Aprea has announced positive data from a completed Phase I/II clinical study with its investigational drug APR-246. The results from the study have been published in the Journal of Clinical Oncology.
Based on the positive data, Aprea is planning to advance APR-246 into a Phase II proof-of-concept study in ovarian cancer. Aprea is a Karolinska Development portfolio company.
In the Phase I/II-trial, escalating doses of APR-246 were administered as monotherapy to 22 patients with advanced blood- or prostate cancer during up to four consecutive days.
Dose limiting toxicity was shown at plasma levels well above predicted therapeutic plasma levels.
The study also demonstrated dose-proportional and time-independent pharmacokinetics for APR-246 over the dose range studied.
The results from the study have been published in the Journal of Clinical Oncology.
In addition to reporting that the drug was well tolerated, the authors conclude from the study that APR-246 induces biological effects and that there are cases of clinical effects on tumor burden.
One patient with advanced blood cancer demonstrated a 50 percent reduction in the number of blast cells in the bone marrow.
Ulf Björklund, CEO, Aprea: “These are very encouraging study results. We are now planning to take the p53-activating compound APR-246 forward into a Phase II-trial in epithelial ovarian cancer with mutated p53 in combination with conventional chemotherapy.”
In the new proof-of-concept study, APR-246 will be administered in combination with the reintroduction of a carboplatin-based regimen.
A striking synergistic effect between APR-246 and the very active and frequently used cancer product carboplatin has been observed in pre-clinical experiments.
Torbjörn Bjerke, CEO, Karolinska Development: “This is indeed promising data. Although it was primarily a safety trial, the data indicates that APR-246 has an anti-tumor effect. APR-246 was well tolerated and, importantly, the safety profile is different from traditional cytostatic drugs. With these new results we can see a clear path forward for APR-246, especially in carboplatin-resistant patients.”