Provention Bio, Inc.

Good morning, my name is Irene and I will be your conference operator for today. At this time, I would like to welcome everyone to the ProVention Bio call. There will be a question and answer session to follow. Please be advised that this call is being recorded at the company's request. I would now like to turn the call over to Mr. Robert Doody, Vice President of Investor Relations for ProVention Bio.

Thank you, Operator, and thank you all for joining us on PreventionBio's fourth quarter and full year 2021 Financial Results Conference Call. Joining today's call from the PreventionBio team is Ashley Palmer, Chief Executive Officer and Co-Founder, Francisco Leon, Chief Scientific Officer and Co-Founder, Jason Hoyt, Chief Commercial Officer, and Siri Chauchet, Chief Financial Officer. Before we begin, Let me remind you that the various remarks we will make today constitute forward-looking statements. These include statements about our future plans and expectations, clinical results, regulatory and other developments, and timelines related to our product candidates, including our plans to continue working with the FDA as they review our BLA resubmission and continuing our efforts for securing a potential FDA approval and commercialization of teplizumab for an at-risk indication, as well as the planned delivery of significant catalysts over the next 24 months, the potential safety, efficacy, and commercial success of teplizumab, and our other product candidates, the potential COVID-19 impact on our clinical studies and business plans, financial projections, including our anticipated use of cash and our cash runway, and and our business plans and prospects and projected timing for the same. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in the risk factors section of our most recent annual report on Form 10-K, which we filed with the SEC this morning, and in other filings that we may make with the SEC in the future. Any forward-looking statements represent our views as of today only. While we may elect to update these forward-looking statements at some point in the future, we specifically disclaim any obligation to do so, even if our views change, except as required by law. Therefore, you should not rely on these forward-looking statements as representing our views as of any date subsequent to today. There is more complete information regarding forward-looking statements, risks, and uncertainties in the reports prevention files of the SEC. These documents are available on prevention's website at www.preventionbio.com under the investor section. We encourage you to review these documents carefully. With that, I will now turn the call over to Ashley.

Thank you, Bob, and thank you to everyone joining us on this morning's call. We are very pleased to be highlighting for you today our progress in the fourth quarter of 2021 and the first two months of 2022. Prevention Bio was founded on our belief that recognizing and intercepting or even preventing autoimmunity before irreversible tissue damage and potential organ failure takes place can fundamentally change the world for millions of patients and their families, thereby enabling those individuals to be less impacted by their disease and more focused on living their lives and realizing their true potential, in turn, making the world a better place for all of us. Autoimmune diseases affect over 23 million patients in the United States alone, and it is our assessment that our industry has today underserved this sizable population and underappreciated the potential associated with innovating to address its considerable unmet needs. Instead, autoimmunity has tended to be viewed by biopharmaceutical companies and the medical system as a whole from the perspective of waiting for symptoms to present to clinicians who are then left with no alternative but to try to manage more advanced stages of disease and their complications, as well as to treat ever-worsening symptoms chronically for a lifetime, often at the cost of significant therapeutic burden to patients and their caregivers, with a corresponding reduction in quality of life for all concerned. We believe there are no better examples of this suboptimal approach than celiac disease and type 1 diabetes. Celiac disease has no approved therapeutic option of any kind. Instead, patients are left to try to manage their disease by attempting to evade contamination from a ubiquitous pathological antigen, gluten. Separately, the first time a patient presents with the symptoms of type 1 diabetes, such as life-threatening diabetic ketoacidosis, they can no longer produce enough insulin to control their blood sugar. This would be akin to a patient with chronic kidney disease finding out that they are sick the very same day they need five hours of dialysis three times a week, every week, just to stay alive. To all of us at Prevention Bio, this is simply unacceptable. These patients and their families deserve better, and it is our strategic intent to make a difference. In fact, a key consideration when we founded Prevention Bio was our belief that we could pioneer conceptually similar immunomodulatory approaches for autoimmune disease. to those we have all witnessed offer such profound effects at the immuno-oncology end of the spectrum of immune-mediated diseases. Using our conceptual platform, our expertise in immunology, and our experience and track record in creative business development and corporate partnering, we have acquired and we are advancing a pipeline of product candidates that are uniquely focused on early autoimmune disease intervention. We have two lead assets, teplizumab and PRV3279, that we believe have potential for applicability across a broad spectrum of autoimmune disorders, both as monotherapies and in combination with other emerging approaches, such as cell therapy, gene therapy, and antigen-specific tolerization. And we are now even more committed and well-positioned to unlock this potential, whether it be through independent development programs or strategic partnerships. Throughout 2021, we made significant progress advancing our two lead therapeutic candidates, along with our other clinical stage pipeline assets, and we were especially delighted to announce earlier this week the resubmission of our teplizumab BLA to delay the onset of clinical type 1 diabetes in at-risk individuals. This resubmission follows very productive and collaborative interactions with the FDA throughout last year, including an advisory committee vote in May in favor of teplizumab's approval in at-risk type 1 diabetes, and a subsequent complete response letter in July that cited no clinical deficiencies related to the efficacy and safety data packages submitted to the original BLA. A BLA that included data from the pivotal NIH-sponsored TN10 clinical trial conducted by TrialNet in which a single 14-day course of teplizumab significantly delayed the onset of clinical stage insulin-dependent type 1 diabetes in at-risk patients by a median of approximately three years. Last week's BLA resubmission followed a type B meeting held three weeks earlier, at which the FDA proposed and the company agreed to use PK modeling to adjust the 14-day dosing regimen for our planned commercial product to match the exposure of material manufactured over a decade ago that was used in prior clinical trials, including the pivotal TN10 study. Under FDA guidelines, the agency now has 30 days to verify that our resubmission is complete and acceptable. and assign a goal date for review completion. It is our understanding this goal date should be set within six months of last week's resubmission date, at which point, if approved, teflizumab has the prospect of becoming the first-ever disease-modifying therapy in type 1 diabetes. Following a potential approval and U.S. launch of teplizumab for at-risk T1B by the end of this year, our future clinical development plans for this indication aim to broaden initial labeling and market potential by exploring younger age groups below eight years, as well as the impact of repeat dosing to potentially extend a single course of therapy's three-year median delay in progression. We next look forward to the top line results of our phase three PROTECT trial of teplizumab in newly diagnosed T1D patients. We completed our target enrollment in quarter three of last year. And out of an abundance of caution, given potential COVID-related challenges for clinical trial follow-up, we proceeded to over-enroll this study by about 10%. We remain on track to deliver top-line results following completion of ProTech's 18-month follow-up period in the second half of next year. Our longer-term plans include the evaluation of subcutaneous formulation, as well as the exploration of potential combinations of teflizumab with other rapidly advancing approaches, such as pancreatic islet and beta cell transplantation. targeting the growing market of 1.8 million established insulin-dependent type 1 diabetics in the United States alone. It is worth noting here that in prior published studies, the addition of teplizumab to islet transplantation induction regimens has been successful in extending the durability of favorable post-transplantation results with 75% of transplanted patients remaining free from the burden of insulin dependency for more than five years. Outside of T1D, we plan to explore the potential use of teplizumab across other autoimmune-related disorders, such as celiac and Crohn's disease, early rheumatoid arthritis, and autoimmune hepatitis. Turning now to the rest of our pipeline, at the end of last year, we were pleased to announce positive interim results for the first in-human study of our PRV-101 vaccine candidate targeting Coxsackievirus B, which is known to trigger both T1D and celiac disease. This phase one trial demonstrated in a dose-dependent fashion PRV101's ability to induce high titers of viral neutralizing antibodies against all of the Coxsackievirus B serotypes targeted by this polyvalent vaccine. We are expecting the final results from this first in human study in the first half of this year. PRV015, which has now been assigned the name is currently being studied in a Phase IIb trial with the goal of becoming the first ever therapy to intercept gluten-free diet non-responsive ciliacizine. Our target date for top-line results from this trial is by the end of next year. Regarding PRV3279, our non-depleting bispecific scaffold targeting B-cell mediated disease, we were very pleased to announce at the beginning of this year that we have successfully initiated our PREVAIL-2 phase 2A trial evaluating 3279 in systemic lupus arithmetosis. And we expect this trial to be completed and read out top line results in the first half of 2024. It is our current intention to hold a series of R&D-related investor events throughout this year to dive more deeply into our pipeline development plan, as well as our plans for Tepelizumab's label expansion and lifecycle management. We are also planning to conduct an investor educational event in the second quarter focused on providing you with more in-depth insights and details regarding teplizumab's potential at-risk T1D launch and commercialization. However, let me now ask Jason to provide you with a brief update to whet your appetite. Jason.