Millendo Therapeutics Announces ENDO 2019 Presentations on Livoletide for Prader-Willi Syndrome and Nevanimibe for Classic Congenital Adrenal Hyperplasia

March 14, 2019 Off By BusinessWire

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 its clinical candidates,
livoletide (AZP-531) and nevanimibe (ATR-101), will be featured in three
poster presentations at ENDO 2019, taking place March 23-26, 2019 in New
Orleans, La.

Livoletide, a potential first-in-class treatment for Prader-Willi
syndrome (PWS), will be featured in two poster sessions. The company
will provide an overview of the study design for the pivotal Phase 2b/3
ZEPHYR clinical trial to evaluate the safety and efficacy of livoletide
for hyperphagia and related food-related behaviors in PWS patients in
one poster session. Data from the livoletide nonclinical safety program
to support its clinical development will be presented in another
session. The company will also give an overview of its ongoing
nevanimibe Phase 2b open-label study in patients with classic congenital
adrenal hyperplasia (CAH) in a third session. Top-line results from the
Phase 2b portion of ZEPHYR and the Phase 2b study for nevanimibe in CAH
are expected in the first-half of 2020.

Details of the poster presentations are as follows:

Title: ZEPHYR, a Pivotal Phase 2b/3 Randomized,
Placebo-Controlled Study of Livoletide, a Novel Unacylated Ghrelin
Analog, for the Treatment of Hyperphagia and Food-Related Behaviors in
Patients with Prader-Willi Syndrome
Session: P05, Obesity
Comorbidities and Therapies
Date/ Time: Saturday, March 23,
2019, 1:00 – 3:00 PM CST
Location: ENDOExpo
Abstract:
https://www.abstractsonline.com/pp8/#!/5752/presentation/18359

Title: Multicenter, Dose-titration, Open-Label Phase 2b Study of
Nevanimibe Hydrochloride, a Novel ACAT1 Inhibitor, for the Treatment of
Classic Congenital Adrenal Hyperplasia
Session: P37, Adrenal
Tumors and Hyperplasia
Date/ Time: Sunday, March 24, 2019,
1:00 – 3:00 PM CST
Location: ENDOExpo
Abstract: https://www.abstractsonline.com/pp8/#!/5752/presentation/19302

Title: Nonclinical Development of AZP-531 (Livoletide): A Peptide
Analog of Unacylated Ghrelin for the Treatment of Hyperphagia in
Prader-Willi Syndrome
Session: P49, Treatment of
Obesity in Unique Populations
Date/ Time: Monday, March 25,
2019, 1:00 – 3:00 PM CST
Location: ENDOExpo
Abstract:
https://www.abstractsonline.com/pp8/#!/5752/presentation/11380

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 Nevanimibe
Nevanimibe decreases adrenal
steroidogenesis through the selective inhibition of ACAT1 and is being
studied for the treatment of two orphan adrenal diseases: classic
congenital adrenal hyperplasia (CAH) and endogenous Cushing’s syndrome
(CS). CAH is a rare, monogenic adrenal disease that requires lifelong
treatment with exogenous cortisol, often at high doses, which can make
it difficult for physicians to appropriately treat CAH without causing
adverse consequences. Millendo has received orphan drug designation for
nevanimibe for the treatment of CAH and CS from the FDA, as well as from
the EMA for the treatment of CAH. In a Phase 2 proof-of-concept clinical
trial, Millendo observed nevanimibe to be associated with clear signs of
clinical activity in seven of 10 treated patients and was reported to be
well tolerated at all dose levels. Millendo initiated a Phase 2b trial
of nevanimibe in CAH in September 2018 (NCT03669549).
A Phase 2 trial of nevanimibe for the treatment of patients with CS is
ongoing (NCT03053271).

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
differ materially from those projected. Millendo undertakes no
obligation to publicly update any forward-looking statement, whether as
a result of new information, future events or otherwise, except to the
extent required by law. Millendo uses words such as “anticipates,”
“believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,”
“will,” “should,” “could,” “estimates,” “predicts,” “potential,”
“continue,” “guidance,” and similar expressions to identify these
forward-looking statements that are intended to be covered by the
safe-harbor provisions of the PSLRA. Such forward-looking statements are
based on Millendo’s expectations and involve risks and uncertainties;
consequently, actual results may differ materially from those expressed
or implied in the statements due to a number of factors, including the
timing of the availability of data from Millendo’s clinical trials.

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
from those contained in any forward-looking statements. Forward-looking
statements included in this press release are based on information
available to Millendo as of the date of this press release. Millendo
disclaims any obligation to update such forward-looking statements to
reflect events or circumstances after the date of this press release,
except as required by applicable law.