SAN DIEGO – November 13, 2018 – Amplyx Pharmaceuticals, a company developing first-in-class products for life-threatening infections, today announced that the first patient has been dosed in the Company’s Phase 2 clinical program to evaluate the efficacy and safety of APX001 in the treatment of infections caused by Candida.
“Initiating our Phase 2 program for APX001 represents a significant milestone for Amplyx and for patients,” said Ciara Kennedy, Ph.D., President and Chief Executive Officer of Amplyx. “We anticipate this trial will validate our robust preclinical and Phase 1 data and demonstrate clinical proof-of-concept for APX001 as a novel treatment for patients with difficult-to-treat and often deadly Candida infections.”
This Phase 2 clinical trial is a multi-center, open-label, single-arm study to evaluate the efficacy and safety of both IV and oral APX001 for the first-line treatment of non‑neutropenic patients with candidemia, with or without invasive candidiasis, that may include patients with suspected or confirmed resistance to standard of care antifungal treatment. The trial is expected to enroll up to 20 adult patients at sites in the U.S. and globally. More information about the trial is available at www.clinicaltrials.gov, identifier NCT03604705.
The need for safe and effective broad-spectrum antifungal agents with favorable pharmacokinetic properties remains high, especially with the growing number of immunocompromised patients who are at increased risk for developing these life-threatening infections. Many existing antifungal agents are poorly tolerated or ineffective against drug resistant strains. APX001A, the active moiety of the prodrug APX001, has a novel mechanism of action, inhibiting the fungal Gwt1 enzyme, and maintains its activity against organisms that have developed resistance to other drug classes. APX001A and APX001 have demonstrated broad spectrum in vitro and in vivo activity against Candida, Aspergillus and the rare molds, including species that are intrinsically resistant to antifungal drugs.
Candidemia is a major cause of morbidity and mortality in the health care setting. Candida spp. are ranked as the fourth main cause of bloodstream infections in hospitals, and the frequency of candidemia has increased in recent years. 1 The mortality rate for candidemia is approximately 30%–60%, and the prognosis is generally poor. 2
APX001 is a highly versatile prodrug of APX001A, which is a first-in-class small molecule drug candidate that inhibits the highly conserved fungal enzyme Gwt1, compromising growth of major fungal pathogens. In multiple nonclinical studies, APX001A has shown broad-spectrum activity against common species of Candida spp., and Aspergillus spp., including multi-drug resistant strains including Candida auris and rare, hard-to-treat molds including Fusarium spp., Scedosporium spp., and fungi from the Mucorales order. APX001 can be delivered in both oral and intravenous formulations.
Invasive infections due to Aspergillus, Fusarium, Scedosporium and fungi from the Mucorales order are especially difficult to treat resulting in high mortality rates (50-80%), even when patients receive standard of care treatment. The frequency of fungi resistant to both the azole and echinocandin classes of drugs is increasing. Thus, there remains a significant unmet medical need for a new broad-spectrum antifungal to treat serious, invasive fungal infections and reduce the existing high morbidity and mortality.
About Amplyx Pharmaceuticals
Amplyx Pharmaceuticals is developing first-in-class products for life-threatening conditions, with a near-term focus on deadly fungal pathogens in vulnerable patients. Amplyx’s drug discovery and development efforts have been supported by significant venture investment and grants from the National Institutes of Health. For more information, please visit www.amplyx.com.
1 Wisplinghoff H, Bischoff T, Tallent SM, Seifert H, Wenzel RP, Edmond MB. Nosocomial bloodstream infections in US hospitals: analysis of 24179 cases from a prospective nationwide surveillance study. Clin Infect Dis. 2004;39(3):309–317.
2 Flevari A, Theodorakopoulou M, Velegraki A, Armaganidis A, Dimopoulos G. Treatment of invasive candidiasis in the elderly: a review. Clin Interv Aging. 2013;8:1199–1208.
Heidi Chokeir, Ph.D.
Amplyx Pharmaceuticals, Inc.