FDA Lifts Hold, Clears IND for Multiple Sclerosis Stem Cell Agent IMS001

March 28, 2020

ImStem Biotechnology plans to initiate a phase 1 study of the human embryonic-mesenchymal stem cell agent sometime in 2020. Preclinical data suggest its potential to reduce MS relapses and disability progression, and encourage disease arrest.

Xiaofang Wang, MD, PhD

ImStem Biotechnology has announced that the FDA recently decided to lift the clinical hold on and clear an investigational new drug (IND) application for the company’s investigational treatment for multiple sclerosis (MS), IMS001.1

The investigational, allogeneic cell product is designed to be administered intravenously (IV), which the company stated it believes is the first such human embryonic-mesenchymal stem cell (hES-MSC). ImStem state that the initiation of a phase 1 clinical study in patients with relapsing-remitting, secondary, and primary progressive forms of MS is planned for this year.

“As an inventor of our proprietary technology, we look forward to building a company whose foundation lies in our science, based upon years of pioneering research, and we are so grateful for the continuous support from the University of Connecticut,” said Xiaofang Wang, MD, PhD, chief technology officer, ImStem Biotechnology, in a statement.

In preclinical data, IMS001 demonstrated immunomodulatory and blood-brain-barrier repair actions, which the company noted might offer therapeutic benefits across a variety of neurological, autoimmune, and rare orphan diseases that have high unmet medical needs. In MS, the properties imply a potential to reduce relapses and disability progression, and induce disease arrest.

“As a company, we are excited to have reached this clinical stage and to move one step closer to providing an effective treatment option for patients suffering from MS,” said Michael Men, MD, chief executive officer, ImStem, in a statement.

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In 2017, the company announced a joint patent for the hES-MSCs and the method for their production. At that time, Wang noted that the current therapies for MS only temporarily treat the symptoms of the disease, with sometimes severe adverse effects and costs as high as $60,000 annually. “ImStem’s technology can offer strong immunosuppression and tissue regeneration with no side effects. It is more robust than other adult stem cell therapies,” Wang said when the patent was issued.2

A 2016 study by Wang and peers of 2 animal models of autoimmune disease suggested that cells like those which comprise IMS001 express high levels of the immune inhibitory molecule programmed death-ligand 1 (PD-L1) and enabled a durable control of destructive immune system responses.3

Additionally, previous work from 2014 conducted by Wang and colleagues from ImStem and the University of Connecticut Health Center suggests that these hES-MSCs outperform MSCs derived from bone marrow in experimental autoimmune encephalitis (EAE) models of MS and are better suited for clinical treatment due to their unlimited and stable supply. Their EAE mouse model data suggest that hES-MSCs can both significantly reduce clinical symptoms and prevent neuronal demyelination. Additionally, they suggest that the disease-modifying effect of is significantly more apparent than that of bone-marrow-derived MSCs.4

“Our evidence also suggests that increased IL-6 expression by BM-MSCs contributes to the reduced anti-EAE therapeutic activity of these cells. A distinct ability to extravasate and migrate into inflamed CNS tissues may also be associated with the robust therapeutic effects of hES-MSCs on EAE,” they wrote.

REFERENCES

1. ImStem Biotechnology Announces FDA has Lifted the Clinical Hold on the Investigational New Drug Application for IMS001 for the Treatment of Multiple Sclerosis [press release]. Farmington, CT: ImStem Biotechnology; Published March 20, 2020. Accessed March 27, 2020. prweb.com/releases/imstem_biotechnology_announces_fda_has_lifted_the_clinical_hold_on_the_investigational_new_drug_application_for_ims001_for_the_treatment_of_multiple_sclerosis/prweb16992369.htm

2. UConn Spin Out Issued Stem Cell Patent for Autoimmune Disease [press release]. Farmington, CT: ImStem and UConn; Published September 12, 2017. Accessed March 27, 2020. innovation.uconn.edu/news/221/UConn%20Spin%20Out%20Issued%20Stem%20Cell%20Patent%20for%20Autoimmune%20Disease

3. Wang X, Lazorchak AS, Song L, et al. Immune modulatory mesenchymal stem cells derived from human embryonic stem cells through a trophoblast-like stage. Stem Cells. 2016;34(2):380—391. doi: 10.1002/stem.2242

4. Wang X, Kimbrel EA, Ijichi K, et al. Human ESC-Derived MSCs Outperform Bone Marrow MSCs in the Treatment of an EAE Model of Multiple Sclerosis. Stem Cell Reports. 2014;3(1):115—130. doi: 10.1016/j.stemcr.2014.04.020