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Alnylam Reveals Positive Initial Data on RNAi Drug ALN-CC5 - Analyst Blog

Alnylam Pharmaceuticals, Inc.ALNY announced initial positive results from an ongoing phase I/II study on ALN-CC5. ALN-CC5 is an investigational RNA interference (RNAi) therapeutic targeting complement component C5 for the treatment of complement-mediated diseases.

Initial results on healthy volunteers (n=12) showed that a single subcutaneous dose administration of ALN-CC5 led to a potent, dose-dependent, durable, and statistically significant knockdown of serum C5 of up to 96%. Moreover, inhibition of serum complement activity of up to 92% was achieved, including an up to 61% inhibition of serum hemolytic activity. Data from these studies revealed an approximate three-to-five-fold increase in potency for ALN-CC5 in humans as compared to non-human primates (NHP).

These data were presented at the recently held congress of the European Hematology Association (EHA). While the safety data are as of a data cut-off date of May 26, 2015, clinical activity data are as of a data cut-off date of Jun 4, 2015. ALN-CC5 has been found to be well tolerated with no adverse events being reported to date.

The phase I/II study is ongoing in both the SAD and multiple ascending dose (MAD) stages in healthy volunteers with additional data expected later this year. The company also remains on track to enroll patients with paroxysmal nocturnal hemoglobinuria (PNH) into the trial by year end.

ALN-CC5 is the company's fourth RNAi therapeutic program to demonstrate clinical activity and translation from NHP to humans.

We note that Alnylam has an agreement with Genzyme Corporation, a Sanofi SNY company, for the development and commercialization of RNAi therapeutics targeting rare diseases.

Alnylam currently carries a Zacks Rank #3 (Hold). Gilead Sciences Inc. GILD and Hospira Inc. HSP are some better-ranked stocks in the health care sector carrying a Zacks Rank #1 (Strong Buy).

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.


The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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