Kevin Boyle, Kuur

Months af­ter a ma­jor re­brand­ing around CAR-NKT cells, Ku­ur Ther­a­peu­tics is of­fer­ing a peek at ear­ly re­sults

Sev­er­al months af­ter a name change and ma­jor re­struc­tur­ing, Ku­ur Ther­a­peu­tics has of­fered an ear­ly glimpse at re­sults from a small num­ber of pa­tients treat­ed with their CAR-NKT cell ther­a­pies de­vel­oped with the Bay­lor Col­lege of Med­i­cine.

Out of 10 evalu­able neu­rob­las­toma pa­tients dosed with Ku­ur’s lead can­di­date, an au­tol­o­gous GD2-tar­get­ing CAR-NKT ther­a­py, re­searchers not­ed one com­plete re­sponse, one par­tial re­sponse, and three pa­tients with sta­ble dis­ease, ac­cord­ing to the Hous­ton-based biotech.

The pa­tient with a com­plete re­sponse had pre­vi­ous­ly achieved a par­tial re­sponse be­fore re­ceiv­ing a sub­se­quent dose of the drug. The drug ap­peared safe, ac­cord­ing to Ku­ur, with one case of Grade 2 cy­tokine re­lease syn­drome (CRS) oc­cur­ring in the Phase I tri­al, dubbed GI­NAKIT2.

“We have some very good ear­ly signs of NKT cells be­ing safe, hom­ing to the site of tu­mor, which is very im­por­tant go­ing af­ter sol­id tu­mors … and now we’re show­ing signs of ef­fi­ca­cy as well,” CEO Kevin Boyle told End­points News. “So, based on that, we want the com­pa­ny to be fo­cused on just this plat­form.”

The drug con­sists of nat­ur­al killer T cells en­gi­neered with a CAR tar­get­ing GD2, a ma­jor gan­glio­side ex­pressed on many neu­rob­las­toma tu­mors. It’s de­signed to se­crete IL-15, which has been shown to bol­ster the ef­fi­ca­cy and per­sis­tence of CAR-NKT cells in the im­muno­sup­pres­sive tu­mor mi­croen­vi­ron­ment.

NKT cells are rare lym­pho­cytes that aren’t fre­quent­ly found in the pe­riph­er­al blood, and as a re­sult, re­searchers tend not to work with them, Boyle ex­plained. The sci­en­tist lead­ing Bay­lor’s re­la­tion­ship with Ku­ur has been work­ing with the cells for two decades, he said.

Kurt Gunter

“One rea­son that we like this cell type is it homes nat­u­ral­ly to the tis­sue in sol­id tu­mors… It al­so has an in­vari­ant T-cell re­cep­tor… and that means it’s the same in every­one so it won’t cause graft-ver­sus-host dis­ease when we use it in an­oth­er per­son as off-the-shelf ther­a­py. And that means we don’t need to gene ed­it the TCR and that puts us in a fun­da­men­tal­ly dif­fer­ent po­si­tion from the peo­ple work­ing with CAR-T cells,” CMO Kurt Gunter said.

Ku­ur’s sec­ond can­di­date, an off-the-shelf CD19-di­rect­ed CAR-NKT ther­a­py, achieved a PR and CR in the two pa­tients with hema­to­log­i­cal ma­lig­nan­cies who were evalu­able in the Phase I study, AN­CHOR. The re­searchers saw no CRS cas­es, and no ev­i­dence of graft ver­sus host dis­ease (GvHD) — a con­di­tion where donor cells at­tack their host, the com­pa­ny said.

Boyle, who took the helm last March dur­ing a ma­jor re­struc­tur­ing, ex­pects to read out full da­ta from both the Phase I stud­ies next year. And if all goes well, he’s plan­ning to launch a Phase II ex­pan­sion tri­al of the AN­CHOR study in 2021.

Un­til last March, Ku­ur went by the name Cell Med­ica. The Lon­don-based up­start had lo­ca­tions across two con­ti­nents and three coun­tries, Boyle said. But in­stead of work­ing on a va­ri­ety of cell ther­a­pies, the com­pa­ny chose to laser-fo­cus on the CAR-NKT pro­gram un­der an ex­clu­sive li­cense with Bay­lor. It set­tled on one lo­ca­tion in Hous­ton, right next door to their col­lab­o­ra­tors, and for­mer CEO Chris Now­ers was re­placed by Boyle, who had pre­vi­ous­ly served as CFO.

The com­pa­ny hopes to close a pri­vate fi­nanc­ing round this quar­ter, Boyle hint­ed, some of which will be used to trans­form the sin­gle-cen­ter stud­ies in­to mul­ti-cen­ter stud­ies.

“The sit­u­a­tion with neu­rob­las­toma is still kind of bleak es­pe­cial­ly for these high risk re­lapsed re­frac­to­ry pa­tients,” Gunter said. “They have gen­er­al sur­vivals of less than one year, once they’ve re­lapsed,” he con­tin­ued, adding that he be­lieves the com­pa­ny can bring a sig­nif­i­cant ben­e­fit to pa­tients.

The top 100 bio­phar­ma VCs, Bob Brad­way places $2B bet in can­cer, gene edit­ing pi­o­neer's new big idea, and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Before diving in, we had some news to share: Endpoints is launching a premium weekly report focusing on all things regulatory. Coverage will be led by our new senior editor, Zachary Brennan, who joins us from POLITICO. Arsalan Arif has more details in his Publisher’s Note.

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Robert Bradway (Photographer: Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Am­gen snaps up can­cer drug play­er Five Prime, adding PhI­II-ready FGFR2b drug in $2B M&A play

Amgen is making a long-awaited move on the M&A side, buying South San Francisco-based Five Prime $FPRX for close to $2 billion and adding a slate of new cancer drugs to the pipeline.

Amgen is paying $38 a share, putting the deal value at $1.9 billion. The stock closed at $21.26 last night, giving investors a 78% premium.

The jewel in the crown of this deal is bemarituzumab, which Amgen describes as a first-in-class, Phase III-ready anti-FGFR2b antibody. Amgen was drawn to the bargaining table by Five Prime’s mid-stage data on gastric cancer, satisfied by PFS and OS data helping to validate FGFR2b as a target. Amgen researchers will now expand on the R&D program in other epithelial cancers, including lung, breast, ovarian and other cancers.

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David Liu (Casey Atkins Photography courtesy Broad Institute)

David Liu has a new big idea: pro­teome edit­ing. It could one day shred tau, RAS and some of the worst dis­ease-caus­ing pro­teins

Before David Liu became famous for inventing new forms of gene editing, he was known around academia in part for a more obscure innovation: a Rube Goldberg-esque system that uses bacteria-infecting viruses to take one protein and turn it into another.

Since 2011, Liu’s lab has used the system, called PACE, to dream up fantastical new proteins: DNA base editors far more powerful than the original; more versatile forms of the gene editor Cas9; insecticides that kill insecticide-resistant bugs; enzymes that slide synthetic amino acids into living organisms. But they struggled throughout to master one of the most common and powerful proteins in the biological world: proteases, a set of Swiss army knife enzymes that cut, cleave or shred other proteins in everything from viruses to humans.

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The 2021 top 100 bio­phar­ma in­vestors: As the pan­dem­ic hit and IPOs boomed, VCs swung in­to ac­tion like nev­er be­fore

The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.

Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.

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Eli Lil­ly claims a TKO in its long-run­ning ti­tle fight with No­vo Nordisk for the block­buster di­a­betes mar­ket — but there’s a hitch

Eli Lilly isn’t just gunning for a better diabetes drug in tirzepatide. They want to cut ahead of Novo Nordisk’s blockbuster rival Ozempic (semaglutide) on the obesity front as well. But a newly-claimed win in a head-to-head Phase III showdown over reducing A1C while shedding pounds — complete with clear evidence of superiority over the approved rival — could prove a tough sell right now.

Let’s start with the latest data from Lilly.

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Hal Barron, Endpoints UKBIO19

GSK, Vir's hopes for a Covid-19 an­ti­body fall flat in NIH 'mas­ter pro­to­col' with no ben­e­fit in hos­pi­tal­ized pa­tients

GlaxoSmithKline and Vir Biotechnology were hopeful that one of their partnered antibodies would carve out a win after getting the invite to a major NIH study in hospitalized Covid-19 patients. But just like Eli Lilly, the pair’s drug couldn’t hit the mark, and now they’ll be left to take a hard look at the game plan.

The NIH has shut down enrollment for GSK and Vir’s antibody VIR-7831 in its late-stage ACTIV-3 trial after the drug showed negligible effect in achieving sustained recovery in hospitalized Covid-19 patients, the partners said Wednesday.

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Eli Lil­ly claims suc­cess in a new JAK in­di­ca­tion: hair loss

Over the last decade, drugmakers have proven JAK inhibitors can treat a smattering of immune-related diseases ranging from rheumatoid arthritis to Covid-19. Now Eli Lilly has pulled out a new one.

Lilly and its biotech partner Incyte announced Wednesday that their JAK inhibitor baricitinib effectively regrew patients’ hair in a Phase III trial for alopecia areata, an autoimmune condition that can cause sudden, severe and patchy hair loss. Lilly didn’t break down the results from the 546-patient trial, but the primary endpoint was improvement on a standard score for alopecia symptoms.

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Bruce Cozadd, Jazz CEO (Jazz Pharmaceuticals)

Jazz CEO Bruce Cozadd cam­paigned for 6 months to buy GW Phar­ma. A 90% pre­mi­um sealed the deal — along with $17.6M in ‘re­ten­tion’ in­cen­tives

Jazz CEO Bruce Cozadd didn’t beat around the bush.

In his first video meeting with GW Pharma chief Justin Gover last July 8, he offered to pay $172 a share to get the company, which had beaten the odds in getting its remarkable cannabinoid drug Epidiolex across the regulatory finish line for epilepsy. GW’s stock closed at $129 that day.

Cozadd had already done his homework on the financing to make sure he could swing it the way he wanted. He just needed to do some due diligence before making the non-binding bid firm.

UP­DAT­ED: Not 3 weeks af­ter tak­ing Hu­ma­cyte pub­lic, Ra­jiv Shuk­la launch­es an­oth­er blank check com­pa­ny

One of biotech’s earliest SPAC investors is back with another blank-check company, less than a month after his last effort announced its intent to merge.

Rajiv Shukla is intending to take a third lucky winner public with Alpha Healthcare Acquisition III, filing to go public Thursday with a $150 million raise penciled in. The move comes just a couple of weeks after Shukla’s second SPAC said it would jump to Nasdaq in tandem with Laura Niklason’s Humacyte in a $255 million new investment.

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