bemdaneprocel (BRT-DA01) / Bayer, BlueRock Therapeutics - LARVOL DELTA

Continued Evaluation of Clinical Outcomes in Participants With Parkinson’s Disease Through 4 Years After Bemdaneprocel Administration (MDS Congress 2026) - No abstract available

Clinical • Clinical data • CNS Disorders • Movement Disorders • Parkinson's Disease

Continued evaluation of participants with Parkinson’s disease 3 Years after bemdaneprocel administration, and design of a phase 3 trial (WPC 2026) - P, P1, P3 | "The favorable safety profile overall and maintained motor improvements observed in the high-dose cohort through 3 years in exPDite support the continued development of bemdaneprocel; however, further studies are needed. exPDite-2 is the first large-scale, potentially registrational trial of stem cell–derived midbrain dopaminergic neuron progenitors for PD."

P3 data • CNS Disorders • Movement Disorders • Parkinson's Disease

Movement Disorders Section Poster Tour (AAN 2026) - "Katie Kompoliti. If you are interested in attending this poster tour, visit the poster hall and look for the tour guide holding the yellow Poster Tour sign. Featured abstracts:Leveraging Markerless Video-based Gait Analysis to Diagnose iNPH.Sleep–Autonomic Interactions in Parkinson’s Disease and Multiple System Atrophy: A Comparative Clinical Study with Structural Equation ModelingDigital Biomarkers of Disease Severity in Progressive Supranuclear PalsySleep Quality in Lewy Body Dementia Family Caregivers: Associations with Caregiver and Care Recipient CharacteristicsDifferentiating White-matter Damage Patterns by Underlying Pathology in Corticobasal SyndromeEffects of Bright Light Therapy on Daytime Sleepiness and Depression in Parkinson’s Disease: A Meta-analysis of Randomized Controlled TrialsexPDite-2: A Phase Three, Randomized, Sham-controlled, Double-blind Trial to Evaluate Bemdaneprocel, a Midbrain Dopaminergic Neuron Progenitor Cell Therapy for Parkinson’s..."

Alzheimer's Disease • CNS Disorders • Dementia • Depression • Lewy Body Disease • Movement Disorders • Multiple System Atrophy • Parkinson's Disease • Progressive Supranuclear Palsy

Continued Evaluation of Safety, Tolerability, and Clinical Outcomes in Participants with Parkinson’s Disease Throughout Three Years After Bemdaneprocel Administration (AAN 2026) - P, P1 | "Overall, outcomes in the low-dose cohort were stable. Conclusions These results support ongoing development of bemdaneprocel for the potential treatment of PD."

Clinical • Clinical data • Cerebral Hemorrhage • CNS Disorders • Hematological Disorders • Movement Disorders • Parkinson's Disease

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 (AAN 2026) - P1, P3 | "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."

Clinical • CNS Disorders • Movement Disorders • Parkinson's Disease

AAN 2026: Parkinson’s cell therapy trial shows sustained benefits (Parkinson's News Today) - "The study met its main goal of demonstrating good safety and tolerability after a year. Data up to two years showed that the therapy tended to ease or stabilize motor symptoms and shorten off episodes...An ongoing Phase 3 trial called exPDite-2 (NCT06944522) is testing the safety and efficacy of that dose in a larger group of people with Parkinson’s. The study, which started dosing last year, is enrolling about 102 participants in the U.S., Canada, and Australia...After the two-year exPDite trial, all participants enrolled in an ongoing extension study (NCT05897957), where safety and clinical outcomes are being monitored for up to five years post-transplant."

P1 data • Trial status • Parkinson's Disease

CONTINUED EVALUATION OF SAFETY, TOLERABILITY, AND CLINICAL OUTCOMES IN PARTICIPANTS WITH PARKINSON'S DISEASE THROUGH 3 YEARS AFTER BEMDANEPROCEL ADMINISTRATION (ADPD 2026) - P, P1 | "These results support ongoing development of bemdaneprocel for the treatment of PD."

Clinical • Clinical data • CNS Disorders • Movement Disorders • Parkinson's Disease

BlueRock Therapeutics investigative cell therapy bemdaneprocel for treating Parkinson’s disease receives Pioneering Regenerative Medical Product designation in Japan (Bayer Press Release)

Japanese regulatory • Parkinson's Disease

Continued Evaluation of Bemdaneprocel Following a Phase 1 Study for Parkinson's Disease: Outcomes Through 3 Years (MDS Congress 2025) - P, P1 | "Bemdaneprocel demonstrated a favorable safety profile and trended toward improvement or stability across clinical assessments through 2 years post transplantation. Results to date support the ongoing development of bemdaneprocel for the treatment of PD. Table 1 Table 2"

P1 data • Cerebral Hemorrhage • CNS Disorders • Epilepsy • Movement Disorders • Parkinson's Disease

BlueRock Therapeutics reports positive 36-month results from Phase I trial of bemdaneprocel for treating Parkinson’s disease (Bayer Press Release) - "The data were presented on October 6th at the International Congress of Parkinson’s Disease and Movements Disorders...Bemdaneprocel’s safety profile at 36 months is consistent with earlier findings, demonstrating that it continues to be well tolerated by patients, with no adverse events reported related to the therapy or surgical procedure....In the high dose cohort, the 36-month measurement of the effects of bemdaneprocel on activities of daily living using the MDS-UPDRS Part II measured a mean reduction of -4.3 points compared with baseline. The low dose cohort showed a mean increase of 0.2 points. Participants in both cohorts continue in the long term Continued Evaluation Study."

P1 data • Parkinson's Disease

Bayer first company to advance cell therapy as well as gene therapy against Parkinson’s disease (Bayer Press Release) - "A first participant received randomized treatment in the pivotal Phase III clinical trial, exPDite-2, of bemdaneprocel, an investigational cell therapy for PD...'Bemdaneprocel aims to replace these lost cells with new dopamine producing neurons and we are excited that the pivotal Phase III exPDite-2 clinical trial to explore this approach is now underway.'"

Trial status • Parkinson's Disease

exPDite-2: A Study to Investigate the Efficacy and Safety of Bemdaneprocel in Adults Who Have Parkinson's Disease (clinicaltrials.gov) - P3 | N=102 | Recruiting | Sponsor: BlueRock Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Movement Disorders • Parkinson's Disease

Phase I trial of hES cell-derived dopaminergic neurons for Parkinson’s disease. (PubMed, Nature) - P1 | "There were no graft-induced dyskinesias. These data demonstrate safety and support future definitive clinical studies."

Journal • P1 data • CNS Disorders • Movement Disorders • Parkinson's Disease • Transplantation

exPDite-2: A Study to Investigate the Efficacy and Safety of Bemdaneprocel in Adults Who Have Parkinson's Disease (clinicaltrials.gov) - P3 | N=102 | Not yet recruiting | Sponsor: BlueRock Therapeutics

New P3 trial • CNS Disorders • Movement Disorders • Parkinson's Disease

BlueRock Therapeutics announces publication in Nature of 18-month data from Phase 1 clinical trial for bemdaneprocel, an investigational cell therapy for Parkinson’s disease (GlobeNewswire) - "BlueRock Therapeutics LP...announced the publication of the 18-month data from its Phase 1 exPDite clinical trial for bemdaneprocel in the journal Nature....The publication, which is available online, confirms that 18 months following surgery, treatment with bemdaneprocel did not cause any serious adverse events related to cell therapy....'Our efforts are now focused on maintaining our forward momentum to initiate and enroll the Phase 3 trial as we advance bemdaneprocel’s development to this exciting next stage'....The Phase 3 trial, called exPDite-2, is expected to be initiated in the first half of 2025."

New P3 trial • P1 data • Parkinson's Disease

Phase I trial of hES cell-derived dopaminergic neurons for Parkinson’s disease (Nature) - P1 | N=12 | NCT04802733 | Sponsor: BlueRock Therapeutics | "Twelve patients were enrolled sequentially in two cohorts-a low-dose (0.9 million cells, n = 5) and a high-dose (2.7 million cells, n = 7) cohort-and all of the participants received one year of immunosuppression. The trial achieved its primary objectives of safety and tolerability one year after transplantation, with no adverse events related to the cell product. At 18 months after grafting, putaminal 18Fluoro-DOPA positron emission tomography uptake increased, indicating graft survival....There were no graft-induced dyskinesias."

P1 data • Parkinson's Disease

Safety, Tolerability, and Clinical Assessment of Bemdaneprocel for Parkinson's Disease: Results Through 24 Months From a Phase 1 Study (S37.005). (PubMed, Neurology) - P, P1 | "An immediate family member of Viviane Tabar has received intellectual property interests from a discovery or technology relating to health care. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff."

Journal • P1 data • Anesthesia • CNS Disorders • Epilepsy • Gastrointestinal Disorder • Hematological Disorders • Infectious Disease • Movement Disorders • Novel Coronavirus Disease • Parkinson's Disease • Transplantation

Safety, Tolerability, and Clinical Assessment of Bemdaneprocel for Parkinson's Disease: Results Through 24 Months From a Phase 1 Study (AAN 2025) - P, P1 | "Bemdaneprocel demonstrated a favorable safety profile and trended toward clinical benefit or stability through 24 months post transplantation. All participants have enrolled in a continued-evaluation study (NCT05897957). These results support the ongoing development of bemdaneprocel in a Phase 3 trial to evaluate efficacy and safety in people with PD."

Clinical • P1 data • Anesthesia • CNS Disorders • Epilepsy • Gastrointestinal Disorder • Hematological Disorders • Infectious Disease • Movement Disorders • Novel Coronavirus Disease • Parkinson's Disease

BlueRock Therapeutics advances investigational cell therapy bemdaneprocel for treating Parkinson’s disease to registrational Phase III clinical trial (Bayer Press Release) - "Bayer AG and BlueRock Therapeutics LP, a clinical-stage cell therapy company and wholly owned subsidiary of Bayer AG,...announced plans to initiate a Phase III clinical trial for bemdaneprocel, its investigational cell therapy for Parkinson’s disease. The registrational trial, named exPDite-2, is expected to begin in the first half of 2025 and will represent a significant milestone in the development of allogeneic cell-based therapies for neurodegenerative disorders....Depending upon the outcome, the results from this trial are intended to be part of a robust data package to support regulatory submissions for marketing authorization."

New P3 trial • Parkinson's Disease

Phase 1 Safety and Tolerability Study of MSK-DA01 Cell Therapy for Advanced Parkinson's Disease (clinicaltrials.gov) - P1 | N=12 | Completed | Sponsor: BlueRock Therapeutics | Active, not recruiting ➔ Completed

Trial completion • CNS Disorders • Movement Disorders • Parkinson's Disease

BlueRock Therapeutics’ Investigational Cell Therapy Bemdaneprocel for Parkinson’s Disease Shows Positive Data at 24-Months (Businesswire) - P1 | N=12 | exPDite (NCT04802733)| Sponsor: BlueRock Therapeutics | "The safety profile at 24 months is consistent with earlier findings, demonstrating that bemdaneprocel continues to be well-tolerated by patients, with no adverse events reported related to bemdaneprocel. Transplanted cells continue to survive and engraft in the brain after discontinuing immunosuppression therapy at 12 months as outlined in the study’s protocol...In the high dose cohort, the 24-month measurement of the effects of bemdaneprocel on motor symptoms using MDS-UPDRS Part III measured in the “OFF”-medication state, showed a mean reduction of 21.9 points compared with baseline. The low dose cohort showed a mean decrease of 8.3 points...In the high dose cohort, the 24-month measurement of the effects of bemdaneprocel on activities of daily living using MDS-UPDRS Part II measured a mean reduction of 3.4 points compared with baseline. The low dose cohort showed a mean increase of 2.0 points."

P1 data • CNS Disorders • Parkinson's Disease

Motor and Non-Motor Outcomes of Bemdaneprocel in People With Parkinson's Disease: Results up to 24 Months From a Phase 1 Study (MDS Congress 2024) - P, P1 | "Overall, trends toward benefit in motor symptoms and stability in non-motor symptoms were reported for participants who received bemdaneprocel. These effects persisted for 6 months post discontinuation of immunosuppression. All participants are expected to be followed in a long-term extension study (NCT05897957)."

P1 data • CNS Disorders • Parkinson's Disease

Safety and Tolerability of Bemdaneprocel in People With Parkinson's Disease: Results up to 24 Months From a Phase 1 Study (MDS Congress 2024) - P, P1 | "Bemdaneprocel was generally safe and well tolerated 18 months post transplantation. All participants are expected to be followed in a long-term extension study (NCT05897957) to monitor safety and durability of effect. These results support the continued development and evaluation of bemdaneprocel for the treatment of people with PD."

Clinical • P1 data • CNS Disorders • Epilepsy • Parkinson's Disease

BlueRock Therapeutics receives FDA Regenerative Medicine Advanced Therapy designation for Parkinson’s disease cell therapy candidate bemdaneprocel (Bayer Press Release) - "Bayer AG and BlueRock Therapeutics LP...announced today that BlueRock’s investigational cell therapy bemdaneprocel for the treatment of Parkinson’s disease has been granted Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA)....Regenerative Medicine Advanced Therapy (RMAT) designation follows phase I clinical trial results demonstrating that bemdaneprocel is well tolerated with no major safety issues through 18 months / Bemdaneprocel is the most clinically advanced investigational cell therapy in the U.S. for treating patients living with Parkinson’s disease."

FDA event • Parkinson's Disease

Cell-Based Therapies and Bembaneprocel (NeurologyLive) - "Daniel Kremens, MD, JD, described positive 18-month data on bemdaneprocel, an investigational cell-based approach for patients with Parkinson disease."

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