→ A long­time re­searcher in car­diac re­gen­er­a­tive med­i­cine has closed a $26 mil­lion Se­ries B to ad­vance his de­vel­op­ment of iP­SC-de­rived car­diomy­ocytes as a treat­ment for heart fail­ure. Kei­ichi Fuku­da con­vinced a num­ber of new in­vestors, in­clud­ing SBI In­vest­ment, JMDC, Gene Tech­no Sci­ence, Nis­say Cap­i­tal and SM­BC Cap­i­tal, to join Astel­las Ven­ture Man­age­ment in back­ing Tokyo-based Heart­seed. The biotech ex­pects to launch its own Phase I/II for the lead drug in HFrEF late next year, while sup­port­ing an­oth­er tri­al for di­lat­ed car­diomy­opa­thy ini­ti­at­ed by Keio Uni­ver­si­ty.

→ Gen­e­sis Ther­a­peu­tics — a spin­out of Vi­jay Pande’s lab at Stan­ford join­ing the rush of putting AI tech to work on drug dis­cov­ery and de­vel­op­ment — has bagged $4.1 mil­lion in seed fund­ing led by An­dreessen Horowitz with Fe­li­cis Ven­tures as a ma­jor in­vestor.

Gen­e­sis founder Evan Fein­berg told End­points News they dif­fer­en­ti­ate them­selves from oth­er AI com­pa­nies by build­ing a neur­al net­work specif­i­cal­ly tai­lored to­wards bio­phar­ma, rather than one based from im­age pro­cess­ing or oth­er forms of AI.

“For many years, both aca­d­e­mics and com­pa­nies work­ing on ma­chine learn­ing for small mol­e­cule drug dis­cov­ery “cut and past­ed” AI tech­niques from these oth­er do­mains of com­put­er vi­sion and nat­ur­al lan­guage and ap­plied them to chem­istry, and many still do,” He wrote in an email to End­points.  “How­ev­er, in our re­search, we re­al­ized that small mol­e­cule drug can­di­dates, which are formed of atoms and bonds, could be more rich­ly rep­re­sent­ed, and those rep­re­sen­ta­tions could be bet­ter lever­aged, by de­vis­ing neur­al net­works that di­rect­ly re­flect sym­me­tries of na­ture”

→ Af­ter pro­vid­ing an up­date on its on­go­ing Phase II study of vosori­tide in chil­dren with achon­dropla­sia, Bio­Marin Phar­ma­ceu­ti­cal has an­nounced that they’ve sub­mit­ted a mar­ket­ing au­tho­riza­tion ap­pli­ca­tion (MAA) to the EMA for its in­ves­ti­ga­tion­al gene ther­a­py, val­oc­toco­gene rox­a­parvovec, for adults with se­vere he­mo­phil­ia A. The com­pa­ny an­tic­i­pates the re­view to take place in Jan­u­ary 2020 un­der ac­cel­er­at­ed as­sess­ment.

A small, Santa Ana-based biotech created in 2017 is looking to enter a SPAC deal as it lays out plans to begin trials in its lead cell therapy candidates and bring on new executives.

Graf Acquisition Corp. IV and NKGen Biotech announced Thursday, with few other details, that the two companies signed a non-binding letter of intent to “pursue a business combination.” Graf Acquisition II and III withdrew their IPOs last year.

The FDA has rejected Incyte’s extended-release formulation of ruxolitinib tablets, in a surprise setback for the company’s plans to build on its blockbuster Jakafi franchise.

The ruxolitinib XR tablets are designed to be taken once a day, whereas Jakafi is indicated for twice daily dosage (although some patients can take it once daily).

According to Incyte, the FDA acknowledged in its complete response letter that the study submitted in the NDA “met its objective of bioequivalence based on area under the curve (AUC) parameters but identified additional requirements for approval.”

Cyclerion Therapeutics may have the design of a Phase IIb study ready to go, but it’s scrambling for a way to fund it.

The company said in a press release that it’s “actively evaluating the best combination of capital, capabilities, and transactions available to it to advance the development of zagociguat,” its lead candidate for a rare, genetic mitochondrial disease known as MELAS.

In a separate SEC filing, Cyclerion once again flagged “substantial doubt about (its) ability to continue as a going concern.” As of the end of 2022, it had cash and cash equivalents of only $13.4 million.