{"success":true,"data":{"pressRelease":{"id":"138941","rtpr_id":"nPNAzhJXSa-20260909","ticker":"KZIA","exchange":"NASDAQ","all_tickers":["KZIA"],"title":"Kazia Therapeutics Announces First Patient Dosed in PNOC035 Clinical Study Evaluating Paxalisib for Recurrent Pediatric AT/RT Brain Cancer","author":"PR Newswire","published_at":"2026-09-09T11:30:00.446Z","article_body":"Kazia Therapeutics Announces First Patient Dosed in PNOC035 Clinical Study\nEvaluating Paxalisib for Recurrent Pediatric AT/RT Brain Cancer\nPR Newswire\n\nSYDNEY, Sept. 9, 2026\n\nMulticenter study to evaluate paxalisib plus gemcitabine for children and\nyoung adults with recurrent or progressive AT/RT, where no approved therapies\ncurrently exist\n\nSYDNEY, Sept. 9, 2026 /PRNewswire/ -- Kazia Therapeutics Limited\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4769279-1&h=2562557461&u=https%3A%2F%2Fwww.kaziatx.com%2F&a=Kazia+Therapeutics+Limited) (NASDAQ:\nKZIA) (\"Kazia\" or the \"Company\"), an oncology-focused biotechnology company\ndeveloping therapies that selectively reprogram cancer biology, restore\nanti-tumor immunity and overcome treatment resistance, today announced that\nthe first patient has been dosed in Arm A of PNOC035, a Phase 2 platform study\nevaluating paxalisib in combination with gemcitabine for patients ages 1 to 39\nwith recurrent or progressive atypical teratoid/rhabdoid tumor (AT/RT). AT/RT\nis widely recognized as one of the most aggressive forms of pediatric brain\ncancer.\n\n\n\nPNOC035 is conducted by the Pediatric Neuro-Oncology Consortium (PNOC), an\nopen-label, multi-center platform study. In Arm A, patients receive oral,\nonce-daily paxalisib in combination with intravenous gemcitabine on Days 1, 8\nand 15 of each 28-day treatment cycle.\n\nThe combination increased median survival nearly four-fold (22 to 82.5 days;\np<0.0001) in an orthotopic AT/RT preclinical model and demonstrated\ncomplementary anti-tumor activity across multiple AT/RT cell lines. These\npreclinical findings provided the rationale for advancement into the\nmulticenter PNOC035 clinical study evaluating paxalisib plus gemcitabine in\nchildren and young adults with recurrent or progressive AT/RT. These findings,\npreviously presented at ISPNO, AACR and Neuro-Oncology meetings, provided the\nscientific rationale for PNOC035 and extend paxalisib's pediatric\nneuro-oncology development beyond diffuse midline glioma into another area of\nsignificant unmet need.\n\nPaxalisib has been granted both Orphan Drug Designation and Rare Pediatric\nDisease Designation by the U.S. Food and Drug Administration (FDA) for AT/RT.\nThese designations do not constitute FDA approval or endorsement of any\ntherapeutic claim. If paxalisib were to receive marketing approval for this\nindication, which would require successful completion of clinical development\nand FDA regulatory approval, Kazia could become eligible to receive a\npediatric priority review voucher from the FDA.\n\n\"AT/RT typically affects infants and very young children, and families facing\nthis devastating diagnosis have limited treatment options,\" said Dr. John\nFriend II, Chief Executive Officer of Kazia Therapeutics. \"Despite decades of\nresearch, there are still no approved therapies for recurrent or progressive\nAT/RT, underscoring the urgent need for new treatment options. We've remained\ncommitted to pediatric brain cancer because this is an area where the need is\nespecially great and the options are often fewest. Reaching this milestone\nwith the PNOC035 team is a meaningful step forward, and we look forward to\nworking closely with PNOC as enrollment progresses.\"\n\nAbout PNOC035\n\nPNOC035 is a Phase 2 platform trial conducted by the Pediatric Neuro-Oncology\nConsortium (PNOC) for patients ages 1 to 39 with recurrent or progressive\nAT/RT. The platform design allows multiple treatment arms to be evaluated\nwithin a shared trial infrastructure, with patients enrolling in an arm based\non availability and arm-specific eligibility criteria. Arm A evaluates\npaxalisib in combination with gemcitabine. Additional information about the\nstudy is available on ClinicalTrials.gov (NCT07447076\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4769279-1&h=500840980&u=https%3A%2F%2Fclinicaltrials.gov%2Fstudy%2FNCT07447076%3Fterm%3DGDC-0084%2520OR%2520paxalisib%2520OR%2520EVT801%26aggFilters%3Dstatus%3A%26viewType%3DTable%26page%3D2%26rank%3D16&a=NCT07447076)).\n\nAbout Kazia Therapeutics\n\nKazia Therapeutics Limited (NASDAQ: KZIA) is an oncology-focused drug\ndevelopment company, based in Sydney, Australia. The Company's lead asset,\npaxalisib, is an investigational brain penetrant inhibitor of the PI3K/Akt\n/mTOR pathway, which is being developed to treat multiple forms of cancer.\nLicensed from Genentech in late 2016, paxalisib is or has been the subject of\nover 15 clinical trials. A completed Phase 2/3 study in glioblastoma (GBM\nAGILE) was reported in 2024, and discussions are ongoing for designing and\nexecuting a pivotal registrational study in pursuit of a standard\napproval. Other clinical trials involving paxalisib are ongoing in advanced\nbreast cancer, brain metastases, diffuse midline gliomas, and primary central\nnervous system lymphoma, with several of these trials having reported\nencouraging interim data. Paxalisib was granted Orphan Drug Designation for\nglioblastoma by the U.S. Food and Drug Administration (FDA) in February 2018,\nand Fast Track Designation (FTD) for glioblastoma in August 2020. Paxalisib\nwas also granted FTD in July 2023 for the treatment of solid tumor brain\nmetastases harboring PI3K pathway mutations in combination with radiation\ntherapy. Additionally, paxalisib was granted Rare Pediatric Disease\nDesignation and Orphan Drug Designation by the FDA for diffuse intrinsic\npontine glioma in August 2020 and for atypical teratoid / rhabdoid tumors in\nJune 2022 and July 2022, respectively. Kazia is also developing EVT801, a\nsmall molecule inhibitor of VEGFR3, which was licensed from Evotec SE in April\n2021. In addition to its clinical-stage programs, Kazia is advancing NDL2, a\npotentially first-in-class intracellular PD-L1 protein degrader program\ntargeting a newly identified mechanism of immunotherapy resistance and\nmetastatic progression, as well as MSETC, a potentially first-in-class SETDB1\ninhibitor program intended to restore immune signaling in tumors that have\nbecome resistant to immunotherapy, including checkpoint inhibitors. Both\nprograms are currently in preclinical development. For more information,\nplease visit http://www.kaziatx.com/ or follow us on X @KaziaTx.\n\nForward-Looking Statements\n\nThis announcement may contain forward-looking statements, which can generally\nbe identified by words such as \"may,\" \"will,\" \"estimate,\" \"future,\" \"forward,\"\n\"anticipate,\" \"expect,\" \"plan,\" \"believe,\" \"potential,\" \"could,\" or similar\nterms. Forward-looking statements in this release include, but are not limited\nto, statements regarding: the conduct, timing and progress of enrollment in\nthe PNOC035 study; the potential therapeutic benefit of paxalisib in AT/RT;\nthe possibility of clinical results that are consistent with the preclinical\ndata; and the possibility that Kazia could become eligible to receive a\npriority review voucher if paxalisib were to receive marketing approval for\nAT/RT, which remains uncertain and is subject to the successful completion of\nclinical development and regulatory review; the design, execution and outcome\nof a potential pivotal registrational study of paxalisib for glioblastoma; the\ntherapeutic potential and clinical development of paxalisib in other\nindications, including advanced breast cancer, brain metastases, diffuse\nmidline gliomas and primary central nervous system lymphoma; and the\npreclinical development and potential of Kazia's earlier-stage programs,\nincluding NDL2 and MSETC. Such statements are based on Kazia's current\nexpectations and are subject to risks and uncertainties that could cause\nactual results to differ materially, including those described in Kazia's\nAnnual Report on Form 20-F and subsequent filings with the SEC. Kazia\nundertakes no obligation to update any forward-looking statement except as\nrequired by law.\n\nView original content to download\nmultimedia:https://www.prnewswire.com/apac/news-releases/kazia-therapeutics-announces-first-patient-dosed-in-pnoc035-clinical-study-evaluating-paxalisib-for-recurrent-pediatric-atrt-brain-cancer-302873110.html\n\nSOURCE Kazia Therapeutics Limited\n\n\n\nPhoto: \nhttps://mmx.prnewswire.com/media/MS1368769/Kazia-Logo.jpg?id=OA2937528\n\nCopyright (c) 2026 PR Newswire Association,LLC. All Rights Reserved.","article_body_html":"","raw_payload":{"data":{"id":"nPNAzhJXSa-20260909","title":"Kazia Therapeutics Announces First Patient Dosed in PNOC035 Clinical Study Evaluating Paxalisib for Recurrent Pediatric AT/RT Brain Cancer","author":"PR Newswire","ticker":"KZIA","created":"2026-09-09T11:30:00.446Z","tickers":["KZIA"],"exchange":"NASDAQ","article_body":"Kazia Therapeutics Announces First Patient Dosed in PNOC035 Clinical Study\nEvaluating Paxalisib for Recurrent Pediatric AT/RT Brain Cancer\nPR Newswire\n\nSYDNEY, Sept. 9, 2026\n\nMulticenter study to evaluate paxalisib plus gemcitabine for children and\nyoung adults with recurrent or progressive AT/RT, where no approved therapies\ncurrently exist\n\nSYDNEY, Sept. 9, 2026 /PRNewswire/ -- Kazia Therapeutics Limited\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4769279-1&h=2562557461&u=https%3A%2F%2Fwww.kaziatx.com%2F&a=Kazia+Therapeutics+Limited) (NASDAQ:\nKZIA) (\"Kazia\" or the \"Company\"), an oncology-focused biotechnology company\ndeveloping therapies that selectively reprogram cancer biology, restore\nanti-tumor immunity and overcome treatment resistance, today announced that\nthe first patient has been dosed in Arm A of PNOC035, a Phase 2 platform study\nevaluating paxalisib in combination with gemcitabine for patients ages 1 to 39\nwith recurrent or progressive atypical teratoid/rhabdoid tumor (AT/RT). AT/RT\nis widely recognized as one of the most aggressive forms of pediatric brain\ncancer.\n\n\n\nPNOC035 is conducted by the Pediatric Neuro-Oncology Consortium (PNOC), an\nopen-label, multi-center platform study. In Arm A, patients receive oral,\nonce-daily paxalisib in combination with intravenous gemcitabine on Days 1, 8\nand 15 of each 28-day treatment cycle.\n\nThe combination increased median survival nearly four-fold (22 to 82.5 days;\np<0.0001) in an orthotopic AT/RT preclinical model and demonstrated\ncomplementary anti-tumor activity across multiple AT/RT cell lines. These\npreclinical findings provided the rationale for advancement into the\nmulticenter PNOC035 clinical study evaluating paxalisib plus gemcitabine in\nchildren and young adults with recurrent or progressive AT/RT. These findings,\npreviously presented at ISPNO, AACR and Neuro-Oncology meetings, provided the\nscientific rationale for PNOC035 and extend paxalisib's pediatric\nneuro-oncology development beyond diffuse midline glioma into another area of\nsignificant unmet need.\n\nPaxalisib has been granted both Orphan Drug Designation and Rare Pediatric\nDisease Designation by the U.S. Food and Drug Administration (FDA) for AT/RT.\nThese designations do not constitute FDA approval or endorsement of any\ntherapeutic claim. If paxalisib were to receive marketing approval for this\nindication, which would require successful completion of clinical development\nand FDA regulatory approval, Kazia could become eligible to receive a\npediatric priority review voucher from the FDA.\n\n\"AT/RT typically affects infants and very young children, and families facing\nthis devastating diagnosis have limited treatment options,\" said Dr. John\nFriend II, Chief Executive Officer of Kazia Therapeutics. \"Despite decades of\nresearch, there are still no approved therapies for recurrent or progressive\nAT/RT, underscoring the urgent need for new treatment options. We've remained\ncommitted to pediatric brain cancer because this is an area where the need is\nespecially great and the options are often fewest. Reaching this milestone\nwith the PNOC035 team is a meaningful step forward, and we look forward to\nworking closely with PNOC as enrollment progresses.\"\n\nAbout PNOC035\n\nPNOC035 is a Phase 2 platform trial conducted by the Pediatric Neuro-Oncology\nConsortium (PNOC) for patients ages 1 to 39 with recurrent or progressive\nAT/RT. The platform design allows multiple treatment arms to be evaluated\nwithin a shared trial infrastructure, with patients enrolling in an arm based\non availability and arm-specific eligibility criteria. Arm A evaluates\npaxalisib in combination with gemcitabine. Additional information about the\nstudy is available on ClinicalTrials.gov (NCT07447076\n(https://edge.prnewswire.com/c/link/?t=0&l=en&o=4769279-1&h=500840980&u=https%3A%2F%2Fclinicaltrials.gov%2Fstudy%2FNCT07447076%3Fterm%3DGDC-0084%2520OR%2520paxalisib%2520OR%2520EVT801%26aggFilters%3Dstatus%3A%26viewType%3DTable%26page%3D2%26rank%3D16&a=NCT07447076)).\n\nAbout Kazia Therapeutics\n\nKazia Therapeutics Limited (NASDAQ: KZIA) is an oncology-focused drug\ndevelopment company, based in Sydney, Australia. The Company's lead asset,\npaxalisib, is an investigational brain penetrant inhibitor of the PI3K/Akt\n/mTOR pathway, which is being developed to treat multiple forms of cancer.\nLicensed from Genentech in late 2016, paxalisib is or has been the subject of\nover 15 clinical trials. A completed Phase 2/3 study in glioblastoma (GBM\nAGILE) was reported in 2024, and discussions are ongoing for designing and\nexecuting a pivotal registrational study in pursuit of a standard\napproval. Other clinical trials involving paxalisib are ongoing in advanced\nbreast cancer, brain metastases, diffuse midline gliomas, and primary central\nnervous system lymphoma, with several of these trials having reported\nencouraging interim data. Paxalisib was granted Orphan Drug Designation for\nglioblastoma by the U.S. Food and Drug Administration (FDA) in February 2018,\nand Fast Track Designation (FTD) for glioblastoma in August 2020. Paxalisib\nwas also granted FTD in July 2023 for the treatment of solid tumor brain\nmetastases harboring PI3K pathway mutations in combination with radiation\ntherapy. Additionally, paxalisib was granted Rare Pediatric Disease\nDesignation and Orphan Drug Designation by the FDA for diffuse intrinsic\npontine glioma in August 2020 and for atypical teratoid / rhabdoid tumors in\nJune 2022 and July 2022, respectively. Kazia is also developing EVT801, a\nsmall molecule inhibitor of VEGFR3, which was licensed from Evotec SE in April\n2021. In addition to its clinical-stage programs, Kazia is advancing NDL2, a\npotentially first-in-class intracellular PD-L1 protein degrader program\ntargeting a newly identified mechanism of immunotherapy resistance and\nmetastatic progression, as well as MSETC, a potentially first-in-class SETDB1\ninhibitor program intended to restore immune signaling in tumors that have\nbecome resistant to immunotherapy, including checkpoint inhibitors. Both\nprograms are currently in preclinical development. For more information,\nplease visit http://www.kaziatx.com/ or follow us on X @KaziaTx.\n\nForward-Looking Statements\n\nThis announcement may contain forward-looking statements, which can generally\nbe identified by words such as \"may,\" \"will,\" \"estimate,\" \"future,\" \"forward,\"\n\"anticipate,\" \"expect,\" \"plan,\" \"believe,\" \"potential,\" \"could,\" or similar\nterms. Forward-looking statements in this release include, but are not limited\nto, statements regarding: the conduct, timing and progress of enrollment in\nthe PNOC035 study; the potential therapeutic benefit of paxalisib in AT/RT;\nthe possibility of clinical results that are consistent with the preclinical\ndata; and the possibility that Kazia could become eligible to receive a\npriority review voucher if paxalisib were to receive marketing approval for\nAT/RT, which remains uncertain and is subject to the successful completion of\nclinical development and regulatory review; the design, execution and outcome\nof a potential pivotal registrational study of paxalisib for glioblastoma; the\ntherapeutic potential and clinical development of paxalisib in other\nindications, including advanced breast cancer, brain metastases, diffuse\nmidline gliomas and primary central nervous system lymphoma; and the\npreclinical development and potential of Kazia's earlier-stage programs,\nincluding NDL2 and MSETC. Such statements are based on Kazia's current\nexpectations and are subject to risks and uncertainties that could cause\nactual results to differ materially, including those described in Kazia's\nAnnual Report on Form 20-F and subsequent filings with the SEC. Kazia\nundertakes no obligation to update any forward-looking statement except as\nrequired by law.\n\nView original content to download\nmultimedia:https://www.prnewswire.com/apac/news-releases/kazia-therapeutics-announces-first-patient-dosed-in-pnoc035-clinical-study-evaluating-paxalisib-for-recurrent-pediatric-atrt-brain-cancer-302873110.html\n\nSOURCE Kazia Therapeutics Limited\n\n\n\nPhoto: \nhttps://mmx.prnewswire.com/media/MS1368769/Kazia-Logo.jpg?id=OA2937528\n\nCopyright (c) 2026 PR Newswire Association,LLC. All Rights Reserved."},"type":"article","timestamp":"2026-09-09T11:30:00.496258837Z","server_sent_at_ms":1788953400496},"received_at":"2026-09-09T11:30:00.565Z","source_url":null},"analysis":{"id":"127782","press_release_id":"138941","analysis_json":{"industry":{"label":"Biotechnology","sector":"Health Care"},"redFlags":["First-patient-dosed milestone only — no human efficacy or safety data yet","Supporting evidence is preclinical, with median survival benefit measured in days (22 to 82.5) in a mouse model; high translational risk","Priority review voucher eligibility is contingent on completing clinical development and receiving FDA marketing approval, which remains uncertain"],"eventType":"clinical_trial","narrative":"Kazia Therapeutics dosed the first patient in Arm A of PNOC035, a Phase 2 platform study testing oral paxalisib combined with intravenous gemcitabine in patients ages 1 to 39 with recurrent or progressive atypical teratoid/rhabdoid tumor (AT/RT), one of the most aggressive pediatric brain cancers with no approved therapies.\n\nPreclinical data underpin the trial: the combination increased median survival nearly four-fold (22 to 82.5 days; p<0.0001) in an orthotopic AT/RT model, findings previously presented at ISPNO, AACR and Neuro-Oncology meetings.\n\nPaxalisib already holds FDA Orphan Drug and Rare Pediatric Disease designations for AT/RT, and marketing approval in this indication could make Kazia eligible for a pediatric priority review voucher — a milestone that extends paxalisib's pediatric neuro-oncology development beyond diffuse midline glioma, though any payoff depends on completing clinical development and winning regulatory approval.","sentiment":"bullish","agentHooks":{"shouldPost":false,"suggestedAngle":"Kazia expands paxalisib into pediatric AT/RT — early-stage pipeline optionality with priority-review-voucher upside if approval ever lands."},"keyFigures":{"drugName":"paxalisib","phaseOfTrial":"Phase 2","customDimensions":{"trial_id":"NCT07447076","indication":"recurrent or progressive atypical teratoid/rhabdoid tumor (AT/RT)","trial_name":"PNOC035","combination":"paxalisib (oral, once-daily) + gemcitabine (IV, Days 1/8/15 of 28-day cycle)","patient_ages":"1 to 39","potential_upside":"pediatric priority review voucher eligibility if marketing approval is received","fda_designations_atrt":["Orphan Drug Designation (July 2022)","Rare Pediatric Disease Designation (June 2022)"],"preclinical_median_survival":"increased nearly four-fold, 22 to 82.5 days (p<0.0001) in orthotopic AT/RT model"}},"quotedText":"AT/RT typically affects infants and very young children, and families facing this devastating diagnosis have limited treatment options","namedEntities":{"people":[{"name":"Dr. John Friend II","role":"Chief Executive Officer, Kazia Therapeutics"}],"products":["paxalisib","gemcitabine","EVT801","NDL2","MSETC"],"companies":[{"name":"Kazia Therapeutics Limited","ticker":"KZIA","relationship":"filer"},{"name":"Pediatric Neuro-Oncology Consortium (PNOC)","relationship":"clinical trial collaborator (conducts PNOC035 platform study)"},{"name":"Genentech","relationship":"licensor of paxalisib (licensed late 2016)"},{"name":"Evotec SE","relationship":"licensor of EVT801 (licensed April 2021)"}],"dollarAmounts":[]},"materialImpact":{"score":2,"reasoning":"First-patient-dosed is an early execution milestone in a Phase 2 platform study (Arm A of PNOC035); there is no clinical data readout yet, only supporting preclinical survival data. Positive for pipeline breadth and pediatric expansion, but not a market-moving binary event."},"tickerRelevance":{"others":[],"primary":"KZIA"},"globalImportance":16,"audienceRelevance":15,"eventTypeSecondary":[],"importanceComponents":{"tickerTier":"micro-cap biotech","eventGravity":"first_patient_dosed_phase2","sectorWeight":"clinical-stage oncology","hasDataReadout":false,"householdBrandBoost":0,"retailFavoriteBoost":0}},"event_type":"clinical_trial","event_type_secondary":null,"sentiment":"bullish","material_impact_score":2,"narrative":"Kazia Therapeutics dosed the first patient in Arm A of PNOC035, a Phase 2 platform study testing oral paxalisib combined with intravenous gemcitabine in patients ages 1 to 39 with recurrent or progressive atypical teratoid/rhabdoid tumor (AT/RT), one of the most aggressive pediatric brain cancers with no approved therapies.\n\nPreclinical data underpin the trial: the combination increased median survival nearly four-fold (22 to 82.5 days; p<0.0001) in an orthotopic AT/RT model, findings previously presented at ISPNO, AACR and Neuro-Oncology meetings.\n\nPaxalisib already holds FDA Orphan Drug and Rare Pediatric Disease designations for AT/RT, and marketing approval in this indication could make Kazia eligible for a pediatric priority review voucher — a milestone that extends paxalisib's pediatric neuro-oncology development beyond diffuse midline glioma, though any payoff depends on completing clinical development and winning regulatory approval.","key_figures":{"drugName":"paxalisib","phaseOfTrial":"Phase 2","customDimensions":{"trial_id":"NCT07447076","indication":"recurrent or progressive atypical teratoid/rhabdoid tumor (AT/RT)","trial_name":"PNOC035","combination":"paxalisib (oral, once-daily) + gemcitabine (IV, Days 1/8/15 of 28-day cycle)","patient_ages":"1 to 39","potential_upside":"pediatric priority review voucher eligibility if marketing approval is received","fda_designations_atrt":["Orphan Drug Designation (July 2022)","Rare Pediatric Disease Designation (June 2022)"],"preclinical_median_survival":"increased nearly four-fold, 22 to 82.5 days (p<0.0001) in orthotopic AT/RT model"}},"named_entities":{"people":[{"name":"Dr. John Friend II","role":"Chief Executive Officer, Kazia Therapeutics"}],"products":["paxalisib","gemcitabine","EVT801","NDL2","MSETC"],"companies":[{"name":"Kazia Therapeutics Limited","ticker":"KZIA","relationship":"filer"},{"name":"Pediatric Neuro-Oncology Consortium (PNOC)","relationship":"clinical trial collaborator (conducts PNOC035 platform study)"},{"name":"Genentech","relationship":"licensor of paxalisib (licensed late 2016)"},{"name":"Evotec SE","relationship":"licensor of EVT801 (licensed April 2021)"}],"dollarAmounts":[]},"model_name":"glm-5.3-flash","prompt_hash":"sha256:727b4b9429a443af","schema_hash":"sha256:05005c02d9cffac9","created_at":"2026-09-09T11:31:47.390Z","global_importance":16,"audience_relevance":15,"importance_components":{"tickerTier":"micro-cap biotech","eventGravity":"first_patient_dosed_phase2","sectorWeight":"clinical-stage oncology","hasDataReadout":false,"householdBrandBoost":0,"retailFavoriteBoost":0}},"durationMs":36824,"modelName":"glm-5.3-flash"}}