Axsome Therapeutics Receives FDA Breakthrough Therapy Designation for AXS-12 for the Treatment of Narcolepsy Designation offers potential for expedited development and review FDA Orphan Drug Designation previously grante

Axsome Therapeutics Receives FDA Breakthrough

Therapy Designation for AXS-12 for the Treatment of Narcolepsy

Designation offers potential for expedited

development and review

FDA Orphan Drug Designation previously

granted to Axsome for AXS-12 in narcolepsy

Third FDA Breakthrough Therapy designation

NEW YORK, August 5, 2020 (Globe Newswire)

- Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company developing novel therapies for the management

of central nervous system (CNS) disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough

Therapy designation for AXS-12 for the treatment of cataplexy in patients with narcolepsy. AXS-12 is a novel, oral, highly selective

and potent norepinephrine reuptake inhibitor. Axsome previously received Orphan Drug Designation from the FDA for AXS-12 for the

treatment of narcolepsy. Narcolepsy is a debilitating, neurological condition characterized by excessive daytime sleepiness (EDS)

and cataplexy, a sudden loss of muscle tone triggered by strong emotions.

A Breakthrough Therapy designation is granted

to potentially expedite development and review timelines for a promising investigational medicine when preliminary clinical evidence

indicates it may demonstrate substantial improvement on one or more clinically significant endpoints over available therapies for

a serious or life-threatening condition. The Breakthrough Therapy designation for AXS-12 for the treatment of cataplexy in narcolepsy

was supported by the positive results from the Phase 2 CONCERT study, a randomized, double-blind, placebo-controlled, crossover,

multicenter U.S. trial. In the trial, 21 patients with a diagnosis of narcolepsy with cataplexy were treated for 2 weeks with AXS-12

or with placebo, followed by a crossover to the other treatment after a 1-week down-titration and washout period. AXS-12 met the

primary endpoint demonstrating a highly statistically significant reduction from baseline in the mean weekly number of cataplexy

attacks, averaged for the 2-week treatment period (overall treatment effect), as compared to placebo (p<0.001), and at the end

of the 2-week treatment period (p=0.002). AXS-12 also significantly improved EDS compared to placebo, as measured by the Epworth

Sleepiness Scale (ESS) and by the frequency of inadvertent naps (p=0.003 and p<0.001, respectively). In addition, AXS-12 significantly

improved cognitive function compared to placebo over the 2-week treatment period as measured by the Ability to Concentrate item

of the Narcolepsy Symptom Assessment Questionnaire (p<0.001). AXS-12 was well tolerated in this trial with the most commonly

reported adverse events being anxiety, constipation, and insomnia.

"This FDA Breakthrough Therapy designation

for AXS-12 for cataplexy in narcolepsy highlights its potential, should it be successfully developed, to provide meaningful benefit

and substantial improvement over currently available treatment options for patients living with this debilitating condition. We

are excited by the burgeoning profile of AXS-12 which has also received Orphan Drug designation for the treatment of narcolepsy,"

said Herriot Tabuteau, MD, Chief Executive Officer of Axsome. "Axsome has now received three FDA Breakthrough Therapy designations,

including designations for AXS-05 in major depressive disorder and for AXS-05 in Alzheimer's disease agitation. These designations

exemplify our commitment to developing potentially life-changing medicines for patients with difficult-to-treat CNS conditions,

our innovative approach to clinical development, and our resulting differentiated and broad late-stage CNS pipeline. We look forward

to meeting with the FDA as soon as possible to discuss the continued development of AXS-12 in light of this significant milestone."

About FDA Breakthrough Therapy Designation

Breakthrough Therapy designation is

granted by the FDA in order to expedite the development and review of drugs for serious or life-threatening conditions. In

order to receive Breakthrough Therapy designation, a drug must demonstrate preliminary clinical evidence that the drug may

have substantial improvement on at least one clinically significant endpoint over available therapy. Breakthrough Therapy

designation provides an organizational commitment involving senior managers from the FDA, more intensive FDA guidance on an

efficient drug development program, and greater access to and more frequent communication with the FDA throughout the entire

drug development and review process. It also provides the opportunity to submit sections of a New Drug Application (NDA) on a

rolling basis, where the FDA may review portions of the NDA as they are received instead of waiting for the entire NDA

submission. In addition, Breakthrough Therapy designated products are eligible for Priority Review, where the FDA has a goal

to take action on an application within six months, as opposed to ten months under standard review. Breakthrough Therapy

designation does not change the standards for approval.

Narcolepsy can be 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.

AXS-12 (reboxetine) is a highly selective

and potent norepinephrine reuptake inhibitor for the treatment of narcolepsy. AXS-12 modulates noradrenergic activity to promote

wakefulness, maintain muscle tone and enhance cognition. AXS-12 has been granted U.S. Food and Drug Administration (FDA) Breakthrough

Therapy designation and Orphan Drug Designation for the treatment of narcolepsy. AXS-12 is an investigational drug product not

approved by the FDA.

About Axsome Therapeutics, Inc.

Axsome Therapeutics, Inc. is a biopharmaceutical

company developing novel therapies for the management of central nervous system (CNS) disorders for which there are limited treatment

options. For the many people facing unsatisfactory treatments for CNS disorders, Axsome accelerates the invention and adoption

of life-changing medicines. Axsome's core CNS product candidate portfolio includes five clinical-stage candidates, AXS-05,

AXS-07, AXS-09, AXS-12, and AXS-14. AXS-05 is being developed for major depressive disorder (MDD), treatment resistant depression

(TRD), Alzheimer's disease (AD) agitation, and as treatment for smoking cessation. AXS-07 is being developed for the acute

treatment of migraine. AXS-12 is being developed for the treatment of narcolepsy. AXS-14 is being developed for fibromyalgia. AXS-05,

AXS-07, AXS-09, AXS-12, and AXS-14 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

Forward Looking Statements

Certain matters discussed in this

press release are "forward-looking statements". We 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 our ongoing clinical trials and anticipated

clinical trials for our current product candidates, including statements regarding the timing of initiation, pace of

enrollment and completion of the trials (including our ability to fully fund our disclosed clinical trials, which assumes no

material changes to our currently projected expenses), futility analyses and receipt of interim results, which are not

necessarily indicative of the final results of our ongoing clinical trials, and the number or type of studies or nature of

results necessary to support the filing of a new drug application ("NDA") for any of our current product

candidates; our ability to fund additional clinical trials to continue the advancement of our product candidates; the timing

of and our ability to obtain and maintain U.S. Food and Drug Administration ("FDA") or other regulatory authority

approval of, or other action with respect to, our product candidates (including, but not limited to, FDA's agreement

with the Company's discontinuation of the bupropion treatment arm of the ADVANCE-1 study in accordance with the

independent data monitoring committee's recommendations); the potential for the MOMENTUM clinical trial to provide a

basis for approval of AXS-07 for the acute treatment of migraine in adults with or without aura, pursuant to our special

protocol assessment; the potential for the ASCEND clinical trial, combined with the GEMINI clinical trial results, to provide

a basis for approval of AXS-05 for the treatment of major depressive disorder and accelerate its development timeline and

commercial path to patients; 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; the Company's anticipated capital requirements,

including the Company's anticipated cash runway; unforeseen circumstances or other disruptions to normal business

operations arising from or related to COVID-19; 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