Regeneron hands off ex-US rights for cholesterol disorder drug to rare disease specialist Ultragenyx
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.