Marin County’s Ultragenyx Pharmaceutical expands loss in Q3 as R&D spending jumps

Ultragenyx Pharmaceutical Inc. (Nasdaq: RARE) on Tuesday reported bigger third-quarter and nine-month losses from a year before amid higher research-and-development spending on its pipeline of treatments for rare diseases.

The Novato-based biotechnology company posted a loss of $72.8 million, or $1.08 a share, for the quarter ended Sept. 30, up 6% from $68.5 million, or $1.13 a share, a year before. The three-quarter loss expanded to $331.6 million, or $4.91 a share, more than double the net loss of $162.5 million, $2.73 a share, from 2020.

Quarterly spending on R&D was up 30% annually, to $113.4 million, and for the first nine months such outlays were up by one-third, to $374.1 million.

“In the third quarter we executed on key commercial and clinical milestones, including the resumption of the GTX-102 study for the treatment of Angelman syndrome and the initiation of our seamless Phase 1/2/3 study of UX701 for the treatment of Wilson disease. Looking ahead, we are preparing to initiate four additional studies across multiple modalities, including two Phase 3 gene therapies, a Phase 2/3 anti-sclerostin monoclonal antibody, and a Phase 1/2 leveraging our first mRNA program,” said Emil D. Kakkis, M.D., Ph.D., CEO and president of Ultragenyx, in the announcement. “The breadth of our clinical programs with the strength of our balance sheet put us in position to deliver disease-modifying therapies across a spectrum of rare diseases.”

The quarterly results exceeded Wall Street expectations, the Associated Press reported. The average estimate of 11 analysts surveyed by Zacks Investment Research was for a loss of $1.43 per share. Eight analysts surveyed by Zacks expected revenue of $78.2 million.