Arrowhead Pharmaceuticals, Inc. ((ARWR)) announced an update on their ongoing clinical study.
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Arrowhead Pharmaceuticals, Inc. is conducting a Phase 1 clinical study titled A Phase 1 Placebo-Controlled Dose Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-ATXN2 in Adult Subjects With Spinocerebellar Ataxia Type 2. The study aims to assess the safety and effectiveness of ARO-ATXN2 in adults with Spinocerebellar Ataxia Type 2 (SCA2), a genetic disorder characterized by progressive problems with movement.
The intervention being tested is ARO-ATXN2, an experimental drug administered via a single intrathecal injection. The purpose of this drug is to target the genetic mutations causing SCA2 and evaluate its safety and pharmacological effects.
This interventional study follows a randomized, parallel assignment model with quadruple masking, meaning neither the participants, care providers, investigators, nor outcomes assessors know who receives the treatment or placebo. The primary goal is treatment-focused.
The study began on November 1, 2024, with the latest update submitted on September 15, 2025. These dates are crucial as they mark the study’s progression and provide investors with a timeline for potential results and further updates.
The progress of this study could significantly impact Arrowhead Pharmaceuticals’ stock performance, as positive results may enhance investor confidence and market position. The competitive landscape in genetic disorder treatments is evolving, and successful outcomes could position Arrowhead as a leader in this niche market.
The study is currently recruiting, and further details are available on the ClinicalTrials portal.