Alnylam Pharmaceuticals ((ALNY)) announced an update on their ongoing clinical study.
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Study Overview: Alnylam Pharmaceuticals is conducting a Phase 2 trial titled ‘A Phase 2, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Intrathecally Administered ALN-APP in Patients With Cerebral Amyloid Angiopathy (CAA)’. The study aims to assess the impact of ALN-APP on CAA progression and evaluate its safety and pharmacodynamics in patients with sporadic and Dutch-type CAA.
Intervention/Treatment: The study tests ALN-APP, an experimental drug administered intrathecally, designed to slow CAA progression. A placebo is also used for comparison.
Study Design: This interventional study uses a randomized, parallel assignment model with quadruple masking, meaning participants, care providers, investigators, and outcomes assessors are unaware of the treatment allocations. The primary goal is treatment-focused.
Study Timeline: The study began on May 17, 2024, with primary completion expected within 24 months, followed by an optional 18-month open-label extension. The last update was submitted on July 15, 2025, indicating ongoing recruitment.
Market Implications: This study could significantly impact Alnylam Pharmaceuticals’ stock performance, as successful results may enhance investor confidence and position the company as a leader in CAA treatment. Competitors in the neurological disorder treatment space will be closely monitoring these developments.
The study is ongoing, with further details available on the ClinicalTrials portal.