Additional Information

TSHR Program

TSHR Program – Oral Small Molecule TSHR NAM for Graves’ Disease and TED

Graves’ disease is one of the most prevalent autoimmune conditions affecting over 2 million patients in the United States and is the leading cause of hyperthyroidism, resulting in symptoms including anxiety, irritability, tremor, and fatigue. Approximately 50% of Graves’ disease patients develop thyroid eye disease (TED) which is a serious, progressive and vision-threatening autoimmune condition that can lead to eye bulging, swelling, pain and blurred or double vision.

Both Graves’ disease and TED are caused by autoantibodies that bind to and activate the thyroid stimulating hormone receptor (TSHR) on thyroid cells in the thyroid gland (leading to Graves’ disease) and fibroblasts located behind the eyes (leading to TED).

We believe an oral small molecule TSHR negative allosteric modulator (NAM) could offer a novel disease-modifying treatment approach that directly addresses the pathobiology of both diseases by blocking TSHR overactivation caused by patients’ autoantibodies. Using our Native Complex PlatformTM we have identified novel TSHR NAMs, and we are currently focused on advancing multiple lead compounds.