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BCLI: Initiation - Potential 10x Return on Successful Phase 3b. Promising Post Hoc and Biomarker Analysis Underpins Our NurOwn® Clinical Effectiveness Expectation

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BrainStorm Cell Therapeutics (NASDAQ: BCLI) is advancing its autologous mesenchymal stem cell (MSC) therapy, NurOwn® (debamestrocel, MSC-NTF), targeting neurodegenerative diseases, primarily ALS. Post hoc analysis of Phase III data reveals significant clinical benefits for early-stage ALS patients, prompting the FDA to back a new Phase IIIb trial under an SPA agreement. The Phase IIIb trial aims to enroll ALS patients with ALSFRS-R scores ≥35, focusing on slowing disease progression. Peer-reviewed studies also highlight NurOwn's positive impact on ALS biomarkers, supporting its potential efficacy. BCLI has met the NASDAQ minimum value compliance and anticipates strong valuation growth if the Phase IIIb trial succeeds.

Positive
  • Statistically significant clinical effects in early-stage ALS patients from Phase III trial data.
  • FDA's commitment to a new Phase IIIb trial under an SPA agreement.
  • Positive impact on ALS biomarkers (NfL, LAP, Galectin-1) found in peer-reviewed research.
  • Regained NASDAQ minimum value compliance.
  • Potential for strong valuation growth if Phase IIIb trial is successful.
Negative
  • Phase III trial results clouded by 23% of participants with advanced ALS, affecting statistical analysis.
  • Primary and secondary endpoints of the Phase III trial impacted by possible floor effects.
  • High reliance on the successful execution of the new Phase IIIb trial for positive valuation inflection.
  • Current market valuation only considers the smaller US market, excluding potential EU/UK market impact.

BrainStorm Cell Therapeutics' new data from their Phase 3b trial for NurOwn® shows promising results for early-stage ALS patients, addressing a significant unmet need in neurodegenerative disease treatment. ALS, often fatal within 2 to 5 years, has limited treatment options, making any progress noteworthy.

Notably, the post hoc analysis revealed that NurOwn® positively impacts biomarkers such as NfL, LAP and Galectin-1, which correlate with slower disease progression. This kind of biomarker data is important because it provides a measurable indicator of the drug's effectiveness, potentially accelerating FDA approval processes if these findings are consistently replicated.

The focus on early-stage ALS patients in the new PIIIb trial, excluding those with more advanced symptoms, increases the likelihood of clearer, more positive results. This change in trial design is a strategic move to avoid statistical noise and floor effects observed in the prior trial.

From an investor perspective, these results are a positive development, indicating that NurOwn® could meet a critical need in ALS treatment and potentially garner significant market share, especially if the new trial meets its endpoints.

BrainStorm Cell Therapeutics' NurOwn® targeting ALS shows signs of becoming a significant revenue driver if clinical success continues. The company regaining compliance with NASDAQ's regulatory minimum value standards is another positive sign, indicating stable financial health amidst clinical developments.

Key to note is the valuation excluding the EU/UK markets, which suggests substantial upside potential if the therapy demonstrates efficacy. The focus on the US market alone may undervalue the full potential of NurOwn® on a global scale. This conservative approach provides a margin of safety for investors.

Positive clinical outcomes in the PIIIb trial could lead to lucrative partnerships or acquisition interest from larger pharmaceutical companies looking to bolster their neurodegenerative disease pipeline, further enhancing shareholder value.

In the short term, investors should monitor the trial's progress and FDA's ongoing commitment. Long-term success will depend on market penetration and acceptance, both of which look promising given the high unmet need in ALS treatment.

FROM ACF EQUITY RESEARCH HEALTHCARE TEAM

NASDAQ: BCLI

READ ACF EQUITY RESEARCH'S INITIATION NOTE HERE 

READ ACF EQUITY RESEARCH'S RELATED THEMATIC RESEARCH NOTE ON REGENERATIVE MEDICINE – NEURODEGENERATIVE DISEASE STEM CELLS & ALS/MND/LOU GEHRIG'S HERE

LONDON, June 14, 2024 /PRNewswire/ -- 

INITIATION NOTE: BrainStorm Cell Therapeutics Inc. (Nasdaq: BCLI) is focused on developing autologous mesenchymal stem cell (MSC) therapies for the treatment of neurodegenerative diseases (NDDs) – BCLI's primary target is the fatal amyotrophic lateral sclerosis (ALS/MND/Lou Gehrig's). Post hoc analysis of BCLI's PIII trial data shows BCLI's NurOwn® (debamestrocel, MSC-NTF), has statistically significant clinical effects on early-stage ALS sufferers and that placebo trialists deteriorate faster. Peer reviewed research (Mar 2024) indicated that BCLI's NurOwn® has a positive impact on NfL biomarkers for ALS. These and other factors have persuaded the FDA to sign up to the SPA binding commitment for a NurOwn® PIIIb ALS trial. BCLI has regained regulatory minimum value NASDAQ compliance. Our valuation captures only the smaller by patients US market and excludes the larger EU/UK market.

Post hoc analysis of BCLI's PIII early stage (mild-moderate) ALS sufferers (baseline ALSFRS-R scores >=35)  treated with BCLI's NurOwn® (debamestrocel, MSC-NTF) revealed positive clinical responses with respect to slowing of ALS disease progression (primary endpoint). However the PIII trial cohort consisted (unexpectedly) of 23% advanced ALS sufferers, clouding the primary and secondary end point statistical analysis (possible floor effects). The new PIIIb trial is designed to recruit a cohort of participants with ALSFRS-R scores >=35. Additionally, more recent peer reviewed research found that certain biomarkers involved in ALS pathology, specifically NfL, LAP and Galectin-1 were found to be predictive of positive clinical outcomes in NurOwn® (debamestrocel, MSC-NTF),-treated participants. If the new trial design is successfully executed, as we forecast, we expect a strongly positive valuation inflection point for BCLI.

ALS: High Unmet Medical Need Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder (NDD) that affects motor nerve cells in the brain and the spinal cord. There are an estimated ~450k ALS patients worldwide (30k US and 51k European). Median survival post diagnosis is 2 to 5 years. Current treatment options have very limited efficacy. Tofersen addresses just 2% of ALS sufferers. There is a high unmet need to slow/reverse ALS progression.

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DISCLOSURES: ACF Equity Research has received cleared funds in advance for a minimum one year service directly from the issuer, from an exchange, from a portfolio manager; from an investor group or and from an investor relations firm. ACF Equity Research has received for any of investment/equity/credit/ESG/sustainability research a fixed paid in full in advance annual fee in the range US$35,000 to US$75,000 service package dependent.

Cision View original content:https://www.prnewswire.com/news-releases/bcli-initiation---potential-10x-return-on-successful-phase-3b-promising-post-hoc-and-biomarker-analysis-underpins-our-nurown-clinical-effectiveness-expectation-302173201.html

SOURCE ACF Equity Research

FAQ

What is the focus of BrainStorm Cell Therapeutics?

BrainStorm Cell Therapeutics focuses on developing autologous mesenchymal stem cell (MSC) therapies for neurodegenerative diseases, primarily ALS.

What are the significant findings from BCLI's Phase III trial?

The Phase III trial showed statistically significant clinical effects on early-stage ALS patients and highlighted the positive impact on ALS biomarkers.

What does the new Phase IIIb trial aim to achieve?

The new Phase IIIb trial aims to enroll ALS patients with ALSFRS-R scores ≥35 and focuses on slowing disease progression.

What is the impact of NurOwn® on ALS biomarkers?

Peer-reviewed research indicates that NurOwn® has a positive impact on ALS biomarkers such as NfL, LAP, and Galectin-1.

Has BCLI met NASDAQ compliance requirements?

Yes, BCLI has met the NASDAQ minimum value compliance requirements.

What is the potential market impact of a successful Phase IIIb trial for BCLI?

A successful Phase IIIb trial is expected to create a strong valuation inflection point, significantly boosting BCLI's market value.

How does the Phase III trial cohort composition affect the results?

The Phase III trial cohort's inclusion of 23% advanced ALS sufferers clouded the primary and secondary endpoint statistical analysis, potentially due to floor effects.

Which geographical markets does the current valuation of BCLI exclude?

The current valuation of BCLI excludes the larger EU/UK market, focusing only on the US market.

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