Policy & Regulation
Basilea Touts Positive Interim Results from Registrational Phase 2 Study with Oncology Drug Candidate Derazantinib in Intrahepatic Cholangiocarcinoma
10 January 2019 - - US-based precision medicine company ArQule, Inc.'s (NASDAQ: ARQL) partner, Switzerland-based Basilea Pharmaceutica Ltd. (SIX: BSLN), has received results from the interim analysis of the registrational Phase 2 study with the orally administered pan-fibroblast growth factor receptor kinase inhibitor derazantinib (BAL087), the company said.

The analysis showed promising activity in patients with FGFR2 gene fusion-expressing intrahepatic cholangiocarcinoma and also confirmed the safety profile and tolerability of the drug candidate observed in previous clinical studies.

This interim analysis in the ongoing registrational Phase 2 study was conducted after 42 patients had been enrolled in the study, with a subset of 29 evaluable patients who had at least one post-baseline imaging assessment.

The objective response rate in the 29 evaluable patients was 21%. The disease control rate, reflecting the proportion of patients with a partial response or with stable disease, was 83%. The safety data obtained from all 42 patients enrolled to date was consistent with the results from previous clinical studies with derazantinib.

ArQule is a biopharmaceutical company engaged in the research and development of targeted therapeutics to treat cancers and rare diseases.

ArQule's mission is to discover, develop and commercialize novel small molecule drugs in areas of high unmet need that will dramatically extend and improve the lives of our patients.

Our clinical-stage pipeline consists of five drug candidates, all of which are in targeted, biomarker-defined patient populations, making ArQule a leader among companies our size in precision medicine.

ArQule's pipeline includes: ARQ 531, an orally bioavailable, potent and reversible inhibitor of both wild type and C481S-mutant BTK, in Phase 1 for patients with B-cell malignancies refractory to other therapeutic options; miransertib (ARQ 092), a selective inhibitor of the AKT serine/threonine kinase, in a Phase 1/2 company-sponsored study for Overgrowth Diseases, in a Phase 1 study for ultra-rare Proteus syndrome conducted by the National Institutes of Health, and in Phase 1b in combination with the hormonal therapy, anastrozole, in patients with advanced endometrial cancer; ARQ 751, a next generation AKT inhibitor, in Phase 1 for patients with AKT1 and PI3K mutations; derazantinib, a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor family, in a registrational trial for iCCA; and ARQ 761, a β-lapachone analog being evaluated as a promoter of NQO1-mediated programmed cancer cell necrosis, in Phase 1/2 in multiple oncology indications in partnership with the University of Texas Southwestern Medical Center.

ArQule's current discovery efforts are focused on the identification and development of novel kinase inhibitors, leveraging the company's proprietary library of compounds.

Derazantinib (BAL087, formerly ARQ 087) is an investigational orally administered small molecule inhibitor of the FGFR family of kinases with strong activity against FGFR 1, 2, and 3.

Therefore, it is called a pan-FGFR kinase inhibitor. FGFR kinases are key drivers of cell proliferation, differentiation and migration.

FGFR alterations, e.g., gene fusions, overexpression or mutations, have been identified as potentially important therapeutic targets for various cancers, including iCCA, bladder, breast, gastric and lung cancers.

Current scientific literature suggests that FGFR alterations exist in a range of 5% to 30% in these cancers.

In iCCA, FGFR2 gene fusions have been reported in 13-22% of the cases4, 5 and FGFR gene mutations have been reported in up to 5% of the cases.

Basilea in-licensed derazantinib from ArQule Inc. in April 2018. The drug candidate has demonstrated favorable clinical data in previous clinical studies, including a biomarker-driven Phase 1/2 study in iCCA patients.

Derazantinib has US and EU orphan drug designation for this disease.

Intrahepatic cholangiocarcinoma is a cancer originating from the biliary system.

The age-adjusted incidence rate of iCCA in the United States has been increasing over the past decade and is currently estimated to be approximately 1.2 per 100,000.

Patients are often diagnosed with advanced or metastatic disease that cannot be surgically removed. Current first-line standard of care is the chemotherapy combination of gemcitabine and platinum-derived agents.

The prognosis for patients with advanced disease is poor, with a median survival of less than one year. There is no proven effective treatment for patients who progress on first-line chemotherapy, thus there is a high unmet medical need.

Basilea Pharmaceutica Ltd. is a commercial stage biopharmaceutical company focused on the development of products that address the medical challenge in the therapeutic areas of oncology and anti-infectives.

With two commercialised drugs, the company is committed to discovering, developing and commercialising innovative pharmaceutical products to meet the medical needs of patients with serious and life-threatening conditions.

Basilea Pharmaceutica Ltd. is headquartered in Basel, Switzerland and listed on the SIX Swiss Exchange (SIX: BSLN).
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