RedHill Biopharma Announces Advancement to Second Stage of Phase IIa Study with YELIVA® for Cholangiocarcinoma
- The first stage of the Phase IIa study with YELIVA® for advanced cholangiocarcinoma (bile duct cancer), a highly lethal malignancy, achieved its pre-specified efficacy goal, and the study can therefore continue to its second stage, enrolling the full cohort of 39 evaluable patients who previous received other treatments
- Complete response (i.e. disappearance of all target lesions and all non-target lesions) reported in
U.S.patient with advanced gallbladder carcinoma treated with YELIVA® as part of RedHill’s Expanded Access Program, allowing compassionate use for eligible patients
The primary efficacy endpoint of the study is defined as either partial or complete response, or stable disease at four months treatment with YELIVA®. Enrollment of all subjects is expected to be completed by mid-2019.
The single-arm Phase IIa study is evaluating the activity of YELIVA® as a single agent in patients suffering from advanced, unresectable intrahepatic, perihilar and extrahepatic cholangiocarcinoma. All subjects enrolled in the study have received up to two lines of other systemic therapy for advanced disease. The study is being conducted at renowned clinical institutions in the
YELIVA® was granted FDA Orphan Drug designation for the treatment of cholangiocarcinoma, providing various development incentives to develop YELIVA® for this indication and, if approved, a seven-year marketing exclusivity period for the treatment of cholangiocarcinoma.
Complete Response Achieved in Patient treated under RedHill’s Expanded Access Program:
Additionally, a patient in the
Cholangiocarcinoma (bile duct cancer) is a highly lethal malignancy for which there is an urgent need for more effective treatments. Approximately 8,000 people are diagnosed with intrahepatic and extrahepatic bile duct cancers annually in the
About YELIVA® (opaganib, ABC294640):
YELIVA® (ABC294640), a new chemical entity, is a Phase II-stage, proprietary, first-in-class, orally-administered, sphingosine kinase-2 (SK2) selective inhibitor with anticancer and anti-inflammatory activities, targeting oncology, inflammatory and gastrointestinal indications. By inhibiting SK2, YELIVA® blocks the synthesis of sphingosine 1-phosphate (S1P), a lipid-signaling molecule that promotes cancer growth and pathological inflammation. SK2 is an innovative molecular target for anticancer therapy because of its critical role in catalyzing the formation of S1P, which is known to regulate cell proliferation and activation of inflammatory pathways. YELIVA® was originally developed by
The Phase IIa study in cholangiocarcinoma was initiated following an extensive pre-clinical program, and a Phase I clinical study with YELIVA® in patients with advanced solid tumors which successfully met its primary and secondary endpoints, demonstrating that the drug is well tolerated and can be safely administered to cancer patients at doses that provide circulating drug levels that are predicted to have therapeutic activity. Of the three patients with cholangiocarcinoma treated in the Phase I study, all of whom had prior therapy, one subject achieved a sustained partial response (overall survival (OS) = 20.3 months) and the other two subjects had prolonged stable disease (OS = 17.6 and 16.3 months).
The ongoing studies with YELIVA® (ABC294640) for cholangiocarcinoma, multiple myeloma and advanced hepatocellular carcinoma (HCC) are registered on www.ClinicalTrials.gov, a web-based service by the
Expanded Access Program (EAP)
RedHill adopted an Expanded Access Program (EAP), allowing patients with life-threatening diseases potential access to RedHill’s investigational new drugs that have not yet received regulatory marketing approval. Expanded access (sometimes referred to as “compassionate use”) is possible outside RedHill’s clinical trials, under certain eligibility criteria, when a certain investigational new drug is needed to treat a life-threatening condition and there is some clinical evidence suggesting that the drug might be effective in that condition. Following the adoption of the program, RedHill continues to receive patient requests to obtain access to investigational drugs. Subject to evaluation of eligibility and all the necessary regulatory and other approvals, RedHill is likely to provide certain patients with an investigational new drug under the EAP. Further information about RedHill’s EAP can be found on the Company’s website at: http://www.redhillbio.com/expandedaccess.
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) the initiation, timing, progress and results of the Company’s research, manufacturing, preclinical studies, clinical trials, and other therapeutic candidate development efforts; (ii) the Company’s ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; (iii) 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; (iv) the manufacturing, clinical development, commercialization, and market acceptance of the Company’s therapeutic candidates; (v) the Company’s ability to successfully promote Donnatal® and Esomeprazole Strontium Delayed-Release Capsules 49.3 mgand commercialize EnteraGam®; (vi) the Company’s ability to establish and maintain corporate collaborations; (vii) the Company's ability to acquire products approved for marketing in the
1 YELIVA® (ABC294640) is an investigational new drug, not available for commercial distribution.
3 Gores GJ. Cholangiocarcinoma: current concepts and insights. Hepatology (
4 Banales JM et al. Expert consensus document: Cholangiocarcinoma: current knowledge and future perspectives consensus statement from the
5 Valle J, Wasan H, Palmer DH, et al. Cisplatin plus gemcitabine versus gemcitabine for biliary tract cancer. New Eng J Med 2010;362:1273-81.
Adi Frish Senior VP Business Development & Licensing RedHill Biopharma+972-54-6543-112 firstname.lastname@example.org IR contact ( U.S.): Timothy McCarthy, CFA, MBA Managing Director, Relationship Manager LifeSci Advisors, LLC+1-212-915-2564 email@example.com
Source: RedHill Biopharma Ltd.