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 preliminary unaudited 2023 revenue results, cash and
investments at year end 2023, and provided 2024 guidance on select
key financial metrics including net cash used in operations.
“2024 is poised to be a year of significant momentum for
Ultragenyx, with clinical catalysts across multiple value-driving
programs, meaningful revenue growth from our commercial products,
and continued focus on financial discipline,” said Emil D. Kakkis,
M.D., Ph.D., chief executive officer and president of Ultragenyx.
“We anticipate several important milestones in the first half of
the year, including completing enrollment of our Phase 3 studies in
osteogenesis imperfecta, interim data on a substantive portion of
patients enrolled in our Phase 1/2 study in Angelman syndrome,
Stage 1 data from our pivotal Phase 1/2/3 study in Wilson disease,
and topline Phase 3 data from our GSDIa gene therapy program.”
Ultragenyx will present at the 42ᵗʰ annual J.P. Morgan
Healthcare Conference on Monday, January 8, 2024 at 3:00 p.m. PT.
The live and archived webcast of the presentation will be
accessible from the company’s website at
https://ir.ultragenyx.com/events-presentations.
Financial Update
2023 Revenue (unaudited) and 2024 Revenue Guidance
|
2023 |
|
2024 |
($ in
millions) |
Preliminary Revenue |
|
Revenue Guidance |
Crysvita |
$325 -
$330 |
|
$375 -
$400 |
Dojolvi |
$70 -
$71 |
|
$75 -
$80 |
Total
Revenue |
$430 -
$435 |
|
$500 -
$530 |
2023 Ending Cash Position (unaudited) and 2024 Cash Used in
Operations Guidance Cash, cash equivalents, and
available-for-sale investments were approximately $776 million as
of December 31, 2023. Cash uses in 2023 included the completion of
construction of our gene therapy manufacturing facility. With
forecasted ~20% increases in revenue and continued focus on key
pipeline value drivers, 2024 net cash use is projected to be less
than $400 million.
The 2023 revenues and cash position included in this release are
preliminary and are therefore subject to adjustment. The
preliminary revenue results are based on management’s initial
analysis of operations for the year ended December 31, 2023. The
Company expects to issue full financial results for the fourth
quarter and fiscal year 2023 in February 2024.
Recent Updates and 2024 Clinical Milestones
UX143 (setrusumab) monoclonal antibody for Osteogenesis
Imperfecta (OI): Phase 3 portion of Orbit study expected to be
fully enrolled in the first quarter of 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 randomized, placebo-controlled Phase 3
portion of the Orbit study is expected to be fully enrolled around
the end of the first quarter of 2024. Additional longer-term Phase
2 data from the Orbit study are expected in 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 aged 2 to <5
years. Cosmic is targeting to enroll approximately 65 patients at
more than 20 global sites and is expected to complete enrollment in
the first half of 2024.
GTX-102 an 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 patients enrolled.
There are a total of 74 patients 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 cohorts plus some new evaluations. The next data
update is expected in the first half of 2024 and is planned to
include at least 20 expansion cohort patients with a minimum of Day
170 data.
UX701 AAV gene therapy for Wilson Disease: Last patient
in Cohort 3 on track to be dosed soon; expect interim Stage 1 data
in the first half of 2024Four of five patients in the
third of three dose escalation cohorts in the pivotal study have
been dosed, with the fifth patient scheduled to be dosed soon.
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 first half of 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 to be presented
at WORLDSymposiumTM in February
2024Ultragenyx will present new data from the ongoing
pivotal Transfer A study evaluating the efficacy and safety of
UX111 in children with MPS IIIA at the 20th Annual
WORLDSymposiumTM. The presentation will show that reductions of
heparan sulfate exposure in cerebrospinal fluid correlate with
improved long-term cognitive function in patients with MPS IIIA
following treatment with UX111. Discussions with FDA seeking an
accelerated review path are ongoing.
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 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.
About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing
novel products to patients for the treatment of serious rare and
ultrarare 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.
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 the Company’s
clinical development programs, commercial success of its products
and product candidates, continued 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 candidate.
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-/).
Contacts Ultragenyx Pharmaceutical
Inc.InvestorsJoshua
Higair@ultragenyx.com
MediaCarolyn Wang media@ultragenyx.com
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