IBD Innovate: Accelerating IBD products through innovation

At the end of 2019, the Crohn’s & Colitis Foundation convened the second annual IBD Innovate, our unique showcase and networking event for innovators working in the inflammatory bowel disease (IBD) space.   

By bringing together developers engaged in the creation of new and innovative products and technologies for IBD patients, including representatives from biotech, pharmaceutical, venture capital, investment funds, academia, patient advocates, and nonprofits, the Foundation continues to pursue its goal of accelerating the pace of research for IBD.  

IBD Innovate builds on our IBD Ventures program, through which the Foundation invests directly in promising technologies with the potential to address the unmet needs of IBD patients in a new and innovative ways. Through IBD Innovate, the Foundation has built a platform to introduce selected innovators with investors and other appropriate resources and partners.  

At IBD Innovate 2019, 17 speakers from start-ups and academia showcased new products in therapeutics, diagnostics, devices, digital health, precision nutrition, and microbiome therapeutics. Four of the presenters were recipients of awards from the Foundation’s IBD Ventures program, and it was especially exciting to see their progress. Two presentations from patients-turned-entrepreneurs were therapeutically innovative as well as inspiring. Following is a synopsis of what’s in the pipeline and how new products are being developed.

New therapeutic tools, thanks to synthetic biology 

Keynote speaker Dr. Pamela Silver from Harvard Medical School highlighted the importance of synthetic biology (a new field of science that marries engineering with biology) in medical innovations. With faster and cheaper DNA sequencing and genome editing available, it is now possible to engineer living organisms (like bacteria and microphages) for therapeutic and other medical purposes. For example, bacteria, which Dr. Silver described as “exquisitely effective in identifying their environment and adapting,” could be used for diagnostic purposes.

Dr. Silver addressing the crowd at IBD Innovate

Dr. Silver’s group is working on designing a kind of living bacterial ‘memory stick’ that could be ingested and used to monitor your internal environment and/or report on your diet. Another idea is to use designer bacteria to serve as an early warning system for patients who are symptom-free but at risk for a flare. Because we currently have no reliable ways to anticipate and prevent IBD-related flares, the Foundation is investing in synthetic biology projects to address this need. As Dr. Silver said, this new field of science offers enormous potential for breakthroughs in medical treatments.  

Dr. Scott Plevy, a long-time scientific advisor to the Crohn’s & Colitis Foundation with decades of experience in gastroenterological research in academia, pharmaceutical, and biotech, echoed this theme in his presentation. “We haven’t had a blockbuster change in IBD treatment for nearly 20 years, since the introduction of biologics. Since then improvements have been incremental, not groundbreaking. That’s why I congratulate the Crohn’s & Colitis Foundation for their vision to fund innovations in this space, because that’s where many transformational ideas will first emerge. And synthetic biology will be the portal for much of it.” 

Bearing in mind that the point of IBD Innovate is to showcase innovative product development, most of the projects presented are still being researched and will not be ready for general use in 2020. But bringing these innovators into the room with pharma, investors, and other stakeholders can accelerate the rate of progress. Just as importantly, symposiums such as this—hosted by the Foundation—can prevent what Dr. Andrés Hurtado-Lorenzo, Senior Director, Translation Research at the Crohn’s & Colitis Foundation, described as “the valley of death,” where promising ideas fail to translate from the laboratory bench to clinical practice because of inadequate resources—and never reach patients. 

Early stage new product highlights from IBD Innovate 2019 

The conference showcased many unique and inspiring products. The following is just a sample of how exciting the future looks for IBD treatments. 

Tissium: Enabling tissue reconstruction 

Tissium, founded in 2013, has received an IBD Ventures investment from the Foundation for their polymer research. Tissium develops polymers (products that act as an adhesive or glue) that can be used safely in the body. Maria Pereira, Chief Innovation Officer at Tissium, told the IBD Innovate audience that sutures (materials used to stitch wounds together) “haven’t radically changed in 5,000 years. We’re still mostly sewing and stitching. It’s time to change that.”

Tissium has a biocompatible and biodegradable polymer product that conforms to and integrates with the tissue surrounding a wound, without harming living tissue. Originally developed at MIT for use in cardiac surgery, this synthetic polymer can be used as a sealant, adhesive, barrier, or even as a vehicle for drug delivery. The folks at Tissium think it can be extremely useful in IBD, including for post-surgical treatment of perianal fistulas (abnormal connections that form between the intestines and another organ or the skin), one of the most pressing challenges faced by many IBD patients.  

• Why it matters: If it works, this polymer could provide an alternative to suturing a fistula that would allow for the fistula to be sealed immediately and healed faster. 
• Where it stands: Tissium is working with surgeons to develop and test its polymer technology for use in fistulas in a preclinical setting. 

Thetis Pharmaceuticals: Using lipids to treat inflammation 

Have you read about the anti-inflammatory properties of omega-3 fatty acids? The folks at Thetis Pharmaceuticals wondered whether lipids (a kind of fat molecule with anti-inflammatory properties) could be used to help manage inflammation in IBD patients. That’s why they developed TP317, a first-of-its-kind lipid-based compound that’s been shown to reduce inflammation and promote healing of the intestinal wall (known as mucosal healing) and tissue repair. Equally importantly, TP317 promotes healing without suppressing the immune system, so it may have a better safety profile than some other drugs currently in use for treatment of IBD.  

• Why this matters: An oral drug that can treat mucosal inflammation and repair tissue without suppressing the immune system might offer an important treatment option for IBD. Thetis hopes that their future clinical trials will support its use early in UC and CD patients, before escalating to immunosuppressive agents. 
• Where it stands: Thetis aims to be in phase 1 clinical trials in 2021. Thetis Pharmaceuticals was a recipient of an IBD Venture investment for this product, and we’re delighted to see their progress. 

Advances in microbiome-targeted therapeutics and diagnostics 

The Crohn’s & Colitis Foundation has been investing in microbiome research since 2008. Our gut microbiome, which is mostly found in the large intestine, is made up of trillions of bacteria, fungi, and other microbes. The microbiome plays a very important role in a person’s health, by controlling digestion and benefiting the immune system. The Foundation was the first organization to dedicate significant ongoing resources towards understanding the impact the microbiome has on human disease. This important effort has been foundational in research work across many diseases and has now gotten us closer to new and innovative products in clinical development for patients with IBD. 

Microbiome-targeted therapy seeks to establish a healthy gut microbiome, and IBD Innovate 2019 showcased several microbiome-centric innovations from various companies. 

Dr. Plevy recalled how early reports on fecal microbiota transplants (FMT) in IBD patients stirred a great deal of excitement. In FMT, fecal bacteria from a healthy person is transplanted into that of someone with an infection or disease with the goal of restoring  bacterial balance in the guts. Dr. Plevy said, “Some of us thought, maybe we’re addressing the root causes in the microbiome for the first time.” But fecal transplants also present some safety risks, since it’s hard to know exactly what microbes are in various batches of fecal transplants. There’s a variety of ways that you could make a product that could provide some of the benefits of FMT with a simpler, more well-defined and targeted, and potentially safer product. That’s where the promise of synthetic biology comes in. Synlogic is designing live therapeutic products containing well-defined, genetically engineered microbes that can be produced and administered in a standardized manner. They are partnering with AbbVie to develop a synthetic biology-inspired therapeutic agent to treat IBD. 

Seres Therapeutics is also using synthetic biology to create what they call EcoBiotic® drugs, which are combinations of ‘designer’ microbes. These compounds are intended to improve the health of the microbiome in order to change the course of disease. Their product, SER-287, is an orally delivered treatment designed to mimic healthy gut bacteria, showed some efficacy in patients with mild-to-moderate UC without any serious drug-related adverse effects. They are now working on a more targeted agent that they hope will be both affordable and effective. 

AlphaBiomics is using microbiome-based data to help direct treatment choices. We know that at least 25% of patients don’t respond to expensive and widely used drugs like biologics. Unfortunately, today we have no way of identifying in advance who will or will not respond. As a result, treatment strategies today are often a matter of trial and error, which can be expensive and time-consuming, as well as very frustrating for patients.

The team at AlphaBiomics, led by co-founder Dr. Angelique van ‘t Wout, have developed a tool that is designed to help identify which patients are not likely to respond to certain biologics. The concept builds on evidence published in 2017 by Foundation-funded academic researchers from Massachusetts General Hospital. 

• Why this matters: A tool that can identify which drugs are less likely to work for specific patients could speed up the time it takes to find the right drug for patients. AlphaBiomics says their cost-benefit analysis indicates that even small improvements in therapy responses can yield significant clinical and economic value. 
• How it works: Patient stool samples are sent to a lab for analysis and results are uploaded to a secure cloud. This information can help physicians and patients make a more informed decision about best treatment options.
• Where it stands: AlphaBiomics has received funding from Johnson & Johnson and the UK’s National Institute for Health Research (the largest funder for health research in the UK). They are now ready to test it in two different patient groups. 

Digital innovations 

Has your life changed as a result of computers and smartphones? The same revolution is happening in medicine. Thanks to a grant from Lilly, IBD Innovate 2019 convened a special panel on digital innovations in IBD care. Part of the panel was devoted to how digital innovation is changing research. But we also heard about a new field of digital therapeutics intended for direct patient use.  

You may already be using digital health apps on your phone or wrist to help your health (such as exercise reminders, step counts, and nutrition calculators). But do they have solid evidence to back up their health claims, and have they been evaluated by the FDA? Digital therapeutics, as opposed to digital health apps, must meet a very high standard of evidence.  

Digital therapeutics are defined as any software that helps to manage, prevent, or treat disease. These products are usually provided to and used by patients. They may be prescribed by physicians and/or used with healthcare providers. Regulatory oversight improves clinical safety and ensures that therapeutic claims are supported by evidence. But FDA clearance is also expensive, time-consuming, and requires rigorous quality control.  

Megan Oser, VP of Clinical Innovation at Mahana Therapeutics, gave us an overview on the state of digital therapeutics for IBD. The good news is that, since 2017, at least eight companies and 10 academic laboratories have published data about the use of digital therapeutics to manage IBD. But as Megan told us, the key question is whether insurance companies (payers) will cover these devices. Insurance companies  want to see cost-savings and effectiveness data. As Megan said, “Evidence gets you in the door, but they want cost-containment data, too.” Much progress has been made in this area and the Foundation will continue to provide updates.  

Patient involvement and engagement is the key 

High quality research and development doesn’t happen without the engagement and dedication of IBD patients. One of the highlights of IBD Innovate 2019 was a presentation from Rolf Benirschke, former NFL placekicker. Rolf was first diagnosed with ulcerative colitis when he was playing football for the San Diego Chargers in 1978. He went on to become the first NFL football player to play with an ostomy pouch! Now he’s a patient advocate and CEO of Legacy Health Strategies as well as a long-time supporter of the Crohn’s & Colitis Foundation.

Rolf Benirschke and patient advocate Tina Aswani Omprakash

Rolf gave an impassioned talk, recounting his experiences and ending with a plea to the attendees to continue to engage patients: “Use us! Let’s not waste our experience. Use us, and let us help design the studies, use our samples, use and share our experiences so we can help others as well as ourselves.”  

The Foundation agrees with Rolf and shares his commitment to ensuring that patients and caregivers are involved in shaping research priorities and clinical trials. Nothing happens without your involvement. One way patients can get involved is to learn more about participating in a clinical trial. Check out the Foundation's Clinical Trial Finder to find information about trials near you. 

The Foundation is excited and proud about the innovative IBD products highlighted at IBD Innovate 2019, and look forward to the next IBD Innovate conference in 2020. The Foundation will keep you updated about progress in the future. 

Sheila Moher, MPH, is a science writer for the Crohn's & Colitis Foundation.