Kite adds promis­ing leukemia re­spons­es in small CAR-T stud­ies, then fires the start­ing gun on its rolling sub­mis­sion

Kite Phar­ma­ceu­ti­cals $KITE has lined up a fresh batch of pos­i­tive ef­fi­ca­cy da­ta for its lead CAR-T for ad­vanced cas­es of acute lym­phoblas­tic leukemia at ASH. And then lat­er Sun­day evening the biotech added that it has launched its rolling sub­mis­sion for a first ap­proval.

David Chang, Kite

In­ves­ti­ga­tors turned up at the big ASH meet­ing to say that the drug spurred a com­plete re­mis­sion in 9 of 11 evalu­able pa­tients — 82% — en­rolled in the ZU­MA-3 and ZU­MA-4 Phase I stud­ies for ALL in a pre­lim­i­nary re­view.

But the treat­ment was not with­out dan­ger. Five of the 13 had se­ri­ous bouts of cy­tokine re­lease syn­drome as well as 5 who had grade three or above cas­es of neu­ro­log­i­cal events. There were no cas­es of cere­bral ede­ma, which have be­dev­iled ri­val Juno, but one of the pa­tients in the tri­als died from CRS.

Kite had long hoped to file its first ap­pli­ca­tion for KTE-C19 be­fore the end of the year, but was forced to de­lay un­til Q1 2017 af­ter some in­ter­ac­tions with the FDA, pre­sum­ably so it would have time to in­clude da­ta on the 6-month re­spons­es of pa­tients in ZU­MA-1. Some an­a­lysts have been skep­ti­cal of the dura­bil­i­ty of the treat­ment af­ter see­ing the ef­fect de­cline over a few months of treat­ment, but com­pa­ny ex­ecs ex­pect to see that re­sponse lev­el plateau at a sig­nif­i­cant lev­el.

Soon af­ter Kite post­ed the lat­est da­ta on their ther­a­py Sun­day, the com­pa­ny an­nounced that it had launched the rolling sub­mis­sion and would get it com­plet­ed by the end of the first quar­ter of 2017. But No­var­tis now may have an edge in fil­ing first, and look­ing for the first ever ap­proval of a CAR-T ther­a­py.

The drug re­mains one of the lead­ing CAR-T ther­a­pies head­ing to the FDA. No­var­tis is now hus­tling along its own CAR-T af­ter reg­is­ter­ing an im­pres­sive 82% com­plete re­mis­sion rate among the 50 chil­dren and young adults with B-cell acute lym­phoblas­tic leukemia.

“In the Phase I por­tion of these stud­ies, we on­ly en­rolled pa­tients with high dis­ease bur­den to rig­or­ous­ly eval­u­ate safe­ty and ef­fi­ca­cy of KTE-C19,” said Kite R&D di­rec­tor David Chang. “We look for­ward to ini­ti­at­ing the Phase II por­tions of these stud­ies in 2017.”

Dan Skovronsky, Eli Lilly CSO

UP­DAT­ED: An­a­lysts are quick to pan Eli Lil­ly's puz­zling first cut of pos­i­tive clin­i­cal da­ta for its Covid-19 an­ti­body

Eli Lilly spotlighted a success for one of 3 doses of their closely-watched Covid-19 antibody drug Wednesday morning. But analysts quickly highlighted some obvious anomalies that could come back to haunt the pharma giant as it looks for an emergency use authorization to launch marketing efforts.

The pharma giant reported that LY-CoV555, developed in collaboration with AbCellera, significantly reduced the rate of hospitalization among patients who were treated with the antibody. The drug arm of the study had a 1.7% hospitalization rate, compared to 6% in the control group, marking a 72% drop in risk.

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#ES­MO20: Alk­er­mes of­fers their first snap­shot of a ben­e­fit for their next-gen IL-2 drug. But why did 1 pa­tient starve to death?

Everyone in the cancer R&D arena is looking to build new franchises around better drugs and combos. And one busy pocket of that space is centered entirely on creating an IL-2 drug that can be as effective as the original without the toxicity that damned it to the sidelines.

Alkermes $ALKS formally tossed its hat into the ring of contenders at virtual ESMO today, highlighting the first glimpse of efficacy for their candidate, ALKS 4230, as both a monotherapy as well as in combination with Merck’s Keytruda.

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Albert Bourla (Photo by Steven Ferdman/Getty Images)

Pfiz­er match­es Mod­er­na with their full Covid-19 tri­al blue­print — As­traZeneca says it will un­veil its pro­to­col 'short­ly'

Yesterday, after sustained public pressure as Moderna released its Phase III Covid-19 trial blueprint, Pfizer released its own full trial design for their vaccine trials. The move was designed to boost transparency and shore up public trust in the vaccines, but it also revealed differences in how the two companies are approaching the much-watched studies while failing to satisfy the demands of the fiercest advocates for transparency.

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#ES­MO20: Re­gen­eron, Sanofi eye an­oth­er first for their PD-1 con­tender Lib­tayo with promis­ing da­ta for on­col­o­gy niche

Regeneron and Sanofi took another step forward in the long march towards a greatly expanded market for their late-bloomer PD-1 checkpoint Libtayo.

The two occasional allies posted an objective response rate of 31% for Libtayo among 84 patients suffering from advanced cases of basal cell carcinoma at virtual ESMO. That spotlights progress for 26 patients, 5 of whom had a complete response. The data also reflect a boost in the number of responses seen from the last cut of the numbers.

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Christian Itin, Autolus CEO (Autolus)

#ES­MO20: Au­to­lus pro­vides glimpse of next-gen­er­a­tion CAR-T pro­gram, show­ing ear­ly pos­i­tive safe­ty da­ta

CAR-T therapies were hailed as a breakthrough when Novartis received the first FDA approval for Kymriah back in 2017. Though highly effective at treating certain types of blood cancers, CAR-Ts are also associated with severe and potentially deadly side effects, including lethal instances of cytokine release syndrome.

With this in mind, Autolus Therapeutics is looking to take a crack at a safer CAR-T and presented Phase II cohort data for its AUTO3 program at virtual ESMO 2020. The data showed that, among the 35 patients in the cohort being treated for r/r diffuse large B cell lymphoma, there were no instances of Grade 3 or higher CRS. Eight individuals saw Grade 1 inflammation while another four patients reached Grade 2.

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Albert Bourla, Pfizer CEO (Steven Ferdman/Getty Images)

Pfiz­er ex­ecs con­fi­dent­ly tap their top 10 block­busters-to-be. But what are the chances of sur­viv­ing PhI­II, let alone hit­ting these big peak sales es­ti­mates?

Pfizer’s top executive team doesn’t lack for confidence.

Where many Big Pharmas would be reluctant to put a peak sales figure on their late-stage drugs, Pfizer CEO Albert Bourla has shrugged off the usual diffidence to outline where the pharma giant expects to get $15 billion-plus.

The list, outlined this week during their investor presentations, is topped by 3 drugs in the $3 billion-plus peak sales category. They are:

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Seat­tle Ge­net­ic­s' Astel­las-part­nered ADC nails con­fir­ma­to­ry PhI­II in urothe­lial can­cer

Nine months after Seattle Genetics nabbed an accelerated approval for its Astellas-partnered antibody-drug conjugate Padcev, the partners said the therapy has nailed a confirmatory Phase III, proving its worth in locally advanced or metastatic urothelial cancer.

Padcev, which has widely been tapped as a potential blockbuster, scored improvements in both overall survival and progression-free survival compared to chemotherapy, causing a 30% reduction in risk of death (p = 0.001) and 39% reduction in risk of disease progression or death (p<0.00001).

Stéphane Bancel, Moderna CEO

UP­DAT­ED: 'The biggest in­flec­tion point ever': On R&D day, Mod­er­na CEO Stéphane Ban­cel dou­bles down on vac­cines and lays out a vi­sion for a '40, 50'-drug biotech

The stakes have always been different for Moderna. They didn’t invest more in the Covid-19 vaccine race than Pfizer, Sanofi or AstraZeneca, and they didn’t have more to lose than Novavax — a $100 million biotech that became a $7 billion company virtually overnight. But they had considerably more to gain than anyone.

Born with grand ambitions and the checkbook to match, Moderna spent its first decade of life attracting as many critics of its vision to remake drug development as it did backers. But the pandemic presented a twin opportunity: to prove its technology on a global stage and to give a still-young biotech billions in annual revenue years before anticipated.

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Steve Hurly, Lava CEO

La­va breaks pro­longed si­lence with an $83M Se­ries C and two I/O pro­grams set for the clin­ic

Lava debuted in May of 2018 with $18.9 million, a platform built around something called gamma delta cells and a plan, apparently, of saying nothing else for the next two years. They never announced a program and they did not issue another press release for the next 20 months.

In May, though, the Dutch-American biotech announced a partnership on cancer bispecifics with J&J. And today, CEO Steve Hurly is ready to talk about what they’ve been working on for the last two years in Utrecht and Philadelphia – as well as the $83 million they raised from Novo, Sanofi and others to bring that work into the clinic next year.