Abstract Details

Title exPDite-2: A Phase Three, Randomized, Sham-controlled, Double-blind Trial to Evaluate Bemdaneprocel, a Midbrain Dopaminergic Neuron Progenitor Cell Therapy for Parkinson’s Disease

Topic Movement Disorders

Presentation(s) P8 - Poster Session 8 (11:45 AM-12:45 PM)

Poster/Presentation
Number 17-008

Objective

To evaluate the efficacy of bemdaneprocel on motor symptoms in participants with Parkinson’s disease (PD) compared with participants who undergo sham surgery in exPDite-2—a Phase 3, multicenter, randomized, sham surgery–controlled, double-blind trial (NCT06944522).

Background Bemdaneprocel, an investigational therapy under development for PD, comprises midbrain dopaminergic neuron progenitors derived from embryonic stem cells. exPDite (NCT04802733), a Phase 1, open-label, 24-month, multicenter study demonstrated bemdaneprocel was generally safe and well tolerated with potential clinical benefit through 24 months post transplantation. The exPDite-2 trial will evaluate the efficacy and safety of bemdaneprocel.

Design/Methods Eligible participants with clinically established PD, ≥45 to ≤75 years of age, ≥4 and <12 years from PD diagnosis with ≥2.5 hours daily OFF time will be randomized to bemdaneprocel or sham surgery in a single surgical session. Bemdaneprocel is administered bilaterally to the postcommissural putamen (3 million cells/putamen); sham surgery consists of bilateral partial burr holes. The procedure will be followed by 12 months of active (bemdaneprocel group) or placebo (sham group) immunosuppression. Sham group participants may be eligible to receive bemdaneprocel during the open-label period based on the primary analysis at Week 78.

Results The primary endpoint is change from baseline at Week 78 in adjusted PD diary ON time without troublesome dyskinesia. Secondary and exploratory endpoints include motor, non-motor, activities of daily living, quality of life, and safety outcomes. All participants receiving bemdaneprocel will be followed for 5 years post transplantation. Approximately 102 participants will be randomized in exPDite-2. The first participant was randomized and underwent surgery in August 2025.

Conclusions exPDite-2 is a Phase 3, randomized, sham surgery–controlled, double-blind trial that will evaluate the efficacy and safety of bemdaneprocel. exPDite-2 will be the first large-scale, potentially registrational trial of stem cell–derived midbrain dopaminergic neuron progenitors in people with PD.

Authors/Disclosures
Nauman Abid, MD (BlueRock Therapeutics) PRESENTER	Dr. Abid has received personal compensation for serving as an employee of BlueRock Therapeutics.
Mathews Adera, MD, MBBS (Ovid Therapeutics)	Dr. Adera has received personal compensation for serving as an employee of Ovid Therapeutics. Dr. Adera has received stock or an ownership interest from Ovid Therapeutics.
Melinda Louie-Gao, PhD	Dr. Louie-Gao has received personal compensation for serving as an employee of Bluerock Therapeutics. Dr. Louie-Gao has received personal compensation for serving as an employee of Wave Life Science.
Gabriel M. Belfort, MD (BlueRock Therapeutics)	Dr. Belfort has received personal compensation for serving as an employee of Praxis Precision Medicines. Dr. Belfort has received stock or an ownership interest from Praxis Precision Medicines. Dr. Belfort has received intellectual property interests from a discovery or technology relating to health care.

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