A decade af­ter it first failed, a Shire-de­vel­oped Take­da an­tivi­ral ap­pears suc­cess­ful in Phase III

Near­ly 20 years af­ter it first en­tered the clin­ic and over a decade af­ter it first flunked a Phase III tri­al, a Take­da-owned drug to treat CMV in­fec­tions has proven ef­fec­tive in a late-stage study.

Take­da didn’t break down all the num­bers, but said that in a 352-per­son tri­al, marib­avir out­per­formed physi­cian’s choice in clear­ing in­fec­tions in trans­plant pa­tients who were re­sis­tant to cur­rent treat­ments. In a state­ment, Obi Umeh, Take­da’s marib­avir pro­gram leader, said they would take the da­ta to reg­u­la­tors in the US and Eu­rope.

CMV, or cy­tomegalovirus, is a com­mon virus that af­fects much of the pop­u­la­tion with­out caus­ing symp­toms, but it can be dan­ger­ous to peo­ple whose im­mune sys­tems are com­pro­mised, such as re­cip­i­ents of bone mar­row or or­gan trans­plants.

Obi Umeh

“To­day, trans­plant pa­tients who do not re­spond or ex­pe­ri­ence ad­verse events re­lat­ed to ex­ist­ing ther­a­pies may be at high­er risk for com­pli­ca­tions from the virus, in­clud­ing trans­plant fail­ure and death,” Umeh said. “Marib­avir has the po­ten­tial to re­de­fine man­age­ment of post-trans­plant CMV in­fec­tions by help­ing pa­tients clear the virus with less treat­ment lim­it­ing tox­i­c­i­ties.”

An ap­proval would give Take­da one of the first big wins from the Shire buy­out and mark the cul­mi­na­tion of a wild, two-decade ride for a drug Glax­o­SmithK­line first syn­the­sized two decades ago. In 2003, af­ter some ear­ly work in the clin­ic, the then an­tivi­ral-fo­cused Big Phar­ma li­censed the drug to the rare dis­ease and in­fec­tious dis­ease-fo­cused biotech Vi­roPhar­ma.

Vi­roPhar­ma ad­vanced the drug, with pos­i­tive re­sults, through Phase II, but in 2009, it missed the pri­ma­ry end­point on a Phase III study, fail­ing to pre­vent CMV in­fec­tions bet­ter than place­bo in pa­tients re­ceiv­ing bone mar­row trans­plants. Vi­roPhar­ma went back to the draw­ing board, even­tu­al­ly ar­gu­ing that a high­er dosage might lead to a bet­ter re­sponse.

In 2013, though, the drug left Vi­roPhar­ma’s hands and passed to Shire, which bought out the en­tire biotech for $4.2 bil­lion. The Irish drug­mak­er upped the dosage and ran the drug through a smat­ter­ing of phar­ma­co­ki­net­ic and ef­fi­ca­cy stud­ies, test­ing whether giv­ing 4 to 12 times the amount of drug might im­prove per­for­mance.

In 2016, they showed ef­fi­ca­cy in a Phase II study that 400mg, 800mg, and 1200mg helped clear in­fec­tion. Based off those re­sults, the FDA hand­ed the com­pa­ny break­through des­ig­na­tion. The most re­cent Phase III used 400 mg, or four times the dosage of the Phase III that failed in 2009.

The same year it re­ceived break­through des­ig­na­tion, the com­pound served as one of the as­sets in Take­da’s de­ci­sion to buy out Shire for $62 bil­lion. Glob­al­Da­ta pro­ject­ed that it could earn around $200 mil­lion per year in sales by 2023.

Still, key ques­tions re­main for the drug. Take­da has yet to break down the safe­ty da­ta, which ap­peared to be a con­cern in a re­cent Phase II study. Last year, the com­pa­ny an­nounced re­sults from a tri­al com­par­ing marib­avir to the Roche an­tivi­ral Val­cyte. Marib­avir was bet­ter at clear­ing in­fec­tion, but Val­cyte ap­peared safer: Two thirds of the marib­avir group saw treat­ment-re­lat­ed ad­verse events, com­pared to one fifth of Val­cyte pa­tients.

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

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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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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