Ascendis Pharma, a renowned biopharmaceutical company working towards finding new therapies for several unmet medical demands, reportedly announced that it would present an outlook for 2020 along with the review progress of Vision 3×3, its strategic growth plan through 2025 in order to attain sustainable growth, during the 38th Annual J.P. Morgan Healthcare Conference which would be conducted in San Francisco.
President and Chief Executive Officer, Ascendis Pharma, Jan Mikkelsen stated that 2019 was a revolutionary year for the company. For Ascendis’ endocrinology rare disease portfolio, the company was successful in its first phase 3 study for TransCon hGH, a product candidate of TransCon. The company also advanced two more programs across the world into phase 2. Additionally, the company also established oncology as its second therapeutic domain with highly differentiated pipeline by the use of TransCon technology.
Mikkelsen further added that 2019 was only the beginning of the company’s path to create a top, fully integrated, global biopharma firm. The company is on track with its Vision 3×3 objectives to offer multiple sources of growth as it seeks to make important differences in the lives of patients.
TransCon hGH is a new long-acting prodrug of hGH (human growth hormone) in phase 3 as a once-weekly therapy for GHD (growth hormone deficiency). TransCon hGH can release unmodified somatropin. The drug has evaluated a statistically substantial growth in height speed compared to a day-to-day hGH during the phase 3 heiGHt study.
The company recently conducted two pre-BLA meetings with the U.S. FDA (Food and Drug Administration) to review the CMC (Chemistry, Manufacturing and Controls), and non-clinical/clinical packages for TransCon as a potential therapy for pediatric GHD.
The company is presently on track to document a BLA (Biologics License Application) with the FDA during the second quarter of 2020. Also, a Marketing Authorization Application is planned to be filed to the European Medicines Agency during the fourth quarter.
Ascendis aims to submit regulatory filings to start a global, phase 3 clinical study in adult GHD in the first quarter. The company also aims to initiate a trial in pediatric GHD in Japan in the fourth quarter.