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

Rhea-AI Summary

Clene presented preliminary data on June 19, 2024, at the International Rett Syndrome Foundation Annual Meeting, showcasing CNM-Au8® as a potential treatment for Rett Syndrome. This rare pediatric neurological disease, primarily affecting females, results in severe developmental impairments. CNM-Au8 demonstrated significant neuroprotective effects and mitochondrial function improvements in in vitro models. Clene aims to extend CNM-Au8’s applications beyond ALS and MS, leveraging its catalytic ability to enhance neuronal survival and remyelination. The study, in collaboration with Dr. Kathrin Meyer and researchers, highlights CNM-Au8's promise in treating multiple nervous system disorders.

Positive

• CNM-Au8 demonstrated significant neuroprotective effects (p<0.01) in an in vitro model of Rett Syndrome.
• CNM-Au8 showed significant improvements in neuron survival (p<0.0001) and neurite lengths (p<0.05).
• Statistically significant mitochondrial respiration improvements observed in patient-derived astrocytes with CNM-Au8 treatment.
• Potential for CNM-Au8 to treat multiple nervous system disorders beyond ALS and MS.

Negative

• Preliminary data only, with no clinical trials or FDA approval for Rett Syndrome treatment yet.
• Partial mitochondrial respiration improvements observed in one Rett line, indicating varied responses.

Insights

Medical Research Analyst

The preliminary data on CNM-Au8 for Rett Syndrome showcases promising neuroprotective effects and improvements in mitochondrial function. These findings suggest potential benefits for patients suffering from this severe neurodevelopmental disorder, which currently has limited treatment options. The emphasis on mitochondrial health is particularly noteworthy, given its central role in cellular energy metabolism and neuronal survival.

In vitro models that demonstrate increased neuronal health and survival provide an encouraging foundation for future clinical trials. However, it's essential to recognize that in vitro results don't always translate to clinical efficacy. The differentiation between full and partial mitochondrial rescue across different Rett patient-derived astrocytes also indicates potential variability in patient response, warranting further investigation.

Overall, these results position Clene's CNM-Au8 as a potential multifaceted therapeutic option not only for Rett Syndrome but also for other neurological conditions marked by mitochondrial dysfunction. If future studies confirm these preliminary results, CNM-Au8 could become a significant addition to the treatment landscape for neurodevelopmental disorders.

Market Research Analyst

From a market perspective, Clene's exploration into Rett Syndrome with CNM-Au8 represents a strategic extension of its existing focus on neurological diseases like ALS and MS. Rett Syndrome, although rare, represents a high unmet medical need. The fact that CNM-Au8 addresses mitochondrial dysfunction—a common thread in several neurological disorders—could broaden the drug's applicability and market potential.

Investors should note the potential for diversification of Clene's therapeutic portfolio. A successful expansion into Rett Syndrome could not only provide new revenue streams but also enhance the company's valuation by showcasing the versatility of its nanotherapeutic platform. However, it's important to exercise caution since these findings are still preliminary and based on in vitro data.

In the short term, continued positive updates and advancements into clinical trials could drive stock price appreciation. In the long term, the commercial viability of CNM-Au8 for Rett Syndrome and other neurological conditions will depend heavily on successful clinical outcomes and regulatory approvals.

• 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<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.
  

“Rett Syndrome has a disease mechanism that shares some common features with both ALS and MS,” said Dr. Ho. “Dysfunctional energy metabolism, glutamate excitotoxicity, demyelination, and mitochondrial dysfunction are all hallmarks of 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. 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.”

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.

About Clene

Clene Inc., (Nasdaq: CLNN) (along with its subsidiaries, “Clene”) and its wholly owned subsidiary Clene Nanomedicine Inc., is a late clinical-stage biopharmaceutical company focused on improving mitochondrial health and protecting neuronal function to treat neurodegenerative diseases, including amyotrophic lateral sclerosis, Parkinson’s disease, and multiple sclerosis. CNM-Au8^® is an investigational first-in-class therapy that improves central nervous system cells’ survival and function via a mechanism that targets mitochondrial function and the NAD pathway while reducing oxidative stress. CNM-Au8^® is a federally registered trademark of Clene Nanomedicine, Inc. The company is based in Salt Lake City, Utah, with R&D and manufacturing operations in Maryland. For more information, please visit www.clene.com or follow us on X (formerly Twitter) and LinkedIn.

Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended, which are intended to be covered by the “safe harbor” provisions created by those laws. Clene’s forward-looking statements include, but are not limited to, statements regarding our or our management team’s expectations, hopes, beliefs, intentions or strategies regarding our future operations. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words “anticipate,” “believe,” “contemplate,” “continue,” “estimate,” “expect,” “intends,” “may,” “might,” “plan,” “possible,” “potential,” “predict,” “project,” “should,” “will,” “would,” and similar expressions may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. These forward-looking statements represent our views as of the date of this press release and involve a number of judgments, risks and uncertainties. We anticipate that subsequent events and developments will cause our views to change. We undertake no obligation to update forward-looking statements to reflect events or circumstances after the date they were made, whether as a result of new information, future events or otherwise, except as may be required under applicable securities laws. Accordingly, forward-looking statements should not be relied upon as representing our views as of any subsequent date. As a result of a number of known and unknown risks and uncertainties, our actual results or performance may be materially different from those expressed or implied by these forward-looking statements. Some factors that could cause actual results to differ include our ability to demonstrate the efficacy and safety of our drug candidates; the clinical results for our drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; our ability to achieve commercial success for our drug candidates, if approved; our limited operating history and our ability to obtain additional funding for operations and to complete the development and commercialization of our drug candidates; and other risks and uncertainties set forth in “Risk Factors” in our most recent Annual Report on Form 10-K and any subsequent Quarterly Reports on Form 10-Q. In addition, statements that “we believe” and similar statements reflect our beliefs and opinions on the relevant subject. These statements are based upon information available to us as of the date of this press release, and while we believe such information forms a reasonable basis for such statements, such information may be limited or incomplete, and our statements should not be read to indicate that we have conducted an exhaustive inquiry into, or review of, all potentially available relevant information. These statements are inherently uncertain and you are cautioned not to rely unduly upon these statements. All information in this press release is as of the date of this press release. The information contained in any website referenced herein is not, and shall not be deemed to be, part of or incorporated into this press release.

Media Contact
Ignacio Guerrero-Ros, Ph.D., or
David Schull
Russo Partners, LLC
Ignacio.guerrero-ros@russopartnersllc.com
David.schull@russopartnersllc.com
(858) 717-2310

Investor Contact
Kevin Gardner
LifeSci Advisors
kgardner@lifesciadvisors.com
(617) 283-2856

FAQ

What did Clene's preliminary data reveal about CNM-Au8 for Rett Syndrome?

Clene's preliminary data revealed that CNM-Au8 demonstrated significant neuroprotective effects and improvements in mitochondrial function in in vitro models of Rett Syndrome.

When and where did Clene present the CNM-Au8 data for Rett Syndrome?

Clene presented the data on June 19, 2024, at the International Rett Syndrome Foundation Annual Meeting in Westminster, Colorado.

How did CNM-Au8 perform in terms of neuron survival in Rett Syndrome models?

CNM-Au8 showed significant improvement in neuron survival with a p-value of less than 0.0001 in Rett Syndrome models.

What are the potential broader applications of CNM-Au8 beyond Rett Syndrome?

CNM-Au8 holds promise for treating multiple nervous system disorders, including ALS and MS, due to its ability to enhance neuronal survival and remyelination.

What are the key findings from Clene's study on CNM-Au8 for Rett Syndrome?

Key findings include significant neuroprotective effects, improved neuron survival, enhanced neurite lengths, and improved mitochondrial respiration in Rett Syndrome models.