BrainStorm Update Ensures Funds for Completion of Phase 3 NurOwn Trial, Announces 2 New Patents

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by José Lopes, PhD |

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BrainStorm Cell Therapeutics’ latest financial results guarantee enough capital to complete the ongoing Phase 3 trial of amyotrophic lateral sclerosis (ALS) therapy candidate NurOwn, according to an update from the company.

In addition, the company recently announced it has received patents in Japan and Europe for NurOwn.

“We are pleased with our accomplishments in the second quarter of 2018,” Chaim Lebovits, BrainStorm’s president and CEO, said in a press release.

BrainStorm’s randomized, double-blind, multicenter Phase 3 clinical trial (NCT03280056) is aimed at assessing the safety and effectiveness of three administrations of NurOwn into the spinal canal in approximately 200 adults with ALS. Participants receive either NurOwn or placebo every two months.

The trial is currently recruiting participants at six U.S. sites. BrainStorm plans to complete the study by July 2019.

NurOwn is an investigative therapy based on patients’ own mesenchymal stem cells (MSCs), which are able to generate various cell types. The treatment is intended to transform these cells into sources of molecules called neurotrophic factors to promote nerve tissue growth and survival after being transplanted back into patients.

The patents Brainstorm recently received for NurOwn from Japan and Europe cover the methods used to treat ALS with MSCs. The Japanese patent, “Methods of generating mesenchymal stem cells which secrete neurotrophic factors,” provides protection until 2033, while the European-wide patent, “ ‘Mesenchymal Stem Cells for the Treatment of CNS Diseases’ for the treatment of Amyotrophic Lateral Sclerosis (ALS) with NurOwn,” is valid until 2029.

“Having a EU patented product should increase our ability to enter into new commercial partnerships for NurOwn in Europe,” Leibovits said in a separate press release.

Earlier this month, BrainStorm announced a partnership with the Connell and O’Reilly Families Cell Manipulation Core Facility at Dana-Farber Cancer Institute for the production of NurOwn. It is now the second U.S. manufacturing site for the therapy candidate after the City of Hope’s Center for Biomedicine and Genetics.

“We continue to execute on the ongoing Phase 3 trial investigating NurOwn in ALS and were pleased to sign Dana-Farber Cancer Institute as the second U.S. manufacturing site for this trial,” Leibovits said.

In its effort to strengthen the senior management team, BrainStorm appointed Susan Ward, PhD, head of clinical operations, and Joseph Petroziello vice president of scientific and corporate communications.

“We are continuing to expand our senior management team with a series of key appointments as we transform BrainStorm to a global biopharmaceutical stem cell therapeutic company and build our U.S. infrastructure,” Leibovits said.

The company’s second quarter financial results, which covers the period between April 1 and June 30, show an increase of $9.6 million in cash, cash equivalents (short-term investments), and short-term bank deposits, as compared with Dec. 31, 2017.

Including other types of investments, the company now has approximately $18.2 million, which does not include a California Institute for Regenerative Medicine (CIRM) grant and other resources.

BrainStorm’s net research and development expenses totaled $1.5 million from April to June and $2.5 million from January to June. The three-month total was $3.3 million excluding the CIRM grant and other funds received, in comparison to $900,000 in the second quarter of 2017.

“We now have sufficient capital to not only complete our ongoing Phase 3 trial of NurOwn in ALS but also to advance the development of other promising [research and development] programs in our pipeline,” Leibovits said.