Denali Therapeutics: FDA Alignment and Positive Trial Results Drive Buy Rating

Denali Therapeutics has achieved alignment with the FDA on an accelerated approval pathway for its DNL126 program targeting Sanfilippo syndrome. The company's ongoing Phase I/II trial for DNL126 has shown consistent results over 49 weeks, supporting the treatment's potential efficacy. The acceptance of the biologics license application for tividenofusp alfa under the accelerated approval pathway for Hunter syndrome adds to the company's positive regulatory momentum. Analyst Myles Minter has reiterated his Buy rating for DNLI stock due to these promising developments.

Denali Therapeutics Inc. (Nasdaq: DNLI) has achieved a significant milestone in its ongoing fight against Sanfilippo syndrome type A (MPS IIIA). The company has aligned with the FDA on an accelerated approval pathway for its DNL126 program, which is designed to treat this rare genetic disorder. This alignment follows the FDA's acceptance of Denali's BLA for tividenofusp alfa, an investigational enzyme replacement therapy for Hunter syndrome, under an accelerated approval pathway. The company's positive regulatory momentum has been further bolstered by the completion of the enrollment of its Phase 1/2 study for DNL126, which demonstrated consistent results over 49 weeks, indicating the potential efficacy of the treatment.

Analyst Myles Minter has reiterated his Buy rating for DNLI stock, citing these promising developments as a strong indicator of the company's potential to deliver meaningful treatments for serious diseases. Denali's ongoing Phase 2/3 COMPASS study for tividenofusp alfa and the planning for a global Phase 3 confirmatory study for DNL126 further underscore the company's commitment to advancing its pipeline of TV-enabled programs.

Denali Therapeutics is a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for the treatment of neurodegenerative diseases and lysosomal storage diseases. The company's focus on rigorously assessing genetically validated targets and engineering delivery across the BBB positions it well to address the unmet needs of patients with these conditions.

References:
[1] https://www.biospace.com/press-releases/denali-therapeutics-reports-second-quarter-2025-financial-results-and-business-highlights