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news.US-based biopharmaceutical firm Alnylam Pharmaceuticals has started a Phase I open-label extension (OLE) trial with ALN-AT3, a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT), to treat hemophilia and rare bleeding disorders.
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The OLE trial is designed to evaluate the long-term safety and efficacy of ALN-AT3 and provide continued dosing for people with hemophilia enrolled in the Phase I study.
Clinical data from the Phase I OLE trial is expected to be reported at least once per year, beginning next year.
Alnylam R&D executive vice-president and chief medical officer Akshay Vaishnaw said: "This Phase I OLE trial brings us one step closer to our goal of providing a safe, subcutaneously administered, long-acting option for hemostasis management to people with hemophilia and other rare bleeding disorders.
"The initiation of this study marks our continued progress with ALN-AT3, and we are pleased to provide hemophilia patients in our Phase I trial the opportunity to receive this investigational medicine on an ongoing basis.
"We believe the ALN-AT3 data presented to date have been very promising, demonstrating potent, dose-dependent, and durable knockdown of AT with the potential to re-balance hemostasis in people with severe hemophilia through normalization of thrombin generation.
"We look forward to providing continued clinical updates, including results for once-monthly subcutaneous dosing in hemophilia patients, later this year."
The open-label, multi-center OLE trial will evaluate the long-term safety and tolerability of ALN-AT3 in hemophilia patients who were previously enrolled in the Phase I trial.
The company noted that eligible patients treated in the Phase I trial can enroll in the OLE trial, where they will receive ALN-AT3 for up to the earlier of two years or until the drug receives regulatory approval and becomes commercially available in their market.
The OLE trial is also designed to evaluate clinical activity of ALN-AT3, as measured by knockdown of serum AT, increases in thrombin generation, and reduction in the frequency of bleeding events.
Last January, Alnylam and Genzyme have formed a multi-product geographic alliance to accelerate and expand the development and commercialization of RNAi therapeutics worldwide in the field of rare diseases.
Antithrombin is a liver expressed plasma protein and member of the serpin family of proteins that acts as a major endogenous anticoagulant by inactivating Factor Xa and thrombin.