Clene claims MS drug win, but COVID disruption masks some tricky data

Clene Nanomedicine has chalked up a win for its gold nanocrystal suspension to treat multiple sclerosis—although it looks like the COVID-19 pandemic may have lent a helping hand.

The oral therapy CNM-Au8 is currently being tested to treat amyotrophic lateral sclerosis, Parkinson’s disease and MS. The study in question was assessing whether CNM-Au8 could improve visual function and bodily coordination in MS patients over 48 weeks.

The phase 2 trial, dubbed Visionary-MS, was stopped prematurely in February due to “operational challenges” resulting from the pandemic, the company said in an update on Monday morning. This meant enrollment only reached 73 of the planned 150 participants.

More significantly, the primary analysis was limited to a modified population, with any “invalid data” removed. While the results from the full population were “directionally consistent” with the reduced pool of participants who comprised the final analysis, the data from the wider group of participants did not actually meet the trial’s goals.

In another sign that the study was being judged in a slightly unorthodox way, the threshold for significance was pre-specified at a p value—the probability of observed effect—of 0.10 prior. For context, the FDA’s threshold for statistical significance is a p value of 0.05 or lower.

Still, Clene was happy to claim a win. The company pointed to a p value of 0.056 when assessing the primary outcome of improvements in how well patients could read letters in a low-contrast letter acuity test.

CNM-Au8 also demonstrated improvements across multiple paraclinical biomarkers, including other visual tests. In contrast, patients on placebo generally worsened during the 48-week period, Clene said.

The therapy also passed secondary endpoints including a 25-foot timed walk and a finger dexterity test, according to Clene.

"In this study, CNM-Au8 demonstrated neurological improvements in people with stable relapsing MS as adjunctive therapy to immunomodulatory [disease-modifying therapies],” Clene’s chief medical officer Robert Glanzman, M.D., said in a statement. “I am very impressed by the consistency of structural and functional improvements demonstrated by CNM-Au8 throughout the neuraxis.”

Armed with these data, Clene looks forward to initiating a phase 3 study in people with MS who “are experiencing progression independent of relapse activity,” Glanzman added.

Clene’s shares were trading down 4% at $3.31 as of 10:18 a.m. ET on Monday.