Potential Congenital Adrenal Hyperplasia Drug Treatment Clears Hurdle

Some promising news for the congenital adrenal hyperplasia (CAH) community!

Spruce Biosciences is one step closer to a treatment for CAH after new phase 2a data showed the drug reduced the level of three hormones overproduced by patients with CAH.

We previously covered the previous stage back in March.

This development tees it up for its next trial, which will gauge whether it can replace the large amounts of steroids currently used to treat the disorder.

That study is slated to start in 2020.

What is CAH?

Congenital adrenal hyperplasia is a group of inherited genetic disorders that affect the adrenal glands, which are walnut-sized organs above the kidneys that help regulate metabolism, the immune system, blood pressure, and other essential functions. Someone with CAF lacks an enzyme that the adrenal glands use to do these vital jobs, in turn affecting the production of steroid hormones, such as cortisol, testosterone, etc.

To read more about CAH, click here.


The drug — tildacerfont — is a daily pill that works by blocking corticotropin-releasing factor (CRF) type 1 receptors to slash the production of adrenocorticotropic hormone (ACTH).

After 12 weeks of treatment, the drug lowered ACTH levels an average of 74% from baseline. The average reduction from baseline was 82% for 17-OHP and 55% for A4.

And more than half of the patients saw their ACTH come down to normal levels!

“Almost all patients tended toward substantial improvement,” Spruce CEO Richard King said. “We did see about 60% of patients normalize for ACTH and 40% for A4 levels in the study. I do anticipate over 16 weeks, those numbers would improve, given that at 12 weeks we are still seeing downward trending.” 

Tildacerfont would be a nonsteroid treatment for patients who rely on high doses of glucocorticoids to manage their disease. These steroids aim to replace the cortisol CAH patients don’t produce as well as tamp down on the excess androgens they do make.

To read more about the results, click here.

Are you a member of the CAH community? Share your stories, thoughts, and hopes with the Patient Worthy community!

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