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CAMBRIDGE, Mass., May 31, 2022 – BlueRock Therapeutics LP, a clinical stage biopharmaceutical company and wholly-owned subsidiary of Bayer AG, 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.
“Patients with Parkinson’s disease are faced with life changing challenges and uncertainties that are not addressed by current treatments,” said Ahmed Enayetallah, M.D., Ph.D., Senior Vice President, Clinical Development, BlueRock Therapeutics. “We are excited that our Phase 1 clinical trial has completed enrollment and believe it is a critical next step toward the development of a novel cell therapy that has the potential to transform the treatment landscape for this devastating disease.”
About the BRT-DA01 Phase 1 Trial
The primary objective of the Ph1 trial is to assess the safety and tolerability of BRT-DA01 transplantation at one-year post-transplant. The secondary objectives of the trial 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.
In the United States, the trial was initiated at Weill Cornell Medicine with Dr. Harini Sarva, M.D. as the Principal Investigator (PI) and first surgeries being performed by Dr. Viviane Tabar, Chair of the Department of Neurosurgery at Memorial Sloan Kettering Cancer Center. Additional Neurology sites were added at the University of California, Irvine, under the guidance of Dr. Claire Henchcliffe, M.D., D.Phil., F.A.A.N., F.A.N.A. (PI), and at the University Health Network (UHN), under the guidance of Dr. Lozano, O.C., M.D., Ph.D., F.R.C.S.C., F.R.S.C., F.C.A.H.S. (PI) and Dr. Alfonso Fasano, M.D., PhD., Chair in Neuromodulation and Multi-Disciplinary Care, UHN and UoT.
More information about the Phase 1 trial is available at clinicaltrials.gov (NCT#04802733)
As a next step, BlueRock Therapeutics plans to initiate a global non-interventional study in the second half of 2022.
More information about the non-interventional study is available at clinicaltrials.gov (NCT#05363046)
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% 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). According to the Parkinson’s Foundation, more than 10 million people worldwide suffer from Parkinson’s disease, with one million living in the United States. BRT-DA01 is being clinically evaluated in a subset of patients with Parkinson’s disease, where regular medications are no longer fully effective.
About BlueRock Therapeutics
BlueRock Therapeutics is a leading engineered cell therapy company with a mission to create authentic cellular medicines to reverse devastating diseases, with the vision of improving the human condition. The Company’s cell+gene platform harnesses the power of cells to create new medicines for neurology, cardiology, immunology, and ophthalmology indications. BlueRock Therapeutics’ cell differentiation technology recapitulates the cells’ developmental biology to produce authentic cells, some of which are engineered for additional therapeutic function. Utilizing these cell therapies to replace damaged or degenerated tissue brings the potential to restore 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 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.
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 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 2021, the Group employed around 100,000 people and had sales of 44.1 billion euros. R&D expenses before special items amounted to 5.3 billion euros. For more information, go to www.bayer.com.
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 the Company’s current expectations of BlueRock 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 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.
This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.
Vice President, Corporate Communications