Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency

Inclusion Criteria:

• Subjects must be naïve to treatment and prepubertal

• Subjects must have a maximal GH response of < 10 ng/mL from 2 prior GH stimulationtests conducted within the preceding 12 months

• Impaired height defined as ≥ 2.0 standard deviations (SDs) below the mean height forchronological age and sex

• Morning or random cortisol level of ≥ 7.0 μg/dL

• ≥ 3.0 years and age ≤ 10.0 years for girls and ≤ 11.0 years for boys

• Baseline height velocity (HV) based on ≥ 6 months of growth assessments < 25thpercentile for age and sex

• Bone Age delay of ≥ 12 months compared to the chronological age

• In girls, have genetic testing results to rule out Turner syndrome. If SHOX genetictesting results are available, they need to be negative.

• Have normal thyroid function. Subjects diagnosed with hypothyroidism must havedocumented successful treatment for at least 3 months prior to Day 1

• Baseline IGF-1 standard deviation score (SDS) ≤ -1.0

Exclusion Criteria:

• Any medical or genetic condition which, in the opinion of the Investigator orMedical Monitor (MM), can be an independent cause of short stature and/or limit theresponse to exogenous growth factor treatment.

• Arm span to height ratio > 2 SDs below the mean for age and sex

• A medical or genetic condition that, in the opinion of the Investigator and/or MM,adds unwarranted risk to use of LUM-201

• Use of any medication that, in the opinion of the Investigator and/or MM, canindependently cause short stature or limit the response to exogenous growth factors

• Current inflammatory diseases requiring systemic corticosteroid treatment for > 2consecutive weeks within the last 3 months prior to the Screening Visit

• Use of hormone replacement therapy for any hormone deficiency other than thyroiddeficiency

• Any ECG at the Screening Visit noted to have a clinically significant abnormality,as confirmed by the MM

• Any subjects suspected of having past or present intracranial tumor growth asconfirmed by brain imaging prior to the Screening or Day 1 Visit

• Any subject suspected of having intracranial hypertension (IH) as confirmed byfundoscopy and other assessments

• Any subject with serum alanine transaminase (ALT), aspartate transaminase (AST), ortotal bilirubin > upper limit of normal (ULN)

• Suspicion of absent pituitary function as evidenced by a maximal stimulated GH ≤ 3.0ng/mL on any prior standard of care GH stimulation test completed within 12 months

• Body weight ≤ 14.0 kg

• BMI < -2 or > +2 SDs for age and sex based on WHO standards

• Birth weight for gestational age < 3rd percentile based on WHO standards

• Treatment with medications known to be moderate or strong inhibitors or stronginducers of cytochrome P450 (CYP) 3A/4

• History of spinal, cranial, or total body irradiation

• Attention deficit hyperactivity disorder (ADHD) diagnosis