3 things to know about the future of small molecule therapeutics

Immunic is changing the game for MS and chronic intestinal diseases.

We see a great demand for effective therapies, particularly for conditions with limited treatment options. Transformative treatments, no matter how small, have the impact potential to improve even the most challenging health conditions.

Immunic is known for innovative work within small molecule therapeutics and immunology. Headquartered in New York City with a subsidiary in Grafelfing, Germany, the company is focused on clinical development for patients with chronic inflammatory and autoimmune diseases.

Since 2016, Immunic has made significant strides to revolutionize therapy programs for patients with rare diseases worldwide. And Immunic has successfully identified several therapeutic targets establishing a promising future for drugs with the potential to alter the course of several difficult-to-treat conditions.

At the Biotechnology Innovation Organization (BIO) 2023 International Convention, we had the chance to sit down with Immunic CEO, President, and Director, Dr. Daniel Vitt, about the latest developments. Here are three things we learned about Immunic and the promise of small molecule therapy.

(Editor’s Note: Conversation has been edited for clarity and length.)

1. Small molecule therapy can restore bowel function – and regenerate bowel epithelium.

IMU-856 was discovered in 2018. As an orally available small molecule modulator, it targets the protein Sirtuin 6 (SIRT6) and acts to restore intestinal barrier function and regenerate bowel epithelium. The benefits of bowel restoration are significant to patients suffering from inflammatory bowel disease, irritable bowel syndrome (IBS), Crohn’s and Ulcerative colitis (UC), and gastrointestinal disease (celiac disease). Preclinical findings also suggest its benefit in maintaining immune surveillance during immunosuppressive therapy.

Celiac disease is another autoimmune condition that stems from an immune reaction to a degradation product of gluten. Its malabsorptive symptoms affect 1 in 100 individuals worldwide, though 60-70% of patients in the United States remain undiagnosed. In May of 2023, positive results from phase 1 trials were reported for IMU-856 surrounding the pathophysiology of celiac disease including histology, biomarkers, nutrient absorption, and patient symptoms. It was notably well-tolerated by subjects and is now being prepared for phase 2 clinical testing.

2. Small molecule therapy shows promise in treating MS.

Another program making excellent progress involves vidofludimus calcium (IMU-838). As a selective immune modulator, IMU-838 inhibits the intracellular metabolism of activated B and T cells by blocking dihydroorotate dehydrogenase (DHODH). Through its effects on the intracellular metabolism of activated immune cells, there is a reduction in inflammation and tissue damage, with potential shown in the treatment of multiple sclerosis.

In May 2023, preclinical data was published in the Journal of Medicinal Chemistry regarding vidofludimus calcium and its additional mechanism of action as a potent nuclear receptor related 1 (NURR 1) activator. The relevance of this finding is contributed to the drug’s postulated neuroprotective effects which are thought to support previous findings of disability-worsening events in MS patients.

The vidofludimus calcium (IMU-838) program is in phase 3 clinical trial for multiple sclerosis (MS) and showed positive therapeutic effects in phase 2 clinical trials in patients with relapsing-remitting MS (RRMS) as well as moderate to severe ulcerative colitis (UC).

3. Immunic is inducing and retaining remission of Crohn’s and UC – and perhaps slowing down the progression of MS.

Given autoimmune diseases arise when the immune system mistakenly attacks healthy cells in the body, Immunic is dedicated to the development of therapies that modulate the immune system’s response to effectively treat these diseases.

According to the Centers for Disease Control and Prevention (CDC), approximately 3.1 million adults in the United States have been diagnosed with inflammatory bowel disease (including Crohn’s disease and ulcerative colitis). Over 70,000 new cases are diagnosed annually. IMU-838 has shown tremendous promise in the treatment of both Crohn’s and UC. Successful clinical trials have demonstrated the potential to induce and maintain remission, leading to improved patient quality of life. Given current treatments often involve immunosuppressive medications with accompanying side effects, Immunic aims to provide enhanced efficacy and a favorable safety profile, minimizing the risks associated with long-term immunosuppression.

The impact of multiple sclerosis as a progressive demyelinating disease is significant, affecting over 1 million individuals in the United States and approximately 2.8 million individuals worldwide. Among progressive (primary and secondary) and relapsing forms, RMS is more common and has sparked a lot of interest in understanding the extent to which disability is secondary to relapse versus an unknown, yet independent variable. As typical detection of MS lesions is measured via MRI with gadolinium, a new treatment would work via a mechanism independent of acute inflammation.  Dr. Vitt’s belief is that many symptoms come from a continuous process that is also immune-driven but not visible on MRI. As there are minimal treatment options available for RRMS, there is a general consensus that treatment must be started early to postpone disability events for years.

By regulating the immune response and reducing inflammation, IMU-838 may help to alleviate symptoms and manage disease progression. As a unique mechanism associated with IMU-838, there is the potential to modify the course of MS by slowing down disease progression, reducing relapses, and ultimately improving long-term outcomes.

Additionally, Immunic therapies are orally administered, offering convenience improving compliance, eliminating discomfort secondary to injections and frequent hospitalizations, and enhancing the patient’s overall treatment experience.

What’s next for Immunic?

Immunic aims to “successfully develop and commercialize therapeutics to improve the lives of those suffering from debilitating disease across the globe,” says Dr. Vitt. By understanding the unique genetic and immunological profiles of patients, the company will have the unique ability to tailor therapies to individual needs, maximizing efficacy and minimizing side effects.

Immunic is undoubtedly a key player in the field of immunology and therapeutics with its conscientious pursuit of innovative solutions for autoimmune and chronic inflammatory disorders. Based on recent developments and success within existing platforms, the company is making its way to transform the lives of millions of patients across the globe.

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