RedHill Biopharma Ltd (RedHill), a biopharmaceutical company primarily focused on the development and commercialization of late clinical-stage, proprietary, orally-administered, small molecule drugs for inflammatory and gastrointestinal diseases, including cancer, has announced encouraging top-line interim results from its ongoing CEASE-MS Phase IIa proof-of-concept (PoC) clinical study evaluating fixed oral dose RHB-104 in patients treated for relapsing-remitting multiple sclerosis (RRMS).

Ira Kalfus, medical director of RedHill and the CEASE-MS study, said: “We are very pleased with the interim results from the ongoing CEASE-MS Phase IIa PoC study with RHB-104 for relapsing-remitting multiple sclerosis (RRMS). The initial findings from the study, including safety, clinical and MRI, support the therapeutic potential of RHB-104 as add-on therapy in RRMS.” Dr. Kalfus added: “Although designed as an exploratory proof-of-concept study in a very small patient population and not powered for efficacy, the study interim results demonstrate positive safety data and clinical signals, supporting additional studies to better investigate the therapeutic potential of RHB-104 in RRMS. 88% of the mITT patient population and 100% of the per-protocol population were relapse free at 24 weeks of treatment. Importantly, a positive efficacy signal was seen in reduction of total T2 lesion volume at 24 weeks compared to baseline. Moreover, EDSS scores were stable with suggestion of improvement. Notably, no patients experienced an increase in total EDSS, further underscoring the therapeutic potential of RHB-104 for the treatment of RRMS. RHB-104 was observed to be safe and well-tolerated with no clinically relevant or unexpected adverse events, further reinforcing our confidence in this therapeutic candidate as we continue to advance the ongoing Phase III MAP US study with RHB-104 for Crohn’s disease. We are looking forward to further analyses of the data upon completion of the 48 week CEASE-MS study. Patients are now completing their 24 week follow up treatment period, off RHB-104, and final results of the completed 48 week study are expected during the second half of 2016. Analysis of the completed study will drive next steps in the development path of RHB-104 for MS including identification of the patient population, comparator and clinical endpoints to be investigated in the next study. We would like to thank the patients, investigators and clinical support staff who are participating in this important ongoing study.”

Dr. Radi Shahien MD of Ziv Medical Center in Safed, Israel, principal investigator of the CEASE-MS study added: “RRMS is a devastating disease with no known cure, limited treatment options and unknown cause, hence the importance of the development of RHB-104, an intracellularly acting, anti-mycobacterial, anti-inflammatory and orally administered drug candidate. CEASE-MS is a small, open-label, exploratory study intended to investigate the groundbreaking hypothesis that a bacterial induced dysregulated immune system plays a critical role in the pathogenesis of MS. RedHill’s CEASE-MS study with RHB-104 is the first clinical study to evaluate the therapeutic potential of a triple antibiotic combination therapy, and specifically these intracellularly acting antibiotics, as add-on therapy in RRMS. The initial analysis of the study’s interim results provides encouraging clinical signals as well as important and reassuring safety data. In particular, the reduction of total T2 lesion burden over baseline, a key MS disease burden measurement, as well as relapse-free and stable EDSS data, are positive and meaningful signals, particularly given the small patient population in the study and the early time point of the data generated to date. I find the interim results very promising and am encouraged by the potential efficacy of RHB-104 in treating MS. I look forward to the completion of the study, further analyses and presentation of the final data at the appropriate international medical and academic forums.”

“We are very encouraged by these findings which further reinforce the potential role of RHB-104 in the field of auto inflammatory diseases. We are conducting the Phase III MAP US study for Crohn’s disease with RHB-104, with interim DSMB analysis expected in the second half of 2016, and the CEASE-MS study adds important information to the body of knowledge about the drug,” stated Dror Ben-Asher, RedHill’s Chief Executive Officer. “With a strong balance sheet and three ongoing Phase III programs in gastroenterology with planned data points in 2016 - RHB-104 for Crohn’s disease, RHB-105 for H. pylori infection and BEKINDA for acute gastroenteritis - RedHill is well positioned for continued growth in 2016 and beyond.”