The FDA Just Blessed A Hemophilia Trial From This Biotech

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Alnylam Pharmaceuticals ( ALNY ) edged up Friday after the Food and Drug Administration lifted its clinical hold on trials of a hemophilia drug, allowing Alnylam to start administering the drug before the year ends.

[ibd-display-video id=3023676 width=50 float=left autostart=true] The announcement is in line with prior commentary from Alnylam that it had reached an agreement with the FDA on risk mitigation measures for the drug known as fitusiran, Leerink analyst Paul Matteis wrote in a note to clients.

Alnylam expects to resume a Phase 2 study and a Phase 3 trial before January. The trials previously were placed on hold after a patient with hemophilia A suffered a fatal blood clot.

Under the new risk mitigation strategy, Alnylam has implemented guidelines for reduced doses of replacement factor or bypassing agents to treat any breakthrough bleeding, the firm said in a news release.

In morning trading on the stock market today , Alnylam shares lifted 0.5%, near 124.30, though they were up as much as 6.7% in earlier trades. Shares of partner Sanofi [ticker symbol=SNY] rose a fraction, near 43.20.

IBD'S TAKE:A number of biotechs either rocketed or tumbled during the American Society of Hematology conference last weekend in Atlanta. Head to IBD Industry Themes for more on how these blood disease-focused biotechs performed.

Fitusiran belongs to a class of drugs known as RNA inhibitors. It targets a small protein molecule known as antithrombin which inactivates several enzymes in the coagulation system. It's being investigated as a treatment for hemophilia A and B.

Matteis noted Roche 's ( RHHBY ) drug, Hemlibra - known in clinical testing as emicizumab - resulted in five thrombotic events in clinical testing. Hemlibra was approved in November to treat some patients with hemophilia A.

"While we believe fitusiran's mechanism of action may by nature impart increased risk of thrombotic events, the level of enthusiasm for Roche's emicizumab does offer evidence that some safety risk is acceptable in hemophilia for new mechanisms if they are effective," he said.

The hemophilia space has become increasingly competitive following recent progress with gene therapies, specifically a treatment from BioMarin Pharmaceuticals ( BMRN ), Matteis said. He kept his market perform rating on Alnylam, though he has an outperform rating on BioMarin.


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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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