logo-loader

Kazia Therapeutics welcomes start of PNOC study in childhood brain cancer, first patient enrolled

Published: 09:50 11 Nov 2021 AEDT

Kazia Therapeutics Ltd - Kazia Therapeutics welcomes start of PNOC study in childhood brain cancer, first patient enrolled

Kazia Therapeutics Ltd (ASX:KZA, NASDAQ:KZIA)’s investigational drug paxalisib is playing a role in PNOC022, a multi-drug phase II study in DIPG and diffuse midline gliomas, that has been initiated at the University of California, San Francisco, with the first patient enrolled to the study.

Diffuse intrinsic pontine glioma (DIPG) is the most common of a group of childhood brain cancers known as diffuse midline gliomas (DMGs). The disease has no FDA approved drug treatments and average survival from diagnosis is approximately 10 months.

Study includes paxalisib

PNOC022 includes paxalisib among the experimental arms and is expected to provide valuable data on the potential use of the drug in this highly aggressive form of childhood brain cancer.

The PNOC022 study (NCT05009992) is sponsored by the Pacific Pediatric Neuro-Oncology Consortium (PNOC), an international consortium focused on the development of new therapies for brain cancer.

It is an adaptive platform study that will examine multiple therapies concurrently, both as single agents and in combination, to determine the optimal approach to treatment.

The lead investigator in the study is Professor Sabine Mueller, a board-certified neurologist and a pediatric neuro-oncologist with a research program focused on novel therapies for pediatric brain tumours.

Applying novel methodologies

Professor Mueller commented, “No individual drug therapy has ever shown convincing efficacy in this group of patients.

"We plan to apply novel trial design methodologies to understand the potential for combination therapy, using some of the most promising agents in the global pipeline, to change the outcome in this devastating disease.”

PNOC022 will enrol children and young adults with diffuse midline gliomas, a category of brain tumours that includes DIPG.

The study will include separate cohorts comprising patients with newly diagnosed disease, patients who have completed initial radiotherapy, and patients who have experienced disease progression after treatment.

At the outset, all patients will be treated with ONC201, combined with either paxalisib or panobinostat. The study employs an adaptive design, in which different arms will be opened and closed based on emerging preclinical and clinical data.

The primary endpoint will be the proportion of patients who are progression-free at six months (PFS6) for newly diagnosed patients and overall survival (OS) for recurrent patients.

“Need for new treatment options”

Kazia CEO Dr James Garner said, “We are proud to be part of this important and innovative clinical study. Preclinical data supports the potential for paxalisib to combine well with certain other therapies, and we are keen to see if this approach is similarly promising in the clinic.

"There remains a desperate need for new treatment options in this disease, and we hope that paxalisib may be able to contribute to better outcomes for patients and their families.”

Kazia’s support of the project will consist primarily in provision of study drug.

Study design

Design of the PNOC022 study has been extensively informed by laboratory research in DIPG, and in particular, by research undertaken at the University of Newcastle, Hunter Medical Research Institute (HMRI) in Australia by Associate Professor Matt Dun and colleagues.

The HMRI team has conducted laboratory research with paxalisib for several years and has generated a powerful body of data combining paxalisib with other investigational drugs.

This research has been partly funded by RUN DIPG, a not-for-profit organisation led by Associate Professor Dun, the DIPG Collaborative, Chad Tough Defeat DIPG Foundation, and the McDonald Jones Foundation.

Associate Professor Dun serves as a scientific advisor to the study.

The study has the potential to recruit up to several hundred patients, with the actual number dependent on emerging results.

About paxalisib

Kazia's lead program is paxalisib, a brain-penetrant inhibitor of the PI3K/Akt/mTOR pathway, which is being developed to treat glioblastoma, the most common and most aggressive form of primary brain cancer in adults.

Paxalisib was granted Orphan Drug Designation for glioblastoma by the US FDA in February 2018, and Fast Track Designation for glioblastoma by the US FDA in August 2020. In addition, paxalisib was granted Rare Pediatric Disease Designation and Orphan Designation by the US FDA for DIPG in August 2020.

Initiation of the PNOC022 trial adds to the pipeline of nine ongoing clinical studies of paxalisib in different forms of brain cancer.

Australian Strategic Materials signs US$600 million LoI

Rowena Smith, CEO and managing director of Australian Strategic Materials Ltd (ASX:ASM, OTC:ASMMF), joins Jonathan Jackson in the Proactive studio to discuss the company’ s Dubbo Project, in Central West New South Wales. This project aims to extract and process critical minerals and rare earth...

12 hours, 46 minutes ago