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Shares of Denali Therapeutics DNLI gained 11.8% on Wednesday after the company announced that it 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). The same has also been received by the Center for Drug Evaluation and Research (CDER) of the FDA.
DNLI reported ongoing collaborative and productive engagement with CDER, aligning with the same on the content of the BLA data package, including the use of CSF HS as a surrogate endpoint for accelerated approval and the path to full approval.
Denali 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. Tividenofusp alfa, an Enzyme Transport Vehicle-enabled iduronate-2-sulfatase (IDS) replacement therapy, enjoys the FDA’s Fast Track and Breakthrough Therapy designations in the United States for MPS II. In the EU, the candidate enjoys the Priority Medicines designation for the same indication.
Year to date, shares of DNLI have lost 32.6% against the industry’s 1.2% growth.
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Updates Regarding DNLI’s Key Pipeline Candidates
Denali continues to enroll patients in the global phase II/III COMPASS study of tividenofusp alfa, which aims to support global regulatory approvals. In January 2025, it expanded target enrollment for neuronopathic participants (Cohort A) to 42 patients, reflecting steady progress in recruitment and data collection. The candidate has not yet been approved for therapeutic use by any health authority.
MPS II is a rare genetic disorder affecting more than 2,000 people worldwide, mostly males, and causes physical, cognitive, and behavioral symptoms.
Denali is also evaluating DNL126 for the treatment of Sanfilippo syndrome type A (MPS IIIA) in a phase I/II study. The candidate enjoys the FDA’s Orphan Disease designation and Fast Track status for this indication.
DNL126 has been selected for the FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) program. This pilot initiative aims to accelerate the development of rare disease treatments.
Based on initial positive phase I/II results demonstrating proof of concept for MPS IIIA, Denali is engaging in productive collaboration with the FDA, under START, to align on a pathway for accelerated development and approval of DNL126 in the United States. This candidate has also not yet been approved for therapeutic use by any health authority.
Denali Therapeutics Inc. Price and Consensus
Denali Therapeutics Inc. price-consensus-chart | Denali Therapeutics Inc. Quote