In a star­tling de­ci­sion, No­vo Nordisk CEO Lars Fruer­gaard Jør­gensen will step down as the com­pa­ny deals with pres­sures on its stock price and GLP-1 busi­ness.

No­vo said Fri­day it start­ed a search for Jør­gensen’s suc­ces­sor, and Jør­gensen will con­tin­ue as CEO un­til one is found. No time­line has been an­nounced.

Jør­gensen’s sur­prise de­par­ture was made in light of “re­cent mar­ket chal­lenges” and the de­cline of No­vo Nordisk’s stock price since mid-2024, ac­cord­ing to a press re­lease. The board of No­vo’s par­ent com­pa­ny, the No­vo Nordisk Foun­da­tion, al­so “ex­pressed a wish” to in­crease its rep­re­sen­ta­tion on No­vo’s board.

Bio­Marin, mul­ti­ple quar­ters in­to its port­fo­lio re­fine­ment, is ink­ing a small ac­qui­si­tion for a rare dis­ease ther­a­py about two years be­fore a po­ten­tial reg­u­la­to­ry ap­proval.

The Cal­i­for­nia drug­mak­er plans to buy Boston-based In­ozyme Phar­ma and its Phase 3 en­zyme re­place­ment ther­a­py for about $270 mil­lion, the com­pa­nies said Fri­day. In­ozyme an­tic­i­pates the first piv­otal tri­al read­out for its ex­per­i­men­tal med­i­cine, dubbed INZ-701, in "ear­ly 2026" and a po­ten­tial launch in 2027.

Bio­Marin Chief Busi­ness Of­fi­cer James Sabry told End­points News that the ac­qui­si­tion is "the be­gin­ning of many deals. You'll see more this year."

In­ozyme is test­ing INZ-701 for in­fants, chil­dren and adults with ENPP1 de­fi­cien­cy, a rare, ge­net­ic con­di­tion that im­pairs blood ves­sels, soft tis­sues and bones. It is as­so­ci­at­ed with height­ened car­dio­vas­cu­lar mor­tal­i­ty, the com­pa­nies said.

NEW OR­LEANS – In an ear­ly-stage clin­i­cal study, three peo­ple with a ge­net­ic heart con­di­tion saw im­prove­ments in heart func­tion af­ter re­ceiv­ing a ther­a­py de­vel­oped by Rock­et Phar­ma­ceu­ti­cals.

It’s the first clin­i­cal da­ta on Rock­et’s gene ther­a­py for what’s called plakophilin-2-re­lat­ed ar­rhyth­mo­genic car­diomy­opa­thy, a heart mus­cle dis­ease caused by mu­ta­tions to the code for a key cell struc­ture pro­tein.

All three pa­tients who re­ceived the gene ther­a­py, known as RP-A601, saw im­prove­ment in their heart’s abil­i­ty to pump blood to the lungs, as well as im­prove­ments in typ­i­cal mea­sures used in heart dis­ease clin­i­cal tri­als, in­clud­ing the Kansas City Car­diomy­opa­thy Ques­tion­naire and New York Heart As­so­ci­a­tion Class.