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
Stocks Under $10 with 50-100% upside potential - 14 days FREE!

Alnylam Advances Development Candidate For ALN-AS1, A Subcutaneously Administered RNAi Therapeutic Targeting Aminolevulinate Synthase 1 (ALAS-1) For The Treatment Of Hepatic Porphyrias

Stocks in this article: ALNY

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it is advancing its Development Candidate for ALN-AS1, an RNAi therapeutic targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of hepatic porphyrias including acute intermittent porphyria (AIP). The new pre-clinical research findings, presented at the 9 th Annual Meeting of the Oligonucleotide Therapeutics Society being held October 6 – 8, 2013 in Naples, Italy, show that subcutaneous administration of a GalNAc-siRNA targeting ALAS-1 leads to rapid, dose-dependent, and long-lasting knockdown of ALAS-1 mRNA and complete inhibition of the toxic intermediates that mediate the symptoms and pathology of AIP. Based on these findings, including results in non-human primate studies, the company has selected its ALN-AS1 Development Candidate and expects to file an Investigational New Drug (IND) application for this RNAi therapeutic in 2014. ALN-AS1 is part of the company’s “Alnylam 5x15” product development and commercialization strategy, in which the company aims to advance five genetic disease target programs into clinical development, including programs in late stages, by the end of 2015. In addition, the company presented new pre-clinical data with its proprietary, clinically validated GalNAc-siRNA conjugate delivery platform for subcutaneous delivery of RNAi therapeutics with a wide therapeutic index.

“We are very pleased to advance ALN-AS1 as a new Development Candidate in our ‘Alnylam 5x15’ pipeline, with the goal of filing an IND in 2014. Our new pre-clinical data show that subcutaneous administration of ALN-AS1 results in complete suppression of the toxic heme biosynthesis intermediates that cause the symptoms and pathology of AIP,” said Rachel Meyers, Ph.D., Vice President of Research and RNAi Lead Development at Alnylam. “ALN-AS1 now becomes our third RNAi therapeutic utilizing our GalNAc-siRNA conjugate delivery platform to enter development stages, extending our progress with ALN-TTRsc for the treatment of transthyretin-mediated amyloidosis, where we intend to soon initiate a Phase II trial, and ALN-AT3 for the treatment of hemophilia, where we plan on filing our IND by the end of this year. With the recent clinical validation of our GalNAc-siRNA conjugate delivery platform, we have increased confidence that ALN-AS1 could become a transformative therapy for patients with AIP, an ultra-rare genetic disease with enormous unmet medical need. We very much look forward to filing our IND for this program in 2014.”

1 of 6

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!

Markets

DOW 17,804.80 +26.65 0.15%
S&P 500 2,070.65 +9.42 0.46%
NASDAQ 4,765.38 +16.9840 0.36%

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