Delving into the Latest Updates on Azetukalner with Synapse

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

AZK, Encukalner, 1OP-2198

+ [6]

Target

Kv7.2 x Kv7.3

Action

modulators

Mechanism

Kv7.2 modulators(Voltage-gated potassium channel subunit Kv7.2 modulators), Kv7.3 modulators(Potassium voltage-gated channel subfamily KQT member 3 modulators)

Therapeutic Areas

Nervous System DiseasesOther Diseases

Active Indication

Bipolar I disorderBipolar II disorderDepressive Disorder, Major+ [3]

Inactive Indication-

Originator Organization

Bausch Health Americas, Inc.

Active Organization

Xenon Pharmaceuticals, Inc.

Inactive Organization

Icahn School of Medicine at Mount Sinai

License Organization

1st Order Pharmaceuticals, Inc.

Bausch Health Cos., Inc.

Icahn School of Medicine at Mount Sinai

+ [1]

Drug Highest PhasePhase 3

First Approval Date-

Regulation-

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Structure/Sequence

Molecular FormulaC23H29FN2O

InChIKeyFJNPZKZPWVVSON-UHFFFAOYSA-N

CAS Registry1009344-33-5

Measurement of androstenedione (4-AD) and 17α-hydroxyprogesterone (17-OHP) in blood is crucial for diagnosing and monitoring endocrine disorders such as congenital adrenal hyperplasia (CAH), which results from enzymatic defects in the steroidogenesis pathway. CAH patients require lifelong hormone replacement therapy to manage adrenal insufficiency and control androgen excess. Microsampling offers a less invasive alternative to frequent venous blood sampling, particularly in paediatric CAH patients, and enables at-home monitoring. Including 11-ketotestosterone (11-KT), an emerging biomarker in CAH, further enhances the clinical relevance of this approach. Liquid chromatography-tandem mass spectrometry-based methods were established and fully validated for the determination of 4-AD, 17-OHP and 11-KT in plasma, blood and 20 μL dried blood microsamples collected by volumetric absorptive microsampling (VAMS). For VAMS samples, we paid particular attention to robustness regarding storage and hematocrit to support future home sampling applications. The method's applicability was evaluated by assessing the agreement with plasma as the reference matrix for steroid hormone determination. A robust extraction procedure was developed, ensuring consistent recoveries across storage conditions and hematocrits (0.20 - 0.60 L/L). The method was successfully validated for 4-AD, 17-OHP and 11-KT in VAMS, blood and plasma samples, achieving lower limits of quantification of 30, 144 and 81 pg/mL in VAMS samples, respectively. Accuracy and precision were within 6.6% and 14.0% for all matrices. VAMS samples remained stable for up to one week at room temperature and after postal sending. A proof-of-concept study showed that plasma concentrations can reliably be derived from VAMS concentrations, when taking into account the hematocrit.

01 Nov 2026·CNS DRUGS

Next-Generation Pharmacotherapies for Unipolar Mood Disorders: A Leading Article on Phase II/III Drug Development Across Diverse Mechanisms

Review

Author: Rhee, Taeho Greg ; King, Alana ; Hardy, Morgan ; Wilkinson, Samuel T

Despite the wide prevalence of mood disorders globally, there is a substantial unmet need for effective, mechanistically distinct treatments. Given that approximately 30% of individuals do not display adequate response to standard antidepressant therapies, the past 15 years have seen a shift away from traditional monoaminergic approaches toward glutamatergic and other novel mechanisms. Following the US Food and Drug Administration (FDA) approval of esketamine and brexanolone in 2019, industry investment in therapeutics with novel mechanisms for mood and anxiety disorders has expanded, catalyzing a new wave of innovative interventions. This review summarizes progress for unipolar mood disorders in four categories-glutamatergic/GABA modulators, ion channel modulators, psychedelics, and other potential therapeutic mechanisms-focusing on Phase II/III industry-sponsored US clinical trials active within the past 3 years. The drug names discussed are ALTO-100, ALTO-203, ALTO-207, ALTO-300, BHV-7000, BI-1569912, BPL-003, COMP360, DT120, ELE-101, GATE-251, GH001, GM-1020, GM-2505, HLP003, MK-1942, NBI-1065845, NBI-1070770, NV-5138, PIPE-307, SEP-363856, SPT-300, VLS-01, and XEN1101. The US Clinical Trials registry (clinicaltrials.gov), MEDLINE, conference proceedings, and company communications such as press releases were consulted to obtain up-to-date trial information and reported outcomes. Overall, the therapeutic landscape shows an expanding range of clinical compounds under active development; nonetheless, persistent challenges remain, including approaches to therapeutics where functional unblinding may occur, the ways in which to consider a novel conceptualization of occupancy-driven versus event-driven pharmacology, and the role of supporting psychotherapy.

26 Oct 2026·CLINICAL CHEMISTRY AND LABORATORY MEDICINE

First performance verification of automated cobas ^® i601 LC-MS/MS in a high-throughput clinical laboratory using 25-hydroxyvitamin D, 5α-dihydrotestosterone, and 17α-hydroxyprogesterone

Article

Author: Lutz, Detlef ; Wieland, Eberhard ; Wildt, Sebastian ; Eckel, Nathalie ; Harzer, Oliver ; Morch, Karina ; Filbrecht, Sarah ; Stemmerich, Karsten ; Shipkova, Maria ; Westenberger, Tanja ; Werle, Annika ; Schmidt, Beate ; Blödel, Anja

Abstract:

Objectives:

Liquid chromatography-tandem mass spectrometry (LC-MS/MS) is the analytical gold standard for measuring many parameters in clinical laboratories but remains labor-intensive, time-consuming, and insufficiently standardized. Automating LC-MS analytics is one way to integrate the advantages of the methodology into laboratory routine. This study aimed to evaluate the performance of the fully automated LC-MS/MS analyzer cobas ® i601 for 25-hydroxyvitamin D (25(OH)D), 5α-dihydrotestosterone (DHT), and 17α-hydroxyprogesterone (17-OHP), and to assess its suitability for routine clinical use.

Methods:

Analytical performance of the cobas ® i601 was verified for 25(OH)D, DHT, and 17-OHP according to established quality standards by assessing precision, accuracy, linearity, limit of quantitation (LOQ), and measurement uncertainty. Comparability with existing certified or validated routine methods as well as consistency of two chromatographic lines within the instrument were assessed using Passing–Bablok regression and Bland–Altman analysis.

Results:

Satisfactory analytical performance was observed for all analytes on both chromatographic lines. Imprecision and bias relative to target values fulfilled the acceptance criteria, with values ≤4.2 % and ≤6.0 %, respectively. Linearity and LOQ claimed by the manufacturer were confirmed. Strong agreement with our conventional LC-MS/MS methods was observed for 25(OH)D and with LC-MS/MS data from external quality assessment schemes for 17-OHP. In contrast, 17-OHP patient sample concentrations were considerably lower and DHT concentrations were slightly lower compared to the radioimmunoassay (DIAsource) and the laboratory-developed LC-MS/MS method we used as references, respectively.

Conclusions:

The fully automated cobas ® i601 LC-MS/MS analyzer provides robust analytical performance and its results are both precise and accurate as well as comparable to established LC-MS/MS methods. It enables, for the first time, an efficient high-throughput vitamin D and steroid testing by random-access mass spectrometry in routine clinical laboratories, without having to rely on specially trained personnel.

118

News (Medical) associated with Azetukalner

03 Nov 2026

·www.biospace.com

Xenon to Present at Upcoming Investor Conferences - November 3, 2026

VANCOUVER, BC and BOSTON, MA, Nov. 03, 2026 (GLOBE NEWSWIRE) -- Xenon Pharmaceuticals Inc. (Nasdaq: XENE), a neuroscience-focused biopharmaceutical company dedicated to drug discovery, clinical development, and commercialization of life-changing therapeutics for patients in need, today announced that the company will present at four upcoming investor conferences in the fourth quarter of 2026. Guggenheim 3 rd Annual Healthcare Innovation Conference, Boston, MA Fireside chat: Tuesday, November 10, 2026, 2:30–2:55pm EST Stifel 2026 Healthcare Conference, New York, NY Fireside chat: Wednesday, November 11, 2026, 4:40–5:10pm EST 2026 Jefferies Global Healthcare Conference in London, London, UK Fireside chat: Wednesday, November 18, 2026, 10:30–10:55am GMT (5:30–5:55am EST) 9 th Annual Evercore Healthcare Conference, Coral Gables, FL Fireside chat: Tuesday, December 1, 2026, 7:55–8:15am EST Details about all company presentations, including webcast information, can be found on the Investors section of Xenon's website. When available, webcasts will be posted for replay following the event. The above-listed dates and times are subject to change. About Xenon Pharmaceuticals Inc. Xenon Pharmaceuticals (Nasdaq: XENE) is a neuroscience-focused biopharmaceutical company dedicated to drug discovery, clinical development, and commercialization of life-changing therapeutics for patients in need. Xenon’s lead molecule, azetukalner, is a novel, potent K V 7 potassium channel opener in late-stage clinical development for the treatment of epilepsy, major depressive disorder and bipolar depression. Xenon is also advancing an early-stage portfolio of multiple promising potassium and sodium channel modulators, including K V 7 and Na V 1.7 programs in Phase 1 development for the potential treatment of pain. Xenon has offices in Vancouver, British Columbia, and Boston, Massachusetts. For more information, visit and follow us on LinkedIn and X . Xenon and the Xenon logo are registered trademarks or trademarks of Xenon Pharmaceuticals Inc. in the US, Canada and elsewhere. All other trademarks belong to their respective owner. Contacts For Investors: Laura Perry/Emily Huang Argot Partners Xenon@argotpartners.com For Media: Colleen Alabiso Senior Vice President, Corporate Affairs media@xenon-pharma.com

Phase 1

02 Nov 2026

·www.biospace.com

Xenon to Report Q3 2026 Financial Results on November 9, 2026

VANCOUVER, BC and BOSTON, MA, Nov. 02, 2026 (GLOBE NEWSWIRE) -- Xenon Pharmaceuticals Inc. (Nasdaq: XENE), a neuroscience-focused biopharmaceutical company dedicated to drug discovery, clinical development, and commercialization of life-changing therapeutics for patients in need, today announced it will report its third quarter 2026 financial results and provide a business update after the close of U.S. financial markets on Monday, November 9, 2026. Company Presentation Details: Date: Monday, November 9, 2026 Time: 4:30pm EST (2:30pm PCT) Webcast: Pre-register here Dial-In: (800) 715-9871 toll-free or (646) 307-1963 for international callers Conference ID: 8984105 A live webcast of the company presentation will be available on the Investors section of Xenon's website and posted for replay following the event. The above-mentioned dates and times are subject to change. About Xenon Pharmaceuticals Inc. Xenon Pharmaceuticals (Nasdaq: XENE) is a neuroscience-focused biopharmaceutical company dedicated to drug discovery, clinical development, and commercialization of life-changing therapeutics for patients in need. Xenon’s lead molecule, azetukalner, is a novel, potent K V 7 potassium channel opener in late-stage clinical development for the treatment of epilepsy, major depressive disorder and bipolar depression. Xenon is also advancing an early-stage portfolio of multiple promising potassium and sodium channel modulators, including K V 7 and Na V 1.7 programs in Phase 1 development for the potential treatment of pain. Xenon has offices in Vancouver, British Columbia, and Boston, Massachusetts. For more information, visit and follow us on LinkedIn and X . Xenon and the Xenon logo are registered trademarks or trademarks of Xenon Pharmaceuticals Inc. in the US, Canada and elsewhere. All other trademarks belong to their respective owner. Contacts: For Investors: Laura Perry/Emily Huang Argot Partners Xenon@argotpartners.com For Media: Colleen Alabiso Senior Vice President, Corporate Affairs media@xenon-pharma.com

Financial StatementPhase 1

01 Nov 2026

·www.biospace.com

Xenon Pharmaceuticals Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

VANCOUVER, BC and BOSTON, MA, Oct. 30, 2026 (GLOBE NEWSWIRE) -- Xenon Pharmaceuticals Inc. (Nasdaq: XENE), a neuroscience-focused biopharmaceutical company dedicated to drug discovery, clinical development, and commercialization of life-changing therapeutics for patients in need, today announced equity inducement grants to nine new non-officer employees consisting of an aggregate of 24,250 share options and 14,250 restricted share units (RSUs). All of the foregoing share options and RSUs were approved by the Compensation Committee of the Company’s Board of Directors with an effective date of October 29, 2026 and were granted as inducements material to the employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4). The share options have an exercise price of $37.54 per common share, which is equal to the closing price per share of Xenon’s common shares on the grant date of October 29, 2026. The share option grants vest over four years, with 25% vesting on the one-year anniversary of the respective employee’s start date and 1/36th of the remaining options vesting monthly thereafter on the last day of each month, subject to such option recipient’s continued service relationship with the Company. Each option has a 10-year term and is subject to the terms and conditions of the share option agreement and the terms of the Company’s Amended and Restated 2025 Inducement Equity Incentive Plan. The RSUs will vest as to 25% of the underlying shares on each of the first four anniversaries of the grant date of October 29, 2026, subject to such employee’s continued service relationship with the Company. Each RSU grant is subject to the terms and conditions of the restricted share unit award agreement and the terms of the Company’s Amended and Restated 2025 Inducement Equity Incentive Plan. About Xenon Pharmaceuticals Inc. Xenon Pharmaceuticals (Nasdaq: XENE) is a neuroscience-focused biopharmaceutical company dedicated to drug discovery, clinical development, and commercialization of life-changing therapeutics for patients in need. Xenon’s lead molecule, azetukalner, is a novel, potent K V 7 potassium channel opener in late-stage clinical development for the treatment of epilepsy, major depressive disorder and bipolar depression. Xenon is also advancing an early-stage portfolio of multiple promising potassium and sodium channel modulators, including K V 7 and Na V 1.7 programs in Phase 1 development for the potential treatment of pain. Xenon has offices in Vancouver, British Columbia, and Boston, Massachusetts. For more information, visit  and follow us on  LinkedIn  and  X . Xenon and the Xenon logo are registered trademarks or trademarks of Xenon Pharmaceuticals Inc. in the US, Canada, and elsewhere. All other trademarks belong to their respective owner. Contacts: For Investors: Laura Perry/Emily Huang Argot Partners Xenon@argotpartners.com For Media: Colleen Alabiso Senior Vice President, Corporate Affairs media@xenon-pharma.com

Phase 1

100 Deals associated with Azetukalner

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External Link

KEGG	Wiki	ATC	Drug Bank
D12890	-	-	-

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Bipolar I disorder	Phase 3	United States	Xenon Pharmaceuticals, Inc.	08 Aug 2025
Bipolar I disorder	Phase 3	Bulgaria	Xenon Pharmaceuticals, Inc.	08 Aug 2025
Bipolar I disorder	Phase 3	Germany	Xenon Pharmaceuticals, Inc.	08 Aug 2025
Bipolar I disorder	Phase 3	Spain	Xenon Pharmaceuticals, Inc.	08 Aug 2025
Bipolar II disorder	Phase 3	United States	Xenon Pharmaceuticals, Inc.	08 Aug 2025
Bipolar II disorder	Phase 3	Bulgaria	Xenon Pharmaceuticals, Inc.	08 Aug 2025
Bipolar II disorder	Phase 3	Germany	Xenon Pharmaceuticals, Inc.	08 Aug 2025
Bipolar II disorder	Phase 3	Spain	Xenon Pharmaceuticals, Inc.	08 Aug 2025
Depressive Disorder, Major	Phase 3	United States	Xenon Pharmaceuticals, Inc.	20 Dec 2024
Epilepsy, Tonic-Clonic	Phase 3	United States	Xenon Pharmaceuticals, Inc.	14 Feb 2023

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Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05376150 (X-NOVA, CTgov)	Phase 2	Depressive Disorder, Major	168	Placebo	qjkhanelin(cywfunqtyt) = dclloirjlu lislspjhbk (ffnbmcaixs, 1.41) View more	-	21 Sep 2026
NCT05614063 (X-TOLE2, AAN2026)	Phase 3	Seizures	380	Azetukalner 25 mg	dwrasgbnpi(vtrbmtlsvd) = pwqfmmvtxb okwndiprsc (ixzrubimnf )	Positive	25 Apr 2026
				Placebo	dwrasgbnpi(vtrbmtlsvd) = bzqvlgaqbq okwndiprsc (ixzrubimnf ) View more
NCT05614063 (X-TOLE2, Company_Website) Manual	Phase 3	Seizures	374	Azetukalner 25 mg	dteljsqggo(doyelsvfbm) = saaekzvoij nhmvkbkwai (hufjeppfgr ) Met View more	Positive	09 Mar 2026
				Azetukalner 15 mg	dteljsqggo(doyelsvfbm) = tmepojzken nhmvkbkwai (hufjeppfgr ) Met View more
NCT03796962 (X-TOLE, GlobeNewswire) Manual	Phase 3	Epilepsies, Partial	131	Azetukalner (open-label extension)	gwbwxfsxdi(xyyeoyggjb) = xvsakzeqmr jqcgrakalg (vxxuoxjkeb ) View more	Positive	05 Dec 2025
X-TOLE (IEC2025)	Phase 3	Epilepsies, Partial	275	Azetukalner 20 mg QD	xkrgegsesp(keahevyyfv) = ubwxxzvbze jssguojaoq (uzrswlvnnm ) View more	Positive	30 Aug 2025
NCT05376150 (X-NOVA, Pubmed \| JAMA Netw Open \| NEWS) Manual	Phase 2	Depressive Disorder, Major	168	AzetukalnerAzetukalner 10 mg	ppscsnrvpi(ntrrrvnste) = aynzdjnrah pwkfhiimwb (rtsrljvxmf, 1.34)	Positive	27 May 2025
				AzetukalnerAzetukalner 20 mg	ppscsnrvpi(ntrrrvnste) = rrknjwgdba pwkfhiimwb (rtsrljvxmf, 1.45) View more
X-TOLE (AAN2025 \| IEC2025)	Phase 2	Seizures	325	Azetukalner 25 mg	errihpmnwg(aokercnvve) = 31.6% nxuoekmohr (nyntjvodev ) View more	Positive	07 Apr 2025
				Placebo
NCT03796962 (X-TOLE, NEWS) Manual	Not Applicable	Epilepsies, Partial	182	Azetukalner	wkithujmzz(xpcaoaghtk) = xdnhoayksl zazywltsyv (tqfrubmjoi ) View more	Positive	06 Dec 2024
NCT03796962 (X-TOLE, CTgov)	Phase 2	Epilepsies, Partial \| Seizures	325	XEN1101 (25 mg XEN1101)	ocgbmwliai(fxwgbztawp) = mayygucguk dukkiohonk (arbngcowkx, nirhbbptxj - ruzvibhpsh) View more	-	24 Sep 2024
				XEN1101 (20 mg XEN1101)	ocgbmwliai(fxwgbztawp) = wzbhnnzyrb dukkiohonk (arbngcowkx, ipyfuvkvxg - cczhrjtchl) View more
ESC2024	Not Applicable	-	-	Recreational drug users	yqwgbidcqb(dxchaurops) = xzsogrtuvo fphujrewxs (ysclitmcef )	-	31 Aug 2024
				Non-users	yqwgbidcqb(dxchaurops) = oabmxihadz fphujrewxs (ysclitmcef )