BioAegis Therapeutics has commenced patient recruitment across 13 countries for its landmark Phase 2b clinical trial evaluating recombinant human plasma gelsolin (rhu-pGSN) as a treatment for acute respiratory distress syndrome (ARDS). The 600-patient study represents a significant milestone in addressing one of critical care medicine's most challenging conditions, where no effective therapies currently exist and mortality rates remain devastatingly high.
Global Trial Design and Scope
The randomized, double-blind, placebo-controlled trial (NCT05947955) will evaluate the efficacy of six doses of rhu-pGSN administered intravenously to hospitalized patients with moderate-to-severe ARDS caused by infection. The primary endpoint focuses on survival without organ failure on Day 28, with the study also measuring safety and tolerability alongside secondary outcomes.
Patient recruitment is underway across 13 countries, including the United States, Canada, United Kingdom, and multiple European Union nations such as Belgium, France, Italy, Germany, Netherlands, and Spain. The study specifically targets patients with P/F ratios ≤150, representing the moderate-to-severe ARDS population most likely to benefit from intervention.
"We are excited with our significant progress in this important study. Focusing this study on the moderate to severe patient who will have severely depleted plasma gelsolin levels promises to address ARDS with a novel approach employing the protein whose role in the body is to balance the inflammation-healing axis," stated Susan Levinson, Ph.D., Chief Executive Officer of BioAegis.
Addressing Critical Medical Need
ARDS represents a severe complication that can develop from sepsis, trauma, pneumonia, or other infectious diseases, resulting in life-threatening lung injury with fluid leakage into the lungs. The condition creates significant healthcare burden, with patients requiring oxygen, mechanical ventilation, and extensive critical care resources.
Current treatment outcomes remain poor, with 40% of ARDS patients failing to survive despite aggressive medical management. Survivors often face long-term complications, including impaired lung function and reduced quality of life. In the United States alone, ARDS affects over 500,000 patients per year, representing roughly 10% of all ICU admissions.
Gelsolin's Multifaceted Mechanism
Gelsolin represents a naturally occurring human protein found in the bloodstream that becomes depleted during inflammatory processes. The therapeutic approach centers on supplementing this critical immune regulatory protein to address the excessive inflammatory response characteristic of ARDS.
The protein demonstrates multiple therapeutic mechanisms, including:
- Modulating activation of the NLRP3 inflammasome
- Enhancing uptake and killing of microbial pathogens by innate immune cells
- Binding to and removing harmful inflammatory mediators and toxic actin released from damaged cells
- Regulating macrophage phenotype to modulate inflammation
Research has demonstrated that depleted gelsolin can be effectively restored in both animals and humans, positioning rhu-pGSN as an immune system regulator that interrupts the NLRP3 inflammasome and boosts macrophage clearance of pathogens.
Federal Support and Development Timeline
The Phase 2b trial has received substantial federal funding support from the U.S. Department of Health and Human Services, Administration for Strategic Preparedness and Response, and the Biomedical Advanced Research and Development Authority (BARDA) under contract number 75A50123C00067.
Results from this landmark study are expected to inform therapeutic strategies beyond ARDS, potentially positioning rhu-pGSN as a promising intervention across a spectrum of acute and chronic inflammatory diseases. The company's portfolio is built around gelsolin as a highly conserved and critical immune regulatory protein that controls dysfunctional inflammation without suppressing immune function.
BioAegis Therapeutics holds exclusive licensing rights to broad, worldwide intellectual property through Harvard-Brigham and Women's Hospital, with over 40 patents issued covering inflammatory disease, infection, renal failure, neurologic disease, and frailty applications.
