On October 15, 2020 BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, reported financial results for the third quarter ended September 30, 2020, and provided a corporate update (Press release, BrainStorm Cell Therapeutics, OCT 15, 2020, View Source [SID1234568538]).
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"The most important near-term event for BrainStorm will be the upcoming top-line data readout for the NurOwn Phase 3 trial in ALS, expected by the end of November. A successful outcome will set us on the path to filing a Biologic License Application (BLA) for what we believe will be a valuable new treatment for ALS," said Chaim Lebovits, Chief Executive Officer of BrainStorm Cell Therapeutics. "In parallel to our preparations for upcoming data read out, we are very busy planning and executing on other pre-BLA activities. On the management front, we appointed William K. White and Dr. Anthony Waclawski, adding valuable commercial and regulatory expertise to our leadership team. This expertise will be crucial as we work towards obtaining regulatory approval for NurOwn and ensuring that, if approved, it will be readily accessible to ALS patients in need of new treatment options for this devastating disease."
NurOwn has an innovative mechanism of action that is broadly applicable across neurodegenerative diseases and BrainStorm continues to invest in clinical trials evaluating the product in conditions beyond ALS to maximize value creation for its various stakeholders. The company remains on track to complete dosing in its Phase 2 clinical trial in progressive multiple sclerosis (PMS) by the end of 2020. In addition, the Company recently unveiled a clinical development program in Alzheimer’s’ disease (AD) and is planning a Phase 2 proof-of-concept clinical trial at several leading AD centers in the Netherlands and France.
Third Quarter 2020 and Recent Corporate Highlights:
Completed dosing of all patients in the ongoing NurOwn Phase 3 clinical trial in ALS and plans remains on track to report top-line data by the end of November 2020.
Announced the publication of a manuscript titled "Effects of MSC-NTF cells on T and B regulatory cell function in ALS" in the journal Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. The manuscript describes NurOwn’s immunomodulatory effects on T and B regulatory cell function, suggesting that its mechanism of action is broadly applicable in ALS, PMS and Alzheimer’s disease.
Presented a scientific poster titled "Advancing NurOwn for ALS: Phase 3 Clinical Trial Design" at the Annual Northeast ALS (NEALS) Meeting.
A Phase 2 trial evaluating NurOwn as a treatment for progressive multiple sclerosis (PMS) is ongoing at 5 leading U.S. multiple sclerosis centers. The Company remains on track to complete dosing by year end 2020.
Presented progressive MS natural history data at the MSVirtual2020 meeting documenting an association between magnetic resonance imaging (MRI) measures and functional improvement in patients matched to the phase 2 NurOwn clinical trial.
Hosted a Key Opinion Leader (KOL) webinar on the Alzheimer’s disease program and the planned European Phase 2 trial. The webinar featured presentations by two lead investigators in the trial: Philip Scheltens, M.D., Ph.D., Professor of Cognitive Neurology and Director of the Alzheimer Centre at the VU University Medical Center in Amsterdam, Netherlands; and Bruno Dubois, M.D., Ph.D., Professor of Neurology at the Neurological Institute of the Salpétrière University Hospital in Paris, France.
Announced a groundbreaking pre-clinical study of a NurOwn derived exosome-based treatment for COVID-19 acute respiratory distress syndrome (ARDS).
Appointed Anthony Waclawski Ph.D. as Executive Vice President, Global Head of Regulatory Affairs.
Appointed William K. White as Senior Vice President, Head of Market Access and Pricing.
Received a non-dilutive bonus payment of $700,000 from California Institute for Regenerative Medicine (CIRM) for treating more California participants than originally proposed in the Phase 3 ALS trial.
Announced the grant of Japanese Patent No. 6,753,887, titled: "Methods of Generating Mesenchymal Stem Cells which Secrete Neurotrophic Factors".
Presented at the following Investor Conferences:
40th Annual Canaccord Genuity Growth Conference
Cash and Liquidity as of October 14, 2020
Total available funding as of October 14, 2020, which includes cash, cash equivalents and short-term bank deposits of approximately $33.1 million as well as remaining non-dilutive funding from CIRM, IIA and other grants, amounts to approximately $36 million.
Financial Results for the Three Months Ended September 30, 2020
Cash and cash equivalents, and short-term bank deposits amounted to approximately $28.8 million at September 30, 2020 compared to $16.2 million at June 30, 2020.
Research and development expenses, net for the three months ended September 30, 2020 and 2019 were $1.87 million and $4.01 million, respectively.
Excluding participation from IIA and CIRM under the grants and proceeds received under the hospital exemption regulatory pathway, research and development expenses decreased by $1.68 million from $5.66 million in the third quarter of 2019 to $3.98 million in the third quarter of 2020.
General and administrative expenses for the three months ended September 30, 2020 and 2019 were $2.62 million and $1.54 million, respectively.
Net loss for the three months ended on September 30, 2020 was $4.49 million, as compared to a net loss of $5.63 million for the three months ended September 30, 2019.
Net loss per share for the three months ended September 30, 2020 and 2019 was $0.14 and $0.25, respectively.
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About NurOwn
NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received acceptance from the U.S. Food and Drug Administration (FDA) to initiate a Phase 2 open-label multicenter trial in progressive multiple sclerosis (MS) and completed enrollment in August 2020.