Septerna has dosed the first subjects in a Phase I clinical trial of its investigational product SEP-786, being developed to treat hypoparathyroidism.
An oral small molecule, SEP-786 works by activating the parathyroid hormone 1 receptor (PTH1R).
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The trial is designed to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of SEP-786 in up to 180 healthy adults.
The randomised, placebo-controlled trial will have single-ascending dose (SAD) and multiple-ascending dose (MAD) components.
Currently, subject dosing has commenced in the SAD part of the trial, which will explore the safety and tolerability of increasing oral doses of SEP-786.
The subsequent MAD portion will investigate the safety and tolerability of once-daily and twice-daily oral doses of the drug over several days.
Secondary endpoints of the study are PK, serum calcium changes, urinary calcium excretion, and other relevant biomarkers.
Hypoparathyroidism is a rare endocrine disorder characterised by insufficient PTH production, leading to low calcium levels and increased phosphorus levels in the blood.
Current treatments mainly involve calcium and vitamin D supplementation, which can be challenging to manage and do not address the underlying PTH deficiency.
Septerna CEO and co-founder Jeffrey Finer said: “An oral small molecule PTH1R agonist has transformative potential as a convenient, disease-modifying treatment option that could provide patients with full-day calcium control, while resolving the serious effects of low blood calcium levels due to PTH deficiency.
“We look forward to assessing the safety of SEP-786 and its potential to normalise serum calcium levels, as we have seen in preclinical models, in this Phase I clinical trial. I am very proud of the rapid execution by our R&D team in achieving this milestone, which underscores the power of our Native Complex Platform to take on GPCR targets which have been historically challenging for small molecule drug discovery.”
