Axsome Therapeutics, Inc. · NASDAQ:AXSM
Axsome Therapeutics — Q1 2025 Earnings Presentation
26 slides from Axsome Therapeutics's earnings presentation, May 6, 2025
Slide 1: 2025 First Quarter Financial Results
May 6, 2025 2025 First Quarter Financial Results Innovating to Transform the Lives of Patients and Their Families Markella EPIDIOLEX® patient diagnosed with Dravet syndrome May 6, 2025
Slide 2: Caution Concerning Forward-Looking Statements
Transforming Lives. Redefining Possibilities. Caution Concerning Forward-Looking Statements This presentation contains forward-looking statements and financial targets, including, but not limited to, statements related to: the Company’s growth prospects and future financial and operating results, in
Slide 3: Non-GAAP Financial Measures
Transforming Lives. Redefining Possibilities. Non-GAAP Financial Measures To supplement Jazz Pharmaceuticals’ financial results and guidance presented in accordance with U.S. GAAP the Company uses certain non-GAAP (also referred to as adjusted or non-GAAP adjusted) financial measures in this present
Slide 4: Introduction and Overview
Introduction and Overview Bruce Cozadd Chairman and Chief Executive Officer 4 May 6, 2025
Slide 5: Strong 2025 Execution and Continued Focus on Growth
Strong 2025 Execution and Continued Focus on Growth COMMERCIAL PIPELINE FINANCIAL Growing and diversified revenues generate Zanidatamab and dordaviprone are Disciplined capital allocation enables significant cash flows near-term opportunities investment in growth Sleep1: Zanidatamab: Affirming 2025
Slide 6: Commercial Performance
Commercial Performance Renée Galá President and Chief Operating Officer 6 May 6, 2025
Slide 7: Xywav: Differentiated by Low Sodium; IH Provides Growth Opportunity
Xywav: Differentiated by Low Sodium; IH Provides Growth Opportunity Sleep2 therapeutic area: • Total sleep2 revenue of $431 million in 1Q25 IH Narcolepsy 14,600 14,150 13,225 13,625 4,225 12,950 3,550 3,900 3,050 3,300 Xywav remains the only low-sodium oxybate therapy • Increase of ~450 net patients
Slide 8: Epidiolex: Durable Growth; High Unmet Need in Pediatric Onset Epilepsy
Epidiolex: Durable Growth; High Unmet Need in Pediatric Onset Epilepsy Revenue: +10% YoY Epidiolex is the #1 branded epilepsy treatment1 • Poised to reach blockbuster status in 2025 $218 Growth opportunities: $199 • Continued education on synergies from treatment in combination Net product sales ($
Slide 9: Rely on Rylaze: Critical Component of U.S. ALL/LBL Treatment Protocols
Rely on Rylaze: Critical Component of U.S. ALL/LBL Treatment Protocols Revenue: -8% YoY Sustained asparaginase activity over the course of therapy essential to treatment success of ALL/LBL patients1 • Only therapy available to patients in the U.S. who have a hypersensitivity $103 reaction to E. coli
Slide 10: Zepzelca: Opportunity to Redefine 1L SCLC Treatment Paradigm
PIPELINE Zepzelca: Opportunity to Redefine 1L SCLC Treatment Paradigm Revenue: -16% YoY Leading treatment in 2L SCLC1 • 1Q25 revenues impacted by 2L competition and treatment protocols delaying progression in 1L LS-SCLC patients to 2L setting $75 Submitted sNDA for use as maintenance therapy in 1L E
Slide 11: Ziihera: BTC Launch Driven by Proven Jazz Oncology Team and Infrastructure
Ziihera: BTC Launch Driven by Proven Jazz Oncology Team and Infrastructure • Proven team with deep oncology experience, including extensive expertise in the HER2 therapy Right Team, Right Capabilities space, helps drive additional adoption and uptake • Infrastructure in place for a successful Ziiher
Slide 12: Research & Development
Research & Development Robert Iannone, M.D., M.S.C.E. Executive Vice President, Global Head of Research & Development, Chief Medical Officer 12 May 6, 2025
Slide 13: Key Pipeline Programs
Key Pipeline Programs Key Clinical Programs PHASE 1 PHASE 2 PHASE 3 PHASE 4 / REGULATORY Recent / Upcoming Milestones 2L BTC (pivotal) Positive CHMP decision in 2L BTC Phase 3 1L GEA (pivotal) Phase 3 top-line PFS readout expected 2H25 Phase 3 1L BTC Phase 3 confirmatory trial in 1L BTC ongoing Zani
Slide 14: Zanidatamab: De-Risked Near-Term Opportunity with $2B+ Peak Potential
Zanidatamab: De-Risked Near-Term Opportunity with $2B+ Peak Potential Continued progress: • Completed recruitment of HERIZON-GEA-01 trial Other HER2-Expressing • Received CHMP positive opinion for the treatment Cancers of advanced HER2-positive BTC Breast Cancer Broad potential beyond BTC, GEA, and
Slide 15: Dordaviprone: Continued Diversification and Near-Term Commercial Opportunity
Dordaviprone: Continued Diversification and Near-Term Commercial Opportunity H3-K27M Mutant Positivity Rate H3-K27M Mutant Glioma by Tumor Location1 • Estimated U.S. incidence of more than 2,000 patients2 • No currently approved systemic therapies for H3 K27M-mutant glioma • Median OS ~1 year3 from
Slide 16: Financial Performance
Financial Performance Phil Johnson Executive Vice President, Chief Financial Officer 16 May 6, 2025
Slide 17: Focus on 2025 Commercial Execution
Focus on 2025 Commercial Execution 1Q25 Total Revenues 1Q25 Non-GAAP ANI2 and EPS2,3 Continue to grow and diversify commercial portfolio: In-line ANI -41% EPS3 -36% • Xywav revenues of $345M in 1Q25, 9% growth YoY • Epidiolex revenues of $218M in 1Q25, $902M $898M $178M 10% growth YoY $2.63 • Oncolo
Slide 18: 2025 Revenue Guidance
2025 Revenue Guidance TOTAL REVENUE Top-line growth driven by diversified portfolio +5%3 $4,150M- $4,400M Leading treatment in Standard of care in $4,069M Potential to be the 2L SCLC1; pediatric ALL/LBL patients therapy of choice in expansion opportunity in with asparaginase multiple HER2+ tumors 1L
Slide 19: 2025 Non-GAAP Adjusted Guidance; Investing to Drive Growth
2025 Non-GAAP Adjusted Guidance ; Investing to Drive Growth 1 SG&A2 R&D2 ANI2,3 Non-GAAP Adjusted: 2025 Guidance1 +22%4 -3%4 -78%4 Effective tax rate2 35% - 45% • Includes: $172M related to certain • Includes: R&D costs associated with • Includes: impacts to SG&A2, R&D2 and Xyrem antitrust litigatio
Slide 20: Closing
Closing Bruce Cozadd Chairman and Chief Executive Officer 20 May 6, 2025
Slide 21: Well-Positioned to Deliver Meaningful Shareholder Value
Well-Positioned to Deliver Meaningful Shareholder Value COMMERCIAL EXECUTION PIPELINE CATALYSTS Executing launch in 2L BTC Zanidatamab Phase 3 1L GEA top-line data expected 2H25 Poised to reach blockbuster status Operational Positive CHMP decision in 2L BTC excellence and a disciplined, strategic Ph
Slide 22: Appendix
Appendix 0 Jazz Pharmaceuticals.
Slide 23: Glossary
Glossary Acronym Definition Acronym Definition 1L First-line HSR Hypersensitivity reaction 2024 Notes Our 1.50% exchangeable senior notes due 2024 IH Idiopathic hypersomnia 2026 Notes Our 2.000% exchangeable senior notes due 2026 IHC Immunohistochemistry 2L Second-line IPR&D In-process research and
Slide 24: Reconciliation of GAAP Reported to Non-GAAP Adjusted Information
Reconciliation of GAAP Reported to Non-GAAP Adjusted Information1 Three Months Ended March 31, 2025 Three Months Ended March 31, 2024 In thousands, except per share amounts (unaudited) Net Income (Loss) Diluted EPS/(LPS)2 Net Income (Loss) Diluted EPS/(LPS)2 GAAP reported $(92,541) $(1.52) $(14,618)
Slide 25: Reconciliation of GAAP to Non-GAAP Adjusted 2025 Guidance
Reconciliation of GAAP to Non-GAAP Adjusted 2025 Guidance1 2025 Guidance1 2025 Guidance1 Net Income / Diluted EPS / In millions, except per share amounts (unaudited) (Loss) (LPS) In millions (unaudited) SG&A R&D 5 6 GAAP2 $(615) - $(450) 3 $(10.00) - $(7.50) GAAP expenses $1,640 - $1,723 $835 - $895
Slide 26: Reconciliation of GAAP Reported to Non-GAAP Adjusted Information
Reconciliation of GAAP Reported to Non-GAAP Adjusted Information1 Year ended December 31, 2024 In thousands, except per share amounts (unaudited) Net Income Diluted EPS2 GAAP reported $560,120 $8.65 Intangible asset amortization 627,313 9.50 Share-based compensation expense 248,045 3.76 Acquisition