Rebiotix, Ferring Announces Positive Phase 3 Data of World’s First Microbiome-Based Therapeutic

By T. Chakraborty, Ph.D.

The human gut comprises of millions of bacteria that harbor a symbiotic relationship with the body. These microbiomes are responsible for regulating the immune system, metabolism, and other normal physiological functions. The microbiome, which plays a very important role in our overall health, are housed most densely in the gastrointestinal tract.

Research from different quarters indicates that gut microbiota can be irritated and disturbed by the presence of antibiotics, viruses, and changes in lifestyle including stress. This disruption is termed as ‘dysbiosis’ which in turn promotes favorable conditions for different infections such as the ones caused by Clostridioides difficile (C. diff)

C. diff, a bacterium responsible for causing diarrhea and colitis is estimated to cause up to half a million illnesses in the US alone every year, which can lead to major complications resulting in death in extreme cases. Antibiotics, although the most common cure to such issues has a problem of recurrence in approximately 15% to 50% of the patients [1,2].

Positive Trial Data of Microbiome-Based Therapy

Rebiotix, a part of the Ferring Pharmaceuticals Group describes itself as a late-stage clinical microbiome company. It is primarily looking into unleashing the power of the human microbiota against many diseases. The company boasts of a huge pipeline of investigational drugs built on its microbiota-based, proprietary MRT™ drug platform. The investigational drug technologies developed by the platform are designed to potentially restore the human microbiome by delivering a broad consortium of live microbes to the patient’s intestinal tract [3].

Both Rebiotix and Ferring are trying to bring potential microbiota-based therapies to the market to counter illnesses caused by C. diff. Their RBX2660 is the first-in-class microbiota-based therapy which has shown positive Phase 3 results by meeting primary efficacy endpoints.

RBX2660 is developed to disrupt the recurrence of C. diff infection. The US Food and Drug Administration has granted this therapy designations such as Fast Track, Orphan, and Breakthrough Therapy. The recent phase 3 trial named as PUNCH CD3, is a randomized, multicenter, double-blinded, placebo-controlled study.

C. diff infection is a significant public health threat that has limited treatment options. This phase 3 trial also embodies a safety assessment which prescribes the following patients for an extended period after receiving the drug. Doing so will help provide the company with invaluable insights into the potential of microbiota-based therapies.

The president and CEO of Ferring, Per Falk said “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. 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.

“The positive preliminary data on the primary efficacy endpoint are a major steppingstone 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.”

Related Article: Korean Firm, Asia’s First to Get FDA Clearance for Testing Anti-Cancer Microbiome Therapeutic

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. https://www.rebiotix.com/news-media/press-releases/rebiotix-announces-worlds-first-positive-pivotal-phase-3-data-investigational-microbiome-based-therapy-rbx2660/

Author

Tulip Chakraborty