• Clene revealed new preliminary data on CNM-Au8 for Rett Syndrome treatment at the International Rett Syndrome Foundation 2024 Annual Meeting in Westminster, Colorado
• The only current FDA-approved drug for Rett Syndrome is trofinetide, approved in 2023
• CNM-Au8 treatment showed improvements in mitochondrial respiration deficits in Rett patient-derived astrocytes

Clene (NASDAQ: CLNN), along with its subsidiary Clene Nanomedicine Inc., a biopharmaceutical company specializing in mitochondrial health to address neurological diseases like amyotrophic lateral sclerosis (“ALS”) and multiple sclerosis (“MS”), recently revealed new preliminary data on CNM-Au8 for Rett Syndrome treatment. The data was presented by Dr. Karen Ho, Clene’s Vice President of Translational Medicine, at the International Rett Syndrome Foundation 2024 Annual Meeting in Westminster, Colorado. The presentation, titled “CNM-Au8, a Candidate First-in-Class Nanotherapeutic for Treatment of Rett Syndrome,” highlighted CNM-Au8’s potential (https://ibn.fm/NAtz7).

Rett Syndrome is a severe and rare pediatric neurological disorder caused by mutations in the MECP2 gene on the X chromosome. It predominantly affects females, occurring in about 1 in 10,000 live female births. Children with MECP2 mutations develop normally for the first six months of life but then experience a regression in skills, leading to various neurological and developmental issues such as loss of hand movement, abnormal gait, respiratory problems, autism spectrum traits, motor dysfunction, loss of verbal skills, seizures, and Parkinson-like features. The disorder also involves microcephaly and myelin loss in the brain. Currently, the only FDA-approved drug for Rett Syndrome is trofinetide, approved in 2023.

CNM-Au8 is an oral nanotherapeutic that enhances energy metabolism in nervous system cells, including neurons and oligodendrocytes, to support neuronal survival and remyelination. Clene has primarily been developing CNM-Au8 for ALS and MS, but its unique mechanism of action has led to considering its potential for treating Rett Syndrome.

The research, conducted in collaboration with Dr. Kathrin Meyer, formerly of Nationwide Children’s Hospital and now Chief Scientific Officer of Alcyone Therapeutics, along with her former postdoctoral researchers Drs. Meysam Ganjibakhsh and Andrea Sierra Delgado, explored the impact of CNM-Au8 on Rett Syndrome. Dr. Delgado is now a Research Assistant Professor at the University of Missouri.

Key preliminary findings include significant improvements 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.

Additionally, CNM-Au8 treatment showed improvements in mitochondrial respiration deficits in Rett patient-derived astrocytes, with full rescue (p<0.0001) of both basal and ATP-linked respiration in one Rett line and partial rescue (p<0.001 improvement in ATP-linked respiration) in another.

Dr. Ho said Rett Syndrome has a disease mechanism that shares some common features with both ALS and MS, with dysfunctional energy metabolism, glutamate excitotoxicity, demyelination, and mitochondrial dysfunction being hallmarks of the challenges faced by the nervous system in all three of these diseases.

“These preliminary data suggest that CNM-Au8 may treat Rett syndrome by potential rescue of mitochondrial dysfunction, thereby promoting neuronal health, survival, and synaptic structure. If CNM-Au8 proves to be a successful treatment for Rett syndrome, this will add further affirmation to Clene’s central thesis: that CNM-Au8—with its versatile and unique catalytic mechanism—holds promise as a potential treatment for multiple diseases of the nervous system, beyond its current targets of ALS and MS,” Dr. Ho added.

The study received support from the Baby Eleanor Foundation, and the team acknowledged the donation of Rett and healthy control cells from anonymous individuals. Dr. Ho emphasized the potential impact of CNM-Au8 on millions of people with difficult-to-treat neurological diseases, highlighting the rewarding nature of their ongoing research. “It’s a truly rewarding time to participate in the development of CNM-Au8, which has strong potential to make a difference to the millions of people living with difficult-to-treat neurologic diseases,” she said.

For more information, visit the company’s website at www.Clene.com.

NOTE TO INVESTORS: The latest news and updates relating to CLNN are available in the company’s newsroom at https://ibn.fm/CLNN

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