Axsome Therapeutics Reports Fourth Quarter and Full Year 2020 Financial Results and Provides Business Update
Company to host conference call today at
“The focused execution of the Axsome team made 2020 a year of significant accomplishments. We had successful pre-NDA meetings with the FDA for AXS-05 in major depressive disorder and for AXS-07 in migraine, reported positive results from the pivotal ADVANCE-1 trial of AXS-05 in Alzheimer’s disease agitation, initiated the second pivotal trial of AXS-05 in this indication, received two new FDA Breakthrough Therapy designations, and built out our commercialization infrastructure,” said
For the many people living with serious CNS disorders, Axsome accelerates the invention and development of life-changing medicines. The Company is developing a portfolio of differentiated, patent-protected, CNS product candidates with four in active clinical development.
- AXS-05: AXS-05 (dextromethorphan-bupropion) 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.
Depression: Axsome has submitted a New Drug Application (NDA) to the FDA for AXS-05 for the treatment of MDD. The Company intends to issue a press release following the FDA’s decision on the filing of the application.
December 2020, Axsome announced positive results from the COMET Phase 3 long-term trial in patients with MDD and the three Phase 2 open-label MDD efficacy trials in patients who had failed one prior antidepressant (COMET-AU), patients who had failed two prior antidepressants (COMET-TRD), and patients with suicidal ideation. Patients treated with AXS-05 in these trials experienced rapid, substantial, and durable improvement in depressive symptoms and functional impairment that was sustained over the 12-month treatment period. AXS-05 was well tolerated over the long-term treatment period with a safety profile consistent with that observed in the previously reported controlled trials.
AD Agitation: In
December 2020, Axsome initiated the ACCORD study, a Phase 3, randomized, double-blind, placebo-controlled, multicenter, randomized withdrawal trial to evaluate the efficacy and safety of AXS-05 in the treatment of Alzheimer’s disease (AD) agitation.
Smoking Cessation: Axsome is scheduled to meet with the FDA in the third 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: Axsome is compiling the NDA for AXS-07 for the acute treatment of migraine. Submission of this NDA is expected in early second quarter.
December 2020, Axsome announced positive results from the MOVEMENT Phase 3 long-term trial of AXS-07 in the acute treatment of migraine. In this trial, treatment with AXS-07 resulted in rapid, substantial, and durable relief of migraine pain and associated symptoms. AXS-07 was well tolerated over the 12-month treatment period with a safety profile consistent with that observed in the previously reported controlled trials.
- 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 Orphan Drug designation for the treatment of narcolepsy as well as Breakthrough Therapy designation for the treatment of cataplexy in patients with narcolepsy.
Narcolepsy: Axsome is planning to initiate a Phase 3 trial of AXS-12 in the treatment of narcolepsy in the second quarter. The planned Phase 3 trial will be a randomized, double-blind, placebo-controlled, parallel-group study.
- 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 second quarter, 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 in a Phase 2 trial for the treatment of fibromyalgia.
- Launch readiness activities continue to advance and all functional commercial leadership is in place. Sales force structure and design have been finalized and the hiring of sales managers and representatives is expected to commence in the coming months. The infrastructure for Axsome’s proprietary DCC™, or digital centric commercialization, platform is in place, and payer engagement activities have started.
January 2021, Axsome appointed Kevin Laliberte, PharmD, as Executive Vice President, Product Strategy. Prior to Axsome, Dr. Lalibertewas Senior Vice President, Product Development at Dova Pharmaceuticals. Prior to Dova, he was Senior Vice President, Product Development and Clinical Operations at United Therapeutics, where he was responsible for all development activities for small molecules. Dr. Laliberteearned his Doctor of Pharmacy degree from the University of Michigan, Ann Arborand completed his post doctorate fellowship at the University of North Carolina, Chapel Hilland GlaxoSmithKline.
- NDA Submissions:
- AXS-07 for the acute treatment of migraine, submission (early 2Q 2021)
- Clinical Trial Readouts:
- Phase 2 MERIT trial of AXS-05 in TRD, topline data (2H 2021)
- Clinical Trial Initiations:
- Phase 3 trial of AXS-12 in the treatment of narcolepsy (2Q 2021)
- FDA Meetings:
- AXS-14 for fibromyalgia (2Q 2021)
- AXS-05 for smoking cessation (3Q 2021)
Financial Results for the Fourth Quarter and Full Year 2020
- Research and development (R&D) expenses: R&D expenses were
$17.4 millionfor the quarter ended December 31, 2020and $19.2 millionfor the comparable period in 2019. The decrease was due to the completion of our Phase 3 efficacy and safety trials for AXS-05 and AXS-07. R&D expenses for the year ended December 31, 2020were $70.2 million, compared to $53.6 millionfor the comparable period in 2019. The increase was primarily due to a one-time upfront charge related to our licensing agreement with Pfizer along with costs associated with NDA preparations.
- General and administrative (G&A) expenses: G&A expenses were
$10.4 millionfor the quarter ended December 31, 2020and $5.2 millionfor the comparable period in 2019. The increase was primarily due to increased personnel costs, mainly associated with stock compensation expense, along with the build-out of the commercial function. G&A expenses for the year ended December 31, 2020were $28.9 million, compared to $13.6 millionfor the comparable period in 2019. The increase was primarily due to an increase in stock compensation expense along with the build-out of the commercial function.
- Net loss: Net loss was
$29.2 million, or $(0.78)per share for the quarter ended December 31, 2020compared to a net loss of $24.8 million, or $(0.71)per share for the comparable period in 2019. Net loss for the year ended December 31, 2020was $102.9 million, or $(2.77)per share, of which $22.3 millionwere non-cash charges including stock compensation expense. Net loss for the year ended December 31, 2019was $68.3 million, or $(2.01)per share, of which $6.1 millionwere non-cash charges consisting of stock compensation expense.
- Cash: At
December 31, 2020, Axsome had $183.9 millionof cash compared to $220.0 millionat December 31, 2019.
- Shares outstanding: At
December 31, 2020, Axsome had 37,374,088 shares of common stock outstanding.
- Axsome expects that its operating expenses will increase year over year in 2021 to support continued pipeline advancement and build-out of the commercial function.
- Axsome believes that its cash at
December 31, 2020, along with the remaining committed capital from the $225 millionterm loan facility, is sufficient to fund anticipated operations, based on the current operating plan which includes costs for the commercial launch of AXS-05 in MDD and AXS-07 in migraine, into at least 2024.
Conference Call Information
Axsome will host a conference call and webcast today at
Forward Looking Statements
Certain matters discussed in this press release are “forward-looking statements”. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. In particular, the Company’s statements regarding trends and potential future results are examples of such forward-looking statements. The forward-looking statements include risks and uncertainties, including, but not limited to, the success, timing and cost of our ongoing clinical trials and anticipated clinical trials for our current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including our ability to fully fund our disclosed clinical trials, which assumes no material changes to our currently projected expenses), futility analyses and receipt of interim results, which are not necessarily indicative of the final results of our ongoing clinical trials, and the number or type of studies or nature of results necessary to support the filing of a new drug application (“NDA”) for any of our current product candidates; our ability to fund additional clinical trials to continue the advancement of our product candidates; the timing of and our ability to obtain and maintain
Selected Consolidated Financial Data
Statements of Operations Information:
|Three months ended||Twelve months ended|
|Research and development||$||17,384,729||$||19,205,271||$||70,244,579||$||53,647,067|
|General and administrative||10,359,507||5,222,899||28,896,749||13,598,030|
|Total operating expenses||27,744,236||24,428,170||99,141,328||67,245,097|
|Loss from operations||(27,744,236||)||(24,428,170||)||(99,141,328||)||(67,245,097||)|
|Interest and amortization of debt discount (expense)||(1,473,989||)||(378,814||)||(2,565,838||)||(1,239,537||)|
|Loss on extinguishment of debt||—||—||(1,247,012||)||—|
|Net loss per common share, basic and diluted||$||(0.78||)||$||(0.71||)||$||(2.77||)||$||(2.01||)|
|Weighted average common shares outstanding, basic and
Balance Sheet Information:
|Cash and cash equivalents||$||183,876,453||$||219,966,167|
|Loan payable, current and long-term||48,321,848||19,934,918|
Source: Axsome Therapeutics, Inc.