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.

Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

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San Diego cou­ple charged with steal­ing trade se­crets, open­ing Chi­nese biotech as DOJ crack­down con­tin­ues

A San Diego couple has been charged with stealing trade secrets from a US hospital and opening a business based off those secrets in China as the controversial industry-wide crackdown on alleged corporate espionage continues. On the same day, the Department of Justice announced they had arrested Beijing representative Zhongsan Liu for allegedly trying to obtain research visas for government recruiters.

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UP­DAT­ED: Bio­gen pulls the plug on prized IPF drug from $562M+ Stromedix buy­out

One of Biogen’s attempts to branch out has flopped as the biotech scraps a mid-stage program for idiopathic pulmonary fibrosis.

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Warts for the win: Aclar­is' lead drug clears piv­otal study

Aclaris Therapeutics has found a way to get rid of the warts and all.

The company — which earlier this month decided to focus on its arsenal of kinase inhibitors — on Monday unveiled positive data from a pivotal study testing its lead experimental drug for use in common warts.

The drug, A-101, was tested in a 502-patient study called THWART-2 — patients enrolled had one to six warts before qualifying for the trial. Patients either self-administered A-101 topical solution or a vehicle twice a week over a two-month period. A higher proportion of patients on the drug (a potent hydrogen peroxide topical solution) saw their warts disappear at day 60, versus the vehicle (p<0.0001) — meeting the main goal of the study.  Each secondary endpoint also emerged in favor of A-101, the company said.

Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Charles Nichols, LSU School of Medicine

Could psy­che­delics tack­le the obe­si­ty cri­sis? A long­time re­searcher in the field says his lat­est mouse study sug­gests po­ten­tial

Psychedelics have experienced a renaissance in recent years amid a torrent of preclinical and clinical research suggesting it might provide a path to treat mood disorders conventional remedies have only scraped at. Now a preclinical trial from a young biotech suggests at least one psychedelic compound has effects beyond the mind, and — if you believe the still very, very early hype — could provide the first single remedy for some of the main complications of obesity.

Ac­celeron drops a de­vel­op­ment pro­gram as #2 drug fails to spark func­tion­al ben­e­fits in pa­tients with a rare neu­ro­mus­cu­lar ail­ment

Acceleron is scrapping a muscular dystrophy development program underway for its number 2 drug in the pipeline after pouring over some failed mid-stage secondary data.

Gone is the ACE-083 project in patients with facioscapulohumeral muscular dystrophy. Their drug hit the primary endpoint on building muscle but flopped on key secondaries for functional improvements in patients, which execs felt was vital to the drug’s success.