Ascendis Pharma A/S (NASDAQ:ASND) Q3 2019 Earnings Conference Call - Final Transcript
Nov 18, 2019 • 04:30 pm ET
in Europe in the second half of 2020. We also advancing our two other endocrinology rare disease programs. For TransCon PTH, we announced last week an expansion of our phase 2 trial, which I will discuss in detail and the indicated as planned our TransCon P [Phonetic] phase 2 trial.
We also continue to make progress with our clients through a status, global, clinical and commercial reach for our endocrinology rare disease pipeline.
As our partner in China, VISEN Pharmaceuticals initiated a phase 3 trial for TransCon Growth Hormone for pediatric growth hormone deficiency. Let me start with an update on TransCon Growth Hormone.
Preparation for our BLA filing in the first half of 2020 and MAA filing in the second half of 2020 are both on track. Last quarter, we discussed the two items remaining to be completed prior to filing. Finalization of our long-term safety data and completion of our PPQ manufacturing validation batches.
As planned, in Q3, we reported the last subject visit for the long-term safety database consisting of around 300 subjects on trial of six months, 120 subjects for 12 months, 45 subjects for two years. We have also now completed the production of our top product validation batches required for regulatory filings. We are now finalizing the associated analytical and qualification reports for the BLA and MAA filings. Recently, the European Commission granted us Orphan Designation for TransCon Growth Hormone for pediatric growth hormone deficiency in Eurpoe. Orphan Designation is provided to therapeutic for certain health condition that affect a limit number of patient, and if a treatment already exist, the major thing must be of significant additional benefit.
This is an early indication that the European health authorities recognize the unmet need in pediatric growth hormone deficiency and the clinical benefits of our once-weekly TransCon Growth Hormone therapy.
In addition, in the US, in the enliGHten Trial around 150 patients have now successfully being transferred over to our intended commercial presentation of TransCon Growth Hormone consisting of our auto injector and dual-chamber cartridge. We believe this patient data will be sufficient to support our auto injector to be part of our initial BLA submission.
In light of recent clinical development with other long-acting growth hormone products, I would like to address the fundamentals of growth hormone treatment. A successful product needs to have both the direct and indirect benefit of growth hormone to be safe and effective long-acting growth hormone therapy.
We have stated for many years that the target product profile for a commercial successful long-acting growth hormone must have all the integrated benefits of daily growth hormone therapy. This benefit of growth hormone treatment include not only catch-up, sustained height velocity but also improved body composition, including fat mass and muscle BMI formalization, improved bone health, bone age advancement and improved quality of life. We believe, maintaining the same mode of action and tissue distribution as daily growth hormone is critical to mimic all these effect of native growth hormone.