NeuroGenesis Reports Positive Results from Phase 2 Extension Study in Progressive Multiple Sclerosis Treated with NG-01 Cell Therapy

2024-03-12

临床2期临床结果细胞疗法

Data presented by the MS team at Hadassah Medical Center, Jerusalem, led by Prof. Dimitrios Karussis, in the Americas Committee for Treatment and Research in Multiple Sclerosis Forum reveal consistent reductions in serum levels of both NfL and GFAP, the most reliable biomarkers for neurodegeneration and prediction of MS progression, in patients with progressive MS following treatment with NG-01 cells

Improvement in physical and cognitive parameters of MS and in patient-reported outcomes, were also observed, following repeated treatments with NG-01

The FDA has cleared a Phase 2b, global, multi-center study with a double-blind, randomized, and placebo-controlled design to test the efficacy and safety of different doses of intrathecal administration of NG-01 in progressive MS patients

ROCHESTER, N.Y., March 12, 2024 /PRNewswire/ -- NeuroGenesis Bio Inc., a clinical-stage biopharmaceutical company advancing innovative cell therapies to combat neuro-inflammation and demyelination in neurodegenerative diseases such as multiple sclerosis (MS) and Amyotrophic Lateral Sclerosis Amyotrophic Lateral Sclerosis (ALS), announced today positive results from the interim analysis of the open-label extension period of a randomized, double-blind, placebo-controlled, Phase 2 clinical trial assessing the long-term effects of its lead asset, NG-01, on the two main serum biomarkers of MS activity and progression, on physical and cognitive assessments, and patient-reported outcomes in progressive MS. The results were recently presented by the MS team at Hadassah Medical Center, Jerusalem, led by Prof. Dimitrios Karussis, at the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum, held in West Palm Beach, Florida on March 2nd, 2024.

"There is a significant unmet medical need for progressive MS, which is a debilitating disease without effective treatment options," said Prof. Karussis, lead principal investigator of the study. "We are therefore excited to see that NG-01 not only prevented the progression of the disease for a rather long period of up to two years, but also led to demonstrable improvement in patient condition measured by multiple parameters— clinical assessments, cognitive tests, and objective biomarkers. Serum levels of NfL and GFAP were evaluated at repeated time points after each NG-01 treatment as the key biomarkers that correlate with neuroinflammation and neurodegeneration in progressive MS. We are encouraged to see that NG-01 treatment consistently reduced the levels of both biomarkers. Additionally, the positive effect on cognitive function after NG-01 treatment may point to a potential role of these cells in targeting compartmentalized CNS lesions, such as PRLs (paramagnetic lesions), that is to be tested in subsequent clinical trials. The extension study results underscore the possibility of a novel, safe, and long-acting therapy for stabilizing and/or reversing the progression of MS, especially in its progressive form, known as PIRA (Progression Independent of Relapse Activity), that advances "silently" and is very resistant to the existing conventional medical treatments."

"We are extremely pleased with these promising results of our NG-01 cells in patients with progressive MS," said Tal Gilat, CEO of NeuroGenesis. "Following the observations on the longitudinal decrease in NfL and GFAP, the FDA has cleared our Phase 2b, global, multi-center study with a double-blind, randomized, and placebo-controlled design to primarily test the efficacy and safety of different doses of intrathecal administration of NG-01 in progressive MS patients. We are now preparing for the Phase 2 double-blinded study and have already initiated work with multiple leading hospitals around the world. We look forward to the continued development of this cutting-edge treatment that has shown promise not only in MS but also in ALS, and thus may offer hope for hundreds of thousands of patients worldwide who currently have no alternative medical solution."

The interim analysis was conducted on 23 out of 40 patients who were given at least two administrations of NG-01,in addition to their standard-of-care, 3-6 months apart with a follow up of 12-18 months. In this pre-specified interim analysis, safety, tolerability and various efficacy measurements— changes in EDSS and Functional systems, timed 25-feet (25FT) walking, cognitive functions, and changes in the serum levels of the biomarkers NfL (neurofilaments-light chains) and GFAP (that indicate neurodegeneration and neuroinflammation)— were assessed.

The main findings include:

Results show a significant reduction in serum levels of NfL and GFAP, the two most reliable biomarkers that are correlated with MS-activity, neurodegeneration and prediction of progression of the disease. The mean reduction in NfL was 33.2% in 20 patients from baseline to the last observation after 2nd dosing (p nd dosing (p cognitive test was improved by a mean of ~3 degrees (p=0.0008). In addition, Patient reported outcomes (mental and fatigue questionaries) were all improved by 18-33% (p nd dose (>12 months).

No serious adverse events were observed during the whole period of the trial. Minor adverse events included headache in 9 subjects and backache in 2 subjects, which were considered by the investigators, as procedure-related and resolved within 48 hours after the lumbar puncture.

The Phase 2, randomized, double-blind, placebo-controlled, clinical trial (NCT02166021), which was performed by the Hadassah-MS team led by Prof. Karussis, Dr. Petrou and Dr. Kassis, assessed the safety, tolerability, and efficacy of transplantation of NG-01 in people with progressive MS. The trial demonstrated significant improvement and repair results which were published in BRAIN 2020 as the Editor's Choice. The original study enrolled 48 participants with progressive MS who were randomized into 3 groups, receiving either an intrathecal (IT) or intravenous (IV) NG-01 injection, or a placebo injection. In the extension phase, patients were treated with 1-3 intrathecal injections of NG-01 at intervals of 3-6 months.

About NG-01

NG-01 is a proprietary population of autologous bone marrow-derived stem cells designed to secrete high levels of remyelinating and neurotrophic factors (NG-01) at the central nervous system site of injury. The NG-01 cells are injected directly into the central nervous system (through the cerebrospinal fluid), where the cells settle close to the damaged areas or lesions in the brain and spinal cord, and exert significant neuroprotective, remyelinating, and anti-inflammatory effects.

About NeuroGenesis

NeuroGenesis is developing cell therapies for neurodegenerative disorders based on a unique approach that allows sustained delivery of high levels of neuroprotective, neurotrophic, and anti-inflammatory proteins at the site of CNS injury using the patient's own stem cells. The company's lead product is NG-01 that is under clinical development for the treatment of progressive Multiple Sclerosis, an indication for which a placebo-controlled Phase 2 study as well as two open label trials have been successfully completed. NG-01 was also tested in two successful Phase 2a trials in ALS patients. To date, more than 160 progressive MS and ALS patients combined, have been treated globally with NeuroGenesis' lead product via clinical trials and compassionate use treatments.

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Tsipi Haitovsky

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NeuroGenesis

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SOURCE NeuroGenesis Bio Inc.