Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical
company focused on the development and commercialization of novel
therapies for serious rare and ultrarare genetic diseases, today
reported its financial results for the quarter and full year ended
December 31, 2023 and reaffirmed its financial guidance for 2024.
“Last year, we made significant progress across our pipeline and
look forward to multiple catalysts in 2024 with substantive data in
Angelman syndrome that could enable a Phase 3 study, pivotal Phase
3 data from our GSDIa gene therapy program, dose-finding data from
our seamless study in Wilson disease, and longer-term Phase 2 data
in Osteogenesis Imperfecta,” said Emil D. Kakkis, M.D., Ph.D.,
chief executive officer and president of Ultragenyx. “In 2023, we
saw increased demand for both Crysvita and Dojolvi, and achieved
multiple regulatory and reimbursement milestones for our commercial
products to support continued revenue growth of approximately 20%
in 2024.”
Fourth Quarter and Full Year 2023 Selected Financial
Data Tables and Financial Results
Revenues
(dollars in thousands), (unaudited) |
|
|
|
|
|
|
|
|
Three Months Ended December 31, |
|
Year Ended December 31, |
|
2023 |
|
2022 |
|
2023 |
|
2022 |
Crysvita |
|
|
|
|
|
|
|
Product sales |
$ |
18,379 |
|
$ |
7,698 |
|
$ |
75,697 |
|
$ |
42,678 |
Revenue in Profit-Share Territory |
|
70,124 |
|
|
66,903 |
|
|
231,574 |
|
|
215,024 |
Royalty revenue in European Territory |
|
5,612 |
|
|
6,058 |
|
|
20,783 |
|
|
21,692 |
Total Crysvita Revenue |
|
94,115 |
|
|
80,659 |
|
|
328,054 |
|
|
279,394 |
Dojolvi |
|
23,286 |
|
|
16,412 |
|
|
70,633 |
|
|
55,612 |
Mepsevii |
|
7,889 |
|
|
4,798 |
|
|
30,441 |
|
|
20,637 |
Evkeeza |
|
2,102 |
|
|
— |
|
|
3,642 |
|
|
— |
Daiichi Sankyo |
|
— |
|
|
1,479 |
|
|
1,479 |
|
|
7,686 |
Total revenues |
$ |
127,392 |
|
$ |
103,348 |
|
$ |
434,249 |
|
$ |
363,329 |
|
|
|
|
|
|
|
|
Total RevenuesUltragenyx reported $127 million in total revenue
for the fourth quarter 2023, which represents 23% growth compared
to the same period in 2022. Fourth quarter 2023 Crysvita revenue
was $94 million, which represents 17% growth compared to the same
period in 2022. This includes product sales of $18 million from
Latin America and Turkey, which represents 139% growth compared to
the same period in 2022. Dojolvi revenue in the fourth quarter 2023
was $23 million, which represents 42% growth compared to the same
period in 2022.
Total revenue for the year ended December 31, 2023 was $434
million, which represents 20% growth compared to the prior year.
Full year 2023 Crysvita revenue was $328 million, which represents
17% growth compared to the same period in 2022. This includes
product sales of $76 million from Latin America and Turkey, which
represents 77% growth compared to the same period in 2022. Dojolvi
revenue in 2023 was $71 million, which represents 27% growth
compared to the same period in 2022.
Selected Financial Data (dollars in thousands, except per share
amounts), (unaudited) |
|
|
|
|
|
Three Months Ended December 31, |
|
Year Ended December 31, |
|
|
2023 |
|
|
|
2022 |
|
|
|
2023 |
|
|
|
2022 |
|
Total revenues |
$ |
127,392 |
|
|
$ |
103,348 |
|
|
$ |
434,249 |
|
|
$ |
363,329 |
|
Operating expenses: |
|
|
|
|
|
|
|
Cost of sales |
|
12,051 |
|
|
|
5,319 |
|
|
|
45,209 |
|
|
|
28,320 |
|
Research and development |
|
160,557 |
|
|
|
170,808 |
|
|
|
648,449 |
|
|
|
705,789 |
|
Selling, general and administrative |
|
76,833 |
|
|
|
72,849 |
|
|
|
309,799 |
|
|
|
278,139 |
|
Total operating expenses |
|
249,441 |
|
|
|
248,976 |
|
|
|
1,003,457 |
|
|
|
1,012,248 |
|
Net loss |
$ |
(123,190 |
) |
|
$ |
(151,833 |
) |
|
$ |
(606,639 |
) |
|
$ |
(707,421 |
) |
Net loss per share, basic and diluted |
$ |
(1.52 |
) |
|
$ |
(2.16 |
) |
|
$ |
(8.25 |
) |
|
$ |
(10.12 |
) |
|
|
|
|
|
|
|
|
Operating Expenses Total operating expenses for the fourth
quarter of 2023 were $249 million, including non-cash stock-based
compensation of $34 million. Total operating expenses for the year
ended December 31, 2023 were $1,003 million, including $135 million
of non-cash stock-based compensation. In 2024, annual operating
expenses are expected to be stable or to decrease as the company
continues to manage its costs, focus its investment on advancing
multiple Phase 3 programs, and execute on commercial product
launches.
Net Loss For the fourth quarter of 2023, Ultragenyx reported net
loss of $123 million, or $1.52 per share basic and diluted,
compared with a net loss for the fourth quarter of 2022 of $152
million, or $2.16 per share, basic and diluted. For the year ended
December 31, 2023, Ultragenyx reported net loss of $607 million, or
$8.25 per share basic and diluted, compared with a net loss the
prior year of $707 million, or $10.12 per share, basic and
diluted.
Net Cash Used in Operations and Cash BalanceNet cash used in
operations for the year ended December 31, 2023 was $475 million.
Cash, cash equivalents, and marketable debt securities were $777
million as of December 31, 2023.
2024 Financial Guidance For the full year 2024,
the company reaffirms:
- Total revenue in the range of $500 million to $530 million
- Crysvita revenue in the range of $375 million to $400 million.
This includes all regions where Ultragenyx will recognize revenue:
product sales in Latin America and Turkey, royalties in Europe,
which have been ongoing, and royalties in North America, which
began in April 2023.
- Dojolvi revenue in the range of $75 million to $80 million
- Net Cash Used in Operations to be less than $400 million
Recent Updates and Clinical Milestones
UX143 (setrusumab) monoclonal antibody for Osteogenesis
Imperfecta (OI): Phase 3 portion of Orbit study expected to be
fully enrolled around the end of the first quarter of
2024Patients are being dosed in the late-stage clinical
trials, Orbit and Cosmic, which are evaluating setrusumab in
pediatric and young adult patients with OI. The randomized,
placebo-controlled Phase 3 portion of the Orbit study is expected
to enroll approximately 150 patients and be fully enrolled around
the end of the first quarter of 2024. Additional longer-term Phase
2 safety and efficacy data from the Orbit study are expected in the
second half of 2024.
The Phase 3 Cosmic study is an active-controlled study
evaluating the effect of setrusumab compared to intravenous
bisphosphonate (IV-BP) therapy on annualized total fracture rate in
patients age 2 to <5 years. Cosmic is expected to enroll
approximately 50 patients or more at more than 20 global sites and
is expected to complete enrollment in the first half of 2024.
GTX-102 antisense oligonucleotide for Angelman syndrome:
Phase 1/2 fully enrolled; expansion data expected in the first half
of 2024Enrollment in the expansion cohorts was completed
in December 2023 with a total of 53 new patients enrolled. A total
of 74 patients are enrolled in the Phase 1/2 study including the
dose-escalation/extension study patients. The expansion cohorts
will evaluate many of the same safety, pharmacokinetic, and
efficacy measures as the previously enrolled
dose-escalation/extension cohorts plus some new evaluations. The
next safety and efficacy update is expected in the first half of
2024 and is planned to include data from at least 20 expansion
cohort patients with a minimum of Day 170 data.
In January 2024, GTX-102 was accepted into the Priority
Medicines (PRIME) program by the European Medicines Agency (EMA).
PRIME is granted by the EMA to medicines that show the potential to
benefit patients with unmet needs based on early clinical data.
Through PRIME, the EMA offers early and proactive support to
optimize development plans and the generation of robust data on a
medicine’s benefits and risks, and enables accelerated assessment
of medicines applications.
UX701 AAV gene therapy for Wilson disease: Last patient
in Cohort 3 dosed; expect interim Stage 1 data in
mid-2024All patients in the three dose-escalation cohorts
of Stage 1 have been dosed. During Stage 1, the safety and efficacy
of UX701 will be evaluated and a dose will be selected for further
evaluation in Stage 2, the pivotal, randomized, placebo-controlled
stage of the study. Data from Stage 1 are expected in mid-2024,
which will be followed by dose selection and initiation of Stage 2
in the second half of 2024.
UX111 AAV gene therapy for Sanfilippo syndrome (MPS
IIIA): Updated data from pivotal Transfer A study presented at
WORLDSymposiumTM New positive data from
the ongoing pivotal Transfer A study evaluating the safety and
efficacy of UX111 in children with MPS IIIA were presented at the
20th Annual WORLDSymposium™ earlier this month showing that a
single infusion of UX111 can substantially correct the underlying
metabolic disease and maintain cognitive function in nearly all
patients. The presentation also showed that the observed reductions
of heparan sulfate exposure in cerebrospinal fluid can predict
improved long-term cognitive function in patients with MPS IIIA
following treatment with UX111. With these data and other data,
discussions are ongoing with the FDA seeking an accelerated review
path for UX111.
DTX401 AAV gene therapy for Glycogen Storage Disease
Type Ia (GSDIa): Dosing in Phase 3 study complete; Phase 3 data
readout expected in the first half of 2024In May 2023,
Ultragenyx announced the last patient had been dosed in the Phase 3
study. The 48-week study has fully enrolled patients eight years of
age and older, randomized 1:1 to DTX401 or placebo. The primary
endpoint is the reduction in oral glucose replacement with
cornstarch while maintaining glucose control. Phase 3 safety and
efficacy data are expected in the first half of 2024.
DTX301 AAV gene therapy for Ornithine Transcarbamylase
(OTC) Deficiency: Phase 3 study dosing patients; expect enrollment
to be completed in the first half of 2024Ultragenyx is
randomizing and dosing patients in the ongoing Phase 3 study. The
pivotal, 64-week study will include approximately 50 patients,
randomized 1:1 to DTX301 or placebo. The primary endpoints are
response as measured by removal of ammonia-scavenger medications
and protein-restricted diet and change in 24-hour ammonia levels.
Enrollment is currently expected to be completed in the first half
of 2024.
Conference Call and Webcast Information
Ultragenyx will host a conference call today, Thursday, February
15, 2024, at 2 p.m. PT/5 p.m. ET to discuss the fourth quarter and
full year 2023 financial results and provide a corporate update.
The live and replayed webcast of the call will be available through
the company’s website at
https://ir.ultragenyx.com/events-presentations. To participate in
the live call, please register by clicking on the following link
(https://register.vevent.com/register/BI177d5c166d3045ddaa4dcd3b3ec136ba)
and you will be provided with dial-in details. The replay of the
call will be available for one year.
About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing
novel therapies to patients for the treatment of serious rare and
ultrarare genetic diseases. The company has built a diverse
portfolio of approved medicines and treatment candidates aimed at
addressing diseases with high unmet medical need and clear biology,
for which there are typically no approved therapies treating the
underlying disease.
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.
Forward-Looking Statements and Use of Digital
Media
Except for the historical information contained herein, the
matters set forth in this press release, including statements
related to Ultragenyx's expectations and projections regarding its
future operating results and financial performance, anticipated
cost or expense reductions, the timing, progress and 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, collaboration with third parties, 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 uncertainty of
clinical drug development and unpredictability and lengthy process
for obtaining regulatory approvals, risks related to serious or
undesirable side effects of our product candidates, the company’s
ability to achieve its projected development goals in its expected
timeframes, risks related to reliance on third party partners to
conduct certain activities on the company’s behalf, our limited
experience in generating revenue from product sales, risks related
to product liability lawsuits, our dependence on Kyowa Kirin for
the commercial supply of Crysvita, fluctuations in buying or
distribution patterns from distributors and specialty pharmacies,
the transition back to Kyowa Kirin of our exclusive rights to
promote Crysvita in the United States and Canada and unexpected
costs, delays, difficulties or adverse impact to revenue related to
such transition, smaller than anticipated market opportunities for
the company’s products and product candidates, manufacturing risks,
competition from other therapies or products, and other matters
that could affect sufficiency of existing cash, cash equivalents
and short-term investments to fund operations, the company’s future
operating results and financial performance, the timing of clinical
trial activities and reporting results from same, and the
availability or commercial potential of Ultragenyx’s 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
Securities and Exchange Commission (SEC) on November 3, 2023, and
its subsequent periodic reports filed with the SEC.
In addition to its SEC filings, press releases and public
conference calls, Ultragenyx uses its investor relations website
and social media outlets to publish important information about the
company, including information that may be deemed material to
investors, and to comply with its disclosure obligations under
Regulation FD. Financial and other information about Ultragenyx is
routinely posted and is accessible on Ultragenyx’s Investor
Relations website (https://ir.ultragenyx.com/) and LinkedIn website
(https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/mycompany/).
Contacts Ultragenyx Pharmaceutical
Inc.InvestorsJoshua
Higair@ultragenyx.com
Ultragenyx
Pharmaceutical Inc. |
Selected
Statement of Operations Financial Data |
(in
thousands, except share and per share amounts) |
(unaudited) |
|
|
|
|
|
|
|
|
|
Three Months Ended December 31, |
|
Year Ended December 31, |
|
|
2023 |
|
|
|
2022 |
|
|
|
2023 |
|
|
|
2022 |
|
Statement of Operations Data: |
|
|
|
|
|
|
|
Revenues: |
|
|
|
|
|
|
|
Product sales |
$ |
51,656 |
|
|
$ |
28,908 |
|
|
$ |
180,413 |
|
|
$ |
118,927 |
|
Royalty revenue |
|
75,736 |
|
|
|
6,058 |
|
|
|
182,652 |
|
|
|
21,692 |
|
Collaboration and license |
|
— |
|
|
|
68,382 |
|
|
|
71,184 |
|
|
|
222,710 |
|
Total revenues |
|
127,392 |
|
|
|
103,348 |
|
|
|
434,249 |
|
|
|
363,329 |
|
Operating expenses: |
|
|
|
|
|
|
|
Cost of sales |
|
12,051 |
|
|
|
5,319 |
|
|
|
45,209 |
|
|
|
28,320 |
|
Research and development |
|
160,557 |
|
|
|
170,808 |
|
|
|
648,449 |
|
|
|
705,789 |
|
Selling, general and administrative |
|
76,833 |
|
|
|
72,849 |
|
|
|
309,799 |
|
|
|
278,139 |
|
Total operating expenses |
|
249,441 |
|
|
|
248,976 |
|
|
|
1,003,457 |
|
|
|
1,012,248 |
|
Loss from operations |
|
(122,049 |
) |
|
|
(145,628 |
) |
|
|
(569,208 |
) |
|
|
(648,919 |
) |
Change in fair value of equity investments |
|
1,889 |
|
|
|
1,840 |
|
|
|
397 |
|
|
|
(19,299 |
) |
Non-cash interest expense on liabilities for sales of future
royalties |
|
(17,328 |
) |
|
|
(15,874 |
) |
|
|
(66,004 |
) |
|
|
(43,015 |
) |
Other income, net |
|
10,596 |
|
|
|
6,378 |
|
|
|
26,351 |
|
|
|
9,508 |
|
Loss before income taxes |
|
(126,892 |
) |
|
|
(153,284 |
) |
|
|
(608,464 |
) |
|
|
(701,725 |
) |
Benefit from (provision for) income taxes |
|
3,702 |
|
|
|
1,451 |
|
|
|
1,825 |
|
|
|
(5,696 |
) |
Net loss |
$ |
(123,190 |
) |
|
$ |
(151,833 |
) |
|
$ |
(606,639 |
) |
|
$ |
(707,421 |
) |
Net loss per share, basic and diluted |
$ |
(1.52 |
) |
|
$ |
(2.16 |
) |
|
$ |
(8.25 |
) |
|
$ |
(10.12 |
) |
Shares used in computing net loss per share, basic and diluted |
|
81,118,873 |
|
|
|
70,152,192 |
|
|
|
73,543,862 |
|
|
|
69,914,225 |
|
|
|
|
|
|
|
|
|
Ultragenyx Pharmaceutical
Inc.Selected Activity included in Operating
Expenses(in
thousands)(unaudited)
|
Three Months Ended December 31, |
|
Year Ended December 31, |
|
2023 |
|
2022 |
|
2023 |
|
2022 |
|
|
|
|
|
|
|
|
Non-cash stock based compensation |
$ |
33,744 |
|
$ |
29,355 |
|
|
$ |
135,213 |
|
$ |
130,368 |
In-process research and development expense from GeneTx
acquisition |
|
— |
|
$ |
(201 |
) |
|
|
— |
|
$ |
75,033 |
UX143 clinical milestone |
|
— |
|
|
— |
|
|
$ |
9,000 |
|
|
— |
|
|
|
|
|
|
|
|
Ultragenyx
Pharmaceutical Inc. |
Selected
Balance Sheet Financial Data |
(in
thousands) |
(unaudited) |
|
|
December
31, |
|
December
31, |
|
|
2023 |
|
2022 |
Balance Sheet Data: |
|
|
|
|
Cash, cash equivalents, and marketable debt securities |
|
$ |
777,110 |
|
$ |
896,732 |
Working
capital |
|
|
451,747 |
|
|
622,689 |
Total
assets |
|
|
1,491,013 |
|
|
1,545,444 |
Total
stockholders' equity |
|
|
275,414 |
|
|
352,494 |
Ultragenyx Pharmaceutical (NASDAQ:RARE)
過去 株価チャート
から 5 2024 まで 6 2024
Ultragenyx Pharmaceutical (NASDAQ:RARE)
過去 株価チャート
から 6 2023 まで 6 2024