Welcome to today’s Biotech Spotlight, a series featuring companies that are creating breakthrough technologies and products. Today, we’re looking at Anokion, a Swiss biotech pioneering a vaccine to treat autoimmune diseases by halting the immune system’s attack on healthy tissues, while allowing it to function normally.
In focus with: Deborah Geraghty, CEO and president, Anokion
Anokion’s vision: Autoimmune diseases present a significant challenge — the bad actor is the patient’s own immune system, waging a misguided attack on a harmless self-antigen in the body. Now, Switzerland’s Anokion is testing “inverse vaccines,” which are designed to stop this harmful immune reaction. The approach has reversed multiple sclerosis-like illnesses in laboratory mice and shown clinical promise in early human trials for multiple sclerosis and celiac disease.
Unlike traditional vaccines that give the immune system a glimpse of a pathogen to prime it to fight, inverse vaccines reintroduce the friendly self-antigen through a process that encourages the immune system to ignore it while functioning normally.
Why it matters: The immune system is a powerful force, so when it mistakes a friendly self-antigen for a foreign invader and mounts an attack, the effects can be devastating. Autoimmune diseases can cause digestive problems, rashes, pain, and damage joints and organs, sometimes leading to disability or death.
About 50 million Americans are affected by more than 80 autoimmune diseases, and treating them costs more than $100 billion annually, according to Anokion. The global market for autoimmune treatments, which includes blockbuster meds like Stelara and Enbrel, is expected to reach $185 billion by 2029.
"What is so exciting about this work is that we have shown that we can treat diseases like multiple sclerosis after there is already ongoing inflammation, which is more useful in a real-world context.”
CEO and president, Anokion
Current treatments most often suppress the immune system, which can help alleviate symptoms, but open the door to infections, cancer and other side effects. And they don’t resolve the underlying immune dysfunction at the core of the disease.
A new spin on an old idea: Retraining the immune system to recognize and ignore harmless self-antigens to treat autoimmune diseases isn’t a new idea, Geraghty said.
“This concept has been around for quite some time,” she said. “Others have taken different approaches using nanotechnology, or just delivering straight antigen, but they’ve run into some hurdles over the years.”
Some experiments in animals succeeded but later fell short in human trials. Often, it’s hard to determine what self-antigen the body is reacting to, or if it’s reacting to more than one.
Anokion is using a new approach pioneered by Jeffrey Hubbell, the company’s co-founder, and colleagues at the University of Chicago’s Pritzker School of Molecular Engineering that Geraghty said may succeed where others failed.
The inverse vaccine directs the self-antigen to the liver — an often-underappreciated immune system player — which can signal the immune system to leave certain cells alone. The Anokion vaccines use engineered proteins and a sugar to encourage the liver to label the self-antigen as a friend, not a foe. This can suspend the immune attack, halting and potentially reversing disease progression, a process outlined in a recent study.
“In the past, we showed that we could use this approach to prevent autoimmunity,” Hubbell said, in a written release. “But what is so exciting about this work is that we have shown that we can treat diseases like multiple sclerosis after there is already ongoing inflammation, which is more useful in a real-world context.”
Testing the concept: Inverse vaccines would likely be given in as an introductory dose followed by regular boosters to keep the immune system from veering off track, Geraghty said. Human trials in celiac disease and multiple sclerosis are underway, and the company has attracted a $35 million equity investment from pharma giant Pfizer, which it is also partnering with on research.
The vaccines have achieved notable milestones in early-stage trials. A phase 1 trial of KAN-101, the celiac disease vaccine, found it was safe and reduced T-cell response following a gluten challenge. Enrollment for the first of two phase 2 celiac studies began earlier this year, with topline data expected in 2024.
A second vaccine, ANK-700, is in phase 1 for multiple sclerosis, a disease of the brain and spinal cord caused by immune attacks on myelin, the protective sheath that encases nerve fibers. Destruction of this coating slows or blocks nerve function, causing a host of symptoms that include numbness, tingling, spasms and balance problems. To combat it, the vaccine delivers a myelin antigen to reduce brain and spinal cord inflammation.
Preliminary biomarker data from the multiple sclerosis phase 1 trial showed a trend toward immune tolerance and bystander suppression, which is an expanded immune tolerance against other peptides or antigens that aren’t in the drug, something that’s crucial when treating this complex condition, Geraghty said.
“We’re the first company that has been able to demonstrate that,” she added. Anokion anticipates having full results in the second half of 2024, and a third vaccine for type 1 diabetes is also in early stages.
The road ahead: The prospect of a durable autoimmune treatment that can reverse symptoms with fewer side effects has generated substantial enthusiasm from patient communities, Geraghty said. But it remains to be seen if these vaccines will deliver.
“Next year is really important,” Geraghty said. “I’m excited to advance these pivotal studies.”
The company is also exploring other mechanisms to reset the immune system, which could broaden its targets, allowing it to target other conditions with large markets, such as Crohn's disease and rheumatoid arthritis, where the antigen target is less clear, said Geraghty.
Anokion’s ultimate goal is to improve patients’ lives, she said, “and to do that in an innovative and novel way that is specific and reduces potential side effects that are associated with some of these broader active agents.