Agenus (AGEN) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Clinical data and efficacy

• BOT/BAL combination showed a 23% response rate and 21-month median overall survival in phase I for relapsed/refractory microsatellite stable colorectal cancer, outperforming standard care options with lower response and survival rates.

• In the neoadjuvant NEST study, five out of nine patients achieved pathologic complete response with BOT/BAL, compared to 4% with standard chemotherapy.

• Phase II data showed a 19.4% response rate in the 75 mg BOT combination arm, with 90% of patients alive at six months, mirroring phase I results.

• Standard of care arm in phase II had a 0% response rate, highlighting BOT/BAL's potential benefit.

• BOT/BAL demonstrated activity across nine IO-refractory or cold tumors, including melanoma, sarcoma, and non-small cell lung cancer.

Mechanism of action and differentiation

• Botensilimab features two Fc region mutations enhancing immune synapse formation and neoantigen recognition, and a third mutation reducing complement-mediated toxicities.

• Enhanced depletion of immunosuppressive T-regulatory cells and lower rates of immune-mediated adverse events compared to first-generation CTLA-4 inhibitors.

Regulatory and development plans

• Agreement with FDA on phase III dose (75 mg botensilimab + 240 mg balstilimab); further data on contribution of components to be presented in November.

• Option to add a monotherapy arm in phase III if needed; minor FDA feedback incorporated into study design.

• FDA did not support accelerated approval with current data; plans to return with more mature data and to seek EMA advice for potential conditional approval in Europe.