ASCENDIS PHARMA A/S - Phase 3 PaTHway Trial top-line results demonstrated potential of TransCon™ PTH to become the first parathyroid replacement therapy for adults with hypoparathyroidism
- TransCon PTH
U.S. FDA regulatory submission on track for Q3 andEU MAA for Q4 2022 - Increasing uptake of SKYTROFA® (lonapegsomatropin-tcgd), with more than 1,200 unique patient prescriptions processed as of
April 29, 2022 - Completed
U.S. $575 million convertible notes offering strengthening balance sheet to build a sustainable leading global biopharma company - Conference call today at
4:30 pm ET
“This continues to be a transformative time for Ascendis as we build on positive Phase 3 data for our first two endocrinology rare disease programs – TransCon hGH and TransCon PTH - and our first product approval and launch,” said
Company Highlights & Progress
- TransCon hGH:
- TransCon hGH is commercially available in the
U.S. under the brand name SKYTROFA (lonapegsomatropin-tcgd) as the only FDA-approved once-weekly treatment for 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. As ofApril 29, 2022 , 1,231 unique patient prescriptions for SKYTROFA have been written by 404 prescribers and processed through Ascendis’ patient hub called the Ascendis Signature Access Program. Nearly 50% of the prescribers have written prescriptions for more than one patient. - Coverage for SKYTROFA continues to increase, with approximately 45% of
U.S. lives covered at the end of April per MMIT, compared to approximately 36% at the end of February. - In
January 2022 , the Company received marketing authorization for Lonapegsomatropin Ascendis Pharma (developed under the brand name of TransCon hGH) in theEuropean Union as a once-weekly subcutaneous injection for the treatment of children and adolescents ages 3 to 18 years with growth failure due to insufficient secretion of endogenous growth hormone. - On track for FDA protocol submission during the second quarter of 2022 to evaluate TransCon hGH for Turner Syndrome.
- The ongoing conflict in the region surrounding
Ukraine ,Russia , andBelarus has impacted our ability to conduct clinical trials or to treat and evaluate patients there. As a result, we have shifted focus for our foresiGHt Trial in adult growth hormone deficiency to other countries and are now targeting completion of enrollment during the fourth quarter of 2022.
- TransCon hGH is commercially available in the
- TransCon PTH:
- On
March 13, 2022 , we announced top-line data from a randomized, double-blind, placebo-controlled Phase 3 Trial (PaTHway) of TransCon PTH in adults with hypoparathyroidism (HP), demonstrating statistically significant improvement with TransCon PTH compared to control for the primary composite endpoint and all key secondary endpoints. As ofMay 1, 2022 , all 79 patients continued in the open-label extension portion of the PaTHway Trial. - On track for planned
U.S. NDA submission during the third quarter of 2022 and expectedEU MAA submission during the fourth quarter of 2022. - After more than two years of treatment in the open-label extension portion of the PaTH Forward Trial, as of
May 1, 2022 , 57 out of 59 original subjects continue in the trial. - In
April 2022 , enrollment completed in the Phase 3 PaTHway Japan Trial. Top-line results expected later this year.
- On
- TransCon CNP:
- Top-line data from the ACcomplisH Trial, a Phase 2 randomized, double-blind, placebo-controlled clinical trial in
North America ,Europe , andOceania in children ages 2-10 years with achondroplasia are expected in the fourth quarter of 2022.
- Top-line data from the ACcomplisH Trial, a Phase 2 randomized, double-blind, placebo-controlled clinical trial in
- TransCon TLR7/8 Agonist:
- Enrollment continues in transcendIT-101, a Phase 1/2 study of TransCon TLR7/8 Agonist with or without pembrolizumab in patients with advanced or metastatic solid tumors. We expect top-line monotherapy and combo-therapy dose escalation data during the third quarter of 2022.
- TransCon IL-2 β/γ
- The Phase 1/2 IL-βelieγe Trial evaluating TransCon IL-2 β/γ monotherapy in patients with locally advanced or metastatic solid tumors continues to enroll patients. Top-line data are expected in the fourth quarter of 2022.
- On track to dose the first patient in checkpoint combination dose-escalation arm of the IL-βelieγe Trial in the second quarter of 2022.
- TransCon TLR7/8 Agonist and TransCon IL-2 β/γ Combination:
- We plan to submit an IND or similar for Phase 2 cohort expansion for TransCon TLR7/8 Agonist and TransCon IL-2 β/γ during the fourth quarter of 2022.
- Ended the first quarter of 2022 with cash, cash equivalents, and marketable securities totaling €1,065 million.
First Quarter 2022 Financial Results
Total revenue for the first quarter was €6.8 million, an increase of €6.1 million compared to €0.7 million in the same quarter of 2021. Revenues for the first quarter include
Research and development (R&D) costs for the first quarter were €83.2 million compared to €88.1 million during the same period in 2021. Following FDA approval of SKYTROFA (lonapegsomatropin-tcgd) in
Selling, general, and administrative (SG&A) expenses for the first quarter were €47.4 million compared to €37.2 million during the same period in 2021. Higher SG&A expenses were primarily due to an increase in commercial and administrative personnel costs as well as an increase in commercial costs.
Net loss of associate was €4.9 million in the first quarter, compared to a net profit of €28.1 million during the same period in 2021.
Net finance income was €7.6 million in the first quarter compared to €33.6 million in the same period in 2021.
For the first quarter of 2022,
As of
Conference Call and Webcast Information
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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) the timing of top-line results from the PaTHway Japan Trial, the ACcomplisH Trial, the transcendIT-101 Trial and the Phase 1/2 IL-eliee Trial, (ii) the timing of patient dosing in the combination dose-escalation arm of the IL-eliee Trial, (iii) Ascendis’ expectations regarding the strength of 2022 and its ability to deliver on components of Vision 3x3, (iv) whether Ascendis is able to achieve sustainable growth and long-term profitability, (v) whether Ascendis has the elements of success in place, including proven technology and algorithm for innovation, the right capabilities, and a strong balance sheet, (vi) Ascendis’ expectations regarding the timing of its regulatory submissions, applications, protocols, clinical trials and the results thereof, (vii) Ascendis’ expectations regarding the regarding the potential for TransCon PTH to become the first parathyroid replacement therapy for adults with hypoparathyroidism, (viii) Ascendis’ expectations regarding the ability of its immuno-oncology programs to transform care for cancer patients, (ix) Ascendis’ expectations regarding its ability to apply its technology platform and algorithm for product innovation to develop highly differentiated product candidates for unmet medical needs, (x) Ascendis’ ability to apply its platform technology to build a leading, fully integrated biopharma company, and (xi) 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, the SKYTROFA® Auto-Injector and other study drug for commercial sales in the
Ascendis,
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 | |||||
| 2022 | 2021 | ||||
| Revenue | 6,828 | 746 | |||
| Cost of sales | 4,246 | - | |||
| Gross profit / (loss) | 2,582 | 746 | |||
| Research and development costs | 83,193 | 88,149 | |||
| Selling, general and administrative expenses | 47,418 | 37,247 | |||
| Operating profit / (loss) | (128,029 | ) | (124,650 | ) | |
| Share of profit / (loss) of associate | (4,873 | ) | 28,106 | ||
| Finance income | 13,044 | 34,430 | |||
| Finance expenses | 5,399 | 869 | |||
| Profit / (loss) before tax | (125,257 | ) | (62,983 | ) | |
| Tax on profit / (loss) for the period | (241 | ) | 191 | ||
| Net profit / (loss) for the period | (125,498 | ) | (62,792 | ) | |
| Attributable to owners of the Company | (125,498 | ) | (62,792 | ) | |
| Basic and diluted earnings / (loss) per share | € (2.21) | € (1.17) | |||
| Weighted average number of shares used for calculation (basic and diluted) | 56,720,063 | 53,759,952 | |||
| Net profit / (loss) for the period | (125,498 | ) | (62,792 | ) | |
| Other comprehensive income / (loss) | |||||
| Items that may be reclassified subsequently to profit or loss: | |||||
| Exchange differences on translating foreign operations | 425 | 1,842 | |||
| Other comprehensive income / (loss) for the period, net of tax | 425 | 1,842 | |||
| Total comprehensive income / (loss) for the period, net of tax | (125,073 | ) | (60,950 | ) | |
| Attributable to owners of the Company | (125,073 | ) | (60,950 | ) | |
| Consolidated Statements of Financial Position | ||||
| (In EUR'000s) | ||||
| 2022 | 2021 | |||
| Assets | ||||
| Non-current assets | ||||
| Intangible assets | 5,161 | 5,272 | ||
| Property, plant and equipment | 127,678 | 126,049 | ||
| Investment in associate | 35,023 | 38,345 | ||
| Other receivables | 1,822 | 1,808 | ||
| Marketable securities | 86,487 | 107,561 | ||
| 256,171 | 279,035 | |||
| Current assets | ||||
| Inventories | 92,436 | 75,405 | ||
| Trade receivables | 5,808 | 2,200 | ||
| Income tax receivable | 1,072 | 893 | ||
| Other receivables | 15,071 | 20,093 | ||
| Prepayments | 27,994 | 25,231 | ||
| Marketable securities | 223,055 | 235,797 | ||
| Cash and cash equivalents | 755,643 | 446,267 | ||
| 1,121,079 | 805,886 | |||
| Total assets | 1,377,250 | 1,084,921 | ||
| Equity and liabilities | ||||
| Equity | ||||
| Share capital | 7,649 | 7,646 | ||
| Distributable equity | 665,167 | 875,989 | ||
| Total equity | 672,816 | 883,635 | ||
| Non-current liabilities | ||||
| Borrowings | 464,736 | 97,966 | ||
| Derivative liabilities | 141,379 | - | ||
| Contract liabilities | 2,964 | 2,964 | ||
| 609,079 | 100,930 | |||
| Current liabilities | ||||
| Borrowings | 8,926 | 6,995 | ||
| Contract liabilities | 265 | 2,601 | ||
| Trade payables and accrued expenses | 70,683 | 59,417 | ||
| Other liabilities | 12,536 | 29,952 | ||
| Income taxes payable | 499 | 198 | ||
| Provisions | 2,446 | 1,193 | ||
| 95,355 | 100,356 | |||
| Total liabilities | 704,434 | 201,286 | ||
| Total equity and liabilities | 1,377,250 | 1,084,921 |
| Investor Contacts: | Media Contact: |
| +1 (650) 374-6343 | +1 (650) 709-8875 |
| tle@ascendispharma.com | media@ascendispharma.com |
| ICR Westwicke | |
| +1 (415) 513-1284 | |
| patti.bank@westwicke.com | |
| ir@ascendispharma.com |
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