SEP-479 (PTH1R)

Hypoparathyroidism

Hypoparathyroidism is a rare endocrine disease characterized by low levels of
parathyroid hormone (PTH) caused by the loss of the parathyroid glands, frequently
after neck surgery. The disease affects approximately 70,000 patients in the United
States and 140,000 patients in Europe. The loss of PTH, which is a master regulator of
calcium homeostasis, leads to an array of challenging symptoms and life-threatening
complications for hypoparathyroidism patients including muscle cramps, fatigue,
cognitive dysfunction and brain fog, cardiac arrhythmias, seizures, kidney calcification
and renal failure. Many patients experience persistent symptoms that negatively impact
quality of life.

SEP-479: Oral Small Molecule PTH1R Agonist for Hypoparathyroidism

Our strategy to address hypoparathyroidism is to develop an oral small molecule that
functionally replaces PTH by activating PTH1R in bones and the kidneys to restore
normal calcium control and provides a convenient oral option for all hypoparathyroidism
patients.