Qureight, a deep-learning analytics company, has achieved a world-first breakthrough in clinical trial design by successfully using AI-powered synthetic control arms to replace human placebo groups in lung disease research. The groundbreaking study, presented at the 2025 American Thoracic Society conference in San Francisco, demonstrates significant clinical efficacy for Avalyn's inhaled pirfenidone (AP01) in treating idiopathic pulmonary fibrosis (IPF), a rare and high-mortality lung disease.
Revolutionary Approach to Clinical Trial Design
Traditional clinical study designs for registrational endpoints require large patient cohorts, often taking multiple years to complete even in early-stage efficacy trials. This creates significant burdens for drug developers in fields where new therapies are urgently needed. Qureight's synthetic study arms represent a novel solution to reduce trial size, duration, and cost while accelerating potential new therapies to patients.
The company has developed the ability to identify placebo or control arm patients who are digitally-twinned with treated subjects using processes that incorporate both clinical data and quantitative image analytics. This approach creates randomized placebo and control arms containing patients with more closely matched disease states than previously possible.
Comprehensive Data Platform and Methodology
Qureight has assembled an extensive biorepository of historic IPF patient clinical and lung image data, stored within its proprietary deep-learning platform. The system enables treated patient groups from clinical trials to be digitally paired with synthetic control groups generated using measures of clinical significance and baseline disease extent.
By simulating disease progression differences between closely matched patient groups, the platform may increase accuracy in endpoint assessment. Patient groups with highly comparable baseline disease states are expected to progress similarly, allowing more precise assessments of treatment effects than relying on randomized placebo comparisons while reducing the need for human placebo recruitment.
Clinical Validation Study Results
The recently published study assessed data from Avalyn's investigational therapy, an optimized inhaled formulation of pirfenidone for IPF treatment. The methodology involved matching AP01-treated patients to real-world, treatment-naïve IPF patients within the Qureight platform, first filtering by clinical trial inclusion criteria, then applying proprietary AI-based quantitative image analytics to generate a tightly matched synthetic control pool.
From this pool, over 10,000 randomly sampled control groups were created, with the top 1,000 that best-matched the treatment group evaluated for comparative efficacy testing. Results showed a statistically significant reduction in lung capacity (FVC) over the 48-week treatment period compared to the 100mg dose of AP01, suggesting significant treatment effect.
Addressing Ethical Challenges in Rare Disease Research
This innovative approach proves particularly impactful for trials in orphan diseases such as IPF, where patient populations are smaller and often unresponsive to standard of care. Access to experimental therapies can extend life in these conditions, making placebo arms less ethical from a patient care perspective.
Dr. Muhunthan Thillai, CEO of Qureight, emphasized the significance of this development: "The launch of Qureight's Synthetic Study Arms is a watershed moment for clinical research in lung diseases. By creating an ethical, image-driven synthetic control arm, we can accelerate trial timelines, reduce costs and, most importantly, spare patients from unnecessary placebo assignments."
Industry Impact and Future Applications
Howard M. Lazarus, MD, FCCP, and CMO of Avalyn, highlighted the broader implications: "Qureight's work helps to validate AP01's treatment effects in IPF patients and has the potential to reshape how we think about and design future studies to help us develop improved treatments and standard of care that patients so urgently need."
The company is currently evaluating wider applications of the platform across broader standards of care with partners, positioning this innovation to transform trial design and usher in a new era of data-driven therapeutics. This breakthrough not only demonstrates the potential for AI-powered synthetic controls in rare disease research but also establishes a new paradigm for ethical and efficient clinical trial conduct in high-mortality conditions.
