Tiffany Brown, Avobis Bio - Implantable Cell Therapy for Crohn's Fistulas | LSI USA '25

Tiffany Brown  0:05  
Good afternoon, everyone. I'm Tiffany Brown with Avobis bio, where we're developing implantable cell therapies to heal tissues and transform lives. Avobis is a clinical stage company focused on debilitating conditions caused by impaired healing. We were founded a little over five years ago as a joint venture between the Mayo Clinic and gore, and are coming up on a really exciting milestone this summer, with the data read out from our phase two trial in Crohn's perianal fistula, our technology overcomes impaired healing through the synergistic combination of cells and materials, and we're led by an experienced team with a global clinical and business track record. We're targeting a $70 million raise for our upcoming phase three pivotal trial. Avobis is passionate about impaired healing because it affects millions of patients and costs billions of dollars. Our initial focus is Crohn's perianal fistula, where a tunneling wound forms from within the anus out to the skin of patients who have Crohn's disease. And while you and I may take healing for granted, it is an incredibly, exquisitely orchestrated cascade, and it can be easily disrupted if a key step is impaired. And in patients who have Crohn's disease, their physiology is altered, and so healing doesn't progress in these perianal fistulas. And life with a perianal fistula is very difficult. Patients experience fecal drainage through that tunneling wound, pain and the constant risk of infection or sepsis. Typically, these fistulas last, on average, for six years and have patients undergo six attempted repairs, and in 25% of the patients the entire rectum is removed in the end. So implantable cell therapy is a novel approach to overcoming impaired healing. We take mesenchymal stem cells and a plug shaped biomaterial and then through a proprietary process, create a cell therapy. This cell therapy is then implanted within the fistula track, where the cells generate tissue and produce healing signals. We have a local therapeutic treatment, and our technology is covered by a number of global patent families and pending applications. Crohn's perianal fistula really represents a significant human and financial opportunity. There's 60,000 patients in the US today, costing nearly $4 billion and the prevalence is only rising, because this complication occurs in 10% of Crohn's patients with a typical average onset age of just 30 years old. In addition to the US market, there's a comparable, slightly larger population in need in Europe as well. So unfortunately, standard treatments infrequently heal. Patients experience ongoing interventions, ongoing hospitalizations and a deteriorating quality of life. All of this adds up to $65,000 a year, just in direct health care costs. So clearly, we need to do better as a healthcare field. And Avobis is striving for durable healing, durable healing using implantable cell therapy. And given our clinical track record to date, we are actually poised for first mover advantage in this market. So I mentioned standard of care. This is typically systemic drugs for inflammation as well as a drain in the fistula to manage the infection risk, but this fails to heal patients in two thirds of the cases and attempts at surgical repair also rarely heal and can cause worsening fecal incontinence, which is really debilitating if you have Crohn's disease. So there have been attempts made in the past at curing this condition. Unfortunately, a recent injectable cell therapy failed their phase three primary endpoint, and the next generation biologic drugs in development are primarily focused on the luminal Crohn's disease itself and not healing of the fistula. So given our promising technology and a clear unmet need, we executed a phase one clinical trial that had compelling results, 20 patients with refractory Crohn's perianal fistulas were treated at the Mayo Clinic, and 76% had healing at one year, we saw healing signals, both clinically as well as radiologically, and excited about our quest for durability. Those patients who had healing at six months had not relapsed again at a year, the product showed a strong safety profile and a reduction in subsequent interventions. So these patients who had been refractory had a 10x reduction in their annualized exams under anesthesia after treatment compared to before, which shows you the impact on a patient's life as well as on health care costs. So with the Phase One results, we move towards a phase two, and this is the data that we're expecting this summer in our phase two trial, 48 subjects were treated across 14 us clinical sites, so patients with refractory Crohn's perianal fistulas were randomized to either the investigational therapy that we call. A VB, 114, or standard of care, given their refractory and we had a strong safety profile. This trial actually also has a crossover design, so patients who are randomized to standard of care initially, if they fail to heal by the primary endpoint, can then elect to receive the investigational product, which gives us the ability to compare the treatment across the groups in part one, and on a per subject basis, before and after in the standard of care arm. So our focus here is combined remission, both clinical and radiologic, and we're capturing a number of secondary endpoints around quality of life, safety and healthcare burden. So we're anticipating this data this summer, following the data, we're planning for a post phase two funding round where we'll be doing longer term follow up of the phase two patients, executing work in the clinical MC and C space, leading up to a phase three ind, and then execution of a phase three trial. But we're excited to build on the milestones that we've achieved with our initial funding from Mayo and gore, including that phase two trial and our recently granted Fast Track designation. Once we achieve that phase three data, we expect there will be opportunities for either additional funding or an exit. So turning to manufacturing. The process involves taking a sample of adipose from the patient, isolating and expanding their cells, and then combining the cells with the scaffold to produce that drug product, a VB, 114, for Phase Three and beyond. We plan on using a commercial contract manufacturer. We already established a partnership with one who's experienced in cell therapy and have had a successful tech transfer of the current process. So we're really poised for future success here, and are continuing to work towards improve scalability, cost and robust manufacturing processes. Looking ahead to commercialization, we're focused on patient and physician access, and most Crohn's perianal fistula interventions occur at Inflammatory Bowel Disease Centers of Excellence. This is where gastroenterologists and colorectal surgeons work together in partnership and are experienced in Crohn's perianal fistula care. Our implantable cell therapy is an outpatient surgical procedure, and it utilize utilizes existing fistula management techniques from a customer support perspective, we're anticipating providing both logistical support as well as therapeutic administration support, and think there may be options, either to develop an Avobis team to do that, or partner with an existing sales and distribution channel. From a financial projection perspective, I mentioned there's 60,000 patients in the US today with Crohn's perianal fistula, the treatable market assumptions are based on those who have stability in their Crohn's disease as well as comparable fistula anatomy. And then the Avobis market takes into account some limitations anticipated from patient access due to coverage limitations and the potential that a biologic drug might get a fistula indication. All told, we expect a five year revenue of 350 million, growing to a 10 year revenue of 550 million. There's also the upside if we address the European market. So our post phase two raise is anticipated to be on the order of 70 million. It'll be dialed in once we have those phase two results, with the emphasis on clinical and CMC development as well as phase three trial execution. Again, we're aiming to achieve a value inflection point with that phase three data, where we could have another round of investment or an exit opportunity. So our track record of success and our vision for the future really built on the passions of our entire team, but we're excited to have an experienced leadership team at the helm who has a track record of billions in Global Product Revenue, securing regulatory and reimbursement, as well as deep scientific knowledge. So avois is a clinical stage implantable cell therapy company, who are approaching our lead asset, pivotal trial, we're poised for first mover advantage and a high potential indication with technology that overcomes impaired healing and have an experienced team passionate about changing lives. So we hope you'll join us on the journey. Thank you very much.