Slides
Page 1
Oncology Leadership in Pretargeted Radioimmunotherapy Platform and Antibody-based Therapies January 2025
Page 2
Disclaimer This presentation contains forward-looking statements within the meaning of the US Private Securities Litigation Reform Act of 1995. The forward-looking statements involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this presentation, including statements about: our business model, preliminary estimated financial results and expectations for the year ended December 31, 2024, including estimated net revenue; implied and express statements regarding the future of the Company’s business; and the Company’s strategies, development, regulatory, commercialization and product distribution plans are forward- looking statements. Words such as “anticipate,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “goal,” “guidance,” “hope,” “intend,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward- looking statements contain these identifying words. Our product candidates and related technologies are novel approaches to cancer treatment that present significant challenges. Actual results may differ materially from those indicated by such forward-looking statements as a result of various factors, including but not limited to: risks associated with our financial condition and need for additional capital; the risk that our reported results may differ materially from our preliminary estimated DANYELZA net product revenue results as a result of the completion of year-end closing procedures, final adjustments, and other developments arising between now and the time that our financial results are finalized; risks associated with our development work; cost and success of our product development activities and clinical trials; the risks of delay in the timing of our regulatory submissions or failure to receive approval of our drug candidates; the risks related to commercializing any approved pharmaceutical product including the rate and degree of market acceptance of our product candidates; development of our sales and marketing capabilities and risks associated with failure to obtain sufficient reimbursement for our products; the risks related to our dependence on third parties including for conduct of clinical testing and product manufacture; risks related to our ability to enter into partnerships; the risks related to government regulation; risks related to market approval; risks associated with protection of our intellectual property rights; risks related to employee matters and managing growth; risks related to our common stock; risks associated with ongoing geopolitical conflicts; and other risks and uncertainties affecting the Company including those described in the “Risk Factors” section included in our Annual Report on Form 10-K for the year ended December 31, 2023, our Quarterly Reports on Form 10-Q for the quarters ended March 31, June 30, and September 30, 2024, and in our other SEC filings. Any forward-looking statements contained in this presentation speak only as of the date hereof, and the Company undertakes no obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. 2
Page 3
3 At Y-mAbs, our mission is to deliver innovative therapeutic solutions for life’s most threatening diseases, helping to improve and extend people’s lives
Page 4
4 Next-Generation Novel Platforms Self-Assembly DisAssembly Pretargeted Radioimmunotherapy (“SADA PRIT”) Platform Monoclonal Antibodies Established Commercial Capabilities DANYELZA (naxitamab- gqgk), Anti-GD2 Approved for R/R High-Risk Neuroblastoma U.S. Commercial Footprint; Ex-U.S. through partnerships, NPPs Radiopharmaceutical Leadership Deep bench of industry leadership and expertise in developing and commercializing radiopharmaceutical oncology therapeutics Broad Pipeline Potential SADA PRIT's proven mechanism of pre-targeted approach carries therapeutic potential beyond oncology Growing Base Business with Potential High Value RIT Platform
Page 5
2024 Achievements 5 DANYELZA remains an important anti-GD2 therapy for patients with HR R/R NB MSK presented DANYELZA osteosarcoma data at CTOS COMMERCIAL PROGRESS Increased DANYELZA vial demand in ex-U.S. markets including China, Brazil and Mexico; NPPs in Europe, Turkey SADA PRIT ADVANCEMENT Activated 4 sites for CD38-SADA Phase 1 trial (Trial 1201) Proof-of-concept of SADA PRIT platform achieved in GD2-SADA Phase 1 trial (Trial 1001) Preliminary estimated unaudited FY2024 total net revenue of ~$88M within final guidance range* FINANCIAL Capital efficient with sound financial structure allowing us to invest in the future *Preliminary results are unaudited and based on management’s initial review of the Company’s results as of and for the year e nded December 31, 2024, and are subject to revision based upon the Company’s year -end closing procedures and the completion of the audit by the Company’s external auditors of the Company’s December 31, 2024 financial statements; Previously announced guidance range during Q3 2024 earnings report.
Page 6
Realignment into 2 Business Units Intended to Accelerate Development in Radiopharmaceuticals and Maximize Value of DANYELZA 6 Expand Radiopharmaceutical Capabilities Align Strategy and Budget Accelerate Execution Capital Efficiency Dedicated Business Units aligned with budget and strategy intended to drive operational excellence and efficiency Leverage DANYELZA cash flows along with alternative funding sources to aggressively advance our Radiopharmaceutical Pipeline Realignment is expected to help accelerate the pace of the advancement of our Radiopharmaceutical Platform and leverage our pre-targeting first-mover advantage Adding dedicated internal resources, increasing flexibility, and optimizing operations is critical to advancing our Radiopharmaceutical Platform
Page 7
7 SADA PRIT Platform Novel Self-Assembly DisAssembly Pre-targeted Radioimmunotherapy Technology Platform
Page 8
Current Radiopharmaceutical Industry Challenges Negatively Impact Patient Care 8 Infrastructure and Manufacturing Physician Participation Administration Sites Continuing Drug Shortages
Page 9
Novel SADA PRIT Platform Aims to Address Key Improvements over Traditional Radiopharmaceuticals 9 SADA PRIT Platform Potential Capabilities ✓ Pre-targeting tumor potentially minimizes toxicity and potentially enhances rapid clearing of unbound protein ✓ Potential to work with short T 1/2 isotopes ✓ Potentially broader site options with protein doses administered by Medical Oncologist ✓ Potential COGS improvements Traditional Radioimmunotherapy Limited dose-to-tumor due to off-target radiation Prone to drug shortages / supply issues with single-isotope only capabilities Limited administration sites with licensed nuclear medicine oncologist High investment needed for specific infrastructure and manufacturing * Pending successful development and approval.
Page 10
Two-Step GD2-SADA PRIT Designed to Selectively Delivers Cytotoxic Radiation to GD2+ Tumor Cells PRIT Step 1 Non-radiolabeled SADA protein is administered at a concentration that favors tetramer formation1,2 and binds with high avidity to targeted tumor cells1,2 Over time, unbound SADA protein disassembles into monomers that are renally cleared1-3 PRIT Step 2 Chelated radioisotope4 binds to the anti- DOTA domain of SADA protein on tumor cells, causing radiation-induced damage1,2 Unbound radioisotope is cleared by the kidneys,1,3 limiting exposure to the blood/marrow compartment DOTA, dodecane tetraacetic acid, tetraxetan; GD2, disialoganglioside; Ln, lanthanide metal; PRIT, pretargeted radioimmunotherapy; SADA, self-assembly and disassembly. 1. Santich BH et al. Clin Cancer Res 2021;27:532-541. 2. Santich BH et al. Cancer Res 2022;82(12 suppl):3309. 3. Capala C, Kunos CA. Clin Cancer Res 2021;27:377-379. 4. Cheal SM et al. Eur J Nucl Med Mol Imaging 2016;43:925-937. DOTA Radioisotope 10
Page 11
11 SADA PRIT Platform GD2-SADA Program Update
Page 12
GD2-SADA Phase 1 Clinical Trial (Trial 1001): Part A Ongoing Theranostic approach using a 30 mCi 177Lu-DOTA imaging dose before exposing to therapeutic dose 12 Part A Determine optimal, well- tolerated GD2-SADA protein dose and dosing interval between GD2- SADA and 177Lu-DOTA Part B Dose ranging to determine maximum tolerable activity of 177Lu-DOTA Part C Repeat dosing to determine (RP2D); early efficacy signals Trial Overview • Solid tumors: SCLC, malignant melanoma, sarcomas, adult neuroblastoma • Completed Cohorts 1 through 5; currently in Cohort 6* • 21 patients dosed* • 6 sites open* • No DLTs or serious instances of treatment- related AEs reported* *As of January 12, 2025 Part A Data Anticipated to be Presented in Q2 2025 × Optimal and safe GD2-SADA protein dose • Dosing interval between GD2- SADA and 177Lu-DOTA • PK dosimetry • Rate of excretion • Concentration in tissue • Tumor burden • PET/CT scan images
Page 13
Trial 1001: Demonstrated Proof-of-Concept in GD2-SADA Across Various Tumor Types Overview of patients who showed tumor uptake Data cut as of January 6, 2025. These early results are not complete and are not necessarily indicative of the full results o r ultimate success of the SADA trials or the SADA development program which is in early development with no guaranty of approval . Cohort 2 (2-day interval) Cohort 3 (5-day interval) Cohort 3 (5-day interval) Cohort 3 (5-day interval) Cohort 4 (5-day interval) Cohorts 5 (4-day interval) Cohort 6 (3-day interval) Cohort 6 (3-day interval) Cohort 6 (3-day interval) Patient Number 3 5 8 9 11 16 18 19 21 Diagnosis Osteosarcoma Osteosarcoma Synovial Sarcoma Uveal Melanoma Leyomyosarc oma Uveal Melanoma Melanoma Uveal Melanoma Melanoma Dose level (mg/kg) 0.3 1.0 1.0 1.0 3.0 1.0 1.0 1.0 1.0 Tumor uptake Yes Yes Yes Yes Yes Yes Yes Yes Yes Total Number of Patients by Tumor Type (N = 21) Sarcoma 11 Melanoma 8 Small Cell Lung Cancer (SCLC) 1 Neuroblastoma (NB) 1 13
Page 14
Trial 1001: SPECT/CT Scan on Osteosarcoma Patient Demonstrating Positive Tumor Uptake After Exposure* Shared patient scan shows: • Patient treated with 0.3 mg/kg GD2-SADA, followed by 200 mCi 177Lu-DOTA (lowest therapeutic radionuclide dose) 48-hours later • Scan performed 24 hours after radionuclide administration • 4 target lesions marked on CT scan (left image) – all targeted by 177Lu-DOTA SADA (right image) *These early results are not complete and are not necessarily indicative of the full results or ultimate success of the SADA trials or the SADA development program, which is in early development with no guaranty of approval. Limitation: Patient-level data are for descriptive purposes and should not be considered indicative of typical product efficacy or duration; interpret with caution. 14
Page 15
Trial 1001: Further Example of Osteosarcoma Patient Demonstrating Positive Tumor Uptake Across All 5 Tumors After 177Lu-DOTA SADA Dose Osteosarcoma Patient with 5 Tumors – SPECT/CT Images 1 2 3 4 5 125g 388g 125g 127g 74g Dosing Interval 2-day dosing interval Protein Dose 0.3 mg/kg Patient Details Osteosarcoma 5 Tumors Patient 3 Patient Details Therapy Dose 200 mCi 177 Lu-DOTA *These early results are not complete and are not necessarily indicative of the full results or ultimate success of the SADA trials or the SADA development program, which is in early development with no guaranty of approval. Limitation: Patient-level data are for descriptive purposes and should not be considered indicative of typical product efficacy or duration; interpret with caution. 15
Page 16
Mean Blood PK Values Indicates Clearance Rates Similar with Different Dose Levels, as Predicted, in Preliminary Data 16 Trial 1001: Blood Pharmacokinetic for GD2-SADA (mean values) *These early results are not complete and are not necessarily indicative of the full results or ultimate success of the SADA trials or the SADA development program, which is in early development with no guaranty of approval. Limitation: Patient-level data are for descriptive purposes and should not be considered indicative of typical product efficacy or duration; interpret with caution. Non-QC data cut as of Company’s Q3 2024 earnings report. normal scale Time (hours) Mean GD2-SADA (ng/mL) Methodology: GD2-SADA concentration in plasma measured via blood draws at various points in time Study measures all forms of SADA, tetramer, monomer and dimer Mean GD2-SADA (ng/mL) GD2-SADA PK (Normal scale) GD2-SADA PK (Semi-logarithmic scale) Time (hours) Mean GD2-SADA over Time 3.0 mg/kg (n=4) 0.3 mg/kg (n=4) 1.0 mg/kg (n=8)
Page 17
Preliminary Part A Data and Anticipated GD2-SADA 2025 Milestones 17 Preliminary Phase 1 Part A Data ✓Well-tolerated GD2-SADA protein dose ✓PK dosimetry ✓Rate of excretion ✓Concentration in tissue ✓Tumor burden ✓PET/CT scan images × Optimal dosing interval between GD2-SADA and 177Lu-DOTA Optimization Workstreams Underway • Optimize DOTA chelator • Refine Lu-177 specific activity Evaluate Optimized GD2-SADA Molecule • Dosing interval between GD2- SADA and 177Lu-DOTA • Well-tolerated GD2-SADA protein dose • PK dosimetry* • Rate of excretion • Concentration in tissue • Tumor burden • PET/CT scan images* Ongoing Trial 1001 (GD2-SADA) Molecule Optimization Planned Trial Q2 2025 * Additional time-point data to be collected These early results are not complete and are not necessarily indicative of the full results or ultimate success of the SADA trials or the SADA development program which is in early development with no guaranty of approval.
Page 18
18 SADA PRIT Platform CD38-SADA Program in Circulating Tumors
Page 19
CD38-SADA Demonstrated Dose-dependent Anti-tumor Activity Against CD38-positive Tumors Anti-Tumor Response of CD38-SADA (two preclinical models) Note: if any mouse was removed from study, calculations for “Days Post Dosing Start” was terminated, resulting in variable “Days Post Dosing Start” 177Lu-DOTA Dose ResponseSADA Dose Response Mean Tumor Volume (mm3) Days Post Dosing StartDays Post Dosing Start 0 5 10 15 20 25 30 35 40 45 50 0 500 1000 1500 2000 Mean tumor volume Day post dosing start Tumor volume (mm3) Group 1: Vehicle Group 2: CD38-SADA (30 mg/kg) Group 3: 177Lu-DOTA (37 MBq) Group 4: CD38-SADA (5 mg/kg)/ 177Lu-DOTA (37 MBq/mouse) Group 5: CD38-SADA (10 mg/kg)/ 177Lu-DOTA (37 MBq/mouse) Group 6: CD38-SADA (30 mg/kg)/ 177Lu-DOTA (37 MBq/mouse) 0 5 10 15 20 25 30 35 40 45 50 0 500 1000 1500 2000 Mean tumor volume Day post dosing start Tumor volume (mm3) Group 1: Vehicle Group 2: CD38-SADA (30 mg/kg) Group 3: 177Lu-DOTA (37 MBq) Group 4: CD38-SADA (5 mg/kg)/ 177Lu-DOTA (37 MBq/mouse) Group 5: CD38-SADA (10 mg/kg)/ 177Lu-DOTA (37 MBq/mouse) Group 6: CD38-SADA (30 mg/kg)/ 177Lu-DOTA (37 MBq/mouse) 0 5 10 15 20 25 30 35 40 0 400 800 1200 1600 Burkitt's Lymphoma model Mean tumor volume Day post dosing start Tumor volume (mm3) 177Lu-DOTA (37 MBq/mouse) CD38-SADA (10 mg/kg) Vehicle CD38-SADA (10 mg/kg) / 177Lu-DOTA (18.5 MBq/mouse) CD38-SADA (10 mg/kg) / 177Lu-DOTA (37 MBq/mouse) ▲ CD38-SADA dosing 177Lu-DOTA dosing ▲ ▲ 0 5 10 15 20 25 30 35 40 0 400 800 1200 1600 Burkitt's Lymphoma model Mean tumor volume Day post dosing start Tumor volume (mm3) 177Lu-DOTA (37 MBq/mouse) CD38-SADA (10 mg/kg) Vehicle CD38-SADA (10 mg/kg) / 177Lu-DOTA (18.5 MBq/mouse) CD38-SADA (10 mg/kg) / 177Lu-DOTA (37 MBq/mouse) ▲ CD38-SADA dosing 177Lu-DOTA dosing ▲ ▲ Mean Tumor Volume (mm3) Burkitt’s Lymphoma (BL) Model Santich, et al. CD38-SADA, a Self-Assembling and Dis-Assembling Bispecific Fusion Protein for Two-Step Pretargeted Radioimmunotherapy of Non-Hodgkin Lymphoma. Poster Presentation. American Society of Hematology. 5 November 2024. San Diego, California. 19
Page 20
CD38-SADA Binds Well to CD38-positive Cell-lines While Not Binding to Negative Cell-lines Binding Characteristics of CD38-SADA in Preclinical Model Colorectal Cancer cell-line Breast Cancer cell-line Concentration CD38-SADA (nM) Mean Fluorescent Intensity CD38-SADA (8DC-7) Binding by CD38 Negative Cell Line Log (Antibody µg/mL) CD38-SADA Binding by CD38 Positive Cell-Line Median Fluorescent Intensity 8DC-7 Daudi 8DC-7 HuT 78 8DC-7 MJ PA-2A Daudi PA-2A HuT 78 PA-2A MJ Cell Line Cancer Type CD38 Expression Daudi Diffuse Large B-cell Lymphoma (NHL) High HuT 78 Cutaneous T-cell Lymphoma (NHL) Moderate MJ Cutaneous T-cell Lymphoma (NHL) Low CD38-SADA (8DC-7) did not bind to either of the CD38-negative cell lines Santich, et al. CD38-SADA, a Self-Assembling and Dis-Assembling Bispecific Fusion Protein for Two-Step Pretargeted Radioimmunotherapy of Non-Hodgkin Lymphoma. Poster Presentation. American Society of Hematology. 5 November 2024. San Diego, California. 20
Page 21
CD38-SADA Phase 1 Clinical Trial (Trial 1201): Trial Design Theragnostic approach using IHC validated CD38 positive tumors and 177Lu-DOTA organ dosimetry before dosing in patients with relapsed or refractory non-Hodgkin Lymphoma 21 Inclusion Criteria • R/R non-Hodgkin Lymphoma and ineligible/ exhausted standard therapeutic options • Fluoro-deoxyglucose (FDG)-avid lymphoma with measurable disease • ECOG performance status score of 0, 1, or 2 • CD38+ tumor Part B N = 12 – 15 177Lu-DOTA therapeutic dose escalation with the CD38-SADA dose determined in Part A Occurrence of DLTs during evaluation period Part A N = 12 – 15 CD38-SADA dose- escalation with fixed imaging and therapeutic 177Lu-DOTA doses Tumor imaging and occurrence of DLTs during evaluation period DESIGNOUTCOME TRIAL UPDATE › IND cleared by U.S. FDA in Q4 2023 › First 6 sites selected; 4 sites activated* UPCOMING CATALYSTS › Anticipate dosing the first patient in Q1 2025 *As of January 12, 2025
Page 22
22 Novel SADA PRIT Platform Potentially Provides Simplicity and Enhanced Precision for Physicians and Patients* GD2-SADA Phase 1 Part A data, optimization data and new high-value targets expected to be presented in Q2 2025 Ongoing GD2-SADA Phase 1 Trial (Trial 1001) › Evidence of tumor uptake › No DLTs observed to date › Demonstrated PoC that GD2-SADA targets and binds to tumor in humans Potential to shift radio- immunotherapy treatment paradigm for patients and physicians with simplicity and enhanced precision of novel SADA PRIT platform CD38-SADA Phase 1 Trial (Trial 1201) › First-in-human trial in patients with R/R non-Hodgkin Lymphoma › Anticipate dosing first patient in Q1 2025 *These early results are not complete and are not necessarily indicative of the full results or ultimate success of the SADA trials or the SADA development program which is in early development with no guaranty of approval
Page 23
23 Radiopharmaceutical Pipeline Expansion Plans
Page 24
Selection Process Potentially Leading to High-Value Targets for Future Development 24 Broad Potential Target Universe Initial Refinement – Cancer types of Interest (~20-30 cancer types) Second Refinement – Targets of Interest (~500-800 in- scope targets) Assessment with Target Prioritization Framework (~40-50 fully characterized targets) (~1,200) Targeting: » Good target fits for SADA PRIT » Mix of archetypes allows for diversification of portfolio risk » Target cross- over capabilities with multiple indications Y-mAbs Expects to Provide Pipeline Update on Selected New Programs in Q2 2025
Page 25
Y-mAbs’ Comprehensive Radiopharmaceutical Target Identification Process Next Cohort of Potential High-Value Oncology Targets for Development with SADA PRIT Key target considerations • Clinical validation (especially via ADCs) • Extracellular localization • High tumor expression (ideally with applicability across tumor types) • Low healthy tissue expression Key commercial considerations • Commercial landscape and competitive intensity • Potential speed to PoC • Organizational capabilities Selection to determine suitability for targeting with SADA PRIT Platform in Mind Prioritized target archetypes for different development strategies Good fit, good validation Targets with ADC / RLT validation and niche commercial opportunity Novel target, high-value Targets with less clinical validation and significant commercial opportunity High-risk, high-reward Targets with strong clinical validation but with a high degree of competition Validation Targets used as benchmarks against current RLTs Franchise 3 Franchise 1 Targets are diversified across tumor types, but also offer vertical franchise opportunities Franchise 2 25
Page 26
26 Commercial Progress DANYELZA® (naxitamab-gqgk) GD2 Antibody for R/R High-Risk Neuroblastoma
Page 27
Solid Drivers of Market Uptake • DANYELZA added to 48 hospital formularies since initial launch in 2021** • 113 HCPs prescribed DANYELZA since launch+ • DANYELZA remains an important therapy in U.S. anti-GD2 market DANYELZA: Only FDA-Approved Medicine for R/R HR NB Patients 27 Neuroblastoma • NB forms in certain types of nerve tissue, most frequently starting from adrenal glands; can also develop in the neck, chest, abdomen or spine • NB is the most common cancer in infants FDA Approval for R/R HR Neuroblastoma (NB) • Differentiated therapy: › Humanized antibody › Rapid infusion, modest toxicity › Administered in outpatient treatment setting • U.S. addressable market: › 2L NB: 300 patients › 40% of NB patients are HR Global Commercial Launch Performance • Preliminary estimated unaudited FY 2024 net sales of approx. $88.0 million* • 68 sites across the U.S. have utilized DANYELZA** • Ex-U.S. commercial ramp progressing in China, Brazil and Mexico • Strong demand through NPP in Europe and Turkey*** * Unaudited, as of January 12, 2025 ** As of November 8, 2024 *** Named Patient Program This indication is approved under accelerated approval. Continued approval for this indication contingent upon verification a nd description of clinical benefit in a confirmatory trial(s). Preliminary results are unaudited and based on management’s initial review of the Company’s results as of and for the year en ded December 31, 2024, and are subject to revision based upon the Company’s year -end closing procedures and the completion of the audit by the Company’s external auditors of the C ompany’s December 31, 2024 financial statements.
Page 28
Pivotal Study 201 Data: Waterfall Plot of Change in Curie Scorein all Relapsed/Refractory Patients withBone Disease(n = 48) Adapted from: Kushner B, et al. Poster presented at ESMO-IO; Geneva, Switzerland; December 6-8, 2023 28
Page 29
Ongoing and Potential New Studies for Naxitamab: Exploring Expansion of Usage in New Indications Cancer Indications Treatable Patient Population (U.S.) GD2 Expression High-Risk Neuroblastoma Relapsed / Refractory 300 ~ 99-100% Front-line Induction 450 Osteosarcoma Relapsed/Recurrent 200 ~ 88% Soft-Tissue Sarcomas Including Ewings 2,900 (1st-line population) > 90% Breast Cancer Triple Negative / Advanced 8,900 (2nd line & 3rd line +) > 50% Melanoma Newly Unresectable and Metastatic 11,400 (2nd line & 3rd line +) > 50% 2022 2023 2024 2025 2026 R/R HRNB Confirmatory Study 201* Relapsed Osteosarcoma MSKCC Study 15-096 Pivotal RCT** ISS – Ongoing Phase 2 (Ewings) ISS – Ongoing Phase 2 ISS – Area of Interest * This indication is approved under accelerated approval. Continued approval for this indication contingent upon verification and description of clinical benefit in a confirmatory trial(s). ** Subject to data readout of MSKCC study 15 -096. 1st line Induction BCC-018 Phase 2 1st line Induction RCT BCC study 29
Page 30
30 DANYELZAAddresses Significant Unmet Needs in R/R High-Risk NB with Expansion Potential Across Broader Patient Populations › U.S. commercialization in HR RR NB › Expanding ex-U.S. reach › Commercially available in China through partner SciClone, LATAM partner Adium in Brazil and Mexico › EU and Turkey access via WEP › Studies 12-230 and 201 formed primary basis of approval in November 2020 › Reached 100 patients in Study 201 › Multiple potential advantages over other anti-GD2 therapies: › Modest toxicity › Shorter infusion time › Ability to be administered in outpatient setting › Granted ODD and BTD › Frontline study ongoing
Page 31
31 Company Takeaways
Page 32
Advancing Focused Pipeline with Multiple Potential Value-Added Catalysts Ahead 32 Study Therapeutic Area Preclinical Phase 1 Phase 2/Pivotal Approved Trial Sponsor Status Lead Programs Naxitamab-gqgk (Anti-GD2) 201 Relapsed/Refractory High-Risk Neuroblastoma (Pediatric) U.S. FDA approved 12-230 Relapsed/Refractory High-Risk Neuroblastoma (Pediatric) U.S. FDA approved BCC018 Front-Line Induction in High-Risk Neuroblastoma (Pediatric) Expect primary completion in 2026 15-096 Relapsed Second-Line Osteosarcoma Potential pivotal trial 17-251 Chemoimmunotherapy for Relapsed/ Refractory High-Risk Neuroblastoma Study completed Butterfly Refractory Ewing’s Sarcoma Expect completion in 2028 Metastatic Breast Cancer Trial initiated in Q2 2024 SADA PRIT (Radioimmunotherapy) 1001 GD2-SADA: Solid Tumors (SCLC, Malignant Melanoma, Sarcoma) Ongoing Part A 1201 CD38-SADA: Non-Hodgkin Lymphoma Expect FPI in Q1 2025 DANYELZA (naxitamab-gqgk) Confirmatory Trial DANYELZA (naxitamab-gqgk) Clinicaltrials.gov: BCC trial NCT05489887, MSK trial NCT02502786, OSU trial NCT06026657 Potential High-Value SADA PRIT Targets and Pipeline to be Presented in Q2 2025
Page 33
33 Anticipated 2025 Milestones Ongoing GD2-SADA Phase 1 Trial (Trial 1001) › Part A data anticipated in Q2 2025 › Optimization data anticipated in Q2 2025 › Part B update anticipated in Q2 2025 Ongoing CD38-SADA Phase 1 Trial (Trial 1201) › First-patient-in anticipated in Q1 2025 New Potential High-Value SADA PRIT and Updated Pipeline in Q2 2025 Anticipate new ex-U.S. marketing approval for DANYELZA High-value opportunity to advance naxitamab in osteosarcoma Provide FY 2025 guidance at FY 2024 earnings report in Q1 2025
Page 34
34 Next-Generation Novel Platforms Self-Assembly DisAssembly Pretargeted Radioimmunotherapy (“SADA PRIT”) Platform Monoclonal Antibodies Established Commercial Capabilities DANYELZA (naxitamab- gqgk), Anti-GD2 Approved for R/R High-Risk Neuroblastoma U.S. Commercial Footprint; Ex-U.S. through partnerships, NPPs Radiopharmaceutical Leadership Deep bench of industry leadership and expertise in developing and commercializing radiopharmaceutical oncology therapeutics Broad Pipeline Potential SADA PRIT's proven mechanism of pre-targeted approach carries therapeutic potential beyond oncology Growing Base Business with Potential High Value RIT Platform
Page 35
THANK YOU