Neurology-focused biopharmaceutical company Xenon Pharmaceuticals Inc. (XENE:NASDAQ), which concentrates its work on addressing unmet medical needs in the area of epilepsy, today announced "positive topline results from the Phase 2b X-TOLE clinical trial, which evaluated the clinical efficacy, safety, and tolerability of XEN1101 – a differentiated Kv7 potassium channel modulator – administered as adjunctive treatment in adult patients with focal epilepsy."
"We have generated strong evidence that supports the efficacy, safety, and tolerability of XEN1101 and depicts a highly favorable product profile for XEN1101."
Xenon Pharmaceuticals advised that XEN1101 successfully met its predetermined primary efficacy endpoint in the multiple-dosage Phase 2b X-TOLE study. In the trial, patients who were administered XEN1101 over a period of four weeks (28 days), showed a statistically significant, dose-dependent reduction from baseline in focal seizure frequency compared to participants given a placebo in both the 20 mg and 25 mg dose groups.
The company noted that in the clinical trial, XEN1101 also met several other primary and secondary endpoints including "a pairwise comparison of each active dose to placebo and a responder analysis with the proportion of patients who achieved a 50% or greater reduction in monthly focal seizure frequency from baseline."
Participants in the trial were given either 10 mg, 20 mg or 25 mg doses of XEN1101 or a placebo. Patients in the 25, mg dose group which consisted of 112 individuals demonstrated a 52.8% median reduction from baseline in monthly focal seizure frequency versus a rate of just 18.2% in the 114 patients in the placebo group. Similarly, the lower dose XEN1101 groups also showed improvement compared to placebo but results appeared to be lower as dosage levels decreased. The 51 patients given a 20 mg dose showed an improvement of 46.4% and the 46 patients given 10 mg dose showed somewhat lower improvement of 33.2%, though both were well above results observed in the control group.
"Many patients today are living with the debilitating impacts of focal seizures, even while taking multiple anti-seizure medications, so there is a substantial need for new, efficacious and well-tolerated therapies. The X-TOLE results generated from this large, multicenter, controlled trial are truly exciting because they demonstrate impressive efficacy of XEN1101 for adult patients with focal epilepsy, including those with seizures that are deemed difficult to treat…" stated Jacqueline A. French, M.D., Professor in the Department of Neurology at NYU Langone Health and Co-director of Epilepsy Clinical Trials at NYU Langone's Comprehensive Epilepsy Center; Founder/Director of the Epilepsy Study Consortium; and Chair of the XEN1101 X-TOLE Steering Committee.
"Physicians and patients could benefit from XEN1101's other important attributes, such as once-a-day dosing in the evening with no titration. With its unique potassium channel mechanism-of-action, the strength of these topline data suggest XEN1101 could play an important role in treating focal epilepsy," asserted French.
Xenon Pharmaceuticals' Chief Medical Officer Dr. Christopher Kenney remarked, "We have generated strong evidence that supports the efficacy, safety, and tolerability of XEN1101 and depicts a highly favorable product profile for XEN1101. Importantly, we saw statistically significant reductions of focal onset seizures compared to placebo across all dose groups, which suggests it is highly active in the central nervous system."
"With these compelling topline results, we are eager to work with the FDA to plan for an expedited development path moving forward," Dr. Kenney added.
XEN1101 was generally well-tolerated in the study and the highest 25 mg dose group displayed a 52.8% median reduction in monthly focal seizure frequency.
The company noted that that the Phase 2b X-TOLE clinical trial was a multicenter study designed to evaluate the efficacy, tolerability and safety of once-daily XEN1101 as an adjunctive treatment in 325 randomized adult patients diagnosed with focal epilepsy. Subjects in the study reportedly averaged 40.8 years of age ranging from 27 to 54 years old. The firm listed that 50.8% patients enrolled in the trial were on and continued taking three stable background anti-seizure medications throughout. In addition, 8.9% were taking one such medicine and 40.3% continued taking two.
The company stated that "the primary objective of the study was to assess the dose response trend of XEN1101 in reducing monthly focal seizure frequency, based on a ranked ANCOVA model." The firm said that XEN1101 was generally well-tolerated in the study and the highest 25 mg dose group displayed a 52.8% median reduction in monthly focal seizure frequency.
Xenon's President and CECO Ian Mortimer commented, "We believe that the X-TOLE topline data support a very attractive clinical profile for XEN1101 with desirable attributes that help differentiate it from other ASMs and suggest XEN1101 could be highly competitive in the future adult focal seizure market. Of importance, the XEN1101 efficacy data are especially compelling given that approximately 50% of the subjects in X-TOLE were using three concomitant ASMs, suggesting that this was potentially an even more challenging patient population than previous studies with other ASMs."
CEO Mortimer continued, "These data signal activity of XEN1101 in the central nervous system, which further supports our plans to develop XEN1101 in other indications, including major depressive disorder and other types of epilepsy."
The company advised that it would continue to work closely with the U.S. Food and Drug Administration and other regulatory bodies to move XEN1101 clinical development forward.
Xenon Pharmaceuticals is a clinical-stage biopharmaceutical company based in Burnaby, B.C., Canada, that concentrates its efforts on development of medicines to address neurological disorders with high unmet medical needs, with a strong emphasis on epilepsy. The firm explained on its website that a significant focus of its discovery efforts has been centered around human channelopathies, which has allowed it to develop strong capabilities in small molecule ion channel drug discovery.
Xenon Pharma started off the day with a market cap of around $641.7 million with approximately 41.13 million shares outstanding and a short interest of about 1.4%. XENE shares opened nearly 70% higher today at $26.51 (+$15.60, +69.94%) over Friday's $15.60 closing price and reached a new 52-week high price this morning of $30.50. The stock has traded today between $25.22 and $30.50 per share and is currently trading at $28.58 (+$12.98, +83.21%).
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