FDA slaps a hold on an AML tri­al as Mark­er scraps a fail­ing ovar­i­an can­cer pro­gram, sink­ing shares

The FDA has placed a hold on a Phase II AML tri­al from the small im­muno-on­col­o­gy biotech Mark­er Ther­a­peu­tics. Mark­er dis­closed the is­sue two weeks af­ter re­spond­ing to FDA con­cerns, adding it to the Q3 re­lease Tues­day. The com­pa­ny al­so an­nounced it was scrap­ping a Phase II ovar­i­an can­cer pro­gram it de­ter­mined was un­like­ly to suc­ceed.

The agency’s con­cern cen­ters around two reagents used in man­u­fac­tur­ing for their tri­al for acute myeloid leukemia pa­tients who have re­ceived a stem cell trans­plant. The reagents are from third par­ties and not present in the fi­nal prod­uct, Mark­er said.

The com­pa­ny said the FDA raised con­cerns fol­low­ing Mark­er’s IND ap­pli­ca­tion. FDA guide­lines say the agency will re­spond with­in 30 days of re­ceiv­ing Mark­er’s re­sponse — which should mean be­fore the end of No­vem­ber.

“The FDA re­viewed our sub­mis­sion and re­quest­ed ad­di­tion­al in­for­ma­tion re­gard­ing cer­tain qual­i­ty and tech­ni­cal spec­i­fi­ca­tions for two reagents sup­plied by third-par­ty ven­dors that are used in our man­u­fac­tur­ing process,” Mark­er wrote. “Be­cause the FDA re­quires these da­ta in or­der to clear the IND, the Mark­er AML tri­al has been placed on clin­i­cal hold un­til our com­plete re­sponse to the tech­ni­cal ques­tions is sat­is­fac­to­ry to the FDA.”

Mark­er’s stock $MRKR fell 27%, from a pre­vi­ous close of $3.92 per share to $2.87.

The FDA halt comes as one of sev­er­al no­table holds the reg­u­la­tor has in­sti­tut­ed over the last few weeks, in­clud­ing two for Re­genxBio, one for No­var­tis and one for Sol­id Bio­sciences, which saw its Duchenne’s mus­cu­lar dy­s­tro­phy held up again yes­ter­day af­ter re­solv­ing a hold just 18 months ago. Re­genxBio re­spond­ed to its holds by su­ing the FDA.

Mark­er spun out of Bay­lor Col­lege of Med­i­cine 4 years ago with a re­port­ed $20 mil­lion in ear­ly back­ing, and it merged last year with TapIm­mune. The AML drug is on Mark­er’s top pro­grams and at­tempts to re­train a pa­tients’ T cells to rec­og­nize spe­cif­ic tu­mor types. They had hoped to dose their first Phase II pa­tient be­fore the end of the year.

With the ovar­i­an pro­gram al­so scrapped, Mark­er is left with a hand­ful of clin­i­cal stud­ies, in­clud­ing a T cell vac­cine pro­gram that is now in Phase II for breast can­cer pa­tients. An ear­ly analy­sis of that study showed  31 out of 34 pa­tients had a “mean­ing­ful” im­mune re­sponse, Mark­er said.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

As­traZeneca caps PD-L1/CT­LA-4/chemo com­bo come­back with OS win. Is treme­li­mum­ab fi­nal­ly ready for ap­proval?

AstraZeneca’s closely-watched POSEIDON study continues to be the rare bright spot in its push for an in-house PD-L1/CTLA-4 combo.

Combining Imfinzi and tremelimumab with physicians’ choice of chemotherapy helped patients with stage IV non-small cell lung cancer live longer, the company reported — marking the first time the still-experimental tremelimumab has demonstrated an OS benefit.

For AstraZeneca and CEO Pascal Soriot, the positive readout — which is devoid of numbers — offers much-needed validation for the big bet they made on Imfinzi plus tremelimumab, after the PD-L1/CTLA-4 regimen failed multiple trials in head and neck cancer as well as lung cancer.

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Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Paul Hastings, Nkarta CEO

With no up­front pay­ment or mile­stones on the line, Nkar­ta and CRISPR join forces on CAR-NK search

Most deals in biotech come with hefty upfront payments attached, and the promise of big biobucks if a program works out. Not this one.

Nkarta has struck what CEO Paul Hastings calls a “real collaboration” with CRISPR Therapeutics to co-develop and commercialize two CAR-NK therapies, in addition to an NK+T program. The duo will split all R&D costs — and any worldwide profits — 50/50, Hastings said.

Brent Saunders (Richard Drew, AP Images)

OcuWho? Star deal­mak­er turned aes­thet­ics czar Brent Saun­ders flips back in­to biotech. But who’s he team­ing up with now?

Brent Saunders went on a tear of headline-blazing deals building Allergan, merging and rearranging a variety of big companies into one before an M&A pact with Pfizer blew up and sent him on a bout of biotech drug deals. That didn’t work so well, so under pressure, he got his buyout at AbbVie — which needed a big franchise like Botox. And it was no big surprise to see him riding the SPAC wave into a recent $1 billion-plus deal that left him in the executive chairman’s seat at an aesthetics outfit — now redubbed The Beauty Health Company — holding a big chunk of the equity.

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Drug pric­ing watch­dog joins the cho­rus of crit­ics on Bio­gen's ad­u­canum­ab: What about charg­ing $2,560 per year?

As if Biogen’s aducanumab isn’t controversial enough, the researchers at drug pricing watchdog ICER have drawn up the contours of a new debate: If the therapy does get approved for Alzheimer’s by June, what price should it command?

Their answer: At most $8,290 per year — and perhaps as little as $2,560.

Even at the top of the range, the proposed price is a fraction of the $50,000 that Wall Street has reportedly come to expect (although RBC analyst Brian Abrahams puts the consensus figure at $11.5K). With critics, including experts on the FDA’s advisory committee, making their fierce opposition to aducanumab’s approval loud and clear, the pricing pressure adds one extra wrinkle Biogen CEO Michel Vounatsos doesn’t need as he orders full-steam preparation for a launch.