Ascendis Pharma Introduces Vision 2030
- Strategic roadmap to achieve blockbuster status for multiple products and expand
the Company’s engine for future innovation
“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
Selected Key Updates
- TransCon™ hGH
(lonapegsomatropin, approved as SKYTROFA® in theU.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 inJanuary 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.
- First launch planned in
- 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.
- During the fourth quarter of 2023, entered into an exclusive license agreement with Teijin Limited for TransCon hGH, TransCon PTH, and TransCon CNP in
- 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.
- Preliminary unaudited
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
- Achieve blockbuster status (>
- 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
A live webcast of the event will be available via the Investors & News section of the
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
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
Ascendis,
and YORVIPATH® are trademarks owned by the
Investor Contacts: | Media Contact: | |
+1 (650) 374-6343 | +1 (650) 709-8875 | |
ir@ascendispharma.com | media@ascendispharma.com | |
ICR Westwicke | ||
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Source: Ascendis Pharma