TransCon™ hGH (lonapegsomatropin) Achieved Statistically Superior Outcomes Compared to Placebo in Phase 3 foresiGHt Trial of Adults with Growth Hormone Deficiency
19 Dezembro 2023 - 10:30AM
Ascendis Pharma A/S (Nasdaq: ASND) today announced positive topline
results from foresiGHt, its Phase 3 randomized, parallel-arm,
placebo-controlled (double-blind) and active-controlled
(open-label) trial to compare the efficacy and safety of TransCon
hGH (lonapegsomatropin) with placebo and daily hGH (human growth
hormone, somatropin) in adults with growth hormone deficiency
(GHD).
The foresiGHt Trial evaluated 259 adults with GHD aged 23 to 80
years old, randomized 1:1:1, titrated to receive a target fixed
dose of TransCon hGH, placebo, or daily hGH based on age and oral
estrogen intake with approximately equivalent hGH mg/week for
TransCon hGH and daily hGH.
- TransCon hGH demonstrated superiority on its primary efficacy
endpoint at Week 38:
- Change from baseline in trunk percent fat as measured by dual
x-ray absorptiometry (TransCon hGH -1.67% vs. placebo +0.37%, LS
mean difference = -2.04%, p < 0.0001)
- TransCon hGH demonstrated superiority on its key secondary
efficacy endpoints at Week 38:
- Change from baseline in total body lean mass (TransCon hGH
+1.60 kg vs placebo -0.10 kg, LS mean difference = 1.70 kg, p <
0.0001)
- Change from baseline in trunk fat mass (TransCon hGH -0.48 kg
vs placebo +0.22 kg, LS mean difference = -0.70 kg, p =
0.0053)
- Exploratory post-hoc analysis at Week 38 demonstrated
comparable treatment effect of TransCon hGH and daily hGH on target
tissues. For patients with IGF-1 SDS levels ≤ 1.75 at Week 38:
- Change from baseline in trunk percent fat (TransCon hGH -2.42%
vs. daily hGH -2.59%)
- Change from baseline in total body lean mass (TransCon hGH
+1.70 kg vs daily hGH +1.37 kg)
- Change from baseline in trunk fat mass (TransCon hGH -0.90 kg
vs daily hGH -0.94 kg)
- TransCon hGH was generally safe and well tolerated, with no
discontinuations related to study drug and with comparable safety
and tolerability to daily hGH.
“TransCon hGH, our once-weekly growth hormone, is designed to
deliver unmodified somatropin, thereby preserving the same overall
endocrine benefits inherent in endogenous and daily hGH,” said Jan
Mikkelsen, Ascendis Pharma’s President and CEO. “Next year, we plan
to submit a supplemental Biologics License Application to the U.S.
FDA for the adult GHD indication and look forward to reporting data
from our Phase 2 program in Turner syndrome.”
About Adult Growth Hormone DeficiencyGrowth
hormone plays an essential role in the health of children and
adults, promoting normal growth in children and maintenance of
normal body composition and cardiometabolic health throughout
adulthood. In adults, growth hormone boosts protein production,
promotes fat utilization, enhances muscle mass, and helps regulate
blood sugar levels. Adult GHD is a condition in which an
individual’s body does not produce enough growth hormone. Symptoms
and morbidity can include central obesity, metabolic syndrome,
decreased bone density, alterations in lipid profile and markers of
cardiovascular risk, fatigue, general weakness, lack of muscle
tone, and psychological symptoms such as cognitive impairment,
social isolation, lack of motivation, and depression.1
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 hGH’s
ability to preserve the same overall endocrine benefits inherent in
endogenous and daily somatropin, (ii) Ascendis’ plan to submit a
supplemental Biologics License Application to the U.S. FDA next
year, (iii) Ascendis’ plan to report data from its Phase 2 program
in Turner syndrome, (iv) 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
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.
Investor Contacts:Tim LeeAscendis Pharma+1 (650)
374-6343tle@ascendispharma.comir@ascendispharma.com |
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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1. Hoffman AR, Mathison T, Andrews D, Murray K, Kelepouris N,
Fleseriu M. Adult Growth Hormone Deficiency: Diagnostic and
Treatment Journeys From the Patients' Perspective. J Endocr Soc.
2022;6(7):bvac077. Published 2022 May 12.
doi:10.1210/jendso/bvac077
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