Alnylam Pharmaceuticals, Inc. ALNY announced that it has started a phase I study on its investigational RNAi therapeutic ALN-AS1 for the treatment of acute hepatic porphyrias including acute intermittent porphyria (AIP).
To begin with, Alnylam intends to conduct the study in AIP patients, who are asymptomatic “high excreters” (ASHE). Later on, the study will enroll AIP patients who experience recurrent porphyria attacks.
The study will be conducted in three parts. Parts A and B will be randomized, single-blind, dose-escalation studies, in which up to a total of 40 ASHE patients will be enrolled. However, Part A will be a single-dose and Part B a multi-dose study. The primary objective of Parts A and B is to evaluate safety and tolerability of single and multiple subcutaneous doses of ALN-AS1. Part C, on the other hand, will be an open-label, multi-dose study, which will assess the safety, tolerability, PK/PD and clinical activity of multiple doses of ALN-AS1 in up to eight AIP patients, who experience recurrent porphyria attacks.
Initial data from the study should be out in early 2016.
According to the American Porphyria Foundation, at present there are limited treatment options for acute hepatic porphyrias, especially for those suffering from recurrent attacks, which often necessitate monthly hospitalizations for administration of hematin and pain management. Hence, there is a need for new therapies in this field.
We remind investors that Alnylam has an agreement with Sanofi SNY for accelerating and expanding the development and commercialization of RNAi therapeutics across the world. While Alnylam retains product rights in North America and Western Europe, Sanofi has the right to access certain programs in Alnylam’s current and future genetic medicines pipeline in the rest of the world.
Alnylam carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector are Actelion Ltd. ALIOF and Gilead Sciences Inc. GILD. Both stocks hold a Zacks Rank #1 (Strong Buy).
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