Amplyx Pharmaceuticals Demonstrates APX001 has Potent Activity Against Deadly Multidrug-Resistant Candida Species

Data from Multiple Preclinical Studies Presented at the 28th European Congress of Clinical Microbiology and Infectious Diseases

SAN DIEGO April 30, 2018 – Amplyx Pharmaceuticals, a company developing first-in-class products for life-threatening infections, today announced that preclinical data demonstrating that APX001 has potent activity against deadly multidrug-resistant Candida species were presented last week at the 28th European Congress of Clinical Microbiology and Infectious Diseases (ECCMID) in Madrid, Spain.

The company presented preclinical data from its APX001 program in multiple oral and poster presentations at the conference, including pharmacokinetics/pharmacodynamics (PK/PD) analysis of APX001 in neutropenic mouse models of invasive candidiasis. Research presented also showed strong activity in preclinical models of some of the hardest to treat Candida species, including infections caused by drug-resistant C. auris and C. glabrata, consistent with a lack of cross-resistance for a novel mechanism of action drug. Additional data highlighted the antifungal activity and penetration of APX001 into the brain and eye in a rabbit model of invasive candidiasis.

These data, taken together with data previously presented by Amplyx, support advancement of APX001 into clinical studies in invasive candidiasis and candidemia, and the company plans to initiate a Phase 2, IV to oral switch, treatment study in patients with candidemia this year. The dose regimen to be evaluated in this study has been studied in Phase 1 trials and achieves the target exposures associated with successful treatment in mouse models of invasive candidiasis.

“The team at Amplyx, along with our collaborators at prominent academic institutions, have generated data that highlight the unique characteristics of APX001 and support its advancement into Phase 2 studies,” said Ciara Kennedy, Ph.D., president and CEO of Amplyx. “The wide distribution and strong antifungal activity of APX001 against multidrug-resistant pathogens, including Candida auris and Candida glabrata, along with robust PK/PD data to support antifungal dose selection, position APX001 as a promising, novel antifungal agent for the treatment of these hard-to-treat, life-threatening fungal infections.”

Posters presented at “Hot Topics in Antifungal Use” session:

Title: Candida auris is highly in vitro susceptible to APX001A in EUCAST antifungal susceptibility testing

Author: Jørgensen, Chowdhary, Meis, Astvad, Arendrup

Title: Pharmacokinetics/pharmacodynamics (PK/PD) of the novel, first-in-class glycosylphosphatidylinositol (GPI) inhibitor APX001 in the neutropenic murine invasive candidiasis model against C. albicans (CA), C. glabrata (CG), and C. auris (CAU)

Author: Lepak, Zhao, VanScoy, Bader, Ambrose, Andes

Oral presentations at “News in Antifungal Therapy” session:

Title: APX001 is effective against echinocandin and multi-drug resistant Candida isolates

Author: Perlin, Zhao, M. Lee, A. Lee, Paderu, Kolesnikova, Dolgov, Senin, Rasic, Park

Title: Efficacy of APX001 in treatment of Candida endophthalmitis and haematagenous meningoencephalitis in experimental non-neutropenic rabbit model

Author: Petraitis, Petraitiene, Maung, Mansbach, Shaw, Walsh

All abstracts are available on the ECCMID website at

About APX001

APX001 is the 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, including Candida and Aspergillus. The novel mechanism of action of APX001 translates into a highly versatile drug that demonstrates activity against drug-resistant strains and can be delivered in both oral and intravenous formulations. 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.

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. As with multidrug-resistant bacteria, the frequency of fungi resistant to both the azole and candin 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 infections, 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


Heidi Chokeir, Ph.D.
Managing Director
Canale Communications
(619) 849-5377