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.
About Narcolepsy
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.
About AXS-12
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 website.
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 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
Chief Operating Officer Axsome Therapeutics, Inc. 22 Cortlandt
Street, 16th Floor New York, NY 10007 Tel: 212-332-3243 Email:
mjacobson@axsome.comwww.axsome.com
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