The main objective is to provide a safe and effective drug for the treatment of Acanthamoeba keratitis tested according to the international regulatory standards of the medicinal products development.
This project will undertake research and technology development activities on the Orphan Drug Polihexanide (polyhexamethylene biguanide / PHMB) towards developing the first approved therapeutic ophthalmic intervention for the rare ocular disease Acanthamoeba keratitis (AK). ODAK work focuses on the preclinical and clinical research and development of the orphan drug PHMB (designation EU/3/07/498).
The ODAK project studies will be carried out an innovative clinical development program with the aim to:
evaluate the efficacy and safety of the current treatments of Acanthoamoeba keratitis through a retrospective clinical trial
estimate with scientific evidence in the proposal clinical trials (phase I/phase III) the efficacy and safety profile in humans of an eye drops solution containing the most well-experimented PHMB concentrations (stable, safe and effective) as resulting from preliminary pharmaceutical technology and non-clinical studies
establish the most effective therapeutic regimen for eradicating Acanthamoeba infection
generate the first clinical documentation for marketing authorization of PHMB eye drops for the treatment of Acanthamoeba keratitis
ODAK studies will ultilise appropriate excipients (buffer agents, viscosizing agents, isotonising agents, etc.) which will be used for the development/selection of the most effective formulation of PHMB able to:
improve the bioavailability of PHMB to the ocular surface
improve the therapeutic index of the PHMB administered in order to minimize potential side effects over empirical formulations
obtain a controlled release of the PHMB administered, also by improving its chemical and physical stability, guaranteeing a prolonged presence in the site of action in an amount sufficient for the desired therapeutic effect
ODAK studies will be examining different PHMB concentrations through state of the art experimental models in order to:
select the safest concentration of PHMB that is effective against Acanthamoeba keratitis
improve the toxicological profile of the selected PHMB compared to current empirical eye drops
Clinical Studies - State of the art
The clinical efficacy of 0.02% PHMB is documented in several literature reports, however, no large clinical trials have ever been conducted and a significant statistical evaluation is absent. In addition, the treatment schedule of PHMB 0.02% employed so far in clinical practice is empirical and there is currently no validated therapeutic regimen suggesting a standard clinical practice in treating Acanthamoeba keratitis.