Clinical Program

Marina Biotech’s first therapeutic candidate, CEQ508 for the treatment of Familial Adenomatous Polyposis (FAP), is now recruiting. CEQ508 will be a first-in-class therapeutic for the treatment of FAP, for which there is no viable pharmaceutical treatment option so that [and for which] surgical intervention is the first-line [primary] treatment approach.

The CEQ508 drug candidate is engineered to enter into dysplastic tissue and release a payload of shRNA, a mediator in the RNAi pathway (see "About RNAi" section). The expressed RNA encodes for the β-catenin gene which is known to be dysregulated in classical FAP. CEQ508 has been shown in the non-clinical setting to reduce the amount of intracellular β-catenin. The clinical trial will be conducted in FAP patients at Massachusetts General Hospital. For more information please contact: clinicaltrials@marinabio.com.

Protocol Title:

 A Phase Ib/IIa Open-Label, Escalating-Doses Study, of the Safety and Tolerability of Single Daily Doses of CEQ508, an RNAi-Based Therapy for Familial Adenomatous Polyposis

Sponsor:

 Marina Biotech, Inc.

Study Site:

 MGH, Boston, MA

Principal Investigator:

 Daniel Chung, MD

Projected Start Date:

 Now Recruiting

Study Phase:

 CEQ508, a tkRNAi drug candidate (live E.coli carrying RNAi to silence β-catenin)

Objectives:

Primary:

The primary study objective is to evaluate/establish general safety for orally administered CEQ508 in a daily dosing schedule and to determine the Maximum Tolerated Dose (MTD) (of 4 planned doses) and/or the Highest Safest Dose.

Secondary:

  • To examine shedding of CEQ508 in the stool of patients during and after daily oral dosing with CEQ508 for 28 days, and the 28 day recovery period
  • To examine gene expression changes after oral dosing of CEQ508 in GI mucosa of FAP patients

Patient Population:
  • Patients with known FAP and attenuated FAP (AFAP)

Sample Size:
  • Maximum of 30 patients
  • Dose Escalation Phase: 12 patients, at least 4 patients from each sex
  • Stable Dose Phase: 6 patients, who may be newly enrolled or re-enrolled from the 12 patients who were part of the Dose Escalation Phase

Inclusion

Exclusion
  • 18-65 years of age
  • Male and female
  • Clinical or genetic diagnosis of FAP
  • Pre and post colectomy; no immediate need for colectomy
  • Known endoscopic history of polyposis
  • Eligible to undergo baseline and endpoint endoscopies
  • Ability to be taken off other chronic FAP medication (Sulindac, Aspirin, etc.)
  • Informed consent
  • Inability to return for scheduled treatment and assessments
  • Chronic or intercurrent acute medical disorder
  • Significant clinical laboratory and hematology observations
  • Pregnancy, nursing (or anticipated pregnancy)
  • Antibiotic use (current or anticipated antibiotic treatment during the study period; antibiotic use within the past 2 weeks)
  • Active ulcerations or inflammation found at baseline endoscopy
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