Ultragenyx Reports Fourth Quarter and Full Year 2018 Financial Results and Corporate Update
“This last year was important for Ultragenyx as we successfully launched two therapies internationally and validated clinical data from our gene therapy platform,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “In 2019 we look forward to expanding the global commercial reach of our approved therapies, submitting a New Drug Application for a third, and advancing our gene therapy platform toward pivotal studies.”
Fourth Quarter and Full Year 2018 Financial Results
For the fourth quarter of 2018, Ultragenyx reported
Net revenue for the year ended
Total operating expenses for the fourth quarter of 2018 were
For the fourth quarter of 2018, Ultragenyx reported a net loss of
Cash, Cash Equivalents and Investments
Cash, cash equivalents and investments were
Crysvita in X-Linked Hypophosphatemia (XLH)
- Longer-term data from the Phase 3 pediatric study demonstrated superior efficacy of Crysvita compared with conventional oral phosphate treatment. After 64 weeks of treatment, patients treated with Crysvita continued to show significantly greater improvement in healing of rickets, growth, and bowing of the legs compared with patients treated with oral phosphate and active Vitamin D, which was the standard of care for over 30 years. The 64-week safety profile was similar to that observed at 40 weeks and in other Crysvita pediatric XLH studies.
- Crysvita was approved and launched in
Canadafor the treatment of XLH in adults and pediatric patients one year of age and older. Canadais included in the North American profit share agreement with our partner Kyowa Hakka Kirin.
UX007 in Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
- Positive topline data from ongoing long-term extension study of UX007 for the treatment of LC-FAOD showed sustained reductions in the duration and frequency of major clinical events (MCEs), primarily hospitalizations for hypoglycemia, cardiomyopathy, and rhabdomyolysis. Patients who had previously been enrolled in the Company-sponsored Phase 2 study (n=24) demonstrated sustained reductions in MCEs after an additional 78 weeks of treatment, and 20 additional patients who had not previously received UX007 demonstrated similar substantial reductions in these MCEs with treatment, further corroborating prior results. The safety profile was consistent with what has been previously observed with UX007.
DTX401 Gene Therapy in Glycogen Storage Disease Type Ia (GSDIa)
- Positive topline results from the first cohort of the Phase 1/2 clinical study of DTX401 gene therapy in GSDIa. All three patients demonstrated a clinical response, reflected by improved glucose control, increased time to hypoglycemia during fasting and reductions in necessary cornstarch dosing. Two patients demonstrated a clinically meaningful improvement in time to hypoglycemia, making it possible to sleep through the night. Typically, for GSDIa patients, cornstarch or tube feeding is required during the night to prevent severe hypoglycemia, which can cause seizures or death. There were no infusion-related adverse events and no treatment-related serious adverse events reported.
Upcoming Key Milestones
UX007 in LC-FAOD
- Ultragenyx is on track to submit a New Drug Application (NDA) to the
U.S. Food and Drug Administration( FDA) in mid-2019. The submission will include data from the company-sponsored Phase 2 study in 29 patients, data from the long-term efficacy and safety extension study in 75 patients, a retrospective medical record review of 20 original compassionate use patients, data from 70 patients treated through expanded access, and a randomized controlled investigator-sponsored study of 32 patients showing an effect of UX007 on cardiac function.
DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency
- Enrollment is ongoing in the third-dose cohort of the Phase 1/2 study. Cohort 3 data expected in mid-2019.
DTX401 Gene Therapy in GSDIa
- Enrollment is ongoing in the second-dose cohort of the Phase 1/2 study. Cohort 2 data expected in mid-2019.
- Ultragenyx plans to host an Analyst Day on
Wednesday, April 17. The event will provide updates and expert commentary on commercial, clinical stage and early pipeline programs. Additional details to come.
Conference Call and 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 serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.
Crysvita® (burosumab) is approved by the U.S.
In addition to the approved treatments for XLH and MPS VII, Ultragenyx has four clinical development programs. Crysvita is being studied for the treatment of TIO, a rare disease that impairs bone mineralization. UX007 is being studied in patients with LC-FAOD, a genetic disorder in which the body is unable to convert long chain fatty acids into energy. The company has two gene therapy pipeline candidates: DTX301 is an AAV8 gene therapy product candidate in development for the treatment of patients with OTC deficiency, the most common urea cycle disorder; and DTX401 is an AAV8 gene therapy product candidate for the treatment of patients with GSDIa.
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 related to Ultragenyx's expectations regarding plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions 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 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 products and 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 Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the
Investors & Media
|Ultragenyx Pharmaceutical Inc.|
|Selected Statement of Operations Financial Data|
|(in thousands, except share and per share amounts)|
|Three Months Ended December 31,||Year Ended December 31,|
|Statement of Operations Data:|
|Collaboration and license||$||12,797||$||2,136||$||41,693||$||2,136|
|Cost of sales||507||1||1,146||1|
|Research and development||71,618||61,527||293,998||231,644|
|Selling, general and administrative||34,476||37,720||127,724||99,909|
|Total operating expenses||106,601||99,248||422,868||331,554|
|Loss from operations||(90,340)||(96,834)||(371,373)||(328,942)|
|Gain from sale of priority review vouchers||—||—||170,322||—|
|Other income (expense), net||2,640||(1,114)||3,954||10,604|
|Loss before income taxes||(87,700)||(97,948)||(197,097)||(318,338)|
|Benefit from (provision for) income taxes||(126)||16,217||(514)||16,199|
|Net loss per share, basic and diluted||$||(1.73)||$||(1.89)||$||(3.97)||$||(7.12)|
|Shares used in computing net loss per share, basic and diluted||50,694,007||43,137,679||49,775,223||42,453,135|
|Ultragenyx Pharmaceutical Inc.|
|Selected Balance Sheet Financial Data|
|December 31,||December 31,|
|Balance Sheet Data:|
|Cash, cash equivalents and investments||$||459,706||$||244,468|
|Total stockholders' equity||608,908||383,454|
Source: Ultragenyx Pharmaceutical Inc.