Regeneron hands over Evkeeza rights to Ultragenyx outside US, with an R&D asset also thrown in

regeneron
Regeneron will continue its work in the U.S., which includes developing Evkeeza for children with homozygous familial hypercholesterolemia as well as adult patients with severe hypertriglyceridemia. (Regeneron)

After nabbing U.S. and European approvals in the past year, Regeneron is shipping the sales and development rights to its ultrarare cholesterol drug Evkeeza over to rare disease specialist Ultragenyx.

The drug launched in the U.S. just under a year ago after nabbing an FDA green light to lower the sky-high cholesterol seen in homozygous familial hypercholesterolemia (HoFH), an ultrarare genetic condition that causes “bad” fats in the blood to shoot up, dramatically raising the risk of heart issues at a young age and fatality before a patient hits 30.  

It affects around four people in every 1 million, and, because of the scarcity of patients, the drug’s cost is enormous, with a list price of about $450,000 per year on average.

Regeneron has another HoFH therapy, PCSK9 drug Praluent, which nabbed approval in the U.S. last April. Both Praluent and Evkeeza rival Amgen’s PCSK9 Repatha, which had that indication before Regeneron.

Regeneron is keen, however, to point out that Evkeeza is a first of its kind therapy and works on a completely different pathway from PCSK9 drugs; thus, it hopes to eke out an advantage in the market.  

The drug also grabbed an approval in Europe last summer, but today the pharma announced it would be handing over Evkeeza rights there and elsewhere outside the U.S. to Ultragenyx.

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The deal breaks down like this: Regeneron gets $30 million upfront from the biopharma but can also look forward to $63 million in biobucks and sales milestones, with Ultragenyx also making payments to Regeneron based on net sales. Ultragenyx can work on the drug outside of the U.S. for HoFH and “other potential indications,” according to a statement.

There’s also a bonus asset thrown into the mix, with Regeneron offering Ultragenyx the opportunity to work on a late-stage antibody, also for ultrarare disease, namely fibrodysplasia ossificans progressiva (FOP), “under terms to be agreed upon by both companies.” The companies did not give further details of the therapy or financials involved.

Ultragenyx has several of its own rare disease drugs including FGF23 hormone blocker Crysvita for tumor-induced osteomalacia and X-linked hypophosphatemia as well as Dojolvi, a metabolic supplement for a rare group of disorders, and Mepsevii to treat children and adults with mucopolysaccharidosis VII.

RELATED: Regeneron's Evkeeza, carrying big price tag, wins FDA approval in ultra-rare cholesterol disease

While clearly wanting the focus in the U.S., and the sales, selling an ultrarare drug outside the country requires a lot of work, contacts and ability to help find and diagnose these patients. Many patients can go years without a diagnosis, all within the constraints of differing marketing challenges, such as in Europe, where direct-to-consumer marketing is not allowed.

The best thing to do? Hire a specialist. A Regeneron spokesperson told Fierce Pharma Marketing that its choice of partner made all the difference. “While we are growing our ex-U.S. presence, in the case of Evkeeza, we saw real benefit with partnering with Ultragenyx, which has a focus on serious genetic diseases and deep relationships in countries outside of the U.S.”

Regeneron said it has “been actively engaging in discussions regarding current needs and efforts underway and are working closely together to ensure an expedited onboarding and smooth transition of responsibilities outside of the United States.”

In the U.S., the pharma is also working on Evkeeza in children with HoFH as well as adult patients with severe hypertriglyceridemia, a more common condition in which triglyceride levels are elevated, to help prevent of recurrent acute pancreatitis.

Whilst nabbing the EU approval last year, the drug is yet to launch, and UltraGenyx isn't expecting revenues from the drug this year. The focus, the company told us, was securing reimbursement across Europe.

Analysts at Jefferies said in a note to clients that: "Though we do not model yet, Evkeeza mkt oppty likely to be modest ex-U.S.; for context, current peak consensus U.S. est is $276M in 2030."