MODEYSO generated an established safety profile1

The safety of MODEYSO was demonstrated across 
pediatric and adult patients with glioma1

The safety of MODEYSO was evaluated in an integrated analysis1

The safety of MODEYSO was evaluated in 376 adult and pediatric patients with glioma across 4 open-label clinical studies. Patients in the safety cohort received MODEYSO once weekly at a dose of 625 mg or scaled by body weight for patients weighing <52.5 kg.1

See additional details about the integrated analysis

POPULATION DEMOGRAPHICS

The safety cohort included 154 pediatric patients and 222 adult patients with glioma who had varying disease characteristics1

BASELINE CHARACTERISTICS IN SAFETY COHORT (N=376)1
  • Median age: 23 years
    • Range: 3 to 80 years
    • 41% of patients under 18 years
  • 52% female, 48% male
  • Race
    • 74% White
    • 10% unknown or not reported
    • 9% Black/African American
    • 4% Asian
    • 2.9% other racial groups
  • Ethnicity
    • 13% Hispanic/Latino
  • KPS/LPS score
    • 66% had 80 to 100
    • 27% had 60 to 70
    • 7% had <60
  • Primary tumor location
    • Midline region: 91%
    • Non-midline region: 9%
  • 33% had DIPG
  • 30% presented with multifocal disease
  • 79% had a known H3 K27M mutation
  • 75% had experienced disease recurrence
  • 35% had treatment exposure for 6 months, and 17% were exposed for 1 year

DIPG, diffuse intrinsic pontine glioma; H3, histone 3; KPS/LPS, Karnofsky Performance Status or Lansky Performance Status, depending on age.

SAFETY PROFILE

Serious adverse reactions occurred in 33% of patients who received MODEYSO. Serious adverse reactions in >2% of patients included hydrocephalus (5%), vomiting (4.3%), headache (3.2%), seizure (2.4%), and muscular weakness (2.1%).1

ADVERSE REACTIONS IN ≥10% OF PATIENTS WITH GLIOMA WHO RECEIVED MODEYSO1

Adverse Reaction
All Grades (%)
Grade 3 or 4 (%)
General Disorders
Fatigue*
34
3.2
Gait disturbance
16
3.7
Nervous System Disorders
Headache†
32
4.3
Cranial nerve disorders‡
16
1.3
Hemiparesis
15
4.5
Dysarthria
13
2.7
Dizziness
13
0.5
Ataxia
10
1.3
Gastrointestinal Disorders
Vomiting
24
2.7
Nausea
24
0.8
Dysphagia
13
2.1
Constipation
11
0
Musculoskeletal and Connective Tissue Disorders
Musculoskeletal pain§
20
2.9
Muscular weakness
13
4.5
Metabolism and Nutrition Disorders
Hyperglycemia
12
0.8
Infections and Infestations
Rash||
11
0.8

*Includes asthenia.1

†Includes head discomfort and sinus headache.1

‡Includes accessory nerve disorder, auditory nerve disorder, facial nerve disorder, facial paralysis, facial paresis, glossopharyngeal nerve disorder, hypoglossal nerve disorder, IIIrd nerve disorder, IIIrd nerve paralysis, IVth nerve disorder, IVth nerve paralysis, tongue paralysis, trigeminal nerve disorder, trigeminal neuralgia, VIth nerve disorder, VIth nerve paralysis, and VIth nerve paresis.1

§Includes back pain, pain in extremity, arthralgia, neck pain, noncardiac chest pain, myalgia, bone pain, musculoskeletal chest pain, musculoskeletal stiffness, and spinal pain.1

||Includes dermatitis, dermatitis acneiform, dermatitis bullous, eczema, erythema multiforme, rash erythematous, rash macular, rash maculopapular, rash papular, rash pruritic, and rash pustular.1

The most common adverse reactions (≥20%) experienced with MODEYSO were fatigue, headache, vomiting, nausea, and musculoskeletal pain.1

Most adverse events were grade 1 or 2 in severity1

Dosage interruptions of MODEYSO due to an adverse reaction occurred in 6% of patients. Adverse reactions that required dosage interruption in >1 patient included increased ALT, increased AST, decreased lymphocyte count, muscular weakness, and aspiration pneumonia.1

Dose reductions of MODEYSO due to an adverse reaction occurred in 2.7% of patients. Adverse reactions that required dose reductions in >1 patient included decreased neutrophil count and increased ALT.1

Permanent discontinuation due to an adverse reaction occurred in 2.1% of patients1

Adverse reactions that resulted in permanent discontinuation of MODEYSO in >1 patient included confusional state.1

The most common (≥2%) grade 3 or 4 laboratory abnormalities were decreased lymphocytes, decreased calcium, and increased ALT.1

Laboratory Abnormality¶,#
All Grades (%)
Grade 3 or 4 (%)
Chemistry
ALT increased
28
2.4
AST increased
22
0.9
Calcium decreased
20
2.7
Sodium decreased
14
0.3
Potassium decreased
13
0.3
Glucose decreased
11
0
ALP increased
11
0.3
Hematology
Hemoglobin decreased
25
0.6
Neutrophils decreased
24
1.5
Lymphocytes decreased
19
7

¶Severity as defined by the National Cancer Institute CTCAE Version 5.0.1

#The denominator for each laboratory parameter is based on the number of patients with a baseline and post-treatment laboratory value available, which ranged from 325 to 330 patients.1

ALP, alkaline phosphatase; ALT, alanine aminotransferase; AST, aspartate aminotransferase; CTCAE, Common Terminology Criteria for Adverse Events.

Discover oral dosing1

References: 1. MODEYSO™. Package insert. Chimerix, Inc; 2025. 2. Arrillaga-Romany I, Gardner SL, Odia Y, et al. ONC201 (dordaviprone) in recurrent H3 K27M–mutant diffuse midline glioma. J Clin Oncol. 2024;42(13):1542-1552. doi:10.1200/JCO.23.01134