– Livoletide pivotal study in
Prader-Willi syndrome (ZEPHYR) on track to report topline results
in 1H20 –
– Nevanimibe CAH Phase 2b study now enrolling
under amended protocol –
– New program, MLE-301, beginning preclinical
development to enable first-in-human study –
Millendo Therapeutics, Inc. (Nasdaq: MLND), a clinical-stage
biopharmaceutical company primarily focused on developing novel
treatments for orphan endocrine diseases with significant unmet
needs, today provided a corporate update and reported financial
results for the quarter ended June 30, 2019.
“Our ZEPHYR pivotal Phase 2b/3 clinical study in PWS patients is
off to a strong start, and we remain on track to report topline
results from the Phase 2b portion of the study in the first half of
2020,” said Julia C. Owens, President and Chief Executive Officer
of Millendo Therapeutics. “In addition, our Phase 2b clinical study
of nevanimibe in CAH is enrolling patients under an amended
protocol. We expect to provide an update on timelines for the CAH
study in the second half of 2019.
“We are announcing today our decision to discontinue the Phase 2
study of nevanimibe in Cushing’s syndrome, based on an analysis of
the feasibility of patient recruitment and a reprioritization of
resources. While this decision was a difficult one, we believe that
we are now able to better optimize investments across our portfolio
of potential novel therapies in areas of significant unmet need. In
line with this commitment, we are excited today to announce the
selection of a new compound to begin preclinical development
required to support a first-in-human study, MLE-301 for the
treatment of vasomotor symptoms associated with menopause. This
selective NK3R antagonist enables us to leverage our expertise in
VMS drug development.”
Second Quarter 2019 and Recent Highlights
- ZEPHYR on track to deliver topline results in 1H20: A
pivotal Phase 2b/3 study of livoletide for the treatment of
Prader-Willi syndrome (PWS) was initiated in 1Q19. Clinical trial
site activation has progressed well since study initiation with 26
sites recruiting in the U.S., Europe and Australia. The Phase 2b
portion of the study has the potential to support an NDA submission
for livoletide.
- Fast Track designation has been granted by the U.S. Food and
Drug Administration (FDA) to Millendo for the development of
livoletide for PWS: This designation is intended to expedite
the development and regulatory review of new drugs that address
unmet medical needs and have the potential to treat serious and
life-threatening conditions.
- Nevanimibe Phase 2b CAH study is enrolling under an amended
protocol: A Phase 2b study of nevanimibe in CAH that was
initiated in 3Q18 is now recruiting patients under an amended
protocol. The protocol amendment includes a 500 mg BID (twice a
day) starting dose and an extension of the study treatment period
to 16 weeks. An update on the CAH study timelines is planned for
2H19.
- Nevanimibe Phase 2 study in Cushing’s syndrome
discontinued: Millendo plans to discontinue the Phase 2 study
of nevanimibe in Cushing's syndrome due to slower than anticipated
enrollment. This strategic decision creates the opportunity for the
company to redirect its resources toward other development
programs.
- New pipeline asset MLE-301 beginning preclinical development
required to support first-in-human study: MLE-301, a neurokinin
3 receptor (NK3R) antagonist, is intended for the treatment of
vasomotor symptoms (VMS), defined as hot flashes and night sweats
in menopausal women. MLE-301 is a novel NK3R antagonist, a target
that Millendo and others have shown plays a key role in regulating
the activity of KNDy (kisspeptin/NKB/dynorphin) neurons, which are
overactive in menopausal women and play an important role in the
generation of VMS. MLE-301 is currently in preclinical development
and is expected to enter clinical trials in 2020.
“With the strategic reprioritization of our portfolio, we are
pleased that our current cash position is expected to support our
capital needs into the fourth quarter of 2020,” commented Louis J.
Arcudi III, Chief Financial Officer.
Second Quarter 2019 Financial Results
Cash Position: Cash, cash equivalents, marketable
securities and restricted cash were $56.6 million at June 30, 2019,
compared to $78.2 million at December 31, 2018.
Research and Development (R&D) Expenses: R&D
expenses were $6.0 million for the second quarter 2019, as compared
to $3.2 million for the same period in 2018. The increase in
R&D expenses was primarily driven by increased spending on the
company’s pivotal Phase 2b/3 study of livoletide in PWS and higher
compensation expenses as a result of increased headcount.
General and Administrative (G&A) Expenses: G&A
expenses were $4.2 million for the second quarter 2019, as compared
to $1.8 million for the same period in 2018. The increase in
G&A expenses was primarily driven by higher compensation
expense as a result of increased headcount and increased
professional and other fees to support ongoing business operations
and obligations associated with being a publicly traded
company.
Net Loss: The company’s net loss for the quarter ended
June 30, 2019 was $9.9 million as compared to $5.0 million for the
same period in 2018.
2019 Financial Guidance
Millendo expects that its cash, cash equivalents, marketable
securities and restricted cash will support the company’s capital
needs into the fourth quarter of 2020, beyond the readout for the
topline results of the Phase 2b portion of the pivotal Phase 2b/3
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. 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 classic congenital adrenal hyperplasia
(CAH). 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. 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 ongoing.
About MLE-301 MLE-301 is an investigational, neurokinin 3
receptor (NK3R) antagonist that acts to normalize the activity of
KNDy (kisspeptin/NKB/dynorphin) neurons, which are overactive in
menopausal women and play an important role in the generation of
vasomotor symptoms (VMS). MLE-301 works by reducing neurokinin B
signaling via KNDy neurons and is hypothesized to reduce the
frequency and severity of VMS. Millendo is advancing MLE-301 for
the treatment of VMS associated with menopause through preclinical
development to enable first-in-human clinical studies.
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 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, 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 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, and the timeline for its Phase 2b clinical study of
nevanimibe in CAH, the potential and timing for MLE-301 to enter
clinical trials, the potential of a future product launch, the
potential advantages of Millendo’s discontinuation of its Phase 2
study of nevanimibe in CS, the potential impact of Fast Track
designation, 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 June 30, 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
Six Months Ended
June 30,
June 30,
2019
2018
2019
2018
Operating Expenses Research and
development
$
5,981
$
3,200
$
12,185
$
5,969
General and administrative
4,179
1,786
8,632
3,405
Loss from operations
10,160
4,986
20,817
9,374
Other (income) expense, net
(289
)
56
(580
)
54
Net loss
(9,871
)
(5,042
)
(20,237
)
(9,428
)
Net loss attributable to noncontrolling interest
-
196
-
321
Net loss attributable to common stockholders
$
(9,871
)
$
(4,846
)
$
(20,237
)
$
(9,107
)
Net loss per share of common stock, basic and diluted
$
(0.74
)
$
(6.82
)
$
(1.51
)
$
(12.82
)
Weighted-average shares of common stock outstanding, basic and
diluted
13,379,842
710,390
13,368,981
710,390
Millendo Therapeutics, Inc. Condensed
Balance Sheet Data (in thousands)
June 30,
December 31,
2019
2018
Cash, cash equivalents, marketable securities and restricted cash
$
56,631
$
78,155
Other assets
11,693
5,919
Total assets
$
68,324
$
84,074
Total liabilities
$
13,299
$
10,952
Total stockholders' equity
55,025
73,122
Total liabilities and stockholders' equity
$
68,324
$
84,074
View source
version on businesswire.com: https://www.businesswire.com/news/home/20190812005267/en/
Millendo Investor Contact: Stephanie Ascher Stern
Investor Relations 212-362-1200 stephanie.ascher@sternir.com
Millendo Media Contact: Betsy Yates MacDougall
781-235-3094 byates@macbiocom.com
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