Ascendis Pharma (ASND) Presents At 38th Annual J.P. Morgan Healthcare Conference - Slideshow

Endocrinology Rare Diseases Pipeline - Ascendis Pharma expects to submit the BLA for TransCon hGH for pediatric growth hormone deficiency in Q2 2020 and the MAA in Q4 2020[7, 23, 50] - The Phase 3 heiGHt Trial demonstrated superior height velocity (AHV) of TransCon hGH in pediatric GHD, with 112 cm/year compared to 103 cm/year for Genotropin, a treatment difference of 086 cm/year[25, 50] - The adverse event profile of TransCon hGH was comparable to daily hGH across Phase 3 trials, with 77% of patients experiencing treatment-emergent adverse events (TEAEs) in the TransCon hGH 024 mg/kg/week group in the heiGHt trial, compared to 70% in the Genotropin group[42] - Ascendis Pharma plans to initiate a Phase 3 trial for adult GHD in Q1 2020 and a Phase 3 trial for pediatric GHD in Japan in Q4 2020[50, 97] - Top-line Phase 2 data for TransCon PTH in hypoparathyroidism is expected by the end of March 2020, with six-month data from the open-label extension expected in Q3 2020[7, 65, 97] - Preliminary data from the first 8 subjects completing 4 weeks follow-up in open-label extension of TransCon PTH trial shows that all subjects are completely off current standard of care, 8 of 8 subjects no longer require active vitamin D, and 7 of 8 subjects no longer require calcium supplements[65] - Ascendis Pharma expects to initiate a Phase 2 trial for achondroplasia (ACH) in China in Q4 2020 through VISEN Pharmaceuticals[7, 70, 97] Oncology Pipeline - Ascendis Pharma anticipates submitting the first IND or equivalent for its oncology program in Q4 2020[7, 95, 97] - Preclinical data for TransCon IL-2 b/g in cynomolgus monkeys showed a single 1 mg dose/animal (~01 mg/kg) resulted in >3-fold enhancement of lymphocyte counts, with minimal effect on eosinophils compared to Aldesleukin[88] Financial Position - As of September 30, 2019, Ascendis Pharma had approximately €659 million in cash and cash equivalents[7]