Ascendis Pharma A/S Reports Third Quarter 2019 Financial Results
– Continued execution of global endocrinology rare disease programs as planned regulatory filings for TransCon™ hGH in 2020 advance on track –
– Expanded PaTH Forward Trial expedites enrollment of subjects previously treated with parathyroid hormone (PTH) –
– Conference call today at
“Our achievements this year reflect our ability to bring a product from idea stage all the way through clinic, as we near the finish line for TransCon hGH and approach our planned regulatory filings in
He continued, “One of our driving values at Ascendis is patient focus. In response to the recent recall of NATPARA® in the U.S., we recently expanded our TransCon PTH Phase 2 PaTH Forward Trial with a protocol addendum to help expedite participation of some of those patients affected by the recall. We believe the expanded trial will provide meaningful clinical data to help patients, both naïve to PTH and previously treated with PTH, and further demonstrate the value of our unique approach to product development.”
Corporate Highlights &
- Completed the last subject visit for the long-term clinical database for TransCon hGH, paving the way for the company’s planned Biologics License Application to the
U.S. Food and Drug Administrationin the first half of 2020 and the Marketing Authorisation Application to the European Medicines Agencyin the second half of 2020. TransCon hGH is a long-acting prodrug of human growth hormone (hGH) in phase 3 development as a once-weekly therapy for pediatric growth hormone deficiency (GHD).
- Completed the manufacturing of drug product Process Performance Qualification (PPQ) batches required to support the planned regulatory filings in 2020 for TransCon hGH. The company is now finalizing the associated analytics and preparing qualification reports.
- Advanced the TransCon hGH program in
Greater Chinawith VISEN Pharmaceuticals, who initiated a phase 3 trial for TransCon hGH in pediatric GHD.
- Received Orphan Designation from the
European Commission for TransConhGH, which is provided to therapies aimed at treating, preventing or diagnosing a disease that is life-threatening or chronically debilitating, affects no more than five in 10,000 persons in the European Unionand which may provide significant additional benefit over existing therapies.
- Announced a protocol addendum in the U.S. designed to expedite enrollment of subjects previously treated with NATPARA in the TransCon PTH PaTH Forward Trial, a global phase 2 trial evaluating the safety, tolerability and efficacy of TransCon PTH in adult subjects with hypoparathyroidism (HP). TransCon PTH is an investigational long-acting prodrug of PTH in development as a potential once-daily replacement therapy for HP, designed to provide physiologic levels of PTH for 24 hours a day, seven days a week. Under the addendum, patients previously treated with NATPARA in the U.S. will now have an expedited pathway to enroll in PaTH Forward. As a result, the company expects to exceed targeted enrollment of 40 subjects, and plans to report top-line data from the expanded trial in first quarter of 2020. The goal of PaTH Forward is to evaluate TransCon PTH control of serum and urinary calcium, and identify a titration regimen for complete withdrawal of standard of care (i.e., active vitamin D and calcium supplements).
- Initiated the ACcomplisH Trial, a global, phase 2, randomized, double-blind, placebo-controlled trial designed to evaluate the safety and efficacy of TransCon CNP, a long-acting prodrug of C-type natriuretic peptide (CNP), at escalating doses in children with achondroplasia (ACH). TransCon CNP is designed to provide continuous exposure to CNP at therapeutic levels with once-weekly dosing.
- Presented preclinical data for TransCon TLR7/8 Agonist, a product candidate in development for oncology, at the
Society of Immunotherapy of Cancerannual meeting. TransCon TLR7/8 Agonist is a prodrug of resiquimod, a small molecule with immune-activating and anti-tumor properties that is transiently conjugated to a hydrogel carrier via a TransCon linker. Administered as an intratumoral injection, TransCon TLR7/8 Agonist delivered sustained local release of resiquimod over weeks directly to the tumor site and demonstrated potent anti-tumor activity as a monotherapy, as well as in combination with interleukin-2 (IL-2).
- Ended the third quarter of 2019 with cash and cash equivalents of €658.7 million.
Third Quarter 2019 Financial Results
For the third quarter,
Revenue for the third quarter was €2.2 million compared to €20 thousand in the same quarter of 2018. The increase relates to our
Research and development (R&D) costs for the third quarter were €46.3 million compared to €31.5 million during the same period in 2018. Higher R&D costs in 2019 reflect an increase in personnel-related costs to support development and manufacturing of TransCon hGH, TransCon PTH and TransCon CNP, increasing clinical trial costs for the PaTH Forward Trial and the ACcomplish Trial, as well as increased costs for other research programs, including oncology.
General and administrative expenses for the third quarter were €10.0 million compared to €6.8 million during the same period in 2018. The increase is primarily due to higher personnel-related costs and other increasing costs of expanding the company and preparing to become a commercial organization.
Conference Call and Webcast information
|Date||Monday, November 18, 2019|
|Time||4:30 p.m. ET|
|Dial In (U.S.)||844-290-3904|
|Dial In (International)||574-990-1036|
A live webcast of the conference call will be available on the Investors and News section of the
Ascendis is headquartered in
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
FINANCIAL TABLES FOLLOW
|Ascendis Pharma A/S|
|Unaudited Condensed Consolidated Interim Statements of Profit or Loss and Other Comprehensive Income / (loss)|
|(In EUR'000s, except share and per share data)|
|Three Months Ended September 30,||Nine Months Ended September 30,|
|Research and development costs||(46,258||)||(31,511||)||(141,343||)||(102,286||)|
|General and administrative expenses||(10,000||)||(6,796||)||(31,396||)||(16,684||)|
|Operating profit / (loss)||(54,015||)||(38,287||)||(161,871||)||(118,904||)|
|Share of profit / (loss) of associate||(1,338||)||-||(5,452||)||-|
|Profit / (loss) before tax||(25,174||)||(34,067||)||(137,850||)||(98,425||)|
|Tax on profit / (loss) for the period||61||100||196||306|
|Net profit / (loss) for the period||(25,113||)||(33,967||)||(137,654||)||(98,119||)|
|Other comprehensive income / (loss)|
|Items that may be reclassified subsequently to profit or loss:|
|Exchange differences on translating foreign operations||37||(9||)||2||(16||)|
|Other comprehensive income / (loss) for the period, net of tax||37||(9||)||2||(16||)|
|Total comprehensive income / (loss) for the period, net of tax||(25,076||)||(33,976||)||(137,652||)||(98,135||)|
|Profit / (loss) for the period attributable to owners of the Company||(25,113||)||(33,967||)||(137,654||)||(98,119||)|
|Total comprehensive income / (loss) for the period attributable to owners of the Company||(25,076||)||(33,976||)||(137,652||)||(98,135||)|
|Basic and diluted earnings / (loss) per share||(0.53||)||(0.81||)||(2.99||)||(2.41||)|
|Number of shares used for calculation (basic and diluted)||47,590,837||41,888,908||46,066,493||40,757,686|
|Ascendis Pharma A/S|
|Unaudited Condensed Consolidated Interim Statements of Financial Position|
|September 30,||December 31,|
|Property, plant and equipment||43,272||4,325|
|Investment in associate||17,073||17,083|
|Income taxes receivable||1,298||849|
|Cash and cash equivalents||658,660||277,862|
|Equity and liabilities|
|Income taxes payable||24||9|
|Total equity and liabilities||734,959||318,968|
Scott T. SmithChief Financial Officer (650) 352-8389 firstname.lastname@example.org Media contact: Ami KnoeflerHead of Global Communications(650) 739-9952 email@example.com Investor contact: Patti Bank Westwicke Partners(415) 513-1284 firstname.lastname@example.org
Source: Ascendis Pharma