DNL126
- Generic Name
- DNL126
Denali Therapeutics' Tividenofusp Alfa Receives FDA Breakthrough Therapy Designation for Hunter Syndrome
• Denali Therapeutics' tividenofusp alfa (DNL310) has received Breakthrough Therapy Designation from the FDA for Hunter syndrome (MPS II).
• The designation aims to expedite the development and review of tividenofusp alfa, a potential improvement over existing therapies.
• Denali plans to submit a Biologics License Application (BLA) in early 2025 for accelerated approval of tividenofusp alfa.
• Tividenofusp alfa is designed to address both neurological and physical symptoms of Hunter syndrome by crossing the blood-brain barrier.
Denali Therapeutics' DNL343 Fails to Meet Primary Endpoint in HEALEY ALS Platform Trial
• Denali Therapeutics' DNL343, an eIF2B agonist, did not meet the primary endpoint of slowing ALS disease progression in a Phase 2/3 trial.
• The HEALEY ALS Platform Trial's Regimen G assessed DNL343's impact on disease severity and survival over 24 weeks compared to placebo.
• While DNL343 was safe and well-tolerated, key secondary endpoints like muscle strength and respiratory function showed no significant difference.
• Further analyses, including biomarker assessments, are planned for 2025 to explore potential subgroup benefits and long-term effects.
Denali Therapeutics Announces Positive Clinical Updates and Financial Results for Q1 2024
• Denali Therapeutics reported positive two-year clinical data for tividenofusp alfa in MPS II, showing sustained normalization of heparan sulfate in CSF and improvements in clinical outcomes.
• The company initiated a Phase 1/2 clinical trial for DNL126, an enzyme replacement therapy for children with MPS IIIA, with biomarker and safety data expected by the end of 2024.
• Enrollment is complete in the Phase 2/3 HEALEY ALS Platform Trial for DNL343, an eIF2B activator being developed for the treatment of amyotrophic lateral sclerosis (ALS).
• Denali completed a private investment in public equity (PIPE) financing with gross proceeds of $500 million and divested its preclinical small molecule portfolio.
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