As­traZeneca’s big bet on a com­bi­na­tion of its check­point in­hibitor Imfinzi and in-house CT­LA-4 drug has proved ex­ceed­ing­ly cost­ly. The British drug­mak­er con­ced­ed on Fri­day that the com­bi­na­tion had failed to im­prove over­all sur­vival in cer­tain pa­tients with head and neck can­cer, in ad­di­tion to the big flop in the keen­ly-watched MYS­TIC lung can­cer tri­al re­port­ed last month.

The com­bi­na­tion — once tout­ed as the cor­ner­stone of As­traZeneca’s $AZN check­point de­vel­op­ment strat­e­gy — did not meet the main goal of help­ing pa­tients with re­cur­rent or metasta­t­ic head and neck squa­mous cell car­ci­no­ma (HN­SCC) whose dis­ease had spread de­spite plat­inum-based chemother­a­py live longer, when com­pared to stan­dard chemother­a­py.

The late-stage EA­GLE tri­al was test­ing Imfinzi monother­a­py, as well as the com­bi­na­tion of Imfinzi and treme­li­mum­ab, ver­sus stan­dard-of-care chemother­a­py. Nei­ther the monother­a­py nor the com­bi­na­tion met the main goals of im­prov­ing over­all sur­vival, the com­pa­ny said, with­out of­fer­ing fur­ther de­tail on the re­sults, or pro­vid­ing any da­ta on sec­ondary end­points such as pro­gres­sion-free sur­vival.

But the com­pa­ny was in­sis­tent it may still find a way for­ward for the com­bi­na­tion.

Sean Bo­hen

“While these re­sults are dis­ap­point­ing, we re­main com­mit­ted to eval­u­at­ing the po­ten­tial of Imfinzi and oth­er in­no­v­a­tive med­i­cines for pa­tients with head and neck can­cer. We look for­ward to see­ing the re­sults of the Phase III KESTREL tri­al of Imfinzi and treme­li­mum­ab in pa­tients who have not re­ceived pri­or chemother­a­py for re­cur­rent or metasta­t­ic head and neck squa­mous cell car­ci­no­ma in the first half of 2019,” chief med­ical of­fi­cer Sean Bo­hen said in a state­ment.

While As­traZeneca’s pipeline has of­ten come up short, there have been a string of ma­jor suc­cess­es in on­col­o­gy for the com­pa­ny that have in­spired a re­turn to sales growth af­ter years of shrink­ing rev­enue. This Imfinzi/treme­li­mum­ab com­bi­na­tion, how­ev­er, was con­sid­ered crit­i­cal to putting the brakes on the blitz­ing gains Mer­ck $MRK and Bris­tol-My­ers Squibb $BMY have made af­ter es­tab­lish­ing their lead in the lu­cra­tive front­line can­cer field. The com­pa­ny has al­so re­cent­ly re­port­ed a string of set­backs for its res­pi­ra­to­ry drugs, cap­ping a year of hits-and-miss­es for CEO Pas­cal So­ri­ot, who as ru­mor has it, may be on his way out.

Im­age: Pas­cal So­ri­ot. AP IM­AGES

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.