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Aprea’s ovarian cancer drug candidate APR-246 gets European orphan drug status

Swedish biotech company Aprea has secured orphan drug designation from the European Medicines Agency (EMA) for its anti-cancer agent APR-246 to treat ovarian cancer.

Currently, the company is conducting a Phase Ib/II trial of APR-246 in combination with carboplatin (AUC 5) and pegylated doxorubicin (30 mg/m2), a second line standard of care chemotherapy in patients with relapsed platinum sensitive high-grade serous ovarian cancer.

The company said that APR-246 has the potential to restore p53 activity in patients where p53 is non-functional or mutated.

Aprea CEO Bruno Lucidi said: "To the company’s knowledge, APR-246 is the only compound in clinical development that can restore normal function of the p53 protein and thereby induce efficient cancer cell death.

"The orphan drug designation offers significant benefits during the forthcoming development and commercialisation of this potentially ground-breaking treatment."

Previously, APR-246 has been tested in a first-in-human dose finding Phase I study as a single agent in a total of 32 patients.

Based on the results from this trial, the company claimed that the safety and pharmacokinetic profile of APR-246 allows for combination with standard of care chemotherapy.

In addition, preclinical ex vivo studies with human ovarian cancer cells treated with APR-246 in combination with chemotherapy showed clear synergistic effects of the two treatments combined.