On November 18, 2019 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to address unmet medical needs, reported financial results for the quarter ended September 30, 2019 (Press release, Ascendis Pharma, NOV 18, 2019, View Source [SID1234551454]).
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"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 the United States (U.S.) and Europe," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "Our recent progress in the TransCon hGH program, including both in Europe and China, also support our strategy to establish global reach. The power of the TransCon technology is enabling us to build a leading fully integrated patient-focused biopharma company, and advance towards our ‘Vision 3×3’ goals."
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 & Progress
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 Administration in the first half of 2020 and the Marketing Authorisation Application to the European Medicines Agency in 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 China with VISEN Pharmaceuticals, who initiated a phase 3 trial for TransCon hGH in pediatric GHD.
Received Orphan Designation from the European Commission for TransCon hGH, 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 Union and 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 Cancer (SITC) (Free SITC Whitepaper) annual meeting. TransCon TLR7/8 Agonist is 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, Ascendis Pharma reported a net loss of €25.1 million, or €0.53 per share (basic and diluted) compared to a net loss of €34.0 million, or €0.81 per share (basic and diluted) for the same period in 2018.
Revenue for the third quarter was €2.2 million compared to €20 thousand in the same quarter of 2018. The increase relates to our November 2018 strategic investment in VISEN Pharmaceuticals.
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.
As of September 30, 2019, Ascendis Pharma had cash and cash equivalents of €658.7 million compared to €690.4 million as of June 30, 2019. As of September 30, 2019, Ascendis Pharma had 47,739,647 ordinary shares outstanding.
Conference Call and Webcast information
Ascendis Pharma will host a conference call and webcast today at 4:30 p.m. ET to discuss its third quarter 2019 financial results. Details include:
Date Monday, November 18, 2019
Time 4:30 p.m. ET
Dial In (U.S.) 844-290-3904
Dial In (International) 574-990-1036
Access Code 5897398
A live webcast of the conference call will be available on the Investors and News section of the Ascendis Pharma website at www.ascendispharma.com. A webcast replay will also be available on this website shortly after conclusion of the event for 30 days.