Mer­ck­'s wom­en's health spin­out snags mid-stage can­di­date for preterm birth; Keytru­da nails down TNBC ap­proval af­ter March CRL

Near­ly two months af­ter spin­ning out from Mer­ck, women’s health busi­ness Organon has struck its first half-bil­lion-dol­lar deal.

Organon $OGN has promised $25 mil­lion up­front and an­oth­er $475 mil­lion in biobucks for world­wide rights to ebopiprant, Ob­sE­va’s in­ves­ti­ga­tion­al treat­ment for preterm la­bor. Ebopiprant, a se­lec­tive prostaglandin F2α (PGF2α) re­cep­tor an­tag­o­nist, was orig­i­nal­ly li­censed from Mer­ck KGaA in 2015. The can­di­date works by re­duc­ing in­flam­ma­tion and uter­ine con­trac­tions.

“This de­vel­op­ment-stage as­set is be­ing stud­ied in one of the most cru­cial un­met needs for women glob­al­ly,” Organon CEO Kevin Ali said in a state­ment. There are cur­rent­ly no ap­proved drugs for the acute treat­ment of preterm la­bor in the US.

Back in No­vem­ber, Ob­sE­va said ebopiprant re­duced the de­liv­ery of preg­nan­cies (not in­clud­ing twin preg­nan­cies) by 55% at 48 hours when ad­min­is­tered with atosi­ban (the ex-US stan­dard of care), com­pared to atosi­ban alone. Over­all, 12.5% of women in the treat­ment arm de­liv­ered ba­bies with­in 48 hours of start­ing treat­ment, com­pared to 21.8% in the place­bo arm, Ob­sE­va said of the Phase IIa tri­al.

“The in­ci­dence of ma­ter­nal, fe­tal and neona­tal ad­verse events were com­pa­ra­ble be­tween sub­jects in the ebopiprant group and the place­bo group,” the com­pa­ny said.

Organon of­fi­cial­ly spun out from Mer­ck on June 3 with a port­fo­lio of more than 60 med­i­cines, in­clud­ing Nex­planon, its long-ac­tive re­versible con­tra­cep­tive. Nex­planon is one of the top hor­mon­al con­tra­cep­tives in the coun­try, ac­cord­ing to IQVIA da­ta, and grew at an an­nu­al com­pound growth rate of 9% be­tween 2014 and 2019. — Nicole De­Feud­is 

Keytru­da nails down TNBC ap­proval af­ter March CRL

Mer­ck’s block­buster check­point in­hibitor Keytru­da has won an­oth­er FDA ap­proval, this time in a line of triple-neg­a­tive breast can­cer.

Reg­u­la­tors hand­ed down the green­light for the treat­ment of pa­tients with high-risk ear­ly-stage TNBC in com­bi­na­tion with chemo, as neoad­ju­vant and then con­tin­ued as a sin­gle agent as ad­ju­vant treat­ment af­ter surgery. The thumbs up marks the 30th in­di­ca­tion for which the Keytru­da is ap­proved in the US, Mer­ck said.

Ad­di­tion­al­ly, the FDA con­vert­ed the Keytru­da-chemo com­bo ac­cel­er­at­ed ap­proval in lo­cal­ly re­cur­rent un­re­sectable or metasta­t­ic TNBC for tu­mors ex­press­ing PD-L1 to full ap­proval.

The TNBC ap­proval comes af­ter the FDA hand­ed Mer­ck a rare CRL for Keytru­da back in March, af­ter an ad­comm unan­i­mous­ly vot­ed to rec­om­mend wait­ing for more da­ta for the study in ques­tion. Ad­vis­ers and reg­u­la­tors point­ed to­ward con­cerns over the tri­al’s event-free sur­vival pri­ma­ry end­point over place­bo, say­ing re­searchers hadn’t watched pa­tients long enough af­ter treat­ment to de­ter­mine EFS and OS.

But a few weeks lat­er, Mer­ck claimed a win in the study af­ter claim­ing it reached the in­ter­im analy­sis check­point. The tri­al, known as KEYNOTE-522, met its dual pri­ma­ry end­points of event-free sur­vival and patho­log­i­cal com­plete re­sponse. — Max Gel­man

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.

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

BREAK­ING: 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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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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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.

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.

Aim­ing for fourth nod, Sarep­ta files an­oth­er DMD gene ther­a­py to FDA; Ax­some head­ed to­ward mi­graine re­sub­mis­sion

Sarepta Therapeutics has filed the data needed for an FDA accelerated approval, which would be the biotech’s fourth if granted by the agency.

The biotech has yet to complete confirmatory trials for those first three conditional nods. The filing for its fourth Duchenne muscular dystrophy treatment, disclosed Thursday, is not a surprise. Sarepta said in late-July it would do so after releasing positive results for the Roche-partnered gene therapy.

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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