Axsome Therapeutics Reports First Quarter 2020 Financial Results and Provides Business Update
Clinical successes highlight Axsome’s accelerated evolution into a leading CNS company
Positive pivotal Phase 2/3 results for AXS-05 in Alzheimer’s disease agitation further deepen innovative pipeline
Positive efficacy results in 5 significant CNS indications with 4 candidates advance broad late-stage pipeline
Two NDA submissions, for AXS-05 in MDD and AXS-07 in migraine, on track for 4Q 2020
Pre-commercialization activities underway for potentially first-in-class or best-in-class CNS therapies
Company to host conference call today at
“The recent clinical successes in our late-stage pipeline, including positive efficacy data in depression, Alzheimer’s disease agitation, migraine, narcolepsy, and fibromyalgia, highlight Axsome’s accelerated evolution into a leading, innovative CNS company,” said
CNS Pipeline Update
Axsome is developing a portfolio of differentiated, patent-protected, central nervous system (CNS) product candidates. For the many people facing unsatisfactory treatments for CNS disorders, Axsome accelerates the invention and adoption of life-changing medicines. The Company’s CNS pipeline includes four differentiated product candidates 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), treatment resistant depression (TRD), Alzheimer’s disease (AD) agitation, and smoking cessation. AXS-05 has been granted
U.S. Food and Drug Administration (FDA) Breakthrough Therapy designation for the treatment of MDD and Fast Track designations for the treatment of TRD and for the treatment of AD agitation.
Depression: Axsome remains on track to submit a New Drug Application (NDA) for AXS-05 in MDD to the FDA in the fourth quarter of 2020. The NDA is supported by positive efficacy results from the ASCEND and GEMINI trials. A Phase 3, open-label, long-term safety extension study of AXS-05 in patients with MDD and TRD is ongoing to further support the NDA filing. To date, more than 800 patients have been dosed in this trial.
InMarch 2020 , Axsome announced results from the Phase 3 STRIDE-1 study, a randomized, double-blind, active-controlled, multicenter,U.S. trial, in patients with confirmed TRD. In this study, AXS-05 met key secondary endpoints by rapidly and statistically significantly improving symptoms of depression as compared to the active comparator bupropion. A second Phase 3 trial of AXS-05 in TRD is planned for the third quarter of 2020.
AD Agitation: InApril 2020 , Axsome announced positive results from the Phase 2/3 ADVANCE-1 study, a randomized, double-blind, controlled, multicenter,U.S. trial in patients with AD agitation. In this study, AXS-05 met the primary endpoint by rapidly, substantially, and statistically significantly improving agitation in patients with AD as compared to placebo. Axsome intends to meet with the FDA to discuss these results and next steps in this development program.
Smoking Cessation: Axsome plans to meet with the FDA in the second half of 2020 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 betweenAxsome andDuke 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: Axsome remains on track to submit an NDA for AXS-07 in the acute treatment of migraine to the FDA in the fourth quarter of 2020. The NDA is supported by positive efficacy results from the MOMENTUM and INTERCEPT trials. A Phase 3, open-label, long-term safety extension study of AXS-07 is ongoing to further support the NDA filing. To date, more than 700 patients have been dosed in this trial.
InApril 2020 , Axsome announced positive results from the Phase 3 INTERCEPT study, a randomized, double-blind, placebo-controlled, multicenter,U.S. trial, in the early treatment of migraine. In this study, AXS-07 met the two co-primary endpoints resulting in significantly greater rates of freedom from migraine pain and most bothersome migraine-associated symptoms as compared to placebo. AXS-07 also substantially and significantly prevented progression of migraine pain intensity. - 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 Orphan Drug Designation by the FDA for the treatment of narcolepsy.
Narcolepsy: Axsome is on track to initiate Phase 3 trials of AXS-12 in the treatment of narcolepsy in the second half of 2020. Axsome previously announced positive results from the Phase 2 CONCERT study, in which AXS-12 significantly reduced the number of cataplexy attacks and excessive daytime sleepiness as compared to placebo in patients with narcolepsy. - 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 plans to meet with the FDA in the second half of 2020 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.
Anticipated Milestones
- NDA Submissions:
• AXS-05 in the treatment of MDD (4Q 2020)
• AXS-07 in the acute treatment of migraine (4Q 2020) - FDA Meetings:
• AXS-14 for fibromyalgia (2H 2020)
• AXS-05 for smoking cessation (2H 2020) - Clinical Trial Initiations:
• Phase 3 trial of AXS-05 in TRD (3Q 2020)
• Phase 3 trials of AXS-12 in narcolepsy (2H 2020)
• Phase 3 trial of AXS-05 in Alzheimer’s disease agitation (2H 2020)
First Quarter 2020 Financial Results
- Research and development (R&D) expenses: R&D expenses were
$27.5 million for the quarter endedMarch 31, 2020 and$7.6 million for the comparable period in 2019. R&D expense in the quarter included a one-time charge of$10.2 million for the Pfizer license agreement, of which$7.2 million was non-cash related. The remaining increase was due primarily to ongoing spend for our active clinical trials in the quarter which included the STRIDE-1, ADVANCE-1, and INTERCEPT trials, close-out costs for our previously completed GEMINI, MOMENTUM, and CONCERT trials, along with costs associated with the AXS-05 and AXS-07 open-label safety studies. - General and administrative (G&A) expenses: G&A expenses were
$5.0 million for the quarter endedMarch 31, 2020 and$2.8 million for the comparable period in 2019. The change was primarily due to personnel costs, mainly associated with an increase in stock compensation expense, along with the build-out of the commercial function. - Net loss: Net loss was
$32.5 million , or$(0.88) per share for the quarter endedMarch 31, 2020 , compared to a net loss of$10.6 million , or$(0.32) per share for the comparable period in 2019. - Cash: At
March 31, 2020 , Axsome had$197.3 million of cash compared to$220.0 million of cash atDecember 31, 2019 . - Shares outstanding: At
March 31, 2020 , Axsome had 37,075,422 shares of common stock outstanding. - Financial guidance: Axsome believes that its cash at
March 31, 2020 will be sufficient to fund the company’s anticipated operations, based on its current operating plans, for at least two years.
Conference Call Information
Axsome will host a conference call and webcast with slides today at
About
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 | ||||||||
2020 | 2019 | |||||||
Operating expenses: | ||||||||
Research and development | $ | 27,521,400 | $ | 7,603,081 | ||||
General and administrative | 4,970,057 | 2,818,392 | ||||||
Total operating expenses | 32,491,457 | 10,421,473 | ||||||
Loss from operations | (32,491,457 | ) | (10,421,473 | ) | ||||
Interest income (expense) | 7,311 | (218,903 | ) | |||||
Net loss | $ | (32,484,146 | ) | $ | (10,640,376 | ) | ||
Net loss per common share, basic and diluted | $ | (0.88 | ) | $ | (0.32 | ) | ||
Weighted average common shares outstanding, basic and diluted | 37,061,356 | 33,052,468 | ||||||
Balance Sheet Information:
Cash and cash equivalents | $ | 197,313,408 | $ | 219,966,167 | |||
Total assets | 197,800,871 | 220,549,760 | |||||
Loan payable, current and long-term | 20,112,570 | 19,934,918 | |||||
Accumulated deficit | (208,379,639 | ) | (175,895,493 | ) | |||
Stockholders’ equity | $ | 156,156,866 | $ | 178,722,389 | |||
Axsome Contact:
Chief Operating Officer
Tel: 212-332-3243
Email: mjacobson@axsome.com
www.axsome.com
Source: Axsome Therapeutics, Inc.