SAN DIEGO, Nov. 16, 2016 /PRNewswire/ -- Amplyx
Pharmaceuticals, a company developing novel antifungal agents for
life-threatening fungal infections, announced today that the U.S.
Food and Drug Administration (FDA) Office of Orphan Product
Development has granted orphan drug designation to APX001, the
company's lead drug candidate. 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.
"These government-granted incentives highlight the significant
need for safe and effective new antifungal treatments," said
Mike Grey, president and CEO of
Amplyx Pharmaceuticals. "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.
About Amplyx Pharmaceuticals
Amplyx Pharmaceuticals is developing novel, broad-spectrum
antifungal agents for the treatment of life-threatening fungal
infections. 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.
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SOURCE Amplyx Pharmaceuticals