Xenon Outlines Key Corporate Milestone Opportunities for 2025
Xenon Pharmaceuticals (XENE) has outlined significant milestones for 2025, highlighting progress in its pipeline programs. The company anticipates topline data from its first Phase 3 FOS study in H2 2025, marking a important step toward NDA filing for azetukalner. The company has initiated the first of three planned Phase 3 MDD studies and is advancing its ion channel portfolio.
Key developments include continued advancement of Phase 3 azetukalner clinical studies in focal onset seizures, with over 600 patient-years of data from the ongoing X-TOLE open-label extension study. The Phase 3 X-ACKT study for primary generalized tonic-clonic seizures (PGTCS) continues enrollment. Additionally, Xenon has launched X-NOVA2, the first of three Phase 3 trials for Major Depressive Disorder (MDD), with topline results from a Mount Sinai investigator-led study expected in H1 2025.
Xenon Pharmaceuticals (XENE) ha delineato traguardi significativi per il 2025, evidenziando i progressi nei suoi programmi in fase di sviluppo. L'azienda prevede di ottenere risultati preliminari dal suo primo studio FOS di Fase 3 nel secondo semestre del 2025, rappresentando un passo importante verso la presentazione della domanda di approvazione (NDA) per azetukalner. L'azienda ha avviato il primo dei tre studi di Fase 3 previsti per il Disturbo Depressivo Maggiore (MDD) e sta progredendo nel suo portafoglio di canali ionici.
I principali sviluppi includono il continuo avanzamento degli studi clinici di Fase 3 per azetukalner in caso di crisi focali, con oltre 600 anni-paziente di dati provenienti dallo studio di estensione open-label X-TOLE in corso. Lo studio X-ACKT di Fase 3 per le crisi tonico-cloniche generalizzate primarie (PGTCS) continua con le arruolazioni. Inoltre, Xenon ha lanciato X-NOVA2, il primo di tre studi di Fase 3 per il Disturbo Depressivo Maggiore (MDD), con risultati preliminari attesi da uno studio guidato da ricercatori del Mount Sinai nel primo semestre del 2025.
Xenon Pharmaceuticals (XENE) ha delineado hitos significativos para 2025, destacando los avances en sus programas en desarrollo. La compañía anticipa datos preliminares de su primer estudio FOS de Fase 3 en la segunda mitad de 2025, marcando un paso importante hacia la presentación de la solicitud de NDA para azetukalner. La empresa ha iniciado el primero de tres estudios de Fase 3 planificados para el Trastorno Depresivo Mayor (MDD) y está avanzando en su cartera de canales iónicos.
Los desarrollos clave incluyen el progreso continuo de los estudios clínicos de Fase 3 de azetukalner en crisis focales, con más de 600 años-paciente de datos del estudio de extensión en abierto X-TOLE en curso. El estudio X-ACKT de Fase 3 para las crisis tónico-clónicas generalizadas primarias (PGTCS) continúa con la inscripción. Además, Xenon ha lanzado X-NOVA2, el primero de tres ensayos de Fase 3 para el Trastorno Depresivo Mayor (MDD), con resultados preliminares esperados de un estudio liderado por investigadores del Mount Sinai en la primera mitad de 2025.
제논 제약(XENE)은 2025년을 위한 중요한 이정표를 설명하며 파이프라인 프로그램의 진행 상황을 강조하였습니다. 회사는 첫 번째 3상 FOS 연구에서 topline 데이터를 2025년 하반기에 수집할 것으로 기대하고 있으며, 이는 azetukalner의 NDA 제출을 위한 중요한 단계로 간주됩니다. 또한 회사는 계획된 3개의 3상 MDD 연구 중 첫 번째를 시작했으며 이온 채널 포트폴리오를 발전시키고 있습니다.
주요 개발 사항으로는 600년 이상의 환자 데이터를 보유한 진행 중인 X-TOLE 공개 라벨 확장 연구와 함께 중심 발작에서 azetukalner 임상 연구가 지속적으로 진전을 보이고 있습니다. 원발성 전신 강직-간질 발작(PGTCS)에 대한 3상 X-ACKT 연구는 등록을 계속 진행하고 있습니다. 또한, 제논은 주요 우울 장애(MDD)를 위한 3개 단계 중 첫 번째인 X-NOVA2를 출시했으며, Mount Sinai 연구진이 주도하는 연구에서의 topline 결과가 2025년 상반기에 예상됩니다.
Xenon Pharmaceuticals (XENE) a défini des jalons significatifs pour 2025, mettant en avant les progrès de ses programmes en développement. La société anticipe des données préliminaires de sa première étude FOS de Phase 3 au second semestre 2025, marquant une étape importante vers le dépôt d'une demande d'autorisation de mise sur le marché (NDA) pour azetukalner. L'entreprise a lancé la première de trois études de Phase 3 prévues pour le Trouble Dépressif Majeur (MDD) et fait progresser son portefeuille de canaux ioniques.
Les développements clés incluent l'avancement continu des études cliniques de Phase 3 pour azetukalner dans le cadre de crises focales, avec plus de 600 années-patient de données provenant de l'étude d'extension ouverte X-TOLE en cours. L'étude X-ACKT de Phase 3 pour les crises tonico-cloniques généralisées primaires (PGTCS) continue son recrutement. De plus, Xenon a lancé X-NOVA2, le premier de trois essais de Phase 3 pour le Trouble Dépressif Majeur (MDD), avec des résultats préliminaires attendus d'une étude dirigée par des chercheurs du Mount Sinai au premier semestre 2025.
Xenon Pharmaceuticals (XENE) hat bedeutende Meilensteine für 2025 umrissen und dabei Fortschritte in seinen Pipeline-Programmen hervorgehoben. Das Unternehmen erwartet Ergebnisse aus seiner ersten Phase 3 FOS-Studie im zweiten Halbjahr 2025, was einen wichtigen Schritt in Richtung NDA-Einreichung für azetukalner darstellt. Das Unternehmen hat die erste von drei geplanten Phase 3 MDD-Studien initiiert und entwickelt sein Portfolio an Ionenkanälen weiter.
Zu den wichtigsten Entwicklungen gehört der kontinuierliche Fortschritt der klinischen Studien der Phase 3 für azetukalner bei fokalen Anfällen, wobei über 600 Patientenjahre an Daten aus der laufenden X-TOLE-Open-Label-Erweiterungsstudie vorliegen. Die Phase-3-Studie X-ACKT für primäre generalisierte tonisch-klonische Anfälle (PGTCS) läuft weiterhin mit der Rekrutierung. Darüber hinaus hat Xenon X-NOVA2 ins Leben gerufen, die erste von drei Phase-3-Studien zum Major Depressive Disorder (MDD), wobei die vorläufigen Ergebnisse einer von Mount Sinai geleiteten Studie im ersten Halbjahr 2025 erwartet werden.
- First Phase 3 topline data readout for azetukalner expected in H2 2025
- Extensive safety data with over 600 patient-years from X-TOLE extension study
- Expansion into MDD market with initiation of Phase 3 trials
- Multiple IND filings planned for 2025 across various indications
- None.
Insights
The outlined Phase 3 clinical trial milestones represent pivotal catalysts for Xenon Pharmaceuticals. The anticipated H2 2025 topline data from the first Phase 3 FOS study for azetukalner is particularly crucial, as positive results could pave the way for an NDA filing. The trial design mirrors the successful Phase 2b study, enhancing probability of success. With 600+ patient-years of existing safety data, the risk-benefit profile appears promising.
The expansion into Major Depressive Disorder (MDD) with three planned Phase 3 studies significantly broadens the commercial potential. The first MDD trial initiation demonstrates execution capability, while the upcoming Mount Sinai study results in H1 2025 could provide early validation for this indication.
The multiple IND filings planned for 2025 across Kv7 and Nav1.7 targets indicate a robust early-stage pipeline, providing additional shots on goal beyond azetukalner.
From a market perspective, Xenon's strategic positioning in both epilepsy and MDD represents significant commercial opportunities. The focal onset seizures market alone is valued at several billion dollars annually. The company's expansion into MDD, a $12+ billion market, substantially increases the total addressable market.
The partnership with Neurocrine Biosciences for Nav1.2/1.6 development provides additional validation and potential milestone revenues. The transition from clinical to commercial stage, contingent on successful Phase 3 results, could trigger substantial revaluation of Xenon's
For retail investors, the multiple catalysts throughout 2025 - including Phase 3 data readouts, IND filings and the Mount Sinai study results - create numerous potential value-driving events. The company's focus on ion channel targets, a historically successful approach in neurology, provides a solid scientific foundation.
– Topline data from the first Phase 3 FOS study anticipated in H2 2025 representing major milestone in support of NDA filing and potential launch of azetukalner
– First of three planned Phase 3 MDD studies now initiated
– Expanding ion channel portfolio includes Kv7 and Nav1.7 candidates advancing towards IND filings in 2025
VANCOUVER, British Columbia and BOSTON, Jan. 13, 2025 (GLOBE NEWSWIRE) -- Xenon Pharmaceuticals Inc. (Nasdaq: XENE), a neuroscience-focused biopharmaceutical company dedicated to discovering, developing, and delivering life-changing therapeutics for patients in need, today outlined progress within its pipeline programs and key milestones for 2025.
“Reflecting on our accomplishments in 2024, we are proud of the advancements in our late-stage clinical programs, culminating in a strong presence at AES where we presented new long-term azetukalner data from our ongoing X-TOLE open-label extension study showing sustained monthly reduction in seizure frequency and impressive seizure freedom rates in patients with epilepsy,” said Ian Mortimer, President and Chief Executive Officer of Xenon. “We also made significant progress within our early-stage portfolio, progressing multiple drug candidates targeting Kv7 and Nav1.7 into IND-enabling studies.”
“Looking ahead to 2025, we anticipate a number of important milestones, particularly the first Phase 3 topline readout of azetukalner in focal onset seizures expected in the second half of 2025, representing a major inflection point for Xenon as we evolve from a clinical to commercial stage organization. Beyond epilepsy, we continue to advance azetukalner in MDD with the recent launch of our first Phase 3 study. Lastly, we expect multiple IND filings in 2025 in our early-stage programs, with the goal of initiating first-in-human trials across multiple targets,” stated Mr. Mortimer.
Program Updates and Anticipated Milestones
- Phase 3 azetukalner clinical studies in focal onset seizures (FOS) continue to advance, with the first topline data readout anticipated in the second half of 2025.
- Building upon more than 600 patient-years of data to date from the ongoing X-TOLE open-label extension study, Xenon continues to generate long-term scientific evidence supporting azetukalner’s compelling efficacy and safety profile.
- The Phase 3 X-ACKT clinical study continues to enroll patients and is intended to support potential regulatory submissions in an additional epilepsy indication of primary generalized tonic-clonic seizures (PGTCS).
- The first of three planned Phase 3 clinical trials evaluating azetukalner in patients with MDD, X-NOVA2, has been initiated to support indication expansion of azetukalner in neuropsychiatry. In addition, topline results from the Mount Sinai investigator-led study of azetukalner in MDD are anticipated in the first half of 2025.
- Xenon continues to expand its portfolio with pre-clinical candidates targeting Kv7, Nav1.7, and Nav1.1 across various indications and anticipates filing multiple INDs, or equivalent, in 2025.
- Within Xenon’s partnered program, Neurocrine Biosciences is now focusing its efforts on progressing a Nav1.2/1.6 inhibitor towards human clinical trials in 2025 as a potential treatment for epilepsy.
About Azetukalner Phase 3 Epilepsy Program
Xenon’s Phase 3 epilepsy program includes three Phase 3 clinical trials in focal onset seizures (FOS) and primary generalized tonic-clonic seizures (PGTCS). Designed closely after the Phase 2b X-TOLE clinical trial, the Phase 3 X-TOLE clinical trials are multicenter, randomized, double-blind, placebo-controlled studies evaluating the clinical efficacy, safety, and tolerability of 15 mg or 25 mg of azetukalner administered orally with food as adjunctive treatment in approximately 360 patients with FOS per study. The primary efficacy endpoint is the median percent change (MPC) in monthly seizure frequency from baseline through the 12-week double-blind period (DBP) of azetukalner compared to placebo. X-ACKT is a multicenter, randomized, double-blind, placebo-controlled study evaluating the clinical efficacy, safety, and tolerability of 25 mg of azetukalner administered with food as adjunctive treatment in approximately 160 patients with PGTCS. The primary efficacy endpoint is the MPC in monthly PGTCS frequency from baseline through the 12-week DBP of azetukalner compared to placebo. Upon completion of the DBP in the Phase 3 epilepsy studies, eligible patients may enter an OLE study for up to three years.
About Azetukalner Phase 3 MDD Program
Xenon’s Phase 3 MDD program includes three multicenter, randomized, double-blind, placebo-controlled clinical trials to evaluate the clinical efficacy, safety, and tolerability of 20 mg of azetukalner administered orally with food over the 6-week double-blind period (DBP) as monotherapy treatment in approximately 450 patients with moderate-to-severe major depressive disorder (MDD) per study. The primary efficacy endpoint is the change from baseline in the HAM-D17 score at week 6 in patients who received azetukalner compared to placebo. Upon completion of the DBP, eligible patients may enter an OLE study for up to 12 months.
About Xenon Pharmaceuticals Inc.
Xenon Pharmaceuticals (Nasdaq: XENE) is a neuroscience-focused biopharmaceutical company dedicated to discovering, developing, and delivering life-changing therapeutics. We are advancing an ion channel product portfolio to address areas of high unmet medical need, including epilepsy and depression. Azetukalner, a novel, highly potent, selective Kv7 potassium channel opener, represents the most advanced, clinically validated potassium channel modulator in late-stage clinical development for multiple indications. For more information, please visit www.xenon-pharma.com.
Safe Harbor Statement
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995 and Canadian securities laws. These forward-looking statements are not based on historical fact, and include statements regarding the timing of and potential results from clinical trials; the potential efficacy, safety profile, future development plans in current and anticipated indications, addressable market, regulatory success and commercial potential of our and our partners’ product candidates; the efficacy of our clinical trial designs; our ability to successfully develop and achieve milestones in our azetukalner and other pipeline and development programs, including the anticipated filing of INDs; the timing and results of our interactions with regulators; our ability to successfully develop and obtain regulatory approval of azetukalner and our other product candidates; and anticipated timing of topline data readout from our clinical trials of azetukalner. These forward-looking statements are based on current assumptions that involve risks, uncertainties and other factors that may cause the actual results, events, or developments to be materially different from those expressed or implied by such forward-looking statements. These risks and uncertainties, many of which are beyond our control, include, but are not limited to: clinical trials may not demonstrate safety and efficacy of any of our or our collaborators’ product candidates; promising results from pre-clinical development activities or early clinical trial results may not be replicated in later clinical trials; our assumptions regarding our planned expenditures and sufficiency of our cash to fund operations may be incorrect; our ongoing discovery and pre-clinical efforts may not yield additional product candidates; any of our or our collaborators’ product candidates, including azetukalner, may fail in development, may not receive required regulatory approvals, or may be delayed to a point where they are not commercially viable; we may not achieve additional milestones in our proprietary or partnered programs; regulatory agencies may impose additional requirements or delay the initiation of clinical trials; the impact of market, industry, and regulatory conditions on clinical trial enrollment; the impact of competition; the impact of expanded product development and clinical activities on operating expenses; the impact of new or changing laws and regulations; the impact of unstable economic conditions in the general domestic and global economic markets; adverse conditions from geopolitical events; as well as the other risks identified in our filings with the U.S. Securities and Exchange Commission and the securities commissions in British Columbia, Alberta, and Ontario. These forward-looking statements speak only as of the date hereof and we assume no obligation to update these forward-looking statements, and readers are cautioned not to place undue reliance on such forward-looking statements.
“Xenon” and the Xenon logo are registered trademarks or trademarks of Xenon Pharmaceuticals Inc. in various jurisdictions. All other trademarks belong to their respective owner.
Contacts:
For Investors:
Chad Fugere
Vice President, Investor Relations
(857) 675-7275
investors@xenon-pharma.com
For Media:
Colleen Alabiso
Senior Vice President, Corporate Affairs
(617) 671-9238
media@xenon-pharma.com
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