Q2 2020 Provention Bio Inc Earnings Call

Please standby.

Good day, ladies and gentlemen, and welcome to your prevention Bio second quarter 2020 earnings Conference call. All lines have been placed on the listen only mode and before the open for your questions and comments following the presentation.

Yes, you should require assistance throughout the conference. Please press star zero at this time it it's my pleasure to turn the floor over to Mr., Andrew Drexler, Sir the floor is yours.

Thank you operator, and thank you all for joining us on prevention bio second quarter financial results Conference call.

Joining today's call from the prevention bio team or my colleagues.

Actually Palmer CEO and co founder.

Jason White, 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 provision under the private Securities Litigation Reform Act at 1995.

These include statements about our future expectations clinical results development, and regulatory matters and timelines, including for the tipple isn't that the L.A. potential safety efficacy and commercial success of diplomat and there are other product candidates the potential cobot 19 impact on our clinical study.

Business plan financial projection, including our anticipated use of cash in the second half. The 2020, you cash runway and our business plans and prospects, including planned pre commercial activities across the company in preparation for the potential approval I wasn't bad and projected timing for the sake.

Actual results may differ materially from those indicated by these forward looking statements as a result to various important factors, including those discussed in the risk factor section of our most recent annual report on form 10-K, which is on file with the S. U C and in other filings that we may make with yes, you see in the future.

Any forward looking statements represent our views as of today only.

Well, we may elect to update these forward looking statements at some point in the future. We specifically disclaim any obligation could 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.

It was more complete information regarding forward looking statements risks and uncertainties in the reports prevention files with the FCC. These documents are available at prevention website at Www Dot prevention bio dot com under the Investor 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.

Thank you include our financial statement 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 Thirtyth 2020, our cash cash equivalents in marketable securities Dallas was 172.2 billion.

This balance includes the net proceeds of 103.3 million from our June follow on equity offering as well as 9.9 million at that age. So the sale of common stock under the aftermarket program during the second quarter prior to the follow on equity offering.

I'm a piano perspective.

We generated a net loss for the second quarter of 22.1 million or 45 cents per basic and diluted share.

The increase in net loss over the prior year was primarily attributable to to pleasant Mab related CNC costs of 7.5 million, which Ashley will detail shortly pre commercial costs of 4.9 billion.

As well as BLE preparation costs and medical affairs expenses.

Our net loss for the first six months of 2020.

It's 34.7 million were 72 cents per basic and diluted share.

Our cash based operating expenses for the first six months ended June Thirtyth 2020 were 33.1 million.

The spend in the first six months and particularly in the second quarter was focused on CMC pre commercial and regulatory activities related to deploy isn't that it was driven by our timelines for took wasn't mabs potential marketing approvals in the U.S. and our positive manufacturing progress to date.

Consistent with our current plans, we expect to use approximately 44 to 52 million up cash for operating needs in the second half a 2020 as we continue to scale and ready our commercial organization continue key manufacturing and regulatory activities and build out our U.S. commercial infrastructure in place.

Operation for a potential launch it's a pleasant back next year.

We expect that our current cash cash equivalent and marketable security will be sufficient to fund projected operating requirements to potential approval and into the launch of to plus about.

With that let me turn the call over to actually for an update on to pause about.

Ashley.

Thank you Andy and thank you all for joining us today.

In quarter, two we made tremendous progress across all areas of our business as we continue to implement our plan to bring our lead candidate to prison mob also known as PRB. So do you want to market. So the delay prevention of insulin dependent.

Type one diabetes.

He wants to be increased symptomatic patients.

We remain on track to achieve a key goal for 2000 didn't 20.

Completing the submission of our rolling B.L.A.'s attempt Lizzy ma'am I'm.

And assuming a priority review.

In the stage for a potential sta 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 three symptomatic patients.

Especially in light of the recently presented clinical data.

Further strengthening the transformational value proposition of diplomat.

Specifically, we will emboldened by the same did follow one data from the at risk T.N. 10 study presented at the <unk> eight years scientific sessions in June.

These data show that a single pools have kept museum.

Consisting of a daily so do you maybe confusion over two weeks continues to significantly be lady on sector insulin dependent. So you wouldn't be increased symptomatic patients by a median of approximately three years as compared to placebo.

I think another 12 months to the previous to your medium delay reported in the New England Gen Live medicine last year.

Not only are these results highly and increasingly clinically relevant they remain highly statistically significant.

We're not talking about the delay of three days.

Three weeks, well three minutes, <unk>, which would be precious.

Ask any t., one patient pool that family.

It's a delay of three years.

Just incredible.

No.

No you safety signals would data were presented at this year's 80 a conference.

So once because emptied the concern the preservation and restoration of beat to sell function.

I was determined twice the pet all right, which is a measure of the person's own insulin production.

Consistent with <unk>, well to reactive T cell destruction of Insulins decreasing beat <unk>, which is the characteristics of type one diabetes.

C peptide levels in the T.N. 10 study decreased impatience administered to see though.

He confessed patients receiving a single cool stepped listen map demonstrated a relative increase in C peptide levels.

Suggesting not only a delay in beat to sell destruction.

But also the recovery of see well dining be to sounds like the restoration of they functionality and insulin secretion.

The follow on T.N. 10 data presented at this year's <unk>.

And the schools the disease modifying potential looked up losing.

We believe that so the first time, our investigational therapy presents an opportunity to we do.

When we set the immune system and delay the progression of a serious life impacting and life threatening auto immune disease.

We are laser focused on getting kept lizzy maps to the T. One de community as quickly as possible and we are making excellent progress with our rolling BLE submission.

We remain on track to submit our clinical module by the end of this quarter and to complete the food B.L.A. submission on schedule in cost of school when we plan to filed the remaining C. N C module.

I cannot speak more highly of the extra ordinary.

Hard work long hours and selfless dedication of our attempt was another B.L.A. rolling submission team.

Prevention bio employee using consultants under the leadership about Chief Medical Officer, Dr., Lenny Ramos and our senior Vice President of regulatory Affairs.

Dr. Sharon Rowlands.

I would also like to highlight the outstanding exits and excellent work being undertaken.

Yeah, our strategic manufacturing partner AGTC biologic.

Let's see what we called AGTC completed an engineering run at the end of 2019.

And then close to one successfully completed a C.G.M.P. run.

Today, we are pleased to report.

Prevention and AG see have achieved yet another mission critical milestone would be on schedule completion of our drug substance P. P Q manufacturing campaign.

He PQ spends to process performance and qualification.

And consists of three back to back commercial scale runs required for the validation about drug substance manufacturing process.

The demonstration of our ability to manufacture consistently batch to batch <unk> commercial scale.

The results from our drug substance P. PQ runs well served as the foundation for all be L.A.'s CMC much Oh.

When we got successful on schedule completion of this campaign, we have addressed a very significant component of the EU operational risk associated with the on time filing about rolling B.L.A. submission.

Especially when taking the current complete 19 pandemic logistical and supply chain challenges into consideration.

Overall, we're pleased with the status of our ongoing rolling B.L.A. submission process and timelines.

As I stated earlier.

You mean that too.

It's period.

They six month priority review, which we are eligible to file for undetected <unk> breakthrough therapy designation.

We could potentially C and D. A decision in the middle of next year.

In transitioning to a commercial organization a hedge as a potential U.S. to please do not launch.

We have been building a strong commercialization team and expanding our executive leadership team and operational infrastructure.

This includes key players across market access.

Sales marketing analytics and forecasting medical Affairs regulatory affairs quality C N C operations and logistics.

Earlier this week, we welcomed Heidi Broking Jones, as our general counsel and Chief legal Officer.

From her prior industry experience Heidi brings to prevention valuable expertise developing commercial compliance programs.

Supporting the launch of therapeutics in the rare disease space.

Hi, These appointment reinforces that prevention continues to attract and secure top talent from across the biotech industry in preparation for next year's anticipated capitalism at launch.

I'm not going to ask Jason to highlight some key findings from our initial market research, which a bubble recognizing emphasize the profound impact, but a three year delay on the onset of clinical stage type one diabetes may have on.

Patients and their families.

Otherwise facing a lifetime of insulin dependent didn't see intensive glucose monitoring and fundamental lifestyle challenges in order to survive.

Jason.

Thanks Ashley.

In addition to building a world class team with extensive biotech launch experience and the first half of this year, we began our engagement with the payer patient and health care 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 T. Lundetrae.

Okay.

In May we convened our first hair advisory board consisting of medical directors from plant that represent greater than 73 million covered lives across our anticipated payer mix.

We are encouraged by the insights we gain from this meeting and then by the end of the meeting we observed close alignment of the participants understanding of C. D being the result of auto and it.

As well as their understanding of the underlying disease process and mechanism of progression.

As expected screening was unanimously support.

Zero push back on coverage during the meeting.

In addition, payers recognize the disease modifying potential such a pleasant.

And reacted favorably to the T.N. 10 study data.

I'd like to point out that this pair advisory board was conducted in that well score knowing the extended 10 10 follow on results showing a median three or rather than two year delay in the progression to end stage insulin dependent you Andy.

The payers in it in attendance anticipated coverage particle isn't.

The label for the vast majority of patients.

As you would expect payers indicated final prior authorization criteria would be determined by pricing.

He noted that denying the disease modifying drought like it wasn't.

Almost certainly the overturned on appeal.

In addition to this advisory board, we've commenced our direct engagement, a key national and regional payers.

And these initial meeting the feedback was overwhelmingly consistent with that of our payer advisement.

And disease education, and corporate overview of prevention provided by our market access team resonates well with our key pair 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 do you want to do.

Which we expect further reinforced the value proposition particular isn't.

In recent months, we've conducted blinded qualitative market research, including patients and their caregivers as well as approximately 100 health care professionals comprised the pediatric and adult endocrinologist and certified diabetes educators.

The transformative potential it took wasn't Bob has been welcomed enthusiastically by these key stakeholders in particular, the initial proposition that a single course, if it wasn't not therapy has the potential to quote by time and quote now by a median of approximately three years before progressing to insulin dependent t. Wendy.

As seen in studies to date resonates with patients and their families as well as medical professionals.

Patients and caregivers shared perspective, supporting that's 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 T. One do you diagnosis.

Our children teenagers and even young adults a three year delay may offer additional time to mature educate themselves and become better position to cope with the challenges and additional responsibilities of insulin dependence.

Health care professionals echo the sentiment recognizing the profound physical and mental impact a multiyear delay it may have on families and patients.

In addition to potential benefits for patients supported by clinical data to date health care providers were comfortable with to Pleasant Mab safety profile reinforced by prevention 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 to pleasant.

Likely would rapidly advance discussion around the need for screening and potentially catalyze its broader utilization.

He's experts are already routinely reviewing screening options with patients and caregivers existing pediatric patients and proactively offerings screening tests to some siblings and other family members.

As you would expect families familiar with T. Wendy are highly motivated just screen the siblings the patients as they clearly have a unique understanding of the challenges of living wet and managing this relentless disease.

Our research confirms that the availability of a potential treatment to delay the onset of insulin dependent T. One D with compelling even more screen.

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 T. One de 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 health care practitioners endocrinologists and family members of Gee, why do you patients through implementation of consumer and health care provider campaigns to increase the understanding of the familial risk of T. One D and the need for increased autoantibody testing.

We plan to launch these prevention bio disease 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 actually.

Thank you Jason.

The advisory boards meetings and discussions we're having with key constituents in the T. One de monkey.

I'll quit equal to the successful launch a prisoner.

It is also worth noting that while this market research is primarily exploring the currently documented three a median delay moved to backed by a single cool so completely man used.

In relevant clinical trials to date.

We believe the reducing tip listen I'm at appropriate time point has the potential to indefinitely delay the onset of insulin dependent disease.

In fact, we believe that at least denoms mechanism of action converting pathological well to reactive T cells into exhausted T cells with the regulators <unk> phenotype provides a useful biomarker for when immune tolerance is starting to wane and another cost of capitalism I maybe wrong.

Quiet.

In addition to read dosing following anticipated launch we will be exploring other label expansion in lifecycle opportunities for simplicity mouth, including studying patients below eight years of age.

Evaluating combinations with had jumped to favorite piece.

Potentially providing concomitant immunomodulatory therapy fuel cell transplantation in established T. Wendy actions.

And of course use to preserve the remaining beat to sell function in newly diagnosed patients.

The newly diagnosed indication is the target for our ongoing global so used three protex study.

I see what we call in March during the early stages of because the 19 pandemic.

We temporarily pools randomization of age.

In conjunction with aren't data monitoring committee, which now includes an infectious diseases, but 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 time pandemic.

And at all times prioritizing the safety about patients. They can't give is the amazing clinical site staff and bonuses associated with the Protex study.

Before we open the call for questions. Let me provide you with a brief update on our other pipeline programs.

[noise] for people to be one or one our investigational polyvision vaccine for the prevention of acute infection by Coxsackie virus be well CVB.

As well as for the prevention of CMV associated with type, one diabetes and Celia disease.

I'm pleased to report, but in conjunction with our strategic partner Intevac.

We have now successfully complete <unk> mm the GMP manufacturing batches for <unk> for the constituency.

And we therefore expect to be on schedule to initiate later this year Oh first in human phase one trial evaluating two dose levels of P. all the while no one 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 PRB, a one five an investigational anti interleukin 15, monoclonal antibody, which we believe could be the first ever therapeutic to be approved for sillier disease.

Having received confirmation from the FDA.

The Central Ethics Committee that I say used to be 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, I don't see at patients not responding to that gluten free diet.

Comparing three P. RV 015 dose levels with the C., but oh, the six month.

Results are expected in the second half of 2022.

And finally, we also continue to advance PRB 30 to 79.

An investigational state of the art by specific scaffold targeting both CD 32, B and C. D 79 be receptive to modulate b cells.

We believe PRB 30 to 79 has broad potential in a number of indications with multiple potential shots on goal, including as a monotherapy for the treatment b cell mediated auto immune diseases, such as lupus.

We are planning to commence a phase two Ole 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 one diabetes.

[noise] throughout the remainder of 2020, we will continue to focus on the completion of our rolling B.L.A. submission and in parallel transition and transform our company into a commercial lines. They shouldn't read you organization in anticipation of the potential launch of kept was the mountain <unk>.

Last year.

In addition to tech is the amount of our pipeline is rich with potential opportunities to fundamentally address the unmet needs associated with other serious auto immune diseases.

And we are passionate about advancing a therapeutic candidates to help both patients and their caregivers.

And now we will open the call up to take your question.

Thank you.

Floors now open for questions. If you do have a question. Please press star one on your telephone keypad at this time.

Since we'll be taking in order. They were received if you are using a speaker phone, we ask that well posing your question you pick up your handset provide favorable sound quality. If at any time. Your question has been answered you can remove yourself from the Q by pressing one again, ladies and gentlemen, if you do have a question or comment. Please press star one on your telephone keypad.

At this time, please hold while we pull for questions.

Our first question comes from Thomas Smith, I've asked me be Leerink. Please state your question.

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 on it sounds like you have resumed enrollment there on a country by country and a site by site basis. Its obviously, a pretty dynamic environment, but can you share. Your your latest thoughts on enrollment cadence here and I guess any updated expectations in terms of potential timing.

For data.

[noise], yes, thanks, Tom So based on current expectations, but obviously the winter months and researchers in the pandemic could change as the situation. We would hope to have enrollment completed by the first half of next year.

Okay, Okay, Great and then I guess.

Shifting to the to put them out the law and the clinical data module, you're expecting to submit a this quarter can you talk about whether you're able to incorporate the updated data from the 10 10 studying that was presented at a D.A. and how this might impact the discussion with the agency is there any chance that those data end up.

Included in the initial label.

The.

Clinical module will be most complete and the target labeling will be for the two years.

We will include as much of the three year to date to as possible.

But obviously in order for the FDIC to review that Dieter it's not just the case of reporting the presentations or a publication data has to be validating you've done 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 they will be determined no at the end of the review period to what extent.

Three years is referenced in the label.

We very clearly established though that was three years is better than two.

Who is.

Absolutely good enough.

Right right, Okay, and I guess, just one last question if you'll allow.

The allowance.

It sounds like you've made a lot of progress on the 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 to close enough.

Jason.

Yeah. Thanks to the question Tom I think you know based on the initial engagements that we've had and based on the outcome of the payer AD Board I think we're even a more enthusiastic and encouraged by the payer response to the profile of to Pleasant mob and the you know the value proposition that will.

Be putting in front of them layer that later this year and once we finish our real world evidence study I.

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 disease. The burden of illness to set up the pricing research that will conduct in the early part of next year.

<unk>.

Got it okay, great well. Thanks, thanks, so much guys for taking the questions and congrats again on the quarter.

Thanks, Tom.

Our next question comes from a leap year young of Cantor. Please state your question.

MS. Young your line is open if he could please check your mute button.

MS younger you on the line.

I'm not hearing no response gentlemen.

Okay Oh. Please go to the next question operator, thank you.

Well go next to a Gregory rental of RBC capital markets. Please state your question.

Good morning, actually Andean Jason that congrats on the progress and thank you for taking the question.

I think.

And just to just to build on some of the on the.

Access a discussion we appreciate the additional color you've provided today I was wondering I'm actually perhaps Jason to maybe put a provide a little additional context on perhaps that incremental receptivity that you have or anticipate garnering from from the payer and reimbursement that community on that on 88.

And that additional year, a delay I know you've alluded to that a little bit <unk> I'm, just curious how that perhaps translates into that potential enthusiasm there. Thank you.

Jason I think it's 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 which is you know as I said earlier, a real wild factor.

In this community is is is really good and really welcome but ER. It's still just based on the two years of very profound.

Impact and something that this community hasn't seen all contemplated you know since insulin was introduced but do you want to say anything more Jason.

Yeah, I think that I think that summarizes it pretty well actually I think you know I. Appreciate the question, Greg when when well we did this advisory board.

No. We were we were focused on the two year data and subsequent to that you know obviously the three year data came out at 88 and have been incorporated into the introductory deck that our team is using to introduce prevention bio introduce type one diabetes the burden of illness. The burden of disease and then ultimately the mechanism of action and the impact of to Pleasant Mab on the at risk we.

The issue with payers.

I'd say that there was the reaction that our team is getting and as we engage payers and those one on one forums is entirely consistent with what we hurting the outboard 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.

The effectiveness analysis the budget impact analysis.

Ah and support you know 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.

That's very helpful. Thank you and then maybe just one more for me and certainly and enthusiasm about the potential for for reducing and I'm curious actually how how is that a recent data.

From a D.A. and at that has evolved over time can help perhaps before that that processes and the potential cadence in timing for you to look at that optimal are reducing and that strategy. There. Thank thank you very much.

Thanks, Greg yet.

So you know obviously, we have to do a lot more work on the re dosing and validate our assumptions, but our perception of the evolution of.

And the T.N. 10 follow on data is that they may in fact, b two populations.

Maybe a population of a individuals who.

Have they will to reactive T cells neutralized.

And at some point and da da presentations also you know indicated this the exhausted T cells.

And that decline. These these are the cells that the will to reactive T cells are converted into the regulators phenotype.

With that decline or may be.

An indicator as to when Ace. He's a second we'll follow up dose could be administered in that that could be repeated in order to indefinitely delay.

The other population, though you know if it is those that.

Our sort of seven and eight years after.

The single course of therapy may be that there are patients who are essentially tolerate rise by a single dose and either the rate of development that disease will be impacted completely man or on their well to reactive T cells is.

Much more sustainable they may never need another dose a tool and remain disease.

These free will 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 you'll be expert in exhausted T cells and.

The analysis the VA later.

Yes, Indeed, just gonna see said than new they that it needs. This year showed that.

Any subject, who had and increasing access to T cells above 10% of their total T cell number.

Did not progress.

Two clinical to Wendy.

In the close of the study and now we had accumulated and have a lengthy follow on follow up so I think the hypothesis is coming up.

We can use these cells to guys read those in some subjects may not me today those.

Well they send it to here sometime or perhaps never.

That's great. Thanks for taking my question.

Thanks, Greg.

Our next question comes from Lithia Young of Cantor. Please state your question.

Hey, guys Gary.

Finally, see a good morning, Jeremy Okay. Okay. So sorry about that so two questions for me one congrats on like the commercial again to the commercials guy kind of one it just.

Is that kind of the big step and you know from here. We're kinda you know somewhat down year on another part of the processes easy and filing but I just wanted to kind of get your favorite on that seem to be a big hurdle on that need to be completed and then the second part which may have gotten asked already but I was down back in I guess I was just trying to wonder where you know.

[noise] [noise].

I think we lost the lithium Ashley you want to take the first part of her question.

Yeah I wasn't sure if it was me [laughter] dropped or leave you know sometimes I.

Especially since a lot of manufacturing is.

Proprietary knowhow and so you have these milestones.

First and.

Engineering ran says G. M. P ran the P P Q ones.

And they are notorious for complications.

And.

Having transferred our process at commercial scale to run three back to back ones.

Without significant issue and for our internal team to feel really good about those P. P. Q runs accomplishing their objectives.

Is yes, I believe a very significant accomplishment that takes a lot of the risk.

Out of the remaining C. M. C module that we have to file we have to do the remaining P. P. Q work on the feeling in the finishing because you can appreciate that.

Is much more is much more for much more straightforward and routine than the complex.

Some some mentation or cellular production of Ah monoclonal antibody with.

Lots of.

Factors that can impact the outcome and.

Undermined the success of the runs back part of our manufacturing.

Is behind Us and now it's about collecting the data from them with samples and submitting that alongside.

Additional data, including P. P Q runs on the field.

Finish in that and putting the final product up on on stability and we're really really pleased with the outcome from accomplishing the three P. P. T runs with drug substance.

And Miss Young your line is open. Please you can go a shake.

Oh can you hear me my is that good question was just to my how do we think about the biology potential Super Responder Sarcoplasm AB.

Thankfully So Francisco do you wanted to talk about.

Super responders.

[noise], yes, so we know that exhausted T cells correlate with <unk>.

No progression too clinical T O N D.

In the natural causes the disease.

That has been Stablish sent published before.

The place I'm not even is introduced.

<unk>.

Or the other side of the coin of the ultra reactive T cells.

So what we're saying now is that when the person that is given to.

Patience with P symptomatic D C S.

Those who achieved great to levels of the sauce.

Did not seem to progress and some of those subjects have been followed for eight years is actually mentioned.

So the possibility of marriages that they are.

Long term thaler iced.

That they're immune system.

Is now able to continuously prevent the ultra reactivity against the beta cells.

If you think about it.

Similar situation in Indiana oncology.

When you look at it kept on Meyer curves.

Before Indiana oncology, they kept on my curse, whereas shifted slightly to the right.

Bye chemotherapy radiotherapy.

But with them in oncology did supposed to ship those.

Serves up.

Meaning some subjects never.

Progressed.

And that's what we're starting to see with <unk>.

And a couple of my curse of survival.

That's not just shift to the right, but also shifts up.

And so you can see in the 2020 updates.

It has become a flatline that most forward.

Into the future without.

Going down to the conversion.

Clinical T. Wendy so it's a it's a very interesting possibility that's hassles song.

Strong biological a fundamental.

Great. Thank you.

Pennsylvania.

Our next question comes from Justin Kim Oppenheimer. Please state your question.

Yeah, Hi.

Congrats on progress honestly Jackie M for Justin.

First I guess.

Listen maps first approval qualify for a pediatric truck vulture.

So.

A pediatric drug.

Voucher I believe requires often status and we've obtained often status for newly diagnosed.

And are working on.

Often for at risk, but we've not.

Not made an announcement that that has been accomplished yet.

Thanks for that.

Maybe it was where Jason on a commercial from you know from market perspective, how accessible from a patient at 10, a vacation perspective, that's the group of familiar stays one patient appear and how important well labeling.

B in trading this patient thanks.

Yeah, So I think nothing's changed with respect to how we view the address of all market for the initial at risk population right. So we see this initial familial direct relative market focus at launch of approximately 30000 patient and.

You know as I mentioned and and are prepared remarks will 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 one diabetes.

And why they should go and check out screening knowing that screening is commercially available to date I would say that you know than it does you know 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'll be a prior authorization to label is.

I mentioned from the one on one engagements we've had repairs as well is there the advisory board that we did it may.

Oh, that's very helpful. On my last question is Florida, Coxsackie Laxson program. Okay, I'll talk about what pieces of evidence gave the team coffee does that you know Italian <unk> now induce all wanted also remarried T X.

Thanks, a lot Francisco that's definitely ears.

Yeah. Thanks for the question Justin.

So we have multiple multiple.

Safeguards for the works.

Oh.

It is in inactivated.

Vaccine, so that viruses that cannot insect.

[noise] Pancras, that's the first thing.

Second.

There have been some epitopes proposed for potential mimicry mimicry has never been proven N T O M D.

To be the cost for two on the outside of soldiers infection, but just to be safe those epitopes or not included in the vaccine and or they are included in other vaccines <unk>.

Such as for example, the polio vaccine and the polio vaccine polio being and enterovirus as well.

[noise] given to children with high risk of two on the end they did not.

Have any interest in two on this so.

We feel quite confident that the vaccine.

Is devoid of this kind of theoretical side effects, but of course, we will.

[noise] assess that in the first thing human study.

Alright, great. Thanks again for the office. Thank you.

Thanks, a lot.

[noise]. Our next question comes from <unk> <unk> of H C. Wainwright. Please state your question.

Good morning.

Good morning. This this month speaking on behalf of them sound raunchy. Thanks for taking my question. So I had a couple of pipeline question. Firstly, and then some queries about marketing and punished ships. 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.

Your patient as being lost.

Follow up since the previous day to release.

We don't anticipate.

Although it's not a data so it's hard to tell but we don't anticipate but that would be any additional updates.

Before the end of the year. It appears that they're on an annual cannot calendar schedule and the C. D. A D. A L. A M you for that.

It's entirely possible that the date that was presented in presentation format H E. A D I.

Is subject.

For a for a potential publication, but again.

Data belongs to trial net.

And and sponsored by N I D D K and we don't control the academic publication side of the equation.

We we do have though following initial publication the.

The rights for the purposes of regulatory submission.

Okay.

M G four see any scope for other auto immune indications for <unk>, a beyond type one diabetes.

We do and we obviously a focused on getting a.

Breakthrough therapy designation for T. One D. You know cross the finish line for us.

We're also in parallel focusing on the possibility of a subcutaneous formulation, which we feel could facilitate expansion outside of type one diabetes and obviously be.

M. A an improvement in terms of therapeutic burden Ah in.

M T won't be itself, although a single cause.

Of.

Two two weeks.

Therapy versus.

A lifetime of.

Of regular Immunosuppressants.

You know <unk>, which is [noise].

Sort of how many auto immune diseases are manage these days is beam to be vastly superior from a therapeutic burden perspective, even though we're administering ivy.

Francisco do you want to just give ah flavor of some of the indications that we could pursue with type Lizzie mab in that context.

[noise], so it will be T cell driven out immune disorders.

Processes Celiac <unk>.

You know hepatitis Psoriatic arthritis, where we already have data it's published.

Rheumatoid arthritis, lupus et cetera, it's it's a wide range of Algerian an inflammatory disorders, the doctors them by T cells.

Fantastic. Thanks for the granularity on that I'm, just a couple of marketing and punish of questions. If I may [noise].

Is the marketing authorization application for a couple of do ma'am for T. One D in Europe still on track for submission.

Ah you're seeing significant partnership interest from X U S countries.

Yes, the E M. A process is on track we're in the stage of.

Having scientific advice meetings scheduled and taking place in Europe.

Continues we anticipated that we're approximately 12 months behind.

The schedule for the United States.

And we have indicated that we will not be.

Tempting to launch to please the map directly outside of the United States and that there are a number of.

Scenarios that can help insure we have the market infrastructure in partnership.

In Europe.

Two two to facilitate commercialization, but we have not made an announcement on the progress of those discussions.

[noise] [noise]. Our next question comes from David Huang S. M. B C. Please state your question.

Hi, guys. Good morning, and thanks for taking my questions. So I had a few so first do you I guess what are your expectations in regards to potentially having an F D a add.

And then could you provide a little bit more color on the patients the address patients under.

Under eight years of age you know what percentage of at risk patients does that represented how about you reach those patients eventually.

Yes. Thanks, So we have not have discussions with the F. D. A regarding an outcome, but as you would imagine.

You know a season.

Management team expects will tries to expect everything and so we have so we are certainly preparing.

And those preparations are.

Alright, actually I'm going in parallel with the the regulatory filing an submissions and you'll be well prepared.

<unk>.

<unk> event, assuming but it happens <unk>.

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 whilst.

The diagnosis.

Of.

Clinical stage T. One D peeks around the ages of 10 to 12.

The appearance of water antibodies.

Clearly.

P as.

<unk> much earlier than that and.

In some instances instances.

As early as two years of age.

We have a pediatric.

Plan, which with some.

<unk> submitted to the F D, a and and will be part of the discussions you know with respect to.

I'm going submission and review and we're committed to to ensuring that we collect data as quickly as possible.

Post.

<unk> post anticipated launch in order to be able to [noise].

Reassure.

The agency inclinations and parents that administering Pep Lizzie ma'am below eight years of age.

It is safe and effective.

[noise], Okay, great. Thanks that that was really helpful. And then I just had one other question and this is in regards to you know how the 14th week course of type lives about it might work in and clinical practice.

Is there any concerned that you know some patient, Maine Miss some of the doses and you know is that something they need to come into the infusion censor or everyday or there are alternatives into in terms of how they can <unk> receive their doses.

So if I may break that question down into two one the scientific side and ask Francisco.

The potential impact of a miss or a delay in.

Any one of those daily 30 minute infusions.

And once he has the address the medical or scientific concerned.

Ask Justin to speak to.

The the commercial leadership teams' strategy with respect to infusions and making infusions.

As as convenient.

Possible for clinicians and patient and and essentially making sure that we provide the facility for them.

To administer the drug wherever their preferences, but first francisco, what if a patient Mrs. A day.

Yes, Thank you David and just to clarify it's it's fucking.

<unk> not not 14 weeks of us to put some.

And we do have data already and face to.

This was explored we know that.

<unk> six days a therapy has an effect, but the fact is not S.

Robust S. It's off or 14 days, that's so weird now using 12 days an hour nearly diagnose program.

So based on our pharmacokinetic Pharmacodynamic analysis.

Leaves patients knit together at least 10 to 12 days of therapy. So if they missed a day they miss two days.

We'll ask them to complete the cause we will try to get in those 14 days, but it will not be an absolute Los altos responsiveness to the drug.

[noise] Yeah. David This is <unk> I'll take the second part of your question, which you know in the commercial context I think the way we're preparing for all eventuality as in terms of.

Product acquisition and site of administration, and so I think there are three potential scenarios right to the person area of the.

[noise] patient gets abused him the infusion center and will provide you know.

Billing encoding guy 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 a specialty pharmacies that have that capability in all 50 States and then the third eventuality is is that they would starting an infusion center under.

<unk> and once the physician in the family feel comfortable with how the infusions are going they transfer from the infusion center to home infusion and we will make sure that we have.

All three of those scenarios cupboard, such that the decision can be made by the health care provider the family and the caregiver as to what works best for them and make sure that we have Ah you know a clear route for product acquisition, an infusion or for all three scenarios.

So we were on the hour and I really appreciate everybody's questions fantastic questions, but I'm gonna. Thank you for joining us today.

And let you know that we look forward to updating you soon on a continued progress when we sincerely hope that you and your loved ones say safe and well throughout the summer. Thank you [noise].

[noise]. Thank you. This does conclude today's teleconference. We thank you for your participation you may disconnect. Your lines at this time and have a great day.

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