Giovanni Caforio, AP Images

Bris­tol My­ers Squibb takes an­oth­er late-stage loss for Op­di­vo-Yer­voy com­bo in head and neck can­cer

Bris­tol My­ers Squibb has worked over­time to hunt down an edge for its PD-(L)1 block­er Op­di­vo over ri­val Keytru­da from Mer­ck, in­clud­ing tak­ing a bold com­bo strat­e­gy with CT­LA4 in­hibitor Yer­voy. But in head and neck can­cer, the com­bo keeps drop­ping duds.

Op­di­vo plus Yer­voy failed to show clin­i­cal ben­e­fit in ex­tend­ing the lives of first-line pa­tients with re­cur­rent or metasta­t­ic squa­mous cell car­ci­no­ma of the head and neck, ac­cord­ing to topline re­sults from the Phase III CHECK­MATE-651 study re­vealed Fri­day.

The tri­al test­ed the duo against what’s known as the EX­TREME reg­i­men, a first-line com­bo of Er­bitux, cis­platin/car­bo­platin and flu­o­rouracil, the drug­mak­er said.

Bris­tol My­ers took a shot at spin­ning a pos­i­tive, say­ing the study da­ta showed a “clear, pos­i­tive trend to­wards over­all sur­vival” for high ex­press­ing PD-L1 pos­i­tive pa­tients and not­ing the study’s con­trol arm per­formed “bet­ter than ex­pect­ed based on his­tor­i­cal da­ta.”

But it’s a fail­ure, nonethe­less, and a missed op­por­tu­ni­ty to gain ground on Keytru­da. In a state­ment, Ab­der­rahim Oukessou, Bris­tol My­ers’ VP of tho­racic can­cers and de­vel­op­ment lead, called the re­sults “dis­ap­point­ing.”

As of now, full re­sults aren’t avail­able, and Bris­tol My­ers said it would con­duct a “full eval­u­a­tion” be­fore al­low­ing the sci­en­tif­ic com­mu­ni­ty to as­sess the dam­age.

The topline re­sults aren’t all that sur­pris­ing giv­en Op­di­vo-Yer­voy al­ready flopped a head-to-head study against Op­di­vo alone in these same pa­tients back in 2019, the first sign the com­bi­na­tion could be in for some tur­bu­lence. The com­bo does hold a sec­ond-line ap­proval in this in­di­ca­tion dat­ing back to 2016 based on a pos­i­tive OS read­out in the CHECK­MATE-141 tri­al.

Op­di­vo, which sports an ap­proval in the first-line set­ting dat­ing back to 2016, could al­so be in trou­ble in head and neck can­cer af­ter drug price watch­dog the Na­tion­al In­sti­tute for Health and Care Ex­cel­lence (NICE) telegraphed in Jan­u­ary that it had stopped rec­om­mend­ing the so­lo check­point in­hibitor’s use on Eng­land’s NHS.

The drug had pre­vi­ous­ly been avail­able as part of NICE’s Can­cer Drug Fund, which al­lows physi­cians to ap­ply for use on a case-by-case ba­sis, but in­ves­ti­ga­tors found the drug’s clin­i­cal ben­e­fit pro­file wasn’t stur­dy enough to even en­dorse lim­it­ed use.

Mean­while, Keytru­da al­so sports an ap­proval in that same in­di­ca­tion dat­ing to rough­ly the same time, but has faced less of an up­hill climb. Part of the rea­son is Mer­ck hasn’t chased a com­bo ap­proval along­side a CT­LA4 block­er like Yer­voy. The drug­mak­er has been adamant that not all can­cers ben­e­fit from the PD-1/CT­LA4 com­bi­na­tion, ef­fec­tive­ly throw­ing cold wa­ter on Bris­tol My­ers’ pipeline strat­e­gy.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

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Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

FDA's ad­vanced ther­a­pies of­fice pro­vides more clar­i­ty on gene ther­a­py CMC con­sid­er­a­tions

As the Office of Tissue and Advanced Therapies (OTAT) transforms into the Office of Therapeutic Products (OTP), with new user fee funds and “super office” status, the department focused on cell and gene therapies also opened its doors to a town hall Thursday offering clarification on guidance and regulations for manufacturers.

Some of the major concerns from manufacturers were the CMC considerations between first-in-human studies and late-phase studies supporting a marketing approval.

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