Wednesday, Spruce Biosciences Inc ( SPRB ) released topline results from its CAHmelia-203 Phase 2b study of tildacerfont in adult classic congenital adrenal hyperplasia (CAH) and its CAHptain-205 study of tildacerfont in pediatric classic CAH.

CAH refers to a group of genetic disorders that involve the adrenal glands, a pair of small organs located above the kidneys.

The CAHmelia-203 study did not achieve the primary efficacy endpoint of the assessment of dose response for the change in A4 from baseline to week 12.

200mg QD of tildacerfont demonstrated a placebo-adjusted reduction from baseline in A4 of -2.6% at week 12 with a non-significant p-value.

 Compliance with study medication and GC was low, with approximately 50% of patients reporting 80% or greater compliance, resulting in lower-than-expected tildacerfont exposure.

 Tildacerfont was generally safe and well tolerated at all doses with no treatment-related serious adverse events (SAEs). Most adverse events were reported as mild to moderate.s

CAHptain-205 study enrolled 30 children.

Tildacerfont was generally safe and well tolerated at all dose ranges with no treatment-related SAEs reported.

 Preliminary pharmacokinetic analysis suggests that tildacerfont is cleared more rapidly in children than in adult CAH patients.

 73% of all patients (22 of 30) met the efficacy endpoint of A4 or GC reduction from baseline at 12 weeks of treatment with tildacerfont.

 70% of patients with elevated baseline A4 values (16 of 23 patients) demonstrated an A4 reduction at week 4.

 The company says data from the CAHptain-205 study supports further dose-ranging across additional cohorts.

The company is also conducting CAHmelia-204 Phase 2b study assessing glucocorticoid (GC) reduction, a potentially registrational endpoint in adult CAH patients on supraphysiologic GC doses with normal or near normal levels of A4. Topline results are expected in Q3 2024.

In January, Spruce Biosciences ( SPRB ) said it completed enrollment in its CAHmelia-204 trial, with 100 patients exceeding the target enrollment of 90.

The company says resource prioritization and cost reductions, including termination of the CAHmelia-203 trial and a reduction in force of approximately 21%, will extend the cash runway through the end of 2025, including beyond anticipated topline results from CAHmelia-204 and additional dose-ranging data from CAHptain-205.

Price Action: SPRB shares are down 77.80% at $1.15 during the premarket session on the last check Thursday.