(RTTNews) - Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company, on Wednesday reported encouraging data from its ongoing clinical program of UX111, a gene therapy for Sanfilippo Syndrome Type A or MPS IIIA, a rare neurodegenerative disorder.
The new data show that treatment with UX111 resulted in significant improvements in cognitive function, communication skills, and motor abilities, as measured by the Bayley-III assessment, compared to natural history data from untreated patients.
According to the company, UX111 demonstrated a 22.7-point improvement in cognitive function, with positive trends also seen in receptive and expressive communication as well as fine motor function.
Furthermore, a substantial reduction in cerebrospinal fluid or CSF-heparan sulfate or HS levels was observed, reflecting a direct impact on the underlying disease process.
Patients treated with the 3x10^13 vg/kg dose showed a 65 percent median reduction in CSF-HS levels at 34 months post-treatment.
Importantly, older patients and those with more advanced disease at the time of treatment retained critical functional abilities, such as communication, ambulation, and self-feeding.
Dr. Eric Crombez, Chief Medical Officer at Ultragenyx, stated, "The data support UX111's potential to provide meaningful benefits to children with Sanfilippo Syndrome, especially for those in advanced stages of the disease."
Safety results also remained favorable, with most adverse events being mild to moderate and resolving over time.
A Biologics License Application seeking accelerated approval for UX111 was submitted to the FDA in December 2024. A decision on the application is expected in the second half of 2025.
RARE closed Tuesday's (FEB.04 2025) trading at $44.18 up by 1.75%. In premarket trading Wednesday the stock is up 2.31% at $45.20.
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