Significant and Sustained Improvements in Renal Function Reported for Adults with Chronic Hypoparathyroidism Treated with TransCon™ PTH (Palopegteriparatide): 2-year Results from Phase 3 PaTHway Trial
May 13 2024 - 8:30AM
Ascendis Pharma A/S (Nasdaq: ASND) today announced 2-year results
from a post-hoc analysis of the Company’s Phase 3 PaTHway Trial
demonstrating significant and sustained improvements in renal
function in adults with chronic hypoparathyroidism treated with
TransCon PTH (palopegteriparatide). The data were shared in an oral
presentation on May 12 by Peter Schwarz, M.D., Professor of
Clinical Medicine at the University of Copenhagen, during the
European Congress of Endocrinology 2024 (ECE 2024), the annual
meeting of the European Society of Endocrinology.
“This unparalleled data showing sustained, clinically meaningful
improvements in kidney function in these patients reinforce the
potential for TransCon PTH to address concerns about soft-tissue
calcifications and decreased kidney function associated with
conventional therapy,” said Aimee Shu, M.D., Ascendis Pharma’s
Senior Vice President of Clinical Development, Endocrine Medical
Sciences.
The post-hoc analysis examined the impact of treatment with
TransCon PTH on renal function using estimated glomerular
filtration rate (eGFR) through Week 104 (n=76) of PaTHway, a Phase
3 double-blind, placebo-controlled trial of 82 dosed adults with
chronic hypoparathyroidism randomized 3:1 (TransCon PTH:placebo;
both arms initially co-administered with conventional therapy of
active vitamin D and oral calcium), with a 26-week blinded period
followed by an ongoing 156-week open-label extension period. Across
both treatment arms, TransCon PTH treatment resulted in a mean eGFR
increase of 8.9 mL/min/1.73m2 (p<0.0001) from baseline at Week
52, sustained at Week 104 with a mean change from baseline of 9.0
mL/min/1.73m2 (p<0.0001). Treatment was generally
well-tolerated, with no new safety signals.
eGFR* Change from Baseline by Study Arm |
|
Baseline |
Week 26 |
Week 52 |
Week 104 |
Study Arm |
eGFR(mL/min/1.73m2) |
N |
Mean(p value) |
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) |
18 |
+13.4(p<0.0001) |
eGFR ≥ 60 |
41 |
+6.3(p=0.0002) |
40 |
+8.2(p<0.0001) |
40 |
+6.9(p<0.0001) |
All |
60 |
+7.9(p<0.0001) |
59 |
+9.3 (p<0.0001) |
58 |
+8.9(p<0.0001) |
Placebo (first 26 weeks) / TransCon PTH** |
eGFR < 60 |
4 |
+0.05 (p=0.9877) |
4 |
+11.7(p=0.0018) |
4 |
+15.6(p=0.0067) |
eGFR ≥ 60 |
15 |
-2.4(p=0.3280) |
15 |
+6.5(p=0.0199) |
14 |
+7.6(p=0.0121) |
All |
19 |
-1.9 (p=0.3468) |
19 |
+7.6 (p=0.0014) |
18 |
+9.4(p=0.0006) |
*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). An eGFR level <60 mL/min/1.73m2 is considered
the threshold for impaired kidney function.
**Patients in the placebo arm switched to TransCon PTH following
the Week 26 visit.
TransCon PTH treatment was associated with clinically meaningful
increases (≥ 5 mL/min/1.73 m2) in eGFR within 26 weeks that were
sustained through Week 104 of PaTHway:
Proportion of Participants (%) with ≥ 5 and ≥ 10
mL/min/1.73 m2 Increases in
eGFR from Baseline through Week
104* |
eGFR Change from Baseline |
All Participants |
TransCon PTH / TransCon
PTH(n=61) |
Placebo (first 26 weeks) / TransCon PTH**
(n=21) |
Week 26 |
Week 52 |
Week 104 |
Week 26 |
Week 52 |
Week 104 |
PTH |
PTH |
PTH |
Placebo |
Switch to PTH |
Switch to PTH |
> 5 mL/min/1.73 m2 |
57% |
64% |
61% |
24% |
52% |
62% |
> 10 mL/min/1.73 m2 |
43% |
43% |
46% |
10% |
39% |
38% |
|
eGFR Change from Baseline |
Participants with Baseline eGFR < 60 mL/min/1.73
m2 |
TransCon PTH / TransCon PTH
(n=19) |
Placebo (first 26 weeks) / TransCon PTH**
(n=4) |
Week 26 |
Week 52 |
Week 104 |
Week 26 |
Week 52 |
Week 104 |
PTH |
PTH |
PTH |
Placebo |
Switch to PTH |
Switch to PTH |
> 5 mL/min/1.73 m2 |
74% |
68% |
74% |
25% |
100% |
100% |
> 10 mL/min/1.73 m2 |
47% |
42% |
53% |
0% |
75% |
75% |
*Percentages were calculated based on all participants. Patients
who did not have an eGFR assessment at the visit were still
included in the denominator.
**Patients in the placebo arm switched to TransCon PTH following
the Week 26 visit.
Highlights from this ECE oral presentation will be made
available on the Investor & News section of the Ascendis
Pharma website
at https://investors.ascendispharma.com.
About HypoparathyroidismHypoparathyroidism is
an endocrine disease caused by insufficient levels of PTH, the
primary regulator of calcium and phosphate balance in the body,
acting directly on bone and kidneys and indirectly on the
intestines. Individuals with hypoparathyroidism may experience a
range of severe and potentially life-threatening short-term and
long-term complications, including neuromuscular irritability,
renal complications, extra-skeletal calcifications, and cognitive
impairment. Post-surgical hypoparathyroidism accounts for the
majority of cases (70-80%), while other etiologies include
autoimmune and idiopathic causes.
About TransCon PTHTransCon PTH
(palopegteriparatide) is a prodrug of parathyroid hormone (PTH
1-34) administered once daily, designed to provide parathyroid
hormone levels within the normal physiological range across the
24-hour dosing period. TransCon PTH was granted marketing
authorization under the brand name YORVIPATH® by the European
Commission (EC) and the European Economic Area (EEA)
in November 2023 and the United Kingdom’s Medicines &
Healthcare Products Regulatory Agency (MHRA) in Great Britain as a
PTH replacement therapy indicated for the treatment of adults with
chronic hypoparathyroidism. In the United States, the Food
& Drug Administration (FDA) has set a PDUFA target action
date of May 14, 2024, to complete their review of
Ascendis Pharma’s New Drug Application for TransCon PTH for adults
with chronic hypoparathyroidism. TransCon PTH is also in
development in Japan through Teijin Ltd. and in
China through VISEN Pharmaceuticals.
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 Europe and the United States. 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
for TransCon PTH to address concerns about soft-tissue
calcifications and decreased kidney function associated with
conventional therapy, (ii) TransCon PTH’s PDUFA date of May 14,
2024, (iii) Ascendis’ ability to apply its TransCon technology
platform to build a leading, fully integrated biopharma company and
(iv) 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. 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 7, 2024 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, TransCon, and YORVIPATH® are trademarks owned by the Ascendis
Pharma group. © May 2024 Ascendis Pharma A/S.
Investor
Contacts: Tim
Lee Ascendis
Pharma +1
(650)
374-6343 tle@ascendispharma.comir@ascendispharma.com |
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Media Contact:Melinda BakerAscendis Pharma+1 (650)
709-8875media@ascendispharma.com |
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Patti BankICR Westwicke+1 (415)
513-1284patti.bank@westwicke.com |
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