Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical
company developing and delivering novel therapies for the
management of central nervous system (CNS) disorders, today
announced topline results from the CRESCENDO (Characterizing
Patient Perspectives on Unmet Needs in Narcolepsy) survey of
patients with narcolepsy type 1 (NT1, i.e., narcolepsy with
cataplexy) receiving treatment, demonstrating high rates of
persistent symptoms and significant patient burden, despite being
on current treatments. CRESCENDO was conducted in partnership with
Narcolepsy Network, a national non-profit patient support
organization for people with narcolepsy, idiopathic hypersomnia,
and related sleep disorders.
The CRESCENDO survey included 203 adult patients
diagnosed with NT1, who collectively have more than 2600 years of
lived experience with the sleep disorder, to understand their
experience and journey, to measure disease symptoms including
cognitive impairment while on treatment, and to assess burden of
illness, comorbidities, and unmet needs and challenges patients
face. To quantify key elements of the narcolepsy patient
experience, CRESCENDO utilized patient reported measures, and
validated scales including the Epworth Sleepiness Scale (ESS) to
assess excessive daytime sleepiness (EDS), and the British Columbia
Cognitive Complaints Inventory (BC-CCI) to assess cognitive
function.
All patients taking part in the survey were
currently undergoing treatment for NT1. The most common treatments
were wake promoting agents (about 53% of surveyed patients),
oxybates (47%), and stimulants (42%).
“Narcolepsy is a debilitating and incurable
orphan neurologic disorder. Its most troublesome symptom is daytime
sleepiness, which can impair social and occupational functioning
and can cause potentially dangerous sleep attacks. Its other
symptoms include cataplexy, episodes in which strong emotion causes
a sudden loss of muscle control, sleep paralysis, sleep-related
hallucinations, and disrupted nighttime sleep,” said Dr. Karl
Doghramji, Professor of Psychiatry, Neurology and Medicine, and
Medical Director of the Jefferson Sleep Disorders Center at Thomas
Jefferson University in Philadelphia, PA. “Unfortunately,
narcolepsy is underrecognized and underdiagnosed by the medical
community and its burden of illness is not widely understood or
appreciated, which is why the CRESCENDO survey is such an important
step forward in raising awareness of narcolepsy and its impact on
patient lives, and in giving voice to the thousands of individuals
living with this debilitating disorder.”
Despite receiving treatment, the majority of
narcolepsy patients continued to experience symptoms. Cataplexy was
reported by 77% of patients while on their current treatment
regimen. EDS, assessed using the ESS (scores > 10), was observed
in 64% of patients despite receiving current treatments. Cognitive
impairment, assessed using the BC-CCI (scores ≥5), was observed in
74% of patients. Depression and anxiety were experienced by about
45% and 57% of patients respectively.
Axsome plans to present the detailed results of the CRESCENDO
survey at upcoming scientific meetings.
Key Topline Findings from the CRESCENDO
Survey
Patient Demographics
- Survey
respondents consisted of a total of 203 adults, aged 18-82 years
(mean 41 years) with a diagnosis of narcolepsy Type 1, all of whom
had experience with FDA-approved treatments for their
narcolepsy.
Current Treatments and Comorbidities
-
Subjects were receiving pharmacotherapy for narcolepsy.
- The
most common narcolepsy-specific treatments were wake promoting
agents (53% of surveyed patients), oxybates (47%), and stimulants
(42%).
- 37% of
participants were diagnosed with depression, of which 80% were
taking medication to manage their depression.
- 37% of
participants were diagnosed with anxiety, of which 72% were taking
medication to manage their anxiety.
Prevalence of Narcolepsy Symptoms While on
Treatment
-
Cataplexy was reported by 77% of patients while on their current
treatment regimen.
- EDS,
assessed using the ESS (scores > 10), was observed in 64% of
patients despite receiving current treatments.
-
Cognitive impairment, assessed using the BC-CCI (scores ≥5), was
observed in 74% of patients on treatment.
- 68%
rated their ability to concentrate as poor, very poor, or
average.
Cataplexy Impacts on Daily Life
- 65% of
participants currently experiencing cataplexy reported that
cataplexy burdens their professional life (e.g. career, school),
despite treatment
- 60% of
participants currently experiencing cataplexy reported that
cataplexy burdens their social life, despite treatment.
- 50% of
patients currently experiencing cataplexy reported that cataplexy
burdens their day-to-day life, despite treatment
- 64%
and 68% of patients experiencing cataplexy reported being
embarrassed by falling down and slurred speech, respectively,
despite treatment.
Time of Conduct
- The
survey was conducted between October-December 2023.
About Narcolepsy
Narcolepsy is a serious and debilitating orphan
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. A majority of narcolepsy patients
report cognitive impairment related to their condition. 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.
About Narcolepsy Network
Narcolepsy Network is a 501(c)(3), member-led
community organization based in the United States that works to
educate, empower, and connect people impacted by narcolepsy,
idiopathic hypersomnia and related sleep disorders. Through the
lens of advocacy, education, awareness, and support, Narcolepsy
Network delivers programs and resources by sharing clinical updates
and research, hosting educational webinars, facilitating support
groups, and providing advocacy opportunities. Narcolepsy Network
works with industry to ensure a neutral and unbiased approach to
engage with the patient community. - Neutrality Statement
(narcolepsynetwork.org)
About Axsome Therapeutics, Inc.
Axsome Therapeutics, Inc. is a biopharmaceutical
company developing and delivering novel therapies for central
nervous system (CNS) conditions that have limited treatment
options. Through development of therapeutic options with novel
mechanisms of action, we are transforming the approach to treating
CNS conditions. At Axsome, we are committed to developing products
that meaningfully improve the lives of patients and provide new
therapeutic options for physicians. 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
continued commercial success of our Sunosi® and Auvelity® products
and the success of our efforts to obtain any additional
indication(s) with respect to solriamfetol and/or AXS-05; 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 revenues or expenses), futility
analyses and receipt of interim results, which are not necessarily
indicative of the final results of our ongoing clinical trials,
and/or data readouts, 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 statements
regarding the timing of any NDA submission; whether issues
identified by FDA in the complete response letter may impact the
potential approvability of the Company’s NDA for AXS-07 for the
acute treatment of migraine in adults with or without aura,
pursuant to our special protocol assessment for the MOMENTUM
clinical trial; 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 products and product
candidates, if approved; the Company’s anticipated capital
requirements, including the amount of capital required for the
continued commercialization of Sunosi and Auvelity and for the
Company’s commercial launch of its other product candidates, if
approved, and the potential impact on the Company’s anticipated
cash runway; unforeseen circumstances or other disruptions to
normal business operations arising from or related to geo-political
conflicts or a global pandemic 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 Contacts:
Investors:Mark JacobsonChief Operating OfficerAxsome
Therapeutics, Inc.One World Trade Center, 22nd FloorNew York, NY
10007Tel: 212-332-3243Email: mjacobson@axsome.com
www.axsome.com
Media:Darren OplandDirector, Corporate CommunicationsAxsome
Therapeutics, Inc.One World Trade Center, 22nd FloorNew York, NY
10007Tel: 929-837-1065Email: dopland@axsome.com www.axsome.com
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