bemdaneprocel (BRT-DA01) / Bayer - LARVOL DELTA

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

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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Phase 1 Data Highlights Sustained Efficacy of Cell Therapy Bemdaneprocel in Parkinson Disease (NeurologyLive) - "'It’s exciting that bemdaneprocel met safety and tolerability criteria at 12 months, and now the 18-month results suggest that these allogeneic cells survive and have potentially positive effects even after discontinuation of immunosuppressants,' principal investigator Claire Henchcliffe, MD...said in a statement."

Media quote

BlueRock Therapeutics phase I clinical trial for Parkinson's disease continues to show positive trends at 18 months (Financial Post) - P1 | N=12 | NCT04802733 | Sponsor: BlueRock Therapeutics | "Bayer AG and BlueRock Therapeutics LP...announced today details of 18-month data from the phase I clinical trial for bemdaneprocel....The data were presented at the Alzheimer’s and Parkinson’s Diseases Conference....The data demonstrate that at 18 months bemdaneprocel continues to be well tolerated with no major safety issues, transplanted cells survive and engraft in the brain and F-DOPA signal continues to increase after stopping immune suppression therapy at 12 months as outlined in the study’s protocol. In addition, exploratory clinical endpoints improved compared to baseline assessments in both cohorts, with participants in the high dose cohort showing greater improvement than those in the low dose cohort....A phase II study for further clinical testing of bemdaneprocel is planned to begin enrolling patients later this year."

New P2 trial • P1 data • CNS Disorders • Parkinson's Disease

Can dopamine be restored in Parkinson's patients? (UCI Health) - "The hope is that infusing the brain with these specialized neural cells - called bemdaneprocel (BRT-DA01) and developed by BlueRock Therapeutics - will restore critical dopamine production and alleviate the progression of Parkinson's disease, says Henchcliffe..."

Media quote

Bayer Opens First Cell Therapy Manufacturing Facility to Advance Regenerative Medicines on a Global Scale (Bayer Press Release) - "Bayer AG announced today the opening of its first Cell Therapy Launch Facility in Berkeley, California to create the capacity to bring cell therapies to patients on a global scale. The $250 million (USD), 100,000-square-foot facility will supply the material required for late-stage clinical trials and potential commercial launch of BlueRock Therapeutics’ bemdaneprocel (BRT-DA01), an investigational cell therapy currently in evaluation for treating Parkinson’s disease. In addition, it includes space for a second module of production suites to support additional cell therapies as Bayer’s portfolio advances."

Commercial • CNS Disorders • Parkinson's Disease

Bayer invests 250 million USD in new cell therapy manufacturing facility in the U.S. (Bayer Press Release) - "Bayer AG has opened a new cell therapy production plant in Berkeley, California, USA, to bring cell therapies to patients on a global scale. The USD 250 million investment will support the production of material required for clinical trials and potential commercial launch of BlueRock Therapeutics’ bemdaneprocel (BRT-DA01), an investigational cell therapy currently in evaluation for treating Parkinson’s Disease. BlueRock Therapeutics LP is a clinical stage cell therapy company and wholly owned, independently operated subsidiary of Bayer AG. The new facility will also support the future production of additional cell therapies as Bayer’s cell therapy portfolio advances."

Commercial • Parkinson's Disease

Dopaminergic neuronal cell therapy for Parkinsons Disease: Results from a Phase 1 study of Bemdaneprocel (CNS Mission 2023) - P=P1 | N=12 | "Results from at least 1 year of follow-up in this phase 1 study will be presented. An acceptable safety profile, evidence of sustained engraftment, and positive exploratory clinical outcomes in this study support further investigation of bemdaneprocel for the treatment of PD. Unpublished data, will be partly shown in a poster at MDS."

P1 data

BlueRock's Phase I study with bemdaneprocel in patients with Parkinson's disease meets primary endpoint (Canada Newswire) - P1 | N=12 | NCT04802733 | Sponsor: BlueRock Therapeutics | "Bayer AG and BlueRock Therapeutics LP...recently announced details of the positive data from the Phase I clinical trial for bemdaneprocel (BRT-DA01), a stem cell derived investigational therapy for treating Parkinson's disease. The data was presented at the International Congress of Parkinson's Disease and Movement Disorders in Copenhagen, Denmark....The study met the primary objective of demonstrating safety and tolerability in all 12 participants in the study's low and high dose cohorts, with no serious adverse events (SAEs) reported related to bemdaneprocel through one year....Secondary exploratory clinical endpoints improved in both cohorts, with participants in the high dose cohort showing greater improvement....Based on these results, planning is underway for a Phase II study that is expected to begin enrolling patients in H1 (first half) 2024."

New P2 trial • P1 data • CNS Disorders • Parkinson's Disease

Parkinson Cell Therapy Bemdaneprocel Heads to Phase 2 After Successful Phase 1 Results (NeurologyLive) - "'The data from this Phase I open label study are extremely encouraging,' principal investigator Claire Henchcliffe, MD...said in a statement.1 'While this is a small open label study, meeting the study’s primary objective for safety and tolerability along with initial improvements seen in clinical outcomes represents a great step forward. The hope now is that these trends continue and translate into meaningful benefit for people with Parkinson’s disease in controlled clinical trials.'"

Media quote

BlueRock’s Parkinson’s disease therapy first to show positive results in Phase I clinical study - "...Participants were followed at clinical sites by...Dr. Claire Henchcliffe, M.D., D.Phil., F.A.A.N., F.A.N.A...."

BlueRock’s neuronal stem cell therapy for Parkinson’s disease is first to show positive results in Phase I clinical study (Bayer Press Release) - P1 | N=12 | NCT04802733 | Sponsor: BlueRock Therapeutics | "Bayer AG and BlueRock Therapeutics LP...today announced positive top-line results from a Phase I clinical trial of investigational drug, bemdaneprocel (BRT-DA01), a potential first-in-class cell therapy for Parkinson's disease. The trial showed that bemdaneprocel was well-tolerated in all 12 patients in the study to date, with no major safety events. In addition, an assessment of the study’s secondary endpoints demonstrated feasibility of transplantation and evidence of cell survival and engraftment in the brain through one year. Based on these results, planning is underway for a Phase II study that is expected to begin enrolling patients in H1 (first half) 2024....Detailed Phase I trial data from primary and secondary endpoints will be presented at the 2023 International Congress of Parkinson’s Disease and Movement Disorders in Copenhagen from August 27 – 31."

New P2 trial • P1 data • CNS Disorders • Parkinson's Disease

Continued Evaluation of Patients With Parkinson's Disease Who Previously Received BRT-DA01 (clinicaltrials.gov) - P=N/A | N=12 | Enrolling by invitation | Sponsor: BlueRock Therapeutics

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

Bayer progressing well with transformation of pharma business and raises peak sales potential of key growth drivers to over twelve billion euros (Market Screener) - "BRT-DA01 has completed enrollment of the Phase I clinical study, expecting data readout in the second half of 2023."

Enrollment closed • P1 data • CNS Disorders • Parkinson's Disease

BlueRock Therapeutics Announces Completion of Enrollment of Phase 1 Trial in Patients with Parkinson’s Disease (PRNewswire) - "BlueRock Therapeutics LP...announced the completion of enrollment of its Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson's disease (PD). The purpose of the Ph1 clinical trial is to evaluate the safety, tolerability, and preliminary efficacy of BRT-DA01 in patients with Parkinson's disease....As a next step, BlueRock Therapeutics plans to initiate a global non-interventional study in the second half of 2022...In the United States, the trial was initiated...Dr. Alfonso Fasano, M.D., PhD..."

CNS Disorders • Parkinson's Disease