• The second independent Data and Safety Monitoring Board (DSMB) meeting of the RHB-104 Phase III study for Crohn’s disease (MAP US study) is planned to be held in late July 2017 and will assess the safety and efficacy of RHB-104 in the first 222 subjects who have completed week 26 assessments
• The DSMB meeting will include an interim efficacy analysis and an evaluation of an option for early stop for success for overwhelming efficacy; its recommendation is planned to be announced by early August 2017
• To date, approximately 300 patients of the planned total of 410 patients have been enrolled in the ongoing Phase III MAP US study
• The MAP US study is a randomized, double-blind, placebo-controlled Phase III study evaluating the safety and efficacy of RHB-104 in patients with moderately to severely active Crohn’s disease, with a primary endpoint of remission at week 26
• An ongoing open-label extension Phase III study (MAP US2 study) is evaluating the safety and efficacy of RHB-104 in patients who have completed 26 weeks of treatment in the Phase III MAP US study and remain with active Crohn’s disease; these patients have the opportunity to receive treatment with RHB-104 for a 52-week period in the Phase III open-label extension study

RedHill Biopharma Ltd. (NASDAQ: RDHL) (Tel-Aviv Stock Exchange: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company primarily focused on the development and commercialization of late clinical-stage, proprietary, orally-administered, small molecule drugs for gastrointestinal and inflammatory diseases and cancer, today announced that the second independent Data and Safety Monitoring Board (DSMB) meeting of the RHB-104 Phase III study for Crohn’s disease (the MAP US study) is expected to convene in late July 2017 and will assess the safety and efficacy of RHB-104 in the first 222 subjects who have completed week 26 assessments. RedHill expects to announce the recommendation of the DSMB meeting by early August 2017.  

The independent DSMB meeting will conduct safety and interim efficacy analyses and will evaluate the option for an early stop for success for overwhelming efficacy, according to a pre-specified statistical significance threshold for overwhelming efficacy of RHB-104 versus placebo at the primary endpoint (two-sided p-value<0.003). The primary endpoint of the MAP US study is disease remission, defined as a reduction in Crohn’s Disease Activity Index (CDAI) to less than 150 at week 26. Assuming the study is not stopped for success or inefficacy following the DSMB meeting, completion of recruitment for the MAP US study is expected by the beginning of 2018.

Additionally, the Company updated that it has completed the enrollment of approximately 300 out of the 410 subjects planned to be enrolled in the RHB-104 Phase III study for Crohn’s Disease (the MAP US study).

RHB-104 is a proprietary, orally-administered, potentially groundbreaking, antibiotic combination therapy with potent intracellular, antimycobacterial and anti-inflammatory properties, targeting a suspected underlying bacterial infectious cause of Crohn’s disease, Mycobacterium avium subspecies paratuberculosis (MAP).

The MAP US study is a randomized, double-blind, placebo-controlled first Phase III study evaluating the safety and efficacy of RHB-104 in patients with moderately to severely active Crohn’s disease (defined as CDAI between 220 and 450). The MAP US study is being conducted in up to 150 clinical sites in the U.S, Canada, Europe, Israel, Australia and New Zealand. Additional clinical data will need to be generated to support a U.S. New Drug Application (NDA) for RHB-104.

In December 2016, a first pre-planned independent DSMB meeting reviewed safety data from the ongoing MAP US study and provided a unanimous recommendation to continue the study as planned. Assuming the Phase III MAP US study is not stopped early, a third, safety-focused, independent DSMB meeting will be held once 75% of the 410 patients planned to be enrolled in the study will have completed 26 weeks of study participation.

RedHill recently initiated an open-label extension Phase III study (MAP US2 study) to the first Phase III MAP US study with RHB-104 for the treatment of Crohn’s Disease. The MAP US2 study is intended to assess the safety and efficacy of RHB-104 in patients who have completed 26 weeks of treatment in the MAP US Phase III study and remain with active Crohn’s disease (CDAI > 150) at week 26, the MAP US study’s primary endpoint. These patients have the opportunity to receive treatment with RHB-104 for a 52-week period in the open-label MAP US2 extension study. The data collected in the MAP US2 study will be supplemental to the MAP US study data. The MAP US2 study’s primary endpoint is disease remission at week 16, defined as CDAI of less than 150. The MAP US2 study is planned to enroll approximately 100 subjects in up to 150 clinical sites in the U.S., Canada, Europe, Israel, Australia and New Zealand. Additional open-label studies with RHB-104 for Crohn’s disease are planned to provide further supportive clinical data for potential future marketing applications.

About RHB-104
Currently in a first Phase III study for the treatment of Crohn’s disease (the MAP US study), RHB-104 is a proprietary, orally-administered, potentially groundbreaking oral antibiotic combination therapy, with potent intracellular, antimycobacterial and anti-inflammatory properties. RHB-104 is based on increasing evidence supporting the hypothesis that Crohn’s disease is related to Mycobacterium avium subspecies paratuberculosis (MAP) infection in susceptible patients. The development of RHB-104 is consistent with the growing awareness of the possibility that a bacterially-induced dysregulated immune system may contribute to the pathogenesis of various autoimmune diseases of unknown etiology. Clinical trials conducted with earlier formulations of RHB-104 include an Australian Phase III study conducted by Pharmacia/Pfizer. RedHill has conducted several supportive studies with the current formulation of RHB-104 and a long-term population pharmacokinetic (pop-PK) study is ongoing as part of the Phase III MAP US study. Additionally, an open-label extension Phase III study (the MAP US2 study) is ongoing to assess the safety and efficacy of RHB-104 in patients who have completed week 26 assessments in the ongoing Phase III MAP US study and remain with active Crohn’s disease (CDAI > 150) at week 26. RHB-104 is covered by several issued and pending patents. RedHill also completed a Phase IIa, proof-of-concept clinical study, evaluating RHB-104 as an add-on therapy to interferon beta-1a in patients treated for relapsing-remitting multiple sclerosis (the CEASE MS study), with top-line final results suggesting meaningful positive safety and clinical signals upon 24 weeks of treatment with RHB-104 as an add-on therapy, thereby supporting further clinical development. RHB-104 was granted Qualified Infectious Disease Product (QIDP) designation by the U.S. FDA for the treatment of nontuberculous mycobacteria (NTM) infections, providing a Fast-Track development pathway, as well as NDA Priority Review and an additional five years of U.S. market exclusivity, if approved. RedHill is in discussions with the FDA regarding the development of RHB-104 development program for NTM infections.

About RedHill Biopharma Ltd.
RedHill Biopharma Ltd. (NASDAQ: RDHL) (Tel-Aviv Stock Exchange: RDHL) is a specialty biopharmaceutical company headquartered in Israel, primarily focused on the development and commercialization of late clinical-stage, proprietary, orally-administered, small molecule drugs for the treatment of gastrointestinal and inflammatory diseases and cancer. RedHill promotes two gastrointestinal products in the U.S. - Donnatal®, a prescription oral adjunctive drug used in the treatment of IBS and acute enterocolitis, and EnteraGam®, a medical food intended for the dietary management, under medical supervision, of chronic diarrhea and loose stools. RedHill’s clinical-stage pipeline includes: (i) TALICIA™ (RHB-105)- an oral combination therapy for the treatment of Helicobacter pylori infection with successful results from a first Phase III study and an ongoing confirmatory Phase III study; (ii) RHB-104- an oral combination therapy for the treatment of Crohn's disease with an ongoing first Phase III study, a completed proof-of-concept Phase IIa study for multiple sclerosis and QIDP status for nontuberculous mycobacteria (NTM) infections; (iii) BEKINDA® (RHB-102)- a once-daily oral pill formulation of ondansetron with successful top-line results in a Phase III study for acute gastroenteritis and gastritis and an ongoing Phase II study for IBS-D; (iv) RHB-106- an encapsulated bowel preparation licensed to Salix Pharmaceuticals, Ltd.; (v) YELIVA® (ABC294640)- a Phase II-stage, orally-administered, first-in-class SK2 selective inhibitor targeting multiple oncology, inflammatory and gastrointestinal indications; (vi) MESUPRON - a Phase II-stage first-in-class, orally-administered protease inhibitor, targeting pancreatic cancer and other solid tumors and (vii) RIZAPORT® (RHB-103) - an oral thin film formulation of rizatriptan for acute migraines, with a U.S. NDA currently under discussion with the FDA and marketing authorization received in two EU member states under the European Decentralized Procedure (DCP).