RARE Daily

Regeneron Hands Off Ex-U.S. Rights for Cholesterol Disorder Drug to Ultragenyx

January 7, 2022

Regeneron Pharmaceuticals and Ultragenyx Pharmaceutical entered into a license and collaboration agreement for Ultragenyx to clinically develop, commercialize, and distribute Evkeeza in countries outside of the United States. 

Photo: George Yancopoulos, chief scientific officer and president of Regeneron

This includes the European Economic area, where Evkeeza was approved in June 2021 as a first-in-class therapy for use together with diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies to treat adults and adolescents aged 12 years and older with homozygous familial hypercholesterolemia (HoFH).

HoFH, also known as homozygous FH, is an ultra-rare inherited condition that affects 1 in 160,000 to 300,000 people worldwide. HoFH occurs when two copies of the familial hypercholesterolemia (FH)-causing genes are inherited, one from each parent, resulting in dangerously high levels (>400 mg/dL) of LDL-C, or bad cholesterol. Patients with HoFH are at risk for premature atherosclerotic disease and cardiac events as early as their teenage years.

Evkeeza is a fully human monoclonal antibody that binds to and blocks the function of angiopoietin-like 3 (ANGPTL3), a protein that plays a key role in lipid metabolism. Regeneron scientists discovered the angiopoietin gene family more than two decades ago. Human genetics research published in New England Journal of Medicine (NEJM) in 2017 by scientists from the Regeneron Genetics Center found that patients whose ANGPTL3 gene did not function properly (called a “loss-of function mutation”) have significantly lower levels of key blood lipids, including LDL-C, and that this is associated with a significantly lower risk of coronary artery disease.

In the U.S., Evkeeza is indicated as an adjunct to other LDL-C lowering therapies for the treatment of adult and pediatric patients, aged 12 years and older with HoFH. The safety and effectiveness of Evkeeza have not been established in patients with other causes of hypercholesterolemia. The effect of Evkeeza on cardiovascular morbidity and mortality has not been determined.

Regeneron discovered and developed Evkeeza, and launched the medicine in the United States in February 2021 when it was approved by the U.S. Food and Drug Administration.

“Evkeeza is a transformational medicine for those living with homozygous familial hypercholesterolemia, as previous therapies were insufficient for many patients who still faced extremely high LDL cholesterol levels and treatment-related tolerability issues,” said George Yancopoulos, chief scientific officer and president of Regeneron. “With its focus on rare, debilitating genetic conditions, Ultragenyx is an ideal partner for us, and we look forward to working together to bring this much needed medicine to patients around the world.”

Under the terms of the agreement, Regeneron will receive a $30 million upfront payment and is eligible to receive up to $63 million in additional potential regulatory and sales milestones. Ultragenyx will receive the rights to develop, commercialize and distribute the medicine in countries outside of the U.S. and make payments to Regeneron based on net sales. Ultragenyx will share in certain costs for global trials led by Regeneron and also have the right to continue to clinically develop Evkeeza in countries outside of the United States for HoFH and other potential indications.

Regeneron has also granted Ultragenyx an exclusive option to negotiate a separate agreement to collaborate on the development and commercialization outside of the United States of Regeneron’s investigational antibody currently in phase 2/3 development for the treatment of the ultra-rare disease, fibrodysplasia ossificans progressiva (FOP) under terms to be agreed upon by both companies.

“Evkeeza utilizes a novel, potent biological mechanism to significantly reduce LDL cholesterol levels beyond historical standard of care for people with HoFH. This is a highly complementary partnership that combines Regeneron’s bold science with our proficiency in rare disease,” said Emil D. Kakkis, CEO and president of Ultragenyx. “We have developed our commercial expertise to support patient identification and access across regions and we believe we can make a real difference for the HoFH community outside of the U.S.”

Author: Rare Daily Staff

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