On August 27, 2020 Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to address unmet medical needs, reported new data from PaTH Forward and financial results for the quarter ended June 30, 2020 (Press release, Ascendis Pharma, AUG 27, 2020, View Source [SID1234564094]).

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"We are executing across the globe on all elements of our Vision 3×3, including preparation for the expected U.S. launch for TransCon hGH following submission of our Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) in June, and preparing to submit the Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for TransCon hGH in Europe planned for third quarter," said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer.

Mr. Mikkelsen continued, "Additionally, with today’s data release, we have, for the first time in a randomized, double-blind, placebo-controlled trial, demonstrated that a therapy for hypoparathyroidism (HP) may have a significant impact on improving quality of life for people living with HP compared to the standard of care. Our new data showed that TransCon PTH demonstrated a statistically significant1 and clinically meaningful impact on the SF-36 Health Survey (SF-36), a quality of life assessment tool validated to measure functional health and well being compared to placebo.2 Analysis of these exploratory endpoints, combined with expected findings from our proprietary patient reported outcome instrument, called Hypoparathyroidism Patient Experience Scale (HPES), will support the emerging body of evidence that TransCon PTH may function as a hormone replacement therapy and make a meaningful difference in improving the lives of people with HP."

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) (Free AACR Whitepaper) Virtual Annual 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 2020 were 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 2020 equity offering.
Second Quarter 2020 Financial Results

For the second quarter, Ascendis Pharma reported a net loss of €94.9 million, or €1.97 per share (basic and diluted) compared to a net loss of €58.9 million, or €1.25 per share (basic and diluted) for the same period in 2019.

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 VISEN Pharmaceuticals.

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.

As of June 30, 2020, Ascendis Pharma had cash, cash equivalents and marketable securities totaling €471.6 million compared to €534.4 million as of March 31, 2020. As of June 30, 2020, Ascendis Pharma had 48,345,782 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 second quarter 2020 financial results. Details include:

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 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.