Ocera Therapeutics

Ocera Therapeutics, Inc. was a clinical-stage biopharmaceutical company that concentrated on creating treatments for liver diseases. The company was founded in 2005 and operated from Redwood City, California. A significant event in the company's history was the merger with Tranzyme, Inc. in July 2013, after which the combined entity continued under the Ocera Therapeutics name, led by President and CEO Linda S. Grais, MD. This merger was followed by a $20 million private placement from several venture capital firms. The company's journey culminated in its acquisition by Mallinckrodt plc, a deal announced in November 2017 and finalized in December 2017. Mallinckrodt acquired Ocera for an initial $42 million, with potential for an additional $75 million based on development and sales milestones. Following the acquisition, Ocera became an indirect, wholly-owned subsidiary of Mallinckrodt and ceased to be a publicly traded company on the Nasdaq exchange.

Ocera's business model was centered on the development and eventual commercialization of its lead drug candidate, OCR-002 (ornithine phenylacetate), targeting orphan and other serious liver diseases with high unmet medical need. The company's market focus was on patients with hepatic encephalopathy (HE), a neuropsychiatric complication of acute or chronic liver disease caused by high ammonia levels (hyperammonemia). The potential U.S. market for HE was estimated to be substantial, with around 200,000 hospitalizations annually.

The company's core product, OCR-002, is an ammonia scavenger designed to lower dangerously high ammonia levels in the blood. It functions through a dual-mode action that does not rely on the liver, which is compromised in these patients. Once administered, OCR-002 breaks down into ornithine and phenylacetate. Ornithine helps convert ammonia into glutamine in the muscles, and phenylacetate combines with this glutamine to form phenylacetylglutamine, which is then excreted by the kidneys. This mechanism provides an alternative pathway for ammonia removal. OCR-002 was developed in both an intravenous (IV) formulation for acute HE in hospitalized patients and an oral version for maintenance therapy to prevent recurrence. The U.S. FDA had granted OCR-002 both Orphan Drug designation and Fast Track status. Despite a Phase 2b trial not meeting its primary endpoint, the drug demonstrated a significant effect on lowering ammonia levels, prompting Mallinckrodt to acquire it with the belief that trial design, not the drug itself, was the issue.

Keywords: Ocera Therapeutics, Mallinckrodt, OCR-002, hepatic encephalopathy, liver disease, ammonia scavenger, biopharmaceutical, ornithine phenylacetate, hyperammonemia, Orphan Drug, clinical stage, Tranzyme merger, Linda S. Grais, acute liver failure, chronic liver disease, intravenous formulation, oral formulation, ammonia reduction, clinical trials, biopharma acquisition