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NOVATO, Calif. - Ultragenyx Pharmaceutical Inc. (NASDAQ: NASDAQ:RARE) has shared promising results from the Phase 1/2/3 Cyprus2+ study of its investigational gene therapy, UX701, for the treatment of Wilson disease, a rare genetic disorder. According to the company's latest update, the study has shown meaningful clinical activity and improvements in copper metabolism among participants.
The ongoing study has completed its first stage, with 15 patients across three dosing cohorts showing responses to the therapy. Notably, six patients have entirely stopped their standard-of-care treatments, which include chelators and zinc therapy. A seventh patient is also in the process of tapering off these treatments. These patients have seen their non-ceruloplasmin bound copper (NCC) levels stabilize to normal ranges, indicating the potential efficacy of UX701.
Ultragenyx plans to add another cohort to the study, testing a moderately higher dose of the gene therapy combined with an optimized immunomodulation regimen. This adjustment aims to enhance the treatment's efficiency and effectiveness and potentially allow a majority of patients to discontinue standard-of-care treatments.
The therapy has been well tolerated thus far, with no unexpected treatment-related adverse events or significant immunological safety issues reported. The company is encouraged by the interim results, which show clear signs of transgene expression and improved copper trafficking in a subset of patients.
UX701 has been designed to deliver a stable expression of the ATP7B copper transporter after a single intravenous infusion. It has received Orphan Drug Designation in the United States and the European Union, as well as Fast Track Designation in the United States.
Wilson disease is caused by mutations in the ATP7B gene, leading to copper accumulation in the liver and other tissues. The condition can result in severe hepatic, neurological, and psychiatric problems. While current treatments focus on reducing or removing excess copper, they can have significant side effects, and some patients continue to experience clinical deterioration.
This news is based on a press release statement from Ultragenyx Pharmaceutical Inc. and does not constitute an endorsement of the company or its products. The study's results are preliminary, and further research is necessary to confirm the therapy's safety and efficacy.
In other recent news, Ultragenyx Pharmaceutical Inc. reported strong Q2 earnings with total revenue reaching $147 million, leading to an upward revision of its annual revenue guidance. The company's earnings call highlighted significant advancements in its clinical pipeline and commercial portfolio, including positive results from Phase III and Phase II trials for key drugs. Ultragenyx also secured an agreement with the FDA on a Phase III study design for GTX-102, a potential treatment for Angelman syndrome.
TD Cowen maintained a positive outlook on Ultragenyx, reiterating a Buy rating buoyed by promising Phase 2 Orbit trial results for setrusumab, a treatment for Osteogenesis Imperfecta. The trial data indicated a significant 67% reduction in the annualized fracture rate and a 22% increase in bone mineral density over 12 months. These results have cemented the 20 mg/kg dosage for the ongoing Phase 3 studies, with indications of greater benefits for younger patients aged between 5 to less than 12 years.
However, the company's interim Stage 1 readout for the Wilson's disease program has been delayed. Despite this, Ultragenyx expressed confidence in the superiority of its data for setrusumab compared to Amgen (NASDAQ:AMGN)'s romosozumab in treating osteogenesis imperfecta. These are among the recent developments that investors are closely monitoring.
InvestingPro Insights
As Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) advances its promising gene therapy for Wilson disease, investors may find additional context from InvestingPro's real-time data and expert tips.
According to InvestingPro data, Ultragenyx has shown strong revenue growth, with a 35.75% increase in quarterly revenue as of Q2 2024. This growth aligns with the company's progress in its clinical trials and potential market expansion. However, it's important to note that the company is not yet profitable, with a negative gross profit margin of -49.52% over the last twelve months.
InvestingPro Tips highlight that 8 analysts have revised their earnings upwards for the upcoming period, suggesting growing confidence in Ultragenyx's pipeline and potential commercialization of UX701. This optimism is reflected in the stock's performance, with a robust 56.66% return over the past year.
Despite the promising clinical results, investors should be aware that Ultragenyx operates with a moderate level of debt and is trading at a high Price / Book multiple of 11.43. This valuation may reflect market expectations for future growth and the potential success of therapies like UX701.
For those considering an investment in Ultragenyx, it's worth noting that InvestingPro offers 10 additional tips for RARE, providing a more comprehensive analysis of the company's financial health and market position. These insights could be particularly valuable as the company progresses through clinical trials and approaches potential commercialization of its gene therapy treatments.
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