Ascendis Pharma A/S (ASND), a clinical-stage biopharmaceutical company focusing on endocrinology rare disease, is all set to report results from a phase II study of TransCon PTH for adult hypoparathyroidism by the end of this month. Full results from this trial are expected in the third quarter.
TransCon PTH is an investigational long-acting prodrug of parathyroid hormone (PTH) in phase II development as a potential once-daily replacement therapy for hypoparathyroidism, dubbed PaTH Forward.
The PaTH Forward Study
In the phase II TransCon PTH trial, 40 adult subjects with hypoparathyroidism treated with Standard of Care are randomized to daily TransCon PTH 15, 18, or 21 µg PTH/d or daily blinded placebo via pen-injector for 4 weeks.
The goal of PaTH Forward is to evaluate TransCon PTH control of serum and urinary calcium and identify a titration regimen for a complete withdrawal of standard of care (i.e., active vitamin D and calcium supplements).
Hypoparathyroidism, or HP in short, is a rare endocrine disorder characterized by insufficient levels of parathyroid hormone (PTH), resulting in low calcium and elevated phosphate levels in the blood.
About 80,000 people in the U.S. have HP. The symptoms of this disorder include weakness, severe muscle cramps (tetany), abnormal sensations such as tingling, burning, and numbness (paresthesia), memory loss, impaired judgment, and headache.
Other Drugs In the Pipeline
— The Company’s most advanced investigational drug is TransCon hGH for pediatric growth hormone deficiency or GHD.
In phase III trials, once-weekly TransCon hGH has demonstrated superior height velocity to a daily growth hormone (Genotropin) in children with pediatric growth hormone deficiency. The Company is planning to file a Biologics License Application for TransCon hGH in the U.S. in the second quarter and seek approval for the same in Europe in the fourth quarter of this year.
The Company plans to submit regulatory filings to initiate a global, phase III clinical trial of TransCon hGH in adult growth hormone deficiency during the first quarter, and to initiate a trial in pediatric GHD in Japan during the fourth quarter.
— TransCon CNP, a long-acting prodrug of C-type natriuretic peptide for the treatment of achondroplasia, is under a phase II trial, dubbed ACcomplisH. This trial was initiated last September and is expected to be completed in June 2021.
Achondroplasia is the most common form of dwarfism, affecting approximately 250,000 people worldwide.
— The Company also has a couple of oncology therapies, all of which are in preclinical testing. The first IND for an oncology drug is expected to be filed in the fourth quarter.
Ascendis Pharma ended September 30, 2019, with cash and cash equivalents of roughly 659 million euros.
ASND has traded in a range of $90.06 to $145.59 in the last 1 year. The stock closed Friday’s (March 6, 2020) trading at $129.93, down 1.43%.
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