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|Protalix BioTherapeutics Announces Additional Positive Results from Final Analysis of the Phase II Clinical Trial of OPRX-106 for the Treatment of Ulcerative Colitis|
Mucosal Improvement Observed in 61% of the Patients and 33% achieved Mucosal Healing
Clinical Responses Observed in 67% of the Patients
89% of the Patients Experienced a Reduction in
No Systemic Absorption Observed
The phase II clinical trial is a randomized, open label, 2-arm study of OPRX-106 for the treatment of ulcerative colitis (UC). A total of 24 patients were enrolled in the study, 18 patients completed the study with 6 patients who did not complete the study. The dropout rate is consistent with other trials in similar patient populations, and none of the patients dropped out due to a side effect or serious adverse event. Patients were randomized to receive 2 mg or 8 mg of OPRX-106, administered orally, once daily, for 8 weeks. The average baseline Mayo score was 7.1 (from a scale of 0-12) and the average baseline mucosal endoscopy sub score was 2.1 (from a scale of 0-3). For the 18 patients who completed the study, 89% had a baseline Mayo score between 6 and 9, which meets the criteria of moderate disease activity, and 84% had a baseline mucosal endoscopy sub score of 2 and above indicating moderate to severe disease based on mucosal appearance.
The key efficacy endpoints of the study were met at week 8:
Clinical response at week 8 is defined as a decrease in the Mayo score of at least 3 points and either a decrease in the sub-score for rectal bleeding of at least 1 point from baseline, or rectal bleeding sub-score of 0 or 1. Clinical remission at week 8 is defined as clinically symptom free, a Mayo score ≤ 2, with no individual sub-score exceeding 1 point after treatment.
In addition to clinical response and remission, efficacy was also observed in mucosal healing, an important prognostic parameter in ulcerative colitis and other inflammatory bowel diseases, measured by endoscopy:
Mucosal improvement is defined as a decrease in endoscopy sub-score at week 8. Mucosal healing is defined as a reduction in, and achievement of, endoscopy sub-score ≤1 at week 8.
Other key efficacy endpoints were also achieved, as follows:
The positive trend in efficacy is consistent in substantially all patients. This trend is demonstrated by 89% of the patients having showed an improvement in Mayo score in both doses, with an average decrease in Mayo score of 46% at week 8 in the 8mg dose and 40% in the 2mg dose. In addition, all of the patients also showed an improvement in at least one of the other efficacy parameters.
No anti-drug antibodies were detected. In addition, no systemic absorption was observed. OPRX-106 was safe and well tolerated with only mild to moderate adverse events, which were transient in nature. Headaches were the most common adverse event reported.
“The full set of data from the study is very compelling, and suggests that OPRX-106 is an effective anti-inflammatory agent. OPRX-106 is delivered orally and is biologically active in the gut without triggering the formation of anti-drug or systemic absorption. OPRX-106 has the potential to address the loss of response and to avoid critical safety concerns of infections and malignancies currently seen in anti–TNF alpha treatment, which is driven by the high presence of neutralizing antibodies and systemic absorption respectively,” said Professor
“We are very pleased by these excellent results, particularly with respect to the significant percent of patients demonstrating mucosal healing and clinical remission at eight weeks,” commented
The presentation made at the DDW 2018 Annual Meeting is available on the Company’s website.
Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx®. Protalix’s unique expression system presents a proprietary method for developing recombinant proteins in a cost-effective, industrial-scale manner. Protalix’s first product manufactured by ProCellEx, taliglucerase alfa, was approved for marketing by the U.S. Food and Drug Administration (
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