There are count­less dis­eases lack­ing a sin­gle good treat­ment, but drug­mak­ers are still herd­ing in­to the same tar­gets, par­tic­u­lar­ly in chas­ing af­ter megablock­busters.

George Yan­copou­los, the out­spo­ken co-founder and chief sci­en­tif­ic of­fi­cer of Re­gen­eron, is one of the loud­est crit­ics of this trend. “Even if they make some­thing you take twice as less fre­quent­ly, is that what they should be do­ing?” he said in an in­ter­view. “Give me a new drug that treats a new dis­ease.”

Herd­ing is­n't new, but it has got­ten worse. On­ly 16% of top drug­mak­er­s' port­fo­lios were in crowd­ed tar­gets in 2000, a per­cent­age that quadru­pled by 2020, a McK­in­sey analy­sis found. The last few years have on­ly brought more at­ten­tion to a hand­ful of pro­grams that have pipeline-in-a-prod­uct op­por­tu­ni­ty like Keytru­da, Dupix­ent and Ozem­pic.

As the cal­en­dar turns from JPM to quar­ter­ly re­ports, earn­ings bell­wether John­son & John­son makes its re­turn for 2026. The fo­cus this time? How bio­phar­ma will re­act to a slate of most fa­vored na­tion deals with the Trump ad­min­is­tra­tion.

J&J ex­ec­u­tives es­sen­tial­ly shrugged off any fi­nan­cial im­pact from its MFN deal that was signed ear­li­er this month, say­ing it will be “even­ly dis­trib­uted through­out the year.” When com­bined with fourth-quar­ter sales that near­ly reached $25 bil­lion — up 9.1% from the same pe­ri­od a year ear­li­er — it sets up a strong 2026 for the com­pa­ny, CFO Joseph Wolk said on Wednes­day’s in­vestor call.

“We an­tic­i­pate fair­ly con­sis­tent op­er­a­tional sales growth through­out the year,” Wolk said.

In­tra­Bio said Wednes­day that its treat­ment im­proved mo­tor func­tion for a rare ge­net­ic dis­or­der called atax­ia-telang­iec­ta­sia as part of a piv­otal study.

The Austin, TX-based biotech told End­points News it plans to sub­mit an ap­pli­ca­tion to the FDA in April that would seek ap­proval for its drug, Aqneur­sa. Known gener­i­cal­ly as N-Acetyl-L-Leucine, Aqneur­sa was first ap­proved in 2024 to treat neu­ro­log­i­cal symp­toms re­lat­ed to an­oth­er rare dis­ease called Nie­mann-Pick dis­ease type C.

There are cur­rent­ly no ap­proved treat­ments specif­i­cal­ly for atax­ia-telang­iec­ta­sia.

Sev­en­ty-three pa­tients with the dis­or­der were en­rolled as part of the Phase 3 tri­al, and they re­ceived ei­ther Aqneur­sa or place­bo every day. Af­ter 12 weeks of treat­ment, pa­tients who re­ceived Aqneur­sa saw a -1.92-point im­prove­ment on a mea­sure of mo­tor func­tion called the Scale for the As­sess­ment and Rat­ing of Atax­ia, ac­cord­ing to the re­sults re­port­ed Wednes­day. Mean­while, pa­tients who re­ceived place­bo saw a -0.14 point score change. That dif­fer­ence was sta­tis­ti­cal­ly sig­nif­i­cant with a p-val­ue of p<0.001, meet­ing its pri­ma­ry end­point.

Plus, news about IO, ErVim­mune, Af­fi­body, Clasp Ther­a­peu­tics and In­fini­topes:

💊 Tan­abe Phar­ma’s pill suc­ceeds in rare blood con­di­tions: The biotech’s oral melanocortin-1 re­cep­tor de­layed the on­set of symp­toms in a Phase 3 study in pa­tients with ery­thro­poi­et­ic pro­to­por­phyr­ia and X-linked pro­to­por­phyr­ia. In these hered­i­tary dis­or­ders af­fect­ing heme for­ma­tion, pa­tients ex­pe­ri­ence se­vere skin pain when ex­posed to sun­light and some ar­ti­fi­cial light. Tan­abe said the drug, der­sime­lagon, de­layed the first hints of ear­ly symp­toms as­so­ci­at­ed with sun ex­po­sure, like burn­ing, tin­gling, itch­ing or sting­ing, ver­sus place­bo. — Eliz­a­beth Cairns