In AMPECT, 47% of patients received FYARRO for ≥6 months and 21% for
>1 year

  • The median number of cycles was 8.5 at time of primary analysis2
  • The safety profile of FYARRO was consistent with other mTOR inhibitors, with no new safety signals1
  • 35% of patients required a dose reduction due to an adverse reaction
  • Three patients (9%) discontinued treatment due to an adverse reaction (pneumonitis, anemia, and noninfective cystitis)

Most adverse reactions were mild or moderate in severity (Grade 1 or 2) and manageable1

  • The most common adverse reactions (≥30%) were stomatitis, fatigue, rash, infection, nausea, edema, diarrhea, musculoskeletal pain, decreased weight, decreased appetite, cough, vomiting, and dysgeusia
  • The most common laboratory Grade 3 to 4 abnormalities (≥6%) were decreased lymphocytes, increased glucose, decreased potassium, decreased phosphate, decreased hemoglobin, and increased lipase

Serious adverse reactions (SARs) occurred in 14 (41%) patients who received FYARRO

  • SARs in >5% of patients included infection, abdominal pain, dehydration, and upper gastrointestinal hemorrhage
  • Fatal adverse reactions occurred in 1 (2.9%) patient who received FYARRO and experienced upper gastrointestinal hemorrhage*

*Unrelated to treatment.1

AMPECT=Advanced Malignant PEComa Trial; mTOR=mechanistic target of rapamycin.

AMPECT: Adverse reactions in ≥10% of patients with PEComa who received FYARRO

ADVERSE REACTION FYARRO (N=34)
ALL GRADES (%) GRADES 3 TO 4* (%)
Gastrointestinal

GASTROINTESTINAL

Stomatitisa 79 18
Nausea 50 0
Diarrheab 47 2.9
Vomiting 32 2.9
Abdominal painc 29 6
Constipation 24 2.9
Dry mouth 15 0
Hemorrhoids 12 0
General Disorders

GENERAL DISORDERS

Fatigue 68 2.9
Edemad 50 2.9
Pyrexia 24 0
Skin & Subcutaneous Tissue Disorders

SKIN &
SUBCUTANEOUS
TISSUE DISORDERS

Rashe 68 0
Alopecia 24 0
Pruritus 18 0
Dry skin 12 0
Nail disorder 12 0
Infections

INFECTIONS

Infectionsf 59 12
Metabolism & Nutrition

METABOLISM & NUTRITION

Decreased appetite 44 0
Dehydration 15 6
Nervous System

NERVOUS SYSTEM

Dysgeusia 32 0
Headache 29 0
Peripheral neuropathyg 15 0
Dizzinessh 12 0
Investigations

INVESTIGATIONS

Weight decreased 47 0
Musculoskeletal & Connective Tissue Disorders

MUSCULOSKELETAL
& CONNECTIVE
TISSUE DISORDERS

Musculoskeletal paini 47 2.9
Respiratory, Thoracic, & Mediastinal Disorders

RESPIRATORY,
THORACIC, &
MEDIASTINAL
DISORDERS

Coughj 35 0
Pneumonitis 18 0
Dyspneak 12 0
Vascular Disorders

VASCULAR
DISORDERS

Hypertension 29 2.9
Hemorrhagel 24 2.9
Psychiatric Disorders

PSYCHIATRIC
DISORDERS

Insomnia 21 2.9
Eye Disorders

EYE DISORDERS

Vision blurred 12 0

Grading according to NCI Common Terminology Criteria for Adverse Events Version 4.03.

a

Includes stomatitis, aphthous ulcer, mouth ulceration, esophageal ulcer.

b

Includes diarrhea and enteritis.

c

Includes abdominal pain, abdominal pain upper, and epigastric discomfort.

d

Includes face edema, generalized edema, edema, edema peripheral, and periorbital edema.

e

Includes dermatitis acneiform, palmar-plantar erythrodysesthesia syndrome, rash, rash erythematous, rash macular, rash maculopapular, rash papular, rash pruritic, and skin exfoliation.

f

Includes all reported infections, including but not limited to, upper respiratory tract infection, urinary tract infection, sinusitis, skin infection, folliculitis, nasopharyngitis, pharyngitis, pharyngitis streptococcal, pneumonia, vaginal infection.

g

Includes dysesthesia, hypoesthesia, neuropathy peripheral, paresthesia, and peripheral sensory neuropathy.

h

Includes dizziness, dizziness postural, and vertigo.

i

Includes arthralgia, back pain, musculoskeletal chest pain, myalgia, neck pain, noncardiac chest pain, pain in extremity.

j

Includes cough, productive cough, and upper-airway cough syndrome.

k

Includes dyspnea and dyspnea exertional.

l

Includes epistaxis, hemorrhoidal hemorrhage, mouth hemorrhage, post procedural hemorrhage, and upper GI hemorrhage. Includes 1 fatal adverse reaction of upper GI hemorrhage (unrelated to treatment).1

*No Grade 4 reactions were reported.

AMPECT=Advanced Malignant PEComa Trial; GI=gastrointestinal; PEComa=perivascular epithelioid cell tumor.

Laboratory abnormalities occurring in
≥10% of patients

LABORATORY ABNORMALITY* FYARRO (N=34)
ALL GRADES (%) GRADES 3 TO 4 (%)
Hematology

HEMATOLOGY

Decreased lymphocytes 82 21
Decreased hemoglobin 68 6
Decreased leukocytes 41 0
Decreased neutrophils 35 0
Decreased platelets 35 0
Chemistry

CHEMISTRY

Increased creatinine 82 0
Increased triglycerides 52 0
Increased cholesterol 48 3
Increased alanine aminotransferase
(ALT)
47

2.9

Decreased potassium 44 12
Decreased magnesium 42 0
Decreased albumin 35 2.9
Increased aspartate transaminase (AST) 32 2.9
Increased alkaline phosphatase 29 0
Decreased sodium 24 2.9
Decreased calcium 15 0
Decreased glucose 15 0
Decreased phosphate 15 9
Increased lipase 12 6
Increased glucose 12 12
Increased sodium 12 0

*

Grading according to NCI Common Terminology Criteria for Adverse Events Version 4.03.

The denominator used to calculate the rate varied from 33 to 34 based on the number of patients with a baseline value and at least 1 posttreatment value.

References: 1. Wagner AJ, Ravi V, Riedel RF, et al. nab-Sirolimus for patients with malignant perivascular epithelioid cell tumors. J Clin Oncol. 2021;39(33):3660-3670. doi:10.1200/JCO.21.01728 2. Data on file. Aadi Bioscience, Inc.; 2021.