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Alnylam’s RNAi Drug Shows Promise in Halting Neuropathy Progression in Patients with a Rare Genetic Disorder

Alnylam Pharmaceuticals’ lead RNAi therapy demonstrated positive results in a mid-stage study in patients with a rare genetic disorder.

The company announced initial 12-month clinical data from its ongoing Phase II OLE study evaluating its patisiran for treatment of transthyretin (TTR)-mediated amyloidosis (ATTR amyloidosis) in patients with familial amyloidotic polyneuropathy (FAP), a rare genetic defect that can lead to life-threatening protein buildups that damage nerve tissue.

In the open-label study, patisiran demonstrated a mean 2.5 point decrease in modified neuropathy impairment score at 12 months. According to the company, this decrease compares favorably with the 13 to 18 point increase in impairment in historical data of untreated FAP patients with similar baseline characteristics. Additionally, the drug achieved a sustained mean serum TTR knockdown at the 80 percent target level for 16 months, with an up to 88 percent mean knockdown achieved between doses. Alnylam said that these results are consistent with the therapeutic hypothesis that TTR knockdown has the potential to halt neuropathy progression in patients with FAP. The drug was found to be generally well tolerated out to 17 months of drug administration. The company plans to report 18-month data from the open-label extension later this year.

The results sent the company’s shares up about ten percent. Last year, Alnylam revealed 6-month results from the same study, in which patisiran demonstrated a mean 1 point reduction in neuropathy impairment.

“I view these new clinical activity and safety data from Alnylam’s ongoing patisiran OLE study as very encouraging. In particular, the possibility of halting neuropathic progression over 12 months of treatment is promising in light of the rapid increase in neuropathy impairment scores observed in analysis of other historical data sets. If these results are replicated in a randomized, double-blind, placebo-controlled study, I believe that patisiran could emerge as an important treatment option for patients suffering from this debilitating, progressive and life-threatening disease,” said David Adams, MD, PhD, Head of Department of Neurology and Coordinator of the French Reference Center for FAP (NNERF)/APHP/CHU Bicêtre/France. “I very much look forward to continuing to participate in the clinical advancement of this investigational RNAi therapeutic, including enrolling patients onto the APOLLO Phase 3 study, as there are currently few options for our patients suffering from FAP.”

The company is actively enrolling FAP patients worldwide in its late-stage study, known as APOLLO. Alnylam aims to obtain definitive evidence for patisiran efficacy and safety in FAP in the Phase III study.

Source: Alnylam Pharmaceuticals

Last updated: 4/21/15; 2:45pm EST

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