Provention Bio, Inc.

Good morning. My name is Kaylee, and I will be your conference operator today. At this time, I would like to welcome everyone to Prevention Bio Call. This 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 hand the conference 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 first quarter 2021 financial results conference call. Joining today's call from the PreventionBio team is Ashley Palmer, Chief Executive Officer and Co-Founder, Andrew Drexler, Chief Financial Officer, and the other members of the PreventionBio leadership team. 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 expectations, clinical results, regulatory, and other developments and timelines related to our product candidates, including for the teplizumab DLA, such as our plans to work with the FDA to resolve their PK comparability concerns and expectations for the upcoming advisory committee meeting. 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 planned pre-commercial activities across the company in preparation for the potential approval of Proclizumab 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 as 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 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 good morning to all of you joining us today. We greatly appreciate your support and attention as we continue our mission to develop and commercialize pioneering therapeutic options to intercept or prevent serious life-threatening and life-impacting autoimmune disease. In founding Prevention Bio just four years ago, our intention was to disrupt how the biopharma industry and healthcare systems around the world typically wait for patients with autoimmune disease to present to clinicians with symptoms. Often by this time, irreversible tissue damage has already taken place. Precious cells and organs have been damaged or destroyed. It's simply too late. It's not good enough. And we believe that patients and their families deserve better in this modern world of medicine. As with all pioneering endeavors, we recognize that our path to accomplishing our mission is not without its obstacles, hurdles, and resistance. As such, we've built our company from the ground up with like-minded professionals and industry experts capable of addressing those challenges head on. We have established a resilient and tenacious culture dedicated to improving the lives of patients and their loved ones. Ultimately, we believe we will prevail. I open our call this morning with these comments to put into context the current status of the ongoing review of our BLA for teplizumab for the delay of progression to stage 3 clinical type 1 diabetes in at-risk individuals. When we acquired teplizumab just three years ago, we took on the challenge of reviving a potential therapeutic option that had been long deprioritized by others. Our original strategic intent was to do so by focusing on the preservation of beta cells in newly diagnosed patients by way of our phase three PROTECT study. At the time of acquiring teplizumab in 2018, The TN10 trial conducted by the NIH-sponsored academic consortium TrialNet had already been fully enrolled and was waiting for the progression of a threshold number of patients to clinical stage disease before opening up the blind. The drug product administered in the TN10 study had been manufactured using drug substance produced by Eli Lilly more than seven years beforehand, now over a decade ago, and involving plant and certain processes that are no longer available. Importantly, when prevention acquired teplizumab, we also acquired the original cell lines used to manufacture the original Lilly drug substance. along with the original batch records, specifications, and other know-how necessary to transfer this process to our manufacturing partner and into the modern biotechnology era. We believe we have produced a quality and comparable teplizumab drug substance by way of an up-to-date, well-controlled, reproducible, and validated commercial scale manufacturing process. Additionally, throughout 2020, all the physicochemical testing and analyses required for our manufacturing partner, AGC Biologics, to release that drug substance for filling met all the requisite specifications and parameters, thereby enabling the completion and submission of our BLA's CMC module. Before introducing new drug product containing AGC drug substance into our phase three PROTECT trial in newly diagnosed T1D patients, we conducted a single low-dose PK-PD bridging study in healthy volunteers. And we observed a PK area under the curve, or AUC, level below the target comparability range, indicating that in this particular study, the new drug product might be clearing from the bloodstream faster than drug product manufactured from the old Lilly drug substance. Importantly, in this study, we believe that other relevant PKPD parameters, such as the peak concentration, or Cmax, the clinically relevant PD marker of transient lymphocyte drop, the immunogenicity, and the safety profile all fell within acceptable ranges of comparability. As we stated previously, based upon very extensive PK PD modeling and taking into account the totality of the information available to us at this time, It is our firm belief that the observed difference in PKAUC is not clinically relevant and should not impact clinical efficacy or safety since the predicted exposure for the intended commercial product remains above the requisite threshold for beta cell protection. Based on its review to date, the FDA is not comfortable with our conclusions. the agency has informed us that it does not yet consider the two drug products to be sufficiently comparable and cannot be certain that the PKAUC shortfall observed in our single low-dose PKPD bridging study in healthy volunteers might not translate into clinical relevance. Nevertheless, under our breakthrough therapy designation, the FDA continues to be very engaged, very helpful, and very cooperative, and has agreed to work closely with us to figure out our next steps and a path forward to a solution, which we anticipate will likely require our provision of additional data to support PK-PD comparability. One potential pathway may be to access PKPD data from patients in our ongoing PROTECT study, which has begun enrolling patients to receive either drug products using the original Lilly drug substance or drug product intended for commercialization using AGC drug substance. We have also stated that we expect the need to provide the agency with additional data will result in a delay of the expected timelines within which teplizumab has the potential to be approved and made available to patients. We are continuing to discuss next steps with the FDA and will keep you apprised and updated as to our progress. These comparability discussions are occurring in parallel with preparations for the upcoming advisory committee meeting in three weeks' time on May 27th. And the FDA notified us of its intention to mention the comparability considerations in its briefing materials, along with an affirmative statement that the agency is actively working with us to find a solution. We believe the FDA's intent is to focus the advisory committee meeting on an examination of type 1 diabetes unmet need and the safety and efficacy of teplizumab from the TN10 trial supported by data from other historic studies in newly diagnosed patients. Our understanding is that since the FDA's comparability considerations do not bear on the benefit-risk assessment of the TN10 study clinical data package, no comparability-related questions or discussion topics are planned for the meeting. We are also fully aligned with the FDA's recommendation to remove the term prevention from the wording of teplizumab's initial indication. and instead focus exclusively on delaying the progression of disease. We believe this will help to reinforce the fact that while pre-symptomatic, T1D patients with two autoantibodies and dysglycemia already have the disease and may benefit from therapeutic options targeting the preservation of functional beta cells. Our team has been working diligently for quite some time now preparing for this advisory committee meeting as we realize the pioneering importance of having an opportunity to present the first disease-modifying therapy for type 1 diabetes to be reviewed by this committee. We look forward to standing alongside scientific key opinion leaders, endocrinologists, immunologists, and other treating physicians, diabetes nurse educators, and other members of the T1D clinical community, as well as patient advocacy organizations, patients and their families, who for so very long have hoped and waited for a disruptive innovation that has the potential to delay clinical stage disease and the burden and risks of insulin dependence. such a therapeutic advance would be a critically important breakthrough, especially for younger patients, otherwise facing a lifetime of continuous blood sugar monitoring and insulin therapy. We understand that much of our investors' focus and attention this year has concentrated on the nearer-term regulatory pathway to the potential commercialization of teplizumab for the at-risk patient population. However, that is certainly not all that Prevention Bio is about, and I would now like to spend a few moments providing you with an update on the progress and momentum that is taking place throughout our organization with our impressive pipeline of other immunology therapeutic programs. Importantly, our previous guidance with respect to our programs remains on track. Beginning with our PROTECT phase three trial of teplizumab in newly diagnosed patients, as you know, randomization into this trial was paused for some time last year due to COVID-19. However, upon resuming randomization, enrollment has steadily increased as various sites and countries have come back online. And we are now on track to complete enrollment of this trial in the second half of this year. Importantly, this will position us to have top-line results available in mid-2023. Now turning to PRV3279. This is our humanized bispecific scaffold targeting both CD32B and CD79B receptors designed to inhibit B-cell function and suppress autoantibody production without causing B-cell or platelet depletion. In addition to the strategic collaboration agreement we announced in the first quarter of this year with Huadong Medicine for development and commercialization in Greater China, we continue to progress preparations towards the initiation of our prevailed Phase IIa lupus trial in the second half of this year. Also, with respect to PRV3279, we reported preclinical data and results from a model of Pompe disease, showing improved efficiency of transfection of a gene therapy product by reducing its immunogenicity with a PRV3279 surrogate. PRV015, our partnered fully human anti-IL-15 monoclonal antibody program, continues enrollment in the Phase IIb proactive trial, and we expect top-line results in 2022. As a reminder, after this Phase IIb trial is completed, Amgen has an option to take the asset back for Phase III development and commercialization. At which point, if exercised, we would receive a payment of $150 million, along with subsequent milestone and royalty payments. Lastly, we are excited to announce that we have completed enrollment in the first in human phase one healthy volunteer trial for PRV101, our vaccine against Coxsackievirus B. We expect to have a top line results for this trial in quarter four of this year. As discussed previously, it is believed that Coxsackievirus B is one of the main triggers of the immune cascade that results in pancreatic beta cell destruction in type 1 diabetes and gluten exposure driven gastrointestinal autoimmunity in celiac disease. we continue to make significant progress advancing all of our autoimmune disease therapeutic programs, whilst at the same time concentrating on the teplizumab regulatory pathway and potential commercialization preparation. I'm now going to turn the call over to Andy to provide you with details on our financial results for the first quarter before returning for closing remarks and questions. Andy.