Q4 2020 Apellis Pharmaceuticals Inc Earnings Call

Ladies and gentlemen, Christmas day part.

Operator: Ladies and gentlemen, thank you for standing by. Your conference call will begin momentarily. Again, thank you for standing by. Your conference call will begin momentarily. Thank you.

On the conference call shall begin momentarily I can't think of the standby. The conference calls have begin momentarily. Thank you.

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Valerie: Good afternoon. My name is Valerie, and I will be your conference operator today. At this time, I'd like to welcome everyone to the Apellis Pharmaceuticals fourth quarter and full year 2020 fiscal results conference call. Today's call is being recorded, and a replay will be available at Apellis.com. I will now turn the call over to Tracy Benitez, Vice President of Communications at Apellis.

Good afternoon, my name of salary and I'll be your conference operator today at this time I would like to welcome everyone should that tell us the pharmaceuticals fourth quarter and full year 2020.

The scope at the conference call.

Tracy Benitez: Thank you, Valerie. Good afternoon, and thank you for joining us today to discuss Apellis's fourth quarter and full year 2020 financial results. With me on the call are co-founder and Chief Executive Officer Dr. Cedric Francois, Chief Medical Officer Dr. Federico Grossi, Chief Commercial Officer Adam Townsend, and Chief Financial Officer Timothy Sullivan.

Today's call is being recorded and a replay will be available out of pellet dotcom.

I would now like to turn the call over to trace the Vineet Vice President of communications on a pallet.

Thank you Valerie good afternoon, and thank you for joining us today to discuss the palaces fourth quarter and full year 2020 financial results with me on the call are co founder and Chief Executive Officer, Dr. Cedric Francois Chief Medical Officer, Dr. Federico Grossi, Chief Commercial Officer, Adam Townsend and Chief financial.

Tracy Benitez: Before we begin, I would like to point out that we will be making forward-looking statements that are based on our current expectations and beliefs. These statements are subject to certain risks and uncertainties, and our actual results may differ materially. I encourage you to consult the risk factors discussed in our SEC filings for additional detail.

<unk> Timothy Sullivan.

Before we begin I would like to point out that we will be making forward looking statements that are based on our current expectations and beliefs. These statements are subject to certain risks and uncertainties and our actual results may differ materially I encourage you to consult the risk factors discussed in our SEC filings for additional detail.

Cedric Francois: Now, I'm pleased to turn the call over to Cedric.

Now I'm pleased to turn the call over to Cedric.

Thank you Tracy and good afternoon to everyone joining us today for our first quarterly conference call.

Cedric Francois: Thank you, Tracy, and good afternoon to everyone joining us today for our first quarterly conference call. 2020 was a defining year for Apellis, marked by positive phase 3 Pegasus data in paroxysmal nocturnal hemoglobinuria, or PNA. The PEGASIS results highlighted the potential of pexitacoplan to elevate the standard of care in PNH and the broad platform potential of targeting C3 for complement-driven diseases. In 2021, we look forward to a transformational year for Appelis as we further build on our global leadership in complement across a broad range of diseases with high unmet needs. As you can see on this slide, our corporate strategy is based on three strategic objectives, and we expect to make significant progress on each of these this year.

2020 was a defining year for at the DS Mark by the positive phase III data in paroxysmal nocturnal hemoglobinuria or P of niche.

The biggest news results highlighted the potential of fixed at the Cook line too.

Elevate the standard of care in P N H and the broad less from potential I'll start with the C. Three core complements driven diseases.

In 2021, we look forward to transformation of the year for Smes as we started the build on our global leadership and complement across a broad range of the diseases with high unmet need.

As you can see on the slide our corporate strategy is based on three strategic objectives, and we expect to make significant progress against each of these this year.

Our first objective is to establish systemic picks at the coupon.

Cedric Francois: Our first objective is to establish a systemic Plexi-etacoplasm. Targeted C3 Therapy as a Disruptive Treatment for Rare Complement-Driven Diseases We have a PDUFA date of May 14th for the potential U.S. approval of Plexidacoblan and PNA, and our Chief Commercial Officer, Adam Townsend, will discuss our work to prepare for a successful commercial launch. We also expect to advance four additional registrational programs for PlexiTacoplan with our partner, Swedish Orphan Biovitrin, or SILVI, as we work to maximize the broad potential of targeting C3.

Target the C three therapy as a disruptive treatments across the rare complements driven diseases.

We havent be due dates of may 14th.

So U S approval of fixed at the Copeland and P of niche and.

And our Chief commercial officer, Adam Townsend will discuss our work to prepare for a successful commercial launch.

We also expect to advance score additional registrational programs of fixed at that group of them with our partner Swedish orphan day, youll be true or sobey as we work to maximize the broad potential of targeting <unk>.

The second the objectives for our company is to be number one in treating retinal diseases.

Cedric Francois: The second objective for our company is to be number one in treating retinal disease. In what will be a seminal event for Apellis, we expect top-line results from our phase 3 clinical studies of flexeta coplan and geographic atrophy, or GA, in the third quarter of this year. GA is a relentless and disabling disease that affects approximately five million people around the world, and we have a unique and exciting opportunity to advance what could become the first drug for people living with GA. Our third objective is to develop new technologies to control complex, Our research team has been very active on a number of fronts, and we look forward to advancing three new product candidates into clinical development by the end of next year. As you can see, we have an Now, let's dive deeper into our first object.

And what will be a seminal event for Applebee's, we expect top line results from our phase III clinical studies of the exit of Copeland and geographic atrophy or <unk> is the third quarter of this year.

G a a relentless and disabling disease that affects approximately 5 million people around the world and we have a unique and exciting opportunity to events what could become the first drug for people living with G.

Our third objective is to develop new technologies to control complements.

Our research team has been very active on the number of slots and we look forward to advancing three new product candidates into clinical development by the end of next year.

As you can see we have an ambitious strategy and several key milestones in 2021 to advance our global leadership of the treatment of complement of driven diseases.

Now, let's dive deeper into arps versus the objectives, establishing systemic picks at the coupon is a disruptive therapies for rare diseases.

Cedric Francois: Establishing systemic pexitacoplam as a disruptive therapy for rare diseases. I will now turn the call over to our Chief Medical Officer, Dr. Federico Grossi, to review our recent data on pexitacopan and PNH.

I'll now turn the call over to our Chief Medical Officer, Dr. Fidel of Eco glossy to review our recent data on the exit that goes on and peonage isn't it.

Federico Grossi: Thank you, Cedric. I'd like to begin by reminding everyone why we believe that Bexeta Coplan has the potential to improve the standard of care for people living with PNH. PNH is characterized by the destruction of oxygen-carrying reptile cells through both extravascular and intravascular hemolysis caused by uncontrolled complement activation. Current treatments inherent to complement cascade downstream at C5, controlling intravascular but not extravascular MR. As a result, while C5 inhibitors offer improvement in patient survival, they do not address many debilitating symptoms from which people living with PNH continue to suffer.

Yeah.

Thank you Cedric.

I would like to begin by reminding everyone why we believe that.

At the top line has the potential to elevate the standard of care for people living with Peonage, DNA, which is characterized by the destruction of boxes in the current rep debt cells through boss extra Boston kind of Intravascular hemolysis.

First by uncontrolled complement activation.

Current treatments and hearing the complement Cascade downstream C. Five.

The controlling intramuscular, but not extra vascular hemolysis.

The result was C. Five inhibitor of <unk> offer improvement patient survival. They do not address many of debilitating symptoms from which people. The one would be an H continued yourself.

Olympics at the copying targets complement centrally.

Federico Grossi: Only Pexeta couple and target complement centrally at C3, controlling both intra and extravascular hemorrhage. As a result, Trixeta Copeland met the primary endpoint in the Phase III Pegasus study and became the first and only investigational therapy to demonstrate superiority compared to Solaris or Eculizumab, with an improvement in adjustment means of 3.8 grams per deciliter of hemoglobin at which as well as The safety profile of Plexivacopter was comparable to Solaris in this. Based on these results, we will receive priority review from the FDA for Plexetacoplin in PNH with a PDUFA date of May 2020. Additionally, in December, we announced top-line data at week 48 from Pegasus Tech.

The controlling both being tracked on the extra vascular hemolysis.

As a result because of the.

Copeland met the primary endpoint in the Phase III Vegas, the study and became the first and only investigational therapy to demonstrate superiority compared to soliris or at least the amount we had.

The improvement in not just the means of three eight grams per deciliter of hemoglobin at week 16.

As well of sustained improvements in all of the key clinical measures.

The safety profile of effects of the cooking was comparable to solar salt life in this thing.

Based on these results we received priority review from the FDA post Brexit the Copeland in P and H with the producer date of May 14.

Additionally, in December we announced top line data with 48 from the Pegasus study.

These long term results show that makes it the Copeland has the potential to help patients gain on.

Federico Grossi: These long-term results show that pexetecoplam has the potential to help PNH patients gain and maintain more complete control of the disease. At week 48, hemoglobin increases were sustained with Bexarthecoplan-treated patients, with a mean improvement from baseline equal to the increase seen at week 16 in Bexarthecoplan-treated patients. Importantly, as you can see in the graph,

Maintain more complete control of the disease.

At week 48, hemoglobin increases were sustained with the exit of cop untreated patients with of many improvement from baseline equal to the increase seen at week 16, and fixed at the corporate untreated patients.

Constantly as you can see on the graph.

Federico Grossi: Solaris-treated patients who switched to Perceptacoplam during the open-label period also experienced sustained improvement in hemoglobin and other key clinical measures compared to simulated patients treated with Paxilla-Tocopan immunotherapy during the randomized control period. Sustained improvements in avoidance, critical site count, lactate dehydrogenase, or LDH levels, and functional assessment of chronic illness therapy, or FASI-FATIG scores, were also observed in patients treated with Bexetacor. At week 48, the safety profile of Paxilotocin was consistent with previously reported data, and no new safety signals were identified. Also in December, at ASH, we presented a Matching Adjusted Indirect Comparison, or MAIC, analysis across the pivotal studies called Pexeta-Coughlin and Altomiris oral regulation. Along with Steve Sites, Inc.

So a lot of treated patients who switched to parts of the Copeland doing the open label period also explained sustained improvement of hemoglobin and all of the key clinical measures simulates the patients treated with Brexit the copper on monotherapy during the randomized control period.

Sustained improvement.

Boyd and political such count.

Take the Hydrogenase LDH levels and functional assessment of chronic illness therapy of fashion, but the scores were also observed in patients treated with fix of the coastline.

A big 48, the checks the profile of Opex at the top line was consistent with previously reported data on.

On non new safety signals.

Thank you Mike.

Also in December.

Cash we presented of matching adjusted in the Euro.

At completion or.

Hey, IC analysis across the pivotal studies called flex it of Copeland on ultra movies or rather in the Qimonda.

Our longer C five inhibitor.

Federico Grossi: In the absence of a clinical head-to-head study, MAIC is a valid and accepted method for comparative effectiveness research used by health technology assessment bodies across the world. The MNIC showed that on pexitocoplan, 76% more patients achieved hemoglobin stabilization compared to patients on ultramarine. Also, 64 more patients than Pexeta-Coplan achieved LDH normalization. This is remarkable because LDH is a biomarker of intravascular molecules. Thank you.

In the absence of clinical head to head study and May I see it's the ballot and accepted method for comparative effectiveness and that's true.

Search used by health technology assessment bodies across the world.

The MAA as the show that Opex of the Copeland, 76% more patients achieved from loving the stabilization compared to patients on the ultra mirrors.

Also 64 more patients infected the captain.

LDH normalization of this is remarkable because LDH is the biomarker of Intravascular hemolysis.

Federico Grossi: Well.

Federico Grossi: Additionally, of major importance to patients' quality of life, 71% more patients treated with hexatococcal were transfusion-free.

Corporate policies the C five inhibitor like called the Myers control well.

Federico Grossi: And there was a 9-point difference in the Thracipatec score of Paxil-Percoplan over Ultimatum. These results reinforce the potential of dextrotocopin to elevate the standard of care by targeting, As with other MNIC analyses, matching may not adjust for confounding factors due to differences inherent in study design and entry criteria. I'd like now to move to our ongoing phase three brainstorming.

Additionally, on major quantum two patients quality of life.

71% more patients treated with picks of the cotton were transfusion free.

And there was a nine point difference on the profit tactics core aspects of the Copeland Cobra automakers.

These results reinforce the potential effects of the coke and elevate the standard of care by targeting the secret.

Federico Grossi: While we believe peptotocopin has the potential to receive broad label based on the results from Pegasus, the print study evaluates peptotocopin in patients that are more representative of the overall treatment-naive population. On this slide, you can see the print study design. Fifty-three treatment-minded PNH patients were enrolled. The two primary endpoints evaluated after 26 weeks are hemoglobin stabilization in the absence of transitions and reductions in LDH. As noted in the slide, patients in the control group have the option to escape to Pexeta-Coupland if their hemoglobin levels drop by two or more grams per deciliter below their baseline.

How should we called on EMEA I see on the Odyssey.

You may now not just for compounding factors due to the fence inherent in the study the science and entry criteria.

I would like now to move to our ongoing phase III study.

While we believe picture at the top line has the potential to receive broad label based on the results from Pegasus the.

Prince study.

Because of the compound in patients that are more representative of the overall treatment naive population.

On the slide you can see the bridge study of this time.

The treatment naive patients and walk the.

The two primary endpoints evaluated after 26 weeks on hand, Mcglothin sterilization and the absence of transfusions and reductions in LDH level.

Federico Grossi: I'd like to discuss a couple of key points about this study. Please use the standard regulatory endpoint for P&H studies that were designed for C5 inhibitors. And therefore, focus on each of us very much. As we all know, PNH is characterized by both intra and exovascular analysis, so we believe that some of the secondary endpoints, such as hemoglobin levels, transfusions, and fasciculatic score, are equally as important since they truly reflect the impact this disease has.

That's not in any of the slight patients in the control group have the option to escape true picks at the Copeland.

Mcgladrey interest levels drop.

By two or more of grams per deciliter below the baseline.

Federico Grossi: Additionally, even on measures of thermolysis, we have set a higher biospecs as a component. For example, patients in PRINCE are considered to have unstable hemoglobin levels if they lose only one gram per deciliter of hemoglobin levels. Historically, in studies with C5 inhibitors, patients were allowed to drop by 2 grams per deciliter before being considered. We have been fortunate to be minimally impacted by COVID-19 across our P&H clinical studies. However, the timing of spring...

Therefore focus on Intravascular hemolysis.

As we all know <unk> is characterized by both the intra and extra vascular hemolysis. So we believe the some of the secondary endpoints such as hemoglobin levels transfusions and fastest got the score are equally as important seems the truly reflect the impact the species cost on.

Patients.

Additionally, even on major you'll find policies, we have set of higher box opex of the company.

Adam J. Townsend: Okay, we'll begin in June, in line with public guidance, but later than initially planned. This is due to COVID-19-related delays in local regulatory approvals of the extension study, which have precluded us from transitioning all patients in two countries and delayed our database launch. We are very excited to see the impact of Paxilotocopin on treatmentized patients and look forward to sharing these results later next quarter. I'll now hand the call over to our Chief Commercial Officer, Adam Townsend, to provide an update on our P&H launch preparation. Adam

Patients from Plains has considered to have on stable hemoglobin levels, if they lose on the one gram per deciliter in hemoglobin levels the.

Historically in the studies with <unk> inhibitors.

We're allowed to drop by two grams per deciliter before being kind of sooner unstable.

Well the unfortunate to be minimally impacted by COVID-19 across our <unk> clinical studies, however, the timing of free.

Okay from the beginning of June.

In line with the public guidance on later than initially expected.

This is due to COVID-19 related delays and local regulatory approvals of the extension study.

Who would have precluded us from transition and quotations in two countries and delayed our database lock.

Adam J. Townsend: Thank you, Fede. The positive Phase 3 Pegasus results showed the potential of Pegcetacroplin to elevate the standard of care in P&H, and we are working hard to prepare for its successful U.S. launch in anticipation of our May PDUFA date. As you can see on this slide, people with PNH continue to suffer from significant unmet need despite their current treatment with C5 inhibitors like cilirus and ultimiris. Clinical data and our own market research have shown that about a third of patients on C5 inhibitors continue to require transfusions to address their falling hemoglobin levels.

We are very excited to see the impact of Tetra Tech all of them on treatment naive patients and look forward to share in these results later next quarter.

I'll now hand, the call over to our Chief commercial officer, Alan downside to provide an update on our <unk> launch preparations Adam.

Thank you Fedex the positive phase III Pegasus results showed the potential of peg such of Copeland to elevate the standard of care in the <unk> and we are working hard to prepare for its successful U S launch in anticipation of a may produce the day.

Adam J. Townsend: Another third of these patients continue to be severely anemic and experience other symptoms like severe fatigue. This has a huge impact on these patients. The final third of patients have hemoglobin levels closer to normal, but they only achieve that at the expense of maximum output of red blood cells from their bone marrow.

As you can see on this slide people with Pn H continue to suffer from significant unmet need despite the current treatment with <unk> inhibitors like Soliris and also mirrors.

Clinical data on our own market research of shutting that about a third of patients on the <unk> inhibitors continue to require transfusions to address the falling hemoglobin levels.

Adam J. Townsend: As these data show, there is an urgent need for new treatments within PNH. Over the last two years, we have built a robust commercial organization in preparation for our first launch of Pexeter-Coplin in P&A. Our integrated team is focused on ensuring that we are ready to effectively address the needs of patients at launch, and our progress is highlighted on this slide. Our Value and Access team is fully staffed and engaging with high-priority payers representing more than 80% of all US P&H patients. Our discussions with those payers have yielded positive feedback on the clinical profile of Pexar. Apellis is also in the late stages of finalizing its distribution model and patient support resources and programs.

Another third of these patients continue to be severely anemic and experience other symptoms like severe fatigue. This has a huge impact on these patients.

The final third of patients have closer to normal hemoglobin levels, but only achieve that at the expense of maximum output of red blood cells from the bone marrow.

As these data show there is an urgent need for new treatments within PMA.

Over the last two years, we have built a robust commercial organization in preparation for our first launch opex such Copeland within <unk>. Our integrated team is focused on ensuring that we are ready to effectively address the needs of patients at launch and our progress is highlighted on this slide.

Adam J. Townsend: We will aim to provide patients with a consistent positive experience, both at the time of treatment initiation with Pexeter-Coplan and long-term assistance as and when needed. As an example, we have established Apellis Assist, a patient-focused program designed to ensure a high-quality patient treatment experience, including the recruitment of our care educator team, which will interact directly with patients through product and drug administration education. In parallel with our commercial activities, our medical team has also been preparing some, As shown on this slide, our medical affairs colleagues have been actively engaging with the top treating physicians via our virtual presence at medical meetings and in-person engagements when appropriate.

All of value and access team is fully staffed and engaging with high priority payers, representing more than 80% of all U S. P N H patients.

Our discussions with those payers have yielded positive feedback on the clinical profile of <unk> sense of comfort.

Our pallets is also in the late stages of finalizing our distribution model and patient support resources and programs.

We will aim to provide patients with a consistent positive experience by the fact the.

The time of treatment initiation with peg set of Copeland as well as long term assistance as and when needed Hasnt.

Adam J. Townsend: They have also initiated an early access program for Pegcetacoplin in the U.S. and already established multiple sites to treat PNH patients who are experiencing ongoing disease activity despite treatment with ciliris or ultimirib. On marketing, early activities with healthcare professionals, or HCPs, have been really strong and positive, showing high engagement above industry benchmarks. Almost 90% of targeted U.S. P&H HCPs have accessed our content focused on the unmet need in P&H, examples of which can be seen on the left-hand side of this slide.

As an example, we have established a palace assist our patient focused program designed to ensure a high quality patient treatment experience, including the recruitment of our cash educated team, which will interact directly with patients through product and drug administration education.

In parallel with our commercial activities.

Medical team has also been preparing for launch.

Shown on this slide on our medical affairs colleagues have been actively engaging with the top treating physicians via our virtual presence at medical meetings and in person the engagements when appropriate.

Adam J. Townsend: Separately, over 2,000 patients, caregivers, advocates, and other P&H community members have opted in to communicate with Apellis via our community outreach and patient marketing efforts, which can be seen on the right side of this slide. Patients and caregivers are also spending considerable time on our website, which tells us they are interested in our information, particularly around the existing unmet needs. Finally, our focused and experienced sales team will be deployed to cover the top 1,000 to 2,000 HCPs, including more than 90 key treatment areas. We are excited about the commercial team we are building and honored to have the opportunity, pending approval, to bring Peg Setter Coplin to P&H patients this year, as well as to prepare to elevate the standard of care for PNH patients.

They have also initiated an early access program for <unk> set of Copeland in the U S and already established of multiple sites to treat <unk> patients who are experiencing ongoing disease activity, despite treatment with soliris or all tumors.

On marketing efforts.

On the activities with health care professionals, or Hcp's had been really strong and positive showing high engagement above industry benchmarks.

Almost 90% of targeted the U S. P N H H Cp's Baxter stock content focused on the unmet need in P. N H examples of which can be seen on the left hand side of the slide.

Separately over 2000 patients caregivers advocates and other pnas community members have opted in to communicate with the palace via our community outreach and patient marketing efforts, which can be seen on the right hand side of the slide.

Adam J. Townsend: We are quietly preparing for future potential approvals in new indications. The commercial organization is excited to see our GA results later in the year. I will now turn the call back over to our Chief Medical Officer, Dr. Federico Grossi, to review the additional systemic program indications, as well as geographic... Fareday.

Patients and caregivers of also spending considerable time on our website, which tells US they are interested in our information, particularly around the existing unmet need.

Finally, our focus on experienced sales team will be deployed to cover the top 1000 to 2000, hcp's, including more than 90 key treatment centers.

Federico Grossi: Thank you, Adam. Beyond P&H, we're advancing four registrational programs of systemic beta-tocoplan in rare diseases with high unmet need with our partner Sobe. As seen on this slide, in the second half of the year, Apellis expects to initiate a phase 3 study to further a registration program in immune-complex membranoproliferative glomerulinositis, or ICMPGN, and G3 glomerulopathy, or C3GF. So we plan to start with an illustration of problems in cholaglutinin disease. TAD, and hematopoietic stem cell transplantation-associated thrombotic microangiopathy, or HSCTDMF. Also, in the second half of this year, Apellis expects to complete enrollment for a potentially registrational Phase II meridian study in amyotrophic lateral sclerosis. All A-L-L-A.

We are excited about the commercial team we are building and honored to have the opportunity pending approval to bring peg sets of Copeland to P and H patients this year.

As well as preparing to elevate the standard of care of P. N. H patients we are quietly preparing for the future potential approvals of new indications. The commercial organization is excited to see RGA results later in the year.

I will now turn the call back of it to our Chief Medical Officer talked of Federico Grossi to review the additional systemic program indications as well as geographic atrophy Fedex.

Thank you Adam.

The lumpy nature of advancing four registrational programs of six that make bets at the top line in rare diseases with high unmet need with a partner of salvi.

As seen on the slide in the second half of the year.

Federico Grossi: In parallel to our work in systemic Pexeta-Coupland, we continue to execute our Phase III studies of intravitreal Pexeta-Coupland in GA, with top-line results expected in the third quarter. We believe that our GA program represents a unique opportunity to make a difference in the lives of 5 million people at risk of blindness, with no treatment options available and few opportunities on the horizon. Results from the largest retrospective study in GA secondary to age-related macular degeneration, or AMD, were presented as a late break at the American Academy of Ophthalmology meeting.

<unk> expects to initiate a phase III study to further our Registrational program the name.

The complex Mcdonough plug of throughout the two of Maryland, nephritis, or IC and PGN on Q3 of them are loyalty or CPG on Shelby bunch of Starwood administration of programs and Golar looting Nbc's T. A D and hematopoietic stem cell transplantation associate.

Thats true, but my current geography or HSC key DMA.

Also on the second half of this year of police expects to complete enrollment.

Or potentially Registrational phase II married in the study in the amyotrophic lateral sclerosis or.

Federico Grossi: This study, which was conducted in partnership with Verona Health, highlighted the disabling impact this disease has on quality of life. The results also reiterated that WETMD is an expected occurrence in GA patients and show that it is observed more frequently if WETMD is present in the fellow eye, with 22% of GA eyes developing WETMD over 24 weeks. These results echo feedback received on our recent GA webcast, which features a panel of leading veteran specialists and further underscores the need for new treatments. I encourage you to listen to the archived recording of this event on our website under events and presentations in the investment section.

A L S.

And part of it to our work in systemic picks of the Copeland, we continued to execute our phase III studies of income vitro opex of the Copeland NGA with top line results expected in the third quarter.

We believe the RG <unk> program represents a unique opportunity to make a difference and the lack of 5 million people of grade school blenders with no treatment options available on a few opportunities in the horizon.

We saw from the largest retrospective study of NGA secondary to age related macular degeneration.

M D with the.

Centered at the late breaker at the American Academy of Lone Star module meeting.

The study, which was conducted in partnership with her on the health.

Federico Grossi: Now to our Pivotal Phase III GA study. Gravionogs is two large, world-controlled studies that compare the efficacy and safety of monthly and every-other-month intravitreal pexilotocoplan with sham treatment in more than 1,200 patients. The primary endpoint of our study is the reduction in gross GA emissions at month 12.

Highlighted the disabling impact this the six past on quality of life.

The results also reiterated that the Wendy.

Expected occurrence in GL patients and showed that it itself sort of more frequently if what MB is present in the fellow eye with 22% of G of animals developing wet AMD over 24 months.

These results Echo feedback received on a recent G. A webcast with feature of panel of leading but specialists and further underscore the need for new treatments.

Federico Grossi: And safety and efficacy will be assessed again at that point. In Philly, we saw the treatment effect of Paxilococcal increase from month 6 to month 12, and we look forward to seeing the results of longer treatment with Pexeta-Coplan in our Phase 3 study. Full studies and details can be seen in this.

Encourage you to listen to the Cub recording of this event on the <unk>.

Website under events and presentations in the investors section.

Now to our pivotal phase III studies.

The way, we announced two large well controlled studies that confer the efficacy and safety of monthly and every other month into the job picks of the Copeland with sham treatment in more than 1200 patients.

Federico Grossi: We're excited to see the top-line results from Derby and Oakes in Q3 and would like to take this opportunity to reiterate why we believe these studies will be successful. First, Debbie and Ox have the same study population and core study design as Phoebe, which demonstrated robust and statistically significant results that were confirmed by multiple sensitivity analysis. Second, they will now include more frequent assessments that will provide an even more complete evaluation of the primary. Third, cases of oxidation require confirmation by the reader, which reduces the potential for bias in the.

The primary end point of both studies is the reduction in gross of G. A mission of months talk.

The studies will continue for a total of 24 months on safety and efficacy will be assets again at that time, Inc.

In Philly instead of the treatment effect of picks of the Copeland increase from month six month Tox.

And we look forward to seeing the results of longer treatment with folks at the top line in our phase III study.

For the study the time details can be seen in the select.

Federico Grossi: In addition, patients experiencing exudations can receive anti-BGF treatments while continuing on Plexus Etacoplus, which we expect to reduce the number of study treatments. Finally, the studies are sufficient with power to meet the primary endpoint given the current rate of misinjections in this group. For all of these reasons, we believe Derby and Knox will be successful, and we are excited to see the results later this year. Success here will position us as a leader in the treatment of retinitis.

We're excited to see the top line results from Derby and Oaks in D. C and we would like to take this opportunity to reiterate why we believe the studies will be successful.

First that'd be a notes have the same study population and of course that it is a sign of spirit, which demonstrated robust kind of statistically significant results.

Income from the multiple sensitivity analysis.

Second the real now to include more frequent assessment that will provide an even more complete the evaluation of the primary endpoint.

<unk> cases of extra dilution the quiet confirmation by the reading Center.

Which reduces the potential for bias in the diagnosis.

Federico Grossi: The last component of our strategy is the addition of a pipeline of several new technologies designed to control companies. We plan to advance three new product candidates into clinical development by the end of next year. The first area of focus is on less frequent dosing while maintaining the strong clinical benefits seen in multiple studies with Bexetec-Ox. Our second focus is to expand our MD offerings to treat all forms of MD and potentially prevent the onset of advanced MD autism.

In addition.

<unk> experiencing excavations can receive anti VEGF treatments, while continuing complex of the covenant.

Which we expect to reduce the number of standard treatment discontinuation.

Finally, the studies of sufficient group power to meet the primary endpoint given the current rate of Mesa injections in the space.

For all of these reasons, we believe there'll be enough will be successful and we are excited to see the results later this year.

Success here will position us as the leader in the treatment of retinal disease.

Federico Grossi: And finally, we believe that you three play a critical role in many neurodegenerative conditions and are pursuing new technologies focused on neurology. We look forward to sharing more about these programs in the months to come. I will now turn the call over to our Chief Financial Officer, Tim Sullivan, who will reveal the financial results.

The last component of our strategy is the addition to apartment of several new technologies the fine to control comprehends the plan to add funds three new product candidates into clinical development by the end of next year.

The first area of focus is on less frequent dosing, while maintaining the strong clinical benefit seen in multiple studies with fix of the coffee.

Timothy E. Sullivan: Thank you, Fede. Since we issued a press release earlier today with the full financial results, I will just focus on the highlights for the full year 2020. As of December 31, 2020, Apellis had $877.6 million in cash, cash equivalents, and short-term marketable securities, compared to $352 million in cash and cash equivalents as of December 31, 2019. This increase primarily reflects the addition of cash from our follow-on offering for gross proceeds of $404 million in January 2020, our convertible offering for gross proceeds of $329 million in May 2020, and also the receipt of $250 million in upfront proceeds for the SOBI transaction in October 2020, less our cash used in operations.

The second focus is.

To expand the offerings.

All forms of the AMD and potentially avoid the onset of add on.

And the on together.

And finally, we believe that cheaply.

Critical role in many neurodegenerative conditions.

The pursuit of new technologies focused on neurology.

We look forward to share more about these programs in the months to come.

I will now turn the call all of the Jordan, Chief Financial Officer, and Sullivan, who our reveal of the financial results.

<unk>.

Thank you said it since we issued a press release earlier today with the full financial results I will just focus on the highlights for the full year 2020.

As of December 31, 2020 of pellets at 877 6 million of cash cash equivalents of short term marketable securities compared to $352 million in cash and cash equivalents as of December 31 2019.

Timothy E. Sullivan: Research and development expenses were $325 million for the full year ending 2020 compared to $221 million for the same period in 2019. The increase in R&D expenses for the full year 2020 was primarily attributable to an increase in manufacturing expenses for our Phase 3 clinical trials and potential commercial launch, costs associated with ongoing and planned clinical trials, compensation, and related personnel costs, primarily due to the hiring of additional personnel in 2020, among others.

This increase primarily reflects the addition of cash from our follow on offering for gross proceeds of $404 million in January 2020, our convertible offering for gross proceeds of $329 million may of 2020, and also the receipt of $250 million in the upfront proceeds from the Soviet transaction in October.

<unk> thousand 20, less our cash used in operations.

Research and development expenses were $325 million per the full year, ending 2020 compared to $221 million per the same period in 2019.

The increase in R&D expense for the full year 2020 was primarily attributable to an increase in manufacturing expenses for our phase III clinical trials and potential commercial launch cost associated with ongoing and planned clinical trials compensation and related personnel costs, primarily due to the hiring of additional personnel in 2020 among other.

Timothy E. Sullivan: General and administrative expenses were $139.4 million for the full year ending 2020 compared to $67 million for the same period in 2019. The increase in general and administrative expenses for the full year 2020 was primarily attributable to an increase in professional and consulting fees, employee-related costs due to the hiring of additional personnel, and directors' stock compensation expense, among others. For the full year ending December 31, 2020, Apellis reported a net loss of $344.8 million, compared to a net loss of $304.7 million for the same period in 2019.

Yeah.

General and administrative expenses were $139 4 million for the full year, ending 2020 compared to 67 million from the same period in 2019.

The increase in general and administrative expenses for the full year 2020 was primarily attributable to an increase in professional and consulting fees employee related costs due to the hiring of additional personnel and director stock compensation expense among others.

For the full year ending December 31, 2020, the palace reported a net loss of $344 8 million compared to a net loss of $304 7 million for the same period in 2019.

We remain well capitalized to execute on the potential launch of TEG seat of cop on and Peanuts and to continue to advance our robust clinical development plan.

Timothy E. Sullivan: We remain well capitalized to execute on the potential launch of Pegcetocoplin and PNH and to continue to advance our robust clinical development plan. Our cash runway is expected to fund operations into the second half of 2022. I will now turn the call back over to Cedric for closing remarks.

Our cash runway is expected to fund operations into the second half of 2022.

I will now turn the call back over to Cedric for closing remarks.

Thank you Tim.

Cedric Francois: Thank you, Tim. As you heard today, we have a transformation of the era ahead. And the key commercial, clinical, and regulatory milestones are shown on this slide. As you can see, we have a busy 2021 as we work to deliver on the full potential of targeting C3 across a broad range of complement-driven diseases. Before we move to Q&A, I would like to thank the patients, investigators, and caregivers who have participated in our clinical trial, and our employees and investors who have helped advance Apellis through this difficult year. We look forward to keeping you updated on our upcoming mouse. And now, operator, please open the call for questions.

As you heard today, we have a transformational year ahead, and the key commercial clinical and regulatory milestones are shown on this slide.

As you can see we have a busy 2021 as we work to deliver on the full potential of targeting <unk> three across the broad range of complement driven diseases.

Before we move to Q&A I would like to think of the patients investigators and caregivers who have participated in our clinical trials and our employees and investors will have helped the advent of babies through this difficult year.

Of course to keeping you updated on our upcoming milestones.

Now operator, please open the call for questions.

Thank you.

Operator: Thank you. Again, ladies and gentlemen, if you'd like to ask a question, please press star then 1 on your telephone. One moment, please. Our first question comes from Anupam Rama of J.P. Morgan. Your line is open.

Ladies and gentlemen, can I ask the questions going on.

The one when you kind of from telephone.

One moment please.

<unk>.

Yeah.

Our first question comes from Anna <unk> Rama of Jpmorgan. Your line is open.

Hi, guys. Thanks, so much for taking the question.

Anupam Rama: Hi guys. Thanks so much.

Anupam Rama: Thank you so much for taking the question. One of the most common questions we've gotten recently is, in Derby and Oaks, how do you think about the potential impact of sort of missed injections and how that impacts the study, the stats plan, how we should be thinking about this? Is the Philly every other month arm maybe a reasonable proxy for how we should be thinking about the potential effect of APL-2 on

One of the most common questions. We've gotten recently is in.

In Derby and Oaks, how do you think about the potential impact of sort of missed the injections and how that impacts of the study the <unk>.

<unk> plan, how we should be thinking about this is the Philly every other month on maybe a reasonable proxy for how we should be thinking about the.

The potential effect.

P L too.

Anupam Rama: Thanks so much.

Missed doses. Thanks, so much.

Cedric Francois: Thank you so much, Anupam, and it's great hearing you, and thank you to everyone for joining this call. The brief answer to this question is that the studies, DERBY and OCHS, continue to be well-powered to show what we intend to show in these studies, in spite of the missed injections that we have seen so far. Obviously, we did have missed injections, and the study was impacted by COVID. But we have a way of looking back, as you alluded to, to the Philly trial and comparing the frequency of missed injections, how these missed injections occurred, and based on that, making an assessment of the quality of the phase three clinical trial. And it is those assessments that make us highly confident that we are in a good place to measure the primary endpoints with good quality.

Thank you so much some of them great hearing you and thank you to everyone for joining the scope.

On the brief answer to this question is debt the studies Derby and Oaks continues to be well covered.

The show, but we intend to show in the studies in spite of the missed injections that we have seen so far.

Obviously, we did have missed injections and the study was impacted by Covid.

But we have a way of looking back as you alluded to to the filly trial.

Compare the frequency of missed the injections. How these missed injections of occurred and based on debt to make an assessment of the quality of the phase III clinical trials and this those assessments that makers had the confidence that we arent the good place to.

The measure of the Premier primary endpoint with good quality.

Anupam Rama: Great. Thanks so much for taking our questions.

Great. Thanks, so much for taking our question.

Thank you and from it.

Cedric Francois: Thank you.

Operator: Thank you, Anupam.

Our next question comes from Nomura.

Umer Raffat: Thank you. Our next question comes from Umer Raffat of Evercore.

Evercore Your line is open.

Hi, Thanks, so much for taking my question [noise] Cedric what percentage of the patients had wet AMD at baseline in the fellow eye on I'd be very curious and also sort of separately for the primary endpoint can you remind us how the auto fluorescence was done in phase two and how it's being done in phase III.

Umer Raffat: Your line is open. Hi Cedric, what percentage of the patients had wet AMD at baseline in the fellow eye? I'd be very curious. And also, sort of separately, for the primary endpoint, can you remind us how the autofluorescence was done in Phase 2?

Umer Raffat: How it's being done in phase three. But I guess what I'm really asking is, are you using...

Well I guess, what I'm really asking is are you using C. S. L O the scanning laser or using the standard funded the camera. Thank you.

Umer Raffat: C S L O

Umer Raffat: SLO, the scanning laser, or using the standard fundus camera. Thank you.

Thank you so much for the question so I'm going to start with the second part of your question, which is that we measure of the fluorescence and exactly the same way in the phase III as we did in the phase II clinical trial, and then I have two.

Cedric Francois: Thank you so much for that question. So I'm going to start with the second part of your question, which is that we measure autofluorescence in exactly the same way in phase 3 as we did in the phase 2 clinical trial. And then I have to ask you to repeat the first part because the line was breaking up a little bit.

Ask you to repeat the first part because the language breaking up a little bit piece of it no problem what percentage of the 600 patients on each trial had wet AMD in the fellow eye because I recall one of the discussions with FDA was whether they should or should not be included in the FDA encourage you guys to put them in.

Umer Raffat: Oh, no problem. What percentage of these 600 patients in each trial had wet AMD in the other eye?

Cedric Francois: One of the discussions with the FDA was whether they should or should not be approved.

Cedric Francois: They encouraged you guys to put them in.

But presumably that will impact the conversion rates. So I'm curious what percentage of habit on baseline.

Cedric Francois: Transcripts provided by Transcription Outsourcing, LLC. Yeah, no, thank you so much.

Yeah no. Thank you so much and the city trial as you May recall based on we had 38 per cent of patients who had geographic atrophy in the study eye and with M D and the control of that it really.

Cedric Francois: So in the Philly trial, as you may recall, at baseline, we had 38% of patients who had geographic atrophy in the study eye and wet AMD in the contralateral eye. That compares to a normal natural frequency of that demographic of approximately 25%. So we had many more patients that came into our Phase II clinical trial having that phenotype present. The reason for that is that back then, there were two large Phase III clinical trials that were enrolling with Lampalizumab, which excluded those patients from their studies.

That compares to a normal natural frequency of the demographic of approximately <unk> 25 per cent. So we had many more patients that came into our into our phase two clinical trial, having seen what types of present. The reason for that is the back then there were two large phase three clinical trials that we're enrolling.

With lump of leasing them up which excluded those patients from their studies.

And the phase three clinical trials, we are not commenting of course, yet on the.

Cedric Francois: And in phase three clinical trials, you know, we are not commenting, of course, yet on the baseline characteristics that will come in due time. But everyone should expect, I think, a lower present and naturally lower occurrence of patients coming into the study with that particular phenotype. The reason simply being that we do not compete anymore with these other studies and that I think we will be closer to that 25% normal demographic spread.

The baseline characteristics that will come and do the same but everyone should expect I think the lower president of naturally lower occurrence of patients coming into the study.

With that particular phenotype of the reason simply being that we do not compete anymore with these other studies and I think we will be closer to the 25% normal demographic spread.

Thank you very much.

Cedric Francois: Thank you very much.

Cedric Francois: Thank you.

Thank you.

Thank you. Our next question comes from Steve Do you have of Raymond James Your line is open.

Steven Seedhouse: Thanks. Our next question comes from Steve Seedhouse of Raymond James. Your line is open. Yeah, thank you. Maybe I'll ask about the COVID data that's coming up. I'm just curious if you have a hurdle that you set that's going to determine next steps. That asset in that indication, I think Alexian, [inaudible]

Yeah. Thank you maybe I'll ask the vote.

The Covid data, that's coming up but I'm just curious if you have a hurdle that you've set the sort of determined next steps.

Net asset in that indication I think alexia.

<unk> has talked about a 20% mortality delta or having statistical confidence at a 20% reduction of mortality of their interim analysis, which ultimately they didn't meet the I'm wondering if you have something similar established.

That'll determine if you have assets or not.

Cedric Francois: Yeah, thank you so much, Steve, for that question as well. So as we have previously publicly reported, I should say, in December, at the end of December, towards Christmas, we completed enrollment in this study. So we are now getting close to the point in time when the Data Safety Monitoring Board will make an assessment, which should be expected in the near future. And then the top line results will be after the full data analysis has been completed.

Yeah. Thank you so much defer that question as well so as we have previously commonly.

The publicly reported I should say.

In December at the end of the December towards Christmas, We completed the enrollment in the study. So we are now getting close to the point in time, where the data safety monitoring board will make an assessment.

We will that should be expected in the near future and then the top line results will be.

After the full data analysis has been completed.

Thank you.

Cedric Francois: Our next question comes from Derek Archila of Siegel, Illinois. Great, hi guys, and thanks for taking the questions.

Our next question comes from Derrick Archie a lot of Stifel. Your line is open.

Great Hi, guys and thanks for taking the questions maybe one on P. N H Cedric could you just provide some color of how you think about the competitive landscape shaping up in that indication, particularly with the oral that are in development.

Derek Christian Archila: Maybe one on PNH. I mean, Cedric, can you just provide some color on how you think about the competitive landscape shaping up in that indication, particularly with the orals that are in development? And then maybe I'll just throw in one more in terms of some of the new programs you might be launching from now to 2022. Is there an oral complement inhibitor in the mix there? Thanks.

And then maybe I'll just throw in one more in terms of some of the the new programs you might be launching.

From now to 2022 is there an oral complement inhibitor in the mix there. Thanks.

Yeah. Thank you so much they reported that question so.

Cedric Francois: Thank you so much, Eric, for that question. So, as we have commented many times, you know, we are excited about the development of new complement inhibitors by others and ourselves. I think we're really just at the beginning of what complement control can do in a wide range of indications. Of course, the launch in PNH being the first one now, where, very importantly, for patients, I believe, and we all believe, that the control of extravascular hemolysis will elevate the standard of care in these patients.

As we have commented many times we.

We are excited about the development of new complement inhibitors.

Others and ourselves I think we're really just at the beginning of what the components can control can do in a wide range of indications of course, the launch of <unk> being the first one now where very importantly for patients I believe and we all believe that the control of extra vascular hemolysis with elevate the standard of.

Care in these patients.

On a couple of years, particularly of the MP of niche we may be looking forward to the introduction of oral products that can control the alturas dispense the way of complement in these patients and thereby the dress extra vascular hemolysis.

Cedric Francois: In a couple of years, particularly in PNH, we may be looking forward to the introduction of oral products that can control the alternative pathway of complement in these patients and thereby address extravascular hemolysis. And while on the surface it may seem much more appealing to have an oral product compared to a twice-a-week subcutaneous product, it's important to bear in mind that in a disease like PNH, there is no room for error.

And while on the surface that may seem much more appealing to have an oral products compared to a twice a week subcutaneous product.

The important to bear in mind that in a disease like <unk> there is no room for error.

I mean bad debt is that when you are.

Cedric Francois: What I mean by that is that when you are reliant on a pill that you need to take twice per day, for example, not getting to take a pill could have important consequences. That is a liability that many patients are not very comfortable with, and neither are physicians. In line with that is the fact that we still need to find out if a majority of patients respond well to these products and whether the control of PNH is durable in the long run.

Reliance on a bill that you need to take twice per day for example.

We're getting to take of film could have important consequences.

That is the liabilities that many patients are not very comfortable with neither on the physicians.

In line with debt also is the fact that we still need to find out is the majority of patients reached 112th of these products and whether the control of the image is durable in the long run. So we look forward to the phase III readouts, but beyond that I'm on to and this is on the positive news I am personally very excited to the both the development of oral products.

Cedric Francois: So we look forward to the phase 3 readouts, but beyond that, I want to end this on a positive note. I am personally very excited about the development of oral products. In PNH, not so much, but I think in other indications where the exquisite control of complement is less important, they will provide a lot of opportunity. As it relates to our own internal programs, we are not yet ready to comment on that, but we look forward to and are excited about sharing more in the months ahead.

And the <unk> not so much but I think in other indications where it kind of the exquisite control of complement is less important it will provide a lot of opportunities.

As it relates to our own internal programs, we are not yet ready to comment on that but we look forward and theyre excited about sharing more of in the months of the comp.

Great. Thanks.

Derek Christian Archila: Great, thanks.

Thank you.

Cedric Francois: Thank you.

Our next question comes from the Jackson County of Oppenheimer <unk> Company. Your line is open.

Justin Alexander Kim: Our next question comes from Justin Kim of Oppenheimer & Company, Eliana Merle. Hi, thanks for taking the question. Just maybe on print, as we prepare for that readout, could you walk us through maybe perhaps what unique insights we may see from this naive to complement treatment population, and particularly maybe where the geographies where APL-2 is being used here may differ from like a standard of care perspective?

Hi, Thanks for taking the question just maybe on on print as we prepare for that readout could you walk us through maybe perhaps what unique insights we may see from the naive to complement treatment population.

And particularly on maybe where the geographies where APL two is being used here.

Differ from like a standard of care perspective.

Thank you so much for the question, Justin I'm going to hand that one over to of Chief Medical Officer adopter of gross Shannon.

Cedric Francois: Thank you so much for that question, Justin. I'm going to hand that one over to our Chief Medical Officer, Dr. Gross.

I think it's out of again think of Justin for free.

Federico Grossi: Thank you, Cedric, and thank you, Justin, for that. We're really looking forward to hearing from you. I'm going to share with you the results. So the study, as you pointed out, is looking at a PNH population that is nave to covalent inhibitors, and the primary endpoint is hemoglobin re-stabilization, which is a typical endpoint for PNH studies. And in addition to that, we're looking at quality of life and transfusion dependency.

What we're really looking forward to.

Sure the outcome of the study and sharing with you the results.

So the study as you point out of just looking at AR.

H population that is naive to complement inhibitors and the primary endpoint.

Instead of a mutation, which is typical of endpoint for <unk> studies and the.

In addition to that.

We're looking at.

Quantity or quality of life on transfusion dependency so that the the population.

This population does not differ from the treatment.

Treatment naive population that you see on.

On the regions, where a producer of mob is available.

Two of the studies in regions, where at least from all of US not available in order to do it but.

Federico Grossi: Yeah.

Federico Grossi: Thank you very much.

But the the population does not differ.

Justin Alexander Kim: Got it. And maybe just a follow-up to the previous question, and maybe ask maybe slightly differently, when you think about pan-AMD sort of therapies, do you, is it, is it a fair assumption that we may see sort of modalities outside of intravitreal injection if we're looking at, you know, populations who maybe have less severe disease?

Okay got it and maybe just a follow up to the previous question and maybe ask maybe slightly differently. When you think about Penn.

N D sort of thing.

Therapies do you is it is it a fair assumption that we may see sort of modalities outside of infra vitriol injection of if we're looking at populations, who maybe have less severe disease.

Justin Alexander Kim: So, can you repeat the question on MD?

So on <unk> can you repeat the question on M D R.

Just wondering as we think about her of new agents in a pan M D.

Federico Grossi: I was just wondering, you know, as we think about sort of new agents in a pan-AMD sort of therapeutic, does that suggest a non-injection-based therapy, potentially?

True therapeutic.

Does that suggest a non injection based therapy potentially.

Federico Grossi: Well, when you look at, this is outside of MD, you know, for diseases in the eye, the level of systemic exposure that you will get from treatments that are not injected into the eye may be too high. So there'll have to be, you know, a therapy that, when administered systemically, you can, you know, achieve good levels in the eye while exposing the patients to, you know, mother the drug and having safety concerns from a systemic perspective. Guys, thank you

When you look at this is outside of M D.

For the systems into the into the eye the.

The you know the level of systemic exposure that you would get from.

Treatments that are not the injected into the eye it might be.

True too high so they'll have to be yeah.

Therapy that.

From the systemic B you can achieve.

Good levels in the eye on exposure to patients true.

The amount of the drug on having safety concerns from average systemic perspective.

Got it thank you.

unknown: Thank you. Now, our next question... Transcripts provided by Transcription Outsourcing, LLC. Hey guys, thanks for taking my questions, and I'm looking forward to a very exciting year ahead for you guys. One, just for me, is on, I mean, obviously, you know, Alexion, you know, is going to be acquired, and sometimes there's disruption which can occur, you know, and I just wanted to get your perspectives on whether, you know, from a human resources standpoint, or even a commercial standpoint, you're sensing any sort of opportunity that you might be able to take advantage of, even though your data is already quite robust.

Thank you our next question.

On Thats from Alethia young of Cantor Fitzgerald of your line is open.

Hey, guys. Thanks for taking my questions and very exciting year ahead of you guys.

One just from me it's on I mean, obviously, you know Alexia on you know kind of.

Gonna be acquired and sometimes there's disruption, which can occur you know and I just wanted to get your perspective on whether you know from the human resource standpoint, or even the commercial standpoint that you're sensing any sort.

Net of opportunity that you might be able to take advantage of of even though your day is already quite robust and then the circa.

unknown: And then the second question I have is just, you know, on the four programs that are kind of moving toward registration. ALIS is always interesting and a little bit challenging. I mean, do you perceive that as being a higher risk, higher reward program, or should we think about them all kind of a little bit more in terms of equivalence?

The question I have is just you know on the floor of programs, they're kind of moving to the Registrational is endless.

This is always interesting and a little bit challenging I mean, do you perceive that as being a higher risk higher reward program or how should we think about the model kind of a little bit more on equivalents. Thanks.

Yeah no. Thank you so much low.

unknown: Thanks.

Starting first with the first question.

Cedric Francois: Well, starting with the first question, our primary goal is to elevate the standard of care in P&H. You know, the competitive landscape has changed, but at the end of the day, we believe that the important unmet need that exists in P&H is going to be the main driver of sales, and we look forward to addressing that.

Our primary goal is to elevate the standard of guarantee of niche the.

Relative landscape has changed but at the end of the day, we believe that the the <unk>.

Fortunately unmet need that exists in <unk> is going to be the main driver of sales and we look forward to them to addressing debt.

Then as it relates to the new and the or the other indications which are currently in Registrational developments of which there are four subsidiary Columbia of lots of tea with Icmp, Jim Yeah, that's cool diluted in the disease and to HFC T associated thrombotic microangiopathy the.

Cedric Francois: Then as it relates to the new or the other indications which are currently in registrational development, of which there are four, so C3 glomerulopathy with ICMPGN, ALS, cold agglutinin disease, and the HSCT-associated thrombotic microangiopathy, the ALS trial has a special place. It has a special place as the first neurological indication that we are targeting. It has a special place as, of course, an indication of an incredible unmet need that we hope to address and also as a place where we believe that C3 offers advantages over other places in the continent cascade where this disease can be controlled.

And this trial has the special place as the specialties as the first neurological indications that we are targeting.

And as the special place as of course.

Vacation with.

On the incredible unmet need that we hope to address.

And also as a place where we believe that <unk> offers advantages over other places on the complement Cascade, where this disease can be controls so.

Cedric Francois: So for us, again, the primary motivation for us was to bring the science together with the molecular entity that we have and our belief that we can address the unmet need. Is this a high-risk program? Of course it is. Any trial in ALS is, with a high reward associated. But one that we do truly believe fits very well into kind of the plethora of indications that we are pursuing with systemic pexita

So for US again, the primary motivation for US was to bring the science together with the molecular entity that we have in our belief that we can address the unmet need.

Is this a high risk program of course, it is any trailing the AOS is.

With the high reward associated but one that we do truly believe fits very well into kind of the the plethora of indications that we are pursuing with systemic fixes that colton.

Thank you. Thank you.

Cedric Francois: Thank you. Our next question comes from Laura Christensen of Cowling, Orlando. Good afternoon. Hi guys.

Our next question comes from Laura Christianson from Cowen Your line is open.

Good afternoon, Hi, guys.

So my question is actually about the allergic reaction to peg that have been sporadically seen in people who have received of the Pfizer pattern of vaccines I believe.

Laura Christensen: So, my question is actually about the allergic reactions to PEG that have been sporadically seen in people who have received the Pfizer and Moderna vaccines. I believe the CDC recommends that anyone who experiences an immediate allergic reaction to PEG should not receive the booster. So, I was just wondering what gives you confidence that patients aren't being sensitized to PEG? And, you know, I assume that is your belief, but if it's not, you know, why that's also the case?

CDC recommends that anyone who are experiencing.

Yet allergic reaction to price should not receive the booster.

So I was just wondering what gives you confidence the patients aren't being sensitized to peg.

And I assume that is your current beliefs, but if it's not you know why that fell from the case.

Cedric Francois: Thank you, Laura, for that question. So, you know, as you correctly mentioned, there are a couple of isolated cases of anaphylaxis that have been associated with text sensitization. This is something that is well known, you know, that can sometimes occur. It's important, therefore, that when you introduce products like ours, especially in the beginning, to make sure that there is good follow-up. What is important to note here, however, as well, is that with the introduction of the vaccines with, you know, the pigulated element in them, there does not seem to be an extra sensitization because the booster vaccines do not seem to be associated with any type of additional immune reactions. So we feel very comfortable with where we stand right now.

Yeah. Thank you Laura for the question so.

Sure.

As you correctly mentioned there are a couple of isolated cases of the unless you'd like six debt have been associated with fixed synthesize. The sensitization. This is something that is well known that can sometimes appear.

As important therefore, the when you introduced products like ours, especially at the beginning to make sure that there is a good follow up.

What is important to note here, however, as well as debt with the introduction of the vaccines with the.

The big related element in them.

There does not seem to be an extra sensitization because of the booster vaccines do not seem to be associated with any type of additional immune reactions. So we feel very comfortable with where we stand right now will be in the future in these rare diseases right I mean, the once in the well see and.

Cedric Francois: Will we in the future, in these rare diseases, once in a while, see an anaphylactoid reaction? That is possible. Thus far, that has not been the case.

And then if you'd like to its reaction that is possible. The sorted that has not been the case.

Laura Christensen: Perfect, that's helpful. Thank you. Thank you.

Okay. Thanks, that's helpful. Thank you thank.

Thank you.

Thank you.

Cedric Francois: Thank you.

<unk>.

Our next question comes from Matthew Luchini of BMO capital. Your line is open.

Matthew Lucchini: Our next question comes from Matthew Lucchini of BMO Capital. Hi, good afternoon.

Hi, good afternoon, thanks for taking the questions.

Matthew Lucchini: Thanks for taking the question. So first, on P&H, commercially, I guess I'd love to get a little sense as to what your internal market research is telling you. Tazeen Ahmad, Annabel Samimy, Yigal Nochomovitz, Steven Seedhouse, Douglas Tsao, Eliana Merle, And then secondly, just a kind of a housekeeping question. It looks like R&D saw a pretty decent sequential step down in 4Q. And I'm just wondering if that is, Um, something that we should think about as sort of the new baseline going forward, or how we should think about that as we think forward into this year and beyond. Thanks.

So first on Pn H.

Commercially I guess I'd love to get a little sense as to what your internal market research Chinese of about.

The initial launch and you know what I'm really looking for is a little bit of more of the color around things like.

Patients or doctors, telling you that the patients are asking for the drug are they planning to call patients and versus waiting for the next visit.

Any kind of color there that could help sort of set of actual patients on the what the initial launch is going to look like and then secondly, just on kind of a housekeeping question. It looks like R&D saw a pretty decent sequential step down in four Q and I'm just wondering if that's.

Something that we should think about as sort of the new baseline going forward on how we should think about that as we think forward into this year and beyond.

Yes.

Cedric Francois: Thank you, Matthew. I will hand the first question over to our Chief Commercial Officer, Adam Townsend, and then Tim will take your second question.

Thank you Matthew I will hand, the first question over to our Chief Commercial Officer, Adam Townsend and then Tim will take your second question.

Adam J. Townsend: Thank you, Cedric. And thank you, Matthew, for the question. So, we've spent a lot of time with the P&H community, patients, caregivers, and everything that surrounds the patient. And we do believe that we expect some patients to have a conversation with their physicians about the potential to improve the standard of care with Pexeter-Coplin. We've got various patient-focused marketing activities out there, and we're getting a great response from patients as they interact with our content.

Thank you Cedric and thank you Matthew for the question. So we've spent a lot of time with the <unk> community Ah patients caregivers and everything that surrounds the patient and we do believe that we expect some patients to have a conversation with the physician about the potential to elevate the standard of care with picks up the content.

We've got various patient focused marketing activities out there and we're getting a great response from them as they interact with our content they truly understand the unmet need that exists within the market. So whilst this is a very much an efficacy driven story for us we think that that launch the physicians will.

Adam J. Townsend: They truly understand the unmet need that exists within the market. So, whilst this is very much an efficacy-driven story for us, we think that at launch, physicians will have identified the patients with the highest unmet needs. And you've seen from our presentation and previous discussions, we look at the market for C5 treated in thirds. So we expect to transition from the patients that have the highest unmet need to the broader patient population and unmet need population as we work through.

Identified the patients with the highest unmet need in <unk>.

And you've seen from our presentation on our previous discussions.

Look at the market of C. Five treated instead, so we expect to transition with the from the patients that have the highest unmet need.

To the broader patient and unmet need population as we work through a core piece of that will be when patients go in.

Adam J. Townsend: A core piece of that will be when patients go in to have a conversation with their physicians, and we also expect some physicians to have already identified the patients and actually potentially call them in. Still a rare disease, it will be a very thoughtful approach to launch. And obviously, this is an important conversation for patients and physicians to have around the potential of pegcetacloprid.

To have a compensation because the physicians and we also expect some physicians who have already identified the patients and actually potentially pull them in.

Still a rare disease it will be a very thoughtful approach to launch and obviously this is an important conversation for patients and physicians to have around the potential of peg sense of comfort.

Matthew Lucchini: Thanks, Matthew, for the question. I'll take the R&D question. So what you're seeing in terms of the 4Q step down in R&D is actually more of an accounting and SOBI-related concept. So SOBI, as you know, from the structure of the deal, will reimburse Apellis for $80 million worth of R&D expenses over the course of the next four years. The way those are accounted for and when those began started in the fourth quarter, primarily in 2020.

Sure.

Matthew for the question on I'll take the R&D question.

So what youre seeing in terms of the <unk> step down in R&D is actually more of an accounting.

So the related concepts so.

So as you know.

From from the structure of the deal will reimburse at palace for $80 million worth of R&D expenses over the course of the next four years.

The way those are accounted for in the Windows began started in the fourth quarter primarily of of 2020, and so that does actually.

Matthew Lucchini: And so that is actually counted as a contra account. And so, from a GAAP perspective, those amounts were deducted from R&D. And that just brings the number down. Ultimately, the reimbursement for that $80 million will come over time over the next four years. So I would look at the true R&D expenses much closer to the third quarter, flat to a little bit up, and probably it will steadily, although not dramatically, rise over the next Great, thank you.

Counted as a contra account and so from a GAAP perspective, those those those amounts were deducted from R&D.

And that just brings the number down ultimately the <unk>.

Reimbursement for that $80 million will come over time over the next over the next four years so I.

I would look at the true R&D expenses.

Much closer to the third quarter flat to a little bit up and probably will it will steadily although not dramatically rise over the next year.

Great. Thank you very helpful.

Matthew Lucchini: Very helpful. Thank you. Again, if you'd like to ask a question, please press star then 1 on your touchtone telephone. Our next question comes from Yigal. Travelers.

Thank you I didn't say that.

I got the question. Please press Star then one on your Touchtone telephone. Our next question comes from the Heck out.

The job of it.

Operator: Thank you. Thank you. Hi, great, thanks for taking the question.

Your line is open.

Alright, great. Thanks for taking the question I had one on Derby and Oaks. So as you know Cedric in Philly the difference in the absolute lesion growth area between the Sham and the monthly.

Yigal Dov Nochomovitz: I had one on DERBY and OCHS. So, as you know, Cedric, in Philly, the difference in the absolute lesion growth area between the sham and the monthly Bexida coplan was 0.66 meters squared. And that was obviously deeply stat-signatured. Now, presumably, the bar is lower in DERBY and OCHS, given a higher power. So could you comment at all on the hurdle that you need to hit for the difference in the GA lesion growth area? For DERBY and OCHS to be successful. Thank you.

The the culprit was six six meters square.

And that was obviously deeply stat Sig now, presumably the bar is lower and the Derby and Oaks, given the higher higher power. So could you comment at all on the hurdle that you need to hit from the difference in the G. A lesion growth area for the army.

To be successful.

Thank you so much you go.

Cedric Francois: Thank you so much, Yigal. So, the phase 3 clinical trials are more than 95% powered to show the same effect that we saw in the phase 2 clinical trial for the monthly-dosed individuals and somewhere between 80 and 90% for every other month-dosed individuals. This is, of course, on a presumption of similar variability and accounts for a p-value of 0.05. So, you know, again, for us to kind of protect the p-value that we got in the phase 2 clinical trial, we made sure to have as few changes as possible. We are studying the same exact patient population. We analyze and read the findings in the same way. You know, all of that was maintained between phase 2 and phase 3.

So the phase III clinical trials are more than 95% powered to show the same effect that we saw on the phase two clinical trial.

I'm sort of the monthly dose individuals' and somewhere between 80 and 90 per cent for every other month of those individuals. This is of course on the presumption of similar variability and.

Comes from the P value of points of price. So you know again for us to kind of protect the P value that we got in the phase two clinical trial.

Made sure to have as few changes as possible. We are studying the same exact patient population.

We analyze and read the findings in the same way.

On all of that was maintained between phase II and phase III.

Yigal Dov Nochomovitz: Okay. Thank you. Thank you.

Okay. Thank you.

Thank you.

Thank you.

Laura Kathryn Chico: All right, the next question comes from Laura Chico-Wedbush, from Eliana Merle. Hey, thanks very much for taking the question. I just wanted to circle back on one with respect to wet AMD.

Our next question of the Laura Chico of Wedbush. Your line is open.

Hey, Thanks very much for taking my question I just wanted to circle back on one with respect of wet AMD such rich I think you indicated the normal frequency is around 25% I'm wondering if you could just comment then around maybe what is an acceptable rate of nuance that explanation that we should be thinking about in Derby and Oaks 80 from Iraq.

Laura Kathryn Chico: Cedric, I think you indicated the normal frequency is around 25%. I'm wondering if you could just comment then on maybe what is an acceptable rate of new onset exudation that we should be thinking about in Derby and Oaks, A, from a regulatory perspective, and B, from a commercial perspective. Just kind of curious if patients do have a lower baseline frequency there. How might that change the expectation? And then a quick follow-up, just with respect to PNH, could you just remind us or talk a little bit about your expectation of whether APL2 labeling would also extend to include ultramarine-treated patients? I think you had a slide there comparing that to ultramarine. So what type of data or guidance might you be able to provide for patients thinking about a transition? Thank you.

The Tory perspective, and B from a commercial perspective, just kind of curious if.

If patients do you have a lower baseline frequency there.

That changed the expectation and then a quick follow up just with respect the Pn H could you just remind us or talk a little bit about your expectation on weather picks up.

ABL to labeling would also extend to include Altamira is treated patients I think you had a slide there of comparing the.

That with Altamira, so what what type of gate data of our guidance might you be able to provide for patients from thinking about the transition. Thank you.

Thank you so much so starting with the first part of your question. The the with M. D of occurrences that we had in the phase II clinical trial.

Cedric Francois: Thank you so much. So, starting with the first part of your question, the wet MD occurrences that we had in the Phase II clinical trial are important to contextualize, right? These were small exudates that did not lead to significant vision loss, and that is why in the Phase III clinical trial, we are studying the exact same patient population as we did in the Phase II. So if your question is what is acceptable from a physician perspective or from a regulatory perspective in terms of exudation, it is the exudation rate that we saw in the phase two clinical trial.

<unk> are important to contextualize right. These were small extra debt, which did not lead to significant vision loss and that is the way in the phase III clinical trial. We are studying the exact the same patient population as we did in the phase two so of your question is what is acceptable from a physician perspective or from a regulatory perspective in terms of the exhibition.

Is this the expedition rates that we saw in the phase II clinical trial.

Cedric Francois: And that exudation rate was arguably artificially higher than what you should expect in phase three because we had so many patients in the study that had already wet the contralateral eye when they came into the study. And also because, with the benefit of hindsight, we had some investigator bias that may have contributed to an increased frequency of treatment with anti-VEGF as well. All of that will be corrected in the phase three clinical trial.

And that extradition rates was arguably artificially higher than what you should expect in the phase III because we had so many patients in the study that had already with in the in the control of that of life. When they came into the study and also because of the phase II with the benefit of hindsight. We have some investigator of bias that may have contributed to an increased frequency of treatment with <unk>.

As well all of that will be corrected in the phase III clinical trial.

Cedric Francois: But I think the most important take-home message here is that our phase three clinical trial, if it hadn't been for COVID, if it hadn't been for the kind of IMPDs that we had to wait for in Europe, would have been completed probably in nine months. I mean, and that gives you a sense of the unmet need that exists and the willingness and desire of physicians to treat these patients. So we start with what I believe is a very strong baseline, and we will see what we see in the phase three clinical trial.

But I think the most important take home message here is that our phase III clinical trial.

It hadn't been before.

But if it hadn't been for the kind of the.

On the A&P d's that we have to wait for in Europe.

Would've been rules, probably nine months I mean in debt.

Gives you a sense of the unmet need that exists and the willingness and desire of physicians to treat these patients. So we are.

We start with a with what I believe is a very strong baseline and we will see what the what we've seen in the phase III clinical trial, but importantly.

Cedric Francois: But importantly, exudations in patients with geographic atrophy are a normal phenomenon. And I would encourage listeners that haven't done that to look at our presentation at the American Academy of Ophthalmology. It was a late-breaking abstract where we looked retrospectively at 69,000 patients with geographic atrophy. It's the largest study of its kind ever done.

Exhibitions and patience the geographic atrophy.

Normal phenomenon and I would encourage listeners that haven't done that to look at our presentation at the American Academy of ophthalmology towards the late breaking abstract where we looked retrospectively at 69000 patients with geographic atrophy of the largest study of its kind of freedom from the third piece, what's the find out.

Cedric Francois: And the purpose was to find out, you know, how frequent it is for patients with geographic atrophy to develop wet AMD. And on a base case, the number to bear in mind is that patients with pure geographic atrophy, meaning no exudations yet anywhere, develop wet AMD based on claims-based data in 8% of cases over two years. If you start off with wet AMD in one eye and GA in the contralateral eye, then that GAI has a 22% odds of developing wet AMD over the course of two years. It is not at all unusual.

How frequent it is for patients with geographic atrophy, the developed with AMD and on the base case, the number to bear in mind is that patients with pure geographic atrophy, meaning no extraditions yet anywhere the vet.

With M D based on claims data.

Eight per cent of cases over two years.

If you start off with wet AMD and one I N G. In the control of lateral line than the GE is the 22%.

Of developing within the over the course of two years, so not at all unusual and again it comes down to what is the nature of these accusations that article observed.

Cedric Francois: And again, it comes down to what the nature of these exudations that are observed is. Then, to get back to your second question as it relates to the label. So we are not yet commenting on labeling. We believe that we will have a broad label when we get our approval. A broad label would mean including treatment-naive patients and, in that particular case, would, of course, be applicable and usable in patients that are on baseline ultramarine.

And then to get back to your second question as it relates to the label. So we are not yet common thing on literally we believe that we will have a breadth of label win.

We get our approval.

The broad label label, which mean, including treatment nave patients.

And in that particular case would of course the.

The bill and usable ambitions that are on the based on Nielsen movies as well.

Laura Kathryn Chico: Thanks very much.

Thanks very much.

Cedric Francois: Thank you.

Thank you.

Cedric Francois: Thank you. I'm showing no further questions at this time. I will turn the call back over to Cedric Francois for any closing remarks.

Thank you I'm showing no further questions at the time I can turn the call back on the sensor Kras law for any closing remarks.

Cedric Francois: Thank you so much, and thank you everyone for joining us on our inaugural financial results conference call. We are excited about the transformational year ahead for us. I would like to close by reiterating our corporate strategy for leadership in companies. We aim to establish systemic Plexi-Tycoplan as a disruptive therapy across rare complement-driven diseases. We plan to become number one in the retina with the first treatment for geographic atresia, and we continue to advance innovative technologies to control complement with a focus on complement factor C. Thank you again for joining us today.

Thank you so much and thank you everyone for joining us on our inaugural financial results Conference call. We are excited about the transformational year ahead for us.

To close by reiterating our corporate strategy for leadership in continent, we aim to establish systemic makes it the coupon.

The disruptive therapy across rare complements driven diseases.

We plan to become number one in the retina with the first the treatment for geographic atrophy.

And we continue to advance innovative technologies to control complements with a focus on complement factor of seating.

Thank you again for joining us today.

Operator: Thank you. Ladies and gentlemen, this does conclude today's conference. Thank you all for participating. You may all disconnect. Have a great day.

Thank you ladies and gentlemen, this does conclude today's conference. Thank you all of the painting you may all disconnect.

Great day.

[music].

unknown: BF-WATCH TV 2021

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