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SOUTH SAN FRANCISCO - The U.S. Food and Drug Administration has accepted Denali Therapeutics Inc.’s (NASDAQ:DNLI) Biologics License Application for tividenofusp alfa with Priority Review, setting a target action date of January 5, 2026, the company announced Monday. The $2.1 billion market cap biotech company, which according to InvestingPro maintains a "FAIR" overall financial health score, has seen 5 analysts revise their earnings expectations upward for the upcoming period.
The application seeks accelerated approval for tividenofusp alfa to treat Hunter syndrome, a rare genetic disorder caused by a deficiency in the iduronate 2-sulfatase enzyme. With the company’s next earnings report scheduled for July 31, investors following this regulatory milestone can access detailed financial analysis and 8 additional key insights through InvestingPro.
Tividenofusp alfa is designed to deliver the missing enzyme across the blood-brain barrier, potentially addressing both neurological manifestations and physical symptoms of the disease. Current therapies do not cross this barrier and cannot address cognitive impacts.
"If FDA-approved, tividenofusp alfa would mark the first significant advancement in nearly two decades for enzyme replacement therapy for individuals living with Hunter syndrome," said Carole Ho, Chief Medical Officer at Denali Therapeutics, in the press release.
The application is supported by data from an open-label, single-arm Phase 1/2 study involving 47 participants with Hunter syndrome. Denali is conducting an ongoing Phase 2/3 COMPASS study to support global regulatory approvals.
The FDA previously granted Fast Track and Breakthrough Therapy designations to tividenofusp alfa, while the European Medicines Agency has given it Priority Medicines designation.
Hunter syndrome is a progressive disorder that causes developmental delays, cognitive decline, behavioral abnormalities, and physical complications including joint stiffness, hearing loss, and organ dysfunction.
Tividenofusp alfa utilizes Denali’s proprietary TransportVehicle platform, which is designed to deliver large therapeutic molecules across the blood-brain barrier. The company maintains a strong balance sheet with more cash than debt, though InvestingPro data indicates it is currently burning through cash reserves - a common characteristic of biotech companies in late-stage development.
In other recent news, Denali Therapeutics has been the focus of analyst discussions and strategic developments. Stifel has maintained its Buy rating for Denali Therapeutics, setting a price target of $37.00. This decision follows insights gained from meetings with Denali’s executive team, where the company expressed confidence in its regulatory processes, particularly for its DNL310 and DNL126 products. On the other hand, H.C. Wainwright adjusted its price target for Denali from $80.00 to $32.00, though it also maintained a Buy rating. This adjustment was influenced by Denali’s recent completion of its Biologic License Application for tividenofusp alfa, with a potential launch anticipated in late 2025 or early 2026.
Denali’s leadership remains optimistic about its platform and pipeline, which includes promising programs targeting diseases such as Pompe and Alzheimer’s. The company is also exploring strategic partnerships to manage cash expenditures effectively. Despite the challenges in the rare disease market, analysts believe Denali holds several strategic advantages, such as dynamic pricing strategies and risk-sharing agreements. These strategies are expected to support the successful launch and adoption of their products. The continued Buy ratings from both Stifel and H.C. Wainwright reflect confidence in Denali Therapeutics’ long-term potential.
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