Cel­lec­tis preps for ground­break­ing US tri­als for an off-the-shelf CAR-T ther­a­py

Cel­lec­tis is ramp­ing up the first-ever US tri­al launch­es for an off-the-shelf CAR-T ther­a­py.

The Paris-based biotech $CLLS, which has a siz­able R&D group in Man­hat­tan, says the FDA has signed off on their IND for a Phase I study of UCART123 in acute myeloid leukemia and blas­tic plas­ma­cy­toid den­drit­ic cell neo­plasm.

The plan now is to get start­ed in the next few months, wrap up their ear­ly-stage work in ear­ly 2018 and then launch what they hope will be a reg­is­tra­tional Phase II tri­al in mid-2018 — pro­vid­ed the FDA signs off, Dr. Loan Hoang-Sayag, Cel­lec­tis’ chief med­ical of­fi­cer, tells End­points News via email.

Servi­er and part­ner Pfiz­er have been col­lab­o­rat­ing with Cel­lec­tis on an on­go­ing Phase I study for the lead ther­a­py, UCART19, in the UK. UCART123 is a sole­ly-owned Cel­lec­tis pro­gram us­ing TAL­EN en­gi­neered T cells.

The biotech is com­ing in be­hind lead au­tol­o­gous CAR-T play­ers Kite and No­var­tis, both of which are an­gling for the first-ever ap­proval this year for reengi­neered cells ex­tract­ed from pa­tients. Cel­lec­tis, helmed by CEO An­dre Chouli­ka, has been steadi­ly ad­vanc­ing these off-the-shelf ther­a­pies as an eas­i­er to man­u­fac­ture and de­liv­er can­cer treat­ment that could be a nat­ur­al suc­ces­sor to the first CAR-Ts to hit the mar­ket.

It’s a tricky process though. Their ther­a­py has been test­ed in com­pas­sion­ate use cas­es, and in one in­stance re­quired steroids to tamp down on an im­mune re­sponse, which is one re­ac­tion they def­i­nite­ly don’t want to see in an al­lo­gene­ic ap­proach. The au­tol­o­gous lead­ers have been us­ing pa­tient-de­rived cells pre­cise­ly in or­der to avoid any im­mune re­sponse.

UCART123 is a gene-edit­ed T-cell in­ves­ti­ga­tion­al drug that tar­gets CD123, an anti­gen ex­pressed at the sur­face of leukemic cells in AML, tu­moral cells in BPD­CN. The clin­i­cal re­search for AML will be led at Weill Cor­nell by prin­ci­pal in­ves­ti­ga­tor Dr. Gail J. Roboz, sirec­tor of the Clin­i­cal and Trans­la­tion­al Leukemia Pro­grams. The UCART123 clin­i­cal pro­gram for BPD­CN will be led at the MD An­der­son Can­cer Cen­ter by Dr. Naveen Pem­mara­ju, an as­sis­tant pro­fes­sor, and Pro­fes­sor Hagop Kan­tar­jian, de­part­ment chair of the De­part­ment of Leukemia, Di­vi­sion of Can­cer Med­i­cine.

Hoang-Sayag called the move “a ma­jor mile­stone not on­ly for the com­pa­ny but al­so for the med­ical com­mu­ni­ty, glob­al biotech and phar­ma­ceu­ti­cal in­dus­tries at large. Cel­lec­tis’ al­lo­gene­ic UCART prod­ucts have the po­ten­tial to cre­ate an im­por­tant shift with re­gard to avail­abil­i­ty, and cost-ef­fec­tive­ness, to make these ther­a­pies wide­ly ac­ces­si­ble to pa­tient pop­u­la­tion across the world.”

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Paul Hastings, Nkarta CEO

With no up­front pay­ment or mile­stones on the line, Nkar­ta and CRISPR join forces on CAR-NK search

Most deals in biotech come with hefty upfront payments attached, and the promise of big biobucks if a program works out. Not this one.

Nkarta has struck what CEO Paul Hastings calls a “real collaboration” with CRISPR Therapeutics to co-develop and commercialize two CAR-NK therapies, in addition to an NK+T program. The duo will split all R&D costs — and any worldwide profits — 50/50, Hastings said.

Brent Saunders (Richard Drew, AP Images)

OcuWho? Star deal­mak­er turned aes­thet­ics czar Brent Saun­ders flips back in­to biotech. But who’s he team­ing up with now?

Brent Saunders went on a tear of headline-blazing deals building Allergan, merging and rearranging a variety of big companies into one before an M&A pact with Pfizer blew up and sent him on a bout of biotech drug deals. That didn’t work so well, so under pressure, he got his buyout at AbbVie — which needed a big franchise like Botox. And it was no big surprise to see him riding the SPAC wave into a recent $1 billion-plus deal that left him in the executive chairman’s seat at an aesthetics outfit — now redubbed The Beauty Health Company — holding a big chunk of the equity.

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Drug pric­ing watch­dog joins the cho­rus of crit­ics on Bio­gen's ad­u­canum­ab: What about charg­ing $2,560 per year?

As if Biogen’s aducanumab isn’t controversial enough, the researchers at drug pricing watchdog ICER have drawn up the contours of a new debate: If the therapy does get approved for Alzheimer’s by June, what price should it command?

Their answer: At most $8,290 per year — and perhaps as little as $2,560.

Even at the top of the range, the proposed price is a fraction of the $50,000 that Wall Street has reportedly come to expect (although RBC analyst Brian Abrahams puts the consensus figure at $11.5K). With critics, including experts on the FDA’s advisory committee, making their fierce opposition to aducanumab’s approval loud and clear, the pricing pressure adds one extra wrinkle Biogen CEO Michel Vounatsos doesn’t need as he orders full-steam preparation for a launch.

'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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