BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading
developer of cellular therapeutics for neurodegenerative diseases,
today issued a Letter to Shareholders.
Dear Valued Shareholders,
2019 was an extraordinary year for BrainStorm
Cell Therapeutics! From a research and development, clinical, and
corporate perspective, I appreciate the ongoing, dedicated support
we have received from our patients and their families. I also want
to acknowledge the many accomplishments of our employees, and the
commitment we have received from our shareholders and our
distinguished clinical partners, as we continue to execute on our
goal of bringing much needed treatments to those suffering from
neurodegenerative diseases.
In the second week of October, we announced full
enrollment of the 200-patient Phase 3 clinical trial (NCT03280056)
evaluating repeat intrathecal administration of NurOwn® cellular
therapeutic (autologous MSC-NTF cells) in amyotrophic lateral
sclerosis (ALS). This clinical milestone is a key inflection point
in the development of NurOwn as an investigational therapeutic and
confirming BrainStorm as the leading biotechnology company
developing autologous stem cell therapy treating neurogenerative
disease.
In late October 2019, the Data and Safety
Monitoring Board (DSMB) completed the second planned interim safety
analysis for the first 106 patients who received repeat dosing of
NurOwn in the Phase 3 ALS trial. The DSMB concluded the trial
should continue as planned without any protocol changes. Dr.
Carlayne Jackson, M.D., chair of the Phase 3 DSMB stated, “After
reviewing all of the safety data as of September 30th, the DSMB has
recommended the study continue without any changes in the protocol.
We did not identify any significant safety concerns.”
At the end of 2018, we announced the expansion
of our pipeline by introducing a new indication for NurOwn in
progressive Multiple Sclerosis (MS). Progressive MS has deeply
affected the lives of many and dramatically impacted family
members, caregivers and others. Today, there is no U.S. FDA
approved therapy addressing more than one progressive form of MS.
In March 2019, we enrolled the first patient in our Phase 2
open-label, multicenter study of repeated intrathecal
administration of autologous MSC-NTF cells in participants with
progressive MS. Throughout the year, due to our dedicated efforts,
we successfully contracted with leading US clinical sites for our
Phase 2 open-label, multicenter clinical trial of repeated
intrathecal administration of autologous MSC-NFT cells in
progressive MS. These clinical sites, which include the Cleveland
Clinic, Stanford University School of Medicine, the University of
Southern California and Mount Sinai Medical Center, are Centers of
Excellence for MS research and treatment and are led by prominent
and well-respected clinical investigators and KOLs in the field of
progressive MS. In November, Our Phase 2 ALS clinical data was
published in “Neurology”, the highly respected peer-reviewed
journal and the official journal of the American Academy of
Neurology. Concurrently, Brainstorm was also selected to be
featured in an editorial by independent reviewers titled “Stem
Cells in Amyotrophic Lateral Sclerosis - Hype or Hope”, which
commented on Brainstorm’s ALS clinical study design and stated:
“Berry and associates are to be commended for presenting the
framework of a well-planned and designed study that should serve as
a framework for other investigators to emulate.”
BrainStorm was recently awarded a grant of $495K
from The National MS Society (NMSS) to support our Phase 2
biomarker study of NurOwn in progressive MS. This funding from the
National MS Society will help us explore key neuroinflammation and
neural repair biomarkers in progressive MS to confirm NurOwn’s
unique mechanism of action and will guide the design of future
clinical trials to address this important unmet patient need.
We also expanded our intellectual patent
portfolio by obtaining additional United States and Japanese
patents for BrainStorm's NurOwn cellular technology for the
treatment of various neurogenerative diseases. These patents add
another layer of protection around our cellular technology and are
the result of our ongoing technical development and clinical
progress.
Lastly, we strengthened our executive team by
bringing on board Dr. Preetam Shah as our Executive Vice President
and Chief Financial Officer to expand our Investor Relations
program and future business development and capital strategy
initiatives.
Our ongoing Phase 3 and Phase 2 clinical
programs investigating NurOwn in ALS and progressive MS have
received extensive and positive news coverage in the mainstream US
media over the last year. I want to make sure that all our
investors have an opportunity to read these articles and view these
news items. The list of selected articles is listed below. The ABC
Nightline segment was broadcast to more than 3 million households
across the US and continues to be a major part of a full form
documentary that will be broadcast by ABC Nightline and partners in
2020.
What is clear from these news items is that ALS
and progressive MS represent significant unmet medical needs and
that, despite the limited options currently available, patients are
highly motivated to seek out new treatments and physicians
recognize the potential of our autologous cellular therapy
technology platform.
Approximately 6,000 people in the U.S. are
diagnosed with ALS each year and more than 30,000 Americans are
currently living with the disease. Worldwide it is estimated that
450,000 patients are living with ALS. Since 1995, only two new
pharmaceuticals have been approved, but the medical need remains.
NurOwn is the first autologous cellular investigational therapy to
have advanced as far in a Phase 3 clinical trial. Progressive MS
represents a US and worldwide addressable market opportunity of
500,000 and 1,250,000, individuals, respectively.
Looking ahead to 2020, we are highly focused on
generating top-line results for our ALS phase 3 and progressive MS
phase 2 trials in the fourth quarter, to initiating the regulatory
submission and eventual commercialization of NurOwn in ALS, to
further advancing our cellular technology and manufacturing
capabilities. We will actively seek additional opportunities to
expand our pipeline to address unmet need and to grow as a
biotechnology company.
I want to personally thank you for your
continued support.
Sincerely,
Chaim
Chaim Lebovits President and CEO BrainStorm Cell
Therapeutics Inc.
CONTACTS
Investors:Preetam Shah, Chief
Financial OfficerBrainStorm Cell Therapeutics IncPhone:
+1-201-488-0460Email: pshah@brainstorm-cell.com
Media:Sean LeousWestwicke/ICR
PRPhone: +1.646.677.1839Email: sean.leous@icrinc.com
Selected Video Clips:
ABC Nightline
ALS patients share about their life 5 years
after 'Ice Bucket Challenge': Part
1https://abcnews.go.com/Nightline/video/als-patients-share-life-years-ice-bucket-challenge-64874152
Part
2https://abcnews.go.com/Nightline/video/als-patient-rides-motorcycle-unable-years-part-64874209
KTLA Southern California
https://ktla.com/2019/11/21/l-a-firefighter-among-patients-undergoing-promising-treatment-for-als/
CBS Philadelphia
https://philadelphia.cbslocal.com/2019/03/11/als-patients-fighting-fda-for-experimental-drug-nurown/
Selected News Articles:
- ALS News Today – Phase 2 Trial Data on NurOwn Therapy,
Supporting Safety And Early Efficacy,
Publishedhttps://alsnewstoday.com/2019/11/25/phase-2-trial-nurown-cell-therapy-published-support-safety-and-early-efficacy-als/
- Multiple Sclerosis News Today – NMSS Awards $495K to Study
Biomarkers in Phase 2 Trial of NurOwn in Progressive
MShttps://multiplesclerosisnewstoday.com/news-posts/2019/11/15/brainstorm-cell-therapeutics-announces-research-grant-award-from-the-national-multiple-sclerosis-society/
- Seeking Alpha – Interview With Chaim Lebovits, CEO Of
Brainstorm Cell
Therapeuticshttps://seekingalpha.com/article/4301794-interview-chaim-lebovits-ceo-of-brainstorm-cell-therapeutics
- ALS News Today – Phase 3 Trial of NurOwn, Cell Therapy for ALS,
Gets Thumbs Up in Safety Review by Independent
Boardhttps://alsnewstoday.com/2019/11/01/nurown-gets-good-safety-review-by-phase-3-trial-monitoring-board/
- ALS News Today – Phase 3 Trial of NurOwn Cell Therapy Fully
Enrolls 200 Patients, BrainStorm
Announceshttps://alsnewstoday.com/2019/10/14/phase-3-nurown-trial-fully-enrolls-200-als-patients-brainstorm-says/
- ALS News Today – Advancing Potential ALS Treatments Is Like
Accelerating a
Glacierhttps://alsnewstoday.com/2019/08/14/stem-cell-research-glacier-clinical-trials-progress/
- Money Week – How stem cells are changing the face of
medicinehttps://moneyweek.com/511814/how-stem-cells-are-changing-the-face-of-medicine/
- MedPage Today – Baby Step Forward for ALS Cell
Therapyhttps://www.medpagetoday.com/meetingcoverage/aan/79680
- Multiple Sclerosis News Today – Progressive MS Phase 2 Trial of
NurOwn Stem Cell Therapy Enrolls 1st
Patienthttps://multiplesclerosisnewstoday.com/2019/03/18/progressive-ms-phase-2-trial-of-nurown-stem-cell-therapy-enrolls-1st-patient/
- Bloomberg – Namesake of ‘Right to Try’ Law Gets ALS Therapy,
Says It’s
Workinghttps://www.bloomberg.com/news/articles/2019-02-05/right-to-try-law-namesake-gets-als-therapy-says-it-s-working
- Multiple Sclerosis New Today – BrainStorm Expands Production of
Autologous Stem Cell Therapy NurOwn for Phase 2 Trial in Multiple
Sclerosishttps://multiplesclerosisnewstoday.com/2019/01/09/brainstorm-expands-production-autologous-stem-cell-technology-nurown-phase-2-trial/
Safe-Harbor Statements
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.
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