On Tuesday, H.C. Wainwright maintained its Buy rating and $95.00 price target for Ultragenyx Pharma (NASDAQ:RARE), representing significant upside from the current trading price of $43.87. According to InvestingPro data, analyst consensus remains strongly bullish, with price targets ranging from $48 to $140. The firm’s endorsement follows Ultragenyx’s recent announcement on December 19, 2024, regarding the commencement of the pivotal Phase 3 Aspire study for GTX-102, its experimental treatment for Angelman syndrome.
The Aspire study, which began dosing its first patient, aims to enroll approximately 120 children between the ages of 4 to 17 who have been genetically diagnosed with a full maternal UBE3A gene deletion characteristic of Angelman syndrome.
While the company posted strong revenue growth of 27.4% in the last twelve months and maintains a healthy current ratio of 2.81, indicating strong liquidity, participants are randomized in a 1:1 ratio to receive either GTX-102 or a sham comparator over a 48-week primary efficacy analysis period. The treatment group will be given three monthly loading doses of GTX-102 at 8 mg, followed by a maximum of 14 mg quarterly during maintenance.
The primary endpoint of the Aspire study is to measure cognitive improvement through the Bayley-4 cognitive raw score. Additionally, a key secondary endpoint involves the Multi-domain Responder Index (MDRI), which assesses five domains: cognition, receptive communication, behavior, gross motor function, and sleep. After the initial 48 weeks, patients who received the sham treatment will be eligible to crossover to GTX-102.
In conjunction with the Aspire study, H.C. Wainwright is also looking ahead to the first potential interim analysis from the Phase 3 portion of the Orbit study for setrusumab, expected around the end of 2024 or early 2025. This analysis will only proceed if the compiled data meets the statistical significance threshold of p
In other recent news, Ultragenyx Pharmaceutical (NASDAQ:RARE) Inc. has made noteworthy strides in its clinical pipeline. The company is seeking FDA approval for its Sanfilippo A gene therapy, UX111, a significant development in the treatment of a rare neurodegenerative disease. This move comes after the FDA acknowledged cerebrospinal fluid heparan sulfate as a biomarker for accelerated approval.
On the financial front, Ultragenyx reported a 42% year-over-year increase in Q3 2024 revenue, totaling $139 million. However, the company also recorded a net loss of $134 million for the same quarter. Despite this, analysts maintain a strong buy consensus on the stock, with price targets ranging from $48 to $140 per share.
Ultragenyx’s CEO, Emil Kakkis, has emphasized the importance of timely execution in rare disease programs and is optimistic about the company’s future. The company anticipates reaching GAAP profitability by the end of 2026 and is preparing for Biologics License Application submissions for treatments targeting rare diseases, with potential for three new therapies launching in upcoming years.
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