BlueRock Therapeutics Receives Permission from Health Canada for DA01 Trial in Parkinson's Disease

CAMBRIDGE, Mass., April 7, 2021 /PRNewswire/ -- BlueRock Therapeutics LP, a clinical stage biopharmaceutical company and wholly-owned subsidiary of Bayer AG, announced that Health Canada has given permission to initiate "Phase 1 Safety and Tolerability Study of MSK-DA01 Cell Therapy for Advanced Parkinson's Disease (PD)." This is the first trial in Canada to study pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson's disease.

"We're excited to initiate this study with our expert collaborators at University Health Network in Toronto as we believe this trial could shift the treatment paradigm for PD patients in Canada and around the world," states Emile Nuwaysir, Ph.D., President and Chief Executive Officer of BlueRock. "Our therapy is intended to replace the midbrain dopaminergic neurons lost in the degenerative condition to rebuild the neural circuit, and thereby restore motor control to Parkinson's patients. If successful, this could demonstrate for the first time that the effects of degenerative disease are, in principle, reversible."

"We are thrilled to be the first national trial site bringing leading-edge clinical research with life-changing potential to the nearly 8 million patients world-wide who are affected by Parkinson's," says Brad Wouters, Ph.D., Executive Vice-President, Science and Research, University Health Network (UHN). "As the largest hospital-based research institution in Canada, UHN is uniquely positioned with both talent and clinical trial infrastructure to support this important research."

"Exploring the potential for a cell therapy that could improve Parkinson's rather than simply slowing its progress is a monumental step – few therapeutic approaches to date have been designed to truly restore function," says the trial's lead investigator Andres Lozano, M.D., Ph.D., Senior Scientist, Krembil Research Institute and RR Tasker Chair in Stereotactic and Functional Neurosurgery, University Health Network. "This is an important step to advance treatment using a novel strategy for a disease that does not yet have a cure and could change lives for many patients."

The trial will enroll ten patients in the United States and Canada, with the Canadian trial site at University Health Network, Canada's largest research and teaching hospital network, located in Toronto, Ontario. The primary objective of the Ph1 study is to assess the safety and tolerability of DA01 cell transplantation at one-year post-transplant. The secondary objectives of the study are to assess the evidence of transplanted cell survival and motor effects at one- and two-years post-transplant, to evaluate continued safety and tolerability at two years, and to assess feasibility of transplantation. 

More information about this trial is available at clinicaltrials.gov (NCT#04802733).

About Parkinson's Disease
Parkinson's disease is a progressive neurodegenerative disorder caused by nerve cell damage in the brain, leading to decreased dopamine levels. The worsening of motor and non-motor symptoms is caused by the loss of dopamine-producing neurons. At diagnosis, it is estimated that patients have already lost 60-80 percent of their dopaminergic neurons. Parkinson's disease often starts with a tremor in one hand. Other symptoms are rigidity, cramping and slowness of movement (bradykinesia). Parkinson's disease is the second most common neurodegenerative disorder, impacting more than 7.5 million people, including 1.3 million in North America.   

About BlueRock Therapeutics
BlueRock Therapeutics is a leading engineered cell therapy company with a mission to develop regenerative medicines for intractable diseases. BlueRock Therapeutics' cell+gene platform harnesses the power of cells to create new medicines for neurology, cardiology, and immunology indications. BlueRock Therapeutics' cell differentiation technology recapitulates the cell's developmental biology to produce authentic cell therapies, which are further engineered for additional function. Utilizing these cell therapies to replace damaged or degenerated tissue brings the potential to restore or regenerate lost function. BlueRock Therapeutics was founded in 2016 by Versant Ventures and Bayer AG and capitalized with one of the largest-ever Series A financings in biotech history by Bayer AG (through its Leaps by Bayer unit) and Versant Ventures. The company was fully acquired by Bayer in 2019. BlueRock Therapeutics' culture is defined by scientific innovation, the highest ethical standards, and an urgency to bring transformative treatments to all who would benefit. For more information, visit bluerocktx.com.

About Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to drive sustainable development and generate 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 2020, the Group employed around 100,000 people and had sales of 41.4 billion euros. R&D expenses before special items amounted to 4.9 billion euros. For more information, go to www.bayer.com.

Forward-Looking Statements 
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as "anticipate," "believe," "forecast," "estimate" and "intend," among others. These forward-looking statements are based on BlueRock's current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, the timing of our clinical trial for DA01; our results regarding the safety, tolerance and efficacy of DA01 cell transplantation for patients with Parkinson's disease; and ongoing FDA, Health Canada and other regulatory requirements regarding the development of DA01. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Except as expressly required by law, BlueRock does not undertake an obligation to update or revise any forward-looking statement. All of the Company's forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date hereof.

SOURCE BlueRock Therapeutics