Encouraging preliminary data from the first part of a clinical study in pouchitis

8 June 2023 

Announcement no. 34 

Encouraging preliminary data from the first part of a clinical study in pouchitis

•  The safety and efficacy of endoscopically administered gel formulation of Granulocyte Macrophage Colony Stimulating Factor (GM-CSF) is being evaluated in patients with pouchitis. 

• The clinical safety and proof-of-concept study (EudraCT no: 2020-000609-10) assesses the combined treatment with GM-CSF, fosfomycin and metronidazole for pouchitis in ulcerative colitis patients after restorative ileal pouch anal anastomosis (IPAA) surgery. 

The study investigates whether GM-CSF in combination with metronidazole and fosfomycin, applied topically in the pouch, can be used in patients with pouchitis. In the first part of the study, a single treatment is applied topically, under endoscopic supervision to 6 patients. In the second part of the study involving 12 patients, repeated dosing will be administered using an enema. The objectives of the study are to determine safety, and changes in the Pouchitis Disease Activity Index. 

“I am thrilled that we have been able to show such a strong improvement on pouchitis symptoms, and objective measures made during endoscopy and histological evaluation in the first 6 patients in our clinical study,” says Prof Ismail Gögenur, University of Copenhagen.  “Seeing such a clear effect after a single treatment brings me real hope that we can develop an effective and safe local treatment for pouchitis. We observed 5 out of 6 patients with improvement in the Pouchitis Disease Activity Index (PDAI) total score. It is really encouraging to see this kind of potent effect in only one treatment and in that many patients. We are now looking forward to the second phase of the study to evaluate the effects of repeated daily treatment.” 

 "We are very excited about these preliminary results in the first part of the study,” says Thomas Kaas Selsø, CEO, “The update from our investigator gives us optimism for the future development of this asset." 

About Pouchitis 

Total colectomy, mucosal proctectomy, and ileal pouch-anal anastomosis (IPAA) has become the surgical procedure of choice for patients with ulcerative colitis and familial adenomatous polyposis. However, approximately 50% of patients go on to develop pouchitis (an inflammation of the ileal remnant). Pouchitis has an estimated prevalence of 250.000 in the European Union, United States and Japan. Symptoms of pouchitis include abdominal cramps, incontinence, bleeding, and tenesmus with considerable impact on patients’ quality of life. Treatment modalities include oral antibiotics as well as immunomodulators, steroids, probiotics, and biological agents, but up to 20% of these patients develop chronic, treatment-resistant pouchitis, which can result in pouch failure and the need for reoperation and the possible creation of an ileostomy.  

About Pharma Equity Group A/S 

Pharma Equity Group A/S is a listed company on the Nasdaq Copenhagen Stock Exchange, with a focus on early investment in innovative life science companies (in or before phase 2) that develop technologies and therapies that have the potential to improve human health and quality of life.  

Pharma Equity Group A/S portfolio currently includes Reponex Pharmaceuticals A/S. Reponex Pharmaceuticals A/S is a clinical-stage biopharmaceutical company dedicated to the development of novel, effective treatments for diseases that have significant patient and social impact.