Whitehawk Therapeutics (WHWK) Jefferies 2024 Global Healthcare Conference summary

Commercial performance and product adoption

• FYARRO, approved for PEComa, generates about $25 million annually with strong market penetration and 70% first-line share among sarcoma treaters in the U.S.

• Sales have stabilized at around $6 million per quarter, with minor Q1 fluctuations attributed to clinical trial enrollment at major centers.

• Anticipates a return to growth in Q2 as the TSC1/TSC2 trial enrollment is now closed.

TSC1/TSC2 tumor-agnostic trial (PRECISION1)

• PRECISION1 is a tumor-agnostic trial enrolling 120 heavily pretreated patients with TSC1 or TSC2 mutations across over 20 tumor types, excluding sarcoma.

• First interim analysis showed a 26% response rate in TSC1 (5/19) and 11% in TSC2 (2/18), with durable responses in a last-line setting.

• Second interim analysis (80 patients, independent review, 6-month follow-up) is expected in Q3, with final data in Q1 next year.

• Even distribution of tumor types was achieved, with no over-enrollment in any single type.

• The trial design and FDA guidance allow for potential independent or combined regulatory submissions for TSC1 and TSC2.

Pipeline expansion and future plans

• Phase 2 trials are ongoing in neuroendocrine tumors (NETs) and endometrioid endometrial cancer (EEC), targeting improved response rates over existing mTOR therapies.

• Both studies are open and enrolling, with part one updates (about 10 patients each) expected by year-end; full enrollment targets about 30 patients per study.

• The company leveraged over 150 sites and next-generation sequencing for rapid, broad enrollment in the tumor-agnostic trial.

• Standard NGS testing for TSC1/TSC2 is already available, supporting future commercial adoption.