FDA Grants Orphan Drug Designation to Potential Pediatric Ulcerative Colitis Treatment

Mathew Shanley

This morning, Seres Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to SER-287 for the treatment of Ulcerative Colitis (UC) in pediatric patients.

The impetus of the designation was a review of Phase 1b clinical data which established a medically plausible basis for the use of SER-287, and highlighted the drug’s potential as a novel treatment modality for patients suffering from UC.

UC is a common inflammatory bowel disease in adults, however, it is incredibly rare in children. The condition is characterized by inflammation and ulcers in the colon and rectum, and can lead to symptoms like abdominal pain, diarrhea and bloody stools. The current standard-of-care for more than 80% of all people with mild-to-moderate ulcerative colitis is anti-inflammatory medication. In more severe cases, though, corticosteroids and/or immunomodulatory agents are recommended.

SER-287 is an oral formulation containing a syndicate of live bacterial spores and is hypothesized to act through a novel mechanism of action by modulating the dysbiotic microbiome thereby reducing inflammation without immunosuppression effects. By restoring good health to the microbiome, integrity will be maintained in the colonic barrier, and signaling by pro-inflammatory molecules produced by certain bacteria will be reduced. Additionally, this positive change will, theoretically, induce regulatory T cells in the colon to modulate immune responses.

“We are pleased to receive FDA Orphan Drug Designation for SER-287 and we look forward to advancing the program into further development for Ulcerative Colitis,” said Roger J. Pomerantz, M.D., President, CEO and Chairman of Seres in a press release. “Based on the highly encouraging Phase 1b clinical results and favorable safety profile, we intend to evaluate SER-287 in a pediatric UC population as part of our overall development plan. Safety is of particular importance to the pediatric population, and given the positive safety profile observed in our clinical trial to date, we believe our microbiome approach may be well suited to address this group.”

Enrollment in the Phase 1b trial is now complete, and the study has primary outcome measures including the safety and tolerability of SER-287 vs. placebo, examination of baseline composition of the intestinal microbiome, and engraftment of SER-287 bacteria into the intestinal microbial community in each of the SER-287 arms compared to the placebo arm. The estimated primary completion date is February 2017.

Inclusion criteria for those enrolled in the study include UC diagnosed by routine clinical, radiographic, endoscopic and pathologic criteria.

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