This is the Year for NurOwn’s Stem Cell Phase 3 ALS Development

Brainstorm Cell Therapeutics (NASDAQ:BCLI) is involved in all stages of autologous cell therapy development for neurodegenerative diseases with a Phase 3 trial currently underway for ALS.

Dr. Ralph Kern, chief medical and chief operating officer at Brainstorm explains his company’s technology, NurOwn, takes patients own autologous stem cells and converts them into differentiated cells. The cells are then returned to the patient to “repair and regenerate the nervous system,” he said. The process involves a short procedure to derive the cells from the patients bone marrow, called a bone marrow aspiration.

The company’s lead program, for ALS, in Phase 3 development is the company’s fourth trial for the program. Taking place in six sites in the US, 200 patients will be enrolled, and is currently half enrolled. Kern said the company expects to finish enrollment by the middle of 2019 and top line data should be released in the middle of 2020.

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The trial length is 11.5 months, and consists of three intrathecal treatments. Due to the treatment schedule and overall logistics of the trial no interim results will be released. However the company did release an interim safety analysis in August 2018 for the cell therapy and ALS. Kern said the results were “reassuring and again confirms our belief that the treatment can be safely administered and well-tolerated by patients.”

Investors can look forward to another Data and Safety Monitoring Board (DSMB) read out for safety towards the second half of 2019.

In December 2018, Brainstorm announced it submitted an investigational new drug (IND) application for the same platform in progressive multiple sclerosis (MS). The US Food and Drug Administration (FDA) accepted the application in mid-December and a Phase 2

trial will begin enrollment in early 2019. There is currently no FDA approved therapy to address more than one progressive form of MS.

The platform has been tested against a number of brain diseases including Parkinson’s disease, ALS, Huntington’s disease, MS and autism among others, Kern said. “We see consistent pre-clinical results with the technology platform,” he said. The company attributes these results to the brain’s limited mechanisms to be injured and response to the injuries.

Kern said most degenerative brain diseases involve brain cell loss, and in this process inflammation occurs, the NurOwn technology addresses both of these. Neurotrophic factors are created in high levels from the cell therapy which are the “most important survival factor for neurons,” he added. And for inflammation, the company’s pre-clinical and biomarker work has aided this, which demonstrated reduction in neuroinflammation, an important driver of the disease.

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Brainstorm is one of many companies developing drugs for neurodegenerative diseases, but Kern said a big differentiating factor is “activating the body’s own repair regenerative system,” through its technology.

Unlike other therapies which are harnessing anti-inflammatory abilities, Brainstorm’s neurotrophic factors are not foreign to the body. They are produced by the body and may offer a more seamless approach to ease of repair and regeneration. Kern said he isn’t aware of another treatment modality with the same concept as NurOwn.

“Our feeling is that cells are hard wired to communicate with each other,” Kern said. We don’t think that we can do better than to activate the cell to cell communication system that our body already has, he added.

Chaim Lebovits, Brainstorm’s CEO, said the company is full financed through Phase 3, with a non-dilutive grant from the California Institute for Regenerative Medicine (CIRM). This is for 50 percent of this trial, and financed through 2019 to after the trial in 2020. CIRM is the world’s largest institution dedicated to cell therapies and awarded Brainstorm US$ 16 million in July, 2017.

Lebovits added Brainstorm continues to look at other indications “very seriously and closely.” This could mean another additional product aside from the current NurOwn platform, Lebovits said to watch out for more news involving pre-clinical research and another product in 2019.

Brainstorm’s is headquartered in New York with its research and development in Israel. The company’s technology has been in development for 10 years and seven years in clinical development. This translates into four clinical trials with the most recent on the go as the Phase 3 ALS trial.

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Securities Disclosure: I, Gabrielle Lakusta, hold no direct investment interest in any company mentioned in this article.

The Investing News Network does not guarantee the accuracy or thoroughness of the information reported in the interviews it conducts. The opinions expressed in these interviews do not reflect the opinions of the Investing News Network and do not constitute investment advice. All readers are encouraged to perform their own due diligence.

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