Clene’s ALS Treatment Reaps Rewards with Promising Results

Clene Inc.’s gold nanocrystal therapy for ALS, although missing the initial mark, still offers hope. On Thursday, the company released encouraging results from an exploratory analysis, which showed that CNM-Au8 had a positive effect on slowing the advancement of the disease in multiple areas for patients.

In October 2022, Clene announced the disappointing news that their groundbreaking drug CNM-Au8 had failed to meet the primary endpoint of the HEALEY ALS trial – a slope of change in the adjusted ALS Functional Rating Scale-Revised. Despite being hailed as a potential breakthrough in the treatment of ALS, the drug ultimately fell short of expectations.

The six-month survival signals were so strong, they begged for further investigation. Taking a closer look revealed even more remarkable results.

Our latest analysis has revealed that treatment with CNM-Au8 has the potential to drastically slow ALS progression. Though the speed of progression varies from patient to patient, this devastating disease usually claims its victims within 2-5 years of diagnosis. With CNM-Au8, patients may be able to prolong this timeline and enjoy a better quality of life for longer.

The HEALEY ALS trial revealed incredible results over a six-month period: Clene’s oral suspension was associated with increased energy production and utilization, resulting in restored neuronal activity. CNM-Au8, a component of Clene, showed promising results in this trial and may be a key player in restoring normal functioning in ALS patients.

A remarkable 74% decrease in the time to ALS clinical worsening was achieved through a composite endpoint.

The risk of death or permanent ventilation has been dramatically reduced by 98% – an incredible result that highlights the significant progress we’ve made in medical science.

Studies have shown that providing early feeding interventions to premature infants can result in a 74% decrease in their risk of needing to be placed on a feeding tube. This is a remarkable finding that has the potential to drastically improve the health outcomes of preterm babies.

A remarkable 84% reduction in risk of hospitalization due to ALS has been observed – a truly incredible finding that could bring hope to many patients.

Building on the results of its extensive research, Clene is now entering in to the final stages of Phase II discussions with the FDA. This is an exciting step forward for the company as it brings them closer to achieving their goals.

This could be a game-changer for people living with ALS, as a confirmatory trial could give them periods of longer independence – a priceless gift for them and their families. Robert Etherington, CEO of Clene, expressed his excitement at the potential implications of this.

On Thursday’s investor call, Merit Cudkowicz, M.D., Director of the Healey Center for ALS and Principal Investigator of the HEALEY ALS trial, made it clear that the FDA’s approval process requires two separate studies for approval, making it unlikely that accelerated approval could be granted on the basis of this study alone.

Due to the missed endpoint in the original study, a confirmatory trial is highly recommended, according to Cudkowicz. This further trial would be a prudent step to ensure the accuracy of the results.

Biomarker data is set to provide further evidence to the clinical findings of slowing progression in ALS patients, as soon as it reveals a drop in their neurofilament levels. High levels of neurofilament are known to be a predictor of death in ALS patients, making this data especially crucial.

In February, Clene made huge strides forward in their quest to utilize CNM-Au8 to treat multiple sclerosis and Parkinson’s disease. MRI results indicated improved daily life for trial patients, with positive changes seen in low contrast vision, cognition, upper body function, and walking. These results build upon the neurological advancements reported back in August 2022, providing further evidence of the drug’s potential.

Following a productive meeting with the Food and Drug Administration, a Phase III clinical trial has been scheduled to evaluate the effectiveness of a potential treatment for multiple sclerosis patients. This is a crucial step in the development of a new therapy and could provide much-needed relief for those suffering from this debilitating condition.

Leave a Comment