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.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

UP­DAT­ED: Boehringer nabs FDA's first in­ter­change­abil­i­ty des­ig­na­tion for its Hu­mi­ra com­peti­tor — but will it mat­ter?

The FDA late Friday awarded Boehringer Ingelheim the first interchangeability designation for its Humira biosimilar Cyltezo, meaning that when it launches in July 2023, pharmacists will be able to automatically substitute the Boehringer’s version for AbbVie’s mega-blockbuster without a doctor’s input.

The designation will likely give Boehringer, which first won approval for Cyltezo in 2017, the leg up on a crowded field of Humira competitors.

Bio­gen hit by ALS set­back with PhI­II fail­ure for tofersen — but fol­lows a fa­mil­iar strat­e­gy high­light­ing the pos­i­tive

Patients and analysts waiting to hear Sunday how Biogen’s SOD1-ALS drug tofersen fared in Phase III didn’t have to wait long for the top-line result they were all waiting for. The drug failed the primary endpoint on significantly improving the functional and neurologic decline of patients over 28 weeks as well as the extension period for continued observation.

In fact, there was very little difference in response.

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Jeffrey Nau, Oyster Point Pharma CEO

FDA OKs an in­haled ver­sion of smok­ing ces­sa­tion drug Chan­tix — for a com­mon eye dis­ease

Oyster Point Pharma now has its first FDA-approved product — Tyrvaya. And the biotech has taken a unique route to get there by using an old drug with a storied past.

The New Jersey biotech announced this morning that the FDA has approved their nasal spray product for dry eye disease on Friday — the first nasal spray to be approved for the disease. The product’s active ingredient is 0.03 mg of varenicline, also known as smoking cessation aid Chantix.

Sheldon Koenig, Esperion CEO

Es­pe­ri­on gets out the bud­get ax, chop­ping 170 staffers as its big drug launch sput­ters

Esperion’s executive team spent years insisting that they had found the sweet spot in the market for their cholesterol drug. But that strategy has soured badly, and after struggling to sell its heart disease pill for more than a year, the biotech says it will cut about 40% of its staff over the next few weeks.

The layoffs will take place across the board, from sales and marketing to R&D, CEO Sheldon Koenig told Endpoints News on Monday. While the chief executive declined to elaborate on how many employees will be affected, an SEC filing stated that approximately 170 staffers are on the chopping block.

Two drug­mak­ers hit with PDU­FA date de­lays from FDA amid back­log of in­spec­tions

As the FDA is weighed down with more and more pandemic responsibilities, the agency is beginning to miss PDUFA dates with more frequency too. Two different companies on Monday said they received notices that the FDA has not completed their drug reviews on time.

The review of an NDA for Avadel Pharmaceuticals’ candidate treatment for narcolepsy is not coming this month, the company said, and the review of UCB’s BLA for bimekizumab, used to treat moderate to severe plaque psoriasis, will miss its target date as well.

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Reshma Kewalramani, Vertex CEO (YouTube)

Ver­tex gets much-need­ed win with ‘ex­tra­or­di­nary’ first pa­tient re­sults on po­ten­tial di­a­betes cure

Vertex said Monday that the first patient dosed with its cell therapy for type 1 diabetes saw their need for insulin injections vanish almost entirely, a key early step in the decades-long effort to develop a curative treatment for the chronic disease.

The patient, who had suffered five potentially life-threatening hypoglycemic — or low blood sugar — episodes in the year before the therapy, was injected with synthetic insulin-producing cells. After 90 days, the patient’s new cells produced insulin steadily and ramped up their insulin production after a meal like normal cells do, as measured by a standard biomarker for insulin production.

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Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; 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.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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Scott Struthers, Crinetics CEO

Cri­net­ics spins out ra­dio­phar­ma ef­forts in­to a new com­pa­ny, high­light­ing the grow­ing field­'s al­lure

Largely known for its nonpeptide small molecule research, Crinetics has been keeping its radiopharma work comparatively under wraps. But that changed Monday afternoon as the California biotech spun out a new company focused solely on the burgeoning field.

Crinetics launched Radionetics after the closing bell Monday, the company announced, seeding the new entity with $30 million raised from 5AM Ventures and Frazier Healthcare Partners. Radionetics will start with its own radiopharma-centric platform and a pipeline of 10 programs aimed at solid tumors.