Today's News

Burnaby, BC, March 4, 2022--(T-Net)--Xenon Pharmaceuticals Inc. (Nasdaq:XENE), a neurology-focused biopharmaceutical company, today reported financial results for the year ended December 31, 2021 and provided a corporate update.

Ian Mortimer, President and Chief Financial Officer, Xenon Pharmaceuticals Inc.

Ian Mortimer, Xenon's President and Chief Executive Officer stated, "We made significant progress in 2021, marked by the positive read-out of strong efficacy data from our XEN1101 Phase 2b X-TOLE clinical trial, which represented a transformative event for Xenon. We have successfully carried this positive momentum into 2022 as we look forward to an end-of-Phase 2 meeting with the FDA in the second quarter and continue to focus on initiation of Phase 3 development for XEN1101 in the second half of 2022. In addition, we have now filed an IND with the FDA to support the initiation of our company-sponsored Phase 2 XEN1101 clinical trial in major depressive disorder, anticipated to commence in the first half of this year."

Mr. Mortimer added, "Patient enrollment is ongoing in our pediatric XEN496 Phase 3 'EPIK' clinical trial, which is expected to be completed in the first half of 2023. Our partnered programs also continue to advance through development with two separate Phase 2 clinical trials underway from Neurocrine Biosciences in adult patients with focal-onset seizures and pediatric patients with SCN8A-related epilepsy."

Highlights and Anticipated Milestones

Proprietary Programs

XEN1101

XEN1101 is a differentiated Kv7 potassium channel opener being developed for the treatment of epilepsy and major depressive disorder (MDD). In October 2021, Xenon announced positive results from its Phase 2b X-TOLE clinical trial, which evaluated the clinical efficacy, safety and tolerability of XEN1101 administered as an adjunctive treatment for adult patients with focal epilepsy. The topline data showed all primary and secondary seizure reduction endpoints were statistically significant across all dose groups, including the primary endpoint of median reduction from baseline in monthly seizure frequency and in the key secondary endpoint of patients with at least a 50% reduction in monthly focal seizure frequency from baseline, with p-values of <0.001 for both the 20 mg and 25 mg dose groups.

Xenon anticipates participating in an "end-of-Phase 2" meeting with the U.S. Food and Drug Administration (FDA) in the second quarter of this year to support the initiation of its Phase 3 XEN1101 clinical program in adult patients with focal epilepsy, estimated in the second half of the year. The X-TOLE open-label extension, which has been extended to three years, is expected to continue to generate important long-term data for XEN1101. Xenon is also evaluating other potential epilepsy indications for the future development of XEN1101.

Xenon continues to execute on its comprehensive strategy to protect and expand the intellectual property portfolio that covers XEN1101. Importantly, two additional U.S. patents were granted in 2021 with claims related to: (1) compositions-of-matter covering four distinct crystalline forms of XEN1101 drug substance (including the forms used in current and future clinical development); and (2) methods of enhancing the bioavailability of XEN1101 by administration with or close to a meal (consistent with the dosing of XEN1101 in clinical studies). These U.S. patents are expected to expire in 2040 and 2039, respectively, absent any extensions of patent term.

In addition, Xenon is collaborating with the Icahn School of Medicine at Mount Sinai to conduct an investigator-sponsored Phase 2 proof-of-concept, multi-site, randomized, parallel-arm, placebo-controlled clinical trial of XEN1101 for the treatment of MDD, with patient enrollment underway. In addition, an investigational new drug (IND) application has been submitted to the FDA to support its plans for a larger company-sponsored clinical study in MDD with XEN1101, which is expected to be initiated in the first half of 2022.

XEN496

XEN496, a Kv7 potassium channel opener, is a proprietary pediatric formulation of the active ingredient ezogabine being developed for the treatment of KCNQ2 developmental and epileptic encephalopathy (KCNQ2-DEE). A Phase 3 randomized, double-blind, placebo-controlled, parallel group, multicenter clinical trial, called the "EPIK" study, is underway to evaluate the efficacy, safety, and tolerability of XEN496 administered as adjunctive treatment in approximately 40 pediatric patients aged one month to less than six years with KCNQ2-DEE. Xenon anticipates that the EPIK study will be completed in the first half of 2023.

Partnered Programs

NBI-921352

Xenon has an ongoing collaboration with Neurocrine Biosciences to develop treatments for epilepsy. Neurocrine Biosciences has an exclusive license to XEN901, now known as NBI-921352, a selective Nav1.6 sodium channel inhibitor. Neurocrine Biosciences is conducting a Phase 2 clinical trial evaluating NBI-921352 in adult patients with focal onset seizures, with data expected in 2023. In addition, a Phase 2 clinical trial is underway evaluating NBI-921352 in patients aged between 2 and 21 years with SCN8A developmental and epileptic encephalopathy (SCN8A-DEE). Pursuant to the terms of the agreement, Xenon has the potential to receive certain clinical, regulatory, and commercial milestone payments, as well as future sales royalties.

PCRX301 (formerly FX301)

In November 2021, Pacira BioSciences, Inc. completed its acquisition of Flexion Therapeutics, Inc., which included Flexion's global rights to develop and commercialize XEN402, a Nav1.7 inhibitor also known as funapide. XEN402 has been formulated for extended release from a thermosensitive hydrogel and is now known as PCRX301 (previously FX301). A Phase 1b proof-of-concept trial is underway evaluating the safety and tolerability of PCRX301 administered as a single-dose, popliteal fossa block in patients undergoing bunionectomy, with data now anticipated in the second quarter of this year. Pursuant to the terms of the agreement, Xenon has the potential to receive certain clinical, regulatory, and commercial milestone payments, as well as future sales royalties.

2021 Financial Results

Cash and cash equivalents and marketable securities as of December 31, 2021 were $551.8 million, compared to $177.0 million as of December 31, 2020. As of December 31, 2021 there were 51,634,752 common shares, 2,775,996 pre-funded warrants and 1,016,000 Series 1 Preferred Shares outstanding. The Series 1 Preferred Shares are convertible into common shares on a one-for-one basis at the option of the holder, subject to certain limitations.

Based on current assumptions, which include fully supporting the planned XEN1101 clinical development program, XEN496, and pre-clinical and discovery programs, Xenon anticipates having sufficient cash to fund operations into at least 2024, excluding any revenue generated from existing partnerships or potential new partnering arrangements.

For the year ended December 31, 2021, Xenon reported total revenue of $18.4 million, compared to $32.2 million for the same period in 2020. The decrease of $13.7 million was attributable to deferred revenue related to the transfer of exclusive licenses and associated technology and know-how for certain compounds under the license and collaboration agreement with Neurocrine Biosciences being fully recognized by December 2020, partially offset by recognition of $5.3 million of milestone revenue in connection with the license and collaboration agreement with Neurocrine Biosciences, $3.0 million of milestone revenue in connection with the agreement with Pacira BioSciences and an increase in research and development services revenue.

Research and development expenses for the year ended December 31, 2021 were $75.5 million, compared to $50.5 million for the same period in 2020. The increase of $24.9 million was primarily attributable to increased spending on Xenon's clinical development product candidates XEN1101 and XEN496 as well as increased spending on pre-clinical, discovery and other internal programs.

General and administrative expenses for the year ended December 31, 2021 were $22.0 million compared to $12.9 million for the same period in 2020. The increase of $9.0 million was primarily attributable to increased salaries and benefits from additional headcount, stock-based compensation expense, market research costs and legal fees for intellectual property protection.

Other income for the year ended December 31, 2021 was $0.1 million compared to $2.2 million for the same period in 2020. The decrease was primarily attributable to a decrease in interest income and an unrealized loss on fair value of marketable securities recognized for the year ended December 31, 2021 due to fluctuations in market interest yields.

Net loss for the year ended December 31, 2021 was $78.9 million, compared to $28.8 million for the same period in 2020. The change was primarily attributable to higher research and development and general and administrative expenses, and lower revenue as compared to the same period in 2020.

At-the-Market Equity Offering

Xenon also announced today that it has amended its at-the-market equity offering sales agreement dated August 6, 2020, with Jefferies LLC and Stifel, Nicolaus & Company, Incorporated, under which Xenon may sell its common shares from time-to-time. Sales of the common shares, if any, will only be conducted in the United States through the Nasdaq or another exchange at market prices. No sales of common shares will be made in Canada.

About Xenon Pharmaceuticals Inc.

Xenon Pharmaceuticals (NASDAQ:XENE) is a clinical stage biopharmaceutical company committed to developing innovative therapeutics to improve the lives of patients with neurological disorders. We are advancing a novel product pipeline of neurology therapies to address areas of high unmet medical need, with a focus on epilepsy.