Novogen's Drug in the FDA Approval Process
This section highlights FDA-related milestones and regulatory updates for drugs developed by Novogen (KZIA).
Over the past two years, Novogen has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
Paxalisib. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Paxalisib FDA Regulatory Timeline and Events
Paxalisib is a drug developed by Novogen for the following indication: Atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Paxalisib
- Announced Date:
- January 27, 2026
- Indication:
- Atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer
Announcement
Kazia Therapeutics provided a clinical update from its ongoing Phase 1b study evaluating paxalisib in combination with pembrolizumab and chemotherapy in patients with late-stage (Stage IV), metastatic triple-negative breast cancer (TNBC).
AI Summary
Kazia Therapeutics reported interim results from its Phase 1b study of paxalisib combined with pembrolizumab and chemotherapy in patients with late-stage (Stage IV) metastatic triple-negative breast cancer (TNBC). Three patients have shown meaningful clinical benefit: two trial participants achieved partial responses and one patient treated under expanded access had a confirmed complete metabolic response. Responses were seen in patients with visceral and multi-organ disease. Median time on treatment so far is about 6.1 months, and all patients remain on therapy. Paxalisib at 30 mg daily has been generally well tolerated; roughly 75% of adverse events were judged unlikely or unrelated to the drug, most drug-related events were mild to moderate, one case of Grade 1 hyperglycemia was noted, and two serious adverse events were deemed unrelated.
The trial is a multi-center, open-label, randomized study started in June 2025, testing paxalisib with either pembrolizumab plus chemotherapy or olaparib. Eligibility for the pembrolizumab arm required PD-L1–positive disease (CPS ≥10). Kazia plans additional site activations through 2026, aims to enroll about 12 TNBC patients by year-end 2026, and expects a topline readout in early 2027. The oral, once-daily dosing may offer a convenient option if benefits are confirmed.
Read Announcement- Drug:
- Paxalisib
- Announced Date:
- December 10, 2025
- Indication:
- Atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer
Announcement
Kazia Therapeutics Limited announced new data from two presentations at the 2025 San Antonio Breast Cancer Symposium (SABCS) providing compelling mechanistic and early clinical evidence supporting the activity of paxalisib, the Company's brain-penetrant dual PI3K/mTOR inhibitor, across both HER2-positive metastatic breast cancer and triple-negative breast cancer (TNBC).
AI Summary
Kazia Therapeutics reported new data at the 2025 San Antonio Breast Cancer Symposium showing compelling mechanistic and early clinical activity for paxalisib, a brain-penetrant dual PI3K/mTOR inhibitor, in both HER2-positive metastatic breast cancer and triple-negative breast cancer (TNBC).
In HER2+ ex‑vivo studies, paxalisib cut single circulating tumor cells (CTCs) by 42% and CTC clusters by 78%, disrupting aggressive Vimentin+/Snail+/NRF2+ cluster biology. Treated samples showed activation of cytotoxic, interferon, chemokine and inflammatory pathways, consistent with immune reinvigoration.
Early TNBC Phase 1b data from the first patient showed a 76% tumor volume shrinkage with large reductions in CTC clusters, epigenetic reprogramming of CTCs toward less aggressive states, and decreased exhausted CD8 T cells alongside restored cytotoxic and antigen‑presentation pathways. When paxalisib was paused, CTC clusters rebounded; restarting paxalisib restored suppression.
These findings suggest paxalisib may both dismantle metastasis‑initiating CTCs and revive anti-tumor immunity, supporting combinations with immunotherapy, PARP inhibitors and chemotherapy across breast cancer subtypes.
Read Announcement- Drug:
- Paxalisib
- Announced Date:
- November 18, 2025
- Indication:
- Atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer
Announcement
Kazia Therapeutics Limited announced that a patient with stage IV triple-negative breast cancer (TNBC) treated under an FDA-authorized single-patient expanded access protocol combining paxalisib with pembrolizumab (Keytruda®) and standard chemotherapy has achieved an initial immune-complete response (iCR) per iRECIST criteria.
AI Summary
Kazia Therapeutics announced that a patient with stage IV triple-negative breast cancer (TNBC) treated under an FDA-authorized single-patient expanded access protocol combining paxalisib with pembrolizumab (Keytruda®) and standard chemotherapy has achieved an initial immune-complete response (iCR) per iRECIST criteria. After three weeks of treatment the patient showed an 86% reduction in tumor burden, and a PET/CT scan after about three months demonstrated complete metabolic resolution of all previously identified lesions. The patient remains on therapy and under active clinical monitoring, and a follow-up scan will be done to confirm the initial iCR under immune-based response guidelines.
Complete responses in stage IV metastatic TNBC are very uncommon across therapies, so this radiologic iCR is an unusual and encouraging result. Although it is a single expanded-access case and requires confirmation, the depth of response suggests the combination may have meaningful anti-tumor activity and supports further study in clinical trials.
Read Announcement- Drug:
- Paxalisib
- Announced Date:
- October 27, 2025
- Indication:
- Atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer
Announcement
Kazia Therapeutics Limited announced its intention to request and hold a follow-up Type C meeting with the U.S. Food & Drug Administration (FDA) to discuss overall survival (OS) findings in newly diagnosed glioblastoma (GBM) patients treated with paxalisib and to seek agency feedback on a potential regulatory pathway aligned with the FDA Oncology Center of Excellence's Project FrontRunner initiative.
AI Summary
Kazia Therapeutics plans to request a follow-up Type C meeting with the FDA. The goal is to discuss overall survival (OS) results in newly diagnosed glioblastoma patients treated with their drug paxalisib.
Kazia will seek FDA feedback on a regulatory pathway aligned with the FDA Oncology Center of Excellence’s Project FrontRunner initiative. This program encourages earlier approval for cancer drugs in front-line settings.
Dr. John Friend, Kazia’s CEO, said the company will propose starting a post-approval, randomized Phase 3 confirmatory study before filing a new drug application. This approach matches the FDA’s emphasis on OS as the most meaningful endpoint for patients and clinicians.
At the meeting, Kazia will present survival data, safety findings, and its planned trial design. The company hopes to secure conditional approval for paxalisib as a front-line GBM treatment under Project FrontRunner’s modernized framework.
Read Announcement- Drug:
- Paxalisib
- Announced Date:
- October 2, 2025
- Indication:
- Atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer
Announcement
Kazia Therapeutics Limited today reported a substantial reduction in tumor burden from a single-patient expanded access case in triple-negative breast cancer (TNBC) treated with a combination regimen that included the Company's investigational pan-PI3K/mTOR inhibitor, paxalisib.
AI Summary
Kazia Therapeutics Limited reported that a single patient with triple-negative breast cancer (TNBC) had an 86% reduction in tumor burden after three weeks of a combination treatment that included the company’s investigational pan-PI3K/mTOR inhibitor, paxalisib, along with immunotherapy and chemotherapy.
The patient, a woman in her forties, was first diagnosed in April 2023 and underwent chemotherapy, immunotherapy, surgery, and radiation. About two years later, her cancer spread to bone and lungs. Under an expanded access protocol, she received the new combination and showed rapid, major tumor shrinkage.
These results mirror the design of Kazia’s ongoing Phase 1b study in advanced breast cancer, which combines paxalisib with Keytruda® and chemotherapy. Company leaders said the speed and size of the response support further testing of paxalisib with immune checkpoint inhibitors.
Read Announcement- Drug:
- Paxalisib
- Announced Date:
- July 9, 2025
- Indication:
- Atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer
Announcement
Kazia Therapeutics announce preliminary results from the first patient in its Phase 1b trial evaluating a combination regimen of Paxalisib, pembrolizumab (Keytruda®), and standard chemotherapy after completing Cycle 1 (21 days) of dosing.
AI Summary
Kazia Therapeutics has announced promising early results from its Phase 1b trial that evaluates a combination of Paxalisib, pembrolizumab (Keytruda®), and standard chemotherapy. In the first patient, a 61-year-old woman with metastatic triple-negative breast cancer, the treatment showed over a 50% decrease in circulating tumor cells (CTCs) and a significant reduction in CTC clusters after just 21 days (Cycle 1).
These preliminary findings suggest that the combination therapy may quickly lower the markers linked to cancer spread, which is important since CTC clusters are known to promote metastasis more efficiently than individual tumor cells. The early data reflect encouraging synergy between Paxalisib and immunotherapy, supporting further investigation in this clinical trial to better understand safety, tolerability, and overall effectiveness against aggressive tumors.
Read Announcement- Drug:
- Paxalisib
- Announced Date:
- June 11, 2025
- Indication:
- Atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer
Announcement
Kazia Therapeutics Limited announced the publication of transformative preclinical research in the journal Molecular Cancer Therapeutics, a journal of the American Association for Cancer Research that underscores the powerful potential of its lead asset, paxalisib, in reshaping the treatment landscape for triple-negative breast cancer (TNBC), one of the most aggressive and treatment-resistant cancer subtypes.
AI Summary
Kazia Therapeutics Limited recently published groundbreaking preclinical research in Molecular Cancer Therapeutics that highlights the strong promise of its lead asset, paxalisib, for treating triple-negative breast cancer (TNBC). This aggressive form of cancer is known for being resistant to treatment. The study, conducted by Professor Sudha Rao at QIMR Berghofer Medical Research Institute, shows that paxalisib can reprogram the tumor microenvironment. It increases the infiltration and activation of CD4+ and CD8+ T cells, which are key players in the body’s immune response.
Additionally, the research found that paxalisib works best when paired with immune checkpoint inhibitors, such as pembrolizumab, demonstrating a synergistic effect in combating the cancer. These findings provide a strong scientific basis for further clinical exploration, positioning paxalisib as a potential game changer for TNBC treatment.
Read Announcement- Drug:
- Paxalisib
- Announced Date:
- June 5, 2025
- Indication:
- Atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer
Announcement
Kazia Therapeutics Limited announced that the first patient has been dosed in a Phase 1b clinical trial sponsored by Kazia
AI Summary
Kazia Therapeutics Limited announced that the first patient has been dosed in a Phase 1b clinical trial sponsored by the company. The trial is testing paxalisib, a dual PI3K/mTOR inhibitor, when used with either olaparib or pembrolizumab for patients with advanced breast cancer. The study aims to evaluate the safety, tolerability, and early signs of effectiveness of these combination therapy regimens. Patients enrolled in the trial will be monitored through biomarker profiling to better understand treatment impacts on tumor metabolism, DNA repair, and immune responses. Dr. John Friend, CEO of Kazia, described this milestone as an important step in broadening the application of paxalisib beyond brain cancer. The study represents a significant effort to explore new therapeutic options for patients with advanced breast cancer, with the hope of improving outcomes and expanding treatment strategies for this challenging disease.
Read Announcement- Drug:
- Paxalisib
- Announced Date:
- February 20, 2025
- Indication:
- Atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer
Announcement
Kazia Therapeutics Limited announced a research grant awarded from The Michael J. Fox Foundation for Parkinson's Research (MJFF) to fund research between The Hebrew University of Jerusalem (Hebrew University) and Kazia to explore the therapeutic potential of paxalisib as a treatment for Parkinson's disease (PD).
AI Summary
Kazia Therapeutics Limited announced it received a research grant from The Michael J. Fox Foundation for Parkinson’s Research. The funding will support a collaborative study with The Hebrew University of Jerusalem aimed at exploring the therapeutic potential of paxalisib as a treatment for Parkinson’s disease. The preclinical research will be conducted in Professor Ronit Sharon’s lab and will investigate how paxalisib, a drug that crosses the blood-brain barrier, may affect specific biochemical, pathological, and molecular biomarkers in mouse models of Parkinson’s. Researchers will evaluate mouse survival, as well as motor and non-motor performances, to determine how the drug impacts pathways involved in the disease. This study seeks to provide new insights into whether paxalisib, a unique PI3K inhibitor, can address the underlying pathophysiology of Parkinson’s disease and ultimately lead to more effective treatments.
Read Announcement- Drug:
- Paxalisib
- Announced Date:
- January 30, 2025
- Indication:
- Atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer
Announcement
Kazia Therapeutics Limited is pleased to announce the regulatory approval and launch of a clinical trial evaluating the combination of paxalisib and immunotherapy in patients with advanced breast cancer.
AI Summary
Kazia Therapeutics Limited has received regulatory approval to start a new clinical trial for advanced breast cancer. The study will evaluate the combination of paxalisib with immunotherapy in patients, aiming to improve treatment options for this aggressive and resistant type of cancer. The trial, known as the ABC-Pax study, is a phase 1b, multi-center study that plans to enroll 24 patients at top cancer centers in Queensland, Australia. Patients in the study will receive the treatment combination for up to 12 months.
The innovative approach combines paxalisib with either pembrolizumab or olaparib. Early preclinical findings suggest that this combination may work by reprogramming dormant cancer cells, making them more visible to the immune system and enhancing the body’s ability to fight the tumor. This trial marks a promising step forward in targeting hard-to-treat breast cancer.
Read Announcement- Drug:
- Paxalisib
- Announced Date:
- December 31, 2024
- Indication:
- Atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer
Announcement
Kazia Therapeutics Limited today provided a regulatory update on paxalisib for the treatment of glioblastoma (GBM) following its Type C clinical meeting with the United States Food and Drug Administration (FDA).
AI Summary
Kazia Therapeutics Limited provided a regulatory update on paxalisib for glioblastoma (GBM) following its recent Type C clinical meeting with the FDA. During the meeting, the FDA explained that overall survival (OS) data, while supportive, would generally not be enough for accelerated approval. However, the agency indicated that these OS results could help support a traditional, standard approval if further confirmed. Both parties reached an agreement on key aspects of a proposed registrational Phase 3 study, including the patient population, the primary endpoint, and the comparator arm. This feedback gives Kazia clear direction on the next steps for paxalisib’s development in newly diagnosed unmethylated GBM patients, emphasizing the need for a pivotal trial to further establish its clinical benefits.
Read Announcement- Drug:
- Paxalisib
- Announced Date:
- December 11, 2024
- Indication:
- Atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer
Announcement
Kazia Therapeutics Limited announced that data from two abstracts will be presented at the San Antonio Breast Cancer Symposium highlighting the activity the Company's lead product candidate, paxalisib, for the potential treatment of immunotherapy-resistant triple negative breast cancer (TNBC) and HER2 positive metastatic breast cancer with active brain metastases.
AI Summary
Kazia Therapeutics Limited announced that two abstracts will be presented at the San Antonio Breast Cancer Symposium. The abstracts highlight the activity of their lead product candidate, paxalisib, for potentially treating difficult forms of breast cancer. One abstract focuses on immunotherapy-resistant triple negative breast cancer, where preclinical studies in a mouse model showed that adding paxalisib to immunotherapy and a PARP inhibitor helped reduce tumor size, lung metastases, and liver inflammation. The second abstract shared clinical data from heavily pretreated patients with HER2 positive metastatic breast cancer and active brain metastases. Patients receiving a combination of paxalisib and trastuzumab had a median overall survival of 16.5 months compared to historical averages. These findings suggest that paxalisib may play an important role in overcoming resistance to standard treatments in these challenging breast cancer types.
Read Announcement- Drug:
- Paxalisib
- Announced Date:
- October 2, 2024
- Indication:
- Atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer
Announcement
Kazia Therapeutics Limited announced the presentation of data from a Phase I study (NCT04192981) evaluating concurrent paxalisib and radiation therapy (RT) in patients for the treatment of solid tumor brain metastases (BM) or leptomeningeal metastases (LM) harboring PI3K pathway mutations at the American Society for Radiation Oncology 66th Annual Meeting (ASTRO 2024), which is taking place from September 29 - October 2, 2024, in Washington, D.C.
AI Summary
Kazia Therapeutics Limited announced that it will present data from its Phase I study (NCT04192981) at the American Society for Radiation Oncology (ASTRO) 2024 Annual Meeting in Washington, D.C., from September 29 to October 2, 2024. The study evaluates the safety and efficacy of combining paxalisib, an investigational PI3K inhibitor, with radiation therapy for patients with solid tumor brain metastases or leptomeningeal metastases that harbor PI3K pathway mutations. Early data from the trial revealed that treatment with 45mg paxalisib alongside radiotherapy resulted in a 67% partial response, with over two-thirds of patients at the maximum tolerated dose showing an intracranial response. These promising early response rates compare favorably to historical outcomes with whole brain radiation therapy alone, suggesting that this combination could provide a viable treatment option for patients facing treatment-resistant brain metastases.
Read Announcement- Drug:
- Paxalisib
- Announced Date:
- July 10, 2024
- Indication:
- Atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer
Announcement
Kazia Therapeutics Limited is pleased to announce results from GBM-AGILE, a phase II/III study that included an evaluation of paxalisib versus standard of care (SOC) for patients with glioblastoma (NCT03522298), a life-threatening brain cancer, where there is an urgent unmet need for new therapeutics.
AI Summary
Kazia Therapeutics Limited announced positive results from its GBM-AGILE trial, a phase II/III study that compared paxalisib with the standard of care for patients with glioblastoma, a deadly brain cancer with few treatment options. The study focused on newly diagnosed patients whose tumors have an unmethylated MGMT promoter status, who currently have limited effective treatments. In a prespecified secondary analysis, paxalisib showed a clinically meaningful improvement in overall survival, with patients in the treatment group experiencing a 3.8-month longer median survival compared to those receiving standard therapy. These promising results support the potential of paxalisib as a new therapeutic option in this challenging patient population. Kazia plans to discuss these findings with the FDA to explore a pathway for accelerated approval, addressing the urgent need for effective treatments in glioblastoma.
Read Announcement- Drug:
- Paxalisib
- Announced Date:
- June 27, 2024
- Indication:
- Atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer
Announcement
Kazia Therapeutics Limited announce the presentation of new data from its lead program, paxalisib, at the upcoming 21st International Symposium on Pediatric Neuro-Oncology (ISPNO 2024) June 29 – July 2, 2024, in Philadelphia, PA. Kazia concurrently announces publication of an article in European Journal of Cancer highlighting the need for evaluating mutation-specific, CNS penetrant, inhibitors to treat pediatric patients with Diffuse Midline Glioma (DMG).
AI Summary
Kazia Therapeutics Limited announced that it will present new data from its lead program, paxalisib, at the 21st International Symposium on Pediatric Neuro-Oncology (ISPNO 2024) in Philadelphia, PA, scheduled for June 29 – July 2, 2024. The company will share findings from its ongoing research on paxalisib, an innovative brain-penetrant inhibitor, aimed at treating pediatric brain cancers such as Diffuse Midline Glioma (DMG).
In tandem, Kazia revealed the publication of an article in the European Journal of Cancer. The article stresses the need to evaluate mutation-specific, CNS-penetrant inhibitors for children with DMG and calls for innovative regulatory approaches. This work highlights a critical direction in pediatric oncology, emphasizing targeted treatment strategies to better address the unique challenges presented by these rare and aggressive tumors.
Read Announcement
Novogen FDA Events - Frequently Asked Questions
As of now, Novogen (KZIA) has not received any FDA approvals for its therapy in the last two years.
In the past two years, Novogen (KZIA) has reported FDA regulatory activity for Paxalisib.
The most recent FDA-related event for Novogen occurred on January 27, 2026, involving Paxalisib. The update was categorized as "Clinical Update," with the company reporting: "Kazia Therapeutics provided a clinical update from its ongoing Phase 1b study evaluating paxalisib in combination with pembrolizumab and chemotherapy in patients with late-stage (Stage IV), metastatic triple-negative breast cancer (TNBC)."
Currently, Novogen has one therapy (Paxalisib) targeting the following condition: Atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer.
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FDA Event Stage Terminology & Abbreviation Guide
- NDA: New Drug Application
- ANDA: Abbreviated New Drug Application
- sNDA: Supplemental New Drug Application
- BLA: Biologics License Application
- sBLA: Supplemental Biologics License Application
- FDA Approved: Approved by the FDA
- EMA: European Medicines Agency
- CE Mark: European Union Certification
- NMPA: China National Medical Products Administration
- MHLW: Japanese Ministry of Health
- FDA Meeting: Consultation with FDA
- Pre-IND: Pre-Investigational New Drug Meeting
- Breakthrough Therapy: Special FDA designation for promising therapies
- Fast Track: Accelerated FDA approval pathway
- Orphan Drug: Designation for rare disease treatments
- RPD: Rare Pediatric Disease Designation
- RMAT: Regenerative Medicine Advanced Therapy
- DSMB Review: Data Safety Monitoring Board Review
- IDMC Review: Independent Data Monitoring Committee
- MAA: MHRA Marketing Authorization Application
- RTF: Refusal to File (Rejected Application)
- 510(k): FDA Clearance for Medical Devices
- Rolling Submission: Staggered regulatory review process
FDA progress for NASDAQ:KZIA last updated on 1/27/2026 by MarketBeat.com Staff. We continuously monitor for new FDA events and market data.
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