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Alnylam Pharmaceuticals ALNY presented new data from its early-stage study of nucresiran (formerly ALN-TTRsc04) for treating transthyretin (ATTR) amyloidosis at a recent medical conference in Chicago. Nucresiran is ALNY’s next-generation RNAi therapeutic, which is being developed leveraging the company’s proprietary IKARIA platform.
The ongoing phase I study evaluates the safety, tolerability, pharmacokinetic (PK) and pharmacodynamic effects of nucresiran in 48 healthy adult subjects, who have been randomized in a ratio of 3:1 to receive either a single ascending dose of nucresiran or placebo. The study's primary endpoint is safety and secondary endpoints include the change from baseline in serum TTR over time, as well as characterization of plasma and urine PK of nucresiran.
ALNY’s Amyloidosis Drug Shows Sustained 6-Month Efficacy
Per the data from an interim analysis of the phase I ATTR amyloidosis study, it was observed that a single 300 mg or higher dose of nucresiran quickly reduced serum TTR levels with low inter-patient variability, by more than 90% within 15 days, sustained through at least six months. At these dosage strengths, the reduction of mean TTR levels peaked at more than 96% by Day 29. Furthermore, serum TTR levels remained significantly reduced, with a mean decrease of more than 70% at the one-year mark after a single 300 mg dose. Alnylam reported that the one-year data for the 600 and 900 mg doses are not yet available.
Year to date, shares of ALNY have gained 23.1% against the industry’s 10.5% decline.
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Management is optimistic about nucresiran's ability to minimize interpatient variability in TTR reduction. Its demonstrated durability suggests the potential for biannual or annual dosing, offering a new approach to treating ATTR amyloidosis. Alnylam expects to share late-stage development plans in the first quarter of 2025.
We remind the investors that Alnylam had earlier reported nucresiran 300 mg six-month data from the phase I ATTR amyloidosis study at its R&D day last year held in December.
Alnylam also reported that all tested doses of nucresiran have been well tolerated in the early-stage ATTR amyloidosis study. Adverse events were mostly mild in severity and none were related to treatment with different doses of the candidate.
ALNY’s Marketed Amyloidosis Drugs
Alnylam currently markets Amvuttra, the company’s lead drug, in the United States for the treatment of adult patients with polyneuropathy of hereditary ATTR (hATTR) amyloidosis. Amvuttra is also available in the EU for treating hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy. Alnylam also markets Onpattro, which is approved in the United States and EU to treat hATTR amyloidosis in adults.