Amplyx’s APX001 gets FDA orphan drug status to treat multiple invasive fungal infections

APX001 has received orphan drug designation for four distinct indications: the treatment of invasive candidiasis, invasive aspergillosis, coccidioidomycosis, and rare mold infections caused by Scedosporium spp., Fusarium spp., and Mucorales fungi (including Mucor spp., and Rhizopus spp.).

Orphan drug designation qualifies APX001 for seven years of market exclusivity in the U.S. upon FDA approval of a new drug application (NDA) for the orphan designated indications.

The company was previously granted Qualified Infectious Disease Product (QIDP) designation for APX001 Injection (for intravenous use) and for APX001 Tablets (for oral use), which provides significant development incentives including eligibility for Fast Track designation, priority review and when combined with orphan drug designation, a total of twelve years of marketing exclusivity.

Mike Grey, president and CEO of Amplyx Pharmaceuticals, said: “These government-granted incentives highlight the significant need for safe and effective new antifungal treatments.

“The incidence and severity of invasive and drug-resistant fungal infections is increasing, including those caused by rare pathogens. This, coupled with the limited number of antifungal drug classes available, creates an urgent need for the development of novel broad-spectrum antifungal agents.”

The company is advancing APX001 in clinical development, having initiated its Phase 1 clinical program earlier this year. The Phase 1 program evaluates APX001 using both intravenous and oral formulations to address the need for treatment in both the hospital setting and continued convenient treatment after discharge from hospital.

The company plans to initiate Phase 2 studies in 2017.

About APX001

APX001 is a first-in-class small molecule drug candidate that targets and inhibits the conserved fungal enzyme Gwt1, compromising fungal growth of major fungal pathogens, including Candida and Aspergillus. APX001 has been evaluated extensively in preclinical studies, and has demonstrated broad-spectrum activity against infections caused by common species of Candida and Aspergillus, including species that are intrinsically resistant to antifungal drugs, 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.

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to APX001 and has also designated APX001 as a Qualified Infectious Disease Product (QIDP) for the treatment of multiple fungal infections.