Denali Therapeutics (DNLI) investor relations material

Executive summary

• Achieved first FDA approval for AVLAYAH (tividenofusp alfa) in March 2026 for Hunter syndrome, marking the first medicine to use transferrin receptor technology to cross the blood-brain barrier; commercial launch began in April 2026 with strong initial uptake.

• Secured $200M in gross proceeds from a synthetic royalty funding agreement with Royalty Pharma, with potential for an additional $75M upon EMA approval.

• Takeda terminated the DNL593 collaboration for strategic reasons, with all rights reverting; not related to efficacy or safety.

• Expanded clinical pipeline with ongoing and planned studies, including first patient dosed with OTV-enabled DNL628 for Alzheimer's and progress in DNL593 for FTD-GRN.

• Awarded a Rare Pediatric Disease Priority Review Voucher by the FDA, which is transferable.

Financial highlights

• Net loss of $128.4M for Q1 2026, compared to $133.0M in Q1 2025.

• Total operating expenses decreased 6% year-over-year to $137.4M, driven by lower R&D costs.

• Research and development expenses decreased to $103.8M from $116.2M year-over-year, mainly due to timing of AVLAYAH supply manufacturing and lower external costs.

• General and administrative expenses rose to $33.5M from $29.4M, reflecting higher headcount for commercial activities.

• Cash, cash equivalents, and marketable securities totaled $1.05B as of March 31, 2026.

Outlook and guidance

• Expect continued operating losses as R&D and commercialization activities expand.

• Product revenue from AVLAYAH expected to grow but not sufficient to offset expenses in the near term.

• Existing cash resources projected to fund operations for at least 12 months from filing.

• Data from the Phase 1/2 DNL593 study for FTD-GRN expected by end of 2026; DNL628 Phase 1b Alzheimer's study data anticipated in first half of 2027.

• Regulatory filing for DNL921 (ATV: Abeta) for Alzheimer's planned for first half of 2026.