Brainstorm Cell Therapeutics(US:BCLI)2024-08-14 12:01Clinical Trials and Regulatory Status - NurOwn® has completed Phase 3 ALS and Phase 2 PMS clinical trials, with positive top-line data announced on March 24, 2021, for PMS[95]. - The FDA granted a Special Protocol Assessment (SPA) agreement for the Phase 3b trial of NurOwn® in ALS on April 9, 2024, validating the clinical trial protocol[101]. - 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[95]. - The BLA for NurOwn® was withdrawn on November 3, 2023, in coordination with the FDA, viewed as a withdrawal without prejudice[95]. - The Phase 3 clinical trial of NurOwn® did not achieve statistically significant results, with 32.6% of participants meeting the primary endpoint compared to 27.7% for placebo (p=0.453)[125]. - In a pre-specified subgroup with ALSFRS-R baseline scores of 35 or greater, NurOwn® showed a 34.6% responder rate versus 15.6% for placebo (p=0.305) and an average change of -1.56 on NurOwn® compared to -3.65 on placebo (p=0.050)[126]. - The Phase 2 trial demonstrated a slower rate of disease progression in ALS patients, with significant improvements in ALSFRS-R and FVC[116]. - NurOwn® treatment resulted in statistically significant increases in neurotrophic factors and reductions in inflammatory markers in cerebrospinal fluid samples[120]. - The Phase 3 trial enrolled a broad set of participants, including those with advanced ALS, which may have impacted the sensitivity of the ALSFRS-R[129]. - The company is actively exploring next steps for NurOwn®, including publication of emerging clinical data and development of a protocol for an additional clinical study[95]. - The FDA invited the company for an expedited meeting to discuss the regulatory path forward for NurOwn® in ALS on October 18, 2023[112]. - The company plans to submit a SPA request for a Phase 3b clinical trial for NurOwn® to discuss protocol design with the FDA[112]. - A meeting with the FDA on December 6, 2023, focused on discussing the regulatory path forward for NurOwn® and plans for a Special Protocol Assessment (SPA) for a Phase 3b trial[134]. - The company received written agreement from the FDA on April 9, 2024, under a SPA for the design of the Phase 3b trial of NurOwn® in ALS, validating the clinical trial protocol[134]. Financial Performance and Funding - The company incurred operating costs of approximately $2,982,000 for the three months ended June 30, 2024, a decrease of 45.1% compared to $5,449,000 for the same period in 2023[185]. - Research and development expenditures, net for the three months ended June 30, 2024 were $922,000, a decrease of 67.1% compared to $2,794,000 for the same period in 2023[187]. - The net loss for the three months ended June 30, 2024 was $2,541,000, compared to a net loss of $5,329,000 for the same period in 2023, representing a reduction of 52.3%[191]. - The company had cash, cash equivalents, and restricted cash amounting to $3,648,000 as of June 30, 2024[197]. - Net cash used in operating activities for the six months ended June 30, 2024 was $4,746,000, primarily attributed to clinical trial costs and payroll expenses[197]. - The company received $6,909,000 from financing activities for the six months ended June 30, 2024, primarily from sales of common stock under the ATM program[197]. - The company anticipates needing to raise substantial additional financing in the future to fund operations, including potential debt and equity offerings[210]. - The Company sold an aggregate of 2,446,641 shares of Common Stock at an average price of $9.45 per share, raising gross proceeds of approximately $23.11 million under the March 6, 2020 ATM[201]. - The Company entered into a Securities Purchase Agreement on July 17, 2023, agreeing to sell 4,054,055 shares of Common Stock at a purchase price of $1.85 per share, yielding gross proceeds of approximately $7.5 million[205]. - On June 27, 2024, the Company agreed to sell 7,918,764 registered shares of Common Stock and pre-funded warrants, yielding gross proceeds of approximately $4.0 million[207]. Legal and Compliance Issues - The company is involved in ongoing litigation, including a putative securities class action complaint alleging violations related to NurOwn® for the treatment of ALS[220]. - The company is facing multiple derivative action complaints alleging breach of fiduciary duty and unjust enrichment against certain officers and directors, with claims related to the internal controls and FDA submissions for NurOwn® for ALS treatment[221]. - The company received a letter from Nasdaq on November 1, 2023, indicating non-compliance with the $1.00 minimum bid price requirement, with a compliance period until April 29, 2024[226]. - Nasdaq determined to delist the company's common stock on May 1, 2024, due to continued non-compliance with the bid price requirement[226]. - The company submitted a hearing request to Nasdaq on May 2, 2024, and was granted a temporary exception to regain compliance by October 21, 2024[227]. - The company received a notice on July 18, 2024, indicating it did not maintain the minimum market value of listed securities of $35 million, with a compliance period until January 14, 2025[228]. - Failure to regain compliance with either the bid price or market value requirements could lead to delisting from Nasdaq, negatively impacting trading and liquidity[229]. - The company is subject to securities class action litigation and other stockholder litigation, which could result in substantial costs and divert management's attention[230]. Company Operations and Workforce - The company currently employs 29 employees in the United States and Israel, with a focus on recruiting and retaining talent to support its objectives[99]. - The company has a GMP manufacturing center in Tel Aviv, enhancing its capacity to manufacture and ship NurOwn® into the EU and local Israeli markets[99]. - The company has developed a validated cryopreservation process for MSC, allowing multiple doses of NurOwn® from a single bone marrow harvest, enhancing manufacturing efficiency[135]. - As of November 1, 2023, the company optimized its manufacturing capabilities by leasing a GMP-certified cleanroom manufacturing center, significantly enhancing production capacity for NurOwn®[138]. - The company has improved the efficiency of NurOwn® production and its stability, allowing centralized manufacturing and distribution to clinical trial sites[183]. Research and Development - The company presented new biomarker data suggesting that ALS patients may benefit from longer-term treatment with NurOwn® at the 3rd Annual ALS Drug Development Summit in May 2024[103]. - The NurOwn® technology has received Fast Track designation and Orphan Drug status from the FDA and EMA for ALS, indicating its potential significance in treating this condition[98]. - The FDA granted Fast Track designation and Orphan Drug Status for NurOwn® in ALS, providing potential for extended exclusivity[112]. - The company is considering advancing NurOwn® as a treatment option for Alzheimer's Disease, focusing on its capability to target multiple biological pathways[161]. - The company received a total of $15,912,390 in grant funding from the California Institute for Regenerative Medicine (CIRM) for the Phase 3 study of NurOwn® for ALS[162]. - The company holds 27 granted patents and 23 patent applications worldwide, protecting its proprietary technologies and products[164]. - The company announced the European grant for NurOwn® and the acceptance of applications for exosomes derived from MSC-NTF cells in Australia and Israel[171]. - The Phase 2 PMS clinical trial compared clinical efficacy outcomes with a matched clinical cohort from the CLIMB Study, demonstrating significant improvements in various measures of function and cognition[156]. - In the Phase 2 study for Progressive Multiple Sclerosis (PMS), 80% of the 20 enrolled patients completed the study, with no deaths or serious adverse events reported[155]. - 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[158].