– Initiated pivotal study of livoletide in Prader-Willi syndrome
(PWS) –
– Established foundation for a commercial organization in Boston –
– Protocol amendment underway for nevanimibe study in congenital
adrenal hyperplasia (CAH) –
ANN ARBOR, Mich.–(BUSINESS WIRE)–Millendo
Therapeutics, Inc. (Nasdaq:MLND), a clinical-stage biopharmaceutical
company developing novel treatments for orphan endocrine diseases, today
provided a corporate update and reported financial results for the first
quarter 2019 ended March 31, 2019.
“Our first quarter accomplishments represent important progress toward
the achievement of our strategic priorities for 2019, including the
initiation of our ZEPHYR pivotal Phase 2b/3 clinical trial in PWS
patients, which has the potential to support an NDA submission for
livoletide,” said Julia C. Owens, President and Chief Executive Officer
of Millendo Therapeutics. “Furthermore, we presented on both trials for
our late stage clinical candidates, livoletide and nevanimibe, at ENDO
and established the foundation for a commercial organization in the
Boston area, ahead of topline data for livoletide in the first half of
next year. We look forward to continuing to make important strides for
patients, families and caregivers throughout 2019.”
First Quarter 2019 and Recent Highlights
-
Initiated ZEPHYR, a Pivotal Phase 2b/3 Clinical Trial of Livoletide
in PWS Patients: In March 2019, Millendo initiated the ZEPHYR
trial, which has the potential to support a New Drug Application (NDA)
submission. Topline data from the Phase 2b portion of the study is
expected in 1H20. -
Initiated Development of a Pen Delivery System for Livoletide: Millendo
has initiated pre-clinical activities in support of the development of
a multi-dose pen device to improve patient and caregiver convenience
and further simplify administration of livoletide. -
Protocol Amendment for Nevanimibe Phase 2b Study in CAH Underway:
Preliminary data with a starting dose of 1000 mg BID showed lower
tolerability than expected based on prior clinical experience with the
drug. Enrollment will be paused while a protocol amendment is
submitted to add a lower starting dose. Patients currently enrolled in
the study will continue per protocol and may dose escalate as planned. -
Delivered Multiple Presentations at ENDO 2019: In March,
Millendo presented three posters at ENDO 2019, including an overview
of the study design for ZEPHYR, data from the livoletide nonclinical
safety program, and an overview of the ongoing nevanimibe Phase 2b
study in CAH. -
Initiated Building a Commercial Organization in the Boston area:
In April, Millendo entered into a lease for offices in Lexington, MA,
and has begun to establish a commercial organization to help prepare
for potential future product launch.
First Quarter 2019 Financial Results
Cash Position: Cash, cash equivalents and marketable securities
were $67.0 million at March 31, 2019, compared to $77.7 million at
December 31, 2018.
Research and Development (R&D) Expenses: R&D expenses were
$6.2 million for the first quarter 2019, as compared to $2.8 million for
the same period in 2018. The increase in R&D expenses was primarily
driven by increased spending on the company’s Phase 2b/3 pivotal study
of livoletide in PWS and higher employee compensation costs due to
increased headcount.
General and Administrative (G&A) Expenses: G&A expenses were
$4.5 million for the first quarter 2019, as compared to $1.6 million for
the same period in 2018. The increase in G&A expenses was primarily
driven by increased costs related to employee compensation and
professional fees to support ongoing business operations and compliance
with obligations associated with being a publicly traded company.
Net Loss: The company’s net loss for the quarter ended March 31,
2019 was $10.4 million as compared to $4.4 million for the same period
in 2018.
2019 Financial Guidance
Millendo expects that its cash, cash equivalents, and marketable
securities will support the company’s capital needs into the second half
of 2020, beyond the readout for the topline results of the Phase 2b
portion of the Phase 2b/3 pivotal study of livoletide in PWS, which is
expected in the first half of 2020. This cash runway guidance is based
on the company’s current operational plans and excludes any additional
funding or business development activities.
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 March 2019, the company initiated a pivotal Phase 2b/3
clinical study of livoletide in patients with PWS. In a previous
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
(FDA) and the European Medicines Agency (EMA) for the treatment of PWS.
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 Nevanimibe
Nevanimibe decreases adrenal
steroidogenesis through the 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 in patients with CAH, Millendo observed nevanimibe to be
associated with clear signs of clinical activity in seven of 10 treated
patients. A Phase 2b trial of nevanimibe in CAH (NCT03669549)
is active with enrollment currently paused as described above, and a
Phase 2 trial of nevanimibe in CS (NCT03053271)
is ongoing.
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 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 Millendo’s expectations regarding the potential for
an NDA submission for livoletide, the timing of data from its clinical
trials, including the timing of topline data from the Phase 2b portion
of its ZEPHYR trial, the potential of a future product launch,
Millendo’s expectations regarding its 2019 and 2020 milestones, and
Millendo’s expectations regarding its cash runway, 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 to the
satisfaction of applicable regulatory authorities, enrollment and
retention of patients in clinical trials is an expensive and
time-consuming process and could be made more difficult or rendered
impossible by multiple factors outside Millendo’s control, Millendo’s
product candidates may cause undesirable side effects or have other
properties that could delay or prevent their regulatory approval, or
limit their commercial potential and Millendo faces substantial
competition. You should refer to the risk factor disclosure set forth in
the periodic reports and other documents we file with the SEC available
at www.sec.gov,
including without limitation our Annual Report on Form 10-K for our
fiscal year ended December 31, 2018 and our Quarterly Report on Form
10-Q for our fiscal quarter ended March 31, 2019.
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.
| Millendo Therapeutics, Inc. | |||||||||
| Condensed Statements of Operations | |||||||||
| (in thousands except share and per share amounts) | |||||||||
| Three Months Ended | |||||||||
| March 31, | |||||||||
| 2019 | 2018 | ||||||||
|
Operating Expenses |
|||||||||
| Research and development | $ | 6,204 | $ | 2,769 | |||||
| General and administrative | 4,453 | 1,619 | |||||||
| Loss from operations | 10,657 | 4,388 | |||||||
| Other (income) expense, net | (291) | (2) | |||||||
| Net loss | (10,366) | (4,386) | |||||||
| Net loss attributable to noncontrolling interest | – | 125 | |||||||
| Net loss attributable to common stockholders | $ | (10,366) | $ | (4,261) | |||||
| Net loss per share of common stock, basic and diluted | $ | (0.78) | $ | (6.00) | |||||
|
Weighted-average shares of common stock outstanding, basic and diluted |
13,357,999 | 710,390 | |||||||
| Millendo Therapeutics, Inc. | |||||||||
| Condensed Balance Sheet Data | |||||||||
| (in thousands) | |||||||||
| March 31, | December 31, | ||||||||
| 2019 | 2018 | ||||||||
| Cash, cash equivalents and marketable securities | $ | 66,987 | $ | 77,671 | |||||
| Other assets | 7,318 | 6,403 | |||||||
| Total assets | $ | 74,305 | $ | 84,074 | |||||
| Total liabilities | $ | 10,619 | $ | 10,952 | |||||
| Total stockholders’ equity | 63,686 | 73,122 | |||||||
| Total liabilities and stockholders’ equity | $ | 74,305 | $ | 84,074 | |||||
Contacts
Millendo Investors:
Stephanie Ascher
Stern Investor
Relations
212-362-1200
stephanie.ascher@sternir.com
Millendo Media:
Betsy Yates
MacDougall
781-235-3094
byates@macbiocom.com