Brainstorm Cell Therapeutics(BCLI) - 2020 Q4 - Earnings Call Transcript

Financial Data and Key Metrics Changes - Research and Development (R&D) expenses for the year ended December 31, 2020, were $22.3 million, an increase from $17.2 million in 2019, primarily due to increased expenses related to materials, consultants, and payroll [18][19][20] - General and administrative expenses rose to approximately $9.4 million in 2020 from $5.8 million in 2019, driven by higher payroll and stock-based compensation [21] - The net loss for the year ended December 31, 2020, was $31.8 million, or $1.07 per share, compared to a net loss of $23.3 million, or $1.06 per share, in 2019 [21] - Cash and cash equivalents were approximately $42 million at December 31, 2020, compared to approximately $0.6 million at the end of 2019 [21][22] Business Line Data and Key Metrics Changes - The company is focused on the development of NurOwn for ALS, with ongoing discussions with the FDA regarding regulatory approval [7][9] - An expanded access program for ALS patients who completed the Phase III trial has been initiated, reflecting the promise of NurOwn's clinical data [10][44] - The company is also progressing in its clinical program for progressive MS, with top-line data anticipated by the end of Q1 2021 [14][46] Market Data and Key Metrics Changes - The company is actively engaging with the ALS community and regulatory bodies, which has increased confidence and support for NurOwn [8][9] - The ALS phase III trial demonstrated a clinically meaningful slowing of disease progression, which is critical for future regulatory discussions [11][12] Company Strategy and Development Direction - The primary focus is on obtaining regulatory approval for NurOwn in ALS, with a commitment to making it available to patients [6][7] - The company is also exploring the development of NurOwn as a treatment for Alzheimer's disease, targeting multiple biological pathways [15][16] - There is an ongoing assessment of the potential for NurOwn-derived exosomes to treat COVID-19 related ARDS, with discussions for development opportunities [17][52] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the efficacy of NurOwn and emphasized the importance of ongoing dialogue with the FDA [8][39] - The company is committed to addressing unmet medical needs in neurodegenerative diseases and is optimistic about the future of its clinical programs [14][15] Other Important Information - The company has raised approximately $21.8 million through the sale of common stock as part of an amended ATM distribution agreement [22] - The expanded access program for ALS patients is set to begin dosing shortly, with specific eligibility criteria in place [44] Q&A Session Summary Question: If NurOwn is granted FDA approval, will it be authorized for a specific subset of patients or all ALS patients? - Management cannot speculate on approval conditions but believes investigators support broad access for all patients [24][25] Question: Will additional trials be needed for the subgroup with clinically meaningful results from the phase III trial? - New analyses have been presented to the FDA, and management is in active dialogue regarding regulatory pathways [26][28] Question: Can you explain the high percentage of patients under 25 at baseline in the phase III trial? - The entry criteria were based on screening scores, and the trial included a broader set of patients compared to other studies [29][30] Question: What caused the high placebo effect observed in the phase III trial? - The placebo effect could stem from various factors, including patient randomization and disease severity [30][31] Question: What biomarkers were evaluated in the phase III trial, and how do they correlate with treatment response? - The trial collected extensive biomarker data, which correlates with clinical responses and supports NurOwn's mechanism of action [32][34] Question: When will the BLA for NurOwn be submitted to the FDA? - The BLA has not yet been submitted, and management is in ongoing discussions with the FDA regarding the regulatory path [35][39] Question: What is the status of the expanded access program for NurOwn? - The program was developed in partnership with the FDA and will initially treat less severely affected ALS patients [43][44] Question: What are the expectations for the progressive MS study's top-line data? - Top-line data is expected by the end of the current quarter, focusing on safety, efficacy, and biomarker assessments [46][47] Question: Has the Phase III Alzheimer's trial begun in Europe? - The trial is in the final steps of initiation, with no patients yet dosed [48] Question: Will the company conduct a clinical trial for the COVID-19 ARDS program? - The company is reviewing the unmet need and considering various partnerships based on promising preclinical results [52]