Provention Bio, Inc.

Good morning. My name is Kate, and I will be your conference operator today. At this time, I would like to welcome everyone to the prevention 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 Prevention Bio.

Thank you, operator. And thank you all for joining us on Prevention Bio's first quarter 2022 financial results conference call. Joining today's call to review our 2022 progress to date and share some insights into what to expect going forward will be Ashley Palmer, Chief Executive Officer and Co-Founder. Siri Shoshay, our Chief Financial Officer, will also be joining us to provide an update on our Q1 financial results and financing options, along with additional members of our leadership team available to address your questions. 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 efforts towards securing a potential FDA approval for 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 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 quarterly report on Form 10-Q, 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 of any date subsequent to today. There is more complete information regarding forward-looking statements, risks, and uncertainties in the Reports Prevention Files with the SEC. These documents are available on Prevention's website at www.preventionbio.com under the Investors 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 excited to speak with you today, not only about the progress made throughout the first quarter, but where that progress and the catalytic potential of our pre-commercial asset, teplizumab, can now take prevention and the patients, investors, and other stakeholders we serve. In January, we participated in a successful Type B meeting with the FDA to review our pharmacokinetic data and modeling, as well as the pharmacodynamic results from a PK-PD substudy conducted under our PROTECT phase 3 trial in newly diagnosed type 1 diabetic patients. The FDA did not request further clinical data, but instead proposed the use of additional modeling to potentially address the PK comparability consideration set out in the agency's complete response letter issued last July. We immediately accepted the FDA's recommendation, deploying a population PK model developed in close collaboration with the agency to demonstrate how modest adjustments to teplizumab dosing used in the at-risk T1D 14-day regimen could match the exposure of prevention's planned commercial product with that of clinical trial material manufactured 11 years ago. Three weeks following the January Type B meeting, our team was able to complete the resubmission of a biologics license application, or BLA, for the delay of clinical Type 1 diabetes in at-risk individuals. And 30 days later, in mid-March, we were delighted to report that the FDA had accepted our resubmission for review assigning a user fee goal date of august 17th during the first quarter we also reported significant progress across our other pipeline programs focused on the prevention or interception of serious autoimmune diseases In January, we announced the initiation of the PREVAIL-2 trial for PRV3279, our investigational humanized bispecific therapeutic scaffold targeting the CD32B and CD79B surface proteins on B cells. PRV3279 has the potential to intercept the underlying pathophysiology of B-cell-mediated autoimmune diseases, such as systemic lupus erythematosus, or SLE. The PREVAIL-2 trial, which we are conducting in conjunction with our PRV3279 Greater China licensee and partner, Guadong Medicine, is a Phase IIa proof-of-concept study enrolling patients with moderate to severe SLE in both the United States and Hong Kong. The trial is targeting enrollment of approximately 100 patients who will receive monthly infusions of active or placebo drug product over a 20-week period, with a primary efficacy readout at 24 weeks. We expect top-line results from this trial in 2024. Additionally, this past quarter, we continued to screen and enroll patients with diet non-responsive celiac disease into the Phase IIb proactive trial of our Amgen-partnered anti-IL-15 human monoclonal antibody, or disecumab, or PRV, Also, as per previous guidance, we reported results from the final analysis of our phase one first in human PROVENT trial for PRV101, a polyvalent Toxsackie virus B vaccine candidate targeting the key viral strains associated with type 1 diabetes. The final results showed this trial's primary endpoint was met and confirmed the tolerability observed in our previously reported interim analysis. In particular, there were no treatment emergent serious adverse events, adverse events of special interest, or adverse events leading to study drug discontinuation or withdrawal. The results also show durability of viral neutralizing antibody responses with 100% of subjects in the high dose arm maintaining high titers for the majority of vaccine targeted serotypes. We consider these results to be extremely encouraging, enabling us to explore prospective partnerships to advance PRV101 towards possibly becoming the first vaccine to prevent Coxsackievirus B infection and its acute morbidity and mortality, as well as potentially decrease the global incidence of associated type 1 diabetes and celiac disease. Earlier, I made reference to the catalytic potential of teplizumab. Since the successful filing of our US breakthrough therapy designation and our prime and innovation passport designations in Europe and the UK, we have not only witnessed considerable growth in teplizumab's potential to bring about fundamental change for the patients and stakeholders we serve, we've actually begun to realize and experience that change. Merely the prospect of an FDA approval for the first disease-modifying therapy to delay clinical type 1 diabetes in at-risk individuals has generated heightened visibility and awareness of the importance and benefits of screening for two autoantibodies, not just for the relatives of existing insulin-dependent patients, but also with respect to general population screening. Medical societies, key opinion leaders, academic consortia, patient advocacy groups and corporations are now engaged in meaningful, collaborative, multi-stakeholder dialogue. This dialogue has shifted from how screening can reduce the incidence of diabetic ketoacidosis to how the revision of screening guidelines can help to identify at-risk T1D patients in anticipation of the potential approval of immunomodulatory therapies like teplizumab. JDRF's T1Detect program and other screening initiatives now bring disease and screening awareness, education, patient support, and affordable T1D autoantibody testing to the kitchen table of at-risk T1D patients and their families. And we are proud to be a founding sponsor of JDRF's efforts. If teplizumab is approved by the FDA in August, now only 15 weeks away, prevention will begin realizing its founding mission to change the world by providing a disease-modifying therapeutic option to a patient and clinical community that has been deeply underserved for decades and highly frustrated and burdened for generations. We believe this fundamental shift in paradigm could have a profound impact on patients. many of whom present with T1D for the first time in life-threatening metabolic crisis. And for those diagnosed under the age of 10, a reduced life expectancy of 16 years. It also represents a major catalyst for change at prevention. On the development front, we believe it will validate our founding conceptual platform and pipeline and our belief that life-threatening and debilitating autoimmune diseases, such as type 1 diabetes and celiac, can potentially have their progression intercepted and delayed. If teflizumab is approved, it will also lay the foundation for what we believe will become a meaningful therapeutic franchise, beginning with at-risk T1D label expansion focused on children under eight years of age, as well as redosing teplizumab to potentially extend the two to three-year median delay in clinical T1D onset that was demonstrated in the TN10 study and its follow-up using a single 14-day course of therapy. In the second half of next year, we expect top-line results from our ongoing teplizumab phase three trial in newly diagnosed patients. And in parallel, we anticipate the continuation or startup of collaborative development programs using teplizumab in combination with tolerogenic platforms like that of our existing partner, ActoBio, as well as pancreatic islet and beta cell transplantation programs targeting the growing market of 1.8 million end-stage 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 regimen 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, especially celiac disease, which to date has no therapeutic option for patients other than attempting to avoid contamination from antigenic gluten by way of dietary control. Preparing for teplizumab's potential approval is also catalyzing significant organizational change within prevention. As we transition to becoming a progressive, highly focused, omnichannel commercial vehicle leveraging capabilities and resources across the ecosystem to create, penetrate, and expand nascent markets and cost effectively commercialize innovative immunomodulatory therapeutics to highly targeted audiences. Rather than go into greater detail regarding our teplizumab commercialization plans on today's call, I'm delighted to announce that later this month, we will be conducting our promised commercial launch investor event. On Thursday, May 19th, prevention's chief commercial officer, Jason Hoyt, and his commercial leadership team will be sharing with you our deep understanding of the critical aspects of the at-risk T1D target market and patient journey. Specific agenda items Jason and his team intend to cover at this event include the evolution and future of patient screening, our extensive market research, which now includes in excess of 1,300 respondents across multiple target audiences, our go-to market strategy, field force deployment, payer research, our supply chain and distribution model, patient services and support, as well as our well-qualified and well-prepared medical affairs infrastructure and team. The potential approval of Teplizimab just over one quarter from now would also be an important financial catalyst for prevention. Before handing over today's call to Thierry to discuss our quarter one results and future financing strategy and optionality, I want to acknowledge his leadership, the hard work and expertise of his finance function, and the professionalism and dedication of the rest of the prevention team for the disciplined manner in which they have managed our business operations throughout the first quarter of 2022. As Thierry will explain, our cash-based operating expenses were well within guidance and we closed the quarter with a cash balance in excess of $113 million, sufficient to take us beyond potential teflizumab launch and into next year, despite the worldwide macroeconomic challenges we are all witnessing. Thierry, over to you.