Denali Therapeutics has announced exciting new results from a study on tividenofusp alfa (DNL310), a promising treatment for Hunter syndrome (MPS II). The findings show that this new therapy could improve both brain function and physical symptoms, bringing fresh hope to patients and families. Denali now plans to seek FDA approval in early 2025, which could make the treatment available as soon as late 2025 or early 2026.
What the Study Found
In a 24-week trial with 47 participants, and a follow-up period of up to four years, researchers saw significant improvements:
- Better brain function – Improvements in thinking, memory, and behavior.
- Hearing improvements – Many participants had better hearing across all frequencies.
- Lower disease markers – Key signs of the disease in spinal fluid and urine dropped to normal or near-normal levels.
- Healthier organs – Liver size returned to normal after 24 weeks.
- Well tolerated – Most side effects were mild or moderate.
Why This Matters
Hunter syndrome is a rare and serious genetic disorder that mainly affects boys. It causes physical problems like joint stiffness and hearing loss, but also impacts brain function, leading to cognitive decline. The current treatment, enzyme replacement therapy (ERT), helps the body but cannot reach the brain, leaving patients with worsening mental symptoms.
Tividenofusp alfa is different—it is designed to cross the blood-brain barrier, meaning it can help both the body and the brain. If approved, it could be a game-changer for patients and families.
What Happens Next?
Denali Therapeutics is now running a larger Phase 2/3 trial called the COMPASS study, which aims to confirm the benefits of this treatment.
- Patients will either receive tividenofusp alfa or the standard treatment to compare results.
- More participants are being added to ensure strong data for global approval.
What Experts Are Saying
“This progress gives real hope to families affected by Hunter syndrome,” said Dr. Joseph Muenzer, a lead researcher. “Seeing these improvements over time is a huge step forward.”
With this new treatment, Denali Therapeutics is working toward changing the future for Hunter syndrome patients, giving them a chance at a healthier and brighter life.
For updates, visit www.denalitherapeutics.com.