CAMBRIDGE, Mass., July 19, 2021 / PRNewswire / – BlueRock Therapeutics LP, a clinical-stage biopharmaceutical company and wholly-owned subsidiary of Bayer AG, announced that the United States Food and Drug Administration (FDA) has granted Fast Track designation for DA01 for the disease Parkinson’s disease (PD). DA01, BlueRock’s pluripotent stem cell-derived dopaminergic neural therapy, is being evaluated in a Phase 1 study.
The FDA’s Fast Track designation is intended to facilitate the development and review of drug candidates that treat serious conditions and address an unmet medical need. A drug candidate who receives the Fast Track designation may be eligible for more frequent interaction with the FDA to discuss the drug candidate’s development plan as well as eligibility for expedited approval and priority review.
“Receiving the Fast Track designation from the FDA is an important milestone, which will help us further accelerate the clinical development of our DA01 cell therapy approach for Parkinson’s disease,” said Joachim Fruebis, Ph.D., Director of Development by BlueRock. “This is another critical step in BlueRock’s mission to create authentic cellular drugs to reverse devastating disease, with the vision to improve the human condition.”
About the trial
The trial will enroll ten patients in the United States and Canada. The main objective of the Ph1 study is to assess the safety and tolerability of DA01 cell transplantation one year after transplant. The secondary objectives of the study are to assess the evidence for transplant cell survival and motor effects at one and two years after transplantation, to assess 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 disease caused by damage to nerve cells in the brain, resulting in decreased levels of dopamine. The worsening of motor and non-motor symptoms is caused by the loss of dopamine-producing neurons. At the time of diagnosis, it is estimated that patients have already lost 60 to 80% of their dopaminergic neurons. Parkinson’s disease often starts with a tremor in one hand. Other symptoms are stiffness, cramps, and dyskinesias. Parkinson’s disease is the second most common neurodegenerative disease, affecting more than 7.5 million people, including 1.3 million people in North America.
About BlueRock Therapeutics
BlueRock Therapeutics is a cell therapy company designed with a mission to develop regenerative drugs for incurable diseases. The company’s cell + gene platform enables the creation, manufacture and delivery of authentic cell therapies with engineered functionality by simultaneously exploiting pluripotent cell biology and genome editing. This allows for an approach where, in theory, any cell in the body can be made and any gene in the genome can be altered for therapeutic purposes. The platform is widely applicable, but the company today focuses on neurology, cardiology, immunology, and ophthalmology. In August 2019, the company was acquired by Bayer Pharmaceuticals, for an enterprise value of $ 1 billion in upfront and milestone payments. For BlueRock, this is the next step in the journey of proving degenerative diseases are reversible and bringing our breakthrough new drugs to patients who desperately need them. For more information, visit www.bluerocktx.com.
Bayer is a global company with core competencies in the fields of life sciences, healthcare and nutrition. Its products and services are designed to help people and the planet prosper by supporting efforts to address the major challenges presented by a growing and aging global population. Bayer is committed to promoting sustainable development and generating a positive impact with its activities. At the same time, the Group aims to increase its profitability and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality around the world. In fiscal year 2020, the Group employed around 100,000 people and generated sales of € 41.4 billion. R&D expenses before exceptional items amounted to 4.9 billion euros. For more information, visit www.bayer.com.
Certain statements contained 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”, “anticipate”, “ estimate ”and“ intention ”, 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 these forward-looking statements. These factors include, but are not limited to, the timing of our clinical trial for DA01; our findings regarding the safety, tolerability and efficacy of DA01 cell transplantation in patients with Parkinson’s disease; and current FDA and other regulatory requirements for the development of DA01. As with any pharmaceutical product under development, there are significant risks in the development, regulatory approval and commercialization of new products. Unless expressly required by law, BlueRock does not undertake to update or revise any forward-looking statement. All of the Company’s forward-looking statements are expressly qualified by all of these risk factors and other cautionary statements. The information contained herein is only valid as of the date hereof.
SOURCE BlueRock Therapeutics