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.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Take­da swoops in to buy lit­tle biotech part­ner and its celi­ac drug poised to 'change stan­dard of care'

Having spent three years carefully grooming PvP Biologics and its drug for celiac disease, Takeda is happy enough with the proof-of-concept data to buy it all.

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Bio­gen touts new ev­i­dence from the gene ther­a­py com­pa­ny it wa­gered $800M on

A year ago, Biogen made a big bet on a small gene therapy company. Now they have new evidence one of their therapies could work.

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Mi­cro­bio­me Q&A: New study maps the vagi­na's 'op­ti­mal mi­cro­bio­ta' — and its im­pli­ca­tions for bio­phar­ma

The widely-held notion that the “optimal” vaginal microbiota is dominated by one strain of lactic-acid producing bacteria has now been challenged in a new paper, published in Nature Communications on Wednesday, which used advanced gene sequencing methods to map out the most comprehensive gene catalog of the human vaginal microbiome.

Things have changed in the more than 50 years since the concept of vaginal microbiota transplants was proposed and subsequently tainted by a Texas-based gynecologist who transplanted the vaginal fluid of women who had bacterial vaginosis into healthy females, suspecting he had isolated the bacteria responsible for the condition.

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In at least one life-sci hub, gen­der and di­ver­si­ty ini­tia­tives haven’t made a dent

Gender and racial diversity at the top of UK life science companies has hardly budged over the last seven years despite repeated advocacy efforts, according to a new report.

The report, from the recruiting firm Liftstream, found that 14.8% of directors on life sciences boards were women and 21.1% of top executives were women. That’s a modest bump from the 9.8% of directors and 18.1% of executives Liftstream identified in their last report from 2014. The percentage of women CEOs moved from 8% to 9.8%.

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Will a 'risk-of­f' mind­set has­ten cell ther­a­py M&A? Io­vance surges on buy­out chat­ter

Is it time for some cell therapy M&A?

Investors of Iovance Biotherapeutics certainly thought so, sending its stock $IOVA up as much as 40% after Bloomberg reported that the cancer-focused biotech is talking to potential buyers.

While 2019 saw a number of high-profile gene therapy company takeovers — led by Roche’s $4.3 billion bid of Spark as Astellas went for Audentes, Biogen snapped up Nightstar and Vertex absorbed Exonics — large players appeared to prefer partnering on the cell therapy front, particularly when it comes to cancer. Hal Barron put his weight behind Rick Klausner’s startup as he rebuilt GlaxoSmithKline’s cancer pipeline. Takeda turned to MD Anderson to license their natural killer cell therapy.

One less ri­val for Im­muno­vant, as Alex­ion aban­dons FcRn in­hibitor

Less than one year after Alexion parted with $25 million upfront to secure access to a second anti-FcRn asset, it is abandoning the experimental drug. The discontinuation, disclosed at the SVB Leerink Global Healthcare Conference in New York during a fireside chat, bodes well for rival Immunovant.

The drug (ABY-039), partnered for development with Sweden’s Affibody, was forsaken on the basis of early-stage data that was not viewed favorably, Baird and SVB Leerink analysts noted.

Clin­i­cal tri­al spon­sors have to dis­close decade’s worth of un­re­leased da­ta, fed­er­al judge rules

A decade’s worth of unreleased trial data may soon see the light of day.

A New York federal judge ruled this week that the FDA and the NIH have for years misinterpreted a law that would require companies, universities and other clinical trial sponsors to release trial data from studies completed between 2007 and 2017. The ruling covers drugs and medical devices that were experimental when the study was completed but have since been approved, potentially putting hundreds of sponsors out of compliance if they don’t put their results on clinicaltrials.gov.

Laurie Glimcher and Ansbert Gadicke (Justin Knight, Dana-Farber Cancer Institute)

Ty­ing ba­sic sci­ence to spin­outs, Dana-Far­ber de­buts sis­ter funds to­tal­ing $126M with MPM Cap­i­tal

As one of the most prestigious cancer institutes in the US, Dana-Farber has enjoyed considerable support for its entrepreneurial pursuits, spinning out about 30 companies in the past 12 years.

“Now where we’ve always struggled — where every cancer center struggled — is support of basic science,” Barrett Rollins, chief scientific officer emeritus, told Endpoints News.

And then two of its trustees had an idea. What if they tied philanthropy to investment in Dana-Farber startups, requiring a donation to basic science as a condition for accessing its brightest biotech venture ideas?