The US Food and Drug Administration (FDA) has granted orphan drug designation for Ocelot Bio’s lead candidate OCE-205 to treat ascites, excluding cancer-associated cases.

The FDA grants orphan drug status for therapies targeting rare diseases or ailments that impact fewer than 200,000 people in the US. 

Ascites is an abnormal fluid accumulation in the abdomen’s peritoneal cavity.

A therapeutic peptide, OCE-205 is designed to selectively target the vasopressin 1a receptor, improving outcomes for patients with end-stage liver disease complications.

A Phase II clinical trial assessing the asset to treat hepatorenal syndrome with acute kidney injury has concluded subject enrolment. The company intends to begin clinical trials of OCE-205 for refractory ascites in 2024.

The asset received orphan drug designation from the FDA to treat hepatorenal syndrome last year. 

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​In preclinical research, OCE produced a decline in portal hypertension and amelioration of ascites volume, backing its potential to treat complications of decompensated cirrhosis, such as ascites.

Ocelot Bio chief medical officer Lise Kjems stated: “The FDA granting orphan drug designation for OCE-205 in ascites is a validation of the tremendous need for improved therapies offering novel approaches for patients and supports our clinical focus on this important indication. 

“This milestone serves as a catalyst as we work with patients, healthcare professionals and health authorities to pave a brighter path forward for these patients who have limited treatment options. 

“We are well positioned to further advance our clinical development programme for OCE-205 and look forward to progressing this programme in ascites.”

Ocelot Bio focuses on the development of drugs to boost outcomes for patients with liver disease.

The company is supported by an investor syndicate comprising Venrock, RA Capital Management and Vivo Capital.

Orphan drug status benefits Ocelot Bio with monetary incentives for clinical development and possible market exclusivity for up to seven years for the therapy subject to approval.