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Results published in Brain show significant beneficial effects in patients with progressive multiple sclerosis after treatment with NG-01 stem cells

NEW YORK, NY: December 8, 2020 – NeuroGenesis, a clinical-stage biopharmaceutical company advancing innovative cell therapies to combat myelin-related neurodegenerative diseases, and Hadassah Medical Organization (HMO) in Jerusalem announced today highly positive results from a placebo-controlled Phase 2 clinical trial. The trial, which was conducted by Prof. Dimitrios Karussis, lead principal investigator and Director of MS Center at Hadassah Hospitals, together with Dr. Petrou Panayiota and Dr. Ibrahim Kassis, assessed the impact of NG-01 autologous proprietary subpopulation of mesenchymal stem cells (MSCs) on patients with progressive multiple sclerosis (MS). 

The results, recently published in Brain, a prestigious peer-reviewed journal published by Oxford University, and highlighted in “Editor’s Choice," show that:

• No serious, treatment-related safety issues were detected;
• Significantly fewer patients experienced treatment failure (disease progression) in the intrathecal (IT) and intravenous (IV) NG-01 treatment groups compared with those in the placebo group (6.7%, 9.7%, and 41.9%, respectively, P = 0.0003 and P = 0.0008);
• 58% of the patients treated intrathecally with NG-01 did not show any evidence of disease activity (NEDA) during the entire the treatment period (vs. 9.7% in the placebo treated group) (P<0.0001);
• NG-01 treatment groups demonstrated a significant improvement in walking ability as measured by 25-foot walking time (P=0.0017); and
• Intrathecal administration of NG-01 was more efficacious than intravenous in several key parameters of the disease: relapse rate (89% decrease in the relapse rate), functional MRI (improvement of motor networks), monthly changes of the MRI T2 lesion load and the 9-hole peg test, as compared to the control (placebo-treated) group.

"The treatment was well tolerated and the trial met all of its primary endpoints,” said Prof. Dimitrios Karussis, lead principal investigator and Director of the MS Center at Hadassah Hospitals. “The patients’ improvement was in many cases quite remarkable and included regain of motor function and noticeable effects on their cognitive abilities.”

Prof. Karussis added, “Although we currently have several good treatment options for relapsing remitting MS, we have fallen short of providing effective treatment for progressive MS that could substantially suppress the progression of disability. This trial provides encouraging results and suggests the potential for a new approach that may not only slow down the progression of the disease but even induce improvement and promote repair mechanisms in progressive MS.”

The technology is now further developed by NeuroGenesis, following a license from Hadasit, Hadassah Hospitals’ Technology Transfer Company.

Neurogenesis' technology entails collecting bone marrow from the patient. Then, by utilizing a proprietary process, a unique subpopulation of bone marrow cells is identified, cultured and enhanced towards remyelinating biofactory cells (NG-01) that also possess neurotrophic immunolatory and neuroprotective properties. The NG-01 cell population is injected directly into the central nervous system (through the cerebrospinal fluid), where the cells home in on the damaged area, take up residence and produce significant amounts of neurotrophic factors.

"Progressive MS is a chronic, debilitating disease with no satisfactory treatment to improve or reverse established disability," said Tal Gilat, CEO of NeuroGenesis. "We are therefore extremely pleased to witness the significant positive effect of our NG-01 cells. Following recent interactions with the FDA, we look forward to confirming and expanding these findings in a large multi-center MS trial, and continuing advanced studies in additional indications such as ALS.”

About the Phase 2 trial of NG-01
The Phase 2, randomized, double-blind, placebo-controlled clinical trial assessed the safety, tolerability and efficacy of transplantation of NG-01 in people with progressive MS. The study enrolled 48 participants with progressive MS who were randomized into three groups each of which received either an intrathecal or intravenous NG-01 injection or a placebo injection.

The two predetermined primary endpoints of the trial were (i) the safety of the intrathecal and intravenous NG-01 treatments assessed by incidence of adverse events versus those in the placebo-treated group and (ii) the differences among the three groups in the Expanded Disability Status Scale (EDSS) score changes and the proportion of patients with treatment failure, as evidenced by an increase in EDSS (disease progression) score at 6 and 12 months. Overall, the study duration was 14 months.

About Multiple Sclerosis 
Multiple sclerosis (MS) is an autoimmune disease that causes damage in the myelin and the nerve cells of the central nervous system (demyelinating plaques in brain and spinal cord), resulting in cumulating neurological disability. The destruction of the myelin (the covering that protects nerves and promotes the efficient transmission of nerve impulses) causes secondary damage to the nerve cells and progressive atrophy. MS often causes sensory disturbances in the limbs, including a prickling or tingling sensation (paresthesia), numbness, pain and itching. Motor problems are common in people with MS. Affected individuals may have tremors, muscle stiffness (spasticity), exaggerated reflexes (hyperreflexia), weakness or paralysis of the muscles of the limbs, difficulty in walking and poor sphincter control. The condition is also associated with visual problems, such as blurred or double vision or partial or complete vision loss. There is no known cure for multiple sclerosis. The existing treatments are mostly aimed to reduce the incidence of relapses of the disease and slow down the rate of neurological deterioration.

About NeuroGenesis
NeuroGenesis is developing cell therapy for neurodegenerative diseases based on a unique approach for sustained delivery of high levels of remyelinating growth factors using the patient’s own stem cells. The technology for this unique approach was licensed from Hadasit, Hadassah Hospitals’ Technology Transfer Company. The Company's lead product is NG-01 for the treatment of progressive multiple sclerosis (in which a placebo-controlled Phase 2 study has been completed and recently published). NG-01 were also tested in two successful Phase 2a trials in ALS patients. Up to today, more than 150 progressive MS and ALS patients from around the world have been treated with Neurogenesis' products via clinical trials (Phase 1 and Phase 2) and compassionate use treatments.

About Hadassah Medical Organization (HMO) and Hadasit
For more than a century, Hadassah Medical Organization (HMO) has set the standard of excellence for medical care and research in Israel. Our doctors and scientists are on the front lines, uniquely positioned to pinpoint ever-evolving medical needs. Their experience and ingenuity have yielded new ideas with huge potential in all areas of medicine, including therapeutics, diagnostic medical devices and digital health. Hadasit is Hadassah Hospitals’ Technology Transfer Company. We transform the cutting-edge research coming out of Hadassah Hospitals into marketable medical technologies. We turn ground-breaking ideas into viable products and services that can change the world and better humanity.