Instant Report
FDF

Date: 04-Jun-2021

The First Hormone Replacement Therapy For Hypoparathyroidism In China Obtains IND Approval For Phase III Clinical Trial

VISEN Pharmaceuticals has obtained the investigational new drug (IND) approval from the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) for the phase III China clinical trial (PaTHway China Trial) of TransCon PTH Solution for Injection (TransCon™ parathyroid hormone).

2. PaTHway China Trial is designed to assess the potential of TransCon™ PTH as a hormone replacement therapy, and the aim is to enable patients to have serum calcium (sCa) within the normal range and independence from usual care.

3. The once-daily Subcutaneous Infection of TransCon™ PTH enables sustained exposure within normal physiological range of PTH concentrations, namely mimic continuous PTH infusion, and it is set to provide a truly ideal hormone replacement therapy for patients with HP.

SHANGHAI, June 3, 2021 /PRNewswire/ -- June 1 is World Hypopara Awareness Day. VISEN Pharmaceuticals, a biotech company focused on developing and commercializing innovative endocrine drugs, announced that it has obtained the investigational new drug (IND) approval from the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) for the phase III China clinical trial (PaTHway China Trial) of TransCon™ parathyroid hormone on June 1 and will soon initiate the study of TransCon™ PTH in patients with hypoparathyroidism (HP) in China.

Hypoparathyroidism (HP) is a set of clinical syndromes due to absent or insufficient production effect of circulating parathyroid hormone (PTH). The clinical manifestations are characterized by hypocalcemia, hyperphosphatemia and the resulting increased complications in neuromuscular excitability and ectopic calcification of soft tissues. The prevalence varies by country or region. For example, the prevalence of HP in the United States is estimated at 37 per 100,000. TransCon PTH has been defined as an orphan drug by FDA and EMA.

PaTHway China Trial is a randomized, double-blind, placebo-controlled, parallel, multi-center phase III study to assess the potential of TransCon™ PTH as a hormone replacement therapy. The primary endpoints of the study include evaluating the safety, tolerability and efficacy of once-daily subcutaneous injection of TransCon™ PTH in adults with HP. The aim is to enable patients to have serum calcium (sCa) within the normal range and, in the meanwhile, independent from usual care.

The key secondary efficacy endpoints in PaTHway China include changes in the scores in the Hypoparathyroidism Patient Experience Scale (HPES) and 36 Item Short Form Health Survey (SF-36) reported by HP patients receiving TransCon™ PTH. As China doesn't have sufficient patient-reported data on the quality of life of HP patients, their employment status, and the impact on their jobs, PaTHway China will leverage the scale to conduct a survey on patients in a bid to obtain more patient-reported data to assess the changes in the survival status of patients before and after the treatment.

Dr. Jun Yang, CMO of VISEN Pharmaceuticals, said, "Hormone replacement has always been the primary treatment option for hormonal deficiency states. However, no effective parathyroid replacement therapy is available for hypoparathyroidism to correct related pathophysiological changes and metabolic disorders. TransCon™ PTH is an investigational, innovative, long-acting prodrug of PTH. It is being developed as a once-daily hormone replacement therapy for adults with HP, designed to replace PTH at physiologic levels for 24 hours every day, and address both short-term symptoms and long-term complications of the disease. It is very encouraging to obtain the IND approval for Phase III clinical trial in China on the World Hypopara Awareness Day, which will inspire us to work hard to accelerate the development of innovative drugs and address unmet clinical needs!"