BridgeBio scores second major 2025 win with trial in rare endocrine disorder

BridgeBio’s drug encaleret succeeded in a late-stage trial, showing strong potential as a new treatment standard for ADH1, a rare inherited endocrine disorder.

Why it matters: Encaleret significantly outperformed current treatments by normalizing calcium and parathyroid hormone levels in most patients, offering hope to thousands with limited options.

Backstory: ADH1, a form of hypoparathyroidism, disrupts calcium balance due to a genetic mutation affecting a key protein. Current treatments rely on supplements and surgery, often with limited success.

Zoom in: ADH1 affects ~12,000 people in the U.S., with 3,000–5,000 having severe disease. On the bright side, encaleret achieved normalized calcium levels in 76% of patients as opposed to 4% on conventional therapy, and 91% showed corrected parathyroid hormone levels after 24 weeks.

Big picture: The company’s shares have more than doubled since January, driven by the early commercial success of its heart drug Attruby. And this week, BridgeBio notched late-stage trial wins for another program, BB-418, a therapy for a form of muscular dystrophy. Both BB-418 and encaleret are seen by analysts as potential $1 billion–plus market opportunities.