Aequus Pharmaceuticals Inc. (TSX-V:AQS, OTCQB:AQSZF) and reVision Therapeutics, Inc. have announced a collaboration to develop a therapy for Stargardt disease, a devastating genetic disorder that affects central vision in children and adults and often leads to blindness.
The agreement allows Aequus the option to acquire the North American commercial rights to REV-0100, reVision’s proprietary Stargardt disease program.
As part of the option terms, Aequus will make an initial US$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 will begin in the near term.
Clinical trials with Stargardt patients are expected to initiate in late 2021 or early 2022.
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“We are excited to partner with Aequus, an established commercial ophthalmics company, to continue developing REV-0100 for Stargardt disease,” said Paul Fehlner, reVision’s co-founder and president.
“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.”
Meaningful market opportunity
“We are very excited to be working with the reVision team to advance this much-needed potential therapy. The REV-0100 mechanism of action suggests the possibility of slowing disease progression as a first-line therapy,” said Doug Janzen, Aequus chairman and CEO.
“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.”
Orphan Drug & Rare Pediatric Disease Drug designations
The US Food and Drug Administration (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 Good Manufacturing Practice (GMP) standards, potentially reducing safety risk and shortening the development timeline.
REV-0100 as potential therapy
REV-0100 is a potential therapy for patients with Stargardt disease that is designed to bind and clear a toxic lipid called lipofuscin.
Accumulation of lipofuscin in Stargardt disease leads to cell death and retinal degeneration. REV-0100 has the potential to reduce lipofuscin levels in the retina.
There are no other known products in development that remove accumulated lipofuscin through this mechanism of action and no other approved treatment for Stargardt disease.
REV-0100 was developed from research from Weill Cornell Medicine in New York City, and is covered by a granted patent and pending patent applications licensed from Cornell.
Stargardt Disease
Stargardt disease is also called Stargardt macular dystrophy, juvenile macular degeneration, or fundus flavimaculatus. The disease causes progressive damage — or degeneration — of the macula, which is an area in the center of the retina that is responsible for sharp, straight-ahead vision.
Stargardt disease is one of several genetic disorders that cause macular degeneration. Experts estimate that one in 8,000-10,000 people have Stargardt disease.
reVision
reVision Therapeutics is a privately-held, early-stage biopharmaceutical company focused on the development and commercialization of innovative therapies for ocular and rare diseases. reVision's lead product candidate REV-0100 is being developed as a treatment for Stargardt disease and dry age-related macular degeneration (AMD).
Aequus
Aequus Pharmaceuticals is a growing specialty pharmaceutical company focused on developing and commercializing high-quality, differentiated products.
Aequus has grown its sales and marketing efforts to include several commercial products in ophthalmology and transplant.
Aequus plans to build on its commercial platform through the launch of additional products that are either created internally or brought in through an acquisition or license; remaining focused on highly specialized therapeutic areas.