Whitehawk Therapeutics (WHWK) Jefferies 2024 Global Healthcare Conference summary

Commercial performance and product adoption

• FYARRO (nab-sirolimus) is approved for PEComa, a rare soft tissue sarcoma, with annual sales around $25 million and strong first-line adoption at 70% share among sarcoma treaters.

• Sales have stabilized at approximately $6 million per quarter, with minor fluctuations attributed to the ultra-rare nature of the indication and clinical trial enrollment overlap.

• Q1 sales saw a temporary dip due to cannibalization from clinical trial enrollment at major centers, but growth is expected to resume in Q2 as the trial is now closed.

TSC1/TSC2 tumor-agnostic trial (PRECISION1)

• TSC1 and TSC2 mutations occur in about 2% of all cancers, representing a potential market of 16,000 new U.S. cases annually, a significant increase over PEComa.

• The PRECISION1 trial enrolled 120 heavily pretreated patients across 20+ tumor types, excluding sarcoma, with even distribution and no over-enrollment in any type.

• First interim analysis showed a 26% response rate in TSC1 (5/19) and 11% in TSC2 (2/18), with durable responses and clinical benefit even in stable disease cases.

• Second interim analysis (80 patients, independent review, 6-month follow-up) is expected in Q3, with final data in Q1 next year; results will inform FDA submission discussions.

• The trial is among the first true tumor-agnostic studies for a targeted therapy, with separate arms for TSC1 and TSC2 as guided by the FDA.

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 oral mTOR inhibitors.

• NETs trial aims for 25-30 patients, seeking better efficacy than current single-digit response rates; EEC trial combines nab-sirolimus with estrogen receptor blockade for second-line therapy.

• Both studies are open and enrolling, with part one updates (about 10 patients each) expected by year-end.

• The company leveraged next-generation sequencing (NGS) for patient identification, with standard testing available in clinical practice, supporting future commercial adoption.

• Over 150 sites were activated for the tumor-agnostic trial, demonstrating robust infrastructure and timely execution.