Rac­ing past a wound­ed Juno, Kite aims to file lead CAR-T for OK by end of 2016

With Juno Ther­a­peu­tics se­ri­ous­ly de­layed by a brief but painful clin­i­cal hold by the FDA, ri­val Kite Phar­ma out­lined plans to­day to shoot for an ac­cel­er­at­ed ap­proval af­ter it gath­ers the first round of da­ta on 50 pa­tients from its piv­otal study of a ri­val CAR-T, due in just a few months. And com­pa­ny ex­ec­u­tives say their man­u­fac­tur­ing op­er­a­tions will be ready to start pro­duc­ing this ther­a­py in com­mer­cial quan­ti­ties be­fore the end of this year.

If suc­cess­ful, Kite could be the first to the mar­ket, es­pe­cial­ly if the FDA opts to act quick­ly for one of its ‘break­through’ ther­a­pies, which pro­vides for a swift re­view. Juno has al­ready said that it won’t be able to reach the mar­ket be­fore 2018. And No­var­tis has yet to de­tail what kind of time­line it is look­ing at.

Their SEC fil­ing in­cludes this state­ment:

“We have com­plet­ed en­roll­ment of all 72 pa­tients in the DL­B­CL (dif­fuse large B cell lym­phoma) co­hort and 20 pa­tients in the PM­B­CL (pri­ma­ry me­di­asti­nal B cell lym­phoma) and TFL (trans­formed fol­lic­u­lar lym­phoma) co­hort in ZU­MA-1. We plan to open an ad­di­tion­al co­hort in ZU­MA-1 to al­low us to con­tin­ue to dose pa­tients with KTE-C19 in the Unit­ed States and to ex­pand the clin­i­cal tri­al to Eu­rope. We plan to re­port ZU­MA-1 top-line da­ta from the first 50 DL­B­CL pa­tients with at least three-months of fol­low-up by the end of the third quar­ter of 2016. If we be­lieve the da­ta are com­pelling, we plan to dis­cuss with the FDA the fil­ing of a Bi­o­log­ics Li­cense Ap­pli­ca­tion, or BLA, for ac­cel­er­at­ed ap­proval of KTE-C19 as a treat­ment for pa­tients with re­frac­to­ry DL­B­CL, PM­B­CL and TFL. Sub­ject to the in­ter­im re­sults and dis­cus­sions with the FDA, we plan to sub­mit the BLA at the end of 2016. If ap­proved, we plan to com­mer­cial­ly launch KTE-C19 in 2017.  We plan to re­port da­ta from ZU­MA-2 and the Phase 2 por­tions of ZU­MA-3 and ZU­MA-4 in 2017. If we be­lieve the da­ta are com­pelling, we plan to pur­sue FDA ap­proval for these ad­di­tion­al in­di­ca­tions.”

The race be­tween Kite and Juno for a pi­o­neer­ing FDA OK has be­come one of the most close­ly fol­lowed ri­val­ries in R&D. Both had been shoot­ing for a 2017 launch. But just days ago Juno ac­knowl­edged in its quar­ter­ly re­view that a 6-day hold on JCAR015 would post­pone any com­mer­cial ef­fort un­til 2018.

An ag­gres­sive Kite is clear­ly tak­ing ad­van­tage of every an­gle it can think of. CAR-T ther­a­pies re­ly on cells ex­tract­ed from pa­tients which are then reengi­neered to in­clude a chimeric anti­gen re­cep­tor that can hunt down can­cer cells.

Juno has said that its prob­lems with JCAR015 were trig­gered by the ad­di­tion of flu­dara­bine to the reg­i­men used to prep pa­tients to bet­ter re­spond to their drug. The biotech fin­gered flu­dara­bine for the deaths of four pa­tients, which spurred the hold. No­tably, while Juno was grap­pling with the FDA, Kite put out an an­nounce­ment that it was stay­ing on track with its lead pro­gram.

Kite CEO Arie Bellde­grun

In a call with an­a­lysts Mon­day evening, Kite CEO Arie Bellde­grun al­so said that while in­ves­ti­ga­tors are us­ing flu­dara­bine in their pre­con­di­tion­ing reg­i­men, they have yet to see any of the un­usu­al­ly lethal ad­verse events with neu­ro­tox­i­c­i­ty that tripped up Juno. “I have to say that ad­verse event pro­file has been very much on par with what we have been pre­dict­ing based on the ear­li­er stud­ies,” the CEO told an­a­lysts.

Less than two months ago, Kite held a rib­bon cut­ting cer­e­mo­ny for its new man­u­fac­tur­ing fa­cil­i­ty, a 43,500-square-foot plant that will be used to make its per­son­al­ized KTE-C19.

“Our com­mer­cial fa­cil­i­ty will have the ca­pac­i­ty to pro­duce up to 5,000 pa­tient ther­a­pies per year and we ex­pect it to be op­er­a­tional in pro­duc­ing clin­i­cal ma­te­ri­als by year-end,” not­ed Bellde­grun. “Over­all, we have con­tin­u­ous­ly been op­ti­miz­ing key as­pect of our man­u­fac­tur­ing, sup­ply chain, and qual­i­ty con­trol and pos­sess a pro­pri­etary process that dra­mat­i­cal­ly re­duces the time to ap­prox­i­mate­ly 14 days for when a pa­tients ma­te­r­i­al are shift to our fa­cil­i­ty to when the en­gi­neered T-cells are re­leased to the pa­tient. This is one of the fastest rates in the in­dus­try.”

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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As­traZeneca trum­pets the good da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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The Avance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

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Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

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Bryan Roberts, Venrock

Ven­rock sur­vey shows grow­ing recog­ni­tion of coro­n­avirus toll, wan­ing con­fi­dence in ar­rival of vac­cines and treat­ments

When Venrock partner Bryan Roberts went to check the results from their annual survey of healthcare leaders, what he found was an imprint of the pandemic’s slow arrival in America.

The venture firm had sent their form out to hundreds of insurance and health tech executives, investors, officials and academics on February 24 and gave them two weeks to fill it out. No Americans had died at that point but the coronavirus had become enough of a global crisis that they included two questions about the virus, including “Total U.S. deaths in 2020 from the novel coronavirus will be:”.

Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

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David Hoey (Vaxxas)

In for the long vac­cine game, Mer­ck buys in­to patch de­liv­ery tech with pan­dem­ic po­ten­tial

When Merck dived into the R&D fray for a Covid-19 vaccine earlier this week, execs made it clear that they’re not necessarily looking to be first — with CEO Ken Frazier throwing cold water on the hotly-discussed 12- to 18-month timelines. But when it does emerge from behind, the pharma giant clearly expects to play a significant part.

Part of that will depend on next-generation delivery technology that reshapes the world’s imagination of a vaccine.

No­var­tis jumps in­to Covid-19 vac­cine hunt, as Big Phar­ma and big biotech com­mit to bil­lions of dos­es

After spending most of the pandemic on the sidelines, Novartis is offering its aid in the race to develop a Covid-19 vaccine.

AveXis, the Swiss pharma’s gene therapy subsidiary, has agreed to manufacture the vaccine being developed by Massachusetts Eye and Ear and Massachusetts General Hospital. The biotech will begin manufacturing this month, while the vaccine undergoes further preclinical testing. They’ve agreed to provide the vaccine for free for clinical trials beginning in the second half of 2020, but have not disclosed financials for after.

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