The path from idea to market in the oncology sector is long and expensive. In the traditional model of drug development, therapies with the forecast of a favorable return on investment are the ones that are brought to trial. The possibility of effective therapies for the treatment of cancers like glioblastoma (GBM) is helping to bring about a new economic model. In this model, different companies will champion a drug at different stages in their development. Rather than adding to the cost of the drug, this model is designed to introduce cost-efficiencies into the cycle while bringing more drugs to market.
Kazia Therapeutics is a company focused on developing anti-cancer drugs. Headquartered in Sydney, Australia, Kazia works with oncology stakeholders worldwide to bring promising candidates through trials in the U.S. Once FDA approval is granted, these drugs are then brought to market. Kazia’s promising new cure, Paxalisib (GDC-0084), is part of the GBM AGILE trial – an international adaptive trial that will test several treatments in one study. The adaptive nature of the trial makes it easier to reduce the cost of the trial by weeding out poor performers as soon as they are identified. The number of participants in any one part of the trial will also vary, depending upon an analysis using Bayesian statistics.
Dr. James Garner, Kazia’s CEO, is optimistic about Paxalisib’s efficacy in treating GBM.
"There’s been this growing understanding that one of the big challenges with brain cancer is that none of our drugs could get to the tumor," Garner said. "Most cancer drugs don’t go through the blood-brain barrier [the semipermeable layer that separates blood in circulation from the brain] at all. So, if the tumor in the brain is behind the blood-brain barrier, your drug doesn’t actually get there. The special thing about our drug is that it crosses the blood-brain barrier. It does it really well. We know that definitively from a lot of experimental data. Therefore, it’s one of the first drugs that can really get to the tumor.”
Garner said that “there was almost a kind of nihilism about GBM in the drug development community. This was seen as a disease where quite a few companies have tried and failed. That’s why people said, “Brain cancer. You couldn’t pick a tougher disease.”
Garner likens it to the attitude toward melanoma eight to 10 years ago. People were sure it couldn’t be fixed. Garner is encouraged by the fact that there are now about ten new drugs that have been approved for melanoma.
“The outlook for a patient with melanoma has fundamentally changed. We’ve seen the whole birth of immuno-oncology," Garner said. "There’s no reason why brain cancer can’t follow that same trajectory.”
Paxalisib is just one of the drugs Kazia will bring to trial. Its business model is to look for drugs that don’t fit their parent companies. These drugs are among the least likely to be brought to market because they are so costly to develop for the companies that discover them.
"Most big pharma companies produce more drugs than they have the resources and the bandwidth to develop," Garner said. "In years gone by, the ones they didn’t select to take forward would tend to sit on the shelf.”
By focusing on one type of drug – oncology – and one part of the development cycle – the path from trial-to-market – Kazia is well-positioned to contribute to significant advances in oncology treatment.
Published on BioSpace