Ascendis Pharma has submitted an investigational new drug (IND) application to the US Food and Drug Administration (FDA) seeking approval to conduct a Phase II clinical trial of Transcon PTH for the treatment of hypoparathyroidism (HP ).
TransCon PTH is designed as a long-acting prodrug of parathyroid hormone (PTH[1-34]). It is intended to provide replacement therapy for HP.
The company’s TransCon technology is used to develop new therapies that optimise therapeutic effect. Molecules created using the platform comprise an unmodified parent drug, an inert carrier to protect it, and a linker that temporarily binds the drug and carrier.
Dubbed PaTH Forward, the new Phase II trial will assess the safety, tolerability, and efficacy of three fixed doses of TransCon PTH in 40 adults who on standard-of-care therapies.
The randomised, double-blind, placebo-controlled, parallel group trial will also assess a titration regimen for complete withdrawal of standard of care.
PaTH Forward will be performed at up to 40 sites globally. It will introduce a pre-filled pen device and evaluate disease-specific patient-reported outcomes.
Following four weeks of dosing, patients could enrol into an open-label extension trial where TransCon PTH is studied for long-term safety and efficacy.
The company expects to report top-line results from the Phase II trial in the fourth quarter of this year.
Ascendis Pharma chief medical officer Jonathan Leff said: “Unlike current therapies for hypoparathyroidism, once-daily TransCon PTH is designed to restore PTH to physiologic levels for 24 hours each day and fully address all aspects of the disease.
“With PaTH Forward, we are applying our TransCon technology to advance a new treatment option for patients with this debilitating condition.”
The Denmark-based company’s portfolio comprises a total of three rare disease endocrinology product candidates.