On September 6, 2018 RedHill Biopharma Ltd. (NASDAQ: RDHL) (Tel-Aviv Stock Exchange: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company primarily focused on proprietary drugs for gastrointestinal diseases, reported that the ongoing single-arm Phase IIa study with orally-administered YELIVA (opaganib, ABC294640)1 for the treatment of advanced cholangiocarcinoma (bile duct cancer) has achieved its pre-specified efficacy goal for the first stage of the two-stage study design, and as a result, the study will continue to its second stage, enrolling the full cohort of 39 evaluable patients (Press release, RedHill Biopharma, SEP 6, 2018, View Source;LNGID=1&TMID=178&FID=1384&PID=0&IID=9184 [SID1234529317])
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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 U.S.
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 U.S. with advanced gallbladder carcinoma, a condition closely related to cholangiocarcinoma, who had progressed following standard-of-care chemotherapy, received treatment with YELIVA as part of RedHill’s Expanded Access Program, which allows compassionate use for eligible patients, and achieved a confirmed complete response, as measured by RECIST criteria (i.e. disappearance of all target lesions and all non-target lesions).
About cholangiocarcinoma:
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 U.S.2, with recent studies showing an increased incidence of cholangiocarcinoma, mainly attributed to recent advancements in the diagnosis of this disease3. Surgery with complete resection remains the only curative therapy for cholangiocarcinoma; however, only a minority of patients are classified as having a resectable tumor at the time of diagnosis4. Additional treatment options include radiation therapy and chemotherapy. Still, the efficacy of these treatments in cholangiocarcinoma patients is also limited and the prognosis for relapse patients who have failed initial chemotherapy is very poor with an overall median survival of approximately one year5. The 5-year relative survival rates of intrahepatic and extrahepatic cholangiocarcinoma patients range between 2% to 30%, depending on the tumor type and stage at diagnosis6.
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 U.S.-based Apogee Biotechnology Corp. and completed multiple successful pre-clinical studies in oncology, inflammation, GI and radioprotection models, as well as the ABC-101 Phase I clinical study in cancer patients with advanced solid tumors. YELIVA received Orphan Drug designation from the U.S. FDA for the treatment of cholangiocarcinoma. The development of YELIVA was funded to date primarily by grants and contracts from U.S. federal and state government agencies awarded to Apogee Biotechnology Corp., including the U.S. National Cancer Institute.
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 U.S. National Institute of Health, which provides public access to information on publicly and privately supported clinical studies.