Strike three: Bris­tol-My­ers’ third I/O shot at glioblas­toma miss­es as Op­di­vo flops yet again on key end­point

Glioblas­toma con­tin­ues to de­fy about every­thing thrown at it.

Bris­tol-My­ers Squibb put out word Thurs­day morn­ing that its third Phase III as­sault on brain can­cer us­ing its star PD-1 Op­di­vo failed the first end­point, with no sig­nif­i­cant im­prove­ment in pro­gres­sion-free sur­vival for pa­tients who face grim odds.

Check­Mate-548 ap­pears head­ed for a com­plete col­lapse, though over­all sur­vival is still be­ing stud­ied. Re­searchers had tried to see if the check­point ther­a­py could out­do the stan­dard of care by adding it to the stan­dard — temo­zolo­mide and ra­di­a­tion ther­a­py. 

They al­ready tried — and failed — ear­li­er this year with Check­Mate-498, a Phase III that com­bined Op­di­vo and ra­di­a­tion against temo­zolo­mide. That tri­al failed the mark on OS in May.

Two years ago the com­pa­ny was forced to con­cede de­feat on Check­mate-143, the first use of a PD-1 against glioblas­toma.

Bris­tol-My­ers has had a string of set­backs in the clin­ic with Op­di­vo, though the phar­ma gi­ant still en­joys block­buster rev­enue even as Mer­ck’s Keytru­da con­tin­ues its clin­i­cal blitz that put them on top of the mar­ket.

In this case, though, the Bris­tol-My­ers team joins a long list of fail­ures in fight­ing brain can­cer, which re­mains one of the tough­est tar­gets in on­col­o­gy. Ab­b­Vie had its own bit­ter set­back to re­port in May when their an­ti­body-drug con­ju­gate de­patux­izum­ab mafodotin (or de­patux-m; pre­vi­ous­ly known as ABT-414) al­so failed in im­prov­ing sur­vival times for brain can­cer vic­tims.

Sev­er­al years ago Ab­b­Vie showed up at AS­CO with ABT-414 and Ro­va-T to tout their po­ten­tial. Ro­va-T failed com­plete­ly for the R&D team, forc­ing a com­plete write-off of a drug they bought for $5.8 bil­lion up­front.

Robert Prins UCLA

A com­plete fail­ure here won’t ex­tin­guish all hope that a PD-1 could yet play a role in glioblas­toma. Robert Prins at the UCLA Jon­s­son Com­pre­hen­sive Can­cer Cen­ter re­port­ed ev­i­dence ear­li­er this year from a tiny ex­plorato­ry tri­al that a check­point may help pa­tients when used ahead of surgery. But it’s a long shot.

One of the few re­main­ing Phase III stud­ies for glioblas­toma like­ly to read out soon be­longs to Toca­gen $TO­CA which is mak­ing the dif­fi­cult as­sault with To­ca 511/FC. Re­searchers an­nounced sev­er­al months ago that they would con­tin­ue to the fi­nal read­out, dash­ing any hopes for an ear­ly suc­cess. There isn’t much con­fi­dence in this drug, though, with the stock falling from a high north of $15 last fall to just above $3 a share this morn­ing. The mar­ket cap is hold­ing at a mi­cro-lev­el $72 mil­lion.

These fail­ures won’t en­cour­age oth­ers to make a try.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Tim Pearson, Carrick Therapeutics CEO

Pfiz­er backs $60M in­fu­sion in­to Car­rick, teams up on breast can­cer treat­ment

In a big week for Carrick Therapeutics, the company announced $60 million in funding for its lead breast cancer drug and development of a second program, as well as a collaboration with Pfizer for combo development.

The $35 million from Pfizer comes with an agreement under which Pfizer will support Carrick’s Phase II study of samuraciclib in combination with Pfizer’s Faslodex for advanced breast cancer. Along with the investment, Adam Schayowitz, vice president and development head of breast cancer, colorectal cancer and melanoma at Pfizer global product development, will join Carrick’s scientific advisory board.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls

Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Paul Hudson, Sanofi CEO (ROMUALD MEIGNEUX/Sipa via AP Images)

Sanofi and Am­gen are bring­ing cash to cov­er the ta­ble stakes for the Hori­zon M&A game

With the market cap on Horizon Therapeutics $HZNP pushed up to the $23 billion mark today, one of the Big Pharmas in the hunt for a major league buyout deal signaled it’s playing the M&A game with cash.

Paris-based Sanofi, where CEO Paul Hudson has been largely focused on some risky biotech acquisitions to win some respect for its future pipeline prospects, issued a statement early Friday — complying with rule 2.12 of the Irish takeover rules — making clear that while the certainty or size of an offer can’t be determined, any offer “will be solely in cash.” And Amgen CEO Robert Bradway came right in behind him, filing a statement on the London Stock Exchange overnight that any offer they may make will “likely” be in cash as well.

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Philip Tagari switch­es Am­gen's dis­cov­ery lab for in­sitro's ma­chine learn­ing tools; CEO Joaquin Du­a­to to chair J&J's board

In February, Philip Tagari will take a few days of retirement and then immediately return to industry. He won’t be leading the therapeutics discovery unit for a large biopharma, though.

He’ll trade in his Amgen hat for chief scientist at a machine learning startup that has reeled in hundreds of millions in capital to lay the groundwork for a much-hyped new model of drug discovery that aims to speed up the time to new clinical assets.

Raul Rodriguez, Rigel Pharma CEO

Rigel Phar­ma scores FDA ap­proval for leukemia, kick­ing off show­down with Servi­er in IDH1

When Rigel Pharma bought olutasidenib from Forma Therapeutics, it acquired a drug that already secured a PDUFA date at the FDA — for February 2023. But regulators are ready to give their OK sooner than that.

The FDA has approved the IDH1 inhibitor as a treatment for adult patients with relapsed or refractory acute myeloid leukemia who have a susceptible IDH-1 (isocitrate dehydrogenase-1) mutation as detected by an FDA-greenlit test. Rigel will market it as Rezlidhia.

Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

Pfizer and Roivant are teaming up to launch a new ‘Vant’ aimed at bringing a mid-stage anti-inflammatory drug to market, the pair announced Thursday.

There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

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Roche HQ in Basel, Switzerland. (Image credit: Kyle LaHucik/Endpoints News)

As com­peti­tors near FDA goal­post, Roche spells out its re­peat Alzheimer's set­back

Before Roche can turn all eyes on a new version of its more-than-once-failed Alzheimer’s drug gantenerumab, the Big Pharma had to flesh out data on the November topline failure at an annual conference buzzier than in years past thanks to hotly watched rivals in the field: Eisai and Biogen’s lecanemab, and Eli Lilly’s donanemab.

There was less than a 10% difference between Roche’s drug and placebo at slowing cognitive decline across two Phase III trials, which combined enrolled nearly 2,000 Alzheimer’s patients. In its presentation at the conference Wednesday, Roche said it saw less sweeping away of toxic proteins than it had anticipated. For years, researchers and investors have put their resources behind the idea that more amyloid removal would equate to reduced cognitive decline.

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