Mer­ck looks to ditch the nee­dles with its next-gen oral PC­SK9 in­hibitor for high cho­les­terol

Af­ter win­ning ap­proval about six years ago, the first wave of PC­SK9 in­hibitors large­ly failed to muster the block­buster sales an­a­lysts had pre­dict­ed. Now Mer­ck is hop­ing its oral op­tion will find bet­ter suc­cess.

The phar­ma gi­ant un­veiled on Mon­day what it says are the first in-hu­man re­sults for an oral PC­SK9 in­hibitor. The can­di­date, MK-0616, was well-tol­er­at­ed in men and women with high cho­les­terol at dos­es up to 300 mg, with no deaths or se­ri­ous side ef­fects, the com­pa­ny said at the Amer­i­can Heart As­so­ci­a­tion con­fer­ence.

In ad­di­tion, the ex­per­i­men­tal drug re­duced blood lev­els of free PC­SK9 by more than 90% from base­line when ad­min­is­tered as a sin­gle dose be­tween 10 and 300 mg, Mer­ck an­nounced. In a mul­ti­ple-dose study, LDL-cho­les­terol (al­so known as bad cho­les­terol) de­creased by about 65% from base­line in pa­tients who took MK-0616 on top of their cur­rent statin ther­a­py for 14 days.

“The ini­tial re­sults are en­cour­ag­ing; how­ev­er, more clin­i­cal stud­ies are need­ed to con­firm these find­ings, giv­en lim­it­ed clin­i­cal ex­pe­ri­ence with the mol­e­cule MK-0616,” lead study au­thor Dou­glas Johns said in a state­ment.

PC­SK in­hibitors work by block­ing a liv­er pro­tein that helps to de­stroy LDL-C re­cep­tors on the sur­face of cells. Those re­cep­tors are re­spon­si­ble for tak­ing cho­les­terol out of the blood stream. There­fore, more ac­tive re­cep­tors means less bad cho­les­terol.

Sanofi and Re­gen­eron’s Pralu­ent and Am­gen’s Repatha were the first to se­cure ap­provals here, mark­ing the first big cho­les­terol drugs since Lip­i­tor. An­a­lysts had pre­dict­ed megablock­buster sales — but pay­ers, weary of fund­ing a chron­ic treat­ment for a large pa­tient pop­u­la­tion, did every­thing pos­si­ble to keep pa­tients on cheap gener­ics. That led to ma­jor price cuts, and in 2019, Sanofi lopped off its car­dio di­vi­sion al­to­geth­er.

No­var­tis shelled out $9.7 bil­lion for The Med­i­cine Com­pa­ny’s PC­SK9 drug in­clisir­an that same year, plan­ning for a swift US ap­proval. But af­ter get­ting slapped with a CRL last year, the com­pa­ny re­leased more post-hoc da­ta this week at AHA in the hopes of fi­nal­ly get­ting it past the goal line. No­var­tis touts in­clisir­an (which is in­ject­ed twice a year) as an eas­i­er op­tion than Repatha and Pralu­ent, which re­quire dos­ing every two weeks or month­ly, re­spec­tive­ly.

Mer­ck, on the oth­er hand, thinks it can skip the nee­dles al­to­geth­er.

“I be­lieve this is the first re­port of suc­cess­ful oral ab­sorp­tion of a syn­the­sized cyclic pep­tide like MK-0616 in peo­ple,” Johns said. “We were pleased that it ap­peared to be con­sis­tent­ly ab­sorbed and con­cen­trat­ed in pa­tients’ blood, and that it re­duced cho­les­terol lev­els so ef­fec­tive­ly.”

Albert Bourla (Photo by Steven Ferdman/Getty Images)

UP­DAT­ED: Pfiz­er fields a CRL for a $295M rare dis­ease play, giv­ing ri­val a big head start

Pfizer won’t be adding a new rare disease drug to the franchise club — for now, anyway.

The pharma giant put out word that their FDA application for the growth hormone therapy somatrogon got the regulatory heave-ho, though they didn’t even hint at a reason for the CRL. Following standard operating procedure, Pfizer said in a terse missive that they would be working with regulators on a followup.

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A new can­cer im­munother­a­py brings cau­tious hope for a field long await­ing the next big break­through

Bob Seibert sat silent across from his daughter at their favorite Spanish restaurant near his home in Charleston County, SC, their paella growing cold as he read through all the places in his body doctors found tumors.

He had texted his wife, a pediatric intensive care nurse, when he got the alert that his online chart was ready. Although he saw immediately it was bad, many of the terms — peritoneal, right iliac — were inscrutable. But she was five hours downstate, at a loud group dinner the night before another daughter’s cheer competition.

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Graphic: Alexander Lefterov for Endpoints News

Small biotechs with big drug am­bi­tions threat­en to up­end the tra­di­tion­al drug launch play­book

Of the countless decisions Vlad Coric had to make as Biohaven’s CEO over the past seven years, there was one that felt particularly nerve-wracking: Instead of selling to a Big Pharma, the company decided it would commercialize its migraine drug itself.

“I remember some investors yelling and pounding on the table like, you can’t do this. What are you thinking? You’re going to get crushed by AbbVie,” he recalled.

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Mar­ket­ingRx roundup: Pfiz­er de­buts Pre­vnar 20 TV ads; Lil­ly gets first FDA 2022 pro­mo slap down let­ter

Pfizer debuted its first TV ad for its Prevnar 20 next-generation pneumococcal pneumonia vaccine. In the 60-second spot, several people (actor portrayals) with their ages listed as 65 or older are shown walking into a clinic as they turn to say they’re getting vaccinated with Prevnar 20 because they’re at risk.

The update to Pfizer’s blockbuster Prevnar 13 vaccine was approved in June, and as its name suggests is a vaccine for 20 serotypes — the original 13 plus seven more that cause pneumococcal disease. Pfizer used to spend heavily on TV ads to promote Prevnar 13 in 2018 and 2019 but cut back its TV budgets in the past two fall and winter seasonal spending cycles. Prevnar had been Pfizer’s top-selling drug, notching sales of just under $6 billion in 2020, and was the world’s top-selling vaccine before the Covid-19 vaccines came to market last year.

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Roy Baynes, Merck

FDA bats back Mer­ck’s ‘pipeline in a prod­uct,’ de­mands more ef­fi­ca­cy da­ta

Despite some heavy blowback from analysts, Merck execs maintained an upbeat attitude about the market potential of its chronic cough drug gefapixant. But the confidence may be fading somewhat today as Merck puts out news that the FDA is handing back its application with a CRL.

Dubbed by Merck’s development chief Roy Baynes as a “pipeline in a product” with a variety of potential uses, Merck had fielded positive late-stage data demonstrating the drug’s ability to combat chronic cough. The drug dramatically reduced chronic cough in Phase III, but so did placebo, leaving Merck’s research team with a marginal success on the p-value side of the equation.

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Florida Gov. Ron DeSantis (AP Photo/Wilfredo Lee, File)

Opin­ion: Flori­da is so mAb crazy, Ron De­San­tis wants to use mAbs that don't work

Florida Gov. Ron DeSantis is trying so hard to politicize the FDA and demonize the federal government that he entered into an alternate universe on Monday evening in describing a recent FDA action to restrict the use of two monoclonal antibody, or mAb, treatments for Covid-19 that don’t work against Omicron.

Without further ado, let’s break down his statement from last night, line by line, adjective by adjective.

Steve Worland, eFFECTOR CEO

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When eFFECTOR Therapeutics went public last summer on the coattails of a reverse merger with Locust Walk’s SPAC, the potential of its lead drug, tomivosertib, as a combo agent with Merck’s flagship PD-1 Keytruda was hailed as the main draw.

But the biotech is now axing those plans and essentially starting over.

In a surprise move, San Diego-based eFFECTOR said it’s halting the development of tomivosertib in non-small cell lung cancer patients who have already progressed on Keytruda monotherapy after running into enrollment challenges in a Phase IIb trial.

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Not cheap­er by the dozen: Bris­tol My­ers be­comes the 12th phar­ma com­pa­ny to re­strict 340B sales

Bristol Myers Squibb recently joined 11 of its peer pharma companies in limiting how many contract pharmacies can access certain drugs discounted by a federal program known as 340B.

Bristol Myers is just the latest in a series of high-profile pharma companies moving in their own direction as the Biden administration’s Health Resources and Services Administration struggles to rein in the drug discount program for the neediest Americans.

Joaquin Duato, J&J CEO (Photo by Charles Sykes/Invision/AP)

New J&J CEO Joaquin Du­a­to promis­es an ag­gres­sive M&A hunt in quest to grow phar­ma sales

Joaquin Duato stepped away from the sideline and directly into the spotlight on Tuesday, delivering his first quarterly review for J&J as its newly-tapped CEO after an 11-year run in senior posts. And he had some mixed financial news to deliver today while laying claim to a string of blockbuster drugs in the making and outlining an appetite for small and medium-sized M&A deals.

Duato also didn’t exactly shun large buyouts when asked about the future of the company’s medtech business — where they look to be in either the top or number 2 position in every segment they’re in — even though the bar for getting those deals done is so much higher.

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