No­var­tis is keep­ing JULI­ET in hid­ing, but the FDA takes a peek and hands over a ‘break­through’ ti­tle

Vas­ant Narasimhan

There’s been con­sid­er­able spec­u­la­tion of late about the da­ta No­var­tis has col­lect­ed from its JULI­ET study in­volv­ing its close­ly-watched CAR-T drug. The phar­ma gi­ant isn’t the best at com­mu­ni­cat­ing with the out­side world and won’t say what it has seen so far or when ex­act­ly it will be put on dis­play. But they have qui­et­ly giv­en the FDA a peek, and reg­u­la­tors seem im­pressed.

In fact, the FDA has now hand­ed over their break­through ther­a­py des­ig­na­tion for CTL019 as a new ther­a­py for dif­fuse large B-cell lym­phoma.

What’s the big deal?

BTDs are in­creas­ing­ly com­mon, but this one is a bit dif­fer­ent than your run-of-the-mill open door promise at the agency. While No­var­tis and Kite have been run­ning neck and neck to get the first pi­o­neer­ing ap­proval for a CAR-T, No­var­tis is tak­ing its first shot at chil­dren with acute lym­phoblas­tic leukemia while Kite shoots for DL­B­CL. Kite has put its DL­B­CL da­ta up for the world to see, at 3 and 6 months, and now CAR-T ob­servers want to see how they match up in the com­ing ri­val­ry.

The FDA’s BTD in­di­cates that No­var­tis has a good shot at it, but a tac­it en­dorse­ment like this is no guar­an­tee that CTL019 is any bet­ter than KTE-C19, al­so known as axi-cel. Af­ter all, the FDA al­ready hand­ed out break­through sta­tus to Kite’s drug for DL­B­CL, trans­formed fol­lic­u­lar lym­phoma and pri­ma­ry me­di­asti­nal b-cell lym­phoma.

We’ll have to wait for an un­spec­i­fied sci­en­tif­ic con­fer­ence to find out what No­var­tis has been keep­ing se­cret.

In the mean­time Juno has been re­or­ga­niz­ing its CAR-T R&D group, a sto­ry End­points News broke on Sun­day evening, with the hire of Sunil Agar­w­al as the new head of re­search. Juno had to shelve its lead pro­gram af­ter it killed 5 pa­tients in clin­i­cal tri­als. The biotech has fall­en more than a year be­hind the two lead­ers in the field.

Forbes‘ Matt Her­p­er (left) with Sean Park­er and Dr. Carl June (right) – screen­shot

“We are en­cour­aged by the FDA’s recog­ni­tion in the po­ten­tial of CTL019 for this in­di­ca­tion, which fol­lows our promis­ing stud­ies of this ther­a­py for ALL and the FDA fil­ing by No­var­tis in pe­di­atric and young adult ALL that re­ceived pri­or­i­ty re­view,” said Penn’s Carl June, di­rec­tor of the Cen­ter for Cel­lu­lar Im­munother­a­pies at the uni­ver­si­ty’s Perel­man School of Med­i­cine.

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.