Denali Therapeutics Inc. DNLI
Denali Therapeutics Inc. (Nasdaq: DNLI) is a rare genetic drug developer. Its lead program, AVLAYAH (tividenofusp alfa-eknm; DNL310), is in Phase 2 for neurologic manifestations of Hunter syndrome (MPS II).
Upcoming catalysts
No catalyst is dated to the next 30 days or to an FDA action date.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| AVLAYAH (tividenofusp alfa-eknm) | Hunter syndrome (MPS II) | protein/peptide | Approved |
| DNL151 (LRRK2 inhibitor) | Parkinson's disease, LRRK2 variant carriers (BEACON Phase 2a) | small molecule | Phase 2 |
| zafinofusp alfa (DNL126) | Sanfilippo syndrome type A (MPS IIIA) | protein/peptide | Phase 1 |
| DNL593 (PTV:PGRN) | FTD-GRN | protein/peptide | Phase 1 |
| DNL952 (ETV:GAA) | Pompe disease | protein/peptide | Phase 1 |
| DNL628 (OTV:MAPT) | Alzheimer's disease | RNA | Phase 1 |
| DNL921 (ATV:Abeta) | Alzheimer's disease | antibody | Phase 1 |
Recent results and decisions
- May 21, 2026missed Phase 2b LUMA (n=648) missed primary (time to confirmed worsening, MDS-UPDRS II+III) and secondary endpoints; development in idiopathic PD discontinued.
The Phase 2b LUMA study of BIIB122 in early-stage Parkinson’s disease did not meet its primary or secondary endpoints
Filing - Mar 25, 2026approval FDA granted accelerated approval to AVLAYAH based on a 91% reduction in CSF heparan sulfate by week 24 (93% of patients within normal range); rare pediatric disease PRV awarded.
Denali Therapeutics Inc. (Nasdaq: DNLI) today announced the U.S. Food and Drug Administration (FDA) has granted accelerated approval for AVLAYAH™ (tividenofusp alfa-eknm), the first FDA-approved biologic specifically designed to cross the blood-brain barrier and reach the whole body, including the b
Filing - Feb 26, 2026positive Preliminary Phase 1/2 data (WORLDSymposium, Feb 2026) showed substantial reductions in CSF heparan sulfate and GM3 and urinary heparan sulfate, with safety consistent with established ERTs.
In February 2026, Denali presented preliminary Phase 1/2 data at WORLDSymposium demonstrating treatment with DNL126 resulted in substantial reductions in disease biomarkers in cerebrospinal fluid (CSF heparan sulfate and GM3) and the periphery (urinary heparan sulfate) with a safety profile generall
Filing
3 more on record.
For subscribers
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Other phase 2 rare genetic companies
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