Aprea Therapeutics (APRE) H.C. Wainwright 28th Annual Global Investment Conference summary

Pipeline overview and strategy

• Focused on synthetic lethality by targeting the DNA damage response pathway, with a pipeline including WEE1 and ATR inhibitors, a p53 reactivator, and a preclinical DYRK1 program.

• WEE1 inhibitor is in clinical dose escalation, ATR inhibitor completed dose escalation and is seeking combination partners.

• Emphasis on a wide therapeutic window to minimize toxicity compared to traditional chemotherapy.

• Cash position of $41 million at last quarter, with a cash runway into Q1 2028 and recent $30 million financing to support clinical milestones.

• Market cap estimated at $40–50 million on a fully diluted basis.

Clinical development and patient data

• Dose escalation established 150 mg as the minimum efficacious dose, with confirmed partial response at 220 mg.

• Patient enrollment strategy includes indication-independent, mutation-specific, and mutation-independent cohorts.

• Recent financing supports enrollment of up to 100 patients, focusing on USC and cyclin E overexpressing platinum-resistant ovarian cancer.

• Dose-response observed: stable disease at 70 mg and 100 mg, partial response at 150 mg, and longer duration/stable disease at 220 mg.

• Drug is orally administered daily and has shown promising responses in heavily pretreated patients.

Safety, efficacy, and future outlook

• Drug has been well-tolerated at 150 mg and 220 mg, with minimal side effects and no significant toxicity.

• Notable patient cases include significant tumor reduction and durable stable disease in patients with PPP2R1A and FBXW7 mutations.

• All efficacy data to date is from monotherapy; combination studies are planned due to favorable safety profile.

• Additional data updates, including durability and response, are expected in Q4 at upcoming conferences.

• ATR inhibitor program seeks external-funded collaborations; p53 program will revisit patient subgroups for potential responders.