There are 2,004 can­cer im­munother­a­pies crowd­ing in­to the pipeline. Now what?

Here’s a sim­ple set of facts with some com­plex im­pli­ca­tions.

There were 469 new PD-1/L1 can­cer check­point stud­ies launched this year, which re­quire 52,000 pa­tients to ful­ly en­roll all of them.

On the one hand, re­searchers for the non­prof­it Can­cer Re­search In­sti­tute say, that sen­tence un­der­scores the boom in im­munother­a­py that’s been trans­form­ing can­cer treat­ment around the world.

But there’s more.

The ex­plo­sion of pre­clin­i­cal and clin­i­cal-stage pro­grams that has erupt­ed in im­munother­a­pies is rais­ing se­ri­ous ques­tions about the in­trin­sic val­ue of each hu­man study be­ing mount­ed for more than 2,000 I/O agents now in de­vel­op­ment. Is there a more ef­fi­cient way to man­age stud­ies, to get the max­i­mum im­pact from every new tri­al? Can you jus­ti­fy all these tri­als, par­tic­u­lar­ly small, sin­gle-site ef­forts?

Be­fore rais­ing the is­sue, the CRI — which spe­cial­izes in im­munother­a­pies — want­ed to present a clear pic­ture of the scene to every­one in the field.

By as­sign­ing two tu­mor im­mu­nol­o­gists to comb through a va­ri­ety of glob­al tri­al data­bas­es over a year’s time, Aiman Sha­l­abi — CRI’s chief med­ical of­fi­cer and di­rec­tor of the An­na-Maria Kellen Clin­i­cal Ac­cel­er­a­tor — be­lieves that they have, for the first time, es­tab­lished a bird’s eye view of the en­tire im­munother­a­py land­scape span­ning the plan­et, from Shang­hai to Boston. Sha­l­abi jour­neyed to Gene­va to share the re­sults this week­end with the Eu­ro­pean So­ci­ety of Med­ical On­col­o­gy IO meet­ing in Gene­va.

It is stag­ger­ing in scope.

“It’s nev­er been seen be­fore in the drug de­vel­op­ment space or the can­cer space,” says Sha­l­abi, and it’s re­shap­ing can­cer R&D in ways that de­mand some new ap­proach­es to de­vel­op­ment. “It’s time to stop putting new sci­ence on top of the old in­fra­struc­ture and do things dif­fer­ent­ly.”

Do­ing that is go­ing to re­ly on more col­lab­o­ra­tive ef­forts in the in­dus­try and acad­e­mia, and he is spear­head­ing a move to do more of that at the CRI af­ter rais­ing the sub­ject in can­cer R&D cir­cles.

Among the high­lights of the CRI re­port:

— There are 2,004 im­munother­a­py agents in de­vel­op­ment.

— 940 of these I/O ther­a­pies are in clin­i­cal-stage de­vel­op­ment, with 1,064 in the pre­clin­i­cal stage.

— There are 164 PD-1/L1 agents in de­vel­op­ment, with 50 in the clin­ic and 5 on the mar­ket. They have in­spired 1,502 tri­als with 1,105 com­bos.

— 344 are can­cer vac­cines in hu­man stud­ies, and 224 are clin­i­cal-stage cell ther­a­pies.

— There are 69 on­colyt­ic virus­es in clin­i­cal de­vel­op­ment, all in the sec­ond wave be­hind Am­gen’s T-Vec; 95 are pre­clin­i­cal.

— There are 99 T cell tar­get­ed im­munomod­u­la­tors in clin­i­cal de­vel­op­ment, 199 in pre­clin­i­cal de­vel­op­ment.

— There are 165 dif­fer­ent tar­gets be­ing com­bined in check­point stud­ies, with 251 in­clud­ing an an­ti-CT­LA-4 and 170 in­volv­ing chemother­a­pies.

— 0f 1,105 PD-1/L1 drug stud­ies CRI ex­am­ined, 60% are small­er, non-in­dus­try sup­port­ed tri­als.

That last point in par­tic­u­lar at­tract­ed Sha­l­abi’s at­ten­tion. These small­er stud­ies of­ten in­volve aca­d­e­m­ic re­searchers in sin­gle-site tri­als, re­ly­ing on mod­est sup­port from the man­u­fac­tur­er. At a time that each new study in the re­cent wave in­volves dwin­dling num­bers of pa­tients, Sha­l­abi sees two un­der­ly­ing trends: The abil­i­ty to track a ben­e­fit with small­er pa­tient groups, and these sin­gle-site af­fairs that are like­ly to de­liv­er da­ta that will be hard­er to in­ter­pret.

Says Sha­l­abi: “It’s go­ing to be a big chal­lenge to re­cruit and then in­ter­pret them.”

“More and more of these stud­ies are just be­ing de­signed lo­cal­ly; there’s an ap­pear­ance of over­crowd­ing,” he says. “I won­der if these small stud­ies are go­ing to make a con­tri­bu­tion.”

Sha­l­abi be­lieves the da­ta un­der­score the need for more col­lab­o­ra­tions, a greater em­pha­sis on mul­ti-site stud­ies with a more care­ful use of um­brel­la tri­al plat­forms to find faster and more ef­fi­cient means of putting I/O agents through hu­man stud­ies.

There’s one oth­er thing that Sha­l­abi doesn’t doubt.

“What we’ve found is just an un­be­liev­able amount of in­no­va­tion out there,” he says. And it is hav­ing a re­al im­pact on the stan­dard of care in can­cer.

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.

As­traZeneca caps PD-L1/CT­LA-4/chemo com­bo come­back with OS win. Is treme­li­mum­ab fi­nal­ly ready for ap­proval?

AstraZeneca’s closely-watched POSEIDON study continues to be the rare bright spot in its push for an in-house PD-L1/CTLA-4 combo.

Combining Imfinzi and tremelimumab with physicians’ choice of chemotherapy helped patients with stage IV non-small cell lung cancer live longer, the company reported — marking the first time the still-experimental tremelimumab has demonstrated an OS benefit.

For AstraZeneca and CEO Pascal Soriot, the positive readout — which is devoid of numbers — offers much-needed validation for the big bet they made on Imfinzi plus tremelimumab, after the PD-L1/CTLA-4 regimen failed multiple trials in head and neck cancer as well as lung cancer.

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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.