Peptide News Digest

#Pediatric

2 stories

Clinical Trials · View digest

Neurocrine CRENESSITY (Crinecerfont) Two-Year Pediatric CAH Data at ENDO 2026 Plus First Retrospective Case Series in 11β-Hydroxylase Subtype

Neurocrine Biosciences (Nasdaq: NBIX) presented two-year data from CAHtalyst Pediatric showing durable hormone control, reduced glucocorticoid exposure, and improved growth measures in pediatric patients with classic congenital adrenal hyperplasia (CAH), with weight, insulin resistance, and bone-age outcomes also improved at year 2. Separately, Neurocrine announced the first retrospective case series of CRENESSITY (crinecerfont) in patients with classic CAH due to 11β-hydroxylase deficiency, the second-most-common form of CAH after 21-hydroxylase deficiency, accounting for roughly 5% of cases. The 11β-OHD subtype was not previously studied in the crinecerfont trials and is characterized by cortisol deficiency plus excess adrenal androgens and accumulation of 11-deoxycortisol and 11-deoxycorticosterone. Crinecerfont is a small-molecule CRF1 receptor antagonist that dampens the CRH-ACTH peptide signaling axis.

Clinical Trials · View digest

FDA Approves Sanofi's Tzield (Teplizumab) for Children Down to Age 1 in Stage 2 Type 1 Diabetes — First Disease-Modifying Therapy for Pediatric T1D

Sanofi announced April 22 FDA approval of an expanded Tzield (teplizumab-mzwv) indication for stage 2 type 1 diabetes patients as young as 1 year old, down from the previous 8-and-up indication. The approval was supported by 1-year data from the PETITE-T1D Phase 4 study (n=23, mean age 4.8 years), showing 89.6% probability of remaining stage-3-progression-free at 1 year. Tzield delays progression from stage 2 to stage 3 T1D by an average of 2 years and is the first disease-modifying therapy for autoimmune T1D — relevant to the broader insulin/peptide endocrinology landscape.