Reshma Kewalramani, Vertex CEO (Vertex via YouTube)

Bat­tling a line­up of skep­tics, Ver­tex claims an­oth­er ear­ly clin­i­cal win — this time in kid­ney dis­ease

Ver­tex claimed its sec­ond ear­ly-stage win of the fall Wednes­day, an­nounc­ing pos­i­tive re­sults in a small study on a ge­net­i­cal­ly de­fined form of kid­ney dis­ease.

The 16-pa­tient, Phase II tri­al fo­cused on pa­tients with fo­cal seg­men­tal glomeru­loscle­ro­sis, a rare dis­ease where kid­neys are un­able to fil­ter blood prop­er­ly. Over 13 weeks on an ex­per­i­men­tal pill, the lev­el of pro­tein in the pa­tients’ urine fell by an av­er­age of 47.6%.

Pro­tein urine lev­els, or pro­tein­uria, is a wide­ly ac­cept­ed bio­mark­er for kid­ney func­tion and a po­ten­tial pre­dic­tor for a pa­tient’s long-term risk of go­ing in­to kid­ney fail­ure.

“As a first re­sult, I think it’s ter­rif­ic,” said Jonathan Him­mel­farb, di­rec­tor of the Kid­ney Re­search In­sti­tute at the Uni­ver­si­ty of Wash­ing­ton, who was not in­volved in the study. “If it’s sus­tain­able and it re­flects im­prove­ment in an in­jury process in the kid­neys, it is po­ten­tial­ly very sig­nif­i­cant.”

Ver­tex sees the study as a proof of con­cept for its ef­forts to tack­le a larg­er set of kid­ney dis­eases dri­ven by the gene APOL1. Vari­ants of the gene, which plays a role in the im­mune sys­tem, can help boost pro­tec­tion against par­a­sitic in­fec­tions, in­clud­ing hu­man African try­panoso­mi­a­sis, al­so known as African sleep­ing sick­ness.

But pa­tients who car­ry one or es­pe­cial­ly two vari­ants are al­so at a high­er risk of de­vel­op­ing kid­ney dis­ease. Ver­tex cites fig­ures that 50% of African Amer­i­cans car­ry at least one vari­ant and 13% car­ry two. It’s one of sev­er­al fac­tors — along­side ac­cess to health­care and oth­er so­cial de­ter­mi­nants of health — that ac­count for the wide dis­par­i­ty in kid­ney dis­ease be­tween white and Black Amer­i­cans.

Ver­tex’s mol­e­cule, VX-147, is de­signed to in­hib­it APOL1 and slow kid­ney in­jury.

“It’s a very im­por­tant tar­get from a pub­lic health per­spec­tive,” Him­mel­farb said.

The da­ta, while ear­ly, will like­ly come as wel­come news for an­a­lysts, many of whom have re­mained skep­ti­cal of the com­pa­ny’s abil­i­ty to re­peat the suc­cess of its cys­tic fi­bro­sis fran­chise. In a note Tues­day, Jef­feries an­a­lyst Michael Yee placed the bar for suc­cess at a 15% re­duc­tion in pro­tein­uria, but “the high­er the bet­ter.”

The 47.6% re­duc­tion came from 13 pa­tients, with Ver­tex ex­clud­ing 3 pa­tients who it said did not com­ply with tri­al pro­to­cols. There were no se­ri­ous ad­verse events, the com­pa­ny said. The most com­mon side ef­fects were headache, back pain and nau­sea, which all oc­curred in over 15% of pa­tients.

It’s the sec­ond suc­cess­ful read­out for Ver­tex this fall af­ter land­mark re­sults on a po­ten­tial cure for type 1 di­a­betes came in Oc­to­ber, al­beit for just one pa­tient.

Ver­tex now plans to move VX-174 in­to a larg­er piv­otal tri­al for mul­ti­ple types of APOL1-me­di­at­ed kid­ney dis­eases. The com­pa­ny did not dis­close de­tails for the study, but the FDA has said that, for cer­tain kid­ney dis­eases, the agency will grant ac­cel­er­at­ed ap­proval based on re­duc­tion of pro­tein­uria, pro­vid­ing a clear path to mar­ket if ear­ly re­sults bear out in a larg­er study.

The com­pa­ny es­ti­mates 100,000 peo­ple in the US and Eu­rope are cur­rent­ly liv­ing with kid­ney dis­ease po­ten­tial­ly ad­dress­able by their mol­e­cule.

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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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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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.

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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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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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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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Steve Worland, eFFECTOR CEO

Sur­prise piv­ot rocks eF­FEC­TOR's I/O plans — al­though ex­ecs promise big­ger slice of the NSCLC mar­ket in the long run

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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