Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical
company focused on the development and commercialization of novel
products for serious rare and ultrarare genetic diseases, today
reported its financial results for the quarter ended September 30,
2023 and provided its financial guidance for the year.
"We're in a strong financial position due to growing demand for
our commercial products, completion of our recent offering, and our
disciplined expense and portfolio management," said Emil D. Kakkis,
M.D., Ph.D., chief executive officer and president of Ultragenyx.
"We've also made significant progress on our key clinical programs
and highlighted new, interim data from UX143 for osteogenesis
imperfecta, GTX-102 for Angelman syndrome, and UX701 for Wilson
Disease at our Analyst Day in October demonstrating promising
therapeutic potential in these larger indications with further
updates to come in 2024."
Third Quarter 2023 Selected Financial Data Tables and
Financial Results
Revenues (dollars in
thousands), (unaudited) |
|
Three Months Ended September 30, |
|
Nine Months Ended September 30, |
|
2023 |
|
2022 |
|
2023 |
|
2022 |
Crysvita |
|
|
|
|
|
|
|
Product sales |
$ |
19,200 |
|
|
$ |
13,184 |
|
|
$ |
57,318 |
|
|
$ |
34,980 |
|
Royalty revenue |
|
35,160 |
|
|
|
— |
|
|
|
64,221 |
|
|
|
— |
|
Non-cash royalty revenue |
|
20,543 |
|
|
|
5,373 |
|
|
|
42,695 |
|
|
|
15,634 |
|
Revenue in profit-share territory |
|
— |
|
|
|
51,348 |
|
|
|
69,705 |
|
|
|
148,121 |
|
Total Crysvita revenue |
|
74,903 |
|
|
|
69,905 |
|
|
|
233,939 |
|
|
|
198,735 |
|
Dojolvi |
|
16,553 |
|
|
|
13,274 |
|
|
|
47,347 |
|
|
|
39,200 |
|
Mepsevii |
|
5,633 |
|
|
|
6,045 |
|
|
|
22,552 |
|
|
|
15,839 |
|
Evkeeza |
|
963 |
|
|
|
— |
|
|
|
1,540 |
|
|
|
— |
|
Daiichi Sankyo |
|
— |
|
|
|
1,479 |
|
|
|
1,479 |
|
|
|
6,207 |
|
Total revenues |
$ |
98,052 |
|
|
$ |
90,703 |
|
|
$ |
306,857 |
|
|
$ |
259,981 |
|
|
|
|
|
|
|
|
|
Total RevenuesUltragenyx reported $98.1 million in total revenue
for the third quarter 2023, which represents 8% growth compared to
the third quarter 2022. Third quarter 2023 Crysvita product sales,
primarily in Latin America, were $19.2 million, which represents
46% growth compared to the same period in 2022 and 14% growth over
the second quarter 2023. Third quarter 2023 also includes Crysvita
royalty and non-cash royalty revenue in North America of $50.2
million, which was impacted by a decrease in channel inventory
related to Kyowa Kirin Co., Ltd.’s (KKC) change from Ultragenyx
labeled product to KKC’s labeled product as part of the transition
of North America commercialization responsibilities for Crysvita
from Ultragenyx to KKC. This one-time change occurred in the third
quarter 2023, and the Company expects Crysvita channel inventories
to increase to more normal levels at the end of the year. Third
quarter 2023 non-cash royalty revenue in Europe was $5.5
million.
|
Selected Financial
Data (dollars in thousands, except per share amounts),
(unaudited) |
|
Three Months Ended September 30, |
|
Nine Months Ended September 30, |
|
2023 |
|
2022 |
|
2023 |
|
2022 |
Total revenues |
$ |
98,052 |
|
|
$ |
90,703 |
|
|
$ |
306,857 |
|
|
$ |
259,981 |
|
Operating expense: |
|
|
|
|
|
|
|
Cost of sales |
|
10,987 |
|
|
|
8,631 |
|
|
|
33,158 |
|
|
|
23,001 |
|
Research and development |
|
157,245 |
|
|
|
237,297 |
|
|
|
487,892 |
|
|
|
534,981 |
|
Selling, general and administrative |
|
74,917 |
|
|
|
69,841 |
|
|
|
232,966 |
|
|
|
205,290 |
|
Total operating expense |
|
243,149 |
|
|
|
315,769 |
|
|
|
754,016 |
|
|
|
763,272 |
|
Net loss |
$ |
(159,649 |
) |
|
$ |
(245,106 |
) |
|
$ |
(483,449 |
) |
|
$ |
(555,588 |
) |
Net loss per share, basic and diluted |
$ |
(2.23 |
) |
|
$ |
(3.50 |
) |
|
$ |
(6.81 |
) |
|
$ |
(7.96 |
) |
|
|
|
|
|
|
|
|
Operating Expenses Total operating expenses for the third
quarter of 2023 were $243.1 million, including non-cash stock-based
compensation of $34.9 million. In 2023, annual operating expenses
are expected to decrease compared to 2022, as the company manages
headcount and increases operational leverage while executing on its
high-value programs.
Net Loss For the third quarter of 2023, Ultragenyx reported net
loss of $159.6 million, or $2.23 per share basic and diluted,
compared with a net loss for the third quarter of 2022 of $245.1
million, or $3.50 per share, basic and diluted.
Cash, Cash Equivalents and Marketable Debt SecuritiesCash, cash
equivalents, and marketable debt securities were $524.2 million as
of September 30, 2023. This excludes net proceeds of $326.5 million
from an underwritten public offering of common stock and pre-funded
warrants that closed in October 2023.
2023 Financial Guidance For the full year 2023,
the company expects:
- Total revenue in the range of $425 million to $450 million
- Crysvita revenue in the range of $325 million to $340 million.
This includes all regions where Ultragenyx will recognize revenue,
including the royalties in Europe, which have been ongoing, and the
royalties in North America, which began in April 2023.
- Dojolvi revenue in the range of $65 million to $75 million
- Net Cash Used in Operations to be around $425 million
Recent Updates and Clinical Milestones
UX143 (setrusumab) monoclonal antibody for Osteogenesis
Imperfecta (OI): Phase 2 demonstrated 67% reduction in annualized
fracture rate and continuous improvements in bone mineral
densityAt the American Society of Bone and Mineral
Research 2023 Annual Meeting (ASBMR), interim data from the Phase 2
portion of the Phase 2/3 Orbit study were presented that
demonstrated treatment with setrusumab significantly reduced
incidence of fractures in patients with OI with at least 6 months
of follow-up and continued to demonstrate ongoing and meaningful
improvements in lumbar spine bone mineral density (BMD). As of the
cut-off date on August 4, 2023 and following at least six months of
treatment with setrusumab, the annualized fracture rate across all
24 patients in the Phase 2 portion of the study was reduced by 67%.
The median annualized fracture rate of 0.72 in the two years prior
to treatment was reduced to 0.00 (n=24, p=0.042) during the mean
treatment duration period of nine months. As of the data cut-off,
there were no treatment-related serious adverse events (SAEs)
observed in the study. Additional longer-term Phase 2 data are
expected in 2024.
Patients are being dosed in the late-stage clinical trials,
Orbit and Cosmic, which evaluate setrusumab in pediatric and young
adult patients with OI. The Phase 3 portion of the Orbit study is
targeting to enroll up to 195 patients at more than 50 sites across
12 countries. 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 aged 2 to <5 years. Cosmic is targeting to enroll
approximately 65 patients at more than 20 global sites.
GTX-102 antisense oligonucleotide for Angelman syndrome:
data from extension cohorts in Phase 1/2 study showed clinically
meaningful improvements in multiple domainsIn October
2023, interim data from the extension cohorts (Cohorts 4-7) in the
ongoing Phase 1/2 for GTX-102 in Angelman syndrome were presented
at an Analyst Day event. The data showed improvements across
multiple domains compared to natural history data, where available,
and clinical changes were associated with quantitative changes in
EEG. Long-term data showed patients who stopped and restarted
treatment reacquired previously gained developmental skills when
they were re-dosed with the current regimen. As of the data
cut-off, there have been no additional treatment-related SAEs,
including lower extremity weakness, since November 2022.
Globally, sites are dosing patients in the expansion cohorts
(Cohorts A-E), which will evaluate the same safety,
pharmacokinetic, and efficacy measures as the extension cohorts.
Data from at least 20 patients enrolled in the expansion cohorts,
who have been on therapy for six months or more, are currently
expected in the first half of 2024.
UX701 AAV gene therapy for Wilson Disease: Stage 1 of
pivotal clinical study dosing patients; expect Stage 1 enrollment
completion around the end of the yearIn October 2023,
interim data from the first dose cohort (5.0 x 10^12 GC/kg) in the
ongoing Cyprus2+ study for UX701 in Wilson disease were presented
at an Analyst Day event. The company announced four out of five
patients in Cohort 1 had reductions in urinary copper and were
tapering off of chelators and/or zinc therapy, including two of
three earlier treated patients in the cohort that are now
completely off standard of care therapy. As of the data cut-off,
UX701 had been generally well-tolerated with no treatment-related
SAEs.
Dosing in the second of three dose escalation cohorts in the
pivotal study has been completed. The data safety monitoring board
(DSMB) is scheduled to meet and will review the available safety
data from Cohort 2 before making a recommendation on escalating to
Cohort 3 of Stage 1 in this study. Stage 1 is currently on track to
complete enrollment around the end of the year and these data are
expected in the first half of 2024. During this stage, the safety
and efficacy of UX701 will be evaluated and a dose will be selected
for further evaluation in the pivotal, randomized,
placebo-controlled stage of the study.
DTX401 AAV gene therapy for Glycogen Storage Disease
Type Ia (GSDIa): Dosing in Phase 3 study completeIn 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 data are
expected in the first half of 2024.
DTX301 AAV gene therapy for Ornithine Transcarbamylase
(OTC) Deficiency: Phase 3 study dosing patientsUltragenyx
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, November
2, 2023, at 2 p.m. PT/5 p.m. ET to discuss the third quarter 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://edge.media-server.com/mmc/p/t97kpsfo), 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 August 4, 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/).
|
Ultragenyx
Pharmaceutical Inc. |
Selected
Statement of Operations Financial Data |
(in
thousands, except share and per share amounts) |
(unaudited) |
|
|
|
|
|
|
|
|
|
Three Months Ended September 30, |
|
Nine Months Ended September 30, |
|
2023 |
|
2022 |
|
2023 |
|
2022 |
Statement of Operations Data: |
|
|
|
|
|
|
|
Revenues: |
|
|
|
|
|
|
|
Product sales |
$ |
42,349 |
|
|
$ |
32,503 |
|
|
$ |
128,757 |
|
|
$ |
90,019 |
|
Royalty revenue |
|
55,703 |
|
|
|
5,373 |
|
|
|
106,916 |
|
|
|
15,634 |
|
Collaboration and license |
|
— |
|
|
|
52,827 |
|
|
|
71,184 |
|
|
|
154,328 |
|
Total revenues |
|
98,052 |
|
|
|
90,703 |
|
|
|
306,857 |
|
|
|
259,981 |
|
Operating expenses: |
|
|
|
|
|
|
|
Cost of sales |
|
10,987 |
|
|
|
8,631 |
|
|
|
33,158 |
|
|
|
23,001 |
|
Research and development |
|
157,245 |
|
|
|
237,297 |
|
|
|
487,892 |
|
|
|
534,981 |
|
Selling, general and administrative |
|
74,917 |
|
|
|
69,841 |
|
|
|
232,966 |
|
|
|
205,290 |
|
Total operating expenses |
|
243,149 |
|
|
|
315,769 |
|
|
|
754,016 |
|
|
|
763,272 |
|
Loss from operations |
|
(145,097 |
) |
|
|
(225,066 |
) |
|
|
(447,159 |
) |
|
|
(503,291 |
) |
Change in fair value of equity investments |
|
(1,419 |
) |
|
|
(1,626 |
) |
|
|
(1,492 |
) |
|
|
(21,139 |
) |
Non-cash interest expense on liability related to the sale of
future royalties |
|
(17,665 |
) |
|
|
(14,505 |
) |
|
|
(48,676 |
) |
|
|
(27,141 |
) |
Other income, net |
|
5,182 |
|
|
|
2,378 |
|
|
|
15,755 |
|
|
|
3,130 |
|
Loss before income taxes |
|
(158,999 |
) |
|
|
(238,819 |
) |
|
|
(481,572 |
) |
|
|
(548,441 |
) |
Provision for income taxes |
|
(650 |
) |
|
|
(6,287 |
) |
|
|
(1,877 |
) |
|
|
(7,147 |
) |
Net loss |
$ |
(159,649 |
) |
|
$ |
(245,106 |
) |
|
$ |
(483,449 |
) |
|
$ |
(555,588 |
) |
Net loss per share, basic and diluted |
$ |
(2.23 |
) |
|
$ |
(3.50 |
) |
|
$ |
(6.81 |
) |
|
$ |
(7.96 |
) |
Weighted-average shares used in computing net loss per share, basic
and diluted |
|
71,664,493 |
|
|
|
70,054,173 |
|
|
|
70,987,801 |
|
|
|
69,834,037 |
|
|
|
|
|
|
|
|
|
|
Ultragenyx
Pharmaceutical Inc. |
Selected
Activity included in Operating Expenses |
(in
thousands) |
(unaudited) |
|
|
|
|
Three Months Ended September 30, |
|
Nine Months Ended September 30, |
|
2023 |
|
2022 |
|
2023 |
|
2022 |
|
|
|
|
|
|
|
|
Non-cash
stock-based compensation |
$ |
34,877 |
|
|
$ |
35,761 |
|
|
$ |
101,469 |
|
|
$ |
101,013 |
|
In-process research and development expense from GeneTx
acquisition |
|
— |
|
|
$ |
75,234 |
|
|
|
— |
|
|
$ |
75,234 |
|
UX143 clinical milestone |
|
— |
|
|
|
— |
|
|
$ |
9,000 |
|
|
|
— |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Ultragenyx
Pharmaceutical Inc. |
Selected
Balance Sheet Financial Data |
(in
thousands) |
(unaudited) |
|
|
September 30, |
|
December 31, |
|
|
2023 |
|
2022 |
Balance Sheet Data: |
|
|
|
|
Cash, cash equivalents, and marketable debt securities |
|
$ |
524,165 |
|
|
$ |
896,732 |
|
Working
capital |
|
|
345,210 |
|
|
|
622,689 |
|
Total
assets |
|
|
1,238,140 |
|
|
|
1,545,444 |
|
Total
stockholders' equity |
|
|
31,714 |
|
|
|
352,494 |
|
|
|
|
|
|
|
|
|
|
Contacts Ultragenyx Pharmaceutical
Inc.InvestorsJoshua
Higair@ultragenyx.com
MediaJeff Blakemedia@ultragenyx.com
Ultragenyx Pharmaceutical (NASDAQ:RARE)
Historical Stock Chart
From Apr 2024 to May 2024
Ultragenyx Pharmaceutical (NASDAQ:RARE)
Historical Stock Chart
From May 2023 to May 2024