Take­da eyes path­way to nat­ur­al killer ther­a­pies, tee­ing up $100M to part­ner with KSQ for its CRISPR-screened mol­e­cules

The po­ten­tial ap­pli­ca­tions for CRISPR/Cas9 con­tin­ue to grow as drug­mak­ers look for nov­el ways to use the pow­er­ful gene edit­ing plat­form to re­work drug dis­cov­ery and de­vel­op­ment. Japan­ese drug­mak­er Take­da has dab­bled on the gene edit­ing front, and now it’s pair­ing up with a biotech us­ing CRISPR screen­ing to laser in on on­col­o­gy can­di­dates.

Frank Stegmeier

Take­da will shell out $100 mil­lion in up­front cash and pre­clin­i­cal de­vel­op­ment mile­stones to part­ner with Boston-area biotech KSQ Ther­a­peu­tics and its CRISPR-screened nat­ur­al killer cell ther­a­pies. The pact will im­me­di­ate­ly in­clude two T cell pro­grams al­ready iden­ti­fied and val­i­dat­ed, KSQ said, with the op­tion to ex­pand in­to two oth­er tar­get ar­eas.

On top of roy­al­ty rights for sales out­side the US, KSQ will al­so have prof­it shar­ing rights on a prod­uct of its choice in the US, the biotech said. In all, each pro­gram could be el­i­gi­ble for up to $400 mil­lion with down­stream mile­stones con­sid­ered.

The crux of the part­ner­ship will use KSQ’s tech — a CRISPR screen­ing plat­form that pars­es through thou­sands of onco­genes in one go to iden­ti­fy pos­si­ble tar­get ar­eas — and Take­da’s de­vel­op­ment re­sources to bring NK can­di­dates to the clin­ic to tar­get a wide va­ri­ety of can­cers and can­cer­ous tu­mors.

In sep­a­rate in­ter­views with End­points News, ex­ec­u­tives from both com­pa­nies were bull­ish about the pos­si­bil­i­ties the col­lab­o­ra­tion will bring not on­ly to their com­pa­nies, but to the on­go­ing fight to cre­ate sub­stan­tive on­co­log­i­cal ther­a­peu­tics.

“It’s a great val­i­da­tion of our plat­form,” said Frank Stegmeier, KSQ’s chief sci­en­tif­ic of­fi­cer. “I think what I’m par­tic­u­lar­ly ex­cit­ed about is it was very clear from the be­gin­ning of our dis­cus­sions, all the way through the end of the dis­cus­sions (with Take­da), that we have a very aligned ex­cite­ment about the po­ten­tial of next gen­er­a­tion on­col­o­gy drugs for can­cer pa­tients.”

Loïc Vin­cent

Once its CRISPR plat­form be­came op­er­a­tional, it quick­ly be­came clear that KSQ was iden­ti­fy­ing more tar­get ar­eas for on­col­o­gy ther­a­peu­tics than it could pos­si­bly ad­dress on its own, Stegmeier said.

“For the first time, it al­lows us to sys­tem­at­i­cal­ly in­ter­ro­gate the func­tion of all 20,000 genes func­tion­al­ly in dif­fer­ent dis­ease mod­els, and this re­al­ly takes the guess­ing out of drug tar­get dis­cov­ery in a way rather than pick­ing one tar­get,” he said. “We can sys­tem­at­i­cal­ly test the func­tion of all 20,000 genes in par­al­lel.”

What the com­pa­ny need­ed, Stegmeier said, was a part­ner to ac­tu­al­ly de­vel­op the drug can­di­dates af­ter CRISPR dis­cov­ered the most promis­ing tar­get ar­eas — a part­ner that “shares our ex­cite­ment” around the ther­a­peu­tic po­ten­tial of the tar­get ar­eas, but is bet­ter equipped in the drug dis­cov­ery are­na.

En­ter Take­da.

As Loïc Vin­cent, head of Take­da’s on­col­o­gy drug dis­cov­ery unit and im­munol­o­gy unit, tells it, the part­ner­ship was sym­bi­ot­ic from the very be­gin­ning.

“What we are go­ing to try to achieve to­geth­er with KSQ is to ad­vance these two nov­el tar­gets. Put our re­sources that we have at Take­da, put our brains to­geth­er to try to drug those tar­gets that are not low-hang­ing fruit,” Vin­cent said. “This is where we are in­ter­est­ed as a com­pa­ny to in­vest on these tar­gets where there are some chal­lenges in drug­ga­bil­i­ty. But where we can in­crease the chance of try­ing to crack the code on … those tar­gets and bring them in­to the clin­ic.”

There was one key el­e­ment that drew Take­da to part­ner with KSQ, Vin­cent said: CRISPR.

“They have this plat­form up and run­ning where they in­vest­ed a lot in terms of en­er­gy, sci­en­tif­ic foun­da­tion and al­so re­sources. Dur­ing the past years, they were able to not on­ly iden­ti­fy tar­gets, but to put to­geth­er a very com­pelling da­ta pack­age to val­i­date the tar­gets,” he said. “You know, it’s easy to do tar­get iden­ti­fi­ca­tion. It’s much more chal­leng­ing to val­i­date the tar­gets, and then to de­fine what are your top tar­gets and where you should de­vel­op drug dis­cov­ery pro­grams.”

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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So what hap­pened with No­var­tis' gene ther­a­py group? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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Dave Lennon, former president of Novartis Gene Therapies

Zol­gens­ma patent spat brews be­tween No­var­tis and Re­genxbio as top No­var­tis gene ther­a­py ex­ec de­parts

Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

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The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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