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Cidara Therapeutics Initiates Phase 3 Trial of Lead Antifungal Rezafungin for Treatment of Invasive Candida Infections

By September 27, 2018No Comments

Cidara Therapeutics, Inc. (Nasdaq: CDTX), a biotechnology company
developing novel anti-infectives including immunotherapies, today
announced that the first trial site has been activated for ReSTORE, a
Phase 3 clinical trial evaluating the efficacy and safety of the
company’s lead antifungal, rezafungin, to treat candidemia and invasive
candidiasis. Rezafungin is a novel echinocandin antifungal being
developed as a once-weekly, high-exposure therapy for the treatment and
prevention of serious invasive fungal infections.

“Given the compelling data from our Phase 2 program, we are very pleased
to initiate our global Phase 3 program of rezafungin,” said Jeffrey
Stein, Ph.D., president and chief executive officer of Cidara.
“Difficult-to-treat, invasive Candida infections are associated
with a high mortality and affect the most vulnerable patients.
Rezafungin has the potential to be the first new antifungal approved for
this indication in over a decade and, given how the drug works, it has
the potential to improve outcomes for patients and allow them to leave
the hospital earlier, resulting in reduced healthcare costs.”

ReSTORE is a global, randomized, double-blind, controlled Phase 3
pivotal clinical trial evaluating the efficacy and safety of once-weekly
intravenous dosing of rezafungin compared to once-daily dosing of
caspofungin in patients with candidemia and/or invasive candidiasis. The
trial design will be similar to the company’s Phase 2 STRIVE study,
which met its primary safety and efficacy objectives. The ReSTORE trial
is designed to evaluate one rezafungin dosing regimen of 400 milligrams
(mg) for the first week followed by 200 mg of rezafungin once weekly for
up to four weeks in total. This treatment arm will be compared to
caspofungin in a 1:1 randomization.

The primary efficacy endpoint of ReSTORE, which will be used for a U.S.
Food and Drug Administration (FDA) New Drug Application submission, is
all-cause mortality at day 30. The primary efficacy endpoint for the
European Medicines Agency (EMA) is expected to be global response at day

“I am very enthusiastic about the initiation of this important trial of
rezafungin as a once-weekly echinocandin which may offer a new solution
for patients and physicians to treat serious invasive Candida
infections,” said Cornelius J. Clancy, M.D., Associate Professor of
Medicine and Director of the Mycology Research Unit and XDR Pathogen Lab
at the University of Pittsburgh. “There is an urgent and growing need
for new therapeutic approaches for these infections, which typically
afflict highly vulnerable patients, including those who are critically
ill and have compromised immune systems. Rezafungin has the potential to
overcome the significant limitations associated with current standards
of care such as sub-optimal dosing, increasing resistance, drug-drug
interactions and toxicities, all of which complicate therapy in patients
who are already ill.”

The ReSTORE trial will enroll approximately 184 modified
intent-to-treat, or mITT, patients and is expected to generate topline
data in 2020. This global trial is anticipated to be conducted at
approximately 100 clinical trial centers across the United States,
Europe, Asia and Australia.

About Candidemia/Invasive Candidiasis

Over 90 percent of invasive fungal infections begin in the hospital
setting. Invasive candidiasis and candidemia comprise the majority of
invasive fungal infections in intensive care units, internal medicine
and hematology wards within hospitals. Invasive candida infections are
considered to be serious and life-threatening and are associated with
high rates of morbidity and mortality. According to the Centers for
Disease Control and Prevention (CDC) surveillance data, the 30-day
all-cause mortality among people with candidemia is approximately 30

About Rezafungin

Rezafungin is a novel antifungal echinocandin being developed as a
once-weekly, high-exposure therapy for the treatment and prevention of
serious invasive fungal infections. Rezafungin has a unique
pharmacokinetic profile with a prolonged half-life and front-loaded
plasma exposure which, in contrast to all other echinocandins, allows
for once-weekly IV therapy. Rezafungin is being studied to address unmet
needs in the treatment of candidemia and invasive candidiasis as well as
for prophylaxis (prevention) of invasive fungal infections, including Candida,
Aspergillus and Pneumocystis in patients undergoing an
allogeneic bone marrow transplant. In the recent Phase 2 STRIVE trial,
data showed that once-weekly intravenous dosing of rezafungin was
generally well tolerated and safe in patients with candidemia and/or
invasive candidiasis.

About Cidara Therapeutics

Cidara is a clinical-stage biotechnology company focused on developing
new anti-infectives that have the potential to transform the standard of
care and save or improve patients’ lives. The company is currently
advancing its novel echinocandin antifungal, rezafungin acetate,
formerly known as CD101 IV, through clinical trials. Rezafungin has
improved pharmacokinetics compared to existing echinocandins and the
potential for expanded utility across patient settings. It is the only
once-weekly product candidate in development for the treatment and
prevention of life-threatening invasive fungal infections. The company’s
Phase 2 STRIVE clinical trial of rezafungin met its primary safety and
efficacy objectives, and provides support for Cidara to initiate Phase 3
pivotal trials in the treatment of candidemia and invasive candidiasis
and the prophylaxis of invasive fungal infections. Cidara is also
leveraging its novel Cloudbreak™ platform to develop antibody-drug
conjugates for the treatment of serious viral and Gram-negative
bacterial infections. Cloudbreak is the first immunotherapy discovery
platform designed specifically to create compounds that directly kill
pathogens and also direct a patient’s immune cells to attack and
eliminate bacterial, fungal or viral pathogens. Cidara is headquartered
in San Diego, California. For more information, please visit

Forward-Looking Statements

Statements contained in this press release regarding matters that are
not historical facts are “forward-looking statements” within the meaning
of the Private Securities Litigation Reform Act of 1995. Because such
statements are subject to risks and uncertainties, actual results may
differ materially from those expressed or implied by such
forward-looking statements. Such statements include, but are not limited
to, Cidara’s ability to successfully commence and complete Phase 3
clinical trials, the design and timing of those clinical trials, the
potential for rezafungin to successfully treat or prevent invasive
fungal infections and represent an improvement over current approaches,
and Cidara’s ability to successfully complete development of rezafungin
and receive regulatory approval for any indication. Risks that
contribute to the uncertain nature of the forward-looking statements
include: the success and timing of Cidara’s preclinical studies and
clinical trials; regulatory developments in the United States and
foreign countries; changes in Cidara’s plans to develop and
commercialize its product candidates; Cidara’s ability to obtain
additional financing; Cidara’s ability to obtain and maintain
intellectual property protection for its product candidates; and the
loss of key scientific or management personnel. These and other risks
and uncertainties are described more fully in Cidara’s Form 10-Q most
recently filed with the United States Securities and Exchange
Commission. All forward-looking statements contained in this press
release speak only as of the date on which they were made. Cidara
undertakes no obligation to update such statements to reflect events
that occur or circumstances that exist after the date on which they were

Cidara Therapeutics, Inc.
Partners, LLC
Robert H. Uhl
Managing Director

Sam Brown Inc.
Christy Curran