Full Press Release Details
Axsome Therapeutics Reports Fourth Quarter and Full Year 2019 Financial Results and Provides Business Update
NDA filing for Breakthrough Therapy designated AXS-05 in MDD expected in 4Q 2020
NDA filing for AXS-07 in migraine expected in 4Q 2020
Topline results for STRIDE-1 Phase 3 trial of AXS-05 in TRD on track for 1Q 2020
Topline results for INTERCEPT Phase 3 trial of AXS-07 in treatment of migraine on track for 1Q 2020
Topline results for ADVANCE Phase 2/3 trial of AXS-05 in Alzheimer's disease agitation anticipated in 3Q 2020
Initiation of Phase 3 trial of AXS-12 in narcolepsy anticipated in 2020
Company to host conference call today at 8:00 AM Eastern
NEW YORK, March 12, 2020 (Globe Newswire) Axsome Therapeutics, Inc. (NASDAQ: AXSM), a clinical-stage biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders, today reported financial results for the fourth quarter and year ended December 31, 2019.
Axsome delivered on several transformative achievements in 2019, including positive NDA-enabling clinical trial readouts for AXS-05 in depression and for AXS-07 in migraine, propelling Axsome potentially towards commercial stage as early as 2021, said Herriot Tabuteau, MD, Chief Executive Officer of Axsome. In addition, our AXS-12 product candidate for narcolepsy achieved positive Phase 2 results and is progressing to Phase 3, and through our agreement with Pfizer earlier this year, we expanded our Phase 3 pipeline with the addition of AXS-14 for the treatment of fibromyalgia.
Dr. Tabuteau said, We expect continued operational momentum in 2020 with the anticipated filing of the two NDAs for AXS-05 in depression and for AXS-07 in migraine, which we expect to occur in the fourth quarter. Furthermore, we are on track for readouts from our STRIDE-1 Phase 3 trial of AXS-05 in treatment resistant depression and our INTERCEPT Phase 3 trial of AXS-07 in early treatment of migraine, both by the end of this month. We expect readouts from our ADVANCE-1 Phase 2/3 trial of AXS-05 in Alzheimer's disease agitation in the third quarter. By providing new mechanisms of action, and potentially faster, greater and broader efficacy as compared to currently available treatments, our investigational medicines have the potential to change the current standard of care for difficult-to-treat CNS disorders and transform the lives of patients living with these conditions.
Axsome is developing a portfolio of differentiated, patent-protected, central nervous system (CNS) product candidates. For the many people facing unsatisfactory treatments for CNS disease, Axsome accelerates the invention and adoption of life-changing medicines. The Company's CNS pipeline includes four differentiated product candidates in active clinical development.
Depression: In December 2019, Axsome announced positive results from the Phase 3 GEMINI study, a randomized, double-blind, placebo-controlled, multicenter, U.S. trial, in patients with confirmed moderate to severe MDD. In this study, AXS-05 met the primary endpoint by rapidly, substantially, and statistically significantly improving symptoms of depression as compared to placebo. The positive results from the GEMINI trial, along with the previously completed ASCEND trial of AXS-05 in MDD, support an NDA filing for AXS-05 in the treatment of MDD, which is anticipated in the fourth quarter of 2020.
Randomization into the STRIDE-1 Phase 3 active-controlled trial of AXS-05 in TRD has been completed. Axsome remains on track to report topline results from STRIDE-1 in the first quarter of 2020.
To support the planned NDA filing of AXS-05 in MDD, a Phase 3, open-label, long-term safety extension study of AXS-05 in patients with MDD and TRD is ongoing.
AD Agitation: Axsome is enrolling the ADVANCE-1 study, a Phase 2/3, randomized, double-blind, controlled, multicenter trial to evaluate the efficacy and safety of AXS-05 in patients with Alzheimer's disease agitation. To date, more than 80% of the target number of subjects have been randomized in this trial. Based on current enrollment, we expect topline results from this trial in the third quarter of 2020.
Smoking Cessation: Axsome plans to meet with the FDA in 2020 to discuss the continued clinical development of AXS-05 as an aid to smoking cessation treatment.
Migraine: In December 2019, Axsome announced positive results from the Phase 3 MOMENTUM study, a randomized, double-blind, placebo- and active-controlled, multicenter, U.S. trial, in migraine patients with a history of inadequate response to prior acute treatments. The study incorporated the potent active comparator rizatriptan. In this study, AXS-07 met the two regulatory 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 met the key secondary endpoint, demonstrating statistically significant superiority to the active comparator rizatriptan on sustained freedom from migraine pain. MOMENTUM was conducted pursuant to an FDA Special Protocol Assessment (SPA). The positive results from the MOMENTUM trial support an NDA filing for AXS-07 in the acute treatment of migraine, which is anticipated in the fourth quarter of 2020.
Enrollment in the Phase 3 INTERCEPT study, a placebo-controlled trial of AXS-07 in the early treatment of migraine has been completed. Axsome remains on track to report topline results from INTERCEPT in the first quarter of 2020.
To support the planned NDA filing of AXS-07 in the acute treatment of migraine, enrollment in a Phase 3 open-label, long-term safety extension study of AXS-07 is ongoing.
Narcolepsy: In December 2019, Axsome announced positive results from the Phase 2 CONCERT study, a randomized, double-blind, placebo-controlled, crossover, multicenter, U.S. trial, of AXS-12 in patients with narcolepsy. In this study, AXS-12 met the prespecified primary endpoint and significantly reduced the number of cataplexy attacks as compared to placebo. AXS-12 also significantly reduced excessive daytime sleepiness (EDS), and improved cognitive function, sleep quality and sleep-related symptoms as compared to placebo.
Axsome plans to initiate Phase 3 trials of AXS-12 in the treatment of narcolepsy in 2020.
Fibromyalgia: In January 2020, Axsome obtained exclusive U.S. rights from Pfizer, Inc. for the development and commercialization of AXS-14 (esreboxetine), which has demonstrated positive and statistically significant results in a Phase 3 and a Phase 2 trial in the treatment of fibromyalgia. In a Phase 3 trial conducted by Pfizer in 1,122 patients with fibromyalgia treated with esreboxetine or placebo for 14 weeks, esreboxetine met the two primary endpoints demonstrating statistically significant improvements compared to placebo in the weekly mean pain score (p<0.001), and the Fibromyalgia Impact Questionnaire (FIQ) total score (p<0.001). Esreboxetine also resulted in statistically significant improvements as compared to placebo in fatigue as measured using the Global Fatigue Index (p=0.001).
Axsome plans to meet with the FDA in 2020 to discuss the further clinical development of AXS-14 for the treatment of fibromyalgia.
Anticipated Milestones
Updating Investor Conferences
Axsome is scheduled to participate in the following upcoming investor conferences over the next two months:
Fourth Quarter 2019 Financial Results
Conference Call Information
Axsome will host a conference call and webcast today at 8:00 AM Eastern to discuss fourth quarter and full year 2019 financial results as well as to provide a corporate update. To participate in the live conference call, please dial (844) 698-4029 (toll-free domestic) or (647) 253-8660 (international), and use the conference ID 2067997. The live webcast can be accessed on the "Webcasts & Presentations" page of the "Investors" section of the Company's website at axsome.com. A replay of the webcast will be available for approximately 30 days following the live event.
About Axsome Therapeutics, Inc.
Axsome Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders for which there are limited treatment options. Axsome's core CNS product candidate portfolio includes five clinical-stage candidates, AXS-05, AXS-07, AXS-09, AXS-12, and AXS-14. AXS-05 is currently in a Phase 3 trial in treatment resistant depression (TRD), a Phase 2/3 trial in agitation associated with Alzheimer's disease (AD), and is being developed for major depressive disorder (MDD). AXS-05 is also being developed for smoking cessation treatment. AXS-07 is currently in a Phase 3 trial for the acute treatment of migraine. AXS-12 is being developed for the treatment of narcolepsy. AXS-14 is being developed for the treatment of fibromyalgia. AXS-05, AXS-07, AXS-09, AXS-12, and AXS-14 are investigational drug products not approved by the FDA. For more information, please visit the Company's website at axsome.com. The Company may occasionally disseminate material, nonpublic information on the company website.
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 U.S. Food and Drug Administration ( FDA ) or other regulatory authority approval of, or other action with respect to, our product candidates (including, but not limited to, FDA's agreement with the Company's plan to discontinue the bupropion treatment arm of the ADVANCE-1 study in accordance with the independent data monitoring committee's recommendations); the potential for the MOMENTUM clinical trial to provide a basis for approval of AXS-07 for the acute treatment of migraine in adults with or without aura, pursuant to our special protocol assessment; the potential for the ASCEND clinical trial, combined with the GEMINI clinical trial results, to provide a basis for approval of AXS-05 for the treatment of major depressive disorder and accelerate its development timeline and commercial path to patients; the Company's ability to successfully defend its intellectual property or obtain the necessary licenses at a cost acceptable to the Company, if at all; the successful implementation of the Company's research and development programs and collaborations; the success of the Company's license agreements; the acceptance by the market of the Company's product candidates, if approved; the Company's anticipated capital requirements, including the Company's anticipated cash runway; and other factors, including general economic conditions and regulatory developments, not within the Company's control. The factors discussed herein could cause actual results and developments to be materially different from those expressed in or implied by such statements. The forward-looking statements are made only as of the date of this press release and the Company undertakes no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstance.
Axsome Therapeutics, Inc.
Selected Consolidated Financial Data
Statements of Operations Information:
| Three months ended | Twelve months ended | ||||||||||||
| December 31, | December 31, | ||||||||||||
| 2019 | 2018 | 2019 | 2018 | ||||||||||
| Operating expenses: | |||||||||||||
| Research and development | $ | 19,205,271 | $ | 7,151,232 | $ | 53,647,067 | $ | 23,495,055 | |||||
| General and administrative | 5,222,899 | 2,299,083 | 13,598,030 | 9,351,522 | |||||||||
| Total operating expenses | 24,428,170 | 9,450,315 | 67,245,097 | 32,846,577 | |||||||||
| Loss from operations | (24,428,170 | ) | (9,450,315 | ) | (67,245,097 | ) | (32,846,577 | ) | |||||
| Interest and amortization of debt discount (expense) | (378,814 | ) | (248,700 | ) | (1,239,537 | ) | (1,127,305 | ) | |||||
| Tax Credit | 139,448 | 217,418 | |||||||||||
| Change in fair value of warrant liability | 102.000 | 2,791,000 | |||||||||||
| Net loss | $ | (24,806,984 | ) | $ | (9,597,015 | ) | $ | (68,345,186 | ) | $ | (30,965,464 | ) | |
| Net loss per common share, basic and diluted | $ | (0.71 | ) | $ | (0.32 | ) | $ | (2.01 | ) | $ | (1.15 | ) | |
| Weighted average common shares outstanding, basic and diluted | 34,757,910 | 29,874,410 | 34,020,257 | 26,883,656 |
Balance Sheet Information:
| December 31, 2019 | December 31, 2018 | ||||||
| Cash and cash equivalents | $ | 219,966,167 | $ | 13,968,742 | |||
| Total assets | 220,549,760 | 15,379,279 | |||||
| Loan payable, current and long-term | 19,934,918 | 6,910,814 | |||||
| Accumulated deficit | (175,895,493 | ) | (107,550,307 | ) | |||
| Stockholders' equity | $ | 178,722,389 | $ | 937,921 |
Chief Operating Officer
Axsome Therapeutics, Inc.
200 Broadway, 3rd Floor
Email: mjacobson@axsome.com