However, results do indicate benefit for a proportion of patients, and Atlantic Healthcare hopes to move forward with the regulatory approval process.
This Phase 3 multi-center, double-blind, randomized controlled trial (NCT02525523) was conducted in 138 patients with chronic antibiotic refractory pouchitis who failed to respond appropriately to one or more courses of antibiotics.
Pouchitis is the most common long-term complication of the surgical procedure known as ileal pouch anal anastomosis (IPAA), a surgery that patients with severe ulcerative colitis, a form of inflammatory bowel disease, must undergo. Pouchitis is characterized by inflammation, ulcers and frequent and urgent emptying of the bowel, often up to 20 times a day and night. The first line of treatment for pouchitis is antibiotic treatment, but a significant proportion of patients fail to respond.
Alicaforsen is a type of drug known as an antisense oligonucleotide, designed to help reduce levels of ICAM-1, a molecule involved in the process of inflammation. It is being investigated as a treatment for several different inflammatory diseases related to gastrointestinal tract, including inflammatory bowel disease.
The co-primary endpoints of this Phase 3 study were the proportion of patients who underwent endoscopic remission and a decrease in stool frequency at week 10.
After conducting primary analysis of the results, the clinical trial did not meet its co-primary endpoints. However, results for stool frequency were encouraging in 34% of patients.
Furthermore, after re-analyzing the endoscopy results using new statistical methods, researchers found that there was a statistically significant endoscopic response in a subgroup of patients.
“Although we are disappointed that the Phase 3 trial did not achieve statistical significance, we believe the percentage of patients achieving remission in stool frequency and the endoscopic response observed in a number of subpopulations of patients could be noteworthy,” Toby Wilson Waterworth, chief executive at Atlantic Healthcare, said in a press release.
Waterworth added that after consulting key opinion leaders of the field and regulatory advisors, they now plan to meet with the United States Food and Drug Administration and European Medicines Agency to discuss potential routes to regulatory approval for alicaforsen.
Previous results from earlier clinical trials have shown that alicaforsen is safe and well-tolerated, with no therapy-related serious adverse events.
Waterworth said, “It is also encouraging to note that the patients who participated in the trial demonstrated strong compliance with alicaforsen enema.”
Professor Brian Feagan, MD, an acknowledged expert in IBD, said: “This study makes a significant contribution to the existing body of knowledge in pouchitis. As long as there is no approved treatment, there remains an unmet medical need. It is therefore encouraging to note that alicaforsen enema may benefit some pouchitis patients.”
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