VISEN Pharmaceuticals: Deepening Expertise and Innovation in Endocrine Therapeutics

“Innovative Drugs” were mentioned in the Government Work Report for the first time in 2024; developing innovative drugs is a major livelihood issue, a timely response to patients’ urgent needs, and an important engine driving China’s new quality productive forces. The China International Import Expo (CIIE), the world’s first national-level exhibition themed around imports, has also become a stage for the global debut of advanced innovative drugs. On the first day of the CIIE, several renowned domestic and foreign innovative pharmaceutical companies signed cooperation agreements on “full-lifecycle services for innovative drugs,” embracing the theme “New Era, Shared Future” to embark on a path of win-win collaboration with domestic pharmaceutical service providers.

Longpei Somatropin Makes Debut at the 7th China International Import Expo

Among the contracted enterprises, the collaboration between VISEN and Shanghai Pharma was highly representative, sparking heated discussion at the event. In fact, this was not the first partnership between VISEN and Shanghai Pharma; their cooperation originated from VISEN’s pivotal Phase III clinical trial of lonapegsomatropin in China. Shanghai Pharma ensured the supply of essential materials for the clinical trial, including the control drug, reference formulations, and reagents, while also providing services such as drug distribution and retrieval, thereby facilitating the successful completion of the trial by VISEN. The results of the Phase III clinical trial of lonapegsomatropin in China demonstrated superiority over the active control group.

The solid cooperation established in the early stages has laid a foundation of trust for both parties to deepen their future commercial collaboration. Recently, VISEN and Shanghai Pharma have initiated discussions on bonded imports, national distribution, supply chain optimization, and innovative value-added services. During the China International Import Expo (CIIE), Shanghai Pharma expressed high recognition of VISEN’s core products. Li Yongzhong, Executive Director and Executive President of Shanghai Pharmaceutical, stated, “VISEN’s products are highly forward-looking and scarce. We believe that VISEN’s lonapegsomatropin will bring significant benefits to children with growth hormone deficiency in China. Shanghai Pharma will fully leverage its platform advantages to facilitate the commercialization of lonapegsomatropin in the Chinese market.” Benefiting from the “spillover” effect of the CIIE, the accessibility of multiple globally leading innovative drugs, for which Shanghai Pharma provides services, has been significantly accelerated in China, helping these innovative drugs achieve a remarkable transformation from “exhibits” to “commercial products.”

This year, VISEN and Shanghai Pharma jointly seized the platform opportunities presented by the China International Import Expo (CIIE) to further finalize the scope of their cooperation: VISEN has designated Shanghai Pharma International Supply Chain Co., Ltd., a subsidiary of Shanghai Pharma, as the exclusive general importer and distributor for its core product, lonapegsomatropin injection for import, along with its dedicated proprietary auto-injector, in mainland China.

According to the official website of the Center for Drug Evaluation (CDE) under the National Medical Products Administration, the marketing authorization application for lonapegsomatropin in China has been accepted. It is foreseeable that in the near future, VISEN will be able to leverage the supply chain advantages of Shanghai Pharma to introduce this novel long-acting growth hormone, which has the potential to become the best-in-class in China, into the domestic market as soon as possible, thereby benefiting more patients.

VISEN Product Pipeline

In terms of pipeline planning, VISEN neither seeks to amass a large quantity of assets nor chases after “star” drugs. At an innovative drug forum, Lu Anbang, CEO of VISEN Pharmaceuticals, publicly outlined the company’s strategy of “specialization, depth, and differentiation.” This focus has enabled the company to concentrate its resources and efforts on in-depth research in the field of endocrinology, thereby gradually building strong professional advantages in specific areas, particularly in growth and development. This confidence stems not only from faith in its products but also, more importantly, from confidence in the TransCon (Transient Conjugation) technology platform underlying these products.

Ascendis Pharma, a licensor and shareholder of VISEN Pharmaceuticals, recently announced that it had licensed Novo Nordisk to use the TransCon technology platform to develop a once-monthly GLP-1 receptor agonist candidate for the treatment of metabolic disorders, including obesity and type 2 diabetes. This licensing deal underscores that the TransCon platform has emerged as a leader in drug long-acting technologies, standing out among its peers. VISEN has already secured three innovative drugs leveraging the TransCon technology. As Lu Anbang, CEO of VISEN Pharmaceuticals, stated, “VISEN currently has only three pipelines, and we select only Best-in-Class, First-in-Class, or even Only-One-in-Class candidates—a strategy that will continue in the future.”

The etiology of endocrine disorders is complex, with over 170 known diseases caused by hormonal imbalances. It can be said that everyone experiences a life “controlled by hormones.” “Growth and development,” a keyword accompanying individuals from birth, naturally shares countless endocrine stories with the growth hormone that regulates it. In 2023, China surpassed the United States to become the largest market for human growth hormone globally. According to Frost & Sullivan, the size of China’s human growth hormone market rapidly increased from RMB 4 billion in 2018 to RMB 11.6 billion in 2023, representing a compound annual growth rate (CAGR) of 23.9%, and is projected to continue growing to RMB 28.6 billion by 2030.

Pediatric Growth Hormone Deficiency (PGHD) requires long-term treatment to maintain patients’ growth hormone levels within an optimal range, thereby imposing higher demands on medication adherence. This need has also shifted the innovation trend in related drug development from once-daily to once-weekly or even longer dosing intervals.

On the production front, VISEN has partnered with WuXi Biologics, signing agreements for the technology transfer and localized manufacturing of lonapegsomatropin, thereby further enhancing drug accessibility. In retrospect, CEO Lu Anbang’s strategic and commercial considerations for VISEN Pharmaceuticals have not only been “steady and precise” but also “far-sighted in planning.”

Currently, the vast majority of growth hormone products marketed in China are short-acting formulations requiring daily injections, with only one weekly formulation achieving long-acting effects through permanent protein modification technology. Despite the proliferation of long-acting technologies, options remain extremely limited for protein and peptide drugs. The key challenge lies in the complex mechanisms of tissue distribution, receptor binding, and metabolic clearance of hormones within the body, making it difficult to achieve prolonged action without compromising efficacy and safety. Market and clinical demands for innovation are driving the expansion and advancement of innovative technologies.

Taking Lonapegsomatropin as an example, it utilizes the innovative long-acting technology platform known as TransCon. This approach differs fundamentally from previous long-acting drug design strategies based on permanent modification. The core of this technology lies in temporarily linking a prototype drug, which is identical to endogenous growth hormone in both molecular structure and pharmacological mechanism, to an inert carrier via a linker moiety. Enabled by this technology, Lonapegsomatropin slowly releases unmodified growth hormone molecules in vivo. While achieving a long-acting effect, its active ingredient—the unmodified growth hormone molecule—exerts a mechanism of action identical to that of “natural” growth hormone. Phase 3 clinical trials conducted globally and in China have demonstrated that Lonapegsomatropin is significantly superior to daily growth hormone formulations in promoting height gain.

According to published reports, Professor Luo Xiaoping, the principal investigator of the Phase 3 clinical trial of lonapegsomatropin in China, provided further interpretation of the primary study data, stating: “Starting from Week 13, the AHV (annualized height velocity, cm/year, i.e., the average height gain of all subjects over a one-year treatment period) was significantly higher in the lonapegsomatropin group than in the daily growth hormone group. Compared with baseline, the height standard deviation score (HV SDS, an indicator measuring the degree of deviation of a child’s height from the average height of children of the same age and sex) at Week 52 increased by 1.01 and 0.83 in the lonapegsomatropin group and the daily growth hormone group, respectively (p=0.0015), showing a statistically significant difference.”

Further evidence indicates that multiple clinical trials conducted both domestically and internationally have demonstrated the positive efficacy and favorable safety profile of lonapegsomatropin in pediatric patients with PGHD, including its Phase 2 and Phase 3 clinical trials. In the known global Phase 3 long-term open-label enliGHten trial, lonapegsomatropin also showed sustained efficacy and long-term safety for up to 5 years. Given its performance across multiple trials, which has led to improved clinical treatment experiences for patients by enhancing adherence and reducing local reactions, the long-term efficacy and safety of lonapegsomatropin have been supported by clinical trials worldwide and recognized by researchers.

Based on precise insights into the diagnostic characteristics of endocrine disorders and growth-related diseases, VISEN has formulated targeted R&D and medical strategies. Starting from clinical research and academic advancements, the company aims to ensure that the unique advantages of TransCon technology and the clinical profile of lonapegsomatropin are widely understood and recognized by specialists through the dissemination of robust clinical study results. In recent years, VISEN has hosted high-level academic conferences^1-3, actively participated in academic conferences organized by various levels of academic societies/associations across China, as well as other newly established academic exchange activities, engaged in in-depth interactions with nearly 30 leading academic figures in the field of pediatric endocrinology nationwide, and gained recognition from top experts in the field.

Over the past six years, VISEN has actively participated in and funded foundational epidemiological research on other rare endocrine disorders. By collaborating with national associations, research institutions, and leading academic experts, VISEN strives to provide the essential scientific data needed to formulate diagnosis and treatment assurance strategies for these conditions. Adhering to the principle of “academics first, science-based,” VISEN has established a strong academic reputation among pediatric endocrinologists across China. For future commercialization, VISEN plans to build a specialized and highly proficient commercial team focused on the clinical data of lonapegsomatropin, dedicated to conveying value to specialists and addressing unmet clinical needs. Starting from the patient journey through diagnosis and treatment, VISEN will gradually establish a closed-loop system ranging from disease awareness education to drug accessibility, while promoting innovative payment models and after-sales service systems. Ultimately, this approach aims to deliver patient-centered, systematic disease management solutions. It is reported that VISEN has entered into a strategic partnership with United Family Healthcare to expand its collaboration in the high-end medical services sector. This partnership seeks to provide Chinese pediatric patients with globally leading treatment options while ensuring they receive high-quality medical care.

As the global healthcare sector continues to advance, the research, development, and application of innovative drugs have become key to improving human quality of life. Against this backdrop, the collaboration between VISEN and Shanghai Pharma represents a strategic move by both parties to seize opportunities and meet challenges.

VISEN’s strategic choices and focused product pipeline not only reflect the company’s profound understanding and precise grasp of clinical needs, but also demonstrate its ambition and determination in the field of innovative medicines. In the future, with the approval and market launch of lonapegsomatropin in China, we have reason to believe that it will bring a revolutionary treatment option to pediatric patients with growth hormone deficiency in the country. VISEN’s focus and professionalism, along with its deep expertise in endocrine disorders, will enable it to establish a strong foothold in this field. As the commercialization of products such as lonapegsomatropin continues to advance, VISEN is poised to play an even greater role in the future treatment of endocrine diseases, bringing more hope to Chinese patients and creating greater value for society.

References

1. https://mp.weixin.qq.com/s/JINr0OAFK2aONCyTN8VYpA

2. https://mp.weixin.qq.com/s/225SkD8yiCS789HMo2XbXg

3. https://mp.weixin.qq.com/s/PYPFMN7qKJfkdcsDNT-pcA