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spk01: Good day, and thank you for standing by. Welcome to the Bluebird BIO fourth quarter and 2023 annual results conference call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you'll need to press star 11 on your telephone. You'll then hear an automated message advising your hand is raised. To withdraw your question, please press star 11 again. Please be advised that today's conference is being recorded. I would now like to hand the conference over to your first speaker today, Courtney O'Leary, Investor Relations. Please go ahead.
spk07: Good morning, everyone, and thank you for joining our fourth quarter and 2023 annual results call today. My name is Courtney O'Leary, Director of Investor Relations at Bluebird Bio. Before we begin, let me review our safe harbor statement. Today's discussion contains statements that are forward-looking under the Private Securities Litigation Reform Act of 1995, including expectations regarding our future financial results and financial position, in addition to statements of the company's plans, expectations, or intentions regarding regulatory progress, commercialization plans, and business operations. Such statements are based on current expectations and assumptions that are subject to risks and uncertainties and involve a number of risk factors that could cause actual results to differ materially from projected results. A description of these risks is contained in our filings with the SEC, which are available on the investor relations section of our website, www.bluebirdbio.com. On today's call, Andrew Obenshain, Bluebird Bio's CEO, will provide a general corporate update. And then Tom Klima, Chief Commercial and Operating Officer, will discuss progress on the commercial launches of Lifgenia, Zinteglo, and SkySona. And finally, Chris Krawchuk, Chief Financial Officer, will provide a financial update before opening the call up for Q&A. With that, I will turn it over to Andrew.
spk02: Thanks, Courtney. And thank you, everyone, for joining our call this morning as we provide an update on our fourth quarter of 2023 and the results and discuss our strategic outlook and plan milestones for 2024. Earlier this morning, we put out an 8K announcing that we will be restating financial information for 2022 and for the first three quarters of 2023, and that we've submitted an extension on our 10-K filing. We now anticipate filing the 10-K, inclusive of that restatement, no later than April 16th. Importantly, the restatement does not impact our cash position or revenue. Chris will discuss this in greater detail later in the call. 2023 was a year of significant progress for Bluebird. We established ourselves as a commercial gene therapy leader with a validated commercial strategy that brought both Zanteglo and Skysona to patients across the U.S. Our recent commercial progress for Lithgenia builds on that foundation. With the FDA approval of Lithgenia for sickle cell disease last December, Bluebird is currently the only commercial gene therapy company with three FDA-approved products. Since then, our commercial team has hit the ground running, position ourselves for a strong Lithgenia launch. We have talked before about the benefits of our significant commercial head start with Centeglo, and today we are seeing the fruits of that labor translate into real momentum for Lithgenia. We have activated 62 qualified treatment centers and have signed our first outcomes-based agreement for Medicaid, extending access to Lithgenia to this critical patient population. And 2024 is also a fast start with nine patient starts already this year. Tom will dive deeper into our commercial progress momentarily. Last week, we also announced that we entered into a five-year term loan with Hercules Capital for up to $175 million. This transaction provides a bluebird with an infusion of capital that is expected to extend our cash runway beyond the next 24 months. Last August, on our Q2 earnings call, we set out a five-year vision for Bluebird as a standalone commercial gene therapy company. We outlined our near-term path to profitability and our expected additional growth and scale over the next five years. This funding forwards that vision to capitalize on the multi-billion dollar opportunity in front of us in the U.S. I will now hand the call over to Tom to highlight the significant progress happening in our commercial launches.
spk15: Thanks, Andrew, and good morning, everyone. As Andrew noted, 2023 was a pivotal year for Bluebird, in which we built our commercial foundation with Enteglo, beta-thalassemia, and SkySona for cerebral adrenal leukodystrophy. Today, we're capitalizing on that head start to accelerate the launch of Lipgenia for sickle cell disease. As a reminder, our launches are focused on three core elements for success. First, establishing a robust network of qualified treatment centers, or QTCs. These are transplant centers with significant experience in cell and gene therapy. As Andrew mentioned, today we have 62 activated QTCs, unparalleled compared to others in the field. Second, ensuring the value of our therapies are recognized and that patients have timely, equitable access. In a moment, I'll dive deeper into our progress on access and reimbursement. And lastly, optimizing the patient and provider experience. Gene therapy is a high-touch business where transparency, collaboration, and understanding the needs of patients, families, and providers is paramount. Our deep understanding of the gene therapy process, our dedicated focus, and our experience over the last 18 months allow us to be a strong partner to our activated QTCs as we help them bring life-changing therapies to their patients. Three months into the Legenia launch, patient demand is strong. I am very excited to share today that we expect our first patient start will be scheduled imminently. And we are extremely encouraged by the number of enrollments and patients preparing for treatment from multiple treatment centers across our QGC network. We anticipate starts for Lipgenia to grow quarter over quarter, with the majority occurring in the second half of the year as momentum builds. We anticipate that the first revenues for Lipgenia will be reported in Q3. Moving to Zynteglo. We continue to see strong linear growth with seven patient starts since the beginning of 2024, in addition to the 20 patient starts completed for Zentegra in 2023. Additionally, we've completed two patient starts for Skysona since the beginning of 2024, in addition to the six patient starts completed for Skysona last year. And as a reminder, patient starts, which is when cell collection occurs, remains the key commercial metric to watch in the early stages of our launches, as this is the value creating moment for the company, with revenue being recognized when the patient is infused. For modeling purposes, you can expect that patients are infused one to two quarters following cell collection. And in our experience, once a patient goes through the cell collection, they continue on the treatment journey. To date, every patient who has started the process has either completed the process or remains in the process. Going forward, we plan to provide quarterly updates on patient starts for each of our therapies as we work towards the 85 to 105 patient starts we guided to in 2024. Turning to access and reimbursement, gene therapies are one-time transformative treatments and bring tremendous value to the healthcare system and to patients. For years, Bluebird has led the way in articulating the value of gene therapies to payers and charting paths to access for patients. We continue to see timely access for Zenteglo and SkySona with zero ultimate denials for either therapy across both Medicaid and commercial payers. Payers are recognizing the value of these therapies and the high burden of disease. And today, we are seeing the same early success with Lefgenia, where our goal is timely, equitable access for patients. And our approach is working. We have designed outcomes-based agreements that are unique to Lefgenia, offering payers meaningful risk sharing tied to VOU-related hospitalizations with patients typically followed for three years. In addition to a commercial offering, we've designed an offering specifically for Medicaid that addresses the need for predictability and operational ease that is essential for states grappling with resource constraints. Earlier this month, We announced that Bluebird signed its first outcomes-based agreement for Lithgenia with Michigan's Medicaid program, demonstrating support from government payers for these types of innovative arrangements. We've also signed a total of four commercial outcomes-based agreements for Lithgenia, covering more than 200 million lives. In three short months, we've already seen close to 90% of coverage policies Covered with Jenia at parity, including policies from major national insurers such as Aetna, Anthem, and Cigna. We continue to be focused on access for Medicaid patients in 2024, and discussions are ongoing with more than 15 Medicaid agencies, representing 80% of individuals with sickle cell disease in the U.S. We are also actively engaged with CMMI on its cell and gene therapy access demonstration that is slated to begin in 2025, which could serve as a bandwidth extender for states who do not have the ability to implement outcomes-based agreements on their own. Moving to our QTC network, one of the most significant advantages of our commercial head start has been our ability to build our synergistic QTC network. We now have 62 activated centers the largest network of qualified treatment centers for hemoglobinopathies, 49 of which are already ready to receive referrals for Lipgenia today. This means that approximately 95% of people living with sickle cell disease are within 200 miles or less of a Bluebird QTC. We anticipate additional QTC expansion across our portfolio in 2024. We also recently signed agreements with QTCs and geographies with important proximity to sickle cell patients, where we are meeting patients where they are in Georgia, across Florida, and throughout the New York metropolitan area. Additionally, five centers have also been activated to administer SkySona for patients with CALD. We were able to quickly activate our Lefgenia QTC due to both learned experience of setting up these centers for Zenteglo and the strong relationships we have built with these centers throughout 2023. From onboarding through Infusion, our QTCs are comfortable with our process and have grown to expect the transparency and the partnership that Bluebird brings to the table. To recap, Zintegro and SkySona launches continue to progress as planned, and we anticipate strong linear growth from Zintegro in 2024. We've made incredible early progress in our Lipgenia launch, building on our validated commercial platform. We are seeing clear demand from patients and physicians Payers are recognizing the value of Lifgenia in providing access, and our Q2C network is fully ready to help patients start their journey with the curative therapy. And now I'd like to turn the call over to Chris.
spk03: Thanks, Tom, and good morning, everyone. Before discussing the details of the restatement and the filing extension we announced this morning, I want to highlight the financial results we reported in our press release. In the fourth quarter, we reported $7.8 million in total revenue, primarily driven from product revenue from Zanteglo and SkySona. As a reminder, we recognized revenue upon infusion of the drug product. Additional drug product for several patients were delivered at the end of December and infused in January. So this revenue we recognized in the first quarter of 2024. For the full year 2023, we reported $29.5 million in net revenue with $16.7 million of revenue attributable to Zintegro and $12.4 million of revenue attributable to SkySona, with cumulative gross to net discounts across the portfolio of two products of approximately 19%. In 2024, we anticipate gross to net discounts in the range of 20% to 25%, with fluctuations based on product and payer mix, as well as utilization of our outcomes-based agreements. Going forward, Bluebird plans to provide revenue by product on a quarterly basis, and as Tom shared earlier, we expect the first Lefgenia patient infusion and revenue recognition in the third quarter of 2024. We ended the year with cash and cash equivalents, marketable securities, and restricted cash of approximately $275 million, which includes $53 million of restricted cash. We're very pleased to announce that we entered into the five-year term loan with Hercules Capital for $175 million. This financing provides an infusion of non-diluted capital for Bluebird, helping fuel the launch of Livchenia and deliver on our mission as a company. The loan provides for up to $175 million in the aggregate, and it's available in four tranches. The first tranche and the amount of $75 million was drawn on closings. we may draw additional two tranches over the next 18 months in aggregate of an amount up to $50 million. And this is subject to the achievement of commercial milestones tied to Lufthansa patient starts and gross profit. The fourth tranche of up to $50 million may be available at the sole discretion of Hercules. Importantly, and to reiterate Andrew's comments, based on our launch estimates and our current business plans and assuming the first three launches are executed, The transaction is expected to extend our cash runway beyond the next 24 months and puts us in a durable capital position as we approach profitability. Lastly, this morning we announced we restate financial information for the full year of 2022 and for the first three quarters of both 2022 and 2023. The restatement relates to the application of our accounting policies for contracts with our contract manufacturing organizations and suppliers. which did not consistently combine lease and non-lease components and was discovered by our internal finance team during the preparation of our annual financial statements. For 2022, we estimate the understatements of lease assets and lease liabilities of approximately $100 to $200 million for the full year, as well as an estimated understatement of lease assets and liabilities of approximately $30 million to $125 million for the first three quarters of 2022 and 2023. While we're still working through the exact details, the lease will be reclassified as finance leases as a result, and we anticipate the increase of non-cash interest expense on our P&Ls. The restatement is not expected to have any impact on our cash position or revenue. In consideration of the time it will take to complete the restatement, we have filed for an extension of our 10-K and we anticipate that our 2023 10-K, inclusive of the restated periods, will be filed April 16th. And with that, let me turn it back over to Andrew.
spk02: Thank you, Chris. And to summarize, Bluebird is deploying a validated commercial gene therapy strategy with three FDA-approved therapies taking flight in 2024 and delivering on our promise to patients and their families. We expect 2024 to be a transformative year for our company, and we look forward to providing more updates in the upcoming quarters as our commercial launch of Let's Genium progresses. And with that, I'd like to open it up for questions. Operator?
spk01: Thank you. As a reminder, to ask a question, you'll need to press star 11 on your telephone. To withdraw your question, please press star 11 again. Please wait for your name to be announced. We ask that you please limit your questions to one and one follow-up until all have had a chance to ask a question. Please stand by while we compile the Q&A roster. One moment for our first question, please. Our first question will come from the line of Jason Gerberry with Bank of America. Your line is now open.
spk09: Hey, guys. Thanks for taking my question. I was wondering maybe if you can just speak to, in the fourth quarter, the quarterly step down in revenue and what you're seeing there, to what extent that may be speaks to sort of the conversion dynamics of these starts to an actual revenue recognition event. And then, can you comment at all just as a follow-up, just directionally, the flow of New Starts first half versus second half and your outlook for the full year? Thanks.
spk02: Yeah, good morning, Jason. Thanks for the question. So I might ask Tom to just talk a little bit about the patient dynamics and how they're coming in and the flow both. the fourth quarter with Zynteglo, and then for the first and the second half of this year with Lipgenia. Go ahead, Tom.
spk15: Yeah, good morning, Jason. Thanks for the question. So, we saw, you know, strong demand and building demand over the course of the year last year for both for Zynteglo and then, as we expected, we started six patients for SkySoda. With Zynteglo, we actually collected a number of patients at the end of the year that elected to be treated after the start of the new year. So although the collections were on track in Q4, many of those patients went on to be infused in Q1. So that was the reason you saw a little bit of a dip in Q4. But we remain very positive on patient demand force in Teglo. We also are seeing just very strong demand force for Lipgenia. We expect a linear build over the course of this year with the vast majority of the Lipgenia starts happening in the second half of the year.
spk02: Jason, just to reiterate what we said earlier, too, we had some drug products that were delivered to the QTCs in Q4, but actually were not infused, and those infusions will happen in Q1 and be recognized there. And I would also say that, you know, quarter to date, where we've had our best, strongest quarter to date on starts, nine total. Got it. Thanks.
spk01: Thank you. One moment for our next question. Our next question comes from the line of Danielle Breaux with Raymond James. Your line is now open.
spk10: Hi, guys. Good morning. Thanks so much for the question. I guess as a follow-up to Jason's, do you still expect Lithgenia to make up over 50% of the total volume this year? And then I know you said that the first patient start is imminent, but I believe in the past you communicated that multiple patients would start in one queue. Just curious what's getting those patients started. Thank you.
spk15: Ask Tom to answer that. Go ahead, Tom. Yeah, so again, we're excited about the momentum and the demand we're seeing with Leukgenia. We do expect roughly half or more of the volume to come from Leukgenia. Obviously, it's a much larger patient population, and we should see a lot of the starts starting to pick up in the second half of the year as we build momentum. We did say that we would have a patient started in Q1. We believe that we'll have our first patient started very soon, imminently. We've seen Again, very strong demand, and what we've seen so far is a number of patients who have enrolled in the process and who are going through the process of getting ready for treatment across a number of different QTCs. And keep in mind that if you look at, just refer you to the package insert for Lipgenia, it takes about two months on average for patients to be medically ready as they have to wash out their hydroxyurea or disease-modifying therapies that they might be on for at least two months. They have to also go through two rounds of transfusions, one month each. little longer to get ready for therapy.
spk01: Got it.
spk10: Thanks so much.
spk01: Thank you. One moment for our next question. Our next question comes from the line of Jack Allen with Baird. Your line is now open.
spk13: Great. Thanks for taking the question. I wanted to ask about your manufacturing capacity and how you think about, you know, potential influx of patients in the quarters and essentially the second half as well. Can you put out some color as it relates to what capacity you have and how could you meet demand as it comes in in different kind of waves as we think about the second half being really the build here as it relates to the genia?
spk02: So, good morning, Jack. I'll take that one. So, first of all, I think the important thing to understand is that the supply chain for Zantegla or SkySona is one supply chain, and there's a supply chain for the genia, so the two don't impede upon each other. And the way we've designed it, and obviously we've designed our capacity for Lex Janian to be larger than our capacity for Centegra and SkySona in anticipation of a larger demand there. And we do on both sides, both with Vector and with drug product, we do anticipate that we have the volume or the capacity to meet the initial launch expectations. And what we also have in place is plans to expand that capacity as we see demand coming in. So we do anticipate that we do have capacity for this year and the launch phase of all of our products.
spk04: Thanks.
spk01: Thank you. One moment for our next question, please. Our next question comes from the line of Jenna Wang with Barclays. Your line is now open.
spk06: Thank you for taking my questions. I wanted to know that now you have a little bit early information real-world experience, what is your expected from start to finish for left genia?
spk15: Good morning, Gina. Tom, go ahead. Yeah, good morning, Gina. So what we're saying is that obviously from cell collection to infusion is one to two quarters. And the biggest part of that time is just the manufacturing time. And the range in the package insert is between 70 and 105 days plus two weeks. So that's the vast majority of the time. And then we obviously ship it back to a QTC and then it's up to the QTC when they infuse it. So we're saying for modeling purposes, one to two quarters.
spk01: Thank you. Thank you. One moment for our next question. Our next question comes from the line of Eric Joseph with JP Morgan. Your line is now open.
spk08: Hi, good morning. Thanks for taking the question. I guess just if we're looking back at 2023, can you just provide a little more color on how many of the 24, I think, total cell collections contributed to revenue generation and whether they're, yeah, explicitly how many patients actually contributed to revenue recognition and then, you know, how average time to treatment between collection and treatment has trended. What avenues do you anticipate there being to sort of improve conversion rates going forward? Thanks.
spk02: So, Eric, good morning. So, with 26 collections in 2023, every single patient that has gone through the process is still in the process. They've either been infused or had the drug product delivered or the product is in process. So, they're all still in the process. And we haven't actually given numbers of confusions, so I'm not going to say that directly, but we said we have, we infused the number in 2023. We have some products always delivered in Q4, but not infused, and you'll see that revenue in Q1, and some of those patients will be infused early this year. In general, you're going to see that the time from collection to infusion is one to two quarters. There will be some outliers there, just as the hospital chooses to infuse late or something else happens, but in general, it's one to two quarters. As we get more collections and as we get more data, we'll be able to narrow that number down for you and give you a kind of more precise number. But still, we're still dealing with relatively small numbers.
spk08: Okay. And to remain in process, I guess, is there a, I guess, a bracket timeframe? I guess, how long can a patient kind of be in process? Is it, you know, is there ultimately a nine, 12-month window?
spk02: So there's really no end date for them being in the process, except for the expiration of the drug product is just about a year, right? Once you manufacture it, that the drug product will expire after a year or a little bit longer. So that would be the end process. We don't anticipate anyone going that far. But again, remember, this is an election that the patient makes to get treated. So very often it will schedule it around life events, et cetera. That's why it's somewhat unpredictable once we deliver the product back to the hospital about when they will get abused. Okay.
spk08: Thanks for taking the question.
spk01: Thank you. One moment for our next question, please. Our next question comes from the line of Manny Furuhar with Lering Partners. Your line is now open.
spk12: Hey, thanks, guys. I want to pivot a little bit to thinking about broader market dynamics. You've obviously talked about finding patient demand, volume demand out there for Virginia, said that that's going to be perhaps half or more of the 8,505 patients. Can you give us a little bit of sense of how deep that backlog is, how much clarity you have into it? I know you can't give us a numerical guidance number of number of patients currently, but can you give us some, can you put some color around exactly what that backlog of identified potential of genia patients looks like? And I guess as a separate follow-up, can you give us a sense of exactly what demand metrics and other data was available to Hercules as part of your engagement with them prior to your transaction with them, just trying to understand their due diligence process as well.
spk02: Yeah. So, I'm going to pass it to Tom to talk about the backlog or the interest in Lufgenia. And Chris, I'll talk about just what we provided Hercules.
spk15: Go ahead, Tom.
spk02: Yeah.
spk15: Good morning, Monty. Keep in mind that if you look back a year, people were asking questions around, could we get to our goal of 40 to 50 QTCs, which was a factor in making sure that patients had access to therapy. People were also asking questions around reimbursement. And then lastly, people were asking about patient demand. Now we have 62 qualified treatment centers up and running. We've seen reimbursement happen. We have... positive coverage policy so far with Lefgenia, so that box is being checked. And now you're looking at patient demand, and we've seen historically in market research that over 70% of patients who have more severe sickle cell disease have said they would seek a gene therapy if recommended by their physician. And now we're seeing that play out in real life where we have multiple enrollments already just in a few months after approval across multiple QTCs. We wouldn't expect a huge bolus because patients, as Andrew mentioned, can get treated per their schedules. And also, I think QTCs are going to want to treat a handful of patients first before they go through their list of patients. But anecdotally, we've heard of long wait lists at QTCs of patients who have sickle cell disease that are looking to be treated. So again, I can't overemphasize how excited we are about the early demand that we're seeing.
spk03: Perfect. Chris, do you want to comment on what the bill has provided Hercules? Thanks for the question. With respect to what Hercules received, so Hercules received customary information as it relates to projections, not just 2024, but beyond that. And the confidence that having a third party come in and scrutinize our projections should provide confidence to the marketplace on the achievability of those, considering they entered into the arrangement for the $175 million financing. What I would say, though, is that the patient start guidance that you saw there for Lefgenia should not be viewed as consensus. That's viewed as progress. So that milestone associated with that is not consensus. We provided our guidance, which is the 85 to 105 starts.
spk04: Okay, that's helpful. Thanks, guys.
spk01: Thank you. One moment for our next question, please. Our next question comes from the line of Jeff Hung with Morgan Stanley. Your line is now open.
spk14: Thanks for taking my questions. For Lipgenia, with 49 QTCs already receiving referrals, what proportion of these sites might have their first patient cell collections in the first half of the year? What are the gating factors that might result in different timelines, particularly for the 35 sites that were online at the beginning of the year? And then I have a follow-up.
spk15: Go ahead, Tom. Yeah, sure. Yeah, we haven't really given specific guidance per QTC, but needless to say, a lot of QTCs were coming on board late last year in anticipation for Lipgenia launch and approval. So we would expect, you know, most of our QTCs to be pretty excited about getting patients started on Lipgenia. In most cases, they are waiting for patients to come in for their regular appointments. And in some cases, if they have a waiting list, say, for a patient that wanted to try a gene therapy through a clinical trial, then those patients might go faster. So really, the dynamic will play out, but we're excited about the early enrollments that we've seen and across multiple QTCs, not just one or two QTCs. And we're excited about the excitement for gene therapy. Okay.
spk14: Okay. Thanks. And then for the third tranche of the term loan facility, what would you need to see in patient starts by the end of the year to be on track to reach the $30 million gross profit milestone by the end of the first half of 25? Thanks.
spk03: Go ahead, Chris. So we haven't commented on that. What we commented on was the exact metric associated with that. But we're not going to comment on the patient start number. Remember, our patient start guidance that we provided in the second tranche was the 35 by September, the 55 by the end of the year for Lipgenia. And then there's a gross profit metric also. And then for the third tranche, but we're not going to provide that level of detail associated with the number of equivalent patient starts to get to the gross profit.
spk02: Sorry, Jeff, you were asking about the first tranche. You said the third tranche. I think you meant the second tranche.
spk14: Yeah, I was talking about the third tranche.
spk02: Yeah. So there's this first tranche of 50, second tranche of 25, which is that it's keyed off of either the 35 or the 55 starts by the end of the year. And then there's a third tranche, 25, that is keyed off of a different metric, which is gross profit.
spk03: Yes. Just to be clear, the first tranche was the 75. The second tranche is the 25. The 25 is attributable to the Lefchania patient starts, as I said, 35 by September, 55 by December 31st. And then the third tranche is associated with gross profit. Right. And then, of course, as I said, the fourth tranche is the 50 that's at the discretion of Hercules.
spk14: Yep. Thank you. Yeah, you answered it. You said that you wouldn't comment on the number of patient starts for the 30 million gross profit. Yep. Okay. Thank you. That's correct. That's correct.
spk01: Thank you. One moment for our next question. Our next question comes from the line of Salveen Richter with Goldman Sachs. Your line is now open.
spk11: Good morning. Thanks for taking my question. Just in line of the trajectory that you're seeing right now and the understanding of you know, the various onboarding dynamics, reimbursement dynamics. How are you thinking about the peak opportunity here for the drug? And just maybe an update for XUS commercialization. Thank you.
spk02: Yeah. So, Sylvia, I was a little hard to hear, but I think you asked what the peak opportunity is for the drug?
spk11: Yes. And XUS commercialization outlook.
spk02: Oh, excuse me. Okay, got it. So let me take that. So we do see this as a multi-billion dollar opportunity at peak in the U.S. alone. Ex-U.S., we do think there's a very attractive opportunity there as well. Right now, we as a company are focused exclusively on the U.S. If we go ex-U.S., we most certainly do that with a partner. We are watching very closely the reimbursement dynamics of gene therapy in Europe as well. That's kind of a key dynamic that will determine the opportunity there.
spk04: Thank you.
spk01: Thank you. One moment for our next question, please. Our next question comes from the line of Sammy Corwin with William Blair. Your line is now open.
spk05: Hi there, thanks for taking my question. I was curious if you could speak a little more as to the cost of good near gross margins and how you expect them to change as your launches across the portfolio continue. And then can you provide any granularity in terms of your assumptions for the Legenia launch this year and the percentage of patients you expect to be covered by Medicaid versus private insurers?
spk02: Yeah, sure. Good morning. So let me have Tom actually answer the second part first, and then I'll go to Chris. Tom, this is the percentage on Lefgenia and Medicaid.
spk15: Yeah, just to give you the stats again, about 50% of patients or people living with sickle cell disease are covered by Medicaid. About 45% are covered by commercial insurance and then a small portion of Medicare. It's hard to predict who the early patients will be, but we would expect over time that the payer mix would play out like we've seen it historically.
spk03: Just covering on the cost of sales, and maybe I'll answer it a slightly different way, which I think is what you're intending, is gross margin. So the gross margin for the company will improve as we scale, and we've said that we would get to a 70% gross margin. And as we scale the company, not only with filling the capacity with infusions, and then continuing to kind of scale the capacity in a gated fashion over time as needed, The gross margin will continue to improve to achieve that 70% that we discussed.
spk01: Great, thanks. Thank you. One moment for our next question, please. Our next question comes from the line of Nazan Yu with Wells Fargo. Your line is now open.
spk00: Hi, thanks for taking our questions. So just curious, when you say multiple patient enrolled for Lithogenia, does enrollment imply approval of insurance? And the time from enrollment to cell collection is the main gaining factor? those washout period and also the pretransfusions if not what might be the the gating factor you see also as a quick follow-up if you can talk about 19% growth to net to date is that different significantly between Vintaglo and SkySona, and any expectation for the growth to net for Lithuania to be very different from that 19% number? Thanks.
spk02: Thanks for the questions. Tom, why don't you address the enrollments, and then we'll have Chris address the growth to net question.
spk15: Yeah. Again, we're very excited about the multiple enrollments across multiple QTCs. I think it's an indicator of strong demand. And the road to yes, as we call it, is, you know, multifaceted. And the two big components there are obviously, as you mentioned, making sure that they're covered by their insurance. And that's, you know, taking approximately what it did was in Taglo, we're saying on average about two weeks. And then the second part of being ready is being medically ready. And this is a little bit different with Lipgenia than it was for Zantegla with Lipgenia. There's a washout period of their other medications. For example, hydroxyurea has to be discontinued for at least two months before they start therapy. And they're also supposed to go for transfusions, at least two transfusions one month apart. So once they're enrolled and once they're going through the process, If they're not already doing transfusions, I mean, if they're already doing transfusions and they're not on one of the medications that needs to be discontinued, they can go a lot faster. So I'm a little hesitant to give you an average right now because it's just going to depend on the patients. And we provided enrollments today. Enrollments is an early indicator of patient demand. The thing to watch going forward is the number of patient starts. That continues to be the value-creating moment for the company. And then, Chris, do you want to comment on that?
spk03: Sure. So the 19% that we discussed between the combined two products, just characteristically the difference between Zintegro and SkySona, Zintegro has an outcomes-based agreement, SkySona does not. And then what to expect on a go-forward basis as Lipgenia comes into the fray is a higher increased Medicaid population associated with Lipgenia. And that's where you saw that we provided guidance to 20% to 25% associated with the combined gross to net population of all three products.
spk00: Got it. Thanks.
spk01: Thank you. One moment for our next question, please. Our next question comes from the line of Luca Issi with RBC Capital Markets. Your line is now open.
spk11: Hi, this is Renan for Luca Issi. Thanks for taking my question. I just want to ask, during the last approval call after not receiving the PRV, you mentioned that you'll engage in discussion with the agency and attempt to achieve a potentially positive outcome, ultimately meaning receiving the PRV. Just wondering if there was any update on that.
spk02: Yeah, thanks for the question. We have submitted a request for reconsideration and discussions with the agency. We're not going to elaborate on ongoing discussions with the agency now, but we believe we have a clear and compelling rationale for why Lithgenia could be awarded a PRV. We'll provide an update when we conclude our discussions.
spk01: Perfect. Thanks for taking my question. Thank you. This will now conclude today's question and answer session. I'll now turn the call back over to Andrew Obenshain for closing remarks.
spk02: Great. Thank you, everyone, for joining our call this morning and for your questions. Our management team is available for a follow-up call today, and please reach out to Courtney if you would like to connect. Thank you very much.
spk01: This concludes today's conference call. Thank you for your participation. You may now disconnect. Everyone have a wonderful day.
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