Mundipharma Reports Positive Phase III ReSPECT Topline Results for ▼REZZAYO (rezafungin) in Preventing Invasive Fungal Diseases in Allogeneic Stem Cell Transplant Patients
Mundipharma has announced encouraging topline results from its pivotal Phase III ReSPECT clinical trial, highlighting the potential of REZZAYO® (rezafungin acetate) as a prophylactic treatment for invasive fungal diseases (IFDs) in adult patients undergoing allogeneic haematopoietic stem cell transplantation (HSCT). The findings mark an important milestone in the development of rezafungin and reinforce its promise as a next-generation antifungal therapy for highly vulnerable, immunocompromised populations.
The global ReSPECT study is among the largest and most comprehensive antifungal trials conducted to date. Designed as a multicentre, randomized, double-blind study, it evaluated both the efficacy and safety of once-weekly rezafungin compared to a standard-of-care regimen (SAR). The trial focused on preventing invasive fungal infections caused by pathogens such as Candida, Aspergillus, and Pneumocystis—organisms that pose a significant threat to patients undergoing allogeneic HSCT.
Patients receiving allogeneic HSCT typically experience prolonged and profound immunosuppression as part of their treatment, leaving them highly susceptible to opportunistic infections. Invasive fungal diseases are a major cause of morbidity and mortality in this population, making effective prophylaxis a critical component of post-transplant care. Current antifungal regimens, while effective, often require frequent dosing and may be associated with drug-drug interactions (DDIs) and tolerability challenges. In this context, therapies offering simplified dosing schedules and improved safety profiles are particularly valuable.
The ReSPECT trial successfully met its primary endpoint, demonstrating non-inferiority of rezafungin compared with the standard antifungal regimen in achieving fungal-free survival at Day 90. Specifically, fungal-free survival rates were 60.7% for patients treated with rezafungin and 59.0% for those receiving the comparator therapy. These results meet the regulatory criteria set by both the U.S. Food and Drug Administration and the European Medicines Agency, providing a strong foundation for future regulatory submissions.
The comparable efficacy observed in the trial underscores the strength of rezafungin’s unique pharmacokinetic and pharmacodynamic (PK/PD) profile. Unlike traditional antifungal agents that often require daily dosing, rezafungin is designed for once-weekly administration. This extended dosing interval is made possible by its long half-life and sustained therapeutic activity, which may improve patient adherence and reduce the burden on healthcare systems. Additionally, the drug’s profile may help minimize drug-drug interactions, a critical consideration for HSCT patients who are often receiving multiple concomitant medications.
Beyond its primary endpoint, the trial also demonstrated comparable outcomes between the two treatment arms in terms of protection against infections caused by Candida, Aspergillus, and Pneumocystis species. Mortality rates were similarly aligned across both groups, further supporting the overall effectiveness of rezafungin in this high-risk setting. Importantly, the study also reported favorable results across several secondary endpoints, particularly those related to safety and tolerability.
Rezafungin was generally well tolerated, with a safety profile that appears well-suited for immunocompromised patients. Notably, the incidence of treatment-emergent adverse events leading to dose reduction, interruption, or discontinuation was lower or more favorable compared to the standard regimen. This suggests that rezafungin may offer a more manageable treatment experience for patients, potentially reducing complications and improving overall quality of care during the post-transplant period.
Dr. Yuri Martina, Chief Development and Medical Officer at Mundipharma, emphasized the significance of these findings, noting that the study achieved all its key endpoints and reflects the collaborative efforts of patients and investigators across more than 50 centers in seven countries. He highlighted that the results represent a meaningful advancement in addressing the unmet needs of patients undergoing HSCT, a group that remains particularly vulnerable to life-threatening infections.
The implications of the ReSPECT trial extend beyond clinical outcomes. If approved, rezafungin could help redefine the standard of care for antifungal prophylaxis in transplant patients by offering a combination of efficacy, convenience, and improved tolerability. Its once-weekly dosing regimen, in particular, could simplify treatment protocols and reduce the logistical challenges associated with frequent drug administration in both inpatient and outpatient settings.
Looking ahead, Mundipharma, in collaboration with its U.S. licensee CorMedix Inc., plans to leverage the ReSPECT data to support regulatory filings. The companies intend to submit a supplemental New Drug Application (sNDA) to the FDA in the second half of 2026. In parallel, Mundipharma is preparing for a submission to the European Medicines Agency, which is expected in the third quarter of 2026. These filings represent the next critical steps toward making rezafungin available to patients in need.
In summary, the positive Phase III results from the ReSPECT trial highlight the potential of REZZAYO® (rezafungin acetate) to become a valuable addition to the antifungal treatment landscape. By demonstrating non-inferior efficacy, a favorable safety profile, and the convenience of once-weekly dosing, rezafungin addresses several key challenges associated with current prophylactic therapies. As regulatory submissions move forward, the therapy holds promise for improving outcomes and quality of life for patients undergoing allogeneic HSCT, ultimately contributing to better standards of care in this high-risk population.
About rezafungin
Rezafungin is a next-generation echinocandin approved for the treatment of candidaemia and invasive candidiasis in adults.2 Invasive candidiasis continues to be an area of significant unmet need, especially for critically ill patients in hospitals and patients with compromised immune systems. Despite a number of available treatments, the mortality rate for patients with invasive candidiasis is as high as 40%.3,4
Rezafunginhas been studied for the prophylaxis of invasive fungal diseases in adults undergoing allogeneic HSCT.
Approximately 1.3 million people worldwide were diagnosed with haematological malignancies in 2019,5 the majority of whom are at risk of treatment‑related neutropenia, leaving them highly vulnerable to invasive fungal infections and underscoring the ongoing unmet need for effective antifungal prophylaxis.
® REZZAYO is a Registered Trademark of Mundipharma, used under licence by Melinta Therapeutics LLC, a wholly owned subsidiary of CorMedix Inc. in the USA.
About Mundipharma
Mundipharma is a global healthcare company with a presence in Africa, Asia Pacific, Canada, Europe, Latin America and the Middle East. In line with its mission, United for Patients, Mundipharma is dedicated to bringing innovative treatments to patients in the areas of pain management, infectious disease, ophthalmology, oncology, respiratory and central nervous system.
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