Aprea Therapeutics Inc.

• US
• Aprea Therapeutics Inc.
• Health-Care

APRE

 We are a clinical-stage biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate mutant p53 tumor suppressor protein. p53 is the protein expressed from the TP53 gene, the most commonly mutated gene in cancer. We believe that mutant p53 is an attractive therapeutic target due to the high incidence of p53 mutations across a range of cancer types and its involvement in key cellular activities such as apoptosis.

Cancer patients with mutant p53 face a significantly inferior prognosis even when treated with the current standard of care, and a large unmet need for these patients remains. Our lead product candidate, APR-246, is a small molecule p53 reactivator that is in late-stage clinical development for hematologic malignancies, including myelodysplastic syndromes, or MDS, and acute myeloid leukemia, or AML. APR-246 has received Orphan Drug and Fast Track designations from the FDA for MDS, and Orphan Drug designation from the EMA for MDS, AML and ovarian cancer, and we believe APR-246 will be a first-in-class therapy if approved by applicable regulators. We have commenced a pivotal Phase 3 trial of APR-246 with azacitidine for frontline treatment of TP53 mutant MDS and expect initial data from this trial in the second half of 2020. Our pivotal Phase 3 trial is supported by data from two ongoing Phase 1b/2 investigator initiated trials, one in the U.S. and one in France, testing APR-246 with azacitidine as frontline treatment in TP53 mutant MDS and AML patients.

        In the ongoing U.S. Phase 1b/2 trial, sponsored by Dr. David Sallman of the H. Lee Moffitt Cancer Center and Research Institute, or Moffitt, we have observed an objective response rate, or ORR, of 88% and a complete remission, or CR, rate of 59% in 41 response-evaluable MDS/AML patients treated with APR-246 and azacitidine, as of July 2019 and we expect a final CR endpoint in the second half of 2019. In MDS patients, the CR rate was 61%, with an additional 27% achieving non-CR responses.

In AML patients, the CR rate was 50%, with an additional 38% achieving non-CR responses. In addition, 46% of evaluable patients were able to discontinue treatment in order to proceed to allogeneic hematopoietic stem cell transplantation, or allo-HCT. Allo-HCT is currently the only recognized therapy believed to increase the likelihood of long term survival for TP53 mutant MDS and AML patients in remission. In the ongoing French Phase 1b/2 trial, sponsored by Groupe Francophone des Myelodysplasies, or GFM, under lead investigator Prof. Pierre Fenaux, we have observed an ORR of 75% and a CR rate of 56% in 16 response-evaluable patients treated with APR-246 and azacitidine, as of July 2019 and we expect a final CR endpoint in the first half of 2020. In MDS patients, the CR rate was 58%, with an additional 17% achieving non-CR responses. In AML patients, the CR rate was 50%, with an additional 25% achieving non-CR responses. We expect the results of our pivotal Phase 3 trial and the U.S. and French Phase 1b/2 trials will be submitted in support of marketing applications to the FDA and EMA.

We are also developing a next-generation small molecule p53 reactivator, APR-548, for potential use in multiple hematologic malignancies and other oncologic indications, and expect to file an IND with the FDA in the first half of 2020. We have assembled a management team with extensive experience in the discovery and development of novel oncology drugs. Our management team is supported by our world-class scientific advisors and leading life science investors in their mission to successfully develop p53-reactivating therapies for cancer patients. 

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