Turning Microbiome Therapy into Modern Panacea

The United States Food and Drug Administration’s (US FDA) approval of Seres Therapeutics’ (USA) VOWST in April 2023 and Ferring Pharmaceuticals’ (Switzerland) REBYOTA in November 2022, has marked a pivotal moment in the microbiome therapeutics landscape. There are many disease areas with significant unmet medical needs that are linked to the state of an individual’s microbiome. In recent years, there has been growing interest and research in the field of microbiome therapies. Researchers are exploring the potential of manipulating the microbiome to prevent, treat, or manage a wide range of health issues, including gastrointestinal disorders, metabolic disorders, autoimmune diseases, and neurological disorders. Let’s explore this new era of microbiome therapeutics and challenges in developing such therapies.

- Ayesha Siddiqui

A new class of drugs, microbiome therapeutics are gaining prominence. The obvious initial therapeutic area for gut microbiome modalities is in infectious diseases as evidenced by the recent approvals. There are three approved microbiome-based drugs. Seres Therapeutics’ VOWST and Ferring Pharmaceuticals’ REBYOTA have been approved by the US FDA. BiomeBank’s Biomictra has received approval from Therapeutic Goods Administration (TGA) in Australia. All three are approved for Clostridium difficile bacteria infection.

According to GlobalData’s Pharma Intelligence Centre, there are currently 388 microbiome therapeutics under development. The most targeted therapy areas for these agents are gastrointestinal conditions, with 106 agents, followed by infectious diseases with 74 agents, and oncology with 65 agents.

Cancer 

Researchers and companies are actively exploring the potential of microbiome-based therapies in oncology. Various studies evaluating microbiome-based therapies to enhance the efficacy of cancer immunotherapy are currently underway.

In 2022, South Korean firm Genome & Company collaborated with American pharma giant Merck, for a phase 2 trial involving its cancer blockbuster drug Keytruda. The trial aimed to evaluate the efficacy of Genome & Company’s lead product, GEN-001, in treating biliary tract cancer patients. Genome & Company’s lead pipeline GEN001 is said to be the first candidate for an immune-oncology microbiome therapeutic in Korea. Currently, clinical studies (Phase I/Ib) targeting multiple solid cancer patients (non-small cell lung cancer, head and neck cancer, urothelial cancer) are being conducted in the US and Korea.