Form 8-K - Current report
01 10월 2024 - 9:00PM
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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the
Securities Exchange Act of 1934
Date of Report (Date of earliest event reported):
October 1, 2024
Brainstorm Cell Therapeutics Inc.
(Exact name of registrant as specified in its
charter)
Delaware |
|
001-36641 |
|
20-7273918 |
(State or other jurisdiction of
incorporation) |
|
(Commission File No.) |
|
(IRS Employer Identification No.) |
1325 Avenue of Americas, 28th Floor |
|
New York, NY |
10019 |
(Address of principal executive offices) |
(Zip Code) |
(201) 488-0460
(Registrant’s telephone number, including
area code)
N/A
(Former name or former address, if changed
since last report)
Check the appropriate box below if the Form 8-K filing is intended
to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
| ¨ |
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
| ¨ |
Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
| ¨ |
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
| ¨ |
Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
Securities registered pursuant to Section
12(b) of the Act:
Title of each class |
Trading Symbol(s) |
Name of each exchange on which registered |
Common Stock, $0.00005 par value |
BCLI |
NASDAQ Stock Market LLC
(Nasdaq Capital Market) |
Indicate by check mark whether the registrant is an emerging
growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities
Exchange Act of 1934 (§240.12b-2 of this chapter).
Emerging growth company ¨
If an emerging growth company, indicate by check mark if the
registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards
provided pursuant to Section 13(a) of the Exchange Act. ¨
Item 7.01 Regulation FD Disclosure.
On October 1, 2024, Brainstorm Cell Therapeutics, Inc. (the “Company”)
released a company presentation. A copy of the company presentation is attached as Exhibit 99.1 to this Current Report on Form 8-K. The
company presentation will also be available in the investor relations section of the Company’s website at https://ir.brainstorm-cell.com/.
Information contained on the Company’s website is not incorporated by reference into this Current Report on Form 8-K, and you should
not consider any information on, or that can be accessed from, the Company’s website as part of this Current Report on Form 8-K.
The information under this Item 7.01, including Exhibit 99.1 hereto,
is being furnished and shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended
(the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference
in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in
such a filing. The Company undertakes no obligation to update, supplement or amend the material attached hereto as Exhibit 99.1.
Item 9.01 Financial Statements and
Exhibits.
(d): Exhibits:
Exhibit No. |
Description |
|
|
99.1 |
Company Presentation. |
|
|
104 |
Cover Page Interactive Data File (formatted as inline XBRL and contained in Exhibit 101) |
SIGNATURES
Pursuant to the requirements of the Securities
Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
|
BRAINSTORM CELL THERAPEUTICS INC. |
|
|
|
Date: October 1, 2024 |
By: |
/s/ Chaim Lebovits |
|
|
Chaim Lebovits |
|
|
Chief Executive Officer |
Exhibit 99.1
| Brainstorm Cell Therapeutics
Nasdaq: BCLI |
| 2
Safe Harbor Statement
Statements in this announcement other than historical data and information 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, risks associated
with Brainstorm's limited operating history, history of losses; minimal working capital, dependence on its license to Ramot's
technology; ability to adequately protect the technology; dependence on key executives and on its scientific consultants;
ability to obtain required regulatory approvals; 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. |
| 3
Brainstorm Cell Therapeutics: A Clinical Stage Biotechnology Company
Our Mission Brainstorm pioneers autologous cell therapies for neurological disorders, focusing on ALS
and MS
Flagship
Product NurOwn® - a cutting-edge autologous cell therapy platform
Strong Pipeline NurOwn® innovative therapy serves as a platform for treating neurodegenerative diseases
Experienced
Leadership Decades of expertise in cell therapy and biopharma commercialization |
| 4
Promising data from our previous trials continues to support the therapeutic potential of
NurOwn®, demonstrating meaningful improvements in key areas.
Robust biomarker data strongly supports NurOwn®’s mechanism of action.
Aligned with FDA on new clinical trial design through the Special Protocol Assessment (SPA),
ensuring regulatory confidence and trial integrity.
Phase 3b plans: Comprehensive plan for Phase 3b trial, optimizing patient selection and
dosing strategies.
Pioneering Breakthrough: First-In-Class Therapy to Transform ALS
Treatment |
| 5
Indication Preclinical Phase 1 Phase 2 Phase 3
ALS*
Progressive MS
Parkinson's Disease
ARDS
NeurOwn® MSC-NTF Cellular Platform
MSC-NTF Exosomes Platform
Innovating Cell Therapies for Neurodegenerative Diseases
*dependent on future funding
Phase 3
Phase 3B |
| 6
Experienced Team in Cell Therapy Development
Proven Expertise Decades of experience in developing innovative cell therapies across multiple therapeutic areas.
Successful Track
Record
Led the development of NurOwn®, producing over 470 GMP clinical batches for 190+ ALS trial.
Extensive experience across all phases of clinical development, from preclinical to commercialization.
Manufacturing
Excellence
Expertise in scaling up complex cell therapy manufacturing processes to meet clinical and regulatory
standards.
Regulatory and
Quality Leadership
Strong regulatory affairs team with a proven history of engaging with global health authorities, including the
FDA and EMA.
Innovation-Driven
Approach Focus on cutting-edge science and advancing new cell therapy technologies to improve patient outcomes. |
| 7
Chaim Lebovits
President and CEO
Bob Dagher, MD
CMO
Haro Hartounian, PhD
COO
Uri Yablonka
CBO
Mary Kay Turner
SVP, Advocacy and Government
Affairs
Netta Blondheim-Sharga
SVP, Research and Development
Yossef Levy, PhD
VP. Cell Production
Leticia Tarilonte
VP, Global Clinical Operations
Proven Leadership with Deep Expertise in Biotechnology |
| 8
Finalize agreements with leading clinical sites, setting the stage for a successful Phase 3b trial.
Looking Ahead: Key Upcoming Expected Milestones
Clinical Trial
Agreements (CTA)
Signed
15 Clinical Sites
Announced
Officially unveil our partnership with 15 premier clinical sites, ensuring broad geographic coverage and
access to top-tier patient care.
GMP Manufacturing in
Full Swing
GMP manufacturing of clinical-grade products is ready to support upcoming trials, ensuring timely
execution and quality.
Engaging Top-Tier CRO Signed an agreement with a leading CRO to manage patient recruitment, trial execution, and regulatory
compliance, maximizing trial success.
First Patient Enrollment
by December
On track to enroll the first patient in December 2024, marking a significant milestone in advancing
NurOwn®. |
| Our Technology
Cutting-Edge Innovation: Advancing the Future of Cell Therapy |
| 10
Developing NurOwn® as a Groundbreaking Platform to Treat ALS
• Survival is 2 to 5 years from onset, with a lifetime risk of 1 in 300 people.
• ALS affects 1.6 per 100,000 people annually worldwide.
• 90% to 95% of ALS cases are sporadic, with no known family history.
• Projected to grow significantly, driven by factors such as advancements in research, rising prevalence due to
aging populations, and emerging therapeutic modalities like cell and gene therapy.
• Valued at approximately $669 million in 2023, is expected to surpass $1.2 billion by 2034, growing at a
compound annual growth rate (CAGR) of around 6.4% (Datam Intelligence (IMARC).
1. Brotman RD, et al. StatPearls. 2020
2. Masrori P, et al. Eur J Neurol. 2020; 27:1918-1929
3. Brown RH, et al. N Engl J Med. 2017; 377:162-172
ALS is a progressive neurodegenerative disease that impairs walking, talking, eating, and breathing,
and is currently 100% fatal |
| 11
Amyotrophic Lateral Sclerosis- ALS
Devastating Disease with Hope on the Horizon through Our Groundbreaking Treatment |
| 12
Intrinsic immunomodulation properties
Tropism to sites of damage
In ALS murine models
• Delay motor neuron degeneration
• Improve motor performance
• Prolong survival
Multiple evidence for neuroprotection
Deficient in several neurodegenerative
diseases, including ALS
Single NTFs tested as potential treatment in
humans (e.g. BDNF & GDNF), with limited
success
MSCs NTFs
MSC-NTFs (NurOwn®)
Enhanced secretion of multiple NTF
(e.g. VEGF, HGF, GDNF, BDNF & Gal-1)
Enhanced immunomodulatory effects
Enhanced effects in ALS and other
neurodegenerative diseases
NurOwn®: Harnessing MSC Characteristics and NTF Production |
| 13
Our Technology: Transforming ALS Treatment with Advanced Cell
Therapy
NurOwn®: Uses the patient’s own cells, reducing immune rejection risks,
contributes to the high tolerance and good safety profile, and enhancing
natural neurorepair.
Introduced to the spinal cord CSF, MSC-NTF cells secrete bioactive
molecules like NTFs, microRNA, and cytokines, activating neuroprotective
and immunomodulatory pathways after intrathecal administration.
FDA-Approved Therapies: Riluzole and Edaravone offer limited benefits,
targeting glutamate toxicity and oxidative stress, respectively.
NurOwn® Platform: Explored for use in other neurodegenerative diseases
like MS and Parkinson's, showing broad potential. |
| 14
Changes in biomarkers across main-pathways following NurOwn treatment
MCP-1
OPG
S100B
SDF-1a
CHI3L1/YKL-40
Chitotriosidase-1
GFAP
ICAM-1
IP-10*
TREM-2
Inflammatory pathway Anti-Inflammatory pathway
Fetuin-A
IL-37
LAP (TGF-β1)
MSR1
hsa-miR-146a-5p
hsa-miR-146b-5p
Neurodegeneration pathway
pNfH
DR6
NfL
TWEAK
Tau
UCH-L1
hsa-miR-142-5p*
Caspase-3
Neuroprotection pathway
VEGF-A
BDNF
G-CSF
GDF-15
Galectin-1
HGF
NMNAT1
Clusterin/ ApoJ
LIF
Bold = significant change from baseline
detected during the study; p<0.05
*= non-significant trend in other direction
Robust biomarker data strongly supports NurOwn®’s mechanism of action |
| 15
Change from placebo,
neuroprotective markers
Change from placebo,
neurodegenerative and
neuroinflammatory
markers
Illustrative approach to demonstrate
the cumulative biomarker changes over
time following NurOwn treatment,
compared to placebo:
Lindborg et al., 2024
Biomarkers Change Following NurOwn Treatment |
| 16
Biomarkers in CSF from ALS patients
demonstrate pathological hallmarks of
active neurodegenerative and
neuroinflammatory processes
CSF biomarker changes following NurOwn
treatment suggest a favorable disruption
of pathological processes:
• Reduction in CSF neuroinflammatory and
neurodegenerative biomarkers
• Increase observed in CSF anti-inflammatory
and neuroprotective biomarkers
NurOwn®: Tipping the Balance to Favor Neuroprotection
Neuroinflammatory
Neurodegeneration pathway
Neuroprotective
Anti-Inflammatory
NurOwn®
Treatment
Neuroinflammatory
Neurodegeneration pathway
Neuroprotective
Anti-Inflammatory |
| 17
The NurOwn® Process: Step by Step
The patient’s bone marrow is harvested and Mesenchymal
Stromal Cells (MSCs) are isolated from the total bone
marrow population.
The MSCs are expanded ex-vivo and cryopreserved.
Cryopreserved MSCs are thawed, expanded and induced
to differentiate into MSC-NTF cells (MSC cells that secrete
Neurotrophic Factors).
The MSC-NTF cells are then injected back into the patient
at or near the site of damage (the spinal cord). |
| 18
Manufacturing Expertise: Delivering Quality and Scale for NurOwn®
470+ GMP Batches Produced: Proven ability to deliver clinical-grade
materials at scale, dosing over 190 patients in multiple trials.
Successfully executed technology transfer across multiple clinical sites for
the production of NurOwn®, ensuring consistency and quality in
manufacturing.
State-of-the-Art Facilities: Advanced manufacturing in the US and Israel
with cutting-edge technology for precision and efficiency.
cGMP Compliance: Fully compliant with cGMP, ensuring consistent product
quality and safety through rigorous quality controls.
Global Manufacturing Footprint: Supporting global clinical and commercial
needs with best-in-class facilities. |
| NurOwn®
A Breakthrough in ALS Treatment
with Promising Clinical Data |
| 20
Phase 1/2 Phase 2a Phase 2 Phase 3 EAP Phase 3b
N=12 N=14 N=48 N=189 N=10 N=200
Orphan Drug
Designation
Fast Track
Designation
FDA SPA
Agreement
Our Milestones: NurOwn® Development Program in ALS |
| 21
Respiratory Function
• Dyspnea
• Orthopnea
• Respiratory insufficiency
Bulbar Function
Gross
Motor Function
Fine Motor Function
48-point scale with 4 domains
Primary Endpoint ALS Functional Rating Scale- Revised (ALSFRS-R)
Validated approvable questionnaire-based tool
Used as basis for approval of Radicava in 2017 with mean score
change at 24 weeks as primary endpoint
Data from the PRO-ACT database shows the average rate of
ALSFRS-R decline is 1.02 points/month
Change in ALSFRS-R slope (rate of disease progression) >20-25% is clinically meaningful2 |
| 22
NurOwn®: Phase 3 Clinical Trial in ALS (BCT-002-US)
Subgroup analysis: positive results in early disease population (N=58)
• Consistent treatment effects across multiple endpoints and over time
• Over ~2 points benefit in function at Week 28
Pre-specified subgroup with baseline ALSFRS-R ≥35
• ALSFRS-R treatment difference nominally significant starting at week-12, through the end of trial
NurOwn®
Placebo
* p ≤ 0.05
CFB in ALSFRS-R (95% CI) |
| 23
NurOwn® demonstrated a significant treatment effect in subgroup of patient with less
advanced disease.
More advanced ALS patients did not show a treatment benefit, likely due to a floor
effect.
Data reinforces the safety of repeat intrathecal administration.
The next trial will focus on enrolling less advanced patients and measuring the delta
between the baseline and week 24 to maximize the statistical power.
Clinical Data Summary |
| NurOwn®:
Optimized Phase 3b Trial in Early ALS Disease |
| 25
Part A:
Double Blind
Part B:
Open Label
NurOwn®: Phase 3 Clinical Trial in ALS (BCT-006-US)
9 weeks 24 Weeks 24 Weeks
R
NurOwn®
Randomization
Screening
Placebo
NurOwn®
NurOwn®
Intrathecal Injection every 8 weeks
Upon successful completion of our double-blind Phase 3b study,
we will be positioned to submit our Biologics License Application
(BLA) for regulatory approval, potentially accelerating our path to
market. |
| 26
Phase 3 (BCT-002-US) Phase 3b (BCT-006)
Patient
population
≥25, followed by 20-week “run-in” period, allowing the
possibility to enroll advanced-disease participants
Early-disease participants: ALSFRS-R criteria targeting less
advanced levels of functional decline (≥ 2 points on each item of the
ALSFRS- R …)
Primary
endpoint
Responder: proportion with ≥1.25 points/month improvement
in the post-treatment slope vs. pre-treatment slope in
ALSFRS-R at week 28
Use of “gold standard” in recent registrational trials (change from
baseline to Week 24 in ALSFRS-R total score)
• p-value derived from inference model of Combined Assessment
of Function and Survival (CAFS)
Study
duration
28-week double blind period 24-week double blind (Part-A) to be followed by 24-week open
label extension (Part B)
• Extended to evaluate long-term effects on survival and
neurodegenerative biomarkers
Screening
period
20 weeks screening, including 3-months “run-in” period to
evaluate pre-treatment disease progression via ALSFRS-R
Elimination of “run-in” and shortening of screening period to
minimize changes between screening and baseline
Regulatory Filing at week-28: Responder analysis in overall population,
including advanced disease
Filing at week-24: Special Protocol Assessment (SPA) agreement
with FDA
NurOwn® Phase 3b (BCT-006): Key Design Criteria
Significant Changes Made to Upcoming Phase 3b Clinical Trial |
| 27
NurOwn®: Positioned for Success
Proven Expertise Our team has extensive experience in conducting clinical trials, including Phase 1, 2, and 3 studies.
Regulatory
Alignment
Strong alignment from the FDA with Special Protocol Assessment (SPA) approval, ensuring trial design
meets regulatory standards and de-risking regulatory aspects of the program.
Data-Driven
Approach
Lessons from prior Phase 3 trials have refined patient selection, endpoints, and trial protocols, increasing
the probability of success.
Robust
Manufacturing
Process
Securing partnerships with leading CDMOs for reliable production of clinical-grade materials, ensuring
smooth execution.
Operational
Excellence
A dedicated team, resources, and a clear execution plan are in place to manage and monitor the trial
effectively. |
| 28
Finalize agreements with leading clinical sites, setting the stage for a successful Phase 3b trial.
Looking Ahead: Key Upcoming Expected Milestones
Clinical Trial
Agreements (CTA)
Signed
15 Clinical Sites
Announced
Officially unveil our partnership with 15 premier clinical sites, ensuring broad geographic coverage and
access to top-tier patient care.
GMP Manufacturing in
Full Swing
GMP manufacturing of clinical-grade products is ready to support upcoming trials, ensuring timely
execution and quality.
Engaging Top-Tier CRO Signed an agreement with a leading CRO to manage patient recruitment, trial execution, and regulatory
compliance, maximizing trial success.
First Patient Enrollment
by December
On track to enroll the first patient in December 2024, marking a significant milestone in advancing
NurOwn®. |
| Thank you
Chaim Lebovits
President & CEO
BrainStorm Cell Therapeutics
1325 Avenue of Americas NY, NY 10019 |
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