Ferring Presents New Phase 3b Safety and Effectiveness Data for Recurrent

Business Wire

05-05-2025

PARSIPPANY, N.J.--(BUSINESS WIRE)--Ferring Pharmaceuticals presented initial findings from the investigational Phase 3b multi-center, single-arm CDI-SCOPE study evaluating the safety and effectiveness of REBYOTA ® (fecal microbiota, live – jslm) when administered by colonoscopy. REBYOTA is the first microbiome-based therapy approved for the prevention of recurrent Clostridioides difficile (C. diff) infection (CDI) in individuals 18 years of age and older, following antibiotic treatment for C. diff infection. These data were presented at Digestive Disease Week 2025 (DDW2025) from May 3-6 in San Diego, CA.
The single-arm CDI-SCOPE study demonstrated that treatment with REBYOTA administered by colonoscopy was safe. Only 5 treatment-emergent adverse events (TEAEs) – occurring in 9.8% (4/41) of participants – were considered possibly related to REBYOTA, all of which were mild in intensity and related to the gastrointestinal tract. Two participants withdrew consent and did not complete the 8-week follow-up assessment.
"Colonoscopies are routinely used by physicians managing patients with recurrent C. difficile infection," said Dr. Paul Feuerstadt, M.D., F.A.C.G., A.G.A.F., Yale School of Medicine and PACT-Gastroenterology Center, a lead investigator of CDI-SCOPE. "The insights gained from this investigative study point to administration via colonoscopy as a promising potential option for physicians to consider for their patients."
A second abstract from the CDI-SCOPE study reported the burden of rCDI on health-related quality of life (HRQoL) and any improvements after treatment among 73.2% (30/41) participants. Prior to REBYOTA treatment, the most experienced rCDI symptoms were diarrhea (100%, 30/30), abdominal pain (70.0%, 21/30), and fatigue or weakness (46.7%, 14/30). All participants (100%, 30/30) reported that CDI recurrence impacted their daily living, including disrupting their sleep and forcing them to be near a bathroom at all times. Most participants also said that rCDI affected their social life and relationships (96.7%, 29/30) and their emotional wellbeing (93.3%, 28/30).
Following treatment with REBYOTA, 89.7% of participants (26/29) saw an improvement in their symptoms within the first month, and more than half (51.7%, 15/29) felt an improvement within the first week. Symptom severity rating scores (0-10, none to worst) decreased significantly at Week 8 for diarrhea (from 8.9 to 1.3), abdominal pain (from 7.5 to 1.6), and fatigue (from 8.1 to 1.7).
'Recurrent CDI is a life-altering condition that many patients in our CDI-SCOPE trial called 'a literal living nightmare' that makes it impossible to function,' said Raza Ahmed, MD, Senior Director of Medical Affairs, Ferring Pharmaceuticals. 'We are proud that REBYOTA has become an important, therapy for many patients who, for too long, were stuck in an agonizing cycle of CDI recurrence. With the data we are presenting this week at DDW, Ferring is continuing its commitment to supporting patients living with rCDI and their physicians by broadening the breadth of evidence.'
A third CDI-SCOPE abstract showed that 90% of the physicians who completed the 41 colonoscopy procedures in the study had a 'positive' or 'very positive' experience across all aspects of administration, including the material preparation time and ease of passage through the colonoscope. One investigator reported their experience as 'somewhat negative' citing difficulty with connecting REBYOTA to the colonoscope. All 39 patients who completed 8-week visits were assessed by the physicians as either having 'much improved' or 'very much improved' on the clinical global impression – improvement scale (CGI-I).
Additional Data from PUNCH™ Clinical Program
Along with the data from CDI-SCOPE, Ferring also reported two analyses with data from the PUNCH™ clinical trial program. This included:
Results from an integrated safety analysis of five clinical trials (n=1192) demonstrating a favorable safety profile with REBYOTA across all trials in the development program, including in participants with inflammatory bowel disease (IBD) and immunocompromising comorbidities. TEAEs were reported in 70.9% of patients who received REBYOTA, most of which were mild or moderate in severity and related to the gastrointestinal tract. Serious TEAEs were reported in 14.3% of patients receiving REBYOTA, most of which were related to rCDI and/or preexisting conditions, with few events (<1%) being considered possibly related to treatment. There was no clustering of serious TEAEs, including in comorbid subgroups.
This new exploratory analysis of the PUNCH™ CD3 trial in which improvements in HRQoL were associated with changes in microbiome and metabolome composition. In a previous exploratory analysis, patients who responded to REBYOTA vs. placebo had a healthier gut microbiome composition with an increase in the relative abundance of beneficial gut microbiota (Bacteroidia and Clostridia) and a decrease in other bacteria (Gammaproteobacteria and Bacilli). This new analysis found that the greatest improvements in HRQoL – as measured using the disease-specific C. difficile Quality of Life Survey (Cdiff32), which examines physical, mental and social domains – were associated with the mental health domain, with the largest improvements associated with higher levels of Bacteroidia and Clostridia and lower levels of other bacteria.
To learn more about REBYOTA and other information, please visit REBYOTA.com or www.REBYOTAHCP.com.
About C. diff infection
C. diff infection is a serious and potentially deadly infection that impacts people across the globe. The C. diff bacterium causes debilitating symptoms, such as severe diarrhea, fever, stomach tenderness or pain, loss of appetite, nausea and colitis (an inflammation of the colon). 1 C. diff infection can be the start of a vicious cycle of recurrence, causing a significant burden for patients and the healthcare system. 2,3 It has been estimated that up to 35% of C. diff infection cases recur after initial diagnosis and people who have had a recurrence are at significantly higher risk of further infections. 4,5,6,7 After the first recurrence, it has been estimated that up to 65% of patients may develop a subsequent recurrence. 6,7 Antibiotics – the current standard of care for treatment of C. diff infection – treat the disease but can also be a contributing factor to the cycle of recurrence. 1
About REBYOTA
REBYOTA is a pre-packaged, single-dose 150 mL microbiota suspension for rectal administration consisting of a liquid mix of up to trillions of live microbes – including Bacteroides. REBYOTA is delivered directly to the gut microbiome and is administered by a healthcare professional in one visit.
INDICATION
REBYOTA (fecal microbiota, live – jslm) is indicated for the prevention of recurrence of Clostridioides difficile (C. diff) infection in individuals 18 years of age and older, following antibiotic treatment for recurrent C. diff infection.
Limitation of Use
REBYOTA is not indicated for the treatment of C. diff infection.
IMPORTANT SAFETY INFORMATION
You should not receive REBYOTA if you have a history of a severe allergic reaction (e.g., anaphylaxis) to REBYOTA or any of its components.
You should report to your doctor any infection you think you may have acquired after administration.
REBYOTA may contain food allergens.
Most common side effects may include stomach pain (8.9%), diarrhea (7.2%), bloating (3.9%), gas (3.3%), and nausea (3.3%).
REBYOTA has not been studied in patients below 18 years of age.
Clinical studies did not determine if adults 65 years of age and older responded differently than younger adults.
You are encouraged to report negative side effects of prescription drugs to FDA. Visit www.FDA.gov/medwatch or call 1-800-332-1088.
Please click to see the full Prescribing Information.
About Ferring Pharmaceuticals
Ferring Pharmaceuticals is a privately-owned, research-driven, specialty biopharmaceutical group committed to building families and helping people live better lives. In the United States, Ferring is a leader in reproductive medicine, and in areas of gastroenterology and orthopaedics. We are at the forefront of innovation in microbiome-based therapeutics and uro-oncology intravesical gene therapy. The company was founded in 1950 and is headquartered in Saint-Prex, Switzerland. Ferring employs more than 7,000 people worldwide and markets its medicines in over 100 countries. Ferring USA is based in Parsippany, New Jersey, and employs more than 900 employees.
For more information, please visit www.ferringusa.com, call 1-888-FERRING (1-888-337-7464), or connect with us on LinkedIn, and X.
About DDW 2025
Digestive Disease Week® (DDW) is the largest international gathering of physicians, researchers and academics in the fields of gastroenterology, hepatology, endoscopy and gastrointestinal surgery. Jointly sponsored by the American Association for the Study of Liver Diseases (AASLD), the American Gastroenterological Association (AGA), the American Society for Gastrointestinal Endoscopy (ASGE) and the Society for Surgery of the Alimentary Tract (SSAT), DDW is an in-person and online meeting from May 3-6, 2025. The meeting showcases more than 5,600 abstracts and hundreds of lectures on the latest advances in GI research, medicine and technology. More information can be found at www.ddw.org.
References:
Centers for Disease Control and Prevention. What is C. diff? 7 Sep. 2022. Available at: https://www.cdc.gov/c-diff/about/index.
Centers for Disease Control and Prevention. 2019 Antibiotic Resistance Threats Report: Clostridioides difficile. 23 Nov. 2021. Available at: https://www.cdc.gov/drugresistance/pdf/threats-report/clostridioides-difficile-508.pdf.
Feuerstadt P, et al. Healthcare resource utilization and direct medical costs associated with index and recurrent Clostridioides difficile infection: a real-world data analysis. J Med Econ. 2020;23(6):603-609.
Riddle DJ, Dubberke ER. Clostridium difficile infection in the intensive care unit. Infect Dis Clin North Am. 2009;23(3):727-743.
Nelson WW, et al. Health care resource utilization and costs of recurrent Clostridioides difficile infection in the elderly: a real-world claims analysis. J Manag Care Spec Pharm. 2021 Jul;27(7):828-838. doi: 10.18553/jmcp.2021.20395. Epub 2021 Mar 11.
Kelly, CP. Can we identify patients at high risk of recurrent Clostridium difficile infection? Clin Microbiol Infect. 2012;18 (Suppl. 6): 21–27.
Smits WK, et al. Clostridium difficile