Ascendis Presents TransCon™ PTH Data at the American Thyroid Association Annual Meeting
02 Outubro 2023 - 9:00AM
Ascendis Pharma A/S (Nasdaq: ASND) presented a poster with results
of a sub-analysis of its Phase 3 PaTHway clinical trial data during
the recent American Thyroid Association (ATA) annual meeting,
showing that 81% of adults with chronic post-surgical
hypoparathyroidism (n = 70) treated with TransCon PTH
(palopegteriparatide) achieved independence from conventional
calcium and active vitamin D therapy while maintaining normal serum
calcium levels during the 26-week blinded portion of the trial,
compared to 6% taking placebo. The results were comparable to those
from the overall PaTHway Trial population (N = 82), which
demonstrated consistency in improved outcomes with TransCon PTH
treatment compared to conventional therapy across disease
etiologies.
TransCon PTH is an investigational prodrug with sustained
release of active parathyroid hormone (PTH [1-34]) administered
once daily. TransCon PTH (palopegteriparatide) has received a
positive CHMP opinion recommending approval in the European Union
and is in development in the United States, and Japan for the
treatment of adults with hypoparathyroidism.
“We are pleased to share with thyroid surgeons and
thyroidologists attending ATA this data demonstrating the safety
and benefits of TransCon PTH seen in clinical trial patients whose
disease arose following neck surgeries,” said Aimee Shu, M.D., Vice
President of Clinical Development, Endocrinology Medical Sciences
at Ascendis Pharma. “Our goal is to provide a treatment option that
can help patients overcome the serious health and quality of life
burdens associated with hypoparathyroidism, regardless of its
cause. We will continue to advance TransCon PTH globally as a
candidate to address the urgent needs of this patient
community.”
PaTHway is a Phase 3 trial with a completed 26-week randomized,
double-blind, placebo-controlled period, followed by an
ongoing 156-week open-label extension period, designed to evaluate
the efficacy, safety, and tolerability of TransCon PTH in adults
with hypoparathyroidism. A total of 84 patients were randomized 3:1
to treatment with TransCon PTH (n = 63) or placebo (n = 21), both
co-administered with conventional therapy. The multi-component
primary efficacy endpoint of PaTHway was defined as normocalcemia
(a normal concentration of calcium in the blood; 8.3–10.6 mg/dL),
independence from conventional therapy (no active vitamin D and ≤
600 mg/day calcium), and no TransCon PTH dose change within the 4
weeks prior to Week 26. Key secondary endpoints included
patient-reported assessments of disease impact using the
Hypoparathyroidism Patient Experience Scale (HPES) and the 36-Item
Short Form Survey (SF-36 version 2). Of the 84 patients randomized,
82 received study drug. Of those, the 70 with post-surgical
hypoparathyroidism (52 TransCon PTH, 18 placebo) were included in
the sub-analysis population.
PaTHway Trial Sub-Analysis HighlightsAcross
multiple evaluated parameters in participants with post-surgical
hypoparathyroidism, treatment with TransCon PTH enabled
independence from conventional therapy (no active vitamin D and ≤
600 mg/day calcium); improvement of hypoparathyroidism-specific
symptoms and their impact on daily life and
physical functioning; and normalization of mean 24-hour urine
calcium excretion. This includes:
- At Week 26, 81% of patients with
postsurgical hypoparathyroidism treated with TransCon PTH in the
trial achieved independence from conventional therapy while
maintaining normocalcemia compared to 6% treated with placebo.
- At Week 26, 81% (42/52) of patients
with chronic post-surgical hypoparathyroidism treated with TransCon
PTH demonstrated significant improvement compared to those on
placebo in disease-related symptoms and the impact of
hypoparathyroidism on physical functioning and daily life (all p
< 0.01).
- Health-related quality of life, as
measured by the SF-36 physical functioning subscale score, improved
significantly with TransCon PTH treatment compared to placebo in
participants with post-surgical hypoparathyroidism (p =
0.0063).
- From baseline to Week 26, patients
with postsurgical hypoparathyroidism treated with TransCon PTH in
the trial showed a normalization and greater reduction in urine
calcium excretion than those on placebo.
- Treatment with TransCon PTH was
generally safe and well-tolerated. Most treatment-emergent adverse
events (TEAEs) were mild or moderate, and no patients discontinued
study treatment or the trial due to a treatment-related TEAE.
About Ascendis Pharma A/SAscendis Pharma is
applying its innovative platform technology 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, the company 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) the potential
value of TransCon PTH, if approved, as a treatment option for
adults with hypoparathyroidism; (ii) the ability of TransCon PTH to
provide stable PTH levels within the physiological range 24
hours/day; (iii) Ascendis’ ability to achieve its goal of providing
a treatment option that can help patients overcome the serious
health and quality of life burdens seen with hypoparathyroidism;
(iv) Ascendis’ plans to continue to advance TransCon PTH globally
as a candidate to address the needs of the hypoparathyroidism
patient community; (v) the potential approval of TransCon PTH in
the European Union; (vi) Ascendis’ ability to apply its TransCon
platform technology to build a leading, fully integrated, global
biopharma company; and (vii) 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. © October 2023 Ascendis Pharma A/S.
Investor Contacts: |
Media Contact: |
Tim Lee |
Melinda Baker |
Ascendis Pharma |
Ascendis Pharma |
+1 (650) 374-6343 |
+1 (650) 709-8875 |
tle@ascendispharma.com |
media@ascendispharma.com |
ir@ascendispharma.com |
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Patti Bank |
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ICR Westwicke |
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