The EMA re­jects XBiotech’s can­cer drug af­ter an em­bar­rass­ing PhI­II — but they’re not giv­ing up

You can file this sto­ry in the Not Sur­prised fold­er.

Austin-based XBiotech’s ap­pli­ca­tion to start sell­ing its lead can­cer ther­a­py has been spurned by Eu­ro­pean reg­u­la­tors.

That couldn’t have been com­plete­ly un­ex­pect­ed as their lead drug — Xilonix — had of­fered up some du­bi­ous da­ta. In­ves­ti­ga­tors re­port­ed that the Phase III was marred by a mix­up in treat­ing the place­bo and drug arms, a num­ber of dropouts and im­prop­er pa­tient eval­u­a­tions. In­ves­ti­ga­tors then used a sur­ro­gate end­point on con­serv­ing pa­tients’ health sta­tus as a mea­sure of Phase III suc­cess.

A month ago, a trend vote by the EMA’s Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use flagged the mem­bers’ opin­ion that the da­ta just wasn’t there for an ap­proval. The for­mal CHMP opin­ion didn’t change. And its opin­ion was bru­tal.

The CHMP had a num­ber of con­cerns. First, the com­mit­tee not­ed that the study did not show clear im­prove­ments in ei­ther lean body mass or qual­i­ty of life. Sec­ond­ly, there was an in­creased risk of in­fec­tion in pa­tients tak­ing the med­i­cine, which was not con­sid­ered ac­cept­able in vul­ner­a­ble pa­tients who will be re­ceiv­ing pal­lia­tive care. Last­ly, there were in­ad­e­quate con­trols of the man­u­fac­tur­ing process to en­sure the med­i­cine would have the same qual­i­ty as the prod­uct used in clin­i­cal tri­als.

There­fore, the CHMP was of the opin­ion that the ben­e­fits of this med­i­cine did not out­weigh its risks and rec­om­mend­ed that it be re­fused mar­ket­ing au­tho­ri­sa­tion.

XBiotech $XBIT, though, in­dig­nant­ly in­sists that the EMA is off base and wants a do-over. The biotech not­ed the re­jec­tion in an SEC fil­ing Thurs­day, say­ing:

The Com­pa­ny plans to prompt­ly no­ti­fy the EMA of its re­quest for a re-ex­am­i­na­tion and will have 60 days to sub­mit its grounds for ap­peal. The Com­pa­ny firm­ly be­lieves it has a strong po­si­tion for re-ex­am­i­na­tion.

XBiotech had burned through about $194 mil­lion by the end of Q1. Its shares are down a lit­tle more than 10% this morn­ing.

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.

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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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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Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.