Pediatric Innovative Medicines Enter Commercialization: An Exclusive Interview with Zhang Cheng of YouEr Pharma

2023 was a milestone year for Pediatrix Therapeutics, a pharmaceutical company in the pediatric field.

In April, Pediatrix Therapeutics' Yunuoqing for the treatment of nocturnal enuresis^®Desmopressin Oral Solution was approved for market launch and successfully passed the national medical insurance negotiation within less than a year, gaining inclusion in the National Reimbursement Drug List; in December, the company’s product Youningrui, indicated for the treatment of attention-deficit/hyperactivity disorder (ADHD) in children aged 6 years and older,^®(Methylphenidate Hydrochloride) Extended-Release Dry Suspension and Extended-Release Chewable Tablets Officially Approved for Market Launch.

Since its establishment in 2021, Pediatrix Therapeutics has brought three pediatric drug products to market in less than three years. Meanwhile, the company completed clinical trials for two additional drugs this year and is poised to file for marketing approval in 2024.

Pediatrix Therapeutics’ commercialization strategy is set to expand further.“For Pediatrix Therapeutics, 2023 was a year in which our team achieved remarkable results through hard-fought efforts. We focused our resources on overcoming various challenges to attain these milestone achievements,” said CEO Zhang Cheng, reflecting on the journey of 2023.

During its first two years, Pediatrix Therapeutics focused primarily on pipeline development.With the product launch in 2023, the company reached its first major turning point—the commencement of commercialization.Through a conversation with Zhang Cheng, CEO of Pediatrix Therapeutics, the company’s keen market insight, steady approach to drug development, mature management, and unique perspectives on the pediatric pharmaceutical sector were gradually revealed.

Zhang Cheng stated that Pediatrix Therapeutics identified unmet clinical needs in the field of pediatrics at the time of its founding.However, the more critical step lies in translating these needs into truly safe and effective drugs that ultimately gain regulatory approval for marketing and are used by the pediatric population.

In April 2021, to prepare for the company’s initial establishment, Pediatrix Therapeutics launched a research project targeting frontline pediatricians. With over 200 participants from hospitals of varying tiers and across different cities in China, the initiative aimed to precisely identify the true unmet clinical needs in the field of pediatric medicines.

This may be the largest-scale survey conducted among pediatricians in China to date.Through market research, Pediatrix Therapeutics identified unmet needs in indications such as nocturnal enuresis and attention-deficit/hyperactivity disorder (ADHD), leading to the initiation of projects for desmopressin and extended-release methylphenidate hydrochloride.

Initially, nocturnal enuresis was not regarded as a disease; with changes in social lifestyles and advances in epidemiology, this condition began to become common among children. However,Previously, only desmopressin tablets for central diabetes insipidus in adults were available in China. Due to the considerable inconvenience of dose titration with these tablets, they are not suitable for pediatric patients, thus leaving a significant gap in the treatment of nocturnal enuresis in children.Based on this requirement, Pediatrix Therapeutics ultimately approved the project to develop a pediatric-friendly dosage form—desmopressin oral solution.

This drug’s project initiation and commercialization have both received strong support at the national policy level. Zhang Cheng introduced that desmopressin is included in China’s second batch of the List of Pediatric Drugs Encouraged for Research, Development, and Application, making it an urgently needed medication for pediatric clinical practice in China. Due to thorough preliminary project research, Pediatrix Therapeutics’ product was exempted from clinical trials, completing the entire process from pharmaceutical development to regulatory approval in just two years. During the national medical insurance negotiations at the end of 2023, the National Healthcare Security Administration provided significant policy incentives for the inclusion of innovative drugs in the National Reimbursement Drug List and vigorously encouraged the application for pediatric drugs and pediatric-specific formulations, thereby promotingDesmopressin Oral Solution Successfully Included in the National Reimbursement Drug List, Further Broadening Pediatrix Therapeutics’ Commercialization Pathway.

The market launch of Desmopressin Oral Solution stands as a testament to Pediatrix Therapeutics’ acute market insight and unwavering commitment to pharmaceutical development, while the approval of Methylphenidate Hydrochloride Extended-Release Chewable Tablets and for Dry Suspension demonstrates the resilience and exceptional execution capabilities of the company’s team.

In China, medications for treating attention deficit hyperactivity disorder (ADHD) in children are scarce; methylphenidate hydrochloride extended-release tablets are a first-line therapy, butThe tablets are large, difficult to swallow, and the dosage cannot be adjusted.Pediatrix Therapeutics aims to address this predicament by providing more and better treatment options.

In fact, the product registration process for methylphenidate hydrochloride extended-release chewable tablets and dry suspensions has not proceeded smoothly compared to that of desmopressin oral solution. Given that both products are classified as controlled psychotropic substances, they are subject to an exceptionally complex and rigorous approval process, distinct from the regulatory pathways for conventional drugs.

To initiate the product development project, it was first necessary to obtain project approval from the Department of Drug Safety Supervision. To this end, Pediatrix Therapeutics invited 13 leading experts in the field of attention-deficit/hyperactivity disorder (ADHD) treatment in China for extensive consultations, securing professional guidance and support for preparing the submission documents, which ultimately led to the successful acquisition of the approval letter. Following project approval, the team focused on exploring and optimizing the drug registration pathway, engaging in active discussions with the Center for Drug Evaluation (CDE) and comprehensively presenting existing clinical data from abroad. Through relentless efforts, methylphenidate hydrochloride extended-release chewable tablets and powder for oral suspension became the second methylphenidate formulation approved in China, marking the entry of pediatric ADHD into an era of precision therapy.

Zhang Cheng told VCBeat that when Pediatrix Therapeutics was first established, its goal was not to become a biotech company, but ratherPositioned as a pharmaceutical company focused on the field of pediatric treatment.

“Therefore, we focus the company’s core competencies on two areas: clinical development capabilities and commercialization capabilities.”

To deepen these two competitive advantages,Pediatrix Therapeutics has adopted a dual-drive strategy of “product in-licensing and independent R&D,” accumulating a pipeline of 11 products.This has laid a solid foundation for the rapid growth of Pediatrix Therapeutics over the past three years and established differentiated advantages for the company’s products.

Desmopressin oral solution, as a novel pediatric formulation, overcomes the difficulty of swallowing associated with tablets and allows for more precise and flexible dose adjustments, thereby reducing the recurrence rate. It not only acts rapidly but also ensures safety.

Methylphenidate Hydrochloride Extended-Release Chewable Tablets and Extended-Release Dry Suspension also feature innovative dosage forms with a more patient-centric design. Leveraging the internationally exclusive Liqui XR extended-release technology patent, both medications achieve a rapid onset of action within 45 minutes and sustain therapeutic effects for 12 hours. They allow for flexible dose adjustments to meet the needs of individualized ADHD treatment, balancing efficacy and safety. Furthermore, Pediatrix Therapeutics has carefully considered the medication-taking habits of pediatric patients. To enhance compliance, the company uses fruit flavors to mask the unpleasant taste of the drugs, thereby increasing children’s willingness to take their medication and improving adherence. These two dosage forms complement each other, catering to the medication needs of children across different age groups.

PT-003, a treatment for systemic acute allergic reactions, completed its pivotal clinical trials in 2023 and is scheduled to file for market approval this year. This drug introduces an innovative approach to epinephrine administration, transitioning from injectable delivery to nasal spray. Zhang Cheng noted that PT-003 will expand the usage scenarios for epinephrine, allowing patients to carry it with them and significantly alleviating the challenge of lacking immediate access to emergency medical care, thereby buying more critical time for first aid.

When asked how Pediatrix Therapeutics managed to achieve so much in just three short years, Zhang Cheng candidly stated that the company’s core competitiveness, combined with the support of its investors, has together built the foundation of Pediatrix Therapeutics.

“Since its inception, Pediatrix Therapeutics has maintained robust communication with investors from firms such as STOA Capital and Hongyuan Capital. We have reached consensus and mutual understanding on multiple fronts, and they have extended significant trust and support to the company.”

In addition to investors staying aligned with the company and placing full trust in the management team, Zhang Cheng noted that the investors of Pediatrix Therapeutics have woven a rich resource network for the company’s development. “The portfolio companies of our investment partners span the globe, forming an invaluable resource pool. It is this authoritative endorsement that has facilitated mutual trust and collaboration between Pediatrix Therapeutics and other enterprises.” Benefiting from the extensive global experience of its founding investors and management team in drug development and commercialization, Pediatrix Therapeutics enjoys unique advantages, enabling it to provide substantial support to children and families in China.

To date, Pediatrix Therapeutics has launched three products, with two additional products poised to submit New Drug Applications (NDAs).This demonstrates the company’s robust and agile clinical development and organizational capabilities, while highlighting promising market prospects by addressing genuine unmet clinical needs. Pediatrix Therapeutics has moved one step closer to its goal of becoming a leading pharmaceutical enterprise in the pediatric medicine sector.

The positive achievements made in 2023 have charted the course for Pediatrix Therapeutics’ corporate planning for the new year.Zhang Cheng summarized the key development theme for 2024 and even the next three years as “innovative business models.”“We have successfully demonstrated Pediatrix Therapeutics’ clinical development capabilities in the field of pediatric medicines. Over the next three years, the company will shift its focus to building its own commercialization capabilities.”

Zhang Cheng has nearly 30 years of extensive experience in commercialization, product introduction, and national medical insurance negotiations.He believes that in today’s China, the commercialization of any product requires exploring innovative business models.Due to shifts in the competitive landscape and drug cost structures, today’s pharmaceutical market is fundamentally different from the past. Adhering to the commercialization pathways of the past two to three decades will inevitably fail to address current market dynamics, akin to marking the boat to find the sword.

Pediatrix Therapeutics has its own unique insights and understanding of how to establish innovative business models. In the future, Pediatrix Therapeutics plans to transform the organizational structure of its sales team,Actively seek collaborative promotion partners,Achieve win-win cooperation in pharmaceutical sales. Meanwhile, given the crucial roles that parents and teachers play in the management of pediatric diseases, Pediatrix Therapeutics hopesCentralize resource allocation with a patient-centric approach, striving to build a comprehensive ecosystem centered on parents that covers the entire continuum from disease education, screening, consultation, diagnosis, and treatment to disease management.We call on more people to prioritize pediatric diseases and focus on superior treatment options. From the perspective of corporate development, pipeline layout is also crucial.Continuous and systematic pipeline arrangement and product lifecycle management,This will ensure the steady growth and sustainable development of the company's commercialization.These transformations and innovations in business models will be the areas that Pediatrix Therapeutics continues to explore in the future.

While setting development goals for the company, Zhang Cheng also holds high expectations for the future of China’s pediatric drug market. He has observed that in recent years, an increasing number of pharmaceutical companies have begun to focus on the pediatric drug sector, with the government providing substantial policy guidance. However, policy incentives alone are insufficient; it is the improvement of laws and regulations that will further facilitate the practical implementation of pediatric drug R&D by pharmaceutical enterprises, thereby achieving true equity in pediatric medication.

Meanwhile, Zhang Cheng hopes that more companies will pay attention to the pediatric market. "To some extent, the development of a therapeutic area benefits from everyone's joint efforts. I hope that more Chinese pharmaceutical companies will focus on children's health and pediatric drugs, so as to form a complete ecosystem."

The story of Pediatrix Therapeutics will continue, growing in tandem with China’s pediatric treatment sector.