8-K: Clene Reports Positive Clinical Data and Full Year 2023 Financial Results, Eyes Accelerated Approval for ALS Treatment

Clene Inc. announced its full year 2023 financial results and highlighted positive clinical data for its lead drug candidate, CNM-Au8, including significant survival benefits in ALS patients and vision improvements in MS patients, while also initiating discussions with the FDA for accelerated approval of its ALS treatment.

Summary

• Clene Inc. reported its full year 2023 financial results, showing a net loss of $49.5 million, or $0.47 per share, compared to a net loss of $29.9 million, or $0.46 per share, in 2022.
• The company's cash, cash equivalents, and marketable securities totaled $35.0 million as of December 31, 2023, which is expected to fund operations into the fourth quarter of 2024.
• Clene is in discussions with the FDA regarding an accelerated approval pathway for CNM-Au8 for the treatment of ALS, with the goal of submitting an NDA in 2024.
• Clinical data from the HEALEY ALS Platform Trial showed statistically significant reductions in plasma neurofilament light chain (NfL) levels and a 60% decreased risk of long-term all-cause mortality for ALS patients treated with CNM-Au8.
• An Expanded Access Program (EAP) demonstrated a statistically significant survival benefit for ALS patients treated with CNM-Au8, with a 57% to 68% decreased risk of all-cause mortality compared to historical controls.
• Long-term data from the VISIONARY-MS trial showed continued vision improvement and cognitive benefits for MS patients treated with CNM-Au8 through 35 months.
• The company received a $45.1 million grant from the National Institute of Neurological Disorders and Stroke (NINDS) to support a new EAP for CNM-Au8 in ALS.
• Two peer-reviewed publications highlighted the brain target engagement and catalytic neuroprotective mechanism of action of CNM-Au8.

Sentiment

Score: 8

Explanation: The document presents a generally positive outlook due to the strong clinical data and progress towards regulatory approval, although the increased net loss and need for additional funding temper the overall sentiment.

Positives

• CNM-Au8 shows significant survival benefits in ALS patients, with a 57% to 68% decreased risk of all-cause mortality in the EAP.
• The drug also demonstrates vision improvement and cognitive benefits in MS patients over a 35-month period.
• Clene has secured a substantial $45.1 million grant to support further research and development of CNM-Au8 in ALS.
• The company has a cash runway into the fourth quarter of 2024, providing financial stability for ongoing operations.
• Clene is actively engaged with the FDA to pursue an accelerated approval pathway for CNM-Au8 in ALS.

Negatives

• Clene reported a net loss of $49.5 million for the year ended December 31, 2023, which is an increase from the $29.9 million loss in 2022.
• The FDA initially deemed the biomarker data insufficient to support accelerated approval for CNM-Au8 in ALS, requiring additional data.
• Total other expense, net, was $9.0 million for the year ended December 31, 2023, compared to total other income, net, of $18.5 million for the same period in 2022, primarily due to a loss on initial issuance of equity and changes in the fair value of contingent earn-out liabilities.

Risks

• The company's ability to obtain FDA approval for CNM-Au8 is not guaranteed and depends on the submission of additional data.
• Clene's financial position is dependent on securing additional funding beyond the fourth quarter of 2024.
• Clinical trials may not always produce positive results, which could delay or halt the development and commercialization of drug candidates.
• Changes in government regulations or healthcare practices could impact the demand for Clene's drug candidates.
• The company has a limited operating history, which makes it difficult to predict future performance.

Future Outlook

Clene expects its resources as of December 31, 2023, will be sufficient to fund its operations into the fourth quarter of 2024 and plans to submit additional data to the FDA to advance the CNM-Au8 accelerated approval discussions in 2024.

Management Comments

• In 2024 we will continue to advance our regulatory discussions with the U.S. Food and Drug Administration (FDA) that we anticipate will include new data on biomarkers, as well as additional clinical function and survival data in people living with ALS, said Rob Etherington, President and CEO of Clene.
• Having held our initial discussion with the FDA in the fourth quarter of last year, we have a clear understanding of the additional data required to support an accelerated approval pathway filing for CNM-Au8.
• We believe that we can provide additional supportive evidence to advance discussions with the FDA with the potential to file an NDA later this year.

Industry Context

Clene's focus on neurodegenerative diseases like ALS and MS aligns with a growing need for effective treatments in these areas, and the positive clinical data for CNM-Au8 positions the company as a potential leader in the field. The company's engagement with the FDA for accelerated approval also reflects the industry's push for faster access to promising therapies for unmet medical needs.

Comparison to Industry Standards

• The reported 57% to 68% decreased risk of all-cause mortality in ALS patients treated with CNM-Au8 in the EAP is a significant result, comparing favorably to other ALS treatments currently available.
• The long-term vision improvement and cognitive benefits observed in the VISIONARY-MS trial are also notable, as many MS treatments focus on slowing disease progression rather than reversing symptoms.
• While direct comparisons are difficult due to varying trial designs and patient populations, Clene's results appear promising when compared to the outcomes of other clinical trials in ALS and MS.
• For example, the HEALEY ALS Platform Trial is a large, multi-center trial that is considered a benchmark for ALS research, and Clene's results within this trial are particularly noteworthy.
• The company's focus on mitochondrial health and the NAD pathway is also a unique approach compared to other treatments in the neurodegenerative space, which often target specific proteins or pathways.

Stakeholder Impact

• Shareholders may view the positive clinical data and progress towards regulatory approval favorably.
• Employees may be encouraged by the company's progress and financial stability.
• Patients with ALS and MS may benefit from the potential availability of new treatment options.
• Creditors may be reassured by the company's cash position and funding runway.

Next Steps

• Clene plans to submit additional data to the FDA to advance the CNM-Au8 accelerated approval discussions in 2024.
• The company will continue to advance its clinical programs in ALS and MS.
• Clene will continue to engage with the FDA regarding the regulatory pathway for CNM-Au8.

Key Dates

Keywords

CNM-Au8, ALS, Multiple Sclerosis, Neurodegenerative Diseases, FDA, Accelerated Approval, Clinical Trials, Survival Benefit, Mitochondrial Health, Biomarkers, Neurofilament Light Chain, Vision Improvement, EAP, NINDS, Grant, Financial Results