Double-blind period completes! Primary endpoint analysis of phase 3 clinical trial to be conducted for China's first hormone replacement treatment targeting hypoparathyroidism

Submission of NDA to the NMPA is planned within 2023 at the earliest

SHANGHAI,Jan. 6,2023 -- VISEN Pharmaceuticals,an innovative biopharmaceutical company focused on endocrine diseases,announced today that it has completed the double-blind period of PaTHway China Trial,a phase 3 China clinical trial for its innovative investigational new prodrug palopegteriparatide on January 4,2023,and it would start to carry out analysis of the primary endpoints. Palopegteriparatide is designed to restore parathyroid hormone (PTH [1-34]) to physiological levels over 24 hours in adult patients with hypoparathyroidism (HP).

PaTHway China Trial is a randomized,double-blind,placebo-controlled,parallel,multi-center phase 3 trial to assess the potential of palopegteriparatide as a parathyroid hormone replacement therapy. The primary endpoints of the study include evaluating the safety,tolerability,and efficacy of once-daily subcutaneous injections of palopegteriparatide in adults with HP by measuring whether palopegteriparatide -treated patients achieve serum calcium (sCa) levels in the normal range (8.3–10.6 mg/dL)and independence from therapeutic levels of conventional calcium and active vitamin D therapy. VISEN obtained investigational new drug (IND) approval from the National Medical Products Administration (NMPA) on May 31,2021,allowing it to initiate clinical research of palopegteriparatide in China. On June 16,2022,VISEN completed patient enrollment for the PaTHway China Trial.

HP is the last endocrine deficiency state,where hormone replacement so far has not been established as standard management. It is a rare endocrine disorder characterized by insufficient levels of parathyroid hormone (PTH),resulting in low calcium and elevated phosphate levels in the blood. Conventional therapy with calcium supplements and active vitamin D does not effectively address the short-term symptoms,long-term complications,or quality-of-life impacts of HP and can lead to impaired renal function. The once-daily subcutaneous injection of palopegteriparatide is designed to provide sustained exposure within normal physiological range of PTH concentrations. If approved,it is expected to become the first hormone replacement treatment targeting HP in China.

Pony LU,CEO and Executive Director of VISEN Pharmaceuticals,said: "The completion of the double-blind period of the phase 3 clinical trial of palopegteriparatide in China means we have moved a step closer towards the success of our research efforts for what we hope will become China's first hormone replacement therapy targeting HP. We will now proceed with the analysis of the primary endpoints of the study and plan to submit a New Drug Application (NDA) to the National Medical Products Administration (NMPA) when all preparation work is completed. We look forward to bringing new treatment options to Chinese HP patients as soon as possible,so that their long-standing suffering could potentially be alleviated sooner."

VISEN Pharmaceuticals is developing palopegteriparatide under license from Ascendis Pharma,who received Priority Review from the U.S. Food & Drug Administration (FDA) in 2022 for its New Drug Application (NDA) for this new drug. Ascendis has said that the U.S. FDA has set a target date for completion of its review of its application by April 30,2023[1]. In addition,Ascendis Pharma also submitted a Marketing Authorization Application (MAA) for palopegteriparatide for adult patients with HP to the European Medicines Agency (EMA)[2].

[1] https://investors.ascendispharma.com/news-releases/news-release-details/fda-accepts-priority-review-ascendis-pharmas-nda-transcontm-pth?fbclid=IwAR3eEnJy3Udn5_-x5yGvmb2FpIzQM_jSin7RJjHmtfK8Uw34VsAs2HMnAmA

[2] https://investors.ascendispharma.com/news-releases/news-release-details/ascendis-pharma-submits-marketing-authorisation-application

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