Crinetics Pharmaceuticals' atumelnant, a novel ACTH antagonist, has shown promising results in a Phase 2 trial for congenital adrenal hyperplasia (CAH), leading to reiterated Buy ratings from analysts at Morgan Stanley, Citi, and H.C. Wainwright. The trial demonstrated significant reductions in androstenedione (A4) levels, a key biomarker for CAH, across various dosages, with the highest dose achieving a notable decrease.
Significant A4 Reductions and Rapid Onset of Action
The Phase 2 TouCAHn trial results indicated that atumelnant achieved substantial A4 reductions as early as Week 2, which were sustained through Week 12. These results compare favorably to existing treatments like crinecerfont, which have shown lower A4 reduction outcomes in similar timeframes. Douglas Tsao, analyst at H.C. Wainwright, noted that the reductions of 60%, 70%, and 80% at week 12, while showing some variability compared to initial data, remain clinically significant.
Clinical Improvements and Potential Disease Modification
Beyond A4 reduction, the trial also indicated potential disease-modifying capabilities. A significant number of female patients experienced normalization of testosterone levels, and some resumed menstruation. David Lebowitz from Citi highlighted that these reductions translated to meaningful clinical benefits, such as improvements in adrenal gland size, hirsutism, acne, and menstrual cycle regularity, underscoring the drug’s unique mechanism of action.
Path to Phase 3 and Future Growth
Crinetics Pharmaceuticals is planning to advance atumelnant into a Phase 3 pivotal trial for adults and a Phase 2b/3 trial for pediatric patients in 2025. This progression underscores the company's confidence in atumelnant's potential. The overall safety profile of atumelnant was deemed acceptable, despite one instance of elevated liver enzymes. Cory Jubinville, PhD from LifeSci Capital, anticipates that these factors collectively underpin the Buy rating given.
Analyst Confidence
Analysts have expressed confidence in Crinetics Pharmaceuticals, with price targets ranging from $70 to $82. The positive trial outcomes and the expected share price return underpin the confident Buy ratings. The company's focus on rare endocrine diseases and endocrine-related tumors, along with its pipeline of oral nonpeptide therapeutics, positions it for continued growth and success.
