Mer­ck’s PhI­II liv­er can­cer study for Keytru­da fails on two key end­points, just three months af­ter the FDA gave it the green light

In a rare set­back, Mer­ck has suf­fered a key Phase III fail­ure for Keytru­da that could put a crimp in its sales prospects.

Roy Baynes

The phar­ma gi­ant re­port­ed af­ter the mar­ket closed on Tues­day that Keytru­da failed the KEYNOTE-240 study for he­pa­to­cel­lu­lar car­ci­no­ma, the most com­mon type of liv­er can­cer. The tri­al missed on both over­all sur­vival as well as pro­gres­sion-free sur­vival. That news ar­rived just 3 months af­ter the FDA of­fered an ac­cel­er­at­ed ap­proval on liv­er can­cer based on their ear­li­er mid-stage da­ta for the drug. 

Stay­ing on the mar­ket re­quires Mer­ck to put up pos­i­tive piv­otal da­ta. Ques­tions about the drug’s fu­ture in liv­er can­cer dinged Mer­ck’s stock $MRK, which dropped slight­ly more than 1% af­ter the re­lease hit.

Mer­ck was quick to note that it’s study­ing Keytru­da in a range of oth­er liv­er can­cer stud­ies, but the ad­van­tage here has clear­ly switched to Op­di­vo for now, which has been drag­ging steadi­ly be­hind Keytru­da af­ter falling short on lung can­cer, where Mer­ck holds first-line ad­van­tage.

This wasn’t the on­ly con­fir­ma­to­ry Phase III study to flop. Just weeks ago Eli Lil­ly had to sus­pend mar­ket­ing of Lartru­vo af­ter its piv­otal for soft tis­sue sar­co­ma failed. These back-to-back set­backs, though, are un­like­ly to slow down the FDA, which has proved ea­ger to hand out ac­cel­er­at­ed OKs — par­tic­u­lar­ly for can­cer drugs — in re­cent years.

“While we are dis­ap­point­ed KEYNOTE-240 did not meet its co-pri­ma­ry end­points, the re­sults for over­all sur­vival, pro­gres­sion-free sur­vival and ob­jec­tive re­sponse rate are gen­er­al­ly con­sis­tent with find­ings from the Phase II study, KEYNOTE-224, which led to the ac­cel­er­at­ed ap­proval of KEYTRU­DA for the treat­ment of pa­tients with he­pa­to­cel­lu­lar car­ci­no­ma who have been pre­vi­ous­ly treat­ed with so­rafenib,” said Roy Baynes, se­nior vice pres­i­dent and head of glob­al clin­i­cal de­vel­op­ment, chief med­ical of­fi­cer, Mer­ck Re­search Lab­o­ra­to­ries.


Pho­to cred­it: AP Im­ages.

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,400+ biopharma pros reading Endpoints daily — and it's free.

The Advance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: Gilead leas­es part­ner rights to TIG­IT, PD-1 in a $2B deal with Ar­cus. Now comes the hard part

Gilead CEO Dan O’Day has brokered his way to a PD-1 and lined up a front row seat in the TIGIT arena, inking a deal worth close to $2 billion to align the big biotech closely with Terry Rosen’s Arcus. And $375 million of that comes upfront, with cash for the buy-in plus equity, along with $400 million for R&D and $1.22 billion in reserve to cover opt-in payments and milestones..

Hotly rumored for weeks, the 2 players have formalized a 10-year alliance that starts with rights to the PD-1, zimberelimab. O’Day also has first dibs on TIGIT and 2 other leading programs, agreeing to an opt-in fee ranging from $200 million to $275 million on each. There’s $500 million in potential TIGIT milestones on US regulatory events — likely capped by an approval — if Gilead partners on it and the stars align on the data. And there’s another $150 million opt-in payments for the rest of the Arcus pipeline.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,400+ biopharma pros reading Endpoints daily — and it's free.

Covid-19 roundup: Roche pairs Actem­ra with remde­sivir in new PhI­II; GSK makes its own 1B vac­cine man­u­fac­tur­ing plan

A month after a small study in France suggested that Roche’s IL-6 inhibitor Actemra helped Covid-19 patients do better — even as Sanofi and Regeneron found somewhat disappointing results with their rival drug — Roche is doubling down on the strategy.

The Swiss pharma giant is kicking off a second Phase III global placebo-controlled study involving Actemra. But rather than testing it as a monotherapy, they will add Gilead’s remdesivir to the regimen.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,400+ biopharma pros reading Endpoints daily — and it's free.

Roger Perlmutter, Merck R&D chief (YouTube)

UP­DAT­ED: Backed by BAR­DA, Mer­ck jumps in­to Covid-19: buy­ing out a vac­cine, part­ner­ing on an­oth­er and adding an­tivi­ral to the mix

Merck execs are making a triple play in a sudden leap into the R&D campaign against Covid-19. And they have more BARDA cash backing them up on the move.

Tuesday morning the pharma giant simultaneously announced plans to buy an Austrian biotech that has been working on a preclinical vaccine candidate, added a collaboration on another vaccine with the nonprofit IAVI and inked a deal with Ridgeback Biotherapeutics on an early-stage antiviral.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,400+ biopharma pros reading Endpoints daily — and it's free.

Bryan Roberts, Venrock

Ven­rock sur­vey shows grow­ing recog­ni­tion of coro­n­avirus toll, wan­ing con­fi­dence in ar­rival of vac­cines and treat­ments

When Venrock partner Bryan Roberts went to check the results from their annual survey of healthcare leaders, what he found was an imprint of the pandemic’s slow arrival in America.

The venture firm had sent their form out to hundreds of insurance and health tech executives, investors, officials and academics on February 24 and gave them two weeks to fill it out. No Americans had died at that point but the coronavirus had become enough of a global crisis that they included two questions about the virus, including “Total U.S. deaths in 2020 from the novel coronavirus will be:”.

Fabrice Chouraqui, Cellarity CEO-partner (LinkedIn)

Drug de­vel­op­er, Big Phar­ma com­mer­cial ex­ec, now an up­start biotech chief — Fab­rice Chouraqui is ready to try some­thing new as a ‘CEO-part­ner’ at Flag­ship

Fabrice Chouraqui’s career has taken some big twists along his life journey. He got his PharmD at Université Paris Descartes and jumped into the drug development game for a bit. Then he took a sharp turn and went back to school to get his MBA at Insead before returning to pharma on the commercial side.

Twenty years later, after steadily rising through the ranks and journeying the globe to nab a top job as president of US pharma for the Basel-based Novartis, Chouraqui exited in another career switch. And now he’s headed into a hybrid position as a CEO-partner at Flagship, where he’ll take a shot at leading Cellarity — one of the VC’s latest paradigm-changing companies of the groundbreaking model that aspires to deliver a new platform to the world of drug R&D.

Al­ny­lam nabs speedy re­view, set­ting up 3rd pos­si­ble ap­proval in 3 years

After nearly two decades in the haze of preclinical and clinical development, things seem to be coming into focus for Alnylam Pharmaceuticals.

Two years ago the company landed the first approved drug for RNA interference (RNAi), a Nobel Prize-winning technique discovered in plants and pioneered around the turn of the century. Then last year, they landed another approval. Now, fresh off a massive investment from Blackstone, they’ve received an FDA priority review designation for a third therapy, setting them up to potentially nab three different approvals in three consecutive years.

FDA ex­plains im­pact of Covid-19 on ap­pli­ca­tions, for­mal meet­ings

The FDA on Tuesday issued immediately effective guidance explaining how the coronavirus disease public health emergency is impacting the conduct of formal meetings and its review of certain user fee-funded applications.

The guidance features 11 questions and answers on how the agency intends to handle disruptions affecting meetings and goals under its user fee programs established by the Prescription Drug User Fee Act (PDUFA), Biosimilar User Fee Act (BSUFA) and the Generic Drug User Fee Amendments (GDUFA).