Phase 2 Cystic Fibrosis Study (Completed)
Corbus Pharmaceuticals reported positive topline data demonstrating that anabasum reduces acute pulmonary exacerbations and multiple inflammatory biomarkers in its Phase 2 study in patients with cystic fibrosis.
Study participants were screened for up to 28 days to ensure they met study entry criteria. Following the initial screening, participants were provided with anabasum or placebo for once a day dosing for 28 days. On the 29th day, participants switched to twice a day dosing for an additional 56 days. The total treatment period was 84 days. After the treatment period, participants were followed for an additional 28 days.
Anabasum successfully achieved the primary objective of the study by demonstrating an acceptable safety and tolerability profile at all doses with no serious or severe adverse events related to the study drug.
During Weeks 1-4, treatment-emergent adverse events (TEAEs) occurred in 14 (54%) of study participants in the anabasum 1 mg cohort, 13 (54%) of the anabasum 5 mg cohort and 15 (43%) of the placebo cohort. During Weeks 5-12, TEAEs occurred in 21 (68%) patients in the anabasum 20 mg once per day cohort, 19 (63%) of the anabasum 20 mg twice per day cohort and 14 (58%) of the placebo cohort. The most common drug-related adverse event that occurred in more than 2 individuals in any treatment group was mild dry mouth observed in 8 (13%) of anabasum patients and no placebo patients. As expected, the respiratory system was the most common source of TEAEs overall. Nine serious adverse events (SAEs) occurred in the anabasum-treated patients and 6 SAEs occurred in placebo-treated patients. Three severe TEAEs occurred in the anabasum-treated patients and 4 in placebo-treated patients. None of the serious or severe TEAEs were assessed by site investigators to be related to study drug.
Treatment with anabasum yielded a reduction in acute pulmonary exacerbations when compared to placebo. Anabasum 20 mg twice a day was associated with a reduction in the rate of pulmonary exacerbations requiring treatment with IV antibiotics, compared to placebo. Similar levels of reduction were also observed in pulmonary exacerbations defined by new or worsening respiratory system symptoms requiring treatment with new antibiotics.
Inflammatory Cells and Biomarkers
Treatment with anabasum 20 mg twice a day showed a consistent reduction in multiple inflammatory cell types in sputum at the end of active treatment compared to placebo, including total leukocytes, neutrophils, eosinophils, lymphocytes and macrophages. Treatment with anabasum 20 mg twice a day resulted in a consistent reduction in inflammatory mediators in sputum including interleukin-8, neutrophil elastase and immunoglobulin G.
The Company plans to engage in further evaluation of the data and design of the next clinical trial in partnership with CF experts, the Cystic Fibrosis Foundation Therapeutics, Inc., Cystic Fibrosis Therapeutic Development Network and European Cystic Fibrosis Society Clinical Trials Network. The Company is also planning to initiate a larger and longer Phase 2b study of efficacy and safety of anabasum for the treatment of cystic fibrosis in the fourth quarter of 2017.
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