Ultragenyx Pharm Stock Price, News & Analysis

Ultragenyx Pharmaceutical (NASDAQ: RARE) announced the grant of stock options and restricted stock units to a new non-executive officer. The compensation committee approved 4,677 stock options and 7,100 restricted stock units under the Ultragenyx Employment Inducement Plan, effective May 16, 2021. The restricted stock units vest over four years, while stock options vest 25% after the first year and monthly thereafter. The exercise price of the stock options is set at $110.88, reflecting the closing price on May 14, 2021. Ultragenyx focuses on novel therapies for rare diseases.

Ultragenyx Pharmaceutical (NASDAQ: RARE) reported positive long-term results from Phase 1/2 trials for its gene therapies DTX401 (for GSDIa) and DTX301 (for OTC deficiency). DTX401 showed durable responses lasting over 2.5 years with a 79% reduction in cornstarch intake, while DTX301 demonstrated stability after more than three years. No serious adverse events were reported in either study. The company is set to initiate Phase 3 studies for both therapies later in 2021, enhancing its proprietary HeLa cell line manufacturing platform, which has improved product yield significantly.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that Mardi Dier, Chief Financial Officer, and Camille Bedrosian, M.D., Chief Medical Officer, will present at the BofA Securities Healthcare Conference on May 12, 2021, at 3:30 PM ET. The live webcast can be accessed from the company's website, with a replay available for 90 days. Ultragenyx focuses on developing therapies for rare genetic diseases and has a diverse portfolio of approved medicines aimed at addressing high unmet medical needs.

Ultragenyx Pharmaceutical reported first quarter 2021 revenue of $99.4 million, with Crysvita revenue in its territories at $42.1 million, marking a 46% increase year-over-year. The company reaffirmed its 2021 Crysvita revenue guidance of $180 million to $190 million. The Dojolvi launch shows strong momentum, currently serving around 180 patients. Operating expenses rose by 31% to $206 million, primarily due to an upfront payment related to a collaboration. As of March 31, 2021, Ultragenyx holds $1.0 billion in cash and equivalents.

Ultragenyx Pharmaceutical (NASDAQ: RARE) announced that Emil Kakkis, M.D., Ph.D., will present at the 7th Annual Truist Securities Life Sciences Summit on May 5, 2021, at 1:00 PM ET. The live and archived webcast of the presentation can be accessed through the company's website. Ultragenyx is dedicated to developing therapies for rare genetic diseases, focusing on addressing significant unmet medical needs. The company employs a management team experienced in creating effective treatments efficiently and urgently. For more details, visit www.ultragenyx.com.

Ultragenyx Pharmaceutical (NASDAQ: RARE) announced a conference call scheduled for May 4, 2021, at 5 PM ET, to discuss its Q1 financial results for the period ending March 31, 2021. The call will be accessible via the company website and phone. Ultragenyx focuses on developing therapies for serious rare and ultra-rare genetic diseases and has a diverse portfolio of approved treatments. The management team emphasizes efficient drug development to meet urgent patient needs.

Ultragenyx Pharmaceutical (NASDAQ: RARE) announced it will present data at the American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting, held virtually from May 11-14, 2021. The presentations will cover their investigational gene therapy programs, including advancements in their HeLa producer cell line for AAV production, and long-term results from Phase 1/2 studies on Ornithine Transcarbamylase (OTC) Deficiency and Glycogen Storage Disease Type Ia (GSDIa). Key presentations are scheduled for May 11, 13, and 14.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced the completion of an End-of-Phase 2 meeting with the FDA for its DTX301 gene therapy aimed at treating ornithine transcarbamylase (OTC) deficiency. The finalized Phase 3 study design includes a 64-week efficacy analysis with approximately 50 patients. Co-primary endpoints will assess plasma ammonia levels and patient response. The trial is set to begin in the second half of 2021, with all initial placebo patients eligible for treatment afterward. DTX301 has shown promising results in earlier trials and has Orphan Drug Designation in both the U.S. and Europe.

The n-Lorem Foundation has partnered with Ultragenyx Pharmaceutical (NASDAQ:RARE) to provide free, lifetime supplies of individualized RNA-targeted medicines for patients with ultra-rare diseases. This collaboration aims to enhance n-Lorem's mission of delivering potentially life-saving treatments for conditions that affect 1 to 30 patients globally. Both organizations aim to expedite access to treatments using advanced genomic diagnostics and antisense oligonucleotides (ASOs). The partnership promises to bring hope and rapid treatment for patients suffering from rare genetic disorders.