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-05 for the treatment of Alzheimer’s disease
(AD) agitation. AXS-05 is a novel, oral, investigational NMDA
receptor antagonist with multimodal activity. This is the second
Breakthrough Therapy designation granted to Axsome for AXS-05.
There is currently no approved treatment for AD agitation.
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-05 in AD agitation was
supported by the recent positive results from the pivotal Phase 2/3
ADVANCE-1 study, a randomized, double-blind, controlled,
multicenter U.S. trial in which 366 Alzheimer’s disease patients
were treated with AXS-05, bupropion, or placebo. In this trial,
treatment with AXS-05 resulted in a rapid, substantial, and
statistically significant improvement in agitation as compared to
placebo. On the primary endpoint, AXS-05 demonstrated a
statistically significant mean reduction from baseline in the Cohen
Mansfield Agitation Inventory (CMAI) total score compared to
placebo at Week 5, with mean reductions of 15.4 points for AXS-05
and 11.5 points for placebo (p=0.010). AXS-05 was also superior to
bupropion on the CMAI total score (p<0.001), establishing
component contribution. AXS-05 was well tolerated and not
associated with cognitive impairment or sedation. The most commonly
reported adverse events in the AXS-05 arm were somnolence (8.2% for
AXS-05 versus 4.1% for bupropion and 3.2% for placebo), dizziness
(6.3%, 10.2%, 3.2%, respectively), and diarrhea (4.4%, 6.1%, 4.4%,
respectively).
“This FDA Breakthrough Therapy designation is an
important milestone in the development of AXS-05 for Alzheimer’s
disease agitation, a serious, prevalent, and debilitating condition
for which there is currently no approved therapy,” said Herriot
Tabuteau, MD, Chief Executive Officer of Axsome. “This marks the
second Breakthrough Therapy designation received by Axsome for
AXS-05, the first being for the treatment of major depressive
disorder, and highlights the potential of AXS-05 to address unmet
medical needs in multiple difficult-to-treat CNS disorders. We look
forward to working with the FDA over the coming months as we
advance the development of AXS-05 for the treatment of Alzheimer’s
disease agitation.”
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 Alzheimer’s Disease (AD)
Agitation
Alzheimer’s disease (AD) is a progressive
neurodegenerative disorder characterized by cognitive decline, and
behavioral and psychological symptoms including agitation. AD is
the most common form of dementia and afflicts an estimated 6
million individuals in the United States, a number that is
anticipated to increase to approximately 14 million by 2050 [1].
Agitation is reported in up to 70% of patients with AD and is
characterized by emotional distress, aggressive behaviors,
disruptive irritability, and disinhibition [2]. Agitation in
patients with AD has been associated with increased caregiver
burden, decreased functioning, accelerated cognitive decline,
earlier nursing home placement, and increased mortality [2-4].
There are currently no therapies approved by the FDA for the
treatment of agitation in patients with AD.
About AXS-05
AXS-05 is a novel, oral, patent-protected,
investigational NMDA receptor antagonist with multimodal activity
under development for the treatment of Alzheimer’s disease
agitation, major depressive disorder, and other central nervous
system (CNS) disorders. AXS-05 consists of a proprietary
formulation and dose of dextromethorphan and bupropion and utilizes
Axsome’s metabolic inhibition technology. The dextromethorphan
component of AXS-05 is a non-competitive N-methyl-D-aspartate
(NMDA) receptor antagonist, also known as a glutamate receptor
modulator, a sigma-1 receptor agonist, an inhibitor of the
serotonin and norepinephrine transporters, a nicotinic
acetylcholine receptor antagonist, and an inhibitor of microglial
activation. The bupropion component of AXS-05 serves to increase
the bioavailability of dextromethorphan, and is a norepinephrine
and dopamine reuptake inhibitor, and a nicotinic acetylcholine
receptor antagonist. AXS-05 is covered by more than 42 issued U.S.
and international patents which provide protection out to 2034.
AXS-05 has been granted U.S. Food and Drug Administration
Breakthrough Therapy designation for major depressive disorder,
Fast Track designation for treatment resistant depression, and
Breakthrough Therapy and Fast Track designations for Alzheimer’s
disease agitation. AXS-05 is 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. 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.
References
- Alzheimer’s Association. 2020
Alzheimer’s Disease Facts and Figures. Alzheimers Dement
2020;16(3):391+.
- Tractenberg RE, Weiner MF,
Thal LJ. Estimating the prevalence of agitation in
community-dwelling persons with Alzheimer’s disease. J
Neuropsychiatry Clin Neurosci. 2002;14:11-18.
- Porsteinsson AP, Antonsdottir IM.
An update on the advancements in the treatment of agitation in
Alzheimer’s disease. Expert Opin Pharmacother.
2017;18:611-620.
- Rabins PV, Schwartz S, Black BS,
Corcoran C, Fauth E, Mielke M, Christensen J, Lyketsos C, Tschanz
J. Predictors of progression to severe Alzheimer's disease in an
incidence sample. Alzheimers Dement. 2013;9:204-207.
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. 200 Broadway, 3rd Floor New York,
NY 10038 Tel: 212-332-3243 Email: mjacobson@axsome.com
www.axsome.com
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