Summary: At its Investor Day on October 1, Harmony Biosciences presented a pipeline update, highlighting progress in its sleep disorder treatments. New data from the orexin-2 agonist BP1.15205 showed potential best-in-class potency, while long-term extension data for pitolisant in idiopathic hypersomnia demonstrated sustained efficacy, improving patients’ Epworth Sleepiness Scale scores by approximately 9 points. Notably, preliminary results revealed that pitolisant is safe at doses up to five times the current labeled amount, positioning the company for the development of pitolisant-HD.
Three Key Takeaways:
- Orexin-2 Agonist Shows High Potency: Harmony’s orexin-2 agonist, BP1.15205, demonstrated greater potency compared to other publicly known orexin-2 agonists, with the potential to be best-in-class.
- Pitolisant Efficacy in Idiopathic Hypersomnia: Long-term data showed that pitolisant significantly improves wakefulness in patients with idiopathic hypersomnia, with benefits persisting for over a year.
- Safety of Pitolisant at High Doses: Preliminary data confirmed pitolisant’s safety at doses up to five times the highest labeled dose, establishing a safety margin for the development of pitolisant-HD.
Harmony Biosciences Holdings Inc provided a pipeline update at its Investor Day event on Oct 1, showcasing new data from its orexin-2 receptor agonist, next-generation pitolisant programs, and long-term extension data from its pitolisant program in idiopathic hypersomnia.
“Harmony Biosciences has transformed into an innovative, catalyst-rich, self-funding biotech company focused on patient impact and long-term value creation,” says Jeffrey M. Dayno, MD, president and chief executive officer of Harmony Biosciences, in a release. “We strategically expanded and advanced our pipeline, with near-term catalysts positioned to deliver one or more new product or indication launches each year over the next five years, positioning Harmony to generate over $3 billion in potential annual revenue.”
The event included presentations from Harmony’s executive leadership team and experts in the field of sleep disorders.
Key Highlights from Harmony’s Sleep/Wake Portfolio:
- Orexin-2 agonist program: New data show BP1.15205 (formerly TPM-1116) is based on a novel chemical scaffold and has demonstrated greater potency compared to all publicly disclosed data on orexin-2 agonists, with the potential to be best-in-class.
- Pitolisant in Idiopathic Hypersomnia: New data show robust and sustained efficacy of pitolisant in patients with idiopathic hypersomnia in the long-term extension study; mean improvement in Epworth Sleepiness Scale was ~9 points from baseline out beyond one year, with the majority of patients achieving normal levels of wakefulness. The company is on track to submit a supplemental new drug application in Q4 2024.
- Pitolisant-HD program: Preliminary data confirm safety up to five times the highest labeled dose of WAKIX, establishing the safety margins for the pitolisant-HD development program. Market research indicates healthcare professionals view HD as a superior product profile and anticipate high uptake of pitolisant-HD for all patients, including patients on WAKIX. Payers also see value in the HD profile and anticipate broad access to pitolisant-HD both pre- and post-WAKIX loss of exclusivity. Harmony is aiming for a Prescription Drug User Fee Act target date in 2028.
Additionally, the company also provided program updates from its acquisitions in Fragile X syndrome and rare epilepsies, which have strengthened its late-stage development pipeline.
The replay webcast and presentation slides from the event will be available on Harmony Biosciences’ investor relations website.
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