Thank you for standing by. My name is Ken and I will be your conference operator today. At this time, I would like to welcome everyone to the bluebird bio Second Quarter 2024 Results Call. [Operator Instructions] I would now like to turn the conference over to Courtney O'Leary from Investor Relations. You may begin..

Good morning, everyone, and thank you for joining our second quarter 2024 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 opening remarks.

Then Tom Klima, Chief Commercial and Operating Officer will provide updates on commercial launches of LYFGENIA, ZYNTEGLO and SKYSONA. And lastly bluebird's CFO, James Sterling will provide a financial update before opening the call up for Q&A. With that, I will turn it over to Andrew..

Thanks, Courtney, and thank you, everyone, for joining our call this morning. Over the past year, bluebird has built what we believe to be an unrivaled commercial gene therapy foundation with three ongoing launches.

We've seen that translate into a robust network of qualified treatment centers, proven access and reimbursement and demonstrated demand for our therapies, for both patients and providers.

Today, we'll provide you with additional insight into our commercial performance, grounded by a real world experience that helps to inform expectations and modeling for our launches. This morning, we announced that, we've renegotiated our agreement with Hercules Capital.

And on today's call, we will also provide you with details on our business execution and cash runway. I'll now hand it over to Tom to discuss the progress in our commercial launches in greater detail..

Thanks, Andrew, and good morning, everyone. Nearly 24-months following our first FDA approval, we have made incredible strides in building and strengthening our gene therapy commercial infrastructure.

We are beginning to see the results of this hard won experience with an unparalleled network of over 70 qualified treatment centers, far beyond our original goal of between 40 and 50 QTCs.

We have clear and established paths to access for all of our therapies and strong demand with rapid acceleration and patient starts on the horizon for both ZYNTEGLO and LYFGENIA. We are extremely encouraged by the excitement from patients and providers.

We continue to hear consistently through direct conversations at QTCs, at conferences and even on recent KLL calls that there is immense enthusiasm for gene therapy among the patient communities we aim to serve. And there is confidence and belief in our data, which recently surpassed more than 1,000 patient years of experience.

Today, we announced 27 patient starts or cell collections have been completed across our portfolio so far in 2024, including 19 for ZYNTEGLO, four for LYFGENIA and four for SKYSONA. Looked at another way, we have completed 23 combined patient starts in the U.S.

alone across beta-thalassemia and sickle cell disease in 2024 with a total of 43 patient starts since approval of both. And our launch momentum continues to build with more than 40 additional patients already scheduled for cell collection across our commercial portfolio to the end of the year and more being added every week.

Importantly, approximately half of these patients are individuals living with sickle cell disease. We are especially encouraged that, because of our extensive QTC network, patients have broad access to our therapies.

Today, patients have initiated or scheduled for treatment across more than 20 unique QTCs and we clearly have tremendous room for additional growth as the 70 plus centers in our network begin to initiate the treatment process.

For ZYNTEGLO, demand continues to be very strong and we anticipate continued growth in the second half of the year following the recent expansion at our Lonza facility, which doubles our manufacturing capacity for ZYNTEGLO and SKYSONA.

All of this reinforces the acceleration we projected in the second half of the year and gives us confidence in our ability to complete approximately 85 starts across our portfolio this year. Additionally, we've recently completed the manufacturing and release testing for the first commercial LYFGENIA patient and first infusion is being scheduled.

This is an incredibly exciting milestone. In this case, we completed this manufacturing and release testing on time and we now anticipate recognizing our first LYFGENIA revenue in the third or fourth quarter. Now moving to access and reimbursement. Our goal has always been timely equitable access to our therapies.

We are extremely encouraged by the speed with which both commercial and many government payers are approving pathways to patient access, particularly with LYFGENIA for sickle cell disease. In just seven months, more than half of sickle cell patients insured by Medicaid live in a state that has affirmed coverage to LYFGENIA.

This includes multiple states including California, Pennsylvania and Louisiana that have published coverage policies that are aligned to clinical trial criteria. 20% of patients live in a state that has already completed prior authorization approval for LYFGENIA for at least one patient.

This means that, Medicaid is moving patients through the process. I specifically want to highlight that, in June, LYFGENIA was placed in as preferred list in state of Florida, a critical step to unlocking access for one of the largest sickle cell Medicaid populations in the country.

We currently have four qualified treatment centers in the state of Florida and patients are eager for treatment. On the commercial payer side, we have multiple outcomes-based agreements with national commercial payers in place for LYFGENIA and published coverage policies in place for more than 200 million U.S. lives.

Additionally, timely access to ZYNTEGLO and SKYSONA has continued with zero ultimate denials for either therapy across both Medicaid and commercial payers. Moving to our QTC network. Today, bluebird has activated over 70 total QTCs for LYFGENIA and ZYNTEGLO in the U.S., approximately 3x the size of others in the field.

Additionally, six of the centers in our network are also activated to administer SKYSONA for patients with CALD. As I mentioned earlier, patients are scheduled for treatment with a bluebird gene therapy at more than 20 QTCs showcasing the benefit of our vast experience network.

We continue to see key differentiators that are driving preference for LYFGENIA.

Most recently in market research that we conducted in Q2 across our QTC network, we found that, the majority preferred LYFGENIA over our competitor across a wide range of key measures, including efficacy, mechanism of action, manufacturing turnaround time, cell collection and manufacturer flexibility and support.

To recap, this ZYNTEGLO launch is outperforming our initial expectations and we anticipate acceleration for remainder of the year with our newly expanded capacity. As projected, the LYFGENIA launch is accelerating with access expanding at a rapid clip and many patients scheduled to begin the treatment journey.

And the SKYSONA launch continues to progress as planned with 5 to 10 patient starts expected this year. As we look towards the back half of the year, we have more than 40 patient starts already scheduled and we are on track for approximately 85 patient starts across our portfolio. And now, I would like to turn the call over to James..

Thank you, Tom, and good morning, everyone. It's great to be at bluebird, and I look forward to getting to know many of you in the months ahead. It's an exciting time to join the company in the midst of three transformative launches.

I first want to acknowledge that, our restatement is ongoing with a tremendous amount of work by our finance and accounting team. We are moving as quickly as we can to complete the restatement, which as we previously stated is not expected to impact our cash or revenue. And we look forward to putting this chapter behind us.

In the meantime, we recognize the importance of providing you with an update on company performance and I'm pleased today to highlight our Q2 results. In the second quarter, we reported $16.1 million of total revenue, up from $6.9 million in the prior year period.

As previously guided, in 2024, we continue to 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. I also wanted to provide a few data points that you might find helpful for modeling.

Since our first FDA approval 24 months ago, we have collected cells from 53 patients across our three products. Cell collections are the value-creating moment. And to date, every patient who has had their cells collected has either completed an infusion or remains in the process.

Of the 53 patients who started cell collection, 31 have been infused, so we've already recognized revenue associated with those patients. We expect more than half of the remaining 22 patients will receive their infusions in the second half of this year. As a reminder, revenue is recognized at the time of infusion.

For additional context, the process for ZYNTEGLO was designed to take between 70 to 90 days from cell collection to delivery.

While we've successfully completed manufacturing and release testing within this timeframe, our real world commercial evidence and the experience to date is showing us that, it is taking about a month longer on average for this first set of patients. We are implementing process improvements and we expect this timeline will improve over time.

Once the hospital receives the drug, the patient then needs to be scheduled for infusion, which is driven by scheduling preferences and availability of both the hospital and the patient. As Tom noted, we've completed drug products manufacturing for the first commercial LYFGENIA run, which is still too early.

While still too early to predict the average real world timelines from manufacturing to infusion for LYFGENIA, for modeling purposes for both ZYNTEGLO and LYFGENIA, we recommend assuming two quarters between initial cell collection and revenue recognition.

Looking ahead, we want to manage expectations that revenue will fluctuate quarter-to-quarter, mainly due to varying manufacturing cycle times.

For example, we anticipate a drop in revenue in the third quarter, which is then projected to rebound in the fourth quarter, based on our latest review of manufacturing schedules and anticipated infusion timing.

We are clearly on the right track with accelerating interest in our therapies, and a clear path to translate patient starts into a growing consistent revenue stream over time. As of June 30, 2024, we had $193.4 million of cash on hand, which is inclusive of $49.2 million in restricted cash.

Based on current operating plans, our cash runway is expected to take us into Q2 of 2025. This runway does not account for the cash minimums required by our covenants with Hercules and also excludes the receipt of any future tranches from them. Factoring in the Hercules minimum cash, the runway takes us to Q1.

This recalibration of our cash runway was primarily driven by two factors. The first being the updated phasing of LYFGENIA starts that pushed out our revenue expectations and the associated cash collections. And second, the exclusion of additional borrowings from Hercules in our near-term runway estimates.

Finally, I'm pleased that we announced this morning that, we renegotiated our debt facility with Hercules to better align with current expectations and timing of LYFGENIA patient starts with our ability to borrow additional amounts.

Under the terms of the revised agreement, we are eligible to receive two funding tranches totaling $50 million, subject to the achievement of patient starts and product delivery milestones and contingent on the completion of additional financing.

A final potential tranche of an additional $50 million at the lender's discretion remains in the agreement. And with that, I will turn it back to Andrew..

Thank you, James. And to close us out for Q&A, I want to underscore the progress we've made in our business. We have three ongoing commercial launches where we've seen significant unrealized value at bluebird.

As you can see from the updates we shared today, we have demonstrated clear patient and provider demand and ability to successfully secure reimbursement for all three of our therapies and our ability to turn patient starts or cell collections into revenue. And we're now leveraging these strengths to our competitive advantage.

We look forward to continuing to update the investor community on our progress, as our business continues to scale. With that, we'd like to open it up for questions.

Operator?.

[Operator Instructions] And your first question comes from the line of Jack Allen of Baird..

Hi, thanks. This is Jack Allen with Baird dialing in. Thanks for taking the questions and congratulations on the progress. I just wanted to ask about these 40 patients that are scheduled to receive or initiate cell collection in the remainder of the year.

I guess, could you speak to the historical experience, as it relates to patients that have been scheduled? Have you seen anyone drop out of the process at this point in the, I guess, cell procurement. I'd love to hear any kind of historical context.

And then I had one follow-up as well on the revenue recognition, as it relates to ZYNTEGLO and SKYSONA..

Okay, great. Good morning, Jack.

Tom, why don't you describe that?.

Yes. Hi, good morning, Jack. Thanks for the question. We feel, I think, very confident with the acceleration in the second half of the year when you look at both LYFGENIA and ZYNTEGLO. We're extremely encouraged that more than 40 patients are scheduled for treatment going forward.

Historically, we haven't given guidance around how many have fallen out of the funnel. But in general, what we've seen is that, it just takes time for patients to get through the process and more often than not, it's more of a timing issue versus a getting treatment or not issue..

Yes. And Jack, I'd layer on that, that's from the time from the collection to actually infusion. In terms of the number of patients that have rescheduled their collection, that's a very small number, very, very small number. So we feel very confident in that 40. Go ahead on the revenue recognition question..

And then as it relates to recognizing revenues, I think James mentioned that there were 30 million patients that had cells procured or had cells infused. And when I go back and look at the revenues, that you estimate that, you've realized since the launch of these therapies is about $65 million, which -- when I calculate it seems like a 25% discount.

I just wanted to rebate, I wanted to understand, I guess, do you expect that 20% to 25% net price to take effect for both ZYNTEGLO and SKYSONA? Or is that more focused on SKYSONA and LYFGENIA?.

Go ahead, James..

Hi, Jack. It should be for both. I think you can apply that percentage estimated range to all three products..

Your next question comes from the line of Eric Joseph with JPMorgan..

Hi. Good morning. Thanks for taking the questions. Just a point on how to think about revenue recognition for the incremental three patients that have started -- incremental three LYFGENIA patients that have started collections this past cycle.

I guess, how should we think about revenue recognition there appreciating that there's been a little bit of an extension with the expected timeline for the first patient? And then just on this metric of 20% of Medicaid insured sickle cell patients living in a state, where prior authorization has been completed.

Can you just talk about the number of states comprised in that metric and where that might be expected to go with the additional LYFGENIA patient starts you anticipate in the back half of the year? Thank you..

Good morning, Eric. James, go ahead and talk about the three patients revenue recognition..

So, we would expect that to come in within six months, two quarters, is what we're thinking between cell collection and revenue recognition or infusion..

Hi, Eric. Good morning. This is Tom. I'll follow-up with the Medicare question. Obviously, it's a very exciting statistic to see that 20% of patients live in a state where at least one prior auth has already been completed. We expect that to continue to grow, as we work on especially targeted states, where people living with sickle cell disease live.

We haven't given a breakdown of that, but it continues to be our goal to provide timely and equitable access for patients..

Okay. Thanks for taking the questions..

Sorry, if I was going to jump in. I think you said something about the first lift patient being delayed. The first lift LYFGENIA patient was actually on time within our guidance of what the 70 to 105 days. Sorry, go ahead, next question..

Your next question comes from the line of Jason Gerberry with Bank of America..

Hi, good morning. This is Dina [Ramadin] on for Jason Gerberry. Congrats on the progress this quarter and thanks so much for taking our question. We just had a question on the kind of the initial feedback on the LYFGENIA launch.

What is driving your anticipation for an acceleration of patient starts in the second half of this year? Is there a bolus of kind of patients waiting to get initiated and that they're just kind of bottlenecks in the process in terms of getting them started? Appreciate any color. Thank you..

Good morning, Dina. Go ahead, Tom..

Hi, Dina. Good morning. So with LYFGENIA, we've always said that we would expect the acceleration to pick up in the second half of the year and continue into next year. And for LYFGENIA, there's just simply a longer path to patient starts.

This includes both going through the payer approval process, but also the steps to clinical readiness within the patient community. And so, it's really just a timing thing. Additionally, we are seeing patients schedule many months in advance.

As they did was integral, but maybe even a little bit more so with LYFGENIA, they're scheduling around life events. So it's not a slower start than expected and it's not a demand issue. Demand is actually strong. It's simply a timing issue..

Got it. Thank you. And just a quick follow-up, are you seeing any competitive dynamics in terms of how prescribers are making decisions on which sickle cell gene therapy to prescribe? Thank you..

Go ahead, Tom..

Yes. It's a good question. I think just I'll point you to a couple of different things. Number one, the size of our QTC network, we have over 70 qualified treatment centers, which is about 3x that of our closest competitor. When you look at number of starts across beta thalassemia and in sickle cell disease, we reported 23 starts in the U.S.

so far this year. So we continue to believe that, we maintain a strong leadership position. Beyond that, when you look at the market research that I talked about, we conducted market research across the QTC network, where we are seeing QTC's preferred LYFGENIA and preferred bluebird.

So we're seeing a number of indicators that lead us to believe that we continue to hold the lead in the market..

Your next question comes from Gena Wang with Barclays..

Maybe I think last quarter you mentioned that, you are expecting 85 to 105 patient starts for all three drugs. Now you have more definitive understanding on 85 patient starts. We still have a few more months.

Like what make you so confident that, you only will have 85 rather than potentially additional patients that could get scheduled? And then my second question is regarding two quarters you said will take from initial cell collection to infusion.

What is your assumption for number of cycles in average for cell collection?.

Okay. Go ahead, Tom..

Yes. Hi. Good morning, Gena. As we move through the year and we have the 27 patients who've already gone through cell collection, we have more than 30 patients that are scheduled to go through cell collection between now and the end of the year and that number continues to grow every week.

But with the timeline, what we're seeing is that, patients are scheduling in advance. So as we get into the second half of the year, you could expect some of those patients to start to schedule into 2025. And so, now that we have visibility, we believe that, demand is strong, but we feel that, we'll come in closer to approximately 85 starts..

And then, Gene, I'll take the recollections are a normal part of the process. We do indeed have experienced recollections, which we do anticipate will continue. We are, and to date roughly 95% of commercial patients have needed only one or two collections in the commercial setting.

So it's really too early to provide an assessment of recollections rate for LYFGENIA. But in clinical trials, it's about 85% patients that require one to two collections..

Your next question comes from the line of Mani Foroohar with Leerink Partners..

Hi, guys. This is Ryan on for Mani. Thanks for taking our question. We just have two. On the last call, you guys talked about how half of the QTC network is in the process of evaluating patients.

Can you kind of talk about if you're seeing a broadening across the network or more heavily concentrated uptake or progress within a select few networks or QTCs? And then I have a second one, but go ahead..

Go ahead, Tom..

Yes. Hi. Good morning, Ryan. We're pleased with not only the size of our qualified treatment center network, but also greater than 20 patients -- 20 QTCs have started the process with the patient. We believe that, a very broad subset of the QTCs are evaluating patients.

Keep in mind that, as they come on board and they do the work to become a qualified treatment center, they don't take that lightly and they really wouldn't commit to being a qualified treatment center, unless they had patients that they openly wanted to treat. So we're pleased with the growth that we're seeing.

I think there's a lot of upside with these 70 QTCs continue to go through the process and we're excited about the acceleration we're seeing..

That's helpful.

And then on the one month delay that you guys are seeing from cell collection to delivery for LYFGENIA, can you just provide any color on what some of the reasons would be for that?.

Yes. We haven't given a breakdown as part of gene therapy recollections, as Andrew talked about a second ago, as part of the process. But we are looking at different ways to improve our process and get back closer to the original guidance.

We're still incredibly encouraged that, the speed at which we can turn things around in the manufacturing process, experience that we're getting with our QTCs, obviously we are working very closely with our QTCs and becoming strong partners with them..

Your next question comes from the line of Jeff Hung of Morgan Stanley..

Thanks for taking my questions. For ZYNTEGLO, the rate of new patient starts came down slightly in May, since May.

Were there any one time that you saw in the quarter? Is that mainly due to timing? And then, is your confidence that it should increase over the remainder of the year? Is that mainly from the patients that are already scheduled? And then I have a follow-up..

Yes. So for your first question that, the diff you saw was just a timing issue. So you called that correctly. Our belief in the acceleration in the second half of the year is not only demand we're seeing, but also the patients that are already scheduled going forward.

And then, also when you look at ZYNTEGLO specifically, we did double our manufacturing capacity at Lonza. And so that will allow more patients that are already waiting to be treated in the second half of the year and going forward. So we have confidence obviously with kind of what we're seeing going forward..

Okay, great. And then you reported restricted cash of around $49 million but last quarter it was around $52 million. Just wondering if you could just talk about the slight difference there? Thanks so much..

Yes. We had a small release of restricted cash this quarter related to some research right, I believe. Go ahead, James..

Yes. And a change in one of the banking relationships, a lot that really sort of permitted that restricted cash..

Your next question comes from the line of Yanan Zhu with Wells Fargo..

Hi. Thanks for taking our question. This is Kwan on for Yanan. I have a question regarding the QTC. I wonder if there are any centers that were clinical sites, but decided not to become a QTC. Thank you..

Yes. Hi, good morning. It's a good question. So all of our clinical trial sites have gone on to be commercial QTCs. Keep in mind that, in some cases they will continue to do clinical trials. They go on to become commercial QTCs, but they continue to do clinical trials with other gene therapies as well. So, they have all converted to commercial QTCs..

Got it. Thank you for that. And one quick question on insurance. So you mentioned that, there was no ultimate denials on ZYNTEGLO and SKYSONA. How about LYFGENIA, any pushback or denial? Thank you..

It's probably too early to tell. What we're seeing now though is, we're encouraged by both commercial insurers as well as government insurers approving therapy for the patients that are going through the process. Again, it's too early to say that, there haven't been any events.

I mean, we will kind of continue to monitor that and provide updates as we see more patients..

Your next question comes from the line of Salveen Richter with Goldman Sachs..

Hi. This is [Shunath Rao] on for Salveen. Thank you for taking our question.

Regarding your debt financing agreement with Hercules, could you provide any additional color on the renegotiated terms? And what would trigger the second two tranches in this agreement?.

Go ahead James..

Yes. Hercules has been a great partner through this and allowing adjustments to give us a chance to access those next two tranches given the update to the LYFGENIA phasing. So the second tranche now becomes available, first, if we secure at least $75 million in gross proceeds from additional financing by December 20th of this year.

And at least 50 LYFGENIA starts by March 31st or 70 LYFGENIA starts by June 30th. That would release tranche two, $25 million. And then tranche three now becomes available.

If we receive at least $100 million in gross proceeds from additional financing by December 20th or at least $125 million by June 30th of next year, and we complete at least 70 drug product deliveries within a six month period no later than December 31th next year..

Your next question comes from Luca Issi with RBC Capital Markets..

Great. Thanks so much for taking my question and congrats on the progress. I have two quick ones. Maybe circling back on the prior questions on sickle cell disease. How should we think about the relative market share between you and Vertex now? Most of you guys have been in the market obviously for eight months.

I think you have four patients start so far versus they actually have 20 cell collected. We don't have the breakdown between beta-thalassemia sickle cell disease for them, but I'm assuming that most of them are sickle cell disease. Maybe if you go by that metric, you suggest that they could be ahead here, but again, we'd love to hear your pushback.

And then maybe a quick one on the P&L.

We obviously have not seen COGS for quite some time, but we'd love to hear kind of at high level, how should we think about where gross margins are today and where you think they can go as the product scales?.

Go ahead, Tom..

Hi, Luca. Good morning. It's Tom. I actually see it much differently than that. Keep in mind that, what it was 23 starts in beta-thalassemia and sickle cell disease and 43 starts since they were both approved, just in the U.S. alone. I have not seen a breakdown from what they reported, but it was 20 global starts from what I remember.

So if you look at both the starts that we're reporting and you look at the size of our QTC network, we continue to believe that, we are securing the lead position in the market..

Go ahead, James, on the next question..

Yes. Since we haven't completed the restatement and so we haven't released the 10-K or the 10-Q yet. I can't comment on specific gross margin right now or COGS. But as we guided previously, we continue to anticipate a gross margin of at least 70% within the next five years.

And I'm happy to walk through the updates to COGS once the restatement is done and we complete the filings..

Your next question comes from the line of Sami Corwin with William Blair..

Hi there. Thanks for taking my questions. I was wondering, if you could discuss the negative opinion by HHS on the fertility treatment and if that's had an impact on LYFGENIA.

And then also, I would love your high level thoughts on obtaining additional sources of funding since that seems to be a largely tied to obtaining those next loan tranches?.

Good morning, Sami. Go ahead, Tom..

Yes. Good morning, Sami. Obviously, fertility preservation is a consideration for patients who would be considering gene therapy. As a reminder, bluebird offers fertility preservation for eligible, commercially-insured patients through our patient support, which is called my bluebird support.

And as you're referencing, on July 22, the OIG released an unfavorable opinion on our request to provide fertility preservation services for patients that are insured through Medicaid and other federal healthcare programs. We continue to focus on equitable access for patients.

We are as bluebird engaged with a diverse group of stakeholders, including patients and advocates to encourage change.

In the meantime, I would say that, both bluebird and our qualified treatment centers are highly experienced with helping patients and their families navigate the coverage process and are quite experienced in navigating fertility preservation..

Go ahead, James..

So we have a wide range of options available to us on the financing. It include other sources of debt, equity, royalty, other alternatives. So it's too early to comment on any specifics, as we contemplate structure to satisfy the Hercules ask which we consider quite appropriate..

I will now turn the conference back over to Andrew Obenshain for closing remarks..

Thank you. Great. Thanks everyone for joining the call this morning and for your questions. Our team is available for follow-up calls today. Please reach out to Courtney, if you would like to connect. Thank you..

Ladies and gentlemen, that concludes today's call. Thank you all for joining. You may now disconnect..