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Kazia Therapeutics (ASX:KZA) - CEO, Dr James Garner
CEO, Dr James Garner
Source: Kazia Therapeutics/Twitter
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  • Kazia Therapeutics (KZA) kicks off its brain cancer study, with the first patient now enrolled
  • The study will enlist up to 216 children and young adults with diffuse midline gliomas, a category of brain tumour
  • The primary endpoint of the study will be the proportion of patients who are progression-free at six months for newly diagnosed patients, and overall survival for recurrent patients
  • The initiation of this trial adds to the pipeline of nine ongoing clinical studies of paxalisib in different forms of brain cancer
  • Kazia Therapeutics was up 1.94 per cent, trading at $1.58 at 3:05 pm AEDT

Kazia Therapeutics (KZA) has kicked off its brain cancer study, with the first patient now enrolled.

The phase II study is investigating diffuse intrinsic pontine glioma (DIPG) and diffuse midline gliomas, a highly aggressive form of childhood brain cancer, using Kazia’s investigational drug, paxalisib.

DIPG has an average survival from diagnosis of around 10-months, with no current FDA-approved drug treatment.

The study will enrol children and young adults with diffuse midline gliomas, a category of brain tumours that includes DIPG. The study has the potential to recruit up to 216 patients, with the actual number dependent on emerging results.

The study will include separate cohorts, made up of patients with the 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 primary endpoint of the study will be the proportion of patients who are progression-free at six months for newly diagnosed patients, and overall survival for recurrent patients.

“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,” said Kazia CEO Dr James Garner.

“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.”

The initiation of this trial adds to the pipeline of nine ongoing clinical studies of paxalisib in different forms of brain cancer.

Kazia Therapeutics was up 1.94 per cent, trading at $1.58 at 3:05 pm AEDT.

KZA by the numbers
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