YMAB
Oncology Leadership in
Pretargeted Radioimmunotherapy
Platform and Antibody-based
Therapies
November 2024
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 regarding our strategy, future operations, future financial position, future revenue, projected costs, prospects, plans and objectives of management and expected market growth are forward-looking statements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "might," "plan," "potential," "predict," "project," "should," "target," "would," "goal," "objective," "guidance," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements include, but are not limited to, statements about early clinical data, regulatory matters, clinical trial timing and plans, the achievement of clinical and commercial milestones, future financial and operating results, including 2024 financial guidance and beyond and anticipated future cash and cash equivalents, business strategies, market opportunities, financing, and other statements that are not historical facts. 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; risks associated with the Company's development work, including any delays or changes to the timing, cost and success of our product development activities and clinical trials including if we encounter difficulties enrolling patients in our clinical trials; the risks of delays in FDA and/or EU approval of our drug candidates or failure to receive approval; the risks related to commercializing any approved new 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; risks related to our dependence on third parties including for conduct of clinical testing and product manufacture; our ability to enter into collaboration or other arrangements with partners; risks associated with protection of our intellectual property rights; 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 fiscal year ended December 31, 2023, our Quarterly Reports on Form 10-Q for the quarters ended March 31, 2024, June 30, 2024 and September 30, 2024, in addition to other reports the Company files from time to time with the Securities and Exchange Commission. 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.
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Strongly Positioned to Drive Future Value
Novel Platforms
Commercial
Anticipated 2024
Capital
in Development
Leverage
Milestones
Efficiency
Self-Assembly
DANYELZA
GD2-SADA Phase 1
Independent
DisAssembly Pretargeted
(naxitamab-gqgk)
Part A Completion
Commercial-stage
Radioimmunotherapy
Biotech Company with
("SADA PRIT")
Anti-GD2 Antibody
CD38-SADA Phase 1
Cash of $68.1 million*
Platform
Marketed for R/R High-
Study Initiation
Risk Neuroblastoma
Financial Runway
Monoclonal Antibodies
MSK Data Readout of
into 2027
FY2024 Total Net
Phase 2 Osteosarcoma
Revenue Guidance of
Trial Anticipated
Strategic Capital
$87-95 million
Deployment
* As of September 30, 2024
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Advancing Focused Pipeline with Multiple Value-Added Catalysts Ahead
Study
Therapeutic Area
Lead Programs
Preclinical
Phase 1
Phase 2/Pivotal
Approved
Trial Sponsor
Status
Relapsed/Refractory High-Risk
201
DANYELZA (naxitamab-gqgk) Confirmatory Trial
Neuroblastoma (Pediatric)
Relapsed/Refractory High-Risk
12-230
DANYELZA (naxitamab-gqgk)
Neuroblastoma (Pediatric)
Front-Line Induction in High-Risk
BCC018
Neuroblastoma (Pediatric)
Naxitamab-gqgk
15-096
Relapsed Second-Line Osteosarcoma
(Anti-GD2)
17-251
Chemoimmunotherapy for Relapsed/
Refractory High-Risk Neuroblastoma
Butterfly
Refractory Ewing's Sarcoma
Metastatic Breast Cancer
1001
GD2-SADA: Solid Tumors (SCLC,
SADA PRIT
Malignant Melanoma, Sarcoma)
(Radioimmunotherapy)
1201
CD38-SADA: Non-Hodgkin Lymphoma
Early Programs
1002
GD2-SADA: Neuroblastoma
SADA PRIT
HER2-SADA
(Radioimmunotherapy)
B7H3-SADA
U.S. FDA approved
U.S. FDA approved
Randomized trial expected 2H 2024
Expected MSK data readout in Q4 2024
Study completed
Initiated in Q4 2023
Expect FPI in 2H 2024
Expect to complete Part A in 2024
Expect FPI in Q4 2024
Expected IND filing in 2025
Expected IND filing in 2025
Expected IND filing in 2025
Clinicaltrials.gov: BCC trial NCT05489887, MSK trial NCT02502786, OSU trial NCT06026657
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Novel SADA Pretargeted
Radioimmunotherapy
Platform
Current Radiopharma Challenges Negatively Impact Patient Care
Infrastructure and
Physician
Administration
Continuing Drug
Participation
Sites
Shortages
Manufacturing
Simpler, more user-friendly solutions greatly needed for physicians and patients
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Potential Improved Capabilities of Novel SADA PRIT 2-Step Approach
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 radiologists
High investment needed for specific infrastructure and manufacturing
* Pending successful development and approval.
Novel SADA PRIT Platform
Potential Capabilities
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Self-Assembly DisAssembly (SADA) Technology: High Affinity for Tumor Targets and Rapid Clearance from Blood Stream
SADA domains uniquely selected to allow proteins to change size based on concentration
Self-Assembled Tetramer
DisAssembled Monomer
~200 kDa
< 70kDa
Strong Tumor Binding
Rapid Clearance
Adapted from Santich et al. Clin Canc Res 2020
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GD2-SADA Achieves High Tumor Uptake with Minimal Exposure to All Other Tissues
Blood Exposure
(Radioactive Payload)
Concentration (Radioactive Payload)
Tumor
1. IgG Bispecific
Time
2. DOTA
Tumor Uptake
(Radioactive Payload)
1. SADA Bispecific
2. DOTA
Time
Adapted from Santich et al. Clin Canc Res 2021
These early results are not complete and are not necessarily indicative of the full results or ultimate success of the trials or SADA development program.
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GD2-SADA Phase 1 Clinical Trial (Study 1001): Dosing Patients in Part A
Theranostic approach using a 30 mCi 177Lu-DOTA imaging dose before exposing to therapeutic dose
Inclusion Criteria
Inclusion Amendment
*Currently in Part A
DESIGN
OUTCOME
Part A*
N ≈ 15 - 18
GD2-SADA protein
dose-escalation from 0.3
mg/kg to 10 mg/kg
+
Therapeutic 177Lu-DOTA
dose of 100-200 mCi
(7.4 GBq)
Determine optimal, safe GD2-SADA protein dose and dosing interval between GD2-SADA and 177Lu-DOTA
Part B
N ≈ 9 - 12
177Lu-DOTA therapeutic
dose escalation from 400 mCi (14.8 GBq) to 750 mCi (27.8 GBq)
Patients to receive up to
2 cycles
Determine maximum tolerable activity of 177Lu-DOTA
Part C
5 cycles
Doses determined in
Part A and B administered
Patients to receive maximum of 5 cycles
Assess safety and AEs following repeat dosing; determine recommended Phase 2 dose (RP2D)
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Disclaimer
Y-mAbs Therapeutics Inc. published this content on November 11, 2024, and is solely responsible for the information contained herein. Distributed by Public, unedited and unaltered, on November 11, 2024 at 20:45:02.607.