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.”

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.

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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Yong Dai, Frontera Therapeutics CEO

Scoop: Lit­tle-known Or­biMed-backed biotech clos­es $160M round to start gene ther­a­py tri­al

Frontera Therapeutics, a China and US biotech, has closed a $160 million Series B and received regulatory clearance to test its first gene therapy stateside, Endpoints News has learned.

Led by the largest shareholder, OrbiMed, the biotech has secured $195 million total since its September 2019 founding, according to an email reviewed by Endpoints. The lead AAV gene therapy program is for an undisclosed rare eye disease, according to the source.

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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.

John Leonard, Intellia CEO

In­tel­li­a's CRISPR pro­gram that ed­its genes di­rect­ly in pa­tients shows dura­bil­i­ty in AT­TR amy­loi­do­sis

The first in vivo CRISPR/Cas9 gene editing program has some new durability data showing sustained reduction of a toxic protein in ATTR amyloidosis at all four dose levels in a small 15-patient study.

Intellia Therapeutics presented the much-anticipated data for its Regeneron-collaborated NTLA-2001 Friday morning, adding to the initial Phase I results it first delivered almost a year ago to the day.