Ultragenyx Pharmaceutical, Inc. (RARE Quick QuoteRARE - Free Report) reported first-quarter 2021 loss per share of $2.03 compared with a loss of $2.05 in the year-ago quarter. The Zacks Consensus Estimate was pegged at a loss of $1.25 per share.

In the first quarter, Ultragenyx reported total revenues of $99.4 million, up 173.7% from the year-ago quarter driven by strong launches for Dojolvi and Crysvita for the second indication. The top line surpassed the Zacks Consensus Estimate of $75 million.

Shares of Ultragenyx have lost 26.4% so far year compared with the industry's decline of 3.0%.

Ultragenyx markets three drugs, Crysvita, Mepsevii and Dojolvi (UX007). Crysvita is approved for the treatment of X-linked hypophosphatemia, an inherited disorder and tumor induced osteomalacia (TIO), an ultra-rare disease. Mepsevii is approved to treat Mucopolysaccharidosis VII (MPS VII), also known as Sly syndrome. Dojolvi was approved last year for all forms of long-chain fatty acid oxidation disorders (LC-FAOD).

Crysvita’s total revenues were $46.0 million, up 46.3% year over year driven by increased demand for both approved indications. Crysvita revenues in Ultragenyx territories rose 46% to $42.1 million in the quarter and included $36.3 million from the North America profit share territory and $5.9 million of net product sales for the drug in other regions. Total royalty revenues related to the sales of Crysvita in the European Territory were $3.9 million Ultragenyx sold its Crysvita rights in the European territory to Royalty Pharma in December 2019.

Mepsevii product revenues were $3.6 million in the quarter, up 5.3% year over year. Dojolvi (UX007) product revenues were $7.0 million versus $1.44 million in the year-ago quarter driven by strong new patient demand. Revenues in the quarter also included $42.8 million related to the collaboration and license agreement with Daiichi Sankyo for Ultragenyx’s proprietary AAV-based gene therapy manufacturing technologies, which was executed in March 2020.

Operating expenses rose 31% to $206 million in the quarter.

2021 Guidance

The company reaffirmed the guidance for Crysvita which it provided at the beginning of 2021 when it released preliminary sales numbers for the drug. This range is $180 million to $190 million in the Ultragenyx territories, representing growth of 30% to 37% year over year. R&D and SG&A costs are expected to increase in 2021 as the company supports its expanding pipeline and launches of Crysvita, Dojolvi and Mepsevii

Pipeline Update

UX007 is currently in a phase III study in patients with glucose transporter type-1 deficiency syndrome (Glut1 DS), a brain energy deficiency, who are experiencing movement disorders.

An investigational new drug (IND) application for UX701, a AAV9 gene therapy for Wilson disease, a larger rare metabolic disease, was submitted in January 2021. The company expects to initiate a seamless single-protocol phase I/II/III study in the first half of 2021. 

In March 2021, the FDA cleared the investigational new drug application for its first mRNA program, UX053, for the treatment of Glycogen Storage Disease Type III (GSDIII). The company plans to begin enrollment in a phase I/II study evaluating in the second half of 2021.

The company also has three gene therapy candidates in its pipeline. These include DTX301, an AAV8 gene therapy for the treatment of Ornithine Transcarbamylase, or OTC, Deficiency, DTX401, a mRNA program for Glycogen Storage Disease Type Ia, and GTX-102, an antisense oligonucleotide for Angelman Syndrome with partner GeneTx. Phase III studies for both DTX401 and DTX301 are expected to begin in the second half of 2021.

Zacks Rank & Stocks to Consider

Ultragenyx is a Zacks Rank #4 (Sell) stock.