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news.Alnylam Pharmaceuticals has started an open-label extension Phase III trial (abbreviated APOLLO-OLE) with patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) to treat TTR-mediated amyloidosis (ATTR amyloidosis) in patients with Familial Amyloidotic Polyneuropthy (FAP).
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The company noted that all patients who complete the APOLLO Phase III trial with patisiran are eligible for enrollmnet in APOLLO-OLE.
The trial is designed to assess the long-term safety and efficacy of patisiran as well as measure effects of treatment toward a number of clinical endpoints, including the modified Neuropathy Impairment Score, or mNIS+7.
mNIS is an evaluation of muscle weakness, sensory and autonomic function, and nerve conductance.
Additionally, the company is assuming positive study results in the APOLLO Phase III trial and expects to file a new drug application (NDA) for patisiran in 2017.
Alnylam R&D executive vice-president and chief medical officer Dr Akshay Vaishnaw said: "Patisiran is the leading investigational candidate in our pipeline of RNAi therapeutics.
"We are very encouraged with the clinical activity and tolerability seen to date with patisiran, including in our Phase 2 OLE study where we have recently reported what we believe to be continued evidence for a possible halting of neuropathy progression after the first 12 months of treatment.
"The APOLLO trial continues enrolling patients with FAP, and, assuming positive results, we expect to be in a position to file an NDA in the 2017 timeframe."
The initiation of the APOLLO Phase III trial is a major step toward its ‘Alnylam 2020’ goals.
By the end of 2020, the company expects to have three marketed products, as well as ten programs in clinical development, including four in late stages of development, across its three Strategic Therapeutic Areas (STArs).
The open-label, multi-center APOLLO-OLE will evaluate the long-term safety and tolerability of patisiran in ATTR amyloidosis patients with FAP who were previously enrolled in the APOLLO Phase III trial.
In the Phase III APOLLO-OLE trial patients will be given patisiran at a dose of 0.3 mg/kg every three weeks for up to the earlier of two years or until the drug is commercially available in their market.
Image: Patients who complete the APOLLO Phase III trial will be eligible for enrollment in APOLLO-OLE trial with patisiran. Photo: Photo: courtesy of Baitong333/ FreeDigitalPhotos.net.