Seres’s success with an industrially made bacterial mix in phase 3 trials against Clostridium difficile infection promises an alternative to fecal microbial transplantation in clinical practice.
The August announcement of positive data from Seres Therapeutics’ phase 3 trial to prevent recurrent Clostridium difficile infections renewed hopes that purified, defined mixtures of bacteria can treat such infections as effectively as — and presumably more safely than — fecal microbial transplantation (FMT). Several other companies have now also shown favorable late-stage trial data in ‘C. diff.’ using customized stool-derived cocktails. Along with Seres, they are teeing up clinical studies of these compounds in a variety of diseases.
……..Two other companies, Rebiotix, and Vedanta Biosciences are pursuing microbe-based drugs to stop C. diff infection recurrences, and both are in late-stage development. Rebiotix is running a pivotal phase 3 study of its commercially prepared FMT in enema form. Vedanta Biosciences is expecting a readout of its phase 2 trial of VE303, which, unlike the other three drug candidates, is a defined consortium of eight types of cultured, clonal commensal bacteria strains.
This is a promising approach to managing CDI. Completion of a Phase 3 study, with positive results, is exciting and holds much promise for patients suffering with recurrent C. diff. infections.
We look forward to the final results and are truly grateful.
Rebiotix and Ferring announce world’s first with positive preliminary pivotal Phase 3 data for investigational microbiome-based therapy RBX2660
Rebiotix and Ferring are the first to announce positive preliminary results on primary
efficacy endpoint from ongoing pivotal Phase 3 clinical trial for RBX2660
RBX2660 is an investigational, non-antibiotic, microbiome-based therapy, developed to
reduce Clostridiodes difficile (C. diff) infection recurrences
The CDC defines C. diff as a major burden to patients and doctors and an urgent healthcare
threat causing an estimated half a million illnesses and thousands of deaths annually in the
US alone ( 1 , 2)
Source: Press Release
Roseville, Minnesota and Saint-Prex, Switzerland – 6 May, 2020, 07:00 EST –
Today, . These preliminary positive efficacy findings mark an important milestone, advancing RBX2660 in its clinical development program with a goal of bringing a US FDA approved therapy to patients. The clinical development program for RBX2660 is the most advanced in the world in evaluating the safety and efficacy of a standardized, non-antibiotic microbiome-based therapy.
RBX2660 is being developed to reduce C. diffinfection recurrences, an urgent unmet need for
patients and healthcare providers worldwide. Antibiotics, the current standard of care, have been shown to disrupt the microbiome and increase the risk of C. diff recurrence. 3
C. diff causes nearly 30,000 deaths each year in the US; in Europe, the incidence of C. diff is increasing, with recurrent bouts of infection representing 10-15% of all healthcare-related infections in hospitals annually. 4 , 5
As a live biotherapeutic, aiming to help restore the gut microbiome community, RBX2660 may bring an innovative therapeutic option to patients suffering from this potentially deadly infection. “C. diff infection is a significant public health threat that has limited treatment options. These positive preliminary findings represent a major step forward towards bringing an innovative, non-antibiotic option to patients that may help restore their gut microbiome, said Per Falk, Ferring’s President and Chief Science Officer. With health systems under increasing pressure due to viruses like COVID-19 and the rising threat of antimicrobial resistance, the need for new therapies is greater than ever. We believe the power of the microbiome has great potential and we look forward to bringing RBX2660 to patients soon.”
“Since founding Rebiotix in 2011, our mission has been to harness the power of the microbiome to treat complex diseases. Our first goal was to address C. diff, which poses a significant health threat to thousands worldwide every year,” said Lee Jones, CEO and founder of Rebiotix, a Ferring company.
The positive preliminary data on the primary efficacy endpoint are a major stepping stone
for the RBX2660 development program, bringing us closer to an approved microbiome therapy
available for healthcare providers to help patients. As a first-in-class, potentially paradigm-changing technology, we look forward to discussing our final data with the FDA in the latter part of this year.” The ongoing Phase 3 trial is a randomized, multicenter, double-blinded, placebo-controlled study. The trial also incorporates a safety assessment intended to follow patients for several months after receiving the investigational drug. The safety data will provide insight into the potential of using microbes as a therapeutic intervention. The full data package is anticipated in the second half of 2020.
This trial builds on nearly a decade of research and evaluation of the formulation, with robust clinicaland microbiome data collected over multiple controlled trials under the proprietary MRT drug platform.
About Clostridioides difficile infection (C. diff)
C. diff is a bacterium that causes diarrhea and colitis (an inflammation of the colon). 6 It is estimated to cause up to half a million illnesses in the US alone every year and is considered an urgent threat to public health by the CDC, and can lead to severe complications, including hospitalization, surgery, and death. 2 While antibiotics are the standard of care to address the infection, they are also the primary risk factor for disease recurrence. 3 Recurrence of C. diff occurs in approximately 15- 50% of patients. 7
About the microbiome
The human microbiome is a complex community of microorganisms which live on every surface of the body. The microbiome aids in the maintenance and development of the immune system,
metabolism, and other functions essential to human life. 8 The gastrointestinal tract houses the most dense and complex population of microbiota, which has an incredible influence over daily health – from aiding in food digestion to fighting disease. Clinical and scientific studies indicate antibiotics, viruses, stress and other factors can disturb the gut microbiota. This disruption, often referred to as “dysbiosis,” may have negative health impacts, and promote conditions for infections like C. dif infection to take hold. 9
Rebiotix and Ferring believe there is tremendous potential in microbiota-based therapies to address such illnesses, and are evaluating this therapeutic option through their
pioneering microbiota-based MRT drug platform, beginning with recurrent C. diff infection.
The investigational RBX2660 formulation is the first-in-class microbiota-based therapy to achieve positive preliminary Phase 3 study results. RBX2660 is being developed to help break the cycle of recurrent C. diff infection. The therapy has been granted Fast Track, Orphan, and Breakthrough Therapy designations from the US FDA. The RBX2660 ongoing pivotal Phase 3 trial, PUNCH CD3, is a randomized, multicenter, double-blinded, placebo-controlled study. For more information about the RBX2660 Phase 3 study, visit http://www.clinicaltrials.gov (NCT03244644).
About Ferring Pharmaceutical 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. 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 http://www.ferring.com, or connect with us on Twitter, Facebook, Instagram, LinkedIn and YouTube.
Rebiotix Inc, part of the Ferring Pharmaceuticals Group, 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 http://www.rebiotix.com, or connect with us on Twitter, Facebook, LinkedIn and YouTube.
For more information, please contact
Rebiotix Inc., a Ferring Company
+1 651 705 8774 (direct)
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 Theriot CM, Young VB. Microbial and metabolic interactions between the gastrointestinal tract and
Clostridium difficile infection. Gut Microbes. 2013;5(1):86-95. doi:10.4161/gmic.27131.
4 Lessa FC, Mu Y, Bamberg WM, et al., Burden of Clostridium difficile Infection in the United States. New
England Journal of Medicine. 2015;372(9):825-834. doi:10.1056/nejmoa1408913.
5 DRG Report 2016 Clostridium Difficile.
6 Centers for Disease Control and Prevention. Clostridiodes difficile Fact Sheet. Available at:
7 Stevens VW, Nelson RE, Schwab-Daugherty EM, et al., Comparative Effectiveness of Vancomycin and
Metronidazole for the Prevention of Recurrence and Death in Patients with Clostridium difficile Infection.
JAMA Intern Med. 2017;177(4):546–553. doi:10.1001/jamainternmed.2016.9045.
8 Mohajeri MH, Brummer RJM, Rastall RA, et al., The role of the microbiome for human health: from basic
science to clinical applications. Eur J Nutr. 2018;57(Suppl 1):1–14. doi:10.1007/s00394-018-1703-4.
9 Quigley EM. Gut bacteria in health and disease. Gastroenterol Hepatol (N Y). 2013;9(9):560–569.
Rebiotix, a Ferring Company, completes enrollment for first-ever, pivotal Phase 3 Clinical Trial of Microbiota -based RBX2660
Enrollment completion for the first Phase 3 clinical trial in microbiome industry
The largest randomized, double-blinded study, with over 300 patients enrolled aimed to demonstrate the potential benefit of RBX2660 in reducing rates of recurrent Clostridioides difficile (C. diff) infection
Rebiotix intends to use the results from the Phase 3 trial to serve as the basis for licensure application to the US Food and Drug Admin (FDA)
Saint-Prex, Switzerland – On February 4, 2020
Rebiotix, a Ferring company, announced today that it has completed enrollment of the pivotal Phase 3 clinical trial for RBX2660, an investigational therapy aimed at breaking the cycle of recurrent Clostridioides difficile (C. diff) infection, which is responsible for the deaths of thousands of people in the US alone. The Centers for Disease Control and Prevention (CDC) has classified C. diff as an urgent public health threat, with limited options for treatment.
RBX2660 was developed under Rebiotix’s investigational microbiota-based MRT drug platform with the goal of delivering standardized, stabilized formulations to meet unmet medical needs. Conducted in the US and Canada, this is the first Phase 3 trial of its kind to be completed using a broad consortia microbiota-based formulation.
“Rebiotix was founded to harness the power of the human microbiome to treat debilitating diseases,” said Lee Jones, Rebiotix Founder, and CEO. “Microbiota-based therapies have shown tremendous potential as an innovative, non-antibiotic therapy, starting with C. diff. The completion of enrollment of this trial is a critical next step in making microbiota-based products accessible to patients – we are excited about this important milestone and look forward to sharing results later this year.”
The Phase 3 trial builds on the company’s extensive history with the formulation, including several hundred participants previously enrolled in multiple Phase 2 clinical trials. The robust data collected over the course of the company’s multi-year clinical development program will be eventually presented to the US FDA as part of a Biological License Application (BLA).
Ferring Pharmaceuticals, also with a rich and vast history of microbiome research of its own, led the industry by becoming the first major pharmaceutical company to acquire a microbiome therapeutics company in April 2018. Headquartered in Saint-Prex, Switzerland, Ferring is expected to have the first regulatory approved microbiota-based therapeutic in the world through the potential approval of the RBX2660 in the US.
About Clostridioides difficile Infection
Clostridioides difficile (also known as C. diff) is a bacterium that causes diarrhea and colitis (inflammation of the colon). C. diff, impacts nearly a half a million people each year in the United States; of those impacted, up to one in five patients will experience a recurrent episode.1 In 2019, the U.S. Centers for Disease Control listed C. diff as an urgent threat to public health.2
RBX2660 is currently in Phase 3 clinical development for the reduction of recurrent Clostridioides difficile (C. diff) infection. RBX2660 has been granted Fast Track, Orphan, and Breakthrough Therapy Status designations from the US FDA. For more information about the RBX2660 Phase 3 study, visit http://www.clinicaltrials.gov (NCT03244644).
Rebiotix Inc., part of the Ferring Pharmaceuticals Group, 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 http://www.rebiotix.com.
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. 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.