SHANGHAI, May 15, 2025 — Shanghai Ark Biopharmaceutical Co., Ltd. (“ArkBio”) announced today encouraging top-line results from its Phase II trial of AK3280, a novel anti-fibrotic drug, for the treatment of idiopathic pulmonary fibrosis (IPF). The study, spearheaded by Professor Huaping Dai from the Department of Pulmonary and Critical Care Medicine at China-Japan Friendship Hospital in Beijing, took place across 31 clinical locations in China.
Global Clinical Challenge: Unresolved Treatment Needs in IPF
Idiopathic Pulmonary Fibrosis (IPF) is a progressive, irreversible, and ultimately fatal lung disease where lung tissue becomes scarred. This leads to respiratory failure, and patients typically survive only 2–5 years after being diagnosed. Existing treatments like pirfenidone and nintedanib have been available for some time, but their effectiveness is limited, they cause significant side effects, and patients often can’t tolerate them long-term. This underscores the pressing need for new and more effective treatments.
Phase 2 Proof-of-Concept Study of AK3280, a New Generation Broadly Active Antifibrotic Drug
AK3280 represents a new generation of anti-fibrotic drugs with broad activity, designed with improved pharmacological and pharmacokinetic properties compared to current IPF medications. Preclinical data suggest it has greater anti-fibrotic activity and better pharmacokinetic properties, without the gastrointestinal issues and other toxicities seen with current treatments. Prior Phase I studies have shown it to be safe, well-tolerated, and to have a favorable pharmacokinetic profile in humans.
The completed Phase II proof-of-concept study was a randomized, double-blind, placebo-controlled trial that assessed the safety, tolerability, and clinical efficacy of AK3280 in Chinese patients with IPF. Participants were randomly assigned to receive either AK3280 (100/200/300/400 mg twice daily) or a placebo for 24 weeks. The study measured clinical efficacy using endpoints such as forced vital capacity of the lung (FVC and %pFVC), diffusing capacity of the lung for carbon monoxide (DLco), the 6-minute walk test (6MWT), and patient-reported St. George’s Respiratory Questionnaire (SGRQ) scores. The higher dose groups showed improvement in FVC from the beginning of the study, particularly the 400 mg group, which saw an absolute FVC increase of 209.4 mL and a 6.4% adjusted %pFVC improvement from baseline. These results were statistically significant compared to the placebo (p=0.002 and 0.004, respectively). Improvements were also observed in other measures of lung and respiratory function. The drug demonstrated a good safety and tolerability profile, without the gastrointestinal issues associated with current IPF therapies.
Implications for Future IPF Therapeutics
This study, conducted over 24 weeks with a rigorous multicenter, randomized, double-blind design, followed by another 24-week open-label study, is the first phase 2 proof-of-concept study of AK3280 in patients with fibrotic conditions. The findings not only indicate AK3280’s potential to improve lung function and respiratory outcomes but also highlight its unique safety profile, which could support long-term use. This positions it as a possible future standard treatment for IPF.
Dr. Huaping Dai, the principal investigator of the phase 2 study and a professor at China-Japan Friendship Hospital, commented, “In the IPF treatment field, there’s an ongoing need for safer and more effective anti-fibrotic drugs. The most promising aspect of this Phase II study is the positive effect AK3280 has on improving pulmonary function. Unlike current treatments that only slow the decline of FVC, the high-dose groups in the phase 2 study achieved a significant absolute increase in FVC over 24 weeks. Furthermore, we saw improvements in other respiratory and lung functions, suggesting substantial symptom relief and quality-of-life benefits for IPF patients. Importantly, AK3280’s good tolerability across all dose levels is crucial for the long-term management of IPF patients. This study offers new scientific and medical insights, and we hope to see this innovation become a standard global treatment option soon.”
About AK3280
AK3280 is a potential next-generation, broad-spectrum anti-fibrotic molecule derived from a marketed drug. It affects several pathways and biomarkers closely related to the fibrotic process, including the expression of genes and proteins associated with fibrosis that are induced by transforming growth factor-beta (TGF-β) and lysophosphatidic acid (LPA). AK3280 works by reducing the proliferation of fibroblast cells and preventing the synthesis and accumulation of the extracellular matrix. Compared to existing treatments, AK3280 offers benefits in terms of safety and tolerability, with the potential for significantly improved clinical efficacy. The Phase II randomized, double-blind, placebo-controlled confirmatory clinical study has been completed, and preparations are underway to begin the pivotal Phase III clinical study.
About ArkBio
ArkBio is a global biotechnology company focused on creating innovative treatments for respiratory, infectious, and pediatric diseases. Founded in 2014, it has developed core technology platforms and a unique R&D pipeline through its own research and development efforts and through external collaborations. Its key drug assets include ziresovir, the first direct-acting RSV antiviral with positive pivotal phase III results, and AK0901, a pediatric ADHD therapeutic drug approved by the FDA. ArkBio has formed strategic partnerships with several multinational pharmaceutical companies and academic institutions, including Roche, Genentech, the Scripps Research Institute, the Institute of Microbiology of the Chinese Academy of Sciences, both domestic and international biotechnology companies, and venture capital firms.
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