REDWOOD CITY, Calif.--(BUSINESS WIRE)-- Ocelot Bio, Inc., a clinical-stage biopharmaceutical company focused on the development of innovative therapeutics to treat complications of end-stage liver disease (ESLD), today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead candidate OCE-205 for the treatment of ascites due to all etiologies except cancer.

“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,” said Lise Kjems, M.D., Ph.D., chief medical officer at Ocelot Bio. “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 is the abnormal accumulation of fluid within the peritoneal cavity of the abdomen and the hallmark of progression into the decompensated phase of cirrhosis. Data suggest that approximately 400,000 people live with ascites, with 60,000 refractory to standard-of-care treatment and who require much more aggressive treatment approaches.^1,2 Current treatments for ascites are limited, are often invasive procedures and may come with significant risks and burden for patients, underscoring the critical need for novel advancements that improve outcomes for those suffering from this devastating condition. Ocelot Bio plans to initiate clinical studies of OCE-205 in refractory ascites in 2024.

The FDA grants Orphan Drug Designation to drugs and biologics intended for the treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides Ocelot Bio certain benefits, including financial incentives to support clinical development and the potential for up to seven years of market exclusivity for the drug for the designated orphan indication in the U.S. if the drug is ultimately approved for its designated indication. This is the second Orphan Drug Designation for OCE-205, which previously received the designation in hepatorenal syndrome.

About OCE-205

Ocelot Bio’s lead asset, OCE-205, is a therapeutic peptide with a differentiated mechanism of action that has potential for improving outcomes for patients with complications of end-stage liver disease (ESLD). OCE-205’s innovation emanates from its design as a mixed agonist-antagonist peptide selective for the vasopressin 1a (V1a) receptor with no vasopressin 2 (v2) receptor activity at drug concentrations well above those used in treating ESLD. By virtue of its mixed V1a agonist/antagonist activity, OCE-205 treatment is designed to relieve portal hypertension while offering a capped maximal efficacy that avoids excessive vasoconstriction seen with full agonists, that leads to ischemic injury and potentially other serious adverse events such as respiratory failure. By eliminating V2 activity, OCE-205 treatment is designed to avoid fluid retention and overload.

A Phase 2 clinical trial has completed enrollment to evaluate OCE-205 for the treatment of hepatorenal syndrome with acute kidney injury (HRS-AKI) (NCT05309200), and clinical studies for ascites are expected to initiate in 2024. The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for OCE-205 in the treatment of hepatorenal syndrome in 2022 and in ascites in 2023.

About Ocelot Bio

Ocelot Bio is a clinical-stage biopharmaceutical company focused on the development of innovative therapeutics designed to significantly improve outcomes for patients with liver disease. The company’s lead clinical candidate, OCE-205, is a peptide therapeutic with a mechanism of action designed to selectively target complications of portal hypertension, such as hepatorenal syndrome with acute kidney injury (HRS-AKI) and ascites, serious and life-threatening consequences of end-stage liver disease (ESLD). The company was founded by leading experts in peptide therapeutic development and is backed by a strong syndicate of investors including Venrock, RA Capital Management and Vivo Capital. For more information, visit www.ocelotbio.com.

References
¹ Siqueira F, et al. Gastroenterol Hepatol (N Y). 2009;5(9):647-656.
² Orman, et al. JAMA Netw Open. 2019;2;2(10):e1913673.

View source version on businesswire.com: https://www.businesswire.com/news/home/20231220466025/en/