Company: DNLI
Filing Date: 2025-04-17
Form Type: DEF 14A
Source: 0001714899-25-000087
Chunk: 41

Company: Denali Therapeutics Inc.
Filing Date: 2025-04-17
Form: DEF 14A
Chunk 41
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accharidosis II ("MPS II", or Hunter syndrome). On April 2, 2025, we announced initiation of a rolling submission of the biologics license application ("BLA") for tividenofusp alfa under the U.S. Food and Drug Administration(FDA)'s accelerated approval pathway, and we are preparing for a potential commercial launch in late 2025 or early 2026. Our TV-enabled clinical development portfolio also includes DNL126 (ETV:SGSH) for MPS IIIA (Sanfilippo syndrome) and DNL593 (PTV:PGRN) for frontotemporal dementia-granulin (“FTD-GRN”). We are also developing BIIB122/DNL151 (small molecule LRRK2 inhibitor) for Parkinson’s disease. We believe the combination of a clinically validated delivery platform and a maturing therapeutic portfolio will position us for long-term success in our goal to deliver barrier-crossing, targeted, and effective medicines.

In 2024 we expanded and progressed our clinical programs for neurodegenerative and lysosomal storage diseases. Some 2024 business milestones include:

Advanced and expanded our TransportVehicle TM clinical portfolio

Tividenofusp alfa (DNL310, ETV:IDS): We announced plans to file a BLA in early 2025 for accelerated approval of tividenofusp alpha for the treatment of MPS II (Hunter syndrome), based on the outcome of a successful meeting with the FDA's Center for Drug Evaluation and Research ("CDER") whereupon alignment was reached that cerebrospinal fluid heparan sulfate ("CSF HS") can be used as a surrogate endpoint. We completed enrollment of 47 participants with MPS II in the Phase 1/2 open-label study. We also completed target enrollment of 33 participants with neuronopathic MPS II in Cohort A of the global Phase 2/3 COMPASS study and continued to enroll participants with non-neuronopathic MPS II in Cohort B.

DNL126 (ETV:SGSH): We announced that dosing commenced in the Phase 1/2 study of DNL126 in participants with MPS IIIA and that DNL126 was selected for the FDA's Support for clinical Trials Advancing Rare disease Therapeutics ("START") program to accelerate the development of rare disease therapeutics. We also announced that preliminary Phase 1/2 data from up to 25 weeks of dosing demonstrated a significant reduction in CSF HS levels from baseline, including