Joyce Shao

Joyce Shao inquired about KOL feedback on the two-year CRENESSITY data, specifically regarding the durability of benefit, and how this data is expected to support and drive strong patient persistence on the drug.

Answer

Chief Medical Officer Sanjay Keswani reported positive clinician support, highlighting durable reductions in ACTH and androgens, weight loss in obese patients, improved insulin tolerance, and attenuation of bone age advancement. CEO Kyle Gano added that the 90% rollover and 80% retention at two years in the open-label extension underscore the excellent safety and tolerability, which is crucial for long-term treatment and bending the disease course.

Joyce Shao asked about KOL feedback on the two-year CRENESSITY data, specifically regarding the durability of benefit and how this data supports and drives strong patient persistence on the drug.

Answer

Sanjay Keswani, Chief Medical Officer, reported positive support from clinicians, emphasizing the importance of chronic androgen reduction and glucocorticoid dose reduction to physiological levels. He highlighted two-year data showing weight reduction in obese patients, improved insulin tolerance, attenuation of bone age advancement in pediatrics, excellent tolerability (80% retention at two years), no new safety signals, and preserved vasopressin-induced ACTH stimulus. Kyle Gano, Chief Executive Officer, added that the high retention rate and long-term efficacy data underscore the benefits of sustained treatment.