Brainstorm Cell Therapeutics(BCLI)2025-05-15 21:10Clinical Trials and Regulatory Status - NurOwn® has completed Phase 3 ALS and Phase 2 PMS clinical trials, with top-line data announced on November 17, 2020, and March 24, 2021, respectively[83]. - The FDA granted Fast Track designation and Orphan Drug status for NurOwn® for ALS, enhancing its regulatory profile[85]. - On September 27, 2023, the Advisory Committee voted 17 no, 1 yes, and 1 abstention, indicating that NurOwn® did not demonstrate substantial evidence of effectiveness for mild to moderate ALS[83]. - The BLA for NurOwn® was withdrawn on November 3, 2023, in coordination with the FDA, viewed as a withdrawal without prejudice[83]. - The company plans to submit a Special Protocol Assessment request to the FDA for a Phase 3b trial of NurOwn® for ALS[83]. - A productive meeting with the FDA on December 6, 2023, focused on plans for a Special Protocol Assessment (SPA) for a Phase 3b trial of NurOwn®[97]. - The FDA granted a meeting to discuss the regulatory path forward for NurOwn® on November 20, 2023, following the withdrawal of the BLA[97]. - The company submitted a BLA to the FDA for NurOwn® for ALS treatment on September 9, 2022, but received a RTF letter on November 10, 2022, indicating the BLA was not sufficiently complete for review[115]. - The FDA granted a Type A meeting on January 11, 2023, where the company was presented with options to return the BLA to regulatory review, including filing over protest[116]. - The BLA was re-filed on February 7, 2023, after the company requested the FDA to file the NurOwn® BLA for ALS over protest[116]. - An amendment to the BLA was submitted on March 7, 2023, addressing the majority of items in the RTF letter, and the FDA confirmed an ADCOM for the BLA on March 22, 2023[117]. - The Advisory Committee voted on September 27, 2023, with 17 voting no, one voting yes, and one abstention, indicating that NurOwn® did not demonstrate substantial evidence of effectiveness for mild to moderate ALS[117]. - The company announced the withdrawal of the BLA for NurOwn® on November 3, 2023, coordinated with the FDA and viewed as a withdrawal without prejudice[117]. - A meeting with the FDA on December 6, 2023, focused on discussing plans for a SPA for a planned Phase 3b registrational trial for NurOwn®[117]. - The SPA request for the Phase 3b clinical trial was submitted on February 23, 2024, and written agreement from the FDA was received on April 9, 2024[118]. Clinical Trial Results - The Phase 2 trial showed that 32.6% of NurOwn® participants met the primary endpoint compared to 27.7% for placebo, but did not reach statistical significance (p=0.453)[112]. - In a pre-specified subgroup with ALSFRS-R baseline scores of 35 or greater, NurOwn® demonstrated a 2.09 point improvement over placebo (p=0.050)[112]. - The Phase 3 trial enrolled rapidly progressing ALS patients but did not achieve statistically significant results, with a responder analysis showing 34.7% for NurOwn® versus 20.5% for placebo (p=0.053)[112]. - The company has completed two Phase 1/2 open-label trials and a Phase 2 double-blind trial, demonstrating tolerability and preliminary signs of activity for NurOwn®[98][99]. - The Phase 2 trial published in JAMA Neurology indicated a slower rate of disease progression in ALS patients treated with NurOwn®[100]. - Key findings from the PMS trial included a 10% mean improvement in the timed 25-foot walk (T25FW) and a 4.8% improvement in the 9-hole peg test (9-HPT) for NurOwn® treated patients compared to historical controls[145]. Financial Overview and Capital Needs - The company aims to raise $2.5 million in stockholders' equity to regain compliance with Nasdaq Listing Rule 5550[87]. - The company reported a net loss of $2,864,000 for the quarter ended March 31, 2025, compared to a net loss of $3,401,000 for the same quarter in 2024, resulting in a net loss per share of $0.45[176]. - Research and development expenditures for the quarter ended March 31, 2025, were $1,304,000, an increase of 35.7% from $961,000 in the same quarter of 2024[172]. - General and administrative expenses increased to $1,785,000 for the quarter ended March 31, 2025, up from $1,513,000 in the prior year, reflecting a rise of 17.9%[174]. - The company had cash, cash equivalents, and restricted cash of $1,826,000 as of March 31, 2025[182]. - Net cash used in operating activities for the quarter ended March 31, 2025, was $1,631,000, primarily due to clinical trial costs and payroll expenses[183]. - The company generated $3,086,000 in net cash from financing activities during the quarter ended March 31, 2025, from sales of common stock and warrants exercise[184]. - The Company anticipates continued losses from clinical development and regulatory activities, necessitating additional capital raises for future trials and commercialization efforts[196]. - Future capital requirements will depend on various factors, including clinical trial costs, regulatory approvals, and commercialization expenses[202]. - The Company expects to incur additional financing needs, potentially through debt or equity, which may dilute existing stockholders[198]. Manufacturing and Production - The NurOwn® manufacturing process is compliant with Good Manufacturing Practice, ensuring quality and safety in production[92]. - The company has developed a validated cryopreservation process for MSC, allowing multiple doses of NurOwn® from a single bone marrow harvest[120]. - The company has contracted with City of Hope and Dana Farber Cancer Institute for the manufacturing of NurOwn® for clinical studies, enhancing production capabilities[121]. - The company has improved the efficiency and stability of NurOwn® production, allowing centralized manufacturing for clinical trials[168]. Intellectual Property and Grants - The company has a comprehensive intellectual property portfolio for NurOwn®, with exclusive commercialization rights held by its Israeli subsidiary[85]. - The Company holds approximately 30 granted patents and is expanding its portfolio to include exosome-based technologies, with a recent patent expected to provide protection until April 10, 2039[150][151]. - The Company was awarded a total of $15,912,390 in grants from the California Institute for Regenerative Medicine to fund the pivotal Phase 3 study of NurOwn® for ALS[147]. Stock and Equity Transactions - The Company entered into a New Distribution Agreement allowing the sale of up to $100 million in Common Stock, with gross proceeds of approximately $706 from 388,059 shares sold under this agreement as of March 31, 2025[191]. - On July 17, 2023, the Company sold 270,270 shares of Common Stock at $27.75 per share, generating gross proceeds of approximately $7.5 million[193]. - A subsequent offering on June 27, 2024, involved the sale of 527,918 registered shares and warrants, yielding gross proceeds of approximately $4.0 million at a purchase price of $5.4 per share[194]. - The Company issued a common warrant to purchase up to 2,762,766 shares of Common Stock on April 1, 2025, subject to stockholder approval[195]. - The exercise price for the Common Warrants from the July 2023 offering is set at $30.00 per share, expiring five years after issuance[193].