disease compared to the expected historical rate of decline.
    • Administration of cerliponase alfa was no walk in the park, however. The most frequent adverse events were pyrexia (fever)(46%), hypersensitivity (33%), seizure (33%), epilepsy (17%), vomiting (13%) and headache (13%).
    • Orphan Drug- and Breakthrough Therapy-tagged cerliponase alfa is an enzyme replacement therapy, specifically a recombinant form of human TPP1.
    • The company intends to file regulatory applications in the U.S. and Europe by mid-year and will request accelerated review for both.
    • Shares are up 2% after hours on light volume.
    | Thu, Mar. 3, 4:10 PM | 2 Comments
  • Tue, Mar. 1, 7:08 PM
    • BioMarin Pharma's (NASDAQ:BMRN) BMN 270 hemophilia A gene therapy has been granted orphan drug designation by the FDA. The company says it's "currently conducting a Phase 1/2 study to evaluate the safety and efficacy of BMN 270 gene therapy in up to 12 patients with severe hemophilia A and will provide a program update in April."
    • BMRN +2.7% after hours to $89.97.
    | Tue, Mar. 1, 7:08 PM | 2 Comments
  • Thu, Feb. 25, 4:08 PM
    • BioMarin Pharmaceutical (NASDAQ:BMRN): Q4 EPS of $0.39 may not be comparable to consensus of $0.99.
    • Revenue of $227.9M (-0.9% Y/Y) beats by $3.52M.
    | Thu, Feb. 25, 4:08 PM | 2 Comments
  • Wed, Feb. 24, 5:35 PM
  • Tue, Feb. 23, 9:14 AM
    • PTC Therapeutics (NASDAQ:PTCT) is getting slammed in premarket trading. Shares are down 42% on robust volume in response to its announcement that it received a Refuse to File letter from the FDA regarding its New Drug Application (NDA) seeking clearance of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
    • Refuse to File means that the application was not complete enough to allow for review. The company is assessing its options.
    • Translarna is a protein restoration therapy designed to enable the formation of a functioning protein in patients with nonsense mutation-caused genetic disorders.
    • This is the second bump in the road for DMD hopefuls. The FDA said no to BioMarin's (NASDAQ:BMRN) Kyndrisa (drisapersen) last month. It's not looking much better for Sarepta's (NASDAQ:SRPT) eteplirsen either. The FDA's briefing document for its upcoming (date unspecified) Ad Comm review is considered overly harsh (bearish) by many observers.
    • Previously: FDA rejects BioMarin's drisapersen for Duchenne muscular dystrophy (Jan. 14)
    • Previously: It ain't lookin' too good for Sarepta's eteplirsen at next week's Ad Comm review; shares down 57% (Jan. 15)
    | Tue, Feb. 23, 9:14 AM | 6 Comments
  • Mon, Feb. 22, 4:01 PM
    • A Phase 1/2 clinical trial is underway assessing Capricor Therapeutics' (CAPR) lead product candidate, Orphan Drug-tagged CAP-1002, for the treatment of patients with Duchenne muscular dystrophy-related cardiomyopathy.
    • The study, called HOPE-Duchenne, will enroll 24 DMD patients. Those randomized for treatment with CAP-1002 will receive the cells in all three coronary arteries which will enable thorough delivery across the myocardium. The primary endpoint is safety and tolerability. Secondary endpoints include measures of cardiac structure and physical function such as the 6-minute walk test, spirometry and Performance of Upper Limb Scale.
    • CAP-1002 is an "off-the-shelf" cell therapy that will be infused via a catheter into the patients' three coronary arteries.
    • According to clinicaltrials.gov, the estimated final data collection date for the primary endpoint is May 2017. The estimated study completion date is July 2017.
    • DMD-related tickers: (NASDAQ:BMRN)(NASDAQ:SRPT)(NYSE:PFE)(NASDAQ:SMMT)(NASDAQ:PTCT)(OTCQB:MRNA)
    | Mon, Feb. 22, 4:01 PM | 1 Comment
  • Tue, Jan. 26, 10:27 AM
    • Non-profit-backed Akashi Therapeutics suspends enrollment and dosing in its clinical trial, HALO, assessing lead product candidate and Orphan Drug- and Fast Track-tagged HT-100 in patients with Duchenne muscular dystrophy (DMD) after one patient developed serious, life-threatening health issues subsequent to receiving the highest dose in the study, 60 ug/kd/day. The company is working with the FDA to analyze the situation.
    • This is the only instance of this severity observed in HT-100's clinical program. An interim safety and efficacy analysis released in June 2015 showed promising therapeutic effects with no serious drug-related adverse events. The company intends to restart the trial after the analysis is complete and the safety issues fully vetted.
    • Earlier this month, the company inked a $100M global agreement with Germany's Grunenthal to develop HT-100 for DMD.
    • 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.
    • Previously: Akashi Therapeutics and Grunenthal to jointly develop DMD med (Jan. 8)
    • Previously: Akashi Therapeutics' Duchenne MD candidate shows encouraging results in early stage study (June 18, 2015)
    • DMD-related tickers: (SRPT -1.5%)(BMRN -0.8%)(PFE +0.1%)(SMMT)(OTCQB:MRNA +18.1%)(PTCT -0.7%)
    • Update: Sadly, the patient died. The company has initiated a comprehensive investigation of all patient records with a particular focus on this patient. It will continue to work closely with the FDA and outside experts in its effort to fully understand all aspects of the situation and to insure that all study participants are safe.
    | Tue, Jan. 26, 10:27 AM | 5 Comments
  • Mon, Jan. 25, 11:30 AM
    • Part A of a Phase 1/2 clinical, MoveDMD, assessing Catabasis Pharmaceuticals' (CATB +15%) Orphan Drug- and Fast Track-tagged CAT-1004 for the treatment of Duchenne muscular dystrophy (DMD) successfully demonstrated an acceptable safety profile 17 ambulatory boys with DMD. Three doses of CAT-1004 (33 mg/kg/day, 67 mg/kg/day and 100 mg/kg/day) were generally well tolerated with no safety signals or serious adverse events observed and there were no drug discontinuations. The majority of adverse events were mild, with the most common being diarrhea. Plasma exposure levels associated with NF-kB inhibition were consistent with those previously observed in adults.
    • The company intends to initiate Part B of MoveDMD in H1, a randomized, double-blind, placebo-controlled study that will assess CAT-1004's safety and efficacy over a 12-week period. The boys in Part A will be asked to participate in Part B.
    • CAT-1004 is a small molecule that inhibits a protein called NF-kB, that plays a key role in muscle health.
    • DMD-related tickers: (BMRN +1.9%)(SRPT +2.9%)(PFE -0.3%)(OTCQB:MRNA -5.5%)(PTCT -2.2%)(SMMT)
    | Mon, Jan. 25, 11:30 AM
  • Fri, Jan. 22, 8:23 AM
    • Credit Suisse analysts have been very busy lately with a long list of initiations: Acceleron Pharma (NASDAQ:XLRN) Outperform with $50 (47% upside) price target; BioMarin Pharmaceutical (NASDAQ:BMRN) Outperform with $110 (36% upside) price target; Amgen (NASDAQ:AMGN) Outperform with $205 (34% upside) price target.
    • Celgene (NASDAQ:CELG) Outperform with $149 (41% upside) price target; Coherus Biosciences (NASDAQ:CHRS) Outperform with $25 (79% upside) price target; Gilead Sciences (NASDAQ:GILD) Outperform with $125 (40% upside) price target.
    • Regeneron Pharmaceuticals (NASDAQ:REGN) Neutral with $552 (19% upside) price target; Vertex Pharmaceuticals (NASDAQ:VRTX) Outperform with $151 (59% upside) price target; Alnylam Pharmaceuticals (NASDAQ:ALNY) Outperform with $154 (117% upside) price target.
    • Alexion Pharmaceuticals (NASDAQ:ALXN) Neutral rating with $201 (29% upside) price target; Biogen (NASDAQ:BIIB) Neutral with $322 (23% upside) price target; XenoPort (NASDAQ:XNPT) Underperform with $4 (16% downside risk) price target.
    • PTC Therapeutics (NASDAQ:PTCT) Outperform with $36 (33% upside) price target; Mesoblast Limited (NASDAQ:MESO) Outperform with $10 (92% upside) price target and Incyte (NASDAQ:INCY) Outperform with $110 (49% upside) price target.
    | Fri, Jan. 22, 8:23 AM | 3 Comments
Company Description
BioMarin Pharmaceutical, Inc. engages in the development and commercialization of innovative pharmaceuticals for serious diseases and medical conditions. It operates its business through one segment, the biopharmaceutical development and commercialization segment. Its company's products include... More
Sector: Healthcare
Industry: Biotechnology
Country: United States