Immunic Therapeutics is a biotechnology company focused on developing innovative, small-molecule therapies for chronic inflammatory and autoimmune diseases, including multiple sclerosis and gastrointestinal disorders.
Why did Immunic Therapeutics decide to focus on small molecules instead of biologics or other advanced therapies for autoimmune diseases?
We believe it is essential to select the right tool for the right problem. When addressing a disease like multiple sclerosis (MS) that affects the brain, spinal cord and optic nerve, large molecules, like biologics, face significant challenges in distribution and crossing the blood-brain barrier. Small molecules, in contrast, have better distribution profiles, making them particularly effective for systemic conditions.
Moreover, small molecules offer a better patient experience. They are easier to administer, often in the form of oral treatments, which enhances compliance and convenience for patients. For example, our lead candidate, vidofludimus calcium, not only meets these criteria but also combines two essential functions in one treatment.
Ultimately, there are no inherently “good” or “bad” therapies—only suitable or unsuitable ones for specific medical challenges. For autoimmune diseases, small molecules clearly meet an important need for both efficacy and practicality.
Vidofludimus calcium is your lead candidate for multiple sclerosis. With significant recent progress in MS treatments, particularly in the field of disease-modifying therapies, what sets this drug apart?
The key unmet need in MS today is addressing relapse-independent neurodegeneration. While current treatments focus primarily on reducing relapses, they do little to halt the progression of disability that occurs independently of these relapses and right from the onset of the disease.
Approximately 50% of MS progression stems from this neurodegeneration, leaving patients increasingly disabled despite treatment. Vidofludimus calcium addresses this critical gap with its dual mode of action.
The drug acts both as an inhibitor of the enzyme DHODH and an activator of the neuroprotective transcription factor Nurr1. By selectively targeting DHODH, the drug can calm overactive immune responses without completely shutting down the immune system, allowing the body to maintain its natural defenses. Activating Nurr1 has shown promise in slowing or even stopping the neurodegenerative processes that lead to disability. This dual mechanism allows us to simultaneously target focal inflammation, which causes relapses, and directly protect neurons from degeneration. This approach goes a step beyond the current standard of care.
Another key benefit of vidofludimus calcium seems to be its safety profile compared to many approved MS treatments. Could you elaborate on that?
Many current treatments for autoimmune diseases, including MS, compromise the overall immune system, leading to side effects like increased infection rates, liver toxicity, and even a higher risk of certain cancers. Patients and physicians are often forced to balance efficacy with these risks, creating a need for safer, better-tolerated therapies. Vidofludimus calcium offers a distinct advantage in this regard.
First, in clinical trials involving over 1,800 individuals so far, the safety and tolerability profile was nearly identical between the drug and placebo groups. Importantly, we observed no signs of hepatotoxicity, no issues with neutropenia or lymphopenia, no cardiovascular side effects, and no increased infection rates. In fact, the mode of action even suggests a lower risk of infections. Coupled with its once-daily oral administration, vidofludimus calcium provides a safe, easy-to-use option that does not require burdensome monitoring, making it practical for both patients and physicians.
Affordability as another important factor. How does vidofludimus calcium address it?
Affordability is increasingly crucial as healthcare systems worldwide struggle to manage rising costs. While innovative technologies like biologics, RNA therapies, and cell therapies offer promise, they are often prohibitively expensive and pose challenges in accessibility. Small molecules, like vidofludimus calcium, can be produced more cost-effectively, enabling broader access to patients globally.
For MS, which already represents a $23 billion market, an affordable and highly effective treatment that also improves safety and convenience can have a transformative impact. By combining neuroprotection with anti-inflammatory and anti-viral effects as well as its favorable safety and tolerability profile, vidofludimus calcium not only advances the standard of care but does so in a way that is accessible and practical for healthcare systems. This ensures that more patients can benefit from cutting-edge treatments without facing financial barriers.
Immunic is also working on a molecule for the treatment of celiac disease. Can you tell us more about that?
Our second molecule, IMU-856, recently demonstrated activity in a Phase 1b study in patients with celiac disease, which represents a high unmet medical need. Millions of people suffer from celiac disease globally, yet there are no approved treatments available and patients do not have any other option than adhering to a strict gluten-free diet. We approach these diseases differently from others, focusing on repairing the underlying cause instead of simply managing symptoms. To use an analogy: if it is raining into a building, it is better to fix the roof than constantly mop up the water.
Mechanistically and clinically, we have shown that this drug can stop symptoms, improve gut function, and prevent structural damage to the gut wall in a gluten challenge trial. This highlights its potential to treat celiac disease effectively without immunosuppressive drugs, which could significantly improve patient outcomes. In addition, based on its mechanism, IMU-856 offers broad potential across various gastrointestinal disorders which we may evaluate in the future, too.
If all goes well and your trials succeed, how do you picture Immunic’s significance for MS patients in a couple of years?
Our focus is on research and development, but we remain open to marketing our drugs ourselves depending on partnering opportunities. We are flexible but firm on ensuring we do not undervalue our innovations. Should our trials succeed, we envision Immunic playing a major role in the MS treatment landscape, particularly within the oral drug segment. Experts predict oral treatments will retain about 40% of the market, and we aim to capture a significant portion of that space.
For progressive MS, where there are currently almost no approved treatments, our Phase 2 study aims to transform the therapeutic landscape by addressing all progressive MS subtypes. If successful, we believe our data could set a new standard for managing this underserved population. With relapsing MS, our Phase 3 trials are on track to conclude by 2026, and we look forward to delivering solutions that meet the most critical unmet needs in MS treatment.
