Product Description
The drug, GDC-0084, is being developed by the Australian biotech Kazia Therapeutics Limited (ASX: KZA; NASDAQ: KZIA) as an alternative treatment option for the two thirds of patients resistant to the mainstay of current pharmacological treatment for glioblastoma, temozolomide. GDC-0084 is being developed as an inhibitor of the PI3K signaling pathway, which is implicated in most cases of glioblastoma. (Sourced from: https://www.kaziatherapeutics.com/site/blog/novel-australian-drug-gdc-0084-for-glioblastoma-brain-cancer-starts-phase-ii-clinical-trial-in-us)
Mechanisms of Action: PI3K Inhibitor, mTOR Inhibitor
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: Fast Track - Brain Cancer|Oncology Solid Tumor UnspecifiedFast Track - GlioblastomaOrphan Drug - Brain CancerOrphan Drug - Glioma *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Company: Kazia Therapeutics
Company Location: Asia Pacific
Company Founding Year: 1994
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: Australia, Canada, France, Germany, Switzerland, United Kingdom, United States
Active Clinical Trial Count: 9
Recent & Upcoming Milestones
- Clinical Outcomes Expected - Kazia Therapeutics announced they will present P1 Breast Cancer results in YE26 for Paxalisib
- Clinical Outcomes Reported - Kazia Therapeutics presented P1 Triple Negative Breast Cancer results on 2026-01-27 for Paxalisib
- Clinical Outcomes Reported - Kazia Therapeutics announced they will present P1 Triple Negative Breast Cancer results in 1Q26 for Paxalisib
Highest Development Phases
Phase 3: Glioblastoma
Phase 2: Brain Stem Cancer|Breast Cancer|Central Nervous System Cancer|Glioma|Hemimegalencephaly|Lung Cancer|Lymphoma, Non-Hodgkin|Primary Central Nervous System Lymphoma|Rhabdoid Tumor|Tuberous Sclerosis
Phase 1: Brain Cancer|Meningeal Carcinomatosis
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
NCT03970447 |
GBM AGILE | P3 |
Recruiting |
Glioblastoma |
2028-06-01 |
42% |
2026-04-04 |
Patient Enrollment|Primary Endpoints|Treatments |
NCT03994796 |
A071701 | P2 |
Active, not recruiting |
Central Nervous System Cancer|Brain Stem Cancer|Breast Cancer|Lung Cancer |
2026-10-01 |
12% |
2026-04-14 |
|
NCT04192981 |
NCT04192981 | P1 |
Active, not recruiting |
Brain Cancer|Meningeal Carcinomatosis |
2026-12-01 |
2025-04-18 |
Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments |
|
NCT07447076 |
ATRT | P2 |
Not yet recruiting |
Rhabdoid Tumor |
2035-12-31 |
2026-03-04 |
Primary Endpoints |
|
NCT07391215 |
5G-PEARL | P2 |
Recruiting |
Glioblastoma|Glioma |
2029-01-19 |
12% |
2026-02-06 |
Primary Endpoints|Treatments |
NCT07287202 |
SVG103-001 | P2 |
Not yet recruiting |
Hemimegalencephaly|Tuberous Sclerosis |
2026-12-01 |
2025-12-18 |
Primary Endpoints|Treatments |
|
NCT04906096 |
NCT04906096 | P2 |
Recruiting |
Lymphoma, Non-Hodgkin|Primary Central Nervous System Lymphoma |
2026-12-01 |
2025-06-27 |
Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments |
|
ACTRN12624001340527 |
PaxPlus-ABC | P1 |
Recruiting |
Breast Cancer |
2026-08-10 |
2026-01-17 |
Patient Enrollment|Treatments |
|
NCT06208657 |
OPTIMISE | P2 |
Recruiting |
Central Nervous System Cancer|Primary Central Nervous System Lymphoma|Brain Stem Cancer |
2030-12-01 |
12% |
2025-11-29 |
Primary Endpoints |