Intrinsic Medicine is focusing on inherently safe compounds, specifically human milk oligosaccharides (HMOs), to treat chronic diseases where immune dysregulation and dysbiosis of the microbiome play central roles. The company aims to address conditions like Parkinson’s disease, irritable bowel syndrome (IBS), inflammatory bowel disease (IBD), and autism spectrum disorder (ASD) by leveraging the unique properties of human milk biology.
Targeting the Gut-Immune-Brain Axis
Intrinsic Medicine is developing a library of pharmaceutical-grade HMOs to target disorders of the "gut-immune-brain axis (GIBA)." According to Alex Martinez, CEO of Intrinsic Medicine, these HMOs are not merely nutritional but possess drug-like properties. The company has two HMOs under active development and is building a research platform around its clinical pipeline.
Lead Compound: 2’Fucosyllactose (2’FL)
The company's lead compound, 2’Fucosyllactose (2’FL), is a synthetic-biology-produced drug identical to the most abundant HMO in human milk, constituting nearly 30% of the total HMO proportion. 2’FL has received regulatory approval in Australia to proceed directly from preclinical to a Phase 2 clinical trial for Parkinson’s disease and IBS. This HMO is known to be critical in establishing immune system homeostasis in early human development. Future indications for 2’FL include IBD, ASD, and immune oncology.
3’Sialyllactose (3’SL) and Autoimmune Disease
Data from the University of California, San Diego (UCSD) indicated that another HMO, 3’Sialyllactose (3’SL), showed efficacy in a mouse model of rheumatoid arthritis. Interestingly, HMOs start circulating in the blood of expectant mothers during the first trimester, coinciding with observations of remission in women with autoimmune diseases during pregnancy. This observation echoes the discovery of cortisol as a treatment for rheumatoid arthritis.
Further research from South Korea compared 3’SL to methotrexate in an animal model of rheumatoid arthritis, demonstrating equivalent efficacy to a high human dose of methotrexate (British Journal of Pharmacology, DOI: 10.1111/bph.14486). Intrinsic Medicine is now investigating a version of 3’SL, structurally identical to the natural form, as a disease-modifying drug for oligoarticular juvenile idiopathic arthritis and atopic dermatitis. Studies have shown that this HMO has a strong, direct anti-inflammatory effect in animals and human in vitro models.
Clinical Trials and Future Directions
Intrinsic Medicine's Phase 2 trial will study 2’FL in 180 Parkinson’s disease patients in Australia. The trial has received ethics approval and is awaiting funding to begin dosing. Additionally, an investigator-initiated study of pediatric IBD at Cincinnati Children’s Hospital is ongoing, involving 200 patients and utilizing the HMO-derived compound.
Martinez emphasizes the importance of considering the immune system in drug development, noting that 70% of immune cells reside in the gastrointestinal tract. He also highlights the potential of synthetic biology to manufacture human-identical HMOs at scale, paving the way for new therapeutic interventions targeting the gut-immune-brain axis.
