ALN-HTT / Regeneron, Alnylam - LARVOL DELTA

Huntington's disease clinical trials update: March 2025. (PubMed, J Huntingtons Dis) - "In this edition of the Huntington's Disease Clinical Trials Update, we expand on the ongoing phase I clinical trial of ALN-HTT02 from Alynlam Pharmaceuticals. We also report on the SAGE-718 (also known as dalzanemdor) program from Sage Therapeutics, with results of the phase II DIMENSION study and the recent termination of the open-label phase III PURVIEW study. Additionally, we discuss recent developments in the regulatory pathway for AMT-130, following discussions between uniQure and the U.S. Food and Drug Administration regarding key aspects of accelerated approval. Finally, we provide a comprehensive listing of all currently registered and ongoing clinical trials in Huntington's disease."

Journal • Huntington's Disease • Movement Disorders

A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease (clinicaltrials.gov) - P1 | N=54 | Recruiting | Sponsor: Alnylam Pharmaceuticals | Not yet recruiting ➔ Recruiting | Trial primary completion date: Dec 2027 ➔ Jul 2028

Enrollment open • Trial primary completion date • Huntington's Disease • Movement Disorders

ALN-HTT02, a novel C16-siRNA conjugate for HTT-lowering in the CNS (HSG 2024) - "Utilizing our C16-siRNA delivery platform, ALN-HTT02 is designed to potently and durably reduce HTT protein expression in neurons, and thereby has the potential to alter the course of HD progression. Here, we highlight the unique properties of the platform, share learnings regarding the tolerability of HTT-lowering in non-human primates, and outline an upcoming first-in-human Phase 1b study that will aim to characterize safety and tolerability of ALN-HTT02 in patients with HD."

Huntington's Disease • Movement Disorders

A Study to Evaluate ALN-HTT02 in Adult Patients With Huntington's Disease (clinicaltrials.gov) - P1 | N=54 | Not yet recruiting | Sponsor: Alnylam Pharmaceuticals

New P1 trial • Huntington's Disease • Movement Disorders

Alnylam Pharmaceuticals reports fourth quarter and full year 2019 financial results and highlights recent period activity (Businesswire) - “Alnylam Pharmaceuticals...today reported its consolidated financial results for the fourth quarter and full year ended December 31, 2019 and reviewed recent business highlights…Reported strong progress in CNS and ocular delivery of RNAi therapeutics with seven initial programs selected as part of Regeneron collaboration…ALN-HTT, in development for the treatment of Huntington’s disease.”

Clinical