Ultragenyx Pharmaceutical Inc. ( RARE Quick Quote RARE - Free Report) reported preliminary 2019 revenues and provided 2020 revenue guidance for one of its lead drugs, Crysvita. Crysvita is an antibody targeting fibroblast growth factor 23 (FGF23), approved in the United States for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients. It is approved in the EU for the treatment of XLH with radiographic evidence of bone disease in children one year of age and older, and adolescents with growing skeletons.
Shares of the company went up 6.6% following the announcement of the preliminary results. However, shares have gained 18% in the past year against the
industry’s decline of 2.4%.
For 2019, Ultragenyx’s preliminary total revenues were $102-$104 million. The Zacks Consensus Estimate for 2019 revenues is $101.37 million. Preliminary Crysvita revenues were $86-$88 million in 2019. This includes collaboration revenues in the North American profit share territory, royalty revenues in the European territory from partner Kyowa Kirin Co. Ltd. and product revenues from Crysvita in other regions.
For 2020, the company expects Crysvita revenues of $125-$140 million. This, however, excludes the European territory royalty revenues, as Ultragenyx sold its royalty interest in Crysvita in the region to Royalty Pharma on Jan 1, 2020, for $320 million.
The company has been in the news, lately, for several reasons.
The company submitted a supplemental biologics license application (sBLA) to the FDA on Dec 18, 2019, for the label expansion of Crysvita. The sBLA is seeking approval for the drug for the treatment of Tumor-Induced Osteomalacia (TIO). The company expects to hear back from the FDA on submission acceptance and review designation in February 2020.
Meanwhile, the FDA accepted for review Ultragenyx’s new drug application (NDA) for UX007 for the treatment of Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD). The FDA set an action date of Jul 31, 2020. The agency indicated that it is currently not planning to hold an advisory committee meeting to discuss the application.
Earlier this week, Ultragenyx announced positive top-line safety and efficacy data from the ongoing phase I/II study of an investigational adeno-associated virus (AAV) gene therapy — DTX301. DTX301 is being evaluated for the treatment of ornithine transcarbamylase (OTC) deficiency. The company announced positive safety and efficacy data from Cohort 3 and longer-term data from Cohort 2. It expects to start the fourth cohort to evaluate prophylactic steroid administration of DTX301 in the first half of 2020. The data from the fourth cohort are expected in the second half of the year.
The company expects to file an investigational new drug (IND) application for GTX-102 in Angelman Syndromein the first half of 2020. The company also expects to file an IND for new gene therapy, UX701, for the treatment of Wilson disease, a larger, rare, metabolic disease in the first half of 2020.
Zacks Rank & Stocks to Consider
Ultragenyx is currently a Zacks Rank #3 (Hold) stock.
A few better-ranked stocks in the medical drugs sector are Alexion Pharmaceuticals Inc.
ALXN, ASLAN Pharmaceuticals Ltd. ASLN and Celsion Corporation CLSN,all sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here .
Alexion’s’ earnings per share estimates have increased from $9.97 to $10.35 for 2019 and from $10.85 to $11.16 for 2020 in the past 90 days. The company delivered a positive earnings surprise in the trailing four quarters by 12.50%, on average.
ASLAN’s loss per share estimates have narrowed from 69 cents to 67 cents for 2019 and from 62 cents to 61 cents for 2020 in the past 90 days. The company delivered a positive earnings surprise in three of the trailing four quarters by 24.65%, on average.
Celsion’s loss per share estimates have narrowed from $1.00 to 94 cents for 2019 and from 77 cents to 66 cents for 2020 in the past 90 days. The company delivered a positive earnings surprise in three of the trailing four quarters by 51.11%, on average.
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