Cerdulatinib is an oral, dual Syk-JAK kinase inhibitor that Portola is developing to treat patients with hematologic cancers, specifically those who have relapsed or who have not responded to prior therapies.
The new data will be presented on Sunday, May 31, in a poster discussion session at the 2015 American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago.
The Company previously presented interim data from the Phase 1 part of the study at the American Society of Hematology (ASH) 2014 Annual Meeting in December 2014.
The results to be presented at ASCO demonstrated evidence of clinical activity in this study of patients with relapsed/refractory B-cell malignancies.
To date, partial responses have been observed, including in patients with chronic lymphocytic leukemia (CLL), follicular lymphoma (FL), and transformed FL. Tumor reductions were seen in multiple patients, including those whose disease progressed on (or who could not tolerate) other BCR pathway inhibitors.
Results also showed that cerdulatinib was well tolerated in these heavily pre-treated patients, with no dose-limiting toxicities identified. Grade 3/4 adverse events included fatigue. Dose-escalation is ongoing in the Phase 1 part of the study as the maximum tolerated dose has not yet been achieved.
"The clinical activity that we have seen with cerdulatinib in this hematologic cancer study is particularly notable given that these patients have advanced disease and have failed previous therapies," said Manish Patel, M.D., an investigator in the Phase 1/2a study and associate director of drug development at Sarah Cannon Research Institute and associate director of research at Florida Cancer Specialists and Research Institute in Sarasota, Fla.
"New treatment options are urgently needed for these patients, and we are looking forward to continuing to study cerdulatinib, a unique dual pathway anti-cancer agent."
"These preliminary results from the ongoing dose-escalation Phase 1 part of the study, including partial responses, provide additional evidence that cerdulatinib is active and well tolerated in patients with hematologic cancers who have failed multiple therapies," said John T. Curnutte, M.D., Ph.D., executive vice president, research and development for Portola.
"We recently opened enrollment in larger expansion cohorts in the Phase 2a part of the study, which will include patients with CLL/small lymphocytic leukemia (SLL) or FL whose cancer has progressed or relapsed following treatment with multiple agents.
"We hope to identify a genetically-defined patient population for whom the inhibition of both Syk and JAK by cerdulatinib may provide clinical benefit beyond current treatment options. If we are successful, we plan to work closely with the FDA to seek an accelerated development path for this agent."