Positive Phase 2 Extension Study Results for NG-01 Cell Therapy in Progressive MS

NeuroGenesis Bio Inc., a biopharmaceutical firm focused on cell therapies for neurodegenerative diseases like multiple sclerosis (MS) and amyotrophic lateral sclerosis (ALS), has reported promising interim results from a Phase 2 clinical trial of its lead product, NG-01. The trial, conducted by the MS team at Hadassah Medical Center in Jerusalem under the direction of Professor Dimitrios Karussis, showed a consistent decrease in serum levels of two key biomarkers, NfL and GFAP, which are indicative of neurodegeneration and MS progression. This reduction was observed in patients with progressive MS who received NG-01 cell therapy.

The treatment also demonstrated improvements in physical and cognitive functions, as well as patient-reported outcomes, following multiple administrations of NG-01. The U.S. Food and Drug Administration (FDA) has given the green light for a Phase 2b study, which will be a global, multi-center trial with a double-blind, randomized, and placebo-controlled design. This next phase aims to evaluate the efficacy and safety of various doses of NG-01 administered intrathecally to patients with progressive MS.

Professor Karussis, the lead investigator of the study, highlighted the significant medical need for effective treatments for progressive MS, a disease that currently lacks satisfactory options. The study's findings suggest that NG-01 not only halts disease progression for up to two years but also leads to noticeable improvements across several metrics, including clinical assessments and cognitive tests. The consistent reduction in biomarker levels after NG-01 treatment is particularly encouraging. Furthermore, the positive impact on cognitive function post-treatment could indicate a potential role for NG-01 in addressing specific central nervous system (CNS) lesions, a hypothesis to be explored in future trials.

Tal Gilat, CEO of NeuroGenesis, expressed satisfaction with the results, noting that the FDA's approval of the Phase 2b study is a significant step forward. The company is gearing up for the double-blind Phase 2 study and is collaborating with top hospitals globally. The development of NG-01, which has shown promise in treating both MS and ALS, offers hope to patients worldwide who are in desperate need of new medical solutions.

The interim analysis involved 23 out of 40 patients who received at least two doses of NG-01, 3-6 months apart, with a follow-up period of 12-18 months. The analysis assessed safety, tolerability, and efficacy, including changes in EDSS and Functional systems, 25-foot walking time, cognitive functions, and serum biomarker levels. Key findings indicated a significant reduction in NfL and GFAP levels, improved walking ability, enhanced cognitive test scores, and improved patient-reported outcomes, with no serious adverse events reported.

NG-01 is a proprietary stem cell therapy derived from a patient's bone marrow, designed to release high levels of remyelinating and neurotrophic factors at the site of CNS injury. The cells are injected directly into the CNS, where they provide neuroprotective, remyelinating, and anti-inflammatory effects.

NeuroGenesis is pioneering a unique approach to treating neurodegenerative disorders by using the patient's own stem cells to deliver sustained levels of neuroprotective and neurotrophic proteins at the site of CNS injury. The company's lead product, NG-01, is in clinical development for progressive MS and has successfully completed a placebo-controlled Phase 2 study and two open-label trials. It has also been tested in two Phase 2a trials for ALS patients, with a total of over 160 progressive MS and ALS patients treated globally through clinical trials and compassionate use programs.