Clene Presents Preliminary Data for CNM-Au8® as a Potential Treatment for Rett Syndrome

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CNM-Au8 demonstrated neuroprotective effects in an in vitro model of Rett Syndrome, a rare pediatric neurodevelopmental disease CNM-Au8 also demonstrated rescue of mitochondrial deficits in induced astrocytes derived from Rett patients Invited oral presentation and poster presented on June 19, 2024 , at the International Rett Syndrome Foundation 2024 Annual Meeting in Westminster, Colorado SALT LAKE CITY , June 21, 2024 (GLOBE NEWSWIRE) -- Clene Inc. (Nasdaq: CLNN) (along with its subsidiaries, “Clene”) and its wholly-owned subsidiary Clene Nanomedicine Inc. , a clinical-stage biopharmaceutical company focused on improving mitochondrial health and protecting neuronal function to treat neurological diseases, including amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS), today 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 th 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.” Rett Syndrome is a severe, rare pediatric neurologic disorder caused by mutations in the X chromosome-linked gene, methyl-CpG binding protein 2 (MECP2). The disorder primarily affects females, with an incidence of ~1:10,000 live female births. Children with mutations in MECP2 develop normally until about 6 months of age, after which they exhibit a regression in acquired skills and begin to display a wide range of neurological and developmental impairments that include hand-wringing with loss of purposeful hand movement, abnormal gait, respiratory dysregulation, autism spectrum features, motor dysfunction, loss of verbal communication skills, seizures, and Parkinson-like features. Microcephaly and white matter (myelin) loss are pathological features of the Rett brain. There is currently only one approved drug for the treatment of Rett Syndrome, trofinetide, which was approved by the U.S. Food and Drug Administration in 2023. 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

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