Oryzon Genomics (ORY) investor relations material

Epigenetic drug development and clinical pipeline

• Focus on potent, selective LSD1 inhibitors for oncology, hematology, and CNS diseases, with well-defined registrational pathways and multiple ongoing Phase I-III trials.

• ladademstat shows strong efficacy in unfit 1L AML (100% ORR, 90% CRs) and is being tested in various combinations for AML, MDS, SCD, ET, and SCLC, leveraging collaborations with NCI and top-tier US institutions.

• Vafidemstat is Phase III-ready for aggression in BPD, with ongoing trials in schizophrenia and ASD, and demonstrated significant reduction in agitation/aggression in PoC studies.

• ORY-4001, a selective HDAC6 inhibitor, is advancing for ALS and CMT, with preclinical efficacy and first-in-man readiness expected in 2H2026.

• Robust financial position with over $60 million raised in 2025, providing cash runway through 1H 2027.

Clinical data and regulatory strategies

• ladademstat achieved 100% ORR and 90% CR in 1L AML, with final ALICE-2 data expected Q4 2026 and a seamless Phase II-III ALICE-3 trial planned for accelerated and full approval.

• RESTORE Phase Ib in SCD is recruiting, with PoC data expected in 2026 and a registrational RESTORE-2 trial planned for 2027.

• IDEAL Phase II trial in ET approved by EMA, planned to start 1Q2026, aiming to follow positive results from competitor LSD1i.

• Vafidemstat's PORTICO Phase III protocol for BPD submitted to FDA, incorporating STAXI-2 Trait Anger and OAS-M as endpoints, with protocol resubmission targeted before year-end.

• EVOLUTION Phase IIb trial in schizophrenia is ongoing, with EU expansion in 2026 and readout expected in 2H2027.

Market opportunities and competitive positioning

• Significant unmet needs in AML, SCD, ET, BPD, schizophrenia, and ASD, with large addressable patient populations and high commercial potential (e.g., BPD market >$3Bn, schizophrenia drug market >$10Bn).

• ladademstat and vafidemstat are positioned as first- or best-in-class assets, with broad applicability across multiple indications and strong safety profiles.

• Strategic collaborations with leading pharma and biotech companies, and engagement with world-renowned clinical and regulatory experts.

• Ongoing expansion of aggression program in ASD and rare neurodevelopmental disorders, supported by EU grants.

• ORY-4001 available for partnering, with grant support from the ALS Association.