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 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.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company specializing in rare and ultrarare diseases, has announced the grant of 62,555 restricted stock units to 16 newly hired non-executive officers. The grants were approved by the company's compensation committee and issued under the Ultragenyx Employment Inducement Plan on September 16, 2024. This action complies with Nasdaq Listing Rule 5635(c)(4), serving as an inducement for new employees joining Ultragenyx. The restricted stock units have a four-year vesting schedule, with 25% of the shares vesting annually, contingent on continuous employment.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on developing therapies for rare genetic diseases, has announced its participation in three upcoming investor conferences in September 2024:
- Morgan Stanley 22nd Annual Global Healthcare Conference on September 4
- Cantor Global Healthcare Conference on September 17
- Bank of America Global Healthcare Conference on September 18
Key executives, including CEO Emil Kakkis, CFO Howard Horn, and CMO Eric Crombez, will participate in fireside chats and host one-on-one meetings. Live and archived webcasts of the fireside chats will be available on the company's investor relations website. Ultragenyx is committed to developing novel products for serious rare and ultrarare genetic diseases, with a diverse portfolio of approved therapies and product candidates.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company specializing in rare and ultrarare disease therapies, has granted 37,975 restricted stock units to 12 newly hired non-executive officers. The awards, approved by the company's compensation committee, were issued under the Ultragenyx Employment Inducement Plan on August 16, 2024. This grant aligns with Nasdaq Listing Rule 5635(c)(4) and serves as an employment inducement. The restricted stock units have a four-year vesting schedule, with 25% of the underlying shares vesting annually, contingent on continuous employment with Ultragenyx.
Amlogenyx Inc., a subsidiary of Ultragenyx Pharmaceutical (NASDAQ: RARE), has successfully closed a $14 million seed funding round led by GordonMD® Global Investments LP. The company is developing novel gene therapies for Alzheimer's disease and other amyloid diseases. Amlogenyx's first product candidate is a protease developed in collaboration with St. Jude Children's Research Hospital, which has shown promising results in breaking down Aβ42, a protein believed to be responsible for Alzheimer's effects.
Dr. Craig Gordon, Founder and CIO of GordonMD®, expressed excitement about the collaboration and the potential of Amlogenyx's Alzheimer's disease therapeutic. The funding round also included participation from Ultragenyx and associated investors, demonstrating strong support for Amlogenyx's innovative approach to treating amyloid diseases.
Ultragenyx reported second quarter 2024 financial results with total revenue of $147 million, up 36% YoY. Key revenue drivers were Crysvita ($114 million, +37%) and Dojolvi ($19 million, +17%). The company raised its 2024 revenue guidance to $530-$550 million from $500-$530 million.
Ultragenyx also disclosed significant clinical advancements including positive data from Phase 1/2 study in Angelman syndrome, Phase 2/3 study in osteogenesis imperfecta, and Phase 3 study in GSDIa. Phase 3 studies for Angelman syndrome and UX111 for Sanfilippo syndrome type A are on track to start by end of 2024.
The company reported a net loss of $132 million, down from $160 million YoY. Operating expenses were $263 million, including $39 million in non-cash stock-based compensation. Cash reserves stood at $874 million as of June 30, 2024.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company specializing in novel products for rare and ultrarare genetic diseases, has announced a conference call scheduled for August 1, 2024, at 5:00 p.m. ET. The call will cover the company's financial results and corporate update for the second quarter of 2024, ending June 30. Investors and interested parties can access the live webcast and replay through the company's investor relations website. The replay will remain available for three months following the call, providing extended access to the information presented.
