phase 1/2 study of ctla-4 inhibitor agen1884 + …agenusbio.com › wp-content › uploads › 2018...

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BACKGROUND Antigen-specific T-cell activation is regulated by a balance of co-stimulatory and co-inhibitory signals, such as those mediated by inhibitory receptors cytotoxic T-lymphocyte–associated antigen 4 (CTLA-4) and programmed cell death protein-1 (PD-1) (Figure 1). 1 Binding of these receptors to their ligands results in impaired T-cell function. For these reasons, antibody blockade of PD-1 and CTLA-4 has been identified as a therapeutic modality to reinvigorate or induce tumor-specific T-cell immunity. 1 Figure 1. Overview of Pathways Affected by CTLA-4 and PD-1 CTLA-4 Inhibition PD-1 Inhibition In clinical trials, the combined inhibition of PD-1 and CTLA-4 pathways by blocking receptor-ligand interactions has resulted in objective clinical response and increased survival in several solid tumor indications, including melanoma and non-small cell lung carcinoma. As a result, ipilimumab (anti–CTLA-4) in combination with nivolumab (anti–PD-1) has been approved as a first line of treatment in patients with metastatic melanoma and is currently being tested in multiple other indications. 2-4 Agenus’ proprietary antibodies, AGEN2034 (anti–PD-1 human IgG4 monoclonal antibody) and AGEN1884 (anti–CTLA-4 human IgG1 monoclonal antibody) are currently under evaluation as monotherapy in phase 1/2 studies in subjects with advanced tumors (NCT03104699 and NCT02694822, respectively). A phase 1/2, open-label, multi-arm trial to investigate the safety, tolerability, pharmacokinetics, and biological and clinical activity of AGEN1884 in combination with AGEN2034 in patients with metastatic or locally advanced solid tumors is currently ongoing (C550-01; ACTRN12618000003279; NCT03495882). OBJECTIVE The objective of this study is to assess safety and tolerability of AGEN1884 in combination with AGEN2034 in patients with advanced/refractory solid tumors, with expansion into select solid tumors. METHODS This is an ongoing phase 1/2, open-label, study of AGEN1884 in combination with AGEN2034 in subjects with advanced solid tumors, including cervical cancer. The study consists of 2 phases: – Phase 1: Dose escalation (focus of this poster) – Phase 2: Expansion in select tumors (ongoing) Phase 1 consisted of a standard 3+3 dose escalation with the following escalating dose levels and schedules: – AGEN2034 1 mg/kg administered every 2 weeks (q2w) in combination with AGEN1884 1 mg/kg administered every 6 weeks (q6w) – AGEN2034 3 mg/kg administered every 2 weeks (q2w) in combination with AGEN1884 1 mg/kg administered every 6 weeks (q6w) AGEN2034 was administered IV over 60 min and AGEN1884 IV over 90 mins. AGEN1884 was to be administered on the same day as AGEN2034 ≥30 min after the completion of AGEN2034 administration. Dose escalation will continue until AGEN2034 3 mg/kg + AGEN1884 1 mg/kg (maximum combination dose level) is shown to be safe or the maximum tolerated dose (defined as the combination below which ≥33% of subjects develop dose-limiting toxicities [DLTs]) is reached. A schematic of the phase 1 dose-escalation design is presented in Figure 2. Figure 2. Dose-Escalation Study Flow Starting dose Treat 3 patients; Assess DLTs after 3 weeks Number of DLTs No Yes ≤1 of 6 0 of 3 ≥2 of 3 ≥2 of 6 1 of 3 1 of 3 Treat additional 3 patients; Assess DLTs after 3 weeks Number of DLTs MTD/maximum CDL to be confirmed on total of 6 patients Initiate phase 2 at maximum planned CDL or MTD Determine MTD Escalate to next CDL Maximum CDL per protocol? Determine MTD CDL, combination dose level; DLT, dose-limiting toxicity; MTD, maximum tolerated dose. Each subject will receive the combination treatment for a maximum of 24 months or until confirmed disease progression, unacceptable toxicity, or any criterion for withdrawal from the trial or the investigational medicinal products occurs. – Patients who do not complete the DLT observation period of 21 days after the first dose, for reasons other than a DLT, will be replaced. – Cohorts will be backfilled to 10 subjects once they have reached DLT or MTD. Eligible patients include adults (aged ≥18 years) with a histologically or cytologically confirmed diagnosis of a metastatic or locally advanced solid tumor for which no standard therapy is available or standard therapy has failed. The treatment phase was divided into 6-week cycles, each beginning with the combined administration of AGEN2034 and AGEN1884 on day 1. Thereafter, AGEN2034 will be administrated q2w, completing the 6-week cycle. – Tumor assessments are to be conducted at 6, 12, and 18 weeks from first dose, and every 9 weeks thereafter until disease progression is confirmed or a new line of therapy is initiated. Phase 1 endpoints: – Primary: Occurrence of DLTs in patients in dose escalation during the first 21 days of treatment – Secondary: Confirmed best overall response per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1, as determined by the investigator Frequency and nature of treatment-emergent adverse events (TEAEs) Pharmacokinetic profile and immunogenicity of AGEN1884 and AGEN2034 RESULTS As of July 16, 2018, the study was ongoing, though total enrollment was achieved in the phase 1 portion (ie, 10 patients in each dose cohort). Data are preliminary as it is being cleaned and/or collected at this time. Five patients had discontinued from the study treatment prior to data extract: – AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w: Progressive disease, n=1; investigator decision, n=1 – AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w: Progressive disease, n=3 There was 1 death not related to the study (66-year-old female with lung cancer treated with AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w died due to disease progression). Patients were Caucasian (100%) and primarily female (75%), with a median of 2.3 months from the most recent recurrence/progression to the administration of the first combination dose (Table 1). The cancer types for the 20 patients were as follows: breast cancer (triple negative, n=2; with lung metastases, n=1; with dermal lymphatics, n=1), ovarian cancer (n=2), mesothelioma (pleural, n=1; peritoneal, n=1), squamous carcinoma of head and neck (tongue; n=1), anal squamous carcinoma (n=1), pleomorphic soft tissue sarcoma (n=1), lung cancer (n=1), metastastic leiomyosarcoma (n=1), advanced gastrointestinal stromal tumor (n=1), rhabdomyosarcoma (n=1), alveolar rhabdomyosarcoma (n=1), recurrent chordoma of the thoracic spine (n=1), chordoma (n=1), colorectal-rectal (n=1), esophageal adenocarcinoma (n=1). Table 1. Demographics and Baseline Characteristics (Safety Set) AGEN2034 1 mg/kg + AGEN1884 1 mg/kg (N=10) AGEN2034 3 mg/kg + AGEN1884 1 mg/kg (N=10) Total Patients (N=20) Age (years) Mean (SD) 58.4 (8.13) 47.6 (19.68) 53.0 (15.66) Min, Max 43, 69 21, 79 21, 79 Female, n (%) 8 (80%) 7 (70%) 15 (75%) BMI (kg/m 2 ) Mean (SD) 28.71 (8.227) 27.48 (7.006) 28.10 (7.464) Min, Max 18.8, 40.9 20.9, 45.5 18.8, 45.5 ECOG performance status, n (%) 0 5 (50%) 7 (70%) 12 (60%) 1 5 (50%) 3 (30%) 8 (40%) Time between most recent progression and first dose date (months) n 6 6 12 Mean (SD) 2.26 (1.665) 2.34 (1.002) 2.30 (1.311) BMI, body mass index; ECOG, Eastern Cooperative Oncology Group. An overview of safety and tolerability is presented in Table 2. Table 2. Overview of Treatment-Emergent Adverse Event (Safety Set) Patients, n (%) AGEN2034 1 mg/kg + AGEN1884 1 mg/kg (N=10) AGEN2034 3 mg/kg + AGEN1884 1 mg/kg (N=10) Total Patients (N=20) Any TEAE 10 (100%) 9 (90%) 19 (95%) Any serious TEAE 3 (30%) 2 (20%) 5 (25%) Any grade ≥3 TEAE 3 (30%) 2 (20%) 5 (25%) Any immune-related TEAE 5 (50%) 4 (40%) 9 (45%) Any treatment-related TEAE 8 (80%) 7 (70%) 15 (75%) Any treatment-related serious TEAE 1 (10%) 2 (20%) 3 (15%) Any treatment-related grade ≥3 TEAE 0 2 (20%) 2 (10%) TEAE, treatment-emergent adverse event. As of July 16, 2018, no DLTs have been observed, and none of the TEAEs led to discontinuation or death. TEAEs were reported in 19 of 20 patients (95%), with the most common MedDRA version 18.1 preferred terms listed in (Table 3). Most TEAEs were mild to moderate in severity (grade 1 or 2); 5 patients experienced TEAEs of grade ≥3. – AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w: Vomiting, n=1; wound and cancer pain, n=1; hypotension, n=1 – AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w: Lower respiratory tract infection and pneumonia, n=1; pulmonary embolism, n=1 – Of these, the events of lower respiratory tract infection and pulmonary embolism were considered related to study treatment by the investigator. Table 3. Most Common* Treatment-Emergent Adverse Event (Safety Set) Patients, n (%) AGEN2034 1 mg/kg + AGEN1884 1 mg/kg (N=10) AGEN2034 3 mg/kg + AGEN1884 1 mg/kg (N=10) Total Patients (N=20) Fatigue 5 (50%) 5 (50%) 10 (50%) Nausea 3 (30%) 4 (40%) 7 (35%) Pruritus 2 (20%) 3 (30%) 5 (25%) Chills 3 (30%) 1 (10%) 4 (20%) Vomiting 2 (20%) 2 (20%) 4 (20%) Back pain 4 (40%) 0 4 (20%) *Occurring in ≥20% of patients. Serious TEAEs occurred in 5 patients: 3 receiving AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w and 2 receiving AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w 3 patients experienced serious TEAEs considered related to study treatment. – AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w: Diarrhea, n=1 – AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w: Lower respiratory tract infection, n=1; pulmonary embolism, n=1 The summary of best overall response is listed in Table 4. The partial response was ongoing in one of the women with metastatic ovarian cancer. For patients with >90 days of treatment, stable disease was ongoing for 1 female with breast cancer (with lung metastases), 1 female with metastatic ovarian cancer, and 1 male with pleomorphic soft tissue sarcoma (with lung metastases). Table 4. Summary of Best Overall Response at Time of Data Cut-off Patients, n (%) AGEN2034 1 mg/kg + AGEN1884 1 mg/kg (N=10) AGEN2034 3 mg/kg + AGEN1884 1 mg/kg (N=10) Total Patients (N=20) Complete response 0 0 0 Partial response 1 0 1 Stable disease 5 1 6 Progressive disease 3 6 9 Not evaluable 1 0 1 Pending 0 3 3 Overall response determined by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. DISCUSSION In the phase 1 portion, which is still ongoing, preliminary results demonstrate that AGEN1884 (1 mg/kg q6w) + AGEN2034 (3 mg/kg q2w) is generally safe, well tolerated, and active in adults with select advanced/refractory solid tumors. The phase 2 portion of this study evaluating AGEN1884 in combination with AGEN2034 in adults with second-line cervical cancer and other solid tumors is ongoing. – The phase 2 recommended dosing was determined as AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w. References 1. Topalian SL, Drake CG, Pardoll DM. Cancer Cell. 2015;27(4):450-61. 2. Hodi FS, Chesney J, Pavlick AC, et al. Lancet Oncol. 2016;17(11):1558-68. 3. Antonia SJ, López-Martin JA, Bendell J, et al. Lancet Oncol. 2016;17(7):883-95. 4. Hellmann MD, Gettinger SN, Goldman JW, et al. J Clin Oncol. 2016;34(15 suppl):3001.5 Disclosures CD Dupont, AM Gonzalez, M Lim, D Savitsky, M Carini, S Hu, O Shebanova, E Dow, W Ortuzar, JS Buell, RB Stein, H Youssoufian: Agenus Inc.: current or former employment/consultancy and stock ownership. Acknowledgments The authors thank Nicole Coolbaugh of The Medicine Group, LLC (New Hope, PA, USA) for providing medical writing and editorial support, which was funded by Agenus Inc. (Lexington, MA, USA) in accordance with Good Publication Practice guidelines. The licensed antibodies AGEN2034 and AGEN1884 were originally developed under a Collaborative Research and Development Agreement between Ludwig Cancer Research, 4-Antibody AG (now Agenus Switzerland Inc.) and Recepta Biopharma S.A. Recepta Biopharma S.A. has exclusive rights to these antibodies in Brazil and five other South American countries. Funding This study was funded by Agenus Inc. (Lexington, MA, USA). Correspondence Waldo Ortuzar: [email protected] Presented at the 2018 Annual Congress of the European Society for Medical Oncology (ESMO), October 19–23, 2018, in Munich, Germany Phase 1/2 Study of CTLA-4 Inhibitor AGEN1884 + PD-1 Inhibitor AGEN2034 in Patients With Advanced/Refractory Solid Tumors, With Expansion Into Second-Line Cervical Cancer and Solid Tumors Jermaine Coward, 1 Charlotte Lemech, 2 Tarek Meniawy, 3 Christopher D. Dupont, 4 Ana M. Gonzalez, 4 Min Lim, 4 David Savitsky, 4 Meg Carini, 4 Shuang Hu, 4 Olga Shebanova, 4 Ed Dow, 4 Waldo Ortuzar, 4 Jennifer S. Buell, 4 Robert Benjamin Stein, 4 Hagop Youssoufian 4 1 Icon Cancer Center, South Brisbane, Australia; 2 Scientia Clinical Research, Sydney, Australia; 3 Linear Clinical Research, Perth, Australia; 4 Agenus Inc. or subsidiary thereof (current or former employee), Lexington, MA, USA 1168P

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Page 1: Phase 1/2 Study of CTLA-4 Inhibitor AGEN1884 + …agenusbio.com › wp-content › uploads › 2018 › 10 › ESMO-C-550-01...1 mg/kg q2w + AGEN1884 1 mg/kg q6w died due to disease

BACKGROUND• Antigen-speci� c T-cell activation is regulated by a balance of co-stimulatory and co-inhibitory signals, such as

those mediated by inhibitory receptors cytotoxic T-lymphocyte–associated antigen 4 (CTLA-4) and programmed cell death protein-1 (PD-1) (Figure 1).1

• Binding of these receptors to their ligands results in impaired T-cell function. For these reasons, antibody blockade of PD-1 and CTLA-4 has been identi� ed as a therapeutic modality to reinvigorate or induce tumor-speci� c T-cell immunity.1

Figure 1. Overview of Pathways Affected by CTLA-4 and PD-1CTLA-4 Inhibition PD-1 Inhibition

• In clinical trials, the combined inhibition of PD-1 and CTLA-4 pathways by blocking receptor-ligand interactions has resulted in objective clinical response and increased survival in several solid tumor indications, including melanoma and non-small cell lung carcinoma. As a result, ipilimumab (anti–CTLA-4) in combination with nivolumab (anti–PD-1) has been approved as a � rst line of treatment in patients with metastatic melanoma and is currently being tested in multiple other indications.2-4

• Agenus’ proprietary antibodies, AGEN2034 (anti–PD-1 human IgG4 monoclonal antibody) and AGEN1884 (anti–CTLA-4 human IgG1 monoclonal antibody) are currently under evaluation as monotherapy in phase 1/2 studies in subjects with advanced tumors (NCT03104699 and NCT02694822, respectively).

• A phase 1/2, open-label, multi-arm trial to investigate the safety, tolerability, pharmacokinetics, and biological and clinical activity of AGEN1884 in combination with AGEN2034 in patients with metastatic or locally advanced solid tumors is currently ongoing (C550-01; ACTRN12618000003279; NCT03495882).

OBJECTIVE• The objective of this study is to assess safety and tolerability of AGEN1884 in combination with AGEN2034 in

patients with advanced/refractory solid tumors, with expansion into select solid tumors.

METHODS• This is an ongoing phase 1/2, open-label, study of AGEN1884 in combination with AGEN2034 in subjects with

advanced solid tumors, including cervical cancer.

• The study consists of 2 phases: – Phase 1: Dose escalation (focus of this poster) – Phase 2: Expansion in select tumors (ongoing)

• Phase 1 consisted of a standard 3+3 dose escalation with the following escalating dose levels and schedules: – AGEN2034 1 mg/kg administered every 2 weeks (q2w) in combination with AGEN1884 1 mg/kg administered every 6 weeks (q6w)

– AGEN2034 3 mg/kg administered every 2 weeks (q2w) in combination with AGEN1884 1 mg/kg administered every 6 weeks (q6w)

• AGEN2034 was administered IV over 60 min and AGEN1884 IV over 90 mins. AGEN1884 was to be administered on the same day as AGEN2034 ≥30 min after the completion of AGEN2034 administration.

• Dose escalation will continue until AGEN2034 3 mg/kg + AGEN1884 1 mg/kg (maximum combination dose level) is shown to be safe or the maximum tolerated dose (de� ned as the combination below which ≥33% of subjects develop dose-limiting toxicities [DLTs]) is reached.

• A schematic of the phase 1 dose-escalation design is presented in Figure 2.

Figure 2. Dose-Escalation Study Flow

Starting dose

Treat 3 patients;Assess DLTs after 3 weeks

Number ofDLTs

No

Yes

≤1 of 6

0 of 3 ≥2 of 3

≥2 of 6

1 of 3

1 of 3

Treat additional 3 patients;Assess DLTs after 3 weeks

Number of DLTs

MTD/maximum CDL to be con�rmed on total

of 6 patients

Initiate phase 2 at maximum planned CDL

or MTD

Determine MTDEscalate to next CDL

Maximum CDL per protocol?

Determine MTD

CDL, combination dose level; DLT, dose-limiting toxicity; MTD, maximum tolerated dose.

• Each subject will receive the combination treatment for a maximum of 24 months or until con� rmed disease progression, unacceptable toxicity, or any criterion for withdrawal from the trial or the investigational medicinal products occurs.

– Patients who do not complete the DLT observation period of 21 days after the � rst dose, for reasons other than a DLT, will be replaced.

– Cohorts will be back� lled to 10 subjects once they have reached DLT or MTD.

• Eligible patients include adults (aged ≥18 years) with a histologically or cytologically con� rmed diagnosis of a metastatic or locally advanced solid tumor for which no standard therapy is available or standard therapy has failed.

• The treatment phase was divided into 6-week cycles, each beginning with the combined administration of AGEN2034 and AGEN1884 on day 1. Thereafter, AGEN2034 will be administrated q2w, completing the 6-week cycle.

– Tumor assessments are to be conducted at 6, 12, and 18 weeks from � rst dose, and every 9 weeks thereafter until disease progression is con� rmed or a new line of therapy is initiated.

• Phase 1 endpoints: – Primary: Occurrence of DLTs in patients in dose escalation during the � rst 21 days of treatment – Secondary:

Con� rmed best overall response per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1, as determined by the investigator

Frequency and nature of treatment-emergent adverse events (TEAEs) Pharmacokinetic pro� le and immunogenicity of AGEN1884 and AGEN2034

RESULTS • As of July 16, 2018, the study was ongoing, though total enrollment was achieved in the phase 1 portion

(ie, 10 patients in each dose cohort). Data are preliminary as it is being cleaned and/or collected at this time.

• Five patients had discontinued from the study treatment prior to data extract: – AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w: Progressive disease, n=1; investigator decision, n=1 – AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w: Progressive disease, n=3

• There was 1 death not related to the study (66-year-old female with lung cancer treated with AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w died due to disease progression).

• Patients were Caucasian (100%) and primarily female (75%), with a median of 2.3 months from the most recent recurrence/progression to the administration of the � rst combination dose (Table 1).

• The cancer types for the 20 patients were as follows: breast cancer (triple negative, n=2; with lung metastases, n=1; with dermal lymphatics, n=1), ovarian cancer (n=2), mesothelioma (pleural, n=1; peritoneal, n=1), squamous carcinoma of head and neck (tongue; n=1), anal squamous carcinoma (n=1), pleomorphic soft tissue sarcoma (n=1), lung cancer (n=1), metastastic leiomyosarcoma (n=1), advanced gastrointestinal stromal tumor (n=1), rhabdomyosarcoma (n=1), alveolar rhabdomyosarcoma (n=1), recurrent chordoma of the thoracic spine (n=1), chordoma (n=1), colorectal-rectal (n=1), esophageal adenocarcinoma (n=1).

Table 1. Demographics and Baseline Characteristics (Safety Set) AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Age (years)

Mean (SD) 58.4 (8.13) 47.6 (19.68) 53.0 (15.66)

Min, Max 43, 69 21, 79 21, 79

Female, n (%) 8 (80%) 7 (70%) 15 (75%)

BMI (kg/m2)

Mean (SD) 28.71 (8.227) 27.48 (7.006) 28.10 (7.464)

Min, Max 18.8, 40.9 20.9, 45.5 18.8, 45.5

ECOG performance status, n (%)

0 5 (50%) 7 (70%) 12 (60%)

1 5 (50%) 3 (30%) 8 (40%)

Time between most recent progression and � rst dose date (months)

n 6 6 12

Mean (SD) 2.26 (1.665) 2.34 (1.002) 2.30 (1.311)

BMI, body mass index; ECOG, Eastern Cooperative Oncology Group.

• An overview of safety and tolerability is presented in Table 2.

Table 2. Overview of Treatment-Emergent Adverse Event (Safety Set)

Patients, n (%)

AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Any TEAE 10 (100%) 9 (90%) 19 (95%)

Any serious TEAE 3 (30%) 2 (20%) 5 (25%)

Any grade ≥3 TEAE 3 (30%) 2 (20%) 5 (25%)

Any immune-related TEAE 5 (50%) 4 (40%) 9 (45%)

Any treatment-related TEAE 8 (80%) 7 (70%) 15 (75%)

Any treatment-related serious TEAE 1 (10%) 2 (20%) 3 (15%)

Any treatment-related grade ≥3 TEAE 0 2 (20%) 2 (10%)

TEAE, treatment-emergent adverse event.

• As of July 16, 2018, no DLTs have been observed, and none of the TEAEs led to discontinuation or death.

• TEAEs were reported in 19 of 20 patients (95%), with the most common MedDRA version 18.1 preferred terms listed in (Table 3).

• Most TEAEs were mild to moderate in severity (grade 1 or 2); 5 patients experienced TEAEs of grade ≥3. – AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w: Vomiting, n=1; wound and cancer pain, n=1; hypotension, n=1

– AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w: Lower respiratory tract infection and pneumonia, n=1; pulmonary embolism, n=1

– Of these, the events of lower respiratory tract infection and pulmonary embolism were considered related to study treatment by the investigator.

Table 3. Most Common* Treatment-Emergent Adverse Event (Safety Set)

Patients, n (%)

AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Fatigue 5 (50%) 5 (50%) 10 (50%)

Nausea 3 (30%) 4 (40%) 7 (35%)

Pruritus 2 (20%) 3 (30%) 5 (25%)

Chills 3 (30%) 1 (10%) 4 (20%)

Vomiting 2 (20%) 2 (20%) 4 (20%)

Back pain 4 (40%) 0 4 (20%)*Occurring in ≥20% of patients.

• Serious TEAEs occurred in 5 patients: 3 receiving AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w and 2 receiving AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w

• 3 patients experienced serious TEAEs considered related to study treatment. – AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w: Diarrhea, n=1 – AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w: Lower respiratory tract infection, n=1; pulmonary embolism, n=1

• The summary of best overall response is listed in Table 4.

• The partial response was ongoing in one of the women with metastatic ovarian cancer. For patients with >90 days of treatment, stable disease was ongoing for 1 female with breast cancer (with lung metastases), 1 female with metastatic ovarian cancer, and 1 male with pleomorphic soft tissue sarcoma (with lung metastases).

Table 4. Summary of Best Overall Response at Time of Data Cut-off

Patients, n (%)

AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Complete response 0 0 0

Partial response 1 0 1

Stable disease 5 1 6

Progressive disease 3 6 9

Not evaluable 1 0 1

Pending 0 3 3Overall response determined by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1.

DISCUSSION• In the phase 1 portion, which is still ongoing, preliminary results demonstrate that AGEN1884 (1 mg/kg q6w) +

AGEN2034 (3 mg/kg q2w) is generally safe, well tolerated, and active in adults with select advanced/refractory solid tumors.

• The phase 2 portion of this study evaluating AGEN1884 in combination with AGEN2034 in adults with second-line cervical cancer and other solid tumors is ongoing.

– The phase 2 recommended dosing was determined as AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w.

References1. Topalian SL, Drake CG, Pardoll DM. Cancer Cell. 2015;27(4):450-61.2. Hodi FS, Chesney J, Pavlick AC, et al. Lancet Oncol.

2016;17(11):1558-68.

3. Antonia SJ, López-Martin JA, Bendell J, et al. Lancet Oncol. 2016;17(7):883-95.

4. Hellmann MD, Gettinger SN, Goldman JW, et al. J Clin Oncol. 2016;34(15 suppl):3001.5

Disclosures CD Dupont, AM Gonzalez, M Lim, D Savitsky, M Carini, S Hu, O Shebanova, E Dow, W Ortuzar, JS Buell, RB Stein, H Youssou� an: Agenus Inc.: current or former employment/consultancy and stock ownership.

Acknowledgments The authors thank Nicole Coolbaugh of The Medicine Group, LLC (New Hope, PA, USA) for providing medical writing and editorial support, which was funded by Agenus Inc. (Lexington, MA, USA) in accordance with Good Publication Practice guidelines. The licensed antibodies AGEN2034 and AGEN1884 were originally developed under a Collaborative Research and Development Agreement between Ludwig Cancer Research, 4-Antibody AG (now Agenus Switzerland Inc.) and Recepta Biopharma S.A. Recepta Biopharma S.A. has exclusive rights to these antibodies in Brazil and � ve other South American countries.

Funding This study was funded by Agenus Inc. (Lexington, MA, USA).

CorrespondenceWaldo Ortuzar: [email protected]

Presented at the 2018 Annual Congress of the European Society for Medical Oncology (ESMO), October 19–23, 2018, in Munich, Germany

Phase 1/2 Study of CTLA-4 Inhibitor AGEN1884 + PD-1 Inhibitor AGEN2034 in Patients With Advanced/Refractory Solid Tumors, With Expansion Into Second-Line Cervical Cancer and Solid Tumors

Jermaine Coward,1 Charlotte Lemech,2 Tarek Meniawy,3 Christopher D. Dupont,4 Ana M. Gonzalez,4 Min Lim,4 David Savitsky,4 Meg Carini,4 Shuang Hu,4 Olga Shebanova,4 Ed Dow,4

Waldo Ortuzar,4 Jennifer S. Buell,4 Robert Benjamin Stein,4 Hagop Youssou� an4

1Icon Cancer Center, South Brisbane, Australia; 2Scientia Clinical Research, Sydney, Australia; 3Linear Clinical Research, Perth, Australia; 4Agenus Inc. or subsidiary thereof (current or former employee), Lexington, MA, USA

1168P

Page 2: Phase 1/2 Study of CTLA-4 Inhibitor AGEN1884 + …agenusbio.com › wp-content › uploads › 2018 › 10 › ESMO-C-550-01...1 mg/kg q2w + AGEN1884 1 mg/kg q6w died due to disease

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of

Pat

hway

s A

ffec

ted

by

CT

LA-4

and

PD

-1C

TLA

-4 In

hib

itio

nP

D-1

Inhi

bit

ion

• In

clin

ical

tria

ls, t

he c

omb

ined

inhi

biti

on o

f PD

-1 a

nd C

TLA

-4 p

athw

ays

by

blo

ckin

g re

cep

tor-

ligan

d

inte

ract

ions

has

res

ulte

d in

ob

ject

ive

clin

ical

res

pon

se a

nd in

crea

sed

sur

viva

l in

seve

ral s

olid

tum

or

indi

catio

ns, i

nclu

din

g m

elan

oma

and

non-

smal

l cel

l lun

g ca

rcin

oma.

As

a re

sult,

ipili

mum

ab (a

nti–

CTL

A-4

) in

com

bin

atio

n w

ith n

ivol

umab

(ant

i–P

D-1

) has

bee

n ap

pro

ved

as

a � r

st li

ne o

f tre

atm

ent

in p

atie

nts

with

m

etas

tatic

mel

anom

a an

d is

cur

rent

ly b

eing

tes

ted

in m

ultip

le o

ther

ind

icat

ions

.2-4

• A

genu

s’ p

rop

rieta

ry a

ntib

odie

s, A

GE

N20

34 (a

nti–

PD

-1 h

uman

IgG

4 m

onoc

lona

l ant

ibod

y) a

nd A

GE

N18

84

(ant

i–C

TLA

-4 h

uman

IgG

1 m

onoc

lona

l ant

ibod

y) a

re c

urre

ntly

und

er e

valu

atio

n as

mon

othe

rap

y in

pha

se 1

/2

stud

ies

in s

ubje

cts

with

ad

vanc

ed t

umor

s (N

CT0

3104

699

and

NC

T026

9482

2, r

esp

ectiv

ely)

.

• A

pha

se 1

/2, o

pen

-lab

el, m

ulti-

arm

tria

l to

inve

stig

ate

the

safe

ty, t

oler

abili

ty, p

harm

acok

inet

ics,

and

bio

logi

cal

and

clin

ical

act

ivity

of A

GE

N18

84 in

com

bin

atio

n w

ith A

GE

N20

34 in

pat

ient

s w

ith m

etas

tatic

or

loca

lly

adva

nced

sol

id t

umor

s is

cur

rent

ly o

ngoi

ng (C

550-

01; A

CTR

N12

6180

0000

3279

; NC

T034

9588

2).

OBJE

CTIV

E•

The

obje

ctiv

e of

thi

s st

udy

is t

o as

sess

saf

ety

and

tol

erab

ility

of A

GE

N18

84 in

com

bin

atio

n w

ith A

GE

N20

34 in

p

atie

nts

with

ad

vanc

ed/r

efra

ctor

y so

lid t

umor

s, w

ith e

xpan

sion

into

sel

ect

solid

tum

ors.

MET

HODS

• Th

is is

an

ongo

ing

pha

se 1

/2, o

pen

-lab

el, s

tud

y of

AG

EN

1884

in c

omb

inat

ion

with

AG

EN

2034

in s

ubje

cts

with

ad

vanc

ed s

olid

tum

ors,

incl

udin

g ce

rvic

al c

ance

r.

• Th

e st

udy

cons

ists

of 2

pha

ses:

–P

hase

1: D

ose

esca

latio

n (fo

cus

of t

his

pos

ter)

–P

hase

2: E

xpan

sion

in s

elec

t tu

mor

s (o

ngoi

ng)

• P

hase

1 c

onsi

sted

of a

sta

ndar

d 3

+3

dos

e es

cala

tion

with

the

follo

win

g es

cala

ting

dos

e le

vels

and

sch

edul

es:

–A

GE

N20

34 1

mg/

kg a

dm

inis

tere

d e

very

2 w

eeks

(q2w

) in

com

bin

atio

n w

ith A

GE

N18

84 1

mg/

kg

adm

inis

tere

d e

very

6 w

eeks

(q6w

) –A

GE

N20

34 3

mg/

kg a

dm

inis

tere

d e

very

2 w

eeks

(q2w

) in

com

bin

atio

n w

ith A

GE

N18

84 1

mg/

kg

adm

inis

tere

d e

very

6 w

eeks

(q6w

)

• A

GE

N20

34 w

as a

dm

inis

tere

d IV

ove

r 60

min

and

AG

EN

1884

IV o

ver

90 m

ins.

AG

EN

1884

was

to

be

adm

inis

tere

d o

n th

e sa

me

day

as

AG

EN

2034

≥30

min

aft

er t

he c

omp

letio

n of

AG

EN

2034

ad

min

istr

atio

n.

• D

ose

esca

latio

n w

ill c

ontin

ue u

ntil

AG

EN

2034

3 m

g/kg

+ A

GE

N18

84 1

mg/

kg (m

axim

um c

omb

inat

ion

dos

e le

vel)

is s

how

n to

be

safe

or

the

max

imum

tol

erat

ed d

ose

(de�

ned

as

the

com

bin

atio

n b

elow

whi

ch ≥

33%

of

sub

ject

s d

evel

op d

ose-

limiti

ng t

oxic

ities

[DLT

s]) i

s re

ache

d.

• A

sch

emat

ic o

f the

pha

se 1

dos

e-es

cala

tion

des

ign

is p

rese

nted

in F

igur

e 2.

Fig

ure

2. D

ose

-Esc

alat

ion

Stu

dy

Flo

w

Sta

rtin

g d

ose

Trea

t 3

pat

ient

s;A

sses

s D

LTs

afte

r 3

wee

ks

Num

ber

of

DLT

s

No

Yes

≤1 o

f 6

0 of

3≥2

of 3

≥2 o

f 6

1 of

3

1 of

3

Trea

t ad

diti

onal

3 p

atie

nts;

Ass

ess

DLT

s af

ter

3 w

eeks

Num

ber

of

DLT

s

MTD

/max

imum

CD

L to

be

con�

rmed

on

tota

l of

6 p

atie

nts

Initi

ate

phas

e 2

at

max

imum

pla

nned

CD

L or

MTD

Det

erm

ine

MTD

Esc

alat

e to

nex

t C

DL

Max

imum

CD

L p

er p

roto

col?

Det

erm

ine

MTD

CD

L, c

omb

inat

ion

dos

e le

vel;

DLT

, dos

e-lim

iting

tox

icity

; MTD

, max

imum

tol

erat

ed d

ose.

• E

ach

sub

ject

will

rec

eive

the

com

bin

atio

n tr

eatm

ent

for

a m

axim

um o

f 24

mon

ths

or u

ntil

con�

rm

ed d

isea

se

pro

gres

sion

, una

ccep

tab

le t

oxic

ity, o

r an

y cr

iterio

n fo

r w

ithd

raw

al fr

om t

he t

rial o

r th

e in

vest

igat

iona

l med

icin

al

pro

duc

ts o

ccur

s.

–P

atie

nts

who

do

not

com

ple

te t

he D

LT o

bse

rvat

ion

per

iod

of 2

1 d

ays

afte

r th

e � r

st d

ose,

for

reas

ons

othe

r th

an a

DLT

, will

be

rep

lace

d.

–C

ohor

ts w

ill b

e b

ack�

lled

to

10 s

ubje

cts

once

the

y ha

ve r

each

ed D

LT o

r M

TD.

• El

igib

le p

atie

nts

incl

ude

adul

ts (a

ged

≥18

year

s) w

ith a

his

tolo

gica

lly o

r cyt

olog

ical

ly c

on� r

med

dia

gnos

is o

f a

met

asta

tic o

r loc

ally

adv

ance

d so

lid tu

mor

for w

hich

no

stan

dard

ther

apy

is a

vaila

ble

or s

tand

ard

ther

apy

has

faile

d.

• Th

e tr

eatm

ent

pha

se w

as d

ivid

ed in

to 6

-wee

k cy

cles

, eac

h b

egin

ning

with

the

com

bin

ed a

dm

inis

trat

ion

of A

GE

N20

34 a

nd A

GE

N18

84 o

n d

ay 1

. The

reaf

ter,

AG

EN

2034

will

be

adm

inis

trat

ed q

2w, c

omp

letin

g th

e 6-

wee

k cy

cle.

–Tu

mor

ass

essm

ents

are

to

be

cond

ucte

d a

t 6,

12,

and

18

wee

ks fr

om �

rst

dos

e, a

nd e

very

9 w

eeks

th

erea

fter

unt

il d

isea

se p

rogr

essi

on is

con

� rm

ed o

r a

new

line

of t

hera

py

is in

itiat

ed.

• P

hase

1 e

ndp

oint

s: –P

rimar

y: O

ccur

renc

e of

DLT

s in

pat

ient

s in

dos

e es

cala

tion

dur

ing

the

� rst

21

day

s of

tre

atm

ent

–S

econ

dar

y:

C

on� r

med

bes

t ov

eral

l res

pon

se p

er R

esp

onse

Eva

luat

ion

Crit

eria

in S

olid

Tum

ors

(RE

CIS

T) v

1.1,

as

det

erm

ined

by

the

inve

stig

ator

Fr

eque

ncy

and

nat

ure

of t

reat

men

t-em

erge

nt a

dve

rse

even

ts (T

EA

Es)

P

harm

acok

inet

ic p

ro� l

e an

d im

mun

ogen

icity

of A

GE

N18

84 a

nd A

GE

N20

34

RESU

LTS

• A

s of

Jul

y 16

, 201

8, t

he s

tud

y w

as o

ngoi

ng, t

houg

h to

tal e

nrol

lmen

t w

as a

chie

ved

in t

he p

hase

1 p

ortio

n (ie

, 10

pat

ient

s in

eac

h d

ose

coho

rt).

Dat

a ar

e p

relim

inar

y as

it is

bei

ng c

lean

ed a

nd/o

r co

llect

ed a

t th

is t

ime.

• Fi

ve p

atie

nts

had

dis

cont

inue

d fr

om t

he s

tud

y tr

eatm

ent

prio

r to

dat

a ex

trac

t:

–A

GE

N20

34 1

mg/

kg q

2w +

AG

EN

1884

1 m

g/kg

q6w

: Pro

gres

sive

dis

ease

, n=

1; in

vest

igat

or d

ecis

ion,

n=

1 –A

GE

N20

34 3

mg/

kg q

2w +

AG

EN

1884

1 m

g/kg

q6w

: Pro

gres

sive

dis

ease

, n=

3

• Th

ere

was

1 d

eath

not

rel

ated

to

the

stud

y (6

6-ye

ar-o

ld fe

mal

e w

ith lu

ng c

ance

r tr

eate

d w

ith A

GE

N20

34

1 m

g/kg

q2w

+ A

GE

N18

84 1

mg/

kg q

6w d

ied

due

to

dis

ease

pro

gres

sion

).

• P

atie

nts

wer

e C

auca

sian

(100

%) a

nd p

rimar

ily fe

mal

e (7

5%),

with

a m

edia

n of

2.3

mon

ths

from

the

mos

t re

cent

rec

urre

nce/

pro

gres

sion

to

the

adm

inis

trat

ion

of t

he �

rst

com

bin

atio

n d

ose

(Tab

le 1

).

• Th

e ca

ncer

typ

es fo

r th

e 20

pat

ient

s w

ere

as fo

llow

s: b

reas

t ca

ncer

(trip

le n

egat

ive,

n=

2; w

ith lu

ng

met

asta

ses,

n=

1; w

ith d

erm

al ly

mp

hatic

s, n

=1)

, ova

rian

canc

er (n

=2)

, mes

othe

liom

a (p

leur

al, n

=1;

per

itone

al,

n=1)

, sq

uam

ous

carc

inom

a of

hea

d a

nd n

eck

(tong

ue; n

=1)

, ana

l sq

uam

ous

carc

inom

a (n

=1)

, ple

omor

phi

c so

ft t

issu

e sa

rcom

a (n

=1)

, lun

g ca

ncer

(n=

1), m

etas

tast

ic le

iom

yosa

rcom

a (n

=1)

, ad

vanc

ed g

astr

oint

estin

al

stro

mal

tum

or (n

=1)

, rha

bd

omyo

sarc

oma

(n=

1), a

lveo

lar

rhab

dom

yosa

rcom

a (n

=1)

, rec

urre

nt c

hord

oma

of t

he

thor

acic

sp

ine

(n=

1), c

hord

oma

(n=

1), c

olor

ecta

l-re

ctal

(n=

1), e

sop

hage

al a

den

ocar

cino

ma

(n=

1).

Tab

le 1

. Dem

og

rap

hics

and

Bas

elin

e C

hara

cter

isti

cs (S

afet

y S

et)

AG

EN

2034

1 m

g/k

g +

A

GE

N18

84 1

mg

/kg

(N=

10)

AG

EN

2034

3 m

g/k

g +

A

GE

N18

84 1

mg

/kg

(N=

10)

Tota

l Pat

ien

ts(N

=20

)

Ag

e (y

ears

)

Mea

n (S

D)

58.4

(8.1

3)47

.6 (1

9.68

)53

.0 (1

5.66

)

Min

, Max

43

, 69

21, 7

921

, 79

Fem

ale,

n (%

)8

(80%

)7

(70%

)15

(75%

)

BM

I (kg

/m2 )

Mea

n (S

D)

28.7

1 (8

.227

)27

.48

(7.0

06)

28.1

0 (7

.464

)

Min

, Max

18

.8, 4

0.9

20.9

, 45.

518

.8, 4

5.5

EC

OG

per

form

ance

sta

tus,

n (%

)

05

(50%

)7

(70%

)12

(60%

)

15

(50%

)3

(30%

)8

(40%

)

Tim

e b

etw

een

mo

st r

ecen

t p

rog

ress

ion

and

� rs

t d

ose

dat

e (m

onth

s)

n6

612

Mea

n (S

D)

2.26

(1.6

65)

2.34

(1.0

02)

2.30

(1.3

11)

BM

I, b

ody

mas

s in

dex

; EC

OG

, Eas

tern

Coo

per

ativ

e O

ncol

ogy

Gro

up.

• A

n ov

ervi

ew o

f saf

ety

and

tol

erab

ility

is p

rese

nted

in T

able

2.

Tab

le 2

. Ove

rvie

w o

f Tr

eatm

ent-

Em

erg

ent

Ad

vers

e E

vent

(Saf

ety

Set

)

Pat

ien

ts, n

(%)

AG

EN

2034

1 m

g/k

g +

A

GE

N18

84 1

mg

/kg

(N=

10)

AG

EN

2034

3 m

g/k

g +

A

GE

N18

84 1

mg

/kg

(N=

10)

Tota

l Pat

ien

ts(N

=20

)

Any

TE

AE

10 (1

00%

)9

(90%

)19

(95%

)

Any

ser

ious

TE

AE

3 (3

0%)

2 (2

0%)

5 (2

5%)

Any

gra

de

≥3 T

EA

E3

(30%

)2

(20%

)5

(25%

)

Any

imm

une-

rela

ted

TE

AE

5 (5

0%)

4 (4

0%)

9 (4

5%)

Any

tre

atm

ent-

rela

ted

TE

AE

8 (8

0%)

7 (7

0%)

15 (7

5%)

Any

tre

atm

ent-

rela

ted

ser

ious

TE

AE

1 (1

0%)

2 (2

0%)

3 (1

5%)

Any

tre

atm

ent-

rela

ted

gra

de

≥3 T

EA

E0

2 (2

0%)

2 (1

0%)

TEA

E, t

reat

men

t-em

erge

nt a

dve

rse

even

t.

• A

s of

Jul

y 16

, 201

8, n

o D

LTs

have

bee

n ob

serv

ed, a

nd n

one

of t

he T

EA

Es

led

to

dis

cont

inua

tion

or d

eath

.

• TE

AE

s w

ere

rep

orte

d in

19

of 2

0 p

atie

nts

(95%

), w

ith t

he m

ost

com

mon

Med

DR

A v

ersi

on 1

8.1

pre

ferr

ed

term

s lis

ted

in (T

able

3).

• M

ost

TEA

Es

wer

e m

ild t

o m

oder

ate

in s

ever

ity (g

rad

e 1

or 2

); 5

pat

ient

s ex

per

ienc

ed T

EA

Es

of g

rad

e ≥3

. –A

GE

N20

34 1

mg/

kg q

2w +

AG

EN

1884

1 m

g/kg

q6w

: Vom

iting

, n=

1; w

ound

and

can

cer

pai

n, n

=1;

hy

pot

ensi

on, n

=1

–A

GE

N20

34 3

mg/

kg q

2w +

AG

EN

1884

1 m

g/kg

q6w

: Low

er r

esp

irato

ry t

ract

infe

ctio

n an

d p

neum

onia

, n=

1;

pul

mon

ary

emb

olis

m, n

=1

–O

f the

se, t

he e

vent

s of

low

er r

esp

irato

ry t

ract

infe

ctio

n an

d p

ulm

onar

y em

bol

ism

wer

e co

nsid

ered

rel

ated

to

stud

y tr

eatm

ent

by

the

inve

stig

ator

.

Tab

le 3

. Mo

st C

om

mo

n* T

reat

men

t-E

mer

gen

t A

dve

rse

Eve

nt (S

afet

y S

et)

Pat

ien

ts, n

(%)

AG

EN

2034

1 m

g/k

g +

A

GE

N18

84 1

mg

/kg

(N=

10)

AG

EN

2034

3 m

g/k

g +

A

GE

N18

84 1

mg

/kg

(N=

10)

Tota

l Pat

ien

ts(N

=20

)

Fatig

ue5

(50%

)5

(50%

)10

(50%

)

Nau

sea

3 (3

0%)

4 (4

0%)

7 (3

5%)

Pru

ritus

2 (2

0%)

3 (3

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Page 3: Phase 1/2 Study of CTLA-4 Inhibitor AGEN1884 + …agenusbio.com › wp-content › uploads › 2018 › 10 › ESMO-C-550-01...1 mg/kg q2w + AGEN1884 1 mg/kg q6w died due to disease

BACKGROUND• Antigen-speci� c T-cell activation is regulated by a balance of co-stimulatory and co-inhibitory signals, such as

those mediated by inhibitory receptors cytotoxic T-lymphocyte–associated antigen 4 (CTLA-4) and programmed cell death protein-1 (PD-1) (Figure 1).1

• Binding of these receptors to their ligands results in impaired T-cell function. For these reasons, antibody blockade of PD-1 and CTLA-4 has been identi� ed as a therapeutic modality to reinvigorate or induce tumor-speci� c T-cell immunity.1

Figure 1. Overview of Pathways Affected by CTLA-4 and PD-1CTLA-4 Inhibition PD-1 Inhibition

• In clinical trials, the combined inhibition of PD-1 and CTLA-4 pathways by blocking receptor-ligand interactions has resulted in objective clinical response and increased survival in several solid tumor indications, including melanoma and non-small cell lung carcinoma. As a result, ipilimumab (anti–CTLA-4) in combination with nivolumab (anti–PD-1) has been approved as a � rst line of treatment in patients with metastatic melanoma and is currently being tested in multiple other indications.2-4

• Agenus’ proprietary antibodies, AGEN2034 (anti–PD-1 human IgG4 monoclonal antibody) and AGEN1884 (anti–CTLA-4 human IgG1 monoclonal antibody) are currently under evaluation as monotherapy in phase 1/2 studies in subjects with advanced tumors (NCT03104699 and NCT02694822, respectively).

• A phase 1/2, open-label, multi-arm trial to investigate the safety, tolerability, pharmacokinetics, and biological and clinical activity of AGEN1884 in combination with AGEN2034 in patients with metastatic or locally advanced solid tumors is currently ongoing (C550-01; ACTRN12618000003279; NCT03495882).

OBJECTIVE• The objective of this study is to assess safety and tolerability of AGEN1884 in combination with AGEN2034 in

patients with advanced/refractory solid tumors, with expansion into select solid tumors.

METHODS• This is an ongoing phase 1/2, open-label, study of AGEN1884 in combination with AGEN2034 in subjects with

advanced solid tumors, including cervical cancer.

• The study consists of 2 phases: – Phase 1: Dose escalation (focus of this poster) – Phase 2: Expansion in select tumors (ongoing)

• Phase 1 consisted of a standard 3+3 dose escalation with the following escalating dose levels and schedules: – AGEN2034 1 mg/kg administered every 2 weeks (q2w) in combination with AGEN1884 1 mg/kg administered every 6 weeks (q6w)

– AGEN2034 3 mg/kg administered every 2 weeks (q2w) in combination with AGEN1884 1 mg/kg administered every 6 weeks (q6w)

• AGEN2034 was administered IV over 60 min and AGEN1884 IV over 90 mins. AGEN1884 was to be administered on the same day as AGEN2034 ≥30 min after the completion of AGEN2034 administration.

• Dose escalation will continue until AGEN2034 3 mg/kg + AGEN1884 1 mg/kg (maximum combination dose level) is shown to be safe or the maximum tolerated dose (de� ned as the combination below which ≥33% of subjects develop dose-limiting toxicities [DLTs]) is reached.

• A schematic of the phase 1 dose-escalation design is presented in Figure 2.

Figure 2. Dose-Escalation Study Flow

Starting dose

Treat 3 patients;Assess DLTs after 3 weeks

Number ofDLTs

No

Yes

≤1 of 6

0 of 3 ≥2 of 3

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1 of 3

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of 6 patients

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or MTD

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Maximum CDL per protocol?

Determine MTD

CDL, combination dose level; DLT, dose-limiting toxicity; MTD, maximum tolerated dose.

• Each subject will receive the combination treatment for a maximum of 24 months or until con� rmed disease progression, unacceptable toxicity, or any criterion for withdrawal from the trial or the investigational medicinal products occurs.

– Patients who do not complete the DLT observation period of 21 days after the � rst dose, for reasons other than a DLT, will be replaced.

– Cohorts will be back� lled to 10 subjects once they have reached DLT or MTD.

• Eligible patients include adults (aged ≥18 years) with a histologically or cytologically con� rmed diagnosis of a metastatic or locally advanced solid tumor for which no standard therapy is available or standard therapy has failed.

• The treatment phase was divided into 6-week cycles, each beginning with the combined administration of AGEN2034 and AGEN1884 on day 1. Thereafter, AGEN2034 will be administrated q2w, completing the 6-week cycle.

– Tumor assessments are to be conducted at 6, 12, and 18 weeks from � rst dose, and every 9 weeks thereafter until disease progression is con� rmed or a new line of therapy is initiated.

• Phase 1 endpoints: – Primary: Occurrence of DLTs in patients in dose escalation during the � rst 21 days of treatment – Secondary:

Con� rmed best overall response per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1, as determined by the investigator

Frequency and nature of treatment-emergent adverse events (TEAEs) Pharmacokinetic pro� le and immunogenicity of AGEN1884 and AGEN2034

RESULTS • As of July 16, 2018, the study was ongoing, though total enrollment was achieved in the phase 1 portion

(ie, 10 patients in each dose cohort). Data are preliminary as it is being cleaned and/or collected at this time.

• Five patients had discontinued from the study treatment prior to data extract: – AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w: Progressive disease, n=1; investigator decision, n=1 – AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w: Progressive disease, n=3

• There was 1 death not related to the study (66-year-old female with lung cancer treated with AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w died due to disease progression).

• Patients were Caucasian (100%) and primarily female (75%), with a median of 2.3 months from the most recent recurrence/progression to the administration of the � rst combination dose (Table 1).

• The cancer types for the 20 patients were as follows: breast cancer (triple negative, n=2; with lung metastases, n=1; with dermal lymphatics, n=1), ovarian cancer (n=2), mesothelioma (pleural, n=1; peritoneal, n=1), squamous carcinoma of head and neck (tongue; n=1), anal squamous carcinoma (n=1), pleomorphic soft tissue sarcoma (n=1), lung cancer (n=1), metastastic leiomyosarcoma (n=1), advanced gastrointestinal stromal tumor (n=1), rhabdomyosarcoma (n=1), alveolar rhabdomyosarcoma (n=1), recurrent chordoma of the thoracic spine (n=1), chordoma (n=1), colorectal-rectal (n=1), esophageal adenocarcinoma (n=1).

Table 1. Demographics and Baseline Characteristics (Safety Set) AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Age (years)

Mean (SD) 58.4 (8.13) 47.6 (19.68) 53.0 (15.66)

Min, Max 43, 69 21, 79 21, 79

Female, n (%) 8 (80%) 7 (70%) 15 (75%)

BMI (kg/m2)

Mean (SD) 28.71 (8.227) 27.48 (7.006) 28.10 (7.464)

Min, Max 18.8, 40.9 20.9, 45.5 18.8, 45.5

ECOG performance status, n (%)

0 5 (50%) 7 (70%) 12 (60%)

1 5 (50%) 3 (30%) 8 (40%)

Time between most recent progression and � rst dose date (months)

n 6 6 12

Mean (SD) 2.26 (1.665) 2.34 (1.002) 2.30 (1.311)

BMI, body mass index; ECOG, Eastern Cooperative Oncology Group.

• An overview of safety and tolerability is presented in Table 2.

Table 2. Overview of Treatment-Emergent Adverse Event (Safety Set)

Patients, n (%)

AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Any TEAE 10 (100%) 9 (90%) 19 (95%)

Any serious TEAE 3 (30%) 2 (20%) 5 (25%)

Any grade ≥3 TEAE 3 (30%) 2 (20%) 5 (25%)

Any immune-related TEAE 5 (50%) 4 (40%) 9 (45%)

Any treatment-related TEAE 8 (80%) 7 (70%) 15 (75%)

Any treatment-related serious TEAE 1 (10%) 2 (20%) 3 (15%)

Any treatment-related grade ≥3 TEAE 0 2 (20%) 2 (10%)

TEAE, treatment-emergent adverse event.

• As of July 16, 2018, no DLTs have been observed, and none of the TEAEs led to discontinuation or death.

• TEAEs were reported in 19 of 20 patients (95%), with the most common MedDRA version 18.1 preferred terms listed in (Table 3).

• Most TEAEs were mild to moderate in severity (grade 1 or 2); 5 patients experienced TEAEs of grade ≥3. – AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w: Vomiting, n=1; wound and cancer pain, n=1; hypotension, n=1

– AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w: Lower respiratory tract infection and pneumonia, n=1; pulmonary embolism, n=1

– Of these, the events of lower respiratory tract infection and pulmonary embolism were considered related to study treatment by the investigator.

Table 3. Most Common* Treatment-Emergent Adverse Event (Safety Set)

Patients, n (%)

AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Fatigue 5 (50%) 5 (50%) 10 (50%)

Nausea 3 (30%) 4 (40%) 7 (35%)

Pruritus 2 (20%) 3 (30%) 5 (25%)

Chills 3 (30%) 1 (10%) 4 (20%)

Vomiting 2 (20%) 2 (20%) 4 (20%)

Back pain 4 (40%) 0 4 (20%)*Occurring in ≥20% of patients.

• Serious TEAEs occurred in 5 patients: 3 receiving AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w and 2 receiving AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w

• 3 patients experienced serious TEAEs considered related to study treatment. – AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w: Diarrhea, n=1 – AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w: Lower respiratory tract infection, n=1; pulmonary embolism, n=1

• The summary of best overall response is listed in Table 4.

• The partial response was ongoing in one of the women with metastatic ovarian cancer. For patients with >90 days of treatment, stable disease was ongoing for 1 female with breast cancer (with lung metastases), 1 female with metastatic ovarian cancer, and 1 male with pleomorphic soft tissue sarcoma (with lung metastases).

Table 4. Summary of Best Overall Response at Time of Data Cut-off

Patients, n (%)

AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Complete response 0 0 0

Partial response 1 0 1

Stable disease 5 1 6

Progressive disease 3 6 9

Not evaluable 1 0 1

Pending 0 3 3Overall response determined by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1.

DISCUSSION• In the phase 1 portion, which is still ongoing, preliminary results demonstrate that AGEN1884 (1 mg/kg q6w) +

AGEN2034 (3 mg/kg q2w) is generally safe, well tolerated, and active in adults with select advanced/refractory solid tumors.

• The phase 2 portion of this study evaluating AGEN1884 in combination with AGEN2034 in adults with second-line cervical cancer and other solid tumors is ongoing.

– The phase 2 recommended dosing was determined as AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w.

References1. Topalian SL, Drake CG, Pardoll DM. Cancer Cell. 2015;27(4):450-61.2. Hodi FS, Chesney J, Pavlick AC, et al. Lancet Oncol.

2016;17(11):1558-68.

3. Antonia SJ, López-Martin JA, Bendell J, et al. Lancet Oncol. 2016;17(7):883-95.

4. Hellmann MD, Gettinger SN, Goldman JW, et al. J Clin Oncol. 2016;34(15 suppl):3001.5

Disclosures CD Dupont, AM Gonzalez, M Lim, D Savitsky, M Carini, S Hu, O Shebanova, E Dow, W Ortuzar, JS Buell, RB Stein, H Youssou� an: Agenus Inc.: current or former employment/consultancy and stock ownership.

Acknowledgments The authors thank Nicole Coolbaugh of The Medicine Group, LLC (New Hope, PA, USA) for providing medical writing and editorial support, which was funded by Agenus Inc. (Lexington, MA, USA) in accordance with Good Publication Practice guidelines. The licensed antibodies AGEN2034 and AGEN1884 were originally developed under a Collaborative Research and Development Agreement between Ludwig Cancer Research, 4-Antibody AG (now Agenus Switzerland Inc.) and Recepta Biopharma S.A. Recepta Biopharma S.A. has exclusive rights to these antibodies in Brazil and � ve other South American countries.

Funding This study was funded by Agenus Inc. (Lexington, MA, USA).

CorrespondenceWaldo Ortuzar: [email protected]

Presented at the 2018 Annual Congress of the European Society for Medical Oncology (ESMO), October 19–23, 2018, in Munich, Germany

Phase 1/2 Study of CTLA-4 Inhibitor AGEN1884 + PD-1 Inhibitor AGEN2034 in Patients With Advanced/Refractory Solid Tumors, With Expansion Into Second-Line Cervical Cancer and Solid Tumors

Jermaine Coward,1 Charlotte Lemech,2 Tarek Meniawy,3 Christopher D. Dupont,4 Ana M. Gonzalez,4 Min Lim,4 David Savitsky,4 Meg Carini,4 Shuang Hu,4 Olga Shebanova,4 Ed Dow,4

Waldo Ortuzar,4 Jennifer S. Buell,4 Robert Benjamin Stein,4 Hagop Youssou� an4

1Icon Cancer Center, South Brisbane, Australia; 2Scientia Clinical Research, Sydney, Australia; 3Linear Clinical Research, Perth, Australia; 4Agenus Inc. or subsidiary thereof (current or former employee), Lexington, MA, USA

1168P

Page 4: Phase 1/2 Study of CTLA-4 Inhibitor AGEN1884 + …agenusbio.com › wp-content › uploads › 2018 › 10 › ESMO-C-550-01...1 mg/kg q2w + AGEN1884 1 mg/kg q6w died due to disease

BACKGROUND• Antigen-speci� c T-cell activation is regulated by a balance of co-stimulatory and co-inhibitory signals, such as

those mediated by inhibitory receptors cytotoxic T-lymphocyte–associated antigen 4 (CTLA-4) and programmed cell death protein-1 (PD-1) (Figure 1).1

• Binding of these receptors to their ligands results in impaired T-cell function. For these reasons, antibody blockade of PD-1 and CTLA-4 has been identi� ed as a therapeutic modality to reinvigorate or induce tumor-speci� c T-cell immunity.1

Figure 1. Overview of Pathways Affected by CTLA-4 and PD-1CTLA-4 Inhibition PD-1 Inhibition

• In clinical trials, the combined inhibition of PD-1 and CTLA-4 pathways by blocking receptor-ligand interactions has resulted in objective clinical response and increased survival in several solid tumor indications, including melanoma and non-small cell lung carcinoma. As a result, ipilimumab (anti–CTLA-4) in combination with nivolumab (anti–PD-1) has been approved as a � rst line of treatment in patients with metastatic melanoma and is currently being tested in multiple other indications.2-4

• Agenus’ proprietary antibodies, AGEN2034 (anti–PD-1 human IgG4 monoclonal antibody) and AGEN1884 (anti–CTLA-4 human IgG1 monoclonal antibody) are currently under evaluation as monotherapy in phase 1/2 studies in subjects with advanced tumors (NCT03104699 and NCT02694822, respectively).

• A phase 1/2, open-label, multi-arm trial to investigate the safety, tolerability, pharmacokinetics, and biological and clinical activity of AGEN1884 in combination with AGEN2034 in patients with metastatic or locally advanced solid tumors is currently ongoing (C550-01; ACTRN12618000003279; NCT03495882).

OBJECTIVE• The objective of this study is to assess safety and tolerability of AGEN1884 in combination with AGEN2034 in

patients with advanced/refractory solid tumors, with expansion into select solid tumors.

METHODS• This is an ongoing phase 1/2, open-label, study of AGEN1884 in combination with AGEN2034 in subjects with

advanced solid tumors, including cervical cancer.

• The study consists of 2 phases: – Phase 1: Dose escalation (focus of this poster) – Phase 2: Expansion in select tumors (ongoing)

• Phase 1 consisted of a standard 3+3 dose escalation with the following escalating dose levels and schedules: – AGEN2034 1 mg/kg administered every 2 weeks (q2w) in combination with AGEN1884 1 mg/kg administered every 6 weeks (q6w)

– AGEN2034 3 mg/kg administered every 2 weeks (q2w) in combination with AGEN1884 1 mg/kg administered every 6 weeks (q6w)

• AGEN2034 was administered IV over 60 min and AGEN1884 IV over 90 mins. AGEN1884 was to be administered on the same day as AGEN2034 ≥30 min after the completion of AGEN2034 administration.

• Dose escalation will continue until AGEN2034 3 mg/kg + AGEN1884 1 mg/kg (maximum combination dose level) is shown to be safe or the maximum tolerated dose (de� ned as the combination below which ≥33% of subjects develop dose-limiting toxicities [DLTs]) is reached.

• A schematic of the phase 1 dose-escalation design is presented in Figure 2.

Figure 2. Dose-Escalation Study Flow

Starting dose

Treat 3 patients;Assess DLTs after 3 weeks

Number ofDLTs

No

Yes

≤1 of 6

0 of 3 ≥2 of 3

≥2 of 6

1 of 3

1 of 3

Treat additional 3 patients;Assess DLTs after 3 weeks

Number of DLTs

MTD/maximum CDL to be con�rmed on total

of 6 patients

Initiate phase 2 at maximum planned CDL

or MTD

Determine MTDEscalate to next CDL

Maximum CDL per protocol?

Determine MTD

CDL, combination dose level; DLT, dose-limiting toxicity; MTD, maximum tolerated dose.

• Each subject will receive the combination treatment for a maximum of 24 months or until con� rmed disease progression, unacceptable toxicity, or any criterion for withdrawal from the trial or the investigational medicinal products occurs.

– Patients who do not complete the DLT observation period of 21 days after the � rst dose, for reasons other than a DLT, will be replaced.

– Cohorts will be back� lled to 10 subjects once they have reached DLT or MTD.

• Eligible patients include adults (aged ≥18 years) with a histologically or cytologically con� rmed diagnosis of a metastatic or locally advanced solid tumor for which no standard therapy is available or standard therapy has failed.

• The treatment phase was divided into 6-week cycles, each beginning with the combined administration of AGEN2034 and AGEN1884 on day 1. Thereafter, AGEN2034 will be administrated q2w, completing the 6-week cycle.

– Tumor assessments are to be conducted at 6, 12, and 18 weeks from � rst dose, and every 9 weeks thereafter until disease progression is con� rmed or a new line of therapy is initiated.

• Phase 1 endpoints: – Primary: Occurrence of DLTs in patients in dose escalation during the � rst 21 days of treatment – Secondary:

Con� rmed best overall response per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1, as determined by the investigator

Frequency and nature of treatment-emergent adverse events (TEAEs) Pharmacokinetic pro� le and immunogenicity of AGEN1884 and AGEN2034

RESULTS • As of July 16, 2018, the study was ongoing, though total enrollment was achieved in the phase 1 portion

(ie, 10 patients in each dose cohort). Data are preliminary as it is being cleaned and/or collected at this time.

• Five patients had discontinued from the study treatment prior to data extract: – AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w: Progressive disease, n=1; investigator decision, n=1 – AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w: Progressive disease, n=3

• There was 1 death not related to the study (66-year-old female with lung cancer treated with AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w died due to disease progression).

• Patients were Caucasian (100%) and primarily female (75%), with a median of 2.3 months from the most recent recurrence/progression to the administration of the � rst combination dose (Table 1).

• The cancer types for the 20 patients were as follows: breast cancer (triple negative, n=2; with lung metastases, n=1; with dermal lymphatics, n=1), ovarian cancer (n=2), mesothelioma (pleural, n=1; peritoneal, n=1), squamous carcinoma of head and neck (tongue; n=1), anal squamous carcinoma (n=1), pleomorphic soft tissue sarcoma (n=1), lung cancer (n=1), metastastic leiomyosarcoma (n=1), advanced gastrointestinal stromal tumor (n=1), rhabdomyosarcoma (n=1), alveolar rhabdomyosarcoma (n=1), recurrent chordoma of the thoracic spine (n=1), chordoma (n=1), colorectal-rectal (n=1), esophageal adenocarcinoma (n=1).

Table 1. Demographics and Baseline Characteristics (Safety Set) AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Age (years)

Mean (SD) 58.4 (8.13) 47.6 (19.68) 53.0 (15.66)

Min, Max 43, 69 21, 79 21, 79

Female, n (%) 8 (80%) 7 (70%) 15 (75%)

BMI (kg/m2)

Mean (SD) 28.71 (8.227) 27.48 (7.006) 28.10 (7.464)

Min, Max 18.8, 40.9 20.9, 45.5 18.8, 45.5

ECOG performance status, n (%)

0 5 (50%) 7 (70%) 12 (60%)

1 5 (50%) 3 (30%) 8 (40%)

Time between most recent progression and � rst dose date (months)

n 6 6 12

Mean (SD) 2.26 (1.665) 2.34 (1.002) 2.30 (1.311)

BMI, body mass index; ECOG, Eastern Cooperative Oncology Group.

• An overview of safety and tolerability is presented in Table 2.

Table 2. Overview of Treatment-Emergent Adverse Event (Safety Set)

Patients, n (%)

AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Any TEAE 10 (100%) 9 (90%) 19 (95%)

Any serious TEAE 3 (30%) 2 (20%) 5 (25%)

Any grade ≥3 TEAE 3 (30%) 2 (20%) 5 (25%)

Any immune-related TEAE 5 (50%) 4 (40%) 9 (45%)

Any treatment-related TEAE 8 (80%) 7 (70%) 15 (75%)

Any treatment-related serious TEAE 1 (10%) 2 (20%) 3 (15%)

Any treatment-related grade ≥3 TEAE 0 2 (20%) 2 (10%)

TEAE, treatment-emergent adverse event.

• As of July 16, 2018, no DLTs have been observed, and none of the TEAEs led to discontinuation or death.

• TEAEs were reported in 19 of 20 patients (95%), with the most common MedDRA version 18.1 preferred terms listed in (Table 3).

• Most TEAEs were mild to moderate in severity (grade 1 or 2); 5 patients experienced TEAEs of grade ≥3. – AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w: Vomiting, n=1; wound and cancer pain, n=1; hypotension, n=1

– AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w: Lower respiratory tract infection and pneumonia, n=1; pulmonary embolism, n=1

– Of these, the events of lower respiratory tract infection and pulmonary embolism were considered related to study treatment by the investigator.

Table 3. Most Common* Treatment-Emergent Adverse Event (Safety Set)

Patients, n (%)

AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Fatigue 5 (50%) 5 (50%) 10 (50%)

Nausea 3 (30%) 4 (40%) 7 (35%)

Pruritus 2 (20%) 3 (30%) 5 (25%)

Chills 3 (30%) 1 (10%) 4 (20%)

Vomiting 2 (20%) 2 (20%) 4 (20%)

Back pain 4 (40%) 0 4 (20%)*Occurring in ≥20% of patients.

• Serious TEAEs occurred in 5 patients: 3 receiving AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w and 2 receiving AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w

• 3 patients experienced serious TEAEs considered related to study treatment. – AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w: Diarrhea, n=1 – AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w: Lower respiratory tract infection, n=1; pulmonary embolism, n=1

• The summary of best overall response is listed in Table 4.

• The partial response was ongoing in one of the women with metastatic ovarian cancer. For patients with >90 days of treatment, stable disease was ongoing for 1 female with breast cancer (with lung metastases), 1 female with metastatic ovarian cancer, and 1 male with pleomorphic soft tissue sarcoma (with lung metastases).

Table 4. Summary of Best Overall Response at Time of Data Cut-off

Patients, n (%)

AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Complete response 0 0 0

Partial response 1 0 1

Stable disease 5 1 6

Progressive disease 3 6 9

Not evaluable 1 0 1

Pending 0 3 3Overall response determined by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1.

DISCUSSION• In the phase 1 portion, which is still ongoing, preliminary results demonstrate that AGEN1884 (1 mg/kg q6w) +

AGEN2034 (3 mg/kg q2w) is generally safe, well tolerated, and active in adults with select advanced/refractory solid tumors.

• The phase 2 portion of this study evaluating AGEN1884 in combination with AGEN2034 in adults with second-line cervical cancer and other solid tumors is ongoing.

– The phase 2 recommended dosing was determined as AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w.

References1. Topalian SL, Drake CG, Pardoll DM. Cancer Cell. 2015;27(4):450-61.2. Hodi FS, Chesney J, Pavlick AC, et al. Lancet Oncol.

2016;17(11):1558-68.

3. Antonia SJ, López-Martin JA, Bendell J, et al. Lancet Oncol. 2016;17(7):883-95.

4. Hellmann MD, Gettinger SN, Goldman JW, et al. J Clin Oncol. 2016;34(15 suppl):3001.5

Disclosures CD Dupont, AM Gonzalez, M Lim, D Savitsky, M Carini, S Hu, O Shebanova, E Dow, W Ortuzar, JS Buell, RB Stein, H Youssou� an: Agenus Inc.: current or former employment/consultancy and stock ownership.

Acknowledgments The authors thank Nicole Coolbaugh of The Medicine Group, LLC (New Hope, PA, USA) for providing medical writing and editorial support, which was funded by Agenus Inc. (Lexington, MA, USA) in accordance with Good Publication Practice guidelines. The licensed antibodies AGEN2034 and AGEN1884 were originally developed under a Collaborative Research and Development Agreement between Ludwig Cancer Research, 4-Antibody AG (now Agenus Switzerland Inc.) and Recepta Biopharma S.A. Recepta Biopharma S.A. has exclusive rights to these antibodies in Brazil and � ve other South American countries.

Funding This study was funded by Agenus Inc. (Lexington, MA, USA).

CorrespondenceWaldo Ortuzar: [email protected]

Presented at the 2018 Annual Congress of the European Society for Medical Oncology (ESMO), October 19–23, 2018, in Munich, Germany

Phase 1/2 Study of CTLA-4 Inhibitor AGEN1884 + PD-1 Inhibitor AGEN2034 in Patients With Advanced/Refractory Solid Tumors, With Expansion Into Second-Line Cervical Cancer and Solid Tumors

Jermaine Coward,1 Charlotte Lemech,2 Tarek Meniawy,3 Christopher D. Dupont,4 Ana M. Gonzalez,4 Min Lim,4 David Savitsky,4 Meg Carini,4 Shuang Hu,4 Olga Shebanova,4 Ed Dow,4

Waldo Ortuzar,4 Jennifer S. Buell,4 Robert Benjamin Stein,4 Hagop Youssou� an4

1Icon Cancer Center, South Brisbane, Australia; 2Scientia Clinical Research, Sydney, Australia; 3Linear Clinical Research, Perth, Australia; 4Agenus Inc. or subsidiary thereof (current or former employee), Lexington, MA, USA

1168P

Page 5: Phase 1/2 Study of CTLA-4 Inhibitor AGEN1884 + …agenusbio.com › wp-content › uploads › 2018 › 10 › ESMO-C-550-01...1 mg/kg q2w + AGEN1884 1 mg/kg q6w died due to disease

BACKGROUND• Antigen-speci� c T-cell activation is regulated by a balance of co-stimulatory and co-inhibitory signals, such as

those mediated by inhibitory receptors cytotoxic T-lymphocyte–associated antigen 4 (CTLA-4) and programmed cell death protein-1 (PD-1) (Figure 1).1

• Binding of these receptors to their ligands results in impaired T-cell function. For these reasons, antibody blockade of PD-1 and CTLA-4 has been identi� ed as a therapeutic modality to reinvigorate or induce tumor-speci� c T-cell immunity.1

Figure 1. Overview of Pathways Affected by CTLA-4 and PD-1CTLA-4 Inhibition PD-1 Inhibition

• In clinical trials, the combined inhibition of PD-1 and CTLA-4 pathways by blocking receptor-ligand interactions has resulted in objective clinical response and increased survival in several solid tumor indications, including melanoma and non-small cell lung carcinoma. As a result, ipilimumab (anti–CTLA-4) in combination with nivolumab (anti–PD-1) has been approved as a � rst line of treatment in patients with metastatic melanoma and is currently being tested in multiple other indications.2-4

• Agenus’ proprietary antibodies, AGEN2034 (anti–PD-1 human IgG4 monoclonal antibody) and AGEN1884 (anti–CTLA-4 human IgG1 monoclonal antibody) are currently under evaluation as monotherapy in phase 1/2 studies in subjects with advanced tumors (NCT03104699 and NCT02694822, respectively).

• A phase 1/2, open-label, multi-arm trial to investigate the safety, tolerability, pharmacokinetics, and biological and clinical activity of AGEN1884 in combination with AGEN2034 in patients with metastatic or locally advanced solid tumors is currently ongoing (C550-01; ACTRN12618000003279; NCT03495882).

OBJECTIVE• The objective of this study is to assess safety and tolerability of AGEN1884 in combination with AGEN2034 in

patients with advanced/refractory solid tumors, with expansion into select solid tumors.

METHODS• This is an ongoing phase 1/2, open-label, study of AGEN1884 in combination with AGEN2034 in subjects with

advanced solid tumors, including cervical cancer.

• The study consists of 2 phases: – Phase 1: Dose escalation (focus of this poster) – Phase 2: Expansion in select tumors (ongoing)

• Phase 1 consisted of a standard 3+3 dose escalation with the following escalating dose levels and schedules: – AGEN2034 1 mg/kg administered every 2 weeks (q2w) in combination with AGEN1884 1 mg/kg administered every 6 weeks (q6w)

– AGEN2034 3 mg/kg administered every 2 weeks (q2w) in combination with AGEN1884 1 mg/kg administered every 6 weeks (q6w)

• AGEN2034 was administered IV over 60 min and AGEN1884 IV over 90 mins. AGEN1884 was to be administered on the same day as AGEN2034 ≥30 min after the completion of AGEN2034 administration.

• Dose escalation will continue until AGEN2034 3 mg/kg + AGEN1884 1 mg/kg (maximum combination dose level) is shown to be safe or the maximum tolerated dose (de� ned as the combination below which ≥33% of subjects develop dose-limiting toxicities [DLTs]) is reached.

• A schematic of the phase 1 dose-escalation design is presented in Figure 2.

Figure 2. Dose-Escalation Study Flow

Starting dose

Treat 3 patients;Assess DLTs after 3 weeks

Number ofDLTs

No

Yes

≤1 of 6

0 of 3 ≥2 of 3

≥2 of 6

1 of 3

1 of 3

Treat additional 3 patients;Assess DLTs after 3 weeks

Number of DLTs

MTD/maximum CDL to be con�rmed on total

of 6 patients

Initiate phase 2 at maximum planned CDL

or MTD

Determine MTDEscalate to next CDL

Maximum CDL per protocol?

Determine MTD

CDL, combination dose level; DLT, dose-limiting toxicity; MTD, maximum tolerated dose.

• Each subject will receive the combination treatment for a maximum of 24 months or until con� rmed disease progression, unacceptable toxicity, or any criterion for withdrawal from the trial or the investigational medicinal products occurs.

– Patients who do not complete the DLT observation period of 21 days after the � rst dose, for reasons other than a DLT, will be replaced.

– Cohorts will be back� lled to 10 subjects once they have reached DLT or MTD.

• Eligible patients include adults (aged ≥18 years) with a histologically or cytologically con� rmed diagnosis of a metastatic or locally advanced solid tumor for which no standard therapy is available or standard therapy has failed.

• The treatment phase was divided into 6-week cycles, each beginning with the combined administration of AGEN2034 and AGEN1884 on day 1. Thereafter, AGEN2034 will be administrated q2w, completing the 6-week cycle.

– Tumor assessments are to be conducted at 6, 12, and 18 weeks from � rst dose, and every 9 weeks thereafter until disease progression is con� rmed or a new line of therapy is initiated.

• Phase 1 endpoints: – Primary: Occurrence of DLTs in patients in dose escalation during the � rst 21 days of treatment – Secondary:

Con� rmed best overall response per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1, as determined by the investigator

Frequency and nature of treatment-emergent adverse events (TEAEs) Pharmacokinetic pro� le and immunogenicity of AGEN1884 and AGEN2034

RESULTS • As of July 16, 2018, the study was ongoing, though total enrollment was achieved in the phase 1 portion

(ie, 10 patients in each dose cohort). Data are preliminary as it is being cleaned and/or collected at this time.

• Five patients had discontinued from the study treatment prior to data extract: – AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w: Progressive disease, n=1; investigator decision, n=1 – AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w: Progressive disease, n=3

• There was 1 death not related to the study (66-year-old female with lung cancer treated with AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w died due to disease progression).

• Patients were Caucasian (100%) and primarily female (75%), with a median of 2.3 months from the most recent recurrence/progression to the administration of the � rst combination dose (Table 1).

• The cancer types for the 20 patients were as follows: breast cancer (triple negative, n=2; with lung metastases, n=1; with dermal lymphatics, n=1), ovarian cancer (n=2), mesothelioma (pleural, n=1; peritoneal, n=1), squamous carcinoma of head and neck (tongue; n=1), anal squamous carcinoma (n=1), pleomorphic soft tissue sarcoma (n=1), lung cancer (n=1), metastastic leiomyosarcoma (n=1), advanced gastrointestinal stromal tumor (n=1), rhabdomyosarcoma (n=1), alveolar rhabdomyosarcoma (n=1), recurrent chordoma of the thoracic spine (n=1), chordoma (n=1), colorectal-rectal (n=1), esophageal adenocarcinoma (n=1).

Table 1. Demographics and Baseline Characteristics (Safety Set) AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Age (years)

Mean (SD) 58.4 (8.13) 47.6 (19.68) 53.0 (15.66)

Min, Max 43, 69 21, 79 21, 79

Female, n (%) 8 (80%) 7 (70%) 15 (75%)

BMI (kg/m2)

Mean (SD) 28.71 (8.227) 27.48 (7.006) 28.10 (7.464)

Min, Max 18.8, 40.9 20.9, 45.5 18.8, 45.5

ECOG performance status, n (%)

0 5 (50%) 7 (70%) 12 (60%)

1 5 (50%) 3 (30%) 8 (40%)

Time between most recent progression and � rst dose date (months)

n 6 6 12

Mean (SD) 2.26 (1.665) 2.34 (1.002) 2.30 (1.311)

BMI, body mass index; ECOG, Eastern Cooperative Oncology Group.

• An overview of safety and tolerability is presented in Table 2.

Table 2. Overview of Treatment-Emergent Adverse Event (Safety Set)

Patients, n (%)

AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Any TEAE 10 (100%) 9 (90%) 19 (95%)

Any serious TEAE 3 (30%) 2 (20%) 5 (25%)

Any grade ≥3 TEAE 3 (30%) 2 (20%) 5 (25%)

Any immune-related TEAE 5 (50%) 4 (40%) 9 (45%)

Any treatment-related TEAE 8 (80%) 7 (70%) 15 (75%)

Any treatment-related serious TEAE 1 (10%) 2 (20%) 3 (15%)

Any treatment-related grade ≥3 TEAE 0 2 (20%) 2 (10%)

TEAE, treatment-emergent adverse event.

• As of July 16, 2018, no DLTs have been observed, and none of the TEAEs led to discontinuation or death.

• TEAEs were reported in 19 of 20 patients (95%), with the most common MedDRA version 18.1 preferred terms listed in (Table 3).

• Most TEAEs were mild to moderate in severity (grade 1 or 2); 5 patients experienced TEAEs of grade ≥3. – AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w: Vomiting, n=1; wound and cancer pain, n=1; hypotension, n=1

– AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w: Lower respiratory tract infection and pneumonia, n=1; pulmonary embolism, n=1

– Of these, the events of lower respiratory tract infection and pulmonary embolism were considered related to study treatment by the investigator.

Table 3. Most Common* Treatment-Emergent Adverse Event (Safety Set)

Patients, n (%)

AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Fatigue 5 (50%) 5 (50%) 10 (50%)

Nausea 3 (30%) 4 (40%) 7 (35%)

Pruritus 2 (20%) 3 (30%) 5 (25%)

Chills 3 (30%) 1 (10%) 4 (20%)

Vomiting 2 (20%) 2 (20%) 4 (20%)

Back pain 4 (40%) 0 4 (20%)*Occurring in ≥20% of patients.

• Serious TEAEs occurred in 5 patients: 3 receiving AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w and 2 receiving AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w

• 3 patients experienced serious TEAEs considered related to study treatment. – AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w: Diarrhea, n=1 – AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w: Lower respiratory tract infection, n=1; pulmonary embolism, n=1

• The summary of best overall response is listed in Table 4.

• The partial response was ongoing in one of the women with metastatic ovarian cancer. For patients with >90 days of treatment, stable disease was ongoing for 1 female with breast cancer (with lung metastases), 1 female with metastatic ovarian cancer, and 1 male with pleomorphic soft tissue sarcoma (with lung metastases).

Table 4. Summary of Best Overall Response at Time of Data Cut-off

Patients, n (%)

AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Complete response 0 0 0

Partial response 1 0 1

Stable disease 5 1 6

Progressive disease 3 6 9

Not evaluable 1 0 1

Pending 0 3 3Overall response determined by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1.

DISCUSSION• In the phase 1 portion, which is still ongoing, preliminary results demonstrate that AGEN1884 (1 mg/kg q6w) +

AGEN2034 (3 mg/kg q2w) is generally safe, well tolerated, and active in adults with select advanced/refractory solid tumors.

• The phase 2 portion of this study evaluating AGEN1884 in combination with AGEN2034 in adults with second-line cervical cancer and other solid tumors is ongoing.

– The phase 2 recommended dosing was determined as AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w.

References1. Topalian SL, Drake CG, Pardoll DM. Cancer Cell. 2015;27(4):450-61.2. Hodi FS, Chesney J, Pavlick AC, et al. Lancet Oncol.

2016;17(11):1558-68.

3. Antonia SJ, López-Martin JA, Bendell J, et al. Lancet Oncol. 2016;17(7):883-95.

4. Hellmann MD, Gettinger SN, Goldman JW, et al. J Clin Oncol. 2016;34(15 suppl):3001.5

Disclosures CD Dupont, AM Gonzalez, M Lim, D Savitsky, M Carini, S Hu, O Shebanova, E Dow, W Ortuzar, JS Buell, RB Stein, H Youssou� an: Agenus Inc.: current or former employment/consultancy and stock ownership.

Acknowledgments The authors thank Nicole Coolbaugh of The Medicine Group, LLC (New Hope, PA, USA) for providing medical writing and editorial support, which was funded by Agenus Inc. (Lexington, MA, USA) in accordance with Good Publication Practice guidelines. The licensed antibodies AGEN2034 and AGEN1884 were originally developed under a Collaborative Research and Development Agreement between Ludwig Cancer Research, 4-Antibody AG (now Agenus Switzerland Inc.) and Recepta Biopharma S.A. Recepta Biopharma S.A. has exclusive rights to these antibodies in Brazil and � ve other South American countries.

Funding This study was funded by Agenus Inc. (Lexington, MA, USA).

CorrespondenceWaldo Ortuzar: [email protected]

Presented at the 2018 Annual Congress of the European Society for Medical Oncology (ESMO), October 19–23, 2018, in Munich, Germany

Phase 1/2 Study of CTLA-4 Inhibitor AGEN1884 + PD-1 Inhibitor AGEN2034 in Patients With Advanced/Refractory Solid Tumors, With Expansion Into Second-Line Cervical Cancer and Solid Tumors

Jermaine Coward,1 Charlotte Lemech,2 Tarek Meniawy,3 Christopher D. Dupont,4 Ana M. Gonzalez,4 Min Lim,4 David Savitsky,4 Meg Carini,4 Shuang Hu,4 Olga Shebanova,4 Ed Dow,4

Waldo Ortuzar,4 Jennifer S. Buell,4 Robert Benjamin Stein,4 Hagop Youssou� an4

1Icon Cancer Center, South Brisbane, Australia; 2Scientia Clinical Research, Sydney, Australia; 3Linear Clinical Research, Perth, Australia; 4Agenus Inc. or subsidiary thereof (current or former employee), Lexington, MA, USA

1168P

Page 6: Phase 1/2 Study of CTLA-4 Inhibitor AGEN1884 + …agenusbio.com › wp-content › uploads › 2018 › 10 › ESMO-C-550-01...1 mg/kg q2w + AGEN1884 1 mg/kg q6w died due to disease

BACKGROUND• Antigen-speci� c T-cell activation is regulated by a balance of co-stimulatory and co-inhibitory signals, such as

those mediated by inhibitory receptors cytotoxic T-lymphocyte–associated antigen 4 (CTLA-4) and programmed cell death protein-1 (PD-1) (Figure 1).1

• Binding of these receptors to their ligands results in impaired T-cell function. For these reasons, antibody blockade of PD-1 and CTLA-4 has been identi� ed as a therapeutic modality to reinvigorate or induce tumor-speci� c T-cell immunity.1

Figure 1. Overview of Pathways Affected by CTLA-4 and PD-1CTLA-4 Inhibition PD-1 Inhibition

• In clinical trials, the combined inhibition of PD-1 and CTLA-4 pathways by blocking receptor-ligand interactions has resulted in objective clinical response and increased survival in several solid tumor indications, including melanoma and non-small cell lung carcinoma. As a result, ipilimumab (anti–CTLA-4) in combination with nivolumab (anti–PD-1) has been approved as a � rst line of treatment in patients with metastatic melanoma and is currently being tested in multiple other indications.2-4

• Agenus’ proprietary antibodies, AGEN2034 (anti–PD-1 human IgG4 monoclonal antibody) and AGEN1884 (anti–CTLA-4 human IgG1 monoclonal antibody) are currently under evaluation as monotherapy in phase 1/2 studies in subjects with advanced tumors (NCT03104699 and NCT02694822, respectively).

• A phase 1/2, open-label, multi-arm trial to investigate the safety, tolerability, pharmacokinetics, and biological and clinical activity of AGEN1884 in combination with AGEN2034 in patients with metastatic or locally advanced solid tumors is currently ongoing (C550-01; ACTRN12618000003279; NCT03495882).

OBJECTIVE• The objective of this study is to assess safety and tolerability of AGEN1884 in combination with AGEN2034 in

patients with advanced/refractory solid tumors, with expansion into select solid tumors.

METHODS• This is an ongoing phase 1/2, open-label, study of AGEN1884 in combination with AGEN2034 in subjects with

advanced solid tumors, including cervical cancer.

• The study consists of 2 phases: – Phase 1: Dose escalation (focus of this poster) – Phase 2: Expansion in select tumors (ongoing)

• Phase 1 consisted of a standard 3+3 dose escalation with the following escalating dose levels and schedules: – AGEN2034 1 mg/kg administered every 2 weeks (q2w) in combination with AGEN1884 1 mg/kg administered every 6 weeks (q6w)

– AGEN2034 3 mg/kg administered every 2 weeks (q2w) in combination with AGEN1884 1 mg/kg administered every 6 weeks (q6w)

• AGEN2034 was administered IV over 60 min and AGEN1884 IV over 90 mins. AGEN1884 was to be administered on the same day as AGEN2034 ≥30 min after the completion of AGEN2034 administration.

• Dose escalation will continue until AGEN2034 3 mg/kg + AGEN1884 1 mg/kg (maximum combination dose level) is shown to be safe or the maximum tolerated dose (de� ned as the combination below which ≥33% of subjects develop dose-limiting toxicities [DLTs]) is reached.

• A schematic of the phase 1 dose-escalation design is presented in Figure 2.

Figure 2. Dose-Escalation Study Flow

Starting dose

Treat 3 patients;Assess DLTs after 3 weeks

Number ofDLTs

No

Yes

≤1 of 6

0 of 3 ≥2 of 3

≥2 of 6

1 of 3

1 of 3

Treat additional 3 patients;Assess DLTs after 3 weeks

Number of DLTs

MTD/maximum CDL to be con�rmed on total

of 6 patients

Initiate phase 2 at maximum planned CDL

or MTD

Determine MTDEscalate to next CDL

Maximum CDL per protocol?

Determine MTD

CDL, combination dose level; DLT, dose-limiting toxicity; MTD, maximum tolerated dose.

• Each subject will receive the combination treatment for a maximum of 24 months or until con� rmed disease progression, unacceptable toxicity, or any criterion for withdrawal from the trial or the investigational medicinal products occurs.

– Patients who do not complete the DLT observation period of 21 days after the � rst dose, for reasons other than a DLT, will be replaced.

– Cohorts will be back� lled to 10 subjects once they have reached DLT or MTD.

• Eligible patients include adults (aged ≥18 years) with a histologically or cytologically con� rmed diagnosis of a metastatic or locally advanced solid tumor for which no standard therapy is available or standard therapy has failed.

• The treatment phase was divided into 6-week cycles, each beginning with the combined administration of AGEN2034 and AGEN1884 on day 1. Thereafter, AGEN2034 will be administrated q2w, completing the 6-week cycle.

– Tumor assessments are to be conducted at 6, 12, and 18 weeks from � rst dose, and every 9 weeks thereafter until disease progression is con� rmed or a new line of therapy is initiated.

• Phase 1 endpoints: – Primary: Occurrence of DLTs in patients in dose escalation during the � rst 21 days of treatment – Secondary:

Con� rmed best overall response per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1, as determined by the investigator

Frequency and nature of treatment-emergent adverse events (TEAEs) Pharmacokinetic pro� le and immunogenicity of AGEN1884 and AGEN2034

RESULTS • As of July 16, 2018, the study was ongoing, though total enrollment was achieved in the phase 1 portion

(ie, 10 patients in each dose cohort). Data are preliminary as it is being cleaned and/or collected at this time.

• Five patients had discontinued from the study treatment prior to data extract: – AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w: Progressive disease, n=1; investigator decision, n=1 – AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w: Progressive disease, n=3

• There was 1 death not related to the study (66-year-old female with lung cancer treated with AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w died due to disease progression).

• Patients were Caucasian (100%) and primarily female (75%), with a median of 2.3 months from the most recent recurrence/progression to the administration of the � rst combination dose (Table 1).

• The cancer types for the 20 patients were as follows: breast cancer (triple negative, n=2; with lung metastases, n=1; with dermal lymphatics, n=1), ovarian cancer (n=2), mesothelioma (pleural, n=1; peritoneal, n=1), squamous carcinoma of head and neck (tongue; n=1), anal squamous carcinoma (n=1), pleomorphic soft tissue sarcoma (n=1), lung cancer (n=1), metastastic leiomyosarcoma (n=1), advanced gastrointestinal stromal tumor (n=1), rhabdomyosarcoma (n=1), alveolar rhabdomyosarcoma (n=1), recurrent chordoma of the thoracic spine (n=1), chordoma (n=1), colorectal-rectal (n=1), esophageal adenocarcinoma (n=1).

Table 1. Demographics and Baseline Characteristics (Safety Set) AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Age (years)

Mean (SD) 58.4 (8.13) 47.6 (19.68) 53.0 (15.66)

Min, Max 43, 69 21, 79 21, 79

Female, n (%) 8 (80%) 7 (70%) 15 (75%)

BMI (kg/m2)

Mean (SD) 28.71 (8.227) 27.48 (7.006) 28.10 (7.464)

Min, Max 18.8, 40.9 20.9, 45.5 18.8, 45.5

ECOG performance status, n (%)

0 5 (50%) 7 (70%) 12 (60%)

1 5 (50%) 3 (30%) 8 (40%)

Time between most recent progression and � rst dose date (months)

n 6 6 12

Mean (SD) 2.26 (1.665) 2.34 (1.002) 2.30 (1.311)

BMI, body mass index; ECOG, Eastern Cooperative Oncology Group.

• An overview of safety and tolerability is presented in Table 2.

Table 2. Overview of Treatment-Emergent Adverse Event (Safety Set)

Patients, n (%)

AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Any TEAE 10 (100%) 9 (90%) 19 (95%)

Any serious TEAE 3 (30%) 2 (20%) 5 (25%)

Any grade ≥3 TEAE 3 (30%) 2 (20%) 5 (25%)

Any immune-related TEAE 5 (50%) 4 (40%) 9 (45%)

Any treatment-related TEAE 8 (80%) 7 (70%) 15 (75%)

Any treatment-related serious TEAE 1 (10%) 2 (20%) 3 (15%)

Any treatment-related grade ≥3 TEAE 0 2 (20%) 2 (10%)

TEAE, treatment-emergent adverse event.

• As of July 16, 2018, no DLTs have been observed, and none of the TEAEs led to discontinuation or death.

• TEAEs were reported in 19 of 20 patients (95%), with the most common MedDRA version 18.1 preferred terms listed in (Table 3).

• Most TEAEs were mild to moderate in severity (grade 1 or 2); 5 patients experienced TEAEs of grade ≥3. – AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w: Vomiting, n=1; wound and cancer pain, n=1; hypotension, n=1

– AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w: Lower respiratory tract infection and pneumonia, n=1; pulmonary embolism, n=1

– Of these, the events of lower respiratory tract infection and pulmonary embolism were considered related to study treatment by the investigator.

Table 3. Most Common* Treatment-Emergent Adverse Event (Safety Set)

Patients, n (%)

AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Fatigue 5 (50%) 5 (50%) 10 (50%)

Nausea 3 (30%) 4 (40%) 7 (35%)

Pruritus 2 (20%) 3 (30%) 5 (25%)

Chills 3 (30%) 1 (10%) 4 (20%)

Vomiting 2 (20%) 2 (20%) 4 (20%)

Back pain 4 (40%) 0 4 (20%)*Occurring in ≥20% of patients.

• Serious TEAEs occurred in 5 patients: 3 receiving AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w and 2 receiving AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w

• 3 patients experienced serious TEAEs considered related to study treatment. – AGEN2034 1 mg/kg q2w + AGEN1884 1 mg/kg q6w: Diarrhea, n=1 – AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w: Lower respiratory tract infection, n=1; pulmonary embolism, n=1

• The summary of best overall response is listed in Table 4.

• The partial response was ongoing in one of the women with metastatic ovarian cancer. For patients with >90 days of treatment, stable disease was ongoing for 1 female with breast cancer (with lung metastases), 1 female with metastatic ovarian cancer, and 1 male with pleomorphic soft tissue sarcoma (with lung metastases).

Table 4. Summary of Best Overall Response at Time of Data Cut-off

Patients, n (%)

AGEN2034 1 mg/kg + AGEN1884 1 mg/kg

(N=10)

AGEN2034 3 mg/kg + AGEN1884 1 mg/kg

(N=10)Total Patients

(N=20)

Complete response 0 0 0

Partial response 1 0 1

Stable disease 5 1 6

Progressive disease 3 6 9

Not evaluable 1 0 1

Pending 0 3 3Overall response determined by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1.

DISCUSSION• In the phase 1 portion, which is still ongoing, preliminary results demonstrate that AGEN1884 (1 mg/kg q6w) +

AGEN2034 (3 mg/kg q2w) is generally safe, well tolerated, and active in adults with select advanced/refractory solid tumors.

• The phase 2 portion of this study evaluating AGEN1884 in combination with AGEN2034 in adults with second-line cervical cancer and other solid tumors is ongoing.

– The phase 2 recommended dosing was determined as AGEN2034 3 mg/kg q2w + AGEN1884 1 mg/kg q6w.

References1. Topalian SL, Drake CG, Pardoll DM. Cancer Cell. 2015;27(4):450-61.2. Hodi FS, Chesney J, Pavlick AC, et al. Lancet Oncol.

2016;17(11):1558-68.

3. Antonia SJ, López-Martin JA, Bendell J, et al. Lancet Oncol. 2016;17(7):883-95.

4. Hellmann MD, Gettinger SN, Goldman JW, et al. J Clin Oncol. 2016;34(15 suppl):3001.5

Disclosures CD Dupont, AM Gonzalez, M Lim, D Savitsky, M Carini, S Hu, O Shebanova, E Dow, W Ortuzar, JS Buell, RB Stein, H Youssou� an: Agenus Inc.: current or former employment/consultancy and stock ownership.

Acknowledgments The authors thank Nicole Coolbaugh of The Medicine Group, LLC (New Hope, PA, USA) for providing medical writing and editorial support, which was funded by Agenus Inc. (Lexington, MA, USA) in accordance with Good Publication Practice guidelines. The licensed antibodies AGEN2034 and AGEN1884 were originally developed under a Collaborative Research and Development Agreement between Ludwig Cancer Research, 4-Antibody AG (now Agenus Switzerland Inc.) and Recepta Biopharma S.A. Recepta Biopharma S.A. has exclusive rights to these antibodies in Brazil and � ve other South American countries.

Funding This study was funded by Agenus Inc. (Lexington, MA, USA).

CorrespondenceWaldo Ortuzar: [email protected]

Presented at the 2018 Annual Congress of the European Society for Medical Oncology (ESMO), October 19–23, 2018, in Munich, Germany

Phase 1/2 Study of CTLA-4 Inhibitor AGEN1884 + PD-1 Inhibitor AGEN2034 in Patients With Advanced/Refractory Solid Tumors, With Expansion Into Second-Line Cervical Cancer and Solid Tumors

Jermaine Coward,1 Charlotte Lemech,2 Tarek Meniawy,3 Christopher D. Dupont,4 Ana M. Gonzalez,4 Min Lim,4 David Savitsky,4 Meg Carini,4 Shuang Hu,4 Olga Shebanova,4 Ed Dow,4

Waldo Ortuzar,4 Jennifer S. Buell,4 Robert Benjamin Stein,4 Hagop Youssou� an4

1Icon Cancer Center, South Brisbane, Australia; 2Scientia Clinical Research, Sydney, Australia; 3Linear Clinical Research, Perth, Australia; 4Agenus Inc. or subsidiary thereof (current or former employee), Lexington, MA, USA

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