Provention Bio, Inc.

Good morning. My name is Sarah, 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 PreventionBio's third quarter 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 Andrew Drexler, 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 for teplizumab, our plans to work with the FDA to address deficiencies identified in its CRL, including their PK comparability and product quality considerations, 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, including with respect to any potential BLA resubmission for teplizumab 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 factor 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 report's prevention files with 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'll now turn the call over to Ashley.

Thank you, Bob, and good morning to everyone joining us on the call today. We've made considerable progress across the company over the past few months. And this morning, I will begin by providing an update on the planned regulatory pathway and next steps relating to our efforts to potentially secure approval for teplizumab for the delay of type 1 diabetes in at-risk individuals. I'll also provide an update on our PROTECT phase three trial of teplizumab for newly diagnosed T1D patients. Jason will then be sharing with you some of the activities and progress our commercialization team has been making in preparation for the potential approval and launch of teplizumab. Francisco will then discuss the exciting first in human interim data that we announced last week pertaining to our polyvalent Coxsackievirus B vaccine candidate, as well as provide status updates on our other pipeline candidates targeting serious autoimmune diseases. Lastly, Andy will summarize our financial results for the quarter before we open up the call for your questions. In September, we issued a press release updating you on the status of our interactions with the FDA in response to considerations noted in the complete response letter we received this past July. Specifically, we took part in a Type A FDA meeting in August to discuss those considerations related to product quality and manufacturing. As noted in the press release, We believe these have either been addressed by amendments filed prior to the issuance of the CRL, which the FDA had not yet reviewed, or are items our team is currently addressing and expects to have completed by the time the BLA is potentially resubmitted. The CRL also noted findings from a then recent inspection at a contract fill finish facility we can confirm this inspection has been closed out by the FDA as expected. Given this progress, we believe the remaining gating factor for approvability of teplizumab is our ability to either demonstrate through PK data derived from therapeutically dosed patients that our to be commercialized drug product is comparable with material used in prior clinical trials manufactured from drug substance produced by Eli Lilly 11 years ago. Or to provide the FDA with sufficient data to confirm that any difference in PK area under the curve seen in a single fractional low dose PK study in healthy volunteers does not translate into efficacy concerns in patients. It is our intention to do both of these things. During the first quarter of this year, we initiated a PK-PD sub-study within the ProTek trial to collect samples from actual patients receiving either the Eli Lilly source material or the 2B commercialized product. We have since completed the collection of samples from approximately 160 patients and currently remain blinded to the PK-AUC data. At the beginning of last month, following the FDA's recommendation, we requested a formal Type A meeting and submitted briefing documents so the FDA can formally agree on the population PK model's design prior to the unblinding of data collected from the PROTECT substudy. The FDA has scheduled this meeting for the second half of this month. Assuming we receive the FDA's agreement at this type A meeting, we will then proceed to populate the PK model, analyze the results, and work with the FDA to determine the regulatory path forward. In our September press release, we also provided preliminary unblinded pharmacodynamic data collected from the PROTECT substudy. Our view is that the relevant PD markers, such as lymphocyte counts, CD3 receptor occupancy, and T cell activation, indicate both drug products act on targeted T cells in a consistent and remarkably similar manner. While we cannot yet claim these markers in isolation are determinative of comparability, we do believe they are robustly supportive and provide strong rationale to present to the FDA why PKAUC comparability differences for this therapeutic antibody may not be clinically relevant. Switching now to teplizumab's potential use in newly diagnosed T1D patients, Our goal for the PROTECT phase three study was to enroll 300 patients within six weeks of their initial diagnosis. Our guidance for some time now has been that completion of target enrollment would occur in quarter three. And I'm delighted to report that we indeed accomplished this objective at the end of August. Out of an abundance of caution, given the challenges the COVID pandemic has presented to clinical trials across our industry, we continued enrolling patients to exceed this target by about 10% to ensure that we will have sufficient, evaluable patients. I do want to acknowledge and applaud our Protect Study team, the collaborators, the investigators and clinical trial sites And most importantly, we want to thank the patients and their families for their efforts and participation. As per prior guidance, we remain on track to deliver top line results from the trial in mid-2023. Before I turn the call over to Jason to discuss our commercialization preparations and progress, I'd like to remind you that our planning approach to date has been carefully gated and aligned with our regulatory risks and milestones. As we gain more clarity and confidence in potentially moving forward, we will begin to increase spending on pre-launch activities. Nevertheless, despite this prudent gating, Jason and his team have been able to make great progress on a number of preparatory fronts. And we would like to update you on this now. Jason.