- Denali's lead program, **tividendifusp alfa pidufidate (TIVI)**, an enzyme replacement therapy for Hunter syndrome, has a **PDUFA date of January 5** and is expected to launch in the **U.S. in 2026**.  **[1714899_tx_382210_0]** **[1714899_tx_382210_1]**  The company anticipates **premium pricing** and **20% COGS** for TIVI.  **[1714899_tx_382210_3]** 
- The **COMPASS confirmatory trial** for TIVI is expected to complete Cohort A by the **end of 2025**, with a full readout anticipated **two years from the end of 2025**, which will support broader global approvals.  **[1714899_tx_382210_4]** 
- Denali is pursuing an **accelerated approval path** for its Sanfilippo (MPS IIIA) program, with a Phase 3 trial expected to begin soon, targeting an estimated **300-400 patients in the U.S.**.  **[1714899_tx_382210_4]** **[1714899_tx_382210_5]** 
- The company is expanding its Enzyme Transport Vehicle (ETV) platform to include a **Pompe program**, focusing on the larger **Late-Onset Pompe Disease (LLPD)** population, and aims to file **one to two Investigational New Drug (IND) applications per year** across its pipeline.  **[1714899_tx_382210_6]** **[1714899_tx_382210_7]** **[1714899_tx_382210_8]**

Denali Therapeutics (DNLI)