Denali Therapeutics (DNLI) Corporate presentation summary

Strategic vision and platform technology

• Focused on transforming treatment for serious diseases by delivering biotherapeutics to the whole body, including the brain, using the proprietary TransportVehicle™ (TV) platform.

• TV platform enables systemic delivery of enzymes, oligonucleotides, and antibodies across the blood-brain barrier (BBB), addressing both central and peripheral disease manifestations.

• Platform is modular, optimized for brain uptake, safety, and manufacturability, with over 350 patents and 20+ publications.

Commercial and pipeline highlights

• Achieved historic FDA approval for AVLAYAH™ (tividenofusp alfa) for MPS II, the first therapy to address both body and brain in this disease.

• Preparing for two near-term launches: AVLAYAH™ in 2026 and DNL126 for MPS IIIA in 2027, targeting a combined $1B+ market opportunity.

• Deep pipeline includes programs for lysosomal storage disorders, Alzheimer's, Parkinson's, Pompe disease, and frontotemporal dementia.

Clinical data and product performance

• AVLAYAH™ demonstrated normalization of CNS and peripheral biomarkers and improvements in adaptive behavior, cognition, and hearing in MPS II patients.

• Safety profile of AVLAYAH™ is consistent with enzyme replacement therapies, with infusion-related reactions decreasing over time.

• DNL126 for MPS IIIA showed substantial reductions in CNS and peripheral biomarkers, with manageable safety profile and no treatment-related serious adverse events.