Ultragenyx Announces Program and Pipeline Updates at Analyst Day Including Interim Data from Ongoing Studies in Osteogenesis Imperfecta (OI), Angelman Syndrome (AS) and Wilson Disease
October 16 2023 - 7:00AM
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today will provide
updates on its development pipeline, including setrusumab (UX143)
for osteogenesis imperfecta (OI), GTX-102 for Angelman syndrome
(AS), UX701 in Wilson disease and the rest of the company’s gene
therapy portfolio at an Analyst Day held in New York City and by
webcast.
“The data we are presenting today show that these
investigational therapies are having meaningful clinical effects on
difficult diseases with limited or no approved treatments and are
potentially transformative for people living with these diseases if
proven safe and effective in Phase 3 studies,” said Emil D. Kakkis,
M.D., Ph.D., chief executive officer and president of Ultragenyx.
“We also have one of the largest, late-stage gene therapy pipelines
in rare disease with full end-to-end capabilities in-house that
uniquely position us to deliver high quality robust commercial
manufacturing at scale to support our large pipeline.”
Analyst Day Updates
UX143 (setrusumab) monoclonal antibody for Osteogenesis
Imperfecta (OI): Interim Phase 2 data from the Phase 2/3 Orbit
study show statistically significant decrease in annualized
fracture rates following at least 6 months of
treatment
- Data presented at the American
Society for Bone and Mineral Research 2023 Annual Meeting (ASBMR)
show that treatment with setrusumab reduced the annualized fracture
rate by 67% and this reduction was associated with continuing large
and meaningful improvements in bone mineral density (BMD).
- Setrusumab was generally well
tolerated with no drug related serious adverse events (SAEs)
reported and no reports of drug-related hypersensitivity.
- The company plans to provide updated
Phase 2 data next year.
GTX-102 antisense oligonucleotide for Angelman syndrome:
Data from the extension cohorts in the Phase 1/2 study show
clinically meaningful improvements in multiple domains
- Quantitative data show improvements
across multiple clinical 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.
- There have been no additional
treatment-related SAEs, including lower extremity weakness, since
November 2022.
- Data from the dose expansion cohorts
on at least 20 patients who have been on therapy for at least 6
months is anticipated in the first half of 2024.
UX701 AAV gene therapy for Wilson disease: Four of five
patients in the lowest-dose cohort of the Phase 1/2/3
Cypress 2+ study show
improvements in tapering standard of care
- Four out of 5 patients in the
low-dose Cohort 1 have had reductions in urinary copper and are
tapering off of chelators and/or zinc therapy, including 2 of 3
earlier treated patients in the Cohort that are now completely off
standard therapy.
- UX701 has been generally well
tolerated with no treatment-related SAEs.
- The seamless study is expected to
complete dosing of all 3 dose cohorts in Stage 1 at the end of 2023
and these data are expected in the first half of 2024.
Company also provided update on other late-stage gene
therapy candidates
- DTX401 AAV gene therapy for
Glycogen Storage Disease Type Ia (GSDIa): The Phase 3
GlucoGene study was fully enrolled in the first quarter of 2023 and
the company plans to provide preliminary data in the first half of
2024.
- UX111 for Sanfilippo
syndrome (MPS IIIA): The pivotal Transpher A study has
been fully enrolled and the company plans to meet with the FDA in
the fourth quarter of 2023.
- DTX301 AAV gene therapy for
Ornithine Transcarbamylase (OTC) Deficiency: The Phase 3
Enh3ance study is expected to complete enrollment in the first half
of 2024.
Analyst Day and Webcast Information
Ultragenyx will host an Analyst Day at 8:30 a.m. ET on Monday,
October 16, 2023 to discuss these data and to provide an update on
the company’s development pipeline. A live video webcast of the
program will be available at
https://www.webcaster4.com/Webcast/Page/359/49192. An archived
version of the remarks will also be available through the
Ultragenyx website.
About UltragenyxUltragenyx is a
biopharmaceutical company committed to bringing novel products to
patients for the treatment of serious rare and ultra-rare 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 ultragenyx.com.
Ultragenyx Forward-Looking Statements and Use of Digital
MediaExcept 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,
business plans and objectives for UX143, expectations regarding the
clinical benefit, tolerability and safety of UX143, future clinical
and regulatory developments for UX143, the clinical benefit,
tolerability and safety of GTX-102, future clinical and regulatory
developments for GTX-102, the clinical benefit, tolerability and
safety of UX701, future clinical and regulatory developments for
UX701, timing for enrollment, dosing and data for Ultragenyx’s
investigational therapies and gene therapy candidates and
regulatory meetings 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, the ability of the
company and Mereo BioPharma to successfully develop UX143, the
company’s ability to achieve its projected development goals in its
expected timeframes, risks related to adverse side effects, risks
related to reliance on third party partners to conduct certain
activities on the company’s behalf, the potential for any license
or collaboration agreement, including the company’s collaboration
agreement with Mereo to be terminated, 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-/).
Contacts
Ultragenyx Pharmaceutical Inc.
InvestorsJoshua Higa415-475-6370ir@ultragenyx.com
MediaCarolyn Wang415-225-5050media@ultragenyx.com
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