Immunic reported data today from its phase 2 CALLIPER trial for its drug vidofludimus calcium (IMU-838) for the treatment of patients with progressive multiple sclerosis (PMS). The data itself was mixed because there was actually a miss on one of the endpoints that was being studied.
It was noted that in terms of the primary endpoint of annualized percent brain volume change, this did not achieve statistical significance. There was only a modest 5% improvement in this endpoint over placebo. The company noted that thalamic brain volume loss being reduced by 20% in patients with PMS compared to placebo is more prominent because it’s more of a sensitive MRI biomarker that is measured. Also, in that it correlates better in terms of the progression of disability in these patients.
That’s not to say that it didn’t have a win with respect to another efficacy endpoint of this phase 2 CALLIPER trial, and this was with respect to 24-week confirmed disability worsening (24wCDW). It was noted that patients who took vidofludimus calcium reduced their risk of confirmed disability worsening by 20% compared to placebo.
The company might be on to something here despite the primary endpoint miss, in that the FDA has been more prone to using confirmed disability worsening for phase 3 PMS registration studies. Plus, there are two other important items to highlight of importance here. The first of which is that the company might be able to gain an audience with the FDA to discuss the possibility of initiating a phase 3 study with confirmed disability worsening as the primary endpoint.
If this were to happen after discussion with regulatory authorities, then definitely there is a path forward for Immunic in advancing vidofludimus calcium for PMS. However, this is an unknown for the time being, and there is no assurance that the FDA will with certainty agree to this being an approvable endpoint for U.S. marketing approval. Secondly, there is only one FDA-approved drug for these PMS patients, and it is quite possible that the FDA might be lenient because of this.
There is one area of concern though, for the company and likely why the stock closed lower today by 22% to $0.99 per share. The company doesn’t have a lot of cash left in its coffers. It is going to need a lot of funds in order to initiate a phase 3 study using this drug to treat these PMS patients. For the quarter ending March 31st of 2025, it noted that it had $35.7 million, which it believes will be enough to fund itself through Q3 of 2025.
The good news is that it has a three-tranche private placement agreement in place of $240 million. When this deal closed in January of 2024, the biotech gained $80 million in gross proceeds from it. The stipulation to get another tranche was based on the release of data from the CALLIPER study. Although it remains to be seen if it receives this tranche, considering that it missed the primary endpoint for this specific trial.
The cash constraint isn’t only on the basis of advancing a phase 3 study of vidofludimus calcium for PMS. That’s because it is also running twin phase 3 studies as part of the ENSURE program. This program is focused on using this drug for another segment of the Multiple Sclerosis (MS) patient population, which is relapsing multiple sclerosis (RMS).
Speaking of these late-stage trials as part of the ENSURE program, it is believed that ENSURE-1 will be completed in Q2 of 2026, and then ENSURE-2 should wrap up in the 2nd half of 2026. If this timeline sticks, then investors will get a glimpse of whether or not this drug can be approved for this segment of the MS patient population.
