SAN DIEGO – June 14, 2016 – Amplyx Pharmaceuticals today announced that the U.S. Food and Drug Administration (FDA) has granted Qualified Infectious Disease Product (QIDP) designation to APX001, the company’s lead product candidate for treatment of three separate fungal infections: invasive aspergillosis; invasive candidiasis; and coccidioidomycosis.
Created under the Generating Antibiotics Incentives Now (GAIN) Act of 2012, the QIDP designation was established for the purpose of encouraging pharmaceutical companies to develop new antimicrobial drugs to treat serious and life-threatening infections, including specific fungal pathogens. The QIDP designation of APX001 provides Amplyx with significant incentives for the development of APX001, including priority FDA review, eligibility for Fast Track designation and a five-year extension of marketing exclusivity under the Hatch-Waxman Act.
The need for improved treatment of invasive fungal infections remains a challenge, particularly with the growing number of immunocompromised patients who are at increased risk for developing these life-threatening infections, and in whom treatment can be complex. Many existing antifungal agents can be difficult to use, poorly tolerated, or ineffective due to drug resistant strains. APX001A, the active moiety of the prodrug APX001, has a novel mechanism of action, inhibiting the Gwt1 fungal enzyme, and therefore maintains its activity against fungal organisms that have developed resistance to other drug classes. APX001 and APX001A have demonstrated broad spectrum activity against Candida and Aspergillus fungi, including species that are intrinsically resistant to antifungal drugs. APX001 is about to undergo human testing and has the potential to be used as a front line agent for the treatment of invasive fungal infections, either alone or in combination with existing antifungal drugs.
Amplyx licensed APX001 from Eisai Co., Ltd in 2015. APX001 has been evaluated extensively in preclinical studies, and has demonstrated broad-spectrum activity against infections caused by common species of Candida and Aspergillus, as well as activity against rare, hard-to-treat molds including Fusarium, Scedosporium and fungi from the Mucorales order. Patients with compromised immune systems due to chemotherapy or other immune-compromising medical treatments are susceptible to these potentially deadly fungal infections, and there are over 600,000 cases of invasive fungal infections due to Candida, Aspergillus and rare molds annually worldwide. Amplyx is developing both IV and oral formulations of APX001 to address the need for hospital administration, as well as continued dosage after hospital discharge.
About Amplyx Pharmaceuticals
Amplyx Pharmaceuticals is developing novel, broad-spectrum antifungal agents for the treatment of life-threatening fungal infections. The company’s lead compound, APX001, is expected to enter clinical development in 2016 and has shown broad-spectrum activity in animal models of invasive fungal infection by common species of Candida and Aspergillus, as well as high potency against rare, difficult-to-treat molds. Amplyx has raised $51.5 million in venture capital and received more than $10 million in grants from the National Institutes of Health to support its drug discovery and development efforts. The company’s research operations are located at Johnson & Johnson Innovation, JLABS in San Diego, Calif.
Heidi Chokeir, Ph.D.
Senior Vice President