The first hormone replacement therapy for hypoparathyroidism in China obtains IND approval for Phase III clinical trial

Rhea-AI Summary

VISEN Pharmaceuticals has received IND approval from the CDE of NMPA for the Phase III PaTHway China Trial of TransCon PTH, targeting patients with hypoparathyroidism (HP). This trial aims to evaluate the efficacy of this once-daily hormone replacement therapy, which mimics continuous PTH infusion, to maintain normal serum calcium levels. The study will also collect patient-reported data to enhance understanding of HP's impact on quality of life. Over 200,000 patients globally suffer from HP, and this development marks a significant step in addressing unmet clinical needs in China.

Positive

• IND approval received for Phase III clinical trial in China.
• TransCon PTH aims to provide a new treatment option for over 200,000 patients with hypoparathyroidism.
• Study designed to enhance patient-reported data collection on HP quality of life.

Negative

• None.

1. 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!"

Previously, Ascendis Pharma conducted the global Phase II clinical trial of TransCon™ PTH (PaTH Forward Study) with data proving the potential of TransCon™ PTH as a replacement therapy for HP. The 58-week preliminary results of the open-label extension in the PaTH Forward Study also support the use of TransCon™ PTH in HP patients with durable benefits, and sound tolerability and safety profiles. VISEN Pharmaceuticals is responsible for the development and commercialization of Ascendis Pharma's endocrine therapies in the Greater China region.

Pony Lu, CEO of VISEN Pharmaceuticals, said: "Over 200,000 patients worldwide suffer from hypoparathyroidism, which can cause severe short-term symptoms and long-term complications, and seriously affect the quality of life. Conventional treatment can't meet the clinical needs. With exclusive rights for R&D and marketing of TransCon™ parathyroid hormone in Greater China, VISEN Pharmaceuticals will carry out the related clinical trial in China, and we hope to provide a new option for Chinese patients with HP as soon as possible."

Reference:

1.    Chinese Society of Osteoporosis and Bone Mineral Research，Chinese society of endocrine metabolic bone disease branch ,Clinical practice guideline for hypoparathyroidism, Chinese journal of osteoporosis and bone mineral salt diseases 2018;11(4):323-338.

2.    Ascendis Pharma Announces Orphan Drug Designation Granted for TransCon PTH as Treatment for Hypoparathyroidism Retrieved Jun 1,2021, from https://ascendispharma.gcs-web.com/news-releases/news-release-details/ascendis-pharma-announces-orphan-drug-designation-granted-0?ID=2353621&c=242088&p=irol-newsArticle

About VISEN Pharmaceuticals

VISEN Pharmaceuticals is committed to the treatment of endocrine-related diseases, introducing the world's leading treatment methods and drugs into the China market, and hoping to provide more Chinese patients quick access to the world's most advanced and reliable treatment solutions.

In 2018, VISEN Pharmaceuticals was formed by Ascendis Pharma A/S (Nasdaq: ASND) and an investor syndicate led by Vivo Capital (along with participation by Sofinnova Investments). In January 2021, VISEN Pharmaceuticals completed series B financing, led by Sequoia China with participation from OrbiMed, Sherpa Healthcare Partners, Cormorant, HBM Healthcare Investments, Pivotal bioVenture Partners China, Logos Capital, and CDG Capital, to develop and commercialize endocrinology therapies in Greater China, which includes mainland China, Hong Kong, Macau, and Taiwan.

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SOURCE VISEN PHARMA

FAQ

What is the purpose of the PaTHway China Trial for ASND?

The PaTHway China Trial aims to assess the efficacy and safety of TransCon PTH as a hormone replacement therapy for patients with hypoparathyroidism.

When did VISEN Pharmaceuticals receive IND approval for TransCon PTH in China?

VISEN Pharmaceuticals obtained IND approval on June 1, 2021.

What is hypoparathyroidism and why is TransCon PTH important?

Hypoparathyroidism is a condition characterized by insufficient parathyroid hormone production, leading to complications. TransCon PTH is significant as it offers a potential hormone replacement therapy, addressing unmet clinical needs.

What are the expected outcomes of the Phase III trial for ASND?

The trial aims to evaluate safety, tolerability, and the ability to maintain normal serum calcium levels, along with collecting patient-reported outcomes.