Alnylam Pharmaceuticals ((ALNY)) announced an update on their ongoing clinical study.
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Alnylam Pharmaceuticals is conducting a Phase 3 study titled ‘HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)’. The study aims to evaluate the efficacy and safety of vutrisiran in treating hATTR amyloidosis, a rare genetic disorder. This research is significant as it could offer a new treatment option for patients with this debilitating condition.
The study tests two interventions: Vutrisiran, administered via subcutaneous injection, and Patisiran, given intravenously. Vutrisiran is intended to reduce the production of transthyretin protein, which is implicated in the disease.
The study is designed as a randomized, open-label trial with a parallel intervention model and no masking. Its primary purpose is treatment-focused, comparing the effects of Vutrisiran with an external placebo group from a previous study.
The study began on February 14, 2019, with primary completion achieved in July 2022. The estimated study completion date is July 2025, with the latest update submitted in July 2025. These dates are crucial for tracking the study’s progress and anticipating results.
This update could positively impact Alnylam’s stock performance and investor sentiment, as successful outcomes may lead to a new marketable treatment. Competitors in the genetic disorder treatment space will be closely watching these developments.
The study is ongoing, with further details available on the ClinicalTrials portal.