Good morning, ladies and gentlemen. Thank you for standing by. Welcome to bluebird bio's Third Quarter 2024 Results Conference Call. At this time, all participants are in a listen-only mode. After the speakers' presentation, there will be a question-and-answer session. [Operator Instructions] Please note that today's conference is being recorded.

I'll now hand the conference over to your host, Courtney O'Leary, Director of Investor Relations. Please go ahead..

Good morning, everyone, and thank you for joining our third 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. With that, I will turn it over to bluebird bio's CEO, Andrew Obenshain..

Thank you, Courtney, and thank you everyone for joining our call this morning.

As a gene therapy pioneer and the only standalone commercial-stage gene therapy company in the market today, we have built what we believe to be an unparalleled foundation over the past two years since our first FDA approval with a robust qualified treatment center network, proven access and reimbursement and demonstrated demand for our therapies from both patients and from providers.

Today, we are seeing the results of those efforts. Since we reported our Q2 earnings in August, we have more than doubled patient starts from 27 to 57 across our portfolio.

We initiated steps to reduce our cash operating expenses by 20% in Q3 2025, and we've laid out a roadmap to financial stability and cash flow breakeven in the second half of next year should we accomplish our goal of securing additional cash resources.

On today's call, Tom Klima, Chief Commercial and Operating Officer, will provide updates on the commercial launches of LYFGENIA, ZYNTEGLO and SKYSONA. Then, bluebird's CFO, James Sterling, will provide an update and some comments on our recent proxy vote before opening the call for Q&A. I will now hand the call over to Tom..

Thanks, Andrew, and good morning, everyone. We continue to see strong growth in our commercial launches in the third quarter, with clear and established paths to access for our therapies and an industry-leading QTC network. As Andrew noted, we more than doubled patient starts since our Q2 call to 57 from 27 in mid-August.

Today, we announced that 74 patient starts have been completed or scheduled to date in 2024 across our portfolio. Of that 74, 57 patient starts have been completed with an additional 17 starts scheduled through the rest of the year. And we continue to see strong and sustained demand into the future, with 30 patient starts already scheduled in 2025.

This trajectory continues to support the potential path to cash flow breakeven in the second half of 2025 as those starts convert to deliveries and infusions. Remember, as we have said, it takes approximately two quarters between a patient start and drug product delivery.

We are particularly encouraged that because of our extensive QTC network, patients have broad access to our therapies. To date, patients have initiated or enrolled for treatment across more than 30 unique QTCs.

You will recall that bluebird has more than 70 total QTCs offering LYFGENIA and ZYNTEGLO in the U.S., highlighting the significant upside potential to come as the remaining centers in our network start their first patient.

We have the manufacturing capacity we need today to achieve our cash flow breakeven point and we have plans to double capacity for LYFGENIA in 2026 based on anticipated demand. This follows a similar launch dynamic as ZYNTEGLO where we scaled capacity commensurate with demand. 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. Focusing on LYFGENIA, more than half of all states have affirmed coverage through a preferred drug list or published coverage criteria.

Additionally, nearly 50% of Medicaid-insured individuals with sickle cell disease in the U.S. live in a state that has already completed prior authorization approval for the use of LYFGENIA for at least one patient.

Commercially, multiple outcomes-based agreements have been published and/or in place for LYFGENIA, representing more than 200 million U.S. lives. And timely access to LYFGENIA, ZYNTEGLO and SKYSONA has continued with zero ultimate denials to date for any of our therapies across both Medicaid and commercial.

Now, I'd like to turn the call over to James to speak to our financials..

Thank you, Tom, and good morning, everyone. I'm pleased this morning to confirm that we are now back on track with normal reporting timelines following the restatement of our financials earlier this year. In the third quarter, we reported $10.6 million in total revenue, down from $16.1 million in Q2.

As we guided on our last earnings call, we anticipated this dip in Q3 revenues due to variation in manufacturing timelines. We expect revenue will rebound nicely in the fourth quarter with net revenue of at least $25 million as more patients are infused.

Importantly, in the third quarter, we recognized our first LYFGENIA revenue, following the completion of the first LYFGENIA infusion well within our target timeline.

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.

On the cost side, SG&A expense was generally consistent with the prior-year period, while R&D expense was down $36.1 million year-over-year as previous R&D expenses were shifted to inventory and cost of products revenue. As of September 30, 2024, we had $118.7 million of cash on hand, which is inclusive of $48 million in restricted cash.

As we announced on our call in late September, we've implemented significant changes to optimize our cost structure. These changes are anticipated to result in a 20% reduction in cash operating expenses when fully realized in Q3 of 2025.

And we continue to anticipate quarterly cash flow breakeven in the second half of 2025, assuming we scale to approximately 40 drug product deliveries per quarter and obtain additional cash resources to extend our runway.

As Tom noted, we already have 30 patients scheduled for cell collection in 2025, and we feel confident that we remain on pace with previous guidance of approximately 40 deliveries per quarter in the second half of next year.

We are engaging collaboratively with Hercules as we work to secure adequate cash runway to obtain additional financing and reach cash flow breakeven in the second half of next year.

Based on our current forecasts, which assumes continued cost saving initiatives, successfully renegotiating key contracts and continued collaborative engagement from Hercules, we expect our existing cash and cash equivalents will enable us to fund our operations into the first quarter of 2025.

I wanted to provide a bit of color on our proxy vote last week. As you may have seen, we adjourned our meeting until December 4th to solicit additional votes to obtain approval of Proposal 4 related to the reverse stock split.

A vote in favor of Proposal 4 would enable us to regain compliance with NASDAQ's minimum bid price and increase the number of shares on a relative basis that we are authorized to issue, a necessary step to enable flexibility with our financing options.

We are very pleased with the votes we've received so far and want to encourage every stockholder to vote in favor of Proposal 4. If any of our shareholders have questions about this or would like to discuss it further, please email us at investor@bluebirdbio.com. With that, I will turn it back to Andrew..

Thanks, James. As we highlighted today, we are now in a period of accelerated growth and we have visibility to cash flow breakeven in the second half of next year as we work to obtain additional sources of capital and execute on our launches across our validated commercial gene therapy platform.

We look forward to continuing to update the investor community on our progress as we move towards changing first tens of hundreds of patients' lives. With that, we'd like to open it up for questions.

Operator?.

Certainly. [Operator Instructions] Our first question coming from the line of Jack Allen with Baird. Your line is now open..

Great. Thanks for taking the questions, and congrats to the team on the progress made over the quarter. I guess, my first one is on the revenues in the third quarter. It seems like there's an incremental stepdown as compared to revenues announced in the second quarter.

And I wanted to ask, what are you seeing as it relates to dynamics around administration of the cells after you deliver them to the treatment centers? Do you still see 100% pull-through in that regard, or are you seeing any kind of dropout from cell manufacturing to administration of cells? And then, I have a quick follow-up as well..

Yeah. Good morning, Jack. Thanks for the question. I'm going to hand it to Tom to respond..

Hey, Jack. Good morning. Yeah, we still see 100% pull-through once we deliver cells to a patient being treated. As you know, there's just variability I think in terms of patient scheduling. And then, once we deliver the final drug product to the QTC, it's in the QTC's hand.

So, it's a little bit out of our control once we deliver it back, but we -- once we deliver it back, we've seen 100% pull-through..

Got it. Great. And then, I guess, maybe I'll just dive in a little bit more.

Are you seeing 100% manufacturing success rate? And then, my follow-up was also, on the 2025 scheduling, any color as it relates to how far out you're scheduling slots into 2025 of those 30 slots?.

Yeah. Let me take the first one, and then I'll hand it -- the second one to Tom. So, no, we do see -- recollections are a natural part of the process, right? So, we do sometimes have to collect more than once for a patient. That is not a manufacturing failure. That is a normal part of the process.

And we've -- and once a patient starts the process, nearly a 100% of those patients go on to an eventual delivery. Now, Tom, go ahead..

Yeah. Just to reiterate, although there's variability in terms of scheduling when the patients are being scheduled, we're seeing patients being scheduled one to two months out in many cases, and we're really pleased that [we have now] (ph) 30 patients scheduled in 2025.

But as Andrew mentioned, there's just some variability and recollection is part of the process, but we do not see patients drop out because of that..

Got it. Thanks so much for the color, and congrats again on the progress..

Thanks, Jack..

Thank you. And our next question coming from the line of Gena Wang with Barclays. Your line is now open..

Thank you for taking my questions. Maybe two.

Regarding the 30 patients already scheduled in 2025, are these mainly in the first quarter? And then, for the LYFGENIA, cumulative -- the 17 patients that completed cell collection, what is the average cycle, cell collection cycle for these patients? And quickly regarding the cash gap, you do have one-quarter, second quarter '25, cash gap.

What could be the strategy in addition to Hercules you could cover this one-quarter cash gap?.

Good morning, Gena. Thanks for the question. So, I'll take the question about -- we'll take the question a little bit out of order. I'll answer the questions about the number of cycles for LYFGENIA. I'll hand it to Tom to talk about kind of how those patients are being scheduled out next year, and then go to James for the cash.

In terms of the number of cycles in the clinical trials for LYFGENIA, we said that 85% of patients either got done in about one or two cycles. We anticipate similar results for the commercial setting. The end is still relatively small, so I can't really comment on that yet.

But maybe Tom, if you could comment on whether those patients to be scheduled in first quarter?.

Yeah. Hey, Gena, good morning. We're really pleased with the sustained demand that we're seeing and we're really excited that 30 patients are already scheduled for next year. The vast majority of those are in Q1, although some are into Q2 as well.

And we're really not commenting on the breakdown between LYFGENIA and ZYNTEGLO, but just an exciting marker of the demand that continues to exist..

Right.

And then, James, if you could comment on the cash gap and plans to fill that?.

Yeah. Hi, Gena. And you asked about Hercules, in particular. So, Hercules has been a great partner for us through this and we're in regular contact with them.

It'd be premature for me to expand on specifics, nature of those conversations, but strategy to extend the runway include renegotiating key contracts and other cost initiatives, and our partnership with Hercules is key in helping move those forward..

Thank you. And our next question coming from the line of Jason Gerberry with Bank of America. Your line is now open..

Hey guys, thanks for taking my questions.

On the scheduled new start disclosure, just wondering what's your internal assumption on conversion rate of that actually being -- [becoming] (ph) a patient that gets their cells collected? And, I guess, the other question that I had was just with the new starts and the scheduled new starts for LYFGENIA specifically, are these concentrated in a few centers, or do you feel like the -- where the start activity is occurring is pretty broadly dispersed amongst centers so far?.

Go ahead, Tom..

Yeah. Hey, good morning, Jason. In terms of a patient being scheduled and converting to a start, obviously, we don't want to get too excited about small ends, but so far it's very high. It's virtually -- it's essentially, 100% are converting to a start. The only variable is just the time.

In some cases, they reschedule because of an event, but they 100% of the time have come back on the schedule, so that the conversion rate is high and we're pleased to see the excitement for gene therapy.

As far as -- and remind me the second part of the question?.

Is it broad based for LYFGENIA?.

Yeah. So, I think it's exciting because we have over 70 QTCs and we said approximately 30 QTCs have initiated for one of our therapies, which is pretty well spread out, but I think the most exciting part of that is we have about 40 QTCs that are looking to start their first patients. So, I think it's a nice indicator of growth ahead..

Okay. Thank you..

Thanks, Jason..

Thank you. And our next question coming from the line of Mani Foroohar with Leerink Partners. Your line is now open..

Hi, good morning. This is CJ on for Mani. Thanks for taking my question. Mine is just regarding, you previously shared that time from cell collection to completion of manufacturing and testing was taking like a month longer than expected. Have you figured how to accelerate this process? If so, when you expect to fully realize this? Thank you..

Thanks for the question. I'm not sure that we've actually reported that before. We are seeing pretty consistent timelines or some variability. We'd say 70, 90 days for ZYNTEGLO, 90 to 105 days for LYFGENIA. There's a bell curve around that. So, some are slower, some are faster.

But in general, we've been very pleased with the quality of our manufacturing, the timeliness. I'll point out that for LYFGENIA, that first patient that we actually delivered, we actually delivered that within that -- slightly less than that timeframe for LYFGENIA. So, in general, we're very pleased with our manufacturing..

Okay. Thank you.

And if I could quickly add another one, like, to what degree is your manufacturing capacity limiting your ability to ramp patient starts and to achieve that 40 drug delivery per quarter that you're in -- by second half next year?.

Yes. So, our current capacity is adequate to get to the projections that we've been talking about and able to support us getting to 40 drug product deliveries per quarter, and ultimately, the cash flow breakeven that we've been talking about. We are working on expanding our capacity for LYFGENIA.

We announced that we would double capacity in 2026 and that's based on our belief that the demand will continue to grow for LYFGENIA in sickle cell disease, but right now, we have adequate manufacturing capacity to hit our projections..

Okay, great. Thank you so much..

Thank you. And our next question coming from the line of Salveen Richter with Goldman Sachs. Your line is now open..

Hi. This is [Lydia] (ph) on for Salveen. Thanks so much for taking our question. I guess just going off the last one, can you speak to what drives your confidence in scaling to that 40 product deliveries per quarter by the second half of 2025? And how you expect this to evolve and ramp over the next few quarters? Thanks so much..

Go ahead, Tom..

Yeah, sure. Good morning, Lydia. It's really a couple of things. Number one, we continue to see strong and steady demand for ZYNTEGLO. We did expand our capacity this year for our manufacturing capacity at Lonza for ZYNTEGLO and SKYSONA, and we continue to see strong growth there and demand is not slowing down.

Number two, when you look at the ramp for LYFGENIA, we always said that it would ramp in the second half of the year and continue into 2025.

As we look at the more than the 30 patients that are already scheduled for next year and as you look at our QTC network of over 70 QTCs, 40 of which are looking to start their first patient, we see nothing but strong signals of demand in the market.

So, we're pleased with our growth, but more importantly there's a lot of unmet need in people living with sickle cell disease..

Thank you. And our next question coming from the line of Eric Schmidt with Cantor Fitzgerald. Your line is now open..

Good morning, and thanks for taking my question. One for James on the shareholder vote that went against you at the last stage.

Can you speak to how many shares are currently authorized to be issued and how many additional shares you're asking for?.

Go ahead, James..

So, about 194 million shares -- sorry, authorized to issue about 35 million shares available right now, to issue under current authority..

And the request for how many? Yeah..

So, the request came in the form of reverse stock split, which has the effect of increasing the number of authorized shares on a relative basis. I don't have the number in front of me....

And that must depend on -- so the Board will be authorized to do a stock split in the range of 15 to 20. It depends on how -- what number that Board chooses.

[indiscernible] 193 million or 197 million outstanding will shrink down, and therefore, that remaining 35 million will be issuable, and [all this be depend] (ph) what -- how that stock shrinks down..

Okay.

35 million will be fixed?.

Yeah..

And then, maybe one quick one for Tom.

In terms of dropouts along the process, can you speak to how many total patients you've lost from the time of scheduling collection all the way through?.

Go ahead, Tom..

Yeah. So, it's a good question, Eric. So, we're actually seeing a higher-than-anticipated pull-through rate. What we're seeing is if a patient comes off the schedule for any reason, they ultimately end up, for the most part, back on the schedule. So, we're not seeing a high dropout rate. And I'll give you some examples.

We did have 14 patients this year who came off the schedule for different reasons. Actually seven of them were hurricane related. They lived in an area that was impacted by hurricane. So, they came off the schedule, but so far all of those patients have come back on the schedule.

So, again, it's small ends, so it's early to give out those predictors, but we're really pleased with the pull-through rate that we're seeing..

And then, if I can comment on the -- from collection to actually delivery, we have -- that is once patient gets collected, that is essentially 100%. The exception would be that for occasionally for a sick patient versus SKYSONA, where we need to accelerate the process and not do a recollection, we might give that product for free for SKYSONA only.

And so that would be the only example of a patient not converting to revenue..

Helpful. Thank you..

Thanks, Eric..

Thank you. And our next question coming from the line of Luca Issi with RBC Capital. Your line is now open..

Oh, great. Thanks so much for taking our questions. This is Lisa on for Luca. Just one on the gross margin. The cost of goods continue to remain higher than the revenue coming in.

So, wondering if you can help us understand which component is driving the high cost of goods? Is this the lentivirus production or the release assays? And what levers can you pull to reduce the expense here?.

Go ahead, James..

So, it's the manufacturing for the most part, and they are high fixed costs associated with leases with our manufacturing contract providers and FTEs associated with the work. And so, you're seeing high costs and negative margins at the moment as a result of the relatively low volumes in these early quarters of commercialization.

We do expect that to change meaningfully as volumes increase now, including going positive in '25 on the gross profit. So, there is, of course, high cost associated with the vector manufacturing as well, like most [indiscernible] manufacturing and testing..

Got it. And then, just a follow-up. Is it -- if there are more sickle cell patients getting treated with LYFGENIA, could the cost of goods come down? If I recall, I believe LYFGENIA uses a slightly different manufacturing protocol versus ZYNTEGLO. So, any color here would be helpful. Thanks..

Yeah, that's correct. Our suspension vector with LYFGENIA is lower cost than ZYNTEGLO's. So, there is an advantage there, and we expect to be able to adopt those savings over to ZYNTEGLO as we switch that..

Thank you. And our next question coming from the line of Sami Corwin with William Blair. Your line is now open..

Good morning. Thank you for taking my question. So, last year, you saw a dip in revenue in Q4 due to some patients delaying their infusions until after the holidays.

So, I guess I was curious how much line of sight you have into infusion timing and what gives you confidence in hitting that $25 million in Q4? And then, a second question is that how you kind of expect access and revenue to change with CMS's gene and cell therapy access model being rolled out to the states next year? And if that might supersede any of the negotiations you already have in place? Thank you..

Go ahead, Tom..

Yeah, sure. Hey, good morning, Sami. Yeah, obviously, we're tracking very closely the number of infusions that have already happened in Q4 along with the infusions that are scheduled right now. That gives us the confidence in getting to the $25 million that we're projecting..

And then, Sami, the second part of your question was -- can you repeat that for us?.

Yeah.

How you expect access and revenue to change with CMS's gene cell therapy access model being rolled out to the states next year? And if that might supersede any negotiations you already have in place?.

Yeah. So, we are -- I mean, we are -- we said before, we are collaborating with CMS on the CMMI program. We would expect to hear sometime in early December about that. That is a national program where they would provide a framework that states could access.

We, of course, already have direct access with the states and have been having conversations with them as well. So, it is more an accelerator, I would call it, than anything one replacing the other..

Great. Thank you..

Thank you. And I'm showing no further questions at this time. I will now turn the call back over to Mr. Andrew Obenshain for any closing remarks..

So, thank you very much everyone for joining the call today, and we look forward to updating you in the first quarter next year..

Ladies and gentlemen, that does conclude our conference for today. Thank you for your participation, and you may now disconnect..