Ascendis Pharma Introduces Vision 2030
07 Janvier 2024 - 11:45PM
Ascendis Pharma A/S (Nasdaq: ASND) today introduced selected 2024
corporate milestones and Vision 2030, its strategic roadmap through
2030. Ascendis President and CEO Jan Mikkelsen will present the
update tomorrow, January 8, at the 42nd Annual J.P. Morgan
Healthcare Conference.
“With unwavering focus on our values of Patients, Science, and
Passion, we remain on track to fulfill Vision 3x3, with the
approval already of two Endocrinology Rare Disease products and
value leadership in the U.S. growth hormone market for SKYTROFA,”
said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive
Officer. “Today we are introducing Vision 2030, our strategic
roadmap to attain blockbuster status for multiple products and
expand our engine for future innovation.”
Selected Key Updates
- TransCon™ hGH (lonapegsomatropin,
approved as SKYTROFA® in the U.S. and EU)
- Following positive topline results
from Phase 3 foresiGHt Trial in adult growth hormone deficiency
(GHD), plan to submit a supplemental Biologics License Application
to FDA in the second quarter of 2024.
- Topline results from Phase 2 trial
in Turner syndrome expected in the fourth quarter of 2024.
- Attained U.S. market value
leadership in 2023.
- SKYTROFA preliminary unaudited
fourth quarter 2023 revenue is expected to be approximately €64
million.
- Full year 2024 SKYTROFA revenue
expected to be €320 million to €340 million (based on average 2023
exchange rates).
- TransCon PTH (palopegteriparatide,
approved as YORVIPATH® in the EU)
- First launch planned in Germany in
January 2024, leveraging the Company’s existing commercial
infrastructure.
- FDA regulatory review for the
treatment of adult patients with hypoparathyroidism continues, with
a Prescription Drug User Fee Act (PDUFA) date of May 14, 2024; if
approved, U.S. commercial launch planned in the third quarter of
2024.
- TransCon CNP (navepegritide)
- Treatment with TransCon CNP at 100
mg/kg/week for one year in all 57 children with achondroplasia (age
2 to 10 years) in ACcomplisH, demonstrated growth consistent with
results from the blinded period and achieved improvements in
health-related quality of life and disease impacts assessed with
SF-10 and Achondroplasia Child Experience Measure.
- During the third quarter of 2023,
submitted Clinical Trial Application (CTA) for infant trial (age
0-2 years).
- Topline results from the pivotal
ApproaCH Trial expected in the fourth quarter of 2024.
- During the fourth quarter of 2024,
plan to submit a New Drug Application to FDA for children with
achondroplasia (age 2-11 years).
- Week 26 topline data from the COACH
Trial (TransCon hGH/TransCon CNP combination) expected in the
fourth quarter of 2024.
- During the fourth quarter of 2024,
plan to submit an Investigational New Drug application or similar
in adults with achondroplasia.
- Global commercial presence in
Endocrinology Rare Disease
- During the fourth quarter of 2023,
entered into an exclusive license agreement with Teijin Limited for
TransCon hGH, TransCon PTH, and TransCon CNP in Japan. Phase 3
PaTHway Japan Trial of TransCon PTH completed, and Phase 3 riGHt
Trial of TransCon hGH fully enrolled.
- VISEN’s TransCon hGH Phase 3 and
TransCon CNP Phase 2 trials in China completed.
- Expanding global reach through
exclusive sales & distribution agreements with geographic
market leaders in International Markets, with three regional
agreements signed to date: Specialised Therapeutics Asia Pte Ltd,
Er-Kim, and Vector Pharma FZCO.
- Oncology
- During the fourth quarter of 2024,
expect to complete enrollment in BelieveIT-201, a Phase 2 trial in
advanced head and neck squamous cell carcinoma (HNSCC).
- During the fourth quarter of 2024,
plan to provide a clinical update from the Phase 2 portion of
indication-specific, dose expansion cohorts in the IL-Believe
Trial.
- Ophthalmology
- Creation of Ophthalmology NewCo,
financed by institutional investors, expected during the first
quarter of 2024.
- Financial Update and Outlook Based
on Current Plans
- Preliminary unaudited December 31,
2023 cash, cash equivalents, and marketable securities of ~€400
million.
- Full year 2024 SKYTROFA revenue
expected to be €320 million to €340 million (based on average 2023
exchange rates).
- Expect to provide YORVIPATH revenue
update during 2024.
- Expect total operating expenses
(SG&A and R&D) of approximately €600 million for 2024.
- Expect to be operating cash flow
breakeven on a quarterly basis by the end of 2024.
Vision 2030: Ascendis Pharma’s 2025–2030 Strategic
Roadmap
Achieve blockbuster status for multiple products and expand our
engine for future innovation.
- Be the Leading Endocrinology Rare Disease Company
- Achieve blockbuster status (>$1B) for TransCon PTH, TransCon
hGH, and TransCon CNP through worldwide commercialization
- Be the leader in growth disorders and hypoparathyroidism,
pursuing clinical conditions, innovative life cycle management, and
complementary patient offerings
- Expand pipeline with Endocrinology Rare Disease blockbuster
product opportunities
- Create Value in Additional Therapeutic Areas through Innovative
Business Models
- Obtain accelerated approval in oncology with registrational
trials ongoing
- Pursue TransCon product opportunities in >$5B
indications
- Maximize value creation of these product opportunities through
collaboration with therapeutic area market leaders
- Differentiate with Ascendis Fundamentals
- Outperform industry drug development benchmarks with Ascendis’
product innovation algorithm
- Remain independent as a profitable biopharma through lean and
flexible ways of working
- Let our values Patients, Science, Passion drive our decisions
to success
Presentation at J.P. Morgan Healthcare Conference on
Monday, January 8th
A live webcast of the event will be available via the Investors
& News section of the Ascendis Pharma website at
https://investors.ascendispharma.com. The presentation will begin
at 12:00 p.m. Eastern Time / 9:00 am Pacific Time. A webcast replay
will be available for 30 days.
The Company’s slides from the J.P. Morgan presentation will be
available on the same Investor Relations website at
https://investors.ascendispharma.com.
About Ascendis Pharma A/S
Ascendis 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 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) Ascendis’ ability to achieve blockbuster status for multiple
products and expand its engine for future innovation, (ii)
Ascendis’ ability to achieve its 2024 corporate milestones and
Vision 2030, (iii) Ascendis’ ability to fulfill Vision 3x3, (iv)
Ascendis’ plan to submit a supplemental Biologics License
Application for TransCon hGH in the second quarter of 2024, (v) the
timing of topline results from the Phase 2 trial in Turner
syndrome, (vi) Ascendis’ expectations with respect to SKYTROFA
revenue for 2023 and 2024, (vii) Ascendis’ plan to launch TransCon
PTH in Germany in January 2024, (viii) TransCon PTH’s PDUFA date of
May 14, 2024, (ix) Ascendis’ plans to launch TransCon PTH in the
U.S. in the third quarter of 2024, if approved, (x) the timing of
topline results from the ApproaCH Trial, (xi) Ascendis’ plan to
submit a New Drug Application for TransCon CNP for children with
achondroplasia, (xii) Ascendis’ plan to submit an Investigational
New Drug application or similar for TransCon CNP in adults with
achondroplasia; (xiii) the timing of topline annualized height
velocity data from the COACH Trial, (xiv) Ascendis’ ability to
expand its global reach through exclusive sales & distribution
agreements with geographic market leaders, (xv) Ascendis’
expectations with regard to the completion of enrollment in
BelieveIT-201, (xvi) Ascendis’ plan to provide a clinical update
from the Phase 2 portion of indication-specific, dose expansion
cohorts in the IL-Believe Trial, (xvii) Ascendis’ expectations
regarding its financial results and performance in 2023 and 2024,
(xviii) Ascendis’ Vision 2030, (xix) Ascendis’ ability to apply its
TransCon technology platform to build a leading, fully integrated
biopharma company, and (xx) 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, SKYTROFA®, and YORVIPATH® are trademarks owned by
the Ascendis Pharma group. © January 2024 Ascendis Pharma A/S.
Investor Contacts: |
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Melinda Baker |
Ascendis Pharma |
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Ascendis Pharma |
+1 (650) 374-6343 |
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+1 (650) 709-8875 |
ir@ascendispharma.com |
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media@ascendispharma.com |
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Patti Bank |
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ICR Westwicke |
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+1 (415) 513-1284 |
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patti.bank@westwicke.com |
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