NurOwn (debamestrocel) / BrainStorm Cell Therap, Tel Aviv University - LARVOL DELTA

BrainStorm Cell Therapeutics Submits IND Amendment to FDA, Paving the Way for Phase 3b Trial of NurOwn in ALS (PRNewswire) - "BrainStorm Cell Therapeutics Inc...today announced the submission of an Investigational New Drug (IND) amendment to the U.S. Food and Drug Administration (FDA) for NurOwn...for amyotrophic lateral sclerosis (ALS). This important milestone sets the stage for the initiation of BrainStorm's highly anticipated Phase 3b clinical trial, which has been designed in collaboration with the FDA under a Special Protocol Assessment (SPA). The SPA agreement confirms that the Phase 3b trial design and statistical analysis plan is in line with the FDA's expectations, validating its potential to support a future Biologics License Application (BLA). The company now anticipates a swift regulatory review of the IND, clearing the way for the trial's launch."

IND • New P3 trial • Amyotrophic Lateral Sclerosis

BrainStorm Cell Therapeutics to Host KOL Webinar on Current Developments in the Treatment of Amyotrophic Lateral Sclerosis (ALS) on December 11, 2024 (PRNewswire) - "BrainStorm Cell Therapeutics Inc...today announced that it will host a key opinion leader (KOL) webinar on Wednesday, December 11, 2024 at 10:00 AM ET. To register, click here. The webinar will feature Dr. Terry Heiman-Patterson...who will discuss the current treatment landscape and advances for patients with amyotrophic lateral sclerosis (ALS). Dr. Heiman-Patterson...who will share updates on the company's planned Phase 3b registrational clinical trial, NurOwn (autologous MSC-NTF cells), and future development plans."

Clinical • Amyotrophic Lateral Sclerosis • CNS Disorders

BrainStorm Cell Therapeutics and Pluri Partner to Support NurOwn Phase 3b Trial Manufacturing (PRNewswire) - "BrainStorm Cell Therapeutics Inc...today announced that it has entered into a Memorandum of Understanding (MOU) with Pluri Inc...to manufacture NurOwn for use in BrainStorm's planned Phase 3b trial in amyotrophic lateral sclerosis (ALS). This MOU enables BrainStorm to begin transfer of its manufacturing technology and start producing NurOwn at Pluri's manufacturing facility upon finalizing the binding definitive agreement. Under the terms of the proposed agreement, Pluri will provide GMP-compliant manufacturing of NurOwn at its facilities in Israel...BrainStorm plans to hold a conference call for investors later in Q4 2024, during which it will provide further updates on the NurOwn Phase 3 clinical program."

Clinical • Commercial • Amyotrophic Lateral Sclerosis • CNS Disorders

BrainStorm Cell Therapeutics Presented Positive Survival Data from NurOwn Expanded Access Program at 2024 Annual NEALS Meeting (PRNewswire) - P=NA | N=NA | NCT04681118 | Sponsor: Brainstorm-Cell Therapeutics | "Ten participants (6 debamestrocel, 4 placebo) from Brainstorm's prior Phase 3 clinical trial (BCT-002-US) were enrolled in an open label Expanded Access Program (EAP)...At the last available visit in the EAP, 9/10 participants were alive. The survival curves revealed a statistically significant difference in favor of debamestrocel (LRT, p= 0.0379) with a median survival time of 46.6 months for the debamestrocel group compared to 41.1 months for the matched control. Among the six 'early-start' participants (those who had received Debamestrocel during the Phase 3 study), a continual reduction in NfL was observed. In contrast, for those who received placebo in the Phase 3, the group median NfL change was 37% by the end of phase 3, indicating worsening neurodegeneration. However, after these participants received debamestrocel in the EAP, the majority showed a stabilization in NfL levels."

Clinical data • Amyotrophic Lateral Sclerosis • CNS Disorders

BrainStorm Cell Therapeutics to Provide Corporate Update on Phase 3b NurOwn Trial at the 2024 Maxim Healthcare Virtual Summit (PRNewswire) - "BrainStorm Cell Therapeutics Inc...today announced its participation in the 2024 Maxim Healthcare Virtual Summit...During the session, Mr. Lebovits will provide an update on the planned Phase 3b clinical trial for NurOwn, the company's investigational cell therapy for ALS. This event presents a valuable opportunity to engage with the investment community and share in-depth insights into the upcoming trial."

Clinical • Amyotrophic Lateral Sclerosis • CNS Disorders

BrainStorm Cell Therapeutics Announces Presentations on NurOwn at 2024 Annual NEALS Meeting (PRNewswire) - "BrainStorm Cell Therapeutics Inc...today announced the acceptance of two abstracts featuring NurOwn (MSC-NTF or Debamestrocel) at the 2024 Annual Northeastern Amyotrophic Lateral Sclerosis Consortium (NEALS) Meeting, to take place October 21 - 24, in-person in Clearwater, Florida and virtually...'Our top priority continues to be the initiation of a Phase 3b clinical trial, in order to confirm NurOwn's efficacy and conclusively demonstrate its efficacy in early stage ALS patients...As we make preparations, we anticipate multiple key milestones in the coming months that we believe will have potential to drive value.'"

Clinical data • Clinical protocol • Amyotrophic Lateral Sclerosis • CNS Disorders

BrainStorm Cell Therapeutics Reaches Alignment with FDA on CMC Aspects of Phase 3b NurOwn Clinical Trial (PRNewswire) - "BrainStorm Cell Therapeutics Inc...announced it has reached alignment with the U.S. Food and Drug Administration (FDA) on the Chemistry, Manufacturing, and Controls (CMC) aspects of Brainstorm's Phase 3b clinical trial for NurOwn, its investigational therapy for amyotrophic lateral sclerosis (ALS). This Type C meeting builds upon the positive momentum established in April 2024, when the FDA granted BrainStorm a Special Protocol Assessment (SPA) agreement for its NurOwn Phase 3b trial."

FDA event • Amyotrophic Lateral Sclerosis • CNS Disorders

BrainStorm Cell Therapeutics to Present New Biomarker Data Suggesting ALS Patients May Benefit From Longer-Term Treatment with NurOwn (PRNewswire) - P3 | N=196 | NCT03280056 | Sponsor: Brainstorm-Cell Therapeutics | "BrainStorm Cell Therapeutics Inc...will present new biomarker data suggesting that ALS patients may benefit from longer-term treatment with debamestrocel (NurOwn)...Recently published NfL data from the Phase 3 trial showed that participants treated with NurOwn had an 11% decline from baseline in NfL...The new EAP data to be presented at the ALS Summit showed that, for participants randomized to NurOwn, there was a 4% decrease from baseline in NfL in Phase 3 and a 27% and 36% decrease from baseline, at the ends of Period 1 and Period 2 of the EAP, respectively. These results suggest continual benefit from extended treatment of NurOwn."

Biomarker • P3 data • Amyotrophic Lateral Sclerosis • CNS Disorders

BrainStorm Cell Therapeutics Announces First Quarter 2024 Financial Results and Provides Corporate Update (PRNewswire) - "BrainStorm will participate in the forthcoming 3rd Annual ALS Drug Development Summit, to take place May 21 to 23, 2024 in Boston MA. Dr. Stacy Lindborg will deliver a presentation Promising Longer-Term Biomarker Data from NurOwn Program in ALS: Spotlight on NfL in EAP Extension Cohort. The new data to be presented suggest that patients may benefit from longer term treatment with NurOwn, as evidenced by changes in neurofilament light (NfL), an important biomarker associated with clinical outcomes in ALS."

Biomarker • P3 data • Amyotrophic Lateral Sclerosis • CNS Disorders

BrainStorm Cell Therapeutics Announces Agreement with FDA on a Special Protocol Assessment (SPA) for Phase 3b Trial in ALS (Yahoo Finance) - "BrainStorm Cell Therapeutics Inc...today announced that it received written agreement from the U.S. Food and Drug Administration (FDA), under a Special Protocol Assessment (SPA), on the design for a Phase 3b trial of NurOwn in amyotrophic lateral sclerosis (ALS)....The SPA agreement with the FDA validates the clinical trial protocol and statistical analysis of the planned Phase 3b trial of NurOwn, demonstrating their adequacy for addressing objectives that support a future BLA (Biologics License Application) in ALS....BrainStorm anticipates commencement of the Phase 3b study in 2024....We've been parallel processing to enable us to deliver the first dose in the trial in 2024...”"

Special protocol assessment • Amyotrophic Lateral Sclerosis • CNS Disorders

BrainStorm Cell Therapeutics Announces Peer-reviewed Publication of Biomarker Data from NurOwn's Phase 3 Clinical Trial in ALS (PRNewswire) - P3 | N=196 | NCT03280056 | Sponsor: Brainstorm-Cell Therapeutics | "BrainStorm Cell Therapeutics Inc...announced the peer-reviewed publication of Phase 3 biomarker data in Muscle and Nerve....The study analyzed forty-five biomarkers and identified three that are predictive of clinical outcomes in debamestrocel-treated participants, including neurofilaments light (NfL), LAP/TGFb1 and Galcetin-1. Treatment with debamestrocel led to significant changes in 64% of the forty-five analyzed biomarkers, spanning various pathways involved in ALS pathology. These changes included increases in anti-inflammatory and neuroprotective markers suggesting a potential ability to reduce inflammation and protect neurons, and decreases in inflammatory and neurodegenerative markers indicating a possible reduction in disease activity and nerve cell damage."

P3 data • Amyotrophic Lateral Sclerosis • CNS Disorders

BrainStorm Cell Therapeutics Submits Special Protocol Assessment (SPA) Request to FDA for Phase 3b Trial of NurOwn in ALS (PRNewswire) - "BrainStorm Cell Therapeutics Inc...announced that it has submitted a Special Protocol Assessment (SPA) request to the U.S. Food and Drug Administration (FDA) for a Phase 3b study of NurOwn, its investigational treatment for amyotrophic lateral sclerosis (ALS)....The submission follows an in-person meeting that BrainStorm held with the FDA in December 2023. The interactions leading up to this submission have provided guidance on advancing the planned Phase 3b trial....The response is expected within 45 days of submission."

New P3 trial • Special protocol assessment • Amyotrophic Lateral Sclerosis • CNS Disorders

BrainStorm Cell Therapeutics to Unveil Phase 3b NurOwn Trial Design at MDA Clinical and Scientific Conference (PRNewswire) - "BrainStorm Cell Therapeutics Inc...today announced that it will present a poster...outlining the proposed design for a Phase 3b trial of NurOwn in amyotrophic lateral sclerosis (ALS) at the MDA Clinical and Scientific Conference, on Monday, March 4 between 6-8 p.m. at the Hilton Orlando Hotel, Florida. The design is for a two-part, multicenter, Phase 3b study to assess the efficacy and safety of NurOwn in participants with ALS. Eligible patients will initially enter a 24-week randomized, double-blind, placebo-controlled period (Part A), followed by a 24-week open-label extension period (Part B). The entry criteria will enroll people living with mild-to-moderate ALS. The primary endpoint will be based on the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)."

Clinical protocol • Amyotrophic Lateral Sclerosis • CNS Disorders

Design of A Phase 3B Trial of Debamestrocel in ALS (MDA 2024) - "Background: Neurotrophic factor (NTF)-secreting mesenchymal stem cells (MSC-NTF) are a novel cell-therapeutic approach aimed at effectively delivering NTFs directly to the site of damage in ALS patients. Participants who are receiving standard of care (e.g., riluzole, edaravone, sodium phenylbutyrate/taurursodiol) must be on a stable dose for at least 60 days before Screening. Outcome Measures: Participants will undergo several safety assessments and efficacy measures including the ALSFRS-R, CAFS, rate of survival, respiratory function (SVC), muscle strength (HHD), patient-reported outcomes such as ALSAQ-40, caregiver burden interview, as well as biomarkers and genetic testing."

P3 data

BrainStorm Granted Patents for Allogeneic Exosome Platform-Product in Neurological Disorder Treatments (PRNewswire) - "Brainstorm Cell Therapeutics Inc...today provided an update on the grant and allowance of three patent applications for NurOwn® and NurOwn-Exosomes. The European Patent office granted patent No. 3105587 titled 'Method of Qualifying of Cells' for a method of qualifying whether a cell population is a suitable therapeutic for treating ALS. The patent also grants claims for an isolated population of mesenchymal stem cells for use in treating ALS. The Australian patent Office granted patent No. 2019252987 titled 'Cell-Type Specific Exosomes and Use Thereof' for an isolated Exosomes population derived from MSC-NTF cells as well as a pharmaceutical composition for the treatment of neurodegenerative diseases. The Israel Patent Office announced the allowance of the same claims for application No. 277447 with an Acceptance date on December 01, 2023."

Patent • Amyotrophic Lateral Sclerosis • CNS Disorders

BrainStorm Cell Therapeutics Announces Outcome of FDA Meeting on NurOwn in ALS (PRNewswire) - "BrainStorm Cell Therapeutics Inc...announced the completion of a productive meeting with the U.S. Food and Drug Administration (FDA) to discuss NurOwn, its investigational treatment for amyotrophic lateral sclerosis (ALS). The primary objective of the meeting was to discuss key considerations for a Special Protocol Assessment (SPA) for a planned Phase 3b registrational trial for NurOwn. As an outcome of the meeting, BrainStorm will submit relevant documentation as outlined by the FDA to support the SPA."

Clinical protocol • FDA event • Amyotrophic Lateral Sclerosis • CNS Disorders

BrainStorm Cell Therapeutics Announces In-Person Meeting with the FDA to Discuss Confirmatory Phase 3 Trial for NurOwn in ALS (PRNewswire) - "BrainStorm Cell Therapeutics Inc...today announced that the US Food & Drug Administration (US FDA) has granted the company a meeting to discuss the regulatory path forward for NurOwn in amyotrophic lateral sclerosis (ALS). The meeting is scheduled to take place on December 6, 2023. Brainstorm will discuss plans for a Special Protocol Assessment (SPA) with the FDA to agree on the overall protocol design for a confirmatory Phase 3 trial in ALS."

Clinical protocol • FDA event • Amyotrophic Lateral Sclerosis

BrainStorm Cell Therapeutics to Present at 6th Annual ALS Research Symposia (PRNewswire) - "BrainStorm Cell Therapeutics Inc...today announced a podium presentation and panel discussion at the 6th Annual ALS Research Symposium hosted by ALS ONE. The presentation will feature new analyses from the NurOwn placebo-controlled Phase 3 amyotrophic lateral sclerosis (ALS) trial that highlight the biological effect of NurOwn through CSF biomarker data. The presentation...will be presented by Bob Dagher, MD, Executive Vice President and Chief Development Officer at BrainStorm."

Biomarker • P3 data • Amyotrophic Lateral Sclerosis • CNS Disorders

NurOwn Autologous Stem Cell Transplant- a Precision Medicine Approach Using Mesenchymal Stem Cell Neurotrophic Factors for ALS (ALS-MND 2023) - "MSC-NTF treatment was well tolerated; there were no safety concerns. 33% of MSC-NTF and 28% of placebo participants met clinical response criteria at 28 wks (odds ratio [OR] = 1.33, p = 0.45); thus, the primary endpoint was not met. A pre-specified analysis of participants with baseline ALSFRS-R 35 (n = 58) showed a clinical response rate at 28 wk of 35% MSC-NTF and 16% placebo (OR = 2.6, p = 0.29)."

Bone Marrow Transplantation • CNS Disorders • Transplantation

BrainStorm Cell Therapeutics Announces Strategic Realignment Prioritizing NurOwn for the Treatment of ALS (PRNewswire) - "BrainStorm Cell Therapeutics Inc...today announced a strategic realignment to enable accelerated development of NurOwn® for the treatment of amyotrophic lateral sclerosis (ALS). This realignment is designed to 1) support the company plans to conduct a double-blind, placebo-controlled Phase 3b U.S. clinical trial for NurOwn in ALS with an open-label extension and 2) continue to publish data from NurOwn's Phase 3 clinical trial on: biomarkers, long-term safety and survival, and the Expanded Access Program, providing transparency around NurOwn data and progressing ALS drug development. In preparation for the trial design and subsequent meeting with FDA, BrainStorm is consulting with the NurOwn Principal Investigators, who are most familiar with NurOwn, an additional panel of independent ALS experts, and a patient advisory group."

Pipeline update • Amyotrophic Lateral Sclerosis • CNS Disorders

BrainStorm Cell Therapeutics to Meet with US FDA to Discuss Development Plan for NurOwn as a Treatment of ALS (PRNewswire) - "BrainStorm Cell Therapeutics Inc...today announced that the US Food and Drug Administration (FDA) has invited the Company to request an expedited face-to-face meeting to discuss the path forward for NurOwn® as a treatment for amyotrophic lateral sclerosis (ALS). BrainStorm remains committed to the ALS Community and is actively exploring the next steps in support of NurOwn, including publication of emerging clinical data and development of a protocol for an additional clinical study....Brainstorm is withdrawing the Biologics License Application (BLA) for NurOwn. The decision to withdraw the BLA was coordinated with FDA and is viewed by FDA as a withdrawal without prejudice."

FDA event • Amyotrophic Lateral Sclerosis • CNS Disorders

BrainStorm Cell Therapeutics Provides Update on FDA Advisory Committee Meeting to Review NurOwn for the Treatment of ALS (PRNewswire) - "BrainStorm Cell Therapeutics Inc...announced the outcome of the U.S. Food and Drug Administration's Cellular, Tissue and Gene Therapies Advisory Committee meeting to review the Biologics License Application (BLA) for NurOwn®, an investigational mesenchymal stem cell therapy for the treatment of amyotrophic lateral sclerosis (ALS). Today the Committee voted that NurOwn did not demonstrate substantial evidence of effectiveness for treatment of mild to moderate ALS."

FDA event • Amyotrophic Lateral Sclerosis

"Hey Dr. Kalambogias, help approve NurOwn!" (@GregHeydetSr)

"my exact thought: federal finding increase when #NurOwn is up for grabs? @alsassociation????" (@HaterAls)

"I may be an asshole but at least I am not taking a trump type (I won the election) to say Nurown met it’s primary and secondary endpoints" (@BrownLN11)