BCLI: Positive Proof-of-Concept Data for NurOwn in Progressive Multiple Sclerosis…

By David Bautz, PhD

NASDAQ:BCLI

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Positive Proof-of-Concept Data for NurOwn in Progressive MS

On March 24, 2021, BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI) announced positive proof-of-concept data for NurOwn in patients with progressive multiple sclerosis (MS). The trial achieved the primary endpoint of safety and encouraging results were seen in multiple secondary, functional endpoints.

The Phase 2 trial was a multicenter, open label trial that enrolled 20 patients, with 18 treated and 16 completing the study. Two of the enrolled patients decided against continuing in the trial before dosing began, with one patient withdrawing following dosing due to non-specific symptoms and another due to back/leg pain. Following a 10-week run-in period, patients were dosed with 1-1.25 x 106 MSC-NTF cells three times at 2-month intervals. Patients were followed for 28 weeks following the first treatment. The secondary functional endpoints examined included:

• Timed 25-Foot Walking Speed (T25FW): Scoring is the average of two trials. A patient is directed to walk 25 feet quickly and as safely as possible from one marked end to the other. The patient then walks back the same distance.

• 9-Hole Peg Test (9-HPT): This test is administered by patients taking pegs from a container, one by one, and placing them in holes as quickly as possible. The patient then removes the pegs one by one and puts them back in the container. Both dominant and non-dominant hands are tested.

• Low Contrast Letter Acuity (LCLA): This is similar to a standard eye chart test but instead of black letters on a white background (100% contrast), low contrast levels are used and the number of letters identified is scored.

• Symbol Digit Modality Test (SDMT): Using a reference key, a test subject has 90 seconds to pair specific numbers with given geometric figures. It is a simple substitution task that normal adults can easily perform and can be utilized to detect changes in cognitive functioning over time.

• 12-item Multiple Sclerosis Walking Scale (MSWS-12): This is a self-reported measure of the impact of MS on a patient’s walking ability.

Baseline characteristics for study participants showed that the mean age was 47, 56% were female, and the mean baseline Expanded Disability Status Scale (EDSS) score was 5.4. The results of the trial were compared to a matched clinical cohort of 48 patients from the Comprehensive Longitudinal Investigations in MS at the Brigham & Woman’s Hospital (CLIMB Study) (Gauthier et al., 2006).

Highlights from the topline results of secondary functional outcomes include:

• 14% and 13% of NurOwn-treated patients showed a 25% improvement in T25FW and 9-HPT, respectively, compared to 0% from the matched cohort from the CLIMB registry.

• 38% of NurOwn-treated patients showed a ≥10-point improvement in the MSWS-12 (CLIMB cohort not analyzed for this outcome).

• 47% of NurOwn-treated patients showed a ≥8-letter improvement in the LCLA (CLIMB cohort not analyzed for this outcome).

• 67% of NurOwn-treated patients showed a ≥3-point improvement in the SDMT (BrainStorm is waiting on the results for SDMT for the CLIMB cohort).

• On average, there was a 10% improvement in T25FW and a 4.8% improvement on the 9-HPT for NurOwn-treated patients, compared to a 1.8% and 1.4% worsening, respectively, in the matched control group from the CLIMB registry.

Previous trials of MS drugs have noted an association between loss of vision, as measured by the LCLA, and diminished quality of life (Chahin et al., 2015), thus the improvements in vision seen in almost half the patients treated with NurOwn are particularly noteworthy.

We anticipate the company presenting the full data set at an upcoming scientific conference and publishing the results in a peer reviewed journal article later this year. Additional analyses that the company has not yet completed include detailed cerebrospinal fluid (CSF) and blood biomarker analyses along with MRI analyses (brain lesions, brain volume, etc.).

Conclusion

We are excited by the proof-of-concept results announced by BrainStorm regarding the use of NurOwn in treating progressive MS. Particularly intriguing are the improvements in vision and cognition, as shown through the LCLA and SDMT tests, since those improvements are less likely to be subject to other factors (i.e., you can see the letters or you can’t) and are correlated with an improved quality of life. While these results are encouraging, our enthusiasm is tempered somewhat since these are only preliminary proof-of-concept data from an open label trial. The hurdle of proof-of-concept has been cleared, however the company will need to perform additional double blind, placebo controlled efficacy trial(s) to prove that NurOwn is effective in treating progressive MS patients. Based on these results we have slightly increased the probability of approval for NurOwn in progressive MS to 25%, which has raised the valuation to $11 per share.

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