Clene Inc. and its wholly-owned subsidiary Clene Nanomedicine Inc. announced it presented new, preliminary data demonstrating the potential of CNM-Au8 as a treatment for Rett Syndrome. Karen Ho, Ph.D., Clene?s vice president of translational medicine, unveiled the data in oral and poster presentations on June 19, 2024 at the International Rett Syndrome Foundation 2024 Annual Meeting in Westminster, Colorado. The presentation was titled, CNM-Au8, a Candidate First-in-Class Nanotherapeutic for Treatment of Rett Syndrome.

CNM-Au8 is an orally administered, catalytic nanotherapeutic that targets energy metabolism via mitochondria in nervous system cells, including neurons and oligodendrocytes, to enhance neuronal survival and function as well as to support remyelination. To date, Clene has focused on the development of CNM-Au8 for the treatment of ALS and MS. The novel mechanism of CNM-Au8, with its catalytic ability to bolster mitochondrial function to aid in the survival and function of neurons, as well as the remyelinating properties of CNM-Au8, led Clene to also consider Rett Syndrome as a possible indication for treatment by CNM-Au8. The project was conducted in collaboration with Dr. Kathrin Meyer, formerly of Nationwide Children?s Hospital in Columbus, Ohio, now Chief Scientific Officer of Alcyone Therapeutics, and her former postdoctoral researchers, Drs.

Meysam Ganjibakhsh and Andrea Sierra Delgado. Dr. Delgado was former Chief Research Associate in the Meyer lab and is now Research Assistant Professor at the University of Missouri. The study?s main preliminary findings are: Statistically significant improvement in neuronal health (p<0.01), neuron survival (p<0.0001), and neurite lengths (p<0.05) in an in vitro model of Rett Syndrome, and; Improvements in the mitochondrial respiration deficits associated with Rett patient-derived astrocytes with CNM-Au8 treatment in vitro, with full rescue (p<0.0001) of both basal and ATP-linked respiration observed in one Rett line, and partial rescue observed in a second Rett line (ns change in basal respiration; p<0.001 improvement in ATP-linked respiration) at one concentration of CNM-Au8 treatment for 24 hours.

All statistical analyses were done using one-way ANOVA, and all conditions were performed with a minimum of three replicates. Support for the study from the Baby Eleanor Foundation, and the donation of Rett and healthy control cells from anonymous individuals, were gratefully acknowledged by the study team.