Ver­tex shut­ters a PhI­II cys­tic fi­bro­sis com­bo study, shares slip

Shares of Ver­tex slid Mon­day af­ter­noon af­ter the com­pa­ny said it will scrap one of its late-stage stud­ies for a com­bi­na­tion ther­a­py for cys­tic fi­bro­sis af­ter it failed to pass muster in a fu­til­i­ty analy­sis.

Ver­tex $VRTX is halt­ing its study of VX-661 and Ka­ly­de­co (iva­caftor) in peo­ple with one copy of the F508del mu­ta­tion and one copy of a mu­ta­tion that re­sults in min­i­mal CFTR pro­tein func­tion (F508del het/min). The study stum­bled in its first phase, with­out an im­prove­ment in lung func­tion.

Shares of Ver­tex slipped 3.4% on the news.

That het/min tar­get rep­re­sent­ed the largest mar­ket op­por­tu­ni­ty for Ver­tex with VX-661, ac­cord­ing to a note from Leerink’s Ge­of­frey Porges this morn­ing. But Porges al­so called it the least like­ly of the four tri­als to suc­ceed. Even so, the re­main­ing late-stage stud­ies in the VX-661 stud­ies of­fer a sub­stan­tial mar­ket op­por­tu­ni­ty. Notes Porges:

Our analy­sis and con­ver­sa­tions with MEDA­Corp spe­cial­ists sug­gest that Vx-661/Ka­ly­de­co could gen­er­ate an im­prove­ment in ppFEV1 in the range of 4-5% in the ho­mozy­gous delF508 pa­tients, com­pared to Orkam­bi’s 2-3%, with greater ef­fects on hos­pi­tal­iza­tions and ex­ac­er­ba­tions as well; Vx-661 should be eas­i­er to take (less drug-drug in­ter­ac­tions, less tol­er­a­bil­i­ty and meta­bol­ic ef­fects) and have more ef­fect than Orkam­bi; this suc­cess should trans­late in­to in­cre­men­tal rev­enue po­ten­tial that we es­ti­mate to be in the range of ~$485mm, as­sum­ing com­pa­ra­ble pric­ing to Orkam­bi in all mar­kets.

Ver­tex Chief Med­ical Of­fi­cer Jef­frey Chodake­witz

“While we rec­og­nize that peo­ple with CF with min­i­mal func­tion mu­ta­tions have a form of the dis­ease that is par­tic­u­lar­ly dif­fi­cult to treat, we be­lieved it was im­por­tant to eval­u­ate whether a dual com­bi­na­tion of VX-661 and iva­caftor could pro­vide some ben­e­fit to these pa­tients giv­en they do not to­day have a med­i­cine to treat the cause of their dis­ease,” said Ver­tex Chief Med­ical Of­fi­cer Jef­frey Chodake­witz. “These re­sults sug­gest that a triple com­bi­na­tion reg­i­men may pro­vide this group of peo­ple with CF the best chance at ob­tain­ing a mean­ing­ful ben­e­fit and we look for­ward to be­gin­ning the first study of a next-gen­er­a­tion cor­rec­tor to­geth­er with VX-661 and iva­caftor in this group of pa­tients lat­er this year, pend­ing da­ta from our on­go­ing Phase 1 stud­ies in healthy vol­un­teers.”

VX-661 is Ver­tex’s sec­ond-gen cor­rec­tor, a suc­ces­sor to the first-gen cor­rec­tor lumacaftor, in­clud­ed in the drug com­bi­na­tion for Orkam­bi. Two third-gen cor­rec­tors, VX-152 and VX-440, have been in ear­ly-stage stud­ies as Ver­tex plumbs new ap­proach­es that can treat CF bet­ter than Orkam­bi, which has suf­fered a high dropout rate. The treat­ment tar­gets the un­der­ly­ing ge­net­ic cause of the dis­ease.

The VX-661 pro­gram in­cludes four Phase III stud­ies in mul­ti­ple dif­fer­ent groups of peo­ple with CF who have at least one copy of the F508del mu­ta­tion.

Ver­tex has a $25.4 bil­lion mar­ket cap.

Tal Zaks, Moderna CMO (Moderna via YouTube)

UP­DAT­ED: NI­AID and Mod­er­na spell out a 'ro­bust' im­mune re­sponse in PhI coro­n­avirus vac­cine test — but big ques­tions re­main to be an­swered

The NIAID and Moderna have spelled out positive Phase I safety and efficacy data for their Covid-19 vaccine mRNA-1273 — highlighting the first full, clear sketch of evidence that back-to-back jabs at the dose selected for Phase III routinely produced a swarm of antibodies to the virus that exceeded levels seen in convalescent patients — typically in multiples indicating a protective response.

Moderna execs say plainly that this first stage of research produced exactly the kind of efficacy they hoped to see in humans, with a manageable safety profile.

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Trans­port Sim­u­la­tion Test­ing for Your Ther­a­py is the Best Way to As­sure FDA Ex­pe­dit­ed Pro­gram Ap­proval

Modality Solutions is an ISO:9001-registered biopharmaceutical cold chain engineering firm with unique transport simulation capabilities that support accelerated regulatory approval for biologics and advanced therapeutic medicinal products (ATMP). Our expertise combines traditional validation engineering approaches with regulatory knowledge into a methodology tailored for the life sciences industry. We provide insight and execution for the challenges faced in your cold chain logistics network.

Tillman Gerngross, Adagio Therapeutics CEO

An­ti­body leg­end Till­man Gern­gross is el­bow­ing his way in­to the Covid-19 R&D cru­sade: 'I don’t see this end­ing any­time soon'

One of the most influential — and outspoken — scientists at work in the field of antibody discovery is jumping into the frenzied race to create new therapeutics to treat and prevent Covid-19. And he’s operating with the conviction that the current outbreak now once again spreading like wildfire will create plenty of demand for what he has in mind.

Dartmouth professor and Adimab CEO Tillman Gerngross tells me he’s raised $50 million from a group of close VCs to spin out a new company — Adagio Therapeutics — with a full C-suite team assembled to hire up a staff and keep rolling toward the clinic.

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Full Bril­in­ta study re­sults show the blood thin­ner re­duces rate of sec­ondary stroke

AstraZeneca once projected its Brilinta drug to peak at $3.5 billion in sales, and though the blood thinner never reached that lofty goal, it received the latest positive signs in a string of recent good news.

The pharma released full details from its THALES study Thursday morning, which measured the effects of Brilinta and aspirin against aspirin alone in treating patients who had an acute ischemic stroke or transient ischemic attack. When taken twice daily with once-a-day aspirin for 30 days, the Brilinta combo reduced the risk of stroke and death by 17 percent, meeting the primary endpoint of the study.

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Who are the women blaz­ing trails in bio­phar­ma R&D and lead­ing the fight against Covid-19? Nom­i­nate them for End­points' spe­cial re­port

One of the many inequalities the pandemic has laid bare is the gender imbalance in biomedical research. A paper examining Covid-19 research authorship wondered out loud: Where are the women?

It’s a question that echoes beyond our current times. In the biopharma world, not only are women under-represented in R&D roles (particularly at higher levels), their achievements and talents could also be undermined by stereotypes and norms of leadership styles. The problem is even more dire for women of color.

Norbert Bischofberger, Kronos CEO

Gilead­'s ex-R&D chief Bischof­berg­er heads back to the biotech gi­ant to pick up a pair of late-stage drugs that had been put aside

Norbert Bischofberger knows entospletinib well.

Back during his long, blockbuster run as head of R&D at Gilead, researchers had once held some high hopes for this drug. But to make it work, he and the team felt it would need a new companion diagnostic to identify patients. There was talk of a combo approach to give it more punch. But the market was small, making them wonder if it would be worth going through a lengthy development cycle to get it through a pivotal.

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The $1B Mer­ck-Bay­er drug that di­vid­ed car­di­ol­o­gists in March gets pri­or­i­ty re­view

Three months after Merck published in the New England Journal of Medicine data that left doctors and investors divided over just how well its experimental heart drug worked, the FDA has handed that drug priority review. A decision is now due by January 20, 2021.

Merck first announced the drug, known as vericiguat, as a Phase III success last November. In 2016, Merck had paid $1 billion upfront for US rights to the Bayer-developed drug. Early projections foresaw a few hundred million a year in sales, but the unspecified late-stage success raised the possibility for far more. After all, Novartis’s flagship heart drug, Entresto, was earning $1.7 billion per year and was expected to reach up to $4 billion in annual sales.

GSK’s Shin­grix leader Guil­laume Pfe­fer has jumped on board Flag­ship to helm a biotech hy­brid as Afeyan’s lat­est CEO-part­ner

After spending 4 years in a senior post with GlaxoSmithKline’s star team positioning Shingrix for a blockbuster approval, Guillaume Pfefer is headed back to the biotech world — in style.

Pfefer has signed on to join Noubar Afeyan’s busy group of partners at Flagship, and he’s taking the helm of an upstart — which today is being merged with another Flagship startup — with some grand plans of its own. The announcement this morning notes that Pfefer will run Kintai Therapeutics, one of the grads of the Flagship labs.

Donald Trump and Anthony Fauci (AP Images)

Covid-19 roundup: Rus­sia hack­ers tar­get US, UK vac­cine and drug re­searchers; Fau­ci fires back at White House cam­paign to un­der­mine him

Russia has tried to steal a Covid-19 vaccine and therapeutics researcher from pharmaceutical and academic institutions in the US, UK and Canada, Britain’s National Cyber Security Centre said Thursday.

The NCSC said that hacking attempts came from a group known as APT129, also known as “Cozy Bear,” that “almost certainly operate as part of Russian intelligence services.” The Canadian Communication Security Establishment, US Department for Homeland Security, the Cybersecurity Infrastructure Security Agency, and the National Security Agency shared the assessment, the NCSC said.

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