Q4 2020 Provention Bio Inc Earnings Call
Good morning, My name is Elisa and I will be your conference operator today at this time I would like to welcome everyone to the prevention bio call. There will be a question and answer session to follow please be advised that this call is being recorded at the company's request.
I would now like to turn the call over to Mr. Robert Doody, Vice President of Investor Relations for prevention of bio.
Thank you operator, and thank you all for joining us on prevention Bio's fourth quarter and full year 2020 financial results Conference call.
Joining todays call from the prevention of bio team as Ashleigh Palmer, Chief Executive Officer and co founder.
And Andy Drechsler, Chief Financial Officer.
Francisco, Leon Chief Scientific Officer, and Jason <unk>, our Chief commercial officer.
Before we begin let me remind you that the various remarks, we will make today constitute forward looking statements.
These include statements about our future expectations.
Clinical results developments and regulatory matters and timelines, including for the the Parisien of <unk> B L. A.
The potential safety efficacy and commercial success of <unk> and our other product candidates.
The potential COVID-19 impact on our clinical studies and business plans.
The financial projections, including our anticipated use of cash and our cash runway.
And our business plans and prospects, including plan pre commercial activities across the company in preparation for the potential approval of the pledge of map and projected timing for the state.
Actual results may differ materially from those indicated by these forward looking statements as.
As a result of various important factors.
<unk> those discussed in the risk factors section of our most recent annual report on form 10-K, which we filed with the SEC. This morning.
The 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.
Specifically disclaim any obligations to do so even if our views change except as required by law.
Therefore, you should not rely on these forward looking statements as representing our views.
Of any date subsequent to today.
There is more complete information regarding forward looking statements risks and uncertainties in the reports prevention filed with the SEC.
These documents are available on prevention web site at Www prevention bio dot com under the investors section we.
We encourage you to review these documents carefully.
With that I'll now turn the call over to Ashley.
Thank you Bob and good morning to all of you joining us today.
To begin with I would like to state that our team here at prevention made tremendous progress last year.
Both the and advancing our efforts to bring to please the map to market for the delay for prevention of clinical type one diabetes.
Risk individuals.
As well as execution across our entire pipeline of clinical stage immuno.
Immunology drug can be day.
And this progress was made despite the enormous logistical and operational challenges presented by the global COVID-19 pandemic the dim.
<unk> at the beginning of 2020, and which still remains today.
The momentum we accelerated throughout 2020 continues to be driven for wood into 2021.
As we announced at the beginning of last month, the Fda's filing of a biologics license application for it to please the mapping all of lead type one diabetes.
Indication.
And this BLA.
The undergoing priority review by the agency with the producer date of July 2nd 2021.
I will go into more detail Lizzie maps near term execution and anticipated commercialization targeting the at risk population in the moment.
However, before doing so I would like to highlight some of the progress being made across our other business priorities.
One of the significant majority of our focus this year will remain on the risk BLA regulatory review and if all goes to plan of potential U S launch in the third quarter.
We also intend to place more emphasis on the steps being taken to expand our breakthrough type lithium the franchise.
As well as continue to advance our other pioneering pipeline opportunities targeting serious autoimmune disease prevention and intersection.
Beginning with potential label expansion opportunities for calculating the map.
Protect study targeting the recruitment of 300 newly diagnosed insulin dependent type one diabetics, aged eight to 17 within six weeks of better initial diagnosis.
Continuous to enroll despite COVID-19 challenge.
We anticipate enrollment completion for the study in the second half of this year.
In this study newly diagnosed <unk> patients receive 212 day courses of active or placebo therapy administered six months of ponds.
The goal of the study is to see of 40% or greater difference in C peptide levels of 18 months.
The difference in C peptide levels is a measure of the preservation of of patients insulin producing beta cells.
The primary endpoint is a couple of in our study with clinically relevant secondary endpoints, such as insulin usage hypoglycemic events H B <unk> C and.
Time in range using continuous blue blood glucose monitoring.
Our expectation is that we will be in a position to report the topline results from this study in mid 2023.
In addition to protect we continue to work on the other important initiatives to support the development of Pep Lizzie might incur.
Including demonstrating safety in pediatric patients below the age of eight years.
Re dosing in the at risk population.
And the evaluating at least the knobs combination with other therapies.
As well as formulation improvements to enhance patient compliance and potentially facilitate the therapies development for autoimmune disorders of the type one diabetes.
In addition to tap Lizzie map as it pertains to our broader development efforts in the T. One of the autoimmunity space.
We also announced late last year the initiation of the first in human study of P. O V. One O one hour of Coxsackievirus B vaccine candidate.
As we've noticed noted in the past Coxsackievirus B infection is first and foremost directly responsible for certain of acute conditions I'm serious complication.
It is also a presumed trigger in the development of both type one diabetes and celiac disease.
Our phase one prevent study is of placebo controlled double blind randomized study in healthy adult volunteers.
We expect the results of the program study in the fourth quarter of this year.
Moving next to Peel off the 30 to 79.
You will recall that last month, we released the key findings from our preclinical proof of concept study for the prevention of Immunogenicity associated with gene therapy.
But those of you that follow the gene therapy space close.
It is well known the patients immune responses to viral vectors and transgene products remain a significant sellable in the delivery of these breakthrough therapies.
Current mitigation strategies to overcome these hurdles also presents significant safety challenges.
Through its mechanism of action inhibiting b cell activation without depleting b cells. We believe P. O. The 32 79 has the potential to be used as adjunctive therapy alongside a number of states of the gene therapy platform.
We plan to present the results of a preclinical proof of concept study at the medical meeting later this year and look forward to potentially collaborating on this program with relevant industry partners and key opinion leaders in the gene therapy field.
We were also very pleased to announce last week, our strategic collaboration with the wide Zone Medicine company.
The developed and commercialized P. All of the 30 to 79 in the greater China region.
In addition to cementing our relationship with an excellent partner for the development and potential commercialization of <unk> $32 79 in China.
Why don't collaboration contributes significantly to funding the P O. The 30 to 79 prevail program.
Progressing a phase Iia proof of concept trial for the prevention of relapse in lupus patients.
We currently expect to initiate this trial in the second half of this year.
We will provide you with more details in the coming months and upon study initiation.
We believe our partnership with far down provides further external validation of our pioneering business model focused on identifying acquiring or licensing developing and as appropriately commercialize in clinical stage assets that have.
The potential for intersecting or preventing serious debilitating and life threatening autoimmune diseases.
Lastly, but certainly not least with respect to our auto of immunology pipeline.
We have P. All of the <unk> five.
Our investigational anti IL 15 monoclonal antibody.
We are developing in collaboration with our partner Amgen.
Prior trials you know the 300 patients have demonstrated P O b of one five to be well tolerated and of.
Stablish of proof of mechanism and proof of concept for this human monoclonal antibody.
We initiated a phase two b trial of <unk> <unk> five for the treatment of non responsive celiac disease in the third quarter of last year.
And we expect topline data from this trial to be available in the second half of 'twenty 'twenty two.
Hence throughout 2020 and into the beginning of this year, we have made great strides advancing our pipeline of auto immunology therapeutic candidates and all other programs are now in active clinical development.
Returning now to type one diabetes or T. One day.
The serious chronic life impacting autoimmune disease with substantial unmet need for which current medical practice weights.
And waits until it's too late until patients finally presents the clinicians with symptoms of irreversible tissue damage and end stage organ failure.
Often in a state of life threatening metabolic crisis called diabetic ketoacidosis.
Following stabilization, usually requiring hospitalization and time in an intensive care unit.
Patients are then referred to an endocrinologist for a lifetime of glucose monitoring and insulin therapy with all of it the accompanying risks and complications.
The lives of these patients and their loved ones will never be the same again.
And this is why it is so important to now look ahead at the IR activities in preparation for the potential launch of <unk> took place the knowhow in the risk T. One day patient population.
As a reminder, the FDA previously granted Pep lithium a breakthrough therapy designation.
And as I noted at the average.
As of this call our priority review produce the date is July 2nd of this year.
We have also announced that the FDA is planning to conduct an advisory Committee meeting.
Tentatively scheduled for May 27.
As discussed in previous calls we have been expecting from the outset. The the agency would likely make use of an advisory Committee meeting.
Given the pioneering nature of the police the amount of potentially.
Potentially the first disease modifying therapy in the type one diabetes treatment space.
Since insulin was introduced a century ago.
Our team is currently preparing for the upcoming Advisory Committee meeting.
And we will continue doing so throughout the coming months.
Before progressing further.
We'd like to address a couple of regulatory disclosures made earlier this year.
The first being whether the FDA will require T. One of the auto antibody tests to be considered companion diagnostics for capitalism mab in the at risk population.
Our belief, which we have shared with the FDA is the auto antibody testing is not the definitive diagnostic tool by which clinicians will ultimately determine if at risk patients should be prescribed please the mab therapy.
What we can share with you today as a result of our ongoing open and constructive dialogue with the FDA.
Is that one of the agencies still considering the smack that.
It remains possible that some companion diagnostic bridging maybe required as a post marketing commitment.
We do not currently believe it will be a gating factor towards a proven ability of the placebo.
The second regulatory consideration I would like to address the change to comparability between drug project previously produced from Eli Lilly drug substance.
And of that produced from our current manufacturing partner AGC biologics.
We believe our assessment of the physio chemical analyses of the two drug products, which we submitted to the FDA in our CMC module.
Demonstrates these drug products to be comparable.
This assessment is also supported by the comparability in PD parameters evaluated in the PK PD bridging study we conducted in healthy volunteers last year.
However, the single administration low dose study also showed a slightly lower than target PK area under the curve for the AGC profit.
Indicating that in that particular study the AGC product may have cleared the path from the bloodstream and the Lilly product.
Based on the understanding of the relevant data and the extensive modeling we've conducted to date, we do not believe this observation will have a clinically relevant impact on either of the safety or efficacy of the AGC product.
As many of you know we had a BLA mid cycle review meeting earlier this month and.
And we had an opportunity to discuss this topic for the first time with the FDA and share our point of view regarding the interpretation of the data we have submitted.
The FDA is still evaluating the PK PD bridging study and we will be conducting its own PK modeling to validate our conclusions.
As a result of our breakthrough therapy designation for the at risk indication.
We continue to enjoy the benefit of frequent constructive and valuable dialogue with the agency on all aspects of our BLA filing and the preparations for 100 of Advisory Committee meeting in May.
Considering the type of lithium that potentially represents the first disease modifying therapeutic advance for T. One day in over a century and given the substantial unmet need that remains for these patients and their families. We look forward to continuing to support the agency.
In its review of our BLA to be able to bring this innovative breakthrough therapy to patients later this year.
And with this in mind I'm now going to turn the call over to Jason Hoyt, Our Chief commercial officer.
To discuss the continued advancement of our commercialization planning and execution of prelaunch activities.
Of the Fda's review of proceeds.
Jason.
Thanks, Ashley and good morning, everyone.
Over the course of 2020, we've made significant progress in building our commercial capabilities as an organization.
As I've discussed in previous calls we've built our core commercial infrastructure with amazing talent from across the biotech industry and I could not be more proud of all of that this team has accomplished in a relatively short period of time.
In addition to the infrastructure advances in talent acquisition, we substantially advanced our launch planning and launch readiness companywide.
Through market research, including over 900 participants from key stakeholder groups, including pediatric and adult endocrinologists pediatricians certified diabetes educators mid level providers patients caregivers and at risk individuals we have of far deeper understanding of the market than ever before.
The insights gleaned from this comprehensive market research have informed our launch planning efforts the design of our patient services program, our distribution model and our go to market strategy. The early learnings from this extensive market research also led to the development and deployment of our two national disease awareness campaigns connected by tier one day.
And type one tested laying the groundwork for helping patients families and caregivers increase their understanding of T. One day and they are a disproportionate risk of being in the early stages of the disease as well as the importance of tier one day screening and available testing options.
As we announced in early December we're also proud to have been the founding sponsors of JD, our apps T. One detect screening initiative.
This groundbreaking program is the first of its kind of into your one day and the only screening program that offers the option for in home testing for tier one day auto antibodies through dry blood spot technology.
In addition to increasing patient and healthcare provider awareness. We've also continued to advance our discussions with payers.
You May recall, we first began our payer engagement last may with an AD board that included medical directors, representing approximately 73 million covered lives.
Since that time, we've conducted numerous individual meetings with payers that cover in excess of 70 million lives. These payors are from across our anticipated payer mix, which from a claims database analysis, we anticipate to be approximately 60% commercial 35 per cent Medicaid and five per cent other.
I'm pleased to share of that the feedback from those meetings has generally been both consistent and positive.
And we also can report that we've seen a further increase in requests for a deeper dive into the day Pleasant map data from our medical affairs team since the BLA was filed and if they do for date assigned.
Beyond our payer engagement, we finalized our distribution model of plans and anticipate a limited network of specialty pharmacies that have home infusion capabilities in all 50 states along with the single specialty distributor.
Finally, we've taken a purposeful approach to our patient services design and are in the process of finalizing it based on our patient journey work and identification of not only the potential pain points in the process, but also at the moment that we believe matter to patients.
We've taken a differentiated approach to the validation of this program design by hosting two separate co creation sessions, one with health care providers and their staff adult and pediatric endocrinologists nurse practitioners and physician assistance and office managers and the second with patients and caregivers we.
We believe this approach will help us create a final program that we plan to offer at the time of of potential launch that will be meaningful and address the needs and concerns of the patients that we are here to serve.
This brief overview of provides a very high level look at some of the initiatives that are going into our preparations for a potential launch but are in no way exhaustive I look forward to updating you on future calls as we prepare to hopefully bring this important medicine to the patients the need it later this year.
With that update I'll now turn the call over to Andy to discuss our financials for the period.
The <unk>.
Thanks, Jason and good morning, everyone.
Before I begin I would encourage you to read our 10-K that was filed today.
The 10-K includes our financial statements.
The factors as well as management's discussion and analysis of our financial condition.
I would also like to call your attention to the earnings press release, which was issued prior to this call.
Let me start with our current cash position and cash projection.
As of December 31, 2020, our cash position was $121 8 million.
We recently completed a follow on offering which generated approximately $102 3 million of net proceeds including a partial exercise of the underwriters option to purchase additional shares.
We expect our current cash cash equivalents and marketable securities will be sufficient to fund projected operating requirements for at least the next 12 months, which will enable us to actively develop all four of our programs. The plays the map for type one diabetes P. R V $32 79 for lupus.
T. R V zero of one five for Celiac and P. R V. One O one of Coxsackie virus vaccine.
Our net cash used in operations for the fourth quarter ended December 31, 2020 was $24 6 million.
We expect to use between 26 and $30 million of cash for operations in the first quarter of 2021.
We plan to provide additional cash guidance on each quarterly call as we continue to progress towards the potential regulatory approval and commercial launch at the Pleasant Mab.
From a P&L perspective, we generated a net loss for the fourth quarter 2020 of $32 6 million or 58 cents per basic and diluted share.
The increase in net loss compared to the fourth quarter of 2019 is attributable to increases in research and development expenses of $12 1 million as well as an increase in general and administrative costs of $9 6 million.
The net loss for the fourth quarter included $8 million of noncash stock based compensation of several vesting milestones related to the BLA for <unk> Pleasant NAV were achieved.
Net loss for the full year of 2020 was $98 6 million.
For $1 88 per basic and diluted share compared to a net loss of $43 3 million for $1.06 per basic and diluted share for the full year of 2019.
The net loss for 2020 included $13 2 million of noncash stock based compensation expense, while the net loss for 2019 included $2 8 million of noncash stock based compensation expense.
The increase of net loss in 2020 as compared to 2019 was attributable to the pleasant Mab related CMC costs.
<unk> phase III study costs.
DLA preparation costs.
The commercial costs and medical affairs expenses.
With that let me turn the call over to Ashley for closing comments Ashley.
Ashley.
Thanks, Andy.
So as you can see 2020 represented a year of exceptional progress for our company and for our mission to fundamentally address the substantial unmet needs associated with serious debilitating and life threatening autoimmune diseases.
That progress has translated into an ambitious 2021 for prevention bio which is off to a really good start.
We look forward to keeping you updated on our continued progress throughout the year.
And to that end I'm also pleased to announce that we are planning to conduct an analyst and Investor day in April to provide you with both a deeper dive into our tech lithium ion program and the T. One of the franchise expansion opportunities as.
As well as shine a spotlight on the other clinical stage auto immunology product candidates in our pipeline.
Aimed at the intersecting serious life, threatening and life impacting conditions.
The details of this event will be sent out soon.
And with that operator, we'd like to take any questions.
We will now begin the question and answer session.
Ask the question you May Press Star then one on your Touchtone phone. If you are using a speakerphone. Please pick up your handset before pressing the keys to it.
All of your question. Please press Star then two at this time, we will pause momentarily to assemble our roster.
Yeah.
The first question today comes from Alethia Young with Cantor. Please go ahead.
Hey, guys. Thanks for taking my question and congrats for the progress maybe two one just can you talk since you have of after the dialog with the FDA can you just kind of help us think about you know some of the things that would be of consideration that the panel I think you you referenced the car for a couple in the in the.
The prepared remarks, but I'm, just kind of dig a little bit more ends of that then maybe the second question is for Jason I know Youre not you know for fair to think about price with some granularity but.
I think you can I'm just curious if you kind of half of perspective on the.
So kind of now that you were saying the median be much longer you know kind of how to think about maximizing that price and what the feedback has been with payers. Thanks.
Yeah.
Thank you very much easier for that for the question. So.
We've always anticipated.
There would be an AD com just by the nature of the this is such a fundamental paradigm shift in how.
The type one diabetes.
Is is being considered not only with respect to of disease modifying therapy.
With respect to identifying patients at the pre symptomatic stage and the administering.
A therapeutic and into intervention.
So I think you know the agency is going to.
Welcome an opportunity to discuss that with key opinion leaders with patients the patient advocacy.
Groups in order to better understand and appreciate the.
The unmet need and the context of their review I also think then that as is typical in an advisory committee they.
They will be seeking guidance from experts in the field with respect to the risk benefit.
<unk> ship.
And we're confident.
And of the results and the data that they will speak for themselves and believe that when you consider the now three medium delay in onset and the fact that we're dealing here with.
A therapeutic the resets the immune system and of discrete 14 day dosing.
Dosing regimen, we're not dealing with all of a therapeutic.
Therapeutics that are chronically suppresses the immune system.
And so that's.
A very good.
The safety profile to be considering.
In the in an intersection context, so we will be prepared for you know.
Experts on the F D. A digging deep into the details of not only the P. M 10 study, but the over 800 patient database that we now have from from previous studies of newly diagnosed looking at safety.
And the you know the.
Consistent signal with respect to C. Peptide is evidence of preserving functional beta cell mass.
Jason could you. Please address the leases a question regarding pricing.
Yeah, absolutely and the hi, Alicia Thanks for the question. So you know with respect to our payer engagement.
Our market access team has really done a phenomenal job over the course of 2020 of ramping up our understanding of how payers are thinking about type one diabetes educating payers around type one diabetes, because I think one thing that I think is reassuring for US is the consistency of the feedback that we're hearing from payers debt as I mentioned in our prepared remarks.
<unk> began with the advisory board that we've discussed previously in May of last year, which was before the Ada meeting in June where the the TN 10, three year data was announced which obviously.
Is more favorable than a two year delay and so the consistency between what we heard in that advisory board and what we heard from the the direct Payor engagement I think is reassuring with respect to how they anticipate covering of drug like the pleasant map how appreciative. They are of of an enhanced understanding of the autoimmune nature of type one.
Diabetes, and the unmet need the daclizumab can ultimately address in the type one space, so going into pricing research, which we just kicked off I think we're really encouraged with how physicians are now both thinking about type one day or how payers are now thinking about both type one diabetes and how they're thinking about the profile of the pleasant.
GAAP, but again, we just initiated that pricing research within the last couple of weeks and it'll be a.
The process that'll that'll go on for the next couple of months as we engage with both payers and HCP as a part of that process to ultimately come to a final decision around where we're going to price to play the map, which we would anticipate announcing at the time of our commercial launch.
The maybe a follow up on the like how do they think about like the cost of insulin and all of that what kind of forgone opportunity caused the you know.
Like what what's the kind of the financial value to them of the delaying of year of you know of lifetime of insulin.
Yeah, It's a great question of at the end.
A big part of the conversation that we're having with payers is to frame the discussion around the totality of the cost of type one diabetes right. So it's not just the cost of insulin for a year or two it's not just the cost of the pumps and the supplies in the CGM. It's the cost of you know things like 50% of patients presenting in day.
Diabetic ketoacidosis, we know that of single hospital admission for diabetic ketoacidosis can cost anywhere from 18 to $27000 those direct health care costs that arent necessarily associated with a therapeutic like insulin or you know of pump that goes along with insulin.
Need to be factored into the equation and frankly, it really isn't on the top of mind of payers right now because when they think about their diabetes budget, 90% of that budget is on type two diabetes. So having that conversation helps them think more holistically around the totality of the cost associated with type one so that we are avoiding them thing.
Looking about this disease just in the context of insulin pricing because that would be a mistake.
Great. Thank you.
The next question is from Gregory of I'm done with RBC capital markets for.
Hi.
Yes.
Thanks, Hi, Ashley and team congratulations on the on the progress Thanks for taking my question.
Actually I just wanted to follow up of a.
Bid on those of FDA interactions to date, and just didnt, regardless of the color on on the mid cycle review of which I totally appreciate just curious if you could just layer in perhaps your expectations.
Around the comparability of timelines that the FDA is engaging into to do their own work there and what what elements of you used to sort of arrived at some of your positioning about its current comparability. Thank you.
Thanks, Greg Good morning, so yes, so so what we're dealing with in terms of the comparability is a very comprehensive panel.
Of Physiochemical analyses the release the product from the manufacturing site within the specification and from.
From a validated process now at AGC biologics and comparing that.
To the specifications.
With regard to the Lilly substance manufactured the decade ago.
And.
In that context, the products are comparable.
That is that is our assessment that is our belief and we believe that the agency will we'll.
We'll see that.
So.
We then had the situation as.
As you May recall, where we started the protect study with.
Lilly manufactured product.
And obviously half two.
The transition to the AGC biologics product.
And we did a single.
And a single dose.
Study in healthy volunteers at the low dose because we obviously couldn't administer a full 14 day therapeutic dose to healthy individuals and we wanted to bridge the.
To the the the new material in our protect study.
As a result of that single dose PK.
P D. The bridging study.
Again.
All of the parameters.
We're within.
Anticipated target.
Especially the the the PD parameters, which are.
You know more indicative of.
The the efficacy and the safety with the exception of this AUC peaks.
P K area under the curve.
And.
That the AGC biologics fell slightly.
Below the target, indicating very clear the little faster.
So what we have done in evaluating internally and in the submissions we've made for the agency.
He is very extensive modeling.
Which is very.
The typical you know in in the in the industry to show what the.
Consequences of that would be how.
How the two products behave when you take into account for 14 days at therapeutic dose and from that.
Analysis from that modeling, we do not believe that the diff.
The difference in the area under the curve.
The result in a clinically relevant.
Difference in the safety of the efficacy of.
All of catalyst demand.
So that we've submitted to the agency the mid cycle review meeting we had was the very first and only time.
But we've had.
To discuss that we laid out all of them.
The results our interpretation.
And.
Obviously.
They they they they were not going to make.
It makes the decision on that.
The meeting and they have indicated to us that we will do their own modeling and we anticipate that they will make information requests in the coming week AR in the coming weeks are in.
In order to enable them to do a comparison between the modeling of our modeling.
And whether they arrive at the same conclusion that we do and we are confident in the interpretation that the that we have.
Submit it to them.
Got it that's helpful. Thank you maybe just just one more on the dimension of the potential.
Thoughts around the companion diagnostics and any post marketing interrogation of potential I'm just curious maybe for Jason how are you.
All of that May shape the.
The the engagement with the weight with patients and physicians.
And as far as that patient journey could be concerned as you'd think more conceptually about what that could look like.
Billing of that necessarily the thank you very much.
So I think the the primary consideration there is one of the.
For the regulatory pathway and.
The first thing.
We you know Hum.
Have sort of brief the agency arm is the <unk>.
You don't do a test of that too.
The patient has to auto antibodies.
And Ah clinician administered a placebo.
It's just the first part of the screening the screening.
The indication that the patient has two of two antibodies.
Should refer that patient to an endocrinologist for a for work up that.
For work up we will obviously include.
The confirmation of the author of antibody status and then the disclosed senior component, which will show that the patients.
<unk> to manage blood glucose levels, when the stress part of glucose tolerance test.
Indicates there of stage two patients and so for those reasons are the the.
What are the antibody tests themselves are not the definitive determinant as to whether a patient will get.
Uh-huh Pep lithium apps and for that reason, we don't believe that the companion diagnostic tsunami of appliance here if the agency.
The difference in that opinion.
They've indicated that this will.
The of post marketing.
The exercise to really look at the tests that were historically done in the P. M 10 study and bridge them to the tests that are available today with respect to.
The two towards the antibody testing and we're obviously preparing.
You know to be able to do that.
But vary.
Uh huh.
Very much comfortable but it is the post marketing.
The bridging type of of of exercise of commitment and not.
A hurdle to the approve ability itself.
Okay.
Great makes sense. Thank you very much actually.
The next question comes from Thomas Smith with SBB Leerink. Please go ahead.
Great. Thank you very much this is don the Queen sitting in for Tom.
Congratulations guys on the on the BLA filing just a couple of follow up questions to greg's questions.
Kind of regarding the companion diagnostic in a way.
Do you see any pathway to kind of getting approval for all to use the push the mab in patients as soon as they present with two or more auto antibodies and maybe if you could just remind us on what the natural history says on weighted patients present with the auto antibodies and how long. It takes for total generally to start showing of it. It's just a question on whether you can intervene.
At that point as opposed to waiting for.
The reach for stage two to Wendy.
And then the.
The second question of rounds the.
The PK PD work in and the agency products, just kind of what do you anticipate.
Corporate and this product into the protect study and are you guys playing on the reserving any of the Eli Lilly product for the second one of the dosing for patients that have already entered the study or are you just going to go ahead and dose them with the agency product for the second round of of dosing. Thank you.
Yeah, So let me.
The first question over to Francisco, but take the second question quickly so.
We don't anticipate the.
This is about giving the second dose.
Oh, the two dose regimen.
Using the the AGC product. This this is about enrolling patients for the first time on the.
The AGC product and so that they would have been we would then have them with debt first and second dose and that would keep it nice and clean. We can you know if necessary do sub analysis of those patients for us is the patients that were given.
The the the Lilly derived material again, we would not be introducing.
A.
The change of transitioning if we didn't believe that the therapy the.
It was comparable and again, we're not saying that the material itself isn't comparable it is what we're saying is that we did of single low dose study and there was.
A deviation from the target AUC profile and when its muddled up two therapeutic dosing.
It is.
We believe that the but that will not result in any clinical.
Well safety efficacy.
The issues and and so you know very often with biologics first and foremost there often.
Greatly overdose day anyway, there are changes in limits the amount as simple as small molecules.
And so this.
This is this is something that is regularly has to be done in the first by the agency as a result of modeling and comparability.
But we are very comfortable.
Transitioning to the new material in the protect study from Cisco with regards to.
The question on.
The.
The two what time of two bodies I'd like you to answer that because really.
It's two.
Towards the antibodies alone the stage one type one diabetes in stage two of these two what amount of people all of these plus disk glycemia.
So.
It's a it's a different indication.
And you would probably best to discuss.
What.
The label expansion.
Evaluation would have to be done to bring the use of pet. Please the mab.
Forward to the stage, one population versus the stage two Francisco.
Yeah. Thanks, Thanks for the question.
So when you have two or more auto antibodies.
You will eventually develop clinical T. One of the it just takes longer because you'd have to first go.
Through the displacing of stage the probability of.
The developing clinical T. One of the.
In stage, one two ultra antibodies is 44% that's five years.
Once you have this glycemia is 75%.
And that is why try on that enrolled these patients in the intent study it's easier to study them because of the faster conversion.
As Ashley mentioned.
The disease is the same it's the continuum of care when the the same mechanism of the sees the same T cells, destroying the beta cells. So we do have an expectation that the place of map.
Potentially help these patients as well, but with regards to studying them. They have to be mindful that this would be a longer study larger study and we feel we have better lifecycle opportunities in the near term of which we have indicated in our materials.
Expanding under age of eight years old Sidra combinations. So it is something that we keep an eye on them, but we don't have any immediate plans to undertake.
Great. Thank you and then one last question real quick for Jason.
Just in terms of of how large of the sales force you anticipate needing in order to the effect of the commercial rollout and what your timing is around.
Fruiting and educating the sales team.
Yes, Thanks, John it's a good.
Good question and I think our you know our original guidance that we're looking at more of the you know of rare disease type sales force is still hold somewhere in the neighborhood of 50 to 75 reps I think you know during the Investor and analyst day that we're planning in April we're going to give.
You know additional insights into our market preparedness go to market strategy and a lot more details around the commercial planning efforts that have been ongoing over the course of the last 15 months or so but somewhere in the neighborhood of 50 to 75.
Yeah I appreciate it thank you very much.
Yeah.
The next question is from Justin Kim with Oppenheimer and company. Please go ahead.
Hi, good morning, Thanks for taking the question maybe just the follow up just wondering how much of the build for that sort of potential commercial launch has already been done and.
How much building might we see out of the potential approval just wondering in terms of the timing of things and make it for modeling purposes as well.
Thanks.
So you know strategically.
Our approach to the scale up has been one of the abated.
Approach that sort of traction is in good alignment with the regulatory pathway and the derisking of that.
So I'll hand, you over to Jason now to give you some specifics.
As much as he can but.
Our intent.
Has not been to put boots on the ground until were.
Very comfortable that.
The the the approval of the launch of you know.
Is going to be on time for for obvious reasons.
But at the same time, Jason is very experienced with this and has done a great job with the core leadership team and all of the preparations necessary to do that in a very.
The effective and efficient manner of Jason.
Yeah, absolutely thanks, Justin.
Like Ashley said, you know, we're taking a very prudent and gated approach to the.
Of the spend associated with deploying the field team and I think in particular in the current situation that we're in with the the <unk>.
<unk>.
Monitoring what access is looking like the physicians as we think about deploying of field team. So as it stands right now we've got you know our <unk>.
Vice President of sales, Chris Farley is been on board since the third quarter of last year. He's done a great job in recruiting or first two regional sales directors that both bring a wealth of experience to to prevention bio and art.
Perfect example of the type of talent that we're looking for coming from both the rare disease in the type one diabetes space and collectively together the three of them have identified a wealth of talent from across the biotech space that you know are lined up and actively being interviewed them. So that when the time comes and we decided to pull the trigger.
We've got you know.
The the top talent from across both rare disease and type, one diabetes, and where the thinking that a significant proportion of those individuals having a direct connection to type one diabetes and having that the passion that goes along with you know the the.
The the potential launch of to pledge them out of and what that could mean for patients.
Got it great and maybe I just wanted to touch on the collaboration with Foregone, maybe could you maybe share more light on the Genesis of the deal and sort of the rationale for sort of having that partnership.
Currently and you know in terms of the timing and how you think about the development or the cost of development for the of our overall program.
Yeah, absolutely. So you know obviously, we have of business development function.
<unk>.
The 10.
The favor.
Focusing on R&D champions in the relationships with companies that for.
Fundamentally understand.
Our pioneering approach of technology and so on.
And we certainly keep an eye out for opportunities to partner with.
You know the the the industry leaders in regions and so.
So part one was just a wonderful opportunity for us.
Two to engage with the company debt.
It is a building in immunology franchise.
<unk> is excited about $32 79 of them is he is he's an excellent partner for that region.
And in the process is willing you know through the transaction compensation.
To put.
Some significant fund.
Sounding behind the the the study in lupus.
But we want to conduct and in doing so this enables us to continue the momentum of that program without competing for.
Funds.
From the of the checklist type of is.
The amount of them.
Our commercialization and subsequent label expansion opportunities. So it was it was a perfect a.
It really was the perfect match, we're very pleased and Francisco I believe it was your R&D championship.
And outreach to have done the initiated the the.
The the the.
<unk>.
Yes.
E M.
We look we don't want to take the glory as you say, we have a strong R&D and and the outreach to lead our business development conversations always absolutely coaches has to start from R&D and <unk> has a strong immunology presence and endocrinology presence.
You might have seen in our press release on February seven to 79 that we had some very nice combination of the subs with Cingal the Miss.
That's part of them is the number one share.
All of them with the company in China. For example, so it's not just the money they provide strategic.
Leverage for us to expand into this region.
Got it got it.
Great.
Just had one maybe just the final question on the Immunogenicity of gene therapy programs.
Is there a target indication that that you think would be a good fit to explore here just wondering when you think about utilizing an agent like.
The third piece have any night of and sort of potentially.
Addressing some of the issues of the Immunogenicity is it is it sort of programs that are experiencing safety issues currently or maybe not achieving sort of.
Adequate efficacy just wondering how you think about the types of programs that you would pursue it and where you think you might first.
Leverage of this agent in what setting.
Francisco.
Yeah.
Yeah, So you're right Justin it's primarily.
The.
For the programs that don't want to push the dose of the vector, which we know.
Ron since the safety issues. In addition to the cost of goods of course.
So the.
Combine them.
And then the associated vector or other types of sectors because at the end of the day the they're all of the immunogenic logically so combining that with pivotal at 279.
We believe would allow to use a lower dose.
And it would allow greater efficacy of the transfection of the tissues grades of expression of the transgene that sort of we put out in our press release. So there are benefits of the safety side the benefits on the efficacy side and also the the potential benefit of allowing read those.
<unk>.
Because of your block the Immunogenicity of the the first of course of NAV.
Of the gene therapy, so that.
The the potential here is quite broad.
And we don't think we are limited to a specific disease its for any systemic delivery of gene therapy product.
But for a vote.
So of any doubts of our.
Intent is to develop this agent for the mono therapy for autoimmune indications and then too.
To negotiate.
The collaborations with gene therapy companies that would need an adjunctive therapy like this or would want to explore it.
For them.
To fund.
Those developments were not going to go out do that speculatively.
Okay got it that's really helpful. Thanks.
The next question is from Ron some of that you H C. Wainwright. Please go ahead.
Thanks very much for taking my questions are just a few here could you comment on whether you expect.
The question around the treatment with the Pleasant Nab and the feasibility of studying the impact of recruitment of surplus and that could be a topic discussed at all at the upcoming Advisory Committee meeting for.
Or do you anticipate that this is not really going to be something that the commodity computers the mi.
I suspect that the.
Observation that what was achieved from the TM 10 study with a single.
And then.
We'll come up with the AD com.
And I think the there will likely be enthusiasm from the had come.
Encouraging us to two two of them immediately on the other side of the potential approval studies re dosing given the the potential too.
The extend the delay or indefinitely the like the progression for the disease I don't think it's a gating item to approve of ability. However, I think the.
I believe that the.
A single dose therapy and the delay of.
A median of free years is sufficient.
In the in and of itself.
Yes.
Do you have any thoughts at this juncture of regarding how the REIT treatment impact might in fact the studied.
For example, as you suggested in the post marketing setting.
Yeah Francisco I'm, probably it's best to speak to this because it can be by a number of of means we see the label evolving and.
We don't anticipate the the the label that.
It might be approved in the summer.
Would specify that you can only give one dose.
So in a lot of instances.
The the need to demonstrate the benefit from re dosing is going to be driven by advising clinicians.
On how and when two of them.
Two to consider giving us a second we'll follow up dose.
And the advising payors.
On the justification for doing so, but Francisco you have perhaps a number of.
The initiatives that can occur post marketing.
From our own sponsored studies two of them.
Of the means to provide that information.
Yeah.
Yes, Hi, Remi.
So we do have already experience with re dosing in the newly diagnosed setting as you know we have over 400 patients who were dosed for a second time.
Between six and 12 months after the first of course and this was done safely knowing no safety issues. So that's very important we know we can't read those saves me.
With regards to at risk, while we have not done it yet we do have an extension study, which is listed in clinical trials, the golf, where we would provide the another close to the patients who eventually convert to clinical team on the.
In the original T. M 10 studies that will give us some information.
And then as we mentioned we have an ongoing biomarker program in our current trials and protect.
That is helping us understand the best timing for re dosing, we believe the base.
Find the agenda with the agency, yeah, but we absolutely anticipate that the patient advocacy organizations of.
<unk> will be eager to contribute to the outcome and the and would welcome them doing so obviously.
Okay, and then just two very quick ones. Firstly, when you talked earlier about the differing pre-k profiled between the Lily generated material M. B a G C generated material in a situation where the F. D. A says to you that they're not sure whether the <unk>.
<unk> sources of material are in fact file of equivalent what might be some of the ways in which you can address that expediently.
So you know we will have those discussions with the agency it it it could range from evaluating the the the material used in the protests study too of post marketing commitment.
But we we we simply have not had <unk> dialogue with the agency I've reported the situation you know following the mid cycle review.
Transparently.
Okay, and then lastly, blood on mentioned I think of the press release. That's you know they anticipate 32 79 being part of the constellation of auto and auto immune disease inflammation disease focus therapeutics can you give us a sense of what they're.
The rest of their portfolio of looks like and what their experience. The track record has been so far in developing and potentially the marketing therapeutics that are in related disease areas for those that 32 79 with target.
Yeah. Thanks for the question I I think Francisco knows wondering best Francisco could you provide them with some color. Please.
Yes, <unk>. This is so public information and and for those website the.
They they are relatively large company. The 20 in China of 12000 employees 6000 sales reps and the strength is besides me in immunology endocrinology and oncology.
Uhm any and all of the <unk>.
They have sales in excess of of billions of dollars for for the excuse to prevent transplant rejection and tried some other out to me I'm just so the the.
They are drugs that having the on the market for awhile. So the partnership with prevention allows them to be more on the innovate the side of of development.
Thank you.
For the next question is from David Fine with S. M. B C. T go ahead.
Hi, guys. Congrats on all the great progress over the past year and thanks for taking my questions. So I just wanted to put in a couple of my first one just on in terms of free Lance screening efforts I know that J E. R. S has the program going on currently.
You know with the fingers day screening test and.
My question is do you have any goals in terms of of getting a certain number of pages of screen prior to to plasma of potentially coming to market. Uhm is there anything you know going on outside of the the J D. R. F in terms of.
Provide the screening tests to to patience and.
You know or is it something that you feel that once the products on the market, you'll get a nice ramp up in terms of the the interest and the screening efforts.
So we'll ask Jason for any specifics that he can give but you know strategically corporately. Our goal is to catalyze change and to use the potential approval of type Lizzie ma'am to bring.
The awareness to to the ecosystem to the community. The narrow screening is meaningful it has the consequence beyond the the primary drive it to this point, which has been to be able to anticipate the possibility of D. K, a and kind of caregivers monitor.
And the individual with the two auto antibodies two of the anybody's of dislike femia closely to avoid that's a cute crisis. If if possible what has been missing from the community for you know up to this point is Amy.
Meaning full infection and we believe that that is also a T to triggering revisions and guidelines around the general population screening and so on which we of driving so uhm out of goal.
Is to use Alice situation as as the first mover as as of disrupt to to to allow the ecosystem to change and shift it's paradigm and thinking of we try and drive.
Wherever we possibly can and the ability to sponsor the J D. R. F. A T. One detect initiative brain.
The the the screening of of of of auto antibodies from what was really of a an academic type of consortium, you know opportunity into the hands of the actual patience themselves, especially during a of Covid pandemic where they.
Can do the testing of home you know, which is very proud of Bam to sponsor that with J D. R S and the and and and begin the fundamental change Jason do you have any more details.
Yeah, Oh, I would all I would add to that Ashley is you know I think with that you wanted the tech program, particularly during the pandemic I think J D. R. F has a really nice initiative here that includes both educational components and obviously that in home drive blood spot screening technology that makes things as simple as possible for patients to go about getting screening.
If they want to I think the two things that I would add that would actually said are that.
That I think you know we know through both claims database analyses and what's been published from academic consortia that even in the absence of the in home drive, let's biotechnology hundreds of thousands of auto antibody tests are being ordered by physicians every year and so there are patients being screen.
And that you know there's also an educational GAAP out there with respect to the disproportionate rest of the patients are and if they have a family member with type one diabetes and so I think there are two components to this that I think of really important one is the unbranded disease awareness campaign that we launched it at the end of the third quarter of last year, and then the educational component of associated with the.
He wanted to attack the all create a lot of noise in the in the space around why people should be screened knowing that there's published literature to suggest the diabetic ketoacidosis can be reduced from 50 per cent down to the single digits, which in and of itself is it is it is a huge advance for patients quality of life.
And the second you know is actually mentioned the approval of ectoplasm App for those patients that don't Wanna be screen until there's a therapeutic intervention that can ultimately address of the problem.
The approval of to put them App should catalyzed even more screening through you know a number of different mechanisms, which you know obviously it is a good thing for a number of reasons. So the those would be the only the only additions I have on on top of of Ashley's comments.
Okay, great. Thanks for that and then maybe just quickly on your conversations with the payers I'm I'm just curious as to whether you feel like they're sort of uhm, you'll criteria for of prior authorization. That's emerging from the discussions and then yeah, how how much I guess, there's the re dosing of part of the conversation.
Is re dosing something you know the payers are factoring in in our office of that you know does that all at all change their thoughts on the value proposition of of the product.
Jason.
Yeah. So you know with respect of the the first part of your question you know we've seen a fair degree of consistency with respect of how payers are thinking about how they would cover of product like to play the map and it's aligned with our strategic objective, which is you know of streamlined prior authorization to label and so what we heard from her.
<unk> early on was that based on the profile of of to push the map. You know this would be of medical benefit that they would anticipate covering with the a prior authorization of label you know unless you know they felt like you know we price this way out of whack and then they would put additional controls on it but you know the the initial thought was prior authorization of late.
<unk> and the one comment that we thought was insightful that they gave us at the advisory Board was that they would anticipate covering this with a P. A the label because of the unmet need and type one that if they were to deny this friday the would almost almost surely expect that it would be overturned on appeal. So our goal you know of similar to any other specialty therapeutic product is that at launch or you know sure.
Lee thereafter, once they've had a chance to review and right of medical policy that we would have you know of streamline prior authorization to label.
Okay, and then just on you know <unk>.
Re dosing is that conversation come up with the payers and and what's their feedback you of any.
Yeah. So we're not we're not the because we don't yet have data on re dosing, it's really not a part of the conversation at this time.
Got it okay, great. Thanks, so much for taking the questions.
It concludes our question and answer the question I would like the kind of conference back over the athletes on there for any current in your back.
Thank you very.
Thank you very much again for your time today and the tension this morning, we.
Yeah, I hope you will have a pleasant day, and we look forward to speaking with you again very soon.
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