FDA quick­ly fol­lows a vol­un­tary pause for CAR-T 2.0 with a clin­i­cal hold to in­ves­ti­gate 2 pa­tient deaths

Celyad’s vol­un­tary pause didn’t last long.

The biotech put out word af­ter the mar­ket closed Mon­day that the FDA has swift­ly fol­lowed up the news of 2 pa­tient deaths and an an­nounced pause with a clin­i­cal hold, shift­ing con­trol over the tri­al halt to fed­er­al reg­u­la­tors.

Ac­cord­ing to the Bel­gian biotech $CYAD, the agency de­cid­ed on the hold “due to in­suf­fi­cient in­for­ma­tion to as­sess risk to study sub­jects.”

Just hours be­fore that the biotech an­nounced that the 2 pa­tients who had died in the col­orec­tal can­cer study had “pre­sent­ed sim­i­lar pul­monary find­ings,” with­out ex­plain­ing what that was pre­cise­ly.

Celyad is part of a whole group of drug de­vel­op­ers look­ing to birth the 2.0 gen­er­a­tion of CAR-Ts, la­bor­ing on off-the-shelf ther­a­pies de­signed to go af­ter sol­id tu­mors with new tech­nol­o­gy that should be eas­i­er and less ex­pen­sive to use.

CAR-T 2.0, though, has proven to be rid­dled with safe­ty is­sues, high­light­ed by a stun­ning col­lapse of Tmu­ni­ty’s first round of ther­a­pies af­ter 2 pa­tients died in one of their stud­ies. Al­lo­gene, one of the most ad­vanced biotechs in al­lo­gene­ic CAR-Ts, al­so suf­fered a tem­po­rary hold, which was re­cent­ly re­moved.

Fil­ip­po Pet­ti

The first gen­er­a­tion of per­son­al­ized ther­a­pies had to sur­vive a va­ri­ety of pa­tient deaths as well, with cy­tokine re­lease syn­drome pre­sent­ing a ma­jor hur­dle all the play­ers had to care­ful­ly cir­cum­nav­i­gate.

Celyad — run by CEO and CFO Fil­ip­po Pet­ti, a for­mer in­vest­ment banker — had start­ed the new year with high hopes of demon­strat­ing that its non-gene edit­ing ap­proach was head­ed for suc­cess, look­ing to re­verse a steady grind­ing away of the stock price that has elim­i­nat­ed 90% of its val­ue in the last 5 years. Celyad went pub­lic in the sum­mer of 2015 and the stock has dropped by about half in the last 6 months, with a fresh plunge on Mon­day that left it with a mar­ket cap of just 34 mil­lion eu­ros.

Susan Galbraith, AstraZeneca EVP, oncology R&D, at EUBIO22 (Rachel Kiki for Endpoints News)

Up­dat­ed: As­traZeneca jumps deep­er in­to cell ther­a­py 2.0 space with $320M biotech M&A

Right from the start, the execs at Neogene had some lofty goals in mind when they decided to try their hand at a cell therapy that could tackle solid tumors.

Its founders have helped hone a new approach that would pack in multiple neoantigen targets to create a personalized TCR treatment that would not just make the leap from blood to solid tumors, but do it with durability. And they managed to make their way rapidly to the clinic, unveiling their first Phase I program for advanced tumors just last May.

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Vi­a­tris with­draws ac­cel­er­at­ed ap­proval for top­i­cal an­timi­cro­bial 24 years lat­er

After 24 years without confirming clinical benefit, the FDA announced Tuesday morning that Viatris (formed via Mylan and Pfizer’s Upjohn) has decided to withdraw a topical antimicrobial agent, Sulfamylon (mafenide acetate), after the company said conducting a confirmatory study was not feasible.

Sulfamylon first won FDA’s accelerated nod in 1998 as a topical burn treatment, with the FDA noting that last December, Mylan told the agency that it wasn’t running the trial.

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Uğur Şahin, BioNTech CEO (ddp images/Sipa USA/Sipa via AP Images)

BioN­Tech bets on dif­fi­cult STING field via small mol­e­cule pact with a Pol­ish biotech

BioNTech is beefing up its relatively thin small molecule pipeline by adding weight to a clinically difficult corner of oncology R&D: STING agonists. To do so, BioNTech is teaming up with a 15-year-old Polish biotech and doling out €40 million, about $41.5 million, to start.

The deal is broken into two parts: First, BioNTech obtains an exclusive global license to develop and market Ryvu Therapeutics’ STING agonist portfolio as small molecules, whether alone or in combination with other agents.

Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

Twit­ter dis­ar­ray con­tin­ues as phar­ma ad­ver­tis­ers ex­tend paus­es and look around for op­tions, but keep tweet­ing

Pharma advertisers on Twitter are done — at least for now. Ad spending among the previous top spenders flattened even further last week, according to the latest data from ad tracker Pathmatics, amid ongoing turmoil after billionaire boss Elon Musk’s takeover now one month ago.

Among 18 top advertisers tracked for Endpoints News, only two are spending: GSK and Bayer. GSK spending for the full week through Sunday was minimal at just under $1,900. Meanwhile, German drugmaker Bayer remains the industry outlier upping its spending to $499,000 last week from $480,000 the previous week. Bayer’s spending also marks a big increase from a month ago and before the Musk takeover, when it spent $16,000 per week.

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Bris­tol My­ers scraps gene ther­a­py deal with uniQure for car­dio­vas­cu­lar dis­eases

Bristol Myers Squibb is hitting the exit on a collaboration with a gene therapy biotech.

The Big Pharma company will no longer partner with uniQure on finding new treatments for cardiovascular diseases, the biotech reported to the SEC last week, following a rocky relationship that saw the pair break off an earlier agreement — before coming back to the table. The deal will officially terminate on Feb. 21, 2023.

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Tom Riga, Spectrum Pharmaceuticals CEO

Spec­trum im­plodes af­ter a harsh pub­lic slap­down and now a CRL from Richard Paz­dur

The FDA has gone out of its way several times to flatten any expectations for Spectrum’s lung cancer drug poziotinib, including slamming the regulatory door in the biotech’s face four years ago when the their executive crew came calling for a breakthrough drug designation and encouragement from the oncology wing of the FDA.

That stinging early rebuke pointed straight down the path to a corrosive in-house agency review of Spectrum’s attempt to land an accelerated approval for the oral EGFR TKI and a public whipping that included a classic takedown by none other than Richard Pazdur, who slammed the company for “poor drug development” that led to confusion over the dose needed for a slice of NSCLC patients harboring HER2 exon 20 insertion mutations.

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Catal­ent to cut about 200 jobs in Mary­land and Texas

Contract manufacturing company Catalent is cutting about 200 jobs in Maryland and Texas, according to WARN notices, trimming back some of its pandemic-era expansion.

The company will cut 77 jobs by Jan. 15 of next year at a cell therapy facility in Webster, TX, just outside of Houston. In Maryland, the company is reducing staff at two locations, with 82 jobs being eliminated at Catalent’s facility in Gaithersburg, and 53 in Rockville. The layoffs go into effect at those locations on Jan. 14.

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iECURE CEO Joe Truitt and founder Jim Wilson

Jim Wil­son biotech iECURE gets fresh $65M to push pe­di­atric liv­er dis­ease gene ther­a­py in­to the clin­ic

Jim Wilson-founded biotech iECURE has wrapped a $65M Series A extension round to get its lead candidate — a gene replacement therapy for a rare inherited liver disease known as ornithine transcarbamylase deficiency, or OTC — into the clinic.

This round was co-led by Novo Holdings and LYFE Capital, followed by initial investors Versant and OrbiMed as well. In September 2021, iECURE raised a $50 million Series A led by the latter two. The new cash infusion will get iECURE through an initial in-human trial, which CEO Joe Truitt told Endpoints News iECURE hopes to read out in 2024.

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