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13 March, 2026
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7 Re­al-World Ways RWE Is Trans­form­ing Health­care
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top stories
1. Roivant CEO, bullish on growth, isn't waiting for a 'mercurial master' to buy up assets
2.
peer review
Flagship startup loses CEO to Ipsen; Ionis to shed nearly six decades of board experience
3. A LAG-3 pioneer fails in Phase 3, pouring more cold water on a once-hot cancer target
4. Ultragenyx’s gene therapy hits key endpoint in Phase 3 trial for blood ammonia disease
5. Danish biotech Gubra plots Roivant-like model to churn out new companies
more stories
 
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Alexis Kramer
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Nominations are now open for Endpoints’ annual LGBTQ+ Leaders in Biopharma report. Have someone in mind? You can nominate them here.

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Alexis Kramer
Editor, Endpoints News
Roivant CEO Matt Gline at #BIO24 (Brian Benton Photography)
1
by Max Bayer

These days, if you ask the CEO of Roivant if he’s open to sell­ing an­oth­er late-stage drug to a larg­er com­pa­ny, he refers you to a spoof video clip.

It’s the scene from "Toy Sto­ry" where the alien toys are watch­ing the ar­cade claw de­scend up­on them, and Woody and Buzz Lightyear are try­ing to es­cape the ma­chine. In the video, the aliens are la­beled as biotechs ripe for a buy­out, the claw is big phar­ma and Woody is Roivant.

Matt Gline’s point? Roivant isn’t beg­ging for a buy­er.

“Build­ing busi­ness­es sit­ting around wait­ing for some oth­er mer­cu­r­ial mas­ter to come around and de­cide whether you're good enough for their ap­peal or not is, in ad­di­tion to be­ing a bad way to build busi­ness­es, it's just bor­ing,” the CEO said in an in­ter­view at the com­pa­ny’s Man­hat­tan head­quar­ters.

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Peer Review: Weekly biopharma job report
2
by Alex Hoffman, Kyle LaHucik, Max Bayer

Michelle Wern­er has stepped down as CEO of Flag­ship start­up All­tr­na, say­ing on LinkedIn that the de­ci­sion "is the right one for me and my fam­i­ly," and she's head­ed to Ipsen as pres­i­dent of North Amer­i­ca on March 23. She clos­es out a four-year tenure af­ter join­ing All­tr­na and Flag­ship from No­var­tis, where she was the glob­al fran­chise head for sol­id tu­mors. Wern­er had worked on ad­vanc­ing a pipeline of tR­NA as­sets at the start­up, with a lead pro­gram cur­rent­ly in IND-en­abling stud­ies.

It's un­clear who may re­place Wern­er at All­tr­na, if any­one. But CFO Joanne Protano has been named pres­i­dent of the com­pa­ny fol­low­ing the de­ci­sion. Lo­visa Afzelius is al­so slight­ly shift­ing po­si­tions, now the ex­ec­u­tive chair of All­tr­na. Wern­er will con­tin­ue to be a mem­ber of the board as well as a com­pa­ny ad­vi­sor.

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3
by Andrew Dunn

A few years ago, the on­col­o­gy world won­dered if it had found its next big break­through: a third ma­jor check­point in­hibitor be­yond PD-1 and CT­LA-4.

The FDA in 2022 ap­proved Bris­tol My­ers Squib­b's LAG-3-tar­get­ing Op­du­alag. Many drug­mak­ers hoped that would be the start of a new class of can­cer im­munother­a­py. But there have been few pos­i­tive up­dates in the LAG-3 world since then.

A tiny Aus­tralian biotech added to the list of fail­ures on Fri­day. Im­mutep said its lead LAG-3 drug can­di­date flunked an in­ter­im fu­til­i­ty analy­sis for a Phase 3 lung can­cer study, trig­ger­ing plans to wind down the tri­al.

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Drug Discovery Day 2026
AI-driven drug discovery is already reshaping pipelines. But algorithms alone won’t get a drug to the clinic. Join us for a free virtual program — then continue the conversation at an in-person only fireside chat and happy hour in Boston. Reserve your spot.
4
by Ayisha Sharma

Ul­tragenyx said its gene ther­a­py can­di­date for a rare meta­bol­ic dis­ease has hit one of two pri­ma­ry end­points in a late-stage study.

The drug is be­ing test­ed in 37 peo­ple with or­nithine tran­scar­bamy­lase de­fi­cien­cy, a rare ge­net­ic dis­ease. Pa­tients have a mu­ta­tion in the OTC gene which, in healthy peo­ple, makes an en­zyme that turns am­mo­nia in­to urea for ex­cre­tion through urine. But in peo­ple with the mu­ta­tion, the com­plete or par­tial lack of the en­zyme leads to the buildup of am­mo­nia in the blood to dan­ger­ous and po­ten­tial­ly fa­tal lev­els.

Ul­tragenyx's ther­a­py, called DTX301, de­liv­ers a func­tion­al copy of the OTC gene. In the Phase 3 tri­al, it led to an 18% re­duc­tion in 24-hour plas­ma am­mo­nia com­pared with place­bo at 36 weeks, the com­pa­ny said in a press re­lease. Ul­tragenyx called the re­sult “sta­tis­ti­cal­ly sig­nif­i­cant and clin­i­cal­ly mean­