• The Phase IIa proof-of-concept study evaluates the safety and potential efficacy of fixed oral dose RHB-104 as an add-on therapy to interferon beta-1a for relapsing-remitting multiple sclerosis (RRMS)
• Analysis of the study is ongoing, with top-line final results expected in the fourth quarter of 2016
• Previously announced interim results after completion of the 24-week RHB-104 treatment period of the study demonstrated positive safety and efficacy signals and support further clinical development
• 2016 U.S. and worldwide sales of multiple sclerosis therapies are estimated to exceed $12 billion and $18 billion, respectively
• RHB-104 is also being evaluated as a treatment for Crohn's disease with an ongoing first Phase III clinical study (the MAP US study) with interim DSMB analysis expected in the fourth quarter of 2016
Tel-Aviv, Israel, August 1, 2016 / B3C newswire / -- RedHill Biopharma Ltd. (NASDAQ: RDHL) (TASE: RDHL) (“RedHill” or the “Company”), a biopharmaceutical company primarily focused on development and commercialization of late clinical-stage, proprietary, orally-administered, small molecule drugs for inflammatory and gastrointestinal diseases and cancer, today announced that the last patient has completed the final scheduled follow-up visit in the Phase IIa proof-of-concept clinical study evaluating RHB-104 in patients treated for relapsing-remitting multiple sclerosis (RRMS).
The open label Phase IIa study (the CEASE-MS study) enrolled eighteen patients suffering from RRMS and was designed with a series of exploratory endpoints to evaluate the safety and potential efficacy of fixed oral dose RHB-104 as an add-on therapy to interferon beta-1a.Patients received treatment with RHB-104 for 24 weeks and were evaluated for an additional 24-week follow-up period during which they were treated with interferon beta-1a without RHB-104 add-on. The analysis of the study is currently ongoing and top-line final results are expected to be announced in the fourth quarter of 2016, subject to completion of review requirements and completion of the clinical study report (CSR).
RHB-104 is a proprietary and potentially groundbreaking antibiotic combination therapy in oral capsule formulation, with potent intracellular, anti-mycobacterial and anti-inflammatory properties. Multiple sclerosis (MS) is a chronic inflammatory, demyelinating disease of the central nervous system (CNS) with an unknown etiology, believed to be multifactorial. Thought to be autoimmune, the MS inflammatory process is also consistent with persistent infection. The 2016 U.S. and worldwide sales of MS therapies are estimated to exceed $12 billion and $18 billion, respectively(1).
RedHill announced in March 2016 encouraging top-line interim results from the single-arm, open-label CEASE-MS study. Top-line interim results, after completion of the 24-week treatment period of the study, demonstrated positive safety and efficacy signals and support further clinical development, based on encouraging preliminary data.
As previously announced, the top-line interim results demonstrated an annualized relapse rate (ARR) at 24 weeks of 0.288 in the modified intent-to-treat (mITT) population and 0.0 in the per-protocol (PP) population, comparing favorably with previously reported pivotal studies of interferon beta-1a therapies Avonex® (0.67) (2) and Rebif® (0.87-0.91) (3).
88% of the mITT patient population and 100% of the PP patient population were relapse free at 24 weeks, comparing favorably with previously reported pivotal data on the use of Rebif® (75%) in comparison with Avonex® (63%) as standalone first-line therapies(4). No patient in the CEASE-MS study relapsed after week 8 of treatment.
Expanded Disability Status Scale (EDSS) scores, a standard measure of MS disability, indicated the disease was stable during the treatment period and there was a signal of improvement; No increase in total EDSS was observed in any of the patients in the study.
With only a single active T1 post gadolinium lesion noted among all patients followed, combined unique active lesions (CUAs) - the primary outcome measure in the CEASE-MS study - were almost entirely MRI T2 lesions. Although not powered for efficacy, a reduction in total MRI T2 lesion volume was observed at 24 weeks, as compared to baseline, suggesting a decreased burden of disease and comparing favorably with previously reported Avonex®(5) and Rebif®(6) data. No clinically significant change was observed for total CUA lesions at week 24, which is supportive of a stable disease state.
RHB-104 was found to be safe and well tolerated, with no drug-related serious adverse events or other clinically relevant or unexpected adverse events.
RHB-104 is a multifaceted drug that, in addition to bactericidal properties against intracellular infections, has potentially distinct mechanisms of action that include both anti-inflammation and neuroprotection. The Phase IIa CEASE-MS study was initiated following several successful pre-clinical studies conducted by RedHill with RHB-104.
RHB-104 is also currently undergoing a first Phase III study for Crohn’s disease in the U.S., Canada, Israel, Australia and Europe (the MAP US study). Interim data and safety monitoring board (DSMB) analysis of the ongoing randomized, double-blind, placebo-controlled MAP US study is expected in the fourth quarter of 2016.
The MAP US Phase III study and the CEASE-MS Phase IIa study are registered on www.ClinicalTrials.gov, a web-based service of the U.S. National Institutes of Health, which provides access to information on publicly and privately supported clinical studies.
About Multiple Sclerosis
Multiple sclerosis (MS) is a chronic inflammatory, demyelinating disease of the central nervous system with an unknown etiology, believed to be multifactorial. A dysfunctional immune system in MS patients causes recurrent inflammatory attacks on the central nervous system (CNS), leading to neurological disability. Diffuse inflammatory and demyelinating lesions, also known as plaques, are the main pathological finding in MS neural tissue. The lesions are primarily found in the spinal cord, optic nerves, brainstem and periventricular white matter. The symptoms of MS are dictated by the location of the lesions within the CNS. Geographic variation in MS distribution, which cannot be solely explained by population genetics, supports the notion that environmental factors also hold etiological importance. There is currently no known cure for MS and available treatments are mainly intended to manage or prevent relapses or reduce symptoms. In 2015, there were estimated to be over 900,000 diagnosed patients with MS worldwide. Approximately 85% of MS patients initially exhibit relapse-remitting disease (RRMS). The 2016 U.S. and worldwide sales of MS therapies are estimated to exceed $12 billion and $18 billion, respectively(7).
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 and potentially groundbreaking oral antibiotic combination therapy, with potent intracellular, anti-mycobacterial and anti-inflammatory properties. RHB-104 is based on increasing evidence supporting the hypothesis that Crohn’s disease is caused by Mycobacterium avium subspecies paratuberculosis (MAP) infection in susceptible patients. Clinical trials conducted with earlier formulations of RHB-104 include an Australian Phase III study conducted by 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. RHB-104 is covered by several issued and pending patents. RedHill is also conducting the CEASE-MS 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 (RRMS), with top-line interim results announced.
About RedHill Biopharma Ltd.
RedHill Biopharma Ltd. (NASDAQ/TASE: RDHL) is a 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 inflammatory and gastrointestinal diseases and cancer. RedHill’s current pipeline of proprietary products includes: (i) RHB-105 - an oral combination therapy for the treatment of Helicobacter pylori infection with successful results from a first Phase III study; (ii) RHB-104 - an oral combination therapy for the treatment of Crohn's disease with an ongoing first Phase III study and an ongoing proof-of-concept Phase IIa study for multiple sclerosis; (iii) BEKINDA™ (RHB-102) - a once-daily oral pill formulation of ondansetron with an ongoing Phase III study in the U.S. 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 uPA inhibitor, administered by oral capsule, targeting gastrointestinal and other solid tumors; (vii) RP101 - currently subject to an option-to-acquire by RedHill, RP101 is a Phase II-stage first-in-class Hsp27 inhibitor, administered by oral tablet, targeting pancreatic and other gastrointestinal cancers; (viii) 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 Germany in October 2015; and (ix) RHB-101 - a once-daily oral pill formulation of the cardio drug carvedilol.
• Analysis of the study is ongoing, with top-line final results expected in the fourth quarter of 2016
• Previously announced interim results after completion of the 24-week RHB-104 treatment period of the study demonstrated positive safety and efficacy signals and support further clinical development
• 2016 U.S. and worldwide sales of multiple sclerosis therapies are estimated to exceed $12 billion and $18 billion, respectively
• RHB-104 is also being evaluated as a treatment for Crohn's disease with an ongoing first Phase III clinical study (the MAP US study) with interim DSMB analysis expected in the fourth quarter of 2016
Tel-Aviv, Israel, August 1, 2016 / B3C newswire / -- RedHill Biopharma Ltd. (NASDAQ: RDHL) (TASE: RDHL) (“RedHill” or the “Company”), a biopharmaceutical company primarily focused on development and commercialization of late clinical-stage, proprietary, orally-administered, small molecule drugs for inflammatory and gastrointestinal diseases and cancer, today announced that the last patient has completed the final scheduled follow-up visit in the Phase IIa proof-of-concept clinical study evaluating RHB-104 in patients treated for relapsing-remitting multiple sclerosis (RRMS).
The open label Phase IIa study (the CEASE-MS study) enrolled eighteen patients suffering from RRMS and was designed with a series of exploratory endpoints to evaluate the safety and potential efficacy of fixed oral dose RHB-104 as an add-on therapy to interferon beta-1a.Patients received treatment with RHB-104 for 24 weeks and were evaluated for an additional 24-week follow-up period during which they were treated with interferon beta-1a without RHB-104 add-on. The analysis of the study is currently ongoing and top-line final results are expected to be announced in the fourth quarter of 2016, subject to completion of review requirements and completion of the clinical study report (CSR).
RHB-104 is a proprietary and potentially groundbreaking antibiotic combination therapy in oral capsule formulation, with potent intracellular, anti-mycobacterial and anti-inflammatory properties. Multiple sclerosis (MS) is a chronic inflammatory, demyelinating disease of the central nervous system (CNS) with an unknown etiology, believed to be multifactorial. Thought to be autoimmune, the MS inflammatory process is also consistent with persistent infection. The 2016 U.S. and worldwide sales of MS therapies are estimated to exceed $12 billion and $18 billion, respectively(1).
RedHill announced in March 2016 encouraging top-line interim results from the single-arm, open-label CEASE-MS study. Top-line interim results, after completion of the 24-week treatment period of the study, demonstrated positive safety and efficacy signals and support further clinical development, based on encouraging preliminary data.
As previously announced, the top-line interim results demonstrated an annualized relapse rate (ARR) at 24 weeks of 0.288 in the modified intent-to-treat (mITT) population and 0.0 in the per-protocol (PP) population, comparing favorably with previously reported pivotal studies of interferon beta-1a therapies Avonex® (0.67) (2) and Rebif® (0.87-0.91) (3).
88% of the mITT patient population and 100% of the PP patient population were relapse free at 24 weeks, comparing favorably with previously reported pivotal data on the use of Rebif® (75%) in comparison with Avonex® (63%) as standalone first-line therapies(4). No patient in the CEASE-MS study relapsed after week 8 of treatment.
Expanded Disability Status Scale (EDSS) scores, a standard measure of MS disability, indicated the disease was stable during the treatment period and there was a signal of improvement; No increase in total EDSS was observed in any of the patients in the study.
With only a single active T1 post gadolinium lesion noted among all patients followed, combined unique active lesions (CUAs) - the primary outcome measure in the CEASE-MS study - were almost entirely MRI T2 lesions. Although not powered for efficacy, a reduction in total MRI T2 lesion volume was observed at 24 weeks, as compared to baseline, suggesting a decreased burden of disease and comparing favorably with previously reported Avonex®(5) and Rebif®(6) data. No clinically significant change was observed for total CUA lesions at week 24, which is supportive of a stable disease state.
RHB-104 was found to be safe and well tolerated, with no drug-related serious adverse events or other clinically relevant or unexpected adverse events.
RHB-104 is a multifaceted drug that, in addition to bactericidal properties against intracellular infections, has potentially distinct mechanisms of action that include both anti-inflammation and neuroprotection. The Phase IIa CEASE-MS study was initiated following several successful pre-clinical studies conducted by RedHill with RHB-104.
RHB-104 is also currently undergoing a first Phase III study for Crohn’s disease in the U.S., Canada, Israel, Australia and Europe (the MAP US study). Interim data and safety monitoring board (DSMB) analysis of the ongoing randomized, double-blind, placebo-controlled MAP US study is expected in the fourth quarter of 2016.
The MAP US Phase III study and the CEASE-MS Phase IIa study are registered on www.ClinicalTrials.gov, a web-based service of the U.S. National Institutes of Health, which provides access to information on publicly and privately supported clinical studies.
About Multiple Sclerosis
Multiple sclerosis (MS) is a chronic inflammatory, demyelinating disease of the central nervous system with an unknown etiology, believed to be multifactorial. A dysfunctional immune system in MS patients causes recurrent inflammatory attacks on the central nervous system (CNS), leading to neurological disability. Diffuse inflammatory and demyelinating lesions, also known as plaques, are the main pathological finding in MS neural tissue. The lesions are primarily found in the spinal cord, optic nerves, brainstem and periventricular white matter. The symptoms of MS are dictated by the location of the lesions within the CNS. Geographic variation in MS distribution, which cannot be solely explained by population genetics, supports the notion that environmental factors also hold etiological importance. There is currently no known cure for MS and available treatments are mainly intended to manage or prevent relapses or reduce symptoms. In 2015, there were estimated to be over 900,000 diagnosed patients with MS worldwide. Approximately 85% of MS patients initially exhibit relapse-remitting disease (RRMS). The 2016 U.S. and worldwide sales of MS therapies are estimated to exceed $12 billion and $18 billion, respectively(7).
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 and potentially groundbreaking oral antibiotic combination therapy, with potent intracellular, anti-mycobacterial and anti-inflammatory properties. RHB-104 is based on increasing evidence supporting the hypothesis that Crohn’s disease is caused by Mycobacterium avium subspecies paratuberculosis (MAP) infection in susceptible patients. Clinical trials conducted with earlier formulations of RHB-104 include an Australian Phase III study conducted by 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. RHB-104 is covered by several issued and pending patents. RedHill is also conducting the CEASE-MS 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 (RRMS), with top-line interim results announced.
About RedHill Biopharma Ltd.
RedHill Biopharma Ltd. (NASDAQ/TASE: RDHL) is a 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 inflammatory and gastrointestinal diseases and cancer. RedHill’s current pipeline of proprietary products includes: (i) RHB-105 - an oral combination therapy for the treatment of Helicobacter pylori infection with successful results from a first Phase III study; (ii) RHB-104 - an oral combination therapy for the treatment of Crohn's disease with an ongoing first Phase III study and an ongoing proof-of-concept Phase IIa study for multiple sclerosis; (iii) BEKINDA™ (RHB-102) - a once-daily oral pill formulation of ondansetron with an ongoing Phase III study in the U.S. 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 uPA inhibitor, administered by oral capsule, targeting gastrointestinal and other solid tumors; (vii) RP101 - currently subject to an option-to-acquire by RedHill, RP101 is a Phase II-stage first-in-class Hsp27 inhibitor, administered by oral tablet, targeting pancreatic and other gastrointestinal cancers; (viii) 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 Germany in October 2015; and (ix) RHB-101 - a once-daily oral pill formulation of the cardio drug carvedilol.
