Patients at the Heart win will help fund PWSA’s sleep disorder effort
Award will enable educational program for healthcare workers, caregivers
The Prader-Willi Syndrome Association USA (PWSA) has again been named a winner of the Patients at the Heart awards, an effort by Harmony Biosciences to help fund nonprofits that work for people with sleep disorders and rare neurological diseases.
The award will fund PWS Sleep Within Reach Resources, an educational program for healthcare workers, caregivers, and others who support the needs of those with Prader-Willi syndrome (PWS) about misunderstood sleep disorders, including those associated with the disease.
“We are honored to be recognized with a Patients at the Heart award for our ‘Reach Resources’ program, which provides free, multi-tiered support to individuals living with and affected by PWS,” Stacy Ward, interim CEO of the PWSA, said in a Harmony press release. “This initiative underscores our commitment to fostering collaborative care, involving professionals, caregivers, and educational staff. Through project elements like ‘PWS ECHO Sleep Within Reach’ and ‘ECHO Debrief,’ alongside the development of educational toolkits, we reaffirm our dedication to making a meaningful impact in the lives of those affected by this rare and challenging condition, as well as their loved ones.”
Launched in April, the ECHO project is made up of a series of webinars that focus on PWS-related healthcare topics for healthcare professionals who work with patients. Two webinars have been made available and more are due.
PWSA was selected as a winner of a Patients at the Heart award in 2021 and 2022.
A Geisinger Health Foundation’s program to improve screening and management of sleep disorders in school-aged children also won a Harmony’s 2023 Progress at the Heart awards. The grants support initiatives to address disparities, injustices, and inequities related to rare neurological diseases and sleep disorders.
“Our Patients at the Heart and Progress at the Heart awards exemplify our commitment to empathy and innovation, catalyzing our commitment to these nonprofit organizations who are leading the way in addressing complex challenges posed by sleep and rare neurological disorders,” said Cate McCanless, Harmony’s chief corporate affairs officer. “We are honored to support these initiatives, which collectively hold the potential to make a lasting impact on individuals and communities facing significant unmet needs, while always placing patients at the heart of everything that we do.”
Clinical trials for pitolisant
Harmony is developing pitolisant to treat excessive sleepiness in PWS. The therapy is approved for excessive daytime sleepiness in people with the sleep disorder narcolepsy. It’s marketed as Wakix.
The company plans to launch a placebo-controlled Phase 3 clinical trial before the end of the year to test the therapy in children, adolescents, and adults with PWS. If positive, data will support an application for its approval for the condition.
The study’s design was based on results from a proof-of-concept Phase 2 clinical trial (NCT04257929), which included 65 people with PWS, ages 6 and older, who were treated with pitolisant at one of two doses, or a placebo, for 11 weeks (about three months).
Top-line data indicated using pitolisant led to a clinically meaningful reduction in daytime sleepiness across all doses and age groups. Differences relative to a placebo reached statistical significance for children and adults and were mainly driven by the higher therapy dose.
Earlier this month, the company presented new trial results at the 2023 Foundation for Prader-Willi Research (FPWR) Symposium and Family Conference.
The results, which focused on secondary goals, showed that children and adults given pitolisant generally saw greater reductions in the Caregiver Global Impression of Severity for excessive daytime sleepiness than those on a placebo.
Children given the higher dose also had greater reductions in behavioral abnormalities, including irritability, social withdrawal, hyperactivity/noncompliance, inappropriate speech, and stereotypic or repetitive behaviors, as assessed with the Aberrant Behavior Checklist-2.
While most patients showed no or mild excessive hunger, a hallmark PWS symptom, at the start of the study, reduction trends with pitolisant relative to a placebo were observed, especially among children. This was assessed with the Hyperphagia Questionnaire for Clinical Trials.
The Phase 2 study wasn’t powered to detect statistically significant differences between the groups. According to Harmony, the data were also somewhat skewed because one teen in the placebo group had unexplained but dramatic improvements. This resulted in a bigger than expected placebo effect in the overall population.
The findings do support pitolisant’s benefits in PWS, however.
After completing the 11-week treatment, most participants entered the study’s open-label extension phase, where they will receive pitolisant for several years.
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