Kazia Therapeutics Ltd (ASX:KZA) (NASDAQ: KZIA) has revealed that St Jude Children’s Research Hospital successfully completed the first stage of its ongoing phase I study of Kazia’s investigational new drug, GDC-0084 in diffuse intrinsic pontine glioma (DIPG) and other diffuse midline gliomas.
Diffuse midline gliomas, including DIPG, represent one of the most aggressive childhood cancers, and several hundred new cases are diagnosed in the US each year.
Median life expectancy from diagnosis is estimated to be 9-11 months, and there is no approved pharmacological therapy.
St Jude hospital’s study has determined an appropriate dose for paediatric use and will now advance into the second stage of the study, which is designed to explore preliminary signals of efficacy in this disease.
Early signs are encouraging
Co-lead investigator on the study Dr. Christopher Tinkle said: “To date, thirteen patients have received GDC-0084 in our study.
“We have determined a maximum tolerated dose (MTD) in the pediatric setting of 27 mg/m2 , which we would expect to be approximately equivalent to the doses currently being explored in adult studies.
“The safety profile of the drug in children appears to be broadly similar to that in adults, and we have encountered no unexpected findings in this regard.
“It would be premature to draw conclusions regarding potential efficacy, but we now embark on an expansion cohort to explore the clinical effects of the drug in this patient group.
“My colleagues and I are very pleased with progress and look forward to reporting further data from the study.”
Acceptable tolerability profile
Kazia CEO Dr James Garner said: “DIPG is a devastating illness with a poor prognosis and a paucity of effective treatments. It has been a privilege to support the St Jude team on this incredibly important project.
“The study has recruited well ahead of our expectations, and we are delighted to have the first part successfully completed.
“Toxicity can be a significant challenge in the treatment of childhood cancer, so it is very encouraging that GDC-0084 appears to have an acceptable tolerability profile. We are excited to see further data in due course.”