Repare Therapeutics (RPTX) Goldman Sachs 45th Annual Global Healthcare Conference summary

Company overview and platform strategy

• Focuses on CRISPR-enabled discovery to identify novel synthetically lethal targets for precision oncology, aiming to selectively target tumor cells based on genetic alterations.

• Pipeline includes three clinical compounds (PKMYT1, ATR, PLK4) and a fourth (Polθ inhibitor) expected to enter the clinic later in 2024.

• Proprietary STEP2 platform expands the range of genetic alterations targeted, increasing patient populations for clinical trials.

• Demonstrated ability to identify additional biomarkers (e.g., PPP2R1A, FBXW7) that predict response to PKMYT1 inhibition, broadening efficacy exploration.

Clinical program updates and data outlook

• Five key clinical readouts for lunresertib (PKMYT1 inhibitor) are expected within the next four quarters, including ovarian and endometrial cancer arms, WEE1, gemcitabine, and FOLFIRI combinations.

• Early data in gynecological tumors showed a 50% RECIST response rate in a small cohort, with expanded studies ongoing.

• FOLFIRI combination data will be presented at ESMO GI in June, with promising tolerability and potential for significant PFS benefit.

• Only one positive result among the five readouts is needed to pursue a registration path in 2025.

• Biomarker gamma H2AX and circulating tumor DNA responses correlate with clinical response, supporting the approach.

Clinical development strategy and investigator engagement

• Over 100 patients have been treated with lunresertib, with clear evidence of activity in both monotherapy and combination settings.

• Investigator enthusiasm is high, enabling rapid patient enrollment and strong support for novel strategies.

• Tolerability profile for lunresertib combinations is favorable, with anemia as the main hematologic concern, managed by tailored dosing schedules.

• Protocols allow inclusion of patients with low hemoglobin, ensuring broad trial access.