Investigational antifungal agent shows promise for blood cancer patients undergoing stem cell transplants

Patients undergoing stem cell transplantation for blood cancers face prolonged immunosuppression that leaves them at high risk for life-threatening fungal infections. New Phase III trial results suggest a potential advancement in easier medication and dosing management for these patients.

The ReSPECT trial evaluated rezafungin, a once-weekly antifungal agent, compared to standard antimicrobial regimens, for preventing invasive fungal diseases in adults undergoing allogeneic hematopoietic stem cell transplantation (HSCT). The study enrolled patients across more than 50 centers in seven countries.

Rezafungin met its primary endpoint for US FDA and EMA standards for fungal-free survival at 90 days, demonstrating comparable efficacy to standard antifungal regimens (60.7% vs. 59.0%, respectively). The treatment showed similar effectiveness against infections from Candida, Aspergillus and Pneumocystis, which are the most common invasive fungal pathogens in this population, with comparable mortality rates between treatment arms.

The once-weekly dosing regimen was well tolerated, with a safety profile comparable to standard antimicrobial regimens.

Approximately 1.3 million people worldwide were diagnosed with hematological malignancies in 2019, the majority of whom face treatment-related neutropenia that significantly increases their risk of invasive fungal infections. Despite available treatments, mortality rates for invasive candidiasis remain as high as 40% in some patient populations.

Patients undergoing allogeneic HSCT, a potentially curative treatment for blood cancers, routinely require extended antifungal prophylaxis during their recovery period, making tolerability and adherence key factors in treatment success.

Regulatory submissions are planned for the second half of 2026 in the United States and the third quarter of 2026 in Europe.