The Company also provided an update to its corporate progress, clinical status and anticipated milestones for anabasum, its novel synthetic oral endocannabinoid-mimetic drug that is designed to resolve chronic inflammation and halt fibrosis.
“We have made significant progress in both the corporate and clinical areas in the first quarter of 2017 and are in a strong financial position, which is expected to take us through key milestones for the Company over the next 20 months,” stated Yuval Cohen, Ph.D., Chief Executive Officer of the Company.
Systemic Sclerosis Clinical Program Update
Corbus reported positive topline data results from a Phase 2 study in diffuse cutaneous systemic sclerosis (“systemic sclerosis”) in November 2016, showing a clear signal of clinical benefit with anabasum. The 12-month open-label extension of this Phase 2 study is ongoing and is designed to capture long-term safety and efficacy data in subjects dosed with anabasum 20 mg twice per day. To date, subjects have been safely dosed with anabasum for up to 10 months and Corbus intends to present data at the 2017 American College of Rheumatology (“ACR”) Annual Meeting.
Following an end-of-Phase 2 meeting with the U.S. Food and Drug Administration (“FDA”), Corbus submitted a protocol to the FDA for its Phase 3 study in systemic sclerosis on March 31, 2017, and is moving forward as planned. The Company expects to commence this study in the fourth quarter of 2017. Protocol assistance from the European Medicines Agency (“EMA”) on the Phase 3 study design is expected in the second quarter of 2017.
The planned Phase 3 study is a double-blind, randomized, placebo-controlled, parallel dose, multi-center study to be conducted in approximately 270 adults with diffuse cutaneous systemic sclerosis. Subjects will be randomized to receive anabasum 20 mg twice per day, anabasum 5 mg twice per day, or placebo twice per day for 52 weeks. The primary efficacy outcome of the planned Phase 3 study will be change from baseline at Week 52 in modified Rodnan Skin Score (“mRSS”), a measure of skin thickening and a validated clinical outcome measure in systemic sclerosis. Topline results of the Phase 2 study showed that the mean improvement from baseline in mRSS for anabasum-treated subjects was greater than for the placebo-treated subjects, and considered medically meaningful. The improvement in mRSS was accompanied by statistically significant improvement in the anabasum arm in patient-reported skin symptoms and reduced expression of genes associated with inflammation and fibrosis in skin biopsies. Corbus expects to complete enrollment of the 52-week study in 2018. The Company also expects results by the end of 2019 and a New Drug Application (“NDA”) application to be filed in 2020.
Anabasum was granted Orphan Drug Designation and Fast Track status for the treatment of systemic sclerosis from the FDA in 2015 and Orphan Designation from the European Medicines Agency (“EMA”) in January 2017. The Company was not granted Breakthrough Designation for systemic sclerosis, however Corbus’ existing Fast Track status already grants it similar eligibility for more frequent meetings with the FDA to discuss the development plan for anabasum, as well as Priority Review and Rolling Reviews of completed sections of the NDA.
Expected Near-Term Milestones:
- Presentation of anabasum Phase 2 systemic sclerosis data at EULAR in June 2017;
- Continue Phase 2 anabasum open-label extension study and present a data update at the 2017 ACR Annual Meeting in November 2017; and
- Commence anabasum Phase 3 study of systemic sclerosis in the fourth quarter of 2017.
Cystic Fibrosis Clinical Program Update
In March 2017, Corbus reported positive topline data from the double-blind, randomized, placebo-controlled Phase 2 study of anabasum for the treatment of cystic fibrosis (“CF”) showing that anabasum had an acceptable safety and tolerability profile, reduced pulmonary exacerbations treated with antibiotics, and reduced multiple inflammatory biomarkers in sputum collected from subjects in the study. The 16-week study was an international, multi-center study supported by a $5 million Development Award from Cystic Fibrosis Foundation Therapeutics, Inc.
Anabasum successfully achieved the primary objective of the study by demonstrating an acceptable safety and tolerability profile with no serious or severe adverse events related to the study drug. Anabasum cohorts showed a reduction in a number (event rate per subject) of pulmonary exacerbations treated with intravenous antibiotics compared to placebo, which was a prespecified event of special interest in the protocol, as well as the number (event rate per subject) of pulmonary exacerbations treated with any new antibiotic. Patients in the highest dose cohort of anabasum (20 mg orally, twice per day) had a 75% reduction in the annualized rate of pulmonary exacerbations requiring IV antibiotics compared to placebo cohort. Forced expiratory volume in 1 second (FEV1) remained stable throughout the study in all treatment cohorts.