Ocera Therapeutics, Inc. (NASDAQ:OCRX) reported financial results for the quarter ended March 31, 2017 and provided updates on its clinical development programs of OCR-002 for the treatment of hepatic encephalopathy (HE), a debilitating liver disorder and significant burden on the healthcare system.

“To date, 2017 has been very eventful for Ocera with a successful Phase 1 study of oral OCR-002 in cirrhotic patients in January followed by the completion of our STOP-HE Phase 2b clinical trial of IV OCR-002 and subsequent data read out,” said Linda Grais, M.D., Chief Executive Officer of Ocera. “The data from STOP-HE confirmed that hyperammonemia is correlated with the severity of HE symptoms and that OCR-002 correlates with faster reduction of ammonia, and with clinical improvement.”

Previously, the company reported top-line data from STOP-HE based on an intent to treat (ITT) population of patients with elevated plasma ammonia levels at screening. Patients receiving OCR-002 demonstrated a 17-hour reduction in time to improvement in HE symptoms over placebo (47 versus 64 hours, respectively) for the primary endpoint, p=0.129.

Today, the company reported new data from STOP-HE. A post-hoc analysis of the same primary endpoint revealed patients with confirmed hyperammonemia at randomization (baseline) improved faster on OCR-002 than placebo (42 versus 63 hours, respectively), with statistical significance, p=0.034.

The findings are based on a retrospective analysis of baseline ammonia levels drawn at time of randomization and determined by a central laboratory. Of the 231 patients in the ITT analysis, 201 were confirmed as hyperammonemic at time of randomization; some patients’ ammonia levels had normalized between screening and randomization under standard of care. This post-hoc per protocol population represents the study’s target population given the ammonia-scavenging mechanism of action of OCR-002.

“We are very encouraged by this additional analysis of the data which reaffirms the clinical benefits attributable to the mechanism of action of OCR-002,” said Stan Bukofzer, M.D., Chief Medical Officer. “STOP-HE demonstrated that rapid reduction of ammonia in hyperammonemic patients leads to faster clinical improvement, which we believe will translate to better outcomes for these patients. We look forward to continuing to develop OCR-002 with the hope of bringing these benefits to patients, their families, and to the healthcare system.”

Select First Quarter Financial Results

  • As of March 31, 2017, Ocera had cash, cash equivalents and marketable securities of $25.2 million.
  • Net loss for the quarter was $6.7 million compared to a net loss of $7.5 million for the same period in 2016. Basic and diluted net loss per share were $0.28 for the quarter compared to basic and diluted net loss per share of $0.36 for the same period in 2016.
  • Research and development (R&D) expense for the quarter decreased to $3.9 million, from $4.7 million for the same period in 2016 due primarily to a decrease in clinical development costs.
  • General and administrative (G&A) expense for the quarter is $2.5 million, essentially flat compared with $2.6 million for the same period in 2016.
  • Net interest expense of $0.3 million for the quarter was primarily attributable to interest and amortization associated with the debt facility which closed in July 2015.
  • Net cash proceeds generated from the Company’s “at the market” equity facility totaled approximately $3.5 million for the quarter.

Financial Guidance

Ocera expects net use of cash for 2017 to be in the range of $24.0 million to $27.0 million, including $3.1 million in scheduled principal repayments on notes payable, and reiterates its previous guidance that it will have sufficient cash to fund operations into the second quarter of 2018 based on its current operating plan.

Shares of Ocera Therapeutics are rising nearly 5% to $1.35 in after-hours trading Tuesday. OCRX has a 1-year high of $3.15 and a 1-year low of $0.52. The stock’s 50-day moving average is $1.27 and its 200-day moving average is $1.62.

Ocera Therapeutics, Inc. is a clinical stage biopharmaceutical company. It engages in development and commercialization of OCR-002 (ornithine phenylacetate0 in intravenous and oral formulations.