Full Press Release Details
Therapeutics Appoints Christophe Arbet-Engels, MD, PhD, as Chief Medical Officer
- Company also provides update
on timeline for pivotal ZEPHYR study -
ANN ARBOR, Mich., February 10, 2020 - Millendo
Therapeutics, Inc. (Nasdaq: MLND), a late-stage biopharmaceutical company primarily focused on developing novel treatments
for orphan endocrine diseases, announced today that Christophe Arbet-Engels, MD, PhD, has joined the company as Chief Medical
Officer. The company also announced that it expects to report topline results from the Phase 2b portion of its pivotal Phase 2b/3
clinical trial for livoletide in patients with Prader-Willi syndrome (PWS), called ZEPHYR, early in the second quarter of 2020.
"We are honored and excited to welcome Christophe to the
team," said Julia C. Owens, PhD, President and Chief Executive Officer of Millendo Therapeutics. "Christophe brings
to Millendo broad experience in endocrinology and guiding clinical stage therapies through registration and lifecycle management.
We believe this expertise will be a significant asset with the data readout from our pivotal clinical trial for livoletide in PWS
anticipated in early second quarter of this year and as we look ahead to potential commercialization of our product candidates,
including livoletide for PWS."
Dr. Arbet-Engels joins Millendo from Poxel Pharmaceuticals
where he was Chief Medical Officer, Executive Vice President Late Development and Medical Affairs with responsibility for all
medical activities for Poxel's portfolio, including driving portfolio strategy and execution of registration programs. Previously,
he served as Vice President, Worldwide Medical, Collaborative Medical Sciences at Biogen, where Dr. Arbet-Engels built, developed
and led global medical research, clinical operations, biostatistics/analytics, communication and expanded access program teams
to advance the medical sciences in multiple therapeutic areas. Dr. Arbet-Engels has also held several senior leadership positions,
globally and locally, in clinical development and medical affairs at pharmaceutical and biotech companies including Boehringer
Ingelheim, Roche, Merck, Aventis (now Sanofi) and Ligand Pharmaceuticals where he led the clinical
development and registration, launch and lifecycle management for several new medicines. Dr. Arbet-Engels has a medical degree
from University of Paris Sud, a PhD in endocrinology/diabetes and metabolism from University of Paris Descartes, and a master's
degree in business administration from Rutgers University. He
will be based in Millendo's Lexington, Massachusetts office.
"This is a very exciting time to join the talented team
at Millendo," Dr. Arbet-Engels added. "With the data readouts from the Phase 2b portion of the study for livoletide
in PWS expected in early second quarter of 2020 and for nevanimibe in classic congenial adrenal hyperplasia (CAH) in the second
half of the year, I look forward to working closely with Julia and the rest of the team to guide the company through the critical
next steps for advancing Millendo's pipeline programs through development and into commercialization."
Inducement Equity Award
On February 10, 2020, the Compensation Committee of Millendo's
Board of Directors approved, effective as of February 10, 2020, the grant of an inducement stock option to purchase 140,000 shares
of the Company's common stock to Dr. Arbet-Engels. The stock option awarded to Dr. Arbet-Engels will have an exercise price
equal to the closing price per share of the Company's common stock on February 10, 2020, and will vest and become exercisable
over four years, with 25% of the shares vesting on February 10, 2021, the one-year anniversary of the vesting commencement date,
and the remaining shares vesting ratably over the subsequent 36 months, subject to Dr. Arbet-Engels' continued service with
the Company as of each such date. The stock option is subject to acceleration if Dr. Arbet-Engels' employment terminates
in connection with a change in control. The stock option has a ten-year term and is subject to the terms and conditions of the
stock option agreement pursuant to which the option was granted.
The stock option was granted as an inducement material to Dr.
Arbet-Engels entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4).
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 previous
randomized, double-blind, placebo-controlled Phase 2 clinical trial in 47 patients with PWS, administration of livoletide once
daily for two weeks was associated with a clinically meaningful improvement in hyperphagia, as well as a reduction in appetite.
Millendo has received both Orphan Drug Designation and Fast Track Designation for livoletide for the treatment of PWS from the
U.S. Food and Drug Administration (FDA) and Orphan Drug Designation from the European Medicines Agency (EMA). For more information
about Millendo's pivotal study of livoletide (ZEPHYR) please visit www.clinicaltrials.gov
(NCT03790865) or the Patients and Families portion of our website.
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 hyperphagia, intellectual disability, short stature and incomplete sexual development, among other symptoms. PWS patients
are at risk of premature mortality, 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 the ZEPHYR study
The ZEPHYR study is a two-part, randomized, double-blind, placebo-controlled
pivotal Phase 2b/3 study. The first part is a Phase 2b study 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 Phase 2b portion of the study, one of the largest global PWS studies ever conducted, has completed
recruitment for patients ages 8 to 65 with over 150 patients across 39 clinical sites in the United States, Europe and Australia.
Millendo continues to implement a protocol amendment globally adding an additional cohort of PWS patients ages four to seven. Sites
that recruit pediatric patients will continue to actively recruit patients for this cohort. 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) during each core
period. ZEPHYR is a pivotal study and the results of the Phase 2b portion of the study may be sufficient to support a new drug
application (NDA) for livoletide.
About Millendo Therapeutics, Inc.
Millendo Therapeutics is a late-stage biopharmaceutical
company primarily 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 seeks to create 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,
nevanimibe for the treatment of classic congenital adrenal hyperplasia and MLE-301 for the treatment of vasomotor symptoms associated
with menopause. 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 27A
of the Securities Act of 1933, as amended and Section 21E of the Securities Exchange Act of 1934, as amended. These include statements
regarding our plans to develop and commercialize our product candidates and the progress and timing of our ongoing and planned
clinical trials for our product candidates, and, therefore, you are cautioned not to place undue reliance on them. In some cases,
you can identify forward-looking statements by the words "may," "might," "will," "could,"
"would," "should," "expect," "intend," "plan," "objective,"
"anticipate," "believe," "estimate," "predict," "project," "potential,"
"continue" and "ongoing," or the negative of these terms, or other comparable terminology intended to
identify statements about the future. 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 that Millendo has incurred significant losses since inception, Millendo has a limited operating
history and has never generated any revenue from product sales, Millendo will require additional capital to finance its operations,
Millendo's future success is dependent on the successful clinical development, regulatory approval and subsequent commercialization
of livoletide, nevanimibe and any future product candidates, preclinical studies or earlier clinical trials are not necessarily
predictive of future results and the results of Millendo's clinical trials may not support Millendo's livoletide or nevanimibe
claims, Millendo may encounter substantial delays in its clinical trials or Millendo may fail to demonstrate safety and efficacy