Ilya Pharma receives BIO-X® grant to complete preclinical work for novel IBD chemokine-based gene therapy candidates

Ilya Pharma a clinical stage biopharma company today announced that it has received a BIO-X® grant that will be matched with company funding to complete preclinical studies for two novel chemokine-based gene therapy candidates, ILP100 and ILP101, for Inflammatory Bowel Disease (IBD). This will represent a major breakthrough since ILP100 and ILP101 are the first drugs based on genetically engineered lactobacilli. The bacteria act as on-site mini-bioreactors to express chemokines in therapeutic quantities. ILP100 and ILP101 represent both a novel drug delivery technology and novel investigational new products (IMP) and are classified as advanced therapy medicinal products by the EMA and as low risk candidates under the gene therapy umbrella by the FDA. Ilya Pharma is aiming for progression to clinic by the end of 2022.

CEO and Co-Founder Evelina Vågesjö comments:

“The incidence of inflammatory bowel disease is currently raising in industrialized countries but <50% patients respond to treatment, including biologics. In addition, the current treatments are associated with several side effects due to systemic exposure as well as high societal- and treatment costs. Hence, we have designed these two new drug candidates – ILP100 and ILP101 – with novel mechanisms of action targeting multiple signaling pathways to dampen intestinal inflammation.”

Until now, chemokines have been underexplored as therapeutic agents for gastrointestinal indications in particular due to their short half-life. ILP100 and ILP101 are genetically modified lactic acid bacteria that express the human chemokines CXCL12 and CXCL17, both of which bind to the CXCR4 receptor. Preliminary data, using the experimental DSS model of colitis, have demonstrated that peroral administration of ILP100 to mice with overt colitis interrupts disease progression and improves symptoms, assessed as body weight loss, disease activity index and fibrosis in the colon. Detailed analysis of the tissues showed that treatment with ILP100 alters infiltrating immune cells in the colonic lamina propria, while no changes were detected in the small intestine or mesenteric lymph nodes, suggesting local effects at the site of inflammation. Moreover, ILP100 treatment increases the number of immune cells that produce TGF-ß and IL-10, as well as other tolerogenic immune cell populations locally. Initial feasibility testing with different doses and formulations as well as safety studies including exposure have been performed. The next steps, which the new grant will support are confirmatory studies, verification in human derived tissues, GLP-studies of pharmacokinetics, pharmacodynamics and toxicity, as well as initial adaptations to the final stages of the already GMP-compliant bioprocess, making IMPs for oral administration.

“Even before founding Ilya Pharma we actually we had good data in animal models of inflammatory bowel disease. However, our strategy was to develop one project to clinical proof of concept as fast and as smartly as possible. We chose a topical approach to skin wounds and it has turned out well. We have had quite some traction on this project for years and it is extremely rewarding to now be given the opportunity to also accelerate development in the gastrointestinal indications. The difference now is that we have built a fantastic team and know what it takes to take the ILP-drug candidates to clinical proof of concept,” says Vågesjö.

About IBD – Inflammatory Bowel Disease (IBD) has evolved into a global disease with rising prevalence. In the United States, more than 3 million adults are estimated to have been diagnosed with IBD, while 2.5-3 million people are affected in Europe. IBD encompass Crohn’s disease and Ulcerative colitis, which are chronic, relapsing and incurable lifelong disorders. Available drugs, the core of IBD treatments, are designed to suppress the immune system, and biologics are today commonly used drugs. However, these treatments have limited success, as only 46% and 25% of the CD and UC patients, respectively, are responding to therapy, in addition to serious side effects and high costs ($22,987 per patient per year). Thus, there is an emerging need to find new therapies targeting additional pathways that are also cost efficient.

About the BIO-X® Accelerate program – BIO-X Accelerate is a further development of BIO-X in close collaboration with Tillväxtverket and Region Uppsala, with Uppsala Innovation Centre as partner, with the goal to accelerate the development of innovative life-science companies in the region. The program utilises an industrial approach to support early-stage, life-science related projects that could fulfil well-defined needs in healthcare and society. Projects receive funding, continued support and access to experts within the BIO-X network. The first critical step towards potential commercialization – ‘proof of concept’ studies should already be performed when entering the program. After BIO-X the project should be ready for investment by for example industry or venture capital companies, for a future new product, method or therapy with a significantly new value for the patients, healthcare or society. BIO-X is a trademark of STUNS Life