Ultragenyx Reports First Quarter 2018 Financial Results and Corporate Update
“With the recent approvals and launches of Crysvita in
First Quarter 2018 Financial Results
For the first quarter of 2018, Ultragenyx reported net income of
For the first quarter of 2018, Ultragenyx reported
Cash, cash equivalents, and investments were
Crysvita® (burosumab) in X-Linked Hypophosphatemia (XLH)
- In the U.S., Crysvita was approved on
April 17, 2018and is now commercially available to adults and children with X-linked hypophosphatemia (XLH). In April, the U.S. Food and Drug Administration( FDA) approved Crysvita for the treatment of XLH in adult and pediatric patients one year of age and older. The first patient has now received commercial treatment with Crysvita.
Europe, burosumab received conditional marketing authorization for the treatment of XLH with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons.
DTX301 Gene Therapy in ornithine transcarbamylase (OTC) Deficiency
- Data from Phase 1/2 study of DTX301, our AAV8 vector in patients with OTC, showed positive topline results including normalization of ureagenesis in one patient in the first, lowest-dose cohort. The first patient’s rate of ureagenesis was normalized, maintained and then substantially increased over 24 weeks. The second and third patients did not show a clinically meaningful change in rate of ureagenesis over 20 weeks and 12 weeks, respectively. There have been no infusion-related adverse events and no serious adverse events reported. All adverse events have been Grade 1 or 2 and have resolved. Two patients have been enrolled in the higher dose Cohort 2 portion of the study, and data from the second cohort are expected in the second half of 2018.
DTX401 Gene Therapy in glycogen storage disease type Ia (GSDIa)
- The U.S.
FDAcleared the Investigational New Drug (IND) application for DTX401 for the treatment of patients with GSDIa. Enrollment in the Phase 1/2 study is expected to begin in the first half of 2018, with data from the first cohort in the second half of 2018.
- PRV sold for
$130 million: In January 2018, we completed the sale of the PRV that we received at the time of the approval of Mepsevii.
- Equity financing of approximately
$271.0 million: In January 2018, we completed an underwritten public offering, with net proceeds of approximately $271.0 million.
Upcoming Key Milestones
Crysvita (burosumab) in XLH
- Data from the Phase 3 study in pediatric patients expected in the second half of 2018. The ongoing Phase 3 randomized open-label clinical study is comparing the efficacy and safety of burosumab to oral phosphate and active vitamin D therapy in pediatric patients with XLH. This study will serve as a confirmatory study in
Crysvita (burosumab) in tumor-induced osteomalacia (TIO)
- Data from all patients in Phase 2 study in TIO expected in mid-2018. This is an open label Phase 2 study evaluating the safety and efficacy of burosumab in 17 adult patients with TIO.
Mepsevii (vestronidase alfa) in mucopolysaccharidosis VII (MPS VII)
Europe, an opinion from the Committee for Medicinal Products for Human Use(CHMP) is expected in mid-2018.
UX007 in long-chain fatty acid oxidation disorders (FAOD) and glucose transporter type-1 deficiency syndrome (Glut1 DS)
- Completing study design of Phase 3 study in patients with FAOD; providing additional data to
FDAfor consideration of early filing based on Phase 2 data. Following an end-of-phase 2 meeting, we are providing additional information to submit to FDAfor consideration of an early filing based on the results from the Phase 2 study. While the FDAstill prefers that a randomized controlled trial be completed before filing, it left open the possibility of filing on the current data. We are simultaneously completing the design of a Phase 3 study that could be used for registration or confirmatory purposes. We expect that a decision on a potential filing for approval based on Phase 2 data will be made in mid-2018.
- Data from the Phase 3 movement disorder study in patients with Glut1 DS. Enrollment is complete and data are expected in the second half of 2018.
Conference Call & Webcast Information
Ultragenyx will host a conference call today,
Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies.
Crysvita® (burosumab) is approved by the U.S.
Ultragenyx is conducting Phase 2 and Phase 3 studies of burosumab in patients with XLH and TIO, both rare diseases that impair bone mineralization; a Phase 2 clinical study of UX007 in patients severely affected by LC-FAOD, a genetic disorder in which the body is unable to convert long chain fatty acids into energy; a Phase 3 study for UX007 in patients with Glut1 DS, a brain energy deficiency, who are experiencing movement disorders; a Phase 1/2 study of DTX301 gene therapy in OTC deficiency, a urea cycle disorder that causes ammonia to accumulate in the blood; and a Phase 1/2 study of DTX401 gene therapy in GSDIa, a disease that arises from a defect in G6Pase, an essential enzyme in glycogen and glucose metabolism.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company’s website at www.ultragenyx.com.
Except for the historical information contained herein, the matters set forth in this press release, including statements regarding Ultragenyx's expectations regarding ongoing or additional studies for its product candidates and timing regarding these studies, the design of clinical studies, the demonstrated impact of clinical data and other information to support approval of product candidates, potential indications for its product candidates, discussions with regulatory authorities, the potential issuance of a priority review voucher, sufficiency for, and timing of, regulatory submissions and approvals, and the timing and locations of commercialization efforts are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of our regulatory filings and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of the company in general, see Ultragenyx's Annual Report on Form 10-K filed with the
|Ultragenyx Pharmaceutical Inc.|
|Selected Statement of Operations Financial Data|
|(in thousands, except share and per share amounts)|
|Three Months Ended March 31,|
|Statement of Operations Data:|
|Collaboration and license||$||9,362||$||—|
|Cost of goods sold||225||—|
|Research and development||75,504||51,269|
|Selling, general and administrative||31,435||18,685|
|Total operating expenses||107,164||69,954|
|Loss from operations||(96,487||)||(69,954||)|
|Gain from sale of priority review voucher||130,000||—|
|Other income (expense), net||(3,221||)||1,664|
|Income (loss) before income taxes||30,292||(68,290||)|
|Income tax provision||(39||)||—|
|Net income (loss)||$||30,253||$||(68,290||)|
|Net income (loss) per share:|
|Shares used in computing net income (loss) per share:|
|Ultragenyx Pharmaceutical Inc.|
|Selected Balance Sheets Financial Data|
|March 31,||December 31,|
|Balance Sheet Data:|
|Cash, cash equivalents and investments||$||571,253||$||244,468|
|Total stockholders' equity||723,657||383,454|
Investors & Media
Source: Ultragenyx Pharmaceutical Inc.