Good morning, and welcome to the Apellis Pharmaceuticals conference call to discuss their collaboration with Sobi. Today's call is being recorded. At this time, I'd like to turn the call over to Tracy Vineis, Vice President of Communications at Apellis.
Good morning, thank you for joining us to discuss Apellis' strategic collaboration with Sobi for systemic pegcetacoplan. For those participating by conference call, slides are available via webcast at apellis.com. 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, our actual results may differ materially. I encourage you to consult the risk factors discussed in our SEC filings for additional detail. On today's call, I am joined by Co-founder and Chief Executive Officer, Dr. Cedric Francois, who will discuss our new collaboration with Sobi. Joining us for Q&A will be our Chief Medical Officer, Dr. Federico Grossi, Chief Financial Officer, Timothy Sullivan, and Chief Commercial Officer, Adam Townsend. Now, I am pleased to turn the call over to Cedric.
Thank you, Tracy. Good morning to everyone on the call and joining via webcast. We are very excited to announce our new collaboration with Sobi for the global co-development and exclusive ex-US commercialization rights for systemic pegcetacoplan, our targeted C3 therapy. As part of the deal, Apellis retains the U.S. commercialization rights for systemic pegcetacoplan, as well as worldwide commercial rights for intravitreal pegcetacoplan in geographic atrophy or GA. Apellis will receive up to $1.25 billion in cash and commercial and regulatory milestone payments, plus tiered double-digit royalties. This transformational collaboration allows us to expand on the broad platform potential of targeting C3 across multiple rare diseases that impact over 275,000 patients around the world. First, it enables us to advance systemic pegcetacoplan in five parallel registrational programs across multiple rare diseases.
These include our existing program in paroxysmal nocturnal hemoglobinuria or PNH, two new registrational programs that we plan to start in hematology, and our recently initiated studies in nephrology and neurology. All five registrational programs will build on the remarkable success of pegcetacoplan in its first positive phase III study in PNH, which validated the platform potential of targeting C3. Second, the deal underscores our confidence in geographic atrophy as Apellis keeps global rights to this fully enrolled phase III program with blockbuster potential. Finally, this collaboration further strengthens our already strong balance sheet, which we believe will extend our cash runway into the second half of 2022. Our collaboration with Sobi is the result of a robust process conducted with numerous medium and large parties that sought to partner with us for our systemic pegcetacoplan program.
We chose Sobi, a global leader in hematology and rare diseases, because this collaboration will be as important to them as it will be to us, and because they are committed to expanding the development of systemic pegcetacoplan. Sobi shares the collaborative spirit that we cherish at Apellis, recognizes the platform potential of systemic pegcetacoplan, and perhaps most importantly, shares our values and deep commitment to patients. Sobi has an established international infrastructure with a strong presence in key global markets that currently commercializes 15 products, and we expect that they will leverage these strengths for the ex-U.S. launches of systemic pegcetacoplan, beginning with the anticipated European launch in PNH. Together with Sobi, we are confident that we can maximize the broad platform potential of our targeted C3 therapy. Our systemic pegcetacoplan programs are targeting rare diseases with significant unmet needs across hematology, nephrology, and neurology.
By controlling complement activation centrally, pegcetacoplan offers the opportunity to become a transformative new therapy in each of these rare diseases where patients have few or no treatment options available today. Also, as shown on the slide, our GA program remains the largest indication for pegcetacoplan. This continues to be an important element of our strategy to fully retain the global rights to this program. We believe that our GA program represents a rare 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. Recent data on intravitreal pegcetacoplan following 18 months of treatment and in early geographic atrophy have further validated targeting C3 in this area of significant unmet need in ophthalmology.
Now I'd like to discuss our expanded clinical development plan for systemic pegcetacoplan with Sobi, which will be overseen by a joint development committee. I'll begin first with hematology. PNH, which affects approximately 15,000 patients worldwide, represents the first potential indication to market for systemic pegcetacoplan and the most advanced program included in our collaboration with Sobi. We recently achieved an important step forward in Europe with the European Medicines Agency validating our Marketing Authorization Application, or MAA, for pegcetacoplan in October. We expect a decision by the U.S. Food and Drug Administration regarding the acceptance of the new drug application and a Prescription User Fee Act, or PDUFA, target action date in the fourth quarter of 2020.
These submissions were based on results from the phase III PEGASUS study, which met its primary endpoint demonstrating the superiority of pegcetacoplan to eculizumab, with a statistically significant improvement in hemoglobin levels at 16 weeks. Moving forward, Sobi will be responsible for all regulatory activities for systemic pegcetacoplan in ex-US markets, and we plan to transfer the European MAA to them. We remain on track to present 48-week top-line results from the PEGASUS study later this year, as well as top-line results from the phase III PRINCE study in treatment-naive patients with PNH in the first half of 2021. As part of our collaboration with Sobi, we will also further explore the potential of systemic pegcetacoplan within hematology. We plan to start two new registrational programs in cold agglutinin disease, or CAD, and in hematopoietic stem cell transplantation-associated thrombotic microangiopathy, or HSCT-TMA.
Sobi, with its world-class hematology franchise, will take operational responsibility for these programs. In CAD, our phase II open-label trial demonstrated increases in hemoglobin levels in patients following treatment with pegcetacoplan, as well as reduced markers for extravascular and intravascular hemolysis. Fatigue scores showed an improvement of 9.7 points from baseline, tripling the three-point improvement threshold that is considered a clinically significant increase. Pegcetacoplan was generally well-tolerated, no serious adverse events were reported. Approximately 10,500 patients in the U.S. and Europe are living with this rare disease, there are currently no approved therapies available. Sobi plans to initiate a phase III trial for CAD in 2021. HSCT-TMA is a new hematological indication for systemic pegcetacoplan. We believe that targeting C3 could help treat conditions associated with TMA, where high levels of complement activation are common.
Our registrational program will study pegcetacoplan in patients with HSCT-TMA, where we believe pegcetacoplan can be a best-in-class life-saving therapy. TMA is a disease where blood clots form in small blood vessels and is an often lethal complication of HSCT. There are approximately 9,000 and 18,000 transplants conducted each year in the U.S. and EU, respectively. TMA incidents can be seen in up to 40% of cases. As you can see in the graph on slide 10, TMA has a significant impact on the mortality of HSCT patients. There is a substantial unmet need for a treatment for these patients, we plan to start a potentially registrational phase II study in 2021. Next, in nephrology, Apellis will continue leading the development activities for the registrational program we recently initiated in immune complex membranoproliferative glomerulonephritis, or IC-MPGN, and C3 glomerulopathy, or C3G.
Primary IC-MPGN is a new indication for Apellis. Like C3G, primary IC-MPGN is a rare disease that often leads to kidney failure. With these trials, Apellis and Sobi will be the only companies actively studying a potential treatment for patients with IC-MPGN, a disease where both the alternative and classical pathways have been implicated. Approximately 18,000 patients in the U.S. and Europe suffer from IC-MPGN and C3G, which have no approved medicines. Our open label phase II discovery trial, which evaluated pegcetacoplan in patients with C3G, was designed with a primary endpoint of change from baseline in proteinuria at week 48. Proteinuria, or loss of blood proteins in the urine, is a common finding in C3G patients and an important marker of kidney damage.
Data at week 48 were recently presented at the American Society of Nephrology Kidney Week and showed that pegcetacoplan demonstrated a 73% reduction in mean proteinuria from baseline, highlighting its potential to address the underlying cause of C3G. Importantly, this reduction in proteinuria was accompanied by a corresponding increase in mean serum albumin. No serious or severe adverse events were reported, and pegcetacoplan was well-tolerated overall. Our registrational program in IC-MPGN and C3G includes phase II and phase III studies. The phase II trial is focused on the histopathology of the kidneys, and the first patient is expected to be dosed by the end of the year. We plan to begin the phase III study in the first half of 2021, with reduction in proteinuria as its primary endpoint.
Apellis will also continue to lead the registrational program we recently initiated with amyotrophic lateral sclerosis, or ALS. A devastating disease with high unmet need and the first indication from our new neurology franchise. As we demonstrated in PNH, we believe that targeting C3 can set itself apart from C5, as well as other more narrow-acting complement inhibitors, as the most effective way of controlling complement in a wide range of serious neurological conditions. In ALS, individuals with the disease have high levels of activated C3 in the neuromuscular junctions, where the neurons communicate directly to the muscle cells. Numerous studies suggest that the elevated levels of C3 present in ALS may be responsible for the chronic neuroinflammation that leads to motor neuron death.
We believe that systemic pegcetacoplan, by targeting C3, may have the potential to slow the progression of ALS, a disease that impacts approximately 225,000 patients worldwide. Our potentially registrational phase II placebo-controlled randomized study is enrolling approximately 200 adults with sporadic ALS, and the first patient is expected to be dosed by the end of this year. The primary endpoint will be the Combined Assessment of Function and Survival, or CAFS rank scores at week 52. We believe that C3 plays a central role in numerous neurodegenerative conditions, and we look forward to becoming a leader in this field. To summarize, we believe that Sobi is the ideal collaboration partner to maximize the broad platform potential of our targeted C3 therapy. Together, we plan to rapidly advance systemic pegcetacoplan in five registrational programs across hematology, nephrology, and neurology.
We expect that this progress will occur in parallel to the commercial preparations for our first potential launch in PNH, with Apellis focusing on the anticipated U.S. launch and Sobi managing the European and rest-of-world markets through its global hematology franchise. We expect that this collaboration will provide Apellis with bandwidth to prepare for the global launch of intravitreal pegcetacoplan in GA, which has blockbuster potential and for which Apellis has retained worldwide rights. Finally, this collaboration further strengthens our already strong financial position, extending our projected cash runway into the second half of 2022. We are proud to collaborate with Sobi and look forward to working together to flawlessly execute the clinical development plan outlined today and deliver on the broad platform potential of our targeted C3 therapy, systemic pegcetacoplan, for patients with serious rare diseases.
For additional information on the financial terms and obligations of this collaboration, I will refer you to our press release issued this morning and the associated filing with the SEC. Before we move to Q&A, I want to thank our employees, as well as the patients, investigators, and investors who helped advance our pipeline to its current stage. We look forward to this new chapter of growth for pegcetacoplan. Now, operator, please open the call for questions.
As a reminder, to ask a question, you will need to press star then one on your touch-tone telephone. To withdraw your question from the queue, please press the pound key. Please keep yourself to one question and one follow-up question before rejoining the queue. Please stand by while we compile the Q&A roster. Our first question comes from Justin Kim with Oppenheimer. Your line is now open.
Hi. Good morning. Thanks for taking my questions, and congratulations on the partnership this morning. We've spoken a lot about pipeline optionality and systemic complement-mediated diseases, and it's been very interesting to see that evolve over the past year. Can you just talk a little bit about how you see C3-targeted approaches being differentiated clinically and commercially for the Sobi-led diseases in CAD and HSCT-TMA?
Sure. Thank you, Justin, and great hearing you. In cold agglutinin disease, we have delivered a proof of concept that I think you are familiar with. We administer pegcetacoplan subcutaneously twice per week, exactly the same way we do in PNH. In our phase III clinical trial, we hope to replicate the efficacy profile combined with the advantageous subcutaneous administration. In the hematopoietic stem cell transplantation setting for thrombotic microangiopathy, we will provide further details, but in general, I would say that C3 is, we believe, a pivotal target within the context of TMA, where C5 and C3 have been studied individually in the role in thrombosis and where C3, we believe, offers significant advantages.
Got it. Maybe just a follow-up. As broadly as you think about the partnership, I know it mentions that APL-9 sort of would not pursue some of these listed indications. Just wondering whether second-generation molecules and molecules that maybe optimize the formulation or the sort of PK properties of pegcetacoplan would also fall under the agreement or not.
The agreement solely covers pegcetacoplan administered twice a week subcutaneous, as it was used in the phase III clinical trial in PNH, not follow-up.
Okay, great. Thanks, guys. Congrats again.
Thank you, Justin.
Thank you. Our next question comes from Anupam Rama with JP Morgan. Your line is now open.
Hey, guys. Thanks so much for taking my question. Congratulations on the collaboration. A quick accounting question. For the $80 million over the next four years, are they tied to development milestones, or are they going to be recorded on a more linear basis? Then just a confirmation question real quick. Cedric, I think you mentioned that there is a joint steering committee. Is that for all the indications or just the Sobi-led indications? Thanks so much.
Thank you so much. I will let Tim take the financial question and then get to the second.
Sure. Thanks, Cedric, and thanks, Anupam. It's the latter. These will get recorded more on a linear basis. These are R&D reimbursements fairly evenly spaced over that four-year period.
Yeah. Thank you, Anupam. The answer to your second question is very simple. We are going to be talking about all of our developmental programs together, in a true partnership fashion.
Great. Thanks so much for taking our questions, and congrats again on the deal.
Thank you. Great hearing you, Anupam.
Thank you. Our next question comes from Jonathan Miller with Evercore ISI. Your line is now open.
Hey, guys. Thanks for taking the question and congrats. I'll join everybody else in saying congrats on what looks like a pretty good deal. Sobi's a good partner for you ex-U.S. here. My question is on ALS. I understand you're starting that trial at a phase II this year. When you get first patient in, what's your expectation for data timing there and the competitive profile of pegcetacoplan in that indication, given the dosing regime relative to other players in that disease?
Yeah. Thank you so much, Jonathan, for that question. ALS is obviously a disease where the unmet need is staggering and where new therapies are needed. The role of C3 has been studied extensively for a very long time in ALS. Within the complement pathways, as a particular target stands out as probably the most attractive one. Now, the twice-a-week subcutaneous dosing is actually well-tolerated and very convenient for patients. We know that from our trials in PNH. We're not guiding towards when these trials will be finished yet, but we believe that enrollment will be relatively straightforward, and we look forward to the outcome.
Thanks. One follow-up, I guess. I understand that you're maintaining your ex-U.S. rights to GA, which obviously is the big indication here. What are your plans for building an ex-U.S. sales force and penetrating the global market there, given your leveraging of ex-U.S. from Sobi for all those other indications?
Yeah. Thank you so much for that question, Jonathan. Geographic atrophy for us is just an extraordinary opportunity, both from a medical and a company perspective. We will have the readout 10 months from now or in the third quarter of next year. That's right around the corner. It's an opportunity where in the past couple of months, we have further discovered efficacy profiles for pegcetacoplan. We had the release with the late-breaking abstract at EURETINA, where we basically showed, in a post-hoc setting, that in retina areas where atrophy has not completely penetrated yet, but where the cells are at risk of dying, we have an effect with pegcetacoplan at reducing the rate of progression to full GA. We also had, of course, the 18-month data on the small safety study that we ran with pegcetacoplan before we engaged in our phase III clinical trial.
All of that has increased our confidence. We are super excited about having the global rights exclusive to ourselves as we go into that readout. For the second part of your question, I will hand it over to Adam Townsend, our Chief Commercial Officer.
Thanks, Cedric. Thanks, Jonathan, for the question. Yeah, we've created a very experienced and lean leadership team in Zug, in Switzerland, that looks at our ex-U.S. commercialization. We'll be partnering in true collaboration style with Sobi, with this team, and then we will transition these great leaders across to really start to prepare for a global worldwide launch of geographic atrophy, which has blockbuster potential. The expertise we hired, we will move across to GA. We were super thoughtful when we were building out the team to make sure that we had all of the right people to help us really explore this GA opportunity.
Thanks so much, guys.
Thank you.
Thank you. Our next question comes from Steve Seedhouse with Raymond James. Your line is now open.
Good morning, and congrats. Can you comment on the anticipated timelines for PNH approvals and launch, obviously in regions beyond U.S. and Europe, given Sobi's controlling that? Also how the regulatory and commercial milestones split between PNH and everything else that's covered. I have a follow-up as well.
Yeah. Thank you so much, Steve. I'm not going to comment too much yet. As you know, and as we've disclosed, our filings were done both with EMA as well as with the FDA in September. The file has been accepted by EMA, and we're waiting to get a PDUFA date from the United States regulators. These other indications, again, kind of part of the, I'd say, the attractiveness of the deal with Sobi was their interest to collaborate with us in exploring these multiple new indications. As you know and can appreciate, it's very difficult to synchronize these indications medically, regulatorily, commercially from a pricing perspective. We found a partner in Sobi that I think is going to allow us to do that really well. Timelines, you should think about the conventional timelines in terms of approvals.
We believe that the PEGASUS study gave us the safety and the efficacy profile that will lead to approvals in the two territories. Look forward to sharing more on that in the years to come.
Okay. Just wondering if you could split out the milestones between PNH and the other indications, if you can. If not, just had a follow-up. I wanted to ask about the anticipated development costs. $80 million is going to be reimbursed to Apellis. It looks like Apellis is reimbursing Sobi for the studies in CAD and TMA. I guess the first question is, what amount do you anticipate reimbursing Sobi for those two studies? Then is the $80 million to be reimbursed to you, basically 50% of the cost of the other programs in PNH, nephrology, and ALS? Or is Sobi share of those costs something other than 50%? Thank you.
Thank you so much. I'm going to hand that over to Tim to answer.
Sure. We haven't broken out the PNH regulatory and commercial milestones versus the total of $950 million, which we've disclosed. We don't have any more color we can offer there. In terms of the development cost, this R&D reimbursement of $80 million over the next four years was intended to be roughly 50/50 of the four trials that have been added in addition to PNH. The CAD, TMA, C3G, IC-MPGN trial, and also the ALS trial. We ended up announcing doing those on our own anyways. What's really nice about this transaction in terms of those $80 million in R&D reimbursement is that it effectively covers on a fully loaded basis, the two additional trials, the CAD and the HSCT-TMA trials over that period of time.
Functionally and transactionally, what happens is they do reimburse us, and in effect, we pay them for almost the same amount for those two trials. It's a really nice part of that story where we get these two trials added on to our existing development franchise for systemic pegcetacoplan.
All right. Thank you so much.
Thank you so much, Steve.
Thank you. Our next question comes from Matthew Luchini with BMO Capital Markets.
Hi, good morning. Thanks for taking the question and congrats on the collaboration. I guess, maybe just stepping back a little bit, why now? What is it about where the company is today versus maybe on the other side of GA or some other time point that made this deal make sense for the company? Then relatedly, it sounds like it was a pretty competitive process. It sounds like the company was pretty adamant about maintaining the rights to GA. I'm just wondering if you could talk a little bit about how important maintaining those rights were as you were working through discussions with other partners. In other words, maybe how much was keeping GA rights a sticking point for you guys in your discussions with others? Thank you.
Thank you so much, Matthew, for that question. Critically important is the answer. We have great belief in the geographic atrophy program, which we believe will be transformational in the retina. We look forward to a long story there in the area of ophthalmology. Now, as it relates to the deal, why now, and why with Sobi? As I mentioned during the call, Sobi, because it is a company for whom this deal is as important as it is to us, right? As you know, when you deal with a large pharmaceutical company, it's easy to become deprioritized. That will not happen here. This is going to be a marriage or a partnership of equals, where we are going to jointly make this drug available to as many patients as possible in as many indications as possible in as many geographies as possible.
That kind of summarizes it there. Why we did this deal in the first place, as I mentioned during the call as well, because we can go wider and faster. I mean, pegcetacoplan, if it has shown us anything so far, is going to be exquisitely promising efficacy and safety profile that we have there. I want to remind you that the mechanism behind, or by which pegcetacoplan exerts its effect is, in our opinion, the magic, right? That is a mechanism that we believe that in many indications, will make a big therapeutic difference. To be able to do that efficiently, quite frankly, requires a partner for us, a committed partner to these multiple therapeutic areas. That opens up the bandwidth for us to be ready for geographic atrophy, which will be, should it be positive, a transformational event for the company.
Of course, the fact that we could further shore up our balance sheet, which was already strong. That's our kind of all the elements that went into play.
Great. Just recognizing everything you just said, does Sobi have even, like, a right of first refusal should your thinking on GA change or anything like that?
No. This deal is exclusively around systemic pegcetacoplan administered twice per week subcutaneously for its systemic indication.
Great. Well, thank you for taking the questions, and congrats again.
Thank you, Matthew.
Thank you. Our next question comes from Madhu Kumar with Baird. Your line is now open.
Hey, guys. This is [Raul] on for Madhu. Congrats on the deal. I was just wondering, how does this impact the partnership with the agreement with SFJ?
Thank you so much for that question. I'm going to pass to Tim.
Sure. Hey. Thanks for the question. Apellis is still responsible for the SFJ payments. Sobi has agreed to fulfill diligence obligations to SFJ. Obviously, SFJ had to give their consent to the transaction, which they did. Other than that, nothing has changed.
Okay, thanks. I was also wondering, what happens if there's interest in additional indications?
You mean to develop a systemic pegcetacoplan?
Yes.
Yeah. In the agreement, we have a mechanism by which new indications can be pursued jointly as well.
All right. Thank you.
Thank you.
Thank you. Our next question comes from Phil Nadeau with Cowen and Company. Your line is now open.
Good morning. Let me add my congratulations on the deal. First, one question on commercialization. How is the commercial price going to be determined in ex-U.S. territories? Is there any mechanism, in the deal to try to coordinate it, one worldwide price, or does Sobi have free rein in how they price, in any individual territory?
Thank you very much, Phil. I'm going to hand that question to Adam. Adam, you are muted. I think, Adam, that your line is muted.
Adam, your line is connected.
Sorry. I think I was on mute. Thanks for the question, Phil. Welcome to conference calls and the mute button. Yes, we've done some great work on pricing, strategically, globally, and ex-U.S. We'll be sharing all of that work through the joint steering committees with our partners, Sobi. They'll apply their expertise and experience as well, and then they'll move forward with choosing their pricing ex-U.S. We've got a great potential partnership and collaboration ahead of us.
They have access to all the market research you've done. Ultimately, though, the pricing decision in their territories is theirs. Is that correct?
Correct.
Correct. Okay. Perfect. Just to follow up on the IC-MPGN and C3G study. I guess from the slides that wasn't clear, is that a combined study with both patient populations, or are there going to be two trials in parallel on kidney disease, run side by side? Thanks.
Thank you so much for that question, Phil. It will be a combined study. Without providing further detail, you should expect that to be a stratification.
Perfect. Thanks for taking my questions.
Thank you so much.
Thank you. Our next question comes from Brian Cheng with Bank of America. Your line is now open.
Hi. Good morning, guys. Congrats on the partnership, and thanks for taking my question. My first question is on the commercial front for PNH. Can you give us a bit more color, granularity on your preparation on the U.S. launch? Any updates on your thinking around the size of the sales force to launch for this indication in both the European regions and also U.S.? There may be some potential synergy with their existing infrastructure in hematology.
Thank you so much, Brian, for that question. I'm going to hand that one to Adam as well.
Yeah. Thank you, Cedric. Thank you, Brian. Yeah, we're flawlessly on plan for the execution of the launch of PNH in the U.S. We are currently in the field, and also where appropriate and virtually interacting with key opinion leaders, patients, and partners appropriately. We're on track to build out our sales force, and we've done some great research, looking at the right type of sales force to address the prescriber base and the patient population. We expect to be launching a sales force that's pretty lean, pretty nimble, but super sophisticated, in these COVID times to be able to balance the in-face meetings where they're appropriate and accepted by the physicians, and also to work in a virtual environment. We're all on track.
We're currently in the field, as I said, through medical affairs, through some commercial infrastructure, plus through some market access infrastructure as well. All plans are pointing in the right directions for our U.S. commercialization. Brian, you also asked about ex-U.S. We had a robust plan again to launch lean and nimble teams. We'll be sharing that plan with our partners, Sobi, and sharing our insights on the prescriber base and the potential in each of those geographies. We'll be by their side as they execute their plans, from an ex-U.S. perspective. Apellis is full steam ahead to commercialize PNH in the U.S., and we're looking forward to helping our partner ex-U.S.
Great. Thanks, Adam. Maybe one quick one on C3G as well. Noticed that the NOBLE study is studying in the post-transplant patients. Is the phase III study that's about to start in first half next year also focused on post-transplant setting, or is it going to be a broader indication?
That is correct. It will also be focused on post-transplant.
Okay. It will include patients before transplant as well, right?
Before and after, yes.
Okay, great. Thanks, Cedric. Thank you.
Thank you.
Thank you. Our next question comes from Alethia Young with Cantor Fitzgerald. Your line is now open.
Hey, guys. Thanks for taking my question. Congrats on the progress and for squeezing me in for my first Apellis call here. Number one, maybe just can you talk a little bit about how decisions were made around who would maintain operational responsibility? I guess, just near term and thinking about upcoming launches, how much having some additional cash upfront helped you accelerate your business overall? Thanks.
Thank you so much, Alethia. As it relates to the operational responsibility, we will act through these joint governance committees, which are across development and commercialization. We will be in charge of the operational developmental responsibilities in C3G and ALS, whereas Sobi will take the lead in cold agglutinin disease and the HSCT-TMA. Then for the second part of your question, I will hand that over to Tim.
Hey, Alethia. Thanks for the question. As you probably know, we ended the second quarter with $833 million in the bank. With this $250 million upfront, that obviously extends our runway significantly, which allows us to support these four new indications that we had just announced, those first two, and then these new two with Sobi. It allows us to execute a commercial launch in PNH in the U.S. next year. It also allows us to accelerate our early research programs, build out general operations, probably most importantly, it allows us to get through our GA readout for a primary endpoint in the third quarter comfortably without feeling like we have to raise capital, also allows us to begin preparing for that global launch of GA that Adam was talking about.
We feel very good about our financial position post this transaction.
Maybe just one follow-up on the first question. I guess my question is, how did you guys make those decisions? One, how did you decide that you would keep ALS for example, versus keeping cold agglutinin? Can you just talk a little about that thought process between the two companies? Thanks.
Sure. Thank you, Alethia. Sobi is a company that has a long and deep story in hematology, so this is a field that they know extremely well and where they are a global leader. It was a natural fit for us to hand that over to them.
Great. Thanks.
You're welcome.
Thank you. I'm showing no further questions in the queue at this time. I'd like to turn the call back to Cedric Francois for any closing remarks.
Thank you so much, operator, for navigating the works here. To everyone on the call, thank you for joining us today. We are excited about this new collaboration with Sobi, and together, I am confident that we will maximize the platform potential of our targeted C3 therapy, systemic pegcetacoplan, across a broad range of serious diseases. We look forward to continuing our progress and keeping you updated on our latest developments along the way. Thank you so much for taking the time today, and we look forward to interacting on a one-on-one basis in the near future. Thank you.
Ladies and gentlemen, thank you for your participation on today's conference. This does conclude your program, and you may now disconnect.