Denali Therapeutics

Denali Therapeutics' Tividenofusp Alfa Receives FDA Breakthrough Therapy Designation for Hunter Syndrome

4 months ago

• 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' ALS Trial Fails to Meet Endpoints, BofA Adjusts Stock Target

5 months ago

• Denali Therapeutics' DNL343, an eIF2B inhibitor, failed to meet primary and key secondary endpoints in a Phase 2/3 trial for amyotrophic lateral sclerosis (ALS) at 24 weeks. • BofA Securities reduced Denali's stock target to $30 from $34, removing DNL343 from their valuation model due to the uncertainty of the drug's future development. • Despite the trial setback, BofA remains optimistic about Denali's brain delivery platform and strong financial position, suggesting potential buying opportunities. • Denali anticipates submitting a BLA for tividenofusp alfa, a Hunter syndrome treatment, in early 2025, potentially transitioning to a commercial organization later that year.

Denali Therapeutics' DNL343 Fails to Meet Primary Endpoint in HEALEY ALS Platform Trial

5 months ago

• 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 Initiates Phase 2a Trial of BIIB122 for LRRK2-Associated Parkinson's Disease

6 months ago

• Denali Therapeutics has dosed the first participant in its Phase 2a BEACON study of BIIB122, a LRRK2 inhibitor, for LRRK2-associated Parkinson's disease. • The BEACON study will assess the safety and biomarker effects of BIIB122 in approximately 50 participants with LRRK2-related Parkinson's. • BIIB122 is also under investigation in the Phase 2b LUMA study for early-stage Parkinson's, with or without LRRK2 mutation, in collaboration with Biogen. • LRRK2 inhibition targets lysosomal dysfunction, potentially modifying the course of Parkinson's disease rather than just treating symptoms.

Multiple Sclerosis Pipeline Shows Promise with Novel Therapies in Development

6 months ago

• The multiple sclerosis (MS) therapeutic landscape is expanding, with over 80 active pipeline therapies currently in development by more than 75 companies. • Recent clinical trials have yielded mixed results, with some therapies showing promise in specific MS subtypes, such as non-relapsing secondary progressive MS (nrSPMS). • Regulatory milestones have been achieved, including FDA approval for new formulations and fast-track designations for therapies targeting progressive MS. • Emerging therapies in the MS pipeline include monoclonal antibodies, oral treatments, and CAR-T cell therapies, offering diverse mechanisms of action and routes of administration.

Denali Therapeutics Plans Accelerated Approval Filing for Tividenofusp Alfa in MPS II

9 months ago

• Denali Therapeutics plans to submit a Biologics License Application (BLA) to the FDA in early 2025 for tividenofusp alfa (DNL310) to treat Mucopolysaccharidosis type II (MPS II). • The FDA has indicated that cerebrospinal fluid heparan sulfate (CSF HS) can serve as a surrogate biomarker to support accelerated approval of tividenofusp alfa in MPS II. • Phase 1/2 data presented at SSIEM 2024 showed a 90% mean reduction in CSF HS and improvements in clinical outcomes with tividenofusp alfa treatment in MPS II patients. • Tividenofusp alfa is currently being evaluated in the Phase 2/3 COMPASS study, with plans for full approval contingent upon the study's completion.

Denali Therapeutics Announces Positive Clinical Updates and Financial Results for Q1 2024

a year ago

• 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.

Denali's Hunter Syndrome Candidate Receives FDA Orphan Drug and Rare Pediatric Disease Designations

6 years ago

Denali Therapeutics Inc. announced that its pipeline candidate, DNL310, has been granted orphan drug status and a rare pediatric disease designation by the FDA for the treatment of Hunter Syndrome, a rare lysosomal storage disease. The company plans to initiate a phase I/II study in 2020, following promising pre-clinical results.