EXACT Therapeutics (EXTX) investor relations material

Strategic and Clinical Focus

• Prioritizing development of a proprietary ultrasound-activated drug delivery platform, PS101, to enhance therapeutic uptake in hard-to-treat cancers, with initial focus on locally advanced pancreatic cancer (LAPC).

• Mission to change the standard of care for LAPC, targeting high unmet need cancers.

• Phase I trial in liver metastasis showed a fourfold increase in tumor shrinkage and excellent safety, supporting transition to phase II in LAPC.

• Broad patent protection and intellectual property coverage secured across major markets, including the US.

• Financial runway extended into 2027 through a successful warrants exercise, covering key readouts from the Phase 2 trial.

Phase II ENACT Trial Progress and Results

• ENACT trial targets first-line LAPC patients, aiming to increase conversion to surgical resection and double median overall survival.

• Initial safety review by the Trial Monitoring Committee found no added safety concerns, recommending dose escalation and inclusion of borderline resectable patients.

• Early efficacy data: ≥85% reduction in CA 19-9 biomarker and significant tumor shrinkage (46% and 90% in first two patients), with one patient successfully converted to curative surgery.

• Interim analysis expected mid-2026, final results in first half of 2027.

• Major milestones achieved include final Phase 1 results, US IND approval, first patient dosed in Phase 2, and key preclinical updates.

Pipeline, Technology, and Expansion

• PS101 pipeline prioritizes pancreatic cancer, with additional programs in liver metastases, glioblastoma, immunotherapy, CNS diseases, and gene therapy.

• PS101 is a non-invasive, ultrasound-activated prodrug that enhances targeted drug delivery by opening biological barriers.

• The treatment process involves a two-step ultrasound activation, enabling prolonged and targeted drug delivery.

• Preclinical data support expansion into CNS cancers, immuno-oncology, and gene therapy, with promising results in glioblastoma and early signals in immunotherapy.

• Ongoing collaborations with academic partners for technology expansion and preclinical research.