Ferring and Rebiotix Present Landmark Phase 3 Data Demonstrating Superior Efficacy of Investigational RBX2660 Versus Placebo to Reduce Recurrence of C. difficile Infection

Ferring Pharmaceuticals and Rebiotix, a Ferring Company, today presented results from the pivotal Phase 3 PUNCH™ CD3 clinical trial, demonstrating superior efficacy and consistent safety of single-dose RBX2660 in reducing recurrence of Clostridioides difficile infection (CDI) over placebo. RBX2660 is an investigational, potential first-in-class microbiota-based live biotherapeutic.

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The trial, presented today at Digestive Disease Week^® (DDW) 2021, successfully met its primary endpoint. RBX2660 demonstrated superior efficacy versus placebo (70.4% and 58.1%, respectively) at 8 weeks post treatment, with a comparable safety profile to placebo. RBX2660 results demonstrated statistical significance with a 98.6% posterior probability of superiority, which exceeded the 97.5% minimum threshold. In addition to these outcomes, RBX2660 provided a relative reduction of recurrence of 29.4% compared to placebo. The majority of treatment emergent adverse events (TEAEs) for RBX2660 were similar to placebo, and mild to moderate in nature. These data add to the large body of evidence showing consistent efficacy and safety in patients who have received RBX2660, which may help address the unmet need for patients who suffer from this debilitating and potentially deadly recurrent infection.

“C. difficile infection is a global public health threat that requires immediate action to halt the unrelenting cycle of recurrence. While necessary to treat initial infection, antibiotics are also a predominant risk factor for recurrence because they can disrupt the gut microbiome, leaving the current treatment paradigm for recurrent infection incomplete,” said Paul Feuerstadt, MD, FACG, AGAF, PACT Gastroenterology, Hamden, Conn., Assistant Clinical Professor of Medicine, Yale University School of Medicine, New Haven, Conn., and RBX2660 clinical trial investigator. “These Phase 3 RBX2660 results, as part of the overall clinical development program, show consistent efficacy as early as a first recurrence of C. difficile infection by delivering a broad consortium of live microbes to the area of active infection.”

“People who suffer from C. difficile infection are devastated when they experience recurrence. Patients have told me that they felt hopeless when the infection returned again and again despite multiple courses of antibiotic treatment. They believed that the infection would never go away,” said Christine Lee, MD, FRCPC, Clinical Professor, Department of Pathology and Laboratory Medicine, UBC Faculty of Medicine, Medical Microbiologist and Researcher, Island Health, Vancouver, and RBX2660 clinical trial investigator who presented the data at DDW. "The findings from this pivotal Phase 3 trial of RBX2660 are very encouraging to both patients and healthcare providers, providing hope this potential new treatment could make a meaningful difference in the lives of patients with recurrent C. difficile infection."

The RBX2660 program is the largest and most robust clinical program ever conducted in the field of microbiome-based therapeutics. The decade-long development program consists of six trials with more than 1,000 patients enrolled; two of these trials are the only ones in the field to include two years of follow-up.

“These Phase 3 results are a testament to a decade of robust clinical research to help address a significant unmet patient need,” said Lee Jones, President and CEO of Rebiotix, a Ferring Company. We are deeply grateful to the patients and clinicians for their years of dedication to this program.”

“At Ferring, we are dedicated to helping people live better lives,” said Per Falk, President of Ferring Pharmaceuticals. “We look forward to sharing our data with the U.S. FDA as we believe, based on the totality of evidence, RBX2660 holds the potential to be an improvement over the standard of care alone for tens of thousands of patients affected every year by recurrent C. difficile infection.”

About the PUNCH™ CD3 Clinical Trial (Clinicaltrials.gov identifier: NCT03244644)

PUNCH™ CD3 is a Phase 3, prospective, multicenter, randomized, double-blinded, placebo-controlled clinical trial evaluating the efficacy and safety of RBX2660 vs. placebo in preventing rCDI. The study included adults ages 18 or older who had at least one recurrence after a primary episode of CDI. Participants were followed up to 8 weeks for the efficacy analysis, and up to six months for the safety analysis.

About RBX2660

RBX2660 is a potential first-in-class microbiota-based live biotherapeutic being studied to deliver a broad consortium of diverse microbes to the gut to reduce recurrent C. difficile infection. RBX2660 has been granted Fast Track, Orphan, and Breakthrough Therapy designations from the U.S. Food and Drug Administration (FDA). The pivotal Phase 3 program builds on nearly a decade of research with robust clinical and microbiome data collected over six controlled clinical trials with more than 1,000 participants.

About the microbiome and C. difficile infection

The microbiome is a highly-diverse microbial community that plays an essential role in human health. There is a growing body of evidence that shows when there is a disruption of the composition and/or diversity of the gut microbiome, there may be an associated risk for serious illnesses, such as C. difficile infection.

C. difficile is a bacterium that causes debilitating symptoms such as severe diarrhea, fever, stomach tenderness or pain, loss of appetite, nausea and colitis (an inflammation of the colon).¹ Estimated to cause up to half a million illnesses and thousands of deaths annually in the U.S. alone every year, C. difficile infection is considered an urgent threat to public health by the CDC and can lead to severe complications, including hospitalization, surgery, sepsis and death.^1,2C. difficile infection is often the start of a vicious cycle of recurrence, causing a significant burden for patients and the healthcare system.^3,4 The use of antibiotics has been shown to disrupt the ecology of the gut microbiome, and are a predominant risk factor for C. difficile recurrence – occurring in up to 35% of patients after initial C. difficile infection diagnosis.^5,6,7 After the first recurrence, it has been estimated that up to 60% of patients may develop a subsequent recurrence.⁸

About Ferring Pharmaceuticals

Ferring Pharmaceuticals is a research-driven, specialty biopharmaceutical group committed to helping people around the world build families and live better lives. Headquartered in Saint-Prex, Switzerland, Ferring is a leader in reproductive medicine and maternal health, and in specialty areas within gastroenterology and urology. Ferring has been developing treatments for mothers and babies for over 50 years and has a portfolio covering treatments from conception to birth. Founded in 1950, privately-owned Ferring now employs approximately 6,500 people worldwide, has its own operating subsidiaries in nearly 60 countries and markets its products in 110 countries. Learn more at www.ferring.com, or connect with us on Twitter, Facebook, Instagram, LinkedIn and YouTube.

Ferring is committed to exploring the crucial link between the microbiome and human health, beginning with the threat of recurrent C. difficile infection. With the 2018 acquisition of Rebiotix and several other alliances, Ferring is a world leader in microbiome research, developing novel microbiome-based therapeutics to address significant unmet needs and help people live better lives. Connect with us on our dedicated microbiome therapeutics development channels on Twitter and LinkedIn.

About Rebiotix

Rebiotix Inc, a Ferring Company, is a late-stage clinical microbiome company focused on harnessing the power of the human microbiome to revolutionize the treatment of challenging diseases. Rebiotix has a diverse pipeline of investigational drug products built on its pioneering microbiota-based MRT™ drug platform. The platform consists of investigational drug technologies designed to potentially rehabilitate the human microbiome by delivering a broad consortium of live microbes into a patient’s intestinal tract. For more information on Rebiotix and its pipeline of human microbiome-directed therapies for diverse disease states, visit www.rebiotix.com, or connect with us on Twitter, Facebook, LinkedIn and YouTube.

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) Institute, the American Society for Gastrointestinal Endoscopy (ASGE) and the Society for Surgery of the Alimentary Tract (SSAT), DDW is a fully virtual meeting from May 21-23, 2021. The meeting showcases more than 2,000 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? 17 Dec. 2018. Available at: https://www.cdc.gov/cdiff/what-is.html.
2. Centers for Disease Control and Prevention. Biggest Threats and Data, 14 Nov. 2019. Available at: https://www.cdc.gov/drugresistance/biggest-threats.html.
3. Centers for Disease Control and Prevention. 24 June 2020. Available at: https://www.cdc.gov/drugresistance/pdf/threats-report/clostridioides-difficile-508.pdf.
4. Feuerstadt P, et al. J Med Econ. 2020;23(6):603-609.
5. Lessa FC, Mu Y, Bamberg WM, et al. Burden of Clostridium difficile infection in the United States. N Engl J Med. 2015;372(9):825-834.
6. Cornely OA, et al. Treatment of First Recurrence of Clostridium difficile Infection: Fidaxomicin Versus Vancomycin. Clinical Infectious Diseases. 2012;55(S2):S154–61.
7. Langdon A, Crook N, Dantas G. The effects of antibiotics on the microbiome throughout development and alternative approaches for therapeutic modulation. Genome Med. 2016;8(1):39.
8. Leong C, Zelenitsky S. Treatment strategies for recurrent Clostridium difficile infection. Can J Hosp Pharm. 2013;66(6):361-368.

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