Novel Compound for Treating TBI Demonstrates Positive Results

Ischemix, Inc. recently announced significant results from a randomized, double-blinded, placebo-controlled preclinical study of its drug, CMX-2043, for the treatment of TBI. The company plans to conduct selected additional preclinical studies that will enable filing of an Investigational New Drug (IND) application and commencement of a Phase 1 trial of the candidate.

CMX-2043, a multi-modal cytoprotective compound based on a naturally occurring molecule present in the human body, was successfully tested in a randomized, placebo-controlled pre-clinical TBI treatment study conducted at Children’s Hospital of Philadelphia (CHOP). The positive results complement the favorable data generated in a prior preclinical study.

The study showed a significant reduction in the volume of brain lesions in the population treated with CMX-2043 for 5 days post-injury versus the placebo control group. The study also showed that the significant increase in night-time hyperactivity in the placebo arm versus baseline was not seen in the arms that received the drug. Increased night-time hyperactivity is a well-recognized symptom of brain injury. Additionally, there was a significant improvement in certain biochemical markers of TBI in subjects treated with CMX-2043 versus placebo. No safety issues were observed in the study.

In the prior preclinical study, CMX-2043 demonstrated a significant increase in mitochondrial bioenergetics, decreased mitochondrial reactive oxygen species and oxidative injury in the treated group versus placebo.

“We are particularly excited to note that the combined results of these two preclinical studies of CMX-2043 are the strongest of any announced preclinical results of a drug candidate currently in development for TBI,” said Reinier Beeuwkes, Chairman and Chief Scientific Officer of Ischemix, Inc. “The objective for developing this compound is to reduce the disabilities associated with TBI and increase the ability of TBI survivors to return to work and complete other activities of daily living, thus reducing economic and social costs to society.”

“The receipt of these data position Ischemix to move aggressively into further development of CMX-2043 as a treatment for TBI,” said David DeWahl, President and CEO of the Company. “We continue to develop relationships with pharmaceutical companies, not-for-profit entities and US government agencies with the objective of exploring potential collaborations to support further studies of CMX-2043.”

As part of a sponsored research agreement with CHOP, Ischemix has evaluated the effects of CMX-2043 in two preclinical studies of the drug candidate in TBI. Positive results from these first two studies established the ability of CMX-2043 to significantly reduce the size of the brain lesions in the five-day treatment group relative to placebo and to significantly improve mitochondrial energetics, reduce reactive oxygen species generation and reduce oxidative injury.

TBI is a condition for which there are currently no approved pharmaceutical therapies available. The Center for Disease Control reports that in the US in 2013, there were about 2.5 million TBI-related emergency department visits. It is estimated that each year TBI is responsible for 50,000 deaths and causes 80,000 individuals to have  permanent disabilities. It is estimated that the annual direct and indirect cost of TBI in the US approaches $77 billion.

CMX-2043 is a multi-modal cytoprotective compound based on a naturally-occurring molecule present in the human body. CMX-2043 exhibits several mechanistic properties that may act in concert to attenuate the biochemical and cellular perturbations associated with injury in TBI.  The drug has compiled a record of safety with no reported serious adverse drug-related events in its use in over 400 patients enrolled in clinical trials. CMX-2043 is the subject of multiple issued composition of matter and use patents around the world.

Ischemix is a privately held drug development company based in Grafton, MA. The company focuses on developing its family of cytoprotective compounds for serious diseases and conditions. The primary focus of the company is the development of its lead compound, CMX-2043, for treatment of TBI. For more information, visit www.ischemix.com.