BrainStorm Issues 2024 Letter to Shareholders

Core Insights - The company achieved a significant milestone by securing a Special Protocol Assessment (SPA) with the FDA for the Phase 3b clinical trial of NurOwn, which de-risks the regulatory aspects of its clinical development pathway [2][4] - The company has demonstrated the potential of NurOwn to reduce neurofilament light (NfL) levels, a key biomarker of neurodegeneration in ALS patients, indicating its ability to mitigate neuronal damage [7] - The leadership team has been strengthened with the addition of Dr. Haro Hartounian as COO and the promotion of Bob Dagher, M.D., to CMO, enhancing operational and clinical development capabilities [8] - The company is advancing its exosome platform, which has significant potential for neurodegenerative and respiratory diseases, supported by a recent Notice of Allowance from the U.S. Patent and Trademark Office [9] - The company has raised approximately $8 million since the ADCOM results and is actively pursuing additional funding sources to support the Phase 3b trial and exosome platform development [10] Research and Development - The Phase 3b trial for NurOwn is focused on early-stage ALS patients, aiming to provide robust evidence of the treatment's efficacy [4] - Exploratory analyses have highlighted potential correlations between NurOwn treatment and other biomarkers associated with inflammation and neuroprotection, reinforcing the therapy's multifaceted benefits [3] Corporate Developments - The company entered a Memorandum of Understanding (MOU) with Pluri Inc. for the clinical manufacturing of NurOwn, which will enhance its manufacturing capabilities [17] - The company has maintained financial discipline and operational excellence despite challenges in the biotechnology sector, including a difficult financing environment [10] Future Outlook - The company is entering 2025 with optimism, focusing on the launch of the Phase 3b trial and the advancement of its exosome platform [12][21] - The recent FDA approval of Ryoncil, the first MSC-based therapy, has inspired confidence in the company's own MSC-based therapeutic approach with NurOwn [21]