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 ended March 31, 2024
and reaffirmed its financial guidance for 2024.
“In the first quarter we continued to see rapid revenue growth
in the international markets with steady progress globally across
our four commercial products. We also just completed enrollment of
our Phase 3 program in osteogenesis imperfecta supported by strong
enthusiasm from treating physicians and the patient community,”
said Emil D. Kakkis, M.D., Ph.D., chief executive officer and
president of Ultragenyx. “Last month we shared new data from the
GTX-102 program for Angelman syndrome and have received an
overwhelming response of excitement and support from the clinical
experts and advocates in the Angelman community. We are on track to
hold regulatory discussions in mid-2024 with the goal of initiating
a Phase 3 trial before the end of the year.”
First Quarter 2024 Selected Financial Data Tables and
Financial Results
Revenues
(dollars in thousands), (unaudited) |
|
|
|
|
Three Months Ended March 31, |
|
2024 |
|
2023 |
Crysvita |
|
|
|
Product sales |
$ |
36,241 |
|
$ |
21,234 |
Revenue in Profit-Share Territory |
|
40,402 |
|
|
49,906 |
Royalty revenue in European Territory |
|
5,942 |
|
|
4,882 |
Total Crysvita Revenue |
|
82,585 |
|
|
76,022 |
Dojolvi |
|
16,362 |
|
|
14,303 |
Mepsevii |
|
6,611 |
|
|
8,480 |
Evkeeza |
|
3,275 |
|
|
212 |
Daiichi Sankyo |
|
— |
|
|
1,479 |
Total revenues |
$ |
108,833 |
|
$ |
100,496 |
|
|
|
|
Total RevenuesUltragenyx reported $109 million in total revenue
for the first quarter of 2024, which represents 8% growth compared
to the same period in 2023. First quarter 2024 Crysvita revenue was
$83 million, which represents 9% growth compared to the same period
in 2023. This includes product sales of $36 million from Latin
America and Turkey, which represents 71% growth compared to the
same period in 2023. In April 2023, commercialization
responsibilities in the Profit-share Territory transitioned to
Kyowa Kirin Co and commensurately, revenue in that region
transitioned from a profit-share to a royalty ranging from the
mid-20’s to 30% calculated based on annual revenue tiers. Dojolvi
revenue in the first quarter 2024 was $16 million, which represents
14% growth compared to the same period in 2023.
Selected Financial Data (dollars in thousands, except per share
amounts), (unaudited) |
|
Three Months Ended March 31, |
|
|
2024 |
|
|
|
2023 |
|
Total revenues |
$ |
108,833 |
|
|
$ |
100,496 |
|
Operating expenses: |
|
|
|
Cost of sales |
|
17,533 |
|
|
|
12,257 |
|
Research and development |
|
178,487 |
|
|
|
165,698 |
|
Selling, general and administrative |
|
78,160 |
|
|
|
76,646 |
|
Total operating expenses |
|
274,180 |
|
|
|
254,601 |
|
Net loss |
$ |
(170,684 |
) |
|
$ |
(163,972 |
) |
Net loss per share, basic and diluted |
$ |
(2.03 |
) |
|
$ |
(2.33 |
) |
|
|
|
|
Operating Expenses Total operating expenses for the first
quarter of 2024 were $274 million, including non-cash stock-based
compensation of $37 million. In 2024, annual operating expenses are
expected to be stable or to decrease as the company continues to
manage its costs and focus its investment on advancing multiple
Phase 3 programs and executing on commercial product launches.
Net Loss For the first quarter of 2024, Ultragenyx reported net
loss of $171 million, or $2.03 per share basic and diluted,
compared with a net loss for the first quarter of 2023 of $164
million, or $2.33 per share, basic and diluted.
Net Cash Used in Operations and Cash BalanceNet cash used in
operations for the quarter ended March 31, 2024 was $191 million.
Cash, cash equivalents, and marketable debt securities were $569
million as of March 31, 2024.
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): Enrollment complete for Phase 3 Orbit and Cosmic
studies; expect additional Phase 2 data update in second half of
2024 The Phase 3 portion of the Orbit study has completed
enrollment with 158 patients. Orbit is a randomized
placebo-controlled study evaluating the effect of setrusumab
compared to placebo on the rate of annualized clinical fractures in
patients aged 5 to <25 years. Additional longer-term safety and
efficacy data from the Phase 2 portion of the Orbit study are
expected in the second half of 2024.
The Phase 3 Cosmic study has also completed enrollment with 66
patients randomized or in screening. Cosmic 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 <7 years.
GTX-102 antisense oligonucleotide for Angelman syndrome:
Positive interim Phase 1/2 data shared in April; expect End of
Phase 2 meeting with Food and Drug Administration (FDA)
in mid-2024New interim data from the Phase 1/2
study were presented at a company presentation and at the 76th
Annual American Academy of Neurology Meeting. Patients in Expansion
Cohorts A & B treated with a set dose and regimen of GTX-102
showed rapid and clinically meaningful improvement across multiple
domains consistent with or exceeding Dose Escalation Cohorts 4-7
data at Day 170. Treatment of the Dose Escalation Cohorts 4-7
showed long-term increasing and sustained clinical benefit far
exceeding Natural History data at Day 758.
As previously reported and as of the data cut-off on April 5,
2024, there were two patients who had mild or moderate lower
extremity weakness across 53 patients who completed the loading
phase in the Expansion Cohorts A-E. Both were in Cohorts A & B
and none reported in Cohorts C–E. The lower extremity weakness
resolved rapidly without sequelae and patients remain in the study
without ongoing safety concerns and are expected to continue
dosing. The FDA and other regulatory agencies were notified and
raised no issues nor required additional actions. There were no
other unexpected serious adverse events.
We expect to share additional long-term safety and efficacy data
from the Phase 1/2 study and plan to continue to provide routine
safety updates with efficacy updates. We currently expect to have
an End of Phase 2 meeting with the FDA in mid-2024 and meetings
with other regulatory agencies in the second half of 2024.
UX701 AAV gene therapy for Wilson disease: Last patient
in Cohort 3 dosed; expect interim Stage 1 data in the second half
of 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 the second
half of 2024, which will be followed by dose selection and
initiation of Stage 2.
UX111 AAV gene therapy for Sanfilippo syndrome (MPS
IIIA): Discussions ongoing with FDA, seeking accelerated approval
pathIn February 2024, Ultragenyx participated in a
workshop hosted by the Reagan-Udall Foundation for the FDA that
brought together FDA representatives, patient advocates,
scientists, and industry leaders to discuss the use of CSF heparan
sulfate as a relevant biomarker to enable accelerated approval in
neuronopathic mucopolysaccharidoses (MPS). Discussions are ongoing
with the FDA in seeking an accelerated review path for UX111.
DTX401 AAV gene therapy for Glycogen Storage Disease
Type Ia (GSDIa): Dosing in Phase 3 study complete; expect Phase 3
data readout in the second quarter of 2024In May 2023,
Ultragenyx announced the last patient was dosed in the Phase 3
study. The 48-week study enrolled 49 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 second quarter of 2024.
DTX301 AAV gene therapy for Ornithine Transcarbamylase
(OTC) Deficiency: Phase 3 study dosing patients; expect enrollment
to be completed in the second 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 second half
of 2024.
Conference Call and Webcast Information
Ultragenyx will host a conference call today, Thursday, May 2,
2024, at 2 p.m. PT/5 p.m. ET to discuss the first quarter 2024
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/BI7c62f78f6df84941acb020e8220f4d81)
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 Annual Report on Form 10-K filed with the
Securities and Exchange Commission (SEC) on February 21, 2024, 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 March 31, |
|
|
2024 |
|
|
|
2023 |
|
Statement of Operations Data: |
|
|
|
Revenues: |
|
|
|
Product sales |
$ |
62,489 |
|
|
$ |
44,229 |
|
Royalty revenue |
|
46,344 |
|
|
|
4,882 |
|
Collaboration and license |
|
— |
|
|
|
51,385 |
|
Total revenues |
|
108,833 |
|
|
|
100,496 |
|
Operating expenses: |
|
|
|
Cost of sales |
|
17,533 |
|
|
|
12,257 |
|
Research and development |
|
178,487 |
|
|
|
165,698 |
|
Selling, general and administrative |
|
78,160 |
|
|
|
76,646 |
|
Total operating expenses |
|
274,180 |
|
|
|
254,601 |
|
Loss from operations |
|
(165,347 |
) |
|
|
(154,105 |
) |
Change in fair value of equity investments |
|
3,746 |
|
|
|
(334 |
) |
Non-cash interest expense on liabilities for sales of future
royalties |
|
(15,847 |
) |
|
|
(15,636 |
) |
Other income, net |
|
7,219 |
|
|
|
6,598 |
|
Loss before income taxes |
|
(170,229 |
) |
|
|
(163,477 |
) |
Provision for income taxes |
|
(455 |
) |
|
|
(495 |
) |
Net loss |
$ |
(170,684 |
) |
|
$ |
(163,972 |
) |
Net loss per share, basic and diluted |
$ |
(2.03 |
) |
|
$ |
(2.33 |
) |
Shares used in computing net loss per share, basic and diluted |
|
84,286,292 |
|
|
|
70,368,478 |
|
|
|
|
|
Ultragenyx Pharmaceutical
Inc.Selected Activity included in Operating
Expenses(in
thousands)(unaudited)
|
|
Three Months Ended March 31, |
|
2024 |
|
2023 |
|
|
|
|
Non-cash stock-based compensation |
$ |
36,934 |
|
$ |
31,939 |
|
|
|
|
Ultragenyx
Pharmaceutical Inc. |
Selected
Balance Sheet Financial Data |
(in
thousands) |
(unaudited) |
|
|
|
March
31, |
|
December
31, |
|
|
2024 |
|
2023 |
Balance Sheet Data: |
|
|
|
|
Cash, cash equivalents, and marketable debt securities |
|
$ |
568,661 |
|
$ |
777,110 |
Working
capital |
|
|
350,392 |
|
|
451,747 |
Total
assets |
|
|
1,307,080 |
|
|
1,491,013 |
Total
stockholders' equity |
|
|
140,264 |
|
|
275,414 |
Grafico Azioni Ultragenyx Pharmaceutical (NASDAQ:RARE)
Storico
Da Ott 2024 a Nov 2024
Grafico Azioni Ultragenyx Pharmaceutical (NASDAQ:RARE)
Storico
Da Nov 2023 a Nov 2024