Ferrer and Prilenia have initiated the confirmatory PRECISE‑HD study, a global phase 3 trial designed to evaluate the efficacy and safety of pridopidine in people living with Huntington’s disease. Recruitment has begun in the US, with sites across the EU, UK and Canada expected to open later in the year.
The 400‑participant randomized, double‑blind, placebo‑controlled study will assess pridopidine’s effect on disease progression, functional capacity, motor function, cognition, speech and quality of life.
The oral investigational drug is taken twice daily. The trial incorporates learnings from previous research and input from patient organisations, the HD‑Community Advisory Board and regulatory authorities.
Participants will include people with early to mid‑stage Huntington’s disease, defined by a Total Functional Capacity score of 7‑13, a Total Motor Score ≥20 and an Independence Scale score ≤90%. The design aims to ensure appropriate assessment of any treatment effect compared with placebo. Up to 75 sites will take part globally.
Astri Arnesen, President of the European Huntington Association, said: “The initiation of PRECISE‑HD is an important and needed step to have the results from previous studies with pridopidine confirmed.
“This study is designed to with the group of participants that has shown positive effect previously. EHA appreciate the involvement of the community in the preparations by consulting the patient organisations as well as HD‑Community Advisory Board. We experienced that Ferrer has taken the feedback very seriously when planning for the trial setup and follow‑up. I hope the study will be well received by the community and that the recruitment goes as smoothly and fast as possible. This is different approach to the other trials ongoing and we need them all.”
Victor Sung, Director of the Division of Movement Disorders at the University of Alabama and PRECISE‑HD Steering Committee Member, said: “Across prior studies, pridopidine has shown meaningful clinical effects in specific circumstances, and this study has been carefully designed to confirm those effects, and uniquely incorporates prior learnings and novel elements specifically designed to provide treatment effect clarity.”
Oscar Pérez, Chief Scientific Officer at Ferrer, said: “The initiation of PRECISE‑HD reflects the continued progress of our work in rare neurological diseases and our commitment to advancing rigorous clinical research where the unmet need remains significant.”
PRECISE‑HD includes a 52‑week placebo‑controlled stage followed by a 104‑week open‑label extension, allowing assessment of potential long‑term effects compared with matched external control cohorts. The primary endpoint is change from baseline to Week 52 in the combined Unified Huntington’s Disease Rating Scale score.
