SAN903 for rare inflammatory and fibrotic disorders

Saniona has selected a drug candidate, SAN903, currently under preclinical development and positioned for treatment of rare inflammatory/fibrotic diseases with no or very inefficient current treatment options. SAN903 inhibits the IK potassium channel (a calcium-activated potassium channel also known as KCa3.1, gene KCNN4), which is important for activation of immune cells in both peripheral tissues (T-cells, macrophages) and brain (microglia), and is also involved in the abnormal production of connective tissue (by fibroblasts), which leads to fibrosis in chronic diseases.

SAN903 is safe and shows excellent efficacy in standard models of inflammatory diseases. Our current focus is idiopathic pulmonary fibrosis (IPF), which is a serious and fatal lung disease with limited treatment options. 30,000-150,000 patients suffer from IPF in the U.S. SAN903 is a new chemical entity covered by a recently filed patent application which may provide composition-of-matter protection until 2039.

Due to the role of the IK potassium channel in immune cell function, we are also actively evaluating SAN903 for potential treatment of a rare haematological disease called hereditary xerocytosis (HX) estimated to affect up to 40,000 patients in the U.S. Recent literature evidence demonstrates that HX is caused by gain-of-function mutations in the IK channel gene or in a closely associated calcium channel and SAN903 is expected to reverse this condition.

A precise pharmacological modulation of the IK channel can thus potentially treat rare diseases, which involve overactive or mis-timed immune reactions, such as IPF and HX.