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 dru­ga­bil­i­ty. 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.”

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Janet Woodcock (AP Images)

End­points poll: Janet Wood­cock takes the (in­ter­im) helm at the FDA. And a large ma­jor­i­ty of our read­ers want her to stay there

It’s official: Janet Woodcock is now the acting chief of the FDA.

And — according to an Endpoints poll — most industry readers would like her to stay there, although a significant minority is strongly opposed.

To recap: Joe Biden is reportedly choosing between Woodcock and former deputy FDA commissioner Joshua Sharfstein as his nominee for the permanent position. Given their respective track records, the decision is set to determine the agency’s lodestar for years to come.

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An Endpoints Zoom meeting; and the email header employees will see if your company is a Premium subscriber

What’s next for End­points — and how to sup­port our in­de­pen­dent bio­phar­ma news mis­sion

The firehose of biopharma news is gushing these days.

That’s why broader and deeper is the theme for 2021 at Endpoints. You can expect new coverage outside our core R&D focus, with deeper reporting in some key areas. When John Carroll and I launched Endpoints nearly five years ago, we were wading in waist-high waters. Now we’re a team of 25 full-time staffers (and growing) with plans to cover the flood of biopharma news, Endpoints-style.

Eli Lil­ly's an­ti­body cuts risk of Covid-19 by up to 80% among the most vul­ner­a­ble — but will it have a place next to vac­cines?

Eli Lilly says bamlanivimab lowered the risk of contracting symptomatic Covid-19 in a first-of-its-kind trial involving nursing home residents and staff, paving the way for a new option to protect against the virus.

But how big of an impact it might have, and what role it will play, at a time vaccines are being rolled out to the exact population it is targeting still remains unclear.

Among 965 participants in the study — all of whom tested negative for the coronavirus at baseline — the number of symptomatic cases reported in the bamlanivimab arm was 57% lower than that in the placebo arm (odds ratio 0.43, p=0.00021). In addition to that primary endpoint, all secondary endpoints reached statistical significance.

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Michelle McMurry-Heath, BIO CEO (BIO via YouTube)

BIO looks to re­struc­ture, lay­ing off staff amid chal­lenge to the trade org's nor­mal face-to-face style

The biopharma industry, on the whole, had a red-letter year in 2020 amid Covid-19, with fundraising at an all-time high and major players speeding vaccines ahead to approval. But for BIO, the industry’s leading trade organization, the pandemic has prompted a reconsideration of the game plan.

BIO will pivot to digital as the Covid-19 pandemic continues to rage, making “some staff reductions” as it looks to bring its roughly 37,000 in-person meetings each year to the web, the organization said Thursday.

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Hal Barron, GSK R&D chief (GSK via YouTube)

Glax­o­SmithK­line's $4B bis­pe­cif­ic can­cer drug al­liance with Mer­ck KGaA hit by big set­back with a PhI­II fail­ure on NSCLC

Close to 2 years ago, GSK’s R&D team eagerly agreed to pay up to $4 billion-plus to ally itself with Merck KGaA on a mid-stage bispecific called bintrafusp alfa, which intrigued them with the combination of a TGF-β trap with the anti-PD-L1 mechanism in one fusion protein.

But today the German pharma company says that their lead study on lung cancer was a bust, as independent monitors said there was no reason to believe that the experimental drug — targeting PD-L1/TGF-Beta — could beat Keytruda.

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Covid-19 roundup: Italy won­ders aloud if it can sue Pfiz­er for vac­cine short­falls; Flood, dead­ly fire threat­en As­traZeneca vac­cine plants

As reports crop up that deliveries of Pfizer and BioNTech’s Covid-19 vaccine are being unexpectedly cut, Italy wonders if it can take the vaccine developers to court, according to the Wall Street Journal. 

After its shipment for this week was cut by 29%, the Italian government consulted its attorney general about taking legal action, the WSJ reported. Pfizer and BioNTech had warned the EU and Canada last week that their allocations would be reduced as Pfizer upgrades its Belgium factory. What Italy says it doesn’t appreciate, though, is the short notice.

Robert Habib (file photo)

Once laser-fo­cused on liv­er, Mi­NA takes swing at neu­rol­o­gy with Servier's back­ing

Once focused on hard-to-treat liver diseases, MiNA Therapeutics is joining forces with Servier to engage its small activating RNA technology on another difficult front: neurology.

MiNA and Servier announced a new research alliance Thursday centered around neurological disorders. While the partners are keeping quiet about their targets for now, MiNA CEO Robert Habib vaguely revealed that the first one was nominated by Servier, which has an option over it. MiNA stands to receive up to $266.3 million (€220 million) in an upfront payment and milestones on that target alone, though they declined to break those numbers down any further.

Mike Grey, Plexium chairman (Horizon Therapeutics)

Plex­i­um adds in­dus­try vet Mike Grey to the brain trust with new in­vestor cash fund­ing its pro­tein degra­da­tion play

About 15 months since closing a $28 million Series A, a San Diego protein-degradation upstart returned to the venture well Thursday with an extension of that round and some new hires, including one of the city’s best-connected biotech execs.

Plexium has bagged an additional $35 million in financing, the biotech said, money that will push undisclosed oncology and immuno-oncology programs into the clinic. In addition, longtime industry vet Mike Grey is jumping on as chairman of the board, and two others from Thursday’s leads — Adam Goulburn from Lux Capital and Rob Hopfner from Pivotal BioVentures — joined the board too.