First Wave BioPharma Begins Patient Screening for Phase 2b Clinical Trial Evaluating Niclosamide in Patients With Ulcerative Proctitis & Ulcerative Proctosigmoiditis

First Wave BioPharma, Inc. recently announced it has begun screening patients for enrollment in a Phase 2b clinical trial investigating a topical formulation of FW-UP as a potential treatment for ulcerative proctitis (UP) and ulcerative proctosigmoiditis (UPS). FW-UP is a proprietary topical formulation of niclosamide, a small molecule anti-inflammatory inhibitor therapy. UP and UPS are two types of ulcerative colitis, a chronic inflammatory bowel disease (IBD) consisting of fine ulcerations in the inner mucosal lining of the large intestine that do not penetrate the bowel muscle wall.

“Advancing the clinical development of FW-UP in ulcerative proctitis and ulcerative proctosigmoiditis to a Phase 2b trial marks an important milestone in our niclosamide pipeline and our overarching mission to bring relief to patients living daily with gastrointestinal disease,” said James Sapirstein, President and CEO of First Wave BioPharma. “Our recently completed acquisition of First Wave Bio added new growth opportunities in the inflammatory bowel disease space where we are exploring the use of niclosamide and its anti-inflammatory properties in several indications. Ulcerative proctitis and ulcerative proctosigmoiditis are two of several IBD indications where we believe our proprietary formulations of niclosamide could address the significant underserved needs of this very large and growing patient population.”

The Phase 2b trial is a placebo-controlled study that will enroll up to 28 patients to compare FW-UP, administered as an enema twice daily at a dose of 450 mg, to placebo enemas twice daily. Patient screening is underway at clinical trial sites in Italy and will start soon in Austria and Germany.

The Phase 2b trial builds on preliminary data from a previous Phase 1a/2a trial evaluating a low-dose (150 mg/twice daily) and high-dose (450 mg/twice daily) of FW-UP in patients with UP and UPS. Data from the first 17 patients treated in the low-dose cohort demonstrated niclosamide to be well tolerated, with a durable therapeutic effect and a clinical remission rate of 59%. Although from early stage data, this rate of remission compares favorably to the rates of 38% to 44% reported for the commonly used steroid, budesonide. In data collected from the first four patients in the high-dose cohort, niclosamide was also shown to be well tolerated.

James Pennington, MD, Chief Medical Officer for First Wave BioPharma, said “There is a great deal to be excited about with the clinical data collected thus far investigating niclosamide’s use in UP and UPS. The drug was well tolerated at both low and high doses, and the data suggest that niclosamide could be more effective than the standard of care corticosteroid. This marks the first proof of principle for niclosamide as a treatment for an IBD. Our plan with the Phase 2b study is to determine whether higher doses of niclosamide have the potential to deliver even better results for patients.”

Niclosamide is a prescription small molecule drug known for its anti-inflammatory and anti-viral properties. Listed as an essential medicine by the World Health Organization (WHO), niclosamide has been safely used on millions of patients for other clinical indications. In the US, niclosamide was approved by the US FDA in 1982 for the treatment of intestinal tapeworm infections.

FW-UP is a niclosamide-based, small molecule anti-inflammatory inhibitor therapy for the treatment of ulcerative proctitis (UP) and ulcerative proctosigmoiditis (UPS). A Phase 1b/2a clinical trial is evaluating the safety and potential efficacy of a topical rectal formulation of FW-UP in patients with UP and UPS, and a Phase 2b trial has begun screening patients for enrollment.

Ulcerative proctitis and ulcerative proctosigmoiditis are two types of ulcerative colitis, a chronic inflammatory bowel disease consisting of fine ulcerations in the inner mucosal lining of the large intestine that do not penetrate the bowel muscle wall. UPS causes inflammation in the colon and rectum, while UP is confined only to the rectum. Symptoms include weight loss, fatigue, abdominal pain and cramps, rectal pain and bleeding, and diarrhea, although constipation can also develop as the body struggles to maintain normal bowel function. UP and UPS can occur at any point throughout life, with a high occurrence in young children and then again around 40-50 years of age. Progression of this disease to ulcerative colitis, extending farther up the bowel to involve the sigmoid colon, occurs in about 30%-50% of patients. Although there is a range of treatments to help ease symptoms and induce remission, there is no cure.

First Wave BioPharma, Inc. is a clinical-stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases. First Wave is currently advancing a therapeutic development pipeline populated with multiple clinical stage programs built around its two proprietary technologies – niclosamide, an oral small molecule with anti-viral and anti-inflammatory properties, and the biologic adrulipase, a recombinant lipase enzyme designed to enable the digestion of fats and other nutrients. First Wave’s niclosamide portfolio is led by three clinical programs: FW-COV, for COVID-19 gastrointestinal infections; FW-UP, for ulcerative proctitis (UP) and ulcerative proctosigmoiditis (UPS); and FW-ICI-AC, for Grade 1 and Grade 2 Immune Checkpoint Inhibitor-associated colitis (ICI-AC) and diarrhea in advanced oncology patients. Two additional formulations of niclosamide, FW-UC (for ulcerative colitis) and FW-CD (for Crohn’s disease) are expected to enter the clinic in 2022 and 2023, respectively. First Wave is also advancing FW-EPI (adrulipase) for the treatment of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis and chronic pancreatitis. The company is headquartered in Boca Raton, FL, with clinical operations in Hayward, CA. For more information, visit www.firstwavebio.com.