RNAi Therapeutics Delivery Advancements
Pushkal Garg, Chief Medical Officer, highlighted Alnylam's excitement about the emerging profile of RNAi therapeutics, which enables sustained knockdown of disease-causing proteins with infrequent administration. The company is exploring new tissues of interest, such as adipose and muscle, with updates expected at an R&D Day early next year.
ALN-HTT02 Phase 1 Study Insights
ALN-HTT02, Alnylam's third CNS program, has shown promising preclinical results in nonhuman primates. Targeting an upstream portion of the Huntington gene, it offers potential unique efficacy. The Phase 1 study, active in the UK, Canada, and the US, will evaluate safety, tolerability, and changes in mutant Huntington levels.
Polyneuropathy Market Dynamics
Despite new competition, Alnylam's products have demonstrated robust growth, attributed to quarterly administration, patient retention, and rapid knockdown capabilities. Tolga Tanguler, Chief Commercial Officer, emphasized the company's clear guidelines to ensure compliance and strong demand in the polyneuropathy market.
Payer Discussions for AMVUTTRA
Tolga Tanguler discussed payer negotiations focusing on the value proposition driven by clinical outcomes. The HELIOS-B results are compelling, with Alnylam's patient access philosophy including value-based agreements and appropriate rebates. More details will be shared closer to the PDUFA date.
SG&A Spending for Upcoming Launches
Jeff Poulton, Chief Financial Officer, indicated that SG&A spending will experience double-digit growth as Alnylam prepares for the US and EU launches of vutrisiran in ATTR-CM. The company has made significant progress in hiring and launch preparations, with further guidance to be provided in February.
