Mycovia Pharmaceuticals has announced enrollment of the first participant in an investigator-initiated Phase 2 clinical trial evaluating oteseconazole for cryptococcal meningitis, marking a significant step toward addressing a critical global health challenge that causes an estimated 112,000 deaths annually.
Trial Design and Leadership
The PLATFORM-CM study is an open-label randomized trial designed by Dr. David Boulware, Professor of Medicine, Infectious Disease and International Medicine at the University of Minnesota School of Public Health, and Dr. David Meya, Associate Professor at the School of Medicine at the College of Health Sciences, Makerere University in Uganda. The trial will compare the efficacy and safety of antifungal investigational therapies, including oteseconazole, to the standard of care WHO first-line therapy.
The study will be conducted at three sites in Uganda and will involve up to 200 participants who will be treated for 18 weeks with oteseconazole. The trial features single or potentially multiple interventional arms to evaluate various treatment approaches.
"We are excited to initiate this important study and enroll our first research participant," said Dr. Boulware. "Our goal is to generate meaningful data that will show a reduction in death for patients that cannot use or who have developed resistance to current standard of care treatments."
Addressing Critical Medical Need
Cryptococcal meningitis represents a severe global health burden, causing an estimated 152,000 cases and 112,000 deaths annually. The infection is one of the leading causes of death among people living with HIV in Africa, highlighting the urgent need for improved treatment options.
Current standard of care therapy faces several significant challenges, including medication access and stability issues, supplier limitations, and substantial toxicity. The World Health Organization has recognized Cryptococcus as a critical Fungal Priority Pathogen, underscoring the importance of developing new therapeutic approaches.
Mechanism and Preclinical Evidence
Oteseconazole is designed to selectively inhibit fungal CYP51, an enzyme required for fungal cell wall integrity. This interaction proves toxic to fungi, resulting in the inhibition of fungal growth. Preclinical studies have demonstrated that oteseconazole shows potency against both fluconazole-sensitive and fluconazole-resistant strains of Cryptococcus, suggesting potential advantages over existing treatments.
"This study will serve to further evaluate oteseconazole beyond its currently approved indication and expand upon the encouraging preclinical data that suggests oteseconazole may have clinical utility in treating this serious fungal infection of the brain and central nervous system," commented Dr. Stephen Brand, Chief Development Officer of Mycovia.
Current Approved Use
Oteseconazole is currently marketed as VIVJOA® capsules, the first FDA-approved product for Mycovia. The drug is indicated to reduce the incidence of recurrent vulvovaginal candidiasis (RVVC) in females with a history of RVVC who are not of reproductive potential. The compound received FDA Qualified Infectious Disease Product and Fast-Track designations to become the first FDA-approved therapy for RVVC.
Global Development Strategy
Mycovia has established international partnerships to expand oteseconazole's reach. In 2019, the company licensed oteseconazole to Jiangsu Hengrui Pharmaceuticals Co., Ltd., to develop and commercialize the drug in China, including mainland China, Hong Kong, Macau and Taiwan.
The company recognizes tremendous potential for its oral fungal inhibitors and acknowledges the growing need to treat a range of multi-drug-resistant fungal pathogens. This cryptococcal meningitis trial represents part of Mycovia's broader commitment to addressing unmet medical needs through clinical research and collaboration.
