Americas Healthcare Biotechnology Takeaways From Endocrinologist Lunch

This report summarizes insights from an endocrinologist regarding the clinical landscape of various rare endocrine diseases. It highlights strong physician interest in emerging therapies that offer less frequent dosing and differentiated mechanisms of action.

Key Takeaways

• 1.KOL sees a 50% response rate as a reasonable trade-off threshold for weekly-injectable therapies in hypoparathyroidism.
• 2.Physician expresses enthusiasm for setmelanotide in Prader-Willi Syndrome, noting weight loss results as unprecedented.
• 3.Potential for atulmenant to differentiate in CAH treatment via convenience and mechanism of action compared to Crenessity.

Table of Contents

• Hypoparathyroidism (ASND, MBX)
• KOL sees a 50% response rate threshold for the weekly-injectables.
• Congenital Adrenal Hyperplasia (NBIX, CRNX)
• Positive experience with Crenessity, atumelnant could address unmet need via differentiated mechanism of action
• Post-Bariatric Surgery Hypoglycemia (AMLX)
• Significant unmet need in PBH population, estimated at 15-20% of patients and reflected in patient demand for new therapeutic options
• Prader-Willi Syndrome (NBIX, CRNX, RYTM)
• Enthusiasm for both Vykat and setmelanotide in this very difficult to treat patient population
• Hypothalamic Obesity (RYTM)
• Thyroid Eye Disease / Graves' Disease (VRDN, ROIV/IMVT)
• Achondroplasia (ASND, BBIO, BMRN, TYRA)
• Oral options could meaningfully expand treatment for achondroplasia
• Acromegaly (CRNX)
• Palsonify used in 10% patients but half may qualify, fasting could be challenging to manage

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