In a stun­ner, FDA im­me­di­ate­ly drops its hold on Juno’s lead CAR-T

So much for bu­reau­cra­cy. In what may well be the short­est high-pro­file FDA clin­i­cal hold in the agency’s his­to­ry, Juno re­port­ed to­day that the FDA has lift­ed the halt on its lead CAR-T pro­gram, sat­is­fied that it found the cause for three pa­tient deaths.

Juno’s bat­tered shares $JUNO im­me­di­ate­ly rock­et­ed back up 28%.

Juno con­found­ed in­vestors last week when it re­port­ed the deaths and the sur­pris­ing hold, cit­ing the re­cent ad­di­tion of the chemother­a­py agent flu­dara­bine to its pre­con­di­tion­ing of pa­tients as a like­ly cul­prit. Some ex­perts, who had used the drug in oth­er stud­ies to help clear the path for a fol­lowup ther­a­py, pub­licly ques­tioned the ra­tio­nale. But the agency had no prob­lem pro­vid­ing a quick green light to re­sume the piv­otal study.

The Phase II ROCK­ET study will now con­tin­ue in adult pa­tients with re­lapsed or re­frac­to­ry B-cell acute lym­phoblas­tic leukemia (r/r ALL), us­ing on­ly cy­clophos­phamide in pre-con­di­tion­ing pa­tients for JCAR015.

Not im­me­di­ate­ly clear is whether Juno will be able to gain an ap­proval in 2017, as it had hoped be­fore the hold, putting it back in a race with ri­val Kite.

CAR-T ther­a­pies like this take cells from pa­tients and reengi­neer them with chimeric anti­gen re­cep­tors so they can be rein­ject­ed in­to pa­tients, mul­ti­ply and swarm can­cer cells. Ear­ly stud­ies have im­pressed in­ves­ti­ga­tors with their po­ten­tial for blood can­cers. But Juno roiled the field just days ago with news that a new kind of neu­ro­tox­i­c­i­ty had killed sev­er­al can­cer pa­tients in a study.

Some in­ves­ti­ga­tors, though, note that flu­darib­ine has been used in many oth­er stud­ies, with­out the kind of tox­i­c­i­ty Juno’s ther­a­py trig­gered. And be­cause it was used to help amp up the ef­fect of the drug, some won­der if Juno’s lead ther­a­py may not mea­sure up to Kite’s, which now ap­pears to be in the lead for the first, pi­o­neer­ing ap­proval.

Brad Lon­car, who man­ages an in­dex of the top 30 im­munother­a­py com­pa­nies on the mar­ket, not­ed that Juno CEO Hans Bish­op in­di­cat­ed that he would up­date in­vestors on the time­line for JCAR015 in Au­gust. Kite, mean­while, is push­ing full steam ahead on their piv­otal study. And No­var­tis in­di­cat­ed back in late May that it is on track to file an ap­proval for CTL019 in ear­ly 2017.

 

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.