Looking to Biogen's success, Clene claims its ALS therapy hits biomarker behind Qalsody approval

Attempting to sail in Biogen’s slipstream, Clene has pointed to data for the same biomarker that got Qalsody approved as a sign that the biotech’s own amyotrophic lateral sclerosis (ALS) drug is heading in the right direction.

Clene’s oral therapy, a gold nanocrystal suspension called CNM-Au8, produced what the company claimed was a “statistically significant” reduction in plasma neurofilament light (NfL)—a biomarker of nerve injury and neurodegeneration—compared to placebo after 24 weeks of treatment. The data come from the HEALEY ALS Platform Trial, which is testing multiple ALS therapies, including a cohort of 161 participants taking CNM-Au8 or placebo.

In its postmarket release Thursday, Clene pointed out that “surrogate biomarkers such as NfL have recently been used to support an FDA approval for the treatment of ALS”—a reference to Biogen’s success in securing accelerated approval for Qalsody in April to treat a subset of ALS patients off the back of a reduction in NfL.

Clene also said it saw a “consistent significant reduction” in plasma NfL levels in specific populations at greater risk of ALS progression, including those more likely to receive an ALS diagnosis and those with a higher mortality risk.

The biotech has already collected data on long-term survival and additional biomarkers from the trial and its open-label extension. It expects to publish this analysis later in the year.

The company is wise not to stash all its chips on getting approval based on NfL reduction. Biogen’s biomarker-based accelerated approval for Qalsody means that the Big Pharma is still on the hook to provide confirmatory evidence of the drug’s clinical efficacy. Whatever happens, at some point Clene will need to prove that its drug actually improves clinical outcomes for ALS patients.

“The statistically significant difference in plasma neurofilament levels, a key marker of neurodegeneration, is another independent indicator of slowed disease progression associated with CNM-Au8 treatment,” Clene’s head of medical Benjamin Greenberg, M.D., said in the release.

“Results with CNM-Au8 treatment in multiple phase 2 trials in ALS previously showed two independent indicators of slowed disease progression—CNM-Au8 decreased time to clinical worsening and improved survival at the 30 mg dose,” Greenberg added. “These independent pieces of evidence strongly support CNM-Au8 as a potential treatment for ALS.”

In his own statement, Clene CEO Rob Etherington also alluded to Qalsody’s biomarker-driven approval and said the biotech is “exploring the possibility of a NDA filing” including a planned meeting with the FDA in the third quarter to go over the existing data. In the meantime, the company is gearing up for a global phase 3 trial of CNM-Au8, he said.