NEW YORK, Oct. 17, 2018 (GLOBE NEWSWIRE) -- Axsome Therapeutics, Inc. (NASDAQ: AXSM), a clinical-stage
biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders, received on
Tuesday, October 16, Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for AXS-12 for the treatment of
narcolepsy. Narcolepsy is a serious, debilitating, neurological condition characterized by excessive daytime sleepiness and
cataplexy, which is a sudden reduction or loss of muscle tone while a patient is awake. AXS-12 is a novel, oral, highly selective
and potent norepinephrine reuptake inhibitor. Yesterday morning, Axsome announced that it plans to initiate a Phase 2 trial of
AXS-12 for the treatment of the symptoms of narcolepsy in the fourth quarter of this year with top-line results anticipated in the
first half of 2019.
“We are very pleased to have received Orphan Drug Designation from the FDA for AXS-12 on the heels of our recent
announcement of this new CNS product candidate for the treatment of narcolepsy,” said Herriot Tabuteau, M.D., Chief Executive
Officer of Axsome. “The designation is an important regulatory milestone in the development of AXS-12 for this debilitating
condition. We look forward to starting our planned Phase 2 trial of AXS-12 in patients with narcolepsy this quarter.”
Orphan Drug Designation is granted by the FDA Office of Orphan Drug Products to promising drugs intended for the
safe and effective treatment of rare diseases, defined as those affecting fewer than 200,000 people in the U.S. This designation
may entitle Axsome to a period of seven years of marketing exclusivity in the U.S. upon FDA approval. Orphan Drug Designation also
confers special incentives to Axsome including tax credits towards the cost of clinical trials and a waiver of the Company’s
obligation to pay the FDA application user fees for the product as required by the Prescription Drug User Fee Act.
Key Opinion Leader Conference Call
On October 16, 2018, Axsome hosted a key opinion leader (KOL) conference call and webcast with slides focusing
on AXS-12 and unmet needs in narcolepsy. The call and webcast featured a presentation from Dr. Michael J. Thorpy, Professor of
Neurology at Albert Einstein College of Medicine, who discussed the clinical features of and current treatment landscape for
narcolepsy. An archived webcast of the call, with slides, can be accessed on the "Webcasts & Presentations" page of the "Investors"
section of the Company's website at axsome.com.
About Narcolepsy
Narcolepsy is a serious and debilitating neurological condition that causes dysregulation of the sleep-wake
cycle and is characterized clinically by excessive daytime sleepiness, cataplexy, hypnagogic hallucinations, sleep paralysis, and
disrupted nocturnal sleep. Narcolepsy afflicts an estimated 185,000 individuals in the U.S. Cataplexy is seen in an estimated 70%
of narcolepsy patients and is a sudden reduction or loss of muscle tone while a patient is awake, typically triggered by strong
emotions such as laughter, fear, anger, stress, or excitement. Narcolepsy interferes with cognitive, psychological, and social
functioning, increases the risk of work- and driving-related accidents, and is associated with a 1.5 fold higher mortality rate.
Depression is reported in up to 57% of patients.
About
AXS-12
AXS-12 (reboxetine) is a novel, oral, investigational medicine in development for the treatment of the symptoms
of narcolepsy. AXS-12 is a highly selective and potent norepinephrine reuptake inhibitor. AXS-12 is an investigational drug product
not approved by the FDA.
About Axsome Therapeutics, Inc.
Axsome Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing novel therapies for the
management of central nervous system (CNS) disorders for which there are limited treatment options. Axsome’s core CNS product
candidate portfolio includes four clinical-stage candidates, AXS-05, AXS-07, AXS-09, and AXS-12. AXS-05 is currently in a Phase 3
trial in treatment resistant depression (TRD), a Phase 2/3 trial in agitation associated with Alzheimer’s disease (AD), a Phase 2
trial in Major Depressive Disorder (MDD), and a Phase 2 trial in smoking cessation. AXS-07 is being developed for the acute
treatment of migraine. AXS-12 is being developed for the treatment of the symptoms of narcolepsy. The Axsome Pain and Primary Care
business unit (Axsome PPC) houses Axsome’s pain and primary care assets, including AXS-02 and AX-06, and intellectual property
which covers these and related product candidates and molecules being developed by Axsome and others. AXS-02 is being developed for
osteoporosis, the pain of knee osteoarthritis, and chronic low back pain. AXS-06 is being developed for osteoarthritis and
rheumatoid arthritis. AXS-02, AXS-05, AXS-06, AXS-07, AXS-09, and AXS-12 are investigational drug products not approved by the FDA.
For more information, please visit the Company’s website at axsome.com. The Company may occasionally disseminate material,
nonpublic information on the company website.
Forward Looking Statements
Certain matters discussed in this press release are “forward-looking statements”. The Company may, in some
cases, use terms such as “predicts,” “believes,” “potential,” “continue,” “estimates,” “anticipates,” “expects,” “plans,”
“intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to
identify these forward-looking statements. In particular, the Company’s statements regarding trends and potential future results
are examples of such forward-looking statements. The forward-looking statements include risks and uncertainties, including, but not
limited to, the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for its current
product candidates, including statements regarding the timing of initiation, interim analyses and completion of the trials,
futility analyses and receipt of interim results, which are not necessarily indicative of the final results of the Company’s
ongoing clinical trials; the Company’s ability to fund additional clinical trials to continue the advancement of its product
candidates; the timing of and the Company’s ability to obtain and maintain U.S. Food and Drug Administration or other regulatory
authority approval of, or other action with respect to, its product candidates; the Company’s expected cash runway; the Company’s
ability to successfully defend its intellectual property or obtain the necessary licenses at a cost acceptable to the Company, if
at all; the successful implementation of the Company’s research and development programs and collaborations; the success of the
Company’s license agreements; the acceptance by the market of the Company’s product candidates, if approved; and other factors,
including general economic conditions and regulatory developments, not within the Company’s control. The factors discussed herein
could cause actual results and developments to be materially different from those expressed in or implied by such statements. The
forward-looking statements are made only as of the date of this press release and the Company undertakes no obligation to publicly
update such forward-looking statements to reflect subsequent events or circumstance.
Axsome Contact:
Mark Jacobson
Senior Vice President, Operations
Axsome Therapeutics, Inc.
25 Broadway, 9th Floor
New York, NY 10004
Tel: 212-332-3243
Email: mjacobson@axsome.com
www.axsome.com
