BrainStorm Expands Clinical Pipeline to Evaluate NurOwn for Alzheimer's Disease

BrainStorm Cell Therapeutics announced a new clinical program focused on the development of NurOwn® as a treatment for Alzheimer's disease. As part of the newly announced program, the Company is planning a multi-national Phase 2 clinical trial to evaluate the safety and efficacy of NurOwn treatment in patients with prodromal to mild Alzheimer's disease.

"Our newly announced Alzheimer's program is an important strategic expansion of BrainStorm's clinical pipeline," said Chaim Lebovits, Chief Executive Officer of BrainStorm. "We see this as an ideal complement to our lead amyotrophic lateral sclerosis (ALS) program, as there are compelling data suggesting that the benefits observed with NurOwn in ALS patients may extend to other neurological conditions, including Alzheimer's disease. As we move towards completion of our pivotal Phase 3 ALS study, the newly announced Alzheimer's program, combined with our ongoing progressive multiple sclerosis (MS) program, will continue to drive the advancement of the NurOwn platform. Through the completion of our clinical trial programs, BrainStorm hopes to advance NurOwn to address unmet needs across a broad population of patients with neurodegenerative disease."

The lead investigators of the trial are Philip Scheltens, M.D., Ph.D., and Bruno Dubois, M.D., Ph.D. Prof. Scheltens, the principal investigator (PI) of the study, is Professor of Cognitive Neurology and Director of the Alzheimer Centre at Amsterdam University Medical Centers. He has extensive experience as PI of many international clinical trials in this field. Prof. Dubois, the French national coordinator of the study, is Professor of Neurology at the Neurological Institute of the Salpétrière University Hospital. He is President of the Scientific Committee of France-Alzheimer and of IFRAD (International Fund Raising for Alzheimer's Disease) as well as a member of the European Alzheimer Disease Consortium (EADC).

"We are very eager to evaluate the efficacy of NurOwn in the treatment of Alzheimer's disease, because of its unique mechanism of action. While many previous Alzheimer's therapies have focused on a single target such as tau or beta-amyloid, NurOwn has the capability to simultaneously target multiple relevant biological pathways and bring a comprehensive approach to this multifactorial disease. Importantly, NurOwn's mechanism of action may also allow the therapy to enable synergistic combinations with anti-tau or anti-beta-amyloid treatments, further underscoring its potential to address critical unmet needs in Alzheimer's disease," Prof. Scheltens said.

"In such a complex disease, addressing inflammation and neuro-protection is an innovative and very interesting approach. It is a first in the world for this technology and Brainstorm is leveraging encouraging results from the Phase 2 study in ALS," Prof. Dubois said.

BrainStorm plans to conduct a 52-week, Phase 2 open-label, proof-of-concept clinical trial to evaluate NurOwn in 40 participants with prodromal to mild Alzheimer's disease. Study participants will receive three intrathecal NurOwn doses 8 weeks apart. In addition to meeting well-defined clinical criteria for prodromal to mild Alzheimer's disease, participants must also meet biomarker defined criteria for Alzheimer's disease. The clinical trial will evaluate safety and pharmacodynamics of NurOwn, including effects on inflammatory, Alzheimer's-specific, neurodegenerative, and synaptic biomarkers, as well as a range of key clinical measures of cognition and function. The clinical trial will be conducted at the Brain research Center affiliated with the Alzheimer Center Amsterdam, Pitié-Salpêtrière Hospital (Paris), and several other clinical trial sites in the Netherlands and France.