EXACT Therapeutics (EXTX) ISTU 2024 presentation summary

Strategic focus and technology

• Developing a precision medicine platform using ultrasound to enhance targeted drug delivery in oncology, with a focus on non-invasive therapies.

• Acoustic Cluster Therapy (ACT) enables and increases local drug delivery by overcoming biological barriers in tumors.

• Proprietary PS101 prodrug uses microbubble and microdroplet clusters activated by ultrasound for targeted delivery, requiring no drug reformulation.

• Treatment involves a two-step ultrasound process for activation and enhancement, resulting in prolonged, localized therapeutic delivery.

• Platform is drug-agnostic and compatible with a wide range of therapeutics.

Clinical and preclinical results

• Phase 1 trial in liver metastases showed over 10x tumor shrinkage in insonated lesions compared to control, with no ACT-related adverse events.

• Preclinical models demonstrated strong efficacy across multiple cancer types, including complete tumor disappearance in a significant proportion of animals.

• ACT has shown the ability to safely and temporarily disrupt the blood-brain barrier, suggesting potential in CNS indications.

• Interim clinical data reviewed by independent, blinded central reviewers confirmed significant anti-tumor activity.

Pipeline and development plans

• Phase 2 ENACT study planned for 2H 2024 in first-line locally advanced or borderline resectable pancreatic cancer, targeting survival prolongation and conversion to resectability.

• Potential for orphan drug designation and expansion into oligometastatic pancreatic cancer.

• Pancreatic cancer represents a significant unmet need, with low 5-year survival rates and increasing incidence.