Ascendis Pharma Reports Third Quarter 2023 Financial Results
European Commissiondecision for TransCon™ PTH expected this month; if approved, first European Unionlaunch planned in Germanyin January 2024
- TransCon PTH NDA resubmission to FDA expected before mid-November
- Completed enrollment in the Phase 3 ApproaCH Trial; initiated TransCon CNP infant trial in the third quarter 2023; expect to initiate combination trial of TransCon CNP and TransCon hGH in the fourth quarter 2023
- SKYTROFA Q3 revenue of €47.0 million, increasing full year 2023 SKYTROFA revenue expectations to €170 – €175 million
- Conference call today at
4:30 pm ET
“This quarter marks an important milestone for Ascendis on our path to become a leading, sustainable biopharma company, where we began our journey to extend our SKYTROFA U.S. market value leadership globally with our first EU product launch in Germany,” said
- TransCon hGH (marketed in the
U.S.and EU as SKYTROFA):
- Third quarter 2023 SKYTROFA revenue totaled €47.0 million, a 31% sequential increase. Increased full year 2023 SKYTROFA revenue expectations from €165 – €170 million to €170 – €175 million.
|SKYTROFA revenue (millions)||€12.3||€17.1||€31.6||€35.9||€47.0|
- Announced results from enliGHten, the Company’s open-label extension trial evaluating the long-term safety and efficacy of TransCon hGH for children and adolescents with growth hormone deficiency (GHD), demonstrating the long-term safety and efficacy of TransCon hGH in patients treated up to six years, with the majority of children meeting or exceeding average parental height SDS at time of treatment completion or last visit.
- Topline results from Phase 3 foresiGHt Trial in adult growth hormone deficiency expected in the fourth quarter of 2023, potentially opening a new label expansion opportunity.
- TransCon PTH:
September 14, 2023, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the approval of TransCon PTH (palopegteriparatide) as a parathyroid hormone replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism. Final European Commissiondecision is expected this month. If approved, the first launch is planned in Germany, leveraging the Company’s existing SKYTROFA commercial infrastructure, in January 2024.
- In the
U.S., expect to resubmit NDA for TransCon PTH for adults with hypoparathyroidism to the FDA before mid-November.
- Presented 52-week data from Phase 3 PaTHway Trial demonstrating that skeletal dynamics of patients with chronic hypoparathyroidism trended toward a new steady state closer to age-appropriate norms with continued use of TransCon PTH. Results confirm trends previously reported in the Phase 2 PaTH Forward Trial.
- As of
September 30, 2023, 145 out of 154 participants continue in the open-label extension (OLE) portions of the Phase 2 PaTH Forward, Phase 3 PaTHway, and PaTHway Japan trials.
- TransCon CNP:
- Completed enrollment in ApproaCH, a Phase 3, global randomized, double-blind, placebo-controlled trial in children ages 2–11 years with achondroplasia. Topline results are expected in the second half of 2024.
- Filed an Investigational New Drug (IND) amendment with the FDA to initiate reACHin, a Phase 2, multicenter, double-blind, randomized, placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg/kg of TransCon CNP once-weekly for 52 weeks in infants aged 0 to < 2 years with achondroplasia.
- One-year follow-up data from AComplisH OLE expected in the fourth quarter of 2023.
- During the fourth quarter of 2023, the Company expects to file an IND amendment or similar for COACH, a combination trial evaluating TransCon CNP and TransCon hGH in children with achondroplasia. The Company believes that this combination therapy may provide greater annualized height velocity than CNP alone, and at the same time, address the comorbidities of achondroplasia.
- TransCon IL-2 β/γ:
- Reported new data from ongoing Phase 1/2 IL-Believe Trial demonstrating clinical activity of TransCon IL-2 β/γ as monotherapy or in combination with a checkpoint inhibitor. Of three small-cell lung cancer patients treated in the combination portion of the trial who had previously progressed on checkpoint inhibitors, a partial response (confirmed) and a complete response (unconfirmed, treatment ongoing) were observed to date.
- Enrollment continues in the Phase 2 portion in indication-specific cohorts; first patient dosed with TransCon IL-2 β/γ and TransCon TLR7/8 Agonist in combination. Initial data from indication-specific cohorts expected in the second half of 2024.
- TransCon TLR7/8 Agonist:
- Enrollment continues in Phase 2 portion of transcendIT-101, a Phase 1/2 trial to evaluate TransCon TLR7/8 Agonist as monotherapy or in combination with pembrolizumab in dose escalation and dose expansion. Initial data expected in the second half of 2024.
- Ended the third quarter of 2023 with cash, cash equivalents, and marketable securities totaling €455.4 million.
Third Quarter 2023 Financial Results
Total revenue for the third quarter of 2023 was €48.0 million compared to €15.3 million during the same period in 2022. The increase was primarily attributable to higher SKYTROFA revenue of €47.0 million compared to €12.3 million in the same period last year.
Research and development (R&D) costs for the third quarter were €111.4 million compared to €97.4 million during the same period in 2022. This increase was primarily due to higher development costs for the Oncology and Ophthalmology programs, increasing clinical trial activities for TransCon CNP, and higher employee-related costs, and was partly offset by lower development costs for TransCon hGH.
Selling, general, and administrative (SG&A) expenses for the third quarter were €63.6 million compared to €60.7 million during the same period in 2022. This increase was primarily due to higher employee related expenses and other expenses attributable to organizational growth.
Net finance expenses were €20.4 million in the third quarter compared to €20.9 million in the same period in 2022.
For the third quarter of 2023,
Conference Call and Webcast Information
To participate in the call, please dial (800) 445-7795 (domestic) or +1 (785) 424-1699 (international), and reference passcode ASNDQ323. The link to the live webcast will also be available on the Investors & News section of the
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 timing of the
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FINANCIAL TABLES FOLLOW
|Consolidated Statements of Profit or Loss and Comprehensive Income / (Loss)|
|(In EUR'000s, except share and per share data)|
|Three Months ended
||Nine Months ended
|Cost of sales||7,388||1,693||24,938||7,025|
|Research and development costs||111,439||97,431||322,573||271,006|
|Selling, general and administrative expenses||63,614||60,671||200,435||164,675|
|Operating profit / (loss)||(134,407||)||(144,505||)||(418,930||)||(414,428||)|
|Share of profit / (loss) of associate||(6,794||)||(3,696||)||(15,471||)||(9,736||)|
|Profit / (loss) before tax||(161,578||)||(169,122||)||(393,056||)||(375,748||)|
|Income taxes (expenses)||(645||)||167||(1,513||)||(28||)|
|Net profit / (loss) for the period||(162,223||)||(168,955||)||(394,569||)||(375,776||)|
|Attributable to owners of the Company||(162,223||)||(168,955||)||(394,569||)||(375,776||)|
|Basic and diluted earnings / (loss) per share||€ (2.88)||€ (3.03)||€ (7.02)||€ (6.70)|
|Number of shares used for calculation (basic and diluted)||56,272,698||55,831,561||56,194,956||56,115,782|
|Net profit / (loss) for the period||(162,223||)||(168,955||)||(394,569||)||(375,776||)|
|Other comprehensive income / (loss)|
|Items that may be reclassified subsequently to profit or loss:|
|Exchange differences on translating foreign operations||571||(2,207||)||(1,232||)||(2,538||)|
|Other comprehensive income / (loss) for the period, net of tax||571||(2,207||)||(1,232||)||(2,538||)|
|Total comprehensive income / (loss) for the period, net of tax||(161,652||)||(171,162||)||(395,801||)||(378,314||)|
|Attributable to owners of the Company||(161,652||)||(171,162||)||(395,801||)||(378,314||)|
|Consolidated Statements of Financial Position|
|Property, plant and equipment||125,535||129,095|
|Investment in associate||8,116||22,932|
|Income tax receivables||1,644||883|
|Cash and cash equivalents||441,268||444,767|
|Equity and liabilities|
|Trade payables and accrued expenses||121,552||101,032|
|Income tax payables||6,478||5,490|
|Total equity and liabilities||873,213||1,089,738|
Source: Ascendis Pharma