Clinical Trials
46
15 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
1996
Active, not recruiting
12
26.1%
Completed
14
30.4%
Enrolling By Invitation
3
6.5%
Recruiting
13
28.3%
Suspended
2
4.3%
Terminated
2
4.3%
No approval data available
- Xenon Pharmaceuticals has submitted a New Drug Application to the FDA for azetukalner in focal seizures, based on the Phase 2b X-TOLE and Phase 3 X-TOLE2 trials. - All four azetukalner doses produced statistically significant reductions in monthly seizure frequency versus placebo, with more than 1,500 patient-years of epilepsy safety data. - Xenon voluntarily paused enrollment of new patients in its MDD and bipolar depression studies after an analysis of neuropsychiatric adverse events not previously seen in Phase 2 X-NOVA. - X-NOVA2 enrollment reached roughly 80% of its 450-patient target, with topline MDD data now expected in the first quarter of 2027.
- SK Biopharmaceuticals has secured exclusive global rights to 1ST-104, a preclinical oral small-molecule dual inhibitor of LRRK2 and c-Abl for Parkinson's disease, from 1ST Biotherapeutics. - The licensing agreement is valued at up to $314.8 million, including a $1.8 million upfront payment, a $1.8 million candidate-selection milestone, and tiered royalties on global net sales. - SK Biopharmaceuticals separately made a $2.2 million strategic equity investment in 1ST Biotherapeutics to support a long-term partnership between the two South Korean companies. - 1ST-104 is a next-generation Type 2 LRRK2 inhibitor designed to avoid pulmonary findings seen with some Type 1 inhibitors, and has shown target selectivity and blood-brain barrier permeability preclinically.
- The FDA issued a partial clinical hold on Biohaven's opakalim (BHV-7000) after nonclinical rodent studies flagged a metabolite with uncertain relevance to human safety. - New patient enrollment is paused across the focal epilepsy program, though dosing continues for more than 600 already-randomized patients, including the fully enrolled RISE-3 study. - Biohaven expects RISE-3 topline data in the second half of 2026, with the hold not affecting that timeline, while enrollment resumption timing remains uncertain. - The hold arrives days after Biohaven licensed global rights to opakalim to SK Biopharmaceuticals for $350 million upfront in a deal worth up to $795 million.
- BIOTECanada named Xenon Pharmaceuticals Inc. as Biotech Company of the Year and Ripple Therapeutics as Promising Biotech Company of the Year for 2026. - Dr. Allen Eaves received the Industry Leadership Award, while Dr. Massimiliano Paganelli and Dr. Claudia Raggi shared the Community Leadership Award for Research & Innovation. - The awards, presented for more than 20 years, recognize leadership, innovation, and impact across Canada's biotechnology sector with global reach. - Winners will be celebrated at the Gold Leaf Awards Ceremony on September 22 at the National Arts Centre in Ottawa, coinciding with BIONATION.
- Xenon Pharmaceuticals' azetukalner demonstrated a 53.2% reduction in seizures among treatment-resistant epilepsy patients, representing the highest placebo-adjusted efficacy ever observed in a pivotal epilepsy study. - The late-stage trial of 380 patients showed 55% of participants experienced at least a 50% reduction in seizure activity, with the drug outperforming placebo by 42.7 percentage points over 12 weeks. - Xenon plans to submit a new drug application to the FDA in the second half of 2026, potentially bringing the 15th B.C.-developed therapy to market for the 3 million Americans living with epilepsy.
- Xenon Pharmaceuticals is approaching a pivotal Phase 3 X-TOLE2 data readout for azetukalner in focal onset seizures, expected in March 2026. - The company has completed enrollment of 380 patients in X-TOLE2 and is conducting five additional Phase 3 studies across epilepsy and neuropsychiatry indications. - A potential New Drug Application submission is planned for the second half of 2026 if Phase 3 results are positive. - Institutional investor Braidwell recently reduced its Xenon position by $74.76 million, maintaining a 2.62% portfolio allocation ahead of the critical data milestone.
- A new catalyst monitor report identifies 13 significant regulatory and clinical trial events expected in Q1 2026, based on key opinion leader interviews and predictive intelligence. - Major regulatory approval decisions are anticipated for GSK's depemokimab in asthma and sinusitis, Regenxbio's gene therapy for Hunter syndrome, and Omeros' narsoplimab for thrombotic microangiopathy. - Ongoing late-stage clinical trials from Xenon Pharmaceuticals, Cerevel, and Neumora are expected to deliver critical data across neurological and psychiatric conditions. - The pipeline momentum spans diverse therapeutic areas including respiratory diseases, rare genetic disorders, neurological conditions, and major depressive disorder.
- Xenon Pharmaceuticals will present seven posters at AES 2025, including new long-term data from the ongoing X-TOLE open-label extension study of azetukalner in patients with focal onset seizures. - The presentations will feature ≥48-month interim analysis data characterizing patterns of seizure freedom epochs with extended use of azetukalner, which could inform future clinical practice. - Additional data will highlight the impact of depression on epilepsy patients and new pre-clinical findings from the company's early-stage NaV1.1 program in Dravet syndrome. - Xenon will also host a symposium with the Epilepsy Foundation of America exploring depression and anxiety in epilepsy, featuring leading epileptologists from major medical centers.
- Design Therapeutics has appointed industry veteran Justin Gover to its Board of Directors, bringing over 25 years of biotechnology leadership experience to the clinical-stage genetic disease company. - Gover previously served as founding CEO of GW Pharmaceuticals for over two decades, leading the company through its $7 billion acquisition by Jazz Pharmaceuticals in 2021 and the successful commercialization of Epidiolex for childhood epilepsies. - The appointment comes as Dr. Arsani William stepped down from the board, having provided strategic guidance since 2021 including through the company's IPO and clinical pipeline advancement. - Design Therapeutics is developing GeneTAC gene targeted chimera small molecules to modulate disease-causing gene expression, with clinical programs targeting Friedreich ataxia and Fuchs endothelial corneal dystrophy.
- Praxis Precision Medicines' mid-stage trial of vormatrigine demonstrated a 56% median reduction in seizure frequency among adults with focal onset and tonic-clonic epileptic disorders after eight weeks of treatment. - The oral sodium channel blocker showed rapid onset of action, with over half of participants achieving 50% seizure reduction within the first week, and 22% achieving complete seizure freedom by week eight. - Despite meeting analyst expectations for "best case" efficacy outcomes, Praxis shares dropped 13% due to competitive concerns and lack of clear differentiation from existing treatments. - The drug was generally well tolerated across 61 patients, though nearly 25% discontinued the study, with common side effects including dizziness, drowsiness, and headache.
