Data from a phase 3 study centered on RHB-104 showed positive safety and efficacy results for the treatment of Crohn’s disease.
The manufacturer, RedHill Biopharma, said the drug was superior to placebo in achieving remission at 26 weeks, according to a press release.
“The robust results of this study demonstrate that RHB-104 could become a leading therapeutic option in Crohn’s disease and bring hope to patients worldwide,” Ira Kalfus, MD, medical director for RedHill said in the release. “The availability of antibiotic therapy for treating Crohn’s disease could be transformative.”
The drug is an orally-administered antibiotic combination therapy that targets Mycobaterium avium subspecies paratuberculosis (MAP), a suspected trigger for CD in susceptible patients.
The MAP US study comprised 331 patients with moderate-to-severe active Crohn’s disease defined as a Crohn’s Disease Activity Index (CDAI) Score between 220 and 450. Investigators randomly assigned patients to receive either RHB-104 or placebo, in addition to baseline background medication including 5-ASAs, corticosteroids, immunomodulators or anti-TNF alpha agents.
Researchers found that the drug met the study’s primary endpoint by demonstrating superiority over placebo in achieving remission at week 26; remission was defined as a CDAI score less than 150. A larger percentage of patients who received RHB-104 achieved remission compared with patients who received placebo (37% vs. 23%, P = .013). More of the RHB cohort achieved early remission at week 16 of the trial (42% vs. 29%, P = .019).
The study also found that the drug achieved secondary endpoints in durable remission and demonstrated safety and tolerability among patients, according to the press release.
Lead investigator David Graham, MD, MACG, said the results of the study could lead to a shift in the treatment of CD.
“Many patients with Crohn’s disease do not achieve remission on current standard-of-care therapies, which are accompanied with poor side effects,” he said in the press release. “RHB-104 appears to have the potential to become a promising, new, orally-administered therapy for this important debilitating disease.”
RedHill plans to meet with officials from the FDA, as well as key leaders in the field of CD, about a potential path to approval. The company expects additional studies will be needed to support a future new drug application, according to the press release.
Disclosures: Kalfus is employed by RedHill Biopharma. Graham is a lead investigator in the MAP US study.