Appili Receives Orphan Drug Designation from FDA for ATI-1501

Treating C. difficile in Children

HALIFAX, Nova Scotia–(BUSINESS WIRE)–Appili Therapeutics Inc., an anti-infective pharmaceutical development
company, announced today that the U.S. Food and Drug Administration
(FDA) has granted orphan drug designation for ATI-1501, a taste-masked
reformulation of metronidazole with the potential to effectively treat Clostridium
infection (CDI) in children.

CDI is one of the U.S. Centers for Disease Control’s most urgent
antibiotic-resistant bacterial threats. CDI affects over half a million
Canadians and Americans each year. It reoccurs in 83,000 patients and is
responsible for 29,000 deaths annually. The incidence of CDI in children
has been steadily increasing over recent years, and thus a taste-masked
metronidazole formulation is urgently needed for this population.

“The bitter metronidazole tablet is highly unpalatable for most
children, so ATI-1501 has the potential to fundamentally improve
compliance rates for these patients who are in need of a safe and
effective treatment for CDI,” said Kevin Sullivan, CEO of Appili

The FDA Orphan Drug Act provides orphan status to new drugs that are
defined as those intended for the safe and effective treatment,
diagnosis, or prevention of rare diseases that affect less than 200,000
people in the United States. This orphan drug designation provides
Appili with several benefits, including seven years of marketing
exclusivity upon approval of ATI-1501, reduced FDA application and
administration fees and tax credits for clinical research in the U.S.

“In just 3 months, we received orphan drug designation from the FDA,”
continued Mr. Sullivan. “Having this quick turnaround is an indication
of the high quality of the regulatory team we have built, and an
important step on our path to becoming the first company to offer an
approved treatment for C. difficile designed specifically for
children affected by this serious infection.”

About Appili Therapeutics

Appili is dedicated to identifying, acquiring and advancing novel
therapeutics in the area of infectious disease and has commenced
development on two anti-infective programs, while continuing its search
to acquire additional high quality anti-infective programs at all stages
of development. The company plans to take its lead drug candidate,
ATI-1501, a potential treatment for C. difficile infection, into
clinical trials by 2017. Appili’s second product, ATI-1503, is a novel
antibiotic with the potential to treat deadly infections such as Klebsiella
. These drug-resistant Gram-negative bacterial infections
have been identified by the U.S. Center for Disease Control (CDC) as
posing the highest threat to human health. Appili is co-founded and
backed by the life-science/healthcare investment bank Bloom Burton & Co.
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Appili Therapeutics Inc.
Media Relations:
Cameron, 902-209-4704

Kevin Sullivan, CEO, Appili Therapeutics