TOP1630 in DES


TOP1630 is our lead candidate for dry eye syndrome (DES) which is being readied for its first clinical study in 2017 in the US. The most common eye disease, DES is a chronic inflammatory disease characterised by eye dryness and which can have a significant impact on the quality of life of a sufferer. There remains a major unmet medical need for more effective treatments, as currently available treatments for DES have limited efficacy (particularly over the long-term), provide only symptomatic improvement and or are associated with safety and tolerability issues, such as burning or stinging, that limit their use. Please see our Disease Focus section for more information on the disease.

Better by design

TOP1630 has been designed to deliver long-term efficacy, improving both signs and symptoms of DES by effectively treating the underlying inflammation. It targets several important kinases (P38, Src family kinases (Src & Lck) and Syk) involved in the signalling cascade in inflammatory diseases. TOP1630 has demonstrated an excellent activity profile in vitro and in vivo, including in models of human disease, and it has been shown to synergistically inhibit key pathways involved in innate and adaptive immunity.

TOP1630 has been designed to act locally at surface of the eye, where the inflammation occurs, following topical delivery using eye drops. The drug is taken up and retained by the target inflammatory cells but otherwise has low bioavailability which, together with the natural physiology of the eye, means that the rest of the body is unlikely to have a significant exposure to the drug. This reduces the likelihood of use-limiting side-effects occurring, as commonly seen with currently available treatments, allowing TOP1630 to be used as an effective long-term treatment.

Clinical development

TOP1630 is currently being evaluated in a Phase 1/2a proof-of-concept (POC) study in DES patients in the US. This study will report in the second half of 2017 and could lead directly into pivotal Phase 3 studies thereafter, potentially accelerating its route to market.

Commercialisation

TopiVert intends to seek an industry partner to complete the development of TOP1630 beyond the current POC study and to undertake the global commercialisation of the product.