Ultragenyx Pharmaceutical, Inc. ( RARE Quick Quote RARE - Free Report) reported a second-quarter 2021 adjusted net loss of $1.35 per share (excluding change in fair value of equity investments), comparing unfavorably with the year-ago quarter’s net loss of $1.15. The Zacks Consensus Estimate was pegged at a loss of $1.28 per share for the quarter.
On a reported basis, net loss per share came in at $1.81 against earnings of 41 cents in the year-ago quarter.
In the second quarter, Ultragenyx’s total revenues were $87 million, up 40.9% year over year, driven by strong launches of Dojolvi and Crysvita (for the second indication). The top line surpassed the Zacks Consensus Estimate of $83.4 million.
Shares of Ultragenyx have lost 41.6% so far this year compared with the
industry's decline of 1.3%. Image Source: Zacks Investment Research Quarter in Detail
Ultragenyx markets three drugs, namely Crysvita, Mepsevii and Dojolvi (UX007). Crysvita is approved for the treatment of X-linked hypophosphatemia (XLH), 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 $49.6 million, up 32.7% year over year, driven by increased demand for both approved indications. Crysvita revenues in Ultragenyx territories rose 38% to $44.7 million in the quarter and included $41.8 million from the North America profit share territory as well as $2.9 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 $4.9 million. Ultragenyx sold its Crysvita rights in the European territory to Royalty Pharma in December 2019.
Mepsevii product revenues were $5.4 million in the quarter, up 29% year over year. Dojolvi (UX007) product revenues were $10 million compared with $1.33 million in the year-ago quarter, driven by strong new patient demand. Revenues in the quarter also included $21.9 million in relation to the collaboration and license agreement with Daiichi Sankyo for Ultragenyx’s proprietary AAV-based gene therapy manufacturing technologies, which were executed in March 2020.
Operating expenses rose 36% to $169.8 million in the quarter due to pipeline advancements.
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-$190 million in the Ultragenyx territories. R&D and SG&A costs are expected to increase in 2021 as the company supports its expanding pipeline and the launches of Crysvita, Dojolvi and Mepsevii.
Crysvita was approved by European Commission (EC) for self-administration as a treatment option for pediatric and adult patients with XLH.
An investigational new drug (IND) application for UX701, an AAV9 gene therapy for Wilson disease, which is a larger rare metabolic disease, was cleared by the FDA in the first quarter. The company expects to initiate a seamless single-protocol phase I/II/III study in the second half of 2021.
In March 2021, the regulatory body cleared the investigational new drug application for its first mRNA program, UX053, to treat Glycogen Storage Disease Type III (GSDIII). The company plans to begin enrollment in a phase I/II study for evaluating the same in the second half of 2021.The candidate was recently granted an orphan drug designation in both the United States and Europe.
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, an mRNA program for Glycogen Storage Disease Type Ia and GTX-102, an antisense oligonucleotide for Angelman Syndrome with partner GeneTx. Phase III studies on both DTX401 and DTX301 are expected to begin in the second half of 2021.
Zacks Rank & Stocks to Consider
Ultragenyx is currently a Zacks Rank #3 (Hold) stock. Some better-ranked stocks in the biotech sector are
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