Immunic’s lead asset may have been successful in multiple sclerosis, but it appears to be a dud in ulcerative colitis (UC), where work will be discontinued.
The biotech revealed that vidofludimus calcium did not spur clinical remission in patients with moderate to severe UC during a phase 2 trial called CALDOSE-1, missing the main goal. No meaningful differences were seen between the three active dose groups nor on any of the secondary measures, including symptomatic remission and endoscopic healing, Immunic said Thursday.
No new safety signals were flagged during the trial, and the most common adverse events noted were anemia, headache and COVID-19.
Immunic blamed the failure on unexpected interference between vidofludimus calcium and the concurrent use of corticosteroids, which are commonly prescribed to treat UC. These meds also are commonly used during clinical trials for the condition.
The company pointed to a pooled analysis of patients who were not taking steroids for signs of efficacy, where an 11.4% “advantage in clinical remission” was seen among treated patients compared to placebo. This advantage measure differed from the trial goal, which was simply whether the patient achieved clinical remission.
“Such a difference in clinical remission between active treatment and placebo would traditionally be considered as confirming therapeutic activity,” Immunic noted.
However, the data appear to show that the rate of clinical remission was lower even in the non-steroid group compared to placebo. In the wider patient population that included those taking steroids, the non-steroid treated group saw a 11.5% clinical remission rate at Week 10 compared to 20.6% in the placebo arm. The non-steroid patients who received vidofludimus calcium had a remission rate of 14.7%.
“We are disappointed with the results of the CALDOSE-1 trial. The interference of vidofludimus calcium with concurrent corticosteroid use is surprising, and we will further explore the mechanism behind this unexpected observation,” said Chief Medical Officer Andreas Muehler, M.D.
With the failure on record, Immunic will discontinue the UC indication unless it finds a partner to continue the work. But vidofludimus calcium will go on in multiple sclerosis, where it previously demonstrated a treatment effect during a phase 2 study. A late-stage trial is underway in the indication as well.
Other assets in Immunic’s pipeline include psoriasis med IMU-935 and IMU-856 for celiac disease.
Immunic's shares tumbled on the news of the UC trial, falling from around $5.95 at close Wednesday to about $3.40 Friday afternoon.
The company reported cash and cash equivalents of $93.1 million as of May 31, which should fund operations through the fourth quarter of 2023.