Curis (CRIS) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Strategic focus and pipeline progress

• Emphasis on IRAK4 as a novel target in AML, NHL, and solid tumors, with growing academic and industry interest.

• Proof-of-concept data sets in AML and NHL expected to reach 15–20 patients by year-end, expanding from initial smaller cohorts.

• Ongoing efforts to identify the fastest regulatory approval paths for both AML and NHL indications.

• Frontline AML study initiated to assess safety of IRAK4 inhibitor in combination with standard of care, with efficacy focus to follow.

• Five investigator-sponsored trials in solid tumors supported by NCI, though current investor focus is on hematologic malignancies.

AML program updates

• Monotherapy in FLT3-mutated AML showed a 30% CR/CRh rate in 11 patients, surpassing the 21% benchmark of gilteritinib, even in a more heavily pretreated population.

• Targeting a 20-patient FLT3 data set by year-end to confirm efficacy and durability, with results to be presented at ASH.

• Combination (triplet) therapy with emavusertib, azacitidine, and venetoclax aims to improve standard of care; initial safety data expected from 5–6 patients by year-end.

• Frontline AML study design includes a safety run-in for patients with residual disease, followed by a broader efficacy study starting from cycle one.

• Ongoing FDA discussions for registrational pathways, with potential for accelerated approval based on ORR as a primary endpoint.

NHL program and regulatory strategy

• IRAK4 inhibition in NHL targets both BCR and toll-like receptor pathways, aiming to improve outcomes over BTK inhibitors alone.

• Primary CNS lymphoma identified as a lead indication due to high unmet need and regulatory attractiveness; early data show ~50% response rate and >4 months duration in initial patients.

• FDA engaged in real-time discussions on pivotal study design, with a single-arm, 45–60 patient study proposed for accelerated approval.

• By year-end, 15–20 patients expected on study, with existing patients potentially counting toward pivotal trial enrollment.

• Market opportunity in NHL is significant, with BTK inhibitor market at $11 billion and potential for emavusertib as an add-on therapy.