Additional Information
SEP-786 (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-786 – 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.
Our lead product candidate, SEP-786, is, to our knowledge, the only clinical-stage, oral small molecule agonist targeting PTH1R for the treatment of hypoparathyroidism. We are currently conducting a Phase 1 clinical trial to assess preliminary safety, tolerability, pharmacokinetics and pharmacodynamics of SEP-786.