Aprea

Aprea Therapeutics (Boston, USA and Stockholm, Sweden) develops novel anticancer drugs targeting the tumour suppressor protein, p53. Mutations of the p53 gene occur in around 50% of all human tumours and are associated with poor overall survival. Aprea’s candidate drug, eprenetapopt (APR-246), has shown an ability to reactivate mutant p53 protein, inducing programmed cell death in many cancer cells. The company has received a breakthrough therapy and orphan drug designation from the American Food and Drugs Administration, the FDA for eprenetapopt. In 2020, positive results were reported from a phase 2 study of eprenetapopt in combination with azacitidine as post-transplant maintenance therapy in patients with p53-mutated MDS and acute myeloid leukaemia (AML). The relapse free survival at 1-year post-transplant was 58% and the overall survival was 79%. In August 2021, FDA issued a clinical hold for Aprea Therapeutics clinical program. The issue caused a pause in the patient enrolment for the company’s clinical trials. On 9 December, the FDA removed the full clinical hold. In the near future, the company will primarily focus on conducting a pivotal study on MDS/AML post transplantation patients. Aprea is listed on the NASDAQ Global Select Market in the USA since October 2019. Eprenetapopt has the potential for use in many different types of cancer as mutations in p53 are found in around 50% of all diagnosed cancers. The lead target indications thus far include blood tumours such as MDS and AML. MDS is an orphan disease and represents a spectrum of hematopoietic stem cell malignancies. Approximately 30-40% of MDS patients progress to AML and mutations in p53 are found in up to 20% of MDS and AML patients, which is associated with poor overall prognosis.