Alnylam Pharmaceuticals, Inc. ALNY announced that it has submitted a Clinical Trial Application (CTA) for ALN-GO1 with the UK Medicines and Healthcare products Regulatory Agency for the treatment of patients with primary hyperoxaluria type 1 (PH1).
The company plans to initiate a phase I study in early 2016, upon approval. Initial data from the study are expected to be out in late 2016.
We note that Alnylam has an agreement with Genzyme Corporation, a Sanofi SNY company, for the development and commercialization of RNAi therapeutics targeting rare diseases. As per the terms of the agreement, Alnylam holds rights in North America and Western Europe, while Genzyme has rights to access certain programs in Alnylam's current and future Genetic Medicines pipeline, including ALN-GO1, in the rest of the world. In certain cases, both companies have global rights to co-develop and co-commercialize the products. In addition, Genzyme has the right to opt in, which can be exercised after it achieves the human proof of principle.
According to information provided by the company, in the world, approximately six to seven people per million are affected by PH1.
Meanwhile, other companies are also looking to bring their own PH1 treatments to market. Dicerna Pharmaceuticals, Inc. DRNA recently initiated the dosing of healthy volunteers in a phase I study on its PH1 treatment candidate, DCR-PH1. In addition, the company expects to commence a phase I study in PH1 patients in early 2016. Data from the study on healthy volunteers is expected in the second half of 2016.
Alnylam currently carries a Zacks Rank #3 (Hold). A better-ranked stock in the health care sector is Anika Therapeutics Inc. ANIK, with a Zacks Rank #1 (Strong Buy).
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