Unlike the majority of biotech players, Kazia Therapeutics has emphasised diversity in its clinical trials, so as to look at the potential of its lead drug paxalisib in a range of different treatment areas.

The company currently has eight clinical trials underway, investigating the potential of the drug in various forms of brain cancer, including brain metastases, glioblastoma, and childhood brain cancer.

Kazia Therapeutics (ASX:KZA) CEO Dr James Garner says it’s all about creating multiple shots on goal – because if you can market the drug for several different diseases it expands your commercial opportunities.

“One of the things we’ve been doing as a company for some time now is trying to diversify what we do with this drug,” he said.

“The reality is that a lot of small biotech companies end up being an all-or-nothing bet on the outcome of a single clinical trial. In that scenario, it’s one drug, one trial, one disease – if it’s successful that’s great, but if it’s a failure, it’s hard to come back.

“We’ve actively tried to avoid that by deliberately looking at the drug in a whole range of different areas – we are really trying to have as many horses in the race as we can.”


Positive response in brain metastases trial

The company recently announced positive results from a phase I clinical trial of paxalisib in combination with radiotherapy for the treatment of brain metastases – namely a 100% overall response rate.

“This means that every evaluable patient has had some meaningful response,” Dr Garner said.

“We don’t see that with radiotherapy by itself, and we don’t see it much with other drugs, so it really gives us a sense that paxalisib makes a meaningful difference when you give it with radiotherapy.

“That’s exciting because this is something where there’s a huge need, with around 200,000 patients diagnosed with brain metastases each year in the United States alone. If our drug can make a difference here, it’s going to potentially be a huge opportunity for us.”

The next step is for the study to recruit another 12 patients, with final data expected in 2023.


GBM Agile trial not all bad news

Dr Garner said that while the company also announced that the paxalisib arm of the GBM AGILE trial did not pass the statistical threshold to move to the second stage, the study has fully recruited the first stage is still ongoing.

“The way the study is designed is in two stages. There’s a first stage of up to 150 patients, and then they do a statistical test at that point. If the drug passes the statistical test, it goes on to recruit another 50 patients in the second stage. If it doesn’t, then it stops at 150,” he said.

“We’ve stopped at 150, but we have no further data at this point and the study is still ongoing. When the study completes – which is still another 12 months or so away – we may well have enough data to get an approval from the FDA.”

Kazia is blinded to the results until the study wraps up, which means they don’t know anything about the efficacy or safety of the drug until the final data is released.

“There’s been a negative market response, which is understandable, because people are so used to bad news out of biotech companies that as soon as they see anything like this, they just assume the worst,” Dr Garner said.

“But, in all honesty, it’s not clear what this means. The study remains ongoing, we’ve got the better part of a year before we see final data and the existing 150 or so patients will continue to mature during follow up, so we’ll wait and see.”

Either way, by pursuing a variety of uses for the drug, Dr Garner says Kazia has mitigated the risk around this trial, and the company has plenty of other opportunities in its drug development pipeline.


This article was developed in collaboration with Kazia Therapeutics Limited, a Stockhead advertiser at the time of publishing.

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.