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

Biotech Investing

Brainstorm Cell Therapeutics’ management team discusses an ongoing ALS Phase 3 clinical trial, the company’s technology and more.

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.

In the interview above, Dr. Ralph Kern, chief medical and chief operating officer at Brainstorm, explains that his company’s technology, NurOwn, takes patients’ 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 bone marrow of patients, called a bone marrow aspiration.

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

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 confirm 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 read out for safety towards the second half of 2019.

In December 2018, Brainstorm submitted an investigational new drug 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 issues. Neurotrophic factors are created in high levels from the cell therapy, and these are the “most important survival factor for neurons,” he added. The company’s pre-clinical and biomarker work has aided with inflammation, demonstrating a reduction in neuroinflammation, an important driver of the disease.

Brainstorm is one of many companies developing drugs for neurodegenerative diseases, but Kern said a differentiating factor is that its technology can “activat[e] the body’s own repair regenerative system.”

Unlike other therapies that 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 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. The company doesn’t think it can do better to activate the cell-to-cell communication system that our bodies already have, he added.

Chaim Lebovits, Brainstorm’s CEO, said the company is fully financed through Phase 3, with a non-dilutive grant from the California Institute for Regenerative Medicine (CIRM). This is for 50 percent of the trial, and the company is 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 that Brainstorm continues to look at other indications “very seriously and closely.” This could mean another additional product aside from the current NurOwn platform. Lebovits also said to watch out for more news involving pre-clinical research and another product in 2019.

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

Editorial Disclosure: 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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