#ASH17: The big los­er in CAR-T, Juno is mak­ing a bid to seize the fast lane to fron­trun­ner sta­tus

Juno Ther­a­peu­tics $JUNO may be play­ing catchup in CAR-T. But if the lat­est cut of its 3-month DL­B­CL da­ta at the top dose holds up, the biotech won’t be play­ing for sec­ond — or third — place.

In their ab­stract out for ASH this morn­ing, Juno ex­ecs spelled out a key piece of da­ta for the high dose arm of the ear­ly study on JCAR017. Ze­ro­ing in on that one snap­shot, re­searchers say they tracked an 80% over­all re­sponse rate and a 73% com­plete re­sponse rate at 3 months for the high dose among a “piv­otal core” group of 15 pa­tients.

Gilead’s Kite and No­var­tis saw a slight de­cline in the CR rates to the low 30s go­ing from 3 to 6 months, leav­ing Juno a win­dow for con­sid­er­able im­prove­ment.

To be sure, Juno still has a long way to go af­ter its lead JCAR015 proved to be a lethal, tox­ic dis­as­ter in what was in­tend­ed to be a piv­otal study. But the biotech isn’t at all con­tent to be re­main­dered to the sec­tion of the in­dus­try that counts the num­ber of pa­tients killed in a failed study. In­stead, Juno be­lieves that its fol­low-up pro­gram will prove that the big sums gam­bled on its tech­nol­o­gy will show that they came up with a bet­ter CAR-T, with a more ef­fec­tive man­u­fac­tur­ing process and a safer pro­file that will ul­ti­mate­ly carve out a big place for it­self in the mar­ket.

In­vestors liked what they were see­ing, dri­ving up Juno’s shares by 10% this morn­ing.

Sunil Agar­w­al

Every­thing is rid­ing on this one. There won’t be much chance for a third shot at suc­cess.

What’s dif­fer­ent this time?

“The anal­o­gy I’ll give you,” says R&D chief Sunil Agar­w­al: “Small changes in the an­ti­body world can make big dif­fer­ences; all CARs are not the same.”

There’s more to come at ASH, he adds, where they can re­view the re­sponse for a big­ger group of pa­tients. But the es­sen­tial ef­fi­ca­cy and safe­ty pro­file on dis­play to­day, he adds, isn’t go­ing to change.

Says Agar­w­al: “I think these da­ta con­tin­ue to sup­port a best-in-class pro­file.”

For one, there’s the switch to the 4-1BB cos­tim­u­la­to­ry do­main, which al­lows for a slow and steady ac­ti­va­tion that sets the foun­da­tion for a more durable re­sponse.

Hans Bish­op

But that’s a fea­ture that No­var­tis’ CAR-T shares as well. Juno CEO Hans Bish­op tells me the “pre­cise pro­duc­tion” val­ues used now in mak­ing the ther­a­py from cells ex­tract­ed from pa­tients is just far more pre­cise.

“We know CD4 and CD8 cells act dif­fer­ent, CD8 cells are more po­tent,” says Bish­op. Get­ting the right mix with a mea­sured ex­pan­sion of cells now is part of a pre­cise­ly arranged pro­duc­tion recipe that dis­tin­guish­es JCAR017 from the rest, he as­serts.

Bish­op isn’t talk­ing ex­act pric­ing yet, and won’t un­til much lat­er in the game. But he does want peo­ple to un­der­stand that to be com­pet­i­tive here in­volves beat­ing out some steep ex­ist­ing costs in treat­ing DL­B­CL or ALL. Safe­ty, he says, will play a big role in that.

The lat­est up­date on the da­ta re­mains with 1 pa­tient suf­fer­ing from cy­tokine re­lease syn­drome, and 14% with neu­ro­tox­i­c­i­ty run­ning from Grade 1 to Grade 4. That’s not a per­fect score, by any means, but in this world Bish­op feels the num­bers give Juno an ad­van­tage over worse is­sues with the mar­ket lead­ers.

Close to two thirds of the pa­tients in their study nev­er ex­hib­it­ed any signs of tox­i­c­i­ty, ei­ther cy­tokine re­lease syn­drome or neu­ro­tox­i­c­i­ty, which ul­ti­mate­ly de­stroyed Juno’s lead ther­a­py, JCAR015, af­ter it killed 5 pa­tients. In this world, any tox, Grade 1 or above, earns pa­tients a one-way trip to the hos­pi­tal, which doesn’t come cheap. By avoid­ing tox, Juno hopes to prove that most pa­tients can be treat­ed in an out­pa­tient set­ting, vast­ly re­duc­ing their over­all cost — which some ex­perts say may well range from $1 mil­lion to $1.5 mil­lion, all in.

It’s a com­pelling ar­gu­ment, and one that Juno has care­ful­ly craft­ed af­ter one of the worst clin­i­cal set­backs in re­cent his­to­ry. Their R&D work here will be care­ful­ly scru­ti­nized at every step. To get on to the mar­ket, they’ll have to pass muster at the hands of a group of reg­u­la­tors em­bar­rassed by their abrupt and in­ex­plic­a­ble de­ci­sion to lift the orig­i­nal hold on JCAR015 af­ter just a few days — al­low­ing more pa­tients to die.

Juno has a high bar to clear, but they’re tak­ing a run­ning leap at it.

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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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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'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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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.

Biden ad­min­is­tra­tion backs a po­lar­iz­ing pro­pos­al to waive IP for all Covid-19 vac­cines

In a surprise U-turn, the Biden administration said Wednesday that it will support a proposal at the World Trade Organization to temporarily waive intellectual property protections on Covid-19 vaccines.

The proposal, backed by South Africa and India at the WTO, seeks to help developing countries with limited vaccine supplies. The US and Europe historically opposed the proposal, saying IP should be protected because it incentivizes new drug and vaccine development.

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