BrainStorm Cell Therapeutics Inc (BCLI)

[Fmello]

AMLX stock is in red territory today on yesterday's news. I would have expected it to shoot up like a rocket. I assumed that the positive AMLX news would provide a halo effect for other companies like Brainstorm (which is currently up 6.71% so far today)...so weird.

[midastouch017]

See #msg-170077027 and thread.
To your question, i forsee a slight
positive reaction in the range of +5%

[Fmello]

Do you think there will be a halo effect with Brainstorm stock tomorrow due to today's AMLX's FDA approval announcement?

[bubbarayjr]

My guess is the BLA was filed around the first of September. If I'm correct we'll hear from the FDA by the end of October. FDA acceptance and Priority Review are a slam dunk. It would be surprising for the FDA not to require an Advisory Panel meeting however given the fact that NurOwn provides STATISTICALLY SIGNIFICANT meaningful outcomes as measured by the ALSFRS-R scale in a very important subset of patients (those with early less severe disease) pre-defined in the PIII study, it's not outside the realm of possibilities. BCLI has been able to link positive outcomes with their critical BioMarker data to reinforce NurOwn's mechanism of action which points directly to why it works best in those with early, less severe disease. All IMO

[Fmello]

- I just saw this on the No More Excuses ALS Watchdog Group Facebook page:

NUROWN Timeline = Later in 2023

Brainstorm announced it will submit a BLA to FDA's CBER

No info if Brainstorm has yet submitted the BLA

Once submitted, CBER has 60 days to decide if to accept Brainstorm's BLA for review

Brainstorm said it will only make a public announcement after FDA decides if to accept the BLA

If accepted, CBER will decide if to grant Priority Review (up to 6 months) or regular review timeline (up to 10 months).

CBER will decide if to grant an AdCom & will set a date

No PDUFA decision date but no BLA decision is possible for NurOwn until well into 2023


If Brainstorm submits the BLA before Halloween ...
And if the FDA grants Priority Review...
And if the FDA approves NurOwn...
The earliest time that NurOwn could be approved would be around June sometime.

[Fmello]

.....

[Fmello]

NurOwn Found Safe, Shows Promise in Phase 2 Trial for Progressive MS

https://multiplesclerosisnewstoday.com/news-posts/2022/09/16/nurown-safe-shows-phase-2-trial-promise-progressive-ms/

[Fmello]

BrainStorm Brings MS Data on Heels of ALS BLA Announcement (Updated)

https://www.biospace.com/article/brainstorm-brings-ms-data-on-heels-of-als-bla-submission-announcement-/

The study compared these 20 patients to 48 matched controls from the Comprehensive Longitudinal Investigation of Multiple Sclerosis (CLIMB) registry of Brigham and Women's Hospital and the Ann Romney Center for Neurologic Diseases. All comparison metrics fell strongly in NurOwn’s favor as the study met all endpoints.

NurOwn demonstrated “large, clinically meaningful improvements in some patients,” across measurements in all endpoints measured, BrainStorm reported.

In the first, 19% of participants treated with NurOwn responded with a 25% or greater improvement in the timed 25-foot walk speed (T25FW) or the 9-hole peg test (9HPT) at 28 weeks compared to baseline. This contrasted with 4% of the matched CLIMB controls.

In another endpoint, 67% of NurOwn-treated patients showed a 3-point or greater improvement in the symbol digit modality test (SDMT) compared to 18% of matched CLIMB registry patients.

Aside from the CLIMB registry, 38% of participants treated with NurOwn showed an improvement greater than or equal to 10-points from baseline in the multiple sclerosis walking scale (MSWS).

The company will also present additional data at the 38th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) conference in October.

[Fmello]

Maxim Group analyst Jason McCarthy maintained a Buy rating on Brainstorm Cell Therapeutics (BCLI – Research Report) yesterday and set a price target of $8.00.

[midastouch017]

BrainStorm Cell Therapeutics Announces Peer Reviewed Publication of Results from the NurOwn® Phase 2 Progressive MS Trial in Multiple Sclerosis Journal

https://finance.yahoo.com/news/brainstorm-cell-therapeutics-announces-peer-100000035.html

Data demonstrate NurOwn's safety and provide preliminary evidence of efficacy in patients with progressive multiple sclerosis

Biomarker analyses show consistent treatment effects in neuroinflammation and neuroprotection pathways

NEW YORK, Sept. 15, 2022 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, today announced the peer reviewed publication of data from the Phase 2 trial of NurOwn in progressive multiple sclerosis (MS) in Multiple Sclerosis Journal. The publication, entitled "Evaluation of neurotrophic factor secreting mesenchymal stem cells in progressive multiple sclerosis", can be found here.

Results from the Phase 2, single-arm, open-label study demonstrated NurOwn's safety and provided preliminary evidence of its efficacy in people with progressive MS. Additionally, biomarker analyses confirmed NurOwn's proposed mechanism of action by showing consistent treatment effects in neuroinflammation and neuroprotection pathways.

Twenty participants were enrolled into the Phase 2 trial, with seventeen receiving all three scheduled NurOwn treatments. The mean age of study participants was 47 years with a mean expanded disability status scale (EDSS) score of 5.4 at screening. Results from the trial were compared to 48 matched control patients who were selected from the from the Comprehensive Longitudinal Investigation of Multiple Sclerosis (CLIMB) registry (Brigham and Women's Hospital and the Ann Romney Center for Neurologic Diseases) at the beginning of the trial.

Treatment with NurOwn resulted in large, clinically meaningful improvements in some patients, as defined by response criteria, across all endpoints measured. These endpoints included timed 25-foot walk speed (T25FW), 9-hole peg test (9HPT), multiple sclerosis walking scale (MSWS), symbol digit modality test (SDMT), and low contrast letter acuity (LCLA). These observed improvements diverged from what was seen in matched patients with progressive MS from the CLIMB registry. Key data from the trial, as well as relevant comparisons to the matched CLIMB registry patients, are shown below.

19% of treated trial participants were responders (as defined by prespecified ≥25% improvement in T25FW or 9HPT at 28 weeks compared to baseline), compared to 4% of the matched CLIMB registry patients.

38% of treated trial participants showed a ≥10-point improvement from baseline in the 12-item MSWS (data for this endpoint were not collected by the CLIMB registry).

27% of treated trial participants showed a ≥8-letter improvement in LCLA binocular at a 2.5% contrast threshold, compared to 6% of the matched CLIMB registry patients.

67% of treated trial participants showed a ≥3-point improvement in the SDMT, compared to 18% of the matched CLIMB registry patients.

47% of treated trial participants showed a ≥8-point improvement in LCLA binocular at a 1.25% contrast threshold (data for this endpoint were not collected by the CLIMB registry).

Across all participants, improvements in function as measured by LCLA, SDMT and MS Functional Composite (MSFC) were observed. Mean improvements from baseline of 3.3 points in the LCLA binocular (2.5% contrast), 3.8 points on the SDMT, and 0.18 points in MSFC were observed in treated trial participants. The corresponding changes in matched CLIMB registry patients estimated at 28 weeks showed declines in function on the LCLA and MSFC. The average change in function decline as measured by T25FW, 9HPT, and EDSS across all treated trial participants demonstrated stabilization of functional decline, with similar or slightly worse findings observed in the matched CLIMB registry patients for the same endpoints.

There were no adverse events related to worsening of MS disease and no clinically significant changes in safety lab results/vital signs, confirming NurOwn's favorable safety profile. Two patients developed symptoms of low back and leg pain, consistent with arachnoiditis, occurring in one of three treatments in both participants.

Treatment also consistently resulted in increases in cerebrospinal fluid neuroprotective factors (VEGF–A, HGF, NCAM-1, Follistatin, LIF and Fetuin–A) and reductions in inflammatory biomarkers (MCP-1, sCD27, SDF-1, and Osteopontin), confirming NurOwn's proposed mechanism of action in progressive MS.

"We were pleased that the study's initial results showed efficacy in patients with progressive MS," said Jeffrey Cohen, M.D., Hazel Prior Hostetler Endowed Chair Professor, Cleveland Clinic Lerner College of Medicine, Director, Experimental Therapeutics, Mellen Center for MS Treatment and Research, and the paper's lead author. "There are both promising biological and preliminary clinical signals of a treatment effect that will require confirmation in a randomized trial."

"There is a high unmet need for better treatments for progressive forms of MS and we congratulate the Brainstorm Cell Therapeutics team for the successful completion and publication of this important study. We look forward to future studies that will help to fully understand the potential of NurOwn and other cell-based therapies for this hard-to-treat form of disease" said Bruce Bebo, EVP Research National MS Society.

Chaim Lebovits, Chief Executive Officer, BrainStorm Cell Therapeutics stated, "Having these data peer reviewed and published in the prestigious Multiple Sclerosis Journal is an important step in the evaluation of NurOwn in progressive MS. We appreciate the expertise and commitment of the study investigators and contributions of study participants to advance our understanding of NurOwn's cellular technology platform. Thanks to their efforts and those of the BrainStorm team, we believe we are closer to providing a meaningful treatment option for those with progressive MS".

Ralph Kern, M.D., MHSc, President and Chief Medical Officer of BrainStorm Cell Therapeutics and co-author of the paper commented, "This publication provides preliminary evidence of NurOwn's potential to modify functional outcomes in progressive MS, which we believe warrants further study. In addition, consistent changes in cerebrospinal fluid neuroinflammation and neuroprotection biomarkers reveal how NurOwn may impact disease mechanisms in progressive MS and are complementary to biomarker results observed in our Phase 3 ALS trial. These observations provide further support for NurOwn as a platform technology with potential broad applications and will bolster BrainStorm's efforts to bring much needed solutions to patients with progressive MS, ALS, and other neurodegenerative diseases."

Study Design

The Phase 2 study (BCT-101) was designed to evaluate the safety, efficacy, and biomarker effects of three intrathecal administrations of NurOwn (MSC-NTF cells), given at two-month intervals, to adults with progressive MS. The trial was conducted at four MS centers of excellence: Cleveland Clinic Mellen Center for MS, Icahn School of Medicine at Mount Sinai, Keck School of Medicine of the University of Southern California, and Stanford University School of Medicine. Twenty participants ages 18-65 with progressive MS were enrolled and 17 received all three treatments and were followed for up to 28 weeks. Participants had baseline EDSS scores of between 3.0 and 6.5, were able to walk 25 feet in 60 seconds or less and had not experienced an MS relapse in the 6 months prior to study enrollment.

The primary efficacy outcome was pre-specified improvement (≥25%) in T25FW or 9-HPT. Additional efficacy endpoints included pre-specified improvements in EDSS, SDMT, LCLA, and MSWS-12. The efficacy outcomes were compared to a pre-specified matched group of progressive MS patients from CLIMB registry (n=48) (Tanuja Chitnis, M.D. Brigham and Women's Hospital and the Ann Romney Center for Neurologic Diseases). The study was sponsored by Brainstorm Cell Therapeutics with additional financial support for biomarker analyses received from the National Multiple Sclerosis Society, Fast-Forward Commercial Research Funding Program. For more information on the trial, visit https://clinicaltrials.gov/ct2/show/NCT03799718.

About NurOwn®

The NurOwn® technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn® technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 pivotal trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive MS and was supported by a grant from the National MS Society (NMSS).

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may," "should," "would," "could," "will," "expect," "likely," "believe," "plan," "estimate," "predict," "potential," and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of BrainStorm's NurOwn® treatment candidate, the success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our products and services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwn® treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture and commercialize the NurOwn® treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation; the impacts of the COVID-19 pandemic on our clinical trials, supply chain, and operations; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations, and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements.

CONTACTS

Investor Relations:
John Mullaly
LifeSci Advisors, LLC
Phone: [phone]
[e-mail]

Media:
Lisa Guiterman
[e-mail]

Logo - https://mma.prnewswire.com/media/1166536/BrainStorm_Logo.jpg

Cision
Cision
View original content:https://www.prnewswire.com/news-releases/brainstorm-cell-therapeutics-announces-peer-reviewed-publication-of-results-from-the-nurown-phase-2-progressive-ms-trial-in-multiple-sclerosis-journal-301625167.html

SOURCE BrainStorm Cell Therapeutics Inc.

[Fmello]

BrainStorm Cell Therapeutics Announces Peer Reviewed Publication of Results from the NurOwn® Phase 2 Progressive MS Trial in Multiple Sclerosis Journal

https://www.prnewswire.com/news-releases/brainstorm-cell-therapeutics-announces-peer-reviewed-publication-of-results-from-the-nurown-phase-2-progressive-ms-trial-in-multiple-sclerosis-journal-301625167.html

Data demonstrate NurOwn's safety and provide preliminary evidence of efficacy in patients with progressive multiple sclerosis

Biomarker analyses show consistent treatment effects in neuroinflammation and neuroprotection pathways

NEW YORK, Sept. 15, 2022 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, today announced the peer reviewed publication of data from the Phase 2 trial of NurOwn in progressive multiple sclerosis (MS) in Multiple Sclerosis Journal. The publication, entitled "Evaluation of neurotrophic factor secreting mesenchymal stem cells in progressive multiple sclerosis", can be found here.

Results from the Phase 2, single-arm, open-label study demonstrated NurOwn's safety and provided preliminary evidence of its efficacy in people with progressive MS. Additionally, biomarker analyses confirmed NurOwn's proposed mechanism of action by showing consistent treatment effects in neuroinflammation and neuroprotection pathways.

Twenty participants were enrolled into the Phase 2 trial, with seventeen receiving all three scheduled NurOwn treatments. The mean age of study participants was 47 years with a mean expanded disability status scale (EDSS) score of 5.4 at screening. Results from the trial were compared to 48 matched control patients who were selected from the from the Comprehensive Longitudinal Investigation of Multiple Sclerosis (CLIMB) registry (Brigham and Women's Hospital and the Ann Romney Center for Neurologic Diseases) at the beginning of the trial.

Treatment with NurOwn resulted in large, clinically meaningful improvements in some patients, as defined by response criteria, across all endpoints measured. These endpoints included timed 25-foot walk speed (T25FW), 9-hole peg test (9HPT), multiple sclerosis walking scale (MSWS), symbol digit modality test (SDMT), and low contrast letter acuity (LCLA). These observed improvements diverged from what was seen in matched patients with progressive MS from the CLIMB registry. Key data from the trial, as well as relevant comparisons to the matched CLIMB registry patients, are shown below.

19% of treated trial participants were responders (as defined by prespecified ≥25% improvement in T25FW or 9HPT at 28 weeks compared to baseline), compared to 4% of the matched CLIMB registry patients.
38% of treated trial participants showed a ≥10-point improvement from baseline in the 12-item MSWS (data for this endpoint were not collected by the CLIMB registry).
27% of treated trial participants showed a ≥8-letter improvement in LCLA binocular at a 2.5% contrast threshold, compared to 6% of the matched CLIMB registry patients.
67% of treated trial participants showed a ≥3-point improvement in the SDMT, compared to 18% of the matched CLIMB registry patients.
47% of treated trial participants showed a ≥8-point improvement in LCLA binocular at a 1.25% contrast threshold (data for this endpoint were not collected by the CLIMB registry).
Across all participants, improvements in function as measured by LCLA, SDMT and MS Functional Composite (MSFC) were observed. Mean improvements from baseline of 3.3 points in the LCLA binocular (2.5% contrast), 3.8 points on the SDMT, and 0.18 points in MSFC were observed in treated trial participants. The corresponding changes in matched CLIMB registry patients estimated at 28 weeks showed declines in function on the LCLA and MSFC. The average change in function decline as measured by T25FW, 9HPT, and EDSS across all treated trial participants demonstrated stabilization of functional decline, with similar or slightly worse findings observed in the matched CLIMB registry patients for the same endpoints.

There were no adverse events related to worsening of MS disease and no clinically significant changes in safety lab results/vital signs, confirming NurOwn's favorable safety profile. Two patients developed symptoms of low back and leg pain, consistent with arachnoiditis, occurring in one of three treatments in both participants.

Treatment also consistently resulted in increases in cerebrospinal fluid neuroprotective factors (VEGF–A, HGF, NCAM-1, Follistatin, LIF and Fetuin–A) and reductions in inflammatory biomarkers (MCP-1, sCD27, SDF-1, and Osteopontin), confirming NurOwn's proposed mechanism of action in progressive MS.

"We were pleased that the study's initial results showed efficacy in patients with progressive MS," said Jeffrey Cohen, M.D., Hazel Prior Hostetler Endowed Chair Professor, Cleveland Clinic Lerner College of Medicine, Director, Experimental Therapeutics, Mellen Center for MS Treatment and Research, and the paper's lead author. "There are both promising biological and preliminary clinical signals of a treatment effect that will require confirmation in a randomized trial."

"There is a high unmet need for better treatments for progressive forms of MS and we congratulate the Brainstorm Cell Therapeutics team for the successful completion and publication of this important study. We look forward to future studies that will help to fully understand the potential of NurOwn and other cell-based therapies for this hard-to-treat form of disease" said Bruce Bebo, EVP Research National MS Society.

Chaim Lebovits, Chief Executive Officer, BrainStorm Cell Therapeutics stated, "Having these data peer reviewed and published in the prestigious Multiple Sclerosis Journal is an important step in the evaluation of NurOwn in progressive MS. We appreciate the expertise and commitment of the study investigators and contributions of study participants to advance our understanding of NurOwn's cellular technology platform. Thanks to their efforts and those of the BrainStorm team, we believe we are closer to providing a meaningful treatment option for those with progressive MS".

Ralph Kern, M.D., MHSc, President and Chief Medical Officer of BrainStorm Cell Therapeutics and co-author of the paper commented, "This publication provides preliminary evidence of NurOwn's potential to modify functional outcomes in progressive MS, which we believe warrants further study. In addition, consistent changes in cerebrospinal fluid neuroinflammation and neuroprotection biomarkers reveal how NurOwn may impact disease mechanisms in progressive MS and are complementary to biomarker results observed in our Phase 3 ALS trial. These observations provide further support for NurOwn as a platform technology with potential broad applications and will bolster BrainStorm's efforts to bring much needed solutions to patients with progressive MS, ALS, and other neurodegenerative diseases."

Study Design

The Phase 2 study (BCT-101) was designed to evaluate the safety, efficacy, and biomarker effects of three intrathecal administrations of NurOwn (MSC-NTF cells), given at two-month intervals, to adults with progressive MS. The trial was conducted at four MS centers of excellence: Cleveland Clinic Mellen Center for MS, Icahn School of Medicine at Mount Sinai, Keck School of Medicine of the University of Southern California, and Stanford University School of Medicine. Twenty participants ages 18-65 with progressive MS were enrolled and 17 received all three treatments and were followed for up to 28 weeks. Participants had baseline EDSS scores of between 3.0 and 6.5, were able to walk 25 feet in 60 seconds or less and had not experienced an MS relapse in the 6 months prior to study enrollment.

The primary efficacy outcome was pre-specified improvement (≥25%) in T25FW or 9-HPT. Additional efficacy endpoints included pre-specified improvements in EDSS, SDMT, LCLA, and MSWS-12. The efficacy outcomes were compared to a pre-specified matched group of progressive MS patients from CLIMB registry (n=48) (Tanuja Chitnis, M.D. Brigham and Women's Hospital and the Ann Romney Center for Neurologic Diseases). The study was sponsored by Brainstorm Cell Therapeutics with additional financial support for biomarker analyses received from the National Multiple Sclerosis Society, Fast-Forward Commercial Research Funding Program. For more information on the trial, visit https://clinicaltrials.gov/ct2/show/NCT03799718.

[midastouch017]

Wrong board, my friend!

[bubbarayjr]

AMLX paves the way for BCLI: 10+ Bagger - Wall Street is asleep on this one.
FDA's Panel nod to AMLX for ALS means FDA acceptance of BCLI's BLA a certainty imo.

The transcript of BCLI's BLA announcement: https://seekingalpha.com/article/4534421-brainstorm-cell-therapeutics-inc-s-bcli-ceo-chaim-lebovits-on-q2-2022-results-earnings-call

Correcting the statistical model for a key Pre-specified Secondary Endpoint resulted in a statistically significant improvement for patients in this sub-group.

This is huge but here's the kicker. The FDA has been putting more emphasis on how ALS treatments affect key Biomarkers. NurOwn improved 3 very important markers in patients who responded favorably. **AND** BCLI will publish this Biomarker data in a PEER REVIEWED JOURNAL in the very near future.

With a Market Cap of $138M, BCLI compared to AMLX Market Cap nearing $2B, it's a 10+ Bagger.

[Fmello]

AMLX is up 77% in pre-market and as expected and is pushing up BCLI as well (+10.25% in pre-market). If my guess is right, I would expect to see Brainstorm stock up 20+% by the closing bell today.

[Fmello]

I'm really curious to see what their stock price will jump to tomorrow. I assume that it will be similar to Brainstorm stock if they get the same kind of good news.

[bubbarayjr]

AMLX FDA 's Positive vote bodes very well for BCLI

[Fmello]

AMLX passed the FDA committee vote 7 yes, 2 no, 0 abstain.

I wonder how much that news improves Nurown's odds for FDA approval.

[Fmello]

https://www.biospace.com/article/als-thought-leaders-weigh-in-ahead-of-second-amylyx-adcomm-/?fbclid=IwAR1Z9kf0p4GnPIiVjIgMMrlSext2UfKhmEhzV-GQIG_hNS4u86nxcRVgziI

The ALS community has been calling for regulatory flexibility since the FDA approved Biogen's Aduhelm for Alzheimer’s in June 2021.

Of the Sept. 7 adcomm, Stacy Lindborg, Ph.D., EVP and chief development officer at BrainStorm Cell Therapeutics said, “We hope that a positive…recommendation would encourage the FDA to exercise regulatory flexibility.”

BrainStorm is in the final stages of completing a BLA for NurOwn, an ALS candidate made up of autologous mesenchymal stem cells (MSCs) and neurotrophic factors (NTFs). Lindborg noted that the totality of evidence for NurOwn “[brings] to light valuable insights on the nature of ALS research and biomarkers as predictors of clinical response.”

[midastouch017]

No comparison intended, hence my
semi off topic remark on my post.

[bubbarayjr]

Will be interesting to see how the committee interprets this but there's a BIG difference between AMLX and BCLI. AMLX needs to convince the FDA that their new model to evaluate efficacy is solid even though their clinical trial was not designed for it and there's not historical data to support doing so.

BCLI's issue is much different. The vendor in charge of analytical analysis of the trial data made a mistake. Correcting this error resulted in A STATISTICALLY SIGNIFICANT outcome in a KEY SECONDARY ENDPOINT which WAS PRE-SPECIFIED in the trial study designed (no smoke and mirrors). AND many of the BIOMARKERS the FDA believes are important to measure disease showed SIGNIFICANT improvement.

Definitely not Apples to Apples, more like Apples to Dollar bills IMO

[midastouch017]

FDA still skeptical of ALS drug ahead of high-stakes meeting

Semi O/T)

https://news.yahoo.com/fda-still-skeptical-als-drug-172758215.html

MATTHEW PERRONE
Fri, September 2, 2022 at 8:27 PM·4 min read
WASHINGTON (AP) — Federal health regulators remain unconvinced about the benefits of a closely watched experimental drug for the debilitating illness known as Lou Gehrig’s disease, even as they prepare to give its drugmaker a rare second opportunity to make a public case for the treatment.

Amylyx Pharmaceuticals' experimental drug has become a rallying cause for patients with the deadly neurodegenerative disease, their families and members of Congress who are pushing the FDA to approve the drug.

But regulators said Friday that the drugmaker's new analyses are not “sufficiently independent or persuasive” to establish effectiveness. The agency posted its review ahead of a Wednesday meeting of its outside advisers, who will vote on whether to recommend approval.

In March, the same panel of neurological experts voted 6-4 that the company’s data failed to show a convincing benefit for ALS, or amyotrophic lateral sclerosis. It’s extremely rare for the FDA to call a second review meeting after its advisers have already voted.

The FDA will ask the panel to review several new statistical analyses, which the company says strengthen the case that its drug prolongs life and delays hospitalization and other severe complications. The FDA says the experts can take into account “the unmet need in ALS," the disease's seriousness and other factors specific to the terminal diseases.

Elsewhere in its review the FDA detailed the flexibility it can apply to drug approval decisions, particularly for deadly diseases, which suggests “there is a chance that the FDA is still looking for a way to approve the product,” SVB analyst Marc Goodman wrote in a note to investors. He gives Amylyx a 50% chance of approval.

ALS destroys nerve cells needed to walk, talk, swallow and — eventually — breathe. There is no cure and most people die within three to five years.

The FDA's review reflects some of the biggest questions facing the agency, including: How strict should it be in enforcing approval standards for drugs against rare, fatal diseases? And how much weight, if any, should be given to outside appeals from patients, advocates and their political allies?

Typically, FDA approval requires two large studies or one study with a “very persuasive” effect on survival.

Amylyx’s data comes from one small, mid-stage trial that showed some benefit in slowing the disease, but which was marred by missing data, implementation errors and other problems, according to FDA reviewers.

Amylyx says follow-up data gathered after the study concluded shows the drug extended life. When the company followed patients who continued taking the drug, they survived about 10 months longer than patients who never took the drug, according to a new company analysis.

But FDA said Friday the new approach “suffers from the same interpretability challenges” as Amylyx’s initial study and that the new analysis “is not independent data.”

The FDA does not publicly explain its rationale for holding meetings. But some outside analysts believe the agency is hoping that more external input will strengthen its hand when it renders its final decision, expected by the end of the month.

Amylyx’s drug is a combination of two older drug ingredients: a prescription medication for liver disorders and a dietary supplement associated with traditional Chinese medicine. The Cambridge, Massachusetts, company has patented the combination and says the chemicals work together to shield cells from premature death. Its co-founders first hit upon the combination as Brown University students.

Some ALS patients already take both pills. FDA approval would likely compel insurers to cover the treatment.

The FDA will hear again from patients and advocacy groups, such as I AM ALS, which has lobbied the FDA and Congress for more than two years to make the drug available. The group’s founder, Brian Wallach, said ALS patients, physicians and researchers believe that the company’s data warrants approval.

“Patients do their homework— we know this isn’t going to cure us,” said Wallach, who was diagnosed with ALS in 2017 and spoke through an interpreter. “But we also know it might keep us here until the next drug comes along and that one might be a cure.”

Wallach currently takes the part of Amylyx’s treatment that is available as a dietary supplement.

Despite the negative FDA review, there are several outside developments that could tip the FDA toward approval.

In June, Canadian regulators approved the drug for ALS patients, the first country to do so. That decision puts FDA regulators in a “precarious position,” says bioethicist Holly Fernandez-Lynch.

"They typically like to be out ahead when making approval decisions," said Fernandez-Lynch, who teaches at the University of Pennsylvania. “They like to make the argument that they are not a barrier to patients accessing things that might help them.”

Shares of Amylyx fell more than 23% to close at $18 in trading Friday.

[bubbarayjr]

FDA has 60 days to accept or decline BLA submission. I believe we'll know the outcome by the end of October.

[Fmello]

[midastouch017]

Quote from Seeking Alpha.

Conclusion:

Brainstorm has decidedon August 15, 2022 that it would soon file a biologics license application for its autologous stem-cell-based treatment candidate NurOwn in ALS. At first sight, that announcement came as a surprise, as the drug had failed to reach a primary endpoint in ALS. At second sight, it may not. Minor corrections to the initial readout, due to the original publication having reported results that unintentionally deviated from the trial's pre-specified statistical analysis, led the company to report that a prespecified subgroup of patients with a baseline ALSFRS-R score of ≥35 did report statistical benefit. Additionally, this benefit seemingly extends to all patients with scores from 26 to 35.
The FDA’s transcripts on Amylyx’s drug candidate Albriova provide guidance as to the importance of neurofilament light chain as a biomarker. The FDA seems to find this biomarker of significant importance, as it is in part because Amylyx’s Albrovia was unable to demonstrate any change over placebo here, that the FDA’s advisory committee voted against the approval of Albriova. Biogen decided to go for accelerated approval of its drug candidate Tofersen in ALS based on measurements of the same biomarker. NurOwn has shown a 82% reduction of this biomarker over the course of 5 months, which is significantly better than Biogen’s Tofersen.
I believe exciting times are ahead for the ALS space. Brainstorm Cell Therapeutics is now ready to file for a BLA for NurOwn. Amylyx’s Albriova is up for a PDUFA on September 29, 2022. The FDA’s call on Tofersen is slated for January 25, 2023. My bet is that, if any of these would make it to approval, NurOwn actually stands the best chances. But in light of new data, it would have to move the FDA to revise its earlier decision on NurOwn.
For all the above, I am rating Brainstorm Cell Therapeutics as a buy.

[Fmello]

Here's the link:

https://seekingalpha.com/article/4535982-brainstorm-cell-therapeutics-upcoming-bla-in-als

[midastouch017]

Brainstorm Cell Therapeutics: Upcoming BLA In ALS

Aug. 21, 2022 3:57 AM ETBrainstorm Cell Therapeutics Inc. (BCLI)

Summary

Brainstorm Cell Therapeutics is a company developing NurOwn as an autologous stem cell-based treatment for ALS and MS.
NurOwn’s mechanism of action relies on bringing down neuroinflammation and altering glial activity, a recipe that seems to be a winner in neurodegenerative diseases.
After its Phase 3 trial in ALS did not reach its primary endpoint, the company announced on August 15, 2022 that it would still file a BLA in ALS.
Upon reanalysis of the data, statistical significance was in fact found in a predefined subgroup as well as in a larger group of ALS patients.
Like Biogen’s Tofersen but unlike Amylyx’s Albrioza, NurOwn significantly reduces the biomarker Neurofilament Light Chain, which the FDA’s AdCom finds of high value.

On August 15, 2022, Brainstorm Cell Therapeutics (NASDAQ:BCLI) caught the investment community by surprise, announcing that it would file a BLA for ALS. Brainstorm’s NurOwn failed to reach a primary endpoint in its Phase 3 trial in ALS. Yet, corrections to the initial data were reported and an erratum to the initial publication was published. Those corrections, although minor, did mean that statistical significance was shown in a predefined subgroup that had a baseline ALSFRS-R score of ≥35. Additionally, NurOwn also showed benefit in patients with ALSFRS-R scores of at least 26-35. Higher ALSFRS-R score mean less neurodegeneration, meaning NurOwn shows benefit in patients that have not yet reached the final stages of ALS disease.

That was all i could copy/paste

[bubbarayjr]

BioSpace: NurOwn A Study in the complexity of ALS trials

https://www.biospace.com/article/nurown-a-study-in-the-complexity-of-als-trials-/

[bubbarayjr]

ALS News: NurOwn

https://alsnewstoday.com/news/nurown-benefits-patients-less-severe-als-analyses-show/

[midastouch017]

I do hope the treatment will be approved,
for my sake as a shareholder as well as
for all ALS patients, but one has to be
realistic and read the following:

DISCLOSURE: Zacks SCR has received compensation from the issuer directly, from an investment manager, or from an investor relations consulting firm, engaged by the issuer, for providing research coverage for a period of no less than one year. Research articles, as seen here, are part of the service Zacks SCR provides and Zacks SCR receives quarterly payments totaling a maximum fee of up to $40,000 annually for these services provided to or regarding the issuer. Full Disclaimer HERE.

Notwithstanding: Go NurOwn® Go!

[Fmello]

BCLI: Filing a BLA for NurOwn® for the Treatment of ALS…

https://finance.yahoo.com/news/bcli-filing-bla-nurown-treatment-141600304.html

This stood out to me:

With our increased confidence in NurOwn’s BLA getting to an Advisory Committee meeting, we have increased the probability of approval to 50%, which has increased our valuation to $21 per share.

[Fmello]

Brainstorm Cell Therapeutics (BCLI) Receives a Buy from Maxim Group

https://www.tipranks.com/news/blurbs/brainstorm-cell-therapeutics-bcli-receives-a-buy-from-maxim-group?mod=mw_quote_news

[midastouch017]

As of 11:08AM EDT. Market open.
3.7477+0.3077 (+8.94%)
Inching up indeed!
Still a long way to go.

[Fmello]

https://www.facebook.com/michelle.lorenz.395/videos/1028900704442247

[Windbag1014]

Might be the reason for the extra chatter from the company and Maxim.
Shares are up over 26% since 7/1. Convenient.

[midastouch017]

No More Excuses! ALS Watch Dog Group
Public group
·
13.8K members
----------------------------------------------------------------------------
I just checked in and joined the group!
The only one to decide if and when to
raise funds is the company itself.

[Fmello]

I don't have a Facebook account so can someone go to the No More Excuses! ALS Watch Dog Group - Facebook page and put up a posting informing them that Brainstorm is running low on cash and that they should do a fundraising event for Nurown. If the FDA approves the Nurown BLA, No More Excuses can use that money to help Brainstorm get up and running as fast as possible.

[midastouch017]

Indeed, If the cash burn continues at current rate,
eg ~ $6 million in a single quarter, BCLI will
need to raise capital much sooner than later.

[Fmello]

The one thing that I'm wooried about:

Cash, cash equivalents, and short-term bank deposits were approximately $12.2 million as of June 30, 2022, compared to $18.4 million as of March 31, 2022.

Hopefully, they can get FDA approval before they run out of cash.

[midastouch017]

I am pretty confident the stock will
start an inching up process the
coming days and weeks!

[midastouch017]

I guess the market decided it won’t get excited until at least the FDA accepts the newly filed BLA.

I concur.

Unlike GMDA which is in a far more advanced stage, the market
is still cautiously optimistic as is reflected in its Market Cap.
MDWD is an excellent example of delayed market reaction to
its progress.

[Fmello]

Brainstorm Cell Therapeutics Inc.'s (BCLI) CEO Chaim Lebovits on Q2 2022 Results - Earnings Call Transcript

https://seekingalpha.com/article/4534421-brainstorm-cell-therapeutics-inc-s-bcli-ceo-chaim-lebovits-on-q2-2022-results-earnings-call?source=content_type%3Aall%7Cfirst_level_url%3Aportfolio%7Csection%3Aportfolio_content_unit%7Csection_asset%3Alatest%7Cline%3A1

[murocman]

I guess the market decided it won’t get excited until at least the FDA accepts the newly filed BLA.

Even then, the price advance will probably be muted until such time approval is granted.

Bottom line, still high risk, high reward based mostly on the uncertainty of the FDA.

Murocman

[Fmello]

https://www.fiercebiotech.com/biotech/after-fda-slammed-breaks-brainstorm-therapeutics-hitting-gas-updated-phase-3-data-its-als

[Fmello]

I'm confused about what happened to the stock price today.

It shoots up to $4.70 with the announcement of the FDA filing and then gets pushed down to where it started by the closing bell. Was that pullback caused by shorters? I was expecting a huge bump to the stock price after that great news...weird.

[midastouch017]

How long does it take the FDA to review a BLA?
The timelines for NMEs and BLAs that fall under PDUFA V's “Program” Review Model are 10-months for standard applications and 6-months for priority reviews from the 60-day filing date (or 12 months and 8 months respectively from the date of submission of the application).

[Fmello]

ABOUT FRIGGIN' TIME!!!

That is fantastic news! Does anyone know approximately how long it would take for the FDA to approve Nurown after Brainstorm's BLA filing?

[midastouch017]

4.1700 +0.7000 (+20.1729%)
Pre-Market: 8:04AM EDT
Action starts?

[midastouch017]

BrainStorm to seek FDA approval for ALS therapy despite Phase 3 setback

Aug. 15, 2022 7:45 AM ETBrainstorm Cell Therapeutics Inc. (BCLI)BIIB, IONS, AMLX

By: Dulan Lokuwithana, SA News Editor

Brainstorm Cell Therapeutics (NASDAQ:BCLI), a biotech focused on neurodegenerative diseases, said on Monday that the company would submit a Biologics License Application (BLA) to the FDA seeking approval of its NurOwn therapy for amyotrophic lateral sclerosis (ALS).

The plan comes nearly two years after Brainstorm (BCLI) announced that a Phase 3 trial for NurOwn did not yield statistically significant results in ALS.

However, Brainstorm (BCLI) said a correction was made on the Muscle and Nerve publication regarding Phase 3 ALS data for NurOwn, indicating a statistically significant treatment difference (p=0.050).

"After carefully considering these learnings, the totality of the evidence from NurOwn's® clinical studies, and the feedback received from key opinion leaders and the broader ALS community, we will submit a Biologics License Application to the FDA," Chaim Lebovits, CEO of BCLI said.

"We intend to provide additional updates upon learning whether the FDA files our BLA submission," he added.

The announcement coincided with Brainstorm's (BCLI) Q2 2022 financials which showed ~$12.2M of cash, cash equivalents, and short-term bank deposits for the company at the end of the quarter.

The ALS space has garnered regulatory interest in recent months. An ALS candidate developed by Biogen (BIIB) and Ionis (IONS) is currently under the FDA's priority review.

Weeks ago, Amylyx Pharmaceuticals (AMLX) announced that an independent panel of experts of the FDA is set to meet in September to discuss its ALS medication AMX0035 despite a negative vote in March.

[midastouch017]

BrainStorm Cell Therapeutics Announces Second Quarter 2022 Financial Results and Provides a Corporate Update

https://finance.yahoo.com/news/brainstorm-cell-therapeutics-announces-second-110500270.html

BrainStorm to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for NurOwn® for the treatment of ALS

New clinical analyses reinforce the conclusions from NurOwn's® Phase 3 clinical trial

Conference call and webcast at 8:00 a.m. Eastern Time today

NEW YORK, Aug. 15, 2022 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced financial results for the second quarter ended June 30, 2022, and provided a corporate update. The company also announced its decision to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration for NurOwn® for the treatment of amyotrophic lateral sclerosis (ALS).

BrainStorm announces decision to submit a BLA to the FDA for NurOwn® for the treatment of ALS

"Brainstorm Cell Therapeutics is at a pivotal moment as a company as we finalize the regulatory filing for NurOwn® in the treatment of ALS. The continued analysis and the feedback received from the many scientific presentations of NurOwn's® Phase 3 data have uncovered key insights that furthered our understanding of the product mechanism of action and therapeutic potential and strengthened the conclusions of NurOwn's® efficacy," said Chaim Lebovits, Chief Executive Officer. "After carefully considering these learnings, the totality of the evidence from NurOwn's® clinical studies, and the feedback received from key opinion leaders and the broader ALS community, we will submit a Biologics License Application to the FDA. We are deeply grateful to the ALS clinical experts, members of the ALS community and faithful investors for their contribution to the development of NurOwn® and what it may mean to those living with ALS. Their contributions and commitment made our current progress possible and continue to inspire us as we prepare for the considerable work ahead. We intend to provide additional updates upon learning whether the FDA files our BLA submission."

New clinical analyses strengthen the conclusions from NurOwn's® Phase 3 clinical trial

A correction was made to the Muscle and Nerve publication from December 2021 describing the results of NurOwn's® Phase 3 clinical trial in ALS following new clinical analyses which strengthen the Company's original conclusions from the trial. The correction results in a statistically significant treatment difference (p=0.050) of more than 2 points for an important secondary endpoint, average change from baseline in ALSFRS-R, in the pre-specified efficacy subgroup of participants with a baseline score of at least 35. Analyses reported in the original publication utilized an efficacy model that unintentionally deviated from the trial's pre-specified statistical analysis plan by erroneously incorporating interaction terms between the subgroup and treatment. The newly published results, which includes supporting information to the publication, employ the efficacy model as pre-specified in the trial's statistical analysis plan, correcting the analyses. The correction also relates to the other subgroup analyses published for this endpoint, demonstrating that all subgroups with ALSFRS-R baseline scores of at least 26 to 35 showed a statistically significant benefit following treatment with NurOwn® (p≤0.050) on this secondary endpoint.

Other Second Quarter 2022 and Recent Highlights

Presented new analyses from NurOwn's® Phase 3 ALS trial that showed a treatment effect in participants predicted by the ENCALS model to have intermediate to very long survival. These analyses confirmed the importance of avoiding potentially misclassifying treatment responses due to the ALSFRS-R floor effect and were presented in a poster presentation at the 2022 European Network for the Cure of ALS (ENCALS) Meeting. The presentation was delivered by Dr. Jonathan Katz, principal investigator on the trial and Chair of the Neurology Department and Director of the Forbes Norris ALS Clinic at the California Pacific Medical Center.

Biomarker analyses from NurOwn's® Phase 3 ALS trial were the subject of an abstract presented at the 2022 American Academy of Neurology Congress by Dr. James Berry, MD, MPH, principal investigator on the trial and Director of the Massachusetts General Hospital Multidisciplinary ALS Clinic and Chief of the Division of ALS and Motor Neuron Diseases. Results of the analyses showed significant changes across multiple cerebrospinal fluid (CSF) biomarkers following NurOwn® treatment, with the strongest effects observed on biomarkers related to neuroprotection and neuroinflammation.

Presented a summary of analyses from the Phase 3 trial of NurOwn® in ALS that highlighted the ability of biomarkers to predict clinical treatment response and provide a window into the complex biological pathways underlying disease progression. The presentation was delivered at the ALS Drug Development Summit by BrainStorm's President and Chief Medical Officer, Dr. Ralph Kern, MD, MHSc.

Reported new results from the open-label Phase 2 study of NurOwn® in progressive multiple sclerosis (MS) that highlighted post-treatment improvements in monocular and binocular low contrast letter acuity (LCLA) outcomes (1.25% and 2.5% thresholds). In contrast, matched patients from the long-term Comprehensive Longitudinal Investigation of Multiple Sclerosis (CLIMB) study and participants from the placebo arm of the SPRINT study showed worsening in LCLA outcomes over a similar time period. The results were presented in a poster presentation delivered by Dr. Kern at the 2022 Consortium of Multiple Sclerosis Centers (CMSC) Meeting.

Presented biomarker analyses from the open-label Phase 2 study of NurOwn® in progressive MS showing consistent post-treatment reductions in CSF inflammatory biomarkers that may be relevant to disease progression and treatment response. The presentation was delivered by Dr. Christopher Lock, PhD, Clinical Associate Professor, Neurology and Neurological Studies, at Stanford School of Medicine, at the CMSC 2022 Meeting.

Preclinical in-vitro data that showed NurOwn® cells maintaining their neurotrophic and immunomodulatory effects in the presence of Siponimod, an S1P modulator recently approved for the treatment of secondary progressive MS, were presented in a poster presentation at the CMSC 2022 Meeting. The presentation was delivered by Dr. Sidney Spector, MD, PhD, Senior Vice President, Medical Affairs and Global Strategy at BrainStorm Cell Therapeutics.

Reported preclinical data from a murine lung injury model (the bleomycin model) that demonstrated intrathecal administration of NurOwn®-derived exosomes (Exo MSC-NTF) may have potential as a clinical therapy for inflammatory pulmonary pathologies and display superior macrophage immunomodulation compared to naïve mesenchymal stem cell-derived exosomes (Exo-MSC). The data were presented at the International Society for Extracellular Vesicles (ISEV) 2022 Annual Meeting by Haggai Kaspi, PhD, Preclinical Research Manager at BrainStorm Cell Therapeutics.

Preclinical in vitro data demonstrating the superior anti-inflammatory effects of Exo MSC-NTF compared to Exo-MSC were featured in a poster at the International Society of Cell & Gene Therapy (ISCT) 2022 Meeting. The poster was presented by Dr. Kim Thacker, Senior Vice President, Medical Affairs and Clinical Innovation at BrainStorm Cell Therapeutics.

Strengthened executive team in preparation for anticipated growth and corporate development, with the appointment of Netta Blondheim-Shraga, PhD, as VP of Research & Development and Antal Pearl-Lendner, Adv., as Chief Legal Counsel

Financial Results for the Second Quarter Ended June 30, 2022

Cash, cash equivalents, and short-term bank deposits were approximately $12.2 million as of June 30, 2022, compared to $18.4 million as of March 31, 2022.

Research and development expenses for the three months ended June 30, 2022, and 2021 were approximately $5.1 million and $3.6 million, respectively.

General and administrative expenses for the three months ended June 30, 2022, and 2021 were approximately $2.5 million.

Net loss for the three months ended June 30, 2022, was approximately $7.0 million, as compared to a net loss of approximately $6.3 million for the three months ended June 30, 2021.

Net loss per share for the three months ended June 30, 2022, and 2021 was $0.19 and $0.17, respectively.

Conference Call and Webcast

August 15, 2022, at 8:00 a.m. Eastern Time

Participant Numbers:
Toll Free: 888-506-0062
International: 973-528-0011
Participant Access Code: 955552

Webcast URL: https://bit.ly/3cXwQkt

Those that wish to listen to the replay of the conference call can do so by dialing the numbers below. The replay will be available for 14 days.

Toll Free: 877-481-4010
International: 919-882-2331

[Fmello]

Just a reminder that Brainstorm's 2nd quarter earnings call is on Monday morning @ 7am central time.

https://www.webcaster4.com/Webcast/Page/2354/46290