Reshma Kewalramani, Vertex CEO (Vertex)

A year out from FDA ap­proval, Ver­tex looks to ex­pand top-sell­ing CF drug to pe­di­atrics

About a year af­ter get­ting the FDA thumbs-up for its block­buster cys­tic fi­bro­sis drug Trikaf­ta, Ver­tex is pick­ing up where it left off.

The Boston-based biotech read out pos­i­tive re­sults from a Phase III study in­volv­ing chil­dren ages 6 to 11, and an­nounced plans to file for a sup­ple­men­tal NDA lat­er this year. Pre­vi­ous­ly, the triplet reg­i­men was on­ly avail­able to pa­tients 12 and up.

Trikaf­ta, Ver­tex’s $VRTX fourth CF drug, is de­signed for those with at least one F508del mu­ta­tion in the CFTR gene. It cov­ers about 90% of CF pa­tients, un­like the com­pa­ny’s Ka­ly­de­co, which on­ly cov­ers 6% of Amer­i­cans with CF, ac­cord­ing to a STAT re­port. With­in weeks of Trikaf­ta’s Oc­to­ber 2019 launch, it be­came Ver­tex’s top-sell­ing drug. Quar­ter­ly US sales hit $420 mil­lion by the end of Jan­u­ary.

The win al­lowed for­mer CEO Jef­frey Lei­den to step down on a high note, pass­ing the torch to cur­rent CEO Resh­ma Ke­wal­ra­mani. Lei­den shift­ed over to ex­ec­u­tive chair­man of the com­pa­ny.

Car­men Boz­ic

“Our aim is to ex­tend el­i­gi­bil­i­ty to all pa­tients who may ben­e­fit from this trans­for­ma­tive med­i­cine, and the pos­i­tive re­sults from the study in chil­dren ages 6 through 11 years old al­lows us to take an­oth­er step for­ward to­ward this goal,” Car­men Boz­ic, Ver­tex CMO and EVP of glob­al med­i­cines de­vel­op­ment and med­ical af­fairs, said in a state­ment.

CF is char­ac­ter­ized by the build-up of sticky mu­cus in the lungs, di­ges­tive sys­tem and oth­er or­gans. It’s caused by a de­fec­tive pro­tein that re­sults from mu­ta­tions in the CFTR gene — the most com­mon mu­ta­tion be­ing F508del. Ac­cord­ing to Ver­tex, a Phase III study with 66 chil­dren pro­duced safe­ty da­ta “con­sis­tent with those ob­served in pre­vi­ous Phase III stud­ies.”

The drug cleared pri­ma­ry safe­ty and tol­er­a­bil­i­ty end­points, and showed “mean­ing­ful im­prove­ment” across sec­ondary ef­fi­ca­cy end­points, in­clud­ing per­cent pre­dict­ed forced ex­pi­ra­to­ry vol­ume in 1 sec­ond (ppFEV 1, a mark­er of cys­tic fi­bro­sis lung dis­ease pro­gres­sion), sweat chlo­ride, Cys­tic Fi­bro­sis Ques­tion­naire Re­vised (CFQ-R) res­pi­ra­to­ry do­main score, and body mass in­dex (BMI) through 24 weeks of treat­ment.

De­spite the an­nounce­ment, Ver­tex’s shares dropped 2.8% on Thurs­day, clos­ing at $255.65 per share.

In April, the com­pa­ny faced crit­i­cism from cost-ef­fec­tive­ness watch­dog ICER that its CF drugs were too ex­pen­sive. Trikaf­ta is priced at more than $311,000 an­nu­al­ly, or around $24,000 for a month’s sup­ply. ICER sug­gest­ed dis­counts of up to 77% to match the prices of Ver­tex’s CF drugs with their clin­i­cal val­ue.

“De­spite be­ing trans­for­ma­tive ther­a­pies, the prices set by the man­u­fac­tur­er – cost­ing many mil­lions of dol­lars over the life­time of an av­er­age pa­tient – are out of pro­por­tion to their sub­stan­tial ben­e­fits,” ICER CMO David Rind said in a state­ment. “When a man­u­fac­tur­er has a mo­nop­oly on treat­ments and is aware that in­sur­ers will be un­able to refuse cov­er­age, the lack of usu­al coun­ter­bal­anc­ing forces can lead to ex­ces­sive prices,” he added lat­er.

Days af­ter Trikaf­ta’s FDA ap­proval, Ab­b­Vie an­nounced plans to snag a port­fo­lio of CF drugs from Gala­pa­gos in a $245 mil­lion deal, and bring in a pre­clin­i­cal CFTR po­ten­tia­tor com­pound from the Cys­tic Fi­bro­sis Foun­da­tion. The Gala­pa­gos da­ta looked weak com­pared to Ver­tex’s, but Ab­b­Vie seemed stead­fast. It cur­rent­ly has a com­bo with one po­ten­tia­tor and two cor­rec­tor mol­e­cules in Phase I de­vel­op­ment.

Tim Van Hauwermeiren, argenx CEO

Ar­genx pur­chas­es $100M+ FDA pri­or­i­ty re­view vouch­er from blue­bird bio

Argenx’s Vyvgart is due for a speedy review at the FDA, thanks to a $102 million priority review voucher (PRV).

The Netherland-based biotech picked up the PRV from bluebird bio, the companies announced on Wednesday. PRVs shorten a drug’s FDA review period from 10 months to 6 months, though they often sell on the open market for around $100 million each.

Argenx plans on using the express ticket on efgartigimod, its neonatal Fc receptor (FcRn) blocker marketed as Vyvgart for adults with generalized myasthenia gravis (gMG). While Vyvgart won its first approval last December for the chronic neuromuscular disease — which is characterized by difficulties with facial expression, speech, swallowing and breathing — CEO Tim Van Hauwermeiren said in a news release that he plans to “be active in fifteen disease targets by 2025.”

Susan Galbraith, AstraZeneca EVP, oncology R&D, at EUBIO22 (Rachel Kiki for Endpoints News)

Up­dat­ed: As­traZeneca jumps deep­er in­to cell ther­a­py 2.0 space with $320M biotech M&A

Right from the start, the execs at Neogene had some lofty goals in mind when they decided to try their hand at a cell therapy that could tackle solid tumors.

Its founders have helped hone a new approach that would pack in multiple neoantigen targets to create a personalized TCR treatment that would not just make the leap from blood to solid tumors, but do it with durability. And they managed to make their way rapidly to the clinic, unveiling their first Phase I program for advanced tumors just last May.

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Ei­sai’s ex­pand­ed Alzheimer’s da­ta leave open ques­tions about safe­ty and clin­i­cal ben­e­fit

Researchers still have key questions about Eisai’s investigational Alzheimer’s drug lecanemab following the publication of more Phase III data in the New England Journal of Medicine Tuesday night.

In the paper, which was released in conjunction with presentations at an Alzheimer’s conference, trial investigators write that a definition of clinical meaningfulness “has not been established.” And the relative lack of new information, following topline data unveiled in September, left experts asking for more — setting up a potential showdown to precisely define how big a difference the drug makes in patients’ lives.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

Twit­ter dis­ar­ray con­tin­ues as phar­ma ad­ver­tis­ers ex­tend paus­es and look around for op­tions, but keep tweet­ing

Pharma advertisers on Twitter are done — at least for now. Ad spending among the previous top spenders flattened even further last week, according to the latest data from ad tracker Pathmatics, amid ongoing turmoil after billionaire boss Elon Musk’s takeover now one month ago.

Among 18 top advertisers tracked for Endpoints News, only two are spending: GSK and Bayer. GSK spending for the full week through Sunday was minimal at just under $1,900. Meanwhile, German drugmaker Bayer remains the industry outlier upping its spending to $499,000 last week from $480,000 the previous week. Bayer’s spending also marks a big increase from a month ago and before the Musk takeover, when it spent $16,000 per week.

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Vi­a­tris with­draws ac­cel­er­at­ed ap­proval for top­i­cal an­timi­cro­bial 24 years lat­er

After 24 years without confirming clinical benefit, the FDA announced Tuesday morning that Viatris (formed via Mylan and Pfizer’s Upjohn) has decided to withdraw a topical antimicrobial agent, Sulfamylon (mafenide acetate), after the company said conducting a confirmatory study was not feasible.

Sulfamylon first won FDA’s accelerated nod in 1998 as a topical burn treatment, with the FDA noting that last December, Mylan told the agency that it wasn’t running the trial.

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Emily Leproust, Twist Bioscience CEO

Twist Bio­science’s 'fac­to­ry of the fu­ture' in Ore­gon could de­liv­er with com­pet­i­tive pric­ing, SVB Se­cu­ri­ties says

The synthetic DNA manufacturer Twist Bioscience has given a peek behind the curtain to several analysts into its “factory of the future” as well as insight into the cost structure, workflow and technology at the site.

The 110,000-square-foot manufacturing site in the city of Wilsonville, OR, just south of Portland, which was announced back in 2020, will double Twist’s production capacity and bring around 400 jobs to the area.

Paul Hudson, Sanofi CEO (Romuald Meigneux/Sipa via AP Images)

Sanofi and DN­Di aim to elim­i­nate sleep­ing sick­ness in Africa with promis­ing Ph II/III re­sults for new drug

The Drugs for Neglected Diseases initiative (DNDi) and Sanofi today said that their potential sleeping sickness treatment saw success rates of up to 95% from a Phase II/III study investigating the safety and efficacy of single-dose acoziborole.

The potentially transformative treatment for sleeping sickness would mainly be targeted at African countries, according to data published today in The Lancet Infectious Diseases medical journal. The clinical trial was led by DNDi and its partners in the Democratic Republic of the Congo (DRC) and Guinea, with the authors noting:

Digital render of CPI's Medicines Manufacturing Innovation Centre in Glasgow, Scotland (Image:

CPI opens the doors to a new $100M+ man­u­fac­tur­ing fa­cil­i­ty in Scot­land

A manufacturing site that has received interest and investments from large pharma companies and the UK government is opening its doors in Scotland.

The manufacturer CPI (Centre for Process Innovation) has opened a new £88 million ($105 million) “Medicines Manufacturing Innovation Centre” in Glasgow, Scotland, to accelerate the development of manufacturing tech and solve longstanding challenges in medicine development and manufacturing.

Lil­ly's Covid-19 mAb no longer au­tho­rized due to Omi­cron sub­vari­ants, FDA says

The FDA on Wednesday announced that Eli Lilly’s Covid-19 drug bebtelovimab is no longer authorized to treat Covid-19 because of the rising numbers of two new subvariants that the drug does not work against.

The Centers for Disease Control and Prevention last week published new estimates that the combined proportion of Covid-19 cases caused by the Omicron subvariants BQ.1 and BQ.1.1 are greater than 57% nationally, and already above 50% in all individual regions but one.