Amakem Therapeutics, a clinical stage ophthalmology company, has presented top-line clinical results for its lead drug candidate, AMA0076 for the treatment of glaucoma, at the Ophthalmology Innovation Summit (OIS) at the 2013 Annual Meeting of the American Academy of Ophthalmology.
Based on Amakem's 'Localized Drug Action' platform, AMA0076 is a highly potent locally-acting Rho Kinase (ROCK) inhibitor that has been designed to reduce intraocular pressure (IOP) while minimizing side effects such as hyperemia ('red eye') that have been observed with all other ROCK inhibitors so far tested clinically. Hyperemia as observed with all other ROCK inhibitors in development is a particularly problematic side effect leading to patient non-compliance.
Dr. Jack Elands, CEO of Amakem, presented top-line results from two studies, a first-in-human study in patients with glaucoma and ocular hypertension and a Phase 1b formulation study in healthy volunteers.
The first-in-human study was a multicenter, randomized, double-masked, placebo-controlled dose-escalation study with AMA0076 applied topically, as eye drops (ClinicalTrials.gov identifier NCT01693315). At the most effective dose, IOP reduction from baseline (the primary endpoint of the study) was evident across visits, diurnal time points and both eyes while no conjunctival hyperemia related adverse events were observed. At the end of treatment, this dose group achieved a decrease in mean diurnal IOP from baseline of 3.7 mmHg (p=0.020 compared to placebo).
Additional work has demonstrated that AMA0076's bioavailability could be further increased and Amakem has conducted a Phase 1b study with several new formulations of AMA0076, all with significantly improved corneal absorption. The optimized formulation led to substantial IOP reduction with no significant hyperemia.
Dr. Elands said: "AMA0076 has demonstrated IOP reduction without significant hyperemia, the first ROCK inhibitor to achieve this goal in the clinic. The data from these two studies therefore provide clinical proof of concept for our lead program as well as for our Localized Drug Action platform. Based on these encouraging results, we have selected an optimized formulation to take into a Phase 2a study in H1 2014. We look forward to building on these early clinical data and advancing what we believe has the potential to be a valuable new treatment option for glaucoma patients."
Dr. Ingeborg Stalmans, Professor, Head of the Glaucoma Unit and Director of Ophthalmology Research Center, University of Leuven, Belgium and a member of Amakem's Clinical Advisory Board, said: "The results seen so far with AMA0076 suggest that the promise seen in pre-clinical development has been carried into the clinic successfully. To see such low levels of hyperemia accompanying meaningful reductions in IOP is a breakthrough in the development of ROCK inhibitors."