Early trial tests safety of potential treatment for hypoparathyroidism
Results for experimental therapy SEP-479 expected late this year or in early 2027
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The first participants have been dosed in a Phase 1 clinical trial that Septerna is conducting to test SEP-479, its experimental oral treatment for hypoparathyroidism.
The Phase 1 study is testing single and multiple doses of SEP-479 in up to 150 healthy volunteers, with the main goal of evaluating the experimental therapy’s safety and pharmacological profile. Biomarker data will also be assessed to determine whether the investigational therapy is working as intended. Septerna said it expects to report results from the trial late this year or early in 2027.
“We look forward to advancing our Phase 1 trial as rapidly as possible,” Jeffrey Finer, MD, PhD, CEO and co‑founder of Septerna, said in a company press release.
SEP-479 designed to mimic function of key signaling molecule
Hypoparathyroidism is a disorder marked by low levels of parathyroid hormone (PTH), a signaling molecule that’s produced by the parathyroid glands, a set of tiny structures in the neck. PTH is key to regulating calcium levels in the body, and treatment generally focuses on normalizing them. This may be done with supplements or with Yorvipath (palopegteriparatide), a daily injection therapy from Ascendis Pharma that works to deliver PTH into the body.
“Hypoparathyroidism is a lifelong condition that patients must manage with either high doses of calcium supplements several times a day or daily parathyroid hormone (PTH) injections, both of which can place a substantial burden on patients over time,” Finer said.
PTH exerts its effects throughout the body by binding to a specialized molecular receptor, aptly named PTH Receptor 1 (PTH1R). SEP-479 is an orally available small molecule that’s designed to act as a PTH1R agonist — in other words, it aims to bind to the receptor and activate it, essentially mimicking the function of PTH. According to Septerna, preclinical tests have shown that the experimental therapy’s effects on the receptor protein are comparable to those of the naturally occurring hormone, with calcium levels normalizing in animal models of hypoparathyroidism.
“We are encouraged by the preclinical data for SEP‑479, which underscore its potential to be a [disease-modifying] therapy for hypoparathyroidism by directly targeting the PTH receptor to provide patients full‑day calcium control and relief from their debilitating symptoms. Based on these findings, we believe SEP‑479 has the potential to be differentiated as a once-daily oral therapy for patients,” Finer said.
