Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) announced that it has discontinued the phase III ENDEAVOUR study on revusiran for the treatment of hereditary ATTR amyloidosis (hATTR) with cardiomyopathy (hATTR-CM), also known as familial amyloidotic cardiomyopathy (FAC). We expect investors to react negatively on the news.
The decision followed the recommendation of a Data Monitoring Committee (DMC) which suggested that the benefit-risk profile of the candidate did not support continued dosing in patients. Moreover, unblinded data from the study revealed an imbalance in mortality in patients dosed with revusiran, compared to placebo.
However, Alnylam stated that this decision will not affect the development of the other late-stage candidate, patisiran, or other RNAi therapeutic programs in its pipeline. Patisiran is currently being evaluated in a phase III study – APOLLO – for the treatment of patients with hATTR amyloidosis with polyneuropathy (hATTR-PN), also known as familial amyloidotic polyneuropathy (FAP).
Despite the latest setback, the company has reaffirmed its target of achieving a portfolio of three marketed products by the end of 2020.
We remind investors that Alnylam has a worldwide collaboration agreement with Sanofi (SNY - Free Report) for the development and commercialization of RNAi therapeutics – patisiran and revusiran.
Note that hATTR represents high unmet need, with approximately 10,000 people affected with FAP and at least 40,000 people suffering from FAC in the world.
Given the lack of treatment options for this devastating disease, the decision to stop evaluating revusiran is highly disappointing. Moreover, given that Alnylam has no approved product in its portfolio, successful development of revusiran was crucial for the company.
Shares of Alnylam tanked during Thursday's pre-market trading session.
The company currently carries a Zacks Rank #3 (Hold).
Stocks to Consider
A couple of better-ranked stocks in the health care sector are Anika Therapeutics, Inc. (ANIK - Free Report) and Geron Corporation (GERN - Free Report) . Both the stocks sport a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Anika Therapeutics’ earnings estimates for 2016 and 2017 were up a respective 9.5% and 12.8% over the last 60 days. The company has beaten earnings estimates in each of the last four quarters with an average surprise of 42.19%. Its share price has jumped 26.1% year to date.
Geron has delivered a positive earnings surprise in each of four trailing quarters, bringing the average beat to 20.78%.
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