Founded in November 2018, we are a late-stage, near-commercialization biopharmaceutical company focused on providing treatments in selected endocrinology diseases in China (including Hong Kong, Macau and Taiwan). We have one Core Product and two other pipeline drug candidates, all of which were in-licensed from our collaboration partner and one of the Controlling Shareholders, Ascendis Pharma. See “Business – Collaborations” for more details. Since our inception until the date of this document, we have been conducting further research and development of such drug candidates.
Our Core Product, lonapegsomatropin, is a once-weekly long-acting growth hormone replacement therapy for the treatment of pediatric growth hormone deficiency (“PGHD”), a common short stature in patients aged under 18 caused by insufficient growth hormone. TransCon CNP (navepegritide), one of our key drug candidates, is a long-acting prodrug of c-type natriuretic peptide for the treatment of achondroplasia (“ACH”), a short-limbed dwarfism which results in severe skeletal complications and comorbidities. Palopegteriparatide, the other key drug candidate, is a once-daily parathyroid hormone (“PTH”) replacement therapy for the treatment of chronic hypoparathyroidism (“HP”), a syndrome of abnormal calcium and phosphorus metabolism caused by decreased secretion or defective function of PTH.
WE MAY NOT BE ABLE TO DEVELOP AND/OR MARKET OUR CORE PRODUCT SUCCESSFULLY.
Our Drug Pipeline
Leveraging our clinical development capabilities, we provide patients in China (including Hong Kong, Macau and Taiwan) with access to the following endocrine solutions: (i) our Core Product, lonapegsomatropin, has completed the Phase 3 pivotal trial in China for the treatment of PGHD; the BLA filing was made on January 18, 2024 and subsequently accepted by the NMPA on March 7, 2024; (ii) TransCon CNP (navepegritide) has completed the double-blind period of Phase 2 clinical trial in China for the treatment of ACH and the last patient last visit of open-label extension (“OLE”) period of this trial was completed in April 2024; and (iii) palopegteriparatide is currently undergoing development in a Phase 3 pivotal trial in China; it has completed the double-blind period in January 2023, and the expected NDA filing to the NMPA will be in 2025.
Our Core Product - Lonapegsomatropin
Lonapegsomatropin is a drug candidate studied by us to treat children aged 3 to 17 years old with GHD in a completed Phase 3 pivotal trial in China, where each subject received treatment for 52 weeks. Lonapegsomatropin demonstrated a greater AHV at 52 weeks for lonapegsomatropin compared to daily human growth hormone (“hGH”), with statistical significance. We in-licensed lonapegsomatropin from Ascendis Pharma in November 2018. Prior to our in-licensing, lonapegsomatropin had been studied by Ascendis Pharma in over 300 children with growth hormone deficiency (“GHD”) across three Phase 3 programs. Leveraging its novel molecular design, lonapegsomatropin is the only long-acting growth hormone (“LAGH”) that releases unmodified hGH in vivo consistently in between weekly doses. Such unmodified hGH is identical in the molecular composition to the endogenous growth hormone secreted by pituitary gland and preserves its original mode of action, with direct action by circulating growth hormone on target tissues and indirect action through promoting insulin-like growth factor-1 (“IGF-1”) production in the liver (via growth hormone receptor). In contrast, modified hGH often substantially alters its molecular size, which changes its receptor binding affinity and its ability to reach the target tissue. Our Core Product provides a convenient once-weekly dosing regimen in injection frequency as compared to once-daily hGH, which may foster increased dosing compliance for pediatric patients in daily lives.
We made the BLA filing with the NMPA on January 18, 2024 for our Core Product for the treatment of PGHD, which was subsequently accepted by the NMPA on March 7, 2024.
Addressable Market and Competitive Landscape
China accounted for the largest share of the global hGH market in 2023, surpassing the United States and representing 34% of the global market. The hGH market in China was RMB11.6 billion in 2023 and is expected to grow to RMB28.6 billion by 2030, at a CAGR of 13.7% from 2023 to 2030, according to Frost & Sullivan. The number of patients receiving hGH therapies largely depends on the number of PGHD patients receiving hGH therapies, which is calculated by multiplying the prevalence of PGHD by the treatment rate. As PGHD affects newborn to children as old as 17, the prevalence of PGHD is expected to remain relatively stable from 2023 to 2030, despite the expected decrease of birth rate. On the other hand, the treatment rate for PGHD is expected to significantly increase from 5.3% in 2023 to 10.7% in 2030, leading to a continuous growth in the number of patients receiving hGH therapies.
The hGH market is characterized by intense competition. We are aware of several pharmaceutical and biopharmaceutical companies that have commenced clinical studies of products or have successfully commercialized products addressing areas that we are targeting, including companies marketing or developing LAGH therapies, such as GeneScience Pharmaceutical Co., Ltd., I-Mab Biopharma Co., Ltd., Xiamen Amoytop Biotech Co., Ltd. and Anhui Anke Biotechnology (Group) Co., Ltd, Novo Nordisk A/S, as well as companies marketing daily hGH, such as GeneScience Pharmaceutical Co., Ltd., Anhui Anke Biotechnology (Group) Co., Ltd and Shanghai United Cell Biotechnology Co., Ltd., and Novo Nordisk A/S. Currently in China, only one LAGH therapy has received marketing approval and several are currently under clinical development. See “Risk Factors – Competition in the biotechnology and biopharmaceutical industries is intense and our competitors may discover, develop or commercialize products that are safer, more effective, more effectively marketed or cost less than ours, or receive regulatory approval or reach the market earlier. If we are unable to compete effectively, our business, results of operations and prospects will suffer” and “Industry Overview – Human Growth Hormone” for more details.
TransCon CNP (navepegritide)
TransCon CNP (navepegritide) is a disease-modifying therapy studied by us to treat children aged 2 to 10 years old with ACH in China, where there is currently no effective disease-modifying therapy approved. A disease-modifying therapy is a treatment that delays, slows, or reverses the progression of a disease by targeting its underlying cause. We in-licensed the TransCon CNP (navepegritide) from Ascendis Pharma in November 2018. Prior to our in-licensing, TransCon CNP (navepegritide) had been studied by Ascendis Pharma in 45 healthy adult male subjects in a Phase 1 global trial. TransCon CNP (navepegritide) is designed to optimize efficacy with a safe and convenient once-weekly dose, and is the first ACH therapy in clinical development in China to date, according to Frost & Sullivan. TransCon CNP (navepegritide) has completed the double-blind period of Phase 2 clinical trial in China for the treatment of ACH.
Addressable Market and Competitive Landscape
Due to the absence of approved therapy to treat the genetic basis of ACH, according to Frost & Sullivan, it is currently unfeasible to determine the market share and market size for ACH as there have been no corresponding sales generated in China. The prevalence of ACH in China was 51.2 thousand in 2023 and is estimated to reach 51.9 thousand in 2030.
There is currently no effective disease-modifying therapy for ACH approved in China.
Palopegteriparatide
Palopegteriparatide is a treatment solution studied by us to treat adults with HP. We in-licensed the palopegteriparatide from Ascendis Pharma in November 2018. Prior to our in-licensing, palopegteriparatide had been studied by Ascendis Pharma in healthy adult volunteers in a Phase 1 trial. The current treatments for HP are inadequate due to their limited therapeutic benefits and the need for chronic administration of calcium in high doses and increased risks of associated complications. Palopegteriparatide is designed to restore physiologic levels and activity of PTH throughout 24 hours per day, thereby addressing full aspects of the disease, including normalizing serum and urinary calcium and serum phosphate levels. We are studying palopegteriparatide in a China Phase 3 pivotal trial, and have completed its double-blind period in January 2023.
Addressable Market and Competitive Landscape
Due to the absence of approved PTH replacement therapy for the treatment of HP in China, according to Frost & Sullivan, it is currently unfeasible to determine the market share and market size for HP as there have been no corresponding sales generated in China. The prevalence of HP in China was 410.1 thousand in 2023 and is estimated to reach 495.6 thousand in 2030.
No PTH replacement therapy has been approved for HP treatment in China. Palopegteriparatide is the only PTH replacement therapy that has initiated clinical development in China. As of the Latest Practicable Date, Eneboparatide (developed by Amolyt Pharma) and CLTX-305 (developed by Calcilytix Therapeutics) are being developed in Phase 3 global clinical trials; EB612 (developed by Entera Bio), MBX 2109 (developed by MBX Biosciences) and AMOR-1 (developed by Amorphical) are being developed in Phase 2 global clinical trials; and EXT608 (developed by Extend Biosciences) and SEP-786 (developed by Septerna) are being developed in Phase 1 global clinical trials. None of these drug candidates have undergone clinical studies in China.
Source: VISEN Pharma-B (02561) Prospectus (IPO Date : 2025/03/13) |