Passionately pursuing a better life for patients with cancer July 2020 Pipeline Overview Exhibit 99.1
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Clinical Preclinical Phase 1 Phase 2 Phase 3 Regulatory Filing Marketed Partner Tivozanib (RCC) VEGFR TKI Tivozanib (HCC) VEGFR TKI Ficlatuzumab Anti-HGF/c-MET mAb AV-203 (CAN017) Anti-ERBB3 mAb Multiple Potential Opportunities for Value Creation TIVO-1 (Phase 3 Registration Study in 1st Line RCC) TIVO-3 (Phase 3 Registration Study in 3rd Line RCC) TiNivo (+OPDIVO® RCC) HNSCC (Ficla +/- ERBITUX®) Oncology Pre-Clinical Tivozanib VEGFR TKI AV-380 Anti-GDF15 mAb AV-353 Anti-Notch 3 mAb Cachexia Oncology DEDUCTIVE (1st line HCC+ IMFINZI®) (Europe) Pancreatic (Ficla + Nab-paclitaxel + Gemcitabine) Non-Oncology CyFi-1 (AML HiDac +/- Ficla)
Ficlatuzumab Anti-HGF/c-MET mAb
Ficlatuzumab: A Potent anti-HGF/c-MET mAb Hepatocyte growth factor (HGF) pathway is frequently dysregulated in a broad range of human cancers HGF can lead to both tumor growth and metastatic progression of cancer cells when dysregulated and is an escape mechanism to CT and targeted therapies, including EGFR inhibitors Ficlatuzumab has demonstrated differentiated inhibition of HGF High affinity (pM) and slow off-rate for HGF High potency (nM) inhibiting all biological activities of HGF, including autocrine/paracrine activation loops Partnered with 1. Christensen JG, et al. Cancer Letters. 225:1-26. 2. Engelman JA, et al. Science. 316:1039-43. 3. Turke AB, et al. Cancer Cell. 17:77-88. 4. Yano S, et al. Cancer Res. 68:9479-87.
HNSCC: Randomized Phase 2 Trial Ongoing (Ficlatuzumab + Erbitux vs. Ficlatuzumab) Promising Phase 1 data - ASCO 2017 Topline Phase 2 data expected in 2021 AML: Randomized Phase 2 Trial On Hold Due to COVID-19 (Ficlatuzumab + Cytarabine vs. Ficlatuzumab) Promising Phase 1/2 data - AACR 2019 Pancreatic Cancer: Phase 1/2 Trial (Ficlatuzumab + Nab-paclitaxel + Gemcitabine) Topline data reported ASCO GI 2020 Ficlatuzumab: Recently Completed and Ongoing Studies in Multiple Tumor Types with Potential to Address Significant Unmet Needs *Source: NIH 2020 SEER Cancer Stats
Ficlatuzumab: Head & Neck Cancer (HNSCC)
Ficlatuzumab: Rationale in HNSCC HGF levels elevated in HNSCC tumor tissue ASCO 2017 Poster
Ficlatuzumab + Cetuximab: Phase 1 HNSCC Study Results in Cetuximab-Resistant Patients Combination was well tolerated with promising activity (ORR 17%, PFS 6mo, OS 8.2mo) ASCO 2017 Poster
Ficlatuzumab: Phase 2 Confirmatory Study Currently Enrolling Cetuximab-Resistant Patients Due to CPI 1st line approvals study is recruiting 3rd line pts after CPI and Erbitux failures. Results expected 2021 * IRB Requiring Prior IO *
Ficlatuzumab: Acute Myeloid Leukemia (AML)
Ficlatuzumab: Rationale in AML ASCO Poster 2017
All enrolled patients experienced induction failure to 1st line treatment where 25-30% CR rate expected from chemo alone Ficlatuzumab CyFi-1 Study: 50% CR Rate in Difficult to Treat AML Population ASCO Poster 2017
Ficlatuzumab: Pancreatic Cancer
Combination showed durable treatment responses Significant overlapping edema, study with alternate chemotherapy combo to be considered when new clinical supply is available ASCO GI Poster 2020
The c-Met-HGF axis promotes aberrant tumor cell growth, migration, proliferation, and angiogenesis Abnormal signaling results from c-Met over-expression, amplification or mutation of the c-Met receptor, or HGF over-expression C-Met expression is associated with expression of immunoregulatory proteins (PD-L1, IDO, IFN-g) that suppress the immune response Limited pre-clinical and clinical data suggest inhibition of c-Met signaling has direct and indirect anti-tumor effects via attenuation of immunosuppression in the tumor microenvironment Checkpoint inhibitors are less effective in tumors with driver mutations, such as c-Met; combination regimens with targeted agents may provide effective concurrent therapies Front Oncol 2020;10:54 Ficlatuzumab: Immunotherapy Combination Opportunities Emerging
Ficlatuzumab Summary Potential Ficlatuzumab (HGF MAb) Milestones 1H 20 2H 20 2021 HNSCC Randomized (Ficlatuzumab +/- ERBITUX®) Ph2 Confirmatory Data Pancreatic (Ficlatuzumab + Nab-paclitaxel + Gemcitabine) Ph1/2 Presentation AML CyFi-2 Randomized (DTIC +/- Ficlatuzumab) Ph2 Study Start HNSCC: Randomized Phase 2 trial ongoing (Ficlatuzumab +/- ERBITUX®) Pancreatic Cancer: Phase 1/2 trial complete (Ficlatuzumab + Nab-paclitaxel + Gemcitabine) AML: Phase 1 trial complete (Ficlatuzumab+DTIC) Explore other combinations with scientific rationale: Checkpoint Inhibitors Expected exclusivity in U.S. into 2033* * Composition of matter to 2028 and a potential 5-year PTE ü On Hold Due to COVID-19
AV-380 Anti-GDF15 mAb
Cancer Cachexia Impacts Patients Across a Broad Range of Tumor Types Cachexia is a complex metabolic syndrome associated with reduced food intake, muscle and adipose tissue loss, inflammation and anemia Approximately 50% of people with cancer will suffer from cachexia* Approximately 30% of all cancer patients die from cachexia* GDF15 is a key driver in cancer cachexia: GDF15 is elevated in cachexic models/ patients (vs. non-cachexic) Expression of GDF15 induces cachexia Inhibition of GDF15 reverses cachexia in animal models Managing cachexia has the potential to improve OS AVEO Market Research * J. Cachexia, Sarcopenia and Muscle 2010
Greater Than 70% of Cancer Patients Have Increased GDF15 Expression, Starting at the Pre-Cachectic Stage ** *** ** In collaboration with A Vigano % Patients Cancer Non-Cachexia Pre-cachexia Cachexia Refractory Cachexia GDF15 < 2.0 (ng/mL) 51% 29% 30% 21% GDF15 > 2.0 (ng/mL) 49% 71% 70% 79% GDF15 Distribution Across Patient Samples Average GDF15 in Patient Samples Adjusted by tumor stage, GDF15 expression is the top factor that predicts cachexia stage with significance
Tumors Engineered to Overexpress GDF15 Cause Severe Weight Loss, Inhibition of GDF15 Reverses the Cachexia Phenotype Induced by Tumor Inhibition of GDF15 Reverses Cachexia in Animal Models 2mpk mIgG 2mpk anti-mGDF15 Ab Expression of GDF15 Induces Cachexia in Animal Models Lerner et al., (2016), J. of Cachexia, Sarcopenia and Muscle, 7: 467-482
GDF15 Has Been Shown to Predict Survival in Cancer Patients Survival analysis showed that GDF15 expression predicted survival adjusting for stage and weight change 1st quartile (low) 2nd quartile 3rd quartile 4th quartile (High) Percent Survival Days In collaboration with A. Vigano
Increased GDF15 protein expression found in airway epithelium (mainly in ciliated cells) of human COPD smokers compared with normal non-smokers1 Cigarette smoke exposure consistently up-regulated GDF15 expression in human airway epithelial cells1 Significant Market Opportunities Outside of Cancer* AVEO has conducted additional analyses of patient samples and animal models to examine the role of GDF15 in cachexia outside of cancer COPD >5x Larger than Cancer Cachexia Market Congestive Heart Failure 2x Larger than Cancer Cachexia Market GDF-15 Levels Predict Mortality in CHF2 Wu, Q et al. Innate Immunity 0(0), 16 Dec 2011 Kempf et al. & Wollert. JACC, 2007 *Partnership opportunity for non-oncology rights
AV-380 Development and Exclusivity Program Development Status – Focused on Cancer Cachexia AV-380 is currently in preclinical development AVEO preparing IND for planned 2020 filing Intellectual Property Estate Method of use patent through 2029 to cover increasing body weight (e.g., treating cachexia) by inhibiting GDF15 Composition of matter on AV-380 antibodies through 2033
Key Potential Clinical and Regulatory Milestones AV-380 (Anti-GDF15 mAb) 1H 20 2H 20 2021 AV-380 IND Submission for Cancer Cachexia CAN017 (AV-203) (Anti-ERBB3 mAb) 1H 20 2H 20 2021 Initiate POC Study upon NRG1 Fusion Biomarker Evaluation Ficlatuzumab (Anti-HGF/c-MET mAb) 1H 20 2H 20 2021 HNSCC (Ficlatuzumab +/- ERBITUX®) Randomized Ph2 POC Data Pancreatic (Ficlatuzumab + Nab-paclitaxel + Gemcitabine) Ph1/2 Presentation ü Tivozanib (VEGFR 123 TKI) 1H 20 2H 20 2021 PDUFA Date (R/R RCC) Final OS HCC Ph1/2 (Durvalumab + Tivo) Ph2 HCC Ph1/2 (Durvalumab + Tivo) Ph1 ü ü Tivozanib (VEGFR 123 TKI, Non Oncology) 1H 20 2H 20 2021 Regulatory/Development Milestone
Passionately pursuing a better life for patients with cancer July 2020 Pipeline Overview