Ascendis Pharma A/S Provides Update on Vision 3x3 Strategic Roadmap at 40th Annual J.P. Morgan Healthcare Conference
11 January 2022 - 12:00AM
Ascendis Pharma A/S (Nasdaq: ASND) today announced that the Company
will today provide an update on its Vision 3x3 and planned 2022 key
milestones at the 40th Annual J.P. Morgan Healthcare Conference.
Details of the update are outlined below, and CEO Jan Mikkelsen
will give a virtual presentation to attendees from 3:00-3:40 p.m.
Eastern Time.
“We took major steps in 2021 to becoming a viable, sustainable,
and profitable biopharmaceutical company,” said Jan Mikkelsen,
Ascendis Pharma’s President and Chief Executive Officer. “A strong
flow of clinical results and our first commercial TransCon™ product
showcased how we are leveraging our technology platform to build a
leading global biopharmaceutical company able to successfully meet
the needs of patients with differentiated product candidates.”
“This year, I am happy to report that our U.S. launch of
SKYTROFA® (lonapegsomatropin-tcgd), the first U.S. FDA-approved
once-weekly treatment for pediatric growth hormone deficiency is
off to a strong start,” continued Mr. Mikkelsen. “In only two short
months since launch, we have already seen 10% of our target
prescribers’ writing prescriptions for SKYTROFA instead of daily
growth hormone.”
“During 2022, we look forward to sharing multiple clinical data
readouts across our high-value endocrinology rare disease and
oncology portfolios,” continued Mr. Mikkelsen. “We believe the
clinical readouts will demonstrate our ability to deliver therapies
that make a meaningful impact on patients’ lives. In addition, we
look forward to announcing a third independent therapeutic area
with its own diversified pipeline in the fourth quarter of
2022.”
Pipeline Updates
- TransCon hGH: TransCon hGH is an
investigational once-weekly prodrug designed to deliver somatropin
over a one-week period. TransCon hGH is approved by the FDA in the
U.S. under the brand name SKYTROFA (lonapegsomatropin-tcgd) for the
treatment of pediatric patients one year and older who weigh at
least 11.5 kg and have growth failure due to inadequate secretion
of endogenous growth hormone.
- In mid-October, the Company commercially launched in the U.S.
TransCon hGH under the brand name SKYTROFA. Since launch, physician
enthusiasm for SKYTROFA is reflected by an increase in
prescriptions, submission of formulary exceptions, and repeat
prescribers. During the fourth quarter, 369 SKYTROFA prescriptions
were written by 139 targeted prescribers, which includes 42% repeat
prescribers.1
- In November 2021, the Company received a positive opinion from
the Committee for Medicinal Products for Human Use (CHMP) of the
European Medicines Agency for TransCon hGH for patients with
pediatric growth hormone deficiency. The European Commission’s
approval of the Marketing Authorisation Application (MAA) is
expected by the end of January 2022.
- During the second quarter of 2022, the Company plans to submit
a protocol to the FDA for TransCon hGH in Turner Syndrome
subjects.
- Ascendis is targeting completion of enrollment in foresiGHt, a
global Phase 3 trial evaluating the safety and efficacy of TransCon
hGH in adult patients with growth hormone deficiency during the
second quarter of 2022.
- TransCon PTH: TransCon PTH is an
investigational long-acting prodrug of parathyroid hormone (PTH) in
development as a potential once-daily replacement therapy for adult
hypoparathyroidism (HP):
- During the first quarter of 2022, top-line results are expected
from PaTHway, a Phase 3 randomized, double-blind,
placebo-controlled clinical trial in North America and Europe,
investigating the safety, tolerability, and efficacy of TransCon
PTH in adults with HP.
- If the Phase 3 PaTHway Trial results are positive, Ascendis
plans to submit a New Drug Application (NDA) to the FDA in the
third quarter of 2022 followed by a MAA submission to the EMA in
the fourth quarter of 2022.
- Top-line results from PaTHway Japan, a single-arm Phase 3 trial
of TransCon PTH in a minimum of 12-Japanese subjects with HP are
expected in the third quarter of 2022.
- Initiation of a pediatric HP program is planned for the fourth
quarter of 2022.
- TransCon CNP: TransCon CNP, an investigational
long-acting prodrug of C-type natriuretic peptide (CNP), as a
potential therapeutic option for patients with achondroplasia
(ACH):
- Top-line data from the ACcomplisH Trial, a Phase 2 randomized,
double-blind, placebo-controlled clinical trial in North America,
Europe, and Oceania in subjects with achondroplasia (age 2–10) are
expected in the fourth quarter of 2022.
- During the second quarter of 2022, the Company plans to file an
Investigational New Drug (IND) application or similar for the
ACcomplisH Infants Trial in subjects with achondroplasia (age
0–2).
- TransCon TLR7/8 Agonist: TransCon TLR7/8
Agonist is an investigational long-acting prodrug of resiquimod, a
small molecule agonist of Toll-like receptors (TLR) 7 and 8
designed to provide sustained activation of intratumoral
antigen-presenting cells driving tumor antigen presentation and
induction of immune stimulatory cytokines for weeks or months with
a single intratumoral injection:
- Enrollment continues in transcendIT-101. Top-line data from
monotherapy and combo-therapy dose escalation expected in the third
quarter of 2022.
- TransCon IL-2 β/γ: TransCon IL-2 β/γ is an
investigational long-acting prodrug designed to improve cancer
immunotherapy by sustained exposure to an IL-2 variant that
selectively activates the IL-2Rβ/γ, with minimal binding to IL-2Rα:
- Top-line monotherapy data from the IL-eliee Trial are
expected in the fourth quarter of 2022.
- The Company expects to dose the first patient in the
combo-therapy and dose escalation arm of the IL-eliee Trial in
the first quarter of 2022.
- TransCon TLR7/8 Agonist and TransCon IL-2 β/γ
Combinations:
- During the fourth quarter of 2022, the Company plans to submit
an IND or similar for Phase 2 cohort expansion for TransCon TLR7/8
Agonist and TransCon IL-2 β/γ.
Presentation at J.P. Morgan Healthcare Conference on
Monday, January 10th The live webcast of the J.P. Morgan
presentation will be available on the Investors & News section
of the Ascendis Pharma website at www.ascendispharma.com. A webcast
replay will be available on this website shortly after conclusion
of the event for 30 days. The Company’s slides from the J.P. Morgan
presentation also will also be available on the Investor Relations
website.
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 Heidelberg and Berlin, Germany; Palo Alto
and Redwood City, California; and Princeton, New Jersey. Please
visit www.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 expected
timing of Ascendis’ announcement of a third independent therapeutic
area, (ii) the expected timing of the European Commission’s final
decision on Ascendis’ Marketing Authorization Application for
TransCon hGH, (iii) Ascendis’ plans to submit a protocol to the FDA
for TransCon hGH in Turner Syndrome subjects, (iv) the expected
timing for completion of enrollment in the foresiGHt Trial, (v) the
expected timing of planned regulatory filings for TransCon PTH to
the FDA and the EMA, (vi) the expected timing of top-line results
from the Phase 3 PaTHway Trial and the PaTHway Japan Trial, (vii)
the expected timing of initiation of a pediatric HP program for
TransCon PTH, (viii) the expected timing of planned regulatory
filings for TransCon CNP, (ix) the expected timing of top-line
results from the ACcomplisH Trial, (x) the expected timing of dose
escalation data for transcendIT-101, (xi) the expected timing of
the IL-eliee Trial, (xii) the expected timing of top-line
monotherapy data for the IL-eliee Trial, (xii) the expected
timing of planned regulatory filings for TransCon TLR7/8 Agonist
and TransCon IL-2 β/γ, (xiv) Ascendis’ ability to apply its
platform technology to build a leading, fully integrated biopharma
company, and (xv) 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
and distributors to supply TransCon hGH, and other study drug for
commercial sales in the U.S. and clinical studies; unforeseen
safety or efficacy results in its oncology programs, TransCon hGH,
TransCon PTH and TransCon CNP or other development programs;
unforeseen expenses related to commercialization of
lonapegsomatropin-tcgd in the U.S., the co-pay program, and the
further development of TransCon hGH, expenses related to the
development and potential commercialization of its oncology
programs, TransCon hGH, TransCon PTH and TransCon CNP or other
development programs, selling, general and administrative expenses,
other research and development expenses and Ascendis’ business
generally; delays in the development of its oncology programs,
TransCon hGH, TransCon PTH and TransCon CNP or other development
programs related to manufacturing, regulatory requirements, speed
of patient recruitment or other unforeseen delays; dependence on
third party manufacturers to supply study drug for planned clinical
studies; Ascendis’ ability to obtain additional funding, if needed,
to support its business activities and the effects on its business
from the worldwide COVID-19 pandemic. 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 March 10, 2021 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. © January 2022 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 |
|
|
Patti Bank |
|
ICR Westwicke |
|
+1 (415) 513-1284 |
|
patti.bank@westwicke.com |
|
ir@ascendispharma.com |
|
1 Fourth quarter SKYTROFA prescription figures have not been
verified by any third party and represent the Company’s estimates.
The Company assumes no obligation to update them.
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