Denali Therapeutics (DNLI) Stifel 2026 Healthcare Conference 2026 summary

AVLAYAH launch and commercial outlook

• Strong launch interest from families and KOLs; feedback indicates impact across a broad patient population with neuropathic symptoms.

• ~70% of commercial lives covered, with Medicaid coverage in 20 states; Q2 commercial revenue reached $7.8M after $3.6M in Q1.

• U.S. Hunter disease population estimated at 500, with ~375 eligible under the label; goal is to transition all eligible patients to AVLAYAH.

• Faster payer coverage and clinical-trial patient transitions accelerated the ramp; Q4 commercial revenue guidance is $30M-$32M, with growth expected to become more measured.

Hunter disease confirmatory study

• COMPASS is designed around prior Phase I/II data from 47 patients, including biomarker, behavioral and cognitive outcomes; management expressed confidence in reproducing findings in a placebo-controlled study.

• Natural-history data inform placebo expectations; the robust study includes cognitive outcomes and Part B assessment of somatic disease aspects.

• COMPASS is expected to complete in 2027, with filing planned in 2028 based on the totality of data; the study could support broader Hunter disease use, including adults.

Sanfilippo program

• FDA discussions have aligned on heparan sulfate reduction as the basis for potential accelerated approval; dose-finding patients showed >80% reduction, with many normalizing on treatment.

• Filing plans are based on a 20-patient cohort that has completed the study; detailed data are planned for WORLDSymposium early next year, with filing targeted during 2027.

• Heparan sulfate is expected to anchor a filing; neurofilament will also be evaluated as a biomarker of downstream disease impact.