No­var­tis wins a key OK for Kym­ri­ah, but con­tin­u­ing man­u­fac­tur­ing woes hob­ble roll­out — ri­val Gilead CAR-T breaks in­to Eu­rope

No­var­tis CAR-T man­u­fac­tur­ing woes are con­tin­u­ing to put a crimp in its abil­i­ty to pro­vide ther­a­py to all po­ten­tial pa­tients.

Vas Narasimhan

The phar­ma gi­ant re­port­ed this morn­ing that the EC has stamped its ap­proval on mar­ket­ing Kym­ri­ah in Eu­rope for B-cell acute lym­phoblas­tic leukemia as well as treat­ment-re­sis­tant adults suf­fer­ing from dif­fuse large B-cell lym­phoma. But No­var­tis can on­ly make it avail­able to chil­dren with ALL now — adults will have to wait.

No­var­tis’s top ex­ecs have worked hard to smooth things over with an­a­lysts, but it’s been be­dev­iled by man­u­fac­tur­ing is­sues through­out the de­vel­op­ment stage and con­tin­ues to be af­fect­ed as it goes toe-to-toe with Gilead’s Kite, ap­proved for large B-cell lym­phoma. And Gilead is still com­ing on strong, not­ing this morn­ing that they re­ceived a Eu­ro­pean OK to mar­ket Yescar­ta for dif­fuse and pri­ma­ry me­di­asti­nal large B-cell lym­phoma.

A month ago No­var­tis CEO Vas Narasimhan not­ed dur­ing the Q2 call that:

I think it’s ear­ly days and we’ve al­ways said this is go­ing to be a five-year jour­ney with Kym­ri­ah to re­al­ly get us to be the glob­al­ly suc­cess­ful brand we want it to be.

On man­u­fac­tur­ing, we have seen some vari­abil­i­ty in our prod­uct spec­i­fi­ca­tions. This is some­thing we’re look­ing at now in DL­B­CL to make sure that we can con­tin­ue to ramp-up the de­mand. We feel con­fi­dent in the over­all longer-term out­look for Kym­ri­ah.

Liz Bar­rett

Liz Bar­rett, who runs No­var­tis on­col­o­gy, had this to say:

I think the most im­por­tant thing to let you know is that the vari­abil­i­ty that we’ve seen in the com­mer­cial spec­i­fi­ca­tions, which isn’t un­usu­al with a new ther­a­py as you launch in­to a new tar­get pa­tient pop­u­la­tion, is that we have still been able to de­liv­er fi­nal prod­uct to the ma­jor­i­ty of pa­tients.

As we’ve re­port­ed, though, the cell man­u­fac­tur­ing chief’s of­fice has been out­fit­ted with a re­volv­ing door at No­var­tis in re­cent years. Karen Walk­er left that post in ear­ly 2017, fol­lowed last spring by Spencer Fisk as he jumped ship to join the No­var­tis ex­ecs at Ru­bius. Those de­par­tures came on top of a ma­jor re­vamp for the cell and gene ther­a­py unit in 2016, which raised ques­tions at the time about No­var­tis’ abil­i­ty to keep up with Kite — though the phar­ma gi­ant wound up with the pi­o­neer­ing first OK. And No­var­tis’ is­sues with man­u­fac­tur­ing cell ther­a­pies ex­tend back to the sna­fus it ex­pe­ri­enced with late de­liv­ery to a small but sig­nif­i­cant group of pa­tients en­rolled in clin­i­cal tri­als.

At one stage in the de­vel­op­ment process, No­var­tis had a 6% fail­ure rate on the man­u­fac­tur­ing process in a key clin­i­cal tri­al, which they lat­er said was large­ly re­solved and brought down to 3%.

This morn­ing No­var­tis is fol­low­ing up with plans to in­vest €78.8 mil­lion in­to their cell and gene ther­a­py man­u­fac­tur­ing op­er­a­tions in Switzer­land in their lat­est stab at fix­ing the prob­lems.

But af­ter record­ing on­ly $16 mil­lion in sales for Q2, the big ques­tion loom­ing for No­var­tis is why pa­tients would want to take a risk with their ther­a­py if an­oth­er provider comes in­to the mar­ket which can do bet­ter on a com­plex task: ex­tract­ing T cells from pa­tients, mod­i­fy­ing them and then in­fus­ing pa­tients with a ther­a­peu­tic punch?

You can be sure that the top crew at Gilead and Kite — work­ing with a be­spoke man­u­fac­tur­ing site — are pay­ing close at­ten­tion.

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Uğur Şahin, BioNTech CEO (Kay Nietfeld/picture-alliance/dpa/AP Images)

De­spite falling Covid-19 sales, BioN­Tech main­tains '22 sales guid­ance

While Pfizer raked in almost $28 billion last quarter, its Covid-19 vaccine partner BioNTech reported a rise in total dose orders but a drop in sales.

The German biotech reported over $3.2 billion in revenue in Q2 on Monday, down from more than $6.7 billion in Q1, in part due to falling Covid sales. While management said last quarter that they anticipated a Covid sales drop — CEO Uğur Şahin said at the time that “the pandemic situation is still very much uncertain” — Q2 sales still missed consensus by 14%.

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Anna Protopapas, Mersana CEO

In $1.36B biobuck deal with GSK, Mer­sana touts 'biggest pre­clin­i­cal ADC deal ever'

Days after Enhertu reeled in another FDA nod, with the first-ever green light for HER2-low breast cancer, another antibody drug conjugate biotech claims it has secured the largest preclinical ADC pact to date for a single asset.

AstraZeneca and Daiichi Sankyo made waves with their nearly $7 billion collaboration back in spring 2019, but at that point, Enhertu was already nearing the FDA’s doors with clinical data. The latest ADC tie-up to enter the biopharma fray centers around a preclinical asset, Mersana Therapeutics’ XMT-2056.

Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

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Pascal Soriot, AstraZeneca CEO (David Zorrakino/Europa Press via AP Images)

As­traZeneca and Dai­ichi Sankyo sprint to mar­ket af­ter FDA clears En­her­tu in just two weeks

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, just two weeks after submitting a supplemental BLA. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.

David Reese, Amgen R&D chief

UP­DAT­ED: In a fresh dis­ap­point­ment, Am­gen spot­lights a ma­jor safe­ty is­sue with KRAS com­bo

Amgen had hoped that its latest study matching its landmark KRAS G12C drug Lumakras with checkpoint inhibitors would open up its treatment horizons and expand its commercial potential. Instead, the combo spurred safety issues that blunted efficacy and forced the pharma giant to alter course on its treatment strategy, once again disappointing analysts who have been tracking the drug’s faltering sales and limited therapeutic reach.

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Sen­ate Dems cling to a sim­ple ma­jor­i­ty to pass some of the biggest drug pric­ing re­forms ever

The Pharmaceutical Research and Manufacturers of America — and their fleet of drug industry lobbyists on Capitol Hill — are known for never losing.

Whenever a big drug pricing bill comes up, an army of the industry group’s lobbyists descend onto the Hill and either smash it outright or dismantle it piece by piece.

But for perhaps the largest drug pricing reforms ever enacted, after more than a decade of Congress trying and failing to allow Medicare to negotiate prescription drug prices, those same lobbyists and their biopharma clients were dealt a stunning blow on Sunday afternoon.

FDA commissioner Rob Califf (Tom Williams/CQ Roll Call via AP Images)

With drug pric­ing al­most done, Con­gress looks to wrap up FDA user fee leg­is­la­tion

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

GSK and IQVIA launch plat­form of US vac­ci­na­tion da­ta, show­ing drop in adult rates

Throughout the Covid-19 pandemic, the issue of vaccine uptake has been a point of contention, but a new platform from GSK and IQVIA is hoping to shed more light on vaccine data, via new transparency and general awareness.

The two companies have launched Vaccine Track, a platform intended to be used by public health officials, medical professionals and others to strengthen data transparency and display vaccination trends. According to the companies, the platform is intended to aid in increasing vaccine rates and will provide data on trends to assist public health efforts.

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