First Real-World Outcomes Data and New Analyses of Data for REBYOTA® (fecal microbiota, live – jslm) Presented at DDW 2024

• First real-world outcomes analysis evaluated efficacy of REBYOTA in patients with recurrent C. diff infection
• Ad-hoc analysis evaluated efficacy and safety of REBYOTA in participants taking common non-antibiotic medications associated with microbiome imbalance and recurrent C. diff infection
• Post-hoc analysis looked at gut microbiome composition and diversity in patients following REBYOTA administration

Ferring Pharmaceuticals today announced three poster presentations at Digestive Disease Week^® (DDW 2024) that add to the robust clinical evidence for REBYOTA^® (fecal microbiota, live – jslm), including the first real-world outcomes data assessing the use of this therapy in patients with recurrent C. difficile (C. diff) infection. REBYOTA is the first and only single-dose microbiome-based therapy approved by the U.S. Food and Drug Administration (FDA) for the prevention of recurrent C. diff infection in individuals 18 years of age and older, following antibiotic treatment for C. diff infection.

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In the real-world experience analysis (poster number Sa1916), medical records were reviewed for patients with recurrent C. diff infection who received REBYOTA in outpatient clinics from February 2023 through November 2023 (n=46). Those with 8-week follow-up were further evaluated and the efficacy of REBYOTA was assessed as the absence of recurrence at 8 weeks after administration. Nearly 74% of these patients (25/34) maintained the effectiveness of REBYOTA at eight weeks, despite having a higher risk of further recurrence.

An ad-hoc subgroup analysis (poster number Sa1901) of the PUNCH CD3-Open-Label Study (OLS) – an ongoing Phase 3 REBYOTA trial – evaluated the efficacy and safety of REBYOTA in participants taking certain medications that may cause an imbalance in the gut microbiome (dysbiosis) and can increase the risk of recurrent C. diff infection. Of 469 adult participants with documented recurrent C. diff infection in the modified intent to treat (mITT) efficacy population, outcomes were assessed in 290 patients who were taking proton pump inhibitors (PPI) (42.1%), statins (48.6%) and/or psychotropic medications, such as antidepressants and antianxiety medicines (55.2%), for a period of two weeks prior and two or more weeks after REBYOTA administration. Treatment success was defined as the absence of CDI recurrence within 8 weeks of REBYOTA administration and participants were monitored for treatment-emergent adverse events (TEAEs) for up to 6 months after administration.

Treatment success rates at 8 weeks were 78.7% of participants taking PPIs, 75.2% of statin users and 75.2% of those receiving psychotropic medications. Participants had comparable treatment success rates whether they were receiving one of these medications or multiple medications concurrently.

“Prospective clinical trials often exclude the types of patients we commonly see in clinical practice including those who are taking additional medication for other conditions that may increase their risk for recurrent C. difficile infection," said Paul Feuerstadt, MD, FACG, AGAF, Yale University School of Medicine. "This analysis provides data on this patient population who clinicians see every day."

In the analysis, most TEAEs were mild or moderate gastrointestinal disorders and related to preexisting conditions or C. diff infection. Serious TEAEs occurred in 15.9% of participants receiving PPIs, 15.9% of participants receiving statins and 15.5% of participants receiving psychotropic medications. All serious TEAEs were assessed as unrelated to REBYOTA administration.

A post-hoc analysis from the PUNCH CD3 Phase 3 clinical trial (poster number Sa1910) examined the gut microbiota composition and diversity among participants who received REBYOTA and had treatment success at eight weeks — defined as remaining free of C. diff infection recurrence. Patients provided stool samples at baseline (before study treatment) and at 1 week, 4 weeks, 8 weeks, 3 months and 6 months after study treatment. The analysis evaluated whether the species of bacteria present after treatment were those present in the REBYOTA dose they received and the durability of those changes.

The composition and diversity of the gut microbiota of participants who received REBYOTA shifted toward the REBYOTA profile as early as one week after administration. By week 1, a median of 10 species (range 0 to 78) clonally engrafted from REBYOTA to participants administered therapy, with significantly greater engraftment in those who were therapy responders versus non-responders. Participants who received placebo had no significant clonal engraftment. For at least six months, the number of REBYOTA responders with engraftment continued to rise and persist. Bacteroidia species showed the highest median engraftment effectiveness.

DDW 2024 has abstracts available on its website.

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 maternal health, and in areas of gastroenterology and orthopaedics. We are at the forefront of innovation in microbiome-based therapeutics and uro-oncology intravesical gene therapy. Our 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 (Twitter).

About DDW

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 18-21, 2024. The meeting showcases more than 4,400 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:

1. Centers for Disease Control and Prevention. What is C. diff? 7 Sep. 2022. Available at: https://www.cdc.gov/cdiff/what-is.html.
2. 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.
3. 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.
4. Riddle DJ, Dubberke ER. Clostridium difficile infection in the intensive care unit. Infect Dis Clin North Am. 2009;23(3):727-743.
5. 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.
6. Kelly, CP. Can we identify patients at high risk of recurrent Clostridium difficile infection? Clin Microbiol Infect. 2012; 18 (Suppl. 6): 21–27.
7. Smits WK, et al. Clostridium difficile infection. Nat Rev Dis Primers. 2016;2:16020. doi: 10.1038/nrdp.2016.20.

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