Q4 2023 Inovio Pharmaceuticals Inc Earnings Call

March 6, 2024

<unk> answer session.

If at any time during this call you require immediate assistance. Please press star zero for the operator.

This call is being recorded on Wednesday March six 2024.

I would now like to turn the conference over to Thomas Hung manager of Investor Relations. Please go ahead.

Good afternoon, and thank you for joining the <unk> 2023 fourth quarter and full year financial results Conference call.

Joining me on today's call are Dr. Jackie Shea, President and Chief Executive Officer, Dr. Michael Sumner, Chief Medical Officer, Mark Twyman, Chief Commercial Officer, and Peter Keyes, Chief Financial Officer.

Today's call will review, our corporate and financial information for the quarter and full year ended December 31, 2023, as well as provide a general business update.

Following prepared remarks, we will conduct a question and answer segment.

During the call, we will be making forward looking statements regarding future events and the future performance of the company.

These events related to our business plans to develop <unk> DNA medicines platform, which include clinical and regulatory developments and timing of clinical data readouts.

Along with capital resources and strategic matters.

All of these statements are based on the beliefs and expectations of management as of today and actual events or results could differ materially.

We refer you to the documents we file from time to time with the SEC, which under the heading risk factors identify important factors that could cause actual results to differ materially from those expressed by the company verbally as well as statements made within this afternoon's press release.

This call is being webcast live and a link can be found on our website IR dot <unk> dot com and a replay will be made available. Shortly after this call has concluded.

I will now turn the call over to <unk>, President and CEO, Dr. Jackie <unk>.

Good afternoon, and thank you to everyone for joining today's call.

The past 12 months have been transformational for <unk>.

Today, we are a company planning to submit our first BLA in the second half of this year and preparing for the potential commercial launch of our first products in 2025.

If approved <unk> seven kudos have become the first non surgical therapeutic option for patients with RP and be the first DNA medicine available in the United States.

Beginning verbally as well as statements made within this afternoon's press release.

To achieve this transformation, we have prioritized our product pipeline to focus on 30, 187, and other promising late stage assets.

This call is being webcast live and a link can be found on our website IR dot <unk> dot com and a replay will be made available. Shortly after this call has concluded.

All with high unmet medical need and strong commercial potential.

I will now turn the call over to <unk>, President and CEO, Dr. Jackie Shay.

We remain committed to financial discipline cutting our operating expenses, mainly in half compared with 2022.

Good afternoon, and thank you to everyone for joining today's call.

The past 12 months have been transformational for <unk> today.

And focused on leveraging the advantages of our platform to deliver on the promise of DNA medicine.

Today, we are a company planning to submit our first BLA in the second half of this year and preparing for the potential commercial launch of our first products in 2025.

In addition to the significant progress made with 30 197, our strategic refocus helped drive progress across the pipeline, including a new clinical collaboration and supply agreement with <unk> Biosciences to develop pioneer 31, 12 in combination with <unk> towards the thread count.

If approved <unk> seven kudos have become the first non surgical therapeutic option for patients with RP.

I'd be the first DNA medicine available in the United States.

Sure.

We also shared encouraging results, Brian a $42 one as <unk> vaccine continued to advance Opex clinical stage candidates and made progress with promising preclinical research opportunities for next generation candidates, adding to the positive momentum.

To achieve this transformation, we have prioritized our product pipeline to focus on 30, 107, and other promising late stage assets.

All with high unmet medical need and strong commercial potential.

We remain committed to financial discipline cutting our operating expenses, mainly in half compared with 2022.

Looking ahead, several key catalysts will help continue that momentum across our pipeline.

I'm focused on leveraging the advantages of our platform to deliver on the promise of DNA medicine.

In addition to submitting our BLA for $31 seven on the Fda's accelerated approval program, we plan to initiate a confirmatory trial in the second half of 2024, and we will continue preparations for a potential 2025 launch.

In addition to the significant progress made with $31 seven our strategic refocus helped drive progress across the pipeline, including a new clinical collaboration and supply agreement with <unk> Biosciences to develop pioneer 31, 12 in combination with block towards the throat counts.

For our immuno oncology candidates, we plan to finalize the trial design for evaluation of 31 12 in combination with <unk> in patients with <unk>.

Sure.

We also shared encouraging results from INR $42, one as <unk> vaccine continued to advance other clinical stage candidates.

As well as determine next steps for 50, 401, and clear Blastoma at deadly form of brain cancer.

<unk> made progress with promising preclinical research opportunities for next generation candidates, adding to the positive momentum.

We also expect some key milestones for our infectious disease candidates, including discussions with collaborators from potential partners around development plans, Brian a 40 201 as an apparel booster vaccine and the first half of 2024 and the REIT asset the first clinical data from the phase one.

Looking ahead, several key catalysts will help continue that momentum across our pipeline.

In addition to submitting our BLA for $31 seven under FDA accelerated approval program, we plan to initiate a confirmatory trial in the second half of 2024, and we will continue preparations for a potential 2025 launch.

Trial evaluating the <unk> demob candidates in the second half of 2024.

I would now like to pass the call over to our CFO, Mike Sumner, who will provide some additional details on our clinical progress over the past year and our goals for the year ahead.

For our immuno oncology candidates, we plan to finalize the trial design for evaluation of 31 12 in combination with <unk> in patients with stroke.

Thank you very much Jackie and greetings everyone.

As Jackie outlined we have made significant progress across our pipeline over the past year. Thanks to a strong strategic vision that prioritizes promising candidates with strong commercial potential.

As well as determine next steps for 50 401 in clear Blastoma at deadly form of brain cancer.

We also expect some key milestones for our infectious disease candidates, including discussions with collaborators from potential partners around development plans, Brian a 40 201 as an apparel booster vaccine and the first half of 2024 and the readout of the first clinical data from the phase one.

One of the highlights has been the significant progress of INR $31 seven for the treatment of recurrent respiratory papillomatosis or RFP.

For those who are not familiar with our LP. It is a devastating rare disease of the respiratory tract caused by HPV six and 11 it.

Trial evaluating the <unk> D Mab candidates in the second half of 2024.

It is characterized by <unk> called <unk> that can develop throughout the respiratory tract, primarily affect allowing some vocal cords.

I'd now like to pass the call over to our CFO, Mike Sumner, who will provide some additional details on our clinical progress over the past year and our goals for the year ahead.

RFP, most commonly causes difficulty speaking or complete voiceless difficulty swallowing shortness of breath and choking episodes in rare cases, papilloma can spread to the lungs or become malignant.

Thank you very much Jackie and greetings everyone.

Incidence and prevalence of RFP is variable globally and depends on several factors. The most widely cited use epidemiology data published in 1995.

One other highlight has been the significant progress of IL $31 seven for the treatment of recurrent respiratory papillomatosis or RFP.

Estimated that there were 14000 active cases for both adults and juveniles and about $1 eight new cases per 100000 adults per year.

For those who are not familiar with our LP. It is a devastating rare disease of the respiratory tract caused by HPV six and 11 it.

The only way to remove the <unk> surgery.

But surgery doesn't address the underlying HPV infection. So the <unk> <unk>, often forcing patients to have multiple surgeries a year.

It is characterized by what like <unk> that can develop throughout the respiratory tract, primarily affect allowing some vocal cords.

In the worst cases that could be hundreds of surgeries over a lifetime.

RFP, most commonly causes difficulty speaking or complete voiceless difficulty swallowing shortness of breath and choking episodes.

This puts an extreme physical and emotional burden on patients, including the threat of permanent vocal cord damage impacting the patient's ability to speak normally ever again.

In rare cases, papilloma can spread to the lungs or become malignant.

A recent study found that even patients who had had less than five surgery face a 50% chance of permanent vocal cord damage.

Incidents from prevalence of <unk> is variable globally and depends on several factors. The most widely cited use epidemiology data published in $19 95.

In patients, who have had 10 or more surgeries that increases to a 98% chance.

Estimated that there were 14000 active cases for both adults and juveniles and about 1.8, new cases per 100000 adults per year.

From our conversations with patients and healthcare providers.

Our LP patients are desperate for a non surgical treatment option.

The only way to remove the <unk> surgery.

As Kim Mcclelland President of the RFP Foundation reiterated last week at the White house for them on rare diseases.

But surgery doesn't address the underlying HPV infection. So the papillon was grow back often forcing patients to have multiple surgeries a year.

Even one less surgery, a year would be life changing for patients.

In the worst cases that could be hundreds of surgeries over a lifetime.

This slide shows the very real impact of INR $31 seven as had in reducing surgeries for <unk> patients in our phase one two trial as.

This puts an extreme physical and emotional burden on patients, including the threat of permanent vocal cord damage impacting the patient's ability to speak normally ever again.

As you can see here the majority of patients 81% saw a reduction in surgery after treatment with 28% needing new surgeries at all during the year after treatment.

A recent study found that even patients who had had less than five surgery face a 50% chance of permanent vocal cord damage.

As a reminder, we counted all surgeries, including any surgeries performed during the dosing window.

In patients who've had 10 or more surgeries that increases to a 98% chance.

We believe 30 107 has the potential to completely change the treatment paradigm for patients as a therapeutic adjunct to surgery.

From our conversations with patients and healthcare providers.

Our LP patients are desperate for a non surgical treatment option.

As Kim Mcclelland President of the RFP Foundation reiterated last week at the White house for them on rare diseases.

As you can see on this updated timeline, we have achieved some major development and regulatory milestones in just over a year.

Even one less surgery, a year would be life changing for patients.

In the first quarter of 2023, we shared positive results from our phase one two trial, which will later published in a leading peer review journal read by our future physician customer base in.

This slide shows the very real impact of INR 30, 107 has had in reducing surgeries for RFP patients in our phase one two trial as.

In the second quarter, we received orphan drug designation in the European Union, followed by breakthrough therapy designation from the FDA in the fourth quarter.

As you can see here the majority of patients 81% saw a reduction in surgery after treatment.

28% needing new surgeries at all during the year after treatment.

We now have an established path to BLA submission under the Fda's accelerated approval program and announced plans earlier this year to submit a BLA in the second half of 2024.

As a reminder, we counted all surgeries, including any surgeries performed during the dosing window.

We believe 30 107 has the potential to completely change the treatment paradigm for patients as a therapeutic adjunct to surgery.

Yeah.

Looking forward some of the key catalysts on the horizon for IL 30, 107 include completing submission of our BLA under the accelerated approval program in the second half of 'twenty four.

As you can see on this updated timeline, we have achieved some major development and regulatory milestones in just over a year.

Initiating a confirmatory trial prior to that submission.

In the first quarter of 2023, we shared positive results from our phase one two trial, which will later published in a leading peer review journal read by our future physician customer base in.

Requesting rolling submission and priority review, which would lead to possible action on our BLA application within six months compared to the usual 10 months review.

In the second quarter, we received orphan drug designation in the European Union, followed by breakthrough therapy designation from the FDA in the fourth quarter.

We will also target publication of immunological data supporting the mechanism of action of <unk> 107 in the second half of the year.

And finally commercial launch in 2025, if we received FDA approval.

We now have an established path to BLA submission under the Fda's accelerated approval program and announced plans earlier this year to submit a BLA in the second half of 2024.

Our chief commercial officer, Mark Twyman, who will provide an update on our commercial preparations and we look forward to accelerating the development of this promising product candidate over the next year.

Yeah.

Looking forward some of the key catalysts on the horizon for IL, 31% or seven include completing submission of our BLA under the accelerated approval program in the second half of 'twenty four.

And ultimately delivering on the promise of that DNA medicine for RP patients across the United States.

Shifting gears now to another late stage DNA medicine.

Initiating a confirmatory trial prior to that submission.

I'd like to spend some time talking about INR 31, 12, and our exciting new clinical collaboration and supply agreement with <unk> Bioscience Sciences that we announced earlier this year.

Requesting rolling submission and priority review, which would lead to possible action on our BLA application within six months compared to the usual 10 months review.

This partnership will allow us to evaluate 31 12 in combination with <unk>.

We will also target publication of immunological data supporting the mechanism of action of <unk> 107 in the second half of the year.

A PD one inhibitor that recently received FDA approval for treatment of nasopharyngeal carcinoma.

And finally commercial launch in 2025, if we received FDA approval.

We will be studying this combination therapy as a potential treatment for patients with local or regionally advanced high risk HPV 16, and 18 positive oral pharyngeal squamous cell carcinoma.

In combination. This therapeutic approach is designed to leverage the antigen specific T cells elicited by 31, 12, and the antitumor immunity generated by <unk> to potentially provide improved patient outcomes.

And ultimately delivering on the promise of our DNA medicine for RP patients across the United States.

Shifting gears now to another late stage DNA medicine.

I'd like to spend some time talking about INR, 31, 12, and our exciting new clinical collaboration and supply agreement with coherent Bioscience Sciences that we announced earlier this year.

Under the terms of the agreement <unk> will provide look towards it for use in the planned phase III clinical trial and provide support for regulatory interactions.

This partnership will allow us to evaluate 31 12 in combination with <unk>.

With this collaboration in place we have submitted our clinical development plans to the FDA.

A PD one inhibitor that recently received FDA approval for treatment of nasopharyngeal carcinoma.

And expect to receive feedback in the second quarter.

So what is or affirm Sheila squamous cell carcinoma.

We will be studying this combination therapy as a potential treatment for patients with local or regionally advanced high risk HPV 16, and 18 positive oral pharyngeal squamous cell carcinoma.

It's the type of head and neck cancer, because in the base of the tongue tonsils and or soft talent and is most commonly referred to as throat cancer.

For cancer is typically closely related to high risk subtypes of HCV, which are responsible for 70% to 80% of all oral pharyngeal cancers diagnosed in the United States.

There are also associated with tobacco and alcohol use.

HPV positive throat cancer is rapidly increasing in incidence.

Under the terms of the agreement <unk> will provide look towards it for use in the planned phase III clinical trial and provide support for our regulatory interactions.

Among patients in high income countries and has surpassed cervical cancer is the most common HPV related cancer diagnosed in the U S with nearly 20000, new cases each year.

With this collaboration in place we have submitted our clinical development plans to the FDA.

Most of our cancer patients are diagnosed with local or regionally advanced disease and the current treatment practice is focused on curative options through the use of multi therapeutic approaches including surgery and chemo radiotherapy.

And expect to receive feedback in the second quarter.

So what is or furniture or squamous cell carcinoma.

It's the type of head and neck cancer that occurs in the base of the tongue tonsils and or soft talent and is most commonly referred to as throat cancer.

This treatment protocol about 75% of patients do very well as measured by a three year progression free survival.

For cancer is typically closely related to high risk subtypes of HCV, which are responsible for 70% to 80% of all oral pharyngeal cancers diagnosed in the United States.

However, those 25% of patients who have their cancer progress.

Very poor clinical outcomes.

And proposed trial of INR 31, 12 in combination with <unk> will be in patients who are HPV 16, or 18 posted and exhibit a high risk of recurrent disease with the goal of preventing disease progression.

There are also associated with tobacco and alcohol use.

HPV positive throat cancer is rapidly increasing in incidence.

Among patients in high income countries and has surpassed cervical cancer is the most common HPV related cancer diagnosed in the U S with nearly 20000, new cases each year.

There are an estimated three to 4000 new patients in the U S. Every year, who are deemed to meet these criteria.

Based on previous trial data and the growing body of research, indicating that DNA medicines are adept at combating HPV related diseases. There is a strong rationale and combining 31 12 with a proven PD one inhibitor.

With this treatment protocol about 75% of patients do very well as measured by a three year progression free survival.

Results from our phase one two trial of 31 12 as a single agent treatment in 'twenty, two HPV positive head and neck squamous cell carcinoma patients.

However, those 25% of patients who have their cancer progress phase.

Very poor clinical outcomes.

Demonstrated T cell responses and infiltration of CDA positive T cells into the tumors.

In early 'twenty three updated results were also published from a different phase one two trial of $31 12 in combination with Astrazeneca PDL, one checkpoint inhibitor development.

There are an estimated three to 4000 new patients in the U S. Every year, who are deemed to meet these criteria.

Showing an overall response rate of 28%, which was comprised of four complete responses and four partial responses in 2009 evaluable patients.

Based on previous trial data and the growing body of research, indicating that DNA medicines are adapted combating HPV related diseases. There is a strong rationale and combining 30 112 with a proven PD one inhibitor.

This was accompanied once again by increased peripheral HBV specific T cells and tumor will CDA positive T cells.

The efficacy of this combination of 30 112 with development.

Results from our phase one two trial of 31 <unk> as a single agent treatment in 'twenty, two HPV positive head and neck squamous cell carcinoma patients.

<unk> and a median overall survival of more than 29 months. This compares favorably to immune checkpoint blockade therapy alone, which reports median overall survival of approximately 12 months.

Demonstrated T cell responses and infiltration of CDA positive T cells into the tumors.

This combined data set provides compelling evidence to support our belief in the potential of IL 31, 12 unlocked tulsi.

In early 'twenty three updated results were also published from a different phase one two trial of $31 12 in combination with Astrazeneca PDL, one checkpoint inhibitor development.

I will now turn the call over to Mark to provide an update on our commercialization efforts for <unk> 31, 7% Mark.

Showing an overall response rate of 28%, which was comprised of four complete responses and four partial responses in 2009 evaluable patients.

Thanks, Mike Hello, everyone.

Pleased to share that our commercial efforts for <unk> 107, Oh, well underway as we continue to prepare for potential 2025 launch while theres still much work to be done I'm confident our commercial launch strategy for several important reasons.

This was accompanied once again by increased peripheral HPV specific T cells, and Tumoral CDA positive T cells.

We have made understanding patients and their experience is a top priority every step of the way to.

The efficacy of this combination of 30 112 with development resulted in a median overall survival of more than 29 months.

To continue building on our understanding of the market. We held an advisory board payroll October of last year with adult patients suffering from RFP.

This compares favorably to immune checkpoint blockade therapy alone, which reports median overall survival of approximately 12 months.

They shared their journeys from diagnosis to treatment and provided invaluable insights into the unmet needs of the RFP patient community at large we have also conducted other advisory boards with healthcare provider specializing in the treatment of RFP and with caregivers or do you have enough pesos.

This combined data set provides compelling evidence to support our belief in the potential of INR 31, 12 unlocked Halsey.

I will now turn the call over to Mark to provide an update on our commercialization efforts for INR 30 107 Mark.

Next we understand that mapping the physician landscape will be critical to our commercial success to this end we have initiated a very comprehensive market analysis of physicians that treat RFP that will allow us to focus field. It will allow us to focus field deployment and make smart investment decisions.

Thanks, Mike Hello, everyone.

I'm pleased to share that our commercial efforts for ion authority of 107, Oh, well underway as we continue to prepare for potential 2025 launch while theres still much work to be done I'm confident in our commercial launch strategy for several important reasons.

We also have an experienced commercial team that is well versed in every aspect of the commercialization process for innovative products, particularly for rare diseases, our collective expertise and putting the plans in our central systems in place for a successful launch has been essential to our progress thus far and will help ensure that we stay on track moving forward.

We have made understanding patients and their experience is a top priority every step of the way.

To continue building on our understanding of the market. We held an advisory board payroll October of last year with adult patients suffering from RFP.

Perhaps most importantly, we believe that IL 30, 107 offers potential compelling clinical and commercial advantages that will serve as an important foundation to our commercial efforts.

As you can see here. The FDA has advised that we can use the data from our completed phase one two trial to submit a BLA under the accelerated approval program.

In the trial <unk> hundred 30 in Windows, seven was immunogenic, and generally well tolerated and over 80% of patients across the disease severity continuum at a reduction of the number of surgeries compared to the previous year.

This data was key in supporting our application for an subsequent receipt of breakthrough therapy designation from the FDA.

From a commercial standpoint, INR $31 seven offer several key differentiators it targets and has shown similar efficacy across both HPV, six and HPV 11, which cause RFP.

Perhaps most importantly, we believe that IL 30, 107 offers potential compelling clinical and commercial advantages that will serve as an important foundation to our commercial efforts.

Patients in our trial had a range of two to eight surgeries in the prior year with efficacy demonstrated across a range of disease severity.

Turning 107 also offers important attributes typical of our DNA medicines platform, including the potential for re dosing and stability for up to three years, a refrigerator temperatures admins.

As you can see here the.

<unk> has advised that we can use the data from our completed phase one two trial to submit a BLA under the accelerated approval program.

And the trial INR 30 in Windows, seven was immunogenic, and generally well tolerated and over 80% of patients across the disease severity continuum at a reduction of the number of surgeries compared to the previous year.

Administration with select you are proprietary electroporation device was well tolerated by patients with easy to use for health care providers in the phase one two trial consistent with its used in other trials.

This data was key in supporting our application for an subsequent receipt of breakthrough therapy designation from the FDA.

Also important for the commercialization of IL 31, seven is a well defined commercial scale manufacturing process as well as orphan drug orphan drug designations in both the U S and EU, which offer the potential commercial incentives and regulatory mechanisms in those markets.

From a commercial standpoint, INR $31 seven offer several key differentiators it targets and has shown similar efficacy across both HPV, six and HPV 11, which cause RFP.

I'll now turn the call back to our CEO Jacqui sure some additional pipeline updates Jackie.

Patients in our trial had a range of two to eight surgeries in the prior year with efficacy demonstrated across a range of disease severity.

Thank you Mark.

Mike has highlighted the important progress we've made with <unk> 31, 7% and INR 31, 12, and we are focusing the majority of our internal resources on advancing those candidates.

<unk> thousand 107 also offers important attributes typical of our DNA medicines platform, including the potential for re dosing and stability for up to three years a refrigerator temperatures.

We have several other late stage clinical stage assets and next generation candidates that we plan to advance through partnerships and collaborations across the industry academia and governmental agencies.

The administration with select you are proprietary electroporation device was well tolerated by patients with easy to use for health care providers in the phase one two trial consistent with its use in other trials.

I'd like to provide a brief update on each of them.

Also important for the commercialization of INR 31, seven is a well defined commercial scale manufacturing process as well as orphan drug orphan drug designations in both the U S and EU, which offer the potential commercial incentives and regulatory mechanisms in those markets.

Earlier I mentioned, the encouraging results, we announced in early 2023 from our phase <unk> clinical trial of <unk> as an Ebola vaccine based candidate.

More recently, we have received feedback from the FDA and identified a potential development pathway and are now in discussions with collaborators from potential partners to define next steps.

I'll now turn the call back to our CEO Jacqui sure some additional pipeline updates Jackie.

Thank you Mark.

Mike has highlighted the important progress we've made with <unk> seven and INR 31, 12, and we are focusing the majority of our internal resources on advancing those candidates.

INR 54, <unk> hundred one has to current disease targets.

With clear plus derma, we're in discussions with our partner Regeneron and investigators about next steps and our goal is to have a finalized plan in the first half of 2024.

The second target is to prevent cancer recurrence of cancer and other cancer or non cancer patients with BRCA, one or two mutations.

I'd like to provide a brief update on each of them.

Earlier I mentioned, the encouraging results, we announced in early 2023 from our phase <unk> clinical trial of 42, everyone as Nate Biolife vaccine based candidate.

Our phase <unk> study is being conducted by the University of Pennsylvania, and we look forward to updates as they become available.

Our partner Apollo Bio is also conducting a phase III trial of <unk> 3100, as a therapeutic treatment and cervical H cell caused by HPV 16 and 18.

More recently, we have received feedback from the FDA and identified a potential development pathway and are now in discussions with collaborators from potential partners to define next steps.

INR 50, 401 has to current disease targets, so clear plus derma, we're in discussions with our partner Regeneron and investigators about next steps and our goal is to have a finalized plan in the first half of 2024.

A phase II trial sponsored by the eighth Malignancy consortium is also underway evaluating <unk> steady 100, and HIV positive participants with annual H sale caused by HPV 16 and 18.

And there was also exciting work advancing next generation DNA medicine technology, including a trial with our collaborators at the Wistar Institute evaluating DNA launch nanoparticles <unk>.

The second target is to prevent cancer recurrence of cancer and other cancer or non cancer patients with BRCA, one or two mutations.

Our phase <unk> study is being conducted by the University of Pennsylvania, and we look forward to updates as they become available.

So I'm pleased that designed to provide high maintain levels of antibody responses.

Our partner Apollo buyer is also conducting a phase III trial for <unk> 3100, as a therapeutic treatment and cervical H cell caused by HPV 16 and 18.

Including neutralizing antibodies, while continuing to generate T cell responses.

The first TNMP to enter clinical trials INR 61, 72 is a preventative HIV vaccine candidate in a phase one trial sponsored and funded by <unk>.

A phase II trial sponsored by the eighth Malignancy consortium is also underway evaluating <unk> steady 100, and HIV positive participants with annual H sale caused by HPV 16 and 18.

And finally western partners from Astrazeneca, the University of Pennsylvania, and Indiana University.

Leading the phase <unk> trial, using <unk> DNA encoded monoclonal antibody or <unk> technology to develop team apps, the space therapeutic and preventive treatments for COVID-19.

And there is also exciting work advancing next generation DNA medicine technology, including a trial with our collaborators at the Wistar Institute evaluating DNA launch nanoparticles <unk>.

We're excited about what the future holds for these innovative technologies and look forward to sharing more in the year ahead.

So lumpy that designed to provide high maintain levels of antibody responses.

I'll now turn the call over to our CFO, Peter Keith for our fourth quarter and full year 2023 financial summary.

Including neutralizing antibodies, while continuing to generate T cell responses.

Sir.

Thank you Jackie today I'd like to provide an overview of <unk> operational highlights and financial condition for the fourth quarter and full year of 2023.

The first TNMP to enter clinical trials INR 61, 72 is a preventative HIV vaccine candidate in a phase one trial sponsored and funded by <unk>.

As Jackie Mike and Mark have noted we have made significant progress across our pipeline over the past year advancing key candidates, while working successfully to reduce our operational spend.

And finally western partners from Astrazeneca, the University of Pennsylvania, and Indiana University.

Leading a phase <unk> trial, using <unk> DNA encoded monoclonal antibody or <unk> technology to develop <unk> based therapeutic and preventative treatments for COVID-19.

After our pipeline re prioritization.

As you can see from this slide we have again reduced our total operating spend.

We're excited about what the future holds for these innovative technologies and look forward to sharing more in the year ahead.

<unk> from $56 1 million in the fourth quarter of 2022.

The $27 5 million in the fourth quarter of 2023.

I'll now turn the call over to our CFO, Peter Keith for our fourth quarter and full year 2023 financial summary.

51% decrease or.

Our full year operating expenses were also.

Sir.

Nearly cut in half from $277 8 million for 2022 to $144 8 million for 2023.

Thank you Jackie today I'd like to provide an overview of <unk> operational highlights and financial condition for the fourth quarter and full year of 2023.

Breaking down operating expenses.

As Jackie Mike and Mark have noted we have made significant progress across our pipeline over the past year advancing key candidates, while working successfully to reduce our operational spend.

More.

Our R&D expenses in the fourth quarter of 2023 totaled $17 3 million compared to $42 1 million for the same period in 2022.

After our pipeline re prioritization.

Our full year R&D expenses for 2023 were $86 7 million compared to $187 7 million for the same period in 2022.

As you can see from this slide we have again reduced our total operating spend dropping from $56 1 million in the fourth quarter of 2022 to $27 5 million in the fourth quarter of 2023.

The year over decrease year over year decrease in R&D expenses was primarily the result of lower drug manufacturing.

51% decrease or.

Clinical trial expenses.

Our full year operating expenses were also.

Outside services and Expensed inventory related to INR 4800, and other COVID-19 studies and lower employee and consulting compensation.

Nearly cut in half from $277 8 million for 2022 to $144 8 million for 2023.

Including stock based compensation among other variances.

Breaking down operating expenses a bit more.

<unk> expenses for the fourth quarter, 2023 were $10 2 million compared to $14 million for the same period in 2022.

R&D expenses in the fourth quarter of 2023 totaled $17 3 million compared to $42 1 million for the same period in 2022.

G&A expenses for the full year of 2023 were $47 6 million compared to $90 2 million for the same period in 2022.

Our full year R&D expenses for 2023 were $86 7 million compared to $187 7 million for the same period in 2022.

Yes.

Revenues for the fourth quarter of 2023 were 103000 compared to 125000 for the same period in 2022.

The year over decrease year over year decrease in R&D expenses was primarily the result of lower drug manufacturing.

Revenues.

Clinical trial expenses.

For the full year of 2023 were 832000 compared to $10 3 million for the same period in 2022.

Outside services and Expensed inventory related to INR 4800, and other COVID-19 studies and lower employee and consultant compensation.

Note that revenues reported for 2022 was associated with a procurement contract with the U S Department of defense for <unk> devices and accessories to be used for the delivery of INR 4800, our COVID-19 vaccine candidate, which we have since discontinued these.

Including stock based compensation among other variances.

G&A expenses for the fourth quarter, 2023 were $10 2 million compared to $14 million for the same period in 2022.

G&A expenses for the full year of 2023 were $47 6 million compared to $90 2 million for the same period in 2022.

These factors combined to bring our net loss for the fourth quarter of 2000 $23 million to $25 million or $1 10 per share basic and dilutive.

Yes.

Revenues for the fourth quarter of 2023 were 103000 compared to 125000 for the same period in 2022.

And our net loss for the full year 2023 to $135 1 million or $6 <unk> per share basic and dilutive.

Revenues.

We finished the fourth quarter of 2023 was $145 3 million in cash cash equivalents and short term investments compared to $253 million as of December 31, 2022.

For the full year of 2023 were 832000 compared to $10 3 million for the same period in 2022.

<unk> cash runway to extend into the second quarter of 2025.

This projection.

<unk> and operational net cash burn estimate of approximately $26 million for.

For the first quarter of 2024, this amount excludes repayment of $17 million in remaining principal and accrued interest on our convertible senior notes that matured on March one 2024 and.

These factors combined to bring our net loss for the fourth quarter of 2000 $23 million to $25 million or $1 10 per share basic and dilutive.

And our net loss for the full year 2023 to $135 1 million or $6 <unk> per share basic and dilutive.

Including the repayment the total net cash burn for the first quarter of 2024 is expected to be approximately $43 million.

We finished the fourth quarter of 2023 with a $145 3 million in cash cash equivalents and short term investments compared to $253 million as of December 31, 2022.

These cash runway projections do not include any funds that may be raised through an app the mark at the market program or other capital raise activities.

As a reminder, you can find our full financial statements in this afternoon's press release as well as in our Form 10-K filed with the SEC.

<unk> cash runway to extend into the second quarter of 2025.

This projection.

<unk> and operational net cash burn estimate of approximately $26 million for.

And with that I'll turn it back over to Jacky.

Thanks, Peter I'd now like to open up the call to answer any questions you might have.

For the first quarter of 2024, this amount excludes repayment of $17 million in remaining principal and accrued interest on our convertible senior notes that matured on March one 2024 and.

Operator.

Thank you ladies and gentlemen, we will now begin the question and answer session.

So do you have a question. Please press the star followed by the one on your Touchtone Paul.

Including the repayment of the total net cash burn for the first quarter of 2024 is expected to be approximately $43 million.

You'll hear a prompt acknowledging your request.

Should you wish to decline from the polling process. Please press the star followed by the two.

You are using a speaker phone please lift the handset before pressing in the keys.

These cash runway projections do not include any funds that may be raised through an aftermarket aftermarket program or other capital raise activities.

Your first question comes from the line of her Taj Singh.

<unk> with Oppenheimer <unk> company. Please.

Please go ahead.

As a reminder, you can find our full financial statements in this afternoon's press release as well as in our Form 10-K filed with the SEC.

Great. Thank you. Thanks for the questions I have a couple actually if you don't mind.

One is I know that the confirmatory trial has been in discussion with the FDA for a little bit.

And with that I'll turn it back over to Jacky.

Thanks, Peter I'd now like to open up the call to answer any questions you might have.

The fact that I believe you are going to start that sometime last year early this year on.

Operator.

The FDA.

Thank you ladies and gentlemen, we will now begin the question and answer session.

Granted accelerated approval process can you, maybe just give us some color on what that looks like and especially how long would it take to recruit.

Should you have a question. Please press the star followed by the one on your Touchtone Paul.

The valuation period, roughly when it could readout and began we are not looking for any guidance.

You'll hear a prompt acknowledging your request.

And then secondly.

Should you wish to decline from the polling process. Please press the star followed by the <unk>.

We talked to a lot of Kols.

Vault is.

You are using a speaker phone please lift the handset before pressing in the keys.

<unk> therapy in ERP.

Thank you Rob we are planting a lot of patients.

Your first question comes from the line of her Taj Singh.

Theres academics literature that suggests that.

<unk> with Oppenheimer <unk> company. Please.

Cost of surgeries 70 to $100000 per patient per year, but it adds up over time, just how you're thinking of pricing comps.

Please go ahead.

Great. Thank you. Thanks for the questions I have a couple actually if you don't mind.

So just any color there or any thoughts there again back to the questions.

One is I know the confirmatory trial has been in discussion with the FDA for a little bit.

Good afternoon <unk>.

Great questions. So.

And I believe you are going to start that sometime last year early this year.

I think we covered and Nicole.

And al BTT multi disciplinary meeting that we held with the FDA just before Christmas we got guidance on the number of different elements that needed to go into our BLA submission and as a part of that with getting some further guidance on the design of that confirmatory trial, so I'm going to hand over to Mike.

The FDA.

Granted accelerated approval process can you, maybe just give us some color on what that looks like and especially how long would it take to recruit.

The valuation period and roughly when it could readout and began we're not looking for any guidance.

And then secondly.

We talked to a lot of Kols.

To provide a bit more detail there and then perhaps mark Kaye can talk about some of the comparative from some clients thing that Mike.

About.

Therapy in an RFP.

Thank you Rob we are planting a lot of patients.

Theres academics literature that suggests that.

Yes, thanks, Jackie and tie how touch.

With respect to the confirmatory trial I think we are in the final stages with discussing the details with the agency.

Cost of surgery, 70 to $100000 per patient per year, but it adds up over time, just how you're thinking of pricing comps.

So I am hoping we will soon be in a position to.

So just any color there or any thoughts there again back to the questions.

Revealed sort of details around AD design and future strategy.

Good afternoon, Hi, Josh that's a great question so.

You also asked about sort of recruitment.

I think we covered and Nicole.

Readouts.

And all our BTT multi disciplinary meeting that we held with the FDA just before Christmas we got guidance on a number of different elements that needed to go into our BLA submission and as part of that with getting some further guidance on the design of that confirmatory trial, so I'm going to hand over to Mike.

Until we get the final on the sample size, it's difficult to totally predict how long, it's going to take but what I. What I can say is we included eight clinical trial sites in our original phase one two study.

We're certainly going to go broader than that.

Confirmatory study.

To provide a bit more detail there and then perhaps mark Kaye can talk about some of the comparisons until at some clients thing that Mike.

And I think we've talked consistently about the patient need and.

Openness to receive treatment and non surgical option.

Yes, thanks, Jackie and tie how touch.

With respect to the confirmatory trial I think we are in the final stages with discussing the details with the agency.

I'm really hoping that we'll be able to recruit the subjects pretty quickly.

Again with respect to readout, that's really going to depend on what the final final design and so.

So I am hoping we will soon be in a position to.

Revealed sort of details around AD design and future strategy.

Length of observation that weekend that need to include.

You also asked about sort of recruitment.

Hopefully next quarterly call I'll be able to provide some more color around that.

Readouts.

Until we get the final on the sample size, it's difficult to totally predict how long it's going to take.

Thanks, Mike Mark on them on the pricing structures <unk> good to talk to you soon.

What I can say is we included eight clinical trial sites in our original phase one two study with.

I think what we've said previously is that <unk>.

<unk> is a rare disease, and we would anticipate rare disease pricing for this asset.

We're certainly going to go broader than that.

I think with that said, though.

Confirmatory study.

There are a number of rare disease analog some of which also are positioned as alternatives to surgery.

And I think we've talked consistently about the patient need and.

Openness to receive treatment a non surgical options.

We're assessing as a part of the pricing work that we're doing.

Really hoping that we'll be able to recruit subjects pretty quickly.

And I would say that as we get closer to launch will be in a better position to say more about that.

Again with respect to readout that that's really going to depend on what the final final design and so.

Mark.

Can I just have a quick follow up on just that.

Will you be publishing Pharmacodynamic data also as you get closer to launching or maybe after that.

Length of observation that weekend that need to include.

130, 107, and thanks for all the questions.

But hopefully next quarterly call I'll be able to provide some more color around that.

I think on <unk> I'm going to direct that question to Mike Mike would you like to respond to that yes, certainly I mean.

Thanks, Mike Mark on them on the pricing of <unk>.

<unk> good to talk to you. So I think what we've said previously is that RP is a rare disease and we would anticipate rare disease pricing for this asset I think with that said, though.

We obviously didn't include too many quality of life or impact on the patients in our phase one two study I mean, we will we are suddenly in the process of assessing how we can document the impact too.

There are a number of rare disease analog some of which also are positioned as alternatives to surgery.

RFP on patients and we will most likely include more measures.

Respectively in that confirmatory study soon.

We're assessing as a part of the pricing work that we're doing.

Then we will have I mean, obviously, we will aim to.

And I would say that as we get closer to launch we'll be in a better position to say more about that.

Have supporting evidence for the price that we eventually set.

Mark.

Can I just have a quick follow up on just that.

Okay. Thank you all.

Will you be publishing Pharmacodynamic data also as you get closer to launching or maybe after that.

Questions.

Comes from the line of Roy Buchanan with citizens.

131, with seven and thanks to all the questions.

I think on <unk> I'm going to direct that question to Mike Mike would you like to respond to that yes, certainly I mean.

Please go ahead.

Hey, Great I guess, maybe just sticking a 31 seven for a second then has a few others.

Yes.

State commercial scale manufacturing, how many patients do you think you can address in 2025, we think he can address the entire park.

Population or just what are you thinking for initial penetration.

RFP on patients and we will most likely include more measures.

Yes.

Do you want to take that one sure.

Take a whack at that and then if there is some additional comments feel free to add I guess the way I'd respond to that question is as you would expect we've done.

Prospectively in that confirmatory study soon.

We will have I mean, obviously, we will aim to.

Sort of a lot of modeling and we've been working closely with our with our manufacturing team in.

Have supporting evidence for the price that we eventually set.

So we're confident that sort of based on our our market projections.

Okay. Thank you all.

<unk>.

Estimates around adoption is etcetera, etcetera that will be able to fully meet the demand for for $31 seven.

Questions.

Comes from the line of Roy Buchanan with citizens.

Yeah, and if I can just add on the manufacturing side.

Please go ahead.

Hey, Great I guess, maybe just sticking a 31 seven for a second then has a few others.

<unk> already got two commercial scale up some other product candidates.

And this is a rare disease in terms of dose requirements. The actual numbers of dose requirements, it's very manageable for us from a manufacturing standpoint.

Yes.

<unk> commercial scale manufacturing how many patients do you think you can address and in 2025 do you think he can address the entire.

Population or just what are you thinking for initial penetration.

So I think we're pretty well positioned from a manufacturing standpoint to be able to build up the launch inventory and be able to support the market need is that last couple of years pretty easily.

Yes.

Do you want to take that one sure.

Take a whack at that and if there is some additional comments feel free to add I guess the way I'd respond to that question is as you would expect we've done.

Alright, perfect switching to the pipeline deeper pipeline.

Sort of a lot of modeling and we've been working closely with our with our manufacturing team in.

I guess for 31 12, 18, you could tell us about the design that you submitted for that.

So we're confident that sort of based on our our market projections.

<unk>.

MS. <unk> do you expect that it can be registrational.

Estimates around adoption is etcetera, etcetera that will be able to sort of fully meet the demand for for $31 seven.

Itself and then the last slide deck, you had eight additional clinical stage candidates can you just help us understand a bit what.

Yeah, and if I can just add on the manufacturing side.

<unk> already got two commercial scale up some other product candidates.

I guess level of development you can do for those candidates given the runway into in the <unk> 25, I guess, what does that runway assume in terms of what you can develop out of the pipeline beyond <unk> 107. Thanks.

And this is a rare disease in terms of dose requirements the actual number.

Yeah, that's great. So.

Mike Why don't you take the 31 12 phase III design and what we're thinking down up then I can talk about the rest of the pipeline candidates.

Yeah. Thank you.

So.

As you heard in the call today, we clearly sort of articulated the patient population that we are going after the high risk.

Patients with local or regionally.

Advanced disease and high risk really comes down to based on tumor size local infiltration local node involvement and also smoking history.

So I think we did a lot of work with Kols on a global basis to to define that population.

I mean, as we think of developing this candidate I mean based on the well known safety profile of PD, one inhibitors and also the well documented safety profile of <unk> DNA medicines, we certainly feel we can.

Do a registration study, obviously that will ultimately be decided by the agency.

And as we as we look to that design, we followed very traditional ways. I mean, obviously the agency is going to want to understand the contribution of components.

That is clearly an element of the design, we submitted to the agency.

And so we're really waiting to have that discussion with them in quarter two two.

And what our actual pathway for would really looks like.

Thanks, Mike.

Just to talk about the rest of the pipeline as I mentioned on the call clearly we are focusing the majority of our internal resources on advancing.

<unk>, 31% to 7% fluids license here and then the launch hopefully next year.

And leaving 30 months fall forward.

We do have a number of partnerships in place with the other assets in our pipeline and we're going to be leveraging those partnerships and non diluted funding mechanisms ground's et cetera to be able to help me if those candidates forward and some of the earlier stage work, particularly the preclinical work we can always.

As I move forward ourselves. So we think we have ways leveraging those partnerships non dilutive funding mechanisms of moving those candidates forward.

To generate some meaningful progress as well.

Okay. Thanks for taking the questions.

Your next question comes from the line of Gregory <unk> with RBC capital markets. Please go ahead.

Hi, guys. Its an issue on for Greg. Thanks for taking my questions. Just a couple for me Firstly on 30 107 in RP.

I'm thinking about thinking ahead to market shaping what some of the pushback you've gotten to date when engaging kols on getting patients onto a therapy like <unk> 37 versus standard of care surgery, and how has that feedback been built into your awareness and education directives and secondly, a question on 31 12, if you could just walk us through how the collaborations.

<unk> came to be the selection of walk towards he is a combination candidate among other potential candidates and how you can best leverage the partnership going forward. Thanks again.

Yeah, Thanks, Sidney staff really great candidates.

Mark's comments on how we are thinking about.

31 <unk>.

Mike maybe you'd like to comment on some of the clinical aspects. There, yes. Thanks, Jackie I guess, the first thing I'll say from a commercial perspective I've never been more excited about launching a new product.

I think it speaks to specifics I am going to address your question.

We've conducted multiple advisory boards with both Hep's theyre treating RP as well as sort of patients we've been.

Closely aligned with listening to what the ERP Foundation suggest is important for probably the last three or four years and one of the things that we've heard is that physicians don't like doing this surgery right, they're looking for an alternative to surgery.

As alluded to I mean surgery doesn't address the underlying infection rates in this cycle of surgical procedure after surgical procedure and that's actually the surgery this driving that morbidity.

So they're looking for an option right and this is sort of unlike other.

Launches, where you're replacing a surgical.

Alternative in that.

Physicians are welcoming it right, there's no disincentive for them to use the service.

107, because the current standard of care is holding an adequate.

And similarly from a from a patient perspective.

As Mike mentioned, it's a devastating disease and even with the reduction of sort of one surgery.

<unk> can be life changing for them so im.

I'm really really confident.

Everything that we've learned about this marketplace pretends that.

We're going to have a really successful launch with 31 seven.

Thanks, Mike cumulative talk about some of those clinical aspects and the needs that the patients and the physicians are highlighting.

Yes, absolutely.

As you heard me talk about today I mean, the latest data published by Simon burst from John Hopkins around the ability to cause permanent local code damaging is really in the mind of all the sort of RFP surgeons, we've spoken to they recognize.

The new while surgery is hugely beneficial to the patients.

They understand that sooner or later, there is going to be damage caused and so from a surge or toll perspective as mark said.

They really would they are just as excited as the patients I think for looking for a non surgical treatment for these patients.

<unk>.

I keep going back to sort of having discussions with Kim Mcclellan.

Patients and the physicians are highlighting.

It really is.

Yes, absolutely.

As you heard me talk about today, I mean that the latest data that was published by a sudden burst from John Hopkins around the ability to cause permanent vocal cord damaging is really in the mind of all the sort of RFP surgeons, we spoken to they they recognize.

Every surgery, just impacts a patient's life.

And so you saw on the graph I showed earlier the tremendous impact we've had on reducing surgical burden in the <unk> population.

And so once again I really feel.

While surgery is hugely beneficial for the patients.

You see that clinical efficacy in <unk> associated with that.

The safety data we have accomplished.

They understand that sooner or later that there is going to be damage caused and so from a surgery coal perspective as Mark said.

Accumulated on 31% and seven to date.

There really isn't any any reason that we've heard from the patients of why they they wouldn't want a non surgical option.

They really would they are just as excited as the patients I think for looking for a non surgical treatment for these patients.

We're very excited to move this program forward.

Hey, Mike can I add one other point real quick.

<unk>.

One other sort of really important dimension of the patient component of this market.

I keep going back to sort of having discussions with Kim Mcclellan.

Unlike in other rare diseases and when I was at sort of Genzyme. This was the case you spend a lot of effort identifying patients and getting them to originate for for therapy.

It really is.

Every surgery, just impacts a patient's life.

And so you saw on the broth I showed earlier the tremendous impact we've had on reducing surgical burden in the P population.

That won't be the case with RFP I mean, these are patients who originate for treatment and the primary reason is because the.

The impact on voice quality right. So.

And so once again I really feel.

Again, the combination of patients who originate four for treatment for RFP.

You see that clinical efficacy in <unk> associated with the.

The safety data we have accomplished it.

Fact that surgery is inadequate and burden and causes a lot of morbidity and that physicians are looking for alternative really puts us in a good position with 31 seven.

Accumulated on 31 or seven to date, there really isn't any any reason that we've heard from the patients of why they they wouldn't want a non surgical option.

No I think those are excellent points mark.

We're very excited to move this program forward.

Moving onto 31 smiles. So I think maybe if we just take a step back here for 31 12, we saw a really encouraging data both as a monotherapy and in combination with Allomap.

If I can I can add one other point real quick.

One other sort of really important dimension of the patient component of this market is that unlike in other rare diseases and when I was at sort of Genzyme. This was the case you spend a lot of effort identifying patients and getting them to originate for for therapy.

And whilst we were disappointed that astrazeneca decided not to move.

That won't be the case with RFP I mean, these are patients who originate for for treatment and the primary reason is because the.

So they map forward in head and neck space for.

I'm excited by what we saw in that study and it really goes back to one of the strengths of the platform our ability to generate these antigen specific cytotoxic CDA T cells that we now are capable of getting to the tumor and we know in some of our other HPV.

The impact on voice quality right. So you know it.

The combination of patients who originate four for treatment for RFP. The fact that surgery is inadequate burden and causes a lot of morbidity and that physicians are looking for alternative really puts us in a good position with 31 seven.

<unk> programs are capable of clearing HPV, precancerous lesions and leading to viral clearance.

No I think that's our excellent points Mark.

So when we wanted to move 31 12 forward, we saw a lot of promise.

Leaving.

Moving onto 31 small so I think maybe if we just take a step back cat 431 swap we saw a really encouraging data both as a monotherapy and in combination with divide them up.

For 31, 12, and we really cannot take quite an extensive analysis looking for the right.

PD, one to using combination and Mike maybe not at the point, where you might want to jump in and talk about some of the reasons why we're so excited about volatility.

And you know Paul we were disappointed that Astrazeneca decided not to move.

They map forward in head and neck space.

No absolutely I mean.

We're excited by what we saw in that study and it really goes back to one of the strengths of the platform our ability to generate these antigen specific cytotoxic CDA T cells that we now are capable of getting to the tumor and we know we announced some of our other HPV dysplasia programs.

Okay.

Ducting any clinical partnership when they need it.

First of all is finding a partner Thats just excited about the program as you are and look.

<unk> works with a lot of the same kols.

That will hopefully eventually prescribed 31 12.

Ah capable of clearing HPV precancerous lesions and they think to viral clearance.

<unk>.

It gives them a unique insight into seeing the value of what we see with 30 112 and look towards.

So when we we wanted to move 31 12 forward, we saw a lot of promise.

And so.

As a partner.

For 31, 12, and we really cannot take quite an extensive analysis looking for the right.

Great to work with they share the enthusiasm for the program, having a very recent approval through a nasopharyngeal carcinoma.

PD one to using.

Combination.

Mike maybe not at the point, where you might want to jump in and talk about some of the reasons why we're so excited about an opportunity.

I think is.

They want to also see the use of look towards the expand.

No absolutely I mean.

And so.

So far everything every interaction we've had with coherence has been very positive they bring.

Conducting any clinical partnership I mean first of all is finding a partner Thats just too excited about the program as you are and the fact that.

Knowledge of the head and neck space to the table as well and so we're just really very encouraged to continue with that partnership and perform the clinical trial.

Look towards he works with a lot of the same kols.

That will hopefully eventually prescribed 31 12.

<unk>.

It gives them a unique insight into seeing the value of what we see with 31 12 and look towards.

Yes.

Thanks, Chris.

Your next question comes from the line of Roger song with Jefferies. Please go ahead.

And so.

As a partner.

Great to work with they share the enthusiasm for the program, having a very recent approval through a nasopharyngeal carcinoma I think is.

Hey, good afternoon, basically downtown core Roger Thank you for taking our questions.

<unk> pharma us.

So regarding to the confirmatory study of $31 seven could you comment on how thinking.

They want to also see the use of look towards the expand.

Thinking about some of the care practice among the different.

And so.

So far everything every interaction we've had with <unk> has been very positive they they bring them.

Site, how do you see that impact.

Study results.

And how.

At this point, what the powering assumptions that they are thinking about for the compound.

Knowledge of the head and neck space to the table as well and so we're just really very encourage to continue with that partnership and perform the clinical trial.

Thank you.

Yeah, Mike can you take that one.

Absolutely.

Thanks Roger.

So we cant yet obviously you talk about the sample size elements I can certainly address the clinical trial site elements.

Yes.

Thanks, guys.

Your next question comes from the line of Roger song with Jefferies. Please go ahead.

In our phase one two study, we actually had eight recruiting side.

Hey, good afternoon, Victor downtown core Roger Thank you for taking our question.

Sites.

And.

The results.

While we only had 32 subjects in the phase one two study we didn't we didn't analysis. There was suddenly no site to site variation. So we believe the results will be very consistent.

Yes for a mark.

So regarding to the confirmatory study of 31 or Kevin could.

Could you comment on how.

Thinking about 10 other care practice the amount of different.

As we look to utilize more sites in a confirmatory study.

Right.

Do you see that impact study.

Study results.

And how.

So.

That is certainly not a concern for us.

At this point, what Florida, what the powering assumptions that you are thinking about for the compliance with study. Thank you.

And obviously as we look to.

Sites using.

Yeah, Mike can you take that one.

PSP device, we have a lot of experience from our previous phase III program with 3100. So again that is of any concern to us.

Absolutely.

Thanks Roger.

So we cant yet obviously you talk about the sample size elements I can certainly address the clinical trial site elements.

Mike maybe I can.

I didn't hear it.

In addition, working with eight sites, we will send more patients across the disease spectrum and we also enrolled patients with both HPV six <unk> 11, So I think <unk> in a phase <unk> study, we built up a good understanding of that variability within that patient population.

We in our phase one two study, we actually had eight recruiting sites.

And.

The results soon.

While we only had 32 subjects in the phase one two study we didn't we didn't analysis. There was certainly no site to site variation. So we believe the results will be very consistent.

Absolutely.

As we look to utilize more sites in a confirmatory study.

Thank you maybe another question if I may so regarding the 31 was seven phase one two data.

That that that is certainly not a concern for us.

One key difference from.

And obviously as we look to.

Studies that included the globe 13th right after <unk>.

Sites using.

Including through the treatment window, so maybe could you remind us what do you see if you do not only.

By PSP device, we have a lot of experience from our previous phase III program with 3100. So again that is of any concern to us.

Only includes categories after involved.

After the treatment window.

Mike maybe I can arm.

Yeah.

I didn't hear it.

Great question Amit.

In addition to working with eight sites will send more patients across the disease spectrum and we also enrolled patients with both HPV six on HPV 11. So I think you know in our phase <unk> study, we built up a good understanding of that variability within that patient population.

Design this trial because every surgery matters to the patients that spoke we've had time after time from the patient separately surgery matters.

Mike do you want to talk about the data because I'm not sure if we've disclosed that yet if it's published.

No. We haven't provided any any details of that and I think also if we were going to do a like to like comparison.

Oh absolutely.

Thank you maybe another question if I may so regarding the 30, while <unk> phase one two data.

We want to include.

A full 52 weeks after excluding any synergies from.

One key difference from well, particularly in studies that included adult upper gains right. After.

The surgical window.

Including through the treatment window, so maybe could you remind us what do you see if you do not only.

So I mean.

We will be looking at collecting that data.

That's not going to be available in the short term.

Only include surgeries I'll turn that off.

After the treatment window.

Got it. Thank you that's a diploma.

Yeah.

Great question.

Okay.

Ladies and gentlemen, as a reminder, should you have a question. Please press the star followed by the one on your Touchtone phone.

We designed this trial because every surgery matters to the patients that spoke we've had time after time from the patient separately surgery matters.

Your next question comes from the line of <unk>, Chen with H C. Wainwright.

Mike do you want to talk about the data because I'm not sure if we've disclosed that yet if it's published.

Please go ahead.

Thank you for taking my questions.

No. We haven't provided any any details of that and I think also if we were going to do a like to like comparison, we'd obviously want to include.

Could you comment on the typical age range of the RFP patients or what percentage of patient population that actually are on Medicare and how much time do you think it will take to secure Medicare reimbursement followings.

A full 52 weeks after excluding any surgeries from.

Following potential FDA approval in 2020 clients. Thank you.

The surgical window.

So I mean.

Yeah, Great question. So Mike do you want to talk about the age range and then Mark maybe you can take the Medicare question.

We will be looking at collecting that data, so, but that's not going to be available in the short term.

Yeah.

<unk> so.

RFP in folds all age age groups.

Got it. Thank you that's it from us.

And there is actually sort of three peaks to the instance, so the first one instance of pediatric repeat which usually is around the sort of five to seven year old.

Ladies and gentlemen, as a reminder, should you have a question. Please press the star followed by the one and you touched on the phone.

Your next question comes from the line of <unk>, Chen with H C. Wainwright.

Mark.

There is a earlier adult peak, which obviously follows.

Please go ahead.

Thank you for taking my questions.

Thanks, well activity onset and Thats, usually around the age of 35 and now what is also developed is a later onset peak, which is usually around the age of 65. So it really encompasses all age groups.

Could you comment on the typical age range.

The RFP patients or what percentage of patient population that actually are on Medicare.

How much time do you think it will take to secure Medicare reimbursement.

Following potential FDA approval in 2025, thank you.

But the vast majority is still.

Yeah, Great question. So Mike do you want to talk about the age range and then Mark maybe you can take the Medicare question.

Actually not the vast majority the majority is still around the 35.

Each group with the elderly population growing rapidly.

Yeah.

<unk> so.

RFP and involves all age age groups.

Hi, its mark and Mark Thanks for the thanks for the question, it's really good question.

There is actually sort of three peaks to the incidence. So the first one is the pediatric repeat which usually is around the sort of five to seven year old.

I think what I've said earlier in my comments is that sort of focus in Brazil is going to be key for our success.

Mark then there is a earlier adult peak, which.

Consistent with that particularly as it pertains to kind of what the.

Mix is.

<unk> follows.

For these patients private versus versus Medicare Medicaid.

Actual activity onset and thats, usually around the age of 35 and now what is also developed is a later onset peak, which is usually around the age of 65. So it really encompasses all age groups.

We're going to get a lot of additional information there from this work that I mentioned earlier not only are we going to be assessing.

Physician landscape, but the analysis all starts from the patient level, and we will be able to sort of identify.

Where claims are being adjudicated, whether it's been adjudicated in the private sector or in the.

But the vast majority is still.

Actually not the vast majority the majority is still around the 35 H.

Medicare or Medicaid and that are really kind of help us focus our efforts on both the commercial payers that are important but also to help us understand.

Age group with the elderly population.

Growing rapidly.

What our approach needs to be from the perspective of Medicare and Medicaid.

Hi, It's Mark Hey, Mark Thanks for the thanks for the question, it's really good question.

There are really three pillars that we're focusing on four for commercial launch.

I think what I've said earlier in my comments is that sort of focus on Brazilian is going to be key for our success.

Distribution.

And then the market access piece as well as field deployment. So we're moving as fast as we can.

Distant with that particularly as it pertains to kind of what the.

Mixes.

I can't give you a specific timeline for when only to say that we.

For these patients private versus versus Medicare Medicaid.

We're going to get a lot of additional information there from this work that I mentioned earlier not all.

We started early with the objective of.

Making sure that patients both.

Or are we going to be assessing sort of physician landscape.

Private and in Medicare Medicaid have access to INR 31, <unk> as quickly as possible.

The analysis all starts from the patient level, and we'll be able to sort of identify.

Thank you could you comment on the regulatory pathway going forward in the European Union.

Where claims are being adjudicated, whether its has been adjudicated in the private sector or in the Medicare.

Medicare or Medicaid and that are really kind of help us focus our efforts on both the commercial payers that are important but also to help us understand.

Hi.

Yes, so we have obviously engage with the European.

European Union.

What our approach needs to be from the perspective of Medicare and Medicaid.

We have not yet disclosed.

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There are really three pillars that we're super focusing on four for a commercial launch.

What the strategy is going to be with with Europe.

We believe there's a pathway forward based on the information we have.

Distribution.

And then the market access piece as well as field deployment. So we're moving as fast as we can.

<unk> received and I think we'll be able to give more details.

I can't give you a specific timeline for when only to say that.

In upcoming calls.

Okay. Thank you.

We started early with the objective.

Yeah, if I can just add Mike.

Making sure that patients both.

It does.

Private and in Medicare Medicaid have access to INR 31, <unk> as quickly as possible.

<unk>. Unfortunately, it <unk>, it's a pretty bad there is strong.

Unmet medical need as well in Europe with the standard of care may differ slightly from from the U S. So that's.

Thank you could you comment on the regulatory pathway going forward in the European Union.

Wholesale important consideration to take into account.

Mike.

We'll certainly be trying to provide some more details on losses as our discussions with the European regulators progress.

Yeah. So we have obviously engage with the European.

European Union.

We have not yet disclosed.

Okay. Thank you.

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Okay.

What that strategy is going to be with with Europe.

Ladies and gentlemen, there are no further questions at this time I will now turn the call back over to Jacky Shea President and CEO for closing remarks. Please go ahead.

We believe there's a pathway forward based on the information we have.

<unk> received and I think we'll be able to give more details.

In upcoming calls.

Thank you.

I open this call by highlighting the transformation of Nokia has done is gone after last year at transformation made possible by our renewed strategic focus on the strengths of our DNA medicines platform and our aim to become a commercial stage company.

Okay. Thank you.

Yeah, if I can just add Mike Subsidised.

Our RPM Fortunately, it's slide <unk>, it's a pretty bad there was strong.

Unmet medical needs as well in Europe with the standard of care may differ slightly from from the U S. So that's wholesale important consideration to take into account.

We have made more than just progress.

We have built a foundation for long term success that we will continue to build on.

And we have advanced DNA medicines to patients around the world that could potentially benefit from its promise.

Well certainly be trying to provide some more details on losses as our discussions with the European regulators progress.

I look forward to sharing more updates on progress with you in the year ahead.

Okay. Thank you.

With that thank you again for your attention and have a great evening everyone.

Okay.

Ladies and gentlemen, there are no further questions at this time I will now turn the call back over to Jacky Shea President and CEO for closing remarks. Please go ahead.

Ladies and gentlemen, this concludes your conference call for today, we thank you for participating and ask that you. Please disconnect your lines.

Thank you.

I open this call by highlighting the transformation of Nokia has undergone after last year.

Our transformation made possible by our renewed strategic focus on the strengths of our DNA medicines platform and our aim to become a commercial stage company we.

We have made more than just progress.

We have built a foundation for long term success that we will continue to build on.

And we have advanced DNA medicine to patients around the world that could potentially benefit from its promise.

I look forward to sharing more updates on progress with you in the year ahead.

With that thank you again for your attention and have a great evening everyone.

Ladies and gentlemen, this concludes your conference call for today, we thank you for participating and ask that you. Please disconnect your lines.

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