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NOVATO, Calif. - Ultragenyx Pharmaceutical (TADAWUL:2070) Inc. (NASDAQ: NASDAQ:RARE), a $4.08 billion market cap biotechnology company with 27% revenue growth in the past year, today unveiled new data indicating that its experimental gene therapy, UX111, has led to significant cognitive improvements in patients with Sanfilippo syndrome type A, compared to untreated patients. According to InvestingPro analysis, the company maintains a strong liquidity position with a current ratio of 2.81, providing adequate resources for its development programs. The findings will be shared at the WORLDSymposium™ 2025, currently underway in San Diego.
The study measured the efficacy of UX111 in treating cognitive, communicative, and motor skills deficits associated with the syndrome. The modified intent-to-treat group showed a 22.7-point increase in the mean Bayley-III cognitive raw score, a statistically significant improvement over natural history data of untreated patients.
The treatment also resulted in a substantial decrease in heparan sulfate levels in the cerebrospinal fluid, which is believed to correlate with the disease’s progression. Patients treated with the highest dose of UX111 saw a median reduction of 65% in heparan sulfate levels.
Notably, older children or those with more advanced disease retained meaningful functional abilities, such as communication and self-feeding, which are typically lost during late childhood and early adolescence in untreated patients.
The safety profile of UX111 has been favorable, with the most common adverse events being mild to moderate elevations in liver enzymes, all of which resolved.
These data were part of a Biologics License Application submitted to the FDA in December 2024, with a decision expected in the second half of 2025. With the company’s next earnings report due on February 13, 2025, investors can gain deeper insights into the development pipeline through InvestingPro’s comprehensive analysis, which includes 7 additional key insights about the company’s financial health and growth prospects.
UX111 is currently in Phase 1/2/3 development and has received several designations from the FDA that could expedite its review process. Sanfilippo syndrome type A is a rare, fatal condition with no approved treatment that affects approximately 3,000 to 5,000 patients in commercially accessible geographies.
Ultragenyx specializes in developing therapies for rare and ultra-rare genetic diseases and has a portfolio of approved medicines and candidates in development.
The information reported is based on a press release statement from Ultragenyx Pharmaceutical Inc.
In other recent news, Ultragenyx Pharmaceutical Inc. has made several significant announcements. The company’s FY25 guidance predicts a 14-20% growth, surpassing both Goldman Sachs and Visible Alpha Consensus Data estimates. Goldman Sachs, maintaining a Buy rating on Ultragenyx, has expressed confidence in the company’s Phase 3 Orbit study of setrusumab for osteogenesis imperfecta. The firm also anticipates further advancements in Ultragenyx’s pipeline, such as the potential commercial launch of gene therapy UX111 for Sanfilippo syndrome.
Meanwhile, RBC Capital Markets has reiterated its Outperform rating on Ultragenyx, expressing optimism about the company’s drug for Osteogenesis Imperfecta. The firm believes the drug offers significant upside potential and has highlighted additional opportunities for Ultragenyx in the fields of Angelman syndrome and gene therapy.
Furthermore, H.C. Wainwright maintained its Buy rating and price target for Ultragenyx following the company’s announcement regarding the commencement of the pivotal Phase 3 Aspire study for GTX-102, an experimental treatment for Angelman syndrome. The firm also looks forward to the first potential interim analysis from the Phase 3 portion of the Orbit study for setrusumab.
In addition, Ultragenyx has submitted a Biologics License Application to the U.S. Food and Drug Administration for UX111, a proposed gene therapy for the treatment of Sanfilippo syndrome type A. If approved, UX111 would be the first therapy authorized in the United States for this rare and fatal condition.
Lastly, Ultragenyx reported a significant increase in revenue for the third quarter of 2024, with a 42% year-over-year growth totaling $139 million. Despite a net loss of $134 million for the quarter, Ultragenyx remains focused on advancing its clinical pipeline and expects to reach GAAP profitability by the end of 2026.
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