In wake of a CAR-T re­struc­tur­ing, No­var­tis says it’s ready to hunt a pi­o­neer­ing OK for lead pro­gram

Bruno St­rig­i­ni, CEO No­var­tis On­col­o­gy

No­var­tis is still in the race to win the world’s first ap­proval for a pi­o­neer­ing CAR-T treat­ment.

The phar­ma gi­ant $NVS pulled back the veil from their Phase II study of their CAR-T CTL019, reg­is­ter­ing an im­pres­sive 82% com­plete re­mis­sion rate among the 50 chil­dren and young adults with B-cell acute lym­phoblas­tic leukemia. And with no deaths, though sig­nif­i­cant ev­i­dence of tox­i­c­i­ty, No­var­tis says it will be ready to file for an ap­proval ear­ly on in a loom­ing 2017.

Forty-one of 50 pa­tients achieved com­plete re­mis­sion or com­plete re­mis­sion with in­com­plete blood count re­cov­ery three months af­ter an in­fu­sion of CTL019, which reengi­neers pa­tients’ T cells in­to a can­cer ther­a­py.

Close to half of the pa­tients ex­pe­ri­enced a grade 3 or 4 case of cy­tokine re­lease syn­drome, not un­com­mon in the field. And 15% per­cent of pa­tients ex­pe­ri­enced grade 3 neu­ro­log­i­cal and psy­chi­atric events in­clud­ing en­cephalopa­thy and delir­i­um. But there were no grade 4 events and no one died.

That’s a sol­id enough ben­e­fit/safe­ty pro­file to take to reg­u­la­tors as No­var­tis hunts up an ac­cel­er­at­ed ap­proval.

No­var­tis man­aged to stun prac­ti­cal­ly every­one in the field when it abrupt­ly dis­band­ed its cell and gene ther­a­py unit three months ago, lay­ing off 120 and in­te­grat­ing an­oth­er 280 back in­to its reg­u­lar R&D ranks as CAR-T chief Us­man ‘Oz’ Azam hit the ex­it. That was wide­ly re­gard­ed as a se­ri­ous set­back that could af­fect fu­ture pro­grams, even if more ad­vanced ef­forts like this one at the Uni­ver­si­ty of Penn­syl­va­nia stayed on track.

Juno has been thrown in­to ut­ter dis­ar­ray with mul­ti­ple deaths de­rail­ing their lead ef­fort for JCAR015. And Kite re­cent­ly not­ed that it would need a lit­tle more time to com­plete a rolling sub­mis­sion for their top ther­a­py, which should now be com­plet­ed in Q1.

“This first-of-its-kind tri­al rep­re­sents ex­cit­ing progress to­ward our goal of help­ing chil­dren and young adults with re­lapsed or re­frac­to­ry B-cell ALL, a pa­tient pop­u­la­tion with an ur­gent need for new treat­ment op­tions,” said Bruno St­rig­i­ni, CEO, No­var­tis On­col­o­gy. “We are com­mit­ted to ad­vanc­ing CTL019 and look for­ward to work­ing close­ly with the FDA and EMA in the com­ing months.”

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Dipal Doshi, Entrada Therapeutics CEO

Ver­tex just found the next big ‘trans­for­ma­tive’ thing for the pipeline — at a biotech just down the street

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

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Christian Itin, Autolus CEO (UKBIO19)

Au­to­lus tips its hand, bags $220M as CAR-T show­down with Gilead looms

The first batch of pivotal data on Autolus Therapeutics’ CAR-T is in, and execs are ready to plot a path to market.

With an overall remission rate of 70% at the interim analysis featuring 50 patients, the results set the stage for a BLA filing by the end of 2023, said CEO Christian Itin.

Perhaps more importantly — given that Autolus’ drug, obe-cel, is going after an indication that Gilead’s Tecartus is already approved for — the biotech highlighted “encouraging safety data” in the trial, with a low percentage of patients experiencing severe immune responses.

Rami Elghandour, Arcellx CEO

Up­dat­ed: Gilead, Ar­cel­lx team up on an­ti-BC­MA CAR-T as biotech touts a 100% re­sponse rate at #ASH22

Gilead and Kite are plunking down big cash to get into the anti-BCMA CAR-T game.

The pair will shell out $225 million in cash upfront and $100 million in equity to Arcellx, Kite announced Friday morning, to develop the biotech’s lead CAR-T program together. Kite will handle commercialization and co-development with Arcellx, and profits in the US will be split 50-50.

Concurrent with the deal, Arcellx revealed its latest cut of data for the program known as CART-ddBCMA, ahead of a full presentation at this weekend’s ASH conference — a 100% response rate among patients getting the therapy. Investors jumped at the dual announcements, sending Arcellx shares $ACLX up more than 25% in Friday’s morning session.

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WIB22: Am­ber Salz­man had few op­tions when her son was di­ag­nosed with a rare ge­net­ic dis­ease. So she cre­at­ed a bet­ter one

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

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Ahead of ad­comm, FDA rais­es un­cer­tain­ties on ben­e­fit-risk pro­file of Cy­to­ki­net­ic­s' po­ten­tial heart drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet next Tuesday to discuss whether Cytokinetics’ potential heart drug can safely reduce the risk of cardiovascular death and heart failure in patients with symptomatic chronic heart failure with reduced ejection fraction.

The drug, known as omecamtiv mecarbil and in development for more than 15 years, has seen mixed results, with a first Phase III readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.

Ab­b­Vie slapped with age dis­crim­i­na­tion law­suit, fol­low­ing oth­er phar­mas

Add AbbVie to the list of pharma companies currently facing age discrimination allegations.

Pennsylvania resident Thomas Hesch filed suit against AbbVie on Wednesday, accusing the company of passing him over for promotions in favor of younger candidates.

Despite 30 years of pharma experience, “Hesch has consistently seen younger, less qualified employees promoted over him,” the complaint states.

Scoop: Gilead ter­mi­nates ear­ly-stage FLT3 tri­al in sol­id tu­mors

Gilead chopped a Phase Ib dose escalation study in recent days, with an update to the federal trials database saying the premature termination followed an “internal safety assessment.”

The IV-administered FLT3 agonist, dubbed GS-3583, was being tested as a monotherapy in 13 patients with advanced solid tumors. The goal of the trial was to find out what dose to test in a Phase II, or maximum tolerated dose.

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Susan Galbraith, AstraZeneca EVP, oncology R&D, at EUBIO22 (Rachel Kiki for Endpoints News)

As­traZeneca’s Su­san Gal­braith high­lights twin wins for the can­cer drug pipeline at SABCS, as oral SERD ex­cels

It’s a good time to be the head of R&D for oncology at AstraZeneca. And no one gets that quite like Susan Galbraith.

Today, Galbraith is at the San Antonio Breast Cancer Symposium, highlighting the data on two key drugs in the cancer pipeline: mid-stage results for its oral SERD camizestrant among patients after one line of therapy, and the AKT drug capivasertib, wrapping the Phase III. Both fall neatly into the range of successes, beating out fulvestrant in hormone receptor-positive, HER2-negative breast cancer.

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