2020 whole income of $271.0 million and 2020 Crysvita1 income to Ultragenyx of $138.9 million
2021 Crysvita1 income in Ultragenyx territories steerage of $180 million to $190 million reaffirmed
Sturdy Dojolvi launch continues with roughly 130 sufferers on reimbursed business remedy in the USA
Six packages in scientific trials in 2021, together with three pivotal stage gene remedy trials
NOVATO, Calif., Feb. 11, 2021 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical firm centered on the event and commercialization of novel merchandise for critical uncommon and ultra-rare genetic illnesses, immediately reported its monetary outcomes for the quarter and full yr ended December 31, 2020 and reaffirmed its monetary steerage for 2021.
“Final yr was a transformative yr as we obtained two approvals, generated clinically significant information in our gene remedy and antisense oligonucleotide packages, and accomplished a number of strategic enterprise growth transactions,” mentioned Emil D. Kakkis, M.D., Ph.D., Chief Government Officer and President of Ultragenyx. “Along with rising our world business footprint in 2021, we can even have a powerful, various, late-stage scientific growth portfolio. We’ll provoke three pivotal gene remedy research, a fourth pivotal research with our just lately in-licensed biologic, setrusumab, for osteogenesis imperfecta, make further progress within the Angelman syndrome research, and convey our first mRNA program to the clinic for glycogen storage illness kind III.”
Fourth Quarter and Full 12 months 2020 Monetary Outcomes
For the fourth quarter of 2020, Ultragenyx reported $91.5 million in whole income. Ultragenyx acknowledged $40.4 million in Crysvita (burosumab) income within the Ultragenyx territories, which incorporates $37.5 million in collaboration income within the North American revenue share territory and internet product gross sales in different areas of $2.9 million. Whole royalty income associated to European Crysvita gross sales have been $3.6 million. Dojolvi (triheptanoin) product gross sales within the fourth quarter of 2020 have been $6.4 million. Mepsevii (vestronidase alfa) product gross sales for the fourth quarter of 2020 have been $3.7 million. Whole income for the fourth quarter additionally consists of $37.5 million of income associated to the collaboration and license settlement with Daiichi Sankyo that was executed in March 2020.
Income for the yr ended December 31, 2020 was $271.0 million, together with $138.9 million in Crysvita income within the Ultragenyx territories. Crysvita collaboration income within the North American revenue share territory was $128.6 million and internet Crysvita product gross sales in different areas have been $10.4 million. Whole royalty income associated to European Crysvita royalties was $14.5 million, which incorporates $1.5 million acknowledged on gross sales that occurred previous to January 1, 2020. Dojolvi product income for the yr ended December 31, 2020 was $13.0 million. Mepsevii product income was $15.3 million. For the yr ended December 31, 2020, income associated to our strategic manufacturing partnership with Daiichi Sankyo was $89.2 million.
Whole working bills for the fourth quarter of 2020 have been $187.6 million, together with non-cash stock-based compensation of $22.8 million. This compares to whole working bills for the fourth quarter of 2019, which have been $130.0 million and whole non-cash stock-based compensation of $19.7 million.
Whole working bills for the yr ended December 31, 2020 have been $601.1 million, which incorporates $25.0 million to keep up the choice to accumulate GeneTx, $13.2 million from a one-time in-process R&D expense associated to the strategic collaboration with Strong Biosciences, $7.0 million to license sure vectors from REGENXBIO, and non-cash stock-based compensation of $85.7 million. That is in contrast with $527.9 million for a similar interval in 2019, which features a $15.6 million analysis and growth expense from the Arcturus collaboration modification, $20.0 million for the upfront cost on the GeneTx settlement, and non-cash stock-based compensation of $82.0 million. The rise in whole working bills was as a result of improve in business, on-going scientific and preclinical growth, and common and administrative prices as the corporate commercializes, grows, and advances its portfolio.
For the fourth quarter of 2020, Ultragenyx reported internet lack of $24.0 million, or $0.37 per share primary and diluted, in contrast with a internet loss for the fourth quarter of 2019 of $93.8 million, or $1.62 per share, primary and diluted. For the yr ended December 31, 2020, internet loss was $186.6 million, or $3.07 per share, primary and diluted, in contrast with a internet loss for a similar interval in 2019 of $402.7 million, or $7.12 per share, primary and diluted. The web loss for the fourth quarter of 2020 and the web loss for the yr ended December 31, 2020 features a $79.1 million and $170.4 million improve within the truthful worth of investments in fairness securities, respectively. Web money utilized in operations for the yr ended December 31, 2020 was $132.2 million, in comparison with internet money used of $345.4 million for a similar interval in 2019.
Money, Money Equivalents and Marketable Debt Securities
Money, money equivalents, and marketable debt securities have been $1.2 billion as of December 31, 2020.
2021 Monetary Steering
Crysvita Steering in Ultragenyx Territories
The corporate reaffirms the 2021 steerage vary for Crysvita that was supplied in the beginning of the yr. This vary is $180 million to $190 million for the North American revenue share area and the opposite areas the place product gross sales are acknowledged (Latin America and Turkey).
Program Updates and Upcoming Milestones
Dojolvi for Lengthy-Chain Fatty Acid Oxidation Issues (LC-FAOD): Launched on July 22, 2020
- 5 months into the USA launch of Dojolvi for LC-FAOD, the corporate has obtained roughly 190 accomplished begin kinds from roughly 90 distinctive prescribers. This has led to roughly 130 sufferers on reimbursed remedy as of the top of December, a 117% improve over the prior quarter.
- In Canada, Dojolvi has been granted precedence overview by Well being Canada and a brand new drug submission was filed in August 2020, with a call anticipated quickly. Dojolvi has been submitted to the Brazilian Well being Regulatory Company (ANVISA) looking for advertising authorization. Discussions with the European Medicines Company (EMA) are ongoing. Responses to named affected person remedy requests proceed within the EU.
Three world, pivotal stage gene remedy scientific research are anticipated in 2021
- DTX401 for Glycogen Storage Illness Kind Ia (GSDIa): Following Scientific Recommendation with the EMA and an Finish of Section 2 (EOP2) assembly with the U.S. Meals and Drug Administration (FDA), the corporate presently expects to provoke a Section 3 research within the first half of 2021.
- DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Based mostly on preliminary Scientific Recommendation from the EMA and following a deliberate EOP2 assembly with the FDA, the corporate presently expects to provoke a Section 3 research within the second half of 2021.
- UX701 for Wilson illness: The Investigational New Drug (IND) utility has cleared and this system just lately obtained Quick Observe Designation from the FDA. The corporate presently expects to provoke a seamless, single-protocol Section 1/2/3 research within the first half of 2021.
GTX-102 for Angelman Syndrome, partnered with GeneTx Biotherapeutics: Examine anticipated to renew in first half 2021 with further information anticipated in second half of 2021
- GeneTx, with Ultragenyx help, submitted to the FDA a considerable data modification to the IND together with follow-up security data for the 5 sufferers dosed and toxicology information in nonhuman primates that display no proof of this security problem at greater repeat dosing. GeneTx has obtained suggestions and questions from the FDA primarily based on this submission and GeneTx and Ultragenyx have filed a response to FDA and are engaged on an modification to the protocol.
- A Medical Trial Utility (CTA) has been submitted in Canada and a protocol and knowledge modification just like what has been proposed to FDA might be submitted. GeneTx, with Ultragenyx help, is within the technique of increasing the research to different nations utilizing the amended dosing and administration plan.
- The businesses presently count on the Section 1/2 research to renew enrollment and dosing within the first half of 2021, following decision of FDA requests and approval to proceed.
UX143 (setrusumab) for Osteogenesis Imperfecta, in collaboration with Mereo BioPharma: Section 2/3 in pediatric sufferers anticipated in second half of 2021
- The businesses count on to conduct a Section 2/3 research in pediatric sufferers that first focuses on figuring out the optimum dose primarily based on will increase in bone manufacturing by way of modifications in serum P1NP ranges over two months and an appropriate security profile. Following willpower of the dose, the research is meant to transition right into a pivotal Section 3 research, evaluating fracture discount over an estimated 15 to 24 months. Closing research design and endpoints are pending regulatory overview.
UX053 for Glycogen Storage Illness Kind III or Debrancher deficiency: IND on observe within the first half of 2021
- An IND for the corporate’s first mRNA program, UX053, for the remedy of GSDIII debrancher deficiency is on observe for the primary half of 2021, with a Section 1/2 research anticipated to provoke within the second half of 2021.
1: Ultragenyx territories embody the collaboration income from the North American revenue share territory (U.S. and Canada) and different areas the place income from product gross sales are acknowledged by Ultragenyx (Latin America, Turkey). This excludes the European territory income, which is acknowledged as non-cash royalty income because the rights have been bought to Royalty Pharma in December 2019.
Convention Name and Webcast Data
Ultragenyx will host a convention name immediately, Thursday, February 11, 2021, at 2 p.m. PT/ 5 p.m. ET to debate the fourth quarter and yr 2020 monetary outcomes and supply a company replace. The reside and replayed webcast of the decision might be accessible by way of the corporate’s web site at https://ir.ultragenyx.com/events.cfm. To take part within the reside name by cellphone, dial (855) 797-6910 (USA) or (262) 912-6260 (worldwide) and enter the passcode 6391931. The replay of the decision might be accessible for one yr.
Ultragenyx is a biopharmaceutical firm dedicated to bringing to sufferers novel merchandise for the remedy of significant uncommon and ultra-rare genetic illnesses. The corporate has constructed a various portfolio of permitted therapies and product candidates geared toward addressing illnesses with excessive unmet medical want and clear biology for remedy, for which there are sometimes no permitted therapies treating the underlying illness.
The corporate is led by a administration crew skilled within the growth and commercialization of uncommon illness therapeutics. Ultragenyx’s technique relies upon time- and cost-efficient drug growth, with the purpose of delivering secure and efficient therapies to sufferers with the utmost urgency.
For extra data on Ultragenyx, please go to the corporate’s web site at www.ultragenyx.com.
Aside from the historic data contained herein, the issues set forth on this press launch, together with statements associated to Ultragenyx’s expectations and projections relating to its future working outcomes and monetary efficiency, anticipated value or expense reductions, the timing, progress and plans for its scientific packages and scientific research, future regulatory interactions, and the parts and timing of regulatory submissions are forward-looking statements inside the which means of the “secure harbor” provisions of the Non-public Securities Litigation Reform Act of 1995. Such forward-looking statements contain substantial dangers and uncertainties that might trigger our scientific growth packages, collaboration with third events, future outcomes, efficiency or achievements to vary considerably from these expressed or implied by the forward-looking statements. Such dangers and uncertainties embody, amongst others, the results from the COVID-19 pandemic on the corporate’s scientific actions, enterprise and working outcomes, dangers associated to reliance on third occasion companions to conduct sure actions on the corporate’s behalf, uncertainty and potential delays associated to scientific drug growth, smaller than anticipated market alternatives for the corporate’s merchandise and product candidates, manufacturing dangers, competitors from different therapies or merchandise, and different issues that might have an effect on sufficiency of present money, money equivalents and short-term investments to fund operations, the corporate’s future working outcomes and monetary efficiency, the timing of scientific trial actions and reporting outcomes from similar, and the provision or business potential of Ultragenyx’s merchandise and drug candidates. Ultragenyx undertakes no obligation to replace or revise any forward-looking statements. For an additional description of the dangers and uncertainties that might trigger precise outcomes to vary from these expressed in these forward-looking statements, in addition to dangers referring to the enterprise of Ultragenyx on the whole, see Ultragenyx’s Quarterly Report on Kind 10-Q filed with the Securities and Alternate Fee on October 27, 2020, and its subsequent annual and periodic reviews filed with the Securities and Alternate Fee.
Contact Ultragenyx Pharmaceutical Inc.
Buyers & Media
|Ultragenyx Pharmaceutical Inc.|
|Chosen Assertion of Operations Monetary Knowledge|
|(in 1000’s, besides share and per share quantities)|
|Three Months Ended December 31,||12 months Ended December 31,|
|Assertion of Operations Knowledge:|
|Collaboration and license||$||75,015||$||28,423||$||219,315||$||83,493|
|Product gross sales||12,960||7,170||38,720||20,221|
|Non-cash collaboration royalty income||3,567||—||12,995||—|
|Value of gross sales||5,481||5,107||6,129||9,008|
|Analysis and growth||131,100||83,061||412,084||357,355|
|Promoting, common and administrative||51,042||41,877||182,933||161,524|
|Whole working bills||187,623||130,045||601,146||527,887|
|Loss from operations||(96,081||)||(94,452||)||(330,116||)||(424,173||)|
|Change in truthful worth of fairness investments||79,055||1,419||170,403||13,413|
|Non-cash curiosity expense on legal responsibility associated to
the sale of future royalties
|Different revenue (expense), internet||1,283||2,924||7,645||12,451|
|Loss earlier than revenue taxes||(23,941||)||(91,244||)||(185,359||)||(399,444||)|
|Provision for revenue taxes||(70||)||(2,561||)||(1,207||)||(3,283||)|
|Web loss per share, primary and diluted||$||(0.37||)||$||(1.62||)||$||(3.07||)||$||(7.12||)|
|Shares utilized in computing internet loss per share, primary and
|Ultragenyx Pharmaceutical Inc.|
|Chosen Stability Sheet Monetary Knowledge|
|December 31,||December 31,|
|Stability Sheet Knowledge:|
|Money, money equivalents, and marketable debt securities||$||1,212,039||$||760,404|
|Whole stockholders’ fairness||1,154,375||653,764|