UK's NICE is­n't con­vinced by Mer­ck­'s Keytru­da in head and neck can­cer

There may be 33 new cas­es of head and neck can­cer in the Unit­ed King­dom each day, but the re­gion’s cost-ef­fec­tive­ness watch­dog NICE is not con­vinced by the da­ta sup­port­ing Mer­ck’s flag­ship im­munother­a­py Keytru­da in the pa­tient pop­u­la­tion.

On Wednes­day the agency said it was not rec­om­mend­ing Keytru­da for pa­tients with un­treat­ed metasta­t­ic or un­re­sectable re­cur­rent head and neck squa­mous cell car­ci­no­ma (HN­SCC), cit­ing un­cer­tain­ty that the tri­al ev­i­dence sup­port­ing the drug does not re­flect the cur­rent treat­ment par­a­digm in Eng­land’s NHS.

The ap­proval for Keytru­da in HN­SCC pa­tients was based on da­ta from the KEYNOTE-048 study, which showed the check­point in­hibitor im­proved over­all sur­vival com­pared to ce­tux­imab (Er­bitux), plat­inum chemother­a­py (cis­platin or car­bo­platin) and 5-Flu­o­rouracil (5-FU). The haz­ard ra­tio was 0.78 (p=0.0171) for PD-L1 pos­i­tive pa­tients in the monother­a­py group and 0.77 (p=0.0067) for the en­tire com­bo co­hort.

In the com­para­tor arm, pa­tients were giv­en ce­tux­imab with plat­inum chemother­a­py and 5-FU re­gard­less of whether the can­cer start­ed in­side or out­side the mouth — this is not es­tab­lished clin­i­cal prac­tice in the NHS in Eng­land, NICE said.

(If the can­cer starts in­side the mouth, NHS Eng­land treats with ce­tux­imab, plat­inum chemother­a­py and 5-FU — if it starts out­side the mouth, the can­cer is ini­tial­ly treat­ed with plat­inum chemother­a­py and 5-FU).

NICE was look­ing for ev­i­dence of Keytru­da’s im­pact strat­i­fied by pa­tients whose can­cer be­gan out­side the mouth and said that in­for­ma­tion about the clin­i­cal and cost-ef­fec­tive­ness for the two dif­fer­ent pa­tient groups (can­cer start­ing in­side or out­side the mouth) was in­com­plete.

The drug se­cured EU ap­proval for head and neck can­cer last No­vem­ber. There are around 11,900 new head and neck can­cer cas­es in the UK every year, that’s 33 every day, ac­cord­ing to es­ti­mates com­piled by Can­cer Re­search UK be­tween 2014-2016.

Keytru­da is how­ev­er rec­om­mend­ed for a range of oth­er can­cers by NICE, on the ba­sis that Mer­ck agreed to a con­fi­den­tial dis­count on the block­buster’s list price of £2,630 per 100 mg vial.

Sep­a­rate­ly, NICE al­so did not en­dorse Astel­las Phar­ma’s can­cer drug, Xospa­ta, in pa­tients with re­lapsed or re­frac­to­ry FLT3 mu­ta­tion-pos­i­tive acute myeloid leukemia (AML).

Al­though the ev­i­dence sug­gests that Xospa­ta can in­crease life ex­pectan­cy by more than three months ver­sus ex­ist­ing treat­ments, there is un­cer­tain­ty around long-term sur­vival, NICE said, adding that its cost-ef­fec­tive­ness es­ti­mates are al­so above the range the NHS con­sid­ers ac­cept­able.

The list price for Xospa­ta is £14,188 for a 28-day pack — but a con­fi­den­tial dis­count is in place for the NHS. The drug scored EU ap­proval in this pa­tient pop­u­la­tion last Oc­to­ber.

In ad­di­tion, NICE did back As­traZeneca’s Lyn­parza in adults who have re­lapsed plat­inum-sen­si­tive ovar­i­an, fal­lop­i­an tube or peri­toneal can­cer with a BR­CA1 or BR­CA2 mu­ta­tion.

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.

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

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

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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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.

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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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.

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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UP­DAT­ED: In­ter­nal Trump-era emails re­veal a pletho­ra of celebs, com­pa­nies vy­ing for FDA’s at­ten­tion

The FDA on Thursday publicly released a trove of about 500 pages of internal and heavily redacted emails, showing how celebrities like Dr. Oz and Laura Ingraham vied for the FDA’s attention on Covid-related issues that ultimately proved to be a waste of time.

Many of the conversations, obtained via the Freedom of Information Act, involve major Trump-era health officials who, in retrospect, made crucial and often ill-advised decisions on increasing access to the over-hyped hydroxychloroquine.

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