Aequus Pharmaceuticals Inc. (TSX-V: AQS, OTCQB: AQSZF) (“Aequus”) and reVision Therapeutics, Inc. (“reVision”) have announced a collaboration on the development of a therapy for Stargardt disease. The two companies will be focused on the development and commercialization of innovative therapies for rare ocular diseases. The agreement allows Aequus the option to acquire North American commercial rights to “REV-0100”, reVision’s proprietary Stargardt disease program. The research program provides a potential therapy for patients to bind and clear lipofuscin which leads to cell death and retinal degeneration in Stargardt disease. Both companies stated that there were “no other known products in development that remove accumulated lipofuscin through this mechanism of action and no other approved treatment for Stargardt disease.
Stargardt disease (STGD1) is caused by mutations in the photoreceptor-specific ABCA4 gene which encodes the adenosine triphosphate-binding cassette, subfamily A, member 4. The ABCA4 gene encodes a transporter protein within retinal photoreceptor cells facilitating the active transport of potentially toxic retinoid compounds removing toxic by-products from the visual cycle. Historically, Stargardt disease, also referenced as fundus flavimaculatus, was first discovered by a German ophthalmologist, Karl Stargardt, in 1909. His report described a juvenile macular dystrophy in patients from two separate families, presenting in the first or second decade of life with a reduction in central visual acuity. However, today, it is the most common form of inherited macular dystrophy, affecting 1 in 10,000 individuals worldwide. Not dissimilar to retinitis pigmentosa with a high degree of clinical heterogeneity, Stargardt patients may end up having severe visual impairment or even complete blindness, due to death of the photoreceptors and the retinal pigment epithelium (RPE) cells.
Following the announcement on their collaboration, Aequus will make an initial $400,000 USD equity investment in reVision with the option to fully fund the development program in return for the North American commercial rights. According to the companies, funds from the initial investment will cover the costs of a pre-clinical toxicology study for REV-0100 while clinical trials with Stargardt patients are expected to commence in late 2021 or early 2022. Commenting on the program, Doug Janzen, Aequus Chairman and CEO stated that, “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. 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”. The US Food and Drug Administration has already designated REV-0100 as an Orphan Drug and a Rare Pediatric Disease Drug for the treatment of Stargardt disease.