NurOwn's Regulatory Crossroads: Why BrainStorm Cell Therapeutics (BCLI) Could Be Poised for a Turnaround

The ALS community has long awaited a breakthrough in treating amyotrophic lateral sclerosis (ALS), a neurodegenerative disease with a median survival of just 2–3 years post-diagnosis. BrainStorm Cell Therapeutics (NASDAQ: BCLI), a small biotech with a novel stem cell therapy called NurOwn, now stands at a pivotal juncture. A citizen petition filed in July 2025, supported by compelling new survival and biomarker data, has reignited hopes for regulatory approval. If successful, this could unlock a near-term catalyst for BCLI's valuation, which has languished amid prior regulatory setbacks.

A Citizen Petition with Teeth
The petition, submitted by ALS patients and advocates, demands a reevaluation of NurOwn's data under the FDA's “totality of evidence” framework. It cites unprecedented outcomes from the Expanded Access Program (EAP) and Right to Try cases, including:
- Survival Data: EAP participants achieved a median tracheostomy-free survival of 6.8 years, far exceeding the 2.5–3-year median in natural history. Ten patients survived five years without tracheostomies, compared to the typical 10% survival rate at this benchmark.
- Biomarkers: NurOwn induced statistically significant changes in 23 CSF biomarkers, including neurofilament light (NfL), signaling reduced neurodegeneration.
- Functional Preservation: Subgroup analyses of early-stage ALS patients (ALSFRS-R ≥35) showed NurOwn recipients retained two more points on the functional scale than placebo groups after 24 weeks.

The petition argues NurOwn meets criteria for accelerated approval via biomarkers (e.g., NfL) and respiratory outcomes, or conditional approval under FDA Commissioner Makary's emphasis on “common sense” decisions for life-threatening diseases. It also requests use of the Commissioner's Priority Voucher to fast-track review.

Phase 3b: The Final Hurdle?
While the petition leans on real-world and subgroup data, BrainStorm's Phase 3b ENDURANCE trial is the linchpin for regulatory approval. Launched under a Special Protocol Assessment (SPA), the trial aims to enroll 200 early-stage ALS patients to validate NurOwn's efficacy. Key features include:
- A 24-week randomized, double-blind, placebo-controlled phase, followed by a 24-week open-label extension.
- Primary endpoint: Change in ALSFRS-R scores at week 24.
- A pre-negotiated design with the FDA, meaning positive results could directly support a Biologics License Application (BLA).

The Phase 3b trial's focus on early-stage patients aligns with NurOwn's subgroup efficacy data and the FDA's growing acceptance of surrogate endpoints for neurodegenerative diseases. If results mirror the EAP's survival and biomarker trends, NurOwn could become the first therapy to demonstrate meaningful functional preservation in ALS—a market with no curative options.

Why This Matters for Investors
The ALS market is underserved and growing, with 50,000+ patients in the U.S. alone. Even a conditional approval could generate immediate demand, especially if NurOwn's survival data holds up. The citizen petition's emphasis on biomarkers—validated in Phase 3b—could accelerate the process, potentially bypassing the need for a second confirmatory trial.

Risks remain:
- FDA Skepticism: The agency previously rejected BrainStorm's BLA in 2021, citing insufficient evidence.
- Financial Constraints: BCLI's market cap ($11M) and negative cash flow ($12M over 12 months) raise concerns about funding the Phase 3b trial.

However, the urgency of ALS patients and NurOwn's unmatched survival data create a compelling narrative. If the FDA grants accelerated approval, BCLI's valuation could surge, especially with a post-marketing Phase 4 study to gather long-term data.

Investment Thesis
- Catalysts: Positive Phase 3b data (potential 2026 readout), FDA feedback on the citizen petition, or a conditional approval decision.
- Upside: If NurOwn gains accelerated approval, BCLI's stock could triple from current levels, assuming a $150M–$200M valuation.
- Risk Management: Investors should monitor FDA communications and Phase 3b enrollment progress.

Final Take
BrainStorm's journey mirrors the high-risk, high-reward dynamics of biotech investing. NurOwn's data suggests it could redefine ALS treatment, but regulatory and financial hurdles loom large. For investors willing to bet on a transformative therapy for a desperate patient population, BCLI's near-term upside—driven by the citizen petition and Phase 3b—could outweigh the risks. The FDA's response to this petition, expected in late 2025, will be the first critical test.

Disclosure: This analysis is for informational purposes only. Consult a financial advisor before investing in BCLI.