Millendo Reports Full Year 2018 Operating and Financial Results

March 29, 2019 Off By BusinessWire

–Initiated ZEPHYR, a pivotal Phase 2b/3 clinical study of livoletide
for patients with Prader-Willi syndrome (PWS), in 1Q19–

–Topline data from Phase 2b Portion of ZEPHYR and Phase 2b clinical
study of nevanimibe for classic congenital adrenal hyperplasia (CAH)
expected in 1H20–

–Completed successful merger and associated financing resulting in
$85M in total proceeds and sufficient cash runway to support late-stage
clinical development–

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 year ended December 31, 2018.

“2018 was a transformative year for Millendo, as we continued to advance
our leading orphan endocrine pipeline, prepared for the initiation of a
pivotal Phase 2b/3 trial of livoletide for PWS, advanced nevanimibe into
a Phase 2b trial in CAH and became a publicly traded company,” said
Julia C. Owens, President and Chief Executive Officer of Millendo
Therapeutics. “Our focus in 2019 is to advance our first-in-class
programs for livoletide and nevanimibe and establish our commercial
capabilities to support our late-stage pipeline assets. In addition, we
expect to report topline data from our PWS and CAH clinical trials in
the first half of 2020.”

Fourth Quarter 2018 and Recent Highlights

- Initiated ZEPHYR, a Pivotal Phase 2b/3 Clinical Study of
  Livoletide: In March 2019, Millendo initiated ZEPHYR, one of the
  largest global PWS studies ever conducted. The company expects to
  recruit 150 patients from up to 40 clinical sites across the United
  States and Europe in the Phase 2b portion of the pivotal study, which
  has the potential to support a New Drug Application (NDA) submission.

- Initiated Phase 2b Clinical Study of Nevanimibe in Patients with
  CAH: In September 2018, Millendo initiated a Phase 2b clinical
  trial of nevanimibe in patients with CAH. This trial was designed
  based on the Phase 2 dose-escalation trial that established
  proof-of-concept for nevanimibe, and is expected to include 20-24
  patients across approximately 10 sites.

- Became a Publicly-Traded Company: In December 2018, Millendo
  completed a successful merger and associated financing resulting in
  total proceeds of $85 million, which will be used to fund the
  company’s late-stage programs in orphan endocrine diseases into the
  second half of 2020.

- Strengthened Executive Leadership Team: In 2018, Millendo
  appointed Louis Arcudi III as Chief Financial Officer and Ryan Zeidan,
  Ph.D., as Senior Vice President, Development.

Anticipated 2019-2020 Milestones

- Establish a commercial organization in the Boston area in the second
  quarter of 2019.

- Report topline data from the Phase 2b study of nevanimibe in patients
  with CAH in the first half of 2020, following a study update in the
  second half of 2019.

- Report topline data from the Phase 2b study of livoletide in patients
  with PWS in the first half of 2020.

Full Year 2018 Financial Results

Cash Position: Cash, cash equivalents and marketable securities
were $77.7 million at December 31, 2018, compared to $17.6 million at
December 31, 2017.

Research and Development (R&D) Expenses: R&D expenses were
$14.4 million for 2018, as compared to $14.5 million for the same period
in 2017. The decrease in R&D expenses was primarily driven by a pipeline
change towards support of the company’s Phase 2 clinical studies in
livoletide and nevanimibe, offset by higher employee compensation costs
due to increased headcount.

General and Administrative (G&A) Expenses: G&A expenses were
$8.7 million for 2018, as compared to $6.0 million for the same period
in 2017. 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 year ended December 31,
2018 was $27.2 million.

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
clinical study of nevanimibe in CAH and the Phase 2b portion of the
Phase 2b/3 pivotal study of livoletide in PWS, which are both 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
that may be received or business development activities that may be
undertaken.

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, a pivotal Phase 2b/3 clinical study of
livoletide in patients with PWS was initiated. 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 Millendo’s expectation
regarding the timing of data from its clinical trials, 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. 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 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. These risks and uncertainties are more fully described in
Millendo’s filings with the Securities and Exchange Commission (the
“SEC”), including in the section entitled “Risks Related to Millendo” in
OvaScience, Inc.’s (with whom Millendo merged as described above) Form
S-4 as filed on September 26, 2018 with the SEC, and subsequent reports
that Millendo files with the SEC.

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)

	Year Ended
	December 31,
	2018			2017
Operating Expenses
Research and development	$	14,425		$	14,526
Acquired in-process research and development		–			63,844
General and administrative		8,691			5,956
Other general expenses		3,758			–
Loss from operations		26,874			84,326

Other (income) expense, net		303			260
Net loss		(27,177	)		(84,586	)
Net (income) loss attributable to noncontrolling interest		(15	)		8
Net loss attributable to common stockholders	$	(27,192	)	$	(84,578	)
Net loss per share of common stock, basic and diluted	$	(17.58	)	$	(321.81	)
Weighted-average shares of common stock outstanding, basic and diluted		1,547,051			262,823


Millendo Therapeutics, Inc.
Condensed Balance Sheet Data
(in thousands)

	December 31,
	2018			2017
Cash, cash equivalents and marketable securities	$	77,671		$	17,578
Other assets		6,403			2,234
Total assets	$	84,074		$	19,812

Total liabilities	$	10,952		$	4,829
Convertible preferred stock		–			132,922
Redeemable noncontrolling interests		–			10,584
Total stockholders’ equity (deficit)		73,122			(128,523	)
Total liabilities, convertible preferred stock, redeemable noncontrolling interests and stockholders’ equity (deficit)	$	84,074		$	19,812