Q2 2023 bluebird bio Inc Earnings Call
Good day, and thank you for standing by welcome to the Bluebird Bio's second quarter results and commercial launch progress call. At this time all participants are in listen only mode. After the speaker's presentation, there will be a question and answer session.
A question during the session you will need to press star one on your telephone.
Then here an automated message advising that your hand is raced please be advised that today's conference is being recorded I would now.
Like to hand, the conference over to MS. Courtney O'leary Investor Relations. Please go ahead.
Good morning, everyone and thank you for joining today's call I'm corneal theory Investor relations at Bluebird bio.
Before I 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.
Statements about the company's plans expectations or intentions regarding our business.
Regulatory progress and commercialization plan 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 as contained in our most recent Form 10-Q, and our other filings with the SEC, which are available on the Investor Relations section of our website Www Dot Bluebird bio dotcom.
With me on the call is Andrew <unk>, our CEO who's going to provide some opening remarks and deliberate strategic position and the debt and then review our Q2 results.
Then Tom cleanup, Chief commercial and operating officer will dive deeper into the positive momentum in our commercial lunches and highlight the love a solid market opportunity ahead of us.
They will then be joined by Chris Koch, Our Chief Financial Officer, and Rich Colvin, Chief Medical Officer for Q&A.
With that I will turn the call over to Andrew.
Thanks, Courtney and good morning, everyone.
I want to ground our call. This morning, and why we're here a few weeks ago, we heard from a young adult with sickle cell disease, who was treated in our H E. B 206 study this was a real privilege.
Reflecting on light switch gene therapy. She shared it's amazing my mind is open to the world of possibilities. So much in front of me I wanted to accomplish there's so much more for me that one of my previous medical teams renovation for me themselves and I'm excited about it.
And this is why we're here and what we mean, when we talk about bringing patients and families more bluebird days.
Over the past decade, Bluebird has established herself or gene therapy leader, proving our clinical regulatory and commercial capabilities.
Clinically we have the longest and most robust gene therapy program in the field with over 180 patients treated across eight clinical trials with up to nine years of follow up in over a decade of gene therapy research.
We have an established regulatory track record with two F. D. A gene therapies on the market in the third BLA currently under priority review.
And now we are making strides commercially.
Activate launching achieving reimbursement treating patients with true therapies today.
We occupy a unique strategic position in the gene and cell therapy industry.
There are only a small handful of companies all large cap. They currently have the commercial it's LNG and Cape G and capabilities that we do.
On the opposite side of the spectrum there are more than 250 companies studying gene therapies pre clinically or clinically who lack our gene therapy infrastructure with new companies being formed every month.
We believe that with our unique capabilities Bluebird isn't an attractive strategic position as one of the only standalone commercial gene and cell therapy companies.
Our extensive platform of gene therapy expertise includes manufacturing experience, particularly with bio production cell processing more than a decade of R&D experience and established commercial infrastructure.
Our strategic vision for futures clear to evolve into an industry, leading gene therapy company with opportunity for expansion and growth for years to come.
We have put in place the pieces that we need to be a successful biotechnology company.
On our path to profitability in the near term, we anticipate additional growth at scale in the next five years.
We can certainly estimate they combined multibillion dollar revenue opportunity in the U S person take low load itself if approved.
We have a focused commercial footprint with the ability to cover U S transplant centers with a relatively modest but effective field presence at.
Our scale, we estimate a gross margin of at least 70% as we invest in manufacturing quality capacity and.
In my comments pertain only to the U S. There's also significant upside potential with wholly owned global rights for all three therapies, particularly prison tableau can Louis L.
Yeah.
I'll move now to how our business is performing today and the updates from the second quarter I will summarize the highlights.
Highlights and additional details can be found in our Q2 release this morning and in our 10-Q.
First it's incredibly exciting to be here almost a year after the FDA approvals and take on Sky sooner.
Initial traction we're seeing in these launches as a testament to the robust commercial viability of our platform.
First in tableau and Sky. So now we have 16 patient starts to date.
All of these collections resulted in infusions this translates to a potential $45 million gross revenue over time.
We see that continuing to accelerate quarter over quarter.
Our therapies are being covered by insurance and there have been no ultimate denials from commercial or government payers for either therapy.
And we've activated 15 to Q T. CS qualified treatment centers, which is sufficient for the sky solar Integra launches, but we anticipate an aggressive ramp up to 450 <unk> by year end to support the potential launch of <unk> next year.
Looking ahead, the lowest held for sickle cell disease.
We have the opportunity to make a tremendous difference to change the lives of the more than 20000 individuals living with severe sickle cell disease in the U S that may be appropriate for gene therapy.
In June our BLA was accepted and granted priority review by the FDA for the treatment of patients 12, and over the history of basal occlusive events or Bo.
At this time the FDA has not requested an advisory committee meeting.
Keep in mind. This is a third <unk> gene therapy. They review from Bluebird, which gives us great confidence and we look forward to a decision from the FDA by the end of this year with a <unk> date of December 20th and anticipate a commercial launch in early 2024.
Additionally, the value of our therapy and need for rapid equitable access for patients is already being recognized.
Just a few weeks ago ISO reported <unk> cost effective up to a price of $2 two $6 million.
In fact, I star rated lagerfeld clinical effectiveness more favorably than direct competitors compared to current standard of care.
And financially we are complementing our lean and efficient business model. We are focused on one mission and have the expertise and the agility to deliver for patients and our business.
For the quarter, we reported $6 $8 million combined product revenue for some turbulent skies Donna.
As of June 30th we had $291 million in cash cash equivalents marketable securities and restricted cash.
We made on track with our full year 2023 cash burn guidance in the range of $270 million to $300 million.
And continue to prudently deploy capital and make investments to maximize stakeholder value as we launch into tableau and sky solar and prepare for the launch of liver cell.
We continue to estimate there'll be the cash runway into the fourth quarter of 2024, including the restricted cash.
As previously disclosed this estimate of cash by the way.
Proximately $45 million of restricted cash, which is currently unavailable for use without the release of our restricted cash our runway through the second quarter of 2024.
Now I would like to turn the call over to Tom to dive deeper into our commercial launch progress and momentum in greater detail.
Thanks, Andrew and good morning, everyone almost a year ago, we discussed our first commercial launch plans. Following <unk> approval. It is exciting to be here. This morning to dive deeper into the progress. We have made since then to bring our onetime treatments to patients.
Our forging the commercial model for ex vivo gene therapy in the U S and this quarter, we continued to make great progress across the key pillars of our launch including patient demand.
Qualified treatment center, or <unk>, onboarding and access and reimbursement.
We want to give some additional insight into the launch dynamic towards intaglio and how the foundation. We have built has laid the groundwork for commercial growth that is projected to accelerate quarter over quarter.
In August 2022, we said, we would launch <unk> with five <unk>, which we accomplished by the end of September today every one of those <unk> have started a patient and in fact most of these centers are treating their second third or even their fourth patient.
Our wave <unk> or next five were activated by the end of last year and.
And we are excited to report that 40% of our wave <unk> have started the patient with the rest on track to start a patient in the coming months. We are currently activating our waves <unk> b centers have more recently been on boarded and are in the process of patient identification enrollment and scheduling.
We are building trust with our <unk> network and they are gaining incredibly valuable experience.
They are understanding our process better they are identifying interested patients and have become more familiar with the reimbursement process.
This all led to an acceleration of our <unk> activation.
As expected to bring the time to activation down for months before as integral two weeks for low to sell and we remain on track to scale. The Bluebird GTC network to between 40 and 50 by the end of this year and will likely continue to expand into 2024 to ultimately reach more patients.
Does the integra launches progressing exactly how we would expect an ex vivo gene therapy, commercial launch wood, which trajectory gradual and linear and momentum continuing to build over time.
Now if we flash forward to early next year as these centers will be able to leverage their experience from administering <unk> and tableau.
And are anticipated to be ready to treat individuals with sickle cell disease. Soon after the loan sell potential approval further capitalizing on our 18 month head start on the competition.
Delivering a consistent and reliable manufacturing process is a key part of delivering gene therapy and is essential for physicians providers and for patients and their families and as a reminder, a key element of ex vivo gene therapy is that the transplant center is an integral part of the supply chain.
We have built a strong reputation with these centers through our commitment to transparency and partnership throughout the entire process from TTC Onboarding, all the way through patient infusion.
Four is integral wholesale collection. It takes on average 70% to 90 days to manufacture test release, and then deliver the therapy back to the hospital.
The infusion of Integra is driven primarily by patients physicians and their schedules.
And at this phase of our launch we continue to focus on patient starts as the lead indicator with the expectation as Andrew noted earlier that starts translate to revenue as the treatment treatment process and infusions are completed and currently bluebird recognized as revenue upon infusion.
This process will largely be the same for low to sell and for other ex vivo gene therapies in this space and is yet. Another example of where blue bird is leading the way.
Transitioning to low yourself for sickle cell disease before I talk about our launch preparations and the commercial opportunity I want to remind everyone of the transformative impact of this therapy as seen in clinical trials.
Our clinical data reflects the most robust data available with the longest follow up across any gene therapy program for sickle cell disease.
More specifically our BLA package includes efficacy data from 36 patients with a median of 32 months of follow up and safety data from 50 patients treated across the entire load with cell program.
From our August 22, 2022 data cut we showed 97% complete resolution of severe basal occlusive events through 24 months.
The majority of adverse events were attributed to underlying sickle cell disease or conditioning with <unk> software.
We continue to work with the FDA through their review of our package and we look forward to our <unk> on December 20 of this year.
Preparations are well underway for the commercial launch in early 'twenty four if approved.
The unmet need for these patients is great sickle cell is a devastating disease that has historically been ignored and its severity under appreciated.
We have performed more than seven years of market research with sickle cell treaters transplant, <unk> and with patients, which has consistently demonstrated that mobile sells a meaningful treatment option for patients, giving us great confidence in the significant opportunity ahead.
Starting on the left hand side of the slide we have consistently seen strong patient excitement for gene therapy, and we have found that more than 70% of patients would consider gene therapy, if recommended by their doctor.
Demand an eagerness for gene therapy is clearly there and continues to grow.
Moving to the middle one of the greatest misperceptions is that patients and providers our parcel to one modality over another the research simply does not show. It in fact market research shows that more than 70% of patients will make a treatment decision based on efficacy and based on long term.
Some follow up but not based on modality.
This is a key differentiating point for low itself, which has the longest and most robust clinical and safety profile of any gene therapy for sickle cell disease.
And finally to our market research over the years, we have seen lower cell capture between a 50% to 65% market share against a direct competitor.
This was established after responded to reviewed both product profiles and then were asked which one they would prefer both who are available on the market.
All of this market research is coupled with a decade long partnership between Bluebird and paste sickle cell patient community.
When I talked earlier about our key pillars of our integra launch, including patient demand, our <unk> network and access and reimbursement and I'm pleased that we're already making great progress on these pillars and finalizing our launch readiness for low to sell.
We are excited about the clear patient and physician demand as evidenced by the research I just discussed and we will continue to partner with the patient community to broaden education around gene therapy.
Our <unk> network is growing and continues to become more efficient gaining experience and momentum.
And finally access to gene therapy is critical for patients Bluebird continues to lead the way on value demonstration and in working with government and private payers on our innovative payment models and we're excited about continued positive feedback from target payers on clinical value for high unmet medical need.
And on our outcomes based approach.
We are confident in our therapies, we are confident in our strategy and we are confident in our team and we're making a difference in the lives of patients today and laying the foundation for many more to come.
Now back to Andrew.
Thanks, Tom before closing the call I want to spend a few minutes on sky sooner.
As I mentioned earlier, we've completed five patient starts activated <unk> and received no ultimate denials from governmental and commercial payers to date for Sky solar.
It's critical to remember that cerebral adrenoleukodystrophy as a very different disease that are other disease areas.
Financial impact of Sky solar is not material to bluebird the impact it can have on the lives of patients and families affected by <unk> is.
So it was approved based on a 24 month improvement in major functional disability free survival. These.
These measures include the ability to communicate the ability to see requirement for tube feeding total cognex wheelchair dependence and the loss of voluntary movement.
You can imagine the profound impact the loss of these functions has on patients and their families.
The label for Sky Solar includes a box warning for Hematological malignancy as three boys treated in our clinical trials developed Myelodysplastic syndrome, Mds, which is believed to be caused by searchable entity vector in July reported two additional cases of MBS in patients who are treated in our clinical studies, bringing the total number to five cases.
And given the known risk we do anticipate additional cases may occur.
Nevertheless, we continue to believe that Sky Stoner remains an important therapy for patients who have no other treatment options and.
And given the difference in the vectors, we do not believe this any read through into our broader <unk> platform, including a tableau or level itself.
The rest of 2023 at the beginning of 2024 are shaping up to be an exciting time for bluebird presence.
<unk>, we continue to aim to scale, a 45 to 50 <unk> by the end of 2023 and prescribe <unk>. We continue to anticipate 5% to 10 patient starts this year.
We also look forward to the <unk> date of December 22023, and as Tom touched on we are actively preparing for the commercial launch expected in early 2024 if approved.
Or move into Q&A I want to summarize some key points.
Bluebird occupies a unique strategic position as a leading gene therapy company, that's going to enable growth and expansion for years to come.
Second we have a strong competitive advantage preparing to maximize our 18 month head start against our next competitor with patient starts accelerating <unk> onboard and becoming more efficient and projected lower sell market share of 50% or more.
And finally with commercial momentum, including 16 patient starts to date and the <unk> BLA under priority review, we are in a path to profitability in the near term anticipate additional growth in scale over the next five years.
Thank you for your continued support for Bluebird for the patients and families. We aim to serve.
With that I'd like to open it up for questions and invite Chris to rich to join us operator.
Thank you as a reminder to ask a question you will need to press star one on your telephone please standby, while we compile the Q&A roster.
Your first question comes from the line of Dane Leon from Rgs. Your line is now open.
Hi, congratulations on all the progress.
And with it.
Early launches of Sky's Donna Peglow.
Could you maybe just expand a little bit in terms of what youre seeing at the top of the funnel with referrals into the current <unk>.
<unk> base that you've built up for us in tableau and just what you are starting to find out now and more recent trends around that referral process, then translating into patients that start to undergo the process for cell collection and ultimately treatment. Thank you.
Thanks, Dan Good morning, I'm going to ask Tom to answer that question, yes.
Yes, Hi, Dan good morning.
We've talked about historically all of our key Tcs are a little bit different in some cases.
He has had their own pent up demand of patients who are excited about <unk> and so they're actually working through their internal patient load before they are starting to think about accepting referrals from outside the <unk>. Other key tcs are both actively treating patients within their ttc's, but also taking referrals from outside the <unk>.
And as you can imagine.
It's exciting for the <unk> to be offering a first of its kind of gene therapy, but also exciting for patients.
The process does take some time it takes time to get through patient identification and then the medical work up and then to get enrolled into the program, but we're excited to see both gtc's burning through their initial patient loads, but also starting to think about referrals from outside their TTC.
Yeah.
Great. Thank you.
Thank you. Your next question is from the line of Dana Rama <unk> from Bank of America. Your line is open.
Oh, Hey, guys.
This is Jason.
Can you talk about maybe the learning so far in the launch what what's been the biggest barriers as you go from collection to infusion like are you, losing any patient and if so why.
And then have you guys discussed with payers the value based model.
If you use with <unk> and I will sell as it pertains to the sickle cell in market and just kind of curious given the larger end market and cycle is that is that the preferred approach for payers. Thanks.
Good morning, Jason then Tom again.
Question just to start with the funnel Jason Good morning, we arent really seeing a lot of patients fall out of the funnel so to speak what we're seeing is that a.
A lot of this will depend on both the physician schedule on the patient's schedule because beta thalassemia is not necessarily an acute disease that that allows for some flexibility with scheduling and so in most cases, when we see patients entered the funnel who are interested they don't necessarily fall outages. It just takes a little time for them to get through the treatment process.
In some cases that happens relatively quickly in other cases patients will schedule around a major event like a wedding or a.
A dance or something like that that they want to get through before they start treatment. So again, it's a small and so we're not ready to make huge predictions on fallout percentages, but right now we're encouraged because we're not seeing a lot of patients fall out they just take time to get through the treatment process.
Moving on to the value based approach for sickle cell.
We're extremely thrilled with how the process went for beta thalassemia and was intaglio and we've received a lot of positive feedback from payers, both government payers and private payers on our approach. However.
Approach towards integral will not be the same approach that we're taking for sickle cell disease.
Done a lot of work with the same payers to understand what might work in a population that has a much larger population with different.
Clinical expressions and different outcomes and we're excited to say that we finalize the details around what our outcomes based agreement is going to look like for sickle cell.
As you might understand are not going to share those details at this time, but we'll show more details as we get closer to approval, but we believe that an outcomes based agreement that fits the purpose for payers and for patients with sickle cell disease is absolutely necessary.
Got it can I just squeeze in a follow up.
Sure.
The comment on gross margin, 70% or higher.
I wouldn't assume that the pricing that you guys have that maybe it might've been a little closer to 80%.
Can you talk about the royalty stack on <unk> and Lasalle I know, there's like a number of different entities low single digits.
I'm wondering if you could sort of give us a sense of how much the royalty stacks are on those two products.
And cumulative.
Let me go to <unk> Tom Hill.
I think Chris Yes, just to remind you that with its integrity.
Sky as soon as is pretty brief with Sky. So no we are not discounting or offering any outcomes based agreement there.
There is a Medicaid discount of approximately 17% for pediatrics and Sky Center within tag low.
We did offer an outcomes based agreement that was tied to transfusion independence. It was a very simple outcomes based agreement, where if a patient did not achieve transfusion independents or maintained transfusion transfusion independence, we would rebate up to 80% of the costs again. It was just a one time upfront payment and then a rebate.
Outcome was not achieved or maintained.
Keep in mind that our clinical trial is approximately 90% of patients achieved transfusion independence and of those who achieved at 100% maintained it so I'm, giving you a little bit of background. There because we don't expect there to be a huge impact on gross to net for his integrity enforce Skype zona and before I turn it over to Chris It's too early to comment on.
Sickle cell disease with low yourself Chris.
Chris.
As it relates to your question on royalties and milestones I don't think that thats going to be a material component.
On a go forward basis, there will be commensurate milestone that you saw Q3.
22, we have milestones associated with approval the royalty drag on these products, we're not going to be significant as it relates to your modeling.
Your next question is from the line of Eric Joseph from Jpmorgan. Your line is open.
Good morning, Thanks for taking the questions.
I guess just clarifying.
I guess the revenue breakdown for this quarter can you just.
Kind of articulate how many patients are reflected in the $6 8 million and what if any latency there is between perhaps.
Patients being treated and revenue recognition this quarter and then.
I guess just.
Just tracking sort of the cadence of new patients coming into Q. It seems like you have added four new patients. This past month since your prior corporate update I'm, just wondering whether we should expect this patient.
Cadence of new patient adds to continue or accelerate perhaps going into the second half. Thank you.
Yes.
Good morning, let me take the second part of that question first just quickly then I'll ask Chris to comment.
Comment on the revenue.
So we do expect to see a linear growth over time, we're not going to we're not forecasting out right now we're not operating but we do expect a linear growth as we had predicted for the launch and that's what we're seeing right now Chris.
We're encouraged by the revenue that's recognized however, I want to remind folks that the kpis for the company is really patient starts and that's where we want to continue to focus your attention.
Given where we are in and the.
Progress of our launches.
So we see continued growth as it relates to our patient starts with both <unk> and <unk>, we're not going to report.
Exact byproduct infusion numbers.
Just as a reminder, certainly as Andrew and Thomas touched on patient starts.
<unk> will ultimately then translate into revenue at some point in time and Thats why the Kpis of the company that we're providing to the investor community is the patient starts.
Okay got it a quick follow up if I could just give us incremental color we've had on the.
The early fill review cycle I'm wondering if you.
Similarly, we have been.
Notified about a potential outcome for <unk>, So I guess, what's your expectation of the dotcom accurate.
That review cycle. Thank you.
Okay.
Yes. So at this time the FDA has not requested an advisory committee meeting.
Okay.
I appreciate it.
Taking the questions guys. Thanks very much.
Thanks, Eric.
Your next question is from Gena Wang from Barclays. Please ask your question.
Thank you maybe I'll just follow up also regarding to add a quick question. When was the last time you discussed with the FDA.
And also when will be the next time you will be.
Talking to FDA.
And then regarding ice two wanted to understand the exact timing for the patients with C. Just thoughts forms.
Juicy.
A couple of product complete.
How long does that take.
You mentioned 70 to 90 days past from test to completion of the manufacturing, but if we come at it from the start form how long does that roughly estimate that.
And then the third question quickly is the Skyscanner.
Mentioned that there is a <unk> patient is mds events.
With the patient community well understood. This is the sky Sonet specific.
Risk.
There is no labor or anything.
Comment on the potential risk with.
Tableau or hopefully in the future.
In the Labor Force Oversell ESMO.
Let me break those down.
The first one.
We're not going to comment on back and forth of the FDA. We just we just don't comment on when what our cadence indications there. It's all I can say at this time. The FDA has not requested an advisory committee meeting.
And then I'll hand, it to Tom to talk about the time from the patients start forms to that drug product manufacturer than.
And then I'll comment on Scott go ahead, yes, hi, good morning, Gina what we're seeing is that the most predictable part is clearly the time from cell collection to the time of.
Drug delivery back to the hospital, which is the 70 to 90 days on average what we're seeing on the front end and on the backend as there is some variability and some flexibility in some cases, we're seeing patients who are eager to be treated that move through that process very quickly.
As part of that process. The qualified treatment center has to get a prior authorization from the insurance company to treat the patient on average that prior authorization or PAA is taking about two weeks.
So the other piece is really depend on the patient's schedule and the schedule at the qualified treatment Center and then the other part that's a little bit variable based on patient and physician schedules is after we deliver the drug product, it's up to the patient and the physician when they actually infuse is integral in sky.
So there's a little bit of variability there. However, what we're seeing is that they're motivated to infuse as soon as they can.
For a lot of reasons number one they are usually scheduled to patients in advance and pursue they usually have.
Our limited time, where they get a an approval from the insurance company and then just I think everyone is motivated to get through the process. So again, the most predictable part is cell collection to drug delivery. The other parts, we will get more data as we mature through our launch but right now it's a little bit variable.
Thanks, Tom and then going back to Scott Scott.
Okay Soni use a different vector that our other therapies as a result, we don't believe there's any redrow to our broader <unk> platform, including a tableau or lower sell through reminded of a no cases of a social oncogenesis and other programs.
The two therapies are manufactured using a totally different multi viral vectors. The different vectors are designed specifically for expression in different cell types that utilize different promoters as a result, they are totally distinct safety profiles. This is well understood by clinicians and the FDA as well.
Thank you and your next question is from Jack Allen from Baird. Please ask your question.
Yes.
Okay.
Great. Thank you so much for taking the questions just two quick ones from our end.
Was wondering if you could step back and think about the AD comm dynamics, whether it's the FDA and provide some historical context at this point in the cycle to the FDA notify you of this integral around calmer.
Do you have any historical context surrounding that interaction and then briefly more of a logistical question on the restricted cash can you just make us aware of what the restrictions are on a marketing that cash and extending that runway.
Thanks, so much.
Good morning, Jack I'll take the first part of the question Manhattan Secondly, Chris on the outcome, we had hertz.
At this time point from <unk> and tableau about the Advisory Committee, we have not at this time. The FDA has not requested advisory committee for low the cell they always could.
So that.
<unk>.
There is no time bound requirement for the FDA for notification.
Chris do you want to comment sure Kash.
The restricted cash predominantly relates to letters of credit as it relates to some of our real estate that we occupy the predominance of the restricted cash relates to an arrangement for a lease for a 50 binney property in Massachusetts.
We leased that from Sanofi, that's approximately $41 million, we then sublease that to meta.
And in lieu of meta going direct with Sanofi, which would release the restricted cash we continue to collect sublease income on that sublease that sublease income is recorded in other income in our financial statements of course, the operating leases recorded above the line in our operations.
The release of the restricted cash is really dependent upon going direct and having medical direct with Sanofi.
Or getting clearance from Sanofi to release that restricted cash, but it's actually tied up until the remainder of that lease expires absent any agreements that we've got from those two companies, which we are actively trying to pursue.
Great. Thanks, so much.
Yes.
Your next question is from Sullivan Ritchie from Goldman Sachs. Your line is now open.
Please.
Thanks, Ravi Thanks for taking our question.
Could you help us understand the cadence of <unk> activation last quarter, you had announced.
And then this quarter it <unk> I guess at this pace in line with your expectations and what are the gating factors to getting to about 40 to 50 by year end and then I just have a second question on just the overall characteristics of the patients that have undergone collection.
Are they more younger patients are.
Are they are there are also adults being treated thank you.
Alright, Thanks, Tom go ahead and comment on the QC activation and patient acres, yes.
Hi, good morning.
Good question. So we are at GTC network was designed our Onboarding process was designed first to establish our network for the launch of the <unk>.
Mostly and then a few centers for Sky Soda and then as we moved into our wave three and wave four Activations, obviously, we looked at integra, but the focus is really becoming preparation for the logos. So launch in sickle cell disease. So we're very pleased to have gotten to <unk> if.
If you look at the way we've structured the waves, we expect a bolus of <unk> to come on in the coming quarter, and we still feel confident that we'll get to between 40% and <unk>.
By the end of this year.
I do believe that since the.
BLA has been accepted by the FDA that Utc's are indeed, more motivated and excited about the possibility of having a treatment for sickle cell disease. So we are seeing <unk> in general start to move faster and again, we're excited to see that at least most all of the wave one and most.
A portion of the <unk> are already treating patients. So we feel good about the progress it's going to plan and we look forward to scaling to between 40 and 50 between now and the end of the year.
And then so collection characteristics I think the most interesting thing I'll just say, it's too early to give common characteristics, we see a wide range of interest from younger patients to older patients from.
Many different states throughout the United States from outside of the United States. So it's really difficult right now to say that there's one or two characteristics that we're seeing when we see the interest for.
In the future as we get bigger and we might be able to nail down more specifics, but I again encourage you that we're seeing a wide range of interest from a wide.
Range of patients.
Okay.
Thank you.
Your next question is from the line of Yaron Werber from TD Cowen. Your line is now open.
Okay.
Hi, This is brendan on pay around thanks for taking the question just a quick one from us and sorry, if I missed this earlier, but kind of looking ahead to love to sell launch.
With the launch really kind of just wanted to get your thoughts on maybe the initial stages of the launch itself are there maybe certain patient subsets of our centers are geographies that you see.
Particularly the low hanging fruit specifically for a gene therapy that could maybe kind of crack the door open to a broader uptake long term really just kind of trying to get a sense of what.
What your initial sales strategy for once you hit the market there.
Yes. Good morning. Thank you for the question and we're excited about the Lotus edge launch we've been really looking at.
The launch will look like as we study kind of not only geographically where patients who have sickle cell disease live and where our treatment centers are going to.
Be activated certainly within those treatment centers. The anecdotally there is a huge unmet need in some cases some of the SKU Tcs are doing clinical trials and they have so many patients they can't even.
They have a backlog just for their clinical trials. So they're really excited about having another option available commercially as soon as something is FDA approved and we look forward to low yourselves potential FDA approval.
We look at a number of things, including probably most importantly, where high.
Patient population for sickle cell disease.
The United States.
And then we look at Q, Tcs, who are willing and setup to treat sickle cell disease, and they understand sickle cell disease. So they need at least a baseline expertise because sickle cell is different than some of the other <unk>.
Transplants and other therapies that they've used historically and then we look at their commercial liability and in some cases some of the <unk> wanted to remain clinical trial sites in other cases, they are pretty savvy. When it comes to commercial launch and so we plan to have a broad network across the country and our goal is to ultimately.
Provide access to any patient in the United States, who needs to be treated in the 40% to 50 gets us into a close proximity of about 95% of the patient population.
Alright, great. Thank you.
Your next question is from Luca <unk> from RBC capital. Please ask your question.
Oh, great. Thanks, so much for taking my question.
Two quick one here, maybe a sickle cell disease.
Your expectations for the label in particular do you anticipated label will include patient between 12, and 18 years of age. The reason why Im asking I believe your competitor does not have patient subgroup.
Primary efficacy at least from the data.
So I'm wondering if it's plausible that youll get a broader label that your competitor and if so what are the implications for the launch and then maybe sort of runway can you just remind me if you do get approved for sickle cell disease will you receive the pier B and if so are you planning to monetize it. Thanks so much.
Alright, thanks, So I'll actually answer the rest of the first part and Chris The second rich, yes, Thanks, Andrew and thanks for that question, Yes, we submitted the BLA for the treatment of patients who are between 12 and 18 and over in that matter. We included patients between 12 and 18, both in our study HCV to six group C as well.
Well as <unk>.
Getting to 10, so for that reason, we have patients who are in that age range and have seen the results from those patients and then I'll turn it back over to Chris as it relates to the <unk> and given Richard's comment it's possible we could receive a PRP. Some slope. So bofa was accepted for priority review for patients 12 and over Howie.
Over.
As it relates to whether or not we'd monetize it we of course would look at the market evaluate the opportunities in the market and then make a decision based upon what we see there I don't want to comment today on whether or not we'd monetize it or not until we know whether or not we'd get a fair price for it.
And just as a reminder, the priv is not factored any potential priv is not factored into our cash runway.
Got it thanks, so much.
Your next question is from Jeff hung from Morgan Stanley . Please ask your question.
Hi, Good morning. This is Katherine on for Josh. Thank you for taking our question. We just wanted to ask if there are any learnings from <unk> or sky solar or other gene therapy launches. How do you plan to apply to <unk> launch either in terms of strategy or from an operation or commercial standpoint.
Yes, hi, good morning.
We obviously one of the big advantages that we feel that we have right. Now is we have almost a year now was intaglio. So we've certainly learned a lot over the last year. Many of those things. We believe we can apply to make them more efficient operational process for logo cell.
I'm not going to get into all the details on this call today, but as I mentioned before not only as bluebird, becoming more efficient and nimble <unk> that we're working with are becoming obviously value partners and more efficient and nimble.
Looking at how we design the GTC network, obviously that was a strategic move on our part to design a <unk> network that serve both in <unk> and lower sales synergistically.
And probably one of the most important learnings as our how we thought about our value based approach to setting the price for his integrity. We will follow that same value based approach when we think about the price of low cell and translating that into access for patients.
Learned a lot through the process as we got ready for this integral about what a innovative payment model could look like we've taken some of that momentum and applied some of those same partnerships to learn about what.
Outcomes based agreement for <unk> in sickle cell it could look like so it won't be the same but we did use a lot of the learnings from our first launch and feel that we've gained great momentum obviously with the patient community great momentum with our <unk> and then feel very confident in the way we are thinking about pricing and outcomes based agreements for load itself.
Okay.
Your next question is from the line of Mani <unk> from Leerink. Your line is now open.
Hi, Good morning. This is on for Marty.
Just maybe a follow up question on the restricted cash linked to the perfect tea leaf.
Reminding us.
<unk> time on the lease and would you expect that to fall winter.
For kind of guidance for cash currently thank you.
Okay I'm going to ask you to repeat the second question, let me answer the first because I couldnt exactly hear you on the second part of the question as it relates to the first it's a 10 year lease and the remainder is approximately seven to eight years can you repeat the second question.
Second part of your question.
That also answers the second part thank you.
Your next question is from the line of Yanan Zhu from Wells Fargo. Your line is now open.
Okay.
Hi, Thanks for taking.
Our questions.
So.
The 11 patient starts for than tableau.
How many of them are from the wave two tcf.
And also wanted to ask about the linear trajectory.
Comment.
I think last quarter as of May nine we've had seven patient starts for the impact of them.
This quarter is 11.
Could we.
Infer from a linear trajectory comment next quarter it might be another four.
So on.
And then.
A question.
The global sale.
Pricing.
You made a comment about the ISR suggested.
Price.
I was just curious.
When you make the decision for pricing is.
The more.
To be.
On par with <unk> or with you.
Target lower price to accommodate the larger patient population. Thank you.
Yes, thanks ill take that.
Part of that question first the.
The first part on pricing on loans are priced where it is not going to comment yes, it's way too early.
And that will be around launch when we discuss pricing go ahead, Tom yes, and maybe to add to that obviously, we are going through the process right now.
We're going to base, our pricing decision on profound benefits that we believe low for cell has in sickle cell disease, obviously, it's a devastating disease and we're looking at the process. Similarly that we looked into the process for his integrity. So we're in a good place, but again, we're not going to comment on the final price.
As far as the launch we've said all along that it will be kind of linear and gradual the thing that I'll add to that is it's tied to Q Tc activations and I'm not going to give the breakdown of the number of patients coming from wave one versus wave two but the way to think about it is that <unk> now most of them onto their second third fourth patients.
Whereas the wave two <unk> are getting through their first patients, but we would expect that to be kind of a build over time, whereas new waves of <unk> come on they are finding new patients while the <unk> that have been on for a while or getting through not only the patients that are in the queue Tc, but also starting to accept the referrals from outside the UTC. So we're.
Excited about the demand that we're seeing and we're excited about the momentum that is starting to build.
Thanks for the color if I may squeeze the last question about the Payor mix, if we look at the $6 8 million dollar revenue.
Could you comment on these patients are paid by government.
Thanks.
Go ahead, Chris look the current payer mix as we've seen it today really ties to the what I would say the onboarding of acute tcs in the function of the patients that they were providing so currently we're seeing a little bit higher indexing towards.
Medicaid, but we continue to believe the stats and the characteristics on the payer mix of Tom has described earlier in his comments.
I will just add one thing to that we've seen.
Patients go through their process and receive ttc's, receiving reimbursement from both private payers and from Medicaid payers. So we're encouraged to see progress.
Private payers, but with Medicaid.
Great. Thanks for the answers.
Your next question is from the line of Sami carbon from William Blair. Your line is now open.
Hi, Thanks for taking my question. This is Brexit Sterling Firstly army.
So given that logo and then peg will follow a slightly different manufacturing process. How do you see this affecting the potential launch of love Lasalle, if any and do you protect predict a similar vein to vein time and are there any other ways you expect to execute the lines of global followed differently.
And look our steel products.
Yes, Hi, this is Tom I'll start with kind of the last part of your question and work my way backwards. The biggest difference with the <unk> launch will be how you think about Q Tc activation and again, starting from scratch youre, starting with the baseline it takes months to onboard a <unk> and a lot of that process.
<unk>, a legal agreement between us and the <unk> and a quality agreement between us and the PTC. So that takes time, what we've done strategically was integra is we've designed our agreements so that they apply to both <unk> and <unk>. So the reason we're trying to get to between 40 and 50 <unk>. This year is to both.
Maximize the opportunity for his integrity, but to make it a smoother transition and set things up for success with logo cell, meaning that the time to get it.
PTC activator for logo will be weeks, whereas it was months for his integrity. So that's probably the first big difference in synergy that Youll see operationally <unk> have learned a lot through the process by administering <unk>. Many of those learnings will apply directly to how they think about those we sell everything from cell collection through it.
And then finally I will just say that we believe that the vein to vein time that you asked about at the time from cell collection to drug delivery of between 70, and 90 days will roughly be the same for but we sell and for any other ex vivo gene therapy in this space.
That's the part that's going to be standard and hard to change for a little while.
There are no further questions at this time I would now like to turn the conference back to our speakers for closing remarks.
Thank you everyone for joining this morning, and just to reiterate we have a we've.
We've made really good progress on our launch to date, we're looking forward to bringing <unk> to patients as well.
Blue Bird continues to build its position is really a unique.
Leading gene therapy company in the industry and we look forward to giving you more updates as we progress. Thank you.
This concludes today's conference call. Thank you all for attending you may now disconnect have a great day.
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