SHANGHAI, April 10, 2026 /PRNewswire/ -- Shanghai Ark Biopharmaceutical Co., Ltd. ("ArkBio") today announced that the first cohort of healthy volunteers has been dosed in a phase I clinical trial of AK0406, a novel long-acting antiviral drug-Fc conjugate (ADFC) drug for influenza infection, following approval by the Human Research Ethics Committee (HREC) in Australia. This milestone marks the first-in-human (FIH) evaluation of AK0406 and an important step in the global clinical development of ArkBio's influenza pipeline.

AK0406 is a next-generation, long-acting ADFC candidate discovered and developed by ArkBio. By conjugating a potent small-molecule antiviral to the antibody Fc domain, AK0406 is designed to combine the direct antiviral effect of the payload with Fc-mediated immune clearance and an extended half-life. Preclinical data show that AK0406 exerts broad-spectrum, high-potency activity against both influenza A and B viruses, maintains immune effector function, and provides prolonged exposure. Compared with first-generation ADFC molecules, AK0406 is engineered to offer an optimized profile for both prophylaxis and treatment of influenza infection.

The phase I trial is a single-center, randomized, double-blind, placebo-controlled study designed to assess the safety, tolerability, and pharmacokinetic (PK) profile of AK0406 in healthy adult participants in Australia. Data from this FIH study will inform subsequent dose selection and future proof-of-concept trials in populations at risk for influenza infection.

Influenza remains a significant global public health burden, causing substantial morbidity and mortality each year and posing an ongoing pandemic threat. Current preventive measures, including seasonal vaccination, are challenged by antigenic drift, strain-selection uncertainty, and reduced effectiveness in the general population, especially in vulnerable groups such as older adults and immunocompromised individuals. With its long-acting, broad-spectrum ADFC design, AK0406 has the potential to address key limitations of existing therapies and offer improved options for both prevention and treatment of seasonal influenza and potential pandemic outbreaks. ArkBio will continue to accelerate the clinical development of AK0406 and work with global partners to advance better prevention and treatment options for patients worldwide.

About ArkBio

ArkBio is a commercial-stage biotechnology company focused on the discovery and development of innovative therapeutics for respiratory/lung and pediatric diseases. Founded in 2014, the company has established proprietary technology platforms and a differentiated R&D pipeline through internal innovation and strategic collaborations.

Key pipeline assets include: ziresovir (AK0529), the first direct-acting antiviral for RSV with positive pivotal phase 3 results; AK3280, a potentially best-in-class anti-fibrotic agent with positive phase 2 results in idiopathic pulmonary fibrosis and is advancing into Phase 3 registrational trials; AK0901, approved and commercialized in China for the treatment of ADHD. The company also has multiple first-in-class or best-in-class innovative candidates in clinical or preclinical development.

ArkBio has established strategic partnerships with multinational pharmaceutical companies including Roche and Genentech, leading academic institutions such as The Scripps Research Institute and the Institute of Microbiology of the Chinese Academy of Sciences, Qilu Pharmaceutical as well as other domestic and international biotech companies and CROs.

For more information, please visit: www.arkbiosciences.com 
Investor Inquiries: IR@arkbiosciences.com 

SHANGHAI, Feb. 12, 2026 /PRNewswire/ -- Shanghai Ark Biopharmaceutical Co., Ltd. ("ArkBio") today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for AK3280, a novel anti-fibrotic therapy for the treatment of idiopathic pulmonary fibrosis (IPF). This clearance enables ArkBio to initiate a Phase 2 proof-of-concept (PoC) clinical trial of AK3280 in the United States.

The Phase 2 study is a multi-center, randomized, partially double-blind, placebo- and active-controlled trial designed to evaluate the efficacy, safety, and pharmacokinetics of oral AK3280 in patients with IPF. Authorization to proceed with a U.S. Phase 2 study represents a significant milestone in the global clinical development of AK3280.

IPF is a progressive, irreversible, and ultimately fatal interstitial lung disease characterized by fibrotic remodeling of lung tissue that leads to respiratory failure. The median survival following diagnosis is approximately 2–5 years. Currently approved therapies, including pirfenidone, nintedanib, and nerandomilast, can slow disease progression; however, their clinical benefit remains limited and they are frequently associated with gastrointestinal adverse events such as diarrhea and nausea. These tolerability challenges contribute to poor long-term adherence in a substantial proportion of patients. There remains a significant unmet need for therapies that provide improved efficacy with enhanced safety and tolerability profiles.

AK3280 is an optimized, small-molecule, broad-spectrum anti-fibrotic agent. In a Phase 2 proof-of-concept study conducted in China, AK3280 demonstrated encouraging clinical activity, including a statistically significant, dose-dependent absolute increase from baseline in forced vital capacity (FVC) at Week 24. Improvements were also observed across additional lung function parameters, suggesting meaningful clinical benefit. Importantly, AK3280 exhibited a favorable safety and tolerability profile, with no apparent increase in the gastrointestinal adverse effects commonly associated with currently available IPF therapies.

FDA clearance of the IND marks a pivotal step in the global development of AK3280. The upcoming international Phase 2 PoC trial is expected to generate critical clinical data to support future regulatory submissions and potential commercialization in the United States and other major markets. ArkBio remains committed to advancing AK3280 globally and to delivering more effective and better-tolerated treatment options for patients living with IPF.

About ArkBio

ArkBio is a commercial-stage biotechnology company focused on the discovery and development of innovative therapeutics for respiratory and pediatric diseases. Founded in 2014, the company has established proprietary technology platforms and a differentiated R&D pipeline through internal innovation and strategic collaborations.

Key pipeline assets include: Ziresovir (AK0529), the first direct-acting antiviral for RSV with positive pivotal Phase 3 results; AK3280, a potentially best-in-class anti-fibrotic agent with positive Phase 2 results in idiopathic pulmonary fibrosis; AK0901, approved in China for the treatment of ADHD and currently commercialized.

ArkBio has established strategic partnerships with multinational pharmaceutical companies and leading academic institutions, including Roche, Genentech, The Scripps Research Institute, and the Institute of Microbiology of the Chinese Academy of Sciences, as well as domestic and international biotech companies and venture capital partners.

For more information, please visit: www.arkbiosciences.com
Investor Inquiries: IR@arkbiosciences.com

SHANGHAI, Jan. 8, 2026 /PRNewswire/ -- Shanghai Ark Biopharmaceutical Co., Ltd. ("ArkBio") today announced that China's National Medical Products Administration (NMPA) has approved the New Drug Application for Serdexmethylphenidate Chloride and Dexmethylphenidate Hydrochloride Capsules (Aizhida) for treatment of Attention Deficit Hyperactivity Disorder (ADHD) in patients 6 years of age and older in China.

Aizhida is a central nervous system stimulant indicated for the treatment of ADHD. Each once-daily capsule contains serdexmethylphenidate (SDX), a prodrug of dexmethylphenidate, co-formulated with immediate-release dexmethylphenidate (d-MPH) in a fixed ratio designed to provide both rapid and extended symptom control.After ingestion, the immediate-release d-MPH component is rapidly absorbed to provide early onset of effect, while SDX is gradually converted to d-MPH in the lower gastrointestinal tract, supporting sustained therapeutic exposure throughout the day. Aizhida was approved by the U.S. FDA in March 2021 as a once-daily treatment option for patients with ADHD 6 years of age and older.

ADHD is a common, chronic neurodevelopmental disorder that typically begins in childhood and may persist into adulthood. It is primarily characterized by age-inappropriate levels of inattention and/or hyperactivity-impulsivity. In China, the prevalence of ADHD in children and adolescents is estimated at approximately 6.4%, affecting over 23 million individuals. Studies have shown that symptoms persist into adolescence for 60%-80% of patients, and into adulthood for roughly half of cases. Current ADHD treatment options in China are limited, and currently available medications do not fully meet clinical needs in terms of both efficacy and safety for many patients. Some patients discontinue treatment due to suboptimal response, side effects, or dosing inconvenience, highlighting the need for additional therapeutic options.

Robust Clinical Data Addresses Unmet Need: In a pivotal Phase III clinical trial in Chinese patients with ADHD, Aizhida met both the primary and key secondary endpoints. The study demonstrated statistically significant and clinically meaningful improvements in core ADHD symptoms compared to placebo at all assessment time points. With limited treatment options in China and historical supply constraints for traditional single-agent therapies, the approval of this innovative combination product offers an important new choice for clinicians and patients.

Unique Dual Mechanism for Rapid Onset and All-Day Coverage: Aizhida combines immediate-release and prodrug-based extended-release technologies. Its 'rapid onset + full-day coverage' profile positions it to reshape the ADHD treatment paradigm in China. As the first ADHD medication in China to deliver both rapid onset and sustained symptom control, it fills a key clinical gap and has the potential to become a preferred treatment option.

Professor Yi Zheng, Chief Expert at Beijing Anding Hospital, Capital Medical University, and Lead Principal Investigator of the Aizhida Phase III trial in China, commented: "ADHD is one of the most common neurodevelopmental disorders in childhood, with potential impact lasting into adulthood, posing long-term challenges to learning, social functioning, and family life. While the prevalence in Chinese children is around 6.4%, treatment options—especially innovative ones balancing efficacy and safety—remain insufficient. The approval of Aizhida provides clinicians with a new therapeutic tool. We anticipate its real-world application will help improve the treatment landscape for ADHD patients in China, offering new hope particularly for those with suboptimal response or tolerance to existing therapies."

Professor Jing Liu, Director of the Child Psychiatry Center, Peking University Sixth Hospital, and Lead Principal Investigator of the Aizhida Phase III trial in China, added: "We are committed to advancing standardized diagnosis and treatment for pediatric mental disorders in China. Comprehensive intervention is essential for ADHD, with pharmacotherapy being a foundational component. The approval of a new drug not only increases options but also prompts deeper reflection on optimizing treatment strategies. We hope the approval of Aizhida will enrich ADHD treatment approaches and contribute to better long-term outcomes, which is the ultimate goal of our clinical research."

About ArkBio

ArkBio is a global biotech company focused on developing innovative therapeutics for respiratory and pediatric diseases. Founded in 2014, it has built core technology platforms and a differentiated R&D pipeline through in-house R&D efforts and external collaboration. Key drug assets include ziresovir (AK0529), the first direct-acting RSV antiviral with positive pivotal phase III results, and AK3280, a potentially best-in-class drug with positive phase 2 results to treatment idiopathic pulmonary fibrosis. Another core product Aizhida (AK0901) was recently approved in China for ADHD and has now entered the commercial launch phase. 

ArkBio has established strategic partnerships with several multinational pharmaceutical companies and academic institutes, including Roche, Genentech, the Scripps Research Institute, the Institute of Microbiology of Chinese Academy of Sciences, domestic and international biotechnology companies, as well as venture capital institutions.

For more information, please visit: www.arkbiosciences.com 

Investor Inquiries: IR@arkbiosciences.com 

SHANGHAI, Oct. 30, 2025 /PRNewswire/ -- Shanghai Ark Biopharmaceutical Co., Ltd. ("ArkBio") today announced the initiation of a Phase II clinical study of AK0610, a fully human monoclonal antibody designed to prevent respiratory syncytial virus (RSV) infection. Building on ArkBio's previous experience and knowledge in development of RSV therapeutics, this milestone marks a major step forward in RSV prevention and enriches the company's innovative anti-RSV drug portfolio.

The multicenter, randomized, double-blind, placebo-controlled, dose-escalation phase II study will assess the safety, tolerability, and pharmacokinetics of AK0610 Injection in healthy infants in China. The study will be conducted at five centers nationwide, jointly led by Professor Xin Ni of Beijing Children's Hospital, Capital Medical University, and Professor Hanmin Liu of West China Second Hospital, Sichuan University.

RSV is a leading cause of acute lower respiratory tract infections (ALRTI) in children under 5 years of age worldwide. More than 80% of children become infected before their first birthday, and nearly all have been exposed at age two. Among those infected, approximately one-third may develop severe ALRTI, such as bronchiolitis or pneumonia. Infants under one year of age, particularly preterm infants, are at greatest risk of developing serious RSV-related complications, often requiring hospitalization. Given China's large infant population and persistent RSV prevalence, the country faces a significant healthcare burden, underscoring the urgent need for novel, long-acting preventive interventions.

AK0610 is a monoclonal antibody targeting the pre-fusion F protein of RSV. It was isolated from a convalescent infant and subsequently engineered to enhance durability. Distinguished by its unique binding epitope and mechanism of action, AK0610 maintains robust neutralizing activity across RSV variants, especially RSV B strains. Preclinical data demonstrated strong antiviral activity with an excellent safety profile. In the completed phase I trial in healthy adults, AK0610 was safe, well-tolerated, and exhibited favorable pharmacokinetic properties. With an extended half-life, a single dose may provide season-long protection, positioning AK0610 as a promising new-generation long-acting antibody for prophylaxis of RSV infection in infants.

Professor Xin Ni stated:
"RSV remains the leading viral cause of hospitalization among infants and young children in China, especially those under one year of age, placing a heavy burden on families and the healthcare system. There is an urgent need for domestically developed innovative preventive antibodies capable of offering durable protection throughout the RSV season. The initiation of AK0610's Phase II clinical study is an important milestone in advancing such preventive strategies. Evaluating its safety and pharmacokinetics in healthy infants is an essential step towards developing this antibody for China as well as the global vast infant population. We are optimistic that the study will pave the way for this promising drug candidate to safeguard the healthy growth of infants globally."

Professor Hanmin Liu added:
"AK0610 is a fully human monoclonal antibody with a unique mechanism of action and excellent clinical potential. Its potent, highly specific neutralizing activity and extended half-life make it a strong candidate for RSV prevention. The rigorously designed phase II study will provide important data on its safety and pharmacokinetic characteristics in infants, establishing a solid foundation for future pivotal trials. Together with participating centers across the nation, we are committed to conducting this clinical trial with scientific rigor with a goal to reduce the burden of RSV-related diseases among infants."

About ArkBio

ArkBio is a global biotech company focused on developing innovative therapeutics for respiratory, infectious, and pediatric diseases. Founded in 2014, it has built core technology platforms and a differentiated R&D pipeline through in-house R&D efforts and external collaboration. Key drug assets include ziresovir (AK0529), the first direct-acting RSV antiviral with positive pivotal phase III results, and AK0901, an ADHD therapeutic drug, both of which have filed NDA to China CDE for market approval.

ArkBio has established strategic partnerships with several multinational pharmaceutical companies and academic institutes, including Roche, Genentech, the Scripps Research Institute, the Institute of Microbiology of Chinese Academy of Sciences, domestic and international biotechnology companies, as well as venture capital institutions.

For more information, please visit: www.arkbiosciences.com 

Investor Inquiries: IR@arkbiosciences.com 

SHANGHAI, July 16, 2025 /PRNewswire/ -- In the newly released "Paediatric drug optimization for respiratory syncytial virus (PADO-RSV) meeting report" by Global Accelerator for Paediatric formulations (GAP-f), the World Health Organization (WHO) host network, ziresovir (10mg) has been included in the PADO-RSV priority list. Ziresovir, developed by Shanghai Ark Biopharmaceutical Co., Ltd. ("ArkBio"), is the first and only RSV antiviral drug to be included in the list. This is the first time WHO has included an anti-RSV drug developed from China in the paediatric drug optimization priority list.

Filling a Global Gap: Ziresovir Leads RSV Antiviral Development

RSV is the leading pathogen of lower respiratory tract infections (LRTIs) in infants and young children globally, causing approximately 33 million cases of LRTIs, 3.6 million hospitalizations, and 101,400 deaths annually, with 97% of deaths occurring in low- and middle-income countries (LMICs). Despite the severe threat, current specific treatment options for RSV are extremely limited, and there is a clinical urgent need for safe, effective, and child-appropriate innovative drugs. The "Paediatric drug optimization for respiratory syncytial virus (PADO-RSV)" project initiated by WHO/GAP-f aims to systematically review the global R&D pipeline, clarify R&D priorities, and promote the development and accessibility of RSV prevention and treatment tools more suitable for the paediatric population. With its clear clinical value, child-friendly design, and good safety profile, ziresovir becomes the only product in the antiviral drug category selected for the PADO-RSV priority list, demonstrating its huge potential in the paediatric RSV treatment globally.

WHO pointed out in the report that ziresovir's research results are of great significance to the global RSV treatment field and recommended conducting diverse clinical studies in broader populations to further verify its applicability and effectiveness.

ArkBio will actively respond to WHO's advice with a plan to expand ziresovir's clinical development in other countries and regions. By collaborating with international scientific institutions and public health organizations, ArkBio aims to rapidly advance the drug's global registration and accessibility. Simultaneously, ArkBio is deeply aware of the importance of global public health, particularly concerned about the challenges faced by low- and middle-income countries. The company will formulate drug accessibility plans specifically for these countries to ensure ziresovir can be supplied at reasonable prices globally wherever it is needed, contributing to the prevention and treatment of RSV infection.

"ArkBio has a mission to 'help patients inspire and thrive'", Dr. Jim Wu, CEO of ArkBio, stated. "The inclusion of ziresovir in the WHO PADO-RSV priority list is not only an affirmation of the years of effort by ArkBio scientists, but also an inspiration for our corporate mission. The recognition of ziresovir would allow us to expedite its global development and realize the potential on the global stage. We will ensure the innovation can not only benefit patients in China but the very needed patients worldwide. This is not just our goal but our social responsibility. ArkBio will strive to contribute more to address global public health issues."

About GAP-f

GAP-f stands for the Global Accelerator for Paediatric Formulations Network. Hosted by WHO, it operates throughout the drug development lifecycle to accelerate the research, development, and dissemination of optimized pediatric formulations. Identifying priorities is the first step toward a targeted R&D approach. Establishing a priority list of the most urgently needed pediatric formulations is crucial, as it helps focus the efforts and resources of researchers and suppliers on specific dosage forms and formulations that address children's most critical needs.

About PADO

PADO stands for Paediatric Drug Optimization. It is a WHO's legally binding international priority (based on WHA69.20 resolution) that accelerates the development of high-demand pediatric drugs by building consensus among regulators, funders, and manufacturers. It aims to identify priority products for R&D and define their ideal characteristics. It has been successfully implemented in areas including human immunodeficiency virus (HIV), hepatitis C, tuberculosis, COVID-19, antibiotics, neglected tropical diseases, and childhood cancers. These initiatives demonstrate that, against the backdrop of a fragmented and small pediatric drug market, such efforts hold potential and deliver tangible impacts in accelerating access to optimized formulations.

About Ziresovir

Ziresovir is a novel small-molecule RSV fusion (F) protein inhibitor. It binds to the pre-F protein conformation of the virus and prevents viral entry into human cells. Ziresovir can also suppress RSV viral transmission by blocking cell-to-cell fusion through the formation of "syncytia", a characteristic event of RSV infection of host cells. It is the first oral anti-RSV drug that has completed a phase 3 pivotal clinical study with positive results. The phase 3 results have been published in The New England Journal of Medicine and The Lancet Child & Adolescent Health. Ziresovir has received Breakthrough Therapy designation by the National Medical Products Administration (NMPA) of China.

About ArkBio

ArkBio is a global biotech company focused on developing innovative therapeutics for respiratory, infectious, and pediatric diseases. Founded in 2014, it has built core technology platforms and a differentiated R&D pipeline through in-house R&D efforts and external collaboration. Key drug assets include ziresovir (AK0529), the first direct-acting RSV antiviral with positive pivotal phase III results, and Azstarys (AK0901), an FDA-approved ADHD therapeutic drug.

ArkBio has established strategic partnerships with several multinational pharmaceutical companies and academic institutes, including Roche, Genentech, the Scripps Research Institute, the Institute of Microbiology of Chinese Academy of Sciences, domestic and international biotechnology companies, as well as venture capital institutions.

For more information, please visit: www.arkbiosciences.com
Investor Inquiries: IR@arkbiosciences.com

SHANGHAI, June 16, 2025 /PRNewswire/ -- Shanghai Ark Biopharmaceutical Co., Ltd. ("ArkBio") today announced that the National Medical Products Administration (NMPA) has officially accepted its New Drug Application (NDA) for Azstarys (serdexmethylphenidate/dexmethylphenidate extended-release capsules) and granted it Priority Review designation. The investigational therapeutic drug is indicated for the treatment of Attention-Deficit/Hyperactivity Disorder (ADHD) in patients aged six years and above.

First Methylphenidate Combination Drug Poised to Benefit Patients in China

Azstarys (AK0901) is the first and currently the sole globally approved ADHD combination therapeutic pairing immediate-release dexmethylphenidate (d-MPH) with the prodrug serdexmethylphenidate (SDX). Approved by the U.S. FDA in 2021, Azstarys represents a new generation of methylphenidate medications, providing a dual-phase release profile that offers both rapid onset and sustained symptom control. This novel formulation sets a new benchmark in the management of ADHD for safety, efficacy, and convenience of administering for patients, making it a potential best-in-class ADHD therapeutic drug.

Pharmacokinetic Property: Biphasic Release for Rapid Onset and Whole-Day Symptom Control

• Rapid onset: Immediate-release d-MPH ensures symptom relief within 30 minutes, offering fast control over core ADHD symptoms.
• Extended control: SDX is metabolized slowly into d-MPH in intestine over time, ensuring up to 13 hours of therapeutic effect with reduced risk of evening rebound symptoms.
• Stable Release Profile: Designed to support the full span of a patient's day—from school or work to evening activities—Azstarys enhances adherence and minimizes sleep disruptions, suiting both pediatric and adult populations.

Abuse-Deterrent Design

Azstarys incorporates an inherent abuse-deterrent design through the inclusion of SDX, which undergoes slow conversion in the gastrointestinal tract. This limits peak plasma concentrations of d-MPH and significantly reduces the potential for misuse via non-oral routes—a key public health consideration in ADHD pharmacotherapy.

Robust Clinical Efficacy and Safety Confirmed in China Phase III Trial

• In a multicenter Phase III trial spanning eight leading clinical hospitals in China, Azstarys demonstrated statistically significant improvements over placebo in primary and secondary efficacy endpoints.
• The study reported no drug related serious adverse events, affirming Azstarys's favorable safety and tolerability profile, critical for long-term use in pediatric settings.

Transforming ADHD Treatment Paradigm in China

As the third-generation methylphenidate therapy, Azstarys delivers a new standard of care in ADHD management by integrating rapid onset and full-day symptom control into a single dose. Its future approval in China would mark the introduction of the first ADHD combination medication to the market, offering a better option for patients, families, and physicians.

Dr. Jim Wu, CEO of ArkBio, stated, 

"The acceptance of Azstarys' NDA and its designation for Priority Review by the NMPA reflect regulatory authority's favorable opinions in our science and clinical study results. ADHD remains critically underserved in China, and Azstarys is designed to directly meet the needs of patients who require both immediate symptom relief and sustained control. We are grateful to our investigators, trial participants, collaborators, and the NMPA for this milestone and we look forward to accelerating access to this important therapeutic in China market."

About ArkBio

ArkBio is a global biotech company focused on developing innovative therapeutics for respiratory, infectious, and pediatric diseases. Founded in 2014, it has built core technology platforms and a differentiated R&D pipeline through in-house R&D efforts and external collaboration. Key drug assets include ziresovir (AK0529), the first direct-acting RSV antiviral with positive pivotal phase III results, and Azstarys (AK0901), an FDA-approvedADHD therapeutic drug. 

ArkBio has established strategic partnerships with several multinational pharmaceutical companies and academic institutes, including Roche, Genentech, the Scripps Research Institute, the Institute of Microbiology of Chinese Academy of Sciences, domestic and international biotechnology companies, as well as venture capital institutions.

For more information about the company, please visit our website：www.arkbiosciences.com

SHANGHAI, May 16, 2025 /PRNewswire/ -- Shanghai Ark Biopharmaceutical Co., Ltd. ("ArkBio") today announced positive top-line Phase II study results for its novel anti-fibrotic drug AK3280 in treatment of idiopathic pulmonary fibrosis (IPF). The study, led by Professor Huaping Dai of the Department of Pulmonary and Critical Care Medicine at China-Japan Friendship Hospital, Beijing, was conducted across 31 clinical sites in China.

Global Clinical Challenge: Unresolved Treatment Needs in IPF

Idiopathic Pulmonary Fibrosis (IPF) is a progressive, irreversible, and fatal interstitial lung disease characterized by fibrotic remodeling of lung tissue, ultimately leading to respiratory failure, with a median survival period of only 2–5 years after diagnosis. Current therapeutics including pirfenidone and nintedanib have been available for years, however, with limited efficacy, significant adverse effects and poor long-term tolerability, highlighting the urgent need for novel and more effective therapeutics.

Phase 2 Proof-of-Concept Study of AK3280, a New Generation Broadly Active Antifibrotic Drug

AK3280 is a new generation broadly active antifibrotic drug that is optimized for pharmacological and pharmacokinetic properties based on current IPF drugs. Preclinical data indicate that it exhibits enhanced anti-fibrotic activity and improved pharmacokinetic properties, without the gastrointestinal tolerability issues and other toxicities associated with current therapeutics. Previous Phase I studies have demonstrated its favorable safety, tolerability, and human pharmacokinetic profile.

The completed randomized, double-blind, placebo-controlled Phase II proof-of-concept study evaluated the safety, tolerability and clinical efficacy of AK3280 in China IPF patients. Participants were randomized to receive AK3280 (100/200/300/400 mg BID) or placebo for 24 weeks. Clinical efficacy endpoints included forced vital capacity of lung (FVC and %pFVC), diffusing capacity of lung for carbon monoxide (DLco), the 6-minute walk test (6MWT) and patient reported St. George's Respiratory Questionnaire (SGRQ) scores. The high dose groups demonstrated FVC improvement from baseline, especially the 400 mg group which had the absolute FVC increased by 209.4 mL and a 6.4% adjusted %pFVC improvement from baseline, which is statistically significant compared to placebo (p=0.002 and 0.004, respectively). Other lung and respiratory functions have also got better. The drug exhibited a good safety and tolerability profile without the gastrointestinal intolerability issues associated with current IPF therapeutics.

Implications for Future IPF Therapeutics

This study with a rigorous multicenter, randomized, double-blind study design for 24 weeks followed by another 24 week's open-label study represents the first phase 2 proof-of-concept study of AK3280 in fibrotic patient populations. These findings not only demonstrate AK3280's potential to improve both lung function and respiratory outcome but also highlight its unique safety profile that may support long-term use, positioning it as a potential future standard-of-care therapy for IPF treatment.

Dr. Huaping Dai, principal investigator of the phase 2 study and professor at China-Japan Friendship Hospital, commented, "In the therapeutic landscape of IPF, the development of safer and more effective anti-fibrotic agents remains urgent. The most encouraging aspect of this Phase II study is AK3280's positive signal in pulmonary function improvement. Unlike existing therapies that merely slow FVC decline, the high dosing groups of the phase 2 study achieved an impressive absolute increase in FVC over 24 weeks. In addition, we observed other respiratory and lung function improvements, indicating the substantial symptom relief and benefits to quality-of-life in the IPF patients. Notably, AK3280's favorable tolerability across all dose levels is essential for the long-term management of IPF patients. This study provides novel scientific and medical perspectives, and we look forward to seeing this innovation to become a global standard treatment option in the near future."

About AK3280

AK3280 is a potential next-generation broad-spectrum anti-fibrotic molecule optimized from the marketed drug. It modulates multiple pathways and biomarkers closely associated with the fibrotic process, including the expression of fibrosis-related genes and proteins induced by transforming growth factor-beta (TGF-β) and lysophosphatidic acid (LPA). AK3280 works by reducing fibroblast cellular proliferation and inhibiting the synthesis and accumulation of extracellular matrix. Compared to current therapies, AK3280 offers advantages in safety and tolerability, with potentially much improved clinical efficacy. The Phase II randomized, double-blind, placebo-controlled confirmatory clinical study has been completed, and preparations are underway to initiate the pivotal Phase III clinical study.

About ArkBio

ArkBio is a global biotech company focused on developing innovative therapies for respiratory, infectious, and pediatric diseases. Founded in 2014, it has built core technology platforms and a differentiated R&D pipeline through in-house R&D efforts and external collaboration. Key drug assets include ziresovir, the first direct-acting RSV antiviral with positive pivotal phase III results, and AK0901, an FDA-approved pediatric ADHD therapeutic drug. ArkBio has established strategic partnerships with several multinational pharmaceutical companies and academic institutes, including Roche, Genentech, the Scripps Research Institute, the Institute of Microbiology of Chinese Academy of Sciences, domestic and international biotechnology companies, as well as venture capital institutions.

For more information about the company, please visit our website: www.arkbiosciences.com  

Investor Inquiries: IR@arkbiosciences.com  

SHANGHAI, April 16, 2025 /PRNewswire/ -- The Lancet Child & Adolescent Health, a leading international journal in pediatric and adolescent medicine, has published the Phase III clinical trial results of ziresovir, a novel RSV-targeted antiviral drug developed by Shanghai Ark Biopharmaceutical Ltd. The article, titled "Efficacy and safety of ziresovir in hospitalised infants aged 6 months or younger with respiratory syncytial virus infection in China: findings from a phase 3 randomised trial with 24-month follow-up", presents detailed subgroup analysis of infants under six months of age, following the primary results published in The New England Journal of Medicine in September 2024.

RSV: An Urgent Unmet Medical Need

Respiratory syncytial virus (RSV) is a leading cause of hospitalization and mortality in children under six years old. With millions of cases annually worldwide, especially among infants under six months, RSV imposes a significant burden on families and healthcare systems. Current treatments are limited to symptomatic and supportive care, with no approved direct-acting antiviral therapies available.

Ziresovir's Phase III results mark a significant milestone, representing the first clinical validation of an antiviral's efficacy in hospitalized infants ≤6 months, and demonstrating long-term benefits in reducing recurrent wheezing and asthma.

Study Design

This multicenter, randomized, double-blind, placebo-controlled Phase III trial was conducted at 30 sites in 28 hospitals across China. The trial included two parts:

• Part 1 assessed safety and pharmacokinetics
• Part 2 evaluated clinical efficacy and antiviral effects

Infants were randomized in a 2:1 ratio to receive ziresovir or placebo for five days, with a 24-month follow-up. The latest publication focuses on infants aged six months or younger.

Key Findings

1. Significant Efficacy

• Improved Bronchiolitis Symptoms: By Day 3, ziresovir-treated infants had a 3.5-point reduction in Wang Bronchiolitis Clinical Score (WBCS), compared to 2.2 points in the placebo group—a 54.5% difference (p<0.001). Improvements in respiratory rate, wheezing, and retractions were also statistically significant (p<0.05).
• Rapid Viral Load Reduction: By Day 5, viral load decreased by 2.51 log₁₀ copies/mL in the treatment group, versus 1.87 log₁₀ in the placebo group (p=0.024), confirming ziresovir's antiviral activity.
• Faster Symptom Relief: Time to wheezing resolution (HR=1.53, p=0.021) and retraction relief (HR=1.49, p=0.018) were shorter in the treatment group. Hospital stay and oxygen use showed a reduction trend, though not statistically significant.

2. Long-Term Safety and Respiratory Health Benefits

• Favorable Safety Profile: Treatment-emergent adverse events (TEAEs) occurred in 18% of the treatment group vs. 11% in placebo. No drug-related severe adverse events or deaths were reported.
• Reduced Risk of Recurrent Wheezing and Asthma:
• Wheezing incidence was 3.6 times lower in the treatment group (0.18% vs. 0.65%, p=0.0048).
• Wheezing episodes were reduced by 2.6 times (1.2 vs. 3.1 episodes).
• Asthma rates were also lower (3% vs. 5%).

These long-term benefits suggest early antiviral intervention may reduce the risk of chronic respiratory conditions.

A New Era in RSV Treatment

Unlike existing RSV treatments focused solely on symptom relief, ziresovir is the first RSV-targeted antiviral therapy with demonstrated clinical efficacy in infants under six months—those most at risk of severe complications. These findings introduce a new therapeutic paradigm for RSV treatment and prevention of long-term sequelae.

The results were presented by Dr. Jim Wu, CEO of ArkBio, at the 13th International RSV Symposium in Brazil, receiving recognition from the global RSV research community.

Prof. Xin Ni, principal investigator of the Airflo study and professor at Beijing Children's Hospital, Capital Medical University, commented:

"This pivotal Phase III trial provides the first solid evidence of an effective antiviral treatment for RSV in high-risk infants. Following our initial publication in The New England Journal of Medicine, this deeper analysis in infants under six months published in The Lancet Child & Adolescent Health further validates ziresovir's safety, efficacy, and long-term benefit. We are proud of our team's commitment and look forward to bringing this drug to patients in China and worldwide."

For public readers, the article can be accessed at the following link:
https://www.thelancet.com/journals/lanchi/article/PIIS2352-4642(25)00067-7/fulltext 

About Ziresovir

Ziresovir is a first-in-class, oral small-molecule inhibitor targeting the RSV fusion (F) protein. It prevents viral entry and cell-to-cell transmission by blocking syncytia formation, a hallmark of RSV infection. It is the first oral RSV antiviral to successfully complete a pivotal Phase III trial with positive results and has been granted Breakthrough Therapy Designation by the National Medical Products Administration (NMPA) of China.

About ArkBio

ArkBio is a global biotech company focused on developing innovative therapies for respiratory, infectious, and pediatric diseases. Founded in 2014, it has built core technology platforms and a differentiated R&D pipeline through in-house research and collaborations. Key assets include ziresovir, the first direct-acting RSV antiviral with positive pivotal phase III results globally, and AK0901, an FDA-approved pediatric ADHD therapy with commercialization rights in Greater China.

ArkBio has established strategic partnerships with several multinational pharmaceutical companies and academic institutes, including Roche, Genentech, the Scripps Research Institute, the Institute of Microbiology, Chinese Academy of Sciences, domestic and international biotechnology companies, as well as venture investment institutions.

For more information about the company, please visit our website: www.arkbiosciences.com 

Investor Inquiries: IR@arkbiosciences.com

SHANGHAI, March 24, 2025 /PRNewswire/ -- Shanghai Ark Biopharmaceutical Co., Ltd. ("ArkBio") today announced the successful completion of the Phase III clinical study of AK0901, a novel drug for the treatment of attention deficit hyperactivity disorder (ADHD). This milestone represents a substantial step forward for ArkBio in the research and development of drugs for pediatric psychiatric disorders, potentially offering new treatment options and improved therapeutic outcomes for children with ADHD in China.

ADHD is a chronic neurodevelopmental disorder characterized by age-inappropriate attention deficits and/or hyperactivity-impulsivity, often persisting into adulthood. Globally, approximately 7.2% of children are affected, with 60%–80% continuing to exhibit symptoms during adolescence and 50.9% into adulthood. About 65% of patients present with one or more comorbidities. ADHD impairs learning and causes multidimensional, lifelong functional challenges. In China, the prevalence of ADHD among children and adolescents is 6.4%, affecting over 23 million individuals. However, current treatment options remain limited, with existing drugs often falling short in terms of efficacy, safety, or convenience of drug administration, leading to poor patient compliance.

AK0901, a novel methylphenidate-based drug, is the first and only drug combining two active components of immediate-release dexmethylphenidate (d-MPH) and prodrug serdexmethylphenidate (SDX). It works by inhibiting the reuptake of dopamine and norepinephrine in the synaptic cleft, enhancing neurotransmitter efficiency to alleviate ADHD symptoms. AK0901 (Azstarys^®) received U.S. FDA approval in March 2021 as the new-generation methylphenidate drug in nearly two decades, demonstrating superior safety and clinical efficacy, and a potential as best-in-class therapeutic drug for the treatment of pediatric ADHD.

The Phase III clinical study of AK0901 in China was led jointly by Beijing Anding Hospital affiliated with Capital Medical University and Peking University Sixth Hospital, with the participation of eight clinical centers nationwide. The study aimed to evaluate the efficacy, safety, and tolerability of oral AK0901 in children with ADHD aged 6 to 12. The study results met both primary and key secondary endpoints and reached statistical significance in all the visiting time points compared to placebo, which provides a strong scientific basis for future treatment of Chinese ADHD patients.  

Professor Zheng Yi, the chief expert of the pediatric team at Beijing Anding Hospital affiliated with Capital Medical University, stated,"The Phase III study of AK0901 has systematically validated its therapeutic value in Chinese children with ADHD through a rigorous multi-center randomized controlled clinical study. As a new generation methylphenidate drug, the synergistic effect of its immediate-release and prodrug components not only enables rapid symptom control but also reduces the compliance issues associated with current methylphenidate drugs due to the fluctuation of blood drug concentration. This should serve better the stepwise needs of school-aged children for 'classroom concentration + after-school behavior management', making disease management more feasible and effective. The research data provide high-quality evidence to support the establishment of a comprehensive management system for ADHD that suits the characteristics of Chinese children. We look forward to its approval and launch in China to upgrade the treatment option that benefits patients and families."

Professor Liu Jing, Director of the Center for Child and Adolescent Mental Health of Peking University, stated, "This study marks a breakthrough in confirmation of the efficacy and safety of AK0901 in Chinese ADHD children. Its unique dual component formulation of immediate-release and prodrug methylphenidate significantly reduces the peak-trough fluctuations of traditional methylphenidate drugs, maintaining stable symptom control while reducing the risk of drug abuse. This is of significant clinical value for ADHD patients who require years of continuous intervention. This achievement not only addresses the core concern of clinical safety for long-term medication in children but also provides a new solution for balancing the efficacy needs with long-term safety of ADHD children. We look forward to the market approval and launch of AK0901 for clinical use in China, allowing more Chinese children with ADHD to benefit and grow up healthily."

Dr. Jim Wu, CEO of ArkBio, stated, "The completion of AK0901 Phase III study marks a critical step forward in addressing unmet needs in ADHD treatment in China. We extend our sincere gratitude to all participants, the physician team and partners involved in this study for their efforts and contributions. This milestone will drive our continued commitment to advancing scientific innovation and drug development of ADHD therapeutics. "

About ArkBio
ArkBio is a global biotech company focusing on discovery and development of innovative drugs for unmet medical needs, especially in the therapeutic areas of respiratory, infectious, and pediatric diseases. Since its inception in 2014, the company has established core technology platforms and built an innovative and highly differentiated R&D pipeline through in-house R&D efforts and external collaborations. Our strong R&D pipeline includes innovative clinical and pre-clinical stage drug candidates with first- or best-in-class potential for global clinical development. As one of the core assets, ziresovir is the first direct-acting RSV antiviral drug that has demonstrated positive results from a pivotal phase III study globally. Another near-commercialization product is AK0901, an FDA-approved novel therapy for pediatric ADHD, with ArkBio holding development and commercialization rights in Greater China.

ArkBio has established strategic partnerships with several multinational pharmaceutical companies and academic institutes, including Roche, Genentech, the Scripps Research Institute, the Institute of Microbiology, Chinese Academy of Sciences, domestic and international biotechnology companies, as well as venture investment institutions. 

For more information about the company, please visit our website: www.arkbiosciences.com
Investor Inquiries: IR@arkbiosciences.com

SHANGHAI, Nov. 25, 2024 /PRNewswire/ -- Shanghai Ark Biopharmaceutical Co., Ltd. ("ArkBio") announced today the successful enrollment and dosing of all participants in a Phase 1 clinical study of AK0610, a fully human monoclonal antibody designed to prevent respiratory syncytial virus (RSV) infection. This milestone marks a significant advancement in ArkBio' RSV portfolio, offering a new potential option for preventing RSV infection for vulnerable infants susceptible to RSV.

RSV is a globally prevalent and highly contagious RNA virus known for its seasonal epidemic patterns. It is one of the primary pathogens responsible for lower respiratory tract infections in children under five years worldwide and the leading cause of hospitalization among infants under one year of age. The virus spreads mainly through direct contact and airborne droplets, with a high rate of infection especially among children, leading to severe health consequences. High-risk individuals often experience more severe symptoms, frequently requiring hospitalization and, in some cases, facing fatal outcomes. RSV infection is also a significant risk factor for recurrent wheezing and asthma in children. The world faces a heavy healthcare burden and treatment challenges related to RSV infection.

AK0610 is a monoclonal antibody targeting the pre-fusion F protein of RSV that was isolated from a recovered infant and engineered. Compared to other RSV-neutralizing antibodies, AK0610 has a unique viral antigen binding epitope and novel mechanism of action. In preclinical studies, AK0610 demonstrated outstanding viral neutralization activity and an extended half-life that offers prolonged protection. It is positioned to become a new generation, long-acting preventive antibody against RSV-induced respiratory infections.

The Phase 1 study of AK0610 is a randomized, double-blind, placebo-controlled clinical trial designed to evaluate safety, tolerability, and pharmacokinetics of AK0610 in healthy adults. All 136 participants successfully completed dosing with the maximum administered dose reaching 3000 mg. Most of the study participants have been followed for up to six months with no observed serious adverse events related to the drug and drug exposure reaching the anticipated effective protective concentration, suggesting a desired profile of safety, tolerability, and human pharmacokinetics in healthy subjects.

Dr. Jim Wu, CEO of ArkBio, stated, "The successful enrollment and dosing completion of all participants in Phase 1 clinical trial of AK0610 represents a significant step forward for ArkBio in the area of RSV prevention. We extend our sincere gratitude to all participants, physician team, and partners involved in this study for their efforts and contributions. ArkBio is dedicated to drug development for RSV treatment that is represented by our leading therapeutic drug ziresovir as well as RSV prevention. The completion of AK0610's Phase 1 enrollment and dosing is another significant milestone. Based on the current Phase 1 clinical data, we are encouraged that AK0610 will become a new generation, long-acting monoclonal antibody for RSV prevention. Prevention and treatment of RSV infection complement each other, especially for high-risk populations like infants, the elderly, and immunocompromised patients. This achievement motivates us to continue developing comprehensive and effective prevention and treatment solutions for high-risk and infected patients worldwide, advancing our progress in RSV drug development."

About ArkBio
ArkBio is a global biotech company focusing on discovery and development of innovative drugs for unmet medical needs, especially in the therapeutic areas of respiratory, infectious, and pediatric diseases. Since its inception in 2014, the company has established core technology platforms and built an innovative and highly differentiated R&D pipeline through in-house R&D efforts and external collaborations. Our strong R&D pipeline includes innovative clinical and pre-clinical stage drug candidates with first- or best-in-class potential for global clinical development. As one of the core assets, ziresovir is the first direct-acting RSV antiviral drug that has been developed by ArkBio.

ArkBio has established strategic partnerships with several multinational pharmaceutical companies and academic institutes, including Roche, Genentech, the Scripps Research Institute, the Institute of Microbiology, Chinese Academy of Sciences, domestic and international biotechnology companies, as well as venture investment institutions. 

For more information about the company, please visit our website: www.arkbiosciences.com 
Investor Inquiries: IR@arkbiosciences.com

SHANGHAI, Sept. 26, 2024 /PRNewswire/ -- Shanghai Ark Biopharmaceutical Co., Ltd. ("ArkBio"), a clinical stage biopharmaceutical company focused on the development of therapeutics in respiratory, infectious and pediatric diseases, announced that the results of the phase 3 clinical trial of ziresovir, an oral drug for treatment of respiratory syncytial virus (RSV) infection in infants and young children, have been published as an original article entitled "Ziresovir in Hospitalized Infants with Respiratory Syncytial Virus"in The New England Journal of Medicine (NEJM). This marks the first time that NEJM has published phase 3 clinical trial results of a direct-acting antiviral drug treating RSV infection. This landmark study, led by Professors Xin Ni and Shunying Zhao from Beijing Childrens'Hospital, Capital Medical University, represents a significant milestone in the field of RSV antiviral development, where worldwide, there are no effective treatments for RSV infections available. In addition, the NEJM publication is accompanied by an editorial written by Professor Edward Walsh entitled "Creeping Towards Effective Antiviral Agents for RSV Infection", which regards the results of the phase 3 ziresovir clinical study to be encouraging.

RSV is the leading cause of hospitalized infants with respiratory infections and can cause serious respiratory complications such as bronchiolitis and pneumonia. For high-risk premature infants and those with congenital conditions, RSV infection can be serious and potentially life-threatening. Moreover, infants who suffer from an RSV infection are at a significantly higher risk of recurrent wheezing and asthma in later childhood. Among adults, severely immunocompromised patients and the elderly are at the highest risk for severe RSV infection, which can lead to hospitalization and death.

A total of 30 leading children's hospitals or pediatric departments of general hospitals across China participated in this randomized, double-blind, placebo-controlled phase 3 clinical trial, enrolling 311 infants or young children aged 1 to 24 months who were hospitalized due to RSV infection. These participants were randomly assigned in a 2:1 ratio to ziresovir or placebo twice a day for a 5-day treatment period. The primary outcome measure was the change in the Wang bronchiolitis clinical score from baseline after 48 hours of treatment. The key secondary endpoint was the change in RSV viral load from baseline after 96 hours of treatment.

Study Results:

• Significant Efficacy: In the ziresovir treatment group, the average reduction in bronchiolitis clinical score was 3.4 points after 48 hours of the first dose, compared to a 2.7-point reduction in the placebo group which reached statistical significance (P=0.002).
• Significant Reduction in Viral Load: The ziresovir treatment group showed an average decrease in RSV viral load of 2.5 log10 copies/mL 96 hours after the first dose, while the placebo group showed a decrease of 1.9 log10 copies/mL, representing a 77% greater reduction in the treatment group which was statistically significant (P=0.006).
• Excellent Safety Profile: The incidence of drug-related adverse events was 16% in the ziresovir treatment group and 13% in the placebo group. No serious drug-related adverse events were observed and there were no deaths.

These results indicate that as a novel direct-acting oral antiviral drug, ziresovir is an effective treatment for infants and young children hospitalized with RSV infection and has a favorable safety profile.

Professor Xin Ni, a leading principal investigator of Beijing Children's Hospital, Capital Medical University, stated: "RSV infection has long been a serious medical challenge in pediatric healthcare. This virus not only poses a threat to children's health, but also places a heavy burden on our healthcare system. For a long time, we have been searching for a safe and effective treatment. Today, our team has made significant progress in the management of this illness. We are grateful to The New England Journal of Medicine for recognition of our clinical study. This is the first successful phase 3 clinical trial of a direct-acting antiviral drug for the treatment of hospitalized infants with RSV infection. This research will bring hope to millions of families with their children being infected by RSV globally. We look forward to the approval of ziresovir for treatment of RSV infection in China and around the world for children to grow up free from the threat of RSV infection".

Dr. Jim Wu, CEO of ArkBio, added："The phase 3 clinical trial results of ziresovir in the treatment of hospitalized infants and young children are encouraging, as the study confirms that ziresovir is a well-tolerated and efficacious antiviral drug for the treatment of RSV infection. As the sponsor of the study, we are deeply grateful to all the patients and parents, the investigators and their teams, and all our partners who actively participated in the study. Under the guidance of the regulatory agencies, we will continue to develop the drug in collaboration with the medical community. We are committed to the research and development of innovative medicines to safeguard the wellbeing and healthy growth of children".

About ziresovir

Ziresovir is a novel small-molecule RSV fusion (F) protein inhibitor. It binds to the F protein of the virus and prevents viral entry into human cells. Ziresovir can also suppress RSV viral transmission by blocking cell-to-cell fusion through the formation of "syncytia", a characteristic event of RSV infection of host cells.
Ziresovir is the first oral anti-RSV drug that has completed a phase 3 pivotal clinical study with positive results. Ziresovir is the first non-oncology investigational product that received Breakthrough Therapy designation by the National Medical Products Administration (NMPA) of China.

About ArkBio

ArkBio is a global biotech company focusing on discovery and development of innovative drugs for unmet medical needs, especially in the therapeutic areas of respiratory, infectious and pediatric diseases. Since its inception in 2014, the company has established core technology platforms and built an innovative and highly differentiated R&D pipeline through in-house R&D efforts and external collaborations. This strong R&D pipeline program includes innovative clinical and pre-clinical stage drug candidates with first- or best-in-class potential for global clinical development. As one of the core assets, ziresovir is the first direct-acting RSV antiviral drug that has been developed by ArkBio.

ArkBio has established strategic partnerships with several global pharmaceutical companies and academic institutes, including Roche, Genentech, the Scripps Research Institute, the Institute of Microbiology of Chinese Academy of Sciences, domestic and international biotechnology companies, as well as venture investment institutions. 

For more information about the company, please visit our website: www.arkbiosciences.com

Investor Inquiries: IR@arkbiosciences.com

Dr. Wu stated："Although there are already two IPF drugs on the market, both suffer from poor tolerability and lower efficacy that do not improve patient's symptoms. Successful completion of patient enrollment for Phase 2 study is an important milestone for AK3280 as a next generation broadly active IPF drug. We extend our sincere gratitude to all the patients and investigators who participated in the trial for their efforts and contribution. The safety and efficacy results from this study will provide key scientific evidence for the continuous clinical development of the drug. We will work closely with investigators to advance clinical development of AK3280 and address the unmet medical need in treatment of IPF. ArkBio commits to the pursuit of innovative drugs for the treatment of respiratory and pulmonary diseases."

About the 8^th Annual IPF Summit

The IPF Summit is the largest IPF drug development forum for the pulmonary fibrosis research community. Its annual conference brings together pharmaceutical and biotech companies, clinicians, and academic researchers from around the globe with the goal to harness emerging biological understanding, apply novel and evolving models, and assess patient populations to propel innovative and efficacious pulmonary fibrosis therapeutics through phase 2 and beyond. For more information about the Annual IPF Summit conference, please visit ipf-summit.com. 

About Idiopathic Pulmonary Fibrosis

Idiopathic Pulmonary Fibrosis (IPF) is a chronic, progressive form of fibrosing interstitial pneumonia of unknown cause. IPF predominantly affects the elderly, with a median onset age of 65 years. The primary symptoms include progressively worsening shortness of breath, accompanied by restrictive ventilatory impairment and gas exchange abnormalities, leading to hypoxemia and even respiratory failure. The disease is confined to the lungs with pathological and/or radiological features consistent with usual interstitial pneumonia (UIP).

IPF has an insidious onset, followed by continuous, unpredictable, and irreversible progression. Its prognosis is worse than that of most lung cancers. The five-year survival rate is only 20% with a median survival time of just 2-3 years from diagnosis, which is why it is often considered a cancer-like disease. Smoking, respiratory viral infections, and environmental factors are potential risk factors for IPF.

About AK3280

AK3280 is a next-generation broad-spectrum anti-fibrotic molecule optimized from the marketed drug pirfenidone. It has the ability to modulate multiple pathways and biomarkers closely associated with the fibrotic process, including the expression of fibrosis-related genes and proteins induced by transforming growth factor-beta (TGF-β) and lysophosphatidic acid (LPA). AK3280 works by reducing cell proliferation and inhibiting the synthesis and accumulation of extracellular matrix. Compared to pirfenidone, AK3280 offers advantages in safety and tolerability, with potentially much better clinical efficacy. It is currently undergoing a phase 2 proof-of-concept clinical trial in patients with IPF in China.

About ArkBio

ArkBio is a global biotech company focusing on discovery and development of innovative drugs for unmet medical needs, especially in the areas of pediatric and respiratory/lung diseases. Since its inception in 2014, the company has established core technology platforms and built an innovative and highly differentiated R&D pipeline through in-house R&D efforts and external collaborations. One of the core drug products, ziresovir, is the first direct-acting RSV antiviral drug that successfully completed phase III clinical trial with positive results. Moreover, ziresovir is the first non-oncology drug that has received a breakthrough designation from NMPA. Another near-commercial drug asset is an attention deficit hyperactivity disorder (ADHD) drug AZSTARYS^® that has been approved by FDA. ArkBio owns the right for its development and commercialization in Greater China. The R&D pipeline program also includes a battery of clinical and pre-clinical stage drug candidates with first- or best-in-class potential for global clinical development. 

ArkBio has established strategic partnerships with several multinational pharmaceutical companies and academic institutes, including Roche, Genentech, the Scripps Research Institute, the Institute of Microbiology of Chinese Academy of Sciences, domestic and global biotechnology companies, as well as venture investment institutions. 

For more information about the company, please visit our website www.arkbiosciences.com 

Investor Inquiries IR@arkbiosciences.com 

RSV is a highly prevalent and infectious RNA virus. Populations at high risk of severe RSV infection include children, the elderly, and individuals with compromised immune systems. RSV is the primary viral pathogen responsible for acute lower respiratory tract infection (ALRTI) in infants worldwide, and a major cause of bronchiolitis and pneumonia. According to Lancet, there were 33 million cases of RSV-ALRTI in children under 5 years of age worldwide in 2019, resulting in approximately 3.6 million hospitalizations and 100,000 deaths. China has one of the highest rates of lower respiratory tract infection (LRTI) in children caused by RSV, accounting for 18-27% of all hospitalizations in children under 5 years old due to RSV-ALRTI. On average, 2,500 children in China are hospitalized daily due to RSV infection ^[2]. At present, there is no approved drug for the prevention of RSV infection in China. 

AK0610 specifically targets the RSV pre-F protein. It demonstrated potent neutralizing effects against RSV both in vitro and in vivo. With a prolonged half-life, it holds promise as a next-generation long-acting antibody drug for RSV prevention.  

Dr. Jim Wu, chairman and CEO of ArkBio, said, "We are excited with the IND approval of AK0610 and its great potential in the field of RSV prevention. We sincerely thank Professor George Fu Gao, a member (academician) of CAS and his team from the Institute of Microbiology, and Professor Zhengde Xie and his team from Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, China, for their professional collaboration and scientific spirits. ArkBio specializes in drug discovery and development in the RSV field with a pipeline in both RSV treatment and prevention. We will strive to provide very needed RSV high-risk population and patients with efficacious prevention and treatment solutions." 

Professor George Fu Gao, a member (academician) of CAS and director of the Institute of Microbiology said, "We are delighted with AK0610's IND approval and excellent scientific results, which are the fruits of our collaboration among the expert team of Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, China and outstanding scientists at ArkBio. We will expedite the clinical development of AK0610 and make this partnership an exemplary case among biotech industry, academia, and medical community. We will strive to lead scientific research towards improvement of public life and health, to direct scientific discovery to patient care at bedside, and to meet the unmet needs of our public." 

Professor Zhengde Xie of Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, China added, "RSV infection severely impacts children's health and lives. There is a huge unmet medical need for pediatric prophylaxis of RSV infection. We are delighted to see the progress of AK0610 towards clinical development and wish it a great success in upcoming clinical trials that will eventually benefit very needed pediatric population." 

About ArkBio 

ArkBio is a global biotech company focusing on discovery and development of innovative drugs for unmet medical needs, especially in the areas of pediatric and respiratory diseases. Since its inception in 2014, the company has established core technology platforms and built an innovative and highly differentiated R&D pipeline through in-house R&D efforts and external collaborations. One of the core drug products, ziresovir, is the first direct-acting RSV antiviral drug that successfully completed Phase III clinical trial with positive results. Its NDA application has been accepted and granted Priority Review by NMPA. Moreover, ziresovir is the first non-oncology drug that has received a breakthrough designation from NMPA. Another near-commercial drug asset is an attention deficit hyperactivity disorder (ADHD) drug AZSTARYS^® that has been approved by FDA. ArkBio owns the right for its development and commercialization in Greater China. The R&D pipeline program also includes a battery of clinical and pre-clinical stage drug candidates with first- or best-in-class potential for global clinical development. 

ArkBio has established strategic partnerships with several multinational pharmaceutical companies and acedmic institues, including Roche, Genentech, Scripps Research Institute, the Institute of Microbiology, Chinese Academy of Sciences, domestic and international biotechnology companies, CROs as well as venture investment institutions. 

For more information, please visit www.arkbiosciences.com 

SHANGHAI, March 16, 2023 /PRNewswire/ -- Shanghai Ark Biopharmaceutical Co., Ltd. (ArkBio), a global biopharmaceutical company focusing on discovery and development of innovative drugs for unmet medical needs in pediatric and respiratory disease areas, today announced that it has renewed its co-development collaboration with Calibr, a division of Scripps Research. The collaboration centers on AK0705, a potential first-in-class drug targeting an enzyme that plays an important role in respiratory inflammation. AK0705 is being developed to treat a broad spectrum of respiratory diseases, including chronic obstructive pulmonary disease (COPD).

Under terms of the agreement, ArkBio will lead the preclinical development activities, with Calibr contributing its expertise to jointly guide the program forward. This renewed collaboration extends an ongoing collaboration that identified AK0705 as the pre-clinical candidate (PCC). Upon completion of the next stage of development, AK0705 is expected to enter human clinical studies in 2024.

"We are delighted to continue our collaboration with Calibr and take AK0705 forward together," said Dr. Jim Wu, Chairman and CEO of ArkBio. "Our teams have made great progress so far, and I am confident that we will continue to unlock the value of AK0705 as a well-differentiated therapeutic option for treating respiratory diseases with high unmet needs."

"New therapies are desperately needed for respiratory diseases resulting from chronic inflammation—the hundreds of millions of people suffering from these disorders globally have few options," said Dr. Peter Schultz, CEO and President of Scripps Research and Calibr. "Through novel chemistry, AK0705 has potential to be a targeted therapy that corrects an enzyme imbalance that underlies multiple respiratory disorders. ArkBio has been an exemplary collaborator, and we look forward to progressing this therapy toward human clinical trials."

About AK0705

AK0705 is a potential first-in-class drug candidate targeting a key enzyme activated under inflammatory conditions. It demonstrated a picomolar potency via a novel reversible covalent binding mechanism.

About ArkBio

ArkBio is a global biopharmaceutical company focusing on discovery and development of innovative drugs for unmet medical needs, especially in pediatric and respiratory disease areas. Since its inception in 2014, the company has established core technology platforms and has created innovative drugs with global patent protection. Through in-house R&D efforts and external collaborations, the company has developed a promising and highly differentiated product pipeline. The core product, ziresovir, is the world's leading anti-RSV therapeutic drug that has received a breakthrough designation from China's regulatory authority NMPA, and currently under NDA review by the China CDE. Another near-commercial asset is an attention deficit hyperactivity disorder (ADHD) drug AZSTARYS^® recently approved by the FDA. ArkBio owns the right for its development and commercialization in Greater China. The pipeline programs also include a battery of clinical and pre-clinical stage drug candidates with first- or best-in-class potential for global development.

ArkBio strives to become a leading global biopharmaceutical company with integrated technology platforms for drug discovery and development, manufacturing, and commercialization in pediatric and respiratory therapeutic areas.

For more information, please visit www.arkbiosciences.com

For investor relation please contact: IR@arkbiosciences.com

About Calibr and Scripps Research

Calibr is a first-of-its kind non-profit translational research institute dedicated to creating the next generation of medicines. Part of Scripps Research situated in the heart of San Diego's Torrey Pines Mesa biomedical research hub, Calibr is uniquely positioned to create and proliferate innovative collaborations. Calibr has created a broad therapeutic pipeline extending from early-stage discovery through clinical-ready programs, including candidate medicines ranging from small molecules and peptides to biotherapeutics and cell-based therapies.

Scripps Research is ranked one of the most influential scientific institutions in the world for its impact on innovation. A non-profit research organization, Scripps Research expands basic knowledge in the biosciences and uses these fundamental advancements to develop profound innovations that improve well-being. Scripps Research scientists lead breakthrough studies that address the world's most pressing health concerns, accelerating the creation and delivery of medical breakthroughs to better human health across the globe. Its educational and training programs mold talented and committed students and postdocs into the next generation of leading scientists.

For more information about Calibr and Scripps Research visit www.calibr.org and www.scripps.edu. Follow @ScrippsResearch on Twitter, Facebook or LinkedIn.