Alnylam Pharmaceuticals, Inc.’s (ALNY - Free Report) shares plunged 15.7% on Thursday in response to a death event reported by the company in an open-label extension phase II study on fitusiran. Alnylam has stopped dosing in all ongoing studies on its hemophilia candidate, fitusiran, following the fatal event.
The company will resume dosing after discussion with global regulatory authorities and proper protocol amendments for enhanced patient safety monitoring in place. Alnylam is currently reviewing the risk-benefit profile and developing a risk mitigation strategy for fitusiran.
However, shares of Alnylam have increased 93.7% so far this year, significantly outperforming the industry’s gain of 16.4%.
Fitusiran, an RNAi based therapy, is being studied in a phase II study on patients with and without inhibitors and a phase III ATLAS study. The ATLAS study is yet to initiate dosing.
Hemophilia is an inherited bleeding disorder due to deficiency of thrombin. It affects the clotting of blood, causing spontaneous bleeding from even minor injuries, which can become potentially fatal.
The patient who reportedly died in the study was suffering from hemophilia A. The death occurred due to clotting of blood inside cerebral venous sinus (thrombotic event).
Alnylam has an agreement with Sanofi (SNY - Free Report) to co-develop RNAi based therapy for rare genetic disorders.
Shire plc (SHPG - Free Report) is also developing its recombinant factor VIII (FVIII) gene therapy candidate, SHP654, to treat patients with hemophilia A.
The company is developing another RNAi based therapy, givosiran, for the treatment of acute hepatic porphyrias. Giovosarin has shown promising results in previous clinical studies and a phase III study is expected to start in late 2017.
Zacks Rank & Stock to Consider
Alnylam has a Zacks Rank #3 (Hold). Ligand Pharmaceuticals Incorporated (LGND - Free Report) is a better-ranked biomedical company with a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Ligand’s earnings estimates increased 10.4% to $2.98 for 2017 and 2.2% to $3.73 for 2018 over the last 60 days. The company delivered an average earnings beat of 6.19% for the four trailing quarters. The stock is up 33.3% so far this year.
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