Despite missing the initial mark, new analysis of Clene’s gold nanocrystal therapy from the HEALEY ALS trial indicates the asset still shows promise.
Pictured: Clene CEO Rob Etherington/company courtesy
Despite missing the initial mark, Clene Inc.’s gold nanocrystal therapy for ALS still shows promise. Thursday, the company shared new results from an exploratory analysis showing CNM-Au8 delayed disease progression across key areas for patients.
In October 2022, Clene reported that CNM-Au8 did not meet the primary endpoint of slope of change in the HEALEY ALS trial based on the adjusted ALS Functional Rating Scale-Revised.
However, the strong survival signals seen at six months warranted a deeper look.
Results of the latest analysis suggest treatment with CNM-Au8 delayed the typical trajectory of the disease across all domains. While disease progression speed varies greatly from patient to patient, ALS typically claims it victims between 2-5 years after diagnosis.
Clene’s oral suspension works to restore neuronal activity by increasing energy production and utilization. Further results from the HEALEY ALS trial over the six-month period showed CNM-Au8 was associated with a:
- 74% drop in the composite endpoint of time to ALS clinical worsening
- 98% decreased risk of death or permanent ventilation
- 74% decreased risk of feeding tube placement
- 84% decreased risk of ALS-related hospitalization
Based on the findings, Clene will move forward in end of Phase II discussions with the FDA.
“When supported by a confirmatory trial, this would give people living with ALS longer periods of independence, which are precious to them and their families,” said Robert Etherington, Clene CEO, in a prepared statement.
On an investor call Thursday, Merit Cudkowicz, M.D., director of the Healey Center for ALS and principal investigator of the HEALEY ALS trial, said the FDA has been “very clear” that companies need to come in with two studies for approval. Accelerated approval based on this study alone would be unlikely.
A confirmatory trial would also be prudent due to the missed endpoint in the original study, Cudkowicz said.
She added that biomarker data will be coming soon to indicate if the neurofilament levels dropped, linking to the clinical findings of slowing progression. Neurofilament levels can predict the prognosis of ALS patients; higher levels are associated with greater risk of death.
Clene is also testing CNM-Au8 in multiple sclerosis and Parkinson’s disease. In February, the company shared MRI results for MS patients that reinforce the neurological improvements reported in August 2022. Phase II results showed improved daily life for trial patients in terms of low contrast vision, cognition, upper body function and walking.
A Phase III trial is also planned for MS patients following a meeting with the FDA.
