Kazia adjusts failed brain cancer trial data for FDA submission

Kazia Therapeutics has unveiled a revised survival analysis from a late-stage brain cancer trial, aiming to salvage positive outcomes after an initial failure. By adjusting the control cohort, the biotech company hopes to present data that could potentially open a pathway to accelerated approval from the FDA.

The data originates from a platform study sponsored by the Global Coalition for Adaptive Research, targeting patients with newly diagnosed and recurrent glioblastoma, a notoriously challenging brain cancer. This study compares multiple experimental treatments against a standard-of-care control arm. Notable participants include prominent pharmaceutical companies like Bayer and Biohaven.

In August 2022, Kazia announced that their PI3K/mTOR inhibitor, discovered by Genentech, did not meet the required criteria for progression in the trial. Despite this setback, Kazia now suggests there is a favorable narrative within the data. Two subgroups of patients were treated with Kazia’s drug candidate, paxalisib. In the first subgroup, consisting of patients with recurrent glioblastoma, paxalisib showed poorer results than the control drug, lomustine. Specifically, the median overall survival (OS) for patients on paxalisib was 8.05 months, compared to 9.69 months for those on the standard treatment. This part of the study involved 213 patients, with a near-even split between the two treatments.

Kazia has shifted its focus to the second subgroup, which included newly diagnosed patients with a glioblastoma variant less responsive to temozolomide, a common chemotherapy drug. Here, paxalisib achieved a median OS of 14.77 months in 54 patients, slightly better than the 13.84 months for those in the temozolomide control group. However, by examining a specific subset of the control group, Kazia presents a more optimistic picture. As paxalisib was introduced to the trial 18 months after it began, about one-third of the 75 newly diagnosed patients in the control arm had been enrolled before Kazia’s candidate was administered.

By narrowing the control group to the 46 patients who participated concurrently with the administration of paxalisib, the median OS was shown to be 11.89 months. This adjustment resulted in what Kazia CEO John Friend, M.D., described as a 3.8-month improvement in overall survival, or approximately a 33% enhancement.

With this refined data, Kazia plans to engage in discussions with the FDA to explore the possibility of accelerated approval for paxalisib. The revised survival figures have had a significant impact on Kazia’s market performance, with the company’s share price surging by approximately 70% in premarket trading, though it still stands at just 33 cents.

In summary, by re-evaluating the control cohort, Kazia Therapeutics has identified a potential positive outcome from their initially unsuccessful brain cancer trial. This new analysis will be pivotal in their upcoming discussions with the FDA, as they seek to determine whether there is a feasible route to accelerated approval for paxalisib.

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