FDA ush­ers in a new era in can­cer treat­ment with 'his­toric' CAR-T ap­proval for No­var­tis

The FDA has ap­proved the world’s first CAR-T ther­a­py, giv­ing a green light to No­var­tis for Kym­ri­ah (ti­s­agen­le­cleu­cel) in what reg­u­la­tors them­selves de­scribe as an his­toric event.

The ear­ly ap­proval — about a month ahead of the PDU­FA date — came through for cer­tain pe­di­atric and young adult pa­tients with a form of acute lym­phoblas­tic leukemia.

No­var­tis set the price for the one-time treat­ment at $475,000, right in line with low-end es­ti­mates and one that will put pres­sure on Gilead to rein in its own price for a ri­val ther­a­py ex­pect­ed to be ap­proved in the near fu­ture.

Scott Got­tlieb, FDA Com­mis­sion­er

None oth­er than FDA com­mis­sion­er Scott Got­tlieb did the hon­ors in the agency’s state­ment, not­ing:

We’re en­ter­ing a new fron­tier in med­ical in­no­va­tion with the abil­i­ty to re­pro­gram a pa­tient’s own cells to at­tack a dead­ly can­cer. New tech­nolo­gies such as gene and cell ther­a­pies hold out the po­ten­tial to trans­form med­i­cine and cre­ate an in­flec­tion point in our abil­i­ty to treat and even cure many in­tractable ill­ness­es. At the FDA, we’re com­mit­ted to help­ing ex­pe­dite the de­vel­op­ment and re­view of ground­break­ing treat­ments that have the po­ten­tial to be life-sav­ing.

A group of bio­phar­ma com­pa­nies have been rac­ing to this break­through point. Kite Phar­ma $KITE, new­ly bought out in a $12 bil­lion deal by Gilead $GILD, is com­ing in a close sec­ond with its own ap­pli­ca­tion for axi­cel.

No­var­tis’ in­ves­ti­ga­tors reg­is­tered a game-chang­ing 83% re­mis­sion rate in its piv­otal study.

CAR-T, though, is al­so as­so­ci­at­ed with se­vere and po­ten­tial­ly dead­ly side ef­fects, in­clud­ing lethal in­stances of cy­tokine re­lease syn­drome with some pa­tients dy­ing from brain swelling in sep­a­rate stud­ies from the No­var­tis drug.

Reg­u­la­tors not­ed that they will re­quire spe­cial train­ing for any­one in­volved in de­liv­er­ing this ther­a­py, while ex­pand­ing the ap­proval of Actem­ra (tocilizum­ab) to treat CAR T-cell-in­duced se­vere or life-threat­en­ing CRS in pa­tients 2 years of age or old­er. “In clin­i­cal tri­als in pa­tients treat­ed with CAR-T cells,” the FDA re­port­ed, “69% of pa­tients had com­plete res­o­lu­tion of CRS with­in two weeks fol­low­ing one or two dos­es of Actem­ra.”

This ther­a­py, which is made us­ing a pa­tient’s own im­mune cells, won’t be cheap.

By its own reck­on­ing, the UK’s tough watch dog on drug pric­ing has said that these drugs would be worth up to $649,000 a year, giv­en the young pa­tients it’s tar­get­ing first. An­a­lysts, mean­while, have pegged the price at any­where from $400,000 to $750,000.

In a call with an­a­lysts, No­var­tis ex­ecs said it would keep the price well be­low the high end, set­ting the cost at $475,000 for a drug that will be avail­able in 20 cen­ters with­in one month, and 35 soon af­ter.

In a state­ment the com­pa­ny said it “is col­lab­o­rat­ing with CMS to make an out­comes-based ap­proach avail­able to al­low for pay­ment on­ly when pe­di­atric and young adult ALL pa­tients re­spond to Kym­ri­ah by the end of the first month. Fu­ture po­ten­tial in­di­ca­tions would be re­viewed for the most rel­e­vant out­comes-based ap­proach.”

Joe Jimenez

Said Joseph Jimenez, CEO of No­var­tis:

Five years ago, we be­gan col­lab­o­rat­ing with the Uni­ver­si­ty of Penn­syl­va­nia and in­vest­ed in fur­ther de­vel­op­ing and bring­ing what we be­lieved would be a par­a­digm-chang­ing im­muno­cel­lu­lar ther­a­py to can­cer pa­tients in dire need. With the ap­proval of Kym­ri­ah, we are once again de­liv­er­ing on our com­mit­ment to change the course of can­cer care.

This ap­proval marks a ma­jor shift in on­col­o­gy, with a tru­ly rev­o­lu­tion­ary ap­proach to treat­ing can­cer. That hasn’t es­caped the no­tice of big play­ers and small, with a host of de­vel­op­ers look­ing to do much bet­ter, more safe­ly, with new drugs in the pipeline.

It’s a big day in the biotech world, but it’s just the of­fi­cial start date for a new era.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Bris­tol-My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

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Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

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Gilead bol­sters its case for block­buster hope­ful fil­go­tinib as FDA pon­ders its de­ci­sion

Before remdesivir soaked up the spotlight amid the coronavirus crisis, Gilead’s filgotinib was the star experimental drug tapped to rake in billions competing with other JAK inhibitors made by rivals including AbbVie and Eli Lilly.

Now, long term data on the drug — discovered by Gilead’s partners at Galapagos and posted as part of a virtual medical conference — have solidified the durability and safety of filgotinib in patients with rheumatoid arthritis, spanning data from three late-stage trials. An FDA decision on the drug is expected this year.