MONGERSEN for Crohn’s Disease
On the Basis of encouraging Phase 1 and 2 data (e.g. NEJM Monteleone, et.al. 372 (12): 1104-1113; 2015), Celgene has launched a global, multicenter phase 3 trial of Mongerson for the treatment of Crohn’s Disease. Mongersen is a unique small molecule, which acts non-systemically within the GI tract. It is a single strand antisense nucleotide that inhibits production of SMAD 7, which itself blocks the anti-inflammatory intracellular pathway of TGF-β. Oral administration has resulted in clinical improvement comparable to that of current biologic therapies. No significant serious adverse events have been reported.
The current trial is a randomized, double-blind, placebo-controlled multicenter study to establish the efficacy and safety of Mongersen for the treatment of subjects with active Crohn’s Disease. Participants must have an established diagnosis of Crohn’s for at least 3 months and failed or experienced intolerance to standard Crohn’s medications, including biologics. Crohn’s disease must be active for inclusion, by symptom and endoscopic measures. Patients may be biologics naïve or experienced, although the availability of this trial for biologic-experienced patients is extremely limited and time-sensitive. Patients will be randomized to 3 dose regimens of Mongersen and placebo in a 3:1 ratio. Patients who do not have an adequate clinical response will have an opportunity for “early escape” after 12 weeks-will then be placed in the Mongersen Long-term extension trial (Those in placebo arm who qualify for “early escape” will then receive study drug in the extension phase).
Key Exclusion Criteria:
Inflammatory Bowel disease other than Crohn’s, GI infections, local complications of Crohn’s Disease such as abscesses, strictures that are obstructing or cannot be passed with a standard colonscope and intestinal surgery within 6 months and/ or colostomy.
- Colonoscopy required at Screening (Week 0), Week 12 and Week 52.