Ascendis Pharma A/S Reports Second Quarter 2020 Financial Results and Announces New Data from the Four-Week, Fixed Dose, Double-Blind Portion of the PaTH Forward Trial
– Approval of Paediatric Investigation Plan by
– New data from the four-week, fixed dose, double-blind portion of PaTH Forward
demonstrated statistically significant and clinically meaningful improvements
in SF-36 functional health and well being outcomes –
– Conference call today at
“We are executing across the globe on all elements of our Vision 3x3, including preparation for the expected
Corporate Highlights & Progress
- Submitted a BLA for TransCon hGH for pediatric growth hormone deficiency to the FDA on
June 25, 2020.
- In July, received approval of its proposed Paediatric Investigation Plan covering ages 6 months to less than 18 years of age from EMA for TransCon hGH and remain on track for a planned third quarter 2020 MAA submission to the EMA for pediatric growth hormone deficiency.
- Today announced new data on exploratory endpoints from the four-week fixed dose, blinded portion of PaTH Forward, a global phase 2 trial evaluating the safety, tolerability and efficacy of TransCon PTH, an investigational long-acting prodrug of parathyroid hormone (PTH), in adult subjects with hypoparathyroidism. Data on TransCon PTH from the validated SF-36 quality-of-life instrument showed a statistically significant improvement related to both the physical (LS mean difference=5.2; p=0.013) and mental (LS mean difference=9.8; p=0.0003) components of the measure compared to placebo.1 Importantly, the results were consistent with a clinically meaningful improvement in functional health and well being for subjects receiving TransCon PTH as compared with placebo after four weeks.2 Detailed results are included in the company’s investor presentation which can be found on the company’s website at www.ascendispharma.com. The company plans to present additional data and analyses from the PaTH Forward trial four-week results at upcoming medical conferences, anticipates announcing the six-month data from the open-label extension portion of the PaTH Forward Trial during the third quarter and is preparing to submit regulatory filings to initiate a phase 3 trial in the fourth quarter.
- In July, received orphan designation by the
European Commission(EC) for TransCon C-Type Natriuretic Peptide (CNP), an investigational long-acting prodrug of CNP in development as a therapy for achondroplasia, the most common form of dwarfism.3
- Presented preclinical data for TransCon IL-2 β/γ, an oncology product candidate designed to provide sustained systemic release of a receptor-biased IL-2 (IL-2 β/γ), at the
American Association of Cancer Research (AACR) VirtualAnnual Meeting II. Results showed that TransCon IL-2 β/γ demonstrated a long in vivo half-life of approximately 32 hours, expected to support potential dosing of every three weeks in patients.4
- After the end of the second quarter, the company announced the completion of its underwritten public offering of 4,859,154 American Depositary Shares (“ADSs”). Net proceeds from this offering in
July 2020were approximately $654.7 million, or approximately €580.7 million based on exchange rates on the date of the closing.
- Ended the second quarter 2020 with cash, cash equivalents and marketable securities totaling €471.6 million, excluding the net proceeds from the
July 2020equity offering.
Second Quarter 2020 Financial Results
For the second quarter,
Revenue for the second quarter was €1.4 million compared to €3.2 million in the same quarter of 2019. The decrease was due to a lower amount of license and service revenue being recognized from the company’s strategic investment in
Research and development (R&D) costs for the second quarter were €63.6 million compared to €43.8 million during the same period in 2019. Higher R&D costs in 2020 reflect an increase in personnel-related costs, overhead costs allocated to R&D, and the continued progress in development of the company’s product candidates.
Selling, general and administrative (SG&A) expenses for the second quarter were €20.8 million compared to €11.0 million during the same period in 2019. The increase is primarily due to additional personnel-related costs, higher IT and other site costs, and continued build out of the company’s commercial capabilities.
Conference Call and Webcast information
|Dial In (
|Dial In (International)||574-990-1036|
A live webcast of the conference call will be available on the Investors and News section of the
About Ascendis Pharma’s Pipeline
- TransCon hGH (lonapegsomatropin), an investigational long-acting prodrug of somatropin (human growth hormone or hGH) that releases somatropin with the identical amino acid sequence and size as daily growth hormone, in phase 3 development as a once-weekly treatment for growth hormone deficiency (GHD).
- TransCon PTH, an investigational long-acting prodrug of parathyroid hormone (PTH) in phase 2 development as a once-daily replacement therapy for hypoparathyroidism (HP) designed to replace PTH at physiologic levels for 24 hours every day, and address both short-term symptoms and long-term complications of the disease.
- TransCon CNP, an investigational long-acting prodrug of C-type natriuretic peptide (CNP) in phase 2 development as a therapy for children with achondroplasia (ACH), the most common form of dwarfism3, for which there is no FDA-approved treatment5. TransCon CNP is designed to provide continuous exposure of CNP at safe, therapeutic levels via a single, weekly subcutaneous dose.
Additionally, the company has established oncology as its second therapeutic area of focus and plans to submit an IND or similar in the fourth quarter of 2020 for TransCon TLR7/8 Agonist, the company’s first oncology product candidate.
About Ascendis Pharma A/S
Ascendis Pharma is applying its innovative platform technology to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, the company utilizes its TransCon™ technologies to create new and potentially best-in-class therapies.
Ascendis Pharma currently has a pipeline of three independent endocrinology rare disease product candidates in clinical development and is advancing oncology as its second therapeutic area of focus. The company continues to expand into additional therapeutic areas to address unmet patient needs.
Ascendis is headquartered in Copenhagen,
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) Ascendis’ plans to submit its marketing application for TransCon hGH in in
Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo and TransCon are registered trademarks owned by the Ascendis Pharma Group. © August 2020 Ascendis Pharma A/S.
FINANCIAL TABLES FOLLOW
|Consolidated Statements of Profit or Loss and Other Comprehensive Income / (loss)|
|(In EUR'000s, except share and per share data)|
|Three Months Ended
||Six Months Ended
|Research and development costs||(63,578||)||(43,826||)||(121,093||)||(95,085||)|
|Selling, general and administrative expenses||(20,805||)||(10,960||)||(38,720||)||(21,396||)|
|Operating profit / (loss)||(82,947||)||(51,575||)||(156,152||)||(107,856||)|
|Share of profit / (loss) of associate||(1,885||)||(2,262||)||(3,400||)||(4,114||)|
|Profit / (loss) before tax||(95,038||)||(58,969||)||(158,432||)||(112,676||)|
|Tax on profit / (loss) for the year||106||65||183||135|
|Net profit / (loss) for the year||(94,932||)||(58,904||)||(158,249||)||(112,541||)|
|Attributable to owners of the Company||(94,932||)||(58,904||)||(158,249||)||(112,541||)|
|Basic and diluted earnings / (loss) per share||€||(1.97||)||€||(1.25||)||€||(3.29||)||€||(2.48||)|
|Number of shares used for calculation (basic and diluted)||48,207,661||47,190,717||48,096,749||45,291,688|
|Net profit / (loss) for the year||(94,932||)||(58,904||)||(158,249||)||(112,541||)|
|Other comprehensive income / (loss)|
|Items that may be reclassified subsequently to profit or loss:|
|Exchange differences on translating foreign operations||(147||)||(594||)||(61||)||(35||)|
|Other comprehensive income / (loss) for the year, net of tax||(147||)||(594||)||(61||)||(35||)|
|Total comprehensive income / (loss) for the year, net of tax||(95,079||)||(59,498||)||(158,310||)||(112,576||)|
|Attributable to owners of the Company||(95,079||)||(59,498||)||(158,310||)||(112,576||)|
|Consolidated Statements of Financial Position|
|Property, plant and equipment||49,743||45,069|
|Investment in associate||14,167||15,538|
|Receivable from associate||588||804|
|Income taxes receivable||1,107||1,473|
|Cash and cash equivalents||240,605||598,106|
|Equity and liabilities|
|Income taxes payable||186||119|
|Total equity and liabilities||560,607||676,732|
1 Data on file.
2 Maruish, M. E. (Ed.). User’s manual for the
3 Horton WA, et al.
5 Pauli RM. J Rare Dis. 2019; 14:1
Source: Ascendis Pharma