New Post Hoc Analysis Showed Substantial Improvement in Observed Estimated Glomerular Filtration Rate (eGFR), in TransCon PTH-Treated Adults with Hypoparathyroidism
05 Setembro 2023 - 5:01PM
Ascendis Pharma A/S (Nasdaq: ASND) today announced new post hoc
analysis showing adults with hypoparathyroidism treated with
TransCon PTH demonstrated substantial improvement in estimated
glomerular filtration rate (eGFR), suggesting improved kidney
function. TransCon PTH (palopegteriparatide) is an investigational
prodrug of parathyroid hormone (PTH 1-34) administered once daily
designed to provide sustained release of active PTH within the
physiological range for 24 hours per day in adult patients with
hypoparathyroidism.
“Chronic kidney disease is one of the most consequential
complications of chronic hypoparathyroidism and its treatment with
conventional therapy. Diminished kidney function is associated with
its own set of complications, including hypertension, edema, and
weakness,” said Dr. Aliya Khan, M.D., Clinical Professor of
Medicine at McMaster University and Director of the Calcium
Disorders Clinic at McMaster University Medical Center. “The
observations in the post hoc analysis of the PaTHway Trial,
suggesting that treatment with TransCon PTH reverses impaired
kidney function in patients with hypoparathyroidism, further
substantiating the promise for a major improvement in treatment
opportunities and outcomes for people living with
hypoparathyroidism.”
In the Phase 3 PaTHway Trial, mean baseline eGFR was 67.3 and
72.7 mL/min/1.73m2 for subjects randomized to TransCon PTH and
placebo, respectively. At Week 26, patients treated with TransCon
PTH experienced a mean increase in eGFR of 7.9 mL/min/1.73m2
compared to baseline (p<0.0001) while those on placebo
experienced a mean decrease in eGFR of -1.9 mL/min/1.73m2 compared
to baseline (p=0.3468). By Week 52, patients treated with TransCon
PTH, including those crossing over from placebo, experienced a mean
increase in eGFR of 8.9 mL/min/1.73m2 compared to baseline
(p<0.0001). The improvement at Week 52 was even greater, with
patients with eGFR <60 at baseline, the threshold for kidney
dysfunction, experiencing a mean increase in eGFR of 11.5
mL/min/1.73m2.
PaTHway: eGFR Change from Baseline by eGFR
Group |
|
Baseline |
Week 26 |
Week 52 |
Study Arm |
eGFR(mL/min/1.73m2) |
N |
Mean(p value) |
N |
Mean(p value) |
TransCon PTH / TransCon PTH |
eGFR < 60 |
19 |
+11.4(p=0.0002) |
19 |
+11.5(p=0.0003) |
eGFR ≥ 60 |
41 |
+6.3(p=0.0002) |
40 |
+8.2(p <0.0001) |
All |
60 |
+7.9(p< 0.0001) |
59 |
+9.3(p<0.0001) |
Placebo (first 26 weeks) / TransCon PTH* |
eGFR < 60 |
4 |
+0.05(p=0.9877) |
4 |
+11.7(p=0.0018) |
eGFR ≥ 60 |
15 |
-2.4(p=0.3280) |
15 |
+6.5(p=0.0199) |
All |
19 |
-1.9(p=0.3468) |
19 |
+7.6(p=0.0014) |
eGFR (an assessment of kidney filtering capacity) was calculated
by the trial’s central lab using the Modification of Diet in Renal
Disease Study Group (MDRD) equation (Levey, Ann Intern Med 2006).
*Patients in the placebo arm switched to TransCon PTH following the
Week 26 visit.
Among subjects with baseline eGFR < 60 mL/min/m2 (considered
the threshold for impaired kidney function), approximately 50% were
able to improve their eGFR to > 60 mL/min with TransCon PTH
therapy.
|
|
Number of Responders* |
Number of Responders* |
|
eGFR < 60 |
(n, %) |
(n, %) |
|
at Baseline (n) |
Week 26 |
Week 52 |
TransCon PTH / TransCon PTH |
n=19 |
n=12 |
n=10 |
63% |
53% |
Placebo (first 26 weeks) / TransCon PTH** |
n=4 |
n=0 |
n=3 |
0% |
75% |
Total PaTHway Trial |
n=23 |
n=12 |
n=13 |
52% |
57% |
eGFR based on central lab data using the MDRD Study Group
formula.* Responders defined as moving from eGFR < 60 to eGFR ≥
60. Units in (mL/min/1.73m2).** Patients in the placebo arm
switched to TransCon PTH following the Week 26 visit.
“We will continue to study this important data and topic, which
suggests the potential of TransCon PTH to address physician and
patient concerns about soft-tissue calcifications and decreased
kidney function associated with conventional therapy,” said Aimee
Shu, M.D., Vice President, Clinical Development, Endocrine &
Rare Diseases at Ascendis Pharma. “In addition to reducing risk for
patients, this could also help significantly reduce the healthcare
burden associated with hypoparathyroidism. We look forward to
presenting detailed results at an upcoming medical conference.”
About Ascendis Pharma A/SAscendis Pharma is
applying its innovative platform technology 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, 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, Berlin 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 StatementsThis 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 PTH’s
ability to sustained release of active PTH within the physiological
range for 24 hours per day in adult patients with
hypoparathyroidism; (ii) the potential of TransCon PTH’s ability to
reverse impaired kidney function in patients with
hypoparathyroidism; (iii) the potential of TransCon PTH to address
physician and patient concerns about soft-tissue calcifications and
decreased kidney function associated with conventional therapy,
(iv) TransCon PTH’s ability to reduce risk for the patients and
help reduce the healthcare burden associated with
hypoparathyroidism, (v) Ascendis’ ability to apply its TransCon
technology platform to build a leading, fully integrated, global
biopharma company, and (vi) 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, 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. © September 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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