Alnylam Pharmaceuticals, Inc. ALNY presented data at the annual meeting of the Oligonucleotide Therapeutics Society.
Alnylam presented pre-clinical data along with program guidance on ALN-TTRsc02 for the treatment of TTR-mediated amyloidosis (ATTR amyloidosis).
Alnylam expects to file an Investigational New Drug (IND) application in early 2016. Initial data from a phase I study is expected in late 2016. The company plans to initiate a phase III study on ALN-TTRsc02 in 2017, assuming that results from the early-stage studies are positive.
According to information provided by the company, approximately 10,000 people and at least 40,000 people in the world are affected by familial amyloidotic polyneuropathy (FAP), and familial amyloidotic cardiomyopathy (FAC), respectively. While, FAP patients have a life expectancy of 5 years to 15 years from symptom onset, FAC is fatal within 2.5 years to 5 years of diagnosis.
We note that Alnylam is making progress on its pipeline for the treatment of ATTR amyloidosis. Currently, the company is evaluating patisiran (phase III) for the treatment of ATTR amyloidosis patients with FAP and revusiran (phase III) for the treatment of ATTR patients with FAC.
Apart from data on the pre-clinical study on ALN-TTRsc02, company presented data on two new platform technologies – Bis-RNAi and Reversir.
The company believes both platforms can expand the opportunities for RNAi therapeutics as a new class of medicines. Alnylam intends to advance the first candidate from both platforms in 2016.
Alnylam carries a Zacks Rank #4 (Sell). Some better-ranked stocks in the health care sector include Anika Therapeutics Inc. ANIK, Endo International plc ENDP and Nexvet Biopharma Public Limited Company NVET, each sporting a Zacks Rank #1 (Strong Buy).
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