RedHill Biopharma Accelerates RHB-104 Phase III Study in Crohn's Disease with Top-Line Results Expected Mid-2018
RedHill has curtailed the target sample size in the ongoing first Phase III study with RHB-104 for Crohn's disease (MAP US) from 410 to approximately 325 subjects, of which 322 have been enrolled to date, while maintaining statistical power of over 80% with a treatment effect of 15%
review of the blended efficacy rate of the current blinded data, as well as additional input from experts, including statisticians and key opinion leaders, suggests that the total number of treatment successes is consistent with predefined expected treatment outcomes and that the study has sufficient enrollment to potentially demonstrate efficacy
Development program will be shortened by approximately one year, with enrollment expected to be completed by November 2017 and top-line results expected in mid-2018
Estimated cost saving is approximately $14 million
- RedHill will host a conference call and webcast today, Monday, October 2, 2017, at 9:00 am EDT
TEL-AVIV, Israel and RALEIGH, N.C., Oct. 02, 2017 (GLOBE NEWSWIRE) -- RedHill Biopharma Ltd. (NASDAQ:RDHL) (Tel-Aviv Stock Exchange:RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company primarily focused on late clinical-stage development and commercialization of proprietary, orally-administered, small molecule drugs for gastrointestinal and inflammatory diseases and cancer, today announced that the Company has accelerated the timelines for the ongoing first Phase III study with RHB-104 for Crohn's disease (MAP US) by curtailing the number of subjects planned to be enrolled in the study from 410 to approximately 325 subjects.
Given that 322 subjects have been enrolled in the MAP US Phase III study to date, the Company expects to complete enrollment by November 2017 and to announce top-line results in mid-2018. RedHill remains blinded to the ongoing data from the study. The protocol amendment implementing the curtailment strategy has been filed with the FDA and healthcare regulators in other relevant countries.
The MAP US study is a randomized, double-blind, placebo-controlled first Phase III study evaluating the safety and efficacy of RHB-104 in subjects with moderately to severely active Crohn's disease (defined as Crohn's Disease Active Index (CDAI) between 220 and 450). The primary endpoint of the MAP US study is disease remission, defined as a reduction in CDAI to less than 150 at week 26. The MAP US study is being conducted in up to 150 clinical sites in the U.S, Canada, Europe, Israel, Australia and New Zealand.
Ira Kalfus, MD, Medical Director at RedHill , commented: "We believe that curtailment to a target sample size of approximately 325 subjects is the preferred approach in our effort to bring this potentially groundbreaking new therapy to the market as soon as possible. A review of the blended efficacy rate of the current blinded data suggests that the total number of treatment successes at this point in the study is consistent with the predefined expected treatment outcome. Upon discussion with internal and external experts, we have concluded that the study has sufficient enrollment to potentially demonstrate efficacy within the protocol-defined 15% treatment effect (RHB-104 36% vs. placebo 21%). The blended remission rate of the currently blinded data has been consistently within or superior to our pre-specified protocol defined assumptions, indicating potential study success at the curtailed sample size of approximately 325 subjects, assuming the placebo and RHB-104 remission rates in our study are in line with trial assumptions. Placebo remission rates in similar, but not identical, pivotal studies in Crohn's disease range from approximately 7% to approximately 25%1 with the two most recently approved therapies at 7% (Entyvio® (vedolizumab))2 and 20% (Stelara® (ustekinumab))3. The Company remains blinded to the data and has no visibility into the actual treatment effect. We are excited about the significant progress achieved with RHB-104 and look forward to top-line results from the MAP US Phase III study, expected in mid-2018."
Professor David Graham, MD, M.A.C.G., renowned researcher and physician at the Baylor College of Medicine and the lead investigator of the RHB-104 MAP US Phase III study, added: "After reviewing the blinded, blended data from the patients enrolled in the Phase III MAP US study, we found that we could curtail the study and complete it earlier while still maintaining power and treatment effect. If the RHB-104 MAP US Phase III study is successful and if the drug is approved after completion of the required regulatory path, this curtailment may allow the treatment to be available earlier than initially planned, potentially changing the treatment paradigm for patients suffering from Crohn's disease."
The curtailment in the number of subjects planned to be enrolled in the MAP US study reflects input from internal and external experts, including statisticians who concluded that curtailment to a target sample size of approximately 325 subjects would maintain statistical power of over 80% with a treatment effect of 15%. Final statistical analysis remains unchanged and will be carried out using a two-sided p-value of 0.049 significance level using the O'Brien-Fleming method, as described in the protocol.
RedHill estimates that the development program will be shortened by approximately one year and the Company will benefit from cost savings of approximately $14 million.
RHB-104 is a proprietary, orally-administered, potentially ground-breaking, 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).
Additional clinical studies are likely to be required to support a U.S. New Drug Application (NDA) for RHB-104. If the MAP US Phase III study results are positive, RedHill will meet with the FDA and key opinion leaders to present the data package and discuss the preferred development path.
Two pre-planned independent DSMB meetings were held to review data from the MAP US study in which unanimous recommendations to continue the study without any changes to the protocol, investigator's brochure, study conduct or informed consent form were given. At the first DSMB meeting, held in December 2016, safety data from the study was reviewed. At the second DSMB meeting, held in July 2017, safety and efficacy data from the first 222 subjects who had completed week 26 assessments of the study was reviewed.
In addition, an open-label extension Phase III study (the MAP US2 study) continues to evaluate the safety and efficacy of RHB-104 in subjects who remain with active Crohn's disease (CDAI greater than or equal to 150) after 26 weeks of blinded study therapy in the ongoing Phase III MAP US study. These subjects 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 50-70 subjects in up to 150 clinical sites in the U.S., Canada, Europe, Israel and New Zealand. Additional open-label studies with RHB-104 for Crohn's disease are being planned to provide further supportive clinical data for potential future marketing applications.
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 also continues to advance the development program for a commercial companion diagnostic for the detection of MAP bacteria in Crohn's disease patients, in collaboration with several U.S. universities and with Q2 Solutions. The Company held a pre-submission meeting with the FDA regarding the diagnostic test in 2015. The development of the commercial companion diagnostic is an extension of RedHill's RHB-104 Phase III development program.
There is currently no validated, FDA-approved, commercially-available method of detecting the presence or absence of MAP in patients suffering from Crohn's disease or other diseases. The development of a commercial companion diagnostic is expected to contribute to the understanding of the role of MAP infection in Crohn's disease and potentially other inflammatory diseases.
The clinical studies with RHB-104 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.
RedHill remains blinded to the data from the ongoing MAP US Phase III study and cannot ascertain the potential impact of the curtailed number of subjects on the study's final outcome.
RedHill will host a conference call and webcast call today, October 2, 2017, at 9:00 a.m. EDT, to review the RHB-104 development program and the changes to the MAP US Phase III study.
The conference call, including a slide presentation, will be broadcasted live and available for replay on the Company's website, http://ir.redhillbio.com/events.cfm, for 30 days. Please access the Company's website at least 15 minutes ahead of the conference call to register, download, and install any necessary audio software.
Participants who wish to ask questions during the event can do so by telephone. To participate in the conference call, please dial the following numbers 5-10 minutes prior to the start of the call: United States: +1-877-280-1254; International: +1-646-254-3367; and Israel: +972-3-763-0145. The access code for the call is 2719928.
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 ground-breaking 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 subjects who have completed week 26 assessments in the ongoing Phase III MAP US study and remain with active Crohn's disease (CDAI greater than or equal to 150) at week 26. RHB-104 is covered by several issued and pending patents. 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. A pivotal Phase III study with RHB-104 for NTM infections is planned to be initiated in the first quarter of 2018. RedHill also completed a Phase IIa, proof-of-concept clinical study, evaluating RHB-104 as an add-on therapy to interferon beta-1a in subjects 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.
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 three gastrointestinal products in the U.S. - Donnatal® , a prescription oral adjunctive drug used in the treatment of IBS and acute enterocolitis, EnteraGam® , a medical food intended for the dietary management, under medical supervision, of chronic diarrhea and loose stools, and Esomeprazole Strontium Delayed-Release Capsules 49.3 mg, a prescription proton pump inhibitor indicated for adults for the treatment of gastroesophageal reflux disease (GERD) and other gastrointestinal conditions. RedHill's clinical-stage pipeline includes: (i) TALICIA(TM) (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 a planned pivotal Phase III study for nontuberculous mycobacteria (NTM) infections; (iii) BEKINDA® (RHB-102) - a once-daily oral pill formulation of ondansetron with successful top-line results from 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). More information about the Company is available at: www.redhillbio.com.
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements may be preceded by the words "intends," "may," "will," "plans," "expects," "anticipates," "projects," "predicts," "estimates," "aims," "believes," "hopes," "potential" or similar words. Forward-looking statements are based on certain assumptions and are subject to various known and unknown risks and uncertainties, many of which are beyond the Company's control, and cannot be predicted or quantified and consequently, actual results may differ materially from those expressed or implied by such forward-looking statements. Such risks and uncertainties include, without limitation, risks and uncertainties associated with (i) changes in the outcomes of our clinical trials resulting from changes in the size of the trials, and the accuracy of projected cost savings from changes to clinical trials; (ii) the initiation, timing, progress and results of the Company's research, manufacturing, preclinical studies, clinical trials, and other therapeutic candidate development efforts and projected cost savings from any changes to these trials; (iii) the Company's ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; (iv) the extent and number of additional studies that the Company may be required to conduct and the Company's receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings, approvals and feedback; (v) the manufacturing, clinical development, commercialization, and market acceptance of the Company's therapeutic candidates; (vi) the Company's ability to successfully market Donnatal® and EnteraGam®, (vii) the Company's ability to establish and maintain corporate collaborations; (viii) the Company's ability to acquire products approved for marketing in the U.S. that achieve commercial success and build its own marketing and commercialization capabilities; (ix) the interpretation of the properties and characteristics of the Company's therapeutic candidates and of the results obtained with its therapeutic candidates in research, preclinical studies or clinical trials; (x) the implementation of the Company's business model, strategic plans for its business and therapeutic candidates; (xi) the scope of protection the Company is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; (xii) parties from whom the Company licenses its intellectual property defaulting in their obligations to the Company; and (xiii) estimates of the Company's expenses, future revenues capital requirements and the Company's needs for additional financing; (xiv) the Company's Expanded Access Program, which allows patients with life-threatening diseases potential access, subject to regulatory and other approvals, to RedHill's investigational new drugs that have not yet received regulatory marketing approval, if a patient suffers an adverse experience using such investigative drug, potentially adversely affecting the clinical development program of that investigational product or the Company generally; (xv) competitive companies and technologies within the Company's industry. More detailed information about the Company and the risk factors that may affect the realization of forward-looking statements is set forth in the Company's filings with the Securities and Exchange Commission (SEC), including the Company's Annual Report on Form 20-F filed with the SEC on February 23, 2017. All forward-looking statements included in this Press Release are made only as of the date of this Press Release. We assume no obligation to update any written or oral forward-looking statement unless required by law.
Senior VP Business Development & Licensing
IR contact (U.S.):
Senior Vice President
The Trout Group
1 Remicade® ((infliximab) package insert: https://www.accessdata.fda.gov/drugsatfda_docs/label/2013/103772s5359lbl.pdf
2 Entyvio® (vedolizumab) package insert: https://www.accessdata.fda.gov/drugsatfda_docs/label/2014/125476s000lbl.pdf
3 Stelara® (ustekinumab) package insert: https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/761044lbl.pdf
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: RedHill Biopharma Ltd. via Globenewswire
Om Nasdaq GlobeNewswire
One Liberty Plaza - 165 Broadway
NY 10006 New York
+1 212 401 8700http://www.nasdaqomx.com
NASDAQ (NASDAQ: NDAQ) is a leading provider of trading, exchange technology, information and public company services across six continents.
Følg saker fra Nasdaq GlobeNewswire
Registrer deg med din epostadresse under for å få de nyeste sakene fra Nasdaq GlobeNewswire på epost fortløpende. Du kan melde deg av når som helst.
Siste saker fra Nasdaq GlobeNewswire
SEMAFO: Armed Incident in Bobo Area17.8.2018 15:48 | Pressemelding
MONTREAL, Aug. 17, 2018 (GLOBE NEWSWIRE) -- SEMAFO Inc. (TSX:SMF) (OMX:SMF) regrets to report that a bus transporting employees from the town of Bobo-Dioulasso to the Mana Mine in Burkina Faso was shot at by armed bandits this morning. In the exchange of fire between the policemen and bandits, one SEMAFO national employee and one sub-contractor employee lost their lives. Operations at the Mana Mine, located 80 kilometers from the incident, are not affected. Early analysis suggests that this incident and the armed incident that occurred in the Est region six days ago are unrelated. However, the Corporation will be increasing its security measures at Mana and take appropriate steps to ensure the safety of the mine and of its employees. The Corporation would like to express its sincere sympathy to families of the victims. About SEMAFO SEMAFO is a Canadian-based mining company with gold production and exploration activities in West Africa. The Corporation operates the Mana Mine in Burkina
ThalesNano Energy Introduces H-Genie™: Revolutionary Compact, High Pressure Hydrogen Gas Generation Platform for Chemists17.8.2018 11:00 | Pressemelding
Improves safety, expands chemistry, saves lives. BUDAPEST, Hungary and BOSTON, Aug. 17, 2018 (GLOBE NEWSWIRE) -- ThalesNano Energy LLC today announced, at the 256th ACS conference (booth: 1615), the launch of the first in the series of revolutionary new gas generators, the H-Genie™. The H-Genie™ is a compact high pressure hydrogen generator designed to be used in any laboratory as a safer and simpler alternative to hydrogen cylinders. Capable of generating hydrogen at up to 1 NL/min and 100 bar (1450 psi), the system can help expand chemists’ limited hydrogenation chemistry capability by providing high purity hydrogen gas, on demand from water, to batch and flow reactors. Neal Langerman, Principal Scientist at Advanced Chemical Safety, comments: “The presence of cylinders of hydrogen raises the risk level of a lab. While the explosion or fire hazard can be mitigated with a gas cylinder cabinet, this takes precious floor space and does not eliminate the 1300 liters of flammable gas from
PayByPhone Supports Additional Languages16.8.2018 19:47 | Pressemelding
Drivers can now use the app in Spanish and Chinese VANCOUVER, British Columbia, Aug. 16, 2018 (GLOBE NEWSWIRE) -- Drivers who pay for their parking with the PayByPhone app can now use the app in Spanish, Traditional Chinese, and Simplified Chinese. Later this year, the leading mobile parking payment service will also be available in Punjabi. The support of multiple languages increases PayByPhone’s usability for its diverse consumer base, who are located across North America, Europe, and Australia. To gain access to the newly supported languages within PayByPhone, drivers can simply download the latest version of the app and set their iOS or Android device to their preferred language. “We’re excited to have expanded our language offerings beyond English and French,” said Barrie Arnold, Chief Commercial Officer, PayByPhone North America. “PayByPhone is available in many multicultural cities worldwide, and this update improves the app’s accessibility. PayByPhone has gained a global presen
Global Dairy Platform Announces Dairy Farmers of America CEO Rick Smith as New Board Chair16.8.2018 17:52 | Pressemelding
ROSEMONT, Ill., Aug. 16, 2018 (GLOBE NEWSWIRE) -- Global Dairy Platform (GDP), a pre-competitive collaboration of dairy sector organizations focused on encouraging the appropriate intake of nutrient-rich dairy foods and demonstrating the sector’s role in sustainable agriculture, today announced the appointment of Rick Smith, President and Chief Executive Officer of Dairy Farmers of America as Chair of the Board of Directors. “It is truly an honor to serve as Chair of GDP’s Board of Directors,” noted Mr. Smith. “We are facing global food and health challenges that need short and long-term solutions. The work of GDP creates an avenue for collaborative action that demonstrates dairy’s valuable contribution to global food systems, healthy diets and sustainable livelihoods.” Mr. Smith will serve on the board along with Fonterra Co-operative Group Chief Executive Officer Mr. Miles Hurrell; China Mengniu Dairy Company Executive Director and Chief Executive Officer Mr. Minfang (Jeffery) Lu; Ro
GridGain Systems Named to Inc. 500 List of America’s Fastest-Growing Private Companies for Second Consecutive Year16.8.2018 09:00 | Pressemelding
GridGain Ranks 17th among Software Companies, 13th in San Francisco Metro Area FOSTER CITY, Calif., Aug. 16, 2018 (GLOBE NEWSWIRE) -- GridGain Systems, provider of enterprise-grade in-memory computing solutions based on Apache® Ignite™, today announced it has been named to the 37th annual Inc. 500 list, a highly regarded ranking of the nation’s fastest-growing private companies. This is the second year in a row GridGain has been named to the list. GridGain’s rank of 158 on this year’s Inc. 500 list is up from number 187 last year. The company is ranked 17th among software companies, 13th in the San Francisco metro area, and 37th in the state of California. The Inc. 500 represents a unique look at the most successful companies within the American economy’s most dynamic segment – its independent small and midsized businesses. GridGain provides an in-memory computing platform that delivers speed, scale and high availability to data-intensive applications. Built on a memory-centric archite
Perceptyx Named One of America’s Fastest Growing Companies for the 4th Consecutive Year16.8.2018 05:37 | Pressemelding
INC 5000 award recognizes Perceptyx’s continued innovation and partnership with many of the world’s best companies SAN DIEGO, Aug. 15, 2018 (GLOBE NEWSWIRE) -- For the 4th consecutive year, Perceptyx has made the prestigious INC 5000 list of America’s fastest growing private companies. A recognized leader in people analytics, Perceptyx specializes in facilitating organizational change through the strategic use of employee surveys and management consulting. Longtime partners with many of the world's largest organizations, Perceptyx is uniquely capable of delivering insights across heavily distributed organizations with complex hierarchies in a way that’s as unique as an organization’s culture and brand. “We’re proud to be recognized among America’s fastest growing companies again,” says John Borland, Co-founder and CEO of Perceptyx. “We believe that our clients are the real heroes of their organizations. It has been our honor to help so many of the world’s best companies realize their g
I vårt presserom finner du alle våre siste saker, kontaktpersoner, bilder, dokumenter og annen relevant informasjon om oss.Besøk vårt presserom