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Renzapride

Status

Phase 2

Gastrointestinal symptoms in cystic fibrosis

Indication

About Renzapride 

Renzapride is an investigational prokinetic agent with a dual mode of action, being both a 5HTP4 agonist and a 5HTP3 antagonist, which can act throughout the gastrointestinal tract.  

 

It presents a promising novel treatment for gastrointestinal (GI) symptoms in cystic fibrosis (CF) with an existing attractive efficacy and safety profile as demonstrated in clinical studies involving >3,000 patients. 


  • Results include three Phase 2 clinical studies in Constipation Predominant Irritable Bowel Syndrome (IBS-c) demonstrating a statistically significant (p<0.05) improvement in bowel motility, and reduction in pain, bloating and feelings of nausea. These are all relevant to CF.

  • These improvements have been reconfirmed in a Phase 3 trial, where they were studied as secondary endpoints. 


Ambrose’s high-level plan for approval within five years will leverage regulatory incentives that allow for accelerated development and approvals of rare disease drug therapies. The next step includes a Phase 2 multi-center, double-blind, cross-over study involving 5 study sites and 24 patients.  

The new Phase 2 study protocol to assess renzapride in GI symptoms for CF has been worked up in conjunction with Ambrose’s network of rare disease experts, including with input from the U.S.

Cystic Fibrosis Foundation, CF gastroenterology centres of excellence and CF GI physicians.

 

An independent report on the potential of renzapride to treat GI symptoms experienced by CF patients is available upon request. 



Indication

Gastrointestinal symptoms in cystic fibrosis.  


There are estimated to be ca. 95,000 cystic fibrosis patients in Europe and the US combined [1,2,3] (including ca. 11,000 in the UK), of which around 90%[4] are believed to suffer from gastrointestinal symptoms, ranging from an inability to digest food, to abdominal pain, bloating and constipation[4,5]. Despite the availability of pancreatic enzyme replacement therapy, a recent prospective study found very high levels (>88%) of dissatisfaction with current treatments resulting in continued gastrointestinal symptoms, with >98% of subjects taking a variety of medications in an attempt to address these symptoms [6]. 


References:  

  1. https://www.ecfs.eu/ecfspr (last updated 21 June 2023) 

  2. https://www.cysticfibrosis.org.uk/sites/default/files/2022-10/CFT_2021-Annual-Data-Report-WEB.pdf 

  3. Cromwell, E.A., Ostrenga, J.S., Todd, J.V., et al (2023) J Cystic Fibrosis 22: 436-442 

  4. Cystic Fibrosis Foundation: The Digestive Tract | Cystic Fibrosis Foundation (cff.org)  

  5. Singh, V.K and Schwarzenberg, S.J. (2017) J Cystic Fibrosis 16: Suppl. 2, S70-S78 

  6. Moshiree, B., Freeman, A.J., Vu, P.T., et al (2023) J Cyst Fibros 22: 266-274 

Publications

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