ORYZON Awarded with a Grant for ORY-4001 From the ALS Association in the U.S.

• To explore Oryzon’s clinical candidate ORY-4001 in ALS
• The ALS Association to provide 500,000 USD funding

MADRID, Spain and BOSTON, Dec. 04, 2023 (GLOBE NEWSWIRE) -- Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with a strong unmet medical need, announced today the start of a preclinical collaboration with the ALS Association. The Association has awarded Oryzon a 498,690 USD grant through its Lawrence and Isabel Barnett Drug Development Program to support the regulatory preclinical development of ORY-4001, a highly selective HDAC6 inhibitor, for amyotrophic lateral sclerosis (ALS).

ALS is a progressive neurodegenerative disease that afflicts approximately 27,000 people in the United States and a comparable number of people in Europe. Approximately 6,300 new cases of ALS are diagnosed each year in the United States. The average life expectancy of a person with ALS is approximately two to five years after diagnosis, and only approximately 10% of people living with ALS survive for more than 10 years. Death is usually due to respiratory failure because of diminished strength in the skeletal muscles responsible for breathing. Few treatment options exist for people living with ALS, resulting in a high unmet need for new therapies to address functional deficits and disease progression.  

HDAC6 inhibition or depletion has been previously described as a potentially effective treatment for ALS, protecting against neurodegeneration in various ALS mouse and human iPSC models. Oryzon recently completed an HDAC6 discovery program, leading to the selection of ORY-4001, a clinical candidate with promising efficacy, selectivity, and safety. ORY-4001 has been shown to improve neuromuscular and neuromotor function, axonopathy, and demyelination in a mouse model of Charcot-Marie Tooth (CMT) type 1, the most common inherited peripheral neuropathy. Due to the key role altered axonal transport and proteostasis play in both CMT and ALS, Oryzon hopes to validate the benefits of inhibiting HDAC6 in ALS mouse models. ORY-4001 is currently advancing in its characterization in IND-enabling toxicology studies.

Dr. Jordi Xaus, Oryzon’s CSO, said: “It is an honor to have the support of the ALS Association. The rationale linking ORY-4001’s mechanism of action to the disease is sound. If these preclinical results are positive, we plan to extend the clinical development of ORY-4001, now in CMT, to include ALS and explore its therapeutical potential for people living with this devastating disease. ORY-4001 would become our second epigenetic program in clinical development in nervous system diseases. This is proof of our capability to efficiently develop, and bring to the clinic, highly selective epigenetic molecules.”

“We need more treatments as urgently as possible to help people living with ALS. We are proud to help drive the crucial transition from preclinical to clinical development for potential new ALS therapies like ORY-4001 through our Lawrence and Isabel Barnett Drug Development Program. Getting promising treatments out of the laboratory and into clinical testing as quickly as possible is key to making ALS a livable disease until we can cure it,” said Kuldip Dave, Ph.D., senior vice president of research at the ALS Association.

Established in 1985, the ALS Association is the largest philanthropic funder of ALS research in the world. The Association funds global research collaborations, assists people with ALS and their families through its nationwide network of certified clinical care centers, and advocates for better public policies for people with ALS. The Association is working to make ALS a livable disease while urgently searching for new treatments and a cure. For more information about the ALS Association, visit .

About Oryzon

Founded in 2000 in Barcelona, Spain, Oryzon (ISIN Code: ES0167733015) is a clinical stage biopharmaceutical company and the European leader in epigenetics, with a strong focus on personalized medicine in CNS disorders and oncology. Oryzon’s team is composed of highly qualified professionals from the pharma industry located in Barcelona, Boston, and San Diego. Oryzon has an advanced clinical portfolio with two LSD1 inhibitors, vafidemstat in CNS and iadademstat in oncology, in several Phase II clinical trials. The company has 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

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