Ultragenyx Pharm Stock Price, News & Analysis

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) reported its 2020 financial results, with total revenue reaching $271.0 million and Crysvita revenue of $138.9 million. The 2021 guidance for Crysvita revenue is reaffirmed at $180 million to $190 million. The company launched Dojolvi in July 2020, serving approximately 130 patients in the U.S. with a significant quarterly growth. Operating expenses were $601.1 million, leading to a net loss of $186.6 million. Key clinical trials are set to advance, including three pivotal gene therapy studies in 2021.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has announced a conference call on February 11, 2021, at 5 pm ET, to discuss its fourth-quarter and full-year financial results for 2020. The call will provide a corporate update and insights into the company’s progress in developing novel treatments for serious rare and ultra-rare genetic diseases. The information will be accessible through their website, with a dedicated phone line for participants.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has received FDA clearance for the IND application of UX701, a gene therapy targeting Wilson Disease. The company plans to initiate a seamless Phase 1/2/3 clinical trial in the first half of 2021, evaluating safety and efficacy across multiple dosing levels. UX701 aims to improve copper regulation by delivering a modified ATP7B copper transporter. This therapy could potentially alleviate unmet medical needs for patients suffering from Wilson Disease, which affects over 50,000 individuals in developed regions.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) reported preliminary unaudited 2020 revenues for Crysvita between $137 million and $139 million, aligned with prior guidance despite COVID-19 challenges. The company projects 2021 revenues for Crysvita between $180 million and $190 million. As of December 31, 2020, their cash position was approximately $1.2 billion. The PR highlights advancements in clinical trials for several therapies, including DTX401 and DTX301, with expectations for Phase 3 studies commencing in 2021.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced favorable long-term safety and efficacy data from Phase 1/2 studies of DTX401 for Glycogen Storage Disease Type Ia (GSDIa) and DTX301 for ornithine transcarbamylase (OTC) deficiency. All patients in these studies showed improved metabolic control, with significant cornstarch dependency reduction. Phase 3 trials for both therapies are set to initiate in 2021. Additionally, Ultragenyx submitted an IND for UX701 targeting Wilson Disease, with clinical entry expected in the first half of 2021, leveraging their HeLa PCL platform for manufacturing.

Ultragenyx Pharmaceutical, a biopharmaceutical firm focused on rare genetic diseases, announced that CEO Emil D. Kakkis will present at the 39th Annual J.P. Morgan Virtual Healthcare Conference. The presentation is scheduled for January 12, 2021, at 2:50 PM EST. The live webcast will be accessible from their website, with a replay available for 30 days. Ultragenyx aims to develop novel therapies to address high unmet medical needs in rare diseases, guided by an experienced management team dedicated to efficient drug development.

Ultragenyx Pharmaceutical (Nasdaq: RARE) has entered a collaboration with Mereo BioPharma for setrusumab, a monoclonal antibody targeting osteogenesis imperfecta (OI). Ultragenyx will lead development, paying an upfront fee of $50 million and up to $254 million in milestones. OI affects around 60,000 patients in developed countries, and there are currently no approved therapies. Setrusumab shows promise in increasing bone density and strength, with plans for pediatric studies set to begin in 2021.

Ultragenyx Pharmaceutical (NASDAQ: RARE) announced that the FDA has granted Orphan Drug Designation to UX701 for treating Wilson disease, a rare metabolic disorder. This designation marks an important milestone in UX701's development, addressing a significant unmet need as current treatments often lead to severe side effects. UX701 is an investigational gene therapy aimed at normalizing copper metabolism in the liver. The company plans to file an IND application by the end of the year.

The press release discusses the presentation of data from a Phase 1/2 study of GTX-102, an experimental treatment for Angelman syndrome, at the FAST Global Summit. Key findings indicate initial clinical activity, as reflected in positive EEG results and improvements in communication scores among patients. Importantly, there were no new serious adverse events reported. The companies aim to resume the trial with a modified design to minimize risks observed at higher doses. GTX-102 has received multiple FDA designations, emphasizing its potential in treating this rare disease.