EXACT Therapeutics (EXTX) CMR Symposium 2024 presentation summary

Technology overview

• Acoustic Cluster Therapy (ACT) is a noninvasive, ultrasound-mediated platform enabling targeted, organ-specific drug delivery for oncology applications.

• ACT uses PS101, a proprietary prodrug consisting of gas bubbles and oil droplets, which is activated by ultrasound to enhance drug delivery.

• The process involves a two-step ultrasound activation and enhancement, resulting in prolonged, targeted delivery of therapeutics.

• Broad intellectual property coverage exists, with seven patent families and recent expansion into immuno-oncology applications.

Preclinical and clinical results

• Preclinical studies show ACT significantly increases complete cancer disappearance rates across multiple tumor types, including pancreatic, breast, colon, and prostate cancers.

• In colorectal cancer models, ACT combined with irinotecan led to a fourfold increase in complete responders compared to irinotecan alone.

• Phase 1 ACTIVATE study in patients with liver metastases from colorectal cancer demonstrated over 10x greater tumor shrinkage in insonated lesions versus controls, with no ACT-related adverse events.

• Dose escalation in Phase 1 showed ACT is safe, deliverable in outpatient settings, and provides proof of concept for enhanced tumor reduction.

Pipeline and future directions

• Phase 2 trial in first-line pancreatic cancer is planned for the second half of 2024, targeting locally advanced or borderline resectable patients.

• Preclinical pancreatic cancer models showed a 50% complete response rate when ACT was combined with standard chemotherapy, compared to 11% with chemotherapy alone.

• Expansion into immuno-oncology and CNS/BBB applications is under evaluation, with recent patents and collaborations supporting growth.

• Orphan drug designation and potential expansion into oligometastatic pancreatic cancer are being considered.