An Inhaled Galectin-3 Inhibitor in COVID-19 Pneumonitis: A Phase Ib/IIa Randomized Controlled Clinical Trial (DEFINE)

Area of Science:

• Pulmonology
• Infectious Diseases
• Pharmacology

Background:

• Elevated galectin-3 levels correlate with severe outcomes in COVID-19 patients.
• GB0139, an inhaled galectin-3 inhibitor, possesses anti-inflammatory and anti-fibrotic properties.
• Investigating GB0139's safety and efficacy in COVID-19 pneumonitis is crucial.

Purpose of the Study:

• To evaluate the safety and tolerability of inhaled GB0139 as an adjunct therapy for hospitalized COVID-19 pneumonitis patients.
• To assess GB0139's pharmacokinetic profile and target engagement in this patient population.

Main Methods:

• A Phase Ib/IIa randomized controlled platform trial was conducted.
• Hospitalized COVID-19 pneumonitis patients received standard of care (SoC) or SoC plus inhaled GB0139.
• GB0139 was administered at 10 mg twice daily for 48 hours, then once daily for up to 14 days or until discharge.

Main Results:

• No treatment-related serious adverse events were reported in the GB0139 group.
• Adverse event incidence was similar between the GB0139 + SoC group (40 patients) and the SoC group (35 patients).
• Plasma GB0139 was measurable, demonstrating target engagement through reduced circulating galectin-3 levels (P=0.0099 vs. SoC).

Conclusions:

• Inhaled GB0139 is well-tolerated in COVID-19 pneumonitis patients.
• The drug achieved therapeutic plasma concentrations and engaged its target, reducing galectin-3.
• These findings warrant larger clinical trials to confirm GB0139's efficacy in treating COVID-19 pneumonitis.