Cidara Therapeutics and Mundipharma have completed recruitment of the pivotal Phase 3 ReSTORE trial evaluating the efficacy and safety of rezafungin as a potential first-line treatment for candidemia and invasive candidiasis.
The trial includes 184 patients diagnosed with candidemia and/or invasive candidiasis.
Candidemia and invasive candidiasis continue 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, mortality rates are as high as 40%.
“Rezafungin is a novel once-weekly antifungal that has the potential to help critically ill, vulnerable patients battling these invasive Candida infections,” said George Thompson, M.D., principal investigator in the ReSTORE trial and associate professor of clinical medicine at the University of California, Davis, School of Medicine.
“Completion of enrollment of the ReSTORE trial is a tremendous milestone for all of us, most importantly our patients.”
Jeffrey Stein, Ph.D., President and CEO of Cidara, added: “We would like to thank our investigators and especially our patients who enabled us to achieve this important milestone.
“If approved, rezafungin would be the first new therapy for the treatment of this deadly disease in over a decade. With the completion of enrollment of ReSTORE, we remain on track to announce top-line data by the end of this year and anticipate filing our New Drug Application (NDA) in the US and similar regulatory filings outside the U.S. in mid-2022.”
Cidara has partnered with Mundipharma who will be responsible for bringing rezafungin to patients outside the U.S. and Japan.
Brian Sheehan, Ph.D., chief scientific officer at Mundipharma, commented: “Despite current advances in antifungal therapy, invasive fungal infections remain an area of significant unmet patient need associated with high mortality, particularly in immunocompromised and critically ill patients.
“We believe rezafungin has the potential to be the first effective and well tolerated once weekly treatment option for patients with candidemia and/or invasive candidiasis and would like to thank the patients and the clinicians who participated in this important trial.”
ReSTORE (NCT03667690) is a global, randomized, double-blind, controlled Phase 3 pivotal clinical trial evaluating the efficacy and safety of once-weekly intravenous dosing of rezafungin compared to once-daily dosing of caspofungin, the current standard of care, to treat patients with candidemia and/or invasive candidiasis.
The trial design is similar to Cidara’s Phase 2 STRIVE trial (NCT02734862), which met its primary safety and efficacy objectives. The ReSTORE trial is designed as a non-inferiority trial to evaluate one rezafungin dosing regimen of 400 milligrams (mg) for the first week followed by 200 mg of rezafungin once weekly for up to four weeks in total. This treatment arm is compared to caspofungin dosed once daily in a 1:1 randomization.
This global trial has been conducted at over 100 clinical trial centres across 18 countries. Study sites in China will continue recruiting patients for submission of rezafungin to the Centre of Drug Evaluation.
The primary efficacy endpoint of ReSTORE, which will be used to support a US Food and Drug Administration NDA submission, is all-cause mortality at Day 30. The primary efficacy endpoint for the European Medicines Agency is global response at Day 14.
The ongoing global Phase 3 ReSPECT trial (NCT04368559) evaluating rezafungin versus the standard antimicrobial regimen to prevent invasive fungal disease due to Candida, Aspergillusand Pneumocystis in subjects undergoing allogeneic bone marrow transplants continues to enroll patients as planned.