8-K: Clene Inc. Announces Full Year 2024 Financial Results and Provides Update on CNM-Au8 Development for ALS

Clene Inc. reports its full year 2024 financial results, highlights progress on its CNM-Au8 program for ALS, and announces plans for an NDA submission in the second half of 2025.

Summary

• Clene Inc. announced its full year 2024 financial results and provided updates on its clinical programs in ALS and MS.
• The company is planning to submit a New Drug Application (NDA) for CNM-Au8 in ALS in the second half of 2025, seeking potential Accelerated Approval.
• Clene intends to initiate the confirmatory Phase 3 RESTORE-ALS trial in subjects with ALS in mid-2025.
• A new $10.0 million debt facility was secured at a lower interest rate to replace the remaining $7.85 million debt balance with Avenue Capital.
• As of December 31, 2024, Clene had $12.2 million in cash, cash equivalents, and marketable securities, including $7.3 million in gross proceeds raised on October 1, 2024.
• Clene is analyzing biomarker NfL data from its NIH-sponsored Early Access Protocol (EAP) for evaluation in the third quarter to satisfy the FDA recommendation for additional neurofilament light chain (NfL) data.
• Research and development expenses were $20.1 million for the year ended December 31, 2024, compared to $26.7 million for the same period in 2023.
• The company reported a net loss of $39.4 million, or $5.67 per share, for the year ended December 31, 2024, compared to a net loss of $49.5 million, or $9.43 per share, for the same period in 2023.

Sentiment

Score: 6

Explanation: The sentiment is neutral to slightly positive. The company is making progress on its clinical programs and has secured new financing, but it is also experiencing financial losses and has limited cash reserves.

Positives

• Clene is progressing towards an NDA submission for CNM-Au8 in ALS, potentially leading to accelerated approval.
• The company is initiating a Phase 3 trial to confirm the efficacy of CNM-Au8 in ALS.
• A new debt facility was secured at a lower interest rate, improving the company's financial position.
• Survival improvements were observed in the HEALEY ALS Platform Trial with CNM-Au8 treatment.
• The company is actively working with the FDA to address their requests regarding NfL data.

Negatives

• Clene reported a net loss of $39.4 million for the year ended December 31, 2024.
• Cash reserves have decreased from $35.0 million in 2023 to $12.2 million in 2024.
• The company expects its resources to be sufficient to fund operations only into the second quarter of 2025.

Risks

• The FDA may not accept NfL as a surrogate endpoint for CNM-Au8 in ALS.
• The company's ability to obtain accelerated approval for CNM-Au8 is uncertain.
• The company's cash reserves may not be sufficient to fund operations beyond the second quarter of 2025, potentially requiring additional financing.
• Clinical trial results may not support further development or marketing approval.

Future Outlook

Clene expects further regulatory guidance in 2025 on the critical next steps required to advance their CNM-Au8 NDA submission for the treatment of ALS under the accelerated approval pathway and anticipates NDA submission in the second half of 2025.

Management Comments

• Rob Etherington, President and CEO of Clene, stated that they plan to meet with the agency in the second quarter of 2025 to align on their statistical analysis plan for their NfL biomarker data being collected from the ongoing NIH-sponsored EAP in ALS patients, and then conduct the resulting analyses in the third quarter.
• Etherington also mentioned that their goal from these additional analyses is to determine concordance of the NIH-sponsored EAP NfL data with NfL results in their Healey Platform Trial.
• Etherington stated that they intend to use this NfL surrogate biomarker data as a basis to file their NDA in the second half of 2025 for potential Accelerated Approval.
• Etherington stated that they are incredibly motivated by their mission to help people suffering with ALS and other neurodegenerative diseases prolong their lifespan and improve their quality of life.

Industry Context

Clene is focused on developing treatments for neurodegenerative diseases, a field with significant unmet medical need. The company's focus on mitochondrial health and the NAD pathway aligns with emerging research in the field. The potential accelerated approval pathway for CNM-Au8 in ALS could provide a significant advancement in treatment options for this debilitating disease.

Comparison to Industry Standards

• The HEALEY ALS Platform Trial is a multi-center trial, similar to the CENTAUR trial of AMX0035 (Relyvrio) by Amylyx Pharmaceuticals, which also sought to demonstrate efficacy in ALS.
• Clene's focus on NfL as a biomarker aligns with industry trends, as companies like Biogen and Ionis Pharmaceuticals have also used NfL in their clinical trials for neurological disorders.
• The 4.1 month survival improvement observed in the HEALEY ALS Platform Trial is within the range of survival benefits seen in other ALS clinical trials, such as the 3-month benefit observed with edaravone (Radicava) in a select patient population.

Stakeholder Impact

• Shareholders: The potential approval of CNM-Au8 could increase shareholder value, but the company's financial losses and limited cash reserves could negatively impact shareholder returns.
• Patients: The development of CNM-Au8 could provide a new treatment option for ALS patients, potentially improving their survival and quality of life.
• Employees: The company's progress on its clinical programs could create job opportunities and provide a sense of purpose for employees.
• Creditors: The new debt facility could increase the company's financial stability and improve its ability to repay its debts.

Next Steps

• Meet with the FDA in the second quarter of 2025 to finalize the statistical analysis plan for the EAP NfL biomarker analyses.
• Conduct NfL biomarker collection and analyses in the third quarter of 2025.
• Submit an NDA for CNM-Au8 in ALS in the second half of 2025.
• Commence the confirmatory Phase 3 RESTORE-ALS trial in mid-2025.

Key Dates

Keywords

CNM-Au8, ALS, Clene, NDA, RESTORE-ALS, NfL, Clinical Trial, Accelerated Approval, Financial Results, Neurodegenerative Diseases