RedHill Biopharma's interim results from Phase IIa PoC study supporting therapeutic potential of RHB-104 in multiple sclerosis

RedHill Biopharma's interim results from Phase IIa proof-of-concept study supporting therapeutic potential of RHB-104 in multiple sclerosis

Ira Kalfus MD, 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 proof-of-concept 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.”

RHB-104 is a proprietary, orally administered, potentially groundbreaking antibiotic combination therapy with potent intracellular, anti-mycobacterial and anti-inflammatory properties. The ongoing CEASE-MS Phase IIa clinical study is a single-arm, open-label PoC study evaluating fixed oral dose RHB-104 as add-on therapy to interferon beta-1a for the treatment of RRMS. This study follows positive pre-clinical research findings and provides clinical evidence of RHB-104’s potential as a treatment for MS. The study was designed as an uncontrolled, non-powered, single-arm, open-label PoC study with the objective of evaluating the safety and potential efficacy of RHB-104 using a series of exploratory measures. Eighteen RRMS patients were enrolled in two clinical centers in Israel. The patients received 24 weeks of treatment with RHB-104 and the interim data are presented in this press release. The patients are currently being evaluated for an additional follow-up period of 24 weeks after completing treatment with RHB-104. Independent third parties provided RedHill with the interim data sets and analysis, which remain subject to finalization and completion of the 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.”