Ambrose Healthcare Ltd (Ambrose Healthcare), an award-winning pharmaceutical company focused on driving change in rare diseases, reports that the option to acquire renzapride has been exercised, providing global ownership rights.
Renzapride is an investigational prokinetic agent Ambrose intends to develop for gastrointestinal (GI) symptoms in cystic fibrosis (CF) – which affect around 90% of CF patients.
Dr Bu Hayee, Consultant Gastroenterologist at King’s College Hospital commented: “GI complications affect a huge number of CF patients and are a key quality of life concern with limited existing treatment options. Renzapride offers a significant opportunity to address this unmet need.”
Renzapride has generated positive human efficacy and safety data in GI clinical trials involving >3,000 patients, with statistically significant improvements reported in endpoints relevant for this new indication.
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 improvements have been reconfirmed in a Phase 3 trial, where they were studied as secondary endpoints.
Toby Wilson Waterworth, CEO and Co-founder of Ambrose, comments: “Renzapride is precisely the kind of asset Ambrose is focusing on for our drug pipeline. It has generated supportive clinical data demonstrating efficacy and an attractive safety profile, but it got stuck. Our due diligence suggests that in our target rare disease indication, and with the right approach, it could reach approval within 5 years and become the first approved therapy for treating GI symptoms in cystic fibrosis patients.”
A new Phase 2 study protocol to assess renzapride for the treatment of GI symptoms experienced by CF patients has been worked up in conjunction with Ambrose’s network of rare disease experts, including input from the U.S. Cystic Fibrosis Foundation, CF gastroenterology centers 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.
About Ambrose
Ambrose Healthcare is an award-winning pharmaceutical company driving change in rare diseases.
With only 5-10% of rare diseases having an FDA-approved treatment, Ambrose is on a mission to get safe and effective therapies onto the market and into the hands of patients quickly and cost-effectively.
Alongside drug development, we collaborate globally on data-driven projects to help overcome hurdles in and improve understanding of rare diseases, their management and patient experience.
We believe this twin-track approach is how we’ll contribute to reshaping the rare diseases landscape.
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 GI symptoms in 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 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.
About 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:
https://www.ecfs.eu/ecfspr (last updated 21 June 2023)
https://www.cysticfibrosis.org.uk/sites/default/files/2022-10/CFT_2021-Annual-Data-Report-WEB.pdf
Cromwell, E.A., Ostrenga, J.S., Todd, J.V., et al (2023) J Cystic Fibrosis 22: 436-442
Cystic Fibrosis Foundation: The Digestive Tract | Cystic Fibrosis Foundation (cff.org)
Singh, V.K and Schwarzenberg, S.J. (2017) J Cystic Fibrosis 16: Suppl. 2, S70-S78
Moshiree, B., Freeman, A.J., Vu, P.T., et al (2023) J Cyst Fibros 22: 266-274
Forward looking statements
While Ambrose Healthcare believes any forward-looking statements in this press release are accurate, the statements represent Ambrose Healthcare’s beliefs only as of the date of this press release. A number of factors could cause actual events or results to differ materially from those indicated by such statements. These forward-looking statements involve risks and uncertainties. In addition, risks and results in clinical trials may not be indicative of risks or results from later stage or larger scale trials, and there is no assurance of regulatory approval. Existing and prospective investors should not place undue reliance on the forward-looking statements contained in a press release and instead should make their own determinations as to the reliability of such statements. Ambrose Healthcare undertakes no intent or obligation to update the information contained in this press release as new information becomes available.
For more information, please contact:
Georgia Wilson Waterworth, Chief of Staff and Co-Founder
Juliet Burns, Corporate Communications