“We continue to enroll participants in our Phase 3 trial of NurOwn in ALS, and were delighted by the successful interim safety analysis conducted by the Data Safety Monitoring Board (DSMB),” commented Chaim Lebovits, President and Chief Executive Officer of BrainStorm Cell Therapeutics. “Moving forward, we are completely focused on taking the steps necessary to cross the finish line towards a potential U.S. FDA approval for NurOwn in ALS, and we expect successful completion of enrollment in the ongoing pivotal trial in the first half of 2019. Our goal is to bring this potentially life-changing innovative cellular therapy to patients with ALS, who are in desperate need of new treatment options.”
“Our NurOwn cellular technology platform has the potential to address the unmet need in other serious neurodegenerative diseases and we are excited to report that following a successful FDA Pre-IND Meeting we are now on track to file an IND in a second clinical indication,” added Mr. Lebovits.
Recent Corporate Highlights:
- The Connell and O’Reilly Cell Manipulation Core Facility at the Dana Farber Cancer Institute in Boston, recently commenced the production of NurOwn and placebo for the Phase 3 ALS pivotal trial as an additional manufacturing site.
- On August 23, 2018, BrainStorm announced a successful interim safety analysis in the ongoing Phase 3 pivotal trial of NurOwn in ALS (ClinicalTrials.gov Identifier: NCT03280056). o The Data Safety Monitoring Board (DSMB) completed the pre-specified interim analysis of safety outcomes. The DSMB concluded there were no significant safety concerns and that the trial should continue as planned. The DSMB Chairperson, Carlayne Jackson MD, FAAN, Professor of Neurology and Otolaryngology at the University of Texas Health Science Center San Antonio (UTHSCSA), stated: “The DSMB appreciates the continued commitment of BrainStorm and the research teams to conducting this trial in such an exemplary manner. We commend them on their outstanding enrollment and the quality of data collection.”
- On September 4, Arturo O. Araya, M.A., M.B.A., was hired as Chief Commercial Officer. o Mr. Araya was previously Global Head of Commercial for the Novartis Cell & Gene Therapies Unit. Mr. Araya led the initial efforts to build the infrastructure for the subsequent FDA approval and U.S. commercialization of KYMRIAH®.
- On September 12, Dr. Ralph Kern, Brainstorm’s CMO and COO, presented at the FDA Rare disease workshop in Washington, D.C. on the “ALS Case Study: Clinical Trial Designs for Small Patient Populations.”
- On October 3, Dr. James Berry of Massachusetts General Hospital (one of the Principal Investigators for the ALS pivotal trial), presented a clinical poster entitled, “MicroRNA Changes in the NurOwn Phase 2 ALS Randomized Clinical Trial: Relationship to Neuroprotection and Innate Immunity” at the Annual Northeast Amyotrophic Lateral Sclerosis (NEALS) conference. o The clinical poster received the “Best clinical abstract award” at the NEALS conference.
- Strengthened intellectual property estate around NurOwn: o Japanese Patent Office (JPO) issued a Decision to Grant notice on a patent that is expected to provide protection in Japan until 2033. o European Patent Office granted European-wide patent that is expected to provide protection in Europe until 2029. o U.S. Patent & Trademark Office (PTO) granted a patent that covers the method for generating NurOwn in industrial amounts for clinical practice.
Financial Results for the Three Months Ended September 30, 2018
- Cash, cash equivalents, and short-term bank deposits were $10.9 million at September 30, 2018, compared with $7.8 million at December 31, 2017. o Cash, Cash equivalents (including short-term bank deposits) and cash commitments (including the remaining commitments of $7 million from the CIRM grant and approximately $1 million under the IIA 2018 grant) amounted to approximately $19 million.
- Research and development expenses, net, for the three months ended September 30, 2018 were $2.0 million, compared to $1.2 million, net for the three months ended September 30, 2017. o Excluding participation from IIA and CIRM under the grants, research and development expenses increased by $2.5 million from $1.6 in the third quarter of 2017 to $4.1 million in the third quarter of 2018.
- General and administrative expenses for the three months ended September 30, 2018 were $1.3 million, in line with the $1.2 million reported in the three months ended September 30, 2017.
- Net loss for the three months ended September 30, 2018 was $3.2 million, or ($0.15) per share, as compared to a net loss of $2.4 million, or ($0.13) per share for the three months ended September 30, 2017.
For further details on Brainstorm’s financials, including financial results for the nine months ended September 30, 2018, refer to form 10Q filed with the SEC.
Conference Call – Monday, October 29, 2018 @ 8:30am Eastern Time
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About BrainStorm Cell Therapeutics Inc.
BrainStorm Cell Therapeutics Inc. is a leading cellular therapy biotechnology company engaged in the development of first-of-its-kind adult stem cell therapies derived from autologous bone marrow cells for the treatment of neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform through an exclusive, worldwide licensing agreement. NurOwn has received Fast Track designation from the U.S. Food and Drug Administration (U.S. FDA) in ALS and has additionally been granted Orphan Status by the U.S. FDA and the European Medicines Agency (EMA). For more information, visit BrainStorm's website at www.brainstorm-cell.com.
About NurOwn Phase 3 Clinical Program in ALS
BrainStorm is currently enrolling a Phase 3 pivotal trial investigating repeat-administration of NurOwn in ALS at six clinical sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The study will enroll 200 ALS patients, randomized 1:1 to receive NurOwn or placebo, and will evaluate the ALS functional rating scale (ALSFRS-R) responder analysis as a primary efficacy outcome measure (NCT03280056). On August 23, 2018, BrainStorm announced a successful pre-specified interim safety analyses by an independent Data Safety Monitoring Board (DSMB). The completion of Phase 3 enrollment is anticipated in first half of 2019 and is expected to support a BLA filing for FDA approval of NurOwn in ALS.
About U.S. Sites for NurOwn Phase 3 Trial in ALS
- University of California Irvine; Principal Investigator: Namita Goyal, M.D.
- Cedars-Sinai Medical Center; Principal Investigator: Robert Baloh, M.D.
- California Pacific Medical Center; Principal Investigator: Robert Miller, M.D.
- Massachusetts General Hospital; Principal Investigator: James Berry, M.D.
- University of Massachusetts Medical School; Principal Investigator: Robert Brown, M.D.
- Mayo Clinic; Principal Investigator: Anthony Windebank, M.D.
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.
CONTACTS
Corporate:
Uri Yablonka
Chief Business Officer
BrainStorm Cell Therapeutics Inc.
Phone: 646-666-3188
uri@brainstorm-cell.com
Investors:
Michael Rice
LifeSci Advisors LLC
Phone: 646-597-6979
mrice@lifesciadvisors.com
| September 30, U.S. $ in thousands | September 30, 2 0 1 8 U.S. $ in thousands | December 31, U.S. $ in thousands | December 31, 2 0 1 7 U.S. $ in thousands | |
|---|---|---|---|---|
| ASSETS | Unaudited | Audited | ||
| Current Assets: | ||||
| Cash and cash equivalents | $ | 697 | $ | 2,483 |
| Short-term deposit | 10,194 | 5,273 | ||
| Account receivable | 492 | 672 | ||
| Prepaid expenses and other current assets | 1,238 | 1,195 | ||
| Total current assets | 12,621 | 9,623 | ||
| Long-Term Assets: | ||||
| Prepaid expenses and other long-term assets | 584 | 1,408 | ||
| Property and Equipment, Net | 564 | 392 | ||
| Total long-term assets | 1,148 | 1,800 | ||
| Total assets | $ | 13,769 | $ | 11,423 |
| LIABILITIES AND STOCKHOLDERS' EQUITY | ||||
| Current Liabilities: | ||||
| Accounts payable | $ | 2,602 | $ | 1,424 |
| Accrued expenses | 428 | 817 | ||
| Deferred grant income | 130 | 2,625 | ||
| Other accounts payable | 631 | 677 | ||
| Total current liabilities | 3,791 | 5,543 | ||
| Total liabilities | $ | 3,791 | $ | 5,543 |
| Stockholders' Equity: | ||||
| Stock capital: | 11 | 11 | ||
| Common stock of $0.00005 par value - Authorized: 100,000,000 shares at each of September 30, 2018 and December 31, 2017; Issued and outstanding: 20,700,713 and 18,976,169 shares at September 30, 2018 and December 31, 2017, respectively. | ||||
| Additional paid-in-capital | 94,199 | 85,944 | ||
| Receipts on account of shares | 4,408 | - | ||
| Accumulated deficit | (88,640) | (80,075) | ||
| Total stockholders' equity | 9,978 | 5,880 | ||
| Total liabilities and stockholders' equity | $ | 13,769 | $ | 11,423 |
| Nine months ended September 30, 2018 Unaudited | Nine months ended September 30, 2017 Unaudited | Three months ended September 30, 2 0 1 8 Unaudited | Three months ended September 30, 2 0 1 7 Unaudited | |
|---|---|---|---|---|
| Operating expenses: | ||||
| Research and development, net | $ 4,433 | $ 2,544 | $ 1,975 | $ 1,168 |
| General and administrative | 4,193 | 2,693 | 1,257 | 1,224 |
| Operating loss | (8,626) | (5,237) | (3,232) | (2,392) |
| Financial expenses (income), net | (61) | (9) | (56) | 11 |
| Net loss | $ (8,565) | $ (5,228) | $ (3,176) | $ (2,403) |
| Basic and diluted net loss per share from continuing operations | $ (0.43) | $ (0.28) | $ (0.15) | $ (0.13) |
| Weighted average number of shares outstanding used in computing basic and diluted net loss per share | 19,754,159 | 18,737,307 | 20,691,900 | 18,783,997 |
BrainStorm Cell Therapeutics Inc.