Denali Therapeutics advances Hunter syndrome treatment BLA

Published 02/04/2025, 13:10
Denali Therapeutics advances Hunter syndrome treatment BLA

SOUTH SAN FRANCISCO - Denali Therapeutics Inc. (NASDAQ: DNLI), a biopharmaceutical company with a market capitalization of $1.8 billion, has initiated a rolling submission for a biologics license application (BLA) for tividenofusp alfa, a potential treatment for Hunter syndrome, to the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research (CDER). The company is on track to complete the BLA submission by the first half of May 2025 and is preparing for a possible U.S. market launch in late 2025 or early 2026. According to InvestingPro data, the stock has declined nearly 40% year-to-date, currently trading near its 52-week low of $12.05.

Tividenofusp alfa is designed to address both cognitive and physical symptoms of Hunter syndrome, a rare genetic disorder primarily affecting males worldwide. The current standard of care does not alleviate cognitive and behavioral symptoms, as it cannot cross the blood-brain barrier. Denali’s approach involves using a proprietary Enzyme TransportVehicle™ to deliver the enzyme iduronate 2-sulfatase into the brain. While the company maintains a strong balance sheet with more cash than debt and a healthy current ratio of 8.46, InvestingPro analysis indicates rapid cash burn, a critical factor for investors monitoring developmental-stage biotech companies.

The FDA has granted the treatment Fast Track and Breakthrough Therapy designations, while the European Medicines Agency has given it Priority Medicines designation. The ongoing Phase 2/3 COMPASS study enrolls participants across North America, South America, and Europe to support global approval.

Denali’s Chief Medical Officer, Dr. Carole Ho, expressed gratitude towards the FDA for their support in advancing tividenofusp alfa towards potential approval. She emphasized the importance of this milestone in providing a new treatment option for those affected by Hunter syndrome.

Additionally, Denali is collaborating with CDER through the START program for the accelerated development and approval of DNL126 for Sanfilippo syndrome. DNL126 is another investigational therapy currently in a Phase 1/2 study to assess safety and efficacy for the treatment of this rare genetic disease.

Denali Therapeutics, headquartered in South San Francisco, focuses on developing treatments for neurodegenerative and lysosomal storage diseases by ensuring delivery across the blood-brain barrier. The company’s product candidates are investigational and have not yet received health authority approval. Analyst sentiment remains optimistic, with price targets ranging from $24 to $80 per share, suggesting significant upside potential. For deeper insights into Denali’s financial health and 12 additional ProTips, visit InvestingPro.

This article is based on a press release statement from Denali Therapeutics Inc. and contains forward-looking statements that involve risks and uncertainties, including the possibility of delay or failure in obtaining regulatory approval and commercializing the product candidates. No endorsement of claims is implied.

In other recent news, Denali Therapeutics Inc. has been the focus of several analyst assessments and regulatory developments. Morgan Stanley initiated coverage on Denali with an Overweight rating, highlighting confidence in the company’s enzyme transport vehicle (ETV) platform and setting a price target of $33.00. Meanwhile, H.C. Wainwright adjusted its price target for Denali to $80.00, down from $87.00, while maintaining a Buy rating, citing operational expenses and market penetration projections for DNL310. Deutsche Bank also began coverage with a Buy rating, setting a $31.00 price target, citing Denali’s extensive pipeline and expected developments in 2025.

Stifel analysts reiterated their Buy rating with a $37.00 price target following the FDA’s Breakthrough Therapy Designation for Denali’s DNL310 for Hunter Syndrome, which is expected to accelerate the approval process. Denali is preparing for a Biologics License Application (BLA) submission for DNL310, aiming for accelerated approval in early 2025. The company is also advancing DNL126 for Sanfilippo Syndrome, supported by the FDA’s recognition of heparan sulfate as a surrogate endpoint. Stifel’s analysis suggests that Denali’s stock holds significant promise, despite a recent decline after the failure of another drug candidate. These developments collectively underscore Denali’s strategic focus on leveraging its ETV platform to address neurological disorders.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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