UConn Spin-off ImStem Biotechnology to Move Forward with Clinical Trial of Investigational New Drug for Treatment of Multiple Sclerosis

Just as the United States was recognizing the severity of the coronavirus and state governments were reacting to the impending health crisis, a Farmington-based company that has been pursuing a cure for another elusive disease received milestone news from the U.S. Food and Drug Administration (FDA).

ImStem Biotechnology, a biopharmaceutical company located in UConn’s Technology Incubation Program (TIP) in Farmington, has been trying to revolutionize how serious diseases are treated with a new generation of regenerative and cellular therapies. 

Their initial target is Multiple Sclerosis, and their years-long research and development efforts were bolstered when the FDA announced on March 20 that the clinical hold on ImStem’s Investigational New Drug (IND) to evaluate their cell product candidate, IMS001, for the treatment of multiple sclerosis (MS) has been given the okay to proceed.

The publication BOLD Business described it this way: “Treatments are available for the disease, but a cure for MS remains out of reach to date. However, companies like ImStem Biotechnologies are investigating new areas of research to hopefully change that. Their research with stem cell treatments looks quite promising.”

The lifetime costs for an individual with MS exceeds $4 million. A recent study found that nearly 1 million people over the age of 18 in the US have been diagnosed with MS.

Multiple sclerosis is an autoimmune disease.  It affects the brain and spinal cord. Antibodies attack the myelin coating on these nerves, which can result in a variety of problems. Paralysis, numbness, incoordination, double vision, and blindness are just a few of the more severe effects. The underlying cause of the disease is still unknown, the business publication explained.

The company plans to initiate a phase 1 clinical study in patients with relapsing-remitting, secondary, and primary progressive forms of MS this year in the U.S.  It is unclear at this time of the coronavirus pandemic may cause a slight delay in those plans. ImStem believes IMS001, is the first allogeneic, human embryonic stem cell derived mesenchymal stem cell therapy of its kind to be accepted for clinical trial by the FDA

ImStem’s first product, it is based on pioneering research from two co-founders, chief technology officer, Dr. Xiaofang Wang, and Dr. Ren-He Xu, former director of the UConn Stem Cell Institute, UConn Today reported last month. Xu is one of a handful of scientists in the world who have generated new human embryonic stem cell lines. Named CT1 and CT2, the lines were a major success for Connecticut’s $100 million stem cell program.

“After our 8-year research on hES-MSC at the University of Connecticut and the University of Macau, this is really fantastic news for us to advance IMS001 as a potential cell drug to a clinical trial and realize the therapeutic value of human embryonic stem cells in patients,” said Dr. Xiaofang Wang, Chief Technology Officer of ImStem Biotechnology.  “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.” 

Since it was incorporated in 2012, the company has focused on the development of human embryonic stem cell derived mesenchymal stem cells (hES-MSC), through a proprietary method for the treatment of neurological, autoimmune, and rare orphan diseases.

The technology builds on previous studies with researchers from UConn Health that demonstrated potential advantages of hESC-MSCs that could lead to effective therapies for MS, such as their potential to stabilize the blood-brain barrier. These mechanistic properties could reduce MS relapses, the progression of disability caused by the disease, and induce disease arrest, according to the ImStem team.

“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, chief executive officer of ImStem.

These mechanistic properties may lead to therapeutic benefits in diseases such as MS, potentially reducing relapses, disability progression, and inducing disease arrest, the company said.  The company aspires to revolutionize how serious diseases with significant unmet needs are treated with a new generation of regenerative and cellular therapies.

Pioneering research by its current founder and Chief Technology Officer Dr. Xiaofang Wang and Dr. Ren-He Xu, former director of UConn Stem Cell Institute, led to the proprietary state-of-the-art pluripotent stem cell technology, enabling off-the-shelf, allogeneic stem cell-derived products to be manufactured in scale, differentiating itself from the typical challenges imposed by autologous adult cell therapy products.

The company’s mission is to advance the science and understanding of human pluripotent stem cell based regenerative cellular therapies through novel and creative development pathways and to fulfill unmet medical needs in serious diseases. And its development strategy focuses on neurologic, autoimmune, degenerative, and rare orphan diseases.