One-Year Data from Phase 3 Trial of TransCon™ PTH in Adults with Hypoparathyroidism Presented at ENDO 2023
17 Junho 2023 - 6:30PM
Ascendis Pharma A/S (Nasdaq: ASND) today reported one-year (Week
52) data from its ongoing Phase 3 PaTHway Trial of TransCon PTH in
adults with hypoparathyroidism. The data showed that treatment with
TransCon PTH resulted in sustained improvements through Week 52, as
well as safety and tolerability similar to that reported for the
initial 26-week blinded portion of the trial. The data were
presented by Bart Clarke, M.D., endocrinologist and Professor of
Medicine at the Mayo Clinic (Rochester, MN), during ENDO 2023, the
annual meeting of the Endocrine Society being held in Chicago.
“We are very pleased to see sustained improvements in clinical
outcomes in this trial, including symptom and health-related
quality of life measures, consistent with those reported earlier
for the initial 26-week blinded portion,” said Jan Mikkelsen,
Ascendis Pharma’s President and Chief Executive Officer. “These
clinical and patient-reported data, and the retention of 145
patients in our ongoing clinical trials, reinforces our confidence
that TransCon PTH has the potential, if approved, to benefit adults
with hypoparathyroidism regardless of disease etiology.”
MethodsPaTHway is a Phase 3 trial of TransCon
PTH with a placebo (PBO)-controlled 26-week blinded portion and a
156-week open-label extension (OLE) portion, designed to evaluate
the long-term efficacy and safety of TransCon PTH as a potential
hormone therapy for those diagnosed with hypoparathyroidism.
Results through Week 52 (26 weeks blinded + 26 weeks OLE) were
reported at ENDO 2023. Of the 82 study participants dosed, 79
completed blinded treatment and entered the OLE, and 78 (59
TransCon PTH/TransCon PTH, 19 PBO/TransCon PTH) completed Week
52.
Week 52 Highlights
- 95% of patients in the OLE (74 out
of 78) achieved independence from conventional therapy (defined as
no active vitamin D and calcium supplements of <600mg/day), and
none required active vitamin D.
- At Week 52, 81% of participants
treated with TransCon PTH achieved both normal serum calcium and
independence from conventional therapy.
- With TransCon PTH treatment, mean
albumin-adjusted serum calcium levels were maintained within the
normal range (8.3–10.6 mg/dL) through Week 52 of the OLE (8.9 mg/dL
at Week 52).
- Patient-reported scores on the
Hypoparathyroidism Patient Experience Scale (HPES) and SF-36 Health
Survey showed sustained improvements in disease-related physical
and cognitive symptoms, as well as physical functioning and daily
life, starting at the first scheduled follow up after randomization
or switching from placebo and sustained through Week 52.
- Bone mineral density (BMD) Z-scores
continued to trend toward age- and sex-matched norms with 52 weeks
of TransCon PTH treatment.
- TransCon PTH normalized 24-hour
urine calcium through Week 52, regardless of initial randomization
(placebo or TransCon PTH).
- TransCon PTH continued to be well-tolerated in the Phase 3
open-label extension, with no new safety signal identified. Most
TEAEs were mild or moderate (Grades 1-2) and none reported during
the open-label extension through Week 52 led to discontinuation of
the study drug or trial.
Slides showing the data presented for Week 52 of the Phase 3
PaTHway trial of TransCon PTH in adults with hypoparathyroidism can
be viewed on the Ascendis Pharma Investors & News website at
https://investors.ascendispharma.com.
About Ascendis Pharma A/SAscendis Pharma is
applying its innovative TransCon technology platform to build a
leading, fully integrated, global 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, Berlin and
Munich) and the United States (Palo Alto and Redwood City,
California, and Princeton, New Jersey). 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
approval of TransCon PTH, (ii) TransCon PTH’s ability to benefit
adults with hypoparathyroidism regardless of disease etiology,
(iii) Ascendis’ ability to apply its TransCon technology platform
to build a leading, fully integrated, global biopharma company, and
(v) 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 its
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, and 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. © June 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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