As­traZeneca's prized Imfinzi/treme­li­mum­ab com­bo fails again — this time in head and neck can­cer

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

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FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

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.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to clinicaltrials.gov, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

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.

Stuart Peltz, former PTC Therapeutics CEO

Stu­art Peltz re­signs as PTC Ther­a­peu­tics CEO af­ter 25 years

Stuart Peltz, the longtime CEO of PTC Therapeutics who’s led the rare disease drug developer since its founding 25 years ago, is stepping down.

Succeeding him in the top job is Matthew Klein, who joined PTC in 2019 and was promoted to chief operating officer in 2022. In a call with analysts, he said the CEO transition has been planned for “quite some time” — in fact, as part of it, he gave the company’s presentation at the JP Morgan healthcare conference earlier this year.

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