Ultragenyx Pharm Stock Price, News & Analysis

GeneTx Biotherapeutics and Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the FDA's removal of the clinical hold on GTX-102, an experimental treatment for Angelman syndrome. This allows GeneTx to commence dosing naïve pediatric patients in a Phase 1/2 study. The trial aims to evaluate the drug's safety and effectiveness, enrolling eight patients aged 4 to <8 years in the U.S. The therapy targets the UBE3A gene, which is crucial for brain function and is absent in individuals with Angelman syndrome. There are currently no approved treatments for this condition.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that CEO Emil D. Kakkis will present at the Morgan Stanley 19th Annual Healthcare Conference on September 14, 2021, at 4:15 PM ET. The presentation will be available via a live and archived webcast on the company’s website, with the replay accessible for 90 days. Ultragenyx focuses on developing therapies for serious rare and ultra-rare genetic diseases, emphasizing novel products with high unmet medical needs. The company aims for efficient drug development to deliver urgent solutions to patients.

On September 1, 2021, Ultragenyx Pharmaceutical (NASDAQ: RARE) announced that CEO Emil D. Kakkis will speak at Citi's 16th Annual BioPharma Healthcare Conference on September 9, 2021, at 4:10 PM ET. The panel, titled Finding a Needle in a Haystack - Drug Development Strategies for Rare Disease, focuses on innovative approaches to developing therapies for rare genetic conditions. A live and archived webcast of the presentation will be available on the company's website for 90 days.

Ultragenyx is committed to addressing unmet medical needs in rare diseases through efficient drug development.

Dojolvi has been approved by Brazil's National Health Surveillance Agency (ANVISA) for treatment of long-chain fatty acid oxidation disorders (LC-FAOD), marking an essential therapeutic advance for patients. This decision is crucial for managing LC-FAOD, which affects 8,000 to 14,000 individuals in developed countries, leading to severe health complications. Ultragenyx Pharmaceutical Inc. aims to secure reimbursement to enhance patient access. The drug is already FDA-approved and available through early access programs outside the U.S.

Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the grant of stock options totaling 14,445 shares and 4,815 restricted stock units to two newly hired employees. This grant, which took place on August 16, 2021, aligns with the company's Employment Inducement Plan and Nasdaq Listing Rule 5635(c)(4). The stock options have a ten-year term and an exercise price of $88.00 per share. Vested shares will be distributed over four years, contingent on ongoing employment. Ultragenyx continues to focus on developing therapies for rare diseases.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that CEO Emil D. Kakkis will participate in a panel discussion titled Miss Con-GENE-iality - Updates in Gene Tx at the Wedbush PacGrow Healthcare Conference on August 11, 2021, at 12:00 PM ET. The event will be accessible via live and archived webcasts on the company’s website for 90 days. Ultragenyx focuses on developing therapies for serious rare and ultra-rare genetic diseases, with a commitment to efficient drug development and addressing unmet medical needs.

Ultragenyx Pharmaceutical reported Q2 2021 total revenue of $87.0 million, reflecting a 38% increase from Q2 2020, driven by strong Crysvita sales of $44.7 million. The guidance for Crysvita remains at $180-$190 million for 2021. Dojolvi continues to gain traction with 220 patients on therapy. Operating expenses rose 36% to $169.8 million. The company expects to initiate four pivotal clinical trials in the next six months, enhancing its late-stage pipeline. As of June 30, cash and equivalents stood at $973.8 million.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) will host a conference call on August 2, 2021, at 5 PM ET to discuss its financial results for Q2 2021, ending June 30. The company is known for developing therapies for serious rare genetic diseases and has a diverse portfolio aimed at unmet medical needs. Interested participants can access the live webcast through their website or dial in by phone. This update underscores the company's commitment to efficient drug development and patient care in the rare disease sector.

Ultragenyx Pharmaceutical announced that the FDA and the European Commission have granted Orphan Drug Designation for UX053, aimed at treating Glycogen Storage Disease Type III (GSDIII), affecting over 10,000 patients in developed nations. Enrollment for a Phase 1/2 clinical trial is anticipated in late 2021, evaluating the drug's safety, tolerability, and efficacy. The Orphan Drug Designation provides significant market exclusivity benefits, including seven years in the U.S. and ten years in the EU.