Immunic's investors appeased by interim biomarker data from phase 2 MS trial

Immunic Therapeutics’ decision to narrow its focus in the spring appears to be paying off, with investors reacting warmly to some early biomarker data from the biotech’s multiple sclerosis (MS) candidate.

The company has been testing an inhibitor of dihydroorotate dehydrogenase called vidofludimus calcium in a phase 2 trial of patients with progressive MS. An early peak at data from 203 patients—around half of the individuals eventually enrolled in the midstage trial—showed that those who received a 45-mg dose of the drug saw a 6.7% drop in serum neurofilament light chain (NfL) compared with a 15.8% increase among the placebo cohort.

Of the 79 patients who could also be assessed at 48 weeks, those on the drug—also known as IMU-838—saw a 10.4% reduction compared to a 6.4% increase among those who received placebo.

The company also broke down the data in subgroups. Of the 59 patients with primary progressive MS (PPMS), those who received the drug ended the 24 weeks with a serum NfL level 18.8% lower than those who received placebo. The only approved drug for PPMS, Roche’s Ocrevus, only demonstrated a 12.4% reduction versus placebo at 24 weeks in the phase 3 ORATORIO trial, Immunic pointed out.

Explaining the significance of this morning’s findings, Jens Kuhle, M.D., Ph.D., head of the multiple sclerosis center at University Hospital Basel in Switzerland, said serum NfL has been “consistently shown to capture disease activity and to predict future disability in MS.”

"Particularly remarkable, the non-active progressive MS population, which represents the highest unmet medical need in MS, also showed differences in NfL levels over this relatively short observation period in favor of vidofludimus calcium,” Kuhle added in the release.

"The clear separation observed in serum NfL for vidofludimus calcium over placebo in the PMS patient population represents another major step forward for, what potentially could be, a first-in-class [nuclear receptor related 1] activator for MS," Immunic CEO Daniel Vitt, Ph.D., added in the release.

Investors seemed impressed, briefly sending the biotech’s stock rocketing over 30% in premarket trading Tuesday before settling around 17% up at $1.86 ahead of market open.

A readout on the study’s primary endpoint of a change in brain volume or key secondary endpoint of change in brain atrophy is yet to come. However, today’s data mark some welcome, if tentative, affirmation for the company’s decision to narrow its therapeutic focus. In April, the New York-based biotech revealed that it was deprioritizing development in psoriasis and castration-resistant prostate cancer and throwing its remaining resources to multiple sclerosis and celiac disease.

Immunic disclosed the pipeline prioritization plans alongside phase 2b results for vidofludimus calcium in ulcerative colitis. In June, the biotech explained that the study had missed its primary endpoint, a setback it blamed on “unexpected interference” between the drug candidate and concurrent use of corticosteroids. The failure prompted Immunic to abandon plans to advance the drug in that indication without a partner.