Ascendis Pharma A/S Reports Full-Year 2019 Financial Results
– On track with 2020 corporate milestones –
– Top-line data from PaTH Forward phase 2 trial on track for mid-April –
– Conference call today at
“Following a transformative 2019, Ascendis remains on track with our corporate milestones for an even stronger 2020,” said
Corporate Highlights & Progress
- TransCon hGH: TransCon hGH is an investigational long-acting prodrug of somatropin (human growth hormone or hGH) that releases somatropin in phase 3 development as a once-weekly treatment for growth hormone deficiency (GHD):
-- Following discussions with the
U.S. Food and Drug Administration(FDA), submitted an Investigational New Drug amendment to initiate the global, phase 3 foresiGHt Trial in adult GHD. The foresiGHt Trial is expected to begin enrollment later this year.
-- Held two pre-BLA meetings with FDA to review the Chemistry, Manufacturing and Controls (CMC), and clinical/non-clinical packages for TransCon hGH as a potential treatment for pediatric GHD. The company is on track for planned submission of a Biologics License Application (BLA) to the FDA in the second quarter of 2020 and a Marketing Authorisation Application (MAA) to the
European Medicines Agencyin the fourth quarter of 2020.
-- Received Orphan Designation from the
European Commission for TransConhGH in pediatric GHD.
-- Advanced TransCon hGH in
Greater Chinafollowing initiation of a phase 3 trial for TransCon hGH in pediatric GHD by VISEN Pharmaceuticals, the company’s strategic investment to establish global reach in Greater China.
- TransCon PTH: TransCon PTH is an investigational long-acting prodrug of parathyroid hormone (PTH) in development as a once-daily replacement therapy for hypoparathyroidism (HP) designed to replace PTH at physiologic levels for 24 hours each day, and address both short-term symptoms and long-term complications of the disease:
-- Completed enrollment of 59 subjects in the PaTH Forward Trial, a global phase 2 trial evaluating the safety, tolerability and efficacy of TransCon PTH in adult HP subjects.
-- The company expanded the trial in
November 2019to expedite the enrollment of subjects affected by the NATPARA® recall. Final enrollment of PaTH Forward included 17 subjects previously treated with NATPARA.
-- The goal of PaTH Forward is to identify a starting dose (15, 18, or 21 µg per day) for a pivotal phase 3 trial, establish a titration regimen for complete withdrawal of standard of care (i.e., active vitamin D and calcium supplements), and evaluate TransCon PTH control of serum and urinary calcium.
-- Following the one-month blinded portion of PaTH Forward, subjects entered an open-label extension where they will receive a customized maintenance dose of TransCon PTH (6 to 30 µg per day) titrated to optimize their calcium control and evaluated on the primary composite endpoint, both as planned for phase 3. Fifty-nine subjects completed the blinded portion, and 58 subjects continued in the open-label extension, with one subject withdrawing for reasons unrelated to safety or efficacy of the study drug.
-- The company expects to report top-line results from the one-month blinded portion of PaTH Forward in mid-April, with six-month data from the open-label extension expected during the third quarter of 2020.
- TransCon CNP: TransCon CNP is an investigational long-acting prodrug of C-type natriuretic peptide (CNP) in development as a therapy for children with achondroplasia (ACH), the most common form of dwarfism, for which there is no FDA-approved treatment. TransCon CNP is designed to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose:
-- The ACcomplisH Trial is a global, phase 2, randomized, double-blind, placebo-controlled trial designed to evaluate the safety and efficacy of TransCon CNP at escalating doses in children (ages 2 to 10 years) with ACH. The company continues to work towards escalating sequential dose cohorts throughout the year, while ensuring the safety of subjects during the current pandemic and access to investigator site staff for future monitoring visits.
VISEN Pharmaceuticalsremains on track to initiate a phase 2 trial in children with ACH during the fourth quarter of 2020.
- Oncology: The company advanced its pipeline of multiple programs in oncology for clinically validated pathways, including TransCon IL-2 b/g, TransCon TLR7/8 Agonist and TransCon VEGF-TKI, with the goal to file an IND or equivalent for the company’s first oncology candidate in the fourth quarter of 2020.
- Corporate milestones remain on track for 2020 despite the current global pandemic. The company continues to monitor and adapt to the impact of COVID-19 and expects to provide further updates to the investment community if the update is warranted.
- Ended 2019 with cash and cash equivalents of €598.1 million.
Full Year 2019 Financial Results
For the full year 2019,
Revenue for 2019 was €13.4 million compared to €10.6 million during 2018. The increase reflects recognition of revenue related to our strategic investment in
Research and development (R&D) costs for 2019 were €191.6 million compared to €140.3 million during 2018. Higher R&D costs in 2019 reflect an increase in personnel and external costs for development and manufacturing of TransCon hGH, TransCon PTH and TransCon CNP, and other research programs, including oncology.
General and administrative expenses for 2019 were €48.5 million compared to €25.1 million during 2018. The increase is primarily due to higher personnel-related costs and other increasing costs of preparing to become a commercial organization.
Conference Call and Webcast information
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|Dial In (International)||574-990-1036|
A live webcast of the conference call will be available on the Investors and News section of the
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For more information, please visit www.ascendispharma.com.
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 our 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) our plans to submit a BLA with the FDA in the second quarter of 2020 and a MAA in
FINANCIAL TABLES FOLLOW
|Consolidated Statements of Profit or Loss and Other Comprehensive Income / (loss)|
|(In EUR'000s, except share and per share data)|
|Research and development costs||(191,621||)||(140,281||)|
|General and administrative expenses||(48,473||)||(25,057||)|
|Operating profit / (loss)||(226,719||)||(154,757||)|
|Share of profit / (loss) of associate||(8,113||)||(321||)|
|Profit / (loss) before tax||(218,250||)||(130,491||)|
|Tax on profit / (loss) for the year||234||394|
|Net profit / (loss) for the year||(218,016||)||(130,097||)|
|Other comprehensive income / (loss)|
|Items that may be reclassified subsequently to profit or loss:|
|Exchange differences on translating foreign operations||(37||)||17|
|Other comprehensive income / (loss) for the year, net of tax||(37||)||17|
|Total comprehensive income / (loss) for the year, net of tax||(218,053||)||(130,080||)|
|Profit / (loss) for the year attributable to owners of the Company||(218,016||)||(130,097||)|
|Total comprehensive income / (loss) for the year attributable to owners of the Company||(218,053||)||(130,080||)|
|Basic and diluted earnings / (loss) per share||(4.69||)||(3.17||)|
|Number of shares used for calculation (basic and diluted)||46,506,862||41,085,237|
|Consolidated Statements of Financial Position|
|Property, plant and equipment||45,069||4,325|
|Investment in associate||15,538||17,083|
|Receivable from associate||804||-|
|Income taxes receivable||1,473||849|
|Cash and cash equivalents||598,106||277,862|
|Equity and liabilities|
|Income taxes payable||119||9|
|Total equity and liabilities||676,732||318,968|
Source: Ascendis Pharma