Phase III exPDite-2 trial follows completion and discussion of Phase I trial data with US FDA under Regenerative Medicine Advanced Therapy (RMAT) designation
exPDite-2 is a randomized, sham surgery-controlled double-blind trial assessing efficacy and safety of bemdaneprocel in people living with moderate Parkinson’s disease
Expected to be initiated in H1 2025, exPDite-2 is the first registrational Phase III clinical trial for an investigational allogeneic pluripotent stem cell derived therapy to treat Parkinson’s disease
BERLIN, Germany, and CAMBRIDGE, MA, USA I January 13, 2025 I Bayer AG and BlueRock Therapeutics LP, a clinical-stage cell therapy company and wholly owned subsidiary of Bayer AG, today 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.
“We are thrilled to take this critical step in our development program towards further investigating a potential new therapeutic option for people living with Parkinson’s disease,” said Amit Rakhit, MD, MBA, Chief Development and Medical Officer at BlueRock Therapeutics. “exPDite-2 is the first registrational Phase III clinical trial for an investigational pluripotent stem cell derived therapy in Parkinson’s disease and we look forward to working closely with clinical investigators and the Parkinson’s disease community as we initiate this trial.”
In a Phase I study with 12 participants, bemdaneprocel demonstrated 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. Building on these results, exPDite-2 is a double-blind trial that will assess the efficacy, safety and overall impact of bemdaneprocel compared to a sham surgery control. The trial is designed to enroll approximately 102 participants with moderate Parkinson’s disease. The primary endpoint of the study is change from baseline to week 78 in PD diary measure of ON-time without troublesome dyskinesia, adjusted for a 16-hour waking day. In addition, the trial will incorporate secondary endpoints designed to capture objective measures of movement, safety and tolerability, and instruments that capture activities of daily living and quality of life.
The advancement to this registrational trial follows discussions with the U.S. Food and Drug Administration (FDA) under the Regenerative Medicine Advanced Therapy (RMAT) designation granted by the FDA in May 2024.
“With the planned initiation of the Phase III clinical trial, we are committed to bringing bemdaneprocel faster to patients in need,” said Christian Rommel, Member of the Executive Committee of Bayer’s Pharmaceuticals Division, and Global Head of Research and Development. “It represents a significant milestone in our efforts to advance our cell and gene therapy pipeline and deliver on our ambition to be an industry leader in this space.”
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.
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 pluripotent stem cells that are 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 and in 2024 a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. Data from the Phase I 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 continue in the 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 III clinical trial in H1 2025. OpCT-001 is expected to begin Phase I 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. For more information, visit www.bluerocktx.com.
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 2023, the Group employed around 100,000 people and had sales of 47.6 billion euros. R&D expenses before special items amounted to 5.8 billion euros. For more information, go to www.bayer.com.
Find more information at https://pharma.bayer.com
Follow us on Facebook: http://www.facebook.com/bayer
Find information at www.bluerocktx.com
Follow us on LinkedIN: www.linkedin.com/company/bluerocktx
SOURCE: Bayer
Post Views: 138
Phase III exPDite-2 trial follows completion and discussion of Phase I trial data with US FDA under Regenerative Medicine Advanced Therapy (RMAT) designation
exPDite-2 is a randomized, sham surgery-controlled double-blind trial assessing efficacy and safety of bemdaneprocel in people living with moderate Parkinson’s disease
Expected to be initiated in H1 2025, exPDite-2 is the first registrational Phase III clinical trial for an investigational allogeneic pluripotent stem cell derived therapy to treat Parkinson’s disease
BERLIN, Germany, and CAMBRIDGE, MA, USA I January 13, 2025 I Bayer AG and BlueRock Therapeutics LP, a clinical-stage cell therapy company and wholly owned subsidiary of Bayer AG, today 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.
“We are thrilled to take this critical step in our development program towards further investigating a potential new therapeutic option for people living with Parkinson’s disease,” said Amit Rakhit, MD, MBA, Chief Development and Medical Officer at BlueRock Therapeutics. “exPDite-2 is the first registrational Phase III clinical trial for an investigational pluripotent stem cell derived therapy in Parkinson’s disease and we look forward to working closely with clinical investigators and the Parkinson’s disease community as we initiate this trial.”
In a Phase I study with 12 participants, bemdaneprocel demonstrated 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. Building on these results, exPDite-2 is a double-blind trial that will assess the efficacy, safety and overall impact of bemdaneprocel compared to a sham surgery control. The trial is designed to enroll approximately 102 participants with moderate Parkinson’s disease. The primary endpoint of the study is change from baseline to week 78 in PD diary measure of ON-time without troublesome dyskinesia, adjusted for a 16-hour waking day. In addition, the trial will incorporate secondary endpoints designed to capture objective measures of movement, safety and tolerability, and instruments that capture activities of daily living and quality of life.
The advancement to this registrational trial follows discussions with the U.S. Food and Drug Administration (FDA) under the Regenerative Medicine Advanced Therapy (RMAT) designation granted by the FDA in May 2024.
“With the planned initiation of the Phase III clinical trial, we are committed to bringing bemdaneprocel faster to patients in need,” said Christian Rommel, Member of the Executive Committee of Bayer’s Pharmaceuticals Division, and Global Head of Research and Development. “It represents a significant milestone in our efforts to advance our cell and gene therapy pipeline and deliver on our ambition to be an industry leader in this space.”
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.
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 pluripotent stem cells that are 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 and in 2024 a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. Data from the Phase I 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 continue in the 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 III clinical trial in H1 2025. OpCT-001 is expected to begin Phase I 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. For more information, visit www.bluerocktx.com.
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 2023, the Group employed around 100,000 people and had sales of 47.6 billion euros. R&D expenses before special items amounted to 5.8 billion euros. For more information, go to www.bayer.com.
Find more information at https://pharma.bayer.com
Follow us on Facebook: http://www.facebook.com/bayer
Find information at www.bluerocktx.com
Follow us on LinkedIN: www.linkedin.com/company/bluerocktx
SOURCE: Bayer
Post Views: 138
