Kazia Doses First Patient in Pediatric AT/RT Study

Sydney, Australia, September 10, 2026

PNOC035 Advances Paxalisib Into Pediatric Brain Cancer

The PNOC035 study is a clinical investigation focused on patients with recurrent or progressive AT/RT, an aggressive central nervous system tumor that predominantly affects young children. Despite advances in multimodal treatment, recurrent disease remains difficult to treat, and therapeutic options for children with relapsed AT/RT are limited. The initiation of patient dosing therefore marks an important step in evaluating a targeted therapeutic approach for this rare pediatric cancer. Paxalisib is an orally administered small-molecule inhibitor designed to target the PI3K/mTOR signaling pathway, which plays an important role in cancer-cell growth, proliferation and survival. Dysregulation of this pathway has been implicated in multiple tumor types, including pediatric brain cancers. By inhibiting signaling through PI3K and mTOR, paxalisib is being investigated for its potential to interfere with mechanisms that support tumor progression. The PNOC035 study is expected to enroll pediatric patients with recurrent or progressive AT/RT following standard treatment. Investigators will evaluate the investigational therapy under a structured clinical protocol to characterize its safety profile and determine whether biological and clinical activity warrants further investigation.

Study Evaluates Safety and Antitumor Activity

The clinical study will generate data across several important parameters, including safety, tolerability, pharmacokinetics and preliminary efficacy. These assessments are particularly important in pediatric oncology because treatment-related toxicity, drug exposure and dosing requirements can differ substantially between children and adults. Paxalisib has previously been evaluated in other oncology settings, including pediatric high-grade glioma. Kazia has been developing the molecule as a potential targeted therapy for brain cancers, where the ability of a drug to reach the central nervous system is a critical consideration. The company’s development strategy has focused on the potential of PI3K/mTOR inhibition to address molecular pathways involved in tumor growth. For AT/RT, the need for new therapeutic approaches is particularly significant. These tumors are often associated with alterations involving SMARCB1 or SMARCA4, genes involved in chromatin remodeling and tumor suppression. The disease can progress rapidly, and recurrence presents a major clinical challenge. PNOC035 provides an opportunity to investigate whether targeting PI3K/mTOR signaling can provide therapeutic benefit in this setting. The study’s early clinical data will be important for determining the appropriate dose and understanding how paxalisib behaves in pediatric patients. However, the first-patient dosing milestone itself does not demonstrate efficacy. Clinical benefit will need to be established through prospective trial data and appropriate follow-up.

Rare Pediatric Cancer Highlights Unmet Need

The initiation of PNOC035 underscores the continuing need for novel targeted therapies for pediatric brain tumors. Children with recurrent AT/RT have few established treatment options, and disease progression after initial therapy can be associated with poor outcomes. Clinical studies conducted through collaborative pediatric oncology networks such as PNOC can help accelerate investigation of promising therapies in rare cancers where individual institutions may have difficulty enrolling sufficient numbers of patients. Kazia’s paxalisib program also reflects the broader pharmaceutical effort to develop therapies capable of targeting signaling pathways that contribute to pediatric central nervous system malignancies. The candidate’s oral administration and ability to target PI3K/mTOR signaling provide a rationale for continued clinical investigation. As PNOC035 progresses, forthcoming safety, pharmacokinetic and preliminary efficacy findings will determine the next steps for paxalisib in recurrent AT/RT. The successful dosing of the first participant represents the transition of this specific program into active clinical evaluation and adds another potential treatment strategy for a pediatric cancer with significant unmet medical need. For Kazia Therapeutics, the milestone expands the clinical development of paxalisib into another difficult-to-treat pediatric brain tumor and reinforces the company’s focus on targeted oncology therapeutics.

Source: Kazia Therapeutics press release