Clene Granted In-Person Meeting With FDA Senior Leadership to Discuss CNM-Au8 Biomarker and Related Clinical and Survival Data

Rhea-AI Summary

Clene Inc. (Nasdaq: CLNN) has secured an in-person meeting with senior FDA leadership to discuss its CNM-Au8 development for ALS. The meeting, scheduled before the end of November 2024, will include key FDA officials and opinion leaders in ALS, biostatistics, and biomarkers. This development comes after the FDA initially communicated that Clene's briefing package did not support an NDA submission under the accelerated approval pathway. However, the FDA agreed to reassess the submission in person.

Clene will present data on ALS biomarkers, clinical endpoints, and survival data for CNM-Au8. The company has accumulated over 700 patient-years of safety data for CNM-Au8, showing no significant safety concerns or trends. No serious adverse events related to CNM-Au8 treatment have been identified by clinical trial investigators to date.

Positive

• Secured in-person meeting with senior FDA leadership to discuss CNM-Au8 for ALS
• FDA agreed to re-evaluate submission under accelerated approval pathway
• Over 700 patient-years of safety data showing no significant safety concerns for CNM-Au8
• No serious adverse events related to CNM-Au8 treatment identified in clinical trials

Negative

• Initial FDA communication indicated briefing package was not supportive of NDA submission under accelerated approval pathway

Regulatory Affairs Specialist

The FDA's decision to grant an in-person meeting with senior leadership is a significant development for Clene. This level of engagement suggests the FDA recognizes the potential importance of CNM-Au8 in ALS treatment. The meeting's composition, including key opinion leaders, indicates a comprehensive review of Clene's data.

However, the initial rejection of the NDA submission under the accelerated approval pathway highlights regulatory hurdles. The subsequent agreement to re-evaluate suggests Clene's data may have merit, but requires further scrutiny. This meeting could be pivotal for Clene's regulatory strategy and timeline for CNM-Au8's potential approval.

Neuroscience Research Analyst

The focus on biomarkers, clinical endpoints and survival data in ALS is crucial. ALS is a complex disease with treatment options, making any potential therapy significant. The extensive safety data (700 patient-years) with no significant concerns is promising, as safety is a primary concern in neurological treatments.

The biomarker discussion is particularly intriguing. If Clene can establish a reliable biomarker for ALS progression, it could revolutionize both drug development and clinical management of the disease. However, the initial rejection of the NDA suggests that the data may not yet be robust enough to satisfy regulatory requirements, highlighting the challenges in ALS drug development.

Biotech Investment Analyst

This meeting represents a critical juncture for Clene. The initial rejection of the NDA submission under the accelerated approval pathway is concerning, as it suggests the data may not meet the FDA's stringent requirements. However, the subsequent granting of a high-level meeting indicates there's still potential.

For investors, this situation presents both risk and opportunity. A positive outcome could significantly boost Clene's prospects, potentially leading to faster approval and market entry. Conversely, if the FDA remains unconvinced, it could lead to delays and additional costly trials. The 700 patient-years of safety data is a strong point, potentially reducing regulatory risk. Investors should closely monitor the outcome of this meeting, as it will likely have a substantial impact on Clene's valuation and future prospects.

SALT LAKE CITY, Sept. 16, 2024 (GLOBE NEWSWIRE) -- Clene Inc. (Nasdaq: CLNN) (along with its subsidiaries, “Clene”) and its wholly owned subsidiary Clene Nanomedicine Inc., a late clinical-stage biopharmaceutical company focused on improving mitochondrial health and protecting neuronal function to treat neurodegenerative diseases including amyotrophic lateral sclerosis (ALS) and multiple sclerosis (MS), announced today that it will meet in-person with the U.S. Food and Drug Administration (FDA) before the end of November 2024 to discuss CNM-Au8 development for ALS.

The meeting will include the FDA’s Director of the Office on New Drugs, the Director of the Office of Neuroscience, and the Division of Neurology 1 (DN1) review team, as well as recognized key opinion leaders in ALS, biostatistics, and biomarkers. Clene is appreciative of the FDA’s use of process and regulatory flexibility to obtain the best outcome for people living with ALS.

By way of background, days prior to DN1 granting the in-person meeting, DN1 communicated that Clene’s briefing package for CNM-Au8 was not supportive of an NDA submission under the accelerated approval pathway. However, after further dialogue, the FDA agreed to meet in person to re-evaluate Clene’s submission under the accelerated approval pathway. Clene looks forward to the opportunity to have experts present their views to the FDA and address questions on ALS biomarkers, related clinical endpoints, and survival data, all of which Clene believes are essential for the understanding of its CNM-Au8.

For CNM-Au8, Clene has more than 700 patient-years of safety data that show no significant safety concerns or safety trends identified. No serious adverse events (SAEs) have been identified as related to CNM-Au8 treatment by any clinical trial investigators to date.

About Clene
Clene 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 and LinkedIn.

About CNM-Au8®
CNM-Au8 is an oral suspension of gold nanocrystals developed to restore neuronal health and function by increasing energy production and utilization. The catalytically active nanocrystals of CNM-Au8 drive critical cellular energy producing reactions that enable neuroprotection and remyelination by increasing neuronal and glial resilience to disease-relevant stressors. CNM-Au8^® is a federally registered trademark of Clene Nanomedicine Inc.

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 the timing of the interaction with the FDA. In addition, any statements that refer to 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. Some factors that could cause actual results to differ include the Company's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its 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; the Company’s ability to achieve commercial success for its drug candidates, if approved; the Company’s limited operating history and its ability to obtain additional funding for operations and to complete the development and commercialization of its 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

When will Clene (CLNN) meet with the FDA to discuss CNM-Au8 for ALS?

Clene will meet in-person with the FDA before the end of November 2024 to discuss CNM-Au8 development for ALS.

What is the purpose of Clene's (CLNN) meeting with the FDA regarding CNM-Au8?

The purpose is to re-evaluate Clene's submission for CNM-Au8 under the accelerated approval pathway for ALS treatment, discussing biomarkers, clinical endpoints, and survival data.

How much safety data has Clene (CLNN) collected for CNM-Au8?

Clene has collected more than 700 patient-years of safety data for CNM-Au8, showing no significant safety concerns or trends.

Have any serious adverse events been linked to CNM-Au8 in Clene's (CLNN) clinical trials?

No serious adverse events (SAEs) have been identified as related to CNM-Au8 treatment by any clinical trial investigators to date.