Ophthamize™ Macregen Platform Technology Plan
The Ophthamize™ is based on Macregen's ownership of proprietary technology that has been acquired and/or developed by the Company.
In addition to the Company conducting Phase I a/b trials to demonstrate that Mitia™ is safe and well-tolerated when administered by intravitreal injection, Macregen is also conducting proof of concept studies in which Mitia™ is used in combination with cell-penetrating peptides (CPPs) to enable topical drug delivery in an eye drop formulation. These proof of concept studies are being performed in the laboratory of Dr. Felicity de Cogan, Macregen's Principal Consultant for Topical Drug Delivery.
Macregen’s acquisition and development of this proprietary platform technology is the basis for the Ophthamize™. The Ophthamize™ envisions a broad spectrum of molecules - from approved drugs and others that are in various stages of human clinical studies - used in combination with the proprietary CPP technology.
The Ophthamize™ has established an experimental algorithm to test whether various ophthalmic drugs, either already on the market or in development, can be delivered topically to specific disease targets in therapeutically-effective doses. The Company has published results demonstrating that approved drugs, such as anti-VEGFs, can be topically administrated and reach the disease target site in the retina at therapeutic levels. As a result, the Ophthamize™ should provide Macregen with a product development pipeline in AMD as well as many other eye diseases.
Cell-penetrating Peptides (CPPs)
CPPs are oligopeptides which are very small proteins that can pass easily in and out of cells without harm. The CPPs can be used to permit therapeutic agents of many compositions and molecular masses to access the different tissues in the eye simply by using a topical eye drop. The CPPs can even allow the agents to pass all the way to the back of the eye without a costly, time-consuming and distressing intravitreal injection.
CPPs are non-toxic and can associate with, and deliver, a wide range of therapeutic molecules for eye diseases. One of Macregen's major areas of clinical research focus will be on the delivery of antibodies and antibody fragments, including vascular endothelial growth factor (anti-VEGF) inhibitors for the treatment of neovascular age-related macular degeneration (nAMD). The use of CPP complexes to deliver anti-VEGF inhibitors in an eye drop will remove many of the adverse clinical costs and consequences associated with intravitreal injections; in turn, this will result in improvements to patient care and reduce medical professional/patient interaction for invasive treatments.
In addition to AMD, the topical delivery capabilities of the CPPs may extend to the treatment of many other eye diseases. The facility of CPPs to transport a range of therapeutic materials into the eye can be exploited to deliver a variety of biologically-active molecules for numerous eye diseases. The cell penetrating nature of these mono-oligopeptides (polyarginines) results in no apparent toxicity toward mammalian cells. Additionally, CPPs have exhibited significant antimicrobial range against both Gram positive and Gram negative bacteria that is similar to that of chloramaphenicol. This beneficial effect could obviate the need for inclusion of harmful cytotoxic preservatives into the eye drop formulation.
The preclinical work Macregen has executed to date has shown the CPP can deliver anti-VEGF drugs with eye drops in mice, rats, rabbits and in ex vivo porcine eyes. Disease models in rodents have shown that anti-VEGFs delivered by CPPs have the same efficacy as intravitreally-injected anti-VEGF drugs.
Licensing and Strategic Partnerships
Macregen’s proprietary topical delivery system is provides opportunities for licensing to other companies wishing to use eye drops for their already approved drugs and for drug candidates in various stages of clinical assessment. Such licensing arrangements can also form the basis of strategic partnerships with Macregen.
Macregen Product Pipeline
Macregen’s Ophthamize™ employing cell-penetrating peptides (CPPs) may enable the use of eye drops to treat eye diseases in addition to AMD.
Macregen is currently investigating CPP-enabled topical delivery of about 20 drugs approved or in development for uveitis, diabetic retinopathy (DR), diabetic macular edema (DME), proliferative vitreal retinopathy (PVR) and retinitis pigmentosa (RP). Each of these drugs are potentially deliverable for the first time as eye drops. This potential should stimulate licensing agreements between Macregen and manufacturers of these drugs.