Ultragenyx Pharm Stock Price, News & Analysis

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.

Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the commercial availability of Dojolvi™ (triheptanoin) for treating long-chain fatty acid oxidation disorders (LC-FAOD) following FDA approval on June 30, 2020. This marks the first FDA-approved therapy for LC-FAOD, a serious condition affecting approximately 2,000 to 3,500 patients in the U.S. Dojolvi aims to provide essential calories and fatty acids, with support available through Ultragenyx's UltraCare program for patient access. Caution is advised due to potential gastrointestinal side effects and contraindications for certain patients.

Ultragenyx Pharmaceutical has received FDA approval for Dojolvi™ (triheptanoin), a treatment for long-chain fatty acid oxidation disorders (LC-FAOD), marking its fourth FDA approval in three years. Dojolvi offers a new source of calories and fatty acids for patients, many of whom face serious health challenges despite existing treatments. The company estimates LC-FAOD affects 2,000 to 3,500 individuals in the U.S. and plans to make Dojolvi available within 30 days through its UltraCare® program for patient support and insurance navigation.

Ultragenyx Pharmaceutical (NASDAQ: RARE) and Kyowa Kirin announced FDA approval for Crysvita (burosumab) as the first therapy for tumor-induced osteomalacia (TIO) that cannot be surgically removed. This decision represents the second FDA-approved indication for Crysvita, previously authorized for X-linked hypophosphatemia (XLH). TIO affects approximately 500-1,000 patients in the U.S., and half are inoperable. Crysvita acts by blocking FGF23, alleviating symptoms like severe hypophosphatemia. The approval follows successful Phase 2 trials, showcasing significant improvements in phosphate levels and bone health.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has announced virtual presentations at investor conferences. CFO Shalini Sharp will present at the Jefferies Healthcare Conference on June 3, 2020, at 2 PM ET, followed by CEO Emil D. Kakkis at the Goldman Sachs 41st Annual Global Healthcare Conference on June 9, 2020, at 3:50 PM ET. Live and archived webcasts will be available on the company's website for 90 days post-event. Ultragenyx is focused on developing novel treatments for rare genetic diseases, aiming for efficient drug development to meet high unmet medical needs.