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    XENE
    Earnings call· Mar 2026(Q1 FY26)

    Xenon Pharmaceuticals Q1 FY26 earnings call XENE

    May 7, 2026 Source

    Executive summary

    Xenon Pharmaceuticals Q1 FY26 — Strong X-TOLE2 Data and Extended Cash Runway

    Xenon Pharmaceuticals reported a strong first quarter, highlighted by positive Phase III X-TOLE2 data for azetukalner in focal onset seizures, reinforcing its potential as a differentiated treatment. The company also secured substantial financing, extending its operational runway and enabling continued advancement of its neuropsychiatry and early-stage pain programs. Management is focused on preparing for AZK's anticipated NDA submission and commercial launch, while also exploring broader therapeutic applications and pipeline expansion.

    Highlights

    4

    • Reported positive Phase III X-TOLE2 study results for azetukalner (AZK) in focal onset seizures, exceeding expectations with a 53.2% median percent change reduction for the 25mg dose.

    • Achieved significant long-term seizure freedom in the X-TOLE OLE data, with 40% of patients treated for at least 48 months being seizure-free for 12 months or more.

    • Successfully completed a $747.5 million financing, extending the cash runway into 2029.

    • Progressed three Phase III depression studies for AZK, with top-line results from X-NOVA2 expected in H1 2027.

    Concerns

    3

    • Anticipated AZK launch timing is end of 2027 or early 2028, subject to standard FDA review and 3-month DEA scheduling.

    • Cross-trial comparisons for seizure freedom data are challenging due to different patient populations and reporting methodologies, particularly when comparing AZK to cenobamate.

    • The Dravet syndrome market is crowded, requiring clear differentiation for the Nav1.1 program.

    Guidance & targets

    7
    CategoryTargetConfidence
    NDA submission
    NDA submission to the U.S. Food and Drug Administration
    high materiality
    High
    Anticipated launch timing
    End of 2027 or early 2028
    high materiality
    Medium
    X-NOVA2 top line results
    First half of 2027
    medium materiality
    High
    Cash runway
    Into 2029
    high materiality
    High
    XEN1701 first-in-human studies completion
    Later this year
    medium materiality
    High
    XEN1120 first-in-human studies completion
    Later this year
    medium materiality
    High
    NBI-921355 Phase Ib data
    Next year
    low materiality
    High

    Operational metrics

    20
    Public offering proceeds
    $747.5 million
    Q1 FY26

    Successful public offering completed in Q1 FY26.

    Cash, cash equivalents and marketable securities
    $1.3 billionvs $586 million as of December 31
    Q1 FY26

    Ended Q1 with strong cash position.

    Median percent change in monthly FOS frequency
    53.2%vs 10.4% for placebo
    Q1 FY26

    Highly statistically significant results from the Phase III X-TOLE2 study.

    Median percent change in monthly FOS frequency
    34.5%vs 10.4% for placebo
    Q1 FY26

    Highly statistically significant results from the Phase III X-TOLE2 study.

    Median percent change in monthly FOS frequency
    10.4%
    Q1 FY26

    Placebo arm results in the Phase III X-TOLE2 study.

    Proportion of participants with at least 75% reduction in monthly seizure frequency
    —
    Q1 FY26

    Observed dose-dependent increases in responder rates.

    Proportion of participants with at least 90% reduction in monthly seizure frequency
    —
    Q1 FY26

    Observed dose-dependent increases in responder rates.

    100% responder rate
    6.5%
    Q1 FY26

    Increased steadily over time.

    100% responder rate
    11.3%
    Q1 FY26

    Increased steadily over time.

    100% responder rate
    13.7%
    Q1 FY26

    Increased steadily over time.

    Reduction in monthly seizure frequency
    91%
    Q1 FY26

    Long-term OLE data demonstrated continued reductions.

    Seizure-free for at least 12 months
    40%
    Q1 FY26

    Remarkable seizure freedom in a treatment-resistant population.

    Seizure-free for at least 2 years
    25%
    Q1 FY26

    Remarkable seizure freedom in a treatment-resistant population.

    Reduction in monthly seizure frequency
    100%
    Q1 FY26

    Demonstrated continued reductions.

    Reduction in monthly seizure frequency
    82%
    Q1 FY26

    Demonstrated continued reductions.

    Median monthly focal seizure frequency at baseline
    13
    Q1 FY26

    Highly treatment-resistant FOS population.

    Median prior ASMs
    5
    Q1 FY26

    Highly treatment-resistant FOS population.

    Proportion of patients on 3 concomitant ASMs
    >50%
    Q1 FY26

    Highly treatment-resistant FOS population.

    Proportion of patients on or tried cenobamate
    ~60%
    Q1 FY26

    Indicates a cenobamate-refractory population; ~40% on background, ~20% tried and failed.

    Medical Science Liaison (MSL) team deployment
    2 years
    Q1 FY26

    Field medical team has been engaging with prescribers for two years.

    Industry KPIs

    4
    MetricValueDetails
    Launch access metricsDiscussions initiated with payers
    Pipeline read out calendarX-NOVA2 top-line results H1 2027; NBI-921355 Phase Ib data next year (2027)
    Regulatory approvals filingsNDA submission for azetukalner
    Clinical trial efficacy safety dataMedian percent change reduction in monthly FOS frequency: 53.2% (25mg AZK), 34.5% (15mg AZK), 10.4% (placebo); 100% responder rate (25mg AZK): 6.5% (12-week), 11.3% (last 6-weeks), 13.7% (last 4-weeks); X-TOLE OLE 48-month seizure reduction: 91%; X-TOLE OLE seizure freedom: 40% (12+ months), 25% (2+ years)%

    Deals & partnerships

    1
    Neurocrine BiosciencesCollaboration on NBI-921355, an investigational selective inhibitor of voltage-gated sodium channels Nav1.2 and Nav1.6, for certain types of epilepsy.

    Neurocrine is progressing a Phase Ib study for NBI-921355, with data expected next year.

    Risks & headwinds

    4
    Anticipated launch timing for AZK is subject to standard FDA review and DEA scheduling.End of 2027 or early 2028 for launch.

    Standard review period (12 months), DEA scheduling (3 months).

    Mitigation: Base case assumption is standard review; ongoing thoughtful interactions with FDA.

    Difficulty in directly comparing AZK seizure freedom data to competitors like cenobamate due to differences in patient populations and reporting methodologies.

    X-TOLE2 population was more refractory (median 13 seizures/month, 5 prior ASMs) than cenobamate's trials; cenobamate's 400mg dose rarely achieved in practice.

    Mitigation: Focus on long-term OLE data and ease-of-use attributes for AZK; providing detailed breakdowns of responder rates.

    The Dravet syndrome space is crowded with existing and developing therapies.

    Multiple people are working on this devastating disorder.

    Mitigation: Differentiating Nav1.1 program by targeting underlying pathophysiology with an oral small molecule, aiming for disease modification beyond seizure control.

    Long-term development strategy for acute vs. chronic pain for XEN1701 and XEN1120 is still being worked out.Coming months and years.

    Still need to figure out chronic versus acute and a whole sorts of different things.

    Mitigation: Initial focus on acute pain proof-of-concept studies; mechanisms are not limited to acute pain, potential for broad development if promising.

    What to watch in Q2 FY26

    4

    Completion of XEN1701 and XEN1120 Phase I studies

    later this year
    CurrentOngoing
    TargetCompletion of first-in-human studies

    Why it matters

    These completions will enable advancement to Phase II proof-of-concept studies in pain, leveraging novel non-opioid approaches.

    We're looking forward to completing our first-in-human studies for our novel pain programs this year.

    Q&A highlights

    7

    Asked about derisking safety issues for Nav1.7 in Phase I and specifics on Phase II acute pain studies (size, scope, arms).

    Ian Mortimer stated they are at high enough exposures for receptor occupancy in Phase I Nav1.7, feeling confident in safe dosing and therapeutic index. Phase I studies for both XEN1701 and XEN1120 will complete this year. Phase II acute pain studies (bunionectomy/abdominoplasty) are planned, with design details (number of arms, doses) still being finalized after Phase I data. Chris Kenney confirmed they have what's needed to proceed to Phase II.

    “I think sitting today, we feel really good that we can safely dose, have the appropriate therapeutic index and give this mechanism a real shot to show proof-of-concept data in Phase II.”

    asked by Paul Matteis · answered by Ian Mortimer

    2 min read4 chapters

    Detailed Narrative

    01

    AZK in Focal Onset Seizures

    The Phase III X-TOLE2 study for azetukalner (AZK) in focal onset seizures (FOS) exceeded expectations, demonstrating a 53.2% median percent change reduction for the 25mg dose and 34.5% for the 15mg dose, compared to 10.4% for placebo. The study population was highly treatment-resistant, with patients experiencing a median of 13 seizures per month and having tried a median of 5 prior ASMs. Data presented at AAN highlighted rapid onset of efficacy, dose-dependent responder rates, and a consistent safety profile, with no titration and once-daily dosing being key differentiating attributes.

    02

    AZK in Neuropsychiatric Indications

    Xenon is broadening AZK's potential beyond epilepsy, with three ongoing Phase III studies in neuropsychiatry: X-NOVA2 and X-NOVA3 in major depressive disorder (MDD), and X-CEED in bipolar depression (BPD). Top-line results from X-NOVA2 are expected in the first half of 2027. The rationale for Kv7 openers in depression is supported by preclinical, clinical, and genetic evidence, with physicians keenly interested in AZK's novel mechanism, rapid onset, and potential benefits on anhedonia.

    03

    Early-Stage Pipeline Advancement

    The company is progressing its early-stage ion channel programs, including first-in-human studies for XEN1701 (targeting Nav1.7) and XEN1120 (targeting Kv7) for pain, with completion expected later this year. These programs aim to address large unmet medical needs for non-opioid pain therapies. Additionally, IND-enabling studies are ongoing for the Nav1.1 program in Dravet syndrome, with preclinical data showing potential for disease modification by targeting the underlying genetics.

    04

    Commercial Readiness and Financial Strength

    Xenon is actively building its commercial infrastructure and go-to-market strategy for AZK's anticipated U.S. launch in late 2027 or early 2028. This includes increasing scientific engagement with HCPs, expanding field-based capabilities, and initiating discussions with payers. A successful public offering of $747.5 million has fortified the balance sheet, providing $1.3 billion in cash, cash equivalents, and marketable securities, extending the cash runway into 2029 and supporting the transition to a commercial-stage company.

    AI-generated summary of the company’s earnings call. Not investment advice.