CTI BioPharma has been poleaxed by the news that its much-anticipated late-stage trial for Pixuvri in non-Hodgkin’s lymphoma (NHL) has ended in failure.
The PIX306 trial paired Pixuvri (pixantrone) – already sold in some European markets as a monotherapy for hard-to-treat NHL – with Roche’s MabThera (rituximab) and compared it to MabThera plus gemcitabine in aggressive B-cell NHL, but found that CTI’s drug was unable to improve progression-free survival (PFS), its primary endpoint.
Shares in CTI were suspended as the news broke but slumped after trading resumed as investors came to terms with both a threat to the drug’s EU approval, and little chance of a route towards US approval.
The Seattle-based company has said for some time that any development of the drug in the US would depend on the outcome of PIX306, and its approval in Europe was conditional, with CTI required to submit the results to the EMA by the end of this year in support of its marketing licence.
It’s also a knock for privately-held French drugmaker Servier in all markets outside the US under the terms of a €103m licensing deal signed in 2014 and a revision to that deal agreed last year that resulted in an additional €12m payment. The new terms put CTI in line for €75m in sales and regulatory milestone payments as well as royalties on net product sales.
“We are disappointed with the outcome of the PIX306 trial and will proceed to conduct a thorough review of clinical data to assess the next steps for the Pixuvri program,” said CTI chief executive Adam Craig in a statement.
Thankfully for CTI, it’s been making progress on main pipeline candidate pacritinib , an orally-active kinase inhibitor that targets JAK2, FLT3, IRAK1 and CSF1R and is in phase III testing for myelofibrosis – having been fast-tracked for this indication by the FDA.
If approved, it will compete with Incyte and Novartis’ Jakafi (ruxolitinib) in myelofibrosis, which is predicted to knock on the door of $1.5bn in sales this year on the back of buoyant growth.
Pacritinib has had its own share of problems, however, having been placed under a clinical hold for almost a year after a brain haemorrhage, cardiac failure and cardiac arrests were seen in CTI’s first phase III trial.
The hold was relaxed last year, and earlier this month the biotech said that interim data from a second study showed no evidence of cardiac or bleeding events and the study would continue as planned through to its planned read-out in late 2018 or early 2019.