Pi­o­neer­ing Mer­ck makes an­oth­er break­through with PD-1 star Keytru­da, grab­bing first OK for a can­cer bio­mark­er

Mer­ck con­tin­ues to be the trend­set­ter to watch in im­muno-on­col­o­gy. On Tues­day the phar­ma gi­ant nabbed a unique first in the field, grab­bing an FDA ap­proval to use Keytru­da to fight can­cer with a par­tic­u­lar bio­mark­er sig­na­ture of its own.

Specif­i­cal­ly, the agency has pro­vid­ed a green light to pre­scribe Keytru­da to fight tu­mors with a bio­mark­er called mi­crosatel­lite in­sta­bil­i­ty-high or mis­match re­pair de­fi­cient.

Richard Padzur

No­tably, can­cer drug czar Richard Paz­dur did the hon­ors in rec­og­niz­ing the achieve­ment in the FDA’s state­ment.

“This is an im­por­tant first for the can­cer com­mu­ni­ty,” Richard Paz­dur, act­ing di­rec­tor of the of­fice of hema­tol­ogy and on­col­o­gy prod­ucts in the FDA’s CDER and di­rec­tor of the FDA’s On­col­o­gy Cen­ter of Ex­cel­lence, said in a state­ment. “Un­til now, the FDA has ap­proved can­cer treat­ments based on where in the body the can­cer start­ed — for ex­am­ple, lung or breast can­cers. We have now ap­proved a drug based on a tu­mor’s bio­mark­er with­out re­gard to the tu­mor’s orig­i­nal lo­ca­tion.”

The agency sin­gled out tu­mors with ab­nor­mal­i­ties that play a role in DNA re­pair, high­light­ing col­orec­tal, en­dome­tri­al and gas­troin­testi­nal can­cers, but al­so less com­mon­ly in can­cers aris­ing in the breast, prostate, blad­der, thy­roid gland and more.

In this case Mer­ck was able to draw on da­ta from five stud­ies show­ing Keytru­da’s abil­i­ty to shrink a sig­nif­i­cant num­ber of tu­mors.

Roger Perl­mut­ter, Mer­ck

Mer­ck has been rolling up a string of im­pres­sive wins on the I/O front with its PD-1 check­point drug Keytru­da. While Roger Perl­mut­ter was lucky enough to have it in the pipeline when he ar­rived as R&D chief, he has pressed every ad­van­tage in ex­pand­ing on its pi­o­neer­ing first ap­proval.

A week ago the agency fol­lowed through with an OK for blad­der can­cer, its sec­ond in a few weeks. Just this morn­ing the phar­ma gi­ant scored an ac­cel­er­at­ed re­view for stom­ach can­cer, a clear sig­nal of fu­ture suc­cess. And Mer­ck re­searchers will present da­ta on 16 can­cer types at AS­CO in ear­ly June.

Re­cent­ly the phar­ma gi­ant leapfrogged Bris­tol-My­ers Squibb on non-small cell lung can­cer, win­ning an OK to use a com­bi­na­tion of Keytru­da and chemo as a front­line ther­a­py. And there are hun­dreds of ad­di­tion­al stud­ies un­der­way to test its ef­fi­ca­cy in com­bo ther­a­pies.

Un­pack­ing the Aduhelm de­ci­sion, Ver­tex's half full glass, a $525M J&J breakup, and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

By now you have surely read about the FDA’s controversial approval of Biogen’s Alzheimer’s drug and all its reverberations. But I’d still recommend checking out the meaty recap below to make sure you didn’t miss all the angles that the Endpoints team has covered. If you’d rather look ahead, look no further than our three-day virtual panels next week at BIO, where we will discuss what the new normal means for every part of the industry.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 109,100+ biopharma pros reading Endpoints daily — and it's free.

What does a clear ma­jor­i­ty of the bio­phar­ma in­dus­try think of the FDA ap­proval of ad­u­canum­ab? 'Hor­ri­fy­ing' 'Dan­ger­ous' 'Con­fus­ing' 'Dis­as­ter'

Over the years, we’ve become used to seeing a consensus emerge early in our industry polls at Endpoints News. And when we took the pulse of drug hunters on the heels of a controversial FDA approval for aducanumab this week, it became immediately apparent that the vast majority of our readers — heavily concentrated among biopharma staffers and execs — were incensed by what they had just witnessed.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 109,100+ biopharma pros reading Endpoints daily — and it's free.

David Knopman (Mayo Clinic via YouTube)

A sec­ond ad­comm mem­ber aban­dons his post in af­ter­math of con­tro­ver­sial ad­u­canum­ab de­ci­sion

As the fallout from the FDA’s approval of Alzheimer’s med aducanumab grows, a second member of the adcomm overseeing that drug’s review has walked away. But even with two experts now having resigned from that committee in protest, is there enough broad-level outrage to prevent another aducanumab from getting approved?

The FDA on Wednesday lost another member of its Peripheral and Central Nervous System Drugs Advisory Committee as Mayo Clinic neurologist David Knopman hit the exit over the agency’s decision to approve Biogen’s Alzheimer’s drug Aduhelm despite the committee’s near-unanimous vote against it.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 109,100+ biopharma pros reading Endpoints daily — and it's free.

Aaron Kesselheim (Scott Eisen/AP Images for AIDS Healthcare Foundation)

Har­vard’s Aaron Kessel­heim re­signs from ex­pert pan­el in wake of ad­u­canum­ab OK, blast­ing FDA for ‘worst drug ap­proval de­ci­sion in re­cent U.S. his­to­ry'

A third member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has resigned in the wake of Biogen’s controversial Aduhelm approval, slamming the agency as he left and further deepening the controversy surrounding the decision.

Harvard University professor Aaron Kesselheim quit in protest Thursday afternoon, calling the Aduhelm OK “probably the worst drug approval decision in recent U.S. history.” Kesselheim follows both Joel Perlmutter, a neurologist from Washington University in St. Louis, and David Knopman, a neurologist from the Mayo Clinic, out the door.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 109,100+ biopharma pros reading Endpoints daily — and it's free.

Janet Woodcock, acting FDA commissioner, at Thursday's Senate Appropriations hearing (Bill Clark/CQ Roll Call via AP Images)

Sen­a­tors lam­bast new Alzheimer’s drug’s price but give Janet Wood­cock a free pass on the ap­proval de­ci­sion

Senate Finance Democrats took aim at Biogen’s pricey new Alzheimer’s drug on Thursday, but members on both sides of the aisle at a separate appropriations hearing didn’t question acting FDA commissioner Janet Woodcock on the approval.

“I was appalled that Biogen priced their Alzheimer’s drug approved by the FDA at $56,000 per year — I’m not going to debate whether this is effective or not, but it’s double the household median income for Michiganders over the age of 65,” Sen. Debbie Stabenow (D-MI) said at the finance hearing.

FDA au­tho­rizes about 10M J&J vac­cine dos­es, trash­es 60M more from trou­bled Emer­gent plant

The FDA on Friday released about 10 million doses of J&J’s vaccine for use, and disposed of another 60 million doses that were manufactured at the now-shuttered Emergent BioSolutions facility in Baltimore where cross-contamination occurred.

The agency said it’s not yet ready to allow the Emergent plant to be included in the J&J EUA, but that may occur soon. FDA came to the decision to authorize some of the doses after reviewing facility records and quality testing results.

Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

Months af­ter FDA re­jec­tion, Sanofi touts piv­otal win for rare dis­ease drug su­tim­limab as it preps to re­file

One of the pillar drugs of Sanofi’s $11.6 billion pickup of Bioverativ hit a big setback late last year when the FDA sent its application for approval back. Now, as Sanofi gears up to resubmit the drug for review, the drugmaker is touting pivotal data it hopes will help take it over the finish line.

Sanofi’s sutimlimab nailed all three of its primary endpoints in its Phase III CADENZA study for patients with cold agglutinin disease, a rare disorder that can cause severe anemia, without a recent history of blood transfusion, the French drugmaker said Friday. The topline results will be presented at this weekend’s virtual EHA meeting.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 109,100+ biopharma pros reading Endpoints daily — and it's free.

Reshma Kewalramani, Vertex CEO (BIO via YouTube)

UP­DAT­ED: Ver­tex strikes out on its lat­est big shot at a rare ge­net­ic dis­ease. But they're go­ing to keep on swing­ing

It’s been several months since Vertex culled one of its small molecules for alpha-1 antitrypsin deficiency (AATD), taking a big hit after evidence of liver damage surfaced in a key Phase II trial. Now we learned that the company has whiffed on its second shot, and there’s nothing left in the clinic to treat the rare genetic disease — but that won’t stop it from trying.

Despite avoiding the safety issues that plagued the last candidate, Vertex $VRTX is taking the axe to VX-864 after Phase II results revealed the magnitude of the drug’s response is “unlikely to translate into substantial clinical benefit.” As a result of the news, the company’s stock fell 12.5% after hours.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 109,100+ biopharma pros reading Endpoints daily — and it's free.

Bris­tol My­er­s' CAR-T Breyanzi busts out a win in ear­li­er-line lym­phoma, po­ten­tial­ly crack­ing open an ex­pand­ed mar­ket

Despite being third to the field in B cell lymphoma, Bristol Myers Squibb has repeatedly argued its CAR-T Breyanzi could have the juice to overtake its older competitors. Going into earlier lines of therapy may be the golden ticket on that front, and now Breyanzi has a late-stage win to back up that effort.

Bristol Myers’s Breyanzi beat out physicians’-choice salvage therapy followed by high-dose chemo and a stem cell transplant — what the drugmaker called a “gold standard treatment” — in second-line patients with relapsed or refractory large B cell lymphoma, according to topline data from the Phase III TRANSFORM study released Thursday.