This Day On The Street
Continue to site right-arrow
ADVERTISEMENT
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
TheStreet Open House

UniQure Initiates Phase I In Acute Intermittent Porphyria

AMSTERDAM, December 11, 2012 /PRNewswire/ --

uniQure B.V., a leader in the field of human gene therapy, today announced the start of its Phase I clinical trial in acute intermittent porphyria (AIP) with the treatment of the first patient. The study is conducted under the aegis of the AIPGENE consortium, a pan-European collaboration funded in part by the European Commission's Seventh Framework Program with the aim to develop a gene therapy for the treatment of AIP, a rare and devastating disease caused by mutations in the porphobilinogen deaminase gene (PBGD). AIP can be life-threatening and the long-term effects include irreversible nerve damage, liver cancer and kidney failure. uniQure was granted orphan drug designation for the treatment of AIP in 2009 from the European Medicines Agency.

"The start of the AIP Phase I study marks the first of four programs that will enter clinical trials over the next 12 months," says Jörn Aldag, CEO of uniQure.  "After AIP we expect clinical trials to be initiated in Parkinson's disease, hemophilia B, and Sanfilippo B. After many years of building and developing our capabilities and competencies, and the approval in November of Glybera for LPLD as the first gene therapy in the Western world, we are highly motivated to expedite the clinical development of our other advanced gene therapies."

About the AIP Phase I study

The Phase I will enroll eight patients with severe AIP at two centers: the Clinical University of Navarra, Pamplona, Spain, and the 12 de Octubre University Hospital,, Madrid, Spain. The study's primary objective is the assessment of safety and determination of the maximum tolerated dose.  Secondary objectives include tolerability of treatment, pharmacokinetics, changes in the levels of surrogate markers of activity including porphobilinogen (PBG) and delta-aminolevulinic acid (ALA), and assessment of symptom control, neuro-psychological changes and quality of life. All patients will be followed for one year, and the interim results of the Phase I are expected in Q3 2013.

About acute intermittent porphyria

1 of 2

Check Out Our Best Services for Investors

Action Alerts PLUS

Jim Cramer and Stephanie Link reveal their investment tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
Quant Ratings

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
Stocks Under $10

Jim Cramer's protégé, 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
Try it NOW
Try it NOW
Try it NOW

Check Out Our Best Services for Investors

Dividend Stock Advisor

Jim Cramer's protégé, 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
  • Updates with exact steps to take - BUY, HOLD, SELL
Trifecta Stocks

Every recommendation goes through 3 layers of intense scrutiny—quantitative, fundamental and technical analysis—to maximize profit potential and minimize risk.

Product Features:
  • Model Portfolio
  • Intra Day Trade alerts
  • Access to Quant Ratings
Options Profits

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

Product Features:
  • Actionable options commentary and news
  • Real-time trading community
Try it NOW
Try it NOW
Try it NOW
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!

Markets

DOW 17,164.95 -251.90 -1.45%
S&P 500 1,994.99 -26.26 -1.30%
NASDAQ 4,635.24 -48.1670 -1.03%

Partners Compare Online Brokers

Free Reports

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