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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549

 

FORM 8-K

 

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): May 05, 2025

 

 

Axsome Therapeutics, Inc.

(Exact name of Registrant as Specified in Its Charter)

 

 

Delaware

001-37635

45-4241907

(State or Other Jurisdiction
of Incorporation)

(Commission File Number)

(IRS Employer
Identification No.)

 

 

 

 

 

One World Trade Center, 29th Floor

 

New York, New York

 

10007

(Address of Principal Executive Offices)

 

(Zip Code)

 

Registrant’s Telephone Number, Including Area Code: (212) 332-3241

 

 

 

(Former Name or Former Address, if Changed Since Last Report)

 

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

☐Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
☐Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
☐Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
☐Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:


Title of each class

 

Trading
Symbol(s)

 


Name of each exchange on which registered

Common Stock, Par Value $0.0001 Per Share

 

AXSM

 

Nasdaq Global Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).

Emerging growth company ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

 


Item 2.02 Results of Operations and Financial Condition.

On May 5, 2025, Axsome Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the three months ended March 31, 2025 and provided an update on the Company’s operations. The Company is furnishing a copy of the press release, which is attached hereto as Exhibit 99.1.

In accordance with General Instruction B.2 of Form 8-K, the information included in Item 2.02 of this Current Report on Form 8-K (including Exhibit 99.1 hereto), shall not be deemed “filed” for the purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference into any filing made by the Company under the Exchange Act or Securities Act of 1933, as amended, except as shall be expressly set forth by specific reference in such a filing.

Item 8.01 Other Events.

On May 5, 2025, the Company updated its corporate presentation and posted such corporate presentation to the Company’s website. The updated corporate presentation is filed as Exhibit 99.2 hereto and incorporated by reference herein.

Item 9.01 Financial Statements and Exhibits.

 

(d) Exhibits

 

Exhibit No.

Description

99.1

Press Release dated May 5, 2025.

99.2

 

Corporate Presentation.

104

Cover Page Interactive Data File (embedded within the Inline XBRL document).

 


SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

 

 

 

Axsome Therapeutics, Inc.

 

 

 

 

Date:

May 5, 2025

By:

/s/ Herriot Tabuteau, M.D.

 

 

Name:

Title:

Herriot Tabuteau, M.D.
President and Chief Executive Officer

 


EX-99.1 2 axsm-ex99_1.htm EX-99.1 EX-99.1

img36028404_0.jpg

Exhibit 99.1

 

Axsome Therapeutics Reports First Quarter 2025 Financial Results and Provides Business Update

Total 1Q 2025 net product revenue of $121.5 million, representing 62% year-over-year growth

AUVELITY® 1Q 2025 net product sales of $96.2 million, representing 80% year-over-year growth

SUNOSI® 1Q 2025 net product revenue of $25.2 million, representing 17% year-over-year growth

SYMBRAVO® approved for the acute treatment of migraine; commercial launch on track for June 2025

NDA for AXS-14 for the management of fibromyalgia submitted to the FDA

Supplemental NDA submission for AXS-05 in Alzheimer’s disease agitation on track for 3Q 2025

NDA submission for AXS-12 for cataplexy in patients with narcolepsy anticipated in 2H 2025

Positive topline results of FOCUS Phase 3 trial of solriamfetol in ADHD announced

Initiation of Phase 3 trial of solriamfetol in major depressive disorder with excessive daytime sleepiness anticipated in 2025

Positive topline results of EMERGE Phase 3 trial of SYMBRAVO in migraine patients with prior inadequate response to oral CGRP inhibitors announced

Company to host conference call today at 8:00 AM Eastern

NEW YORK, May 5, 2025 (Globe Newswire) – Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company leading a new era in the treatment of central nervous system (CNS) disorders, today announced financial results for the first quarter of 2025 and provided a general business update.

“The first quarter was strong and eventful, with the approval of SYMBRAVO for the acute treatment of migraine, continued growth of AUVELITY and SUNOSI, and significant advancements across our broad late-stage neuroscience pipeline,” said Herriot Tabuteau, MD, Chief Executive Officer of Axsome Therapeutics. “Commercial availability of SYMBRAVO is on track for this June, and preparations are underway to enable a successful launch. We have submitted our NDA for AXS-14 for the management of fibromyalgia, and are on track for our planned NDA submissions this year for AXS-05 in Alzheimer’s disease agitation and for AXS-12 in cataplexy in patients with narcolepsy. Our development programs with solriamfetol continue to advance, with the recently announced Phase 3 topline results in ADHD and MDD, and progress across the Phase 3 trials in binge eating disorder and excessive sleepiness associated with shift work disorder. Overall, our neuroscience portfolio positions us to deliver potentially five marketed products across ten indications that collectively impact over 150 million patients in the U.S. alone. We look forward to continued momentum over the balance of 2025 as we execute on our mission to deliver transformative medicines to patients living with serious CNS conditions.”

First Quarter 2025 Financial Highlights

Total net product revenue was $121.5 million for the first quarter of 2025, representing 62% year-over-year growth. Total net product revenue for the comparable period in 2024 was $75.0 million.
AUVELITY net product sales were $96.2 million for the first quarter of 2025, representing 80% year-over-year growth. AUVELITY net product sales for the comparable period in 2024 were $53.4 million.

1


 

 

SUNOSI net product revenue was $25.2 million for the first quarter of 2025, representing 17% year-over-year growth, which consisted of $24.1 million in net product sales and $1.1 million in royalty revenue associated with SUNOSI sales in out-licensed territories. SUNOSI net product revenue for the comparable period in 2024 was $21.6 million, which consisted of $20.7 million in net product sales and $0.9 million in royalty revenue.
Total cost of revenue was $9.8 million for the first quarter of 2025. Total cost of revenue for the comparable period in 2024 was $6.3 million.
Research and development (R&D) expenses were $44.8 million for the first quarter of 2025, compared to $36.8 million for the comparable period in 2024. The increase was primarily related to the Company’s Phase 3 trials of solriamfetol, manufacturing costs for the planned launch of SYMBRAVO, and higher personnel costs, including non-cash stock-based compensation, associated with organizational growth.
Selling, general, and administrative (SG&A) expenses were $120.8 million for the first quarter of 2025, compared to $99.0 million for the comparable period in 2024. The increase was primarily related to commercialization activities for AUVELITY, including sales force and marketing expenses, pre-launch activities for SYMBRAVO, and higher personnel costs, including non-cash stock-based compensation, associated with organizational growth.
Net loss for the first quarter of 2025 was $59.4 million, or $(1.22) per share, compared to a net loss of $68.4 million, or $(1.44) per share, for the comparable period in 2024. The net loss in the first quarter of 2025 includes $26.2 million in non-cash charges, comprised primarily of $23.3 million in stock-based compensation expense.
Cash and cash equivalents totaled $300.9 million at March 31, 2025, compared to $315.4 million at December 31, 2024.
Shares of common stock outstanding were 49,216,759 at March 31, 2025.

Financial Guidance

Axsome believes that its current cash is sufficient to fund anticipated operations into cash flow positivity, based on the current operating plan.

Commercial Highlights

AUVELITY

Approximately 167,000 prescriptions were written for AUVELITY in the first quarter of 2025, representing an increase of 76% compared to the same period in 2024, and an increase of 5% compared to the fourth quarter of 2024.
Payer coverage for AUVELITY across all channels is approximately 78% of all covered lives. The proportion of lives covered for AUVELITY in the commercial and government (Medicare and Medicaid) channels are approximately 63% and 100%, respectively.

SUNOSI

Approximately 46,000 prescriptions were written for SUNOSI in the U.S. in the first quarter of 2025, representing an increase of 12% compared to the same period in 2024, and a decrease of 5% compared to the fourth quarter of 2024.
Payer coverage for SUNOSI across all channels is approximately 83% of all covered lives. The proportion of lives covered for SUNOSI in the commercial and government channels are approximately 95% and 60%, respectively.

2


 

 

SYMBRAVO

In January 2025, the U.S. Food and Drug Administration (FDA) approved SYMBRAVO for the acute treatment of migraine with or without aura in adults. SYMBRAVO represents a novel, multi-mechanistic approach to treating migraine that targets multiple pathways underlying a migraine attack, and is engineered with Axsome’s patented MoSEIC™ (Molecular Solubility Enhanced Inclusion Complex) rapid absorption technology. Preparations for the commercial launch of SYMBRAVO remain on track, with commercial availability anticipated in June 2025.

Development Pipeline

Axsome is advancing an industry-leading neuroscience pipeline of multiple, innovative, late-stage, patent-protected product candidates addressing a broad range of serious psychiatric and neurological conditions. Recent and anticipated progress for key pipeline programs is summarized below.

AXS-05

AXS-05 (dextromethorphan-bupropion) is Axsome’s novel, oral, investigational N-methyl-D-aspartate (NMDA) receptor antagonist, sigma-1 agonist, and aminoketone CYP2D6 inhibitor being developed for the treatment of Alzheimer’s disease (AD) agitation and smoking cessation. AXS-05 has been granted FDA Breakthrough Therapy designation for AD agitation.

Alzheimer’s Disease Agitation: In March 2025, Axsome received formal pre-NDA meeting minutes from the FDA for AXS-05 in AD agitation, supporting a supplemental NDA (sNDA) submission. AXS-05 was granted Breakthrough Therapy designation for the treatment of AD agitation in June 2020. The Company is on track to submit the sNDA in the third quarter of 2025.
Smoking Cessation: Axsome plans to initiate a pivotal Phase 2/3 trial of AXS-05 in smoking cessation in 2025.

Solriamfetol

Solriamfetol is Axsome’s dopamine and norepinephrine reuptake inhibitor (DNRI), TAAR1 agonist, and 5-HT1A agonist being developed for the treatment of attention deficit hyperactivity disorder (ADHD), major depressive disorder (MDD) with EDS, binge eating disorder (BED), and excessive sleepiness associated with shift work disorder (SWD).

Attention Deficit Hyperactivity Disorder: In March 2025, Axsome announced positive topline results from the FOCUS Phase 3 trial evaluating the efficacy and safety of solriamfetol in ADHD in adults. The study achieved its primary and key secondary endpoints demonstrating substantial and statistically significant improvements in ADHD symptoms and disease severity with solriamfetol compared to placebo. Solriamfetol was safe and well tolerated in the trial, with a side effect profile consistent with the established safety profile of solriamfetol.

The Company plans to initiate a Phase 3 trial of solriamfetol in ADHD in pediatric patients in 2025.

Major Depressive Disorder: In April 2025, Axsome announced topline results from the PARADIGM Phase 3 proof-of-concept trial evaluating the efficacy and safety of solriamfetol in MDD with and without excessive daytime sleepiness (EDS). In the prespecified subgroup of patients with severe EDS, solriamfetol led to numerically greater improvements in depressive symptoms. Solriamfetol was safe and well tolerated in the trial, with a side effect profile consistent with the established safety profile of solriamfetol.

The Company plans to initiate a Phase 3 trial of solriamfetol in MDD patients with EDS in 2025.

3


 

 

Binge Eating Disorder: Axsome is conducting the ENGAGE study, a Phase 3, randomized, double-blind, placebo-controlled, multicenter trial evaluating the efficacy and safety of solriamfetol in BED. The Company anticipates topline results from the ENGAGE Phase 3 trial in 2026.
Shift Work Disorder: Axsome is conducting the SUSTAIN study, a Phase 3, randomized, double-blind, placebo-controlled, multicenter trial evaluating the efficacy and safety of solriamfetol in SWD in adults. The Company anticipates topline results from the SUSTAIN Phase 3 trial in 2026.

SYMBRAVO

SYMBRAVO (MoSEIC™ meloxicam-rizatriptan) is Axsome’s novel, oral, rapidly absorbed, multi-mechanistic, selective COX-2 inhibitor and 5-HT1B/1D agonist indicated for the acute treatment of migraine with or without aura in adults.

Migraine: In February 2025, Axsome announced positive topline results from the EMERGE Phase 3 open-label trial of SYMBRAVO in patients experiencing inadequate response to oral CGRP inhibitors. The study achieved its primary endpoint by demonstrating statistically significantly greater migraine treatment response with SYMBRAVO compared to prior oral CGRP inhibitors, as assessed by the Migraine Treatment Optimization Questionnaire (mTOQ-4). In the trial, SYMBRAVO rapidly and substantially improved migraine pain and most bothersome symptoms.

 

AXS-12

AXS-12 (reboxetine) is Axsome’s novel, oral, investigational, highly selective and potent norepinephrine reuptake inhibitor and cortical dopamine modulator being developed for the treatment of narcolepsy. AXS-12 has been granted FDA Orphan Drug designation for narcolepsy.

Narcolepsy: Axsome previously announced the completion of its clinical program of AXS-12 in patients with narcolepsy with cataplexy consisting of three controlled Phase 2 and Phase 3 efficacy trials and a long-term safety trial. AXS-12 achieved the primary endpoints and rapidly and substantially reduced cataplexy attacks, improved excessive daytime sleepiness, and improved cognitive function compared to placebo in all three efficacy trials (CONCERT, SYMPHONY, and ENCORE). The Company anticipates submitting an NDA to the FDA in the second half of 2025.

AXS-14

AXS-14 (esreboxetine) is Axsome’s novel, oral, investigational, highly selective and potent norepinephrine reuptake inhibitor being developed for the management of fibromyalgia. Esreboxetine, the SS-enantiomer of reboxetine, is more potent and selective than racemic reboxetine.

Fibromyalgia: Axsome has submitted an NDA to the FDA for AXS-14 for the management of fibromyalgia. The Company anticipates the FDA’s decision on acceptance of the filing in the second quarter of 2025.

Scientific Presentations

In April 2025, Axsome presented the positive topline results from the ACCORD-2 Phase 3 trial of AXS-05 in Alzheimer’s disease agitation and the SYMPHONY Phase 3 trial of AXS-12 in narcolepsy at the 2025 American Academy of Neurology (AAN) Annual Meeting. Additional presentations included findings from a new network meta-analysis comparing the efficacy of SYMBRAVO to oral CGRPs in the acute treatment of migraine.

4


 

 

Corporate Update

In March 2025, Axsome announced that it resolved patent litigation with Hikma Pharmaceuticals USA, Inc. (Hikma) related to Hikma’s Abbreviated New Drug Application (ANDA) for a generic version of SUNOSI. The Company entered into a license and settlement agreement with Hikma, which permits Hikma to begin selling its generic version of SUNOSI on or after September 1, 2040, if pediatric exclusivity is granted, or on or after March 1, 2040, if no pediatric exclusivity is granted, subject to FDA approval and conditions and exceptions customary for agreements of this type.

 

Anticipated Milestones

Regulatory and Commercial:
o
SYMBRAVO for the acute treatment of migraine, commercial launch (June 2025)
o
AXS-14 for fibromyalgia, FDA filing acceptance decision (2Q 2025)
o
AXS-05 for Alzheimer’s disease agitation, sNDA submission (3Q 2025)
o
AXS-12 for narcolepsy, NDA submission (2H 2025)
Clinical Trial Topline Results:
o
Phase 3 ENGAGE trial of solriamfetol in binge eating disorder (2026)
o
Phase 3 SUSTAIN trial of solriamfetol in shift work disorder (2026)
Clinical Trial Initiations and Progress:
o
Pivotal Phase 2/3 trial of AXS-05 in smoking cessation, initiation (2025)
o
Phase 3 trial of solriamfetol in ADHD in pediatric patients, initiation (2025)
o
Phase 3 trial of solriamfetol in MDD with EDS, initiation (2025)

Conference Call Information

Axsome will host a conference call and webcast today at 8:00 a.m. Eastern Time to discuss its first quarter 2025 financial results and provide a business update. To participate in the live conference call, please dial (877) 405-1239 (toll-free domestic) or +1 (201) 389-0851 (international). A live webcast of the conference call can be accessed on the “Webcasts & Presentations” page of the “Investors” section of the Company’s website at axsome.com. A replay of the conference call will be available for approximately 30 days following the live event.

About Axsome Therapeutics

Axsome Therapeutics is a biopharmaceutical company leading a new era in the treatment of central nervous system (CNS) conditions. We deliver scientific breakthroughs by identifying critical gaps in care and develop differentiated products with a focus on novel mechanisms of action that enable meaningful advancements in patient outcomes. Our industry-leading neuroscience portfolio includes FDA-approved treatments for major depressive disorder, excessive daytime sleepiness associated with narcolepsy and obstructive sleep apnea, and migraine, and multiple late-stage development programs addressing a broad range of serious neurological and psychiatric conditions that impact over 150 million people in the United States. Together, we are on a mission to solve some of the brain’s biggest problems so patients and their loved ones can flourish. For more information, please visit us at www.axsome.com and follow us on LinkedIn and X.

5


 

 

Forward Looking Statements

Certain matters discussed in this press release are “forward-looking statements”. The Company 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 commercial success of the Company’s SUNOSI®, AUVELITY®, and SYMBRAVO® products and the success of the Company’s efforts to obtain any additional indication(s) with respect to solriamfetol and/or AXS-05; the Company’s ability to maintain and expand payer coverage; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including the Company’s ability to fully fund the Company’s disclosed clinical trials, which assumes no material changes to the Company’s currently projected revenues or expenses), futility analyses and receipt of interim results, which are not necessarily indicative of the final results of the Company’s ongoing clinical trials, and/or data readouts, 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 the Company’s current product candidates; the Company’s ability to fund additional clinical trials to continue the advancement of the Company’s product candidates; the timing of and the Company’s ability to obtain and maintain U.S. Food and Drug Administration (“FDA”) or other regulatory authority approval of, or other action with respect to, the Company’s product candidates, including statements regarding the timing of any NDA submission; 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 products and product candidates, if approved; the Company’s anticipated capital requirements, including the amount of capital required for the commercialization of SUNOSI, AUVELITY, and SYMBRAVO and for the Company’s commercial launch of its other product candidates, if approved, and the potential impact on the Company’s anticipated cash runway; the Company’s ability to convert sales to recognized revenue and maintain a favorable gross to net sales; unforeseen circumstances or other disruptions to normal business operations arising from or related to domestic political climate, geo-political conflicts or a global pandemic 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 circumstances.

6


 

 

Axsome Therapeutics, Inc.

Selected Consolidated Financial Data

Axsome Therapeutics, Inc.

Consolidated Balance Sheets

(In thousands, except share and per share amounts)

 

 

March 31,
2025

 

 

December 31,
2024

 

 

 

(Unaudited)

 

 

 

 

Assets

 

 

 

 

 

 

Current assets:

 

 

 

 

 

 

Cash and cash equivalents

 

$

300,910

 

 

$

315,353

 

Accounts receivable, net

 

 

161,398

 

 

 

142,001

 

Inventories, net

 

 

16,319

 

 

 

15,732

 

Prepaid and other current assets

 

 

16,007

 

 

 

11,978

 

Total current assets

 

 

494,634

 

 

 

485,064

 

Equipment, net

 

 

773

 

 

 

584

 

Right-of-use asset - operating lease

 

 

23,294

 

 

 

5,383

 

Goodwill

 

 

12,042

 

 

 

12,042

 

Intangible asset, net

 

 

45,322

 

 

 

46,894

 

Non-current inventory and other assets

 

 

20,606

 

 

 

18,531

 

Total assets

 

$

596,671

 

 

$

568,498

 

Liabilities and stockholders’ equity

 

 

 

 

 

 

Current liabilities:

 

 

 

 

 

 

Accounts payable

 

$

85,659

 

 

$

71,997

 

Accrued expenses and other current liabilities

 

 

149,199

 

 

 

147,987

 

Operating lease liability, current portion

 

 

454

 

 

 

1,835

 

Contingent consideration, current

 

 

8,602

 

 

 

8,285

 

Total current liabilities

 

 

243,914

 

 

 

230,104

 

Contingent consideration, non-current

 

 

90,590

 

 

 

91,680

 

Loan payable, long-term

 

 

181,377

 

 

 

180,710

 

Operating lease liability, long-term

 

 

23,905

 

 

 

6,046

 

Finance lease liability, long-term

 

 

3,680

 

 

 

2,943

 

Total liabilities

 

 

543,466

 

 

 

511,483

 

Stockholders’ equity:

 

 

 

 

 

 

Preferred stock, $0.0001 par value per share (10,000,000 shares authorized, none issued and outstanding)

 

 

 

 

 

 

Common stock, $0.0001 par value per share (150,000,000 shares authorized, 49,216,759 and 48,667,587 shares issued and outstanding at March 31, 2025 and December 31, 2024, respectively)

 

 

5

 

 

 

5

 

Additional paid-in capital

 

 

1,235,400

 

 

 

1,179,797

 

Accumulated deficit

 

 

(1,182,200

)

 

 

(1,122,787

)

Total stockholders’ equity

 

 

53,205

 

 

 

57,015

 

Total liabilities and stockholders’ equity

 

$

596,671

 

 

$

568,498

 

 

7


 

 

Axsome Therapeutics, Inc.

Consolidated Statements of Operations (Unaudited)

(In thousands, except share and per share amounts)

 

 

Three months ended March 31,

 

 

 

2025

 

 

2024

 

Revenues:

 

 

 

 

 

 

Product sales, net

 

$

120,358

 

 

$

74,096

 

Royalty revenue

 

 

1,105

 

 

 

903

 

Total revenues

 

 

121,463

 

 

 

74,999

 

Operating expenses:

 

 

 

 

 

 

Cost of revenue (excluding amortization and depreciation)

 

 

9,789

 

 

 

6,297

 

Research and development

 

 

44,785

 

 

 

36,830

 

Selling, general and administrative

 

 

120,787

 

 

 

98,970

 

Loss (Gain) in fair value of contingent consideration

 

 

1,512

 

 

 

(1,412

)

Intangible asset amortization

 

 

1,572

 

 

 

1,589

 

Total operating expenses

 

 

178,445

 

 

 

142,274

 

Loss from operations

 

 

(56,982

)

 

 

(67,275

)

Interest expense, net

 

 

(2,431

)

 

 

(1,082

)

Loss before income taxes

 

 

(59,413

)

 

 

(68,357

)

Income tax expense

 

 

 

 

 

 

Net loss

 

$

(59,413

)

 

$

(68,357

)

Net loss per common share, basic and diluted

 

$

(1.22

)

 

$

(1.44

)

Weighted average common shares outstanding, basic and diluted

 

 

48,871,163

 

 

 

47,393,563

 

 

 

Investors:

Mark Jacobson

Chief Operating Officer

(212) 332-3243

mjacobson@axsome.com

Media:

Darren Opland

Director, Corporate Communications

(929) 837-1065

dopland@axsome.com

8


EX-99.2 3 axsm-ex99_2.htm EX-99.2

Slide 1

Corporate Presentation May 5, 2025


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Forward Looking Statements & Safe Harbor Certain matters discussed in this presentation are “forward-looking statements”. The Company 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 commercial success of the Company’s SUNOSI®, AUVELITY®, and SYMBRAVO® products and the success of the Company’s efforts to obtain any additional indication(s) with respect to solriamfetol and/or AXS-05; the Company’s ability to maintain and expand payer coverage; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including the Company’s ability to fully fund the Company’s disclosed clinical trials, which assumes no material changes to the Company’s currently projected revenues or expenses), futility analyses and receipt of interim results, which are not necessarily indicative of the final results of the Company’s ongoing clinical trials, and/or data readouts, 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 the Company’s current product candidates; the Company’s ability to fund additional clinical trials to continue the advancement of the Company’s product candidates; the timing of and the Company’s ability to obtain and maintain U.S. Food and Drug Administration (“FDA”) or other regulatory authority approval of, or other action with respect to, the Company’s product candidates, including statements regarding the timing of any NDA submission; 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 products and product candidates, if approved; the Company’s anticipated capital requirements, including the amount of capital required for the commercialization of SUNOSI, AUVELITY, and SYMBRAVO and for the Company’s commercial launch of its other product candidates, if approved, and the potential impact on the Company’s anticipated cash runway; the Company’s ability to convert sales to recognized revenue and maintain a favorable gross to net sales; unforeseen circumstances or other disruptions to normal business operations arising from or related to domestic political climate, geo-political conflicts or a global pandemic 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 presentation and the Company undertakes no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances. This presentation contains statements regarding the Company’s observations based upon the reported clinical data. This presentation also contains estimates and other statistical data made by independent parties and by us relating to market size and other data about the Company's industry. This data involves a number of assumptions and limitations, and you are cautioned not to give undue weight to such estimates. Neither we nor any other person makes any representation as to the accuracy or completeness of such data or undertakes any obligation to update such data after the date of this presentation. In addition, these projections, assumptions and estimates are necessarily subject to a high degree of uncertainty and risk. Axsome, AUVELITY, SUNOSI, SYMBRAVO, and MoSEIC, are trademarks or registered trademarks of Axsome Therapeutics, Inc. or its affiliates. Except as with respect to AUVELITY and SUNOSI for their approved indications, the development products referenced herein have not been approved by the FDA.


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Our Mission Develop and deliver transformative medicines for the hundreds of millions of people impacted by central nervous system conditions


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Potential to reach >150M people in the U.S. across 10 serious CNS conditions We focus on therapeutic areas with critical gaps in care and a significant unmet need for new treatment options… Major Depressive Disorder Alzheimer’s Disease Agitation Smoking Cessation ADHD Binge Eating Disorder Neurology Obstructive Sleep Apnea Migraine Narcolepsy Fibromyalgia Shift Work Disorder 21M+ ~ People in the U.S. live with MDD of patients fail to achieve remission from initial therapy 4M+ 1 people with Alzheimer’s disease experience agitation FDA-approved product 34M+ ~70% adults in the U.S. currently smoke cigarettes of smokers say they want to quit 22M+ ~ adults and children in the U.S. live with ADHD of adult ADHD patients do not receive any type of treatment 7M+ 2-3x people in the U.S. experience BED in their lifetime more likely to have psychiatric and medical comorbidities 22M+ ~80% U.S. adults are affected by OSA of patients remain undiagnosed 39M+ >70% adults in the U.S. suffer from migraine of migraine sufferers are not fully satisfied with their current treatment 185K ~70% people in the U.S. are affected by narcolepsy of patients suffer from cataplexy 17M+ >15 people in the U.S. have fibromyalgia years since the last FDA-approved therapeutic 15M+ 0 working Americans suffer from shift work disorder new medications approved in nearly two decades Psychiatry


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…And lead in innovation to expand the therapeutic possibilities for CNS conditions Multi-mechanistic approaches Metabolic pharmacokinetic modulation Clinical trial innovation Molecular drug delivery First-in-class mechanisms of action


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Well-positioned to deliver significant near- and long-term value to patients and shareholders through 2040s and beyond In-market innovations NDA-stage product candidates Phase 3 development programs ongoing Potential new product/indication launches through 2027 Highly prevalent and/or difficult-to-treat CNS conditions 3 3 4 7 10 + + + +


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NMDA = N-methyl-D-aspartate; CYP2D6 = Cytochrome P450 Family 2 Subfamily D Member 6; DNRI = Dopamine-norepinephrine reuptake inhibitor; TAAR1 = Trace amine-associated receptor 1; 5-HT = 5-Hydroxytryptamine; NRI = Norepinephrine reuptake inhibitor; Please see full Prescribing Information for AUVELITY, SUNOSI, and SYMBRAVO at www.AUVELITY.com, www.SUNOSI.com, and www.SYMBRAVO.com, respectively. Phase 1 Phase 2 Phase 3 NDA Marketed Advancing an industry-leading neuroscience pipeline FDA Breakthrough Therapy Designation FDA Orphan Drug Designation Major Depressive Disorder Alzheimer’s Disease Agitation Smoking Cessation Attention Deficit Hyperactivity Disorder Binge Eating Disorder Major Depressive Disorder with EDS EDS in Narcolepsy or OSA Migraine Narcolepsy Fibromyalgia Shift Work Disorder Psychiatry Neurology AXS-05 (dextromethorphan-bupropion) NMDA antagonist, sigma-1 agonist, and aminoketone CYP2D6 inhibitor Solriamfetol DNRI , TAAR1 agonist, 5-HT1A agonist AXS-12 (reboxetine) Highly selective NRI, dopamine mod. AXS-14 (esreboxetine) [S,S]-enantiomer of AXS-12 Solriamfetol DNRI , TAAR1 agonist, 5-HT1A agonist


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$16.5B peak sales potential driven by current commercial and late-stage assets AXS-05 AD Agitation $0.5-$1B AXS-14 Fibromyalgia AXS-12 Narcolepsy $0.5-$1B $1.5-$3B Solriamfetol MDD with EDS Solriamfetol ADHD $1-$3B $0.5-$1B Solriamfetol BED $0.3-$0.5B Solriamfetol SWD $0.5-$1B AXS-05 Smoking Cessation $1-$1.5B $0.3-$0.5B $1-$3B $0.5-$1B


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1Q 2025 highlights Rapidly advancing late-stage pipeline 1Q 2025 total net product revenue of $121.5M represents 62% YoY growth vs. 1Q 2024 AUVELITY: $96.2M SUNOSI: $25.2M SYMBRAVO approved in the U.S. for the acute treatment of migraine with or without aura in adults; commercial launch on track for June 2025 $300.9M cash and cash equivalents as of March 31, 2025 Current cash expected to fund operations into cash flow positivity NDA for AXS-14 for the management of fibromyalgia submitted to the FDA Supplemental NDA submission for AXS-05 in Alzheimer’s disease agitation on track for 3Q 2025 NDA submission for AXS-12 for cataplexy in patients with narcolepsy anticipated in 2H 2025 Positive topline results announced for FOCUS Ph 3 trial of solriamfetol in ADHD Initiation of Ph 3 trial of solriamfetol in MDD with EDS anticipated in 2025 Positive topline results announced for EMERGE Ph 3 trial of SYMBRAVO in oral CGRP non-responders Strong commercial execution Financial strength and operational excellence


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Maintaining momentum into 2025 with catalyst-rich path ahead 2025 & 2026 Clinical Trial Topline Results Clinical Trial Initiations & Progress Updates Positive topline results from EMERGE Ph 3 trial of SYMBRAVO in oral CGRP non-responders (1Q 2025) Positive topline results from FOCUS Ph 3 trial of solriamfetol in ADHD in adults (1Q 2025) Topline results from PARADIGM Ph 3 trial of solriamfetol in MDD (1Q 2025) ENGAGE Ph 3 trial of solriamfetol in BED (2026) SUSTAIN Ph 3 trial of solriamfetol in SWD (2026) 2025 Initiate Ph 2/3 trial of AXS-05 in smoking cessation (2025) Initiate Ph 3 trial of solriamfetol in ADHD in pediatric patients (2025) Initiate Ph 3 trial of solriamfetol in MDD with EDS (2025) SYMBRAVO approved in the U.S. for the acute treatment of migraine with or without aura in adults (January 2025) Commercial launch of SYMBRAVO in the U.S. (June 2025) FDA filing acceptance decision for NDA for AXS-14 in fibromyalgia (2Q 2025) sNDA submission for AXS-05 in Alzheimer’s disease agitation (3Q 2025) NDA submission for AXS-12 in narcolepsy (2H 2025) Regulatory & Commercial


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Net product revenue $121.5 $75.0 62% AUVELITY net product sales $96.2 $53.4 80% SUNOSI net product revenue* $25.2 $21.6 17% R&D expense $44.8 $36.8 22% SG&A expense $120.8 $99.0 22% 1Q 2025 financial summary 1Q = three months ended March 31; *Includes royalty revenue associated with sales in out-licensed territories 1Q 2025 1Q 2024 % change $ millions


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Cash Balance: (as of March 31, 2025) $300.9 M Debt (Face Value): (as of March 31, 2025) $180 M Market Cap: (as of May 2, 2025) $5.5 B Shares Outstanding: (as of March 31, 2025) 49.2 M Options, RSUs, and Warrants Outstanding*: 9.5 M Runway to reach cash flow positivity, based on the current operating plan Financial snapshot *Consists of 8.3M options, 1.0M RSUs, 0.08M warrants, and 0.06M ESPP as of March 31, 2025


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Commercial Highlights


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Building a leading commercial portfolio in CNS MAJOR DEPRESSIVE DISORDER EDS IN NARCOLEPSY OR OSA MIGRAINE WITH OR WITHOUT AURA Approved in the U.S. on January 30, 2025 EDS = Excessive daytime sleepiness; OSA = Obstructive sleep apnea Rx, sales, and revenue growth vs. comparable periods in 2024 +76% TRx growth in 1Q +80% Sales growth in 1Q +12% TRx growth in 1Q +17% Revenue growth in 1Q Commercial launch anticipated in June 2025


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Rapid acting NMDA receptor antagonist and sigma-1 receptor agonist for MDD1 Rapid remission as early as week 2, sustained and increased vs. control through week 63 Rapid symptom improvement starting at week 1, sustained at week 6 vs. placebo1 Novel and differentiated oral treatment for major depressive disorder in adults1,2 NMDA = N-methyl-D-aspartate; MDD = Major depressive disorder 1. AUVELITY [Prescribing Information]. Axsome Therapeutics, Inc., New York, NY; 2. Thomas, D. & Wessel, C. BIO (2017); 3. Iosifescu, D.V. et al. J Clin Psychiatry (2022)


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AUVELITY Growth driven by robust, growing underlying demand Source: Symphony METYS. Weekly TRx Launch to Date ~190,000 new patients and >36,000 unique writers since launch ~78% of all covered lives between commercial and government (Medicare and Medicaid) channels Key drivers of prescribing AUVELITY – fast acting, lack of weight gain or sexual dysfunction, improved daily functioning and quality of life ~50% of prescriptions from 1st or 2nd line usage ~55% of patients start on AUVELITY as a monotherapy (i.e., new patient or switch) TRx = Total prescriptions


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AUVELITY quarterly net sales performance 3Q 2024 net sales of $80.4M represents 113% year-over-year growth vs. 3Q 2023 1Q 2024 net product sales of $96.2M represents 80% year-over-year growth +80%


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First and only wakefulness promoting agent proven to improve wakefulness through 9 hours1 Improvements in cognitive functioning vs. placebo demonstrated in clinical trials 90% of patients reported feeling better with SUNOSI 150 mg2 First and only DNRI approved for EDS associated with narcolepsy or OSA1 EDS = Excessive daytime sleepiness; OSA = Obstructive sleep apnea; DNRI = Dopamine-norepinephrine reuptake inhibitor 1. SUNOSI [Prescribing Information]. Axsome Therapeutics, Inc., New York, NY; 2. Schweitzer, P.K. et al. Am J Resp Crit Care Med. (2019)


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Continued success supported by strong fundamentals SUNOSI Quarterly nTRx Launch to Date Source: Symphony METYS. nTRx normalizes number of pills in each Trx for 30-day period. ~85,000 new patients and >14,000 unique writers since initial launch ~83% of all covered lives between commercial and government channels High patient satisfaction for SUNOSI – drivers include minimal or no side effects, low abuse potential, does not interfere with nighttime sleep, and durable reduction in daytime sleepiness >50% of patients who switch or add on to current treatment with SUNOSI come from other WPA agents nTRx = Normalized total prescriptions


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SUNOSI quarterly net revenue performance 3Q 2024 net revenue of $24.4M represents 21% year-over-year growth vs. 3Q 2023 1Q 2025 net product revenue of $25.2M represents 17% year-over-year growth +17%


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Single, oral dose provided rapid migraine pain freedom and return to normal functioning within 2 hours1 Harnesses Axsome’s rapid absorption technology to target multiple pathways underlying a migraine attack Superior efficacy demonstrated across a broad range of migraine severity (mild, moderate, severe)1 Novel, multi-mechanistic approach for the acute treatment of migraine1 1. SYMBRAVO [Prescribing Information]. Axsome Therapeutics, Inc., New York, NY


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Positioning SYMBRAVO for immediate impact and long-term growth 1. Symphony Health Claims, New to Brand Patients (2022-2023); 2. Lipton et al. Neurology (2015) Launch readiness High patient dissatisfaction due to limited efficacy and/or burdensome side effects >80% of patients discontinue their acute migraine treatment in the first 12 months1 Inadequate acute treatment is associated with increased risk of progression to chronic migraine2 Unmet needs in migraine Launch preparations on track Initial launch strategy prioritizing headache centers and headache specialists Actively engaged with payers across channels to ensure patient access Positive feedback from healthcare providers highlight unique profile of SYMBRAVO Commercial availability anticipated in June 2025


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Development Pipeline


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AXS-05 modulates the function of neurotransmitters implicated in Alzheimer’s disease (glutamate, sigma-1, norepinephrine, and dopamine)1-4 In Alzheimer’s disease, insoluble Aβ production and accumulation triggers secondary steps leading to synaptic loss and neuronal cell death1,2 Reductions in certain neurotransmitters are thought to contribute to cognitive and behavioral symptoms including agitation and aggression1-4 Brain regions implicated in AD agitation4 AXS-05 pharmacological actions5,6 Potentially first-in-class, best-in-class treatment for Alzheimer’s disease agitation AXS-05 (dextromethorphan-bupropion) 1. Cummings, J.L. N Engl J Med. (2004); 2. Querfurth, H.W. & LaFerla, F.M. N Engl J Med. (2010); 3. Porsteinsson, A.P. & Antonsdottir, I.M. Expert Opin Pharmacother. (2017); 4. Rosenberg, P.B., Nowrangi, M.A., & Lyketsos, C.G. Mol Aspects Med. (2015); 5. Stahl, S.M. CNS Spectr. (2019); 6. Cheng, W. et al. Mol Med Rep. (2015)


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Number of U.S. adults aged 65+ with Alzheimer’s dementia expected to double by 20601 Alzheimer’s disease (AD) agitation Alzheimer’s disease (AD) is the most common form of dementia, affecting approximately 7M people in the U.S.1 AD agitation is associated with accelerated cognitive decline, increased caregiver burden, and increased mortality3 Agitation is reported in ~70% of people with AD and is characterized by emotional distress, verbal and physical aggressiveness, disruptive irritability, and disinhibition1,2 1. Alzheimer’s Association (2024); 2. Tractenberg, R.E. et al. J Neuropsychiatry Clin Neurosci. (2002); 3. Porsteinsson, A.P. & Antonsdottir, I.M. Expert Opin Pharmacother. (2017)


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Statistically significant and clinically meaningful improvements in Alzheimer’s disease agitation CMAI = Cohen-Mansfield Agitation Inventory p=0.069 p=0.007 p=0.010 0 Weeks from Randomization Probability of Freedom from Relapse (%) ACCORD-2 100 75 50 25 0 2 4 6 8 10 12 14 16 18 20 22 24 26 + Censored Log-Rank p-value: 0.001 AXS-05 Placebo Hazard Ratio for Time to Relapse Hazard Ratio (95% CI) 0.276 (0.119-0.641) p-value 0.001 Supplemental New Drug Application (sNDA) submission anticipated 3Q 2025 Supplemental New Drug Application (sNDA) submission on track for 3Q 2025


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70% of smokers want to quit2 Only 3-5% who attempt to quit without assistance are successful for 6-12 months2 Smoking cessation Single largest cause of preventable disease and death in the U.S., accounting for nearly 1 in 5 deaths1 Associated with over $300 billion in annual costs in the U.S.1 ~34M adults in the U.S. smoke cigarettes, ~50% of whom live with a smoking-related disease1 1. U.S. Department of Health and Human Services (2020); 2. Hughes J.R. et al. Addiction (2004)


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Unique pharmacology of solriamfetol supports potential utility in a broad range of CNS conditions 1. Raony, I. et al. Prog Neuropsychopharmacol Biol Psychiatry (2022); Halff, E.F. et al. Trends Neurosci. (2023) Solriamfetol was initially developed as a dopamine and norepinephrine reuptake inhibitor (DNRI) with wake-promoting effects Preclinical and clinical evidence1,2 suggest TAAR1 plays a role in neuropsychiatric conditions related to the dysregulation of monoaminergic transmission Multimodal activity of solriamfetol inhibits the reuptake of dopamine and norepinephrine and exhibits agonist activity at TAAR1 receptors in the brain


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Solriamfetol Phase 3 development programs Topline data 2026 Topline data 2026 Complete Complete Substantial and statistically significant improvements in ADHD symptoms and disease severity Initiation of Phase 3 pediatric trial anticipated in 2025 Solriamfetol FOCUS Phase 3 (N=516) PARADIGM Phase 3 (EDS subgroup n=51) ENGAGE Phase 3 (N=450) SUSTAIN Phase 3 (N=450) Numerically greater improvements in depressive symptoms in prespecified subgroup of patients with severe EDS Initiation of Phase 3 trial in MDD with EDS in 2025 Efficacy and safety of solriamfetol vs. placebo in adults with binge eating disorder 12-week, double-blind, randomized, placebo-controlled, parallel group trial Efficacy and safety of solriamfetol vs. placebo in adults with shift work disorder 12-week, double-blind, randomized, placebo-controlled, parallel group trial ADHD MDD BED SWD ADHD = Attention deficit hyperactivity disorder; MDD = Major depressive disorder; BED = Binge eating disorder; SWD = Shift work disorder


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Attention deficit hyperactivity disorder (ADHD) Chronic neurobiological and developmental disorder affecting an estimated ~22M people in the U.S.1, including ~7M children aged 3-17 years old2 Characterized by a persistent pattern of inattention and/or hyperactive-impulsive behaviors3 Associated with significant impairment in social, academic, and occupational functioning and development3 1. “Facts About ADHD in Adults.” CDC (2024); 2. “Data and Statistics on ADHD.” CDC (2024); 3. “Attention-Deficit/Hyperactivity Disorder.” NIMH (2024)


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Phase 3 pediatric trial initiation anticipated in 2025 Statistically significant improvements in ADHD symptoms Primary endpoint: Change from baseline in the AISRS total score at Week 6 Substantial improvement in ADHD symptoms observed as early as Week 1 (p=0.036, solriamfetol 150 mg) Significantly greater percentage of patients achieved a clinical response (≥30% reduction in AISRS) vs. placebo (p=0.024, solriamfetol 150 mg) Statistically significantly reduced overall ADHD disease severity as measured by the CGI-S score vs. placebo (p=0.017, solriamfetol 150 mg) Well tolerated with a side effect profile consistent with the established safety profile of solriamfetol 1. p-values shown for solriamfetol 150 mg dose vs placebo only (primary analysis) p=0.036 p=0.041 p=0.039


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Major depressive disorder (MDD) ~2/3 of patients experience inadequate response to first-line treatment3 1. “Major Depression.” NIMH (2023); 2. Hasin, D.S. et al. JAMA Psychiatry (2018); 3. Rush, A.J. et al. Am J Psychiatry (2006). 4. Hein et al. J Affect Disord. (2019). Serious and chronic mental health condition causing persistently low or depressed mood and a loss of interest or pleasure in daily activities, and may impair one’s sleep, appetite, ability to concentrate, and/or self-worth1 Approximately 50% of patients with MDD also experience excessive daytime sleepiness (EDS)4, for which there are no approved treatments One of the most common mental disorders in the U.S., impacting ~21M adults each year1,2


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Unmet medical need associated with a 2- to 3-fold increased risk of psychiatric and medical comorbidities3 Binge eating disorder (BED) Solriamfetol’s dopamine, norepinephrine, and TAAR1 mechanisms appear relevant to the pathophysiology of BED4-6 ~7 million people in the U.S. have BED2 BED is 1.75x more common in women than in men2 Binge eating disorder (BED) is the most common eating disorder and is thought to involve issues with food reward processing, impulse control, and appetite regulation1,2 1. Kessler, R.M. et al. Neurosci Biobehav Rev. (2016); 2. Hudson, J.I. et al. Biol Psychiatry (2007); 3. McElroy, S.L. et al. J Clin Psychiatry (2020); 4. Giel, KL. et al. Nat Rev Dis Primer (2022); 5. Bello, N.T. & Hajnal, A. Pharmacol Biochem Behav. (2010); 6. Pruccoli et al. Int J Mol Sci. (2021)


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Evaluating solriamfetol as a potential treatment for BED Solriamfetol (150 mg) Solriamfetol (300 mg) Placebo 1:1:1 R Screening (4 weeks) Double-blind Phase (12 weeks) Follow-up (1 week) Baseline ENGAGE Phase 3 Trial N=450 Key eligibility criteria 18-55 years of age with diagnosis of BED (DSM-5) Primary endpoint Change from baseline in days with binge eating episodes Solriafemtol’s dopamine, norepinephrine, and TAAR1 mechanisms appear relevant to the pathophysiology of BED1-3 Topline results from the ENGAGE Phase 3 trial of solriamfetol in binge eating disorder anticipated in 2026 TAAR1 = Trace amine-associated receptor 1 1. Giel, K.E. et al. Nat Rev Dis Primers (2022); 2. Bello, N.T. & Hajnal, A. Pharmacol Biochem Behav. (2010); 3. Pruccoli et al. Int J Mol Sci. (2021)


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Shift work has long been associated with multiple serious health complaints and a 23% greater risk of sustaining a work-related injury4-5 Shift work disorder (SWD) No new medications approved since 2007 and considerable residual sleepiness reported when medication is used6 ~15 million U.S. workers may suffer from SWD Approximately 1 in 3 people working in the U.S. work an alternate shift2 10-43% have SWD1,3 Shift work disorder (SWD) is a combination of excessive sleepiness during wakefulness and persistent insomnia during daytime sleep when working outside a 7 a.m. to 6 p.m. workday1 1. Sateia, M.J. Chest (2014); 2. Alterman, T. et al. Am J Ind Med. (2013); 3. Wickwire, E.M. Chest (2017); 4. Smith, L. et al. Lancet (1994); 5. Akerstedt, T. & Wright, KP. Sleep Med Clin. (2009); 6. Czeisler, C.A. et al. N Engl J Med. (2005)


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Evaluating solriamfetol as a potential treatment for SWD Solriamfetol (150 mg) Solriamfetol (300 mg) Placebo 1:1:1 R Screening (1-4 weeks) Double-blind Phase (12 weeks) Follow-up (1 week) Baseline SUSTAIN Phase 3 Trial N=450 Key eligibility criteria 18-65 years of age with diagnosis of SWD (ICSD-2 or ICSD-3) Primary endpoint Change from baseline in CGI-C score Topline results from the SUSTAIN Phase 3 trial of solriamfetol in shift work disorder anticipated in 2026 CGI-C = Clinical Global Impression - Change


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Norepinephrine and dopamine play important roles in sleep-wake regulation (both) and in maintaining muscle tone during wakefulness (norepinephrine)1-3 AXS-12 inhibits the reuptake of both neurotransmitters, improving both norepinephrine and cortical dopamine signaling in the brain The loss of orexin input inhibits the production of these neurotransmitters1,2 Decreased norepinephrine signaling is thought to contribute to cataplexy, EDS, and cognitive impairment1,4-7 Decreased dopamine signaling is thought to contribute to EDS and cognitive impairment1,4 AXS-12 (reboxetine) Novel pharmacological approach for the treatment of narcolepsy 1. Szabo, S.T. et al. Sleep Med Rev. (2019); 2. Krahn, L.E., Zee, P.C., & Thorpy, M.J. Adv Ther. (2022); 3. Scammell, T.E. N Engl J Med. (2015); 4. Stahl, S.M & Grady, M.M. J Clin Psychiatry (2003); 5. Burgess, C.R. & Peever, J.H. Curr Biol. (2013); 6. Wu, M.F. et al. Neuroscience (1999); 7. Bruinstroop, E. et al. J Comp Neurol. (2012)


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Narcolepsy Rare and debilitating neurological condition that affects approximately 185,000 people in the U.S.1 Characterized by cataplexy, excessive daytime sleepiness (EDS), hypnagogic hallucinations, sleep paralysis, and disrupted nocturnal sleep2-4 An estimated 70% of patients suffer from cataplexy, or the sudden reduction or loss of muscle tone while awake5 1. “About Narcolepsy.” Narcolepsy Network (2024); 2. Sateia, M.J. Chest (2014); 3. “Narcolepsy.” NINDS (2024); 4. España, R.A. & Scammell, T.E. Sleep (2011); 5. Swick, T.J. Nat Sci Sleep (2015)


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Rate Ratio* p=0.007 p=0.006 p=0.031 p=0.031 p=0.018 *Ratio of change in the AXS-12 group divided by the ratio of change in the placebo group SYMPHONY Primary endpoint: Change from baseline in weekly cataplexy attacks Statistically significant reductions in cataplexy frequency CONCERT p<0.001 p=0.002 Baseline Week 1 Week 2 4.0 0.0 -4.0 -8.0 -12.0 -16.0 -20.0 Change in Weekly Cataplexy Attacks Change in Weekly Cataplexy Attacks New Drug Application (NDA) submission anticipated 2H 2025 New Drug Application (NDA) submission anticipated 2H 2025


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Fibromyalgia pain is thought to be partially caused by dysregulated signaling in the descending analgesic system Norepinephrine, one of the key neurotransmitters in this pathway, has predominantly pain-inhibitory effects AXS-14 is a more potent and selective enantiomer of racemic reboxetine that inhibits the reuptake of norepinephrine, resulting in increased norepinephrine activity and decreased pain signaling AXS-14 (esreboxetine) Novel pharmacological approach for the management of fibromyalgia (FM) Adapted from Siracusa, R. et al. Int. J. Mol. Sci. (2021)


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Fibromyalgia (FM) Chronic and debilitating neurological syndrome impacting ~17M people in the U.S.1 Characterized by widespread musculoskeletal pain, fatigue, disturbed sleep, depression, and cognitive impairment2 Limited treatment option with only 3 approved agents of variable and/or inadequate efficacy, with no novel therapeutics in over 15 years 1. Vincent et al. Arthritis Care Res (Hoboken) (2013); 2. Bair, M.J. & Krebs, E.E. Ann Intern Med. (2020)


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Positive clinical data demonstrate statistically significant improvements in symptoms of fibromyalgia ~1,000 individuals with fibromyalgia dosed with esreboxetine across Phase 2 and Phase 3 clinical trials for up to 14 weeks Statistically significant and clinically meaningful reductions in pain scores, overall symptom severity, and improvements in patient-reported global functioning and fatigue p<0.001 p<0.001 p=0.025 Phase 3 Efficacy Results (N=1,122) p<0.001 p<0.001 p=0.023


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Strong intellectual property and barriers to entry Protected by a robust patent estate extending out to at least 2043; Multiple pending Proprietary drug product formulation >99 issued U.S. patents and >131 issued O.U.S. patents Claims extending to at least 2040; Multiple pending Proprietary MoSEICTM formulation and drug product formulation Protected by a robust patent estate extending out to at least 2042 >36 issued U.S. patents and >100 issued O.U.S. patents; Multiple pending Proprietary drug substance and drug product formulation Orphan Drug Designation 8 issued U.S. patents and >2 issued O.U.S. patents Claims extending to at least 2039; Multiple pending Proprietary drug substance and drug product formulation >140 issued U.S. patents and >93 issued O.U.S. patents Claims extending to at least 2043; Multiple pending Proprietary drug product formulation Multiple pending U.S. patents Proprietary drug substance and drug product formulation AXS-05 AXS-12 AXS-14


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Leadership team Roger Jeffs, PhDCEO, Liquidia CorporationFormer President, Co-CEO, Director United Therapeutics Corp. Prior positions at Amgen and Burroughs Wellcome Herriot Tabuteau, MDFounder & CEO Management Board of Directors Nick Pizzie, CPA, MBAChief Financial Officer Mark Jacobson, MAChief Operating Officer Hunter Murdock, JDGeneral Counsel Ari MaizelChief Commercial Officer Mark Saad CEO, NuLids, LLCFormer COO of the Global Healthcare Group at UBS Mark Coleman, MDMedical Director, National Spine and Pain CentersDiplomat of the American Board of Anesthesiology Susan Mahony, PhD Former SVP of Eli Lilly and President Lilly Oncology Prior positions at BMS, Amgen and Schering-Plough Herriot Tabuteau, MD Chairman


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Thank you