To mark World Brain Day on July 22, Immunic Inc (NASDAQ:IMUX)'s chief scientific officer Hella Kohlhof joined Proactive to discuss the company’s lead asset vidofludimus calcium which is being developed to treat the neurological disease multiple sclerosis (MS).
She highlighted that this drug has a dual mode of action, with neuroprotection, anti-inflammatory, and antiviral properties.
Clinical trials have returned promising results, with Phase 3 studies in relapsing MS underway and expected to conclude in 2026.
Proactive: July the 22nd is World Brain Day, an annual event raising awareness for neurological disorders. When people think about MS or multiple sclerosis, they think about the disability and serious consequences for patients' everyday lives. However, people often don't know that MS actually is a neurological disease, a disease of the brain and spinal cord. With more, I'm joined by Immunic’s chief scientific officer Hella Kohlhof. Could you start by talking a bit about the development and origin of MS?
Hella Kohlhof: I'm sure happy to do so. Multiple sclerosis is, as you said, a chronic and unpredictable disease of the central nervous system. It's unpredictable. If you have mild or very severe symptoms or very severe cause it so and as you said, it affects the brain, the spinal cord, and also the optic nerve. It can have a negative impact on different parts of your body. It can have a negative impact on the ability to see, to write, to speak, and to walk. And you can have, in addition, fatigue and depression. It's definitely much more than a muscle disorder.
And you asked as well about the origin and pathogenesis of MS. So actually, in multiple sclerosis, myelin, the coating that protects the nerves, is attacked by and damaged by the immune system. Therefore, MS is considered an immune-mediated demyelinating disease of the central nervous system. And the bad thing is that MS is also a progressive disease. So, without any effective treatment, this leads to severe disability. And to be honest, the cause of multiple sclerosis is not yet fully understood. But there's luckily a lot of ongoing research and, we really want to know what triggers the neural destruction. There's a lot of work to do because we really want to understand in order to get very good treatments to treat multiple sclerosis.
Immunic is developing its lead asset vidofludimus calcium for the treatment of relapsing and progressive MS. Tell us a bit more about the forms of MS and also the prevalence.
Usually, if you hear MS, everybody thinks about relapsing MS because this is the most prevalent with 85% of MS cases, but there are different forms of MS. First of all, you have the early form, what is called clinical isolated syndrome is CIS. But then you have the relapsing form and the progressive form.
About relapsing MS, as I said, 85% of MS patients develop as RMS. The characterization of this is really having relapses. And these are clearly defined attacks of the immune system towards the neurons. Therefore, you have increasing neurological symptoms and these relapses can be followed by periods of remission. There can be even partial or complete recovery during these remissions, all symptoms can disappear but sometimes the symptoms may continue or become permanent. But what is actually quite new for the understanding of relapsing MS is that in between the relapses, the worsening is ongoing. That's quite new, what we learned over the last years actually.
And on the other hand, we have the progressive MS which includes the primary progressive (PPMS) and the secondary progressive (SPMS). Primary progressive is characterized by a steady worsening of the neurological function from the onset of the symptoms without initial relapses. So it's really just worsening over time without having relapses for SPMS. Usually, you have first RMS and then you develop into secondary progressive MS. So, in principle, skipping the relapses but still having the worsening of the disease. The prevalence, about 1 million people in the US have MS and 2.8 million people worldwide, and it can occur at any age, but mainly young adults are affected and it's a lot more common in women compared to men.
Diving deeper into your asset vidofludimus calcium, can you walk us through how the drug works in MS?
Yes. Happy to do so. That's my favourite topic to be honest. So vidofludimus calcium has a dual mode of action. It's an activator of this neuroprotective product in Nurr1 and at the same time inhibitor of DHODH enzyme. It means in principle we have a triple effect with targeting the two targets here because on the one hand side we have neuroprotection, we have anti-inflammatory properties and antiviral properties.
So, for neuroprotective, it means vidofludimus calcium improves the survival, the differentiation, and the function of the neurons. In addition to this, we have the anti-inflammatory properties via DHODH inhibition. It means that via this route, we are reducing the aggressiveness of the auto-reactive immune cells. And with having the antiviral properties via DHODH inhibition, this prevents the reactivation of Epstein-Barr virus and therefore the ongoing destructive attack of the in this case misled, immune system on the nerves.
In which types of MS are you testing vidofludimus calcium and where do the trials stand at the moment?
We started several years ago was testing vidofludimus calcium in relapse-remitting multiple sclerosis. This phase two study was finished in over 200 relapsing MS patients and to be honest, we had wonderful statistically significant efficacy and a super clean safety profile. And therefore, we went on into Phase 3 studies in relapsing MS. This is the ENSURE program. It's a pivotal study into ongoing relapsing MS. We have two parallel studies, ongoing recruiting and planned for overture for around 2,000 patients. And yeah, the completion of both trials is expected in 2026, which will be a major milestone for Immunic as you can imagine. We want to confirm what was seen in the Phase 2 study and hopefully get regulatory approval afterwards.
In addition to that, we have the ongoing CALLIPER study. It's in progressive MS. It's fully recruited in 467 patients, and the top-line data are expected in April 2025. This is another important milestone for us. And next important inflection point we are really waiting for because we had already in last October positive interim biomarker data. CALLIPER could create a huge upside scenario for us when we are able to show something helpful in progressive MS.
This year's World Brain Day focuses on brain health and prevention. What can everybody do to keep their brains healthy?
It sounds really easy - eat and live healthy, do sports, sleep well. But this is actually not just esoteric chatter, it's backed by science as well. So you might have heard already about the gut-brain axis. It really counts what you eat because it can have an impact on the gut and then on the brain as well.
What I think is really interesting about sports is it is known that, for example, the brain-derived neurotrophic factor BDNF is produced in the muscles as well, but it can act on the brain supporting the function of and survival of the brain cells. And of course, train your brain. Don't be too lazy because in principle it acts like a muscle as well. And in general, be happy. It's always good for your immune system.
Quotes have been edited for clarity and style