Septerna Announces Initiation of Phase 1 Clinical Trial with SEP-786, a Novel Oral Small Molecule PTH1R Agonist for the Treatment of Hypoparathyroidism
September 27 2024 - 7:00AM
Business Wire
Trial Initiation Marks Septerna’s Transition to
a Clinical-Stage Company Developing a Portfolio of Oral Small
Molecule GPCR-Targeted Medicines
Phase 1 Clinical Trial Designed to Evaluate the
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of
SEP-786 in Healthy Volunteers
Septerna, a clinical-stage biotechnology company pioneering a
new era of GPCR-targeted drug discovery, today announced the dosing
of the first participants in its Phase 1 clinical trial of SEP-786,
its novel, potent and selective, oral small molecule parathyroid
hormone 1 receptor (PTH1R) agonist being developed for the
treatment of patients with hypoparathyroidism. The Phase 1 clinical
trial is a single-ascending dose (SAD) and multiple-ascending dose
(MAD) clinical trial to evaluate the safety, tolerability,
pharmacokinetics (PK) and pharmacodynamics (PD) of SEP-786 in
healthy adult volunteers.
Hypoparathyroidism is a debilitating endocrine disease caused by
a deficiency of the parathyroid hormone (PTH) that results in a
range of symptoms, including muscle cramps, fatigue, cognitive
dysfunction, and life-threatening complications, such as cardiac
arrhythmias, seizures, and renal failure. Conventional therapy
requires patients take high doses of calcium and Vitamin D
supplements orally several times daily or life-long daily
injections of PTH replacement therapy.
“GPCR-targeted drugs have been the most productive target class
in drug discovery history, with about one-third of all FDA-approved
drugs targeting these receptors. Our proprietary Native Complex
Platform™ fuels the discovery of oral small molecule compounds for
a wide range of GPCRs, such as PTH1R, a validated receptor target
for hypoparathyroidism,” said Jeffrey Finer, M.D., Ph.D., Chief
Executive Officer and Co-founder of Septerna. “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
normalize serum calcium levels, as we have seen in preclinical
models, in this Phase 1 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.”
The randomized, placebo-controlled, SAD and MAD Phase 1 clinical
trial is expected to enroll up to 180 healthy adult participants.
Dosing is underway in the SAD portion of the clinical trial, which
will evaluate the safety and tolerability of SEP-786 at escalating
oral doses. The MAD portion of the clinical trial is designed to
evaluate the safety and tolerability of once-daily and twice-daily
oral dosing of SEP-786 over multiple days of treatment with
secondary endpoints including PK, changes in serum calcium and
urinary calcium, and other biomarkers.
About SEP-786 and Hypoparathyroidism
Septerna is developing SEP-786, a novel, potent and selective
oral small molecule parathyroid hormone 1 receptor (PTH1R) agonist
for the treatment of patients with hypoparathyroidism, a rare
endocrine disease characterized by a deficiency of the parathyroid
hormone (PTH). Hypoparathyroidism results in a wide range of
debilitating symptoms, including muscle cramps, fatigue, cognitive
dysfunction and life-threatening complications, such as cardiac
arrhythmias, seizures, and renal failure. Currently available
treatments include supplements that only partially address PTH
deficiency, or PTH peptide replacements, which require daily
injections. Preclinical safety studies support that SEP-786 has
demonstrated the ability to normalize serum calcium and was
generally well-tolerated in 28-day Good Laboratory Practice
toxicology studies. SEP-786 has the potential to be a
differentiated treatment for hypoparathyroidism.
About Septerna
Septerna, Inc. is a clinical-stage biotechnology company
pioneering a new era of G protein-coupled receptor (GPCR) oral
small molecule drug discovery powered by its proprietary Native
Complex PlatformTM. Its industrial-scale platform aims to unlock
the full potential of GPCR therapies and has led to the discovery
and development of its deep pipeline of product candidates focused
initially on treating patients in three therapeutic areas:
endocrinology, immunology and inflammation, and metabolic diseases.
Septerna was launched in 2022 by preeminent drug discovery company
builders and scientific leaders in the biochemistry, structural
biology, and pharmacology of GPCRs. For more information, please
visit www.septerna.com.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240927704554/en/
Renee Leck THRUST Strategic Communications
renee@thrustsc.com