Immunic Inc (NASDAQ:IMUX) CEO Dr Daniel Vitt talked with Proactive about new positive data from the company’s phase 2 EMPhASIS trial of vidofludimus calcium in relapsing-remitting multiple sclerosis (RRMS).
Proactive: Hello you're watching Proactive, I'm joined by Immunic CEO Dr Daniel Vitt. Daniel, very good to speak with you. You reported positive data from your phase 2 EMPhASIS trial of vidofludimus calcium in relapsing remitting multiple sclerosis. The data shows a low rate of confirmed disability worsening in patients treated with vidofludimus calcium. That must be encouraging?
Dr Daniel Vitt: Oh it's super encouraging. Specifically in the context of the recently announced good data on disability protection for our CALLIPER phase 2 in progressive MS. This is another confirmation or corroboration of the potential neuroprotective effect of the drug. I think it's interesting to see how well patients continue with long-term treatment post the double-blind, placebo-controlled phase of the EMPhASIS study. Up to five and a half years now—that really is an exciting data point for vidofludimus calcium.
Can you tell us more about the safety profile of vidofludimus calcium for long-term use?
Well, you see, it's confirming what we have seen before. There is really little ongoing there. I think the best confirmation of a wonderful safety and perceived tolerability is the high number of patients still on treatment. From the initially 268 patients randomized, 254 went into open-label extension after the double-blind phase. Of those, in January of this year, there were still 182 on active treatment. I think this is outstanding and really confirms that patients feel obviously well on the drug. That makes me very bullish about the safety and tolerability.
Maybe to take a step backwards, could you remind us about the overall EMPhASIS trial, including the trial design and primary outcomes?
Initially, this was really designed to demonstrate that the drug is capable of stopping MS in relapsing MS patients, specifically based on inflammation and relapses. The primary endpoint was a reduction of inflammatory lesions on MRI. This was a randomized, double-blind, placebo-controlled study. We had three doses tested—10, 30, and 45 milligram—compared with placebo. If you look back, the key result was that 30 and 45mg both showed a dramatic reduction of these lesions by 76% and 78% on cumulative active lesions or gadolinium-enhancing lesions. This was a very nice proof with high statistical significance that the drug does its job reducing inflammation and slowing relapses. We also saw initial hints of neuroprotection, such as a reduction of disability progression by even more than 50%.
Despite small numbers, this was a very positive data set. That drove our conviction that the drug has more effects than just reducing inflammation—it goes beyond that. We wanted to test the effect on long-term protection of disability progression, independent of inflammation.
Why is the confirmed disability worsening or CDW important for MS patients? And what does this mean for the overall development program?
This is maybe the elephant in the room for patients. If you get diagnosed with multiple sclerosis, regardless if it's relapsing or primary progressive MS, the biggest fear is losing independence over time. The quantitative assessment of that is the EDSS or disability score, and patients converting from stage 1 to 10 over time once diagnosed. The key unmet need is to stop or slow down that effect. In the past, companies have been successful in developing anti-inflammatory drugs which stop relapses, but there were little effects seen on stopping progression independent of relapse activity. Therefore, I'm very excited about the data we have here.
Daniel, can you take us through the next steps for vidofludimus calcium in MS?
As most people may have heard already, based on the EMPhASIS original data, we decided to test it in two phase 3 studies in relapsing MS to confirm the data in a bigger cohort and to qualify for approval. The ENSURE studies are ongoing. They have recently completed enrollment—1,122 patients recruited across the two studies. These studies are expected to read out end of next year. We believe that ultimately this should give us the opportunity to file for approval of the drug post positive data.
In these studies, and this ties back to our press release this week, we also want to observe the long-term neuroprotective effect as a secondary endpoint. This will also be very important in positioning the drug and communicating with patients and doctors. On top of that, we wanted to test whether the neuroprotective hints from the EMPhASIS study translate into a real pure neuroprotective effect in progressive forms of MS.
As part of that, we performed the CALLIPER phase 2 study, which read out last April. In that study, we confirmed in a pretty impressive way that patients treated with 45mg had a substantially lower rate of disability worsening compared to placebo. To remind everybody, we had a 24% reduction in overall CALLIPER CDW24 and in the primary progressive MS subgroup even a 32% reduction. I think these are impressive numbers and show that all the data follow together and form a nice picture that vidofludimus ultimately can be a very efficacious neuroprotective treatment.
Quotes have been lightly edited for clarity and style