ALNY - Alnylam halts study for rare disease therapy citing U.S. drug pricing reforms

RNAi therapeutics company Alnylam Pharmaceuticals ( NASDAQ: ALNY ) announced Thursday it dropped plans to begin a late study to find a potential therapy for the rare eye disorder Stargardt Disease as it evaluates the impact of the recently enacted Inflation Reduction Act in the U.S.

The company previously announced that the Phase 3 trial, designed to evaluate RNAi therapeutic vutrisiran in Stargardt Disease, would begin in late 2022.

With the passage of the Inflation Reduction Act, Alnylam ( ALNY ) is now weighing options to decide the best path forward for advancing an RNAi therapeutic for the genetic disorder, the company said.

The historic legislation passed in August allows Health and Human Services Secretary to negotiate prices for Part B and Part D Medicare drugs covered under Medicare, among other measures.

Turning to Q3 2022 financials, Alnylam ( ALNY ) reported $232.3M of net product revenue for the quarter with ~39% YoY growth thanks to hATTR amyloidosis therapies ONPATTRO and AMVUTTRA, which added $145M and $25M, respectively.

Meanwhile, net loss for the period climbed ~99% YoY to $405.9M as SG&A expenses and R&D expenses rose ~66% YoY and ~26% YoY, respectively. The company reaffirmed the previously issued guidance for net product revenue at $870M – $930M.

Alnylam ( ALNY ) also reiterated plans to submit a marketing application for a label expansion of Onpattro by the year-end targeting ATTR amyloidosis with cardiomyopathy for which a topline readout from a Phase 3 trial sent ALNY shares sharply higher in August.

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