Ascendis Pharma Announces Dosing of Subjects in Phase 1 Trial

Ascendis Pharma A/S recently announced it has dosed subjects in a Phase 1 trial of TransCon PTH, a long-acting prodrug of parathyroid hormone (PTH) in development for the treatment of hypoparathyroidism. The single and multiple ascending dose Phase 1 trial is designed to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of TransCon PTH in healthy adults. The trial evaluates single ascending doses and 10 multiple daily doses of TransCon PTH.

“This Phase 1 trial will provide us key insights regarding the potential of TransCon PTH as a treatment for hypoparathyroidism. By replacing PTH at physiological levels, we have designed TransCon PTH to normalize serum and urinary calcium levels, as well as serum phosphate levels, and to improve quality of life,” said Jonathan Leff, MD, Ascendis Pharma’s Chief Medical Officer. “Based on extensive clinical experience with PTH replacement therapies, we believe that if this Phase 1 trial is successful, we can advance TransCon PTH directly into a Phase 3 pivotal trial with the ultimate goal of helping patients who suffer from this debilitating disease.”

Hypoparathyroidism is a rare endocrine disorder characterized by insufficient levels of PTH resulting in low calcium and elevated phosphate levels in the blood. In the short-term, symptoms include weakness, muscle cramps, abnormal sensations such as tingling, burning, and numbness (paresthesias), loss of memory, impaired judgment, and headaches. This complex disorder can increase the risk of major long-term complications, such as extraskeletal calcium deposition, including within the brain, the kidneys, and the lens of eye, leading to impaired renal function and quality of life. Hypoparathyroidism affects approximately 77,000 patients in the US, the majority of whom develop the condition following damage or accidental removal of the parathyroid glands during thyroid surgery.

Ascendis Pharma is applying the TransCon technology platform to build a leading rare disease commercial company. The company utilizes its innovative TransCon technology to address significant unmet medical needs in rare diseases by improving clinically validated parent drugs and creating therapies with potential for best-in-class efficacy, safety, and/or convenience. Ascendis Pharma has a wholly owned pipeline of rare disease endocrinology programs, including once-weekly TransCon Growth Hormone, which is currently being evaluated in the Phase 3 heiGHt Trial for children with growth hormone deficiency (GHD), TransCon PTH, a long-acting prodrug of parathyroid hormone for hypoparathyroidism currently in a Phase 1 trial, and TransCon CNP, a long-acting prodrug of C-type Natriuretic Peptide for achondroplasia. Additionally, Ascendis Pharma has multi-product collaborations with Sanofi in diabetes and Genentech in the field of ophthalmology. For more information, visit www.ascendispharma.com.