Welcome to our dedicated page for Ultragenyx Pharm news (Ticker: RARE), a resource for investors and traders seeking the latest updates and insights on Ultragenyx Pharm stock.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) delivers innovative therapies for rare genetic disorders through targeted research and biomarker-driven clinical programs. This page consolidates official announcements and verified updates about the company's therapeutic advancements, regulatory progress, and strategic initiatives.
Investors and medical professionals will find timely updates on clinical trial outcomes, FDA designations, and partnership developments. The curated news collection includes earnings reports, research milestones, and corporate updates relevant to rare disease treatment innovation.
Key content focuses on therapy approvals, orphan drug status achievements, and collaborations with patient advocacy networks. All materials adhere to regulatory disclosure standards while maintaining accessibility for both clinical experts and general investors.
Bookmark this page for centralized access to Ultragenyx's latest developments in addressing unmet medical needs through precision biopharmaceutical solutions.
Ultragenyx Pharmaceutical (NASDAQ: RARE) has submitted a Biologics License Application (BLA) to the FDA for UX111, an AAV gene therapy targeting Sanfilippo Syndrome Type A (MPS IIIA). If approved, UX111 would become the first authorized treatment in the U.S. for this condition.
The submission follows FDA's acceptance of cerebral spinal fluid (CSF) heparan sulfate (HS) as a surrogate endpoint for accelerated approval. Data from the ongoing pivotal Transpher A study showed that UX111 treatment led to rapid and sustained decreases in CSF HS levels, correlating with improved long-term cognitive development compared to natural history data. Most treatment-related adverse events were mild to moderate liver enzyme elevations that resolved.
Ultragenyx Pharmaceutical (NASDAQ: RARE) has announced its participation in three major investor conferences in December 2024. The company will attend Citi's 2024 Global Healthcare Conference in Miami, the 7th Annual Evercore ISI HealthCONx Conference in Coral Gables, and Piper Sandler's 36th Annual Healthcare Conference in New York.
Key executives including CEO Emil Kakkis, CFO Howard Horn, and CMO Eric Crombez will participate in fireside chats and host one-on-one meetings across these events. Live and archived webcasts will be available on the company's investor relations website.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the grant of 20,820 restricted stock units to 13 newly hired non-executive officers. The compensation committee approved these awards under the Ultragenyx Employment Inducement Plan, with a grant date of November 16, 2024. The stock units will vest over four years, with 25% of shares vesting annually, contingent on continuous employment. This grant was made in accordance with Nasdaq Listing Rule 5635(c)(4) as an employment inducement.
Ultragenyx Pharmaceutical (NASDAQ: RARE) presented positive Phase 1/2 data for GTX-102, its investigational treatment for Angelman syndrome, at the FAST Global Science Summit. The data supports the upcoming Phase 3 Aspire study, showing significant improvements across multiple domains at Week 48. Patients (n=40) demonstrated a mean change of +6.7 in Bayley-4 Cognition GSV score and +10.9 in Raw score. The Phase 3 study is powered at 95% to detect treatment effects. In Expansion Cohorts A&B, 80% of patients (22 of 28) achieved clinically meaningful improvement in at least one domain, with a total net response of +2.0. The Phase 3 trial will enroll 120 patients and is set to begin by year-end.
Ultragenyx Pharmaceutical (NASDAQ: RARE) reported strong Q3 2024 financial results with total revenue of $139 million, up 42% year-over-year. Crysvita revenue reached $98 million (+31% YoY), while Dojolvi revenue was $21 million (+29% YoY). The company reaffirmed its 2024 revenue guidance of $530-550 million. Despite revenue growth, Ultragenyx reported a net loss of $134 million ($1.40 per share), improved from $160 million loss in Q3 2023. The company received Breakthrough Therapy Designation for setrusumab in osteogenesis imperfecta and reported positive DTX401 Phase 3 data showing 62% mean reduction in cornstarch for GSDIa patients.
Ultragenyx Pharmaceutical (NASDAQ: RARE), a biopharmaceutical company specializing in treatments for rare and ultrarare genetic diseases, has announced a conference call scheduled for November 5, 2024, at 5:00 p.m. ET. The call will discuss the company's third quarter 2024 financial results and provide a corporate update for the period ending September 30, 2024. The event will be accessible via webcast through the company's investor relations website, with a replay available for three months.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company specializing in rare and ultrarare disease therapies, has announced the grant of 22,405 restricted stock units to six newly hired non-executive officers. The grants were approved by the company's compensation committee and issued under the Ultragenyx Employment Inducement Plan on October 16, 2024.
These stock units are designed as an employment inducement, aligning with Nasdaq Listing Rule 5635(c)(4). The vesting schedule spans four years, with 25% of the shares vesting annually on the grant date anniversary, contingent on continuous employment with Ultragenyx.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has received Breakthrough Therapy Designation from the FDA for setrusumab (UX143) to reduce fracture risk in patients 2 years and older with osteogenesis imperfecta (OI) Type I, III, or IV. This designation is based on promising clinical evidence from the Phase 2 portion of the Orbit study and the completed Phase 2b ASTEROID study, which showed a rapid and significant decrease in fracture rates.
Dr. Eric Crombez, chief medical officer at Ultragenyx, emphasized the importance of this designation in recognizing the seriousness of OI and the potential impact of setrusumab. The Breakthrough Therapy Designation aims to expedite development and review of drugs for serious diseases that show substantial improvement over existing therapies. Setrusumab has also received Orphan Drug Designation in the US and EU, rare pediatric disease designation in the US, and acceptance into the EMA's Priority Medicines program.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has reported promising results from the Phase 1/2/3 Cyprus2+ study of its UX701 gene therapy for Wilson Disease. The study has shown meaningful clinical activity and improvements in copper metabolism in Stage 1. Notably, six patients have completely tapered off standard-of-care treatment, with responses observed across all three dose cohorts.
The company plans to enroll an additional cohort in Stage 1 with a moderately increased dose and an optimized immunomodulation regimen to enhance the therapy's efficiency and efficacy. The goal is to have the majority of patients discontinue standard-of-care treatment before selecting a dose for the randomized placebo-controlled stage.
UX701 has been well-tolerated, with no unexpected related treatment emergent adverse events or significant immunologic safety issues reported. The study's design includes three stages, with the primary efficacy endpoints being change in 24-hour urinary copper concentration and percent reduction in standard-of-care medication by Week 52 of Stage 2.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced it will present seven abstracts related to its setrusumab (UX143) program and osteogenesis imperfecta (OI) at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting. The presentations include a late-breaker oral presentation of 14-month data from the Phase 2/3 Orbit study, which demonstrated a rapid and clinically meaningful increase in bone mineral density and a corresponding decrease in annualized fracture rate.
The abstracts cover various aspects of OI, including:
- Integrated bone biomarker analyses
- Burden of illness in Ontario, Canada
- Manifestations and comorbid conditions among OI patients
- Fracture rates in OI patients
- Population pharmacokinetics analyses
- Healthcare resource use and costs for OI patients
The ASBMR 2024 Annual Meeting will be held from September 27-30, 2024, in Toronto, Canada.
FAQ
What is the current stock price of Ultragenyx Pharm (RARE)?
What is the market cap of Ultragenyx Pharm (RARE)?
