FDA grants breakthrough therapy designation to APR-246, azacitidine combination for myelodysplastic syndrome
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The FDA granted breakthrough therapy designation to APR-246 in combination with azacitidine for the treatment of patients with myelodysplastic syndromes with a susceptible TP53 mutation, according to a company-issued press release.
APR-246 (Aprea Therapeutics) is an investigational small molecule therapy that targets the tumor suppressor protein, p53. The agent has demonstrated preclinical and antitumor activity for a variety of cancers, including myelodysplastic syndrome, acute myeloid leukemia and ovarian cancer.
Azacitidine — an antimetabolite that inhibits cells from making DNA and may kill abnormal blood cells or cancer cells — is commercially approved by the FDA for the treatment of myelodysplastic syndrome.
“Outcomes for [patients with myelodysplastic syndrome] with a TP53 mutation are poor and there are no current therapeutic options specifically for these patients,” Christian S. Schade, CEO of Aprea, said in a company-issued press release. “We look forward to continued interaction with FDA regarding our ongoing phase 3 clinical study and our clinical development program to advance APR-246.”
APR-246 received FDA’s fast track and orphan drug designations last April, as previously reported by Healio.
A pivotal phase 3 clinical trial of APR-246 plus azacitidine for myelodysplastic syndrome is currently enrolling patients. APR-246 also is currently in phase 2 trials for AML and as maintenance therapy after hematopoietic stem cell transplantation for myelodysplastic syndrome and AML.