Millendo Therapeutics Initiates Pivotal Phase 2b/3 Clinical Study of Livoletide for the Treatment of Prader-Willi Syndrome

March 19, 2019 Off By BusinessWire

Top-line Phase 2b results are expected in 1H 2020 and may support an
NDA submission for livoletide

ANN ARBOR, Mich.–(BUSINESS WIRE)–Millendo
Therapeutics, Inc. (Nasdaq: MLND), a clinical-stage
biopharmaceutical company developing novel treatments for orphan
endocrine diseases, announced today that it has initiated its pivotal
Phase 2b/3 clinical study investigating livoletide (AZP-531) in patients
with Prader-Willi syndrome (PWS). The study, called ZEPHYR, will
evaluate the safety and efficacy of livoletide on food-related behaviors
in PWS patients. The primary endpoint is an assessment of livoletide’s
impact on hyperphagia, the excessive hunger which is a hallmark symptom
of the disease. ZEPHYR is one of the largest global PWS studies ever
conducted and its Phase 2b portion aims to recruit 150 patients from up
to 40 clinical sites in the United States and Europe.

Maithé Tauber, M.D., Professor of Pediatrics at the University of
Toulouse and Chief of Endocrinology and Medical Genetics at the
Children’s Hospital of Toulouse noted, “Livoletide is an exciting
investigational drug for Prader-Willi syndrome that has the potential to
treat hyperphagia, the unrelenting hunger that often leads to excessive
eating and is a root cause of morbidity and mortality in PWS patients.
Current strategies to manage the disease present a heavy burden on
caregivers, and do not address the underlying hyperphagia experienced by
patients. Livoletide has the potential to be an important new treatment
option that could positively impact the lives of patients and their
caregivers.”

The ZEPHYR study is a two-part, randomized, double-blind,
placebo-controlled pivotal Phase 2b/3 study. The study will begin with a
Phase 2b portion that includes a three-month double-blind,
placebo-controlled core period in which patients receive one of two
doses of livoletide or placebo followed by a nine-month extension period
in which all patients receive livoletide. The second part is a Phase 3
study that will consist of a six-month double-blind, placebo-controlled
core period in which patients will receive livoletide or placebo
followed by a six-month extension period in which all patients receive
livoletide. The study’s primary endpoint measures the change in
food-related behaviors using the validated Hyperphagia Questionnaire for
Clinical Trials (HQ-CT). ZEPHYR is a pivotal study and the results of
the Phase 2b portion of the Phase 2b/3 study may support a new drug
application (NDA) for livoletide.

Pharis Mohideen, M.D., Chief Medical Officer at Millendo, further noted,
”The initiation of our pivotal trial for livoletide is an important
step toward our mission to bring life-changing therapies to market for
rare endocrine diseases with significant unmet medical needs, like
Prader-Willi syndrome. We are pleased that multiple clinical sites are
actively enrolling patients, and we expect to report topline results
from the Phase 2b portion of ZEPHYR in the first half of 2020.”

Additional details about the ZEPHYR Study can be found at www.clinicaltrials.gov,
NCT03790865
and millendo.com/patients-and-families.

About Livoletide
Millendo’s lead asset, livoletide, is an
unacylated ghrelin analogue in late-stage clinical development for the
treatment of Prader-Willi syndrome (PWS), a rare genetic disease
characterized by hyperphagia, a chronic unrelenting hunger that leads to
obesity, metabolic dysfunction, reduced quality of life and early
mortality. In a randomized, double-blind, placebo-controlled Phase 2
clinical trial in 47 patients with PWS, administration of livoletide
once daily was associated with a clinically meaningful improvement in
hyperphagia, as well as a reduction in appetite. Millendo has received
orphan drug designation for livoletide from the U.S. Food and Drug
Administration, or FDA, and the European Medicines Agency, or EMA, for
the treatment of PWS. For more information about Millendo Therapeutics’
pivotal study of livoletide (ZEPHYR) please visit www.clinicaltrials.gov
(NCT03790865).

About Prader-Willi Syndrome
Prader-Willi syndrome (PWS) is a
genetic disease caused by the lack of expression of several genes on
chromosome 15, which leads to intellectual disability, short stature,
incomplete sexual development and hyperphagia, among other symptoms. PWS
patients are at risk of premature mortality, usually by the age of
30-40, mainly from obesity related conditions such as cardiovascular
disease, respiratory distress and from accidents. The incidence of PWS
is approximately 1 in 15,000 births. The prevalence of PWS is estimated
between 8,000-11,000 patients in the United States and 13,000-18,000 in
Europe. Currently, there is no effective or approved treatment for
hyperphagia and abnormal eating behaviors associated with PWS. Growth
hormone is used for improvement in height, cognition and body
composition, but has no effect on appetite and over-eating. The only way
to effectively manage hyperphagia, obesity and related complications of
PWS is strict control over access to food, creating significant burden
for families and caregivers.

About Millendo Therapeutics, Inc.
Millendo Therapeutics is a
late-stage biopharmaceutical company focused on developing novel
treatments for orphan endocrine diseases where current therapies do not
exist or are insufficient. As a leading orphan endocrine company,
Millendo creates distinct and transformative treatments where there is a
significant unmet medical need. The company is currently advancing
livoletide for the treatment of Prader-Willi syndrome and nevanimibe for
the treatment of classic congenital adrenal hyperplasia and endogenous
Cushing’s syndrome. For more information, please visit www.millendo.com.

Cautionary Statement Regarding Forward-Looking Statements
Certain
statements contained in this press release regarding matters that are
not historical facts, are forward-looking statements within the meaning
of Section 21E of the Securities Exchange Act of 1934, as amended, and
the Private Securities Litigation Reform Act of 1995, known as the
PSLRA. These include statements regarding management’s intentions,
plans, beliefs, expectations or forecasts for the future, and,
therefore, you are cautioned not to place undue reliance on them. No
forward-looking statement can be guaranteed, and actual results may
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consequently, actual results may differ materially from those expressed
or implied in the statements due to a number of factors, including
Millendo’s plans to develop and commercialize its product candidates,
including livoletide and nevanimibe; the timing of initiation of
Millendo’s planned clinical trials; the timing of the availability of
data from Millendo’s clinical trials; the timing of any planned
investigational new drug application or new drug application; Millendo’s
plans to research, develop and commercialize its current and future
product candidates; the clinical utility, potential benefits and market
acceptance of Millendo’s product candidates.

New factors emerge from time to time and it is not possible for Millendo
to predict all such factors, nor can Millendo assess the impact of each
such factor on the business or the extent to which any factor, or
combination of factors, may cause actual results to differ materially
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except as required by applicable law.