Paquinimod (57-57) – a high medical need
Paquinimod is a compound primarily for the treatment of Systemic Sclerosis (SSc), a rare autoimmune disease. In February 2011, paquinimod was granted Orphan Medicinal Product status for the indication Systemic Sclerosis.
SSc - Systemic sclerosis - is a rare autoimmune clinically heterogeneous disease. SSc is characterized by disturbances of the immune system, alterations of the microvasculature and by overproduction and deposition of various proteins in particular collagen leading to inflammation and abnormal growth of the connective tissue of the skin and internal organs such as gastrointestinal tract, lungs, heart and kidney (Gabrielli 2009, Postlethwaite 2010). Even though the precise links of these disease processes are less well understood it seems as the immunological activity in SSc is a key stimulator of vascular abnormalities and fibrosis. There are two types of SSc that are differentiated by the extent of skin and organ involvement. Limited cutaneous SSc tends to be confined to the skin of the fingers and face whereas diffuse cutaneous SSc is a more severe disease with involvement of internal organs. SSc patients with the limited cutaneous form have a more benign disease course with lower incidence of renal involvement and restrictive pulmonary disease, with a much better prognosis. In contrast, patients with diffuse cutaneous SSc have a much more rapidly progressing form of the disease, with large areas of the skin affected, and a higher incidence of renal, cardiac and pulmonary involvement. The pattern of serum anti-nuclear antibodies usually differs between the two forms of SSc, and specific patterns of anti-nuclear antibodies are used for diagnosis as well as predicting prognosis.
Common clinical manifestations in SSc include Raynaud’s phenomenon, calcinosis, articular involvement, esophageal dysmotility, lung fibrosis, isolated pulmonary arterial hypertension, heart involvement and renal disease with Raynaud’s phenomenon being observed in 94-100 % of the SSc patients. The disease may lead to severe dysfunction and can be life threatening due to failure of internal organ.
There is at present no cure for SSc and existing treatments are focused on controlling symptoms and preventing complications. There is a high unmet medical need for new targeted therapies of SSc.
In 2011 and 2014, paquinimod was granted Orphan Medicinal Product Status in Europe and the US, respectively, for the indication systemic sclerosis. The Orphan Medicinal Product designation is implemented to promote the development of drugs that may provide significant benefit to patients suffering from rare diseases identified as life-threatening or chronically debilitating. Orphan Medicinal Product Status can provide ten years and seven years, in the respective geographic areas, of potential market exclusivity if the product candidate is approved for treatment.
In June 2014, data from a clinical study of systemic sclerosis was presented at the scientific conference EULAR (EUropean League Against Rheumatism). The results demonstrated that paquinimod was well tolerated and effects on biomarkers relevant for systemic sclerosis were observed during treatment. Data from a pre-clinical trial was also presented, showing that paquinimod reduces development of skin fibrosis in an experimental disease model for systemic sclerosis.
The next step in clinical development is to verify these effects in a controlled Phase II study that can form the basis for a pivotal study in this patient group. Active Biotech has initiated discussions with potential partners for the continued clinical development of paquinimod. The company will not commence the further clinical development of paquinimod on an independent basis.