Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
FDA Posts Briefing Documents for Advisory Committee Meeting Reviewing Vimizim(TM) for the Treatment of Morquio A Syndrome
Endocrinologic and Metabolic Drugs Advisory Committee Meeting November 19, 2013
SAN RAFAEL, Calif., Nov. 15, 2013 (GLOBE NEWSWIRE) --
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today the U.S. Food and Drug Administration (FDA) posted briefing materials for the November 19, 2013 Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) meeting to review the Vimizim™ (elosulfase alfa) Biologics License Application (BLA). Vimizim, an enzyme replacement therapy, is being developed for the treatment of patients with the lysosomal storage disorder Morquio A syndrome, also called Mucopolysaccharidosis Type IVA (MPS IVA).
The EMDAC meeting is scheduled for November 19, 2013 at 8:00am ET. The briefing materials and webcast information can be found on the FDA website at:
http://www.fda.gov/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/EndocrinologicandMetabolicDrugsAdvisoryCommittee/ucm331504.htm
The Prescription Drug User Fee Act date for completion of the review of the company's BLA for Vimizim is February 28, 2014.
About Morquio A Syndrome
Mucopolysaccharidosis IVA (MPS IVA, also known as Morquio A syndrome) is a disease characterized by deficient activity of Nacetylgalactosamine- 6-sulfatase (GALNS) causing excessive lysosomal storage of glycosaminoglycans such as keratan sulfate and chondroitin sulfate. This excessive storage causes a systemic skeletal dysplasia, short stature, and joint abnormalities, which limit mobility and endurance. Malformation of the chest impairs respiratory function, and looseness of joints in the neck cause spinal instability and potentially spinal cord compression. Other symptoms may include hearing loss, corneal clouding, and heart disease. Initial symptoms often become evident in the first five years of life. The disease substantially limits both the quality and length of life of those affected.
About BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse® (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include Vimizim (N-acetylgalactosamine 6-sulfatase), formally referred to as GALNS, which successfully completed Phase 3 clinical development for the treatment of MPS IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase 3 clinical development for the treatment of PKU, BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase 3 clinical development for the treatment of germline BRCA breast cancer, BMN 701, a novel fusion of acid alpha glucosidase (GAA) with a peptide derived from insulin like growth factor 2, which is currently in Phase 1/2 clinical development for the treatment of Pompe disease, BMN 111, a modified C-natriuretic peptide, which is currently in Phase 1 clinical development for the treatment of achondroplasia and BMN 190, a recombinant human tripeptidyl peptidase-1 (rhTPP1) for the treatment of late-infantile neuronal ceroid lipofuscinosis (CLN2), a form of Batten Disease. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.
Investors:
Traci McCarty
BioMarin Pharmaceutical Inc.
(415) 455-7558
Media:
Debra Charlesworth
BioMarin Pharmaceutical Inc.
_________________________________________________________
http://www.globenewswire.com/news-release/2013/11/15/590285/10058116/en/FDA-Posts-Briefing-Documents-for-Advisory-Committee-Meeting-Reviewing-Vimizim-TM-for-the-Treatment-of-Morquio-A-Syndrome.html
BMRN
BioMarin Announces French ATU Granted for Vimizim(TM) for the Treatment of Morquio A Syndrome
SAN RAFAEL, Calif., Nov. 15, 2013 (GLOBE NEWSWIRE) --
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today that the French National Agency for Medicines and Health Products Safety (ANSM) has granted an Autorisation Temporaire d'Utilisation de cohorte (ATU cohort), or Temporary Authorization for Use, for patient sales of Vimizim for the treatment of Morquio A Syndrome. An ATU is the regulatory mechanism used by the ANSM to make non-approved drugs available to patients in France when a genuine public health need exists. This ATU allows patients with Morquio A Syndrome in France to receive treatment with Vimizim before marketing authorization for the product is granted in the European Union. Government allocations to hospitals allow payment for Vimizim for patients included in the ATU program. The company expects to book revenue on named-patient basis sales and will continue to assist patients in France wherever possible prior to full market product approval in the European Union.
"With a significant number of all Morquio A patients living throughout Europe, we are pleased to be able to provide Vimizim to patients in France under the ATU program," said Jeff Ajer, Senior Vice President, Global Commercial Operations at BioMarin. "We look forward to working closely with European regulatory authorities through the final stages of full market approval in this region."
About Morquio A Syndrome
Mucopolysaccharidosis IVA (MPS IVA, also known as Morquio A syndrome) is a disease characterized by deficient activity of Nacetylgalactosamine- 6-sulfatase (GALNS) causing excessive lysosomal storage of glycosaminoglycans such as keratan sulfate and chondroitin sulfate. This excessive storage causes a systemic skeletal dysplasia, short stature, and joint abnormalities, which limit mobility and endurance. Malformation of the chest impairs respiratory function, and looseness of joints in the neck cause spinal instability and potentially spinal cord compression. Other symptoms may include hearing loss, corneal clouding, and heart disease. Initial symptoms often become evident in the first five years of life. The disease substantially limits both the quality and length of life of those affected.
About BioMarin
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse® (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include Vimizim™ (N-acetylgalactosamine 6-sulfatase), formally referred to as GALNS, which successfully completed Phase 3 clinical development for the treatment of MPS IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase 3 clinical development for the treatment of PKU, BMN 673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase 3 clinical development for the treatment of germline BRCA breast cancer, BMN 701, a novel fusion of acid alpha glucosidase (GAA) with a peptide derived from insulin like growth factor 2, which is currently in Phase 1/2 clinical development for the treatment of Pompe disease, BMN 111, a modified C-natriuretic peptide, which is currently in Phase 1 clinical development for the treatment of achondroplasia and BMN 190, a recombinant human tripeptidyl peptidase-1 (rhTPP1) for the treatment of late-infantile neuronal ceroid lipofuscinosis (CLN2), a form of Batten Disease. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.
Forward-Looking Statement
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the regulatory status of Vimizim, possible sales of Vimizim under the ATU program and expectations about the commercialization of Vimizim generally. These risks and uncertainties include, among others: the ability of hospitals to use the ATU program and the funding for the ATU program generally; results and timing of current and planned preclinical studies and clinical trials of Vimizim; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning Vimizim; and those factors detailed in BioMarin's filings with the Securities and ExchangeCommission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's 2012 Annual Report on Form 10-K, and the factors contained in BioMarin's reports on Form 10-Q. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin®, Naglazyme®, Kuvan® and Firdapse™ are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.
CONTACT: Investors:
Traci McCartyBioMarin Pharmaceutical Inc.
(415) 455-7558
Media:
Debra CharlesworthBioMarin Pharmaceutical Inc.
(415) 455-7451
Source: BioMarin Pharmaceutical Inc.
This article appears in: News Headlines
Read more: http://www.nasdaq.com/press-release/biomarin-announces-french-atu-granted-for-vimizimtm-for-the-treatment-of-morquio-a-syndrome-20131115-00266#ixzz2kikKvYt4
______________________________________________________
(This is the drug which is the subject of the Tuesday meeting)
BMRN
Discussion in recent increase in shorts:
BioMarin Pharmaceutical. On top of an 11 percent rise in the previous period, short interest in this San Rafael, Calif.-based company grew about 12 percent to total more than 5.82 million shares. That was the greatest number since March, and it was about four percent of the float. Days to cover was more than three.
This biopharmaceuticals company has a market cap of more than $8 billion. Third-quarter revenue grew less than expected, but BioMarin reaffirmed its full-year guidance. The long-term EPS growth forecast is about 25 percent, though the return on equity and operating margin are both in the red.
Nine of the 22 polled analysts rate the stock at Strong Buy, and another eight also recommend buying shares. The analysts see room for shares to run, as the mean price target is about 23 percent higher than the current share price. But note that shares traded higher than that target back in September.
Shares have pulled back more than eight percent in the past month. The share price still is up more than 19 percent year to date.
Read more: http://www.benzinga.com/news/13/11/4073760/gilead-sciences-leads-in-rising-short-interest-among-biotechs#ixzz2kWoA33Xu
________________________________________________
BMRN
Whomever purchased the big block is currently down $720,000 at this point today.
Why down? Saw elsewhere the thought it is sympathy with SRPT, but not sure it makes sense.
BMRN
AstraZeneca added 14p to £32.92 as it announced a £120m investment in its manufacturing site in Macclesfield. There was also renewed talk the company was interested in US group BioMarin Pharmaceutical, although many believed Swiss giant Roche could beat the UK group to the punch with a near $11bn offer.
________________________________________________________
http://www.theguardian.com/business/marketforceslive/2013/nov/11/ftse-100-higher-bskyb-rsa-insurance-fall
BMRN
400,000 share purchase just made @ $62.00 (8:31:58 am). Got to be the bottom.
BMRN
Thanks -- great find. Need a moderator to stickie it.
BMRN
It looks like there will be a free webcast. Here is the link from the FDA sight it seems the link will be posted nearer to the date.
http://www.fda.gov/AdvisoryCommittees/Calendar/ucm371064.htm
ghmm, I haven't seen anything about a webcast. Company's website has no info. Hopefully, something will be announced as the date gets closer.
Should be a good week for BMRN!
BMRN
Do you (or anyone) know if their will be a (free) live webcast of the panel? Any link would be appreciated TIA
A little over a week away:
"11/19/2013 Biomarin Pharmaceutical Inc. BMRN Vimizim (BLA) FDA panel to review Vimizim for the treatment of Mucopolysaccharidosis Type IVA (Morquio A syndrome)"
BMRN
BioMarin Begins Phase 3 Trial Of BMN 673 For Metastatic GBRCA Breast Cancer
10/31/2013 5:31 AM ET
BioMarin Pharmaceutical Inc.(BMRN: Quote) Thursday said it has dosed the first patient in its Phase 3 program to study its poly ADP-ribose polymerase or PARP inhibitor, BMN 673, in the treatment of metastatic germline BRCA mutated breast cancer. The primary objective of the study is to measure progression free survival, while the secondary objectives include evaluating the objective response rate and the overall survival.
BRCA1 and BRCA2 are human genes that belong to a class of genes known as tumor suppressors. Mutation of these genes has been linked to hereditary breast and ovarian cancer.
Click here to receive FREE breaking news email alerts for Biomarin Pharmaceutical Inc. and others in your portfolio
by RTT Staff Writer
For comments and feedback: editorial@rttnews.com
_________________________________________________
http://www.rttnews.com/2213556/biomarin-begins-phase-3-trial-of-bmn-673-for-metastatic-gbrca-breast-cancer.aspx?type=qf&utm_source=google&utm_campaign=sitemap
BMRN
From Sheff:
"BMRN..$65.55..is down $1.40 today or 2% today. Hesitant to add with the market up but this one is on watch for an add in the New Economy Portfolio."
BMRN
Biotechs With FDA Review Dates In November 2013
Oct 25 2013, 13:23 | includes: BMRN, xxxx, xxxx
Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. (More...)
The following companies have binary events in November that could move their stocks significantly. They all have upcoming FDA review dates for their new drugs. An FDA approval could cause a sizable rise in the stocks, while a rejection or Complete Response Letter could cause a sell-off. Occasionally, the decision date is postponed or moved up to a later date. November presents an interesting mix of a small-cap, a mid-cap, and a large-cap company.
(other stock info deleted)
Biomarin Pharmaceuticals (BMRN) is the mid-cap company among these three firms. It has an FDA review date on 11/19/13 for Vimizim with an indication for Mucopolysaccharidosis Type IVA (Morquio A syndrome). People with Morquio A do not produce enough of the enzyme that is needed to breakdown long chains of sugar carbohydrates into simpler molecules. This results in cellular damage which affects appearance, physical abilities, organ and system functioning, and mental development. Vimizim works by acting as an enzyme replacement which should halt the progression of the disease and alleviate some symptoms. Morquio A is estimated to occur in 1 of every 200,000 births. Currently, there are no specific treatments for Morquio A syndrome. This bodes well for Biomarin as the company has the potential to be the go-to firm for treating this disorder.
Biomarin can be considered of medium to high risk among these three companies. It has four drugs already on the market for other rare diseases and another five in its pipeline being developed in addition to Vimizim. Despite having four drugs on the market, Biomarin is not yet profitable due to high R&D and other costs. The company just reported revenue growth of 6.9% and a loss of $0.38 per share for Q3 2013. Earnings missed the consensus estimates by $0.08 per share.
Biomarin is expected to grow earnings annually by 25% for the next five years. The 22 analysts covering the company have a price target of $79 for the stock which is currently trading at $69. (EDIT: ~$66)
Conclusion
The good thing about biotechs is that they are developing products to improve and extend the lives of individuals. Conservative investors may want to consider Sanofi as it pays a dividend, is established and profitable, and has a sizable portfolio of products on the market. Investors who are willing to take on more risk with more reward potential, may want to consider Biomarin or Vanda, which may see a spike in stock price if the drugs being reviewed become approved by the FDA. However, be prepared for a sell-off in the event that the drugs are not approved or if the decision dates are delayed.
_________________________________________________________
http://seekingalpha.com/article/1774492-biotechs-with-fda-review-dates-in-november-2013?source=feed_f
BMNR
Sell-side adjusts BioMarin targets after earnings • 11:15 AM
Below is a roundup of purported analyst price target changes for BioMarin (BMRN -3.5%) post-earnings.
Barclays raises target to $75 from $70.
Jefferies lowers target to $83 from $86.
Wells Fargo increases target range to $72-77.
Leerink hikes target to $76 from $72.
More: BMRN Q3 report, highlights, CC transcript
BMRN
Roche talks rare diseases, won't comment on Alexion, BioMarin speculation: Reuters • 11:47 AM
Is Roche (RHHBY +0.4%) really interested in Alexion (ALXN -0.4%) or BioMarin (BMRN +0.1%)? CEO Severin Schwan isn't telling, but he did say at a Paris conference that the company will "go where the science takes it, wherever it is, independent from the size of the patient population," suggesting a move into rare and orphan diseases.
Regarding the company's attempt to expand beyond core cancer treatments, Schwan mentioned autoimmune, inflammatory diseases, and neuroscience, noting that in these "three areas naturally [Roche] is a partner of choice for many companies [and] if there was an interesting opportunity outside of those established areas, I would be very open to look at it."
More on Roche/ALXN speculation here, and on BMRN chatter here
BMRN
Roche talks rare diseases, won't comment on Alexion, BioMarin speculation: Reuters • 11:47 AM
Is Roche (RHHBY +0.4%) really interested in Alexion (ALXN -0.4%) or BioMarin (BMRN +0.1%)? CEO Severin Schwan isn't telling, but he did say at a Paris conference that the company will "go where the science takes it, wherever it is, independent from the size of the patient population," suggesting a move into rare and orphan diseases.
Regarding the company's attempt to expand beyond core cancer treatments, Schwan mentioned autoimmune, inflammatory diseases, and neuroscience, noting that in these "three areas naturally [Roche] is a partner of choice for many companies [and] if there was an interesting opportunity outside of those established areas, I would be very open to look at it."
More on Roche/ALXN speculation here, and on BMRN chatter here
BMRN
Nice article on JJ/the company
http://www.burrillreport.com/content/BR_0913.pdf
BioMarin Surges on Report Roche Is Seeking Bid Financing
By Trista Kelley & Meg Tirrell - Sep 19, 2013 8:18 AM ET
BioMarin Pharmaceutical Inc. (BMRN) gained after DealReporter said Roche Holding AG (ROG) is lining up $15 billion in debt financing for an acquisition of the maker of drugs to treat rare diseases.
Roche may have to pay $95 to $105 a share for the Novato, California-based company, DealReporter said, citing people familiar with the situation. The lower end of the range would be a 23 percent premium to BioMarin’s closing level yesterday of $77.49, and would value the company at about $13.5 billion.
Enlarge image Roche Holding AG Reported to Seek Financing for BioMarin
A logo sits above office windows outside the headquarters of Roche AG in Basel. Photographer: Gianluca Colla/Bloomberg
A takeover would give Roche a company that drew $500.7 million in 2012 revenue from medicines for diseases including mucopolysaccharidosis VI, or MPS VI, a rare, life-threatening lysosomal storage disorder. BioMarin also has a slate of experimental treatments for other rare diseases and cancer, and analysts expect its revenue to grow by at least 20 percent in 2014 and 2015.
“BioMarin is a very interesting company, great products, excellent pipeline in good markets, and a focus on orphan drugs,” said Connie Schuemann, who helps manage about $36 billion at Raiffeisen Capital Management in Vienna. “But it comes at a high price.”
BioMarin climbed 9.5 percent to $84.88 at 7:57 a.m. New York time in trading before U.S. markets opened.
Roche, the world’s biggest maker of cancer drugs, climbed less than 1 percent to 239.10 Swiss francs. Silvia Dobry, a spokeswoman for the Basel, Switzerland-based company, declined to comment on the DealReporter article. BioMarin spokeswoman Debra Charlesworth didn’t immediately respond messages left before business hours.
$5 Billion
UBS AG (UBSN) is leading the financing and has indicated it will lend $5 billion to Roche for the takeover, DealReporter said. Citigroup Inc. is advising Roche on the potential sale while JPMorgan Chase & Co. (JPM) and Goldman Sachs Group Inc. may assist in financing, DealReporter said.
Roche considered a bid for Alexion Pharmaceuticals Inc. (ALXN), another maker of medicines for rare diseases, people with knowledge of the matter said in July. Alexion engaged Goldman as an adviser to prepare for a possible bid from Roche, the people said at the time.
Companies like BioMarin and Alexion develop medicines for as few as 5,000 to 10,000 patients worldwide, and charge as much as $400,000 a year per patient.
Contradict History
A bid for BioMarin would contradict Roche’s history of “financially disciplined” acquisition negotiations, said Andrew Weiss, an analyst at Bank Vontobel AG in Zurich.
If the BioMarin report is true, “this would be a waste of money” at 40 percent more expensive than Alexion, as measured by enterprise value to sales, said Weiss. “Orphans are not a strategic imperative for Roche. They look for things that make sense and if something happens to be an orphan, so be it.”
Medicines for such small patient populations, known as orphan drugs, get exclusive marketing rights as an incentive for their development in the U.S. and Europe, while the small patient populations mean fewer participants are needed for clinical trials. They’ve drawn the interest of large pharmaceutical companies looking to fend off a drop in sales as patents on blockbuster drugs expire.
Yes, a lot of love passed out by Cramer last night because his charting guy liked the setup. We'll see, today is a tough day to judge any stock by with all the political crap going on.
Cramer just told everyone to buy - RIGHT HERE RIGHT NOW !!!
This should be interesting
SHAREHOLDERS, WRITE THIS COMPANY AND DEMAND THEY HELP SOMEONE WITH THEIR DRUGS... THE COMPANY SHOULD SHOW SOME CONFIDENCE IN THEIR DRUGS AND NOT BE AFRAID IT WONT WORK...
Q2 Earnings
Aldurazyme net product revenue recorded was 17.5 million in the quarter
http://www.earningsimpact.com/Transcript/82569/BMRN/BioMarin-Pharmaceutical-Inc----Q2-2013-Earnings-Call
Goldman-Sachs Healthcare-Conference
Interesting Read..
http://www.earningsimpact.com/Transcript/82030/BMRN/BioMarin-Pharmaceutical-Inc----Goldman-Sachs-34th-Annual-Global-Healthcare-Conference
They did a PR much later then I thought! I was beginning to think we wouldn't get one till the actual data release. So the abstract does have less patients then the actual presentation.
Asco abstract-
A video mainly about one of the patients on 701 but also about the New Zealand governments unwillingness to pay for Pompe drugs (in adults).
http://www.3news.co.nz/Life-death-and-Pompe-disease/tabid/367/articleID/297947/Default.aspx
I wonder if there will be some IP issues with BMRN and this company
http://www.callidusbiopharma.com/
I just saw a PR of theirs and they seem to be approaching LSD's exactly how BMRN-701 is.
An NIH PKU conference from February 2012
http://videocast.nih.gov/summary.asp?Live=12444&bhcp=1
Ultragenyx raises $75 million to push 'Lazarus-like' 3rd drug into mid-stage study
http://www.bizjournals.com/sanfrancisco/blog/biotech/2012/12/ultragenyx-rare-disease-funding.html?ana=yfcpc&page=all
Looks like 2014 may be the IPO year for Kakkis's company
An updated story on one of the Pompe trial participants. I believe she was on the 10mg/kg dose. Normally I wouldn't post patient info but since she and others I've posted about here have news paper stories I feel OK doing so.
http://www.stuff.co.nz/national/health/8021801/Drug-trial-gives-hope-to-Pompe-sufferer
Just Banked Short POS here +$$$$::)))
Love the atm here today!!!::)))Trading window is so wide...49.18 - 48.21
::)))
Below 48 EOD.::)))
:05AM Biomarin Pharm's Phase 3 study of GALNS for the treatment of MPS IVA meets primary endpoint; co Plans to submit marketing applications starting in 1Q13 (BMRN) 37.42 : Co announced today that the pivotal Phase 3 study of GALNS met the primary endpoint of change in six-minute walk distance compared with placebo at 24 weeks in subjects receiving weekly infusions of GALNS at the dose of 2 mg/kg. MOR-004 was a randomized, double-blind, placebo-controlled study evaluating two doses of GALNS for the treatment of patients with the rare lysosomal storage disorder Mucopolysaccharidosis Type IVA, also called Morquio A Syndrome. Patients dosed with GALNS at 2 mg/kg every other week did not show a meaningful or statistically significant change from baseline compared to placebo.
The company also announced preliminary data from the MOR-005 extension study which suggests that clinical benefits continue to improve with further dosing with GALNS. Only a limited number of patients have reached the 36 or 48 week points of total time on treatment in the extension study, and the results will be updated when the study is completed. The company confirmed that based on the results of MOR-004, and following planned discussions with regulatory authorities, it expects to submit marketing applications starting in the first quarter of 2013.
The primary endpoint of the study, change in six-minute walk distance at 24 weeks, was statistically significant in patients dosed with GALNS at 2 mg/kg every week with a mean increase of 22.5 meters (p=0.0174) over placebo.
On the secondary endpoint of three-minute stair climb, patients dosed with GALNS at 2 mg/kg every week showed a trend toward improvement at 24 weeks of 1.1 additional stairs per minute over placebo.
In MOR-004, GALNS was generally well-tolerated and adverse events were similar to those seen in clinical trials of other enzyme replacement therapies.
For long term investors I think the data compare well with other ERT's and the drug should gain approval. This will also be a quicker ramp and probably equal (or exceed) the revenue for all of their current products combined in a few years.
If you are a short term trader I can't say what the stock will do. However they also have important data from two other programs in the coming months (PARP and Pompe). What they have said about the PARP program seem promising. Pompe we only know about 3 patients in the low dose. So something negative could turn up to hurt the stock but the data could continue to be positive as well!
Payday !
congrats
MK
A buy here for a galns move to 45-50 by November 16 looks decent.
Haven't posted here much lately for those who monitor this board a couple of things from the Q3 call/PR:
1-GALNS data before thanksgiving
2-PARP program doesn't get much press but it could advance to Phase 3 in as early as end of '13 which is quite fast! That would likely be for BRCA mutations.
3-Pompe program has fully enrolled (16) patients in the highest dosing cohort (20mg/kg) and data is expected in Q1 '13.
Oct 05, 2012 (Marketintelligencecenter.com via COMTEX) --
BioMarin Pharmaceutical (NASDAQ:BMRN) closed Thursday's positive trading session at $40.87. In the past year, the stock has hit a 52-week low of $30.06 and 52-week high of $44.18. BioMarin Pharmaceutical (BMRN) stock has been showing support around $40.33 and resistance in the $41.39 range. Technical indicators for the stock are Bullish and S&P gives BioMarin Pharmaceutical (BMRN) a positive 4 STARS (out of 5) buy rating. For a hedged play on BioMarin Pharmaceutical (BMRN), look at the Jan '13 $39.00 covered call for a net debit in the $34.87 area. That is also the break-even stock price for this trade. This covered call has a duration of 106 days, provides 14.68% downside protection and an assigned return rate of 11.84% for an annualized return rate of 40.78% (for comparison purposes only). BioMarin Pharmaceutical (BMRN) has a current trailing average dividend yield of 0%.
MarketIntelligenceCenter.com
________________________________________________________
http://futures.tradingcharts.com/news/futures/Potential_BioMarin_Pharmaceutical__BMRN__Trade_Targets_40_78__Return_186772532.html
BMRN
Jefferies Says GALNS to drive 30% Upside in BioMarin (BMRN)
Jefferies reiterated a Buy on BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) and raised its price target to $46.00 (from $44.00).
Jefferies said their experts’ discussions indicate probability of successful GALNS Phase 3 for Morquio A syndrome (MPS IV) at 60-85 percent.
"If successful, we forecast 25-30% upside from current levels," said analyst Eun K. Yang.
"BMRN expects GALNS BLA filing in 1Q13, with potentially regulatory approval by 4Q13. We forecast GALNS to turn BMRN to profitability, with peak annual sales potential of $500M," he said.
For an analyst ratings summary and ratings history on BioMarin Pharmaceutical Inc. click here. For more ratings news on BioMarin Pharmaceutical Inc. click here.
Shares of BioMarin Pharmaceutical Inc. closed at $40.97 yesterday, with a 52 week range of $29.50-$44.18.
_______________________________________________________
http://www.streetinsider.com/Analyst+Comments/Jefferies+Says+GALNS+to+drive+30%25+Upside+in+BioMarin+(BMRN)/7768341.html
BMRN
BioMarin Pharmaceutical (BMRN) Showing Bullish Technicals But Could Break Through $40.68 Resistance
Oct 01, 2012 (Marketintelligencecenter.com via COMTEX News Network) -- BioMarin Pharmaceutical (NASDAQ: BMRN) closed Friday's trading session at $40.26. In the past year, the stock has hit a 52-week low of $29.50 and 52-week high of $44.18. BioMarin Pharmaceutical (BMRN) stock has been showing support around $39.62 and resistance in the $40.68 range. Technical indicators for the stock are Bullish and S&P gives BioMarin Pharmaceutical (BMRN) a positive 4 STARS (out of 5) buy rating. For a hedged play on BioMarin Pharmaceutical (BMRN), look at the Jan '13 $37.00 covered call for a net debit in the $33.46 area. That is also the break-even stock price for this trade. This covered call has a duration of 110 days, provides 16.89% downside protection and an assigned return rate of 10.58% for an annualized return rate of 35.11% (for comparison purposes only). BioMarin Pharmaceutical (BMRN) has a current trailing average dividend yield of 0%.
MarketIntelligenceCenter.com
(C) Copyright 2012, Marketintelligencecenter.com. All rights reserved.
_____________________________________________________________
http://www.citiadr.idmanagedsolutions.com/news/global_story.idms?type=scrolling&ID_NEWS=247194430
BMRN
Summer Street Sets Biomarin Pharm Price Target at $52.00 (BMRN)
September 28th, 2012 by Stacy Sanders
Investment analysts at Summer Street boosted their target price on shares of Biomarin Pharm (NASDAQ: BMRN) from $45.00 to $52.00 in a note issued to investors on Friday. The firm currently has a “buy” rating on the stock.
Shares of Biomarin Pharm traded down 0.30% during mid-day trading on Friday, hitting $40.08. Biomarin Pharm has a one year low of $29.50 and a one year high of $44.18. The company’s market cap is $4.952 billion.
Biomarin Pharm last issued its quarterly earnings data on Wednesday, August 1st. The company reported ($0.27) EPS for the quarter, missing the Thomson Reuters consensus estimate of ($0.19) by $0.08. The company’s quarterly revenue was up 12.1% on a year-over-year basis. On average, analysts predict that Biomarin Pharm will post $-0.87 earnings per share for the current fiscal year.
BMRN has been the subject of a number of other recent research reports. Analysts at William Blair raised their price target on shares of Biomarin Pharm from $46.00 to $49.00 in a research note to investors on Thursday. They now have an “outperform” rating on the stock. Separately, analysts at Brean Murray raised their price target on shares of Biomarin Pharm from $41.00 to $47.00 in a research note to investors on Thursday. They now have a “buy” rating on the stock. Finally, analysts at RBC Capital reiterated an “outperform” rating on shares of Biomarin Pharm in a research note to investors on Monday.
BioMarin Pharmaceutical Inc. (BioMarin) develops and commercializes pharmaceuticals for serious diseases and medical conditions.
Keep up with the latest analysts' ratings by subscribing to Daily Political's daily email update. With your complimentary subscription, you will receive a concise summary of stock analysts' upgrades, downgrades and new coverage.
_________________________________________________________
http://www.dailypolitical.com/finance/stock-market/summer-street-sets-biomarin-pharm-price-target-at-52-00-bmrn.htm
BMRN
Twin Orphan Drug News
By Brian Orelli, The Motley Fool
Posted 1:26PM 09/27/12
BioMarin Pharmaceuticals (NAS: BMRN) keeps chugging along, pushing its orphan drug pipeline forward. Yesterday, the biotech announced data from two trials, giving the company confidence to move two programs to the next step.
First, phase 2 data for PEG-PAL, which treats phenylketonuria, looked good. The drug reduced blood Phe levels to 68% of pre-treatment baseline levels in patients treated for one year. And all those patients had Phe measurements below 600 umol/L, substantially below the 900 umol/L recommended by the National Institutes of Health.
BioMarin already has an approved drug to treat phenylketonuria, but Kuvan isn't nearly as good. Only 20% of patients reached their Phe target and the overall reduction was only 29%. PEG-PAL may cannibalize some of the Kuvan sales, but since it's much more effective, PEG-PAL will likely capture patients who would otherwise try to treat their phenylketonuria with a low Phe diet.
BioMarin plans to move PEG-PAL into phase 3 in the second quarter of 2013 after it meets with the Food and Drug Administration to get its input on the trial design.
The second trial was of the early, phase 1 variety, testing BMN-111 for achondroplasia, the most common form of dwarfism. The drug is designed to indirectly inhibit fibroblast growth factor receptor 3, which is overactive in patients with achondroplasia.
This was only a phase 1 trial in healthy patients to determine the proper dose to move into phase 2 development, but at least no safety issues were identified and BioMarin can work on designing the phase 2 proof-of-concept trial.
Moving these two drugs along is important for the overall growth of the company. BioMarin, Alexion Pharmaceutical (NAS: ALXN) , ViroPharma (NAS: VPHM) , and Shire (NAS: SHPG) have made big bucks selling drugs for orphan diseases that affect relatively few individuals. But it's easy to saturate the market when there isn't any competition, leaving new products as the obvious driving force for higher revenue.
While BioMarin rules the orphan drug space, these three companies are setting themselves up to dominate retail. Find out what they are and why Fool analysts like them so much in the new free report, "3 Companies Ready to Rule Retail." Get your copy for free by clicking here now.
The article Twin Orphan Drug News originally appeared on Fool.com
__________________________________________________________
http://www.dailyfinance.com/2012/09/27/twin-orphan-drug-news/
BMRN
Followers
|
16
|
Posters
|
|
Posts (Today)
|
0
|
Posts (Total)
|
694
|
Created
|
01/10/07
|
Type
|
Free
|
Moderators |
http://finance.yahoo.com/q/ks?s=BMRN
BioMarin Pharmaceutical Inc. develops and commercializes biopharmaceuticals for serious diseases and medical conditions. Its product portfolio comprises approved products and multiple investigational product candidates. The company?s approved products include Naglazyme (galsulfase), a recombinant form of N-acetylgalactosamine 4-sulfatase enzyme used to treat genetic disease called mucopolysaccharidosis (MPS)VI; Kuvan (sapropterin dihydrochloride), a proprietary synthetic oral form of 6R-BH4, a naturally occurring enzyme co-factor for phenylalanine hydroxylase indicated for patients with Phenylketonuria (PKU); and Aldurazyme (laronidase) that is used to treat mucopolysaccharidosis I caused by the deficiency of a lysosomal enzyme called alpha-L-iduronidase. Its clinical development products comprise various product candidates for the treatment of genetic diseases, which include PEG-PAL, an enzyme substitution therapy under Phase I clinical development to treat phenylketonurics, which are not responsive to Kuvan; 6R-BH4, a Phase II clinical development product for the treatment of multiple cardiovascular indications, including sickle cell disease; and GALNS, an enzyme replacement therapy for MPS Type IV or Morquio Syndrome Type A (MPS IV A), a lysosomal storage disease. In addition, the company is conducting preclinical development of various other enzyme product candidates for genetic and other diseases, including a small molecule for the treatment of Duchenne muscular dystrophy. Further, BioMarin holds rights to receive royalties related to Orapred and Orapred ODT as a result of sublicense of North American rights. The company sells its products to specialty pharmacies, hospitals acting as retailers, and pharmaceutical wholesalers in the United States, Europe, and internationally. It has a strategic alliance with Merck Serono S.A. and joint venture with Genzyme Corporation. The company was founded in 1996 and is headquartered in Novato, California.
Volume | |
Day Range: | |
Bid Price | |
Ask Price | |
Last Trade Time: |