Ascendis Pharma A/S Reports First Quarter 2019 Financial Results
– Continued execution of global endocrinology rare disease programs, following validation of TransCon™ platform in phase 3 heiGHt Trial –
– R&D Day on
– Conference call today at
“In my view, 2019 has been the most transformative period in Ascendis history as we validated our TransCon platform with clinical evidence from the phase 3 heiGHt and fliGHt Trials for TransCon Growth Hormone, while continuing to execute on all three of our global endocrinology rare disease programs,” said
Corporate Highlights &
- Reported preliminary results from the phase 3, open label, single arm, fliGHt Trial of TransCon Growth Hormone (hGH), a long-acting growth hormone therapy in development as a once-weekly treatment for pediatric growth hormone deficiency (GHD). Data from this 26-week trial showed TransCon hGH was safe and well-tolerated in pediatric subjects previously treated with commercially-available daily growth hormone therapy. These data also include new information demonstrating safety, efficacy and tolerability in treatment-naïve subjects under three years of age. Detailed results from the fliGHt Trial will be presented at the
June 26R&D Day. Data from the heiGHt and fliGHt Trials, and long-term safety data from the ongoing enliGHten (long-term extension) Trial, will form the safety database that supports submission of a Biologics License Application (BLA) with the U.S. Food and Drug Administration( FDA) for TransCon hGH to treat pediatric GHD in the first half of 2020.
- Presented additional top-line analyses from the pivotal, phase 3 heiGHt Trial for TransCon hGH, which were consistent with the previously reported top-line results and demonstrated that TransCon hGH had comparable safety and tolerability to a daily hGH (Genotropin®), with a significantly greater annualized height velocity over the one-year study period. The additional data were presented at the
Pediatric Endocrinology Nursing Society(PENS) and Pediatric Endocrine Society(PES) annual meetings.
- Continued initiating sites to expand reach in the global PaTH Forward phase 2 clinical trial of TransCon PTH, a long-acting parathyroid hormone therapy in development for the treatment of adult hypoparathyroidism, providing physiologic levels of PTH for 24 hours a day. In addition, presented three posters relating to TransCon PTH and hypoparathyroidism: one poster highlighted the short-term symptoms and burden of hypoparathyroidism at
ENDO2019; a second poster presented design of the PaTH Forward trial at the European Calcified Tissue Society(ECTS) meeting; and a third poster presented new data on the economic burden of symptoms of hypoparathyroidism at the International Society for Pharmacoeconomics and Outcomes Research(ISPOR) meeting.
- Completed analysis of data from the phase 1 trial of TransCon CNP, a long-acting prodrug of C-type natriuretic peptide (CNP) in development as a once-weekly therapeutic option for achondroplasia, in healthy subjects following preliminary data reported in
November 2018. The results showed that TransCon CNP provided continuous exposure to CNP with a pharmacokinetic profile designed to provide therapeutic levels of CNP with once-weekly dosing. TransCon CNP was generally well tolerated at doses up to 150 µg/kg, with no serious adverse events reported, and the resting blood pressure and heart rate were unchanged from predose at all time points, in all cohorts.
- Received Orphan Drug Designation (ODD) from the U.S.
FDAfor TransCon CNP in achondroplasia. The company is preparing to initiate a phase 2 trial of TransCon CNP in children with the condition in the third quarter of this year.
- Continued ongoing discussions with regulatory agencies to establish global reach for all three of the company’s endocrinology rare disease clinical programs and advance the global clinical development of TransCon hGH, TransCon PTH and TransCon CNP.
- Ended the first quarter of 2019 with cash and cash equivalents of €696.7 million.
First Quarter 2019 Financial Results
For the first quarter,
Revenue for the first quarter was €5.4 million compared to €28 thousand in the same quarter of 2018. The increase reflects recognition of revenue from the sale of licenses in connection with the formation of the strategic investment in
Research and development (R&D) costs for the first quarter were €51.3 million compared to €30.5 million during the same period in 2018. Higher R&D costs in 2019 reflect an increase in costs for the manufacturing of validation batches of TransCon hGH required as part of the regulatory approval process, partly offset by decreasing costs for the phase 3 clinical program; for TransCon PTH, an increase in costs associated with continued development and progress, including manufacturing of clinical material and pen device, and initiation of the phase 2 PaTH Forward clinical trial; for TransCon CNP, lower manufacturing and preclinical costs, partly offset by phase 2 enabling activities; and increased headcount in R&D functions.
General and administrative expenses for the first quarter were €10.4 million compared to €4.7 million during the same period in 2018. The increase is primarily due to higher personnel costs and other increasing costs of preparing to become a commercial organization.
Realizing Vision 3x3: R&D Day on
Conference Call and Webcast information
|Date||Thursday, May 30, 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 March 31,|
|Research and development costs||(51,259||)||(30,540||)|
|General and administrative expenses||(10,436||)||(4,662||)|
|Operating profit / (loss)||(56,281||)||(35,174||)|
|Share of profit / (loss) of associate||(1,852||)||-|
|Profit / (loss) before tax||(53,707||)||(41,482||)|
|Tax on profit / (loss) for the period||70||107|
|Net profit / (loss) for the period||(53,637||)||(41,375||)|
|Other comprehensive income / (loss)|
|Items that may be reclassified subsequently to profit or loss:|
|Exchange differences on translating foreign operations||559||(9||)|
|Other comprehensive income / (loss) for the period, net of tax||559||(9||)|
|Total comprehensive income / (loss) for the period, net of tax||(53,078||)||(41,384||)|
|Profit / (loss) for the period attributable to owners of the Company||(53,637||)||(41,375||)|
|Total comprehensive income / (loss) for the period attributable to owners of the Company||(53,078||)||(41,384||)|
|Basic and diluted earnings / (loss) per share||(1.24||)||(1.07||)|
|Number of shares used for calculation (basic and diluted)||43,371,559||38,699,204|
|Ascendis Pharma A/S|
|Unaudited Condensed Consolidated Interim Statements of Financial Position|
|March 31,||December 31,|
|Property, plant and equipment||24,032||4,325|
|Investment in associate||17,476||17,083|
|Income taxes receivable||962||849|
|Cash and cash equivalents||696,664||277,862|
|Equity and liabilities|
|Income taxes payable||39||9|
|Total equity and liabilities||761,939||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 BankWestwicke Partners (415) 513-1284 firstname.lastname@example.org
Source: Ascendis Pharma A/S