Paquinimod is a once-daily, oral immunomodulatory compound in development for treatment of systemic sclerosis, a rare autoimmune disease of the connective tissue with a high unmet medical need. Paquinimod shows potent effects on fibrosis and inflammation in experimental models of systemic sclerosis and has been granted orphan drug designation in both EU and the US, which provides for seven to ten years of market exclusivity in the event of future registration.
Active Biotech is seeking a collaboration partner with the relevant expertise to further develop paquinimod in systemic sclerosis.
The clinical development program to establish clinical dose, tolerability and pharmacokinetics has been carried out with paquinimod in healthy subjects, in systemic lupus erythematosus (SLE) patients (Bengtsson et al, 2012) and in systemic sclerosis patients (ClinicalTrials: NCT01487551). Results from the clinical study in systemic sclerosis demonstrated a favorable safety profile and effects on disease-related biomarkers in line with paquinimod’s mode of action. The next step in clinical development is to verify these effects in a controlled phase 2 study that can form the basis for a pivotal study in this patient group.