Ocera Therapeutics Inc (NASDAQ:OCRX) reported top-line data from its exploratory STOP-HE Phase IIb trial assessing the safety, tolerability and efficacy of intravenously-given Ornithine Phenylacetate in hospitalized patients with HE.
Although not statistically important, OCR-002 established a 17-hour decline over placebo for the primary endpoint. It was time to progress in HE symptoms, p=0.129 and hazard ratio 1.25. Additionally, OCR-002 showed a 15-hour reduction compared to placebo for the secondary endpoint. It marked median time to close response in HE symptoms, hazard ratio 1.16 and p=0.361. Consistent with its action mechanism, OCR-002 exhibited a statistically considerable decline over placebo for the trial’s pre-specified exploratory objective which was time to reach normal plasma ammonia levels, hazard ratio 1.69 and p=0.028.
Linda Grais, M.D., the CEO of Ocera, said that it is a major achievement for company. The data validated that OCR-002 safely and rapidly lowered ammonia and demonstrated a dose-related clinical advantage. The patients at the upper doses 15 and 20 grams showed faster clinical improvement and increased complete response rates against the patients on the lowest dose of 10 grams and those on placebo.
This released data will be integral in deciding dose levels for future trials. Grais added that they want to thank the caregivers, investigators and patients and their families for their participation in STOP-HE.
Initial analysis indicates that a higher percentage of subjects respond as the dosage of OCR-002 increases, nevertheless that the clinical objectives did not achieve statistical significance. The same was also noted for the highly statistically considerable ammonia reduction.
Furthermore, the safety profile displays no safety indications for OCR-002 as against placebo. At the highest medication doses, the favorable differences were seen as against placebo in the frequency of grave life threatening safety events and deaths. They anticipate further exploratory assessments will provide increased clarity for their objective of getting intravenous OCR-002 into Phase III development.