We use cookies to understand how you use our site and to improve your experience.
This includes personalizing content and advertising.
By pressing "Accept All" or closing out of this banner, you consent to the use of all cookies and similar technologies and the sharing of information they collect with third parties.
You can reject marketing cookies by pressing "Deny Optional," but we still use essential, performance, and functional cookies.
In addition, whether you "Accept All," Deny Optional," click the X or otherwise continue to use the site, you accept our Privacy Policy and Terms of Service, revised from time to time.
You are being directed to ZacksTrade, a division of LBMZ Securities and licensed broker-dealer. ZacksTrade and Zacks.com are separate companies. The web link between the two companies is not a solicitation or offer to invest in a particular security or type of security. ZacksTrade does not endorse or adopt any particular investment strategy, any analyst opinion/rating/report or any approach to evaluating individual securities.
If you wish to go to ZacksTrade, click OK. If you do not, click Cancel.
DNLI's BLA for Hunter Syndrome Drug Gets FDA's Priority Review
Read MoreHide Full Article
Key Takeaways
FDA accepted DNLI's BLA for tividenofusp alfa in Hunter syndrome, with Priority Review granted.
Tividenofusp alfa uses DNLI's TV platform to address both cognitive and physical symptoms of MPS II.
A potential approval would be DNLI's first, significantly advancing its move to a commercial biotech.
Denali Therapeutics, Inc. (DNLI - Free Report) announced that the FDA has accepted for review its biologics license application (BLA) for lead-pipeline candidate tividenofusp alfa.
The BLA is seeking accelerated approval of the candidate for the treatment of Hunter syndrome (mucopolysaccharidoses type II, or MPS II), a rare and progressive genetic disorder. The regulatory body has granted Priority Review to the application and has set a target action date of Jan. 5, 2026.
The FDA acceptance of the BLA is a significant boost to DNLI’s path toward becoming a commercial-stage biotech company.
Shares of DNLI have lost 30.1% year to date compared with the industry’s 0.9% decline.
Image Source: Zacks Investment Research
More on DNLI’s Hunter Syndrome Drug
In January 2025, the FDA granted Breakthrough Therapy Designation for tividenofusp alfa for the treatment of individuals with Hunter syndrome.
Hunter syndrome is a debilitating and rare genetic disorder caused by a deficiency in the iduronate 2-sulfatase (IDS) enzyme. The human body needs this enzyme to break down complex sugars called glycosaminoglycans, which build up in the brain and body, starting at a young age.
While enzyme replacement therapies (ERTs) have been available for years, they are unable to cross the blood-brain barrier, leaving the central nervous system (CNS) manifestations, such as cognitive decline and behavioral changes, untreated.
Built using Denali’s proprietary TransportVehicle (TV) platform, tividenofusp alfa is designed to deliver the IDS enzyme into both the brain and peripheral tissues, thereby addressing both cognitive and physical symptoms.
The BLA submission is supported by data from the open-label, single-arm phase I/II study of tividenofusp alfa in 47 participants with Hunter syndrome.
Meanwhile, the ongoing phase II/III COMPASS study, comparing tividenofusp alfa to standard-of-care idursulfase, is expected to generate pivotal data to support global regulatory filings.
Denali currently has no approved drug in its portfolio, and hence, a potential approval of tividenofusp alfa would be a significant boost.
DNLI Makes Encouraging Pipeline Progress
Denali is evaluating DNL126 for the treatment of Sanfilippo syndrome type A (MPS IIIA). DNLI earlier announced productive collaboration and discussions with the FDA under the START program (Support for Clinical Trials Advancing Rare Disease Therapeutics) around the potential for an accelerated development and approval path for DNL126 in the treatment of Sanfilippo syndrome Type A.
Another candidate in DNLI’s pipeline is TAK-594/DNL593. Denali has collaborated with Takeda for the co-development and co-commercialization of DNL593, a therapeutic candidate engineered for the delivery of progranulin (PGRN) across the blood-brain barrier and into lysosomes for the treatment of frontotemporal dementia (FTD) associated with a mutation in the granulin (GRN) gene. Denali is conducting the ongoing phase I/II study of DNL593 in FTD-GRN.
Denali and Biogen (BIIB - Free Report) are jointly evaluating an LRRK2 inhibitor, BIIB122/DNL151, in development to treat Parkinson’s disease (PD).
Biogen is leading the global phase IIb LUMA study, evaluating BIIB122's impact on disease progression in early-stage PD. Biogen recently announced that the LUMA study was fully enrolled, with a readout expected in 2026.
Meanwhile, Denali is conducting the phase IIa BEACON study, specifically enrolling participants with LRRK2-associated PD to assess how LRRK2 inhibition may impact this disease. Dosing is currently ongoing.
DNL343, an eIF2B activator, is being evaluated in a phase II/III HEALEY study to treat amyotrophic lateral sclerosis (ALS).
However, in January 2025, Denali announced that the primary endpoint was not met in the HEALEY ALS platform trial.
Denali and partner Sanofi (SNY - Free Report) were co-developing SAR443820/DNL788. We remind investors that Sanofi had earlier discontinued the development of SAR443820/DNL788 for the treatment of ALS based on the results of the phase II HIMALAYA study, which did not meet the primary endpoint.
Denali also has multiple early-stage clinical and preclinical programs in its pipeline.
The company’s sound-cash position is a positive and ensures its ability to fund ongoing programs.
Image: Bigstock
DNLI's BLA for Hunter Syndrome Drug Gets FDA's Priority Review
Key Takeaways
Denali Therapeutics, Inc. (DNLI - Free Report) announced that the FDA has accepted for review its biologics license application (BLA) for lead-pipeline candidate tividenofusp alfa.
The BLA is seeking accelerated approval of the candidate for the treatment of Hunter syndrome (mucopolysaccharidoses type II, or MPS II), a rare and progressive genetic disorder. The regulatory body has granted Priority Review to the application and has set a target action date of Jan. 5, 2026.
The FDA acceptance of the BLA is a significant boost to DNLI’s path toward becoming a commercial-stage biotech company.
Shares of DNLI have lost 30.1% year to date compared with the industry’s 0.9% decline.
Image Source: Zacks Investment Research
More on DNLI’s Hunter Syndrome Drug
In January 2025, the FDA granted Breakthrough Therapy Designation for tividenofusp alfa for the treatment of individuals with Hunter syndrome.
Hunter syndrome is a debilitating and rare genetic disorder caused by a deficiency in the iduronate 2-sulfatase (IDS) enzyme. The human body needs this enzyme to break down complex sugars called glycosaminoglycans, which build up in the brain and body, starting at a young age.
While enzyme replacement therapies (ERTs) have been available for years, they are unable to cross the blood-brain barrier, leaving the central nervous system (CNS) manifestations, such as cognitive decline and behavioral changes, untreated.
Built using Denali’s proprietary TransportVehicle (TV) platform, tividenofusp alfa is designed to deliver the IDS enzyme into both the brain and peripheral tissues, thereby addressing both cognitive and physical symptoms.
The BLA submission is supported by data from the open-label, single-arm phase I/II study of tividenofusp alfa in 47 participants with Hunter syndrome.
Meanwhile, the ongoing phase II/III COMPASS study, comparing tividenofusp alfa to standard-of-care idursulfase, is expected to generate pivotal data to support global regulatory filings.
Denali currently has no approved drug in its portfolio, and hence, a potential approval of tividenofusp alfa would be a significant boost.
DNLI Makes Encouraging Pipeline Progress
Denali is evaluating DNL126 for the treatment of Sanfilippo syndrome type A (MPS IIIA). DNLI earlier announced productive collaboration and discussions with the FDA under the START program (Support for Clinical Trials Advancing Rare Disease Therapeutics) around the potential for an accelerated development and approval path for DNL126 in the treatment of Sanfilippo syndrome Type A.
Another candidate in DNLI’s pipeline is TAK-594/DNL593. Denali has collaborated with Takeda for the co-development and co-commercialization of DNL593, a therapeutic candidate engineered for the delivery of progranulin (PGRN) across the blood-brain barrier and into lysosomes for the treatment of frontotemporal dementia (FTD) associated with a mutation in the granulin (GRN) gene. Denali is conducting the ongoing phase I/II study of DNL593 in FTD-GRN.
Denali and Biogen (BIIB - Free Report) are jointly evaluating an LRRK2 inhibitor, BIIB122/DNL151, in development to treat Parkinson’s disease (PD).
Biogen is leading the global phase IIb LUMA study, evaluating BIIB122's impact on disease progression in early-stage PD. Biogen recently announced that the LUMA study was fully enrolled, with a readout expected in 2026.
Meanwhile, Denali is conducting the phase IIa BEACON study, specifically enrolling participants with LRRK2-associated PD to assess how LRRK2 inhibition may impact this disease. Dosing is currently ongoing.
DNL343, an eIF2B activator, is being evaluated in a phase II/III HEALEY study to treat amyotrophic lateral sclerosis (ALS).
However, in January 2025, Denali announced that the primary endpoint was not met in the HEALEY ALS platform trial.
Denali and partner Sanofi (SNY - Free Report) were co-developing SAR443820/DNL788. We remind investors that Sanofi had earlier discontinued the development of SAR443820/DNL788 for the treatment of ALS based on the results of the phase II HIMALAYA study, which did not meet the primary endpoint.
Denali also has multiple early-stage clinical and preclinical programs in its pipeline.
The company’s sound-cash position is a positive and ensures its ability to fund ongoing programs.
Zacks Rank
Denali currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.