Immunic reported data today from its 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 is more of a sensitive MRI biomarker that is measured. Also, in that it correlates better in terms of disability progression for these patients.
That’s not to say that it didn’t have a win in this study with respect to another efficacy endpoint of this phase 2 CALLIPER trial, and this was with respect to the endpoint of 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.
It this were to happen after discussions with regulatory authorities, then there would definitely be 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 of this drug to treat these PMS patients. Secondly, there is only one FDA-approved drug to help these 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 it is 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 31, 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 for $240 million. When this deal closed in January of 2024, the biotech gained $80 million as a payment from it. In order to get another tranche payment, the stipulation was based on the release of this CALLIPER study data. Although, it remains to be seen if it receives this tranche, considering that it missed the primary endpoint of this specific mid-stage trial.
The cash constraint isn’t only because it might want to run a phase 3 study of vidofludimus calcium for PMS. That’s because it is already in the process of running twin phase 3 studies as part of the ENSURE program, which is to use this drug to treat another segment of multiple sclerosis (MS) patients, which is relapsing multiple sclerosis (RMS).
Speaking of these late-stage studies, as part of the ENSURE program, the ENSURE-1 study is expected to be completed in Q2 of 2026. From there, the ENSURE-2 study is expected to be done by the 2nd half of 2026. If this timeline sticks, then investors will get a glimpse of some data in 2026. This is going to be instrumental in determining if vidoufludimus calcium is successful in being able to treat patients with RMS.