Companies collaborating on Stargardt disease program

Aequus Pharmaceuticals Inc. and reVision Therapeutics Inc. will collaborate to develop a therapy for Stargardt disease.

The agreement allows Aequus the option to acquire North American commercial rights to REV-0100, reVision’s proprietary Stargardt disease program.

Stargardt disease is a genetic disorder that affects central vision in children and adults, often leading to blindness. There are currently no approved treatment options.

According to the companies, REV-0100 is based on important discovery research from Weill Cornell Medicine in New York City that shows REV-0100 can reduce elevated levels of toxic lipid material called lipofuscin in pre-clinical studies.

reVision is thus poised to demonstrate the benefit of reducing levels of lipofuscin to alter the course of Stargardt disease progression.

Paul Fehlner, reVision’s co-founder and president, said the company is excited to partner with Aequus to continue developing REV-0100 for Stargardt disease.

“We believe that the existing efficacy data in animal models of Stargardt disease and established safety profile of the REV-0100 drug substance provide real hope for Stargardt disease patients who presently have no approved therapeutic options,” he said in a statement.

The companies noted that the FDA has already designated REV-0100 as an Orphan Drug and a Rare Pediatric Disease Drug for the treatment of Stargardt disease.

These designations support accelerated development of REV-0100, expediting review and evaluation amongst other benefits, including Orphan Drug market exclusivity upon successful program completion.

In addition, the drug substance has an established safety profile and is manufactured to GMP standards, potentially reducing safety risk and shortening the development timeline.

Doug Janzen, Aequus chairman and CEO, said in a statement that working with revision will help the company advance the therapy.

“Besides representing a meaningful market opportunity, there are a number of advantages ranging from orphan market exclusivity, potential for accelerated regulatory review and the opportunity to be eligible for a Priority Review Voucher,” he said in the statement.

As part of the option terms, Aequus will make an initial $400,000 equity investment in reVision with the option to fully fund the development program in return for the North American commercial rights.

Funds from the initial investment are earmarked to cover the costs of a pre-clinical toxicology study for REV-0100, which we begin in the near term.

Clinical trials with Stargardt patients are expected to initiate in late 2021 or early 2022.

Aequus is a specialty pharmaceutical company with multiple commercial eye care products and reVision is a privately-held, biopharmaceutical company focused on the development and commercialization of innovative therapies for rare ocular diseases.