NOVATO, Calif. - Ultragenyx Pharmaceutical Inc. (NASDAQ: NASDAQ:RARE) today shared new data from a Phase 1/2 study of GTX-102, a treatment for Angelman syndrome, showing significant patient improvements.
The study revealed that patients in the Expansion Cohorts A & B experienced rapid, clinically meaningful progress across multiple domains by Day 170, aligning with or surpassing earlier Dose-escalation Cohorts 4-7 data. Furthermore, long-term data from Dose-escalation Cohorts 4-7 indicated sustained clinical benefits through Day 758.
These findings will be presented in more detail during a corporate presentation today at 8:00 a.m. ET and at the 76th Annual American Academy of Neurology Meeting in Denver on April 16, 2024. Dr. Kemi Olugemo, M.D., FAAN, will lead the presentation at the conference.
Angelman syndrome currently lacks an approved disease-modifying treatment. The syndrome leads to significant impairments, with individuals experiencing cognitive, communicative, and motor difficulties, among other challenges. Dr. Erick Sell, M.D., a principal investigator in the study, noted the significance of the multidomain improvement observed and the potential for increased independence in patients.
The new data include Day 170 results from 24 patients in the Expansion Cohorts and up to Day 758 results from 15 patients in the Dose-escalation Cohorts. Key improvements were seen in cognition, behavior, hyperactivity, noncompliance, sleep, and receptive communication.
Eric Crombez, M.D., chief medical officer at Ultragenyx, highlighted the broad developmental gains and their impact on patients and families, with children now able to communicate their needs and achieve further developmental milestones.
The study reported no unexpected serious adverse events. Three patients experienced mild to moderate serious adverse events related to lower extremity weakness, which resolved rapidly without ongoing safety concerns.
Ultragenyx aims to discuss these results with the FDA and other health authorities to expedite the initiation of a Phase 3 pivotal study. GTX-102 has received Orphan Drug Designation, Rare Pediatric Disease Designation, and Fast Track Designation from the FDA, as well as Orphan Designation and PRIME designation from the EMA.
This article is based on a press release statement from Ultragenyx Pharmaceutical Inc.
InvestingPro Insights
As Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) continues to make strides in the development of GTX-102 for Angelman syndrome, the company's financial health and market performance are key factors for investors to consider. According to the latest InvestingPro data, Ultragenyx has a market capitalization of $3.87 billion, reflecting its position in the biopharmaceutical industry. Despite not being profitable over the last twelve months, with a negative Price/Earnings (P/E) ratio of -6.38, the company's revenue has experienced a growth of 19.52% in the same period, indicating an increasing interest in its product pipeline.
One InvestingPro Tip that stands out is the company's Price/Book (P/B) ratio, which at 14.05 suggests that Ultragenyx is trading at a high valuation relative to its book value. This could be indicative of the market's optimistic view of the company's future prospects, despite the current lack of profitability. Additionally, Ultragenyx has seen a substantial 49.63% price uptick over the last six months, which may attract growth-oriented investors.
Investors may also note that Ultragenyx operates with a moderate level of debt and has liquid assets that exceed its short-term obligations, offering some financial stability as it advances its clinical programs. While the company does not pay dividends, reflecting a reinvestment strategy into its research and development efforts, these financial metrics and strategic decisions are crucial for stakeholders evaluating the company's long-term potential.
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