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Ischemix, Inc. Completes Successful Phase 1 Trial of Novel Compound to Treat Traumatic Brain Injury (TBI); Preparing for Conduct of Phase 2 Trial

August 7, 2023

CMX-2043, a novel, proprietary cytoprotective drug candidate, demonstrated excellent safety and tolerability in a Phase 1 single ascending and multiple ascending dose (SAD-MAD) clinical trial.

CMX-2043 has previously demonstrated safety and efficacy in extensive preclinical pharmacology, toxicology and TBI translational studies

CMX-2043 will be Phase 2-ready by the first quarter of 2024

GRAFTON, MA - Ischemix, Inc., a privately-held company focused on developing cytoprotective therapeutics for serious medical conditions, today announced that it has completed an 80-subject Phase 1 clinical trial to evaluate safety, tolerability and pharmacokinetics of its drug candidate, CMX-2043, in healthy human subjects.

The Phase 1 study was a two-part, double-blinded, randomized, placebo-controlled trial in which single ascending doses (SAD) of CMX-2043 were administered by brief intravenous infusion to 40 healthy male and female subjects. After completion of the SAD portion of the trial, 40 additional healthy male and female subjects were randomized to receive multiple ascending doses (MAD) of CMX- 2043 or placebo by daily intravenous infusion for five days. “This Phase 1 trial demonstrates that CMX-2043 has the potential for an excellent safety profile when given intravenously over a wide range of doses. There were no serious adverse events and all treatment-emergent adverse events were mild and self-limiting. Importantly, the Principal Investigator determined that few adverse events were drug-related.” said Jerry O. Stern, MD, Chief Medical Officer of Ischemix. CMX-2043 also was shown to have a well characterized pharmacokinetic profile demonstrating dose proportionality over the dose ranges studied.”

“Successful accomplishment of the Phase 1, along with the anticipated completion of other Phase 2-enabling non-clinical studies, will allow us to conduct a meeting with FDA early in the first quarter of 2024.” said David DeWahl, President and CEO of the Company. “We are currently in the process of designing the protocol for a Phase 2 trial of CMX-2043 in TBI patients.”

CMX-2043 has previously demonstrated safety and efficacy in rodent models at two laboratories and in porcine models at two additional laboratories. These successful preclinical efficacy results were achieved in endpoints measuring cognition, behavior, motor skills and MRI analysis.

Altasciences Company, Inc. a contract research organization offering a broad array of drug development services, performed the Phase 1 trial at its Montreal, Quebec, Canada facility.

The Phase 1 trial and certain related costs were funded in part by an award granted to Ischemix by US Army Medical Research and Development Command (USAMRDC), Combat Casualty Care Research Program (CCCRP), in collaboration with the Medical Technology Enterprise Consortium (MTEC), a 501(c)(3) biomedical technology consortium. (a)

About TBI

There are currently no TBI-specific pharmaceutical therapies approved for the treatment of patients suffering a brain injury. The Center for Disease Control reports that in the US in 2014, there were approximately 2.9 million TBI- related emergency department visits. It is estimated that each year TBI is responsible for approx. 60,000 deaths and causes 80,000 individuals to have permanent disabilities. The annual direct and indirect costs of TBI in the US are believed to be approx. $77 billion.

About CMX-2043

CMX-2043 is a pleiotropic (multi-modal), cytoprotective, small molecule drug. The compound exhibits a number of mechanistic properties that may act in concert to attenuate the secondary biochemical and cellular dysfunctions following acute TBI.

CMX-2043 is the subject of issued composition-of-matter and use patents in territories around the world.

About Ischemix, Inc.

Ischemix is a privately-held drug development company based in Grafton, MA. The primary focus of the Company is the development of its lead compound, CMX-2043, for treatment of TBI.

About U.S. Army Medical Research and Development Command

The U.S. Army Medical Research and Development Command is the Army's medical materiel developer, with responsibility for medical research, development, and acquisition. USAMRDC produces medical solutions for the battlefield with a focus on various areas of biomedical research, including military infectious diseases, combat casualty care, military operational medicine, and medical chemical and biological defense..

About Neurotrauma at Combat Casualty Care Research Program

The Neurotrauma and Traumatic Brain Injury Portfolio is a requirements- based program that seeks to improve the far-forward capabilities of military diagnostic and treatment for the spectrum of TBI severities, with emphasis on moderate and severe TBI and polytrauma under Multi-Domain Operations in prolonged field care scenarios.

About MTEC

MTEC is a biomedical technology consortium collaborating with multiple government agencies under a 10-year renewable Other Transactional Agreement with the U.S. Army Medical Research and Development Command. MTEC is managed by Advanced Technology International. To find out more about MTEC, visit mtec-sc.org.

For media questions, please contact:

David A. DeWahl, Jr. President and CEO 203-561-0806 or ddewahl@ischemix.com

(a) This work was supported by the Assistant Secretary of Defense for Health Affairs endorsed by the Department of Defense, in the amount of $2,966,519, through the Medical Technology Enterprise Consortium under Award No. MT20015.039. Opinions, interpretations, conclusions and recommendations are those of the author and are not necessarily endorsed by the Assistant Secretary of Defense for Health Affairs or the Department of Defense.

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