Ocelot’s Rare Kidney Disease Candidate Receives Orphan Drug Designation Going Into Phase 2 Trials

San Diego-based Ocelot Bio announced on August 22 that its lead candidate, OCE-205, received the FDA’s Orphan Drug Designation to treat hepatorenal syndrome (HRS). The designation comes at a crucial time for the drug’s development as Ocelot is enrolling in Phase 2 clinical trials to study OCE-205’s safety and efficacy in patients with cirrhosis with ascites who have developed HRS with acute kidney injury. 

Developing a Therapy for Deadly Disease with Limited Options

HRS is a rare form of kidney dysfunction affecting approximately 50,000 Americans annually that can occur in patients with advanced liver disease. The disease has a high mortality rate of about 50% within 90 days of diagnosis. 

In hopes of providing a safe and effective treatment option for HRS, Ocelot is developing OCE-205, which it says targets serious hemodynamic complications while avoiding excessive vasoconstriction, or blood flow restriction, by capping maximal efficacy. Ocelot also designed OCE-205 to avoid fluid retention, often found in patients with cirrhosis with ascites, which can exacerbate kidney and liver problems. 

President and Chief Executive Officer at Ocelot, Katherine Vega Stultz, said, “Receiving Orphan Drug Designation for OCE-205 underscores the urgent unmet medical need for patients with hepatorenal syndrome. We are actively engaged with leading medical centers to advance our Phase 2 clinical study, and our team is working towards better understanding patient outcomes to continue to progress with urgency our development program for OCE-205.”

The Orphan Drug Designation program is a huge stepping stone for OCE-205’s development because it comes with certain benefits like financial incentives and, most importantly, market exclusivity for seven years for HRS treatment if the drug receives approval. 

Related Article: Everest, Calliditas Win First Approval for Rare Kidney Disease Drug in Europe

Current Treatment Options and their Shortcomings

Currently, there are no FDA-approved therapies for HRS, but common treatments include liver transplants and vasoconstrictors, which cause the blood vessels to narrow. Another investigational technique is transhepatic portosystemic shunts (TIPS),  where a surgeon inserts a stent into the patient’s liver, increasing blood flow. 

Some researchers criticize the most common vasoconstrictor, terlipressin, as a dangerous treatment option because of the risks of ischemic events where a patient’s blood flow to the heart is restricted. Still, with so few options available, many look to terlipressin as a treatment option. Mallinckrodt, a company specializing in generic medicines, resubmitted a New Drug Application for the drug in June after receiving a Complete Response Letter from the FDA in February outlining issues with the company’s packaging and labeling facility. 

Vasoconstrictors and TIPS are only investigational therapies at the moment, and the only standard, curative therapy available to HRS patients is a liver transplant. Even with higher success rates, liver transplants pose issues such as limited donor pools and the chance that a patient’s kidneys may not fully recover if a liver transplant is not performed soon enough. Many patients still require dialysis with investigational therapies and liver transplants to supplement liver and kidney function. 

With limited treatment options for patients with HRS, Ocelot’s OCE-205 recent Orphan Drug Designation is a hopeful step in the right direction for a safe and effective treatment. Ocelot’s Phase 1 trial showed promise, and the upcoming Phase 2 trial will provide a better outlook on the future of the drug and its potential to treat HRS. 

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Author

Reed Slater