Zevra adds Edsivo for vEDS to its rare disease portfolio in new deal

Company acquires Acer, Phase 3 program of therapy for COL3A1 mutations

Andrea Lobo, PhD avatar

by Andrea Lobo, PhD |

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Zevra Therapeutics is adding Edsivo (celiprolol), an experimental therapy for vascular Ehlers-Danlos syndrome (vEDS), to its clinical pipeline of rare diseases, as part of an agreement in which the company will acquire Acer Therapeutics,

The new deal means Zevra will take over the ongoing Phase 3 program testing the treatment candidate in people with vEDS caused by mutations in the COL3A1 gene. That program was announced last year by Acer, which had developed the therapy.

Edsivo has the potential to reduce the risk of arterial rupture, or events in other internal organs such as the intestine, in patients with these mutations.

“Acer would bring unique rare disease operations and capabilities that would serve as a foundation to support the commercialization of Zevra’s pipeline as it advances,” Joshua Schafer, chief commercialization officer and executive vice president of business development at Zevra, said in a press release.

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Ehlers-Danlos syndrome (EDS) includes genetic disorders that affect the body’s connective tissue, which provides structure to blood vessels, skin, and different organs.

vEDS, the most severe form of EDS, is generally caused by mutations in the COL3A1 gene that result in reduced collagen production. The disease is characterized by thin, easily bruised skin, as well as fragile arteries, muscles, and organs.

According to Acer, the disease is estimated to affect 7,500 people in the U.S., based on patient databases and U.S. population data.

There are no approved pharmacological therapies to treat vascular EDS, and treatment focuses mainly on addressing symptoms and preventive measures.

However, in Europe, celiprolol, a beta-1 receptor antagonist approved to treat high blood pressure, is used off-label — meaning a drug used for diseases it’s not approved to treat — as the standard of care for people with vEDS, according to Acer.

The therapy works by promoting the production of collagen in blood vessels, easing the pressure in those that are prone to rupture.

As a possible therapy for people with vEDS, Edsivo was tested in the BBEST Phase 4 clinical trial (NCT00190411), which wrapped up in 2011. That trial showed that Edsivo could reduce the risk of arterial, intestinal, and uterine rupture in these patients.

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Supported by the trial’s findings, Acer in 2018 requested the approval of Edsivo from the U.S. Food and Drug Administration (FDA) for treating vEDS. That application was rejected by the agency, which cited a need for additional data from an adequate and well-controlled trial demonstrating Edsivo’s efficacy. A formal appeal of the rejection was denied.

In 2022, Edsivo was granted breakthrough therapy designation by the FDA for vEDS with mutations in the COL3A1 gene. This designation is meant to accelerate the development of medicines aiming to treat a serious condition.

Moreover, the FDA also approved the pivotal Phase 3 DiSCOVER clinical trial testing Edsivo in vEDS patients with COL3A1 mutations. The primary goal of the trial, in which Acer planned to enroll 150 patients, is to determine whether Edsivo can effectively reduce the occurrence of vEDS clinical events requiring medical intervention, compared with a placebo.

The newly announced merger transaction for Acer’s acquisition includes nearly 3 million shares of Zevra common stock, valued at $15 million. It also spells out potential cash payments upon the achievement of certain regulatory and commercial milestones for Edsivo, among other therapies developed by Acer.

“This merger would support Zevra’s vision of becoming a leading rare disease company bringing life-changing therapies to patients with a significant unmet need,” said Christal Mickle, interim CEO and chief development officer at Zevra.