Kite Phar­ma hits its Q1 dead­line on fil­ing a BLA for its pi­o­neer­ing CAR-T, posts im­proved da­ta

Kite CEO Arie Bellde­grun

With the clock tick­ing down the fi­nal hours of Q1, Kite Phar­ma­ceu­ti­cals $KITE has kept its pledge to file an ap­pli­ca­tion for its pi­o­neer­ing CAR-T ther­a­py in the first quar­ter. The move puts Kite one step be­hind No­var­tis $NVS, which two days ago an­nounced that it had the FDA’s com­mit­ment for a speedy re­view of its own CAR-T.

The ques­tion now is whether the FDA will give the Kite’s ap­pli­ca­tion a pri­or­i­ty re­view as well, per­haps lin­ing up back-to-back ad­vi­so­ry com­mit­tee meet­ings to ex­am­ine the pros and cons of this new wave of can­cer ther­a­py.

Kite filed the BLA for axi­cab­ta­gene ciloleu­cel (KTE-C19, des­ig­nat­ed as a break­through ther­a­py by the FDA) as a treat­ment for pa­tients with re­lapsed or re­frac­to­ry ag­gres­sive non-Hodgkin lym­phoma who are in­el­i­gi­ble for au­tol­o­gous stem cell trans­plant.

Kite was held up a few months by the need to com­plete 6-month da­ta on axi­cab­ta­gene ciloleu­cel, a goal it ac­com­plished a month ago.

At six months, the ORR in dif­fuse large B-cell lym­phoma (DL­B­CL) hit 36%, down on­ly three points from month three. The CR rate was 31%, down two points. Big­ger drops were al­so record­ed for pri­ma­ry me­di­asti­nal B-cell lym­phoma (PM­B­CL) and trans­formed fol­lic­u­lar lym­phoma (TFL) en­rolled in Co­hort 2, but the six-month num­bers post­ed were all clear­ly sta­tis­ti­cal­ly sig­nif­i­cant, which helped qui­et some dogged crit­i­cism of the drug’s abil­i­ty to pro­vide a durable re­sponse.

On Fri­day, Kite al­so up­dat­ed its da­ta at AACR in an oral ple­nary pre­sen­ta­tion. And the num­bers had im­proved with more pa­tients to re­port on:

The ORR in the mITT analy­sis set of 101 pts was 82% (CR 54%, PR 28%), and was con­sis­tent across key co­vari­ates in­clud­ing dis­ease sub­type, re­frac­to­ry sta­tus, stage, and IPI score. At a me­di­an fol­low up of 8.7 m, 44% of pts were in re­sponse and 39% were in CR.

That’s up from 41% and 36% re­port­ed in Feb­ru­ary.

Kite — led by CEO Arie Bellde­grun — and No­var­tis are rac­ing to be right at the fore­front of in­tro­duc­ing per­son­al­ized cell ther­a­pies that reengi­neer pa­tients’ im­mune cells to tar­get can­cer. And it’s of­fered some sig­nif­i­cant re­spons­es for a num­ber of pa­tients with ad­vanced cas­es of blood can­cer.

Now Kite and No­var­tis will try to out­do each oth­er on man­u­fac­tur­ing and de­liv­ery, with the phar­ma gi­ant and the biotech need­ing to show that they can pro­vide quick and re­li­able treat­ment.

That may be a par­tic­u­lar­ly dif­fi­cult ques­tion for No­var­tis. As we re­port­ed Thurs­day af­ter­noon, the phar­ma gi­ant’s head of cell man­u­fac­tur­ing — Karen Walk­er, re­spon­si­ble for CAR-T — is leav­ing the com­pa­ny to­day af­ter a lengthy stint. Her de­par­ture fol­lows the un­ex­pect­ed move at No­var­tis to dis­solve its cell ther­a­py unit last sum­mer, lay­ing off staffers and ab­sorb­ing the group in its on­col­o­gy R&D di­vi­sion.

Walk­er and two oth­er ex­ecs were put in charge of No­var­tis’ ef­forts on CAR-T last fall, af­ter the com­pa­ny chopped up the sep­a­rate cell and gene ther­a­py group that had been cre­at­ed specif­i­cal­ly to dri­ve these new ther­a­pies to com­mer­cial­iza­tion. That move fol­lowed the de­par­ture of Us­man “Oz” Azam, who left No­var­tis in the wake of the re­or­ga­ni­za­tion.

Left on the side­lines to­day: Juno Ther­a­peu­tics. Once a leader in the CAR-T area, Juno was forced to shelve its lead ther­a­py af­ter it killed a string of pa­tients. Two of those pa­tients died af­ter a brief R&D halt or­dered by the FDA. But Juno nev­er was able to firm­ly es­tab­lish why the CAR-T caused lethal cas­es of brain swelling. Now the biotech has moved on to a fol­low-up pro­gram, putting the com­pa­ny well be­hind the fron­trun­ners.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; 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.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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GSK says its drug for chron­ic hep B could ‘lead to a func­tion­al cure’ — but will it be alone or in com­bi­na­tion?

GSK, newly branded and soon-to-be demerged, shared interim results from its Phase II trial on its chronic hepatitis B treatment, one that it says has the “potential to lead to a functional cure.”

At a presentation at the EASL International Liver Congress, GSK shared that in around 450 patients who received its hep B drug bepirovirsen for 24 weeks, just under 30% had hepatitis B surface antigen and viral DNA levels that were too low to detect.

Sanofi, GSK tout 72% Omi­cron ef­fi­ca­cy in PhI­II tri­al of next-gen, bi­va­lent shot — with an eye to year-end roll­out

Sometimes, being late can give you an advantage.

That’s what Sanofi and GSK are trying to say as the Big Pharma partners report positive results from a late-stage trial of their next-gen bivalent Covid-19 vaccine, which was designed to protect against both the original strain of the SARS-CoV-2 virus and the Beta variant. Specifically, against Omicron, they note, the vaccine delivered 72% efficacy in all adults and 93.2% in those previously infected.

Matt Kapusta, uniQure CEO

In trou­bled Hunt­ing­ton’s space, uniQure’s gene ther­a­py shows ear­ly promise

In randomized clinical trial data from a small number of patients, Dutch biotech uniQure shared that its gene therapy for Huntington’s disease seems to reduce the amount of the mutant protein responsible for the disease over the course of a year.

In seven patients with early-stage Huntington’s — four who got the treatment and three who got a placebo — mutant huntingtin protein levels in the cerebrospinal fluid decreased by an average of just over 50% in patients who got the gene therapy compared to around a 17% drop in patients who got the placebo after a year.

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De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.