CRI re­port tracks ex­plo­sive growth of cell ther­a­pies in the glob­al pipeline, as Chi­na re­searchers mus­cle to­ward the front

The Can­cer Re­search In­sti­tute is shin­ing a light on the ex­plo­sive growth of cell ther­a­pies in the clin­ic, spot­light­ing Chi­na’s vir­tu­al overnight suc­cess in grab­bing a lead role in the field. And they’re ask­ing the world’s can­cer drug de­vel­op­ment com­pa­nies to adopt a new ap­proach that will help the in­dus­try man­age a fi­nite num­ber of po­ten­tial tri­al pa­tients while point­ing re­searchers to the most like­ly av­enue for do­ing some­thing im­por­tant — rather than just frit­ter­ing away bad­ly need­ed re­sources.

The non­prof­it CRI re­cent­ly quan­ti­fied the pro­lif­er­a­tion of I/O agents in the clin­ic, cap­tur­ing our at­ten­tion with the news that 164 PD-1/L1 ther­a­pies were in the pipeline, look­ing to whit­tle away at the block­buster lead­ers from Mer­ck and Bris­tol-My­ers Squibb. Now we learn from an up­date in Na­ture Re­views Drug Dis­cov­ery — penned by a team that in­cludes Jill O’Don­nell‐Tormey and Aiman Sha­l­abi — that they tracked down 753 cell ther­a­pies, with 375 in the clin­ic.

That fig­ure is up 87% in just 6 months, with 350 added to the pipeline around the world.

The tal­ly re­veals that “378 are in pre­clin­i­cal phase, 160 in phase I, 205 in phase II, 6 in phase II and 4 ap­proved (2 in the US, 1 in South Ko­rea and 1 in Italy),” re­ports the CRI. “In­ter­est­ing­ly, 404 of the 753 agents are chimeric anti­gen re­cep­tor (CAR) T cell ther­a­pies.”

Not sur­pris­ing­ly, the US is in the clear lead here, with 344 pro­grams. But Chi­na is num­ber two, and catch­ing up fast, with 203. In a field where aca­d­e­m­ic cen­ters fre­quent­ly play a lead role in demon­strat­ing their po­ten­tial, 125 of Chi­na’s pro­grams — 62% — are in the hands of aca­d­e­mics.

Here’s a look at the glob­al play­ing field:

In the mean­time, it’s get­ting rapid­ly eas­i­er to get these ther­a­pies pro­duced for clin­i­cal tri­als vir­tu­al­ly any­where. “(W)ith the rapid ad­vance­ment of lo­cal cell pro­duc­tion tech­nolo­gies, such as Clin­i­MACS Prodi­gy (Mil­tenyi Biotec), Co­coon (Lon­za) and Bea­con (Berke­ley Lights), we an­tic­i­pate a low en­try hur­dle for lo­cal man­u­fac­ture of cells for in­ves­ti­ga­tion­al clin­i­cal use.”

There are 113 tar­gets on the radar in this field, with 73 in the clin­ic. CD19 rules the roost, for now, with BC­MA a grow­ing but still rel­a­tive­ly small seg­ment. Here’s a look at the top 30:

The boom­ing on­col­o­gy re­search field has de­liv­ered some im­por­tant gains in the last two years. But at this rate, the gold rush for ad­vanc­ing new drugs is spurring the CRI to out­line some bad­ly need­ed re­forms. Here are three:

  • They’re ask­ing the R&D ex­ecs here to shift more of their fo­cus to in­tro­duc­ing ge­net­ic con­structs in­to cells, rather than con­cen­trate on vi­ral vec­tors, where a pro­duc­tion bot­tle­neck has de­vel­oped.
  • Rather than squan­der the lim­it­ed ac­cess to pa­tient vol­un­teers on a mas­sive num­ber of new stud­ies, they want de­vel­op­ers to move to adap­tive tri­al de­signs that ac­com­mo­date mul­ti­ple drugs and tar­gets at once.
  • And with the big push on to ex­pand in­to sol­id tu­mors, CRI wants de­vel­op­ers to fo­cus on the most im­por­tant tar­gets not cur­rent­ly be­ing treat­ed well with ex­ist­ing drugs.

There’s no R&D czar in the world, though, that can de­mand dis­ci­pline in this field. Large play­ers have been re­sis­tant to be­ing told what to do as much as the small­er play­ers. And every­one wants to con­trol their own eco­nom­ics. The mar­ket is dri­ving the growth of the field, but it can’t triage how it grows — un­less in­vestors start hold­ing back funds in search of more com­mon sense.

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

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I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

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Re­gen­eron's Evkeeza shows promise in curb­ing high triglyc­erides, but will ge­net­ic dis­par­i­ties lim­it use?

When Regeneron scored an early approval for lipid lowering antibody Evkeeza back in February, the drugmaker cracked open a new pathway to lower abnormally high cholesterol levels. Now, Regeneron is chasing high triglycerides as well with some promising mid-stage data — but will genetic restrictions limit the drug’s use?

Regeneron’s Evkeeza (evinacumab) cut median triglyceride levels by more than 800 mg/dL (57%) in patients with a rare disorder causing abnormally high triglyceride levels compared with an overall increase of 50 mg/dL (1.8%) in participants on placebo, according to Phase II data presented Sunday at the virtual American College of Cardiology meeting.

Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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As­traZeneca's Farx­i­ga missed big on Covid-19 study, but it's tak­ing SGLT2 safe­ty da­ta as a sil­ver lin­ing

AstraZeneca hasn’t seen many setbacks in recent months for SGLT2 inhibitor Farxiga, which broke ground in heart failure and kidney disease regardless of diabetes diagnosis. But the British drugmaker had to admit defeat in taking Farxiga into Covid-19, but follow-up results add a bit of a silver lining to that trial’s safety data.

Of hospitalized Covid-19 patients dosed with AstraZeneca’s Farxiga, 11.2% experienced an organ failure or died after 30 days of therapy compared with 13.8% of those given placebo, according to follow-up data from the DARE-19 study revealed Sunday at the virtual American College of Cardiology meeting.

Pfiz­er, Bris­tol My­er­s' Eliquis flops in post-heart surgery pa­tients, spurring an 'un­ex­plained sig­nal' in cer­tain deaths

Pfizer and Bristol Myers Squibb’s non-warfarin blood thinner Eliquis has raced out to become the most prescribed drug of its class on the market — even overtaking warfarin’s long-time lead. But in tricky-to-treat patients after a valve replacement, an investigator-sponsored study couldn’t turn up benefit and raised a troubling safety signal.

Eliquis failed to show benefit over standard of care in preventing serious clinical outcomes after a transaortic valve replacement (TAVR) and was linked to an “unexplained signal” in a subset of populations with a higher rate of non-CV deaths who did not need blood thinners apart from the surgery, according to data presented Saturday at the virtual American College of Cardiology meeting.

Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.