Alnylam Pharmaceuticals has revealed new interim data from its Phase I trial of nucresiran, a ribonucleic acid interference (RNAi) therapeutic for transthyretin (ATTR) amyloidosis treatment.
The study showed significant reductions in serum TTR levels in subjects after a single dose, with sustained effects over time.
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It evaluated nucresiran’s safety, pharmacokinetics and pharmacodynamics in healthy individuals. The outcomes showed that a single 300mg dose led to a mean serum TTR reduction of over 90% at day 15, which was maintained through day 180.
At day 360, the reduction remained substantial at over 70%. Data for the higher doses of 600mg and 900mg cohorts are pending.
The trial also reported low inter-patient variability in TTR reduction, with the 300mg cohort experiencing a 96% to 96.7% reduction by day 29.
The 600mg cohort saw reductions between 96.6% and 98.6%, and the 900mg cohort between 96% and 97.3%.
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By GlobalDataThe study also highlighted nucresiran’s tolerability, with most adverse events classified as mild and unrelated to the treatment.
Nucresiran is part of the company’s IKARIA platform, designed to provide deeper and more durable TTR knockdown, potentially allowing for less frequent dosing.
However, the safety and efficacy of nucresiran have not been established by health authorities such as the Food and Drug Administration (FDA) or the European Medicines Agency (EMA).
The company anticipates its Phase III development plans for nucresiran in the first quarter (Q1) of 2025.
Alnylam Pharmaceuticals chief medical officer Pushkal Garg said: “We are very excited by these new Phase I data with nucresiran, our next-generation TTR-targeting RNAi therapeutic, which demonstrated that a single dose of ≥300mg achieved rapid knockdown of TTR greater than 90% from say 15 that was sustained to at least six months.
“Furthermore, we are encouraged by the potential of nucresiran to reduce interpatient variability in TTR lowering. Nucresiran utilises our IKARIA platform, which has now demonstrated the potential to achieve durability supportive of biannual or annual dosing, representing a potential new paradigm in the treatment of ATTR amyloidosis.”
ATTR amyloidosis is a fatal condition characterised by the accumulation of misfolded TTR proteins as amyloid deposits, affecting multiple body parts.
