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.”

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

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I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

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Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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BAR­DA slows its $9B en­gine for new Covid-19 ther­a­peu­tics

The Biomedical Advanced Research and Development Authority is cooling its jets in looking for new, potential Covid-19 treatments, at least in the near term.

An HHS spokesperson told Endpoints News via email, “to date, BARDA has obligated more than $9 billion for the development and/or purchase of 13 therapeutics, beginning in February 2020 with support to develop Regeneron’s monoclonal antibody therapeutic. Therapeutics are an important element of the COVID-19 response, and we are focused on the programs currently underway and/or in negotiation using the funds available to us.”

Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.

Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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Marshall Fordyce, Vera CEO

Gene ther­a­py play­er turned kid­ney spe­cial­ist Ve­ra drops a dud in lead­up to Nas­daq, pric­ing well be­low range

Vera Therapeutics took a big risk at the start of the year, pivoting away from its gene editing mission statement to chase a lead kidney drug instead — and they doubled down with an IPO just months later. But investors don’t seem impressed with Vera’s promise, and now the biotech is looking at a far more scaled-back offering.

On Friday, Vera priced its 4.35-million-share IPO at $11 per share, well below its targeted range of $14 to $16 and good for $47.58 million in proceeds. The biotech will start trading Monday under the ticker $VERA.

Darren Ji, Elpiscience CEO (Lilly Asia Ventures)

Kept an ocean away from its sci­en­tif­ic ad­vi­sors, Shang­hai's Elpi­science keeps up the clin­i­cal progress, re­fu­els for its I/O pipeline

When Elpiscience pooled $100 million for its Series B in late 2019, CEO Darren Ji promised to move what he described as one of the broadest immuno-oncology pipelines swiftly through the clinic in both the US and China.

Then a pandemic got in the way — but not by much. The Shanghai-based biotech managed to keep testing its 4-1BB/PD-L1 drug, get an OX40 agonist cleared for clinical trials (nabbing a collaboration with Junshi in the process), while in-licensing a Phase I bispecific from California’s TRIGR Therapeutics.

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