Ex­elix­is’ tri­al of Cabome­tyx and Tecen­triq for pa­tients with metasta­t­ic non-small cell lung can­cer failed to meet its goal of ex­tend­ing over­all sur­vival com­pared to chemother­a­py.

The com­pa­ny shared the fail­ure in a brief press re­lease Thurs­day af­ter­noon. It said it will share oth­er, sec­ondary out­comes at the tri­al at a fu­ture med­ical meet­ing.

The 366 pa­tients in the tri­al re­ceived Ex­elix­is’ drug Cabome­tyx in com­bi­na­tion with Roche’s Tecen­triq or do­c­etax­el. All of the pa­tients had been treat­ed pre­vi­ous­ly with an­ti-PD-1 or PD-L1 drugs that failed to stop their can­cers.

Ex­elix­is has arrange­ments with Ipsen, Take­da Phar­ma­ceu­ti­cal and Roche to help de­vel­op Cabome­tyx, which is ap­proved in the US for the treat­ment of sev­er­al oth­er can­cers in­clud­ing ad­vanced re­nal cell car­ci­no­ma, he­pa­to­cel­lu­lar car­ci­no­ma and ad­vanced or metasta­t­ic dif­fer­en­ti­at­ed thy­roid can­cer.

Howard May­er

“The re­sults from the CON­TACT-01 clin­i­cal tri­al have shown the chal­lenge of treat­ing NSCLC pa­tients af­ter pri­or lines of treat­ment have failed,” Howard May­er, ex­ec­u­tive vice pres­i­dent and head of re­search and de­vel­op­ment at Ipsen, said in a state­ment. “While the find­ings of the study have not met the pri­ma­ry end­point in this set­ting, we re­main con­fi­dent in the clin­i­cal ef­fi­ca­cy of cabozan­ti­nib alone and in com­bi­na­tion with an­oth­er treat­ment in ex­ist­ing in­di­ca­tions in dif­fi­cult-to-treat tu­mor types.”

In 2016, Ipsen struck a deal with Ex­elix­is for ex­clu­sive de­vel­op­ment and com­mer­cial­iza­tion rights to Cabome­tyx out­side of the US and Japan. Take­da has the rights in Japan, which it snapped up in 2017.

The drug was first ap­proved for sec­ond-line treat­ment of kid­ney can­cer in 2016, two years af­ter a prostate can­cer fail­ure forced Ex­elix­is to lay off 70% of its staff. The com­pa­ny’s re­cent strat­e­gy has been push­ing com­bi­na­tion treat­ments, such as with Tecen­triq, and seek­ing new in­di­ca­tions for the drug.

In March of this year, the com­pa­ny said an­oth­er com­bi­na­tion tri­al of Cabome­tyx and Tecen­triq failed to beat over­all sur­vival out­comes from the drug Nex­avar as a first-line treat­ment for pa­tients with ad­vanced he­pa­to­cel­lu­lar car­ci­no­ma. It said at the time that it would not to seek an FDA ap­proval for that use.

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.