– Pre-launch activities continue in preparation for a potential FDA approval of TransCon™ hGH (lonapegsomatropin) for pediatric growth hormone deficiency; PDUFA date of June 25, 2021 –
– 58-week data from PaTH Forward demonstrated durable benefit of TransCon PTH signifying potential as a hormone replacement therapy for patients with hypoparathyroidism –
– First-in-human dosing for Ascendis Oncology portfolio with the transcendIT-101 clinical trial moving forward –
– Conference call today at 4:30 p.m. Eastern Time –
COPENHAGEN, Denmark, May 27, 2021 (GLOBE NEWSWIRE) — Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon™ technologies to create product candidates that address unmet medical needs, today announced financial results for the first quarter ended March 31, 2021.
“With potential U.S. FDA approval for TransCon hGH now less than a month away, we believe we are one step closer to fulfilling our Vision 3×3 to build a leading biopharma company. Guided by our values of patients, science and passion, we have built a pipeline of three differentiated endocrinology rare disease product candidates by applying our TransCon technology to clinically validated parent drugs and/or targets. Each candidate targeting substantial unmet medical needs in large markets where we have the potential to become the market leader. We plan to replicate that success in oncology where we have dosed our first patients this past quarter in the transcendIT-101 Trial of TransCon TLR7/8 Agonist and expect to file an IND or similar for our second oncology product candidate TransCon IL-2 β/γ in Q3 2021. Improving patient lives is the driving force behind our dedicated employees,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer.
Company Highlights & Progress
- TransCon hGH (lonapegsomatropin)
- Continued pre-launch commercial activities in preparation of the PDUFA date on June 25, 2021 and an expected subsequent commercial launch of lonapegsomatropin in the United States in the third quarter of 2021 for the treatment of pediatric patients with growth hormone deficiency (GHD). All FDA information requests relating to our BLA for TransCon hGH for the treatment of pediatric GHD have been responded to with no questions currently outstanding.
- European Commission decision on the company’s Marketing Authorisation Application (MAA) is anticipated in the fourth quarter of 2021 for the treatment of pediatric patients with GHD.
- A recent study published online in the Journal of Managed Care & Specialty Pharmacy1 and presented at ENDO 2021 highlighted the economic burden of GHD in the U.S. pediatric population. The study demonstrated that pediatric GHD is a significant healthcare burden, and many patients remain untreated or undertreated with high rates of non-adherence (70-80%).
- Continued execution in the ongoing foresiGHt Trial, a global phase 3 trial in adults with GHD, complete enrollment expected by late 2021 or early 2022.
- Continued execution in the ongoing riGHt Trial, a Japanese phase 3 trial for pediatric GHD.
- In Greater China, VISEN Pharmaceuticals completed the patient enrollment of 154 treatment-naïve, prepubertal children for the ongoing phase 3 pivotal trial of lonapegsomatropin in patients with pediatric GHD.
- TransCon PTH
- Announced preliminary 58-week results from the continuing open-label extension (OLE) portion of the PaTH Forward Trial, a global phase 2 trial evaluating the safety, tolerability, and efficacy of its investigational product candidate TransCon PTH in adult subjects with hypoparathyroidism (HP). The results demonstrated that TransCon PTH was well tolerated at all doses administered and provided durable benefit in adults with HP. As of May 25, 2021, 58 out of the 59 randomized subjects continue in the OLE portion of the phase 2 PaTH Forward Trial.
- Filed a Clinical Trial Notification with the Pharmaceuticals and Medical Devices Agency in Japan to initiate the company’s phase 3 clinical trial of TransCon PTH in adult subjects with HP, the PaTHway Japan Trial.
- Continued execution in the ongoing North American and European phase 3 PaTHway Trial in adults with HP with topline results expected in the fourth quarter of 2021.
- TransCon CNP
- Continued execution in the ongoing phase 2 ACcomplisH Trial and ACcomplisH China Trial to evaluate the safety and efficacy of TransCon CNP in children ages two to ten with achondroplasia.
- On track for clinical program update on both randomized, double-blind, placebo-controlled trials expected in the fourth quarter of 2021.
- TransCon TLR7/8 Agonist
- Continued execution in the ongoing phase 1/2 trial, transcendIT-101.
- TransCon IL-2 β/γ
- IND filing or similar planned in the third quarter of 2021.
- Ended the first quarter of 2021 with cash, cash equivalents and marketable securities totaling €771.1 million.
First Quarter 2021 Financial Results
For the first quarter, Ascendis Pharma reported a net loss of €62.8 million, or €1.17 per share (basic and diluted) compared to a net loss of €63.3 million, or €1.32 per share (basic and diluted) for the same period in 2020.
Revenue for the first quarter was €0.7 million compared to €2.2 million in the same quarter of 2020. The decrease was due primarily to a lower amount of license revenue being recognized, as well as lower sale of clinical supply and services to VISEN compared to the same period the prior year.
Research and development (R&D) costs for the first quarter were €88.1 million compared to €57.5 million during the same period in 2020. Higher R&D costs in 2021 reflect an increase in personnel-related costs and continued progress in development of the company’s product candidates.
Selling, general and administrative expenses for the first quarter were €37.2 million compared to €17.9 million during the same period in 2020. The increase is primarily due to higher personnel-related costs, continued build out of the company’s commercial capabilities, and a change in expense reallocation of shared services.
Net profit of associate for the first quarter was €28.1 million compared to a net loss of €1.5 million in the same quarter of 2020. Net profit of associate for the first quarter included a non-cash gain of €42.3 million as a result of the Series B financing in VISEN on January 8, 2021, partly offset by our share of VISEN’s net loss for the first quarter of €14.2 million.
As of March 31, 2021, Ascendis Pharma had cash, cash equivalents and marketable securities of €771.1 million compared to €834.1 million as of December 31, 2020. As of March 31, 2021, Ascendis Pharma had 53,829,379 ordinary shares outstanding.
Conference Call and Webcast Information
|Date||Thursday, May 27, 2021|
|Time||4:30 p.m. Eastern Time/1:30 p.m. Pacific Time|
|Dial In (U.S.)||844-290-3904|
|Dial In (International)||574-990-1036|
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 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 and one oncology product candidate in clinical development:
- 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, is designed as a once-weekly treatment for GHD and is currently under regulatory review for pediatric GHD by the U.S. Food and Drug Administration and the European Medicines Agency.
- TransCon PTH, an investigational long-acting prodrug of parathyroid hormone (PTH) in phase 3…