Immunic’s multiple sclerosis med trounces placebo in phase 2

Chalk one up for Immunic Therapeutics’ lead asset—the drug outshone placebo at reducing damage to myelin, the fatty substance that coats and protects nerve fibers, in patients with the relapsing form of multiple sclerosis. With these topline data in hand, the company will continue gearing up for a phase 3 program.

The phase 2 study tested two dose levels of the treatment, IMU-838, against placebo in 209 patients. After six months of treatment, patients taking the higher dose, 45 mg, saw a 62% drop in the total number of lesions, or demyelinated areas, in the brain and spinal cord, as measured by magnetic resonance imaging (MRI). Patients taking the lower dose, 30 mg, saw a 70% drop in the same metric.

In multiple sclerosis, the immune system attacks myelin, resulting in the destruction of the fatty sheath and damage to the nerve cells it protects. There is no test that can diagnose MS on its own but using MRI to find areas of damage in the central nervous system, can be part of forming a diagnosis.

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Patients taking IMU-838 and placebo experienced side effects at roughly the same rate: 43% and 44%, respectively. And serious side effects affected 3 of 140 patients taking the drug (2%) versus 1 of 69 patients on placebo (1%). The drug’s safety profile is consistent with that seen in studies of other diseases, the company said in a statement.

Immunic is developing IMU-838 for COVID-19 infection, as well as for the bile duct disease, primary sclerosing cholangitis, ulcerative colitis and Crohn’s disease. It works by blocking the enzyme dihydroorotate dehydrogenase, or DHODH, which plays a role in the metabolism of activated T cells and B cells. The hope is that targeting DHODH will block the release of pro-inflammatory cytokines and tamp down on inflammation.

“We believe that these phase 2 data of IMU-838 speak volumes about its potential to provide a new, convenient, once daily oral front-line treatment option to patients suffering from [relapsing-remitting multiple sclerosis], bolstered by a unique combination of potential efficacy, safety and tolerability,” said Immunic CEO Daniel Vitt, Ph.D., in a statement. Given the strength of these top-line results, we will continue to prepare a clinical phase 3 program for IMU-838 in RRMS and, after a full review of the data, anticipate providing a further update on development strategy.”

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Part of its development strategy includes a study extension, which gives patients who participated in the phase 2 trial the option to keep receiving treatment for up to nine and a half years. Beyond MS, Immunic plans to report data from other phase 2 studies, including in primary sclerosing cholangitis, ulcerative colitis and COVID-19, Vitt said.

This summer, Immunic started testing IMU-838 in patients with moderate COVID-19 infection. In July, a pair of U.K. hospitals started testing a combination of IMU-838 and Tamiflu in patients with moderate to severe COVID-19.

Shares in the biotech jumped 20% in Monday morning trading.