HARMONY BIOSCIENCES HIGHLIGHTS NEW DATA, ROBUST LATE-STAGE PIPELINE WITH NEAR-TERM VALUE CREATION OPPORTUNITIES AND ITS BOLD NEW VISION AT INVESTOR DAY
Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) presented a comprehensive pipeline update at its Investor Day event, showcasing new data and program updates. Key highlights include:
1. Orexin-2 agonist program: BP1.15205 demonstrated greater potency compared to other orexin-2 agonists.
2. Pitolisant in Idiopathic Hypersomnia: Long-Term Extension study showed sustained efficacy.
3. Pitolisant-HD program: Preliminary data confirmed safety up to five times the highest labeled dose of WAKIX.
4. Fragile X syndrome program: Phase 3 RECONNECT study on track for topline data in mid-2025.
5. EPX-100 development program: Favorable risk/benefit profile for Dravet syndrome and Lennox-Gastaut syndrome.
Harmony aims to deliver one or more new product or indication launches each year over the next five years, potentially generating over $3 billion in annual revenue.
Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) ha presentato un aggiornamento completo del suo pipeline durante l'Investor Day, mostrando nuovi dati e aggiornamenti sui programmi. I punti salienti includono:
1. Programma dell'agonista Orexin-2: BP1.15205 ha dimostrato una maggiore potenza rispetto ad altri agonisti dell'orexina-2.
2. Pitolisant nell'ipersonnia idiopatica: Lo studio di estensione a lungo termine ha mostrato un'efficacia sostenuta.
3. Programma Pitolisant-HD: Dati preliminari hanno confermato la sicurezza fino a cinque volte la dose più alta etichettata di WAKIX.
4. Programma per la sindrome dell'X fragile: Lo studio di Fase 3 RECONNECT è in programma per i dati principali a metà del 2025.
5. Programma di sviluppo EPX-100: Profilo rischio/beneficio favorevole per la sindrome di Dravet e la sindrome di Lennox-Gastaut.
Harmony mira a lanciare uno o più nuovi prodotti o indicazioni ogni anno nei prossimi cinque anni, con la possibilità di generare oltre 3 miliardi di dollari di entrate annuali.
Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) presentó una actualización integral de su pipeline en su evento Investor Day, mostrando nuevos datos y actualizaciones de programas. Los aspectos destacados incluyen:
1. Programa de agonista Orexin-2: BP1.15205 demostró una mayor potencia en comparación con otros agonistas de orexina-2.
2. Pitolisant en hipersomnia idiopática: El estudio de extensión a largo plazo mostró eficacia sostenida.
3. Programa Pitolisant-HD: Datos preliminares confirmaron seguridad hasta cinco veces la dosis más alta etiquetada de WAKIX.
4. Programa de síndrome del X frágil: El estudio de Fase 3 RECONNECT está en camino para datos principales a mediados de 2025.
5. Programa de desarrollo EPX-100: Perfil de riesgo/beneficio favorable para la síndrome de Dravet y la síndrome de Lennox-Gastaut.
Harmony tiene como objetivo lanzar uno o más nuevos productos o indicaciones cada año durante los próximos cinco años, lo que podría generar más de 3 mil millones de dólares en ingresos anuales.
하모니 바이오사이언스 홀딩스, Inc. (Nasdaq: HRMY)는 투자자 날 행사에서 포괄적인 파이프라인 업데이트를 발표하며 새로운 데이터와 프로그램 업데이트를 선보였습니다. 주요 하이라이트는 다음과 같습니다:
1. 오렉신-2 작용제 프로그램: BP1.15205는 다른 오렉신-2 작용제에 비해 더 큰 효능을 입증했습니다.
2. 아이디오패틱 하이퍼소미아에서의 피톨리상: 장기 연장 연구는 지속적인 효능을 보여주었습니다.
3. 피톨리상-HD 프로그램: 초기 데이터는 WAKIX의 가장 높은 라벨 용량의 최대 5배까지 안전성을 확인했습니다.
4. 취약 X 증후군 프로그램: 3상 RECONNECT 연구가 2025년 중반에 주요 데이터를 목표로 진행 중입니다.
5. EPX-100 개발 프로그램: 드라베 증후군과 레녹스-가스타 증후군에 대한 호의적인 위험/이익 프로필.
하모니는 향후 5년 동안 매년 하나 이상의 새로운 제품 또는 적응증을 출시해 30억 달러 이상의 연간 수익을 창출하는 것을 목표로 하고 있습니다.
Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) a présenté une mise à jour complète de son pipeline lors de son événement Investor Day, mettant en avant de nouvelles données et actualisations de programmes. Les points clés incluent :
1. Programme de l'agoniste Orexin-2 : BP1.15205 a montré une plus grande puissance par rapport à d'autres agonistes de l'orexine-2.
2. Pitolisant dans l'hypersomnie idiopathique : L'étude d'extension à long terme a montré une efficacité soutenue.
3. Programme Pitolisant-HD : Les données préliminaires ont confirmé la sécurité jusqu'à cinq fois la dose la plus élevée étiquetée de WAKIX.
4. Programme pour le syndrome de l'X fragile : L'étude de Phase 3 RECONNECT est en bonne voie pour des données principales à la mi-2025.
5. Programme de développement EPX-100 : Profil risque/bénéfice favorable pour le syndrome de Dravet et le syndrome de Lennox-Gastaut.
Harmony vise à lancer un ou plusieurs nouveaux produits ou indications chaque année au cours des cinq prochaines années, avec le potentiel de générer plus de 3 milliards de dollars de revenus annuels.
Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) präsentierte ein umfassendes Pipeline-Update bei seiner Investor Day-Veranstaltung und stellte neue Daten sowie Programmaktualisierungen vor. Die wichtigsten Highlights umfassen:
1. Orexin-2-Agonisten-Programm: BP1.15205 zeigte eine höhere Potenz im Vergleich zu anderen Orexin-2-Agonisten.
2. Pitolisant bei idiopathischer Hypersomnie: Die Langzeitverlängerungsstudie zeigte eine anhaltende Wirksamkeit.
3. Pitolisant-HD-Programm: Vorläufige Daten bestätigten die Sicherheit bis zum Fünffachen der höchsten ausgewiesenen Dosis von WAKIX.
4. Programm für das fragile X-Syndrom: Die Phase-3-RECONNECT-Studie ist auf Kurs für die Hauptdaten Mitte 2025.
5. EPX-100-Entwicklungsprogramm: Günstiges Risiko-Nutzen-Profil für das Dravet-Syndrom und das Lennox-Gastaut-Syndrom.
Harmony strebt an, in den nächsten fünf Jahren jedes Jahr ein oder mehrere neue Produkte oder Indikationen einzuführen, was potenziell über 3 Milliarden Dollar an jährlichen Einnahmen generieren könnte.
- BP1.15205 demonstrated greater potency compared to all publicly disclosed orexin-2 agonists
- Pitolisant showed sustained efficacy in idiopathic hypersomnia patients in Long-Term Extension study
- Pitolisant-HD program established safety margins up to five times the highest labeled dose of WAKIX
- Market research indicates high anticipated uptake of pitolisant-HD by healthcare professionals
- Phase 3 RECONNECT study for Fragile X syndrome on track for topline data in mid-2025
- EPX-100 showed favorable risk/benefit profile for Dravet syndrome and Lennox-Gastaut syndrome
- Company aims to deliver one or more new product or indication launches annually for the next five years
- Potential to generate over $3 billion in annual revenue
- None.
Insights
Harmony Biosciences has unveiled a robust pipeline with significant near-term catalysts. The company's orexin-2 agonist, BP1.15205, shows promise as a potential best-in-class treatment, demonstrating greater potency than competitors. This could lead to a strong market position in sleep disorders.
The long-term extension data for pitolisant in idiopathic hypersomnia is particularly impressive, with sustained efficacy and substantial improvements in wakefulness. This bodes well for potential label expansion and market growth.
The pitolisant-HD program's preliminary safety data and positive market research suggest a strong potential for cannibalization of WAKIX and expansion of the patient base. This could drive revenue growth both pre- and post-loss of exclusivity.
With multiple late-stage assets targeting rare diseases like Fragile X syndrome and epilepsies, Harmony is diversifying its portfolio and reducing risk. The company's projection of
Harmony's pipeline shows promise across several neurological indications. The orexin-2 agonist program could represent a significant advancement in sleep-wake disorders if its potency translates to clinical efficacy.
The long-term data for pitolisant in idiopathic hypersomnia is particularly noteworthy. A sustained 9-point improvement on the Epworth Sleepiness Scale over a year is clinically meaningful and could significantly impact patients' quality of life.
In Fragile X syndrome, the
The EPX-100 program for Dravet and Lennox-Gastaut syndromes is also promising, with a potentially favorable safety profile compared to existing treatments. These rare epilepsies have significant unmet needs and a new, well-tolerated option could be transformative for patients.
"Harmony Biosciences has transformed into an innovative, catalyst-rich, self-funding biotech company focused on patient impact and long-term value creation," said Jeffrey M. Dayno, M.D.,
The event will feature presentations from Harmony's executive leadership team and experts in the fields of sleep disorders, Fragile X syndrome and developmental epileptic encephalopathies, highlighting Harmony's expansion and diversification of its pipeline, continued commitment to innovation, and focus on driving value for patients and shareholders.
Key Highlights:
- 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 (ESS) was ~9 points from baseline out beyond one year, with the majority of patients achieving normal levels of wakefulness.
- 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 LOE.
- Fragile X syndrome program (ZYN-002): A prespecified ad hoc analysis from the Phase 2 CONNECT-FX in Fragile X syndrome study (published in 2022) showed that patients with complete methylation of FMR1 gene showed a
40% median improvement in socially avoidant behaviors (p=0.027) on the Aberrant Behavior Checklist-Community FXS Specific (ABC-CFXS) Social Avoidance subscale; this and other learnings from this trial have informed the design and selection of the primary endpoint for the Phase 3 RECONNECT study, which is on track for topline data in mid-2025. - EPX-100 development program: New safety and tolerability data on EPX-100, a potent, oral, centrally acting serotonin (5HT2) agonist currently in a pivotal registrational trial (ARGUS) for Dravet syndrome (DS), shows a favorable risk/benefit profile for EPX-100 compared to select approved drugs for DS and Lennox-Gastaut syndrome (LGS); topline data in DS is expected in 2026, and a pivotal Phase 3 trial for LGS is on track to initiate later this year.
The replay webcast and presentation slides from this event will be available on Harmony Biosciences' investor relations at https://ir.harmonybiosciences.com/.
About WAKIX® (pitolisant) Tablets
WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy and for the treatment of EDS in pediatric patients 6 years of age and older with narcolepsy. It was granted orphan drug designation for the treatment of narcolepsy in 2010, and breakthrough therapy designation for the treatment of cataplexy in 2018. WAKIX is a selective histamine 3 (H₃) receptor antagonist/inverse agonist. The mechanism of action of WAKIX is unclear; however, its efficacy could be mediated through its activity at H₃ receptors, thereby increasing the synthesis and release of histamine, a wake promoting neurotransmitter. WAKIX was designed and developed by Bioprojet (
INDICATIONS AND USAGE
WAKIX is indicated for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy and for the treatment of excessive daytime sleepiness (EDS) in pediatric patients 6 years of age and older with narcolepsy.
IMPORTANT SAFETY INFORMATION
Contraindications
WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation. Anaphylaxis has been reported. WAKIX is also contraindicated in patients with severe hepatic impairment.
Warnings and Precautions
WAKIX prolongs the QT interval. Avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval.
The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment. WAKIX is contraindicated in patients with severe hepatic impairment and not recommended in patients with end-stage renal disease (ESRD).
Adverse Reactions
In the placebo-controlled clinical trials conducted in adult patients with narcolepsy with or without cataplexy, the most common adverse reactions (≥
In the placebo-controlled phase of the clinical trial conducted in pediatric patients 6 years and older with narcolepsy with or without cataplexy, the most common adverse reactions (≥
Drug Interactions
Concomitant administration of WAKIX with strong CYP2D6 inhibitors increases pitolisant exposure by 2.2-fold. Reduce the dose of WAKIX by half.
Concomitant use of WAKIX with strong CYP3A4 inducers decreases exposure of pitolisant by
H1 receptor antagonists that cross the blood-brain barrier may reduce the effectiveness of WAKIX. Patients should avoid centrally acting H1 receptor antagonists.
WAKIX is a borderline/weak inducer of CYP3A4. WAKIX may reduce the effectiveness of sensitive CYP3A4 substrates, including hormonal contraceptives. Patients using hormonal contraception should be advised to use an alternative non-hormonal contraceptive method during treatment with WAKIX and for at least 21 days after discontinuing treatment.
Use in Specific Populations
There is a pregnancy exposure registry that monitors pregnancy outcomes in women who are exposed to WAKIX during pregnancy. Patients should be encouraged to enroll in the WAKIX pregnancy registry if they become pregnant. To enroll or obtain information from the registry, patients can call 1-800-833-7460.
The safety and effectiveness of WAKIX have not been established for treatment of excessive daytime sleepiness in pediatric patients less than 6 years of age with narcolepsy.
The safety and effectiveness of WAKIX have not been established for treatment of cataplexy in pediatric patients with narcolepsy.
WAKIX is extensively metabolized by the liver. WAKIX is contraindicated in patients with severe hepatic impairment. Dosage adjustment is recommended in patients with moderate hepatic impairment.
WAKIX is not recommended in patients with end-stage renal disease. Dosage adjustment of WAKIX is recommended in patients with eGFR <60 mL/minute/1.73 m2.
Dosage reduction is recommended in patients known to be poor CYP2D6 metabolizers; these patients have higher concentrations of WAKIX than normal CYP2D6 metabolizers.
Please see the Full Prescribing Information for WAKIX for more information.
To report suspected adverse reactions, contact Harmony Biosciences at 1-800-833-7460 or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
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 Idiopathic Hypersomnia
Idiopathic Hypersomnia (IH) is a rare and chronic neurological disease that is characterized by excessive daytime sleepiness (EDS) despite sufficient or even long sleep time. EDS in IH cannot be alleviated by naps, longer sleep or more efficient sleep. People living with IH experience significant EDS along with the symptoms of sleep inertia (prolonged difficulty waking up from sleep) and 'brain fog' (impaired cognition, attention, and alertness). The cause of IH is unknown, but it is likely due to alterations in areas of the brain that stabilize states of sleep and wakefulness. IH is one of the central disorders of hypersomnolence and, like narcolepsy, is a debilitating sleep disorder that can result in significant disruption in daily functioning.
About ZYN-002
ZYN-002 is the first-and-only pharmaceutically manufactured synthetic cannabidiol devoid of THC and formulated as a patent-protected permeation-enhanced gel for transdermal delivery through the skin and into the circulatory system. The product is manufactured through a synthetic process in a cGMP facility and is not extracted from the cannabis plant. ZYN-002 does not contain THC, the compound that causes the euphoric effect of cannabis, and has the potential to be a nonscheduled product if approved. Cannabidiol, the active ingredient in ZYN-002, has been granted orphan drug designation by the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of FXS and for the treatment of 22q. Additionally, ZYN-002 has received FDA Fast Track designation for the treatment of behavioral symptoms in patients with FXS.
About Fragile X Syndrome
Fragile X syndrome (FXS) is a rare genetic disorder that is the leading known cause of both inherited intellectual disability and autism spectrum disorder. The disorder negatively affects synaptic function, plasticity and neuronal connections, and results in a spectrum of intellectual disabilities and behavioral symptoms, such as social avoidance and irritability. While the exact prevalence is unknown, upwards of 80,000 patients in the U.S. and 121,000 patients in the European Union and the UK are believed to have FXS, based on FXS prevalence estimates of approximately 1 in 4,000 to 7,000 in males and approximately 1 in 8,000 to 11,000 in females. There is a significant unmet medical need in patients living with FXS as there are currently no FDA approved treatments for this disorder.
FXS is caused by a mutation in FMR1, a gene which modulates a number of systems, including the endocannabinoid system, and most critically, codes for a protein called FMRP. The FMR1 mutation manifests as multiple repeats of a DNA segment, known as the CGG triplet repeat, resulting in deficiency or lack of FMRP. FMRP helps regulate the production of other proteins and plays a role in the development of synapses, which are critical for relaying nerve impulses, and in regulating synaptic plasticity. In people with full mutation of the FMR1 gene, the CGG segment is repeated more than 200 times, and in most cases causes the gene to not function. Methylation of the FMR1 gene also plays a role in determining functionality of the gene. In approximately
About Clemizole Hydrochloride (EPX-100)
EPX-100, clemizole hydrochloride, is under development for the treatment of Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS). EPX-100 acts by targeting central 5-hydroxytryptamine receptors to modulate serotonin signaling. The drug candidate is administered orally twice a day in a liquid formulation and has been developed based on a proprietary phenotype-based zebrafish drug screening platform. DS is caused by a loss of function mutation in the SCN1A gene, and scn1 mutant zebrafish replicate the genetic etiology and phenotype observed in the majority of DS patients. The scn1Lab mutant zebrafish model that expresses voltage gated sodium channels has been used for high-throughput screening of compounds that modulate Nav1.1 in the central nervous system.
About Dravet Syndrome
Dravet syndrome (DS) is a severe and progressive epileptic encephalopathy that begins in infancy and causes significant impact on patient functioning. DS begins in the first year of life and is characterized by high seizure frequency and severity, intellectual disability, and a risk of sudden unexpected death in epilepsy. Approximately
About Lennox-Gastaut Syndrome
Lennox-Gastaut syndrome (LGS) is a rare and drug-resistant epileptic encephalopathy characterized by onset in children between 3-5 years of age. The underlying cause of LGS is unknown and can be related to a wide range of factors including genetic differences and structural differences in the brain. As a result, patients experience multiple seizure types, including atonic seizures, and developmental, cognitive, and behavioral issues. LGS affects approximately 48,000 patients in the U.S.
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
Forward Looking Statement:
This presentation includes forward‐looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in these materials or elsewhere, including statements regarding Harmony Biosciences Holdings, Inc.'s (the "Company") future financial position, business strategy and plans and objectives of management for future operations, should be considered forward-looking statements. Forward-looking statements use words like "believes," "plans," "expects," "intends," "will," "would," "anticipates," "estimates," "may," "could," "might," "continue," "potential," and similar words or expressions in discussions of the Company's future operations, financial performance or the Company's strategies, but the absence of these words does not mean that a statement is not forward-looking. These statements are based on current expectations or objectives that are inherently uncertain. These forward-looking statements involve significant risks and uncertainties that could cause the actual results to differ materially from the expressed or implied forwarding-looking statements, including, but not limited to the risk factors discussed under the caption "Risk Factors" in the Company's Annual Report on Form 10-K filed with the
This presentation includes information related to market opportunity as well as cost and other estimates obtained from internal analyses and external sources. The internal analyses are based upon management's understanding of market and industry conditions and have not been verified by independent sources. Similarly, the externally sourced information has been obtained from sources the Company believes to be reliable, but the accuracy and completeness of such information cannot be assured. Neither the Company, nor any of its respective officers, directors, managers, employees, agents, or representatives, (i) make any representations or warranties, express or implied, with respect to any of the information contained herein, including the accuracy or completeness of this presentation or any other written or oral information made available to any interested party or its advisor (and any liability therefore is expressly disclaimed), (ii) have any liability from the use of the information, including with respect to any forward-looking statements, or (iii) undertake to update any of the information contained herein or provide additional information as a result of new information or future events or developments.
Harmony Biosciences Investor Contact:
Brennan Doyle
484-539-9700
bdoyle@harmonybiosciences.com
Harmony Biosciences Media Contact:
Cate McCanless
202-641-6086
cmccanless@harmonybiosciences.com
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FAQ
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