APRE

[COMPANY] Announces Results of Primary Endpoint from Phase 3 Trial of [DRUG] in TP53 Mutant Myelodysplastic Syndromes (MDS)

[DATE]

The trial failed to meet its primary endpoint of complete remission (CR) rate
CR rate was 53% higher in [DRUG] with AZA arm compared to AZA alone, but did not reach statistical significance
[COMPANY], a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that target the mutant tumor suppressor protein, p53, today announced results of the primary data cut from its Phase 3 clinical trial evaluating the safety and efficacy of [DRUG] with azacitidine (AZA) versus AZA alone in TP53 mutant myelodysplastic syndromes (MDS). The trial did not meet the predefined primary endpoint of complete remission (CR) rate. Analysis of the primary endpoint at this data cut demonstrated a higher CR rate in the experimental arm receiving [DRUG] with AZA versus the control arm receiving AZA alone, but did not reach statistical significance. In the intention-to-treat population of 154 patients, the CR rate in the [DRUG] with AZA arm was 33.3% (95% CI: 23.1% - 44.9%) compared to 22.4% (95% CI: 13.6% - 33.4%) in the AZA alone arm (P = 0.13).

While analysis of certain secondary endpoints (ORR and duration of responses) appears to favor the experimental arm at this data cut, they are not significantly different. The median duration of overall survival at the primary data cut was similar between the arms. Additional patients in the study who have not achieved a CR remain on study treatment and the data will be analyzed at future pre-specified timepoints as set forth in the statistical analysis plan. The combination of [DRUG] with AZA appeared well-tolerated, with an adverse event profile that was similar to the Company's prior Phase 2 clinical trials. Subsequent analyses of the trial data, including secondary endpoints, will be conducted as the duration of patient follow-up increases. The Company expects to present the data at a future scientific conference.

"Though we are disappointed the topline results did not reach statistical significance, we continue to believe that [DRUG] can offer clinical benefit to patients with TP53 mutant malignancies," said [EXECUTIVE], Chief Medical Officer of [COMPANY]. "We will continue to analyze data as it matures and follow patients who are still receiving study treatment. Our other clinical trials continue to progress and we remain committed to pursuing our clinical development programs."

About the Phase 3 Trial in TP53 Mutant MDS

The Phase 3 trial enrolled 154 TP53 mutant MDS patients, randomized 1:1 to either the [DRUG] with AZA arm or the AZA alone arm. Response criteria are those defined by International Working Group 2006 (IWG 2006) and include measures of peripheral blood counts and bone marrow blasts.

About p53, [DRUG] and [DRUG_2]

The p53 tumor suppressor gene is the most frequently mutated gene in human cancer, occurring in approximately 50% of all human tumors. These mutations are often associated with resistance to anti-cancer drugs and poor overall survival, representing a major unmet medical need in the treatment of cancer.

[DRUG] is a small molecule that has demonstrated reactivation of mutant and inactivated p53 protein – by restoring wild-type p53 conformation and function – thereby inducing programmed cell death in human cancer cells. Pre-clinical anti-tumor activity has been observed with [DRUG] in a wide variety of solid and hematological cancers, including MDS, AML, and ovarian cancer, among others. Additionally, strong synergy has been seen with both traditional anti-cancer agents, such as chemotherapy, as well as newer mechanism-based anti-cancer drugs and immuno-oncology checkpoint inhibitors. In addition to pre-clinical testing, a Phase 1/2 clinical program with [DRUG] has been completed, demonstrating a favorable safety profile and both biological and confirmed clinical responses in hematological malignancies and solid tumors with mutations in the TP53 gene.

A pivotal Phase 3 clinical trial of [DRUG] and azacitidine for frontline treatment of TP53 mutant MDS has been completed and additional clinical trials in hematologic malignancies and solid tumors are ongoing. [DRUG] has received Breakthrough Therapy, Orphan Drug and Fast Track designations from the FDA for MDS, Fast Track designation from the FDA for AML, and Orphan Drug designation from the European Medicines Agency for MDS, AML and ovarian cancer.

[DRUG_2] is a next-generation small molecule p53 reactivator. [DRUG_2] has demonstrated high oral bioavailability, enhanced potency relative to [DRUG] in TP53 mutant cancer cell lines and has demonstrated in vivo tumor growth inhibition following oral dosing of tumor-bearing mice. Enrollment in a Phase 1 clinical trial of [DRUG_2] is anticipated to begin in [DATE].