ORY-4001 / Oryzon - LARVOL DELTA

Earlier stage programs (Oryzon Press Release) - "ORY-4001, Oryzon’s highly selective histone deacetylase 6 (HDAC6) inhibitor for neurological disorders, continues IND-enabling studies to enable future clinical trials. The program remains focused on potential applications in Amyotrophic Lateral Sclerosis (ALS), Charcot-Marie-Tooth disease (CMT) and other neurological disorders"

Preclinical • Amyotrophic Lateral Sclerosis • Genetic Disorders

ORYZON raises €12 million and signs a financing agreement with COFIDES to strengthen its balance sheet and accelerate its clinical programs (Oryzon Press Release) - "The funds raised will be used to: Strengthen the Company's balance sheet to support corporate development initiatives in anticipation of future partnership discussions; Advance the clinical development of iadademstat for the treatment of acute myeloid leukemia; Progress the other ongoing clinical programs in hematology and psychiatry; Cover general and administrative expenses and financial obligations....This commitment will apply to any future capital increase that Oryzon may undertake within six months of the execution of the share subscription agreement. This period may be extended by mutual agreement between the parties."

Financing • Acute Myelogenous Leukemia • Amyotrophic Lateral Sclerosis • CNS Disorders

ORY-4001, A Potent And Selective Inhibitor Of HDAC6, Improves ALS-Related Pathology In Different Models Of ALS. (ENCALS 2026) - "Finally, human ALS patient-derived lymphocytes treated with ORY-4001 partially reverted TDP-43 mislocalization and reduced the formation of cytoskeletal protrusions. Conclusion ConclusionsORY-4001, a novel oxadiazole-based HDAC6 inhibitor with good selectivity and safety profiles, showed in vivo efficacy in different ALS experimental models, regardless of their genetic background."

Amyotrophic Lateral Sclerosis • CNS Disorders • FUS • TARDBP

Earlier stage programs (Oryzon Press Release) - "ORY-4001, Oryzon’s highly selective histone deacetylase 6 (HDAC6) inhibitor nominated as a clinical candidate for the treatment of certain neurological diseases such as Charcot-Marie-Tooth disease (CMT), Amyotrophic Lateral Sclerosis (ALS) and others, continues to progress through IND enabling studies to prepare it for clinical studies."

Clinical • Amyotrophic Lateral Sclerosis • Genetic Disorders