Systemic sclerosis
Systemic sclerosis is a rare autoimmune disease affecting approximately 1.47 million people around the world. Causing skin tightening and impaired blood flow, it commonly is associated with digital ulcers – painful, chronic wounds typically impacting a patient’s fingers. For this debilitating condition there is currently no effective approved treatment and no late-stage topical therapy in development.
Diabetic foot ulcers
Patients suffering from diabetes often experience impaired microcirculation and nerve damage (diabetic neuropathy), leading to diabetic foot ulcers. Approximately 18.6 million people worldwide are affected each year. These ulcers can escalate into severe infections and even amputations – with a lower limb amputation occurring every 20 seconds globally. Current solutions and late-stage developments are not yet targeting the underlying microcirculation dysfunction.
TOP-N53 is a topical therapy currently in Phase 2a development for digital ulcers in systemic sclerosis (NCT06954597) and Phase 2 ready for the treatment of diabetic foot ulcers.
In a completed Phase 1 clinical trial, our lead compound has demonstrated improved local microcirculation and an excellent safety profile. In preclinical models, TOP-N53 has shown strong efficacy and no adverse effects at exposure levels well above the anticipated therapeutic dose in humans. These results point to a strong safety margin and high therapeutic potential.
TOP-N53 holds orphan drug designation from the American Food and Drug Administration (FDA) and European Medicines Agency (EMA) for digital ulcers in systemic sclerosis, underscoring its promise in this rare condition.
