- RVU305, a potentially best-in-class, brain-permeable MTA-cooperative PRMT5 inhibitor, demonstrates strong and selective antitumor efficacy in MTAP-deleted glioblastoma models, with confirmed brain exposure and a favorable safety profile supporting IND/CTA-enabling studies.
- ONCO Prime - an integrated synthetic lethality discovery platform leveraging patient-driven models, engineered systems, and high-throughput CRISPR/omics analytics – uncovers the identification of novel targets across various cancer types, including colorectal cancer (CRC) in genetically defined patient populations.
KRAKOW, Poland, October 27, 2025 / Biotech Newswire / -- Ryvu Therapeutics (WSE: RVU), a clinical-stage drug discovery and development company focusing on novel therapies that address emerging targets in oncology, is presenting preclinical data on RVU305 (PRMT5i) and the ONCO Prime Platform at the 2025 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics, October 22-26, 2025, in Boston, MA.
"We are excited to share new data that underscores the strength and breadth of Ryvu’s novel targets platform - ONCO Prime. Our preclinical findings demonstrate how rational design and deep biological understanding can lead to differentiated therapeutic candidates such as RVU305, as well as novel discovery platforms that uncover cancer-specific vulnerabilities. These advances reinforce our commitment to developing precision medicines that address significant unmet needs in oncology.” said Krzysztof Brzózka, Ph.D., Chief Scientific Officer of Ryvu Therapeutics.
Details on the poster presentations are as follows:
Poster Title: “RVU305, a brain-penetrant MTA-cooperative PRMT5 inhibitor, shows efficacy in GBM preclinical models”
Poster Number: B037
Session date and time: Friday, October 24, 12:30 PM - 4:00 PM EST
RVU305, a potentially best-in-class, brain-permeable MTA-cooperative PRMT5 inhibitor, demonstrates significant promise in the treatment of MTAP-deleted cancers. Preclinical studies show that RVU305 selectively inhibits PRMT5 activity in MTAP-deleted cells, inducing a strong synthetic lethal effect while sparing regular counterparts. Mechanistically, RVU305 causes a dose-dependent reduction in SDMA-modified proteins, validating PRMT5 inhibition at the molecular level. Moreover, RVU305 delivers selective inhibition of PRMT5 in MTAP-deleted tumor cells. Its BID oral dosing and MTA-cooperative mechanism enable potent, on-target tumor inhibition while sparing healthy tissue. This translates into >100% tumor growth inhibition and multiple complete responses in MTAP-deleted models.
RVU305 demonstrates potent antiproliferative activity across multiple MTAP-deleted glioblastoma (GBM) cell lines with minimal effects on MTAP wild-type lines. In vivo, RVU305 showed significant tumor growth inhibition (TGI) and good tolerability in an orthotopic U87-LUC glioblastoma mouse model. RVU305 demonstrated CNS penetration with predicted efficacious exposure in the brain of cynomolgus monkeys. Kp,uu modeling indicates brain target coverage significantly superior to clinical stage comparator.
Together, these findings position RVU305 as a promising therapeutic candidate capable of delivering targeted, brain-penetrant efficacy for MTAP-deleted gliomas, addressing a critical unmet medical need in GBM treatment. GLP toxicology studies for RVU305 have been completed with no major toxicology findings and favorable safety profile which supports planned completion of IND/CTA-enabling studies in Q4 2025.
Ryvu Therapeutics S.A. has received the European Union grant for the phased implementation of the project “New targeted therapy for tumors with MTAP gene deletion – Phase II”. The EU funding for Phase II will span 2023-2028 under grant agreement no.: FENG.01.01-IP.01-1012/23.
Poster Title: “Identification of novel molecular vulnerabilities in colorectal cancer through integrated transcriptomic profiling and functional genomics”
Poster Number: B050
Session date and time: Friday, October 24, 12:30 PM - 4:00 PM EST
This study describes the development of a comprehensive discovery platform designed to identify synthetic lethal (SL) interactions linked to major oncogenic drivers in colorectal cancer (CRC), including APC, KRAS, and SMAD4. By combining CRISPR-based functional screening with genomic and transcriptomic analyses across human intestinal stem cell (hISC)-derived isogenic models, patient-derived cells (PDCs), and clinical tumor specimens, the team uncovered novel molecular vulnerabilities specific to genetically defined CRC subtypes. Machine learning applied to RNA sequencing data enabled precise molecular subtyping and validation of model fidelity, while candidate targets were selected based on therapeutic relevance and selectivity for cancer cells. Through this integrated approach, three categories of actionable targets were identified:
- first-in-class synthetic lethal targets currently under active drug discovery and optimization,
- next-wave novel dependencies representing promising directions for future oncology programs,
- novel indications for FDA-approved drugs supporting opportunities for drug repurposing.
Together, these findings establish a robust framework for precision oncology, supporting the discovery of new targeted therapies for CRC and offering broad applicability to other cancer types.
The ONCO Prime project is co-financed by the European Union under the Operational Programme European Funds for Modern Economy 2021-2027. Project title: "ONCO Prime: new possibilities for personalised anti-cancer therapy based on patient-derived primary cell cultures, omics characterisation, and functional assays". Grant Agreement no: FENG.01.01-IP.02-0095/23.
All posters are now available online and can be obtained from the conference site:
https://www.aacr.org/ and https://ryvu.com/
About Ryvu Therapeutics
Ryvu Therapeutics is a clinical-stage drug discovery and development company focused on novel oncology therapies that address emerging targets in oncology. Internally discovered pipeline candidates at Ryvu use diverse therapeutic mechanisms driven by emerging knowledge of cancer biology, including small molecules and antibody-drug conjugates directed at kinases, synthetic lethality, and immuno-oncology targets.
Ryvu’s most advanced program is romacyclib (RVU120, SEL120), a selective CDK8/CDK19 kinase inhibitor with the potential to treat hematological malignancies. RVU120 is currently in Phase II development (i) in combination with venetoclax for the treatment of patients with r/r AML – the RIVER-81 study, (ii) as a monotherapy for the treatment of patients with lower-risk myelodysplastic syndromes (LR-MDS) – the REMARK study, (iii) as a monotherapy and in combination with ruxolitinib for the treatment of patients with myelofibrosis (MF) – the POTAMI-61 study. Dapolsertib (MEN1703, SEL24) is a dual PIM/FLT3 kinase inhibitor licensed to the Menarini Group that is currently being investigated in a Phase II study in diffuse large B-cell lymphoma (DLBCL) – the JASPIS-01 study. RVU305, a potentially best-in-class, brain-permeable PRMT5 inhibitor aiming to treat multiple solid tumors, is currently in IND/CTA-enabling studies. Ryvu Therapeutics is also engaged in oncology collaborations with BioNTech and Exelixis.
Ryvu was founded in 2007 and is headquartered in Kraków, Poland. It is listed on the Warsaw Stock Exchange and is a component of the sWIG80 index.
Contact
Ryvu Therapeutics
Anna Wilk
+48 532 698 425
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Keywords: Congresses as Topic; Glioblastoma; Synthetic Lethal Mutations; Brain; Colorectal Neoplasms; Ryvu Therapeutics; RVU305; PRMT5 inhibitor; MTA-cooperative PRMT5i; brain-permeable inhibitor; MTAP-deleted cancers; synthetic lethality; antitumor efficacy; CNS penetration; IND/CTA-enabling studies; AACR-NCI-EORTC International Conference; ONCO Prime Platform; high-throughput CRISPR analytics; omics characterization; precision oncology; drug discovery and development; oncology therapeutics; Krzysztof Brzózka; preclinical data; poster presentation; tumor growth inhibition; SDMA-modified proteins; PRMT5 inhibition validation; MTA-deleted gliomas; CRISPR-based functional screening; drug repurposing; high-impact oncology research
Source: Biotech Newswire