BlueRock Therapeutics LP, a clinical-stage cell therapy company and wholly owned subsidiary of Bayer AG, announced the publication of the 18-month data from its Phase 1 exPDite clinical trial for bemdaneprocel in the journal Nature.
“The concept of “rebuilding” brain networks that have been lost to disease is compelling,” said Claire Henchcliffe, MD, chair of the UC Irvine School of Medicine’s Department of Neurology at the University of California, Irvine and one of the study’s Principal Investigators, “The results of this early phase clinical trial demonstrate the promise of regenerative medicine and should provide hope for Parkinson’s disease patients and their families.”
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. In addition, positron emission tomography (PET) imaging techniques demonstrated sustained neuron cell engraftment in the trial’s high and low dose cohorts following discontinuation of immunosuppression after one year post-treatment. The publication also reports encouraging trends in secondary and exploratory subjective and objective endpoints related to motor function. These data were first presented in March 2024 at the Alzheimer’s and Parkinson’s Diseases Conference in Lisbon, Portugal.
“Cell therapy is a potential new treatment option for individuals with Parkinson’s disease and our team is immensely proud of having bemdaneprocel’s 18-month Phase 1 data published in such a prestigious journal as Nature,” said Amit Rakhit, MD, MBA Head of Development and Chief Medical Officer at BlueRock Therapeutics. “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.
About bemdaneprocel (BRT-DA01)
Bemdaneprocel (BRT-DA01) is an investigational cell therapy designed to replace the dopamine-producing neurons that are lost in Parkinson’s disease. These dopaminergic neuron precursors are derived from human embryonic stem cells, developing into mature dopamine neurons after implantation. In a surgical procedure, these neuron precursors are implanted into the brain of a person with Parkinson’s disease. When transplanted, they have the potential to re-form neural networks that have been severely affected by Parkinson’s disease and to potentially restore motor and non-motor function to patients. In 2021 bemdaneprocel received Fast Track designation followed in 2024 by a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. Data from the Phase 1 trial’s 12 participants presented at the 2024 International Congress of Parkinson’s Disease and Movement Disorders (MDS) demonstrated good tolerability, with no serious adverse events related to drug product at 24 months post-surgery. Further, encouraging trends were observed in secondary endpoints related to motor impairments at 24 months post-surgery. These participants remain in a long-term Continued Evaluation Study. Bemdaneprocel has not been approved for treatment of any disease or medical condition by any health authority.
About Parkinson’s disease
Parkinson’s disease (PD) is a progressive neurodegenerative disease. It has a significant impact on a person’s daily life. In PD, the death of dopamine producing nerve cells in the brain leads to the continuous loss of motor function. Symptoms include tremors, muscle rigidity, and slowness of movement. Additionally, people with PD experience non-motor symptoms, including fatigue and lack of energy, cognitive issues, and depression. Symptoms typically intensify over time and make everyday tasks demanding. The prevalence of PD has doubled over the past 25 years. Today, more than 10 million people worldwide are estimated to be living with PD. This makes it the world’s second most prevalent neurodegenerative disease. It is also the most frequent movement disorder. At present there is no cure, and current treatment options lack the holistic management of symptoms so new therapies are needed.
About BlueRock Therapeutics LP
BlueRock Therapeutics LP is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for people suffering from neurological, ophthalmic, cardiovascular and immunological diseases. Two of our novel investigational cell therapies, bemdaneprocel (BRT-DA01) for the treatment of Parkinson’s disease and OpCT-001 for the treatment of primary photoreceptor disease are clinical stage programs. Bemdaneprocel has RMAT (Regenerative Medicine Advanced Therapy) and Fast Track designation from the US FDA (Food and Drug Administration) and is expected to begin a Phase 3 clinical trial in H1 2025. OpCT-001 is expected to begin Phase 1 clinical testing in H1 2025. BlueRock was founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, the impact-investing arm of Bayer AG that invests in paradigm-shifting breakthrough innovation. In late 2019, BlueRock became a wholly owned, independently operated subsidiary of Bayer AG as a cornerstone of its newly formed Cell & Gene Therapy platform. Our culture is defined by the courage to persist regardless of the challenge, the urgency to transform medicine and deliver hope, integrity guided by mission, and community-mindedness with the understanding that we are all part of something bigger than ourselves.
About Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2024, the Group employed around 93,000 people and had sales of 46.6 billion euros. R&D expenses amounted to 6.2 billion euros.
