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Ref Type Journal Article
PMID (40939237)
Authors Schram AM, Takebe N, Chen A, Zhou Q, Iasonos A, Silber J, Reynolds M, Hussain S, Gavriliuc M, Smyth LM, Garrison D, Dumbrava EE
Title A phase I study of AZD8186 in combination with docetaxel in patients with PTEN-mutated or PIK3CB-mutated advanced solid tumors.
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Abstract Text Loss of PTEN activity is common in solid tumors and promotes cancer growth through activation of the PI3K pathway. PTEN-deficient tumors have increased dependence on PI3Kβ and are sensitive to PI3Kβ inhibition in preclinical models. Efficacy is further enhanced by the addition of taxane chemotherapy. We conducted a phase I trial of AZD8186, a small molecule inhibitor of PI3Kβ and PI3Kδ, in combination with docetaxel (Taxotere) (NCI 10131; NCT03218826).Patients with advanced PTEN- or PIK3CB-mutated solid tumors identified through local testing were eligible. Treatment included docetaxel intravenously every 21 days and AZD8186 orally twice daily, 5 days on and 2 days off. Primary objectives were safety, tolerability, and maximum tolerated dose (MTD) as determined by a 3 + 3 dose-escalation design. Secondary objectives included assessment of antitumor activity.Twenty-three patients were enrolled with 11 distinct tumor types across 5 dose levels. Clinically significant neutropenia led to dose-level adjustment and the addition of prophylactic growth factor. The MTD was not reached and AZD8186 120 mg twice daily with docetaxel 75 mg/m2 was named the recommended phase II dose. The most common treatment-emergent adverse events (TEAEs) were anemia (57%), diarrhea (43%), and fatigue (43%). The most common grade ≥3 TEAE was neutropenia (30%). One patient with docetaxel-naive prostate cancer had a prolonged partial response (overall response ratio 5.6%); clinical benefit rate was 22.2%.The combination of AZD8186 and docetaxel was generally well tolerated, with the exception of neutropenia, which was effectively managed with the use of growth factor. Limited clinical activity was observed.

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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
PTEN inact mut Advanced Solid Tumor no benefit AZD8186 + Docetaxel Phase I Actionable In a Phase I trial, treatment with the combination of AZD8186 and Taxotere (docetaxel) was generally well tolerated but demonstrated limited efficacy with an overall response rate of 5.6% (1/18), clinical benefit rate of 22.2% (4/18), and median progression-free survival of 3 months in patients with advanced solid tumors harboring PTEN inactivating mutations (n=21) or PIK3CB activating mutations (n=3) (PMID: 40939237; NCT03218826). 40939237