On March 10, 2021 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to create product candidates that address unmet medical needs, reported financial results for the full year ended December 31, 2020 (Press release, Ascendis Pharma, MAR 10, 2021, View Source [SID1234576387]).

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"2020 was a year to remember for Ascendis as our global workforce delivered on time or ahead of schedule on all of our corporate milestones, advancing Vision 3×3, the company’s strategic roadmap through 2025 to build a leading biopharma company by achieving sustainable growth through multiple approaches," said Jan Mikkelsen, Ascendis’ President and CEO. "As we look to 2021, we expect to report important clinical updates across our entire investigational product portfolio, including Endocrinology Rare Disease and Oncology, while also achieving key commercial milestones including the anticipated regulatory approval and commercial launch in the United States, and regulatory approval in Europe, of TransCon hGH for pediatric growth hormone deficiency."

Company Highlights & Progress

TransCon hGH (lonapegsomatropin)
○ Continued to build the global commercial infrastructure in preparation for the PDUFA date of June 25, 2021, and expected subsequent commercial launch of lonapegsomatropin in the United States in the third quarter of 2021. European Commission decision is anticipated in the fourth quarter of 2021 for pediatric patients with growth hormone deficiency (GHD).
○ Reported two year data from the enliGHten long-term extension trial demonstrating children with GHD initially treated with lonapegsomatropin maintained an advantage in height SDS (standard deviation score) improvement.
○ Continued execution in the ongoing foresiGHt Trial, a global phase 3 study in adults with GHD, complete enrollment expected by late 2021 or early 2022.
○ Submitted Clinical Trial Notification (CTN) to initiate pediatric GHD phase 3 riGHt Trial in Japan.

TransCon PTH
○ Submitted regulatory filings in North America and Europe to initiate PaTHway, a phase 3 clinical trial evaluating the safety, tolerability and efficacy of TransCon PTH in adults with hypoparathyroidism (HP). Topline results are expected from PaTHway in the fourth quarter of 2021.
○ Announced six-month data from the PaTH Forward Trial open-label extension (OLE), which demonstrated TransCon PTH replaced standard of care, normalized quality of life and urinary calcium. These results will be presented in an oral presentation on March 23, 2021 at ENDO 2021.
○ As of March 10, 2021, 58 out of the 59 randomized subjects continue in the phase 2 PaTH Forward clinical trial.
○ On track to file CTN to initiate adult HP phase 3 trial in Japan in the second quarter of 2021.

TransCon CNP
○ Continued dose escalation in the phase 2 ACcomplisH Trial to evaluate the safety and efficacy of TransCon CNP in children ages two to ten with achondroplasia.
○ IND approved for the ACcomplisH China Trial, a phase 2 trial to enable dose expansion cohorts, in children ages two to ten, conducted in Greater China by Visen Pharmaceuticals (VISEN).
○ Clinical program update on both randomized, double-blind, placebo-controlled trials expected in the fourth quarter of 2021.

TransCon TLR7/8 Agonist
○ Filed IND application with the FDA to initiate the transcendIT-101 clinical trial.
○ Initial results from the first part of transcendIT-101, the monotherapy dose escalation, are expected in the fourth quarter of 2021.
○ Expect to initiate the second part of transcendIT-101, dose escalation of TransCon TLR7/8 Agonist in combination with a checkpoint inhibitor, in the second quarter of 2021.

TransCon IL-2 β/γ
○ New preclinical data demonstrated a single dose of TransCon IL-2 β/γ resulted in durable and robust increases in the ratios of CD8+ T cells and NK cells over Treg cells in non-human primates.
○ IND filing or similar planned in the third quarter of 2021.

Ended 2020 with cash, cash equivalents and marketable securities totaling €834.1 million.
Full Year 2020 Financial Results

For the full year 2020, Ascendis Pharma reported a net loss of €419.0 million, or €8.28 per share (basic and diluted) compared to a net loss of €218.0 million, or €4.69 per share (basic and diluted) for the same period in 2019.

Revenue for 2020 was €7.0 million compared to €13.4 million in 2019. The decrease was due to lower license and service revenue, partly offset by sale of clinical supply, to VISEN.

Research and development (R&D) costs for 2020 were €260.9 million compared to €191.6 million in 2019. Higher R&D costs in 2020 reflect an increase in personnel-related costs, expansion of R&D facilities and the continued progress in development of the company’s product candidates.

Selling, general and administrative expenses for 2020 were €76.7 million compared to €48.5 million in 2019. The increase is primarily due to higher personnel-related costs, higher IT costs and continued build out of the company’s commercial capabilities.

Net loss in associate was €9.5 million for 2020 compared to €8.1 million in 2019, representing the company’s share of net result from VISEN.

Net finance expenses were €79.0 million for 2020 compared to net finance income of €16.6 million in 2019, primarily reflecting negative exchange rate fluctuations compared to 2019 when the company recognized a gain.

As of December 31, 2020, Ascendis Pharma had cash, cash equivalents and marketable securities totaling €834.1 million compared to €598.1 million at the beginning of the year. As of December 31, 2020, Ascendis Pharma had 53,750,386 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 full year 2020 financial results.

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.