The trial, called EMPhASIS, also exceeded its target number of participants: Immunic set an initial recruitment goal of 195 patients by mid-2020, and enrolled 210 at sites across four European countries.
“Achievement of this milestone for our lead program, IMU-838, in RRMS, substantially ahead of prior expectations, reflects not only our team’s strength in executing on the company’s drug development strategy, but also the urgent need from patients for a safer oral treatment option,” Daniel Vitt, PhD, Immunic’s CEO and president, said in a press release.
IMU-838 (vidofludimus calcium) is a small, selective immunomodulator being developed, as an oral tablet, to treat RRMS, inflammatory bowel disease (IBD), and other chronic inflammatory and autoimmune diseases. It works by blocking an enzyme called dihydroorotate dehydrogenase (DHODH), which dampens inflammation mediated by immune T- and B-cells.
Immunic suggests that a key advantage of DHODH inhibition is its selectivity toward metabolically active immune cells, leaving other bone marrow cells unaffected.
EMPhASIS (NCT03846219) will evaluate the safety and efficacy of two oral doses of IMU-838 (30 or 45 mg/day) given once daily against placebo in RRMS patients for 24 weeks. All enrolled in the trial have evidence of active disease, as assessed through clinical evidence of relapse and signs of active lesions (gadolinium positive) on magnetic resonance imaging (MRI) scans.
Patients were enrolled at 36 centers across Romania, Bulgaria, Ukraine, and Poland starting in February.
The primary efficacy measure is the effects of treatment on disease progression, based on the cumulative number of combined unique active MRI lesions up to week 24.
Those completing the trial can choose to continue or start treatment (placebo group) in an open-label extension study meant to assess the long-term safety and tolerability of IMU-838.
Top-line study results are anticipated to become available during the third quarter of 2020.
Two Phase 1 studies of single or repeated once-daily doses of IMU-838 in healthy volunteers were done in 2017, and showed repeated daily doses of up to 50 mg of the potential therapy were safe and tolerable.
“Backed by newly released preclinical data confirming the superior profile of IMU-838 versus the currently approved DHODH inhibitor, teriflunomide [Aubagio], we remain highly enthusiastic about the potential of IMU-838 to become an important new best-in-class oral therapeutic treatment alternative for patients with RRMS,” Vitt said.
IMU-838 is also being investigated as a treatment for ulcerative colitis (a form of IBD) in a Phase 2 trial, and as a treatment for primary sclerosing cholangitis (a disease of the bile ducts) in a proof-of-concept trial.