Ocelot Bio, a clinical-stage biopharmaceutical company focused on developing innovative therapeutics for complications of end-stage liver disease (ESLD), has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for its leading candidate, OCE-205, in the treatment of ascites excluding cancer.

Lise Kjems, M.D., Ph.D., Chief Medical Officer at Ocelot Bio, expressed excitement about the FDA's recognition, stating, "The FDA granting Orphan Drug Designation for OCE-205 in ascites is 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 program for OCE-205 and look forward to progressing this program in ascites."

Ascites, characterized by abnormal fluid accumulation in the abdominal cavity, is a key indicator of decompensated cirrhosis. Approximately 400,000 people live with ascites, and 60,000 are refractory to standard-of-care treatment, necessitating more aggressive approaches. Current treatments are limited and often involve invasive procedures with significant risks, highlighting the critical need for novel advancements. Ocelot Bio plans to initiate clinical studies of OCE-205 in refractory ascites in 2024.

Orphan Drug Designation is granted by the FDA to drugs intended for rare diseases affecting fewer than 200,000 people in the United States. It provides certain benefits to Ocelot Bio, including financial incentives for clinical development and potential market exclusivity for up to seven years if the drug is approved for its designated indication. OCE-205 had previously received Orphan Drug Designation for hepatorenal syndrome.

OCE-205 is Ocelot Bio's lead asset, a therapeutic peptide with a unique mechanism of action offering potential improvements for patients with complications of ESLD. Its design as a mixed agonist-antagonist peptide selective for the vasopressin 1a (V1a) receptor, without vasopressin 2 (v2) receptor activity, sets it apart. The Phase 2 clinical trial for hepatorenal syndrome with acute kidney injury (HRS-AKI) has completed enrollment (NCT05309200), and clinical studies for ascites are anticipated to commence in 2024. OCE-205 holds promise in addressing the challenges associated with ESLD complications, providing hope for improved patient outcomes.

 

Source: businesswire.com

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