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 reported Q3 2020 revenues of $81.5 million, with Crysvita generating $37.3 million. The company raised the lower end of its Crysvita revenue guidance to $130 million from $125 million, with a total expected range of $130 million to $140 million. The launch of Dojolvi yielded $3.9 million in sales. However, operating expenses were $131.8 million, leading to a net loss of $68.8 million for the quarter. Notably, the interim data for GTX-102 shows promise for Angelman Syndrome, though serious adverse events were reported.
GeneTx Biotherapeutics and Ultragenyx Pharmaceutical (NASDAQ: RARE) announced positive interim data from the Phase 1/2 study of GTX-102, an investigational treatment for Angelman syndrome. All five patients treated showed significant improvements in communication, behavior, and motor functions by day 128. However, serious adverse events related to lower extremity weakness were noted at the highest doses, prompting a pause in dosing. The study protocol will be amended to lower dosages and minimize side effects. Further data will be presented at the FAST Global Summit in December 2020.
Ultragenyx Pharmaceutical and Solid Biosciences announced a strategic collaboration to develop gene therapies for Duchenne muscular dystrophy. The agreement includes a $40 million upfront investment from Ultragenyx, with potential milestone payments totaling up to $255 million and tiered royalties. Solid retains exclusive rights to its microdystrophin technology for other uses. The collaboration aims to harness Solid's microdystrophin construct and Ultragenyx’s HeLa PCL platform to enhance treatment efficacy. The partnership is positioned to leverage both companies' strengths in advancing therapies for rare diseases.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has announced a conference call scheduled for October 27, 2020, at 5 PM ET to discuss its third quarter 2020 financial results and provide a corporate update. The call will be accessible via the company's website, and participants can join by phone. Ultragenyx focuses on developing and commercializing novel therapies for serious rare and ultra-rare genetic diseases, aiming for efficient drug development to address high unmet medical needs.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announces CEO Emil D. Kakkis will present virtually at the Jefferies Virtual Gene Therapy/Editing Summit on October 2, 2020, at 1:00 PM ET. The presentation will be accessible live and as an archive on their website. Ultragenyx focuses on developing treatments for serious rare and ultra-rare genetic diseases, with a commitment to efficient drug development. They aim to provide safe and effective therapies to patients with high unmet medical needs. Visit their website for more details.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that CEO Emil D. Kakkis will present at three upcoming investor conferences. The events include the Citi’s 15th Annual BioPharma Virtual Conference on September 9, 2020, the Morgan Stanley Virtual 18th Annual Global Healthcare Conference on September 15, 2020, and the BofA Securities Virtual Global Healthcare Conference on September 16, 2020. Live and archived webcasts of these presentations will be available on the company’s website for 90 days.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has appointed Mardi C. Dier as Executive Vice President and Chief Financial Officer, effective November 2, 2020. Dier will oversee corporate finance, strategy, investor relations, corporate communications, and information technology. She joins from Portola Pharmaceuticals, where she served as CFO, enhancing her experience in financial leadership. Ultragenyx aims to grow its commercial opportunities and pipeline for rare diseases, while thanking outgoing CFO Shalini Sharp for her contributions.
Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the appointment of Ernie Meyer as the new Executive Vice President and Chief Human Resources Officer, effective September 1, 2020. In this role, Meyer will oversee Global Human Resources and Facilities operations, reporting directly to CEO Emil D. Kakkis. Meyer brings extensive experience from previous roles at Portola Pharmaceuticals and Celgene, where he contributed to significant organizational growth. His leadership is expected to enhance Ultragenyx's commitment to addressing rare genetic diseases as it enters a new growth phase.
Ultragenyx Pharmaceutical reported $61.7 million in total revenue for Q2 2020, with $32.4 million from Crysvita. The company achieved two FDA approvals: Dojolvi for LC-FAOD and Crysvita for TIO. Net income was $25.3 million ($0.42/share), a significant recovery from a $99.2 million loss in Q2 2019. Operating expenses totaled $124.8 million, influenced by increased costs in commercial and development activities. Ultragenyx maintains its full-year guidance for Crysvita revenue between $125 million and $140 million, despite COVID-19 impacts.
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced a conference call set for July 30, 2020, at 5pm ET to discuss its second-quarter 2020 financial results and provide corporate updates. The call will be accessible via phone and through a live webcast on the company’s website. Ultragenyx is dedicated to developing treatments for rare genetic diseases, featuring a diverse portfolio of approved therapies and product candidates aimed at high unmet medical needs. The management team emphasizes efficient drug development to deliver therapies urgently.
