Akari Therapeutics (AKTX) investor relations material

Payload innovation and scientific differentiation

• PH1 payload targets RNA splicing, inducing potent cytotoxicity and activating both innate and adaptive immunity, with a differentiated safety profile compared to existing ADCs.

• Unique mechanism increases mis-spliced RNAs, leading to cell death and immune activation via protein neoantigens.

• Preclinical data show superior tumor regression and immune response versus leading Trop-2 ADCs, with reduced off-target effects due to a proprietary non-cleavable linker.

• No neutropenia, mucositis, or interstitial lung disease observed in non-human primate studies; safety findings are manageable and reversible.

Lead program and clinical development

• Lead candidate AKTX-101 (Trop-2 PH1 ADC) is entering IND-enabling studies, with Phase 1 filing expected in Q4 2026 and first-in-human trial start in ~12 months.

• Initial clinical focus is on urothelial cancer, with rapid expansion planned into other Trop-2 expressing solid tumors such as gastric, lung, and breast cancers.

• Phase 1a/1b trial will assess safety, dosing, and early efficacy in multiple tumor types, with initial data expected 18 months from trial start.

• AKTX-101 has demonstrated significant preclinical activity in aggressive urothelial cancer models, outperforming standard therapies.

Market opportunity and value proposition

• Large addressable market across multiple solid tumors expressing Trop-2, with significant unmet needs in urothelial cancer.

• Second-line urothelial cancer represents a valuable entry point, with 75,000 patients annually in key markets and limited effective therapies.

• Comparable ADC companies have seen 5-10x valuation increases post-Phase 1 data, suggesting significant upside potential within 12-18 months.

• Multiple value inflection points anticipated as clinical milestones are reached over the next 18 months.