Topline Knowledge from Part Three ALS Trial Earlier than Finish of November 2020
BrainStorm Cell Therapeutics, Inc. (NASDAQ:BCLI) is at present conducting a Part Three medical trial of NurOwn® in sufferers with amyotrophic lateral sclerosis (ALS) (NCT03280056). A complete of 200 sufferers have been randomized 1:1 to obtain NurOwn® or placebo within the randomized, double blind, placebo managed, multi-dose trial. Cells have been extracted as soon as from every affected person previous to remedy, with all administrations of NurOwn® derived from the identical extraction of cells as a result of a cryopreservation course of the corporate developed for long-term storage of mesenchymal stem cells (MSC). Simply as with the corporate’s prior research, there was a Three-month run-in interval previous to the primary remedy with two further NurOwn® remedies occurring two and 4 months following the primary remedy. The corporate is focusing the trial on faster-progressing ALS sufferers since these sufferers demonstrated superior outcomes within the Part 2 trial of NurOwn®. The first consequence of the trial is the ALSFRS-R rating responder evaluation and we now anticipate topline outcomes earlier than the top of November 2020.
The next picture supplies an illustration of the responder evaluation. Every individuals ALSFRS-R knowledge is match with a linear regression mannequin for the pre-treatment interval (12 weeks) and the post-treatment interval (28 weeks). This offers an estimate of illness development over time. A responder is outlined as a participant with a ≥1.25 factors/month enchancment in ALSFRS-R post-treatment slope in comparison with the pre-treatment slope. The graphs proven are for illustration functions solely and don't characterize precise knowledge from the trial.
Replace on Part 2 Progressive A number of Sclerosis Trial
BrainStorm is presently conducting a Part 2 medical trial of NurOwn® in sufferers with progressive a number of sclerosis (MS) (NCT03799718). The trial is an open label, single arm research that's enrolling sufferers with progressive MS with Expanded Incapacity Standing Scale (EDSS) scores of three.zero – 6.5. The first endpoint of the research is the security and tolerability of three doses of NurOwn® with secondary endpoints analyzing the timed 25-foot strolling velocity or 9-hole peg check (each validated MS medical consequence assessments) together with paired cerebrospinal fluid (CSF) and blood biomarker evaluation. The Nationwide A number of Sclerosis Society awarded the corporate a $zero.5 million grant to assist fund the research.
The trial is now absolutely enrolled and we anticipate dosing to be accomplished for all sufferers by the top of 2020. Whereas the corporate had beforehand thought-about performing an interim evaluation, since topline knowledge can be out there quickly after an interim evaluation might be carried out the corporate has determined towards performing an interim evaluation and can as an alternative report topline knowledge for all 20 sufferers when it turns into out there.
Enlargement of Administration Workforce
In the course of the third quarter of 2020, BrainStorm introduced the hiring of two further members of the administration group as the corporate strikes towards turning into a business stage entity.
• On September, 2, 2020, BrainStorm announced the appointment of Anthony Waclawski, PhD as Government Vice President, International Head of Regulatory Affairs. Dr. Waclawski has over 35 years of multinational pharmaceutical expertise within the FDA regulatory approval course of, together with most just lately as Vice President and Head, Regulatory and Pharmaceutical Sciences at Bristol-Myers Squibb. His expertise is throughout a number of therapeutic areas and geographies that features executing profitable international regulatory methods.
• On September 29, 2020, BrainStorm announced the appointment of William White as Senior Vice President, Head of Market Entry and Pricing. Mr. White has over 25 years of expertise main profitable product commercializations, together with most lately with Avexis (now Novartis Gene Remedy) as Vice President of Affected person and Market Entry. He additionally held management positions in managed care and affected person entry at Insys Therapeutics and Lundbeck.
On October 15, 2020, BrainStorm announced monetary outcomes for the third quarter of 2020. As anticipated, the corporate didn't report any revenues through the third quarter of 2020. Internet R&D bills for the third quarter of 2020 have been $1.9 million, in comparison with $four.zero million for the third quarter of 2019. The lower was primarily because of decreased prices associated to the Part Three and Part 2 medical trials and elevated participation of the Israel Innovation Authority (IIA) and California Institute for Regenerative Drugs (CIRM) beneath numerous grant awards. Excluding participation from the IIA and CIRM, R&D bills have been $four.zero million within the third quarter of 2020 in comparison with $5.6 million within the third quarter of 2019. G&A bills for the third quarter of 2020 have been $2.6 million in comparison with $1.5 million for the third quarter of 2019. The rise was primarily because of elevated payroll, PR, advisor, and stock-based prices.
The corporate exited the third quarter of 2020 with roughly $28.eight million in money, money equivalents, and short-term investments. Subsequent to the top of the quarter, the corporate raised gross proceeds of roughly $5.1 million by way of the amended ATM facility initially entered into in March 2020 and amended on September 25, 2020. From September 25, 2020 via Oct. 14, 2020, the corporate bought 294,723 shares at a mean worth of $17.21 per share and as of October 14, 2020 the corporate has roughly $33.1 million in money, money equivalents, and brief time period deposits.
As of September 31, 2020, the corporate had roughly 31.6 million shares excellent and, when factoring in warrants and inventory choices, a totally diluted share rely of roughly 37.1 million.
The subsequent anticipated information from the corporate is the topline knowledge launch for the Part Three ALS trial, with these outcomes anticipated earlier than the top of November 2020. The corporate has already begun planning for a BLA submitting upon constructive outcomes from the Part Three trial and is contemplating potential paths to expediting the approval course of. As well as, given the current delay in submitting a BLA introduced by Iovance Therapeutics (IOVA) for its cell remedy on account of an lack of ability to return to an settlement with the FDA on a required efficiency assay (a requirement for a BLA submitting), BrainStorm is conscious of the significance of such an assay for NurOwn and might be discussing the specifics of such an assay with the FDA previous to submitting a BLA. We're assured the corporate may have a professional assay in place in time for a BLA submitting such that a delay like Iovance’s won't happen. With no modifications to our mannequin our valuation stays at $33 per share as we anxiously await the outcomes of the Part Three ALS trial.
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