All Diseases May Not Begin in the Gut, But Many of Them Will End There

By Adam Goulburn and Josh Wolfe

Diabetes. Parkinson’s. Depression. Anxiety. NASH. ADHD. Multiple gastrointestinal disorders and inflammatory disorders. All are illnesses that are linked to problems in the gut-brain axis. Treatment for any one of these diseases would be a multi-billion-dollar opportunity. A company that figures out how to tap into the many pathways between the gut and our internal organs could potentially develop a therapeutic solution for every one of these diseases — and become the next biotech titan.

A New York-based drug discovery biotech startup named Kallyope (cali-OHpay) aims to do just that. Today Kallyope announced a $66-million series B round of funding, bringing the total raised to $110 million. Two new investors, Euclidean Capital and Two Sigma Ventures, joined previous investors including our firm, Lux Capital, as well as Alexandria Ventures, The Column Group, Illumina Ventures, and Polaris.

Kallyope is led by two female scientists, Nancy Thornberry and Ann Weber, who together led the team that discovered the diabetes drug JANUVIA — Merck’s highest-selling drug ever, generating nearly $6 billion in revenue in 2017.

Five years ago my partners and I were intrigued by the prospect of accessing the body and the brain through the gut. Based on a new field of research, we held a fundamental belief that the microbiome and the idea of the gut as a sensing organ will change how we view and act upon human physiology. Yet very few biotech companies were paying attention to the mechanism of action.

We scoured the field for 24 months and discovered the work of three incredible scientists: Charles Zuker, Tom Maniatis (winner of a 2012 Lasker Award) and Richard Axel (winner of a 2004 Nobel Prize). Together, they presented a new approach to treating the brain — a method in which you could use the body’s circuitry to treat illnesses pharmacologically. Their research came at a time when the speed and progression of technologies such as single-cell sequencing, bioinformatics, gene editing technologies such as CRISPR (to enable high-throughput animal-model generation), and optogenetics (to demonstrate functional animal models) — made it possible to create something truly groundbreaking.

Once we found the scientific founders, we embarked on a search for business leaders who would be able to build on their research, work at the cutting edge of science and tech, think big, attract an all-star team of drug developers and create a world-class biotech company. We combed the industry for the right candidates and found it in Nancy Thornberry, CEO, and Ann Weber, SVP Drug Discovery. Over the past two and half years, they have built a data-driven, interdisciplinary team of single-cell sequencing researchers, gut biologists, chemists, neuroscientists, bioinformaticians, and genetic engineers.

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Kallyope views the gut as a window to the rest of the body, and is taking advantage of a paradigm shift in the way we treat metabolic and CNS disorders. It is a completely new way to target diseases: targeting gut-brain circuits with gut-restricted molecules. What does this all mean? With drugs restricted to the gut, there will be limited side effects. Without systemic distribution, therapeutic action will be delivered via the body’s internal circuits. CNS drugs will no longer need to cross the blood brain barrier to have therapeutic impact. All are potentially radical breakthroughs in medicine.

More than 2,000 years ago, Hippocrates said: “All disease begins in the gut.” Here’s hoping they’ll end there, too.

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