Immunic, Inc. Announces Positive Top-Line Data From Investigator-Sponsored Phase 2 Proof-of-Concept Clinical Trial of IMU-838 in Primary Sclerosing Cholangitis
NEW YORK, NY, USA - February 18, 2021 - Immunic, Inc. (Nasdaq: IMUX), a clinical-stage biopharmaceutical company developing a pipeline of selective oral immunology therapies aimed at treating chronic inflammatory and autoimmune diseases, today announced positive top-line data from an investigator-sponsored phase 2 proof-of-concept clinical trial of IMU-838 in primary sclerosing cholangitis (PSC). This single-arm, open-label, exploratory study was designed to investigate IMU-838's potential to improve various biochemical parameters in PSC patients and help determine whether any such activity warrants further investigation in randomized PSC trials. As previously announced, due to the COVID-19 pandemic, only 18 of the targeted 30 patients were enrolled in the study (intent-to-treat population, ITT), of whom only 11 patients completed the full IMU-838 treatment course and were evaluable over the 24-week treatment period (per-protocol population, PP).
The PP population experienced a statistically significant decrease in serum alkaline phosphatase (ALP) levels (p=0.041) after 24 weeks of treatment using 30 mg IMU-838 once daily, as compared to baseline. A consistent individual pattern of a stable decrease in ALP values was observed in the PP population between baseline and week 24, without any single patient showing an increase of more than 20% of ALP. As per the definition of the primary objective of the study, 27.3% of the patients in the PP population had a clinically relevant reduction of serum ALP higher than 25% at week 24, without an increase in liver biochemistry of more than 33%, as compared to baseline. Biochemical endpoints, such as changes in serum ALP, have been used in PSC trials performed by third parties.
Regarding the secondary objectives of the study, no changes in aspartate aminotransferase (AST), alanine aminotransferase (ALT), or total, direct or indirect bilirubin were observed in the ITT or PP populations, as compared to baseline. In addition, despite the limited scope of the data, encouraging results were observed regarding symptoms of inflammatory bowel disease, a common comorbidity for PSC patients, and patient assessments of health-related quality of life. The study also found that IMU-838 is a safe and well-tolerated oral drug for PSC patients and treatment-emergent adverse events were rare and generally mild.
"I am very excited about the effects we have seen in this highly underserved patient population where there is only a small number of cases worldwide and where no pharmaceutical treatment option is currently available," noted Daniel Vitt, Ph.D., Chief Executive Officer and President of Immunic. "We are also very pleased to see that IMU-838's safety and tolerability profile was confirmed in this patient group. The results from this small, open-label study suggest that IMU-838 merits further clinical testing in PSC. We are in discussions with investigators and leading clinical experts to further evaluate the data set and to explore potential next steps for this indication."
"Currently, no effective treatment options are available for PSC patients and the hepatology community is very keen to see new approaches and clinical programs for the investigation of promising new approaches. I am grateful that Mayo Clinic and Immunic are collaboratively exploring this underserved indication for which liver transplantation is often the only effective option," stated Keith Lindor, M.D., Professor of Medicine Emeritus and former President of the American Association for the Study of Liver Diseases. "Although we are mindful of the small size of this dataset, I do believe the results are noteworthy and merit further exploration. Notable in this small patient cohort is the absolute consistency with which these patients experienced decreases in serum alkaline phosphatase at the 24-week time point."
About Primary Sclerosing Cholangitis (PSC)
PSC is a rare liver disease with a prevalence of approximately 4.15 per 100,000 in the United States, in which the bile ducts in the liver become inflamed, narrow and prevent bile from flowing properly. The exact cause and disease mechanism of PSC are still unknown, but an autoimmune mechanism may play a role. There is an association with inflammatory bowel diseases, most often with ulcerative colitis and less commonly with Crohn's disease. PSC is a progressive disease and, other than liver transplantation, there are currently no approved therapies that have been shown to improve survival in patients with PSC. The estimated time from diagnosis of PSC to death or liver transplant has been shown to be less than 15 years.
About IMU-838
IMU-838 is an orally available, next-generation selective immune modulator that inhibits the intracellular metabolism of activated immune cells by blocking the enzyme dihydroorotate dehydrogenase (DHODH). IMU-838 acts on activated T and B cells while leaving other immune cells largely unaffected and allows the immune system to stay functioning, e.g. in fighting infections. In previous trials, IMU-838 did not show an increased rate of infections compared to placebo. In addition, DHODH inhibitors, such as IMU-838, are known to possess a host-based antiviral effect, which is independent with respect to specific virus proteins and their structure. Therefore, DHODH inhibition may be broadly applicable against multiple viruses. IMU-838 was successfully tested in two phase 1 clinical trials in 2017 and is currently being tested in a phase 2 trial in patients with ulcerative colitis. In the third quarter of 2020, the company reported positive results from its phase 2 EMPhASIS trial of IMU-838 in relapsing-remitting multiple sclerosis, achieving both primary and key secondary endpoints with high statistical significance. In the first quarter of 2021, Immunic announced that IMU-838 has shown evidence of clinical activity in its phase 2 CALVID-1 trial in hospitalized patients with moderate COVID-19. Also, in the first quarter of 2021, the company reported positive top-line data from an investigator-sponsored phase 2 proof-of-concept clinical trial of IMU-838 in primary sclerosing cholangitis which was conducted in collaboration with Mayo Clinic. To date, IMU-838 has been tested in more than 800 individuals and has shown an attractive pharmacokinetic, safety and tolerability profile. IMU-838 is not yet licensed or approved in any country.
About Immunic, Inc.
Immunic, Inc. (Nasdaq: IMUX) is a clinical-stage biopharmaceutical company with a pipeline of selective oral immunology therapies aimed at treating chronic inflammatory and autoimmune diseases, including relapsing-remitting multiple sclerosis, ulcerative colitis, Crohn's disease, and psoriasis. Immunic is developing three small molecule products: its lead development program, IMU-838, is a selective immune modulator that inhibits the intracellular metabolism of activated immune cells by blocking the enzyme DHODH and exhibits a host-based antiviral effect; IMU-935 is an inverse agonist of RORγt; and IMU-856 targets the restoration of the intestinal barrier function. For further information, please visit: www.imux.com.
SOURCE: Immunic
