Alnylam Unveils Positive Initial Data on Acute Hepatic Porphyrias Treatment

Alnylam Pharmaceuticals reported over the weekend positive initial results from two cohorts that are part C of a phase 1 study evaluating givorisan as a treatment of acute hepatic porphyrias.

The company said the initial results showed that the treatment was generally well tolerated with no drug-related serious adverse events. Alnylam noted that givorisan achieved a 74% mean decrease in the annualized attack rate in porphyria patients with recurrent attacks.

The company said it plans to start a phase 3 study in late 2017.

Separately, Alnylam also unveiled positive interim phase 1 results for investigational RNAi therapeutic fitusiran in patients with hemophilia with inhibitors. The data showed that the treatment achieved a median estimated annualized bleeding rate of zero in patients with hemophilia A or B with inhibitors. The therapeutic was also generally well tolerated through the Oct. 6 cut-off date, with no thromboembolic events.

Alnylam said it is on track to start a phase 3 program on fitusiran early next year.

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