NEW YORK, NY — Axsome Therapeutics, Inc., 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.
Contacts
Investors
Mark Jacobson – Chief Operating Officer
Tel: 212-332-3243
Email: [email protected]
Media
Darren Opland
Director, Corporate Communications
Tel: 929-837-1065