Shanghai Ark Biopharmaceutical Co., Ltd. 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.