BlueRock Therapeutics Announces First Patient Dosed in Canada in Phase 1 Trial in Patients with Advanced Parkinson's Disease

CAMBRIDGE, Mass., Jan. 18, 2022 /PRNewswire/ -- BlueRock Therapeutics, a clinical stage biopharmaceutical company and wholly-owned subsidiary of Bayer AG, announced today the dose administration for the first patient in Canada in partnership with the University Health Network in a Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with advanced Parkinson's disease. The purpose of the Ph1 clinical trial is to evaluate the safety, tolerability and preliminary efficacy of DA01 in patients with Parkinson's disease. 

"It is incredibly exciting to be a part of delivering this first-of-its-kind authentic cellular medicine to patients in Canada, with the hope of regenerating lost function and possibly improving and reversing Parkinson's disease," said Joachim Fruebis, Ph.D., Chief Development Officer of BlueRock.

Dr. Andres Lozano, O.C., M.D., Ph.D., F.R.C.S.C., F.R.S.C., F.C.A.H.S., Principal Investigator at the Krembil Brain Institute, University Health Network states, "with this surgery, our objective is to use authentic cells, to have them integrate into the brain and restore lost physiologic function. If successful, this treatment could have implications for the Parkinson's community both in Canada and beyond."

About the DA01 Phase 1 Trial
In Canada, the trial is being conducted in partnership with University Health Network, Canada's largest research and teaching hospital network, located in Toronto, Ontario under the guidance of Dr. Lozano.

This surgery marked the closure of the first cohort in this trial. Enrollment is currently open for the second and final high-dose cohort in both the United States and Canada. The primary objective of the Ph1 trial is to assess the safety and tolerability of DA01 cell 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 and first surgeries being performed by Dr. Viviane Tabar, Chair of the Department of Neurosurgery at Memorial Sloan Kettering Cancer Center. An additional Neurology site was 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. (Principal Investigator).

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% 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. DA01 is being clinically evaluated in a subset of patients with advanced 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 cell's 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.

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.

About University Health Network
University Health Network consists of Toronto General and Toronto Western Hospitals, the Princess Margaret Cancer Centre, Toronto Rehabilitation Institute, and The Michener Institute of Education at UHN. The scope of research and complexity of cases at University Health Network has made it a national and international source for discovery, education and patient care. It has the largest hospital-based research program in Canada, with major research in arthritis, cardiology, transplantation, neurosciences, oncology, surgical innovation, infectious diseases, genomic medicine and rehabilitation medicine. University Health Network is a research hospital affiliated with the University of Toronto.

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 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.

SOURCE BlueRock Therapeutics