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Lead Product Candidate

Ischemix' lead compound, CMX-2043, has successfully completed a Phase 1 safety trial in healthy volunteers and a 142-patient Phase 2a trial demonstrating prevention of cardiac injury in PCI patients. In a 360-patient Phase 2 trial (CARIN) CMX-2043 demonstrated safety but did not meet pre-specified endpoints regarding prevention of contrast-induced acute kidney injury (CI-AKI) or cardiac injury in cardiac catheterization lab subjects. The company is now conducting preclinical studies to test the hypothesis that the pharmacokinetics of the drug in the trial were significantly altered by the renal insufficiency status of trial subjects. CMX-2043 is also currently in additional preclinical development to confirm initial studies that showed efficacy of the drug for treatment of traumatic brain injury.


CMX-2043 is a new chemical entity based on alpha lipoic acid (ALA). It has been demonstrated to be more potent than ALA in many cell regulatory pathways. As with ALA, CMX-2043 has a multi-modal mechanism of action and has activity in multiple tissue types. Because it is a small molecule, CMX-2043 is inexpensive to manufacture relative to the cost of biologics or large peptide drugs.

Ischemix has been granted a composition of matter patent in the US on CMX-2043, with coverage through the year 2031 (US Patent No. 7,928,067 B2 Compositions and Methods for Treating Ischemia and Ischemia-Reperfusion Injury). A second composition of matter patent on analogs of CMX-2043 has been granted in the US, and additional patents have been filed in multiple jurisdictions around the world (US Patent No. 8,815,937 B2 Lipoyl Compounds and Their Use For Treating Ischemic Injury).

Engineered for Safety & Efficacy

The molecular structure of CMX-2043 is shown below.

  • Alpha lipoic acid (blue) - safe, active, naturally-occurring compound
  • CMX-2043 (red added) - new chemical entity; more potent
  • Multi-modal mechanism of action maintained
  • Active in multiple tissue types
  • Small molecule, injectable formulation