UP­DAT­ED: Take­da suc­ceeds where oth­er phar­mas have failed with PhI­II win for cy­tomegalovirus castoff marib­avir

Marib­avir, once a tri­al dud and mul­ti­ple-time castoff, re­ceived new life from the FDA right be­fore Take­da picked up Shire in 2019. Now, Take­da is post­ing win­ning late-stage da­ta from the drug that could help it suc­ceed where so many oth­ers have failed.

In a 352-per­son tri­al com­par­ing marib­avir to physi­cian’s choice an­tivi­rals, more than twice as many pa­tients on marib­avir achieved CMV viremia clear­ance at eight weeks — the study’s pri­ma­ry end­point, ac­cord­ing to Take­da. Of the 235 pa­tients who re­ceived marib­avir, 131 (or 55.7%) achieved clear­ance ver­sus 28 of 117 (or 23.9%) of those on con­ven­tion­al an­tivi­ral ther­a­pies (p<0.001). Viremia is the iso­la­tion of CMV by cul­ture.

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

Vol­un­teers in Take­da’s open-la­bel tri­al, dubbed SOL­STICE, ran­dom­ly re­ceived ei­ther marib­avir, or one or a com­bi­na­tion of com­mon­ly used an­tivi­ral ther­a­pies like gan­ci­clovir, val­gan­ci­clovir, fos­car­net or cid­o­fovir. The par­tic­i­pants, who were hematopoi­et­ic cell trans­plant and sol­id or­gan trans­plant re­cip­i­ents, were treat­ed for eight weeks, then fol­lowed for an­oth­er 12.

Break­ing it down fur­ther, Take­da said 55.6% of sol­id or­gan trans­plant pa­tients on marib­avir saw CMV viremia clear­ance at week eight, com­pared with 26.1% of those on oth­er ther­a­pies. And of the hematopoi­et­ic cell trans­plant pa­tients, 55.9% achieved clear­ance at week eight com­pared to 20.8% on oth­er drugs.

In ad­di­tion to the pri­ma­ry end­point, Take­da says it met a key sec­ondary end­point: im­prove­ment in the clear­ance of CMV viremia and as­so­ci­at­ed symp­tom con­trol through four months And those who took marib­avir showed a low­er in­ci­dence of treat­ment-re­lat­ed tox­i­c­i­ties com­mon with oth­er an­tivi­rals, ac­cord­ing to the com­pa­ny.

Treat­ment-re­lat­ed se­ri­ous ad­verse events led to the deaths of one pa­tient in each treat­ment group, ac­cord­ing to Take­da, though it’s un­clear what caused the deaths. As of press time, the com­pa­ny de­clined to pro­vide more in­for­ma­tion.

Take­da of­fered a first glimpse at the Phase III re­sults back in De­cem­ber, and an­nounced that it would take the da­ta to reg­u­la­tors in the US and Eu­rope. In an emailed state­ment, Take­da said it plans to file with the FDA in the first half of FY 2021.

An ap­proval would be a sweet end to marib­avir’s wild-two-decade ride. Glax­o­SmithK­line first syn­the­sized the drug 20 years ago and did some ear­ly clin­i­cal work, be­fore li­cens­ing it to the rare dis­ease and in­fec­tious dis­ease-fo­cused biotech Vi­roPhar­ma.

Vi­roPhar­ma suc­cess­ful­ly took it through Phase II, then missed the pri­ma­ry end­point in a Phase III study in 2009. Marib­avir failed to pre­vent CMV in­fec­tions bet­ter than place­bo in pa­tients re­ceiv­ing bone mar­row trans­plants. Vi­roPhar­ma sug­gest­ed a high­er dose might lead to a bet­ter re­sponse — but end­ed up pass­ing the can­di­date to Shire dur­ing a $4.2 bil­lion buy­out in 2013.

Shire ran with Vi­roPhar­ma’s idea, giv­ing 4 to 12 times more drug in var­i­ous phar­ma­co­ki­net­ic and ef­fi­ca­cy stud­ies. They got the re­sults they were look­ing for in 2016, prov­ing in a Phase II study that 400mg, 800mg, and 1200mg helped clear in­fec­tion. The FDA com­mend­ed the re­sults with a break­through des­ig­na­tion in 2018. And the fol­low­ing year, Take­da com­plet­ed its ac­qui­si­tion of Shire for $62 bil­lion.

Marib­avir has come a long way in­deed, but it’s not over the goal line just yet.

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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