Investigational ROCK inhibitor has positive clinical results

Researchers presented positive top-line clinical results for AMA0076 (Amakem Therapeutics), a possible new drug candidate for the treatment of glaucoma at the 2013 annual meeting of the American Academy of Ophthalmology.

AMA0076 is a highly potent locally-acting Rho kinase (ROCK) inhibitor designed to reduce IOP and minimize side effects including hyperaemia, a particularly problematic side effect observed with all other ROCK inhibitors that leads to patient non-compliance.

"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 hyperaemia accompanying meaningful reduction in IOP is a breakthrough in the development of ROCK inhibitors," said Prof. Ingeborg Stalmans, professor and head of the Glaucoma Unit, and director of the Ophthalmology Research Centre, University of Leuven, Belgium, and a member of Amakem's clinical advisory board.

Jack Elands, CEO of Amakem, presented data 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 former was a multicentre, randomized, double-masked, placebo-controlled dose-escalation study with AMA0076 applied topically as eye drops. At the most effective dose of AMA0076, IOP reduction from baseline was seen across visits, diurnal time points and in both eyes, with no conjunctival hyperaemia. At the end of treatment, the group dosed with the most effective dose had a decrease in mean diurnal IOP from baseline of 3.7 mmHg (P = 0.020, compared with placebo).

In the phase 1b study, several new formulations of AMA0076 brought about significantly improved corneal absorption. The optimized formulation led to substantial reductions in IOP with no significant hyperaemia.

"AMA0076 has demonstrated IOP reduction without significant hyperaemia, the first ROCK inhibitor to achieve this goal in the clinic. The data from these two studies therefore provide clinical proof of concept that our lead programme as well as for our Localized Drug Action platform," said Elands. "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," he concluded.