Drugs / Paxalisib
last change Aug 2026 re-read 9 days ago

Paxalisib

also known as GDC-0084 · G-02441729 · RG 7666 · Sim-0395

Small molecule targets MTOR via inhibition

Developed for
glioblastoma · metastatic malignant neoplasm in the brain · adult glioblastoma · atypical teratoid rhabdoid tumor · brain cancer · breast cancer · central nervous system neoplasm · childhood malignant neoplasm
+6 more · diffuse midline glioma, H3 K27M-mutant · grade III glioma · neoplastic meningitis · non-Hodgkin lymphoma · primary central nervous system lymphoma · refractory malignant neoplasm
Investigated by
Kazia Therapeutics Limited · Genentech, Inc. · Alliance for Clinical Trials in Oncology · AstraZeneca · Australian & New Zealand Children's Haematology/Oncology Group · Bayer
+38 more · Biohaven Pharmaceuticals, Inc. · Bristol-Myers Squibb · C17 Council · CV Biomanufacturing · Cambridge University Hospitals NHS Foundation Trust · Cancer Research UK · Dana-Farber Cancer Institute · Eli Lilly and Company · Global Coalition for Adaptive Research · Institute of Cancer Research, United Kingdom · Jazz Pharmaceuticals · Kintara Therapeutics, Inc. · Knoa Pharma LLC · Lakshmi Nayak, MD · Medical Research Future Fund · Memorial Sloan Kettering Cancer Center · Minderoo Foundation · Mirati Therapeutics Inc. · Mithil Prasad Foundation · National Cancer Institute (NCI) · National Institute of Neurological Disorders and Stroke (NINDS) · Neeve Kolte and Brave Ronil Foundation · Pacific Pediatric Neuro-Oncology Consortium · Polaris Group · Quotient Sciences · Rally Foundation · Royal Marsden NHS Foundation Trust · Sabine Mueller, MD, PhD · St. Jude Children's Research Hospital · Stand Up To Cancer · Storm the Heavens Fund · The ChadTough Defeat DIPG Foundation · The Hospital for Sick Children · Tough2gether Foundation · University of California, San Francisco · Vigeo Therapeutics, Inc. · Weill Medical College of Cornell University · Will Meeker Foundation

Trials 14

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 14 trials
Phase 1 NCT05012670 Aug 2021 → Mar 2023 — Kazia Therapeutics Limited Completed No outcome recorded
Phase 1 NCT04192981 Dec 2019 → Dec 2026 expected metastatic malignant neoplasm in the brain, neoplastic meningitis Memorial Sloan Kettering Cancer Center Active not recruiting No outcome recorded
Phase 1 NCT03696355 Nov 2018 → Apr 2021 central nervous system neoplasm St. Jude Children's Research Hospital Completed No outcome recorded
Phase 1 NCT01547546 Apr 2012 → Jan 2015 glioblastoma Genentech, Inc. Completed No outcome recorded
Phase 1/22 trials
Phase 1/2 NCT07391215 Jan 2026 → Jan 2029 expected glioblastoma Institute of Cancer Research, United Kingdom Recruiting No outcome recorded
Phase 1/2 NCT06208657 Jul 2024 → Dec 2030 expected brain cancer, childhood malignant neoplasm, refractory malignant neoplasm Australian & New Zealand Children's Haematology/Oncology Group Recruiting No outcome recorded
Phase 27 trials
Phase 2 NCT07447076 Jun 2026 → Dec 2035 expected atypical teratoid rhabdoid tumor Sabine Mueller, MD, PhD Recruiting No outcome recorded
Phase 2 NCT05183204 Feb 2022 → Aug 2026 glioblastoma Weill Medical College of Cornell University Completed No outcome recorded
Phase 2 NCT05009992 Oct 2021 → Dec 2027 expected diffuse midline glioma, H3 K27M-mutant, grade III glioma University of California, San Francisco Recruiting No outcome recorded
Phase 2 NCT04906096 Jun 2021 → Dec 2026 expected non-Hodgkin lymphoma, primary central nervous system lymphoma Lakshmi Nayak, MD Recruiting No outcome recorded
Phase 2 NCT03994796 Oct 2019 → Nov 2025 overdue NTRK fusion positive cancer, metastatic malignant neoplasm in the brain Alliance for Clinical Trials in Oncology Active not recruiting No outcome recorded
Phase 2 NCT03765983 Feb 2019 → Apr 2024 breast cancer Dana-Farber Cancer Institute Terminated No outcome recorded
Phase 2 NCT03522298 May 2018 → Mar 2023 adult glioblastoma Kazia Therapeutics Limited Completed No outcome recorded
Phase 2/31 trial · 1 missed
Phase 2/3 NCT03970447 Jul 2019 → Jun 2028 expected glioblastoma Global Coalition for Adaptive Research Recruiting Missed primary

Evidence & citations 9 cited values

Every value below carries the sentence it was read from. 17 sources stand behind the page.

FieldValueCited text
Known as Paxalisib ClinicalTrials.gov intervention name — accepted as the source's own label NCT05009992 ↗
9

NCT03994796 ↗

NCT03522298 ↗

NCT04906096 ↗

NCT07447076 ↗

NCT05012670 ↗

NCT03970447 ↗

NCT07391215 ↗

NCT06208657 ↗

NCT05183204 ↗

Known as G-02441729 ChEMBL registry synonym — accepted as the source's own label CHEMBL3813842 ↗
Known as GDC-0084 “Patients will be dosed orally with paxalisib (GDC-0084) capsules (15-mg each) at the dose and schedule to which they are assigned.” NCT03522298 ↗
4

NCT01547546 ↗

NCT03696355 ↗

NCT03765983 ↗

NCT04192981 ↗

Known as RG 7666 ChEMBL registry synonym — accepted as the source's own label CHEMBL3813842 ↗
Known as Sim-0395 ChEMBL registry synonym — accepted as the source's own label CHEMBL3813842 ↗
Action Inhibit “inhibitor of PI3K and mTOR” PMID 32269051 ↗ Apr 2020
Modality Small molecule “small-molecule inhibitor” PMID 32269051 ↗ Apr 2020
1

CHEMBL3813842 ↗

Route Oral “GDC-0084 was administered orally, once daily” PMID 31937616 ↗ Jan 2020
Target MTOR “inhibitor of PI3K and mTOR” PMID 32269051 ↗ Apr 2020