Share price of Ultragenyx Pharma RARE has surged 66.9% year to date compared with the industry’s growth of 8.4%. A few factors have led to the price increase.
The company is also looking to expand the label of Crysvita, an antibody targeting fibroblast growth factor 23 (FGF23), which was developed for the treatment of tumor-induced osteomalacia (TIO). In January 2020, the company submitted the supplemental Biologics License Application (sBLA) to the FDA after completing a pre-sBLA meeting with the FDA authorities and signing an agreement on the filing package. Subsequently, the FDA accepted the sBLA under priority review in February 2020, with a decision expected on Jun 18.
Crysvita is approved in the United States for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients. The drug’s performance has been strong. The drug received European conditional marketing authorization, in February 2018, for the treatment of XLH with radiographic evidence of bone disease in children one year of age and older, and adolescents with growing skeletons.
An important pipeline candidate is UX007, developed for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD). A new drug application seeking approval of UX007 for LC-FAOD is under standard review with the FDA, with a decision expected on Jul 31.
During the first-quarter 2020 earnings call, management confirmed that the FDA reviews of Crysvita for TIO and UX007 for LC-FAOD are on track with decisions pending on the scheduled dates.
The company also has some gene therapy candidates in its pipeline. These include DTX301, which is an adeno-associated virus 8 (AAV8) being evaluated for the treatment of patients with Ornithine transcarbamylase (OTC) deficiency. In January, Ultragenyx reported positive top-line results from the safety and efficacy data of Cohort 3 and longer-term data of Cohort 2. In May, the company announced positive longer-term safety and efficacy data from the first three cohorts of the ongoing phase I/II study on DTX301. The company is initiating a fourth cohort (n=3) using prophylactic steroids at the same dose as Cohort 3. Data from the prophylactic steroid cohort are expected in the second half of 2020.
DTX401 is also an AAV8 gene-therapy candidate, currently being evaluated for the treatment of patients with glycogen storage disease type Ia, (GSDIa). In May, Ultragenyx announced positive initial data from the confirmatory third cohort and longer-term data from the first two cohorts of the ongoing phase I/II study of DTX401 for GSDIa. Following the release of the confirmatory cohort report, a phase III study could be initiated in the second half of 2020. The FDA also granted DTX401a Fast Track status for the treatment of GSDIa.
In May, the FDA granted Fast Track designation to its investigational antisense oligonucleotide, GTX-102, for the treatment of Angelman syndrome (AS). The candidate is currently being evaluated in a phase I/II study for the treatment of AS.
Ultragenyx Pharmaceutical Inc. Price
Ultragenyx Pharmaceutical Inc. price | Ultragenyx Pharmaceutical Inc. Quote
Zacks Rank & Stocks to Consider
Ultragenyx currently is a Zacks Rank #3 (Hold) stock.
Some better-ranked stocks in the biotech sector are Akcea Thrapeuticss Inc. AKCA, Axcella Health Inc. AXLA and Aytu BioScience Inc. AYTU, all carrying a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Akcea’s loss per share estimates have narrowed from $2.07 to $1.37 for 2020 and from $2.46 to $1.85 for 2021 in the past 60 days.
Axcella’s loss per share estimates have narrowed from $1.14 to 79 cents for 2020 and from $3.30 to $3.03 for 2021 in the past 60 days.
Aytu’s loss per share estimates have narrowed from $1.26 to 12 cents for 2020 in the past 60 days.
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