Clene reports CNM-Au8 shows promise in Rett Syndrome study By

SALT LAKE CITY – Clene Inc. (NASDAQ:CLNN) and its subsidiary Clene Nanomedicine Inc., a clinical-stage biopharmaceutical company, announced new preliminary data suggesting CNM-Au8, an investigational nanotherapeutic, may offer treatment potential for Rett Syndrome, a rare neurodevelopmental disorder. The findings were presented Monday at the International Rett Syndrome Foundation 2024 Annual Meeting in Westminster, Colorado.

Rett Syndrome is characterized by severe neurological and developmental impairments that emerge after a period of normal development in early childhood. Currently, there is only one FDA-approved drug for the condition. CNM-Au8 is designed to improve mitochondrial function and neuronal survival, which could be beneficial in treating Rett Syndrome as well as other neurological diseases like ALS and MS.

The study, conducted in collaboration with researchers from Nationwide Children’s Hospital and the University of Missouri, showed that CNM-Au8 improved neuron health, survival, and neurite lengths in an in vitro model of Rett Syndrome. Additionally, it demonstrated the ability to rescue mitochondrial respiration deficits in astrocytes derived from Rett patients.

Dr. Karen Ho, Clene’s vice president of translational medicine, highlighted the drug’s potential to treat mitochondrial dysfunction, a common challenge in neurodegenerative diseases. While these results are preliminary, they indicate CNM-Au8’s potential as a versatile treatment option for multiple nervous system diseases.

Clene has been developing CNM-Au8 primarily for ALS and MS, but the recent findings encourage its application for Rett Syndrome. The study received support from the Baby Eleanor Foundation and utilized cells donated by anonymous individuals.

With its headquarters in Salt Lake City and operations in Maryland, Clene focuses on therapies that target mitochondrial health to treat neurodegenerative diseases. CNM-Au8 is a central piece of their development portfolio, representing a novel approach to improving central nervous system function.

The company’s announcement is based on a press release statement and represents a step forward in the potential treatment options for Rett Syndrome, which may also reinforce Clene’s broader therapeutic strategy for neurological conditions.

InvestingPro Insights

Clene Inc. (NASDAQ:CLNN) is actively pursuing innovative treatments in the neurodegenerative disease space with its lead candidate, CNM-Au8, showing promise in early data for Rett Syndrome. As investors consider the potential impact of these developments on the company’s valuation, it is important to look at the financial health and market performance of Clene.

InvestingPro Data indicates a market capitalization of $50.24 million, reflecting the size of the company in the competitive biopharmaceutical landscape. Despite the potential of CNM-Au8, analysts are cautious about the company’s financial outlook, with an adjusted P/E Ratio for the last twelve months as of Q1 2024 standing at -0.98, suggesting that the company is not currently profitable. Moreover, the Price / Book ratio is high at 11.78, which could indicate that the stock is trading at a premium compared to the company’s book value.

The InvestingPro Tips highlight some challenges facing Clene: analysts expect a sales decline in the current year and do not anticipate the company will be profitable this year. Clene is operating with a moderate level of debt and has been trading at a high revenue valuation multiple. These factors may raise concerns about the company’s near-term financial trajectory, especially considering that Clene does not pay a dividend to shareholders, which can be a downside for income-focused investors.

For those interested in a deeper dive into Clene’s financials and future prospects, additional InvestingPro Tips are available, offering a comprehensive analysis of the company’s performance and potential. Readers can find out more about these insights by visiting, and can use the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription. There are a total of 8 additional InvestingPro Tips listed for Clene Inc., which could provide valuable context for investors looking to make informed decisions.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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