Ascendis Pharma Announces Once-weekly TransCon? Growth Hormone Demonstrated Superiority on Primary Endpoint Compared to a Daily Growth Hormone in Phase 3 heiGHt Trial for Pediatric Growth Hor
?TransCon hGH demonstrated comparable safety and tolerability to a daily hGH -
- Potential once-weekly treatment option to overcome long-standing challenges with administration of daily hGH -
- Currently no available long-acting growth hormone treatment in the US or?Europe?-
-?Ascendis Pharma?to host conference?call and webcast today at?8:00 a.m. Eastern time?-
COPENHAGEN, Denmark, - ?Ascendis Pharma A/S(Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technology to address unmet medical needs, today announced positive top-line results from the phase 3 heiGHt Trial, a randomized, open-label, active-controlled trial that compared once-weekly TransCon Growth Hormone (hGH) to a daily growth hormone (Genotropin?) in children with pediatric growth hormone deficiency (GHD). The trial met its primary objective, demonstrating that TransCon hGH was observed to be non-inferior and, additionally, superior to the daily hGH on the primary endpoint of annualized height velocity (AHV) at 52 weeks. In the primary analysis of the intent-to-treat population using ANCOVA, TransCon hGH demonstrated an AHV of 11.2 cm/year compared to 10.3 cm/year for the daily hGH. The treatment difference was 0.86 cm/year with a 95 percent confidence interval of 0.22 to 1.50 cm/year. The AHV for TransCon hGH was significantly greater than the daily hGH (p=0.0088). The AHV was greater for TransCon hGH than for the daily hGH at each visit, with the treatment difference reaching statistical significance from and including week 26 onward. The incidence of poor responders (AHV < 8.0 cm/year) was 4 percent and 11 percent in the TransCon hGH and daily hGH arms, respectively. All sensitivity analyses completed from the trial support the primary outcome, indicating the robustness of these results. Results from the trial indicate that TransCon hGH was generally safe and well-tolerated, with adverse events consistent with the type and frequency observed with daily hGH therapy and comparable between arms of the trial. Key safety observations:- No serious adverse events related to study drug were observed in either arm
- One serious adverse event was observed in each arm (representing 1.0 percent for TransCon hGH and 1.8 percent for daily hGH), both determined to be unrelated to study drug
- No treatment-emergent adverse events leading to discontinuation of study drug were observed in either arm
- No neutralizing antibodies detected, and low level (<10 percent) of low-titer non-neutralizing antibodies was similar between the two arms
- Height standard deviation score (SDS) at 52 weeks increased over baseline by 1.05 for TransCon hGH and by 0.94 for the daily hGH, and the treatment difference in height SDS increased at each visit over 52 weeks
- Body Mass Index (BMI) SDS was stable over 52 weeks and was -0.03 for TransCon hGH and -0.40 for the daily hGH at week 52
- Mean hemoglobin A1c values were generally stable over the course of the trial and remained within the normal range for both arms
- Observed peak and trough insulin-like growth factor-1 (IGF-1) SDS values were 1.3 and -0.5 over 52 weeks, respectively, for TransCon hGH compared to an approximate average IGF-1 SDS of 0.0 for the daily hGH at week 52
- In a pre-defined subset of 11 subjects, IGF-1 levels were assessed during week 13 and results were similar to those reported in the TransCon hGH pediatric phase 2 trial
- Consecutive IGF-1 SDS values >2.0 were uncommon (<10 percent of subjects) and IGF-1 SDS >3.0 were rare (<3 percent of subjects)
- Adverse events leading to dose reduction (IGF-1 levels or clinical symptoms) occurred twice in the TransCon hGH arm (representing 1.9 percent) and once in the daily hGH arm (representing 1.8 percent)
- Two subjects in each treatment arm experienced mild injection site reactions that were considered adverse events
Date | March 4, 2019 |
Time | 8:00 a.m. ET |
Dial In (U.S.) | 844-290-3904 |
Dial In (International) | 574-990-1036 |
Access Code | 9556035 |
Internal contact: Scott T. Smith Chief Financial Officer (650) 352-8389 ir@ascendispharma.com Media contact: Ami Knoefler Head of Global Communications (650) 739-9952 ack@ascendispharma.com Investor contact: Patti BankWestwicke Partners (415) 513-1284 patti.bank@westwicke.com