Denali Therapeutics (DNLI) TD Cowen 46th Annual Health Care Conference summary

Strategic overview and 2026 outlook

• Anticipates a pivotal year in 2026 with the awaited PDUFA date for tividenofusp alfa for Hunter syndrome on April 5th.

• DNL126 for Sanfilippo syndrome is on track for an accelerated approval filing and potential launch in 2027.

• Two key clinical data readouts expected: LRRK2 inhibitor in Parkinson’s mid-year and progranulin program in FTD later in the year.

• Plans to initiate three new clinical studies, including two Alzheimer’s programs and an enzyme program for Pompe disease.

• Well-capitalized with nearly $1 billion in cash and an additional $200 million contingent on approval.

Regulatory and clinical progress

• Ongoing, productive FDA dialogue for tividenofusp alfa, with label and post-marketing discussions underway.

• Three-month PDUFA delay was due to a corrected molecular weight error, now resolved with no impact on efficacy or safety.

• Differentiation from competitors highlighted by robust, long-term data and validated biomarkers.

• Strong genetic classification process supports regulatory confidence.

Commercial readiness and launch strategy

• Commercial infrastructure and experienced field team in place, with targeted approach due to known patient locations.

• Productive payer engagement, with payers recognizing the unmet need and robust data.

• Pricing strategy will depend on final label; consensus is growing that all Hunter syndrome patients may benefit.

• Adoption expected to start with newborns and severe cases, with an S-shaped adoption curve and revenue inflection in 2027.