Basilea Pharmaceutica International plans to develop and commercialize ArQule’s Phase III intrahepatic cholangiocarcinoma (iCCA) candidate derazantinib (ARQ087) worldwide except greater China, through an exclusive licensing agreement that ArQule said today could generate for it up to $336 million-plus.

Derazantinib is a pan-fibroblast growth factor receptor (FGFR) inhibitor now in a registrational trial (NCT03230318) in patients with FGFR2 fusion-positive inoperable or advanced iCCA, a form of biliary tract cancer, in the U.S. as well as Canada and Europe. The primary endpoint is anticancer activity measured by Objective Response Rate.

Under the companies’ agreement, ArQule said, Basilea plans to continue the estimated 100-patient trial as well as further development of derazantinib in iCCA and other tumor types with FGFR dysregulation.

Derazantinib is in two additional clinical studies that were active but not recruiting patients as of January 12: A Phase II trial in patients with iCCA with FGFR2 fusions and a Phase Ib trial in patients with adrenocortical tumors and other tumors with FGFR translocations, amplification, and mutations (both combined under NCT0175290).

The licensing agreement covers those territories as well as Japan and rest of the world—except for China, Hong Kong, Macau, and Taiwan, where Roivant Sciences subsidiary Sinovant Sciences holds rights to develop and exclusively commercialize the drug.

Basilea agreed to pay ArQule $10 million upfront and up to $326 million tied to achieving regulatory and commercial milestones. ArQule is also entitled to receive staggered single-digit to double-digit royalties on net sales upon commercialization.

Basilea also agreed to oversee all all costs and expenses of development, manufacture, and commercialization in its territory—though ArQule said it may have the opportunity “under certain circumstances” to promote derazantinib in the U.S. directly.

Both the FDA and European Medicines Agency have granted ArQule their orphan drug designations for derazantinib in iCCA.

“An Ideal Match”

“Derazantinib is an ideal match for our existing clinical oncology portfolio,” Basilea CEO Ronald Scott said in a statement. “It is a targeted therapy building on a solid biomarker approach in an area where patients currently have limited treatment options. This transaction underscores our continued commitment to expand our R&D portfolio with novel compounds focused on overcoming the clinical problem of resistance in oncology and infectious diseases.”

Added ArQule CEO Paolo Pucci: “Partnering with Basilea, a company with global drug development experience and expertise, will propel the advancement of derazantinib in ways we could not have achieved independently.”

Derazantinib joins Basilea’s portfolio of products focused on cancer as well as bacterial infections and fungal infections—specifically, increasing resistance and nonresponse to current treatment options. In cancer, Basilea is developing two clinical-phase candidates, BAL101553 and BAL3833.

BAL3833 is a Phase I oral panRAF/SRC kinase inhibitor designed to block BRAF and CRAF and also inhibits the SRC kinase family.

BAL101553 is a small-molecule tumor checkpoint controller being developed as a potential therapy for diverse cancers, including tumor types unresponsive to standard therapeutics. In releasing 2017 results on February 27, Basilea said it had advanced development of BAL101553 last year by:

  • Entering into a clinical study collaboration with the Adult Brain Tumor Consortium, funded by the NIH’s National Cancer Institute.
  • Launching a Phase I study in combination with radiotherapy in patients with newly diagnosed glioblastoma who have a reduced sensitivity to standard chemotherapy.
  • Completing Phase I dose-escalation and establishing clinical dose ranges in two Phase I/IIa studies in patients with advanced solid tumors with daily oral administration and weekly 48-hour intravenous infusion, respectively. Basilea plans to launch a Phase IIa expansion with weekly 48-hour IV infusion in patients with recurrent glioblastoma and ovarian cancer.

In addition, Basilea said, it is exploring BAL101553 as a once-daily oral treatment in patients with recurrent or progressive glioblastoma in a separate arm of the ongoing Phase I/IIa study.

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