According to CEO Daniel Vitt, clinical and disability-related outcomes are more relevant than brain volume change for drug development in multiple sclerosis.
Immunic’s oral multiple sclerosis drug failed to significantly slow the loss of brain volume in patients with a progressing form of the neurodegenerative disorder during a Phase II clinical trial. But according to CEO Daniel Vitt, it’s no big deal.
“Nobody should care at all about whole brain volume change anymore,” Vitt said during an investor call on Wednesday to discuss the mid-stage data, noting that having brain volume as a primary endpoint of the Phase II study was just a “leftover” of a trial design that the biotech settled on “five years ago.”
Instead, the field has now focused on using disability as an endpoint, Vitt said, noting that Immunic is taking the same approach, particularly for its Phase III trial. In that regard, Vitt asserted that Wednesday’s readout was a “very, very clear positive outcome.”
Leerink analysts said the same thing, calling the CALLIPER results for the therapy, a Nurr1 activator called vidofludimus calcium, “positive” and “competitive” in an investor note on Wednesday.
Vidofludimus calcium, according to the analysts, “demonstrated a competitive risk/benefit profile” in primary progressive multiple sclerosis (MS), as compared with Roche’s Ocrevus, and in non-active secondary progressive MS, relative to Novartis’ Mayzent and Sanofi’s tolebrutinib. Of course, this analysis comes with “usual cross-trial comparison caveats,” Leerink cautioned.
“We believe the molecule has now demonstrated disease-modifying activity in a difficult-to-treat patient population with limited active lesions,” the analysts added. Immunic is running the Phase III ENSURE program to assess vidofludimus calcium, with data expected next year, Vitt said on the call. Leerink is likewise bullish about Immunic’s prospects here, with its analysts expecting a 50% probability of success.
Data from CALLIPER showed that vidofludimus calcium improved the annualized rate of brain volume change by 5% relative to placebo—an effect that failed to reach statistical significance.
Immunic, however, chose to focus on the clinical outcomes. The likelihood of having disability worsening events at 24 weeks, for instance, was 20% lower in the vidofludimus calcium group, as compared with placebo. This effect remained consistent or increased even further in various patient subgroups, including in those with primary progressive MS and in those without inflammatory lesions at baseline.
As for safety, there were no new signals of concern with vidofludimus calcium. Severe side effects were “rare,” according to Immunic, and occurred at similar frequencies between the treatment arms: 8.1% in patients on vidofludimus calcium and 6.5% of placebo counterparts.
