Alnylam Pharmaceuticals, Inc.’s (NASDAQ:ALNY) shares jumped 10.7% after the company announced preliminary results from the ongoing phase II open-label extension (OLE) studies on its pipeline candidates, patisiran and revusiran. Data are being presented at the XV International Symposium on Amyloidosis.
We note that both patisiran and revusiran are experimental RNAi therapeutics targeting transthyretin (TTR) and are being evaluated for the treatment of hereditary TTR-mediated amyloidosis (hATTR amyloidosis).
Data from the single-arm phase II OLE study showed that following 24 months of treatment with patisiran, hATTR patients with polyneuropathy (hATTR-PN; also known as familial amyloidotic polyneuropathy) witnessed improvement or no change in the mean neuropathy impairment score. Overall, these data support the therapeutic hypothesis that reduction of mutant and wild-type TTR with patisiran has the potential to halt or improve neuropathy progression in patients with hATTR-PN.
Alnylam also presented baseline demographics from the APOLLO phase III study on patisiran in hATTR-PN patients, which showed that a significant number of patients (54%) demonstrated evidence of cardiac disease. The company believes this patient subset should allow for a controlled evaluation of effects of patisiran on cardiac parameters. Alnylam expects to report data from the APOLLO study in mid-2017.
In addition, initial 12-month data from the phase II OLE study revealed that revusiran continued to show robust and sustained knockdown of serum TTR. Moreover, 6-minute walk distance results were found to be generally stable in the majority of evaluable hATTR patients with cardiomyopathy (also known as familial amyloidotic cardiomyopathy).
Alnylam also announced that it expects to complete enrollment in the ENDEAVOUR phase III study on revusiran this summer, ahead of schedule. Data readout is now anticipated in early 2018. Previously, the company had expected to complete enrollment in the study in late 2016 while results were projected in mid-2018.
Considering that Alnylam has no approved product in its portfolio, successful development of both patisiran and revusiran is crucial for the company. We expect investor focus to remain on updates pertaining to their development.
Alnylam is a Zacks Rank #3 (Hold) stock. Some better-ranked stocks in the healthcare sector include ANI Pharmaceuticals, Inc. (NASDAQ:ANIP) , Innoviva, Inc. (NASDAQ:INVA) and Pfizer Inc. (NYSE:PFE) . While ANI Pharmaceuticals and Innoviva are Zacks Rank #1 (Strong Buy) stocks, Pfizer sports a Zacks Rank #2 (Buy).
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