United CEO Martine Rothblatt

With pipeline set­backs mount­ing, Unit­ed snares pri­or­i­ty vouch­er for in­haled for­mu­la­tion of PAH med Ty­va­so

De­spite a big lead in pul­monary ar­te­r­i­al hypterten­sion, Unit­ed Ther­a­peu­tics is un­der the gun as it looks to stave off com­peti­tors and build a suc­cess­ful ex­pan­sion over in on­col­o­gy. So far, those re­sults have been mixed. But now, Unit­ed is on the cusp of adding a new for­mu­la­tion to its suc­cess­ful Ty­va­so fran­chise that could spur growth.

For a cool price tag of $105 mil­lion, Unit­ed Ther­a­peu­tics has snared a pri­or­i­ty re­view vouch­er (PRV) for rare pe­di­atric dis­eases from Y-mAbs Ther­a­peu­tics that it in­tends to use to help take an in­haler ver­sion of its hy­per­ten­sion med Ty­va­so across the fin­ish line, the com­pa­ny said Mon­day. The vouch­er was orig­i­nal­ly in­tend­ed for Y-mAbs’ ap­pli­ca­tion for Danyelza in neu­rob­las­toma and wasn’t used for the FDA’s ap­proval in No­vem­ber.

Tag­ging Ty­va­so DPI — a drug-de­vice com­bo that us­es in­haled dry pow­der for de­liv­ery — with a PRV would short­en its FDA re­view from the req­ui­site one year to eight months, Unit­ed said. The com­pa­ny is hop­ing to start that process ear­ly next year and will chase in­di­ca­tions in PAH and pul­monary hy­per­ten­sion as­so­ci­at­ed with in­ter­sti­tial lung dis­ease.

The cur­rent­ly ap­proved ver­sion of Ty­va­so, which has the FDA’s green light in WHO Group 1 PAH and is un­der re­view to ex­pand in­to PH with in­ter­sti­tial lung dis­ease, is dosed through a more re­stric­tive neb­u­liz­er for­mu­la­tion. Adding an in­haler op­tion, Unit­ed be­lieves, would be an ob­vi­ous ad­van­tage for pa­tients who can take the drug on the go.

The new Ty­va­so for­mu­la­tion is the prod­uct of a co-de­vel­op­ment and li­cens­ing deal with MannKind Cor­po­ra­tion, dat­ing back to 2018. The Dream­boat de­vice set to be re­viewed in Ty­va­so is al­so used in MannKind’s in­haled in­sulin prod­uct Afrez­za, ap­proved back in 2014.

Unit­ed’s path to a new ap­proval for Ty­va­so and hopes for growth out­side its ag­ing PAH port­fo­lio comes af­ter one ma­jor fail­ure in that in­di­ca­tion last year and a cat­a­stro­phe in on­col­o­gy ear­li­er this year.

In April 2019 — in a terse mes­sage — Unit­ed said it would drop PAH hope­ful es­uber­aprost from its pipeline af­ter the drug flopped as a com­bo ther­a­py with Ty­va­so in a phase III head-to-head study over its old­er med Re­mod­ulin. Then, in Feb­ru­ary, Unit­ed ad­mit­ted a Phase II/III tri­al com­bin­ing their Uni­tux­in an­ti­body with a stan­dard treat­ment, irinote­can, failed to help small cell lung can­cer pa­tients live longer.

Unit­ed got Uni­tux­in, known chem­i­cal­ly as din­u­tux­imab, ap­proved for child­hood neu­rob­las­toma in 2015. They have since tried to get the orig­i­nal drug ap­proved for new can­cers, while al­so push­ing a hu­man­ized ver­sion for­ward.

De­spite be­ing an ear­ly en­trant in­to PAH af­ter Re­mod­ulin’s ap­proval way back in 2002, Unit­ed has strug­gled to ex­pand out­side that in­di­ca­tion with oth­er meds, and now com­peti­tors are an­gling for its mar­ket share. Ac­celeron, for in­stance, re­cent­ly un­veiled pos­i­tive Phase II re­sults back in Jan­u­ary for so­tater­cept, a drug they’re billing as the first to treat the cause of PAH. With im­pres­sive enough da­ta un­der its belt to pique in­ter­est, so­tater­cept could hit a sales peak of $3 bil­lion, an­a­lysts have said.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Ron Cooper, Albireo CEO

Al­bireo just ad­vanced down to the 10-yard line at the FDA. And Ron Coop­er’s team is get­ting prepped for the next big play

When Albireo Pharma’s board $ALBO moved to bring in Ron Cooper as the CEO more than 5 years ago, the development-stage company went with an experienced commercial player who had a big-time position on his resume after running Bristol Myers’ commercial ops in Europe.

Now, after successfully navigating a pivotal study, putting them in a foot race with a rival toward an FDA OK, Cooper is getting a boost from regulators on the last drive back to an arena he understands completely.

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Matt Gline (L) and Vivek Ramaswamy

Scoop: Vivek Ra­maswamy is hand­ing the CEO job to a top lieu­tenant at Roivant — but he’s not ex­act­ly leav­ing the biotech scene

Over the past 7 years since founding Roivant, Vivek Ramaswamy has been a constant blur of biotech building motion.

He launched his first biotech with an Alzheimer’s drug he picked up cheap, and watched the experiment implode in one of the highest profile pivotal disasters seen in the last decade. But it didn’t slow the 30-something exec down; if anything, he hit the accelerator. Ramaswamy blazed global paths and went on to raise billions to spur the creation of a large lineup of little Vants promising big things at a fast pace. He sold off a section of the Vant brigade to Sumitomo Dainippon for $3 billion. And more recently the relentless dealmaker has been building a computational discovery arm to add an AI-driven approach to kicking up new programs and companies, supplementing the in-licensing drive while pursuing advances that have created more than 700 jobs at Roivant, with $2 billion in reserves.

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News brief­ing: Jef­frey Lei­den to chair Tmu­ni­ty board of di­rec­tors; Op­di­vo wins new ap­proval in ad­vanced RCC

Longtime Vertex CEO Jeffrey Leiden is taking on a new role.

Leiden has been appointed chairman of Tmunity’s board of directors, the company announced Monday. The move comes about a year and a half after Leiden announced he’d be stepping down from his position at Vertex.

Vertex saw immense growth under Leiden, leading the company from its exit out of hepatitis C, when cures were moving in, and into cystic fibrosis. The company’s cystic fibrosis triple combo therapy Trikafta is already its best-seller, reaching the distinction just six weeks after launch and recording the strongest first quarter of sales for any drug, per some estimates.

IPO track­er: 2021 gets start­ed with a flur­ry of new of­fer­ings

A global pandemic couldn’t slow down what turned out to be a record year for biotech IPOs. With the calendar turning toward 2021, the Endpoints News team is prepped to track each new filing this year, and the outcome. We’re off to another hot start at least.

Below, you’ll find the companies that have filed to go public, in addition to those that have already priced. Through the first two business weeks of January, there have already been 9 biotechs that have filed or priced, and the number is only expected to grow. We’ll keep the tracker updated as it does.

Matt Gline (L) and Vivek Ramaswamy

Vivek Ra­maswamy and Matt Gline pen share­hold­er let­ters about the changes now un­der­way at Roivant

Friends and colleagues,

I am writing to provide our annual update on Roivant. These updates are usually restricted to our shareholders, but we are sharing this year’s letter more broadly to announce an upcoming change in my role from CEO to Executive Chairman and the promotion of Matt Gline to Chief Executive Officer.

Reflections on 2020

Much has transpired in the world and at our company since my last annual update in January 2020. One year ago we had just completed our $3 billion transaction with Sumitomo Dainippon Pharma (DSP), and we were evaluating how to reinvest in our business. At the same time, SARS-CoV-2 was still a distant virus barely on our minds. Today it has afflicted the entire world sparing literally no one from its effects.

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José Baselga, AstraZeneca cancer chief (Brent N. Clarke/FilmMagic via Getty Images)

As­traZeneca's Calquence nabs an­oth­er win against Im­bru­vi­ca, but Eli Lil­ly is on its heels

Three years after first launching Calquence as a second generation BTK inhibitor, AstraZeneca continues to tout new data to compete with J&J and AbbVie’s first generation blockbuster Imbruvica.

The British pharma announced on Monday that Calquence passed a head-to-head Phase III study against Imbruvica in chronic lymphocytic leukemia, proving non-inferior — i.e. just as good — as the older drug. Although AstraZeneca did not break down any of the numbers, they said the drug proved superior on safety, triggering fewer cases of atrial fibrillation, an irregular heartbeat that can lead to stroke or heart failure.

Janet Woodcock (AP Images)

End­points poll: Janet Wood­cock takes the (in­ter­im) helm at the FDA. And a large ma­jor­i­ty of our read­ers want her to stay there

It’s official: Janet Woodcock is now the acting chief of the FDA.

And — according to an Endpoints poll — most industry readers would like her to stay there, although a significant minority is strongly opposed.

To recap: Joe Biden is reportedly choosing between Woodcock and former deputy FDA commissioner Joshua Sharfstein as his nominee for the permanent position. Given their respective track records, the decision is set to determine the agency’s lodestar for years to come.

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