Zacks Small Cap Analysis – BCLI: Making ready to Provoke Part 3b Trial of NurOwn™ Earlier than Finish of 2024… – Cyber Tech

By David Bautz, PhD

NASDAQ:BCLI

READ THE FULL BCLI RESEARCH REPORT

Enterprise Replace

Able to Transfer Forward with Part 3b Trial for NurOwn in ALS

On July 8, 2024, BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI) carried out a convention name to supply an replace on the corporate’s NurOwn™ program. The corporate is at present making ready for a Part 3b medical trial of NurOwn in sufferers with amyotrophic lateral sclerosis (ALS). We anticipate the trial initiating earlier than the top of 2024. Over the previous few months, BrainStorm has made appreciable progress in trial preparation, together with the next:

– In March 2024, the corporate offered the proposed design for the Part 3b trial in a poster on the MDA Scientific and Scientific Convention. The Part 3b trial can be a two-part, multicenter examine that may enroll ALS sufferers who’re earlier in the midst of their illness (onset of ALS signs, together with limb weak point, throughout the prior 24 months), with all ALSFRS-R objects ≥ 2 at screening, and upright gradual important capability ≥65% of predicted for gender, top, and age. Half A of the trial could have a double blind, placebo managed interval of 24 weeks. As much as roughly 200 sufferers are anticipated to be enrolled and randomized 1:1 to obtain NurOwn or placebo. There can be a screening interval of six to 9 weeks, throughout which period eligible members will bear a single bone marrow aspiration to obtain the mesenchymal stem cells (MSCs) to supply NurOwn at some point of the trial. Individuals will obtain both NurOwn or placebo through three repeated intrathecal injections as soon as each eight weeks. All members who full Half A can be eligible to enroll into Half B, an open label extension interval of 24 weeks the place all members will obtain three repeated injections of NurOwn as soon as each eight weeks. The first endpoint of the trial would be the change within the Revised Amyotrophic Lateral Sclerosis Practical Ranking (ALSFRS-R) whole rating from baseline to Week 24. Cerebrospinal fluid (CSF) and blood samples can be collected for evaluation of biomarkers of neuroinflammation, neurodegeneration, and neuroprotection. An unbiased Information Monitoring Committee can be established to observe the security of trial members.


– In April 2024, the corporate introduced it had acquired written settlement from the U.S. Meals and Drug Administration (FDA) beneath a Particular Protocol Evaluation (SPA) on the design of the Part 3b trial. The SPA settlement signifies that the FDA concurs with the adequacy and acceptability of particular parts of a protocols total design to make sure that the trial carried out beneath the protocol might be thought of an sufficient and well-controlled examine to assist advertising approval.


– In June 2024, the corporate introduced it reached alignment with the FDA on the Chemistry, Manufacturing, and Controls (CMC) side of the Part 3b trial. Which means that the corporate has resolved all earlier excellent CMC points that the FDA. Given the complexities of cell remedy merchandise, reaching alignment with the FDA forward of the Part 3b trial is a further means to decrease the regulatory threat for NurOwn.

New Biomarker Information Introduced at ALS Drug Growth Summit

In Could 2024, BrainStorm offered new biomarker information suggesting that ALS sufferers might profit from longer-term remedy with NurOwn. The information was offered on the third Annual ALS Drug Growth Summit and included consequence information from the Expanded Entry Program (EAP) together with information from the Part 3 trial.

The EAP was carried out over two intervals of 28 weeks every, throughout which members acquired three doses of NurOwn every interval for a complete of six doses. Everybody within the EAP acquired NurOwn, even those who acquired placebo throughout the Part 3 trial. 13 cerebrospinal fluid (CSF) samples have been drawn throughout the Part 3 trial and the EAP and ranges of neurofilament gentle (NfL) have been monitored. Ten trial members who accomplished the Part 3 trial enrolled within the EAP. Eight members accomplished EAP Interval 1 and 6 accomplished EAP Interval 2. The members within the EAP had decrease NfL values at Part 3 baseline as in comparison with the whole Part 3 inhabitants.

The information from the EAP confirmed that for members randomized to NurOwn there was a 4% lower from baseline in NfL in Part 3 and a 27% and 36% lower from baseline on the finish of EAP Interval 1 and Interval 2, respectively. For sufferers randomized to placebo within the Part 3 trial and subsequently handled with NurOwn within the EAP, there was a 37% improve in NfL from baseline to the top of the Part 3 examine and following remedy with NurOwn within the EAP the identical sufferers had a 17% improve in NfL from baseline throughout Interval 1 and a 5% lower from baseline in NfL throughout Interval 2. These outcomes counsel that NurOwn is contributing to a lower in NfL, with current printed research displaying an affiliation between NfL ranges and medical development.

New COO Additional Reduces CMC Threat

In June 2024, BrainStorm introduced the appointment of Hartoun Hartounian, PhD as its new Government Vice President and Chief Working Officer. Dr. Hartounian has over 30 years of expertise within the biopharmaceutical business, with a concentrate on cell and gene remedy. He based BioCentriq, a CDMO firm for cell and gene remedy that centered on all phases of course of growth and medical manufacturing, the place he served as Common Supervisor from 2015 to 2022 after which as its President, CEO, and a member of the Board of Administrators earlier than it was acquired for $73 million by GC Holdings (Korea). Previous to that, Dr. Hartounian was CEO, Co-Founder, and a member of the Board of Administrators at DiaVacs, an organization specializing in cell remedy for kind 1 diabetics. We imagine Dr. HartounNNian’s huge expertise and experience in cell remedy can be crucial for BrainStorm and additional decreases the CMC threat as the corporate advances towards the upcoming Part 3b trial of NurOwn and its potential commercialization.

Monetary Replace

In Could 2024, BrainStorm introduced monetary outcomes for the primary quarter of 2024. As anticipated, the corporate didn’t report any revenues throughout the first quarter of 2024. Internet R&D bills for the primary quarter of 2024 have been $1.0 million, in comparison with $2.9 million throughout the first quarter of 2023. The lower was primarily because of decreased prices associated to the Part 3 medical trial and payroll bills. G&A bills for the primary quarter of 2024 have been $1.5 million in comparison with $2.3 million for the primary quarter of 2023. The lower was primarily because of decrease payroll, lease, and different prices.

The corporate exited the primary quarter of 2024 with roughly $1.0 million in money, money equivalents, and short-term deposits. In June 2024, BrainStorm introduced a $4.0 million registered direct providing by the sale of roughly 11.1 million shares of frequent inventory at a purchase order worth of $0.36 per share together with roughly 16.7 million warrants with an train worth of $0.3912 per share. We estimate that BrainStorm at present has roughly 81.2 million frequent shares excellent and, when factoring in inventory choices and warrants, a totally diluted share depend of roughly 103.7 million.

Conclusion

We’re happy to see the progress that BrainStorm has remodeled the previous few months as the corporate prepares for the Part 3b trial of NurOwn in ALS sufferers. At this level the corporate simply must acquire the financing needed to finish the trial, with the current $4.0 million increase a step in the proper route. We had already factored within the anticipated prices of the Part 3 examine into our mannequin, nonetheless with market situations not bettering we now have made a slight improve into the anticipated dilution for the following capital increase, which has decreased our valuation barely to $2.40 per share.

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