Ultragenyx Pharmaceutical (RARE - Free Report) reported loss per share of $1.74 in the third quarter of 2018, which was narrower than the loss of $1.87 in the year-ago quarter. Loss was also narrower than the Zacks Consensus Estimate loss of $1.94.
For the third quarter, Ultragenyx reported $11.8 million in total revenues. Revenues beat the Zacks Consensus Estimate of $11.2 million.
Total revenues included $3.6 million received from Bayer (BAYRY - Free Report) in relation to Ultragenyx’s research agreement with the former to develop adeno-associated virus gene therapies. Ultragenyx recognized $5.4 million in profit sharing and royalty revenues from Japanese collaboration partner Kyowa Hakko Kirin for Crysvita. Mepsevii product revenues were $2.1 million, and UX007 revenues were $0.4 million.
Please note that though UX007 is not an approved product, the company recognizes sales from the candidate on a “named patient” basis. This is allowed in certain countries, prior to the commercial approval of a product.
We remind investors that the FDA approved Crysvita in April for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients aged one year or older. Further, Mepsevii, an enzyme replacement therapy, is the first and the only medicine approved for the treatment of children and adults with mucopolysaccharidosis VII (MPS VII) in the United States.
Shares of Ultragenyx increased 14.2% year to date, against the industry’s decline of 16.6%.
The company submitted regulatory filings in Canada, Brazil and Colombia for Crysvita, for the treatment of XLH. Regulatory decisions in these markets are expected in 2019.
The company is in discussions with the FDA regarding the regulatory pathway for Crysvita, for the treatment of Tumor-Induced Osteomalacia (TIO).
The European Commission (EC) approved the Marketing Authorization Application (MAA) for Mepsevii, under exceptional circumstances for the treatment of non-neurological manifestations of MPS VII. Mepsevii is now approved for use in all 28 EU countries along with Iceland, Liechtenstein and Norway. The drug has also been launched in Germany.
The FDA accepted Ultragenyx’s proposal to submit a new drug application (NDA) for UX007, for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD) based on currently available data. The company will provide further details following a pre-NDA meeting, which is scheduled to take place by the end of 2018. Additionally, discussions are progressing with the European Medicines Agency, regarding a potential conditional marketing authorization in Europe.
In October 2018, Ultragenyx announced that the phase III study, evaluating UX007 in patients with Glut1 DS, did not achieve its primary endpoint compared to placebo. The company plans to discontinue further Glut1 DS clinical development for UX007, and expects no impact on plans for the LC-FAOD indication.
Positive top-line data from the first two dose cohorts of the phase I/II study of DTX301 gene therapy in ornithine transcarbamylase (OTC) deficiency showed normalization of ureagenesis in two patients and further support proof-of-concept.The first patient in cohort 3 has been dosed. Data from higher dose cohort 3 are expected in mid-2019.
Ultragenyx exercised its option with REGENXBIO Inc. (RGNX - Free Report) to develop a gene therapy to treat patients with CDKL5 Deficiency Disorder (CDD), using REGENXBIO’s adeno-associated virus (AAV) vectors including AAV9.
Zacks Rank & Other Stock to Consider
Ultragenyx currently carries a Zacks Rank #2 (Hold).
A better-ranked stock worth considering is Bristol-Myers Squibb Company (BMY - Free Report) , sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Bristol-Myers’ earnings per share estimates have increased from $3.59 to $3.81 for 2018 and from $3.83 to $4.03 for 2019 over the past 60 days. The company delivered a positive earnings surprise in all of the trailing four quarters with an average beat of 11.99%.
Today's Stocks from Zacks' Hottest Strategies
It's hard to believe, even for us at Zacks. But while the market gained +21.9% in 2017, our top stock-picking screens have returned +115.0%, +109.3%, +104.9%, +98.6%, and +67.1%.
And this outperformance has not just been a recent phenomenon. Over the years it has been remarkably consistent. From 2000 - 2017, the composite yearly average gain for these strategies has beaten the market more than 19X over. Maybe even more remarkable is the fact that we're willing to share their latest stocks with you without cost or obligation.
See Them Free>>