Actuate Therapeutics (ACTU) Corporate presentation summary

Therapeutic pipeline and clinical results

• Elraglusib, a GSK-3β inhibitor, shows significant survival benefits in multiple hard-to-treat cancers, including metastatic pancreatic ductal adenocarcinoma (mPDAC), with a novel multimodal mechanism of action and synergy with standard-of-care therapies and RAS/RAF/MEK inhibitors.

• In a Phase 2 randomized controlled trial, elraglusib plus gemcitabine/nab-paclitaxel doubled one-year overall survival in mPDAC patients compared to standard therapy, with a 44.1% vs 22.3% one-year survival rate and a 40% improvement in median overall survival (HR: 0.62; p=0.02).

• Greater drug exposure correlates with improved outcomes; 60% of weekly IV patients and 85% of oral dosing patients reach target exposure, suggesting oral tablets may further enhance efficacy.

• IND clearance received for oral tablet formulation, with plans to expand into blockbuster indications such as first-line mPDAC, metastatic melanoma, colorectal cancer, and NSCLC.

Pediatric and rare disease programs

• Elraglusib demonstrates durable responses in heavily pre-treated Ewing Sarcoma patients, including complete responses and long-term disease-free survival.

• In neuroblastoma, 38% of advanced relapsed/refractory patients achieved stable disease or better; a BEACON-2 trial is planned in collaboration with Cancer Research UK.

• Accelerated regulatory pathways and priority review voucher eligibility for pediatric indications, with a combined market size of $700M–$1B.

Development strategy and milestones

• Strategy includes expanding elraglusib’s pipeline with oral tablets, advancing combination regimens in PDAC, and progressing pediatric indications.

• Key milestones through 2027 include nonclinical data on RAS/MEK combinations, initiation of pivotal studies, and interim safety data in pediatric trials.