James Dentzer, Curis CEO

'Rapid' path for Curis' lead leukemia drug hits road­block with par­tial clin­i­cal hold af­ter pa­tient death

A pa­tient has died in a Phase I/IIa tri­al of Curis’ lead drug af­ter ex­pe­ri­enc­ing rhab­domy­ol­y­sis,  “among sev­er­al con­di­tions.” As a re­sult, FDA has slapped a par­tial clin­i­cal hold on the study.

The hold could throw the biotech’s plans for a po­ten­tial “rapid reg­u­la­to­ry path” of the drug in­to ques­tion. Just six weeks ago, Curis said it ex­pect­ed to meet with the FDA to dis­cuss the reg­is­tra­tional route for emavusert­ib as a monother­a­py in the first half of this year.

But the agency wants more da­ta from the open-la­bel study, which is test­ing emavusert­ib, al­so known as CA-4948, in pa­tients with re­lapsed or re­frac­to­ry acute myeloid leukemia, or high-risk myelodys­plas­tic syn­drome. The oral small mol­e­cule is be­ing test­ed alone and in com­bi­na­tion with azac­i­ti­dine or vene­to­clax. Emavusert­ib is an in­hibitor of In­ter­leukin-1 re­cep­tor-as­so­ci­at­ed ki­nase 4.

The news sent Curis’ stock $CRIS slid­ing near­ly 30% be­fore the open­ing bell Mon­day.

The FDA wants more in­for­ma­tion on the death of the pa­tient, who had R/R AML. The pa­tient had rhab­domy­ol­y­sis, “which has pre­vi­ous­ly been iden­ti­fied as a dose-lim­it­ing tox­i­c­i­ty of emavusert­ib,” Curis said in its Mon­day morn­ing state­ment. Pro­teins and elec­trolytes from dam­aged mus­cle tis­sue are re­leased in­to the blood in rhab­domy­ol­y­sis, which can lead to heart and kid­ney dam­age that re­sults in per­ma­nent dis­abil­i­ty or death. The reg­u­la­tor al­so wants safe­ty, ef­fi­ca­cy and oth­er da­ta, in­clud­ing Curis’ de­ter­mi­na­tion of the rec­om­mend­ed Phase II dose.

With the par­tial hold, the TakeAim Leukemia study can­not en­roll new pa­tients. Ex­ist­ing study pa­tients are al­lowed to con­tin­ue treat­ment at dos­es of 300mg BID or low­er. Curis was plan­ning to study the oral drug up to 400mg BID, ac­cord­ing to the tri­al list­ing. Clin­i­cal­Tri­als.gov es­ti­mates that 178 pa­tients have been en­rolled.

“Giv­en the clin­i­cal pro­file of emavusert­ib ob­served to date, we are hope­ful that the study can be re­sumed soon, af­ter ap­pro­pri­ate re­view. We con­tin­ue to be con­fi­dent in the po­ten­tial of emavusert­ib to ad­dress the high un­met need of pa­tients with AML or MDS,” CEO James Dentzer said in the news re­lease. Curis is com­mit­ted to pa­tient safe­ty, he added.

Pa­tients and in­vestors will have to wait for more de­tails about when Curis will dis­cuss emavusert­ib’s reg­is­tra­tional path with the FDA.

The com­pa­ny is al­so paus­ing en­roll­ment in its Phase I/II study of emavusert­ib in pa­tients with B-cell ma­lig­nan­cies. The par­tial hold doesn’t ap­ply to that tri­al, but Curis de­cid­ed to take the ac­tion as it awaits res­o­lu­tion of the hold.

Curis gained rights to emavusert­ib in ex­change for near­ly 20% of its out­stand­ing com­mon stock to Au­ri­gene in Jan­u­ary 2015. Curis is al­so on tap to dole out up to $52.5 mil­lion in mile­stone pay­ments for the AML pro­gram.

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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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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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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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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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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John Carroll with David Chang, Allogene CEO (Credit: Jeff Rumans Photography)

Al­lo­gene takes the stage in New York to go deep on its off-the-shelf cell ther­a­pies — de­clar­ing a first for sol­id tu­mors

NEW YORK — In most cases, a biotech like Allogene would wait until the next big science conference to offer its latest series of snapshots of its data. But most biotechs aren’t like Allogene, where the veteran leaders from Kite garnered a substantial number of kudos over the years for their in-depth reviews of the company’s progress.

So on Tuesday, the leaders at Allogene converged on Manhattan once again to give a detailed breakdown of their latest steps forward, looking to stay out front in the busy off-the-shelf cell therapy arena, keep a clean bill of health on the safety front and prove that they can not only match the autologous pioneers they helped create but make the all-important leap into solid tumors. It’s another step forward in a journey that has a long way to go before even the first big regulatory finish lines appear on the track. But for CEO David Chang, who spent some time with me running through the data ahead of the Tuesday session, it all amounts to forward momentum toward the desired goal.

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UK reg­u­la­tor warns of se­vere eye re­ac­tions fol­low­ing use of Sanofi and Re­gen­eron's Dupix­ent

The UK’s Medicines and Healthcare Regulatory Agency (MHRA) on Tuesday warned of some new and serious eye-related side effects following the use of Sanofi and Regeneron’s atopic dermatitis and asthma treatment Dupixent (dupilumab).

While Dupixent is already associated with cases of conjunctivitis and allergic conjunctivitis, dry eye and with infrequent cases of keratitis and ulcerative keratitis, the MHRA is calling on health professionals to be on the lookout for any of these eye-related side effects as “it is not currently possible to predict who may experience the rarer and most severe ocular adverse reactions, such as ulcerative keratitis.”

Mar­ket­ingRx roundup: Pfiz­er, BioN­Tech re-up iHeartRa­dio hol­i­day spon­sor­ship; WHO re­names mon­key­pox to 'm­pox'

It’s that time of year again for pop music fans with the return of the iHeartRadio Jingle Ball tour — and Pfizer and BioNTech’s sponsorship. For the second year, the Covid-19 vaccine collaborators are the pharma national sponsors among consumer brand partners, including ESPN, Dunkin, M&Ms, Mercedes and Pepsi.

Pfizer and BioNTech are also sponsoring the official Jingle Ball Radio streaming station on iHeart’s network, programmed with music from past and present concert performers. This year they include Lizzo, Dua Lipa, Dove Cameron and Charlie Puth. Pfizer-sponsored radio ads and online video and digital banner ads encourage listeners to get updated Covid-19 booster shots.

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