{"id":8868,"date":"2024-03-06T05:00:45","date_gmt":"2024-03-06T10:00:45","guid":{"rendered":"https:\/\/www.bluerocktx.com\/?p=8868"},"modified":"2024-07-17T15:30:14","modified_gmt":"2024-07-17T19:30:14","slug":"bluerock-therapeutics-phase-i-clinical-trial-for-parkinsons-disease-continues-to-show-positive-trends-at-18-months","status":"publish","type":"post","link":"https:\/\/www.bluerocktx.com\/bluerock-therapeutics-phase-i-clinical-trial-for-parkinsons-disease-continues-to-show-positive-trends-at-18-months\/","title":{"rendered":"BlueRock Therapeutics phase I clinical trial for Parkinson\u2019s disease continues to show positive trends at 18 months"},"content":{"rendered":"
Berlin, Germany and Cambridge, MA USA, March 6, 2024 \u2013 <\/span><\/b>Bayer AG and BlueRock Therapeutics LP, a clinical stage cell therapy company and wholly owned independently operated subsidiary of Bayer AG, announced today details of 18-month data from the phase I clinical trial for bemdaneprocel, an investigational allogeneic stem cell derived cell therapy for treating Parkinson\u2019s disease. The data were presented at the Alzheimer\u2019s and Parkinson\u2019s Diseases Conference in Lisbon, Portugal.<\/span>\u00a0<\/span><\/p>\n The data demonstrate that at 18 months bemdaneprocel continues to be well tolerated with no major safety issues, transplanted cells survive and engraft in the brain and F-DOPA signal continues to increase after stopping immune suppression therapy at 12 months as outlined in the study\u2019s protocol.\u00a0<\/span>\u00a0<\/span><\/p>\n In addition, exploratory clinical endpoints improved compared to baseline assessments in both cohorts, with participants in the high dose cohort showing greater improvement than those in the low dose cohort. These were assessed by the MDS-Unified Parkinson\u2019s Disease Rating Scale Part III (MDS-UPDRS Part III) and the Hauser Diary, which are tools used to assess Parkinson\u2019s disease severity in motor symptoms.<\/span>\u00a0<\/span><\/p>\n \u201cIt\u2019s exciting that bemdaneprocel met safety and tolerability criteria at 12 months, and now the 18-month results suggest that these allogeneic cells survive and have potentially positive effects even after discontinuation of immunosuppressants,\u201d said Claire Henchcliffe, MD, chair of the UCI School of Medicine Department of Neurology at the University of California, Irvine and one of the study\u2019s Principal Investigators. \u201cWe should not overinterpret results of a phase I study, but this is a promising step that deserves to be followed up with further studies.\u201d<\/span>\u00a0<\/span><\/p>\n Using the Hauser Diary, which categorizes patients as being in the \u201cON\u201d state when their symptoms are well controlled and in the \u201cOFF\u201d state when they experience a worsening of their symptoms, participants in the high dose cohort showed a mean increase of 2.7 hours in time spent in the \u201cGood ON\u201d state time compared with baseline after 18 months. Time spent in the \u201cOFF\u201d state showed a mean decrease of 2.7 hours after 18 months. Participants in the low dose cohort showed a mean improvement of 0.2 hours in the \u201cGood ON\u201d state time compared with baseline and a corresponding mean decrease of 0.8 hours in \u201cOFF\u201d state time.\u00a0<\/span>\u00a0<\/span><\/p>\n In the high dose cohort, an 18-month measurement of the effects of bemdaneprocel using MDS-UPDRS Part III measured in the \u201cOFF\u201d-medication state, showed a mean reduction of 23 points compared with baseline. The low dose cohort showed a mild improvement, with a mean decrease of 8.6 points.<\/span>\u00a0<\/span><\/p>\n \u201cWe are excited to see the continued positive trends in the data from bemdaneprocel\u2019s phase I trial at 18 months,\u201d said Ahmed Enayetallah, Senior Vice President and Head of Development at BlueRock Therapeutics. \u201cWhile it is still early days and there is more work to do, we look forward to further investigating bemdaneprocel in clinical studies.\u201d<\/span>\u00a0<\/span><\/p>\n A phase II study for further clinical testing of bemdaneprocel is planned to begin enrolling patients later this year.<\/span>\u00a0<\/span><\/p>\n \u201cWe are on the leading edge in the research for new treatment options for Parkinson\u2019s disease as bemdaneprocel, the most clinically advanced pluripotent stem derived cell therapy candidate to date for this disease, continues to show positive trends,\u201d said Christian Rommel, Member of the Executive Committee of Bayer\u2019s Pharmaceuticals Division and Head of Research and Development. \u201cThere are good reasons to be optimistic about these early data, and we are excited to move to phase II later this year.\u201d<\/span>\u00a0<\/span><\/p>\n About bemdaneprocel <\/span><\/b>(BRT-DA01) and the phase I trial<\/span><\/b>\u00a0<\/span><\/p>\n Bemdaneprocel (BRT-DA01) is an investigational cell therapy designed to replace the dopamine producing neurons that are lost in Parkinson\u2019s disease. These dopaminergic neuron precursors are derived from pluripotent stem cells that are human embryonic stem cells. In a surgical procedure, these neuron precursors are implanted into the brain of a person with Parkinson\u2019s disease. When transplanted, they have the potential to reform neural networks that have been severely affected by Parkinson\u2019s and restore motor and non-motor function to patients. Bemdaneprocel has not been approved for treatment of any disease or medical condition by any health authority.<\/span>\u00a0<\/span><\/p>\n This phase I study is a multi-center, multi-site, open-label, non-randomized, non-controlled study. Twelve (12) subjects diagnosed with Parkinson\u2019s disease received surgical transplantation of 1 of 2 different dose levels of bemdaneprocel cells to the post-commissural putamen bilaterally, and administration of a 1-year immunosuppression regimen. Cohort A (5 subjects) received a dose of 0.9 million cells per putamen. Cohort B (7 subjects) received 2.7 million cells per putamen. Safety and tolerability were assessed at 1 year as the primary endpoint, along with evidence of cell survival and motor effects. The feasibility of transplantation was also assessed. Assessments were repeated at 18 months. All assessments will continue over 2 years.\u00a0<\/span>\u00a0<\/span><\/p>\n The transplant surgeries were performed by Dr. Viviane Tabar, MD, Chair of the Department of Neurosurgery at Memorial Sloan Kettering (MSK) Cancer Center and Dr. Andres Lozano, M.D., Ph.D., F.R.C.S.C., F.R.S.C., F.C.A.H.S., Neurosurgeon and Senior Scientist, Krembil Brain Institute, University Health Network (UHN), Alan & Susan Hudson Cornerstone Chair in Neurosurgery, Toronto Western Hospital, University Health Network and Chairman of the Division of Neurosurgery at the University of Toronto (UoT). Participants were followed at clinical sites by Dr. Harini Sarva, M.D. at Weill Cornell Medicine, Dr. Claire Henchcliffe, M.D., D.Phil., F.A.A.N., F.A.N.A. at the University of California, Irvine, and Dr. Alfonso Fasano, M.D., PhD., Chair in Neuromodulation and Multi-Disciplinary Care at the University Health Network (UHN) and UoT.\u202f<\/span>\u202f<\/span>\u00a0<\/span><\/p>\n Disclosure:\u202f\u00a0<\/span><\/i>\u00a0<\/span><\/p>\n Memorial Sloan Kettering (MSK): Dr. Tabar has financial interests related to BlueRock. MSK has institutional financial interests related to BlueRock. Note the foregoing institutional disclosure language is included because the referenced study relates to MSK technology licensed to BlueRock.\u202fUniversity Health Network (UHN): UHN has institutional financial interests related to BlueRock.<\/span><\/i>\u00a0<\/span><\/p>\n More information about the Phase I trial is available at <\/span>clinicaltrials.gov<\/span><\/a> (<\/span>NCT04802733<\/span><\/a>).\u202f<\/span>\u00a0<\/span><\/p>\n About Parkinson\u2019s disease<\/span><\/b>\u00a0<\/span><\/p>\n Parkinson\u2019s disease is a progressive neurodegenerative disorder caused by the death of nerve cells in the brain, leading to decreased dopamine levels. At diagnosis, it is estimated that patients have already lost 50-80% of their dopaminergic neurons. The loss of these neurons leads to a progressive loss of motor function and symptoms such as tremors, muscle rigidity, and slowness of movement. Even with medication, the symptoms of Parkinson\u2019s disease can fluctuate during the course of the day. According to the Parkinson\u2019s Foundation, more than 10 million people worldwide suffer from Parkinson\u2019s disease, with approximately one million living in the United States. There is no cure, and the effectiveness of current treatments decreases over time.\u00a0<\/span>\u00a0<\/span><\/p>\n About BlueRock Therapeutics LP<\/span><\/b>\u00a0<\/span><\/p>\n BlueRock Therapeutics LP is a clinical stage cell therapy company focused on creating cellular medicines to reverse devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for patients suffering from neurological, cardiovascular, immunological, and ophthalmic diseases. Our lead clinical program, bemdaneprocel, (BRT-DA01) is in Phase I clinical trials for Parkinson\u2019s disease. We were 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 <\/span>www.bluerocktx.com<\/span><\/a>\u00a0<\/span><\/p>\n About Bayer<\/span><\/b>\u00a0<\/span><\/p>\n Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, \u201cHealth for all, Hunger for none,\u201d the company\u2019s 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 <\/span>www.bayer.com<\/span><\/a>.<\/span>\u00a0<\/span><\/p>\n BlueRock Therapeutics Media Contact:<\/span>\u00a0<\/span> Bayer Media Contact:<\/span> Bayer U.S. Media Contact:<\/span> Find more information at <\/span>www.bluerocktx.com<\/span><\/a> Find more information at <\/span>https:\/\/pharma.bayer.com\/<\/span><\/a> Forward-Looking Statements<\/span><\/b>\u00a0<\/span>\u00a0<\/span><\/p>\n Certain statements in this press release are forward-looking which may be identified by the use of forward-looking words such as \u201canticipate,\u201d \u201cbelieve,\u201d \u201cforecast,\u201d \u201cestimate\u201d and \u201cintend,\u201d among others. These forward-looking statements are based on BlueRock\u2019s 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 outcomes from our clinical trials and ongoing FDA and other regulatory requirements. 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 BlueRock\u2019s 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.<\/span>\u00a0<\/span><\/p>\n 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\u2019s public reports which are available on the Bayer website at <\/span>www.bayer.com<\/span><\/a>. The company assumes no liability whatsoever to update these forward-looking statements or to conform<\/span> them to future events or developments.\u00a0<\/span>\u00a0<\/span><\/p>\n","protected":false},"excerpt":{"rendered":" Investigational cell therapy bemdaneprocel continues to be well tolerated with no major safety issues in all 12 participants in low and high dose cohorts through 18 months\u00a0 After stopping the 12-month immune suppression regimen, assessment of 18-month data shows evidence of sustained cell engraftment […]<\/p>\n","protected":false},"author":2,"featured_media":7775,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[45],"tags":[52,56,61],"class_list":["post-8868","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-press-releases","tag-announcement","tag-neurology","tag-parkinsons-disease"],"acf":[],"yoast_head":"\n
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