Re­gen­eron has held high hopes for evinacum­ab, a rare cho­les­terol dis­or­der drug that earned a first-in-class nod from the FDA ear­ly last year, as a cen­ter­piece of its car­diometa­bol­ic fran­chise. Now, the com­pa­ny is tap­ping an ul­tra-rare spe­cial­ist to take evinacum­ab to the next lev­el abroad.

Re­gen­eron will re­ceive $30 mil­lion in up­front cash and a po­ten­tial $63 mil­lion in down­stream mile­stones from Ul­tragenyx for ex-US li­cens­ing rights to Evkeeza (evinacum­ab), an ANGPTL3 block­er with an FDA ap­proval to treat a rare cho­les­terol dis­or­der along­side LDL-C low­er­ing ther­a­py and di­et, the com­pa­nies said Fri­day.

Ul­tragenyx, a small phar­ma known best for its ul­tra-rare dis­ease drugs, will pick up de­vel­op­ment and com­mer­cial­iza­tion costs for evinacum­ab in pa­tients with the con­di­tion known as ho­mozy­gous fa­mil­ial hy­per­c­ho­les­terolemia (HoFH), which is be­lieved to af­fect any­where be­tween 1 in 160,000 and 300,000 peo­ple world­wide.

In ad­di­tion, Re­gen­eron and Ul­tragenyx have set pre­lim­i­nary terms for an ex-US li­cens­ing deal for one of Re­gen­eron’s in­ves­ti­ga­tion­al drugs, an an­ti­body de­signed to treat the ul­tra-rare dis­ease fi­brodys­pla­sia os­si­f­i­cans pro­gres­si­va (FOP). Terms for that po­ten­tial part­ner­ship have yet to be set.

Evinacum­ab was ap­proved last Feb­ru­ary at the hefty price tag of an av­er­age of $450,000 per year. It was the first drug ap­proved to in­hib­it ANGPTL3, or an­giopoi­etin-like 3, a pro­tein in­volved in lipid me­tab­o­lism.

In piv­otal da­ta back­ing the ap­proval, pa­tients tak­ing evinacum­ab saw a 47% re­duc­tion in LDL lev­els from base­line, com­pared to a 2% in­crease in the con­trol. The drug arm al­so saw sig­nif­i­cant re­duc­tions in lev­els of apolipopro­tein B, an in­di­ca­tor of vas­cu­lar dis­ease, as well as triglyc­erides and over­all cho­les­terol.

Evinacum­ab was en­vi­sioned as a sort of nat­ur­al fol­low-up for Re­gen­eron’s Pralu­ent, a part­nered PC­SK9 block­er with Sanofi ap­proved to low­er LDL-C. That drug has been locked in a years­long pric­ing bat­tle with Am­gen’s Repatha in what has ef­fec­tive­ly be­come a war of at­tri­tion over price de­creas­es in the pricey PC­SK9 class.

Ul­tragenyx, mean­while, scored its first FDA ap­proval some five years ago with Mep­se­vii, a drug ap­proved to treat an ul­tra-rare ge­net­ic en­zyme dis­or­der called MPS VII. Since then, the drug­mak­er has earned its stature as an ul­tra-rare spe­cial­ist, bring­ing two ad­di­tion­al drugs on­to the mar­ket in Crysvi­ta, ap­proved to treat two forms of hy­pophos­phatemia, and Do­jolvi, an en­gi­neered medi­um-chain fat­ty acid de­signed as a sup­ple­ment for pa­tients with mol­e­c­u­lar­ly con­firmed long-chain fat­ty acid ox­i­da­tion dis­or­ders.

A lack of diversity in clinical trials has persisted despite decades of initiatives to try to turn the tide.

In a recent review of 17 years of clinical trials, drugmaker GSK found that there were some mismatches between the demographics of its US-based trials and how prevalent diseases were in those populations.

The results, the company says, will help GSK and others design studies that better represent epidemiological rates within races and ethnicities.

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.