An Efficacy and Safety Study of Mongersen (GED-0301) in Subjects With Active Ulcerative Colitis

Inclusion Criteria

Inclusion Criteria

• Subjects must satisfy the following criteria to be enrolled in the study:
• Subject is ≥ 18 years of age at the time of signing the informed consent form (ICF).
• Subject is able to understand and voluntarily sign an informed consent form (ICF)prior to conducting any study related assessments/procedures.
• Subject is willing and able to adhere to the study visit schedule and other protocol requirements.
• Subject must have diagnosis of Ulcerative Colitis (UC) with a duration of at least 3 months prior to screening.
• Subject must have moderate to severe Ulcerative Colitis (UC), defined as Modified Mayo score (MMS) ≥ 4 to ≤ 9 with rectal bleeding subscore (RBS) ≥ 1 at screening.
• Subject must have a Mayo endoscopic subscore ≥ 2 at screening.
• Subject must have failed or experienced intolerance to at least one of the following:

aminosalicylates; budesonide; systemic corticosteroids; immunosuppressants (eg,6-mercaptopurine (6-MP), or azathioprine (AZA)) or Tumor necrosis factor (TNF)-α blockers (eg, infliximab, adalimumab, or golimumab)

• Subject must meet the following laboratory criteria:
• White blood cell count ≥ 3000/mm3 (≥ 3.0 X 109/L)
• Platelet count ≥ 100, 000/mm3 (≥ 100 X 109/L)
• Serum creatinine ≤ 1.5 mg/dL (≤ 132.6 μmol/L)
• Aspartate transaminase (AST/serum glutamic oxaloacetic transaminase (SGOT)) and alanine transaminase (ALT/serum pyruvic transaminase (SGPT)2.5 X upper limit of normal (ULN)
• Total bilirubin ≤ 2 mg/dL (≤ 34 μmol/L) unless there is a confirmed diagnosis of Gilbert's disease
• Hemoglobin ≥ 9 g/dL (≥ 5.6 mmol/L)
• Activated partial thromboplastin time (APTT) 1.5 X ULN
• Females of childbearing potential (FCBP) must have a negative pregnancy test at the Screening and Baseline Visits. While on Investigational Product (IP)and for at least 28 days after taking the last dose of Investigational Product (IP), females of childbearing potential (FCBP) who engage in activity in which conception is possible must use one of the approved contraceptive options described below: Option 1: Any one of the following highly effective methods: hormonal contraception (oral, injection, implant, transdermal patch, vaginal ring); intrauterine device (IUD); tubal ligation; or partner's vasectomy; OR Option 2: Male or female condom PLUS 1 additional barrier method: (a) diaphragm with spermicide; (b) cervical cap with spermicide; or (c) contraceptive sponge with spermicide.
• Male subjects (including those who have had a vasectomy) when engaging in sexual activity with females who are able to become pregnant must use barrier contraception (male latex condom or nonlatex condom NOT made out of natural [animal] membrane [for example, polyurethane]) while on Investigational Product (IP) and for at least 28 days after the last dose.

Exclusion Criteria

• The presence of any of the following will exclude a subject from enrollment:
• Subject has a diagnosis of Crohn's Disease (CD), indeterminate colitis, ischemic colitis, microscopic colitis, radiation colitis or diverticular disease-associated colitis.
• Subject has ulcerative colitis restricted to distal 15 cm or less (eg, ulcerative proctitis).
• Subject had surgery as a treatment for ulcerative colitis (UC)or who, in the opinion of the Investigator, is likely to require surgery for ulcerative colitis (UC) during the study.
• Subject has clinical signs suggestive of fulminant colitis or toxic megacolon.
• Subject is stool positive for any enteric pathogen or Clostridium difficile (C. difficile) toxin at screening.
• Subject has history of colorectal cancer or colorectal dysplasia.
• Prior treatment with more than 2 Tumor necrosis factor (TNF)-α blockers (eg, infliximab, adalimumab, or golimumab).
• Prior treatment with any integrin antagonists (eg, natalizumab or vedolizumab).
• Use of Tumor necrosis factor (TNF)-α blockers within 8 weeks of the screening.
• Subject had prior treatment with mycophenolic