Significant Health and Quality of Life Improvements Achieved in Children with Achondroplasia Treated for One Year with TransCon™ CNP (Navepegritide) at 100 µg/kg/week
December 20 2023 - 8:30AM
Ascendis Pharma A/S (Nasdaq: ASND) today announced new analyses
from the blinded and ongoing open-label extension (OLE) portions of
ACcomplisH, the Company’s Phase 2 randomized, double-blind,
placebo-controlled, dose-escalation trial of TransCon CNP in
children ages 2-10 years with achondroplasia. In the trial, all 57
patients have now completed one year of treatment with TransCon CNP
(navepegritide) at 100 µg/kg/week, the dose agreed with regulatory
agencies for the active arm in the pivotal ApproaCH Trial.
Ascendis analyzed available data for patients who only received
TransCon CNP at the 100 µg/kg/week dose in either blinded or OLE
part and were treated for one year (n=19), compared to those
administered placebo for one year (n=15). Results showed that these
TransCon CNP treated patients (data available for 9-16 patients)
showed significant improvements in health-related quality of life
and disease impacts compared to those receiving placebo (data
available for 5-13 patients).
Assessments were performed with the SF-10 and Achondroplasia
Child Experience Measure (ACEM), with statistically significant
improved outcome in TransCon CNP treated versus placebo for:
- SF-10 Physical Summary (p=0.002, ages 5 years and older)
- ACEM Daily Living Function (p=0.047)
- ACEM Emotional Well-being (p=0.045)
The 46 children switching from placebo or a lower dose of
TransCon CNP to the 100 µg/kg/week dose in the OLE demonstrated
improved growth after one year of treatment, similar to the growth
benefits seen in the 11 children treated with 100 µg/kg/week in the
one-year randomized, double-blind period of ACcomplisH.
“These data support our aspirations for TransCon CNP, the first
investigational product to go beyond height by improving the signs
and symptoms of achondroplasia. We believe that the observed
treatment benefits are likely contributing to the continued strong
patient retention in our trials,” said Jan Mikkelsen, President and
Chief Executive Officer at Ascendis Pharma. “We remain confident
that TransCon CNP has the potential to deliver meaningful benefits
to children with achondroplasia, and we will explore effects of
TransCon CNP in adults with achondroplasia in the years ahead.”
TransCon CNP (navepegritide) is an investigational prodrug of
CNP, administered once weekly and designed to provide sustained
release of active CNP supporting continuous exposure for the
treatment of children with achondroplasia.
Further details of the results will be shared early next
year.
About Ascendis Pharma A/SAscendis Pharma is
applying its innovative TransCon technology platform to build a
leading, fully integrated biopharma company focused on making a
meaningful difference in patients’ lives. Guided by its core values
of patients, science and passion, Ascendis uses its TransCon
technologies to create new and potentially best-in-class therapies.
Ascendis is headquartered in Copenhagen, Denmark and has additional
facilities in Germany (Heidelberg and Munich) and the United States
(Palo Alto and Redwood City, California, and Princeton, New
Jersey). Please visit ascendispharma.com to learn more.
Forward-Looking Statements This press release
contains forward-looking statements that involve substantial risks
and uncertainties. All statements, other than statements of
historical facts, included in this press release regarding
Ascendis’ future operations, plans and objectives of management are
forward-looking statements. Examples of such statements include,
but are not limited to, statements relating to (i) TransCon CNP’s
ability to go beyond height by improving the signs and symptoms of
achondroplasia, (ii) Ascendis’ belief that the treatment benefits
of TransCon CNP contributed to the continued strong patient
retention in Ascendis’ trials, (iii) TransCon CNP’s ability to
deliver meaningful benefits to children with achondroplasia, (iv)
Ascendis’ intent to explore the effects of TransCon CNP in adults
with achondroplasia in the years ahead, (v) TransCon CNP’s ability
to provide sustained release of active CNP thereby supporting
continuous exposure of CNP for the treatment of children with
achondroplasia, (vi) the timing for sharing further details of the
results of the ACcomplisH Trial, (vii) Ascendis’ ability to apply
its TransCon technology platform to build a leading, fully
integrated global biopharma company, and (viii) Ascendis’ use of
its TransCon technologies to create new and potentially
best-in-class therapies. Ascendis may not actually achieve the
plans, carry out the intentions or meet the expectations or
projections disclosed in the forward-looking statements and you
should not place undue reliance on these forward-looking
statements. Actual results or events could differ materially from
the plans, intentions, expectations and projections disclosed in
the forward-looking statements. Various important factors could
cause actual results or events to differ materially from the
forward-looking statements that Ascendis makes, including the
following: dependence on third party manufacturers, distributors
and service providers for Ascendis’ products and product
candidates; unforeseen safety or efficacy results in Ascendis’
development programs or on-market products; unforeseen expenses
related to commercialization of any approved Ascendis products;
unforeseen expenses related to Ascendis’ development programs;
unforeseen selling, general and administrative expenses, other
research and development expenses and Ascendis’ business generally;
delays in the development of its programs related to manufacturing,
regulatory requirements, speed of patient recruitment or other
unforeseen delays; Ascendis’ ability to obtain additional funding,
if needed, to support its business activities; the impact of
international economic, political, legal, compliance, social and
business factors, including inflation, the effects on its business
from the worldwide COVID-19 pandemic and ongoing conflicts such as
that in the region surrounding Ukraine and Russia. 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 Ascendis’ business in
general, see Ascendis’ Annual Report on Form 20-F filed with the
U.S. Securities and Exchange Commission (SEC) on February 16, 2023
and Ascendis’ other future reports filed with, or submitted to, the
SEC. Forward-looking statements do not reflect the potential impact
of any future licensing, collaborations, acquisitions, mergers,
dispositions, joint ventures, or investments that Ascendis may
enter into or make. Ascendis does not assume any obligation to
update any forward-looking statements, except as required by
law.
Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company
logo, and TransCon are trademarks owned by the Ascendis Pharma
group. © December 2023 Ascendis Pharma A/S.
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Investor Contacts: |
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Media Contact: |
Tim Lee |
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Melinda Baker |
Ascendis Pharma |
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Ascendis Pharma |
+1 (650) 374-6343 |
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+1 (650) 709-8875 |
tle@ascendispharma.com |
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media@ascendispharma.com |
ir@ascendispharma.com |
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Patti Bank |
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ICR Westwicke |
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+1 (415) 513-1284 |
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patti.bank@westwicke.com |
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