Back when Long­wood un­veiled its lat­est im­munother­a­py start­up, Im­mu­ni­tas, the VC’s co-founder Lea Hachi­gian talked at length about how its sin­gle-cell ge­nomics analy­sis plat­form could en­able re­searchers to “ask ques­tions of the im­mune sys­tem we weren’t able to ask be­fore,” un­der­stand hu­man bi­ol­o­gy on a much deep­er lev­el and iden­ti­fy tar­gets that no­body else was work­ing on. They just couldn’t dis­close the pre­cise tar­get for the lead pro­gram.

Kai Wucherpfen­nig

This Feb­ru­ary, Im­mu­ni­tas fi­nal­ly did — pub­lish­ing a pa­per in Cell that ze­roes in on CD161 as a re­cep­tor on T and NK cells that sup­press­es their abil­i­ty to kill glioma cells.

Months lat­er, the start­up is back with $58 mil­lion in fresh cash to steer a CD161-block­ing an­ti­body to­ward the clin­ic while fu­el­ing a slate of oth­er pro­grams be­hind it. The goal, Im­mu­ni­tas CEO Jef­frey Gold­berg told End­points News, is to keep ex­pand­ing the pipeline at a rate of about one pro­gram per year.

Kai Wucherpfen­nig, chair of Dana-Far­ber Can­cer In­sti­tute’s de­part­ment of can­cer im­munol­o­gy and vi­rol­o­gy, co-found­ed Im­mu­ni­tas with Aviv Regev (be­fore Roche’s Genen­tech re­cruit­ed her to run R&D), Dane Wit­trup of MIT and Mass Gen­er­al’s Mario Su­và.

For Wucherpfen­nig, the dis­cov­ery of CD161 was a tes­ta­ment to the key ad­van­tages of the sin­gle-cell plat­form he helped in­vent, which start­ed out an­a­lyz­ing hun­dreds of cells but can now work with hun­dreds of thou­sands at a time, at high res­o­lu­tion. Through iso­lat­ing T cells from pa­tients’ tu­mor sam­ples (sep­a­rate from, say, stro­mal cells, tu­mor cells, and so on), clus­ter­ing them by gene ex­pres­sion and then ex­am­in­ing dif­fer­ent pop­u­la­tions, his team es­sen­tial­ly came up with an at­las of im­mune cells in glioma — all based on hu­man sam­ples.

“We’ve been asked mul­ti­ple times, why do oth­er peo­ple not pick it up? And the rea­son ac­tu­al­ly is that the bi­ol­o­gy is very dif­fer­ent be­tween mice and hu­mans,” he said. “Most peo­ple start with mouse mod­els, and then in mice there are ac­tu­al­ly mul­ti­ple genes that en­code CD161 like mol­e­cules. Some of them are in­hibito­ry, some just are ac­ti­vat­ing re­cep­tors, and the bi­ol­o­gy is very con­fus­ing. And in hu­mans it’s ac­tu­al­ly more straight­for­ward. There’s a sin­gle gene that en­codes an in­hibito­ry re­cep­tor, OK. And so most peo­ple have looked at this — I know some peo­ple have looked at this and said, nev­er mind. And they nev­er re­al­ly looked at the hu­man bi­ol­o­gy.”

As Im­mu­ni­tas — now grown to 20-plus staffers — car­ried on the work, once again re­ly­ing on the sin­gle-cell se­quenc­ing and an­a­lyt­ics tech, they found out that the re­cep­tor is not just as­so­ci­at­ed with glioma. CD161 ap­pears to be in play in T and NK cell in­ter­ac­tions with mul­ti­ple can­cer types span­ning sol­id and liq­uid tu­mors, giv­ing the biotech mul­ti­ple op­tions to ex­plore in the first hu­man tri­als, with an IND slat­ed for the first half of next year.

Specif­i­cal­ly, Im­mu­ni­tas’ IMT-009 is de­signed to bind to CD161 on T and NK cells and pre­vent it from in­ter­act­ing with CLEC2D on tu­mor cells, there­by restor­ing the im­mune cells’ can­cer-killing pow­er.

Wucherpfen­nig and Gold­berg fig­ure there are many more im­muno-on­col­o­gy tar­gets out there sim­i­lar­ly lurk­ing in cor­ners tra­di­tion­al dis­cov­ery meth­ods can’t un­cov­er.

The promise has drawn a siz­able syn­di­cate. Agent Cap­i­tal led the round, which fea­tured Med­ical Ex­cel­lence Cap­i­tal, 120 Cap­i­tal, So­las­ta Ven­tures, Mi­rae As­set, Ono Ven­ture In­vest­ment, The Mark Foun­da­tion for Can­cer Re­search, NS In­vest­ment, Bright­Edge (Amer­i­can Can­cer So­ci­ety) and the Leukemia & Lym­phoma So­ci­ety Ther­a­py Ac­cel­er­a­tion Pro­gram. Ex­ist­ing back­ers al­so re­turned: They are Alexan­dria Ven­ture In­vest­ments, Evotec, Leaps by Bay­er, M Ven­tures, No­var­tis Ven­ture Fund and, of course, found­ing in­vestor Long­wood.

Be­yond that, the sci­en­tist al­so sees the up­com­ing work as part of a big­ger ex­per­i­ment to test the un­der­ly­ing plat­form as a com­pre­hen­sive tool to close the loop on trans­la­tion.

“We’re al­so think­ing about how we can use sin­gle cell tech­nolo­gies to ac­tu­al­ly un­der­stand what the drug does in pa­tients — you know, once we start a clin­i­cal tri­al,” he said. “There ob­vi­ous­ly are some lo­gis­ti­cal chal­lenges with sam­ple pro­cure­ment, et cetera, but it could ac­tu­al­ly be an ex­cit­ing op­por­tu­ni­ty. So we — we use sin­gle cell tech­nolo­gies to dis­cov­er it, we use sin­gle cell tech­nolo­gies to de­fine in­di­ca­tions, and then al­so study mech­a­nism of ac­tion in peo­ple.”

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.