DNLI's Hunter Syndrome Drug Gets Breakthrough Therapy Designation

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Denali Therapeutics Inc. DNLI announced that the FDA has granted Breakthrough Therapy Designation to its pipeline candidate, tividenofusp alfa (DNL310), for the treatment of individuals with Hunter syndrome (MPS II).

Denali’s wholly owned program, DNL310 or tividenofusp alfa, is an Enzyme Transport Vehicle-enabled iduronate-2-sulfatase (IDS) replacement therapy in development for MPS II (Hunter syndrome).

Shares of DNLI have lost 1.9% in the past six months compared with the industry’s 8.3% decline.

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More on DNLI’s Tividenofusp Alfa

The FDA's above-mentioned designation is intended to expedite the development and review of therapeutic drugs being evaluated for the treatment of serious or life-threatening conditions.

Breakthrough Therapy Designation is generally granted to a drug when evidences suggest that the investigational drug may provide substantial improvement over available therapy on at least one clinically significant endpoint.

Data from the open-label phase I/II study have shown promising results, with positive effects on evidence-based surrogate endpoints and early signs of improved clinical outcomes in participants with Hunter syndrome.

This designation will provide Denali with more intensive FDA guidance, including involvement of senior reviewers, and eligibility for rolling and priority review of the marketing application.

In 2021, the FDA granted Fast Track designation to tividenofusp alfa for the treatment of patients with Hunter syndrome. In 2022, the European Medicines Agency granted Priority Medicines designation to tividenofusp alfa the.

In September 2024, Denali announced the outcome of a successful meeting with the FDA, providing a path to filing a biologics license application (BLA) for tividenofusp alfa for accelerated approval and subsequent conversion to full approval for the treatment of Hunter syndrome.

Denali expects to submit a BLA for tividenofusp alfa in early 2025 for regulatory review under the accelerated approval pathway.

DNLI’s Recent Pipeline Updates

Denali recently initiated dosing in a global mid-stage study, BEACON, on BIIB122 (DNL151).

BIIB122, an investigational drug leucine-rich repeat kinase 2 (LRRK2) inhibitor, is being evaluated in participants with LRRK2-associated Parkinson’s disease (LRRK2-PD).

The phase IIa study will evaluate safety and biomarkers associated with oral daily dosing of BIIB122 in approximately 50 participants with Parkinson’s disease and LRRK2 pathogenic mutations confirmed by genetic testing.

BIIB122 is also being evaluated in the ongoing global phase IIb LUMA study in participants with early-stage Parkinson’s disease with or without an LRRK2 mutation in collaboration with Biogen BIIB.