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Ultragenyx Pharmaceutical: Ultragenyx Reports Third Quarter 2015 Financial Results And Corporate Update

The following excerpt is from the company's SEC filing.

NOVATO, CA November 9, 2015

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today reported its financial results and corporate update for the quarter ended September 30, 2015.

We have continued to make progress across our product portfolio, including filing for conditional approval in Europe with Ace-ER, reporting interim data for UX007 in long chain fatty acid oxidation disorders (LC-FAOD) and in five infants with cardiomyopathy due to LC-FAOD, announcing plans to initiate a Phase 3 study with UX007 in Glut1 DS patients with the movement disorder phenotype, and initiating a multi-target collaboration for messenger RNA (mRNA) therapeutics, said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. We look forward to closing out the year with the 36-patient data from the Phase 2 study of KRN23 in pediatric XLH patients and Ultragenyxs first R&D day in New York on December 3.

Third Quarter 2015 Financial Results

For the third quarter of 2015, Ultragenyx reported a net loss attributable to common stockholders of $39.2 million, or $1.03 per share, basic and diluted, compared with a net loss attributable to common stockholders for the third quarter of 2014 of $15.8 million, or $0.50 per share, basic and diluted. For the nine months ended September 30, 2015, net loss attributable to common stockholders was $90.4 million, or $2.51 per share, basic and diluted, compared with a net loss attributable to common stockholders for the same period in 2014 of $47.9 million, or $1.73 per share, basic and diluted. Net loss attributable to common stockholders differs from net loss due to dividends and other charges related to outstanding preferred stock, which was converted into common stock upon the companys initial public offering.

Total operating expenses for the third quarter of 2015 were $39.9 million compared with $15.8 million for the same period in 2014. Non-cash stock-based compensation accounted for $7.9 million and $1.7 million of total operating expenses in the third quarter of 2015 and 2014, respectively. Total operating expenses for the nine months ended September 30, 2015 were $91.6 million compared with $39.8 million for the same period in 2014. Non-cash stock-based compensation accounted for $15.4 million and $3.4 million of total operating expenses in the first nine months of 2015 and 2014, respectively. The increase in total operating expenses is due to the increase in development, commercial, and general and administrative costs as the company grows and advances its pipeline, as well as related increases in stock compensation expenses.

Cash, cash equivalents, and investments were $581.9 million as of September 30, 2015.

Recent Highlights & Upcoming Milestones

Corporate Updates

Research collaboration and license agreement initiated for messenger RNA (mRNA) therapeutics

. Ultragenyx entered into an agreement with Arcturus Therapeutics, Inc. to develop mRNA therapeutics for two selected rare disease targets, with the option to expand to eight additional targets. The collaboration is intended to address a wider range of rare diseases than is possible with currently available enzyme replacement therapy approaches.

Research & Development Day to be held on December 3, 2015

. The company will hold its first Research & Development Day for analysts and investors on Thursday, December 3, 2015 at 12:30 p.m. ET in New York, NY. An update on the companys pipeline will be provided.

KRN23 anti-FGF23 Monoclonal Antibody in X-Linked Hypophosphatemia (XLH) and Tumor-Induced Osteomalacia (TIO)

Phase 2 40-week data in 36 pediatric XLH patients expected by the end of 2015

. Safety and efficacy data, including rickets scores, from 36 patients in the pediatric Phase 2 study are expected by year-end. The study has been expanded to enroll a total of 52 patients, and 40-week results for the fully expanded study are expected in mid-2016. Based on the results of the 36-patient data, the company will make a determination on filing for conditional approval in Europe based on prior scientific advice received.

Phase 3 program in adult XLH patients expected to initiate by year-end 2015

. Ultragenyx continues initiation activities for the Phase 3...


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