ORYZON to present positive preclinical efficacy data of ORY-4001 in Charcot-Marie-Tooth (CMT) disease at the 2023 Peripheral Nerve Society (PNS) Annual Meeting

Oryzon Genomics, S.A., a clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with a strong unmet medical need, announced today that it will present positive preclinical efficacy data of ORY-4001 in a CMT model at the 2023 Peripheral Nerve Society (PNS) Annual Meeting, which will be held June 17-20 at the Bella Center in Copenhagen, Denmark. The scientific committee of the 2023 PNS Annual Meeting has selected “ORY-4001, a Novel Potent and Selective Oxadiazole-Based HDAC6 Inhibitor Shows Pre-Clinical Therapeutic Efficacy in CMT1A” for an oral presentation, which will take place on Tuesday, June 20, at 08:40 am CEST.

In 2022, Oryzon and the (CMTRF), a U.S.-based patient-led, non-profit organization focused on delivering treatments and cures for CMT, entered into an agreement to explore the therapeutic potential of Oryzon’s histone deacetylase 6 (HDAC-6) inhibitors. Following positive preclinical results obtained under this collaboration, Oryzon selected a candidate for clinical development, ORY-4001. This compound is a HDAC6 inhibitor with excellent pharmacology and very high selectivity against other HDAC classes, resulting in a remarkable safety profile avoiding hematotoxicity. The compound exhibits strong anti-inflammatory properties and positive in-vivo data in inflammatory models. In the study to be presented at the 2023 PNS Annual Meeting, ORY-4001 showed multiple positive responses in a validated CMT1A peripheral neuropathy in vivo model which effectively mimics many of the symptoms of this condition in humans.

Dr. Jordi Xaus, Oryzon’s CSO, said: “Although this is only a first proof-of-concept preclinical model of a peripheral neuropathy, the robustness of the data obtained in this CMT1A model is really encouraging and prompts us to intensify our efforts to pursue IND enabling studies to support first-in- human administration of ORY-4001 in the following quarters.”

CMT is a progressive, degenerative disease that causes damage to the peripheral nerves. It affects 150,000 Americans and more than 3 million people around the world. It is one of the most prevalent conditions among rare diseases and currently lacks effective treatments or cures. CMT is caused by a variety of genetic mutations. CMT1A is the most prevalent form, accounting for approximately half of all people with CMT.

For more information about PNS-2023, please visit the PNS-2023 website.

About Oryzon

Founded in 2000 in Barcelona, Spain, Oryzon (ISIN Code: ES0167733015) is a clinical stage biopharmaceutical company considered as the European leader in epigenetics. Oryzon has one of the strongest portfolios in the field, with two LSD1 inhibitors, iadademstat and vafidemstat, in Phase II clinical trials, and other pipeline assets directed against other epigenetic targets. In addition, Oryzon has a strong platform for biomarker identification and target validation for a variety of malignant and neurological diseases. For more information, visit www.oryzon.com

About ORY-4001

ORY-4001 is a highly selective histone deacetylase 6 (HDAC-6) inhibitor. Due to its positive pharmacological properties, it was recently nominated as a clinical development candidate for the treatment of certain neurological diseases such as Charcot-Marie-Tooth disease (CMT), Amyotrophic Lateral Sclerosis (ALS), and others. HDAC6 inhibitors have been previously proposed as potentially effective treatments for CMT, ALS, and other neurological disorders that lack effective treatments.

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