EndeavourRx is developing a class of proprietary compounds known as AMPAkines, which are PAMs of the AMPA glutamate receptor (see Illustration). AMPAkines are small molecule compounds that enhance the excitatory actions of glutamate at the AMPA receptor complex, which mediates most excitatory transmission in the central nervous system (“CNS”). Through an extensive translational research effort from the cellular level through Phase 2 clinical trials, we have developed a family of AMPAkines, including CX717, CX1739 and CX1942 that may have clinical application in the treatment of CNS-driven neurobehavioral and cognitive disorders, SCI, neurological diseases, and certain orphan indications. These compounds belong to a new class of low impact AMPAkines that do not display the electrophysiological and biochemical effects that have led to undesirable side effects, namely convulsive activities, previously reported in earlier generations of such compounds. Thus, our compounds can be viewed as being kinder or gentler than the earlier generations.
CX717 and CX1739, our lead clinical compounds, have successfully completed multiple Phase 1 clinical safety trials with no drug-associated serious adverse events. Through an extensive translational research effort from the cellular level through Phase 2 clinical trials, CX717 and CX1739 were efficacious in treating drug induced respiratory depression caused by opioids without altering the analgesic effects of the opioids. The results of the preclinical research studies have been replicated in three separate Phase 2A human clinical trials with CX717 and CX1739, confirming the translational mechanism and target site engagement and demonstrating proof of principle that ampakines act as PAMs of AMPA receptors in humans.