Denali Therapeutics Initiates BLA Submission for Tividenofusp Alfa

By Tamara Parker • Wednesday, April 02 15:15 • Permanent Link

Denali Therapeutics Inc. (NASDAQ: DNLI) has announced the initiation of a rolling submission of a Biologics License Application (BLA) for accelerated approval of Tividenofusp alfa for the treatment of Hunter syndrome (MPS II). This milestone brings the company closer to potentially delivering a new treatment option to the Hunter syndrome community.

The company's Chief Medical Officer, Carole Ho, M.D., expressed gratitude to the FDA for their ongoing support of the BLA filing and dedication to advancing new medicines. The submission of the BLA signals progress in the expansion of Denali's enzyme transport vehicle franchise of next-generation enzyme replacement therapies to treat the brain and body.

Tividenofusp alfa, also known as DNL310, has been granted fast track and breakthrough therapy designations by the U.S. Food and Drug Administration, as well as priority medicines designation by the European Medicines Agency. The phase 2/3 Compass study, which is enrolling participants across North America, South America, and Europe, aims to support global approval of Tividenofusp alfa.

Meanwhile, Denali continues to have positive ongoing interactions with the FDA on DNL126 through the START program, which aims to establish an accelerated development and approval path for DNL126 for the potential treatment of Sanfilippo syndrome.

Hunter syndrome (MPS II) is a rare genetic disease that affects over 2,000 individuals worldwide, primarily males, and leads to physical, cognitive, and behavioral symptoms. The current standard of care, enzyme replacement therapy, only partially treats physical symptoms and does not address cognitive and behavioral symptoms due to its inability to cross the blood-brain barrier.

Denali's efforts in developing Tividenofusp alfa and DNL126 aim to address the unmet need for therapies that can comprehensively treat the range of cognitive, behavioral, and physical manifestations of Hunter syndrome and Sanfilippo syndrome.

Denali Therapeutics is a biopharmaceutical company focused on developing a broad portfolio of product candidates engineered to cross the blood-brain barrier for neurodegenerative diseases and lysosomal storage diseases. The market has reacted to these announcements by moving the company's shares 14.3% to a price of $14.05. For the full picture, make sure to review Denali Therapeutics's 8-K report.

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