As­traZeneca gets a green light on Imfinzi for small-cell lung can­cer — set­ting the stage for a bat­tle with Roche's Tecen­triq

The stan­dard-of-care for small-cell lung can­cer (SCLC) for decades was plat­inum-based chemother­a­py, but that par­a­digm shift­ed in 2018, with the ap­proval of Bris­tol My­ers Squibb’s check­point in­hibitor Op­di­vo in the sec­ond-line set­ting. Since then, a slate of im­munother­a­pies have se­cured SCLC ap­provals, and the lat­est is As­traZeneca’s flag­ship Imfinzi, which was ap­proved on Mon­day in com­bi­na­tion with stan­dard chemother­a­py as the first line of de­fense in ex­ten­sive-stage SCLC pa­tients.

The ap­proval was based on the re­sults of the late-stage, 805 pa­tient-CASPI­AN tri­al, which test­ed Imfinzi plus stan­dard-of-care chemother­a­py, or Imfinzi and chemother­a­py with the ad­di­tion of As­traZeneca’s in-house CT­LA-4 drug treme­li­mum­ab, ver­sus chemother­a­py alone.

The pri­ma­ry end­point was over­all sur­vival in each of the two ex­per­i­men­tal arms. The com­bi­na­tion of Imfinzi and chemother­a­py cut the risk of death by 27% (HR: 0.73; p=0.0047). Pa­tients on the Imfinzi arm lived a me­di­an 13 months longer, ver­sus 10.3 months in the chemother­a­py group.

The arm with treme­li­mum­ab failed to achieve the pri­ma­ry goal. The Imfinzi/treme­li­mum­ab com­bo, once tout­ed as the cor­ner­stone of As­traZeneca’s check­point de­vel­op­ment strat­e­gy, has al­ready seen a raft of fail­ures in the past — it did not im­prove over­all sur­vival in cer­tain pa­tients with head and neck can­cer, in ad­di­tion to the big flop in the keen­ly-watched MYS­TIC non-small cell lung can­cer (NSCLC) tri­al. Al­though the duo has, when com­bined with chemother­a­py, worked in de­lay­ing dis­ease pro­gres­sion in front­line cas­es of stage 4 NSCLC.

Roche’s Tecen­triq in com­bi­na­tion with chemother­a­py was ap­proved first-line treat­ment for ex­tend­ed stage SCLC last Sep­tem­ber, based on a 30% re­duc­tion in risk of death ob­served in the piv­otal IM­pow­er133 tri­al. Months ear­li­er in June, Mer­ck’s key­stone check­point in­hibitor Keytru­da had scored FDA ap­proval as a monother­a­py for the treat­ment of SCLC pa­tients who had pro­gressed on or af­ter plat­inum-based chemother­a­py and at least one pri­or line of ther­a­py.

On Mon­day, the FDA ap­proved the Imfinzi plus chemother­a­py com­bi­na­tion as a first-line treat­ment for ex­tend­ed-stage SCLC pa­tients, pit­ting against Tecen­triq it in the ex­act same pa­tient pop­u­la­tion.

Mean­while, Phar­ma­Mar’s lur­binecte­din re­ceived pri­or­i­ty re­view last month for the treat­ment of SCLC pa­tients who pro­gressed on plat­inum ther­a­py based on da­ta from a sin­gle-arm, mid-stage study show­ing an over­all re­sponse rate (ORR) of about 35%.

Lung can­cer is the lead­ing cause of can­cer death glob­al­ly, ac­cord­ing to the WHO. The two main types of lung can­cer are non-small cell and small cell, and NSCLC ac­counts for about 85% of all cas­es. About two-thirds of SCLC pa­tients are di­ag­nosed with ex­tend­ed stage-SCLC, in which the can­cer has spread wide­ly through the lung or to oth­er parts of the body.

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.

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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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.

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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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, 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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Eu­ro­pean Com­mis­sion de­lays pro­pos­al for ma­jor changes to phar­ma leg­is­la­tion

The European Commission has once again delayed the release of its proposal for an overhaul of the continent’s pharmaceutical legislation.

The release, previously anticipated on March 29, will occur “slightly later” than expected due to the “very busy College agendas of the last few weeks,” a Commission spokesperson told Endpoints News via email.

While the agency hasn’t provided an updated timeline, the spokesperson said the agenda is “always indicative and adoption dates of Commission proposals may change any time, especially when these proposals concern reforms of complex legislations of major importance.”

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.