Scott Wolchko, Fate Therapeutics CEO

Fate's close­ly watched stem cell-de­rived NK pro­gram rais­es red flag over dura­bil­i­ty of re­sponse

At the cut­ting edge of on­col­o­gy re­search, biotechs like Fate Ther­a­peu­tics are look­ing for ways to craft next-gen cell ther­a­pies with­out re­ly­ing on pa­tients’ own cells. An ear­ly peek at the com­pa­ny’s clin­i­cal da­ta looks promis­ing, but will iffy dura­bil­i­ty re­sults prove a road­block?

Fate on Thurs­day re­vealed an ear­ly cut of Phase I da­ta for its FT596 and FT516 in­duced pluripo­tent stem cell-de­rived nat­ur­al killer cell pro­grams that re­vealed po­ten­tial red flags on one of the ther­a­pies’ dura­bil­i­ty over time.

At a three-month check in, eight of 11 B cell lym­phoma pa­tients who re­ceived FT516, off-the-shelf NK cells en­gi­neered with a “non-cleav­able” CD16 Fc re­cep­tor, re­port­ed an ob­jec­tive re­sponse with no ma­jor safe­ty sig­nals. But at a Ju­ly 7 cut­off, just five pa­tients (45%) main­tained those re­spons­es, with two com­plete re­spon­ders see­ing their dis­ease progress and a par­tial re­spon­der forced to re­ceive an “ad­di­tion­al an­ti-can­cer ther­a­py,” Fate said.

It’s a trou­bling sign for FT516’s dura­bil­i­ty but not a death knell for the pro­gram. The ther­a­py’s safe­ty pro­file, for in­stance, with no FT516-linked se­ri­ous side ef­fects and no FT516-re­lat­ed Grade 3 events or high­er, should warm in­vestors’ hearts some­what. But im­me­di­ate­ly fol­low­ing the news, shares of $FATE sunk around 10% af­ter the bell, po­ten­tial­ly un­der­scor­ing share­hold­ers’ wor­ries about the drug’s abil­i­ty to main­tain ef­fect.

On a call with an­a­lysts Thurs­day, the Fate team field­ed ques­tions on FT516’s dura­bil­i­ty, par­tic­u­lar­ly com­pared to re­sults from CD19 CAR-T ther­a­pies.

“It’s too ear­ly to know ul­ti­mate­ly what the long term dura­bil­i­ty of FT516 is and how that com­pares to CAR-T ther­a­py,” CEO Scott Wolchko said. “We’re very en­cour­aged by the dura­bil­i­ty we’ve seen as we fol­low these pa­tients.”

Fate is one of a group of biotechs lean­ing on the pos­si­bil­i­ty of en­gi­neer­ing donor stem cells to cre­ate pow­er­ful tu­mor fight­ers. NK cells, which are as­so­ci­at­ed with a low­er risk of cy­tokine re­lease syn­drome than T cell ther­a­pies, hold promise as an op­tion for late-stage can­cer pa­tients, many of whom have pre­vi­ous­ly failed on pri­or im­munother­a­pies and/or cell ther­a­pies.

The Phase I study for FT516 dosed the ther­a­py across co­horts up to two treat­ment cy­cles in­clud­ing a reg­i­men of three days of con­di­tion­al chemother­a­py fol­lowed by a sin­gle dose of Roche’s Rit­ux­an and three week­ly dos­es of vary­ing strength of FT516 and IL-2 cy­tokine sup­port. Pa­tients had re­ceived a me­di­an of three pri­or lines of ther­a­py and a me­di­an of two pri­or lines of CD20-tar­get­ed ther­a­py. Of the 11 pa­tients in the study, eight had ag­gres­sive B cell lym­phoma, five pa­tients didn’t re­spond to their most re­cent pri­or ther­a­py, and four pa­tients were pre­vi­ous­ly treat­ed with au­tol­o­gous CD19 CAR-T cell ther­a­py.

FT516’s re­sults in pa­tients with pri­or CD19 CAR-T ther­a­py — two of four post­ed a com­plete re­sponse — spurred Fate to an­nounce a dose-ex­pan­sion co­hort in that pop­u­la­tion, which the biotech thinks could ad­dress a grow­ing mar­ket need.

Mean­while, FT596 post­ed ear­ly da­ta of its own show­ing promis­ing ef­fi­ca­cy. The ther­a­py us­es the same plat­form as FT516 with an ad­di­tion­al CAR con­struct en­gi­neered on­to the cells in an at­tempt to ad­dress het­ero­ge­neous tu­mor types and anti­gen es­cape.

In­ter­im da­ta showed 10 of 14 pa­tients across two dose-es­ca­la­tion co­horts post­ed an ob­jec­tive re­sponse, in­clud­ing sev­en com­plete re­spon­ders, in­clud­ing two of three pa­tients who were treat­ed with a com­bo of FT596 and Rit­ux­an af­ter au­tol­o­gous CD19 CAR-T ther­a­py.

Fate ex­pects to present fur­ther da­ta from both stud­ies at the up­com­ing ASH con­fer­ence in De­cem­ber, in­clud­ing a first look at dura­bil­i­ty of re­sponse for FT596.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

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Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

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Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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Yong Dai, Frontera Therapeutics CEO

Scoop: Lit­tle-known Or­biMed-backed biotech clos­es $160M round to start gene ther­a­py tri­al

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Sanofi, GSK tout 72% Omi­cron ef­fi­ca­cy in PhI­II tri­al of next-gen, bi­va­lent shot — with an eye to year-end roll­out

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In trou­bled Hunt­ing­ton’s space, uniQure’s gene ther­a­py shows ear­ly promise

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John Leonard, Intellia CEO

In­tel­li­a's CRISPR pro­gram that ed­its genes di­rect­ly in pa­tients shows dura­bil­i­ty in AT­TR amy­loi­do­sis

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Intellia Therapeutics presented the much-anticipated data for its Regeneron-collaborated NTLA-2001 Friday morning, adding to the initial Phase I results it first delivered almost a year ago to the day.

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Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

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Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

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After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.