Study of SPR001 in Adults With Classic Congenital Adrenal Hyperplasia

This is a 6-week, multiple-dose, dose escalation study of SPR001 for the treatment of adults
with classic CAH. After screening, eligible patients will be enrolled into a 6-week treatment
period followed by a 4-week washout/safety follow-up period.

It is initially planned that up to approximately 18 patients in 2 dose cohorts will be
enrolled. Additional patients or dose groups may be considered based upon specific safety,
PK/PD, and/or efficacy findings, or if an active dose has not yet been reached.

SPR001 will be administered as an oral daily dose. Patients will undergo titration of SPR001
through three escalating dosage strengths at 2-week intervals. Patients will have overnight
PK/PD assessments performed at baseline, which include an pre-dose overnight assessment and a
post-dose overnight assessment for PK/PD following administration of the first dose. At the
end of each 2-week dosing period, patients will return for single overnight visits for
steady-state PK/PD assessments.

A follow-up outpatient visit will occur 30 days after their last dose.

Inclusion Criteria:

- Male and female patients age 18 or older.

- Documented diagnosis of classic CAH due to 21-hydroxylase deficiency

- Elevated 17-OHP at screening

- On a stable glucocorticoid replacement regimen for a minimum of 30 days

Exclusion Criteria:

- Clinically significant unstable medical condition, illness, or chronic disease

- Clinically significant psychiatric disorder.

- Clinically significant abnormal laboratory finding or assessment

- History of bilateral adrenalectomy or hypopituitarism

- Pregnant or nursing females

- Use of any other investigational drug within 30 days

- Unable to understand and comply with the study procedures, understand the risks,
and/or unwilling to provide written informed consent.