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.

Achiev­ing Dig­i­tal Trans­for­ma­tion: Un­lock­ing Cost Re­duc­tion, Clin­i­cal Ex­cel­lence & Pre­ci­sion Ther­a­peu­tics Man­u­fac­tur­ing

In the dynamic landscape of pharmaceutical and biotech industries, L7 Informatics is leading the charge in transforming traditional operations through digital innovation. With a firm focus on cost reduction, clinical improvements, reducing tech-transfer times, L7 Informatics is revolutionizing the way companies research, develop, and manufacture while also prioritizing the well-being of patients and providers thereby paving the way for a future where patients receive better treatments at lower costs.

End­points 20(+2) un­der 40, 2023; Bio­phar­ma's high­est-paid CEOs; N-of-1 CRISPR sto­ry goes on af­ter tragedy; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

We will be off Monday in observance of Memorial Day — and when we get back, it will be a straight march to ASCO, BIO and more. Enjoy the (long) weekend!

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,200+ biopharma pros reading Endpoints daily — and it's free.

Douglas Love, Annexon CEO

An­nex­on’s GA drug miss­es on pri­ma­ry goal but win on vi­su­al acu­ity will be fo­cus of planned late-stage tri­al

Annexon’s complement inhibitor didn’t prove better than sham at reducing lesion growth in a leading cause of blindness, but the biotech still plans to move forward on the back of secondary endpoints showing visual acuity preservation, which will “certainly” be the primary goal in a late-stage trial to be discussed shortly with the FDA, CEO Douglas Love told Endpoints News. 

The California biotech’s ANX007 was not statistically significant compared to pooled sham, the comparator, at 12 months in patients with geographic atrophy, per a Wednesday presentation. In every-month dosing, the GA lesion area changed about 6.2% from baseline (p=0.526) and 1.3% (p=0.896) in the every-other-month group. In a March note, Jefferies analyst Suji Jeong said a reduction of 20% to 30% would be “encouraging.”

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Teresa Bitetti, Takeda's president of the global oncology business unit

Take­da wins pri­or­i­ty re­view for $400M col­orec­tal can­cer drug, li­censed from Hutchmed in Jan­u­ary

Takeda and Hutchmed scored a priority review Thursday afternoon for a colorectal cancer drug, the companies announced.

The experimental drug in question is fruquintinib, previously approved in China in 2018 to treat metastatic colorectal cancer. Takeda and Hutchmed are aiming to bring fruquintinib to the US and other countries outside China in the same indication, and the FDA set its decision date for Nov. 30 of this year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,200+ biopharma pros reading Endpoints daily — and it's free.

Rich Horgan (R) with his late brother, Terry

Rich Hor­gan spear­head­ed a gene ther­a­py for his broth­er. The tri­al end­ed in tragedy, but the work con­tin­ues for more pa­tients

Rich Horgan’s quest to create a custom gene therapy for his brother, Terry, ended in tragedy. But Horgan doesn’t believe it’s the end of the story.

Terry, a 27-year-old patient with Duchenne muscular dystrophy, died last October just eight days after receiving the therapy in a clinical trial in which he was the only participant. The case raised questions about the safety of certain gene therapies and what would happen to other drug programs under a nonprofit that Horgan created, called Cure Rare Disease.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,200+ biopharma pros reading Endpoints daily — and it's free.

Bio­phar­ma's 20 high­est-paid CEOs of 2022, each bring­ing in $20M+ pay­days

Even in a down year for much of the biopharma market, 20 CEOs brought in pay packages valued at more than $20 million, an Endpoints News analysis found.

Endpoints collected data on more than 350 CEO compensation packages, covering a wide range of pharma, biotech, and life sciences companies. All told, the 20 largest earners made over $725 million in 2022 — an average package of $36.4 million. Three brought in paydays over $50 million, and one CEO broke the $100 million mark.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,200+ biopharma pros reading Endpoints daily — and it's free.

The 20(+2) un­der 40: Your guide to the next gen­er­a­tion of biotech lead­ers

This year’s list of 20 biotech leaders under the age of 40 includes a huge range of ambitions. Some of our honorees are planning to create the next big drug giant. Others are pushing the bounds of AI. One is working to revolutionize TB testing. All are compelling talents who are still young in age, but already far along in achievement.

And, as in years past, we went over. The 20 are actually 22 because of two double profiles that reflect how important teamwork is in the industry. As one of our honorees, Joe Illingworth of DJS Antibodies, told me in our interview, “It takes a village to raise a biotech.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,200+ biopharma pros reading Endpoints daily — and it's free.

FDA ap­proves Lex­i­con’s heart-fail­ure drug af­ter de­feat in di­a­betes

The FDA on Friday approved Lexicon’s heart failure drug sotagliflozin following a string of setbacks for the pharma company, including an FDA rejection in diabetes and the loss of a development deal with Sanofi.

The dual SGLT1 and SGLT2 inhibitor will be marketed as Inpefa and is a once-daily tablet. It’s been approved to reduce the risk of cardiovascular death and heart failure-related hospitalization or urgent visits in adults with heart failure or type 2 diabetes mellitus, chronic kidney disease, and other cardiovascular risk factors. The label spans the range of left ventricular ejection fraction, including preserved ejection fraction and reduced ejection fraction, as well as patients with or without diabetes, Lexicon said Friday.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,200+ biopharma pros reading Endpoints daily — and it's free.

Eu­ro­pean Com­mis­sion to re­ceive few­er Pfiz­er-BioN­Tech vac­cine dos­es un­der amend­ed con­tract

The European Commission has made a few changes to its vaccine contract with Pfizer and BioNTech, reducing the dose volume while extending the delivery timeline to cope with “evolving public health needs.”

The Commission previously struck a contract in May 2021 for 900 million doses, with the option to purchase another 900 million. Of those, 450 million were expected to be delivered in 2023, though an amendment now calls for fewer doses. While neither the Commission nor Pfizer and BioNTech have revealed an exact amount, an unnamed source told Reuters that the amendment reduces the remaining expected doses by about a third.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 170,200+ biopharma pros reading Endpoints daily — and it's free.