This section highlights FDA-related milestones and regulatory updates for drugs developed by Denali Therapeutics (DNLI).
Over the past two years, Denali Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
BEACON, DNL126, DNL310, DNL343, and tividenofusp. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
BEACON - FDA Regulatory Timeline and Events
BEACON is a drug developed by Denali Therapeutics for the following indication: For Parkinson's Disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- BEACON
- Announced Date:
- December 5, 2024
- Indication:
- For Parkinson's Disease
Announcement
Denali Therapeutics Inc announced initiation of dosing in a global Phase 2a clinical study, BEACON, of the investigational drug leucine-rich repeat kinase 2 (LRRK2) inhibitor BIIB122 (DNL151) in participants with LRRK2-associated Parkinson's disease (LRRK2-PD).
AI Summary
Denali Therapeutics has begun dosing in its global Phase 2a clinical study, BEACON, to evaluate the investigational LRRK2 inhibitor BIIB122 (DNL151) in patients with LRRK2-associated Parkinson’s disease. This study will involve approximately 50 participants who have confirmed LRRK2 mutations through genetic testing. The trial is designed with a three‐month double-blind treatment period, followed by an open label extension, to assess the safety, tolerability, and biomarker responses to daily oral dosing of BIIB122.
The study aims to explore whether targeting LRRK2 can slow the progression of Parkinson’s disease by addressing underlying lysosomal dysfunction. Denali holds the Investigational New Drug application for this trial and is leading its design and execution in collaboration with a third-party partner, marking an important step in the continued search for disease-modifying therapies for Parkinson’s patients with genetic risk factors.
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DNL126 - FDA Regulatory Timeline and Events
DNL126 is a drug developed by Denali Therapeutics for the following indication: For MPS IIIA (Sanfilippo Syndrome Type A).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- DNL126
- Announced Date:
- January 13, 2025
- Indication:
- For MPS IIIA (Sanfilippo Syndrome Type A)
Announcement
Denali Therapeutics Inc. announced key anticipated milestones for 2025 across its portfolio. Chief Executive Officer, Ryan Watts, Ph.D., will highlight these priorities during a corporate presentation at the 43rd Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, at 11:15 a.m. PDT.
AI Summary
Denali Therapeutics Inc. has outlined its key milestones for 2025, focusing on advancing its portfolio of therapies. One major highlight includes the submission of a biologics license application (BLA) for tividenofusp alfa for Hunter syndrome on an accelerated approval pathway in early 2025, with preparations for a U.S. commercial launch planned for late 2025 or early 2026. The company is also pushing forward with efforts to align with the FDA for accelerated approval of its second program, DNL126, targeting Sanfilippo syndrome Type A. These programs are central to Denali’s strategy to build a broad franchise of TransportVehicleTM enabled enzyme replacement therapies. Chief Executive Officer Ryan Watts, Ph.D., will discuss these priorities and other anticipated milestones during a corporate presentation at the 43rd Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, at 11:15 a.m. PDT.
Read Announcement- Drug:
- DNL126
- Announced Date:
- June 3, 2024
- Indication:
- For MPS IIIA (Sanfilippo Syndrome Type A)
Announcement
Denali Therapeutics Inc announced that the U.S. Food and Drug Administration (FDA) has selected DNL126 for participation in the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program.
AI Summary
The FDA has selected Denali Therapeutics Inc.’s investigational therapy DNL126 to participate in the START Pilot Program, a new initiative designed to speed up the development of treatments for rare diseases. DNL126 is an enzyme replacement therapy engineered to cross the blood-brain barrier and is being studied for its potential to treat MPS IIIA, also known as Sanfilippo syndrome type A. This selection is based on the drug’s promise for clinical benefit and Denali’s capacity to advance the therapy toward a marketing application. Being part of the START Pilot Program will give Denali access to more frequent and flexible communications with FDA review staff, which is expected to help overcome development challenges and accelerate the overall process. This step underscores both Denali’s commitment to finding treatments for rare neurodegenerative diseases and the FDA’s support for innovative drug development.Read Announcement
DNL310 - FDA Regulatory Timeline and Events
DNL310 is a drug developed by Denali Therapeutics for the following indication: Gene therapy that reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the deficient protein in hemophilia B.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- DNL310
- Announced Date:
- February 6, 2025
- Indication:
- Gene therapy that reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the deficient protein in hemophilia B.
Announcement
Denali Therapeutics Inc
AI Summary
Denali Therapeutics Inc. recently shared encouraging long-term results from their Phase 1/2 study in 47 patients with Hunter syndrome (MPS II). The study found that treatment with tividenofusp alfa led to significant and long-lasting reductions in key biomarkers, with many patients showing improvements in hearing, cognition, and adaptive behavior. These benefits were maintained over a median follow-up period of two years and even up to four years in some cases. The data supports Denali's plan to seek accelerated approval, with a biologics license application expected in early 2025 and a planned U.S. launch for late 2025 or early 2026. Denali is working hard to provide this promising treatment to families affected by MPS II, and the positive results not only highlight progress in addressing both neurocognitive and physical symptoms but also pave the way for further advancements in their lysosomal storage disease programs.
Read Announcement- Drug:
- DNL310
- Announced Date:
- September 3, 2024
- Indication:
- Gene therapy that reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the deficient protein in hemophilia B.
Announcement
Denali Therapeutics Inc announced the outcome of a recent successful meeting with the Center for Drug Evaluation and Research (CDER) division of the U.S. Food and Drug Administration (FDA) providing a path to filing a biologics license application (BLA) for accelerated approval and subsequent conversion to full approval for tividenofusp alfa (DNL310) for the treatment of MPS II (Hunter syndrome).
AI Summary
Denali Therapeutics announced a positive outcome from a recent meeting with the FDA’s Center for Drug Evaluation and Research (CDER). During this meeting, the FDA provided clear guidance on filing a biologics license application (BLA) for tividenofusp alfa (DNL310) under the accelerated approval pathway for treating MPS II (Hunter syndrome). The discussions focused on using cerebrospinal fluid heparan sulfate (CSF HS) as a surrogate biomarker that is reasonably likely to predict clinical benefit. This supportive feedback paves the way for Denali to submit the BLA in early 2025, aiming for accelerated approval with plans to convert to full approval after additional clinical validation. This positive development marks an important step toward addressing the unmet needs of patients with MPS II and reflects collaborative efforts among the drug developers, clinicians, and the broader patient community.
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DNL343 - FDA Regulatory Timeline and Events
DNL343 is a drug developed by Denali Therapeutics for the following indication: Amyotrophic lateral sclerosis (ALS).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- DNL343
- Announced Date:
- March 6, 2025
- Indication:
- Amyotrophic lateral sclerosis (ALS)
Announcement
Denali Therapeutics Inc. provided an update that further analyses from Regimen G of the Phase 2/3 HEALEY ALS Platform Trial evaluating eIF2B agonist DNL343 in the treatment of amyotrophic lateral sclerosis (ALS) did not demonstrate a treatment effect on neurofilament light (NfL), a biomarker of neuronal damage, over the 24-week, double-blind period and in a subset of participants that completed an additional 28 weeks in the open-label active treatment extension.
Read Announcement- Drug:
- DNL343
- Announced Date:
- January 6, 2025
- Indication:
- Amyotrophic lateral sclerosis (ALS)
Announcement
Denali Therapeutics Inc announced topline results from an analysis of Regimen G of the Phase 2/3 HEALEY ALS Platform Trial evaluating eIF2B agonist DNL343 in the treatment of amyotrophic lateral sclerosis (ALS).
AI Summary
Denali Therapeutics Inc. announced topline results from Regimen G of the Phase 2/3 HEALEY ALS Platform Trial, which evaluated the eIF2B agonist DNL343 for treating amyotrophic lateral sclerosis (ALS). The study did not meet its primary endpoint of slowing disease progression, as measured by changes in the ALS Functional Rating Scale-Revised (ALSFRS-R) and overall survival at 24 weeks. Additionally, key secondary endpoints such as measures of muscle strength and respiratory function showed no significant difference compared to placebo.
Despite these outcomes, DNL343 was found to be safe and well tolerated by study participants. Denali plans to share further analysis later in 2025, including data on neurofilament light (NfL) and other biomarkers, along with additional subgroup and extended treatment findings, to better understand the therapeutic potential of DNL343 in addressing ALS.
Read Announcement- Drug:
- DNL343
- Announced Date:
- January 6, 2025
- Indication:
- Amyotrophic lateral sclerosis (ALS)
Announcement
Denali Therapeutics Inc announced topline results from an analysis of Regimen G of the Phase 2/3 HEALEY ALS Platform Trial evaluating eIF2B agonist DNL343 in the treatment of amyotrophic lateral sclerosis (ALS).
AI Summary
Denali Therapeutics Inc. announced topline results from Regimen G of the Phase 2/3 HEALEY ALS Platform Trial, which evaluated the eIF2B agonist DNL343 for the treatment of amyotrophic lateral sclerosis (ALS). The study compared 186 patients treated with DNL343 to 139 patients on placebo. Results showed that DNL343 did not slow disease progression over 24 weeks, as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) and survival, and it did not improve muscle strength or respiratory function compared to the placebo group.
Despite the lack of efficacy in these primary and key secondary endpoints, DNL343 was found to be safe and well tolerated. Additional analysis, including tests of neurofilament light and other biomarkers, as well as subgroup findings and long-term data from an extension period, are expected to be released later in 2025.
Read Announcement
tividenofusp - FDA Regulatory Timeline and Events
tividenofusp is a drug developed by Denali Therapeutics for the following indication: For the treatment of Hunter syndrome (MPS II).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- tividenofusp
- Announced Date:
- July 7, 2025
- Estimated Event Date Range:
- January 5, 2026 - January 5, 2026
- Target Action Date:
- January 5, 2026
- Indication:
- For the treatment of Hunter syndrome (MPS II)
Announcement
Denali Therapeutics Inc. announced that The FDA granted the BLA Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of January 5, 2026.
AI Summary
Denali Therapeutics Inc. recently announced significant progress in its development of tividenofusp alfa for treating Hunter syndrome. The U.S. Food and Drug Administration (FDA) granted Priority Review for the Biologics License Application (BLA) under the Prescription Drug User Fee Act (PDUFA). This review comes with a target action date of January 5, 2026, for the agency’s decision.
The FDA’s Priority Review designation highlights the potential impact of tividenofusp alfa as a new treatment option for a rare genetic disorder that affects both cognitive and physical functions. With this milestone, Denali is preparing for a possible commercial launch in the United States, marking an important step forward in addressing an unmet medical need for patients with Hunter syndrome.
Read Announcement- Drug:
- tividenofusp
- Announced Date:
- July 7, 2025
- Indication:
- For the treatment of Hunter syndrome (MPS II)
Announcement
Denali Therapeutics Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) seeking accelerated approval for tividenofusp alfa for the treatment of Hunter syndrome (mucopolysaccharidoses type II, or MPS II), a rare and progressive genetic disorder.
AI Summary
Denali Therapeutics Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for tividenofusp alfa for review. This application is seeking accelerated approval to treat Hunter syndrome (mucopolysaccharidoses type II, or MPS II), a rare and progressive genetic disorder caused by a deficiency in the iduronate 2-sulfatase enzyme. The investigational therapy is designed to deliver the missing enzyme throughout the body and across the blood-brain barrier, addressing both physical symptoms and the neurological aspects of the disease. If approved, tividenofusp alfa could become the first significant advancement in enzyme replacement therapy for Hunter syndrome in nearly two decades, providing a potentially transformative option for patients affected by this challenging condition.
Read Announcement- Drug:
- tividenofusp
- Announced Date:
- April 2, 2025
- Indication:
- For the treatment of Hunter syndrome (MPS II)
Announcement
Denali Therapeutics Inc announced that the company's initiation of a rolling submission of a biologics license application (BLA) for accelerated approval of tividenofusp alfa for the treatment of Hunter syndrome (MPS II) has been received by the Center for Drug Evaluation and Research (CDER) of the U.S. Food and Drug Administration (FDA).
AI Summary
Denali Therapeutics Inc. announced that the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research has received its rolling submission for a biologics license application. This submission is for accelerated approval of tividenofusp alfa, a new treatment aimed at patients with Hunter syndrome (MPS II). The application includes data that uses cerebrospinal fluid heparan sulfate as a surrogate endpoint, which may help speed up the approval process.
The company has been working closely with the FDA and is aligned on the data package needed for both accelerated and full approval. Denali expects to complete its BLA submission by the first half of May 2025 and is preparing for a potential U.S. commercial launch in late 2025 or early 2026. This milestone brings Denali one step closer to providing a new treatment option for the Hunter syndrome community.
Read Announcement
