Harmony Biosciences Presents Preclinical Data Demonstrating Significant Wake-Promoting and Cataplexy-Suppressing Effects of BP1.15205 in Narcolepsy at SLEEP 2025

Business Wire

a day ago

PLYMOUTH MEETING, Pa.--(BUSINESS WIRE)--Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) today announced the presentation of preclinical pharmacological effect data for BP1.15205, an investigational, highly potent, and potentially best-in-class orexin 2 receptor (OX2R) agonist, which demonstrated significant wake-promoting and cataplexy-suppressing effects in a standard transgenic mouse model of narcolepsy type 1. The data will be presented at the 39 th Annual Meeting of the Associated Professional Sleep Societies (APSS) 'SLEEP' on Wednesday, June 11, 2025, at 10:00 AM PDT in Seattle.
'We are encouraged by the robust preclinical data being presented at SLEEP, highlighting BP1.15205 as a potentially best-in-class OX2R agonist,' said Kumar Budur, MD, MS, Chief Medical and Scientific Officer at Harmony Biosciences. 'BP1.15205 is a new and unique chemical scaffold optimized for high potency that demonstrated statistically significant wake-promoting effects at very low doses administered orally in the standard transgenic mouse model. These findings are supportive of dosing flexibility to potentially treat all three central disorders of hypersomnolence at low doses, which could offer an optimized benefit / risk profile. The 3-month GLP toxicity study in two species revealed no concerning adverse events and supports a favorable safety and tolerability profile.'
Orexin receptor functional studies showed that BP1.15205 is a highly potent, selective OX2R receptor agonist with no off-target effects expected and a >600-fold selectivity over human OX1R. BP1.15205 is orally bioavailable and has the potential for once-daily dosing.
Absorption, distribution, metabolism, and excretion (ADME) properties and preliminary toxicology studies showed that BP1.15205 is a differentiated OX2R agonist drug candidate. In the GLP toxicity study, no adverse events or biochemical changes were observed following a 3-month treatment period at doses up to 300 mg/kg/day, pending histopathology data.
An Investigational Medicinal Product Dossier (IMPD) application with the European Medicines Agency (EMA) is being completed for BP1.15205. A first-in-human study is planned to begin in 2H 2025 with topline data anticipated in 2026. Additionally, an Investigational New Drug (IND) application for BP1.15205 will be filed with the U.S. Food and Drug Administration (FDA).
'We are very excited to advance our potentially best-in-class OX2R program and support the strategic expansion of our sleep-wake franchise. We are dedicated to investigating this potential new solution further with the hope of bringing a novel treatment to market that can help even more people with narcolepsy and other central disorders of hypersomnolence,' Budur added.
The poster entitled, 'BP1.15205, a Novel Orexin-2 Receptor Agonist, Demonstrates Pharmacological Effects in a Mouse Model of Narcolepsy,' will be presented at P-38; Poster Board Number 1; on June 11, 2025, at 10:00 AM PDT.
KEY FINDINGS FROM THE STUDIES INCLUDE:
In Vitro Orexin Receptor Functional Studies:
BP1.15205 is a highly potent agonist at OX2R receptors: EC 50 = 0.015 nM.
BP1.15205 is highly selective for human OX2R receptors: >600-fold selectivity over human OX1R receptors.
Minimal interspecies difference in agonist functional properties was observed between human and mouse orexin-2 receptors.
In Vivo Pharmacology Studies:
Single oral dose administration of BP1.15205 in transgenic mice at the beginning of the 12-hour dark period of a 24-hour light/dark cycle produced significant and dose-dependent increases in total wakefulness time and sleep latency at every dose tested beginning at 0.03 and 0.1 mg/kg, respectfully, as compared to vehicle-treated animals.
Significant and dose-dependent decreases in the total number and duration of cataplexy-like episodes were measured following single dose, oral administration of BP1.15205 beginning at 1mg/kg, as compared to vehicle.
About Narcolepsy
Narcolepsy is a rare, chronic, debilitating neurological disease of sleep-wake state instability that impacts approximately 170,000 Americans and is primarily characterized by excessive daytime sleepiness (EDS) and cataplexy – its two cardinal symptoms – along with other manifestations of REM sleep dysregulation (hallucinations and sleep paralysis), which intrude into wakefulness. EDS is the inability to stay awake and alert during the day and is the symptom that is present in all people living with narcolepsy. In most patients, narcolepsy is caused by the loss of hypocretin/orexin, a neuropeptide in the brain that supports sleep-wake state stability. This disease affects men and women equally, with typical symptom onset in adolescence or young adulthood; however, it can take up to a decade to be properly diagnosed.
About Harmony Biosciences
Harmony Biosciences is a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases who have unmet medical needs. Driven by novel science, visionary thinking, and a commitment to those who feel overlooked, Harmony Biosciences is nurturing a future full of therapeutic possibilities that may enable patients with rare neurological diseases to truly thrive. Established by Paragon Biosciences, LLC, in 2017 and headquartered in Plymouth Meeting, Pa., we believe that when empathy and innovation meet, a better future can begin; a vision evident in the therapeutic innovations we advance, the culture we cultivate, and the community programs we foster. For more information, please visit www.harmonybiosciences.com.
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