On May 19, 2020 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 March 31, 2020 (Press release, Ascendis Pharma, MAY 19, 2020, View Source [SID1234558277]).
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"We continue to execute on our vision to build a leading biopharma company. In addition to remaining on track to achieve our corporate milestones for 2020, we continue to expand our organization to support the planned global launch of our first commercial product, TransCon Growth Hormone," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. "We are preparing to submit our marketing application in the United States in the second quarter and have advanced the European submission now to be in the third quarter."
He continued, "Additionally, we recently reported positive top-line data from the fixed dose, blinded portion of PaTH Forward evaluating TransCon PTH. This portion of the phase 2 trial met its key objectives, marking a major potential advance in helping hypoparathyroidism patients who are in urgent need of an effective PTH replacement therapy. And, with the appointment of Jesper Høiland as Global Chief Commercial Officer, we have added significant commercial expertise related to our growth hormone and PTH product candidates."
Corporate Highlights & Progress
For TransCon hGH, an investigational long-acting prodrug of somatropin (human growth hormone or hGH), remain on track for planned submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) in the second quarter, and a Marketing Authorisation Application (MAA) to the European Medicines Agency now in the third quarter, both for pediatric growth hormone deficiency (GHD).
Submitted an Investigational New Drug (IND) amendment to initiate the global, phase 3 foresiGHt Trial evaluating TransCon hGH in adults with GHD. Enrollment is expected to begin later this year.
Remain on track to initiate a phase 3 trial with TransCon hGH in pediatric GHD in Japan in the fourth quarter.
Received Orphan Drug Designation from the FDA for TransCon hGH as a treatment for GHD.
Announced positive top-line results 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 showed that within only four weeks TransCon PTH replaced the current standard of care for 82 percent of patients with hypoparathyroidism.
Remain on track to report six-month data from the open-label extension portion of the PaTH Forward Trial during the third quarter, and to initiate a global phase 3 trial of TransCon PTH in North America, Europe and Asia in the fourth quarter.
Remain on track to submit the first IND or equivalent in oncology in the fourth quarter for TransCon TLR7/8 Agonist.
Announced the appointment of Jesper Høiland as Global Chief Commercial Officer. Mr. Høiland has over 25 years of global senior leadership experience in biopharma, including over 20 years of global commercial experience in growth hormone.
Opened the company’s new facility in Redwood City, California to advance its pipeline of oncology programs.
Ended the first quarter 2020 with cash and cash equivalents of €534.4 million.
First Quarter 2020 Financial Results
For the first quarter, Ascendis Pharma reported a net loss of €63.3 million, or €1.32 per share (basic and diluted) compared to a net loss of €53.6 million, or €1.24 per share (basic and diluted) for the same period in 2019.
Revenue for the first quarter was €2.2 million compared to €5.4 million in the same quarter of 2019. The decrease was due to a lower amount of license and service revenue being recognized, partly offset by sale of clinical supply to Visen.
Research and development (R&D) costs for the first quarter were €57.5 million compared to €51.3 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.
General and administrative expenses for the first quarter were €17.9 million compared to €10.4 million during the same period in 2019. The increase is primarily due to additional personnel-related costs, higher IT and facility costs, and continued build out of the company’s commercial capabilities.
As of March 31, 2020, Ascendis Pharma had cash and cash equivalents of €534.4 million compared to €598.1 million as of December 31, 2019. As of March 31, 2020, Ascendis Pharma had 47,985,837 ordinary shares outstanding.
Conference Call and Webcast information
Ascendis Pharma will host a conference call and webcast today at 4:30 p.m. Eastern Time (ET) to discuss its first quarter 2020 financial results. Details include:
Date May 19, 2020
Time 4:30 p.m. ET
Dial In (U.S.) 844-290-3904
Dial In (International) 574-990-1036
Access Code 8295749
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.
About Ascendis Pharma’s Pipeline
Ascendis Pharma currently has three product candidates in clinical development in rare endocrine diseases:
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 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.
Additionally, the company has established oncology as its second therapeutic area of focus and plans to submit an IND or equivalent in the fourth quarter of 2020 for its first oncology product candidate.