This Day On The Street
Continue to site
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

Alnylam And Collaborators Publish Clinical Trial Results With RNAi Therapeutics Targeting Transthyretin (TTR) For The Treatment Of TTR-Mediated Amyloidosis (ATTR) In The New England Journal Of Medicine

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today the publication of complete study results from Phase I trials with ALN-TTR01 and ALN-TTR02 in the New England Journal of Medicine. The paper, titled “ Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis” (Coelho et al., N Engl J Med 2013;369:819-29), shows that RNAi therapeutics targeting transthyretin (TTR) achieved rapid, dose-dependent, durable, and specific knockdown of TTR, the disease-causing protein in TTR-mediated amyloidosis (ATTR). These results document the most robust proof of concept for RNAi therapy in man to date, including knockdown of serum TTR protein levels of up to 94% after just a single dose of drug.

“Our ATTR program is the lead effort in our ‘Alnylam 5x15’ product development and commercialization strategy, which is focused on advancing innovative RNAi therapeutics toward genetically defined targets for the treatment of diseases with high unmet medical need. Our overall clinical trial experience with RNAi therapeutics for the treatment of ATTR now includes a Phase I trial with ALN-TTR01, Phase I and II trials with ALN-TTR02, and a Phase I trial with ALN-TTRsc, a subcutaneously administered RNAi therapeutic utilizing our proprietary GalNAc conjugate delivery platform,” said Akshay Vaishnaw, M.D., Ph.D., Executive Vice President and Chief Medical Officer of Alnylam. “The Phase I studies of ALN-TTR01 and ALN-TTR02 demonstrate key human proof of concept for RNAi therapeutics targeting TTR but also for liver-expressed target genes in general, and we are thrilled to be publishing these landmark data in the New England Journal of Medicine. Notably, this is the first time that clinical results with an RNAi therapeutic have been published in the Journal, and we are proud to be associated with this milestone as we lead the clinical translation of RNAi therapeutics as a whole new class of innovative medicines.”

“This new paper describes our clinical trial experience with ALN-TTR01 and ALN-TTR02, which utilize first- and second-generation lipid nanoparticle formulations, respectively. Relative to ALN-TTR01, the results for ALN-TTR02 showed exceptional improvement in potency, without any apparent loss in tolerability, thus documenting the important advances made by Alnylam scientists and collaborators on systemic delivery of RNAi therapeutics. Indeed, with ALN-TTR02 we achieved very robust effects, including up to 94% reduction of serum TTR and a nearly 80% level of suppression sustained at one month with just a single dose. Further, ALN-TTR02 was also found to be generally safe and well tolerated,” said Jared Gollob, M.D., Vice President, Clinical Research at Alnylam. “Knockdown of circulating TTR is expected to result in improved clinical outcomes for patients with ATTR based on data from patients receiving liver transplants. Further, evidence from other systemic amyloidotic diseases shows that as little as a 50% reduction of the disease-causing protein can result in disease improvement or stabilization. Accordingly, we believe the findings published today in the New England Journal of Medicine suggest that the robust, sustained knockdown of TTR levels observed in our clinical trials with ALN-TTR02 may ameliorate the course of disease in patients with ATTR.”

ALN-TTR01 and ALN-TTR02 are systemically delivered RNAi therapeutics using first- and second-generation lipid nanoparticle (LNP) formulations, respectively, that encapsulate an identical siRNA targeting both wild-type and mutant TTR mRNA. The ALN-TTR01 Phase I trial enrolled participants from July 2010 through September 2011, and the ALN-TTR02 Phase I trial enrolled participants from March through May 2012. Both trials were designed as multi-center, randomized, single-blind, placebo-controlled, dose-ranging studies in patients with ATTR (n=32) – in the case of ALN-TTR01, or in healthy adult volunteers (n=17) – in the case of ALN-TTR02. The primary objective of each study was to evaluate the safety and tolerability of a single dose of ALN-TTR01 or ALN-TTR02. In addition, preliminary clinical activity of each drug was evaluated based on measurements of serum TTR protein levels.

1 of 7

Check Out Our Best Services for Investors

Action Alerts PLUS

Portfolio Manager Jim Cramer and Director of Research Jack Mohr 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

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

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
14-Days Free
Only $9.95
14-Days Free
Dividend Stock Advisor

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
Real Money

More than 30 investing pros with skin in the game give you actionable insight and investment ideas.

Product Features:
  • Access to Jim Cramer's daily blog
  • Intraday commentary and news
  • Real-time trading forums
Only $49.95
14-Days Free
14-Days Free
AAPL $92.90 -0.77%
FB $117.30 -0.64%
GOOG $700.75 0.73%
TSLA $212.09 -4.70%
YHOO $36.96 2.70%


Chart of I:DJI
DOW 17,630.96 -20.30 -0.12%
S&P 500 2,048.14 -2.98 -0.15%
NASDAQ 4,715.7220 -9.9170 -0.21%

Free Reports

Top Rated Stocks Top Rated Funds Top Rated ETFs