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.”

NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data are messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data are exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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No­vo CEO Lars Fruer­gaard Jør­gensen on R&D risk, the deal strat­e­gy and tar­gets for gen­der di­ver­si­ty

 

I kicked off our European R&D summit last week with a conversation involving Novo Nordisk CEO Lars Fruergaard Jørgensen. Novo is aiming to launch a new era of obesity management with a new approval for semaglutide. And Jørgensen had a lot to say about what comes next in R&D, how they manage risk and gender diversity targets at the trendsetting European pharma giant.

John Carroll: I’m here with Lars Jørgensen, the CEO of Novo Nordisk. Lars, it’s been a really interesting year so far with Novo Nordisk, right? You’ve projected a new era of growing sales. You’ve been able to expand on the GLP-1 franchise that was already well established in diabetes now going into obesity. And I think a tremendous number of people are really interested in how that’s working out. You have forecast a growing amount of sales. We don’t know specifically how that might play out. I know a lot of the analysts have different ideas, how those numbers might play out, but that we are in fact embarking on a new era for Novo Nordisk in terms of what the company’s capable of doing and what it’s able to do and what it wants to do. And I wanted to start off by asking you about obesity in particular. Semaglutide has been approved in the United States for obesity. It’s an area of R&D that’s been very troubled for decades. There have been weight loss drugs that have come along. They’ve attracted a lot of attention, but they haven’t actually ever gained traction in the market. My first question is what’s different this time about obesity? What is different about this drug and why do you expect it to work now whereas previous drugs haven’t?

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Marty Duvall, Oncopeptides CEO

On­copep­tides stock craters as it pulls can­cer drug Pepax­to from the mar­ket

Shares of Oncopeptides crashed more than 70% in early Friday trading after the company said it’s pulling its multiple myeloma drug Pepaxto (melphalan flufenamide) from the US market after failing a confirmatory trial. The move will force the company to close its US and EU business units and enact significant layoffs.

The FDA had scheduled an adcomm meeting next Thursday to discuss Pepaxto, which first won accelerated approval in February and costs about $19,000 per course of treatment. The committee was to weigh in on whether the confirmatory trial demonstrated a worse overall survival in the treatment arm compared to the control arm.

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Oc­u­lar Ther­a­peu­tix ham­mered by a PhII fail­ure in dry eye dis­ease — shares tank

Ocular Therapeutix $OCUL has had its ups and downs in the 7 years since it went public. Friday was one of those down days.

The Bedford, MA-based biotech reported that its lead experimental eye drug, OTX-CSI (cyclosporine intracanalicular insert), failed a Phase II trial for dry eye disease. And the stock experienced one of its periodic meltdowns, dropping more than 30% ahead of the bell.

The therapy flat failed the primary endpoint: increased tear production at 12 weeks as measured by the Schirmer’s Test compared to the vehicle control group. And while investigators called out an improvement from baseline in “signs of dry eye disease as measured by total corneal fluorescein staining (CFS) and symptoms of dry eye disease as measured by the visual analogue scale (VAS) eye dryness in subjects treated with the OTX-CSI insert,” it wasn’t statistically significant.

Pfiz­er pitch­es its Covid-19 vac­cine for younger chil­dren ahead of ad­comm next week

Pfizer will present its case to the FDA’s vaccine adcomm next week, seeking authorization for a lower-dose version of its Covid-19 vaccine for kids ages 5 through 12, which the Biden administration said will likely begin rolling out early next month.

Two primary doses of the 10 µg vaccine (the dose for those ages 12 and up is 30 μg) given 3 weeks apart in this group of children “have shown a favorable safety and tolerability profile, robust immune responses against all variants of concern including Delta, and vaccine efficacy of 90.7% against laboratory-confirmed symptomatic COVID-19,” the company said in briefing documents ahead of next Tuesday’s meeting of the FDA’s Vaccines and Related Biological Products Advisory Committee.

Sanofi, Re­gen­eron etch out an­oth­er PhI­II vic­to­ry for Dupix­ent, eas­ing se­vere itch and clear­ing le­sions

Sanofi and Regeneron can boast of another inflammatory disease where Dupixent has proven effective.

The best-selling drug, which targets both IL-4 and IL-13, has delivered a clean sweep in a Phase III trial for prurigo nodularis, a chronic disease characterized by itch so intense that it can affect patients’ sleep and psychology. Thick skin lesions can cover most of the body.

On the primary endpoint, 37% of patients taking Dupixent saw a clinically meaningful reduction in itch compared to 22% of those on placebo (p=0.0216) at week 12. All secondary endpoints were also met, including clearance of skin lesions and improvement in quality of life.