Company: DNLI
Filing Date: 2025-02-27
Form Type: 10-K
Source: 0001714899-25-000066
Chunk: 122

Company: Denali Therapeutics Inc.
Filing Date: 2025-02-27
Form: 10-K
Item: Item 1
Chunk 122
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 (dB eHL) and any available standard hearing or behavioral audiometry test (in dB HL) are pooled. ABR is prioritized if more than one test is available within the same participant and analysis visit. ABR, auditory brainstem response; BL, baseline; CFB, change from baseline; dB, decibels; eHL, estimated hearing level; HL, hearing level.

Long-term data demonstrated continued improvements from baseline in cognition and adaptive behavior (Figure 11). 

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Figure 11: Cognition and Adaptive Behavior. aBased on MMRM model with fixed effects for visit week, age group at tividenofusp alfa initiation, genotype, and baseline BSID-IIII cognitive raw or Vineland-3 adaptive behavior raw composite score. Only visits with number of observation ≥ 5 were included in the statistical model. bAdaptive behavior raw composite is the derived sum of raw scores of eight subdomains (receptive, expressive, personal, interpersonal relationships, play and leisure, coping skills, fine motor, and gross motor). To evaluate change in adaptive behavior across both instruments, scores were converted from VABS-II to Vineland-3 using an errors-in-variables linear regression model adjusted by age. BL, baseline; BSID-III, Bayley Scales of Infant and Toddler Development, third edition; CFB, change from baseline; CI, confidence interval; MMRM, mixed model for repeated measures; VABS-II, Vineland Adaptive Behavior Scales, second edition; Vineland-3, Vineland Adaptive Behavior Scales, third edition; W, week.

Administration of tividenofusp alfa was generally well tolerated in study participants. Most treatment-related adverse events ("TEAEs") were mild or moderate, including IRRs, anemia, vomiting, pyrexia, respiratory infections and rash. Serious TEAEs in three participants (6.4%) were manageable, with resolution or stabilization with continued treatment. One participant discontinued treatment for reasons related to a moderate IRR and other non-treatment-related adverse events.

2025 expected progress and milestones:

•Submit BLA for tividenofusp alfa in MPS II under the accelerated approval pathway in early 2025

•U.S. commercial launch in late 2025 or early 2026

DNL126 (ETV:SGSH) Program for MPS IIIA (Sanfilippo Syndrome A)

MPS III, also called Sanfilippo syndrome, is a rare, genetic