Mer­ck’s check­point star Keytru­da grabs an­oth­er FDA OK, steer­ing past wreck­age of a Roche dis­as­ter

Mer­ck’s Keytru­da con­tin­ues to make a rapid ad­vance to new ap­provals for ex­pand­ed use. This time the FDA is ap­prov­ing their PD-1 drug for sec­ond-line as well as some first-line cas­es of blad­der can­cer — while Roche’s land­mark OK for the ri­val Tecen­triq (ate­zolizum­ab) in the same in­di­ca­tion has been threat­ened by a stun­ning Phase III fail­ure.

Reg­u­la­tors came up with an ac­cel­er­at­ed ap­proval for Keytru­da for pa­tients whose ad­vanced cas­es of urothe­lial can­cer have pro­gressed af­ter chemother­a­py as well as any front­line pa­tients who aren’t el­i­gi­ble for cis­platin-con­tain­ing chemother­a­py. KEYNOTE-045 pro­vid­ed the da­ta for the sec­ond-line OK, with an ob­jec­tive re­sponse rate of 21% for Keytru­da, close to twice that achieved by chemother­a­py. Those num­bers un­der­score the promise for this field, as well as the need for com­bi­na­tion ther­a­pies that can do much bet­ter. And Mer­ck has hun­dreds of those un­der­way now.

Those com­bos will be telling as the PD-1/PD-L1 check­point field con­tin­ues to get more and more crowd­ed. As­traZeneca al­ready has an ap­proval for Imfinzi (dur­val­um­ab) in blad­der can­cer, along­side Mer­ck KGaA/Pfiz­er’s Baven­cio (avelum­ab) and Bris­tol-My­ers Squibb’s Op­di­vo. And a whole tsuna­mi of new check­points from oth­er play­ers are com­ing along in the pipeline as well.

While Keytru­da goes from ad­vance to ad­vance — most re­cent­ly high­light­ed with a front­line OK for a com­bi­na­tion of Keytru­da and chemo for firstling use against lung can­cer — this field has been roiled by star­tling set­backs. Roche was stunned when its con­fir­ma­to­ry Phase III for Tecen­triq failed in blad­der can­cer, un­able to sig­nif­i­cant­ly im­prove the over­all sur­vival rate of pa­tients af­ter the FDA had of­fered an ear­ly ap­proval. That fail­ure may well force the FDA to yank the ap­proval, putting the block­buster con­tender un­der a cloud. Bris­tol-My­ers Squibb has al­so felt the heat af­ter stum­bling bad­ly in lung can­cer, giv­ing Mer­ck the op­por­tu­ni­ty to leapfrog its lead po­si­tion.

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

Re­gen­eron's Evkeeza shows promise in curb­ing high triglyc­erides, but will ge­net­ic dis­par­i­ties lim­it use?

When Regeneron scored an early approval for lipid lowering antibody Evkeeza back in February, the drugmaker cracked open a new pathway to lower abnormally high cholesterol levels. Now, Regeneron is chasing high triglycerides as well with some promising mid-stage data — but will genetic restrictions limit the drug’s use?

Regeneron’s Evkeeza (evinacumab) cut median triglyceride levels by more than 800 mg/dL (57%) in patients with a rare disorder causing abnormally high triglyceride levels compared with an overall increase of 50 mg/dL (1.8%) in participants on placebo, according to Phase II data presented Sunday at the virtual American College of Cardiology meeting.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

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I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

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As­traZeneca's Farx­i­ga missed big on Covid-19 study, but it's tak­ing SGLT2 safe­ty da­ta as a sil­ver lin­ing

AstraZeneca hasn’t seen many setbacks in recent months for SGLT2 inhibitor Farxiga, which broke ground in heart failure and kidney disease regardless of diabetes diagnosis. But the British drugmaker had to admit defeat in taking Farxiga into Covid-19, but follow-up results add a bit of a silver lining to that trial’s safety data.

Of hospitalized Covid-19 patients dosed with AstraZeneca’s Farxiga, 11.2% experienced an organ failure or died after 30 days of therapy compared with 13.8% of those given placebo, according to follow-up data from the DARE-19 study revealed Sunday at the virtual American College of Cardiology meeting.

Pfiz­er, Bris­tol My­er­s' Eliquis flops in post-heart surgery pa­tients, spurring an 'un­ex­plained sig­nal' in cer­tain deaths

Pfizer and Bristol Myers Squibb’s non-warfarin blood thinner Eliquis has raced out to become the most prescribed drug of its class on the market — even overtaking warfarin’s long-time lead. But in tricky-to-treat patients after a valve replacement, an investigator-sponsored study couldn’t turn up benefit and raised a troubling safety signal.

Eliquis failed to show benefit over standard of care in preventing serious clinical outcomes after a transaortic valve replacement (TAVR) and was linked to an “unexplained signal” in a subset of populations with a higher rate of non-CV deaths who did not need blood thinners apart from the surgery, according to data presented Saturday at the virtual American College of Cardiology meeting.

Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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In­cyte’s PD-(L)1 in­hibitor head­ed for an ODAC show­down next month

The FDA’s Oncologic Drugs Advisory Committee will spend a half day on June 24 reviewing Incyte’s PD-(L)1 inhibitor retifanlimab as a treatment for locally advanced or metastatic squamous carcinoma of the anal canal (SCAC) for those who have progressed on or who are intolerant of platinum-based chemotherapy.

The eighth PD-(L)1 entrant in January nabbed a priority review and an orphan designation from the FDA, which sets the agency’s final decision date as July 25.