Q4 2021 Provention Bio Inc Earnings Call
Good morning, My name is Irene and I'll be your conference operator for 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 buyer.
Thank you operator, and thank you all for joining us on prevention Bio's fourth quarter and full year 2021 financial results Conference call.
Joining todays call from the prevention bio team as Ashleigh Palmer, Chief Executive Officer and co founder.
Cisco Leon Chief Scientific Officer, and co founder, Jason Hoyt, Chief Commercial officer and theory, So Shea Chief Financial Officer.
Before we begin let me remind you that the various remarks, we will make today constitute forward looking statements.
These include statements about our future plans and expectations.
Clinical result, regulatory and other development and timeline.
<unk> to our product candidates.
Including our plan to continue working with the FDA as they review our BLA Resubmission.
Our effort towards securing a potential FDA approval and commercialization.
<unk> proposes the mab or an at risk indication.
As well as the planned delivery of significant catalysts over the next 24 months.
The potential safety efficacy and commercial success of the plays the Mab and.
And our other product candidates.
The potential COVID-19 impact on our clinical studies and business plan.
Financial projections, including our anticipated use of cash and our cash runway.
And our business plans and prospects and projected timing for the St.
Actual results may differ materially from those indicated by these forward looking statements as a result of various important factors.
Including those discussed in the risk factors section of our most recent annual report.
On Form 10-K .
We filed with the SEC this morning.
And in other filings that we may make with the SEC in the future.
Any forward looking statements represent our views as of today only.
While we may elect to update these forward looking statements at some point in the future.
We specifically disclaim any obligation to do so.
Even if our views change except as required by law.
Therefore, you should not rely on these forward looking statements as representing our views as of any date subsequent to today.
There is more complete information regarding forward looking statements risks and uncertainties in the reports prevention filed with the SEC.
These documents are available on prevention website.
At Www prevention bio dot com under the investors section.
We encourage you to review these documents carefully.
With that I will now turn the call over to Ashley.
Thank you Bob.
Thank you to everyone joining us on this morning's call.
We are very pleased to be highlighting to you today.
Our progress in the fourth quarter of 2021.
First two months of 2022.
Prevention bio was founded.
Belief, recognizing and intersecting or even preventing autoimmunity before irreversible tissue damage and potential organ failure takes place.
Fundamentally change the world for millions of patients and their families.
Thereby enabling those individuals to be less impacted by their disease and more focused on living their lives and realizing their true potential.
In ton, making the world a better place for all of us.
Or kill immune diseases affect over 23 million patients in the United States alone.
Our assessment that our industry has to date.
This sizable population.
Appreciate the potential associated with innovating to address its considerable unmet need.
Instead.
<unk> immune T has tended to be viewed by biopharmaceutical companies in the medical system as a whole from the perspective of waiting for symptoms to present to clinicians.
Ill, then left with no alternative but to try to manage more advanced stages of disease and that complication as.
As well as to treat ever worsening symptoms chronically for a lifetime.
Often the cost of significant therapeutic burden to patients and their caregivers with a corresponding reduction in quality of life.
<unk> consent.
We believe there are no better example of this sub optimal approach.
Im celiac disease and type one diabetes.
Really that disease has no approved therapeutic option of any kind.
Instead patients are left to try to manage their disease by attempting to eat.
Contamination from the from a ubiquitous pathological antigen.
Clinton.
Separately, the first time, a patient presents with the symptoms of type one diabetes such.
Such as life threatening diabetic ketoacidosis.
You can no longer produce enough insulin to control their blood sugar.
<unk> came to a patient with chronic kidney disease finding out they have to take the.
The very same day, they need five hours of dialysis three times a week.
Every week just to stay alive.
To all of US at prevention buyout. This is simply unacceptable.
These patients in less time lease deserve better and it is our strategic intent to make a difference.
In fact, the key consideration when we founded prevention bio with our belief that we could pioneer.
Julie similar immuno modulator approaches.
So immune disease.
So those we have witnessed after such profound effect.
Immuno oncology end of the spectrum of immune mediated diseases.
Using our conceptual platform.
Expertise in immunology, and our experience and track record and creative business development and corporate partnering.
We have acquired and we are advancing a pipeline of product candidates that are uniquely focused on early autoimmune disease intervention.
We have to lead acid.
<unk> and PR of the $32 79.
We believe half.
Tension replicability across a broad spectrum of autoimmune disorders.
Both as monotherapy.
And in combination with other emerging approaches such as cell therapy.
Gene therapy, an antigen specific tolerance Asian.
And we are now even more committed and well positioned to unlock this potential whether it be through independent development program.
All strategic partnership.
Throughout 2021, we made significant progress advancing our two lead therapeutic candidates along with our other clinical stage pipeline assets.
And we were especially delighted to announce earlier this week the re submission of our Pep lithium our BLA.
Delay the onset of clinical type one diabetes in at risk individuals.
This resubmission followed was very productive and collaborative interactions with the FDA throughout last year include.
Including an advisory committee vote in May in favour of <unk> approval in at risk type one diabetes and.
And a subsequent complete response letter in July that cited no clinical deficiencies related to the efficacy and safety data packages submitted to the original BLA.
Our BLA that included data from the pivotal NIH sponsored <unk> 10 clinical trial conducted by <unk>.
In which a single 14 day course of catalyst demob significantly delayed the onset of clinical stage insulin dependent type one diabetes in at risk patients.
By a median of approximately three years.
Last week's BLA Resubmission, followed a type B meeting held three weeks earlier at which the FDA proposed and the company agreed to use PK modeling to adjust the 14 day dosing regimen for our planned commercial product.
To match the exposure of material manufactured over a decade ago.
We've used in prior clinical trials, including the pivotal <unk> study.
Under FDA guidelines. The agency now has 30 days to verify that our Resubmission is complete and acceptable.
At this time our goal date for review completion.
It is our understanding this goal date should be set within six months of last week's Resubmission date.
At which point if approved <unk> has the prospect.
Becoming the first ever disease modifying therapy in type one diabetes.
Following a potential approval in U S launch of complete demand for at risk <unk> by the end of this year.
Our future clinical development plans for this indication aimed to broaden initial labeling and market potential by exploring younger age group below eight years.
As well as the impact of repeat dosing to potentially extend a single course of therapies three year medium delay in progression.
We next look forward to the topline results of our phase III protect trial of <unk> in newly diagnosed <unk> patient.
We completed our target enrollment in quarter three of last year.
And out of an abundance of caution given potential COVID-19 related challenges for clinical trial follow up.
We proceeded to over enroll this study by about 10%.
We remain on track to deliver topline result, following completion of protect 18 months follow up period in the second half of next year.
Our longer term plans include the evaluation of subcutaneous formulation.
As well as the exploration of potential combinations of <unk> with other rapidly advancing approaches.
Such as pancreatic islet and beta cell transplantation.
Targeting the growing market of $1 8 million established insulin dependent type one diabetic Indian 19 states alone.
It is worth noting here that in prior published studies. The addition of <unk> to islet transplantation induction regimen.
It's been successful in extending the durability of favorable post transplantation result.
With 75% of transplanted patients remaining free from the burden of insulin dependency for.
More than five years.
Outside of <unk>, we plan to explore the potential use of <unk> across other autoimmune related disorders, such as celiac and Crohn's disease.
Early rheumatoid arthritis.
In autoimmune hepatitis.
Turning now to the rest of our pipeline.
The end of last year, we were pleased to announce positive interim results for the first in human study of our <unk> One O one vaccine candidate targeting coxsackievirus B.
Which is known to trigger both <unk> and celiac disease.
This phase one trial demonstrated in a dose dependent fashion.
<unk> 100 one's ability to induce high titers of viral neutralizing antibodies against all of the Coxsackievirus. These serotypes.
Good bye this polyvalent vaccine.
We are expecting the final results from this first in human study in the first half of this year.
Beyond the <unk>, five which has now been assigned the name or the <unk> is currently being studied in a phase <unk> trial with the goal of becoming the first ever therapy to intersect gluten free diet Nonresponsive celiac.
Thanks.
Our target date, the topline results from this trial is by the end of next year.
Regarding <unk> $32 79, our non depleting bispecific scaffold targeting b cell mediated disease.
We were very pleased to announce at the beginning of this year.
We have successfully initiated our prevail two phase Iia trial evaluating $32 79 in systemic lupus arithmetic.
And we expect this trial to be complete didn't read out topline results.
In the first half of 2024.
It is our current intention to hold a series of R&D related investor events throughout this year.
<unk> more deeply into our pipeline development.
As well as our plans for Pep lithium arbs label expansion and lifecycle management.
We are also planning to conduct an investor educational event in the second quarter focused on providing you with more in depth insights and details.
Regarding <unk> potential risks.
One the launch and commercialization.
However, let me now ask Jason to provide you with a brief update to whet your appetite.
Jason.
Thank you Ashley and good morning, everyone.
Im excited to speak with you all today and provide you with an update on the great progress, we're making in preparation for a potential commercialization of duvelisib for at risk to one day.
Over the course of 2021, our commercial team has substantially enhanced our launch readiness and added both breadth and depth to our understanding of the market in at risk patient.
To date, we have conducted market research with more than 1300 participants across key stakeholder audience.
Including healthcare providers patients caregivers payors and others.
In a recent blinded quantitative study of both pediatric and adult endocrinologists when shown at target product profile for <unk> Pleasant Mab in at risk patient they.
They indicated their intent to prescribe the product for 93%, but their next 10 patient.
Which is quite remarkable.
In addition, we conducted a quantitative market research study with more than 200 patients caregivers and at risk individuals.
And after reviewing a target product profile they provided insights into two pieces of important information.
They stated that with an approved treatment nearly 89% of them would get screened for tier one auto antibody if recommended by their health care provider.
When talking about <unk> patients and caregivers only that percentage increases to 96%.
When the same group of market research participants were asked about the likelihood of taking that pleasant map after seeing its target product profile.
81% said, they would take the treatment with their doctor's recommendation.
Finally in another quantitative market research study that included both pediatric and adult endocrinologist after viewing a target product profile Practicalism app, 76% of endocrinologists indicated they will increase screening of first degree <unk> family members at the time that they have an FDA approved intervention to offer their patients.
To overcome the largest barrier to daclizumab views based on market research 14 consecutive days of infusion.
Both healthcare providers and consumers alike become much more comfortable when presented with the potential for multiple scenarios as it relates to site of care.
To further our understanding of the health care provider environment, we deployed a 12 person pilot team strategically placed across the country in the middle of 2021.
The goals for this team has been twofold, Firstly education.
Educate healthcare providers about the benefits of screening family members, such as avoiding diabetic ketoacidosis and secondly to learn to further enhance our understanding of the tier one <unk> treating health care providers.
The team has interacted with over 1300 providers, including more than 2200 educational calls on type one diabetes treating physicians.
This team has gathered valuable insights, which are enhancing our commercial planning.
As I've mentioned on previous call. We've had two complementary disease awareness campaigns in the market since late 2020, and we continue to see a high level of engagement with these sites.
In the fourth quarter, we also launched Twitter and read it accounts to enhance and drive disease screening awareness.
As we have seen social media channels to be highly engaging with our consumer audience driving the lion's share of traffic to our consumer disease awareness type one test that dot com.
Our social content educates on tier one de risk an auto antibody testing connect.
Connect and engages with the <unk> community drives people to the unbranded website to learn more.
Our Facebook and Instagram account.
<unk> seen substantial traffic with over 400000 engagements of our unique content.
As we enter 2022, we intend to launch additional social media channels for health care providers and the <unk>.
In addition to our engagement of healthcare providers and consumers in 2020, we also continue to engage with payers through both blinded market research and direct engagement of our market access.
We continue to be encouraged by the feedback we've heard from payers and their positive feedback on the profile of <unk> and the unmet need of targets in tier one day.
We look forward to furthering our dialogue with payers once a new action date has been set by the FDA.
Finally in regard to the build out of our limited distribution model, we have contracts in place with our third party logistics provider specialty distributor and limited network of specialty pharmacies.
And are on track with implementation to be ready at the time of a potential FDA approval.
We look forward to continuing to update the market on our commercial plans that build out as we move further into 2022.
I'll now turn the call over to Terry to discuss our financial results.
Gary.
Thank you Jason.
Before I begin discussing the financials for the quarter I would encourage you to read our 10-K that was filed today.
The 10-K includes our financial statements risk 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, 2021, our cash cash equivalence and marketable securities position was hardware and $27 1 million.
Our cash based operating expenses for the fourth quarter ended December 31, 2021 was $23 9 million.
We expect our cash based operating expenses to be between $25 million and $29 million.
In the first quarter of 2022, as we continue to prudently gate our expenses.
Based on our current business plan, we believe that our cash cash equivalence and marketable securities on hand at December 31, 2021 are sufficient to meet our operating requirements for at least the next 12 months. However.
However, the depletion that BLA is approved.
We will need additional capital to for increases in costs related to commercialization and the payment of potential milestone triggered under our current agreement, including with Macrogenics.
We will provide additional cash guidance on these quarterly calls as we continue to progress towards the potential regulatory approval and commercial launch of depletion that.
From a P&L perspective, we generated a net loss for the fourth quarter of two of 2021.
I'll start again 123.
From a P&L perspective, we generated a net loss for the fourth quarter of 2021 of $25 8 million or <unk> 41 per basic and diluted share compared to a net loss of $32 6 million or <unk> 58 per basic and diluted share for the fourth quarter of 2020.
The decrease in net loss compared to the fourth quarter of 2020 is attributable to lower noncash stock based compensation expense, primarily driven by stock option was performance based metrics.
<unk> in the prior year period.
Also contributing to the decrease in net loss were lower manufacturing costs for <unk> hundred one and lower regulatory costs.
This was partially offset by increased costs for our pre commercial activities and our recently initiated <unk>, 30% to 79 prevailed two phase Iia study.
Our fourth quarter net loss included $2 6 million noncash stock based compensation.
The net loss for the full year of 2021 was $114 4 million.
Or $1 81 per basic and diluted share compared to a net loss of $98 6 million or $1 88 per basic and diluted share for the full year of 2020.
The increase in net loss of about 2020.
It was attributable to an increase in study cost will protect proactive which was initiated in August 2020, and prevail too.
Start up activities began in two in the second half of 2021.
So an increase in pre commercial medical affairs, and general and administration expenses as we build the organization in preparation for <unk> commercialization.
Partly offset by lower manufacturing costs with depletion Mab, and <unk> 101, and lower regulatory costs with depletion that.
The net loss for 2021 included $11 8 million of noncash stock based compensation expense.
One were recognized $1 $4 million of collaboration revenue under our license agreement with <unk>.
I will now turn the call back over to Ashley.
Thank you Jason and theory.
We made a great deal of progress in 2021.
This year is already off to a tremendous start with prevention.
Having resubmitted our BLA, we are very excited to move forward with our launch planning and the potential for <unk> to be approved for the delay in clinical stage <unk>.
At risk individuals.
Would validate our conceptual platform focused on early interception or prevention of autoimmune disease.
Adult irreversible tissue damage and possible organ failure.
We believe we are pioneering and catalyzing a potential paradigm shift in the management of autoimmunity, whereby individuals will be less impacted by the risk disabilities and therapeutic burden of advanced stage disease.
In so doing we will be allowing them and their loved ones.
Focus on living their lives and realizing their true potential.
In terms of making the world a better place for all of it.
With that operator wed like to take any questions.
Thank you we.
We will now begin the question and answer session to ask a question you might play Star then one on your telephone keypad.
You are using a speakerphone please pick up your handset before pressing the keys.
To withdraw your question please stay star and thank to remove yourself from the question queue.
At this time, we will post entertainment to assemble our roster.
Our first question is from Gregory Windsor.
RBC capital markets. Please go ahead.
Hey, good morning, Ashley and team congrats on the progress the refill this weekend and thanks for taking my questions.
Actually maybe I'll just start with Europe , you had mentioned.
Investor Education, and the appetite to do that over the course of the year.
Helpful to hear Jason can touch on that as well I'm curious if you can elaborate on any specific components.
Of that pleasant that an at risk population that you think doesn't merit clarification or as perhaps misunderstood by those stakeholders and we can start there. Thank you.
Thanks very much for the question, Greg, we're not really seeking to correct misunderstanding.
We're really wanting to provide more detail for our pipeline products.
As you can appreciate during last year the focus was on <unk>.
In at risk individuals. So we want to make sure that during the course of this year, we give our investors an opportunity to understand the potential.
Pep lithium app from a label expansion perspective.
A lifecycle management perspective.
Side of <unk> as well as leveraging <unk>.
Mechanism of action to enable the companies.
Technological platforms in combination therapy, and so on and then we wanted to do justice to the potential of $32 79.
In particular and the rest of our pipeline and then I think from <unk>.
Jason perspective, with respect to providing commercialization details in quarter two.
Again, obviously, our focus has been on the regulatory pathway.
We had questions from investors and focused on conference calls throughout the second half of last year.
<unk>.
Detailed aspects of.
Regulatory considerations and we think.
Got you and.
The rest of our investors and analysts will appreciate and opportunity to dive deeper into commercial preparations in.
In the next quarter.
That's really helpful. We look forward to those to those update and maybe just on the re file I'm. Just curious if you could perhaps provide maybe some some guidance on when we will learn more about the dosing level being contemplated with respect to your work with the FDA.
And the new dosing regimen is that something that we would expect to hear.
In a short course of time or are we waiting for FDA comfort on that just any color on your disclosure plans would be great. Thank you.
Yes, Thanks again for the follow up question there.
So obviously, we resubmitted.
Last week.
Next milestone would be the fda's acceptance within 30 days.
And then.
We'll begin to.
Review.
Process in earnest assuming that they expected.
Certainly during the course of that review process we.
Hope to be interacting with them and getting feedback and if there's anything material that we would do.
<unk> disclosed that.
Normal part of our.
Quarterly updates or will by exception at some point.
We hope the agency will.
Fixed on a.
A dosage regimen adjustment that.
Well, perhaps to begin to be included in labeling discussions and negotiations.
And I think as soon as we get to that point.
We would be comfortable.
Beginning to disc.
To discuss that more publicly is the potential.
Dosage regimen.
For potential approval.
That's great. Thanks, Ashley we look forward to the updates and thanks for taking my question.
Thank you Greg.
Again, if you have a question. Please press star and then one.
Our next question is from Thomas Smith of SVP.
Please go ahead.
Hey, guys. Good morning, Thanks for taking the questions and congrats on the progress and the BLA Resubmission.
I just wanted to ask one on the regulatory side with.
With respect to protect maybe.
Maybe if you could just outline what the plans are for engaging with regulators in any kind of dialogue with respect to the dosing and protected are there any plans to implement our study the PK modeled dosing regimen in this study.
Thanks, Tom So we're obviously closely monitoring.
Our dialogue with the agency in the context of at risk.
This is clearly a little different.
That we have.
Both therapeutic.
Drug products.
In.
That study we believe so.
He is well powered and as you as I mentioned in my opening remarks.
<unk>.
Over enrolled.
10% to make sure that we have sufficient patient.
To do.
The necessary analysis.
We still feel very strongly that despite differences in PK between.
The drug product the material intended to be commercialized in the material that had been used 11 years.
11 years ago that was <unk> 10.
That.
They lead to similar Pharmacodynamic.
Impact and impact on the immune system.
And.
And therefore that they are both efficacious, we hope the protect study will perhaps confirm that.
Also recall that we're giving two courses of therapy in protect whereas.
CN Penn only gave one.
And.
Also may be helpful. In mitigating some of the Fda's PK comparison.
Compatibility considerations.
I think the agency is concerned we can turn and given the materials manufactured.
11 years ago.
The <unk> study.
It was sort of seven year duration study.
Hey.
Like we wanted to make sure that the.
Patients that get this single course of 14 days getting.
Enough exposure.
Going forward matching exposure.
That <unk> 10.
Patients received to get the three medium delay.
<unk>.
With the <unk> potentially to be commercialized product.
And so a modest adjustment in dosage regimen accomplishes that without.
Creating any concerns and I think that's a different situation.
The one.
That we're in where.
We.
Conducting the protect phase III trial with two courses of therapy with some patients receiving <unk>.
One product in the other group receiving proceeding receiving the to be commercialized does that help.
Okay, Yes, no that's helpful. Thanks, Ashley and then.
Just a question in terms of commercial planning and looking forward to the event.
Here in the <unk>.
Next quarter can you talk a little bit about the commercial manufacturing activities and where do you feel like you are in terms of potential launch supply.
Yes, thanks for that question and I'm going to hand that over to <unk>.
So Jason Jason could you answer that question. Please.
Yeah, absolutely. So we've obviously been working with AGC biologics and then our fill finish manufacturer and feel that we are adequately stocked too to supply. The launch later this year.
Okay, Great and then just.
Lastly, if you could just Ashley maybe give us an update on.
Any of the other potentially.
Potentially outstanding issues that were cited in the CRM I know they were manufacturing.
And other CMC issues that were cited but you also had the site they need exactly in August just I guess, the latest on where you stand with respect to those issues.
Yes, Thanks, Tom So if you recall some of those were.
Issues, we felt we'd already addressed but the agency didn't have time to review by the time the CRA.
Issued and then some.
Sort of Rep.
Represented for the first time in the Cri, which we thought were addressable.
Out of.
An abundance of caution we had the type a meeting in August two.
Walk through our action plan to address those.
With the agency and that was done too.
I believe best satisfaction, and so on the basis of that.
We focused in on the PK comparability and.
And resubmitted the BLA.
Last week.
To address.
All of the.
<unk> consideration.
Got it okay, great. Thanks, guys for taking the questions and congrats again on the progress.
Thank you Tom.
Our next question is from Justin Kim of Oppenheimer <unk> co.
Hi, good morning, Thanks for taking the questions and let me add my congrats on the progress.
Quarter.
As you approach potential figures.
How do you think about the timing and initiation of studies that explore label expansion.
I know the expectation.
The.
Postmarks post approval and potentially maybe even included as post marketing commitments, but curious how small the delay between approval and label expansion studies might be and whether there any items that could be done ahead of us.
Let's say that event.
Yes, Justin Thank you very much so.
In their spare time.
While they have been.
Re submitting a BLA and interacting with the agency on the modeling and the PK comparability.
Our team has been very much working on protocol.
Design and.
Already to have discussions with.
The agency regarding.
For example, the under eight years.
And.
Can not just be something we addressed by way of a specific study.
But also a registry to.
To capture.
Information from from patient.
Post marketing.
If we're fortunate enough to have the approval.
The same with re dosing.
We don't necessarily believe that the label will specify.
A single dose.
Can only be a single dose.
But we obviously have to provide data for the.
Agency to include follow up dosing to obtain specific outcomes.
We have to present that data to the agency and get the agency's approval to include it in the label.
Certainly payers would be expecting that type of information to inform them with respect to that decision.
We have been thinking about this.
Very very thoroughly and I think that.
Upon.
A potential approval you would see a relatively.
Smooth and.
Expedited process to get those studies and initiatives up and running.
I understand I understand.
And what's really interesting this year the work on creating profitability inside of care.
Hi, Jason would you be able to share any additional color on the potential scenarios being pursued.
Right.
Jason.
Yeah, absolutely. Thanks for the question Justin I think I've mentioned on previous calls before that our intent with respect to site of care or site of infusion is that.
It's a decision that really needs to be made by the patient <unk> caregiver in consultation with their health care provider and.
And as such our intent is to.
Ensure that we are making to pleasant mab as available and.
And that we are being as flexible in terms of product acquisition as we possibly can be so.
So let me expand on that a little bit so.
For example, a provider that wants to infuse the patient in their hospital infusion Center can go ahead and acquire the product through our specialty distributor.
And then bill a patient's insurance, we know obviously that launching it wasn't that will be launching with a miscellaneous J code in some infusion centers may not want to take that risk. So they will have the ability to wipe bag the product Youre one of our limited network of specialty pharmacies.
Should a patient and or health care provider.
Want to administer the first few days under observation in an infusion center or however, many days that maybe they can go ahead and do that and we will call that something like a hybrid model, where they started an infusion center and then transition to home infusion I think one of the benefits of the two specialty pharmacies that we have in our network is that they have home infusion capabilities in all 50 states.
And then lastly, if a health care provider <unk> patient wants to pursue home infusion. We obviously have that option available as well, we think that will be more of a longer term.
Site of care alternative that patients and healthcare providers will take we think that initially with a novel therapeutic that most will want to start in an infusion center, but our intent is to put this decision in the hands of those that should be making the decision to health care providers and the patients.
And doing everything we can to remove as many barriers as possible.
Got it okay, great great and just a final.
Sort of curiosity I mean with regard.
Macrogenics payment.
Associated with the potential approval of <unk>.
Any potential to restructure that agreement or <unk>.
Payment, specifically to better align with the business cash needs.
Yeah.
There is always potential too.
Discuss those types of things.
If that takes place we'd be updating the.
You and the investors.
Accordingly.
Great. Thanks, Thanks, so much enrollment Matt Matt Congrats on the program.
Thank you Justin.
Our next question is from David Wang of <unk>.
Hi, Ashley and team congrats.
Congrats on the progress and thanks for taking my question.
I just wanted to ask first on the <unk>.
In terms of the.
Eventual commercial rollout if we do get that.
Date assigned an <unk>.
<unk> approval in the next six months or so how quickly do you feel that you can get the drug into the channel and available to patients.
When would you I guess considered a sales force to be add.
100% capacity.
Thanks for that question, David obviously.
Two very discreet questions there.
Ensuring that patients have.
Access with the appropriate support being one and then the other.
The full scale up of.
The sales force.
Infrastructure.
Those over to Jason, but you can imagine that the accent, one is especially important to us given the potential breakthrough nature of.
This is Jason.
Yes. Thanks for the question David So our intent is the short answer is our intent is to get the product into the channel and made available to.
Providers and patients as quickly as we possibly can but with the right levels of support in the background to make sure that that initial experience is a positive one and so as we've mentioned in the past where gating, our hiring and our spend to regulatory clarity throughout the course of the year.
And as such.
Based on upcoming dates and milestones, we will be able to provide additional clarity, but our goal is to shorten the window.
Much as we possibly can to launch as fast as we possibly can after a new FDA action date.
Great. Thanks for that and just a follow up if.
If I may in terms.
Pricing.
And such.
Imagine you would probably want to announce that closer at the time of launch.
And just is.
Is that kind of how youre thinking about it and then in terms of the payer discussions.
Anything to update us there in regards to sort of their feedback or.
Color code from these meetings in terms of their expectation.
On.
Restrictions for any restrictions were at a project or any.
Kind of label expectation.
Yes.
Payers see it.
Thanks, David Yes, so you're exactly right with respect to defense.
Pricing in the decision point.
But I think Jason you would agree that.
Youre seeing a very encouraging.
Feedback from payers do you want to give some details to David.
Yes, absolutely. Thanks, Thanks again David.
Youre spot on we would anticipate announcing.
At the time of a potential approval down the road.
We've completed our pricing research our pricing committee will set a price relatively close to an action date. So we haven't set a price yet at the time of approval, we would anticipate announcing that.
But again.
I continue to be.
Really encouraged with the consistency of the feedback that we've heard from payers over the course of the last two years I think that what we've most recently heard is that the.
The vast majority of payers anticipate covering this is a medical benefit and look to cover this with a prior authorization to label, which is strategically what we intend to and are hoping and planning for.
Okay, great. Thanks, so much for taking the questions.
Thank you.
My last question is from Alethia Young of Cantor. Please go ahead.
Hey, guys. Thanks for taking my question and congrats.
Getting back to the <unk>.
Starting line of our lives.
Hi.
First question is a big picture question, which is and maybe it's for Jayson Ashley just.
And I know you've had a year to really kind of prep and think about things like what do you think is the key the main challenge that kind of keeps you in that kicks yet, but you know you know that.
That's the main heard a lot about the slot that you have to get over and maybe kind of front running it but like I guess I'm curious when you think about screening and what's going on today.
Just can you talk a little bit about how we get to the screening to be very very standardized in do you feel that.
It's a function of the drug has to be in the market are doing.
Do you feel like Theres any forward progress over the past 12 months and getting People's head wrapped around the fact that there just seems to be a greater degree of screenings that people will know that.
Another auto antibody.
Hi, Alicia Thanks for the question, so you're spot on at screening screening screening.
Finding the patients and making sure that we use lithium app catalytic really leverage it.
Pioneering.
<unk>.
Two.
Introduce a paradigm shift.
And I think that you mentioned.
Back to the starting line and I appreciate the perspective that leads you to say that but we are not we have way beyond that starting line with many respects to the potential introduction of the first disease modifying therapy in tier one day and although we have had a higher hiatus with respect to the <unk>.
<unk> pathway for Pep lazy map.
In at risk individuals during that time.
The entire community.
Hum.
<unk> been making forward progress and momentum.
The prospect.
Tablet demand following the advisory Committee meet.
Meeting and those those.
The way the community rallied around the open mic to express.
Unmet need here and so on has really how the catalytic effect on.
The ecosystem and has.
No.
At the <unk>.
Aspect of a potential approval and launch.
We'll have more momentum behind it would have had.
I'm going to hand over to Jason to give you some more specifics, but I think what I <unk>.
Personally.
Away rather than sort of lie awake at night worrying apparent is that we have already <unk>.
<unk>.
Change.
With some of the work we've been doing with.
With the J D. RF I think Jason should speak to the sponsorship of tier one detect making screening more accessible.
But we've already had an effect, even if a therapy doesn't get approved.
Certainly hope and believe it will.
Just bringing awareness to type one diabetes for the fact that you can identify before.
Lots of stages and if that helps.
The populations.
Appreciate the impact of diabetic ketoacidosis.
Brings their attention too early.
Symptoms, so that the diagnosis of clinical stage disease.
As a more gentle.
Then we've accomplished something but we anticipate being able to do so much more Jason.
Yeah, absolutely. Thanks for the question of Lithia, and I think I think absolutely spot on right I think.
We know that there are there are families out there in patients out there that.
Currently walk to know their Ottawa auto antibody status, so that they can adequately and appropriately prepare for what the transition to insulin dependence looks like we also know that there are other patients and families out there that in the absence of a therapeutic intervention.
I don't want the anxiety of knowing.
And so as you saw from some of the market research data that I highlighted during our prepared remarks, both physicians with a 76% intent to increase screening and patients with an 89% acceptance of auto antibody screening in the context of an approved drug highlights highlights.
Catalytic nature of Daclizumab that Ashley was speaking too so I think in the short term.
Activate the <unk>.
At risk individuals that were reluctant to be screened in the absence of an intervention that can address the problem that they are screening for but then in terms of our longer term strategy.
The the introduction of a novel immuno modular Tory therapeutic intervention like the prism adds.
Really catalyzes that conversation around population based screening and how we ultimately make that a standard of care during routine pediatric well visits in the United States moving forward.
Hum.
Humbly with so.
Grateful to the way in which the community has rallied around.
This this opportunity this prospect.
Working with the patient advocacy groups.
Yes.
Is a thrill to see their excitement and the potential.
You know that.
That we bring to.
To change that.
Circumstances.
Do you want to mentioned <unk> J.
Jason because I think that that.
Especially.
Good initiative, we sponsored.
Run by J D RF brings screening to the kitchen table.
<unk>.
The relatives of patients.
Yes, absolutely awesome and thanks for the reminder, there so <unk> detected the JD RF one run program that allows for dry blood spot auto antibody screening in the comfort of a patients own home and I think the fact that J D. RF launch this program.
Over a year ago now.
During the height of the Covid pandemic made it all that much more relevant and I think there are three really core components of the program that <unk> rolled out that are important to highlight the first of which being the educational component or the core constituent audience of J D. RF, which is the already diagnosed type one community right encouraging them to get their loved.
One screen, making this easy and accessible as possible through dry blood spot technology that comes in a kit with Atlanta to be able to assess your auto antibody status the linkage to care on the backend is critically important and then lastly, and I think most importantly.
Affordability component of that.
The complete kit that comes to a patient's home through the tier one detect program only cost $55 and if that $55 cost is too much for a patient if they can't afford to $55 JD RF will buy that down to 10.
For a cost of $10 a patient can be screened.
As Ashley mentioned at their kitchen table, which I think makes screening that much more accessible to a broader population of patients. So I think it's a really novel program and we're incredibly proud to have been the founding sponsors of that with J D.
Yes, that's a really cool and also yes, absolutely yeah starting lineup.
Oh, sorry line, but just.
Kind of back to finally, we can get ready to focus on launch and commercial but obviously you guys have been shopping and incredible amount what in the past 12 months.
I guess the follow up to that is I was kind of also around the sensibility in pricing you talked about a little bit of a basin does now with being able to cut costs.
How do you think about like the cost to these patients.
I mean, I know that kind of out of pocket costs and I mean, some of it that screening and so it sounds like maybe 10, but are there any other things like as far as like when the first one goes to get the drug administered but you think about.
And then just I guess the other piece of the question.
It's a different obviously different approach to treating patients and preventative approach do you think that people and parents I guess, namely.
Have any kind of concern or hesitation about kind of using an antibody treatment and their children.
Or kind of how you guys are talking to managing that thanks.
Great follow up question. Thank you, Jason do you want to answer both of those.
Yeah, So absolutely Alicia so maybe I'll answer the second question first and say that.
When looking at a target product profile there are questions around safety, but once we talk through.
The the nuances of the product what the unmet need is how the product.
Ultimately addresses that need you see.
And quantitative research after viewing of TPP that 81% of patients intend to accept treatment with daclizumab after our health care providers recommendation. So I think that number is.
That number reflects exactly the question that Youre asking right and then.
With respect to our patient support program, we're still in the final stages of designing that but I think it's important to note that our anticipated payer mix is 60% commercial 35% Medicaid and then about 5% other.
Our intent will be to do what we can to support out of pocket costs for those commercial patients where we can.
And Jason respond to.
So the first part.
Is one of the reasons, we're so excited with respect the prospect of combination therapies with.
Emerging therapies like cell therapy beta cell transplantation and so on.
Trading off.
Time of insulin therapy for a lifetime.
Transplantation.
Immune suppression.
To keep that transplant.
<unk>.
It is something that hangs in the balance.
Because we administer.
Discrete cost of therapy.
Potentially.
Protect.
Only the beta cells that a patient has.
Their own but potentially those that might be transplanted in from a 14 day course of therapy to respect and reboot. The immune system. We are very excited about the prospects of <unk>.
Working with the companies, we admire in that space that are driving that technology forward.
If the combination with Pep lithium app can give them.
A good outcome relative to some of their other options.
Awesome. Thank you.
Thank you Lisa Lithia.
Yeah.
This concludes our question and answer session I would now like to turn the conference back over to Ashley Palmer for any closing remarks.
Thank you Irene.
And thanks for the questions and thank you all again for your time and attention. This morning, we very much look forward to keeping you all updated on our progress throughout 2022.
The conference has now concluded. Thank you for attending today's presentation you may now disconnect.
Okay.
Okay.
On a stay on it.
Okay.
[music].