Ultragenyx Pharm Stock Price, News & Analysis

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) has announced plans to construct a 100,000 square foot gene therapy manufacturing facility in Bedford, Massachusetts. This state-of-the-art facility will allow in-house production of their AAV-based gene therapies, including DTX301, DTX401, and UX701. The facility is projected to create 100 to 150 jobs over five years. Construction is underway, with completion expected in 2023. The facility will improve production processes and scalability to meet demand for Ultragenyx's clinical and approved products.

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced that CEO Emil D. Kakkis will present at two upcoming investor conferences: the Credit Suisse 29th Annual Virtual Healthcare Conference on November 10, 2020, at 2:00 PM ET, and the Barclays Gene Editing & Gene Therapy Summit on November 16, 2020, at 3:00 PM ET. Live and archived webcasts of the presentations will be available on the company's website for 90 days. Ultragenyx focuses on developing therapies for serious rare and ultra-rare genetic diseases, with an experienced management team committed to urgent and efficient drug development.

Ultragenyx Pharmaceutical (NASDAQ:RARE) has successfully completed an underwritten public offering of 5,111,110 shares of common stock at $90.00 per share, raising approximately $435.4 million in net proceeds after underwriting costs. The offering included the full exercise of an option for an additional 666,666 shares by underwriters. J.P. Morgan, Goldman Sachs, BofA Securities, and Cowen served as joint book-running managers for this offering. Ultragenyx focuses on developing novel therapies for rare genetic diseases, reinforcing its commitment to urgent drug development.

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) has announced the pricing of an underwritten public offering of 4,444,444 shares at $90.00 per share, aiming to raise approximately $400 million in gross proceeds. The offering's close is anticipated around November 2, 2020, subject to customary closing conditions. Underwriters include J.P. Morgan, Goldman Sachs, BofA Securities, and Cowen, with an option to purchase an additional 666,666 shares. This strategic capital raise aligns with the company's commitment to developing therapies for rare genetic diseases.

Ultragenyx Pharmaceutical (NASDAQ:RARE) announced a public offering of up to $400 million in common stock, with an option for underwriters to purchase an additional $60 million within 30 days. The offering is subject to market conditions and does not guarantee completion. Major banks involved include J.P. Morgan, Goldman Sachs, BofA Securities, and Cowen. The funds raised will be utilized for the development of therapies aimed at serious rare genetic diseases, reinforcing Ultragenyx's commitment to addressing high unmet medical needs.

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.