Kazia Therapeutics Announces Successful Endpoint in Phase I Trial

On February 21, 2024, Kazia Therapeutics Limited, an Australian biotech firm listed on NASDAQ (KZIA), announced the premature completion of a Phase I clinical trial due to favorable safety outcomes and clinical responses. The study focused on the use of paxalisib, an oral medication that inhibits both PI3K and mTOR, alongside radiation therapy for patients with brain metastases from solid tumors that have a PI3K pathway mutation.

The first part of the trial determined the maximum tolerated dose (MTD) of paxalisib when combined with radiation and showed potential clinical benefits in all nine patients assessed. The second part expanded the cohort to further study the MTD's safety and efficacy in up to 12 more patients. Preliminary results from Part II showed that the study's primary endpoint was met, with continued positive clinical responses observed.

Kazia's CEO, Dr. John Friend, expressed optimism regarding the results, which indicate both safety and potential effectiveness of paxalisib combined with radiation therapy. The company plans to consult with the FDA to discuss the findings and seek guidance for a pivotal study to further the development of paxalisib as a more effective treatment for brain metastases.

Research has indicated that the PI3K pathway is often activated in brain metastases, potentially causing resistance to radiation treatment. This provides a rationale for combining paxalisib with radiation therapy to enhance the sensitivity of tumor cells to radiation. Paxalisib received Fast Track Designation from the FDA in the previous year based on the initial clinical data for treating brain metastases with PI3K pathway mutations.

In the United States, around 200,000 cancer patients develop brain metastases annually, with radiotherapy being the primary treatment method. However, the effectiveness of whole brain radiotherapy (WBRT) varies, and the need for new treatment options is evident due to the increasing prevalence and low response rates to current treatments.

Kazia Therapeutics is also developing EVT801, a VEGFR3 inhibitor, which has shown activity against various tumor types and potential synergy with immuno-oncology drugs. A Phase I study for EVT801 is underway, with preliminary data expected in 2024.

The company's lead product, paxalisib, has received several designations from the FDA, including Orphan Drug Designation for glioblastoma and Fast Track Designation for specific brain cancer treatments. 

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!