NeuroGenesis and Hadassah Report Positive NF-L Biomarker Results from a Phase 2 Study in Progressive Multiple Sclerosis Treated with NG-01 Cells Therapy

NeuroGenesis, a clinical-stage biopharmaceutical company advancing innovative cell therapies to combat myelin-related neurodegenerative diseases, and Hadassah Medical Centre announced last week positive results from a placebo-controlled Phase 2 clinical trial assessing the impact of NG-01 autologous proprietary subpopulation of bone marrow cells on the expression levels of NF-L, a commonly-used biomarker for multiple sclerosis (MS) and other neurogenerative diseases, in patients with progressive MS.

NF-L is a protein released into the cerebrospinal fluid (CSF) by damaged neurons and is a reliable biomarker for neuronal damage in patients with MS and other neurodegenerative diseases. High levels of NF-L are consistently found in the CSF of MS patients and can be used as a biomarker of MS disease activity as well as of the response to various MS-treatments.

The Clinical Trial

• The Phase 2, randomised, 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 which were randomised into 3 groups, receiving either an intrathecal (IT) or intravenous (IV) NG-01 injection, or a placebo injection.
• CSF samples were obtained from the study participants at baseline and at 6 months following treatment with NG-01 or placebo, and the level of the NF-L biomarker was assessed in each sample. The samples of 4 patients were not technically testable for NF-L
• The trial was headed by Prof. Dimitrios Karussis, together with Dr. Petrou Panayiota and Dr. Ibrahim Kassis, all from Hadassah Medical Centre in Jerusalem. The results were recently published in Stem Cells Translational Medicine.

The results

• Reduction of NF-L levels correlated with significant functional improvement. Eight out of the 9 patients in the IT group that experienced a >50% reduction in the NF-L levels were stable or improved in EDSS at 6 months and experienced an improved disability score even after a follow up of 12 months (end of study).
• In the same study, Hadassah researchers also reported a clear trend of reduction in the proinflammatory chemokine in the CSF following intrathecal treatment with NG-01, indicating local immunomodulation in the CNS, induced by the stem cell therapy.
• The NF-L results provide the first reported objective evidence of neuroprotection induced by the intrathecally injected stem cells and are in line with the encouraging NG-01 clinical results in two long term trials in progressive MS and ALS published in Frontiers of Neurology[i] and Frontiers in Bioscience[ii], respectively.
• The MS trial showed that repeated NG-01 treatments were safe at the short/intermediate term and induced clinical benefits, that lasted for up to four years, especially in patients treated with more than two NG-01 injections.
• More information on the long-term trials can be found in www.ClinicalTrials.gov (identifier of the MS trial: NCT04823000 and identifier of the ALS trial: NCT04821479).

Professor Dimitrios Karussis, lead principal investigator and Director of MS Centre at Hadassah Medical Centre, Jerusalem:

“Progressive MS is a debilitating disease without effective treatment options that can substantially suppress the progression of disability. This novel trial evaluated not only the clinical effects induced by the stem cell therapy, but also the effects on the most reliable and accepted biomarker of neuro-degeneration, NFL. ‘’

‘’We therefore believe that our findings are extremely encouraging and taken together with the long-term clinical results, that showed stabilization and even functional improvement in 22 out of the 24 participants up to 4 years after the initiation of treatment, provide substantial optimism for the future of management of progressive MS and neurodegenerative diseases in general.”

“We are extremely pleased to witness the significant protective effect of our NG-01 cells on NF-L and the very encouraging long term clinical results in MS and ALS,” said Tal Gilat, CEO of NeuroGenesis. We are currently working on expanding our manufacturing capacity in the US in preparation for our multi-center Phase 2b trial in secondary progressive MS that is planned to begin in late 2022.”

NeuroGenesis’ technology entails collecting bone marrow from the patient. Then by utilising 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 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.

NeuroGenesis contact:

Tsipi Haitovsky, Global Media Liaison

+972-52-5989-892 or Tsipihai5@gmail.com

Hadassah Medical Organisation contact:

Hadar Elboim, Spokeswoman

+ 972- 2-6776079 or hadare@hadassah.org.il

Editors Notes:

NEWS PROVIDED FOR PRNEWSWIRE.COM BY: NeuroGenesis, Hadassah Medical Centre, 01 March, 2022

NeuroGenesis and Hadassah Medical Center Report Positive NF-L Biomarker Results from a Phase 2 Study in Progressive Multiple Sclerosis Treated with NG-01 Cells Therapy (prnewswire.com)

Results, published in Stem Cells Translational Medicine, show significant reduction of NF-L, a biomarker for neuronal damage, in patients with progressive multiple sclerosis after treatment with NG-01 stem cells. Long term NG-01 trials in progressive MS and ALS were also published in Frontiers of Neurology and Frontiers in Bioscience, respectively.