On 29 September 2025, at the European Respiratory Society (ERS) Conference held in Amsterdam, the Netherlands, Vicore Pharma presented results from the Phase IIa AIR trial using synthetic control arms (SCA) to demonstrate efficacy of its pipeline asset buloxibutid in idiopathic pulmonary fibrosis (IPF). Findings from the study revealed a statistical difference between the mean change in forced vital capacity (FVC) at 36 weeks of treatment between the AIR cohort and the SCAs, demonstrating successful use of SCAs for efficacy testing in IPF and reiterating the asset’s potential as a promising candidate that aims to stimulate lung repair and improve vascular function in the lungs.
The Phase IIa AIR trial (NCT04533022) was an open-label trial evaluating 100mg buloxibutid, twice daily, for up to 36 weeks in IPF patients who were treatment-naïve. Using real-world data via the Qureight platform, 20,000 control arms were generated by Monte Carlo Cross Validation (MCCV), resulting in the creation of 408 matched controls against 48 buloxibutid-treated patients. The results showed a mean change in FVC for the treatment group (N=48) after 36 weeks of 23.2ml compared to a value of negative 114.8ml across the 408 SCAs, with this trend in the active treatment group similar to the final Phase IIa AIR data readout presented in May 2024 that demonstrated a mean FVC change from baseline of 216ml at 36 weeks.
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In addition to these results highlighting the potential of buloxibutid in IPF, the innovative clinical design that was utilised further supports the use of external control arms such as SCAs with broad implications for rare disease drug development. For drug developers within the rare disease space, use of SCAs can potentially facilitate the drug development process. For a rare disease where trial enrolment challenges are prevalent, SCAs can minimise the need for placebo controls and increase the efficiency of trial recruitment by potentially reducing trial size, duration and costs. It is worth noting that buloxibutid’s ongoing Phase IIb ASPIRE trial (NCT06588686) is also implementing Qureight’s imaging platform to investigate the asset in patients with IPF.
The results presented at ERS 2025 focus on a similar theme highlighted at the American Thoracic Society 2025 conference, as exemplified by Avalyn’s use of Qureight’s SCAs to validate treatment efficacy of inhaled pirfenidone (AP01) in IPF. Looking ahead, it is becoming evident that use of synthetic control arms offers competitive advantage, particularly in rare indications. Companies demonstrating successful synthetic control implementation may gain preferred status with regulatory agencies, facilitating faster approvals and expanded indications. Industry-wide adoption of synthetic controls could fundamentally reshape drug development through multiple means, including enabling smaller biotech companies to gain access to cost-effective trial methodologies, accelerating drug development and improving patient access by reduced development costs and timelines. Companies that act decisively to incorporate synthetic control capabilities into their development strategies will be best positioned to capitalise on this transformative approach to clinical trials.
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