News from the Pediatric Endocrine Society Conference

Earlier this month, the annual conference of the Pediatric Endocrine Society (PES) was held in Chicago. PES has over 1600 members representing multiple disciplines of pediatric endocrinology, including bone, thyroid, diabetes, pituitary, adrenal and growth. It's Special Interest Groups provide a forum for interested members to collaborate and facilitate discussion in a particular area of pediatric endocrinology, like obesity, PCOS, Turner Syndrome etc. This year's annual conference brought together experts and collaborators in several different fields-here are a few highlights from some exciting presentations and posters.

New options for treatment of congenital adrenal hyperplasia were showcased, after multicenter research trials led by Spruce Biosciences explored tildacerfont, a novel CRF-1 receptor antagonist. This is a non-steroid alternative for CAH treatment and specially attractive for those requiring high dose steroids to keep adrenal hormone levels in target range. It is taken once daily by mouth and aims to address unmet needs in CAH in both children/adolescents (CAHptain trial) and adults (CAHmelia trial). Data presented showed that tildacerfont was safe and well tolerated. About 70% of participants showed improved CAH control or ability to reduce steroid dosing after 12 weeks of treatment with the new agent, although with suggestions that a higher or more frequent dose may be needed, specially in children.

Of note, a competitor Crincerfont produced by Neurocrine Biosciences with the same mechanism of action also has Phase 3 data from it's CAHtalyst and CHAMPAIN trials which the company presented in Chicago and is also planning on presenting at the European Congress of Endocrinology Conference in Sweden this week. The data from these studies has also been positive and the company has submitted applications to obtain a New Drug Application to the FDA in April 2024.

Rhythm Pharmaceuticals Inc. presented information on their anti-obesity medication setmelanotide (Imcivree) which is the only FDA approved option for 4 genetic reasons of obesity which are resistant to diet and lifestyle modifications, and typically present at a very early age with extreme weight gain. These include Bardet-Biedl Syndrome (BBS), POMC deficiency, LEPR mutation and PCSK1. This agent works on MC4R signaling in the brain to reduce hunger. Label expansion to younger ages (2-6 years) is being actively sought by the manufacturer. It is a once daily subcutaneously administered injection requiring monitoring for certain specific side effects.

Another exciting novel agent in development is an antibody that targets the downstream effects of insulin and can alleviate the (unpredictable and sometimes severe) low blood sugars that people with hyperinsulinism, insulinoma and some pancreatic tumors suffer from. Results from the RIZE study Rezolute, Inc. have shown improvement in hypoglycemia in the past, as noted via CGM trends, and at the Chicago meeting results from overnight studies from pediatric patients were presented. The overnight phase is a specially vulnerable time with fasting and low awareness during sleep increasing the risk of hypoglycemia. Previous studies in children as young as 2 and older have shown safety and tolerability and abstracts have been presented from global studies at other conferences.

More to come from this exciting conference that also discussed thyroid hormone monotherapy, early puberty, bone disease and challenges in transgender health.