Axsome Therapeutics Reports Third Quarter 2020 Financial Results and Provides Business Update
COMET Phase 3 long-term safety trial of AXS-05 in MDD, and MOVEMENT Phase 3 long-term safety trial of AXS-07 in migraine completed
NDA submissions for AXS-05 in depression expected in
Launch readiness activities progressing with buildout of Digital-Centric Commercialization (DCC™) platform
Efficacy results from three Phase 2 open-label efficacy trials of AXS-05 in TRD, antidepressant unresponsive MDD, and suicidal ideation, on track for 4Q 2020
Efficacy results from MOVEMENT Phase 3 open-label trial of AXS-07 in migraine expected in 4Q 2020
Phase 3 trial of AXS-05 in Alzheimer’s disease agitation on track for initiation in 4Q 2020
Phase 3 trial of AXS-12 in narcolepsy on track for initiation in 1Q 2021
FDA meeting for AXS-14 in fibromyalgia scheduled for 1Q 2021
Company to host conference call with slides today at
“Over the past several months, we continued to advance our AXS-05 and AXS-07 product candidates towards NDA submissions in major depressive disorder and migraine, and intensified our commercial launch readiness activities,” said
CNS Pipeline Update
For the many people living with serious CNS disorders, Axsome accelerates the invention and adoption of life-changing medicines. The Company is developing a portfolio of differentiated, patent-protected, central nervous system (CNS) product candidates with four in active clinical development.
- AXS-05: AXS-05 (dextromethorphan/bupropion modulated delivery tablet) is Axsome’s novel, oral, investigational NMDA receptor antagonist with multimodal activity being developed for the following indications: major depressive disorder (MDD), Alzheimer’s disease (AD) agitation, and smoking cessation. AXS-05 has been granted
U.S. Food and Drug Administration(FDA) Breakthrough Therapy designations for MDD, and for AD agitation; as well as Fast Track designations for treatment resistant MDD (TRD), and for AD agitation.
Depression: Pre-submission activities for Axsome’s New Drug Application (NDA) to the FDA for AXS-05 for the treatment of MDD are nearing completion. Due to a COVID-related logistical delay from one vendor, the Company now expects to submit the NDA in January instead of by year-end.
Axsome has completed the COMET (Clinical Outcomes with NMDA-based Depression Treatment) Phase 3 open-label, long-term safety trial to support the planned NDA filing of AXS-05 in MDD. The three Phase 2 open-label efficacy sub-studies of the COMET trial have also been completed. These sub-studies are evaluating the efficacy and safety of AXS-05 in three clinically pertinent MDD patient populations: the COMET-TRD trial in treatment resistant MDD (TRD), the COMET-AU trial in antidepressant unresponsive MDD, and the COMET-SI trial in MDD with suicidal ideation. Efficacy results from these studies are on track to be reported in the fourth quarter of 2020.
Axsome is conducting the MERIT (Mechanistic Evaluation of Response in TRD) trial, a Phase 2, double-blind, placebo-controlled, randomized withdrawal study in patients with TRD. Results from the MERIT trial are on track to be reported in the first half of 2021.
AD Agitation: In
June 2020, Axsome received FDA Breakthrough Therapy designation for AXS-05 for the treatment of AD agitation. The designation was supported by the positive results from the pivotal ADVANCE-1 study.
August 2020, Axsome announced the results of an FDA Breakthrough Therapy meeting for AXS-05 for the treatment of AD agitation. Results of the meeting confirmed the pivotal status of the ADVANCE-1 trial, and the establishment of the superiority of AXS-05 over its components (component contribution) in the treatment of AD agitation. Consequently, only one additional Phase 3 efficacy trial is needed to support the filing of an NDA and only a placebo control will be required for this trial. This additional Phase 3 efficacy trial will be conducted using a randomized-withdrawal design, in which all patients are first treated with open-label AXS-05, with the patients experiencing a treatment response being subsequently randomized in a double-blind fashion to continued treatment with AXS-05 or to switch to placebo. Axsome is on track to initiate this Phase 3 trial this quarter.
Smoking Cessation: Axsome is scheduled to meet with the FDA in the first quarter of 2021 to discuss the continued clinical development of AXS-05 as an aid to smoking cessation treatment. Axsome previously announced positive results from a Phase 2 trial of AXS-05 for smoking cessation treatment conducted under a research collaboration between
Axsomeand Duke University.
- AXS-07: AXS-07 (MoSEIC™ meloxicam/rizatriptan) is Axsome’s novel, oral, rapidly absorbed, multi-mechanistic, investigational medicine for the acute treatment of migraine.
Migraine: Pre-submission activities for the Company’s NDA for AXS-07 in the acute treatment of migraine are progressing with major NDA-related items on track for completion by year-end. Axsome now plans to submit the NDA to the FDA in the first quarter of 2021, versus previous guidance of the fourth quarter of 2020, to allow for inclusion of supplemental manufacturing information to ensure a robust submission package.
Axsome has completed the MOVEMENT (Multimechanistic Treatment Over Time of Migraine Symptoms) Phase 3 open-label, long-term safety trial to support the planned NDA filing of AXS-07 in the acute treatment of migraine. Axsome expects to announce efficacy results from this trial this quarter.
- AXS-12: AXS-12 (reboxetine) is Axsome’s novel, oral, potent, and highly selective norepinephrine reuptake inhibitor for the treatment of narcolepsy. AXS-12 has been granted FDA Breakthrough Therapy designation for the treatment of cataplexy in patients with narcolepsy and Orphan Drug Designation for the treatment of narcolepsy.
August 2020, Axsome received FDA Breakthrough Therapy designation for AXS-12 for the treatment of cataplexy in patients with narcolepsy. The designation was supported by the positive results from the Phase 2 CONCERT study.
September 2020, Axsome announced the expedited development status and plan for AXS-12 for the treatment of narcolepsy following an FDA Breakthrough Therapy meeting. The expedited development plan includes one Phase 3 efficacy trial, which, along with the previously completed Phase 2 CONCERT trial, will be used to support the filing of an NDA for approval of AXS-12 for the treatment of cataplexy in narcolepsy. The planned Phase 3 trial will be a randomized, double-blind, placebo-controlled, parallel-group study. Axsome is on track to initiate this Phase 3 trial in the first quarter of 2021.
- AXS-14: AXS-14 (esreboxetine) is Axsome’s novel, oral, potent, and highly selective norepinephrine reuptake inhibitor for the treatment of fibromyalgia. Esreboxetine, the SS-enantiomer of reboxetine, is more potent and selective than racemic reboxetine.
Fibromyalgia: Axsome is scheduled to meet with the FDA in the first quarter of 2021 to discuss the further clinical development of AXS-14 for the treatment of fibromyalgia. AXS-14 has previously met the primary endpoints and demonstrated positive and statistically significant results in a Phase 3 and a Phase 2 trial in the treatment of fibromyalgia.
- NDA Submissions:
° AXS-05 in the treatment of MDD (
° AXS-07 in the acute treatment of migraine (1Q 2021)
- Clinical Trial Readouts:
° Phase 2 COMET-TRD trial of AXS-05 in TRD, topline data (4Q 2020)
° Phase 2 COMET-AU trial of AXS-05 in antidepressant unresponsive MDD, topline data (4Q 2020)
° Phase 2 COMET-SI trial of AXS-05 in MDD with suicidal ideation, topline data (4Q 2020)
° Phase 2 MERIT trial of AXS-05 in TRD, topline data (1H 2021)
- Clinical Trial Initiations:
° Phase 3 trial of AXS-05 in AD agitation (4Q 2020)
° Phase 3 trial of AXS-12 in narcolepsy (1Q 2021)
- FDA Meetings:
° AXS-05 for smoking cessation (1Q 2021)
° AXS-14 for fibromyalgia (1Q 2021)
November 2020, Axsome and Veeva Systems announced a strategic partnership to augment the build of Axsome’s Digital-Centric Commercialization (DCC™) platform. The partnership with Veeva is part of the continuing build of Axsome’s DCC platform in preparation for the potential launches of AXS-05 for depression and AXS-07 for migraine. The Axsome DCC platform is a proprietary approach to commercialization that incorporates specialized digital tools, proprietary data and analytics, integrated systems, and an intelligent operating model. Through this digital platform, Axsome aims to optimize physician and patient engagements, enhance engagement quality, and increase the effectiveness of promotional efforts as compared to traditional approaches.
September 2020, Axsome secured a $225 millionterm loan facility with Hercules Capital. Under the terms of this facility, $60 millioncan be drawn at closing; $115 millionmay be drawn at the Company’s option, in three separate tranches, upon approval of AXS-05 in MDD, upon approval of AXS-07 in migraine, and upon certain combined sales criteria for AXS-05 and AXS-07; and an additional $50 millionis available, subject to the approval of Hercules Capital, to support future strategic initiatives, including further pipeline advancement or expansion. The committed capital strengthens the Company’s balance sheet through the anticipated commercial launches of AXS-05 for MDD and AXS-07 for migraine, and extends its cash runway into at least 2024, based on current operating plans.
Third Quarter 2020 Financial Results
- Research and development (R&D) expenses: R&D expenses were
$14.8 millionfor the quarter ended September 30, 2020and $15.8 millionfor the comparable period in 2019. The decrease of $1.0 millionwas driven by the completion of several clinical trials which were ongoing in the comparable prior period.
- General and administrative (G&A) expenses: G&A expenses were
$6.3 millionfor the quarter ended September 30, 2020and $3.1 millionfor the comparable period in 2019. The change was primarily due to an increase in stock compensation expense, along with the build-out of the commercial function.
- Net loss: Net loss was
$22.9 million, or $(0.61)per share, which includes a $1.3 millionone-time charge related to the extinguishment of the previous debt facility, for the quarter ended September 30, 2020, compared to a net loss of $19.1 million, or $(0.56)per share for the comparable period in 2019.
- Cash: At
September 30, 2020, Axsome had $202.4 millionof cash compared to $190.7 millionof cash at June 30, 2020.
- Shares outstanding: At
September 30, 2020, Axsome had 37,344,201 shares of common stock outstanding.
- Financial guidance: Axsome believes that its cash at
September 30, 2020along with the committed capital from its $225 millionterm loan facility will be sufficient to fund the Company’s anticipated operations, based on its current operating plans, into at least 2024.
Conference Call Information
Axsome will host a conference call and webcast with slides today at
Forward Looking Statements
Certain matters discussed in this press release are “forward-looking statements”. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. In particular, the Company’s statements regarding trends and potential future results are examples of such forward-looking statements. The forward-looking statements include risks and uncertainties, including, but not limited to, the success, timing and cost of our ongoing clinical trials and anticipated clinical trials for our current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including our ability to fully fund our disclosed clinical trials, which assumes no material changes to our currently projected expenses), futility analyses and receipt of interim results, which are not necessarily indicative of the final results of our ongoing clinical trials, and the number or type of studies or nature of results necessary to support the filing of a new drug application (“NDA”) for any of our current product candidates; our ability to fund additional clinical trials to continue the advancement of our product candidates; the timing of and our ability to obtain and maintain
Selected Consolidated Financial Data
Statements of Operations Information:
|Three months ended|
|Research and development||$||14,795,493||$||15,835,573|
|General and administrative||6,331,308||3,111,662|
|Total operating expenses||21,126,801||18,947,235|
|Loss from operations||(21,126,801||)||(18,947,235||)|
|Interest income (expense)||(551,002||)||(327,825||)|
|Loss on extinguishment of debt||(1,247,012||)||—|
|Net loss per common share, basic and diluted||$||(0.61||)||$||(0.56||)|
|Weighted average common shares outstanding, basic and diluted||37,311,726||34,445,489|
Balance Sheet Information:
|Cash and cash equivalents||$||202,360,292||$||219,966,167|
|Loan payable, current and long-term||48,026,108||19,934,918|
Source: Axsome Therapeutics, Inc.