Kite catch­es the in­side track on the last leg of a race to the fin­ish line with No­var­tis on a CAR-T first

David Chang, Kite

The race be­tween No­var­tis $NVS and Kite $KITE to win the first FDA ap­proval of a CAR-T for can­cer is now in the fi­nal stretch, and Kite just short­ened its dis­tance to the fin­ish line.

The biotech an­nounced this morn­ing that the FDA is pro­vid­ing a pri­or­i­ty re­view for their drug KTE-C19 (axi­cab­ta­gene ciloleu­cel), which will cut the time it takes for reg­u­la­tors to com­plete their ex­am­i­na­tion from 10 months down to 6. The FDA’s PDU­FA date is No­vem­ber 29.

Kite’s big ri­val No­var­tis won a pri­or­i­ty re­view two months ago, which should pro­vide a com­fort­able lead for the first de­ci­sion on its land­mark drug. Both rep­re­sent the cut­ting edge of a new class of drug that ex­tracts cells from pa­tients and then reengi­neers them in­to an at­tack weapon tar­get­ing can­cer cells.

Both KTE-C19 and No­var­tis’ CTL019 are tapped as two of the top block­busters in the in­dus­try pipeline. They’re go­ing af­ter dif­fer­ent in­di­ca­tions on the first round, with Kite ini­tial­ly tak­ing on drug-re­sis­tant non-Hodgkin lym­phoma. But No­var­tis — tak­ing its first shot at chil­dren with acute lym­phoblas­tic leukemia — will switch fo­cus to its JULI­ET study for some head-to-head com­par­isons on dif­fuse large B-cell lym­phoma that will at­tract plen­ty of in­tense at­ten­tion, as well as spec­u­la­tion over the im­pact it will have on Kite’s stock price.

That new da­ta — which al­ready earned a spe­cial break­through ti­tle at the FDA — should ar­rive next month.

In the mean­time, reg­u­la­tors will be fol­low­ing through on re­views for two des­ig­nat­ed ‘break­through’ drugs, mak­ing it at least pos­si­ble for the two drugs to reach an ex­pert pan­el at about the same time.

Kite had man­aged to get through its reg­is­tra­tion pro­gram with­out any ex­cep­tion­al safe­ty is­sues — right un­til it was hit a few weeks ago with the death of a pa­tient due to cere­bral ede­ma. The biotech went to some pains to note just how sick this pa­tient was when he died, but its stock slid as in­vestors con­sid­ered the im­pli­ca­tions. Juno’s lead CAR-T was knocked out of the race when 5 pa­tients died of cere­bral ede­mas, or brain swelling, in­clud­ing 2 who died af­ter the FDA de­cid­ed to lift a clin­i­cal hold on the pro­gram af­ter just a few days.

Those deaths and all the safe­ty is­sues fac­ing CAR-T ther­a­pies will now get care­ful scruti­ny at the FDA.

“Pa­tients with re­frac­to­ry ag­gres­sive NHL face a dire prog­no­sis with on­ly a 50 per­cent chance of sur­viv­ing six months. This un­der­scores the ur­gent med­ical need for these pa­tients and why every day mat­ters, from de­vel­op­ment to man­u­fac­tur­ing to clin­i­cal ex­pe­ri­ence,” said David Chang, ex­ec­u­tive vice pres­i­dent of re­search and de­vel­op­ment and CMO of Kite. “We firm­ly be­lieve in the po­ten­tial for axi­cab­ta­gene ciloleu­cel to ad­dress this need and forge a new path for the fu­ture of cell ther­a­py.”

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Aymeric Le Chatelier, Ipsen

A $1B-plus drug stum­bles in­to an­oth­er big PhI­II set­back -- this time flunk­ing fu­til­i­ty test -- as FDA hold re­mains in ef­fect for Ipsen

David Meek

At the time Ipsen stepped up last year with more than a billion dollars in cash to buy Clementia and a late-stage program for a rare bone disease that afflicts children, then CEO David Meek was confident that he had put the French biotech on a short path to a mid-2020 launch.

Instead of prepping a launch, though, the company was hit with a hold on the FDA’s concerns that a therapy designed to prevent overgrowth of bone for cases of fibrodysplasia ossificans progressiva might actually stunt children’s growth. So they ordered a halt to any treatments for kids 14 and under. Meek left soon after to run a startup in Boston. And today the Paris-based biotech is grappling with the independent monitoring committee’s decision that their Phase III had failed a futility test.

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UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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Roche's check­point play­er Tecen­triq flops in an­oth­er blad­der can­cer sub­set

Just weeks after Merck’s star checkpoint inhibitor Keytruda secured FDA approval for a subset of bladder cancer patients, Swiss competitor Roche’s Tecentriq has failed in a pivotal bladder cancer study.

The 809-patient trial — IMvigor010 — tested the PD-L1 drug in patients with muscle-invasive urothelial cancer (MIUC) who had undergone surgery, and were at high risk for recurrence.

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Gilead claims Tru­va­da patents in HHS’ com­plaint are in­valid

Back in November, the Department of Health and Human Services took the rare step of filing a complaint against Gilead for infringing on government-owned patents related to the HIV drug Truvada (emtricitabine/tenofovir disoproxil fumarate) for pre-exposure prophylaxis (PrEP).

But on Thursday, Gilead filed its own retort, making clear that it does not believe it has infringed on the Centers for Disease Control and Prevention’s (CDC) Truvada patents because they are invalid.

Gilead dusts off a failed Ebo­la drug as coro­n­avirus spreads; Ex­elix­is boasts pos­i­tive Ph I/II da­ta

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.

Alex Karnal (Deerfield)

Deer­field vaults to the top of cell and gene ther­a­py CD­MO game with $1.1B fa­cil­i­ty at Philadel­phi­a's newest bio­phar­ma hub

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.

Tri­fec­ta of sick­le cell dis­ease ther­a­pies ex­tend life ex­pectan­cy, but are not cost-ef­fec­tive — ICER

Different therapeutic traits brandished by the three approved therapies for sickle cell disease all extend life expectancy, but their impact on quality of life is uncertain and their long-term cost-effectiveness is not up to scratch according to the thresholds considered reasonable by ICER, the non-profit concluded in a draft guidance report on Thursday.

Sickle cell disease (SCD), which encompasses a group of inherited red blood cell disorders that typically afflict those of African ancestry, impacts hemoglobin — and is characterized by episodes of searing pain as well as organ damage.