Ryvu Therapeutics (RVU) investor relations material

Pipeline and clinical development

• Lead asset romaciclib (RVU120), a first-in-class oral CDK8/19 inhibitor, is in Phase II for AML and myelofibrosis, with promising efficacy and safety data, including 43% CR/CRi in VEN-refractory AML and spleen volume reductions in MF.

• RVU305, a best-in-class, brain-penetrant PRMT5 inhibitor, is advancing through IND-enabling studies, showing superior potency and efficacy in MTAP-deleted cancers and strong CNS penetration.

• Dapolsertib, a dual PIM/FLT3 inhibitor, is in Phase II for DLBCL in partnership with Menarini, with additional opportunities in hematologic malignancies.

• ONCO Prime platform is generating novel precision oncology targets, including synthetic lethal targets, with initial data in colorectal cancer and collaborations with BioNTech and Exelixis.

• Novel ADC payloads and immunocytotoxic strategies are being developed, including STING-activating ADCs, with multiple first-in-class candidates advancing.

Clinical results and regulatory progress

• Romaciclib demonstrated the ability to overcome VEN resistance in AML, with a favorable safety profile and no significant increase in myelosuppression.

• In myelofibrosis, romaciclib showed clinically meaningful spleen volume reductions and symptom improvements, especially in patients with ASXL1 and CALR mutations.

• FDA Type C meeting in January 2026 provided guidance for U.S. expansion of romaciclib trials, with pivotal studies planned for 2026 and potential approval as early as 2028.

• Medulloblastoma Phase I study in children is fully financed and set to start in 2026, with strong preclinical rationale and parallel U.S. study in preparation.

Market opportunity and financials

• High unmet need in AML and MF, with global AML and MF markets projected to reach $6.6B and $2.9B by 2031, respectively.

• Romaciclib positioned to set a new standard of care in post-VEN AML and post-JAKI MF, with strong commercial potential.

• 2025 revenues were €20.7M, with €14.7M from partnerships and €6.0M from grants; cash position as of March 2026 was €20.7M, plus €20.9M in non-dilutive grant funding.

• Key partnerships with BioNTech and Exelixis have brought significant upfront payments, research funding, and milestone opportunities exceeding €50M and $400M, respectively.