Xenon Outlines Key Upcoming Milestones at the 2026 J.P. Morgan Healthcare Conference

Rhea-AI Summary

Xenon Pharmaceuticals (Nasdaq: XENE) outlined near-term clinical and regulatory milestones at the 2026 J.P. Morgan Healthcare Conference. Key timelines include X-TOLE2 topline data in March 2026 for azetukalner in focal onset seizures (380 patients randomized) and an anticipated NDA submission in H2 2026. Five additional placebo-controlled Phase 3 azetukalner studies remain active across epilepsy and neuropsychiatry, with X-NOVA2 topline data expected H1 2027 for major depressive disorder. Phase 1 SAD/MAD data for pain candidates XEN1701 (NaV1.7) and XEN1120 (KV7) are expected in 2026 to support Phase 2 proof-of-concept studies. The company also reported long-term OLE results showing sustained seizure reductions and detailed plans to support regulatory filings in multiple regions.

Positive

• X-TOLE2 completed enrollment with 380 randomized patients
• Topline X-TOLE2 data expected March 2026, enabling potential H2 2026 NDA submission
• Five additional placebo-controlled Phase 3 azetukalner studies actively enrolling
• X-NOVA2 topline data for MDD expected in H1 2027
• Phase 1 SAD/MAD data for XEN1701 and XEN1120 expected in 2026 to support Phase 2 pain studies
• Open-label extension data show >90% reduction in monthly FOS frequency for participants treated ≥48 months

Negative

• None.

News Market Reaction – XENE

-2.18%

1 alert

-2.18% News Effect

On the day this news was published, XENE declined 2.18%, reflecting a moderate negative market reaction.

Data tracked by StockTitan Argus on the day of publication.

Key Figures

X-TOLE2 enrollment: 380 patients X-TOLE2 topline timing: March 2026 Azetukalner NDA plan: H2 2026 +5 more

8 metrics

X-TOLE2 enrollment 380 patients Randomized in Phase 3 FOS study; topline data expected March 2026

X-TOLE2 topline timing March 2026 Expected Phase 3 X-TOLE2 topline data in focal onset seizures

Azetukalner NDA plan H2 2026 Anticipated NDA submission for azetukalner after X-TOLE2 readout

X-NOVA2 topline timing H1 2027 Expected Phase 3 topline data in major depressive disorder

X-TOLE3 Japan enrollment 60 of 360 patients Planned Japanese participants in global X-TOLE3 FOS study

Seizure freedom ≥12 months 38.2% of participants X-TOLE OLE patients treated ≥48 months with ≥12-month freedom

Long-term seizure reduction Over 90% reduction Monthly FOS frequency drop from DBP baseline at ≥48 months OLE

U.S. adults with epilepsy Approximately 3 million Prevalence of epilepsy in the United States cited in release

Market Reality Check

Price: $42.72 Vol: Volume 1,977,485 is about...

high vol

$42.72 Last Close

Volume Volume 1,977,485 is about 2.05x the 20-day average of 965,223, indicating elevated interest into the JPM update. high

Technical Shares at $42.15 are trading above the 200-day MA $36.77 and sit 9.55% below the 52-week high of $46.60.

Peers on Argus

XENE was up 2.26% on strong volume while several close biotech peers were down (...

1 Up 1 Down

XENE was up 2.26% on strong volume while several close biotech peers were down (e.g., RARE -4.05%, SLNO -6.17%, IMVT -2.40%), with only SRRK modestly higher at +1.05%, pointing to a stock-specific reaction to its JPM milestones rather than a broad sector move.

Common Catalyst Multiple peers (RARE, SLNO, SRRK) also issued updates tied to the J.P. Morgan Healthcare Conference, highlighting a conference-driven news cluster rather than unified price direction.

Historical Context

5 past events · Latest: Jan 05 (Positive)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Jan 05	Conference appearance	Positive	-6.1%	Announcement of upcoming J.P. Morgan Healthcare Conference presentation.
Dec 05	Clinical data update	Positive	+0.7%	48‑month OLE data showing durable seizure reductions for azetukalner.
Dec 04	Equity grants	Positive	+0.7%	Inducement option and RSU grants to new non‑officer employees.
Dec 03	Investor webinar	Positive	+3.3%	Planned investor webinar reviewing AES 2025 epilepsy data and commercialization prep.
Nov 25	Conference data preview	Positive	+4.5%	Announcement of new azetukalner OLE epilepsy data to be presented at AES 2025.

Pattern Detected

Recent Xenon news, mainly positive clinical and corporate updates, has usually aligned with share price moves, with only one notable divergence on a prior JPM presentation announcement.

Recent Company History

Over the last few months, Xenon has steadily highlighted progress for azetukalner and its ion-channel pipeline. AES 2025 presentations showed long-term seizure reduction and seizure‑freedom data, followed by an investor webinar and additional OLE updates in late Nov–Dec 2025. There were also equity inducement grants and conference appearance notices. Most of these events led to modest positive price reactions, suggesting investors have generally rewarded clinical and commercialization progress similar to that outlined in today’s JPM conference update.

Market Pulse Summary

This announcement outlines a dense calendar of late‑stage milestones, including X‑TOLE2 Phase 3 topl...

Analysis

This announcement outlines a dense calendar of late‑stage milestones, including X‑TOLE2 Phase 3 topline data in March 2026 and an anticipated NDA in H2 2026, plus broader Phase 3 programs in epilepsy and mood disorders and early pain assets advancing through Phase 1. Historically, Xenon’s stock has often responded positively to concrete clinical progress. Investors may focus on the robustness of upcoming Phase 3 results, regulatory interactions, cash usage, and execution on planned Phase 2 pain studies.

Key Terms

nda, open-label extension, double-blind, adjunctive therapy, +4 more

8 terms

nda regulatory

"followed by anticipated NDA submission in H2 2026"

An NDA, or nondisclosure agreement, is a legal contract that keeps certain information private between parties. It’s like a promise not to share sensitive details, helping protect business ideas, strategies, or data from being leaked or used without permission. For investors, NDAs help ensure that confidential information remains secure, enabling trust and open communication during business discussions.

open-label extension medical

"open-label extension (OLE) rollover rate in X-TOLE2 are consistent"

An open-label extension is a continuation of a clinical trial where all participants and researchers know which treatment is being given, often after an initial blinded phase. It allows further study of a drug's long-term safety and effectiveness. For investors, it can indicate ongoing interest and confidence in a product's potential, influencing perceptions of its future value.

double-blind medical

"from double-blind period (DBP) baseline among participants treated ≥48 months"

A double-blind process means that neither the people conducting an activity nor the people involved know certain key details, such as who is receiving a treatment or a placebo. This approach helps prevent bias from influencing the results, making the outcome more trustworthy. For investors, it ensures that decisions or judgments are based on unbiased information rather than preconceived opinions or expectations.

adjunctive therapy medical

"administered as an adjunctive therapy in patients with focal onset seizures"

Adjunctive therapy is a treatment given in addition to a primary therapy to boost its effectiveness, reduce side effects, or treat related symptoms — like adding a side dish to make a main meal work better. For investors, evidence that an adjunctive therapy improves outcomes, safety, or convenience can expand a product’s use, support stronger regulatory labeling and reimbursement, and increase commercial potential by reaching more patients or strengthening a therapy’s market position.

single ascending dose medical

"Phase 1 Single Ascending Dose (SAD)/Multiple Ascending Dose (MAD) study"

A single ascending dose is a method used in testing new medicines where small amounts are given to participants, gradually increasing each time to find the safest and most effective dose. For investors, it provides important information about a drug’s safety and potential, helping gauge the progress and prospects of a pharmaceutical development.

multiple ascending dose medical

"Single Ascending Dose (SAD)/Multiple Ascending Dose (MAD) study in healthy"

A multiple ascending dose is a method used in testing new medicines where small groups of people receive gradually larger amounts of the drug over time. This approach helps researchers find the safest and most effective dose without causing too many side effects. For investors, it signals ongoing steps in drug development that can impact a company's potential success or approval prospects.

ind-enabling regulatory

"IND-enabling studies are ongoing for the Company’s NaV1.1 program"

Ind-enabling describes the preclinical tests and safety work a drug candidate must pass before a company can ask regulators for permission to start human trials (an Investigational New Drug or IND filing). Think of it as the mechanical inspection and crash-testing a prototype car needs before it can legally be driven on public roads; for investors, successful ind-enabling work reduces technical and regulatory risk and makes clinical progress and potential value creation more likely.

clinicaltrials.gov regulatory

"The X-TOLE2 clinical trial (ClinicalTrials.gov ID: NCT05614063) is a randomized"

clinicaltrials.gov is a publicly accessible U.S. government database that lists details, timelines and status updates for medical studies testing drugs, devices or procedures. For investors it acts like a public calendar and scoreboard—showing when trials start, are delayed, or report results—so it helps gauge a company’s development progress, regulatory risk and potential value impact before official earnings or approvals are announced.

AI-generated analysis. Not financial advice.

• Phase 3 X-TOLE2 topline data for azetukalner in FOS expected March 2026 followed by anticipated NDA submission in H2 2026
• Five additional Phase 3 azetukalner studies continuing to enroll patients in multiple indications in epilepsy and neuropsychiatry; Phase 3 X-NOVA2 topline data in MDD expected in H1 2027
• Data from two Phase 1 studies of novel Na_V1.7 (XEN1701) and K_V7 (XEN1120) candidates expected in 2026 to support Phase 2 proof-of-concept studies in pain
  
  

VANCOUVER, British Columbia and BOSTON, Jan. 12, 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 outlined forward momentum across the Company’s Phase 3 portfolio and discussed pipeline progress at the 2026 J.P. Morgan Healthcare Conference.

“We’re approaching an important inflection point as we expect to report topline data for our X-TOLE2 Phase 3 study of azetukalner in focal onset seizures later this quarter, a major milestone on the path toward regulatory submission, and ultimately, approval and our first commercial launch,” said Ian Mortimer, President and Chief Executive Officer of Xenon. “We are excited about the anticipation in the epilepsy community and our potential opportunity to bring a different mechanism to the epilepsy treatment paradigm. Beyond our Phase 3 X-TOLE2 program, we continue to build momentum through progress across five additional placebo-controlled Phase 3 studies of azetukalner in epilepsy and neuropsychiatry, and through our maturing pipeline with multiple studies underway in our early-stage pain portfolio.”

The Company's presentation at the J.P. Morgan Healthcare Conference will take place on Monday, January 12^th at 9:00 am PST. A live webcast will be available on the Investors section of Xenon's website and posted for replay following the event.

Phase 3 Azetukalner Program in Epilepsy & Depression

• Phase 3 X-TOLE2 study of azetukalner in focal onset seizures (FOS) has completed enrollment with 380 patients randomized, and final data are being collected to support the topline data readout anticipated in March 2026. Patient baseline characteristics and the open-label extension (OLE) rollover rate in X-TOLE2 are consistent with the Phase 2b X-TOLE study.
• Xenon recently presented 48-month data from the ongoing X-TOLE OLE study of azetukalner in patients with FOS at the American Epilepsy Society (AES) meeting. These data highlighted the long-term efficacy and safety of azetukalner, with reductions in monthly FOS frequency of over 90% from double-blind period (DBP) baseline among participants treated ≥48 months. Seizure freedom for any ≥12, ≥24, ≥36, and ≥48-month consecutive duration was attained by 38.2%, 25.2%, 19.8%, and 10.7% of participants treated for ≥48 months, respectively. These data, along with an update on Xenon’s progress preparing for potential commercialization of azetukalner, were also highlighted in a webinar for investors in December 2025.
• Phase 3 X-TOLE3 study of azetukalner in FOS continues to enroll and is intended to support regulatory submissions outside the United States. In support of a potential regulatory submission in Japan, Xenon has completed an ethnobridging study and shared the results in a recent meeting with Japan’s Pharmaceutical and Medical Devices Agency (PMDA). Xenon has aligned with PMDA to enroll approximately 60 of the planned 360 X-TOLE3 participants in Japan. Enrollment of non-Japanese participants in X-TOLE3 is expected to complete in 2026.
• Phase 3 X-ACKT study of azetukalner in Primary Generalized Tonic-Clonic Seizures (PGTCS) continues to enroll and is intended to support regulatory submissions for an additional epilepsy indication.
• Phase 3 X-NOVA2 and X-NOVA3 studies are ongoing as the first two of three planned Phase 3 clinical studies evaluating azetukalner in patients with major depressive disorder (MDD). Topline data from X-NOVA2 are expected in H1 2027.
• Phase 3 X-CEED study evaluating azetukalner in patients with bipolar depression (BPD) I or II is underway.
  
  

Broader Pipeline Opportunity

• Phase 1 Single Ascending Dose (SAD)/Multiple Ascending Dose (MAD) study in healthy adult participants is underway for XEN1701 targeting the sodium channel Na_V1.7. Preliminary Phase 1 data from the SAD portion of the study suggest that XEN1701 has reached drug concentrations that are predicted to achieve receptor occupancies required for therapeutic activity based on human genetic data. Study completion is expected in 2026 to support initiating a Phase 2 proof-of-concept study in acute pain. Na_V1.7 is an important target for pain based on strong human genetic validation and may represent a new class of pain medicines without the limitations of opioids.
• Phase 1 SAD/MAD study in healthy adult participants is underway for XEN1120 targeting K_V7. Preliminary Phase 1 data from the SAD portion of the study suggest that XEN1120 has reached drug concentrations that are consistent with pain reductions in preclinical models. Study completion is expected in 2026 to support initiating a Phase 2 proof-of-concept study in acute pain.
• IND-enabling studies are ongoing for the Company’s Na_V1.1 program for the treatment of Dravet syndrome. Pre-clinical data suggest that targeting Na_V1.1 could potentially address the underlying cause and symptoms of Dravet syndrome.
• In collaboration with Neurocrine Biosciences, a Phase 1 study is ongoing for NBI-921355, an investigational, selective inhibitor of voltage-gated sodium channels Na_V1.2 and Na_V1.6 in development for the potential treatment of certain types of epilepsy.
  
  

About Azetukalner
Azetukalner is a novel, potent K_V7 potassium channel opener currently in Phase 3 clinical trials for the treatment of epilepsy, major depressive disorder (MDD) and bipolar depression (BPD). It represents the most advanced, clinically validated potassium channel modulator in late-stage clinical development. Azetukalner is designed to open potassium channels in the central nervous system, allowing potassium ions to flow and hyperpolarizing neurons. This process helps reduce excessive neuronal firing, which is a key contributor to several neurologic and psychiatric disorders. It is the only K_V7 potassium channel opener in development for multiple indications that is backed by long-term efficacy and safety data in epilepsy patients and proof-of-concept data in MDD patients.

About the X-TOLE2 Phase 3 Study
The X-TOLE2 clinical trial (ClinicalTrials.gov ID: NCT05614063) is a randomized, double-blind, placebo-controlled, multicenter Phase 3 study evaluating the efficacy, safety, and tolerability of azetukalner, administered as an adjunctive therapy in patients with focal onset seizures (FOS). The study was designed to randomize approximately 360 participants in a blinded manner to one of two active treatment groups or placebo in a 1:1:1 fashion (azetukalner 25 mg : 15 mg : placebo). The trial is designed to assess the median percent change in monthly seizure frequency from baseline through the double-blind treatment period (DBP). During the DBP, participants will be instructed to orally take azetukalner or placebo once daily with food. Participants who complete the 12-week DBP may enroll in a separate open-label extension (OLE) study for continued treatment with azetukalner.

About Epilepsy and Focal Onset Seizures
Epilepsy is a neurological condition characterized by abnormal electrical activity in the brain that leads to spontaneous, recurrent and unprovoked seizures. It is the fourth most common neurological condition and affects approximately 3 million adults in the U.S. Focal epilepsy is characterized by recurrent seizures that originate in a specific area of the brain (i.e. “focal onset seizures”), leading to various symptoms depending on the affected region. Despite the availability of multiple anti-seizure medications, existing treatment options fail around a third of people living with epilepsy, underscoring a persistent, substantial unmet medical need.

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_V7 potassium channel opener in Phase 3 clinical trials for the treatment of epilepsy, major depressive disorder (MDD) and bipolar depression (BPD). Xenon is also advancing an early-stage portfolio of multiple promising potassium and sodium channel modulators, including K_V7 and Na_V1.7 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 www.xenon-pharma.com 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.

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 studies; 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 study designs; our ability to successfully develop and achieve milestones in our azetukalner and other pipeline and development programs, including the anticipated filing of INDs and NDAs; the timing and results of our interactions with regulators, including the timing of any NDA submission; 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 studies 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 studies 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 study results may not be replicated in later clinical studies; 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 or completion of clinical studies; the impact of market, industry, and regulatory conditions on clinical study 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.

Contacts

For Investors:
Tucker Kelly
Chief Financial Officer
investors@xenon-pharma.com

For Media:
Colleen Alabiso
Senior Vice President, Corporate Affairs
media@xenon-pharma.com

FAQ

When will Xenon (XENE) report topline data for the X-TOLE2 Phase 3 study?

Topline data for X-TOLE2 are expected in March 2026.

What is the planned timing for an NDA submission for azetukalner (XENE)?

Xenon anticipates an NDA submission in H2 2026, contingent on X-TOLE2 results.

How many patients were randomized in X-TOLE2 for azetukalner (XENE)?

The X-TOLE2 study completed enrollment with 380 patients randomized.

When does Xenon expect topline data for azetukalner in MDD (X-NOVA2)?

Topline data from X-NOVA2 are expected in H1 2027.

What near-term data are expected in 2026 for Xenon’s pain programs (XENE)?

Preliminary Phase 1 SAD/MAD data for XEN1701 and XEN1120 are expected in 2026 to support Phase 2 proof-of-concept studies.

What long-term efficacy data did Xenon report for azetukalner (XENE)?

OLE data showed >90% reduction in monthly focal onset seizure frequency for participants treated ≥48 months, with seizure freedom rates reported by duration.