Ascendis Pharma Presents Results from Long-Term enliGHten Trial of TransCon™ hGH in Pediatric Growth Hormone Deficiency
23 Setembro 2023 - 8:00AM
Ascendis Pharma A/S (Nasdaq: ASND) today announced results from
enliGHten, the Company’s open-label extension trial evaluating the
long-term safety and efficacy of TransCon hGH as a once-weekly
treatment for children and adolescents with growth hormone
deficiency (GHD). Results were shared today in Poster LB-17 at ESPE
2023, the annual meeting of the European Society for Paediatric
Endocrinology being held in The Hague, September 21-23.
The enliGHten trial enrolled 298 participants (mean age 10.3
years) from the Phase 3 heiGHt Trial of treatment-naïve pediatric
GHD patients and the Phase 3 fliGHt Trial of pediatric GHD patients
switching from daily somatropin treatment. Patients in these trials
received a total of up to 6 years of treatment with TransCon
hGH.
At the time of the enliGHten Trial conclusion, 81 participants
were designated as treatment completers, based on their physician’s
determination that treatment for pediatric GHD was no longer
required. Of these treatment completers, 59% met or exceeded their
average parental height SDS, with mean TransCon hGH treatment
duration of 3.2 years. Treatment completers’ baseline mean height
standard deviation score (SDS) at the beginning of the open-label
extension trial was -1.6, compared to mean height SDS of -0.4
(achieving height similar to their parents’) at their final study
visit. At the time of final visit, all treatment completers were
Tanner stage IV or V, a categorization of physical development
during puberty.
“As an investigator in the enliGHten Trial, I am pleased to see
results confirming that treated children and adolescents have
continued to grow well, achieving statures in line with those of
their parents,” said Aristides K. Maniatis, M.D., F.A.A.P.,
pediatrician and endocrinologist at Rocky Mountain Pediatric
Endocrinology. “Additionally, these results demonstrate that
long-term treatment goals can be safely reached with TransCon hGH
administered once weekly.”
TransCon hGH was generally safe and well-tolerated. The most
commonly reported adverse events over the course of the trial were
categorized as infections, injury, and respiratory/thoracic/medical
disorders. The majority of adverse events were mild in severity and
unrelated to treatment. No adverse events led to discontinuation of
the study treatment.
About TransCon hGH (Lonapegsomatropin)TransCon
hGH (lonapegsomatropin) is a prodrug of somatropin administered
once weekly, designed to provide sustained release of active,
unmodified somatropin. The unmodified, unbound somatropin released
from lonapegsomatropin has the identical 191 amino-acid sequence
and size (22 kDa) as endogenous growth hormone. TransCon hGH is
approved and marketed as SKYTROFA® (lonapegsomatropin-tcgd) in the
United States and as SKYTROFA® (lonapegsomatropin) in the European
Union as a once-weekly treatment for children and adolescents with
GHD.
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) TransCon hGH’s
ability to provide sustained release of active, unmodified
somatropin; (ii) Ascendis’ ability to apply its TransCon platform
technology to build a leading, fully integrated, global biopharma
company, and (iii) 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, TransCon, and SKYTROFA® are trademarks owned by the Ascendis
Pharma group. © September 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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