On Thursday, Leerink Partners upheld its Outperform rating for Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), despite new data from a competitor's clinical trial. The firm remains optimistic about the potential of Ultragenyx's treatment for Angelman syndrome, GTX-102, in light of recent developments from Ionis Pharmaceuticals (NASDAQ:IONS).
Ionis revealed topline results from the Phase 1/2a trial of its own antisense oligonucleotide therapy, ION582, indicating plans to advance to a pivotal study. The preliminary data suggest consistent effects across multiple measures, yet detailed outcomes are anticipated to be presented at the Angelman Syndrome Foundation meeting in July 2024.
Ultragenyx, on the other hand, shared updated data from its Phase 1/2 study of GTX-102 last month, with preparations to consult with the FDA and initiate a pivotal study by the end of 2024. Leerink Partners notes that while the exact comparison between ION582 and GTX-102 is currently difficult, today's news does not alter their cautiously optimistic view of Ultragenyx's program.
Biogen (NASDAQ:BIIB) has chosen not to license ION582, leaving Ionis to proceed independently. This decision comes after the HALOS study showed promising improvements in Angelman syndrome patients, particularly in cognitive functions, over a six-month period. The safety profile was consistent with expectations for the patient population and the nature of the treatment.
Leerink Partners forecasts that Ultragenyx's GTX-102 could achieve gross peak sales of approximately $1.2 billion in the US and EU markets, with a 40% probability of success. The firm emphasizes the significance of GTX-102 in Ultragenyx's pipeline, especially as investors look for substantial products to emerge amidst concerns over increasing expenditures.
The reaffirmed Outperform rating reflects confidence in the drug's market potential and Ultragenyx's strategic position.
InvestingPro Insights
As Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) progresses with its GTX-102 therapy for Angelman syndrome, a glance at the company's financial health and market position could offer investors additional context. According to real-time data from InvestingPro, Ultragenyx has a market capitalization of $3.52 billion, indicating a significant presence in the biotech industry.
Despite challenges, including a high Price/Book multiple of 25.06 and a negative Gross Profit Margin of -60.26% for the last twelve months as of Q1 2024, the company's liquid assets exceed its short-term obligations, suggesting a stable liquidity position.
InvestingPro Tips reveal that analysts are cautious about Ultragenyx's profitability in the short term, as the company is not expected to be profitable this year and has not been profitable over the last twelve months. Moreover, the company does not pay a dividend, which may influence investor decisions depending on their preference for income-generating investments.
Still, the company's commitment to advancing GTX-102 through clinical trials demonstrates a focus on long-term growth and potential market capture, aligning with Leerink Partners' outlook. Investors interested in deeper analysis can find more InvestingPro Tips, offering a comprehensive view of Ultragenyx's financials and market potential, at https://www.investing.com/pro/RARE. For those considering an InvestingPro subscription, use coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription, unlocking access to over six additional tips that can inform and guide investment decisions.
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