Ocelot Bio has received the Orphan Drug Designation from the US Food and Drug Administration (FDA) for its lead candidate OCE-205 to treat ascites due to all etiologies except cancer.
Ocelot is a clinical-stage biopharmaceutical firm dedicated to the development of therapies for complications of end-stage liver disease (ESLD).
The US FDA grants this status to drugs that target rare diseases or ailments that impact fewer than 200,000 people in the US.
OCE-205 is a therapeutic peptide with a differentiated mechanism of action. It is designed to relieve portal hypertension while offering a capped maximal efficacy.
In preclinical studies, it has shown a decrease in portal hypertension and amelioration of ascites volume.
The biopharmaceutical company plans to start clinical trials of OCE-205 in refractory ascites next year.
Ocelot Bio chief medical officer Lise Kjems said: “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 benefits from Orphan Drug status, which includes financial incentives to support clinical development and the possibility of up to seven years of market exclusivity for the drug in the US for the designated orphan indication.
This represents the second Orphan Drug Designation for the asset, which previously secured the status in hepatorenal syndrome in 2022.
Additionally, a Phase 2 clinical study has completed enrolment to assess OCE-205 to treat hepatorenal syndrome with acute kidney injury.
