Michael Morrissey, Exelixis CEO

Ex­elix­is drug fails PhI­II lung can­cer tri­al with Cabome­tyx and Tecen­triq com­bo

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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

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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