Image source: The Motley Fool.
Provention Bio, Inc (PRVB)
Q2Â 2020 Earnings Call
Aug 7, 2020, 9:00 p.m. ET
Contents:
- Prepared Remarks
- Questions and Answers
- Call Participants
Prepared Remarks:
Operator
Good day, ladies and gentlemen and welcome to your Provention Bio's Second Quarter 2020 Earnings Conference Call. [Operator Instructions]
At this time, it is my pleasure to turn the floor over to Mr. Andrew Drechsler. Sir, the floor is yours.
Andrew Drechsler -- Chief Financial Officer
Thank you, operator. And thank you all for joining us on Provention Bio's second quarter financial results conference call. Joining today's call from the Provention Bio team are my colleagues, Ashleigh Palmer, CEO and Co-Founder; and Jason Hoitt, Chief Commercial Officer.
First, let me remind you that the various remarks we will make today constitute forward-looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. These include statements about our future expectations, clinical results, developments and regulatory matters and time lines, including for the teplizumab BLA; 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 in the second half of 2020 and cash runway; and our business plans and prospects, including planned precommercial activities across the Company in preparation for the potential approval of 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 Factors section of our most recent annual report on Form 10-K, which is on file with the SEC 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.
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 Provention files with the SEC. These documents are available at Provention's website at www.proventionbio.com, under the Investors section. We encourage you to review these documents carefully.
I would also encourage you to read our earnings release and our 10-Q that was filed today. The 10-Q includes our financial statements, risk factors as well as management's discussion and analysis of our financial condition.
With that being said, please allow me to provide our Q2 financials. As of June 30, 2020, our cash, cash equivalents and marketable securities balance was $172.2 million. This balance includes the net proceeds of $103.3 million from our June follow-on equity offering as well as $9.9 million of net proceeds from the sale of common stock under the at-the-market program during the second quarter prior to the follow-on equity offering.
From a P&L perspective, we generated a net loss for the second quarter of $22.1 million or $0.45 per basic and diluted share. The increase in net loss over the prior year was primarily attributable to teplizumab-related CMC cost of $7.5 million, which Ashleigh will detail shortly, precommercial costs of $4.9 million as well as BLA preparation costs and medical affairs expenses. Our net loss for the first six months of 2020 was $34.7 million or $0.72 per basic and diluted share. Our cash-based operating expenses for the first six months ended June 30, 2020 were $33.1 million. The spend in the first six months and particularly in the second quarter was focused on CMC, precommercial and regulatory activities related to teplizumab and was driven by our time lines for teplizumab's potential marketing approvals in the US and our positive manufacturing progress to date.
Consistent with our current plans, we expect to use approximately $44 million to $52 million of cash for operating needs in the second half of 2020 as we continue to scale and ready our commercial organizations, continue key manufacturing and regulatory activities and build out our US commercial infrastructure in preparation for a potential launch of teplizumab next year. We expect that our current cash, cash equivalents and marketable securities will be sufficient to fund projected operating requirements to potential approval and into the launch of teplizumab.
With that, let me turn the call over to Ashleigh for an update on teplizumab. Ashleigh?
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
Thank you, Andy and thank you all for joining us today. In quarter 2, we made tremendous progress across all areas of our business as we continue to implement our plan to bring our lead candidate teplizumab, also known as PRV-031 to market for the delay or prevention of insulin-dependent type 1 diabetes, or T1D, increase symptomatic patients. We remain on track to achieve our key goal for 2020, completing the submission of our rolling BLA for teplizumab and assuming a priority review, setting the stage for a potential FDA approval in mid-2021.
In parallel, we are building our organization and laying the groundwork for potential launch. Initial market research, some of which we will share with you today has reinforced our confidence in the initial launch opportunity targeting presymptomatic patients, especially in light of the recently presented clinical data, further strengthening the transformational value proposition of teplizumab. Specifically, we were emboldened by the extended follow-on data from the At-Risk TN-10 Study presented at the ADA's 80th scientific sessions in June. These data show that a single course of teplizumab, consisting of a daily 30-minute infusion over two weeks, continues to significantly delay the onset of insulin-dependent T1D in presymptomatic patients by a median of approximately three years as compared to placebo, adding another 12 months to the previous two-year median delay reported in the New England Journal of Medicine last year.
Not only are these results highly and increasingly clinically relevant, they remain highly statistically significant. We're not talking about a delay of three days, three weeks or three months, all of which would be precious. Ask any T1D patient or their family. It's a delay of three years, just incredible. Wow. No new safety signals or data were presented at this year's ADA conference, but data was presented that confirmed the preservation and restoration of beta cell function as determined by C-peptide, which is a measure of a person's own insulin production.
Consistent with the autoreactive T cell destruction of insulin-secreting beta cells, which is the characteristic of type 1 diabetes, C-peptide levels in the TN-10 Study decreased in patients administered placebo. In contrast, patients receiving a single course of teplizumab demonstrated a relative increase in C-peptide levels, suggesting not only a delay in beta cell destruction, but also the recovery of sick or dying beta cells and the restoration of their functionality and insulin secretion.
The follow-on TN-10 data presented at this year's ADA further underscores the disease-modifying potential of teplizumab. We believe that for the first time, our investigational therapy presents an opportunity to reboot or reset the immune system and delay the progression of a serious life-impacting and life-threatening autoimmune disease. We are laser-focused on getting teplizumab to the T1D community as quickly as possible, and we are making excellent progress with our rolling BLA submission.
We remain on track to submit our clinical module by the end of this quarter and to complete the full BLA submission on schedule in quarter 4, when we plan to file the remaining CMC modules. I cannot speak more highly of the extraordinary efforts, hard work, long hours and selfless dedication of our teplizumab BLA rolling submission team of Provention Bio employees and consultants, under the leadership of our Chief Medical Officer, Dr. Leni Ramos; and our Senior Vice President of Regulatory Affairs, Dr. Sharon Rowland.
I would also like to highlight the outstanding efforts and excellent work being undertaken by our strategic manufacturing partner, AGC Biologics. As you will recall, AGC completed an engineering run at the end of 2019 and in quarter 1, successfully completed a cGMP run. Today, we are pleased to report that Provention and AGC have achieved yet another mission-critical milestone with the on-schedule completion of our drug substance PPQ manufacturing campaign.
PPQ stands for process, performance and qualification, and consists of three back-to-back commercial scale runs required for the validation of our drug substance manufacturing process and the demonstration of our ability to manufacture consistently, batch to batch, at commercial scale. The results from our drug substance PPQ runs will serve as the foundation for our BLA's CMC module. And with our successful on-schedule completion of this campaign, we have addressed a very significant component of the overall operational risk associated with the on-time filing of our rolling BLA submission, especially when taking the current COVID-19 pandemic logistical and supply chain challenges into consideration.
Overall, we are pleased with the status of our ongoing rolling BLA submission process and time line. As I stated earlier, assuming a two-month acceptance [Phonetic] period and a six-month priority review, which we are eligible to file for under teplizumab's breakthrough therapy designation, we could potentially see an FDA decision in the middle of next June. In transitioning to a commercial organization ahead of a potential US teplizumab launch, we have been building a strong commercialization team and expanding our executive leadership team and operational infrastructure. This includes key hires across market access, sales, marketing, analytics and forecasting, medical affairs, regulatory affairs, quality, CMC, operations and logistics.
Earlier this week, we welcomed Heidy Abreu King-Jones as our General Counsel and Chief Legal Officer. From her prior industry experience, Heidy brings to Provention valuable expertise developing commercial compliance programs and supporting the launch of therapeutics in the rare disease space. Heidy's appointment reinforces that Provention continues to attract and secure top talent from across the biotech industry in preparation for next year's anticipated teplizumab launch.
I'm now going to ask Jason to highlight some key findings from our initial market research, which, above all, recognize and emphasize the profound impact that a three-year delay on the onset of clinical stage type 1 diabetes may have on patients and their families, otherwise facing a lifetime of insulin dependency, intensive glucose monitoring and fundamental lifestyle challenges in order to survive. Jason?
Jason Hoitt -- Chief Commercial Officer
Thanks, Ashleigh. In addition to building a world-class team with extensive biotech launch experience, in the first half of this year, we began our engagement with the payer, patient and healthcare provider communities in order to assist us in developing the most compelling messaging to effectively communicate our vision of a new standard of care for T1D treatment.
In May, we convened our first payer advisory board, consisting of medical directors from plans that represent greater than 73 million covered lives across our anticipated payer mix. We are encouraged by the insights we gained from this meeting, and that by the end of the meeting, we observed close alignment of the participants' understanding of T1D being the result of autoimmunity as well as their understanding of the underlying disease process and mechanism of progression. As expected, screening was unanimously supported, with 0 pushback on coverage during the meeting.
In addition, payers recognize the disease-modifying potential for teplizumab, and reacted favorably to the TN-10 Study data. I would like to point out that this payer advisory board was conducted in May, before knowing the extended TN-10 follow-on results, showing a median three-year rather than two-year delay in the progression to end-stage insulin-dependent T1D. The payers in attendance anticipated coverage for teplizumab to label for the vast majority of patients. As you would expect, payers indicated final prior authorization criteria would be determined by pricing. They noted that denying a disease-modifying drug like teplizumab would almost certainly be overturned on appeal.
In addition to this advisory board, we've commenced our direct engagement of key national and regional payers. In these initial meetings, the feedback was overwhelmingly consistent with that of our payer advisory board. The disease education and corporate overview of prevention provided by our market access team resonates well with our key payer audience. In addition to continuing key payer meetings, we also plan to continue our health economic modeling work, a real-world evidence study examining both direct and indirect medical costs associated with T1D, which we expect will further reinforce the value proposition for teplizumab.
In recent months, we've conducted blinded qualitative market research, including patients and their caregivers as well as approximately 100 healthcare professionals, comprised of pediatric and adult endocrinologists and certified diabetes educators. The transformative potential of teplizumab has been welcomed enthusiastically by these key stakeholders. In particular, the initial proposition that a single course of teplizumab therapy has the potential to "buy time" now by a median of approximately three years before progressing to insulin-dependent T1D as seen in studies today, resonates with patients and their families as well as medical professionals.
Patients and caregivers shared perspective supporting that such a significant delay would provide valuable time for them to prepare, logistically and psychologically. But the dramatic lifestyle change that accompanies a clinical stage T1D diagnosis. For children, teenagers and even young adults, a three-year delay may offer additional time to mature, educate themselves and become better positioned to cope with the challenges and additional responsibilities of insulin dependence.
Healthcare professionals echo these sentiments, recognizing the profound physical and mental impact a multi-year delay may have on families and patients. In addition to potential benefits for patients supported by clinical data to date, healthcare providers were comfortable with teplizumab's safety profile, reinforced by Provention's clinical database of over 800 patients dosed to date. Endocrinologists and diabetes educators that participated in market research also generally agree that the commercial availability of teplizumab likely would rapidly advance discussion around the need for screening and potentially catalyze its broader utilization. These experts are already routinely reviewing screening options with patients and caregivers of existing pediatric patients and proactively offering screening tests to some siblings and other family members.
As you would expect, families familiar with T1D are highly motivated to screen the siblings of patients, as they clearly have a unique understanding of the challenges of living with and managing this relentless disease. Our research confirms that the availability of a potential treatment to delay the onset of insulin-dependent T1D would compel even more screening. During the remainder of this year, we will conduct additional market research with these key constituent groups in both the traditional blinded fashion as well as a series of upcoming advisory boards. Ultimately, our goal is to work with key T1D stakeholders to support and make broad-based population screening a reality in the United States.
In the near term, we intend to continue to engage healthcare practitioners, endocrinologists and family members of T1D patients through implementation of consumer and healthcare provider campaigns to increase the understanding of the familial risk of T1D and the need for increased autoantibody testing. We plan to launch these Provention Bio's disease and screening awareness campaigns by the end of the third quarter. We look forward to providing you with additional details as they become available.
Back to you, Ashleigh.
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
Thank you, Jason. The advisory board's meetings and discussions we are having with key constituents in the T1D market are critical to the successful launch of teplizumab. It is also worth noting that while this market research is primarily exploring the currently documented three-year median delay brought about by a single course of teplizumab used in relevant clinical trials to date, we believe that redosing teplizumab at appropriate time point has the potential to indefinitely delay the onset of insulin-dependent disease. In fact, we believe that teplizumab's mechanism of action, converting pathological autoreactive T cells into exhausted T cells with a regulatory phenotype, provides a useful biomarker for when immune tolerance is starting to wane and another course of teplizumab may be required.
In addition to redosing, following anticipated launch, we will be exploring other label expansion and life cycle opportunities for teplizumab, including studying patients below eight years of age, evaluating combinations with adjunctive therapies, potentially providing concomitant immunomodulatory therapy for cell transplantations in established T1D patients and, of course, use to preserve the remaining beta cell function in newly diagnosed patients.
The newly diagnosed indication is the target for our ongoing global Phase III PROTECT study. As you will recall, in March, during the early stages of the COVID-19 pandemic, we temporarily paused randomization of patients. In conjunction with our data monitoring committee, which now includes an infectious diseases expert, we have since resumed randomization on a country by country, site-by-site, case-by-case basis, depending on the ever-changing status of the global pandemic and, at all times, prioritizing the safety of our patients, their caregivers and the amazing clinical site staff and monitors associated with the PROTECT study.
Before we open the call for questions, let me provide you with a brief update on our other pipeline programs. For PRV-101, our investigational polyvalent vaccine for the prevention of acute infection by coxsackievirus B, or CVB, as well as for the prevention of CVB-associated type 1 diabetes and celiac disease, we are pleased to report that in conjunction with our strategic partner, Intravacc, we have now successfully completed all the GMP manufacturing batches for the constituent serotypes [Phonetic], and we, therefore, expect to be on schedule to initiate later this year our first in-human Phase I trial evaluating two dose levels of PRV-101 in healthy adult volunteers for the purposes of safety, tolerability and immunogenicity. Initial data is expected in the second half of 2021.
In collaboration with Amgen, we are developing PRV-015, an investigational anti-interleukin 15 monoclonal antibody, which we believe could be the first-ever therapeutic to be approved for celiac disease. Having received confirmation from the FDA and the Central Ethics Committee, that our Phase IIb PROACTIVE study may proceed in the United States, we now plan to initiate patient screening in the coming weeks. The PROACTIVE study is targeting the enrollment of 220 adult celiac patients not responding to their gluten-free diets, comparing three PRV-015 dose levels with placebo over six months. Results are expected in the second half of 2022.
And finally, we also continue to advance PRV-3279, an investigational state-of-the-art bispecific scaffold, targeting both CD32B and CD79B receptors to modulate B cells. We believe PRV-3279 has broad potential in a number of indications with multiple potential shots on goal, including as a monotherapy for the treatment of B cell-mediated autoimmune diseases such as lupus. We are planning to commence a Phase IIa trial in lupus patients in the first half of 2021.
So in summary, we are driven by the ever-increasing possibility of pioneering the first disease-modifying therapy for type 1 diabetes. Throughout the remainder of 2020, we will continue to focus on the completion of our rolling BLA submission and in parallel, transition and transform our Company into a commercialization-ready organization in anticipation of the potential launch of teplizumab next year. In addition to teplizumab, our pipeline is rich with potential opportunities to fundamentally address the unmet needs associated with other serious autoimmune diseases, and we are passionate about advancing our therapeutic candidates to help both patients and their caregivers.
And now we will open the call up to take your questions.
Questions and Answers:
Operator
Thank you. The floor is now open for questions. [Operator Instructions] Our first question comes from Thomas Smith of SVB Leerink. Please state your question.
Thomas Smith -- SVB Leerink -- Analyst
Hey, guys. Thanks for taking the questions. And congrats on all the progress during the quarter. Just a couple of questions on my end. First, I guess, on the PROTECT study. It sounds like you've resumed enrollment there on a country-by-country and a site-by-site basis. It's obviously a pretty dynamic environment, but can you share your latest thoughts on enrollment cadence here? And I guess, any updated expectations in terms of potential timing for data?
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
Yes. Thanks, Tom. So based on current expectations, but obviously, the winter months and resurges in the pandemic could change the situation, we would hope to have enrollment completed by the first half of next year.
Thomas Smith -- SVB Leerink -- Analyst
Okay. Okay. Great. And then I guess shifting to the teplizumab BLA and the clinical data module you're expecting to submit this quarter. Can you talk about whether you're able to incorporate the updated data from the TN-10 Study that was presented at ADA? And how this might impact the discussion with the agency? Is there any chance that those data end up included in the initial label?
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
The clinical module will be mostly complete and the target labeling will be for the two years. We will include as much of the three-year data as possible, but obviously, in order for the FDA to review that data, it's not just the case of reporting the presentations or a publication, that data has to be validated and inspected, and it may be that we're unable to get all of that data and those requirements in place for the initial submission. But we will continue to update the submission throughout the review period, and it will be determined at the end of the review period to what extent the three years is referenced in the label. We've very clearly established, though, that while three years is better than two, two is absolutely good enough.
Thomas Smith -- SVB Leerink -- Analyst
Right. Right. Okay. And I guess just one last question, if you'll allow. It sounds like you've made a lot of progress on the payer side. And I guess now that you've had the initial ad board meeting and some direct engagement with some of the payers, has anything changed in terms of your thoughts around potential pricing for teplizumab?
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
Jason?
Jason Hoitt -- Chief Commercial Officer
Yeah. Thanks for the question, Tom. I think based on the initial engagements that we've had and based on the outcome of the payer ad board, I think we're even more enthusiastic and encouraged by the payer response to the profile of teplizumab and the value proposition that we'll be putting in front of them later this year once we finish our real-world evidence study.
I would say, to that extent, nothing has really changed with respect to price because the focus of the meetings that we've had so far really haven't been about the pricing. They've been about the profile of the product, the burden of the disease, the burden of illness, to set up the pricing research that we'll conduct in the early part of next year.
Thomas Smith -- SVB Leerink -- Analyst
Got it. Okay. Great. Well, thanks so much, guys for taking the questions and congrats again on the quarter.
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
Thanks, Tom.
Operator
Our next question comes from Alethia Young of Cantor. Please state your question. Ms. Young, your line is open, if you can please check your mute button. Ms. Young are you on the line. I am not hearing any response, gentlemen.
Andrew Drechsler -- Chief Financial Officer
Okay. Well, please go to the next question, operator. Thank you.
Operator
We'll go next to Gregory Renza of RBC Capital Markets. Please state your question.
Gregory Renza -- RBC Capital Markets -- Analyst
Good morning, Ashleigh, Andy and Jason. Congrats on the progress and thank you for taking the question. Just to build on some of the access discussion, we appreciate the additional color you've provided today. I was wondering, Ashleigh and perhaps, Jason, too, maybe provide a little additional context on perhaps that incremental receptivity that you have or anticipate garnering from the payer and reimbursement community on that ADA data and that additional year of delay. I know you've alluded to that a little bit. I'm just curious how that perhaps translates into that potential enthusiasm there. Thank you.
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
Jason? I think it's more a case of a very strong reinforcement of our confidence as opposed to a fundamental change. Again, the additional year over the two years, which is, as I said earlier, a real wow factor in this community, is really good and really welcome. But it's still, just based on the two years, a very profound impact and something that this community hasn't seen or contemplated since insulin was introduced. But do you want to say anything more, Jason?
Jason Hoitt -- Chief Commercial Officer
Yeah. I think that summarizes it pretty well, Ashleigh. I think -- I appreciate the question, Greg. When we did this advisory board, we were focused on the two-year data. And subsequent to that, obviously, the three-year data came out at ADA and have been incorporated into the introductory deck that our team is using to introduce Provention Bio, introduce type 1 diabetes, the burden of illness, the burden of disease, and then ultimately, the mechanism of action and the impact of teplizumab on the at-risk patient population with payers.
And I would say that the reaction that our team is getting, and as we engage payers in those one-on-one forums, is entirely consistent with what we heard in the ad board. And I think the three year data further enhance our enthusiasm around the value proposition that will come from that real-world evidence study when we model out the burden of illness, the cost effectiveness analysis, the budget impact analysis, and support the underlying value proposition that we put in front of payers later this year and early next year when we do the formal pricing research.
Gregory Renza -- RBC Capital Markets -- Analyst
That's very helpful. Thank you. And then, and maybe just one more for me. And certainly, some enthusiasm about the potential for redosing. And I'm curious, Ashleigh, how that recent data from ADA and as that evolved over time can help perhaps inform that process and the potential cadence and timing for you to look at optimal redosing in that strategy there? Thank you very much.
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
Thanks, Greg. Yeah. So obviously, we have to do a lot more work on the redosing and validate our assumptions. But our perception of the evolution of the TN-10 follow on data is that there may in fact, be two populations. There may be a population of individuals who have their autoreactive T cells neutralized and -- at some point, and the ADA presentations also indicated this, the exhausted T cells and their decline, these are the cells that the autoreactive T cells are converted into with a regulatory phenotype. But that decline may be an indicator as to when a second or follow-up dose could be administered, and that, that could be repeated in order to indefinitely delay.
The other population, though, is those that are sort of seven and eight years after the single course of therapy, it may be that there are patients who are essentially tolerized by a single dose, and either the rate of development of their disease or the impact of teplizumab on their autoreactive T cells is much more sustainable and that they may never need another dose at all and remain disease-free or end-stage disease free so to speak. But again, it may be the exhausted T cells that help us understand that. Francisco, are you on the line? You're the expert in exhausted T cells and the analysis of the ADA data.
Francisco Leon -- Chief Scientific Officer and Co-Founder
Yes, indeed. Just as you said, the new data release this year showed that any subject who had an increase in exhausted T cells above 10% of their total T cell number did not progress to clinical T1D in the course of the study. And now we're accumulating a very lengthy follow-on -- follow-up. So I think the hypothesis is framing up that we can use these cells to guys redosing and some subjects may not need to be redosed for very extended periods of time or perhaps never.
Gregory Renza -- RBC Capital Markets -- Analyst
That's great. Thanks for taking my question.
Francisco Leon -- Chief Scientific Officer and Co-Founder
Thanks, Greg.
Operator
Our next question comes from Alethia Young of Cantor. Please state your question.
Alethia Young -- Cantor Fitzgerald -- Analyst
Can you guys hear me?
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
Hi, Alethia. Good morning. How are you?
Alethia Young -- Cantor Fitzgerald -- Analyst
Can you guys hear me? Okay, cool. Okay. So sorry about that. So two questions from me. One, congrats on like the commercial -- again, to the commercial scale. I kind of wanted to just -- is that kind of the big step from here, we're kind of somewhat down here on the note part of the process is easy and filing, but I just wanted to kind of get your flavor on that? That seems to be a big hurdle that need to be completed. And then the second part, which may have gotten asked already, but I was going back in, I guess I was just trying to wonder where, you know...
Andrew Drechsler -- Chief Financial Officer
I think we lost, Alethia. Ashleigh, do you want to take the first part of her question?
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
Yeah, I wasn't sure if it was me who dropped or Alethia, so thanks for chiming in there, Andy. Yeah. So many of our investors have expressed due concern over the manufacturing process because they see it as a black box. Companies do not go into great detail about manufacturing, especially since a lot of manufacturing is proprietary know-how. And so you have these milestones. First engineering runs, first GMP run, the PPQ runs. And they are notorious for complications. And having transferred our process at commercial scale to run three back-to-back runs without significant issue and for our internal team to feel really good about those PPQ runs accomplishing their objectives is, yes, I believe, a very significant accomplishment that takes a lot of the risk out of the remaining CMC module that we have to file.
We have to do the remaining PPQ work on the filling and the finishing because you can appreciate that is much more straightforward and routine than the complex fermentation or cellular production of a monoclonal antibody with lots of factors that can impact the outcome and undermine the success of the runs. That part of our manufacturing is behind us. And now it's about collecting the data from all the samples and submitting that alongside additional data, including PPQ runs on the fill, finish and putting the final product up on stability. And we're really, really pleased with the outcome from accomplishing these three PPQ runs with drug substance.
Operator
And Ms. Young, your line is open. Please you can go ahead and speak.
Alethia Young -- Cantor Fitzgerald -- Analyst
Can you hear me? My second question was just who might -- how do we think about the biology of potential super responders for teplizumab?
Andrew Drechsler -- Chief Financial Officer
Thanks, Alethia. So Francisco, do you want to talk about super responders?
Francisco Leon -- Chief Scientific Officer and Co-Founder
Yes. So we know that exhausted T cells correlate with no progression to clinical T1D in the natural course of the disease, that has been established and published before teplizumab even is introduced. So these cells are the other side of the coin of the ultrareactive T cells. So what we are seeing now is that when teplizumab is given to patients with presymptomatic disease, those who achieve greater levels of T cells do not seem to progress, and some of those subjects have been followed for eight years, as Ashleigh mentioned. So the possibility emerges that they are long-term tolerized, that their immune system is now able to continuously prevent the ultrareactivity against the beta cells.
If you think about a similar situation in immuno-oncology, when you look at the Kaplan-Meier curves, before immuno-oncology, the Kaplan-Meier curves were shifted slightly to the right by chemotherapy, radiotherapy, but what the immuno-oncology did was to shift those curves up, meaning some subjects never progressed. And that's what we are starting to see with teplizumab. The Kaplan-Meier curve of survival does not just shift to the right, but also shift up. And as you can see in the 2020 update, it has become a flat line that moves forward into the future, without going down to the conversion into clinical T1D. So it's a very interesting possibility that has a strong biological fundamental.
Alethia Young -- Cantor Fitzgerald -- Analyst
Great. Thank you.
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
Thanks, Alethia.
Operator
Our next question comes from Justin Kim of Oppenheimer. Please state your question.
Yichuan Yan -- Oppenheimer -- Analyst
Yes. Hi. Good morning. Congrats on the progress. This is Jackie on for Justin today. First I guess, would the teplizumab's first approval qualified for pediatric drug voucher?
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
So a pediatric drug of voucher, I believe, requires orphan status, and we've obtained orphan status for newly diagnosed and are now working on orphan for at-risk, but we've not made an announcement that, that has been accomplished yet.
Yichuan Yan -- Oppenheimer -- Analyst
Got it. Got it. Thanks for that. Maybe one for Jason on the commercial front. From a market perspective, how accessible from a patient identification perspective, that the group of familial stage 1 patient up here? And how important will labeling be in treating these patients? Thanks.
Jason Hoitt -- Chief Commercial Officer
Yeah. So I think nothing's changed with respect to how we view the addressable market for the initial at-risk population, right? So we see this initial familial direct relative market focus at launch of approximately 30,000 patients. And as I mentioned in our prepared remarks, we'll be rolling out awareness campaigns later this quarter to specifically raise the level of awareness and engagement of those populations for why they're at disproportionate risk for being in the early stages of type 1 diabetes and why they should go and seek out screening, knowing that screening is commercially available to date. I would say that, -- then it does, as you mentioned, come down to labeling and what the final label looks like, I think, is how we will expect that payers will be covering the product, right? That we expect there will be a prior authorization to label, as I mentioned, from the one-on-one engagements we've had with payers as well as the advisory board that we did in May.
Yichuan Yan -- Oppenheimer -- Analyst
Got it. That's very helpful. And my last question is for the Coxsackie vaccine program. Can you talk about what pieces of evidence gave the team confidence that candidate would now induce unwanted autoimmunity? Thanks.
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
Thanks a lot. Francisco, that's definitely yours.
Francisco Leon -- Chief Scientific Officer and Co-Founder
Yes. Thanks for the question, Justin. So we have multiple safeguards for the vaccine. So it is an inactivated vaccine so that viruses that cannot infect the pancreas, that's the first thing. Second, there have been some epitopes proposed through potential mimicry. Mimicry has never been proven in T1D to be the cause for T1D [Indecipherable] infection. But just to be safe, those epitopes are not included in the vaccine and/or they are included in other vaccines, such as, for example, the Polio vaccine. The Polio vaccine, Polio being and enterovirus as well has been given to children with high-risk of T1D, and they did not have any increase in T1D. So we feel quite confident that the vaccine is devoid of this kind of theoretical side effects. But of course, we will assess that in the first inhuman study.
Yichuan Yan -- Oppenheimer -- Analyst
Great. Great. Thanks again for that. Thank you.
Francisco Leon -- Chief Scientific Officer and Co-Founder
Thanks a lot.
Operator
Our next question comes from Raghuram Selvaraju of H.C. Wainwright. Please state your question.
Edward Marks -- H.C. Wainwright -- Analyst
Good morning.
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
Good morning.
Edward Marks -- H.C. Wainwright -- Analyst
Good morning. This is Marks speaking on behalf of Ram Selvaraju. Thanks for taking my question. So I had a couple of pipeline questions firstly and then some queries about marketing and partnerships. So in terms of the pipeline, how many additional releases of data from the At-Risk Study might you expect to occur before the end of the year? And how many patients you've lost as a follow-up since the previous data release?
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
We don't anticipate -- although it's not our data. So it's hard to tell, but we don't anticipate that there will be any additional updates before the end of the year. It appears that they're on an annual calendar schedule and see the ADA as a venue for that. It's entirely possible that the data that was presented in presentation format at the ADA is subject for a potential publication. But again, that data belongs to TrialNet, and sponsored by NIDDK, and we don't control the academic publication side of the equation. We do have, though, following initial publication, the rights for the purposes of regulatory submission.
Edward Marks -- H.C. Wainwright -- Analyst
And do you foresee any scope for other autoimmune indications for teplizumab beyond type 1 diabetes?
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
We do. And we obviously are focused on getting our breakthrough therapy designation for T1D cross the finish line first. We're also, in parallel, focusing on the possibility of a subcutaneous formulation, which we feel could facilitate expansion outside of type 1 diabetes and obviously be an improvement in terms of therapeutic burden in T1D itself. Although a single course of two weeks therapy versus a lifetime of regular immunosuppressants, which is sort of how many autoimmune diseases are managed these days is deemed to be vastly superior from a therapeutic burden perspective, even though we're administering IV. But Francisco, do you want to just give a flavor of some of the indications that we could pursue with teplizumab in that context?
Francisco Leon -- Chief Scientific Officer and Co-Founder
It will be T cell driven autoimmune disorders, trans [Phonetic] disease, celiac, autoimmune hepatitis, psoriatic arthritis, where we already have data. It's published. Rheumatoid arthritis, lupus, etc. It's a wide range of autoimmune and inflammatory disorders that are driven by T cells.
Edward Marks -- H.C. Wainwright -- Analyst
Fantastic. Thanks for the granularity on that. And just a couple of marketing and partnership questions, if I may. Is the marketing authorization application for teplizumab for T1D in Europe still on track for submission? And are you seeing significant partnership interest from ex US territories?
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
Yes, the EMA process is on track. We're in the stage of having scientific advice meetings scheduled and taking place in Europe, and that continues. We've anticipated that. We're approximately 12 months behind the schedule for the United States. And we have indicated that we will not be attempting to launch teplizumab directly outside of the United States, and that there are a number of scenarios that can help ensure we have the in-market infrastructure and partnership in Europe to facilitate commercialization, but we've not made an announcement on the progress of those discussions.
Operator
Our next question comes from David Hoang of SMBC. Please state your question.
David Hoang -- SMBC Nikko Securities -- Analyst
Hi, guys. Good morning, and thanks for taking my question. So I had a few. So first, do you -- I guess, what are your expectations in regards to potentially having an FDA ad com? And then could you provide a little bit more color on the patients -- the at-risk patients under eight years of age? What percentage of at-risk patients does that represent? And how might you reach those patients eventually?
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
Yes. Thanks. So we have not had discussions with the FDA regarding an ad com. But as you would imagine, a seasoned management team expects or tries to expect everything. And so we have -- we are certainly preparing. And those preparations are actually ongoing in parallel with the regulatory filing and submissions, and we'll be well prepared for that event, assuming that it happens.
Regarding under eight years of age, yes, this is a very significant opportunity for at-risk -- patients who are at risk of end-stage disease, because while the diagnosis of clinical stage T1D peaks around the ages of 10 to 12, the appearance of autoantibodies clearly appears much earlier than that, in some instances, as early as two years of age. We have a pediatric plan, which we've submitted to the FDA and will be part of the discussions with respect to the ongoing submission and review. And we're committed to ensuring that we collect data as quickly as possible post the anticipated launch in order to be able to reassure the agency and clinicians and parents that administering teplizumab below eight years of age is safe and effective.
David Hoang -- SMBC Nikko Securities -- Analyst
Okay. Great. Thanks. That was really helpful. And then I just had one other question, and this is in regards to how the 14-week course of teplizumab might work in clinical practice. Is there any concern that some patients may miss some of the doses? And is that something they need to come into the infusion center for every day? Or there are alternatives in terms of how they could receive their doses?
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
So if I may break that question down into two. One, on the scientific side, and that's Francisco. The potential impact of a miss or a delay in any one of those daily 30-minute infusions. And once he's addressed the medical or scientific concerns, ask Justin to speak to the commercial leadership team's strategy with respect to infusions and making infusions as convenient as possible for clinicians and patients and essentially making sure that we provide the facility for them to administer the drug wherever their preference is. But first Francisco, what if a patient misses a day?
Francisco Leon -- Chief Scientific Officer and Co-Founder
Yes. Thank you, David. And just to clarify, it's 14 days, not 14 weeks of teplizumab. And we do have data already in Phase II. This was explored. We know that as little as six days of therapy has an effect, but the effect is not as robust as 12 or 14 days. That's why we are now using 12 days in our nearly diagnosed program. So based on our pharmacokinetic, pharmacodynamic analysis, we believe patients need to get at least 10 to 12 days of therapy. So if they missed a day, they missed two days, we will ask them to complete the course. We will try to give them those 14 days, but it will not be an absolute loss of that responsiveness to the drug.
Jason Hoitt -- Chief Commercial Officer
David, this is Jason. I'll take the second part of your question, which, in the commercial context, I think the way we're preparing for all eventualities in terms of product acquisition and site of administration, and so I think there are three potential scenarios, right? The first scenario, a patient gets infused in the infusion center, and we'll provide the billing and coding guide so that they know how to go about acquiring the product directly from our specialty distributor. Second scenario is the potential for home infusion, which we would be preparing for through limited network of specialty pharmacies that have that capability in all 50 states.
And then the third eventuality is that they would start in an infusion center under observation and once the physician and the family feels comfortable with how the infusions are going, they transfer from the infusion center to home infusion, and we'll make sure that we have all three of those scenarios covered such that the decision can be made by the healthcare provider, the family and the caregiver as to what works best for them and make sure that we have a clear route for product acquisition and infusion for all three scenarios.
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
So we're on the hour, and I really appreciate everybody's questions, fantastic questions. But I'm going to thank you all for joining us today, and let you know that we look forward to updating you soon on our continued progress, and we sincerely hope that you and your loved ones stay safe and well throughout the summer. Thank you.
Operator
[Operator Closing Remarks]
Duration: 61 minutes
Call participants:
Andrew Drechsler -- Chief Financial Officer
Ashleigh Palmer -- Chief Executive Officer and Co-Founder
Jason Hoitt -- Chief Commercial Officer
Francisco Leon -- Chief Scientific Officer and Co-Founder
Thomas Smith -- SVB Leerink -- Analyst
Gregory Renza -- RBC Capital Markets -- Analyst
Alethia Young -- Cantor Fitzgerald -- Analyst
Yichuan Yan -- Oppenheimer -- Analyst
Edward Marks -- H.C. Wainwright -- Analyst
David Hoang -- SMBC Nikko Securities -- Analyst
