FDA grants accelerated approval of AVLAYAH for Hunter syndrome (MPS II)
The FDA approved Denali’s AVLAYAH (tividenofusp alfa-eknm) for Hunter syndrome (MPS II) on Mar 25, 2026. The company can now sell it in that market.
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- Company
- Denali Therapeutics Inc. DNLI
- Drug
- AVLAYAH (tividenofusp alfa-eknm)
- For
- Hunter syndrome (MPS II)
- Stage
- Phase 1/2 · an early study that checks safety and takes a first look at effect
- When
- Mar 25, 2026
- Source note
- Denali's tividenofusp alfa-eknm became the first FDA-approved brain-penetrant enzyme replacement therapy for neurologic manifestations of MPS II, cleared on a CSF heparan sulfate surrogate endpoint (91% reduction by week 24). A rare pediatric disease priority review voucher came with the approval.
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Denali expects to report results from its Phase 1/2 study of DNL593 (PTV:PGRN) for frontotemporal dementia with granulin mutations in the second half of 2026 (no exact date yet). Phase 1/2 is an early study that checks safety and takes a first look at effect.
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- Entry
- DNL593 Phase 1/2 results in FTD-GRN · Clinical trial results (readout / topline data)
- Drug
- DNL593 (PTV:PGRN)
- For
- frontotemporal dementia with granulin mutations
- Stage
- Phase 1/2 · an early study that checks safety and takes a first look at effect
- When
- H2 2026 · the company gave a window, not a day
- Source note
- Company guidance (Q1 2026 PR): "results are expected by the end of 2026." Note: Takeda terminated the DNL593 co-development collaboration in April 2026.
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- ClinicalTrials.gov ↗ — trials of this drug
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Denali expects to report results from its Phase 2a study of DNL151 (BIIB122, LRRK2 inhibitor) for Parkinson's disease in pathogenic LRRK2 variant carriers in the first half of 2027 (no exact date yet). This is the first look at whether the drug did what the company hoped.
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- Entry
- BEACON Phase 2a data for DNL151 in LRRK2-variant Parkinson's disease · Clinical trial results (readout / topline data)
- Drug
- DNL151 (BIIB122, LRRK2 inhibitor)
- For
- Parkinson's disease in pathogenic LRRK2 variant carriers
- Stage
- Phase 2a
- When
- H1 2027 · the company gave a window, not a day
- Source note
- Denali continues BEACON independently after the Phase 2b LUMA miss in idiopathic PD (May 2026); BEACON data expected in 1H 2027 per Q2 2026 update.
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Denali expects to report results from its Early-stage clinical study of DNL628 for Alzheimer's disease in the first half of 2027 (no exact date yet). This is the first look at whether the drug did what the company hoped.
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- Entry
- DNL628 Alzheimer's disease study data · Clinical trial results (readout / topline data)
- Drug
- DNL628
- For
- Alzheimer's disease
- Stage
- Early-stage clinical
- When
- H1 2027 · the company gave a window, not a day
- Source note
- Company guidance: "Denali expects data from this study in 1H 2027."
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DNL126 BLA submission and potential accelerated approval in Sanfilippo syndrome type A, expected during 2027 (no exact date yet). A company milestone for Denali (DNL126 (ETV:SGSH) for Sanfilippo syndrome type A (MPS IIIA)): a step in the program rather than a decision or a data readout.
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- Entry
- DNL126 BLA submission and potential accelerated approval in Sanfilippo syndrome type A · Company milestone
- Drug
- DNL126 (ETV:SGSH)
- For
- Sanfilippo syndrome type A (MPS IIIA)
- Stage
- Phase 1/2 · an early study that checks safety and takes a first look at effect
- When
- 2027 · the company gave a window, not a day
- Source note
- Company guidance: "Denali expects a Biologics License Application (BLA) submission and potential accelerated approval for DNL126 for Sanfilippo syndrome type A in 2027" (year-level guidance only).
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- ClinicalTrials.gov ↗ — trials of this drug
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Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page
Denali expects to report results from its Phase 1/2 study of DNL593 (PTV:PGRN) for frontotemporal dementia-GRN during 2027 (no exact date yet). Phase 1/2 is an early study that checks safety and takes a first look at effect.
More details
- Entry
- DNL593 FTD-GRN Phase 1/2 results pushed to 2027 · Clinical trial results (readout / topline data)
- Drug
- DNL593 (PTV:PGRN)
- For
- frontotemporal dementia-GRN
- Stage
- Phase 1/2 · an early study that checks safety and takes a first look at effect
- When
- 2027 · the company gave a window, not a day
- Source note
- changed date for existing DNLI|readout|2026-H2 DNL593 item. Q2 2026 PR (Aug 6, 2026) moves expected DNL593 FTD-GRN results from end of 2026 to 2027.
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Denali expects to report results from its Phase 1/2 study of DNL952 (ETV:GAA) for Pompe disease during 2027 (no exact date yet). Phase 1/2 is an early study that checks safety and takes a first look at effect.
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- Entry
- DNL952 ETV:GAA Pompe disease initial clinical data · Clinical trial results (readout / topline data)
- Drug
- DNL952 (ETV:GAA)
- For
- Pompe disease
- Stage
- Phase 1/2 · an early study that checks safety and takes a first look at effect
- When
- 2027 · the company gave a window, not a day
- Source note
- Q2 2026 PR (Aug 6, 2026): initial clinical data from the DNL952 Pompe program expected in 2027.
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- Source: biospace.com ↗ — where this entry was taken from
- SEC filings (8-K) ↗ — press releases are attached to 8-K filings
- ClinicalTrials.gov ↗ — trials of this drug
- DNLI headlines on this site
Plain-English summary generated from this entry’s own fields (drug, condition, stage, date). Glossary · Open this catalyst’s page