This account is pending registration confirmation. Please click on the link within the confirmation email previously sent you to complete registration.
Need a new registration confirmation email? Click here
Stocks Under $10 with 50-100% upside potential - 14 days FREE!

BioMarin Shares Jump On Positive GALNS Study Results

Updated with new information, stock price/

SAN RAFAEL, Calif. (TheStreet) --BioMarin Pharmaceuticals (BMRN) said Monday that patients with a rare genetic disorder known Morquio A Syndrome were able to walk further following treatment with the company's experimental drug GALNS, satisfying the main goal of a late-stage study.

Based on the positive results from the Morquio A study, BioMarin plans to seek regulatory approval for GALNS in the first quarter of 2013.

BioMarin shares opened for regular trading Monday up 25% to $46.80. GALNS is seen as the most important drug in the BioMarin's pipeline, with the potential to double the company's revenue.

"We view the result as clearly positive for BioMarin and see it as a best-case outcome," writes JP Morgan analyst Cory Kasimov in a note to clients. Kasimov expects BioMarin shares to move higher not only because of GALNS' sales potential but because generalist investors will likely start buying BioMarin given the orphan drug experience with the Genzyme unit of Sanofi (SNY)and Alexion Pharmaceuticals (ALXN).

In patients with Morquio syndrome, also known as MPS IV, a missing enzyme prevents the breakdown of keratan sulftate in cells, leading to progressive skeletal and spinal cord malformation. GALNS is a replacement enzyme that reduces the harmful levels of keratin sulfate.

Morquio syndrome is a rare disease, with an estimated prevalence of between 1,000 and 1,500 patients in the U.S., Europe and Japan and between 1,500 and 2,000 patients in the rest of the world, BioMarin estimates.

In the phase III study, patients treated with a weekly infusion of GALNS were able to walk an average of 22.5 meters further in six minutes compared to patients treated with a placebo. The benefit in walking distance, the study's primary endpoint, was statistically significant.

BioMarin also tested an every other week dose of GALNS but walking distance was not improved compared to placebo.

Treatment with GALNS every week also demonstrated improved trends in three-minute stair climb and a statistically significant 41% reduction in keratan sulfite levels in the urine. Lung function in patients was also improved.

GALNS was shown to be generally well tolerated with adverse events similar to what's observed in other enzyme replacement therapies, BioMarin said.

-- Reported by Adam Feuerstein in Boston.

Adam Feuerstein writes regularly for TheStreet. In keeping with company editorial policy, he doesn't own or short individual stocks, although he owns stock in TheStreet. He also doesn't invest in hedge funds or other private investment partnerships. Feuerstein appreciates your feedback; click here to send him an email.

Select the service that is right for you!

COMPARE ALL SERVICES
Action Alerts PLUS
Try it NOW

Jim Cramer and Stephanie Link actively manage a real portfolio and reveal their money management tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
  • Weekly roundups
TheStreet Quant Ratings
Try it NOW
Only $49.95/yr

Access the tool that DOMINATES the Russell 2000 and the S&P 500.

Product Features:
  • Buy, hold, or sell recommendations for over 4,300 stocks
  • Unlimited research reports on your favorite stocks
  • A custom stock screener
  • Upgrade/downgrade alerts
Stocks Under $10
Try it NOW

David Peltier, uncovers low dollar stocks with extraordinary upside potential that are flying under Wall Street's radar.

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
  • Weekly roundups
Dividend Stock Advisor
Try it NOW

Jim Cramer's protege, David Peltier, identifies the best of breed dividend stocks that will pay a reliable AND significant income stream.

Product Features:
  • Diversified model portfolio of dividend stocks
  • Alerts when market news affect the portfolio
  • Bi-weekly updates with exact steps to take - BUY, HOLD, SELL
Real Money Pro
Try it NOW

All of Real Money, plus 15 more of Wall Street's sharpest minds delivering actionable trading ideas, a comprehensive look at the market, and fundamental and technical analysis.

Product Features:
  • Real Money + Doug Kass Plus 15 more Wall Street Pros
  • Intraday commentary & news
  • Ultra-actionable trading ideas
Options Profits
Try it NOW

Our options trading pros provide daily market commentary and over 100 monthly option trading ideas and strategies to help you become a well-seasoned trader.

Product Features:
  • 100+ monthly options trading ideas
  • Actionable options commentary & news
  • Real-time trading community
  • Options TV
To begin commenting right away, you can log in below using your Disqus, Facebook, Twitter, OpenID or Yahoo login credentials. Alternatively, you can post a comment as a "guest" just by entering an email address. Your use of the commenting tool is subject to multiple terms of service/use and privacy policies - see here for more details.
Submit an article to us!
DOW 17,098.45 +18.88 0.11%
S&P 500 2,003.37 +6.63 0.33%
NASDAQ 4,580.2710 +22.5760 0.50%

Brokerage Partners

Rates from Bankrate.com

  • Mortgage
  • Credit Cards
  • Auto

Free Newsletters from TheStreet

My Subscriptions:

After the Bell

Before the Bell

Booyah! Newsletter

Midday Bell

TheStreet Top 10 Stories

Winners & Losers

Register for Newsletters
Top Rated Stocks Top Rated Funds Top Rated ETFs