DNLI Begins Rolling BLA Submission for Hunter Syndrome Drug, Stock Up

Core Viewpoint - Denali Therapeutics (DNLI) has initiated a rolling submission of a biologics license application (BLA) for accelerated approval of tividenofusp alfa for the treatment of Hunter syndrome (MPS II), leading to an 11.8% increase in its shares [1] Company Developments - DNLI is engaged in ongoing collaboration with the FDA's Center for Drug Evaluation and Research (CDER) regarding the BLA data package, including the use of CSF HS as a surrogate endpoint for accelerated approval [2] - The company expects to complete its BLA submission by mid-May 2025 and is preparing for a potential U.S. commercial launch in late 2025 or early 2026 [3] - Tividenofusp alfa has received Fast Track and Breakthrough Therapy designations from the FDA for MPS II, and it holds the Priority Medicines designation in the EU [3] - DNLI has lost 32.6% in share value year-to-date, contrasting with a 1.2% growth in the industry [4] Clinical Trials and Pipeline - Denali is currently enrolling patients in the global phase II/III COMPASS study of tividenofusp alfa to support global regulatory approvals, with an expanded target enrollment for neuronopathic participants to 42 patients [6][7] - The candidate has not yet received approval for therapeutic use by any health authority [7] - Denali is also evaluating DNL126 for the treatment of Sanfilippo syndrome type A (MPS IIIA) in a phase I/II study, which has received Orphan Disease designation and Fast Track status from the FDA [8] - Initial positive phase I/II results for DNL126 have led to productive collaboration with the FDA under the START program to align on a pathway for accelerated development and approval [9]