Q4 2022 bluebird bio Inc Earnings Call
Good morning, ladies and gentlemen, thank you for standing by and welcome to the Bluebird Bio's fourth quarter and full year 2022 earnings conference call. At this time, all participants on a listen only mode. After the speaker's presentation. There will be a question and answer session. That's a question session you will need to press.
Star one one on your telephone you well done.
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Please note that today's conference maybe recorded I will now hand, the conference over to speak of host Gordon the only area of Investor Relations. Please go ahead.
Good morning, everyone and thank you for joining today's call.
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. In addition to statements of the company's plans expectations.
Asian or intentions regarding regulatory progress in commercialization plans.
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 Dot Bluebird bio dot com.
Today's agenda is as follows Andrew <unk>, our CEO is going to provide some brief opening remarks and discuss our love us all BLA submission.
Then Tom Klema, Chief commercial and operating officer.
Well highlight positive momentum from ours, and tableau and discussed on our commercial launches and finally, Chris <unk>, Our Chief Financial Officer will provide some color on our finances before opening the call up for Q&A with the team will be joined by Rich Goldman Chief Medical officer with that I will turn the call over to Andrew.
Thanks, Courtney and thank you everyone on the line for joining our call. This morning.
Bluebird vision and mission are stronger than ever.
Built on a strong foundation of research.
Today, Bloomberg is making gene therapy, a reality for patients and families in the real world.
Bensley proud of the efforts of our entire company as we continue to lead the way for the gene therapy field proven commercial model for ex vivo gene therapy was the typhoons petrodelta and continue to make meaningful progress towards bringing gene therapies to patients and their families with sickle cell disease in the U S.
As Courtney mentioned, Tom and Chris will share updates there are commercial progress and our financial position.
I will focus my comments on the Logan L regulatory path.
As noted in our press release. This morning, we continue to progress our <unk> BLA.
Speaking plainly, we will likely missed the Q1 2023 submission goal.
Our file is completely written and ready for submission, but we are awaiting feedback from the FDA on CMC.
To provide context I will give a timeline of our recent interactions with the FDA.
In December we completed drug products comparability analyses and provide the FDA with a snapshot of comparability data.
February received feedback and questions from the FDA and a request that these responses will be provided prior to the submission to be alive.
Our team did incredible work and pull forward the full CMC module, along with additional information provided them to the agency in the first week of March.
We believe that this complete data package supports comparability and addresses the questions asked.
Now while there is no set time line to the updated respond the agency has conveyed its commitment to a timely response and we believe that this could ultimately set the stage first of all to review.
We anticipate feedback from the FDA within a matter of weeks and we'll move quickly to expedite our BLA pending the resolution of the comparability questions, but I will reiterate the file is otherwise complete and ready for submission.
We are grateful to the FDA for the ongoing open dialogue and appreciate the importance of ensuring the agency understands the full context of our analysis.
It takes a step back I'd like to remind everyone of the depth preparation that has gone into both the manufacturing and the clinical development of <unk> and the work that will be reflected in the BLA.
First manufacturer.
CMC modules or the largest section to any gene therapy BLA with.
That in mind Bluebird has an active and collaborative dialogue with the FDA on comparability over the past several years, you've also incorporated learnings from Sky zone is integral into our BLA for <unk> itself.
Now as a reminder for those newer to the Bluebird story through the course of clinical development for Louis L. We made improvements in the manufacturing of our led to viral vector and of our logo cell drug product, including changing manufacturing processes manufacturing facilities and testing sites.
This included changing from adherent to suspension process to assure that we would meet the patient demand at launch with a robust scalable and commercially compliant process for the 20000 patients. We believe may be eligible for gene therapy.
Some of these changes occurred following the completion of our age to be 206 study, which forms the primary evidence base for safety and efficacy to support the BLA.
Therefore, the industry required that we demonstrated vector and drug product comparability will be collected and analyzed all the manufacturing data generated from our commercial processes in our commercial facilities and compare them to the manufacturing data generated from our clinical trials.
Facilities.
These analyses were completed at the end of last year.
We remain extremely confident in the quality of our overall BLA submission package.
And as part of that May be moved to our impressive clinical data.
Our clinical data reflects the most robust data available with the longest follow up across any gene therapy program for sickle cell disease and that includes 150 patients treated in multiple multi.
Multiple patients followed for more than six years.
Strength of this package has been reinforced by positive interactions with the SEC yet.
The BLA submission will be based on efficacy results from 36 patients in the <unk> 206 group C cohort.
<unk>, a median of 32 months of follow up and more than four and a half.
Here is a follow up for some patients.
We anticipate that the BLA submission will also include efficacy results from two patients with 18 months of follow up in the <unk> study.
We plan to request priority review for patients 12 and older with the history. These are inclusive of that's.
Importantly at this time, we are not forecasting any change to our commercial plans for <unk> and are continuing to anticipate in early 2024 launch.
And we are really looking forward with delivering on the promise of gene therapy for sickle cell patients who've been waiting for therapies like this.
As Tom will discuss the foundation that we're building today springs integral to patients.
Directly translate into our ability to bring the lowest delta patients.
The same treating physicians the same PTC network and the same payer relationships.
Bluebird has over a year head start launching a gene therapy inherited hemoglobin disorders versus any other gene therapy program.
And with that I will turn it over to Tom to highlight how our launches are progressing.
Thanks, Andrew and good morning, everyone. It's exciting to be here. This morning to discuss the strong momentum we built for our two approved gene therapies <unk> and sky.
There's a lot of attention and noise in gene therapy. These days, but the reality is that Bluebird, we're living it every day and bringing these important onetime treatments to patients.
During the recent field visit I receive feedback from healthcare professionals in our Q Tcs.
<unk> Bluebird for our continued partnership and for leading the way with our deep expertise and through transparency.
They reinforce that our trusted partnership over the years will be an important differentiator.
As a reminder, our commercial strategy is built on three key pillars, which are fundamental to success in gene therapy launches.
<unk> patients and their families are at the center of everything we do and they are the ultimate decision maker.
Our continued engagement over the past decade, and our ongoing education about gene therapy are critical for adoption.
Second and unique to ex vivo gene therapies.
Building a network of qualified treatment centers or <unk>.
The cell collection and infusion of our therapies takes place.
These centers of excellence are both part of our supply chain, but theyre also ambassadors for patient care and ultimately for our therapies.
And finally access and reimbursement and area, where we have innovated as pricing and securing coverage for a onetime therapy is certainly not the same as pricing of chronic therapy.
Importantly, we are seeing success in all three areas since launch with momentum continuing to build.
For <unk>, we are seeing significant patient demand and uptake.
Highlighting the tremendous unmet need and interest in gene therapy from patients with beta thalassemia.
To date, there have been five patient starts or sell collections for patients with beta thalassemia coming from our early <unk>.
This momentum will continue to build as we grow our <unk> network.
Notice, notably as launch progresses, we are already advancing our plans to expand our manufacturing capacity to meet growing projected demand.
On the reimbursement front, we're pleased to report that things continue to go very well on.
On average prior authorization is taking only two weeks.
<unk> indicated that payers recognize the value of <unk> in a rare disease with significant unmet medical need.
Important we continue to see zero ultimate denials.
Switching to <unk>, we've expanded our <unk> network as planned with 12 centers activated to date, representing a mix of both pediatric and adult centers.
Approximately 30 additional <unk> are in the Onboarding stage or MSA negotiation phase and the company remains on track to scale to between 40 and 50 <unk>.
By the end of 2023.
This progress is because of our preparation and is also a signal of patient interest and enthusiasm among physicians for ex vivo <unk> gene therapy.
Importantly, as Andrew mentioned the efforts were making was integral will directly translate to the potential launch of <unk> for sickle cell disease in three key ways first and very simply the treating physicians are the same.
Second the Bluebird <unk> network will be the same this.
This means that from an operational standpoint, our expectation is that we will reduce the lead time for Q Tc activation for months two weeks because of the synergies.
We're really doing the groundwork now so that we will be ready to begin treating patients shortly after approval.
And lastly, with payers were already being recognized as leading the way with our approach to value demonstration and innovation with our outcomes based agreements and establish credibility will carry forward.
We will continue to provide updates on key metrics from this integral launch including number of patient starts as launch progresses. We believe the most important metric for you to keep an eye on is the number of patient starts.
For Sky Soda cell collection has been completed for two patients.
And on March 16th the first commercial infusion was completed at Boston Children's Hospital.
I cannot overstate, what an incredible moment. This was for this patient and his family.
For the clinicians and researchers and for the entire <unk> community.
<unk> is a devastating disease and this was a milestone celebrated by all those who have advocated invested and fought for treatment options for many many years.
It is difficult to summarize the passion and the pride, we all have and being able to deliver sky zone, it to that young boy and his family.
In conclusion, I am extremely proud of the progress and dedication of our team.
How we are leading the way as a commercial gene therapy company.
With that I'll turn it over to Chris to talk through the financials.
Thanks, Tom and good morning, everyone.
It's gratifying to be speaking with you publicly as Bluebird CFO for the first time since I joined the company back in November of 2022.
We now stand in 2023, and the strongest financial position Bluebird as head.
Since we emerged as a dedicated a gene therapy company in November of 2021.
I'm immensely proud of the work our Bluebird team has done to get us to this point and over the past few months.
During the five months beginning in November of 2022, we were able to raise $326 million in net proceeds from the sale of our <unk>.
And our equity offering.
These actions extended our cash runway into the fourth quarter of 2020 floor 24, when including our restricted cash.
Retiring our near term balance sheet risk has been a tough priority for Bluebird and I'm pleased we're able to provide financing for over 50% of our market cap with just 17% dilution.
We will continue to remain opportunistic and looking at options to extend that cash runway even further.
Okay.
We remain on track with our full year 2023 cash burden guidance in the range of $270 million to $300 million.
And continue to prudently deploy capital as we launch our first.
Alright, sorry, as we launch our two first in class gene therapy.
And prepare when <unk> is approved and available for patients.
As a reminder, it's premature to provide revenue and top line guidance at this point in our launches the.
The company anticipates its first commercial revenue we report Q1 results in May.
Lastly.
I'll touch briefly on the Silicon Valley Bank and its impact of Bluebird.
Following the bank collapse earlier. This month, we were pleased to see the FDIC announcements and that the impact appears to be minimal for the industry.
For Bluebird, we remain in a strong financial position and have diverse banking relationships with controls in place for continuous monitoring of a host of these organizations.
However of note.
Phebe does hold approximately $43 million in restricted cash for Bluebird, primarily related to the collateralized letter of credit in connection with the execution of our sublease four.
Or the 50 Binney Street lease in Cambridge, Massachusetts.
We continue we continue to work on releasing the restricted cash and we will provide updates as and when they become available.
With that I'll turn it back to Andrew.
Thanks, Chris and thanks, Tom for walking us through those updates.
It is a really exciting time at Blue Bird, we are in the midst of two first in class launches showing up for patients with <unk> and with beta thalassemia and Theyre, so close to bringing our gene therapy to individuals living with sickle cell disease.
With that I'd like to invite.
Richard Colvin to join me, Chris and Tom for questions.
Operator.
Thank you, ladies and gentlemen to ask a question you will need to press star one on your telephone and wait for your name to be announced to withdraw your question with Westar. One one again, please standby, while we compile the Q&A roster.
And our first question coming from the line of Jack Allen with Baird. Your line is now open.
Great. Thank you so much for taking my question and congratulations on the progress over the course of the quarter I guess my first question is around the <unk> updates.
Could you provide some comments around the number of patients that were treated with the suspension process and then just to clarify is the FDA asking for additional comparability data or is this a discussion of existing data.
Then finally any analogous situations that you can point towards that as it relates to these these comments.
So much.
Hi, Jack Thanks for the questions and good morning.
So on the last one I cant really portending analogous once in terms of because I think it's the there's been a CMC with gene therapy in general has been something that the FDA has been focused on there are plenty of other examples of people who have switched manufacturing plants.
Et cetera, but I'm not going go into details of other analogs here.
The.
Your question regarding.
Sorry, what was your second question can you repeat that Jack Smith.
The second question was about just to clarify is that they are asking for additional data here.
Gotcha of existing data as it relates to the sponsor.
Well, let me just walk through the timeline again, we provide a snapshot of our data in a package of our data that the FDA in January they came back with questions about that data and request for more information we provided that.
In early March for them to review.
Okay.
And then a question about that.
We're not going to get the details right now on the package about how many suspension ah patients over our versus adhering to the details of that package right.
Okay, great. Thank you so much.
Okay.
Thank you and most of our next question.
Next question coming from the line of Jan Lundberg from Cowen Your line is open.
Hi, guys. This is brendan unfair on thanks for taking my question.
I guess, just really quickly looking ahead to potential <unk> launch what can you kind of helping out a little bit early here, but.
I am really based on your conversations around <unk> and tableau now what are you kind of thinking what can you tell us how youre thinking about pricing and reimbursement and maybe how those conversations for the first two drugs have kind of guided your thinking.
Recent month.
I'd like to hand, it over to Tom to respond yes. Good morning, and thank you for the question. We've been obviously working on the potential price for load with sell for quite some time.
Clearly, we will take the same data driven approach to assessing the value of the cell that we took to approach the value is in Taiwan Sky soda.
Taking into consideration obviously, the positive clinical outcomes quality of life improvements health system cost savings and potential societal impact of patients and families.
Again, we will go through the process, it's probably a little bit too early to give you much more detail than that but we I will say that we are already working with payers and talking to them about what an outcomes based agreement might look like.
Keep in mind that was in tableau.
The outcomes based agreement is pretty straightforward and with the larger population in sickle cell disease that will be it will we'll come up with something different that meets the needs of that population.
Thank you and one for next question.
And our next question coming from the line of Jason Goldberg with Bank of America. Your line is open.
Hey, guys. Good morning, Thanks for taking my questions.
A follow up just one on the regulatory point. So is there is no additional CMC work, it's purely just kind of waiting for a response from the FDA at this point and then one commercial question.
Within the context of sickle cell, how would you envision centers sort of embracing and adopting to competing gene therapies.
We'll be rolling out around the same time, just kind of curious if that presents anything unique in its centers might only choose one and any color in terms of how youre thinking about that process evolving. Thanks.
Yes, good morning, Jason I'll take the first part of the question and hand, the second part over to Tom.
So in terms of we believe that we have.
We've submitted a complete data package at this point, we're confident in what we've put together its in the hands of the FDA right now and we are waiting for their response.
To make sure that they agree with that.
And then I'll hand, it over to Tom for them.
Yes, hi, good morning, Jason.
I think it's a good question.
I think the first important thing to point out is that we are actively treating patients today and these <unk> for beta thalassemia and we'll have over a year head start and we believe based on feedback that that will translate into an advantage for our therapies with the experienced and synergies.
We don't believe that most centers will be exclusive to one therapy. The other if you look at other other markets like the car T market. For example, most centers of excellence like.
Like to offer all available, especially all available innovative therapies. So.
So we wouldn't anticipate centers offering just one of the therapies.
Got it thanks a lot.
Thank you and our next question coming from the line of <unk> <unk> with Wells Fargo. Your line is open.
Hi, Thanks for taking the questions.
So first of all of the regulatory process for level of sale I was wondering.
What questions.
Or what type of questions.
Did the FDA race.
If you have any additional color on that thanks.
Good morning, and we're not going to get into the details of the questions right now to say they did have some questions and ask for some more data that we have.
Believe we fully responded to those questions and submitted the data that.
We have some we submitted its really the full CMC package from the BLA that we pulled forward plus additional data. So it's a very robust package that we submitted.
Got it got it.
Then.
The integral launch.
A couple of questions here.
Hi.
How many patients have verified.
Benefit or a cost rather have forgotten prior authorization.
At this point.
And.
With regard to the five five patient starts.
Do you still estimate 70% to 90 days of manufacturing time, and any estimate on when the first infusion.
And then tackle might occur thank you.
So I'm going to pass that over to.
Tom Yes.
Hi, good morning.
We really are pleased with how the launch is going and we continue to see consistent volume of patients who are initiating benefits verification and moving through to the prior authorization phase.
We at launch provided that as a metric because it was really the only thing that we had that was the indicator of demand.
We've moved now to focusing on the number of patient starts are those that have moved through that process and actually started.
Therapy with the cell collection process. So.
We are providing the updates I will just say that we feel very strong about how things are going and that number continues to increase and be steady.
With regard to the five patients. We did report this morning that the one patient was infused with Sky solar at Boston Children's we reported that one because that was publicized by Boston children's but we're not providing quarterly.
Quarter by quarter guidance on how many patients are being infused.
And then Tom you ask a question about the manufacturing time as well and we still believe that 70 to 90 days Thats correct worth US Integra was 79 days in for Sky Zone, It's a little bit less around 55 to 60 days.
Oh I see got.
Got it that's helpful and maybe lastly.
The label potential label.
You are working towards with the logo cell submission I think you mentioned this.
This is the patient greater than.
12 years of age.
But I guess how.
I think it is it.
Are there two patients with.
Age range.
2018.
In the package and in general how confident are.
You are that you could get a label that go down to.
<unk> that.
Age of 12 years once you submit the Lula field.
BLA.
Yes, so there's more than two patients and that we're not going to get into the actual split the numbers right here, but we are we are submitting for the 12 and over they have study this patient group, where there's a clinical benefit for this group as well.
Do you want to make any more comments that no. Thanks, Andrew you know we have just what Andrew said there have been more than two patients. We have studied <unk> in study 206 and also in <unk>.
Got it got it yes, yes, so I I only got the 10 number yes.
Thank you that's helpful.
Thank you and our next question coming from the line of Shlomi Cohen with William Blair. Your line is now open.
Hi, there. Thanks for taking my question I know previously you've stated that there are some patient care will initiate benefits verification for <unk> receive private prior authorization, but will not proceed with treatment and I guess I was just curious if you're collecting any data with that regard as two reasons patients or Chris.
Leading with treatment.
And then just a second lines and tableau. What's your current number at TTC is in place and how many patients are you able to reach with TTC is in place and how many will you be able to reach if you hit your goal of 40 to 50 centers by the end of the year.
Okay.
Go ahead, Tom Yeah, Great question. So we do collect information on patients who start the both the benefits verification prior authorization part of the process and should they choose not to go forward with therapy, we do collect and monitor the different reasons why.
I won't get into a comprehensive list today, but it could be something as simple as you know.
They are finding out that <unk> is going to be in their states. Soon today. They want to wait until <unk> is closer to where they live or they want to wait for it to get through an event in their life like a play or school gratulation or something like that so we aren't seeing a lot of patients completely fall out of the funnel so to speak but we are seeing.
More kind of continuation type of reasons at least in the early phase.
And the second was the second part of your question.
How many patients are able to reach with your current PTC is in place and how many you'll be able to reach if you hit your call. It 40 to 50.
Yes, so of the 500 patients in the U S.
TVT.
Our initial <unk> network, we thought we could reach about 50 patients that was with our first $5 <unk>. We're now at 12 <unk> and ultimately our goal is to get to between 40, and 50, <unk>, which would give us access to the roughly.
850 patients that we feel fall within our label. So that's the ultimate goal by the end of the year.
Great. Thank you.
Yeah.
Thank you and our next question coming from the line of monies for Ohio with SBB Securities. Your line is now open.
Hi, This is Jamie on for money, maybe just wondering if you could provide any clarity on this includes between beta thal and sickle cell disease.
T. A L D on time between new patient start forms and recognize revenue recognition and if so what are the key variables that would drive any decline slightly.
Yes, So let me turn that over to Tom just to talk about the kind of the the.
<unk> and TEG low in the Sky and the manufacturing times, just from kind of initiative from the time of a new patient start form until they get over the back of that maybe I'll hand, it over to Chris just to talk about revenue recognition.
Yes, so we're intentionally building in a lot of synergies so that we both maximize our current launches, but set ourselves up for success for logo cell. So the a lot of the operational stuff that we're going through now becomes very simple when we get to low <unk>. So we feel that we will be able to reduce kind of acute Tc onboarding.
Time, when we get to two low sell two weeks.
The turnaround time from the time, we collect cells until the time, we are ready to ship themselves back to UTC is between 70 to 90 days on average for us and TEG low and 55 to 60 days for Skype Zona at the majority of that time is based on the testing in the release.
The testing assays in the release that we do after we manufacture.
We believe the time for <unk> will be roughly the same but we don't have that finalized yet so stay tuned for that one but operationally how they enroll for treatment.
Is going to be very much the same with the same treating physicians. So keep in mind. The physicians are getting their experience so that as we get to sickle cell disease. They already have those experiences and the learnings from the Fintech loans got down to watch and.
Chris you want to talk about revenue I sure. Thanks, Tom and with respect to revenue recognition will recognize revenue on Bluebird financial statements when the patients infused.
Thanks, Thats very helpful.
Thank you.
Our next question coming from the line of Andrew Young from Raymond James Your line is open.
Alright, Thank you for taking the questions and sorry to rehash it again, but.
Obviously there is.
<unk>.
With some of the history of the CMC discussion that the blueberry team has had with the FDA specifically can you just kind of break it down really simply.
Why this is not a situation of years past.
From a technical perspective really the differences here of what you what you have to resolve and your communications versus.
Some of the hiccups that you've experienced before that you actually had to take corrective action on before having an accepted filing.
Thank you.
Good morning, Dan.
Good question.
For the last couple of years, the entire gene therapy space has gone through a learning period right and Bluebird we.
Innovators, we've always been on the first to experience okay.
And you've seen that over the last couple of years ago.
And both the FDA and the industry gene therapy industry would have matured in terms of mutual understanding what is required for CMC.
And so over the last two years, we have been in constant dialogue with the FDA about.
Comparability and how to approach it in the conversations over the last couple of years have been focused on what we would need to do the recipe that we need to follow how we do it.
Compensation today is about the content of the analysis and what we submitted so that's the difference.
So just to be perfectly clear and again, sorry to rehash that there is.
No suggestion or thought that you would need to run any additional assays or do assay work.
There is a request for additional information that you have in house.
So I cannot predict the FTAA as close as we are to them one of the things that will never get on a conference call and say is the FDA will not come back and ask for more data I just cant right that I think thats, but we are extremely confident in the package that we put together the entire BLA CMC package plus additional information we've been working on this for a year.
Now we think we've been trying to see around every corner we can.
So I think what youre hearing from the Bluebird side, we have a lot of confidence I cannot speak for the FDA.
Okay. Thank you.
Thank you and our next question coming from the line of Gena Wang with Barclays. Your line is now open.
Hi, thank.
Thanks for taking my question and this is shuang.
Two questions from US. The first question is about the patient.
<unk> been talking about.
So.
If I remember correctly your first patient sale connection for named <unk>.
Are they in the Sky. So now so if you think about the time difference from collection to the Doj asked about <unk>.
Shifting to maybe.
<unk> 30 days.
We assume.
The first patient had been seen with.
<unk> or any other factors in play.
About the timeline here.
We've had that Tom to respond yes, so we don't plan on updating.
Month by month or quarter by quarter on number of infusions.
Keep in mind that the part we can control is the.
Vein to vein time at the time from <unk> to the time, we ship the cells back we cannot control the time after Q Tc gets the cells back when they might infuse that.
We did talk about the Sky zone, one because it was made public by Boston Children's hospital, they actually publicized on it quite a bit and announced it at a meeting.
We had committed to being on track to recognize our first infusion commercially in Q1, and we feel happy that we achieved that.
But yes youre right in that the main difference is that as in tableau lead.
Lead time is 70 to 90 days on average in Sky sounded a little bit shorter. So that's the one variable and keep in mind that Sky Center.
Boys those guys don't have progressed rapidly so theres much more of an urgency to treat with sky solar.
Thank you if I may ask a second question.
Yes.
In my understanding at Blue Bird, we all get paid directly from acuity. So how much out of the data price aiming that can't get for example.
So what is the payer agreed to pay to the QD Stifel two eight.
Patient and acuity is they want to make some profit out of it what will be the discount too.
Blue Bird.
Stan will all QD based under the Master service agreement.
Yes.
Im understanding your question correctly.
Buber, just receives the the price of the drug.
<unk> through the as they go through the prior authorization process are securing.
<unk> commitment to pay for not only the drug but also the cost of the procedure and then they negotiate a case rate for themselves. So they negotiate usually something around the drug in something around the procedure the transplant.
But that is very private information to <unk> don't share that information with us and parents don't share that information with us.
So that's a bit of an unknown to us, but they do they do that inside Ford therapies like this.
Thank you very much.
Youre welcome.
Thank you and our next question coming from the line of.
From RBC capital your line is open.
Yeah.
Oh, great. Thank you so much for taking our question. This is Lisa on for Luka.
Just a couple on Zen Tycho here.
Just wondering if you can add some more color on the five patients who had style collection.
These collections occur at more than one of the 12 key pieces that are currently online.
And what is the.
And also as well if you could add more color on the growing patient demand that you are seeing is this coming from a handful of doctors in a localized geography or is this coming from more broadly throughout the U S. Thank you.
Yes sure good questions.
Yes.
We won't get into a center by center, but the five patients that we reported.
Coming from multiple centers from our early wave <unk> Tcs and Thats why we are confident as we bring on more <unk>. We continue to see demand in each PTC that we bring onboard so the growing demand.
<unk> is coming from not only the existing <unk> that have already started treating patients, but as we bring on each new <unk> they have.
Patients to be treated so the answer to that is both and keep in mind. Our plan was to start with the <unk> and roughly we thought about 50 patients at.
At launch and then to rapidly grow that to between 40 and 50 <unk> by the end of the year.
Also I think maximizes opportunity and sets us up for success was intaglio, but also really lays the foundation for lower sell in sickle cell disease.
Thank you that's helpful. And then just maybe a quick one on sickle cell.
Are there plans to expand beyond the 40% to 50 TTC centers planned already for 2023, when sickle cell potentially launches in 2024.
Yes.
Yes, it's a good question right now our goal is to get to between 40 and 50 <unk>. This year by the end of 'twenty three.
We're learning and what we know about Ah patients in both with both beta thalassemia and sickle cell disease is that some patients are willing to travel, but some patients would prefer to get treated closer to their home.
Our goal ultimately will be to bring our treatments to patients. So that they don't have to travel very far for treatment.
But we're not going to say, what we're growing to beyond the $40 to 50 right now it's fair to assume that we'll probably end up with higher than 50, but we're not commenting on how many at this time.
Thank you and our next question coming from the line of Sylvan Richter with Goldman Sachs. Your line is now open.
Good morning. This is <unk>, we have two questions. The first one on Lasalle in light of this delay how are you thinking about the competitive positioning.
Lasalle and then on is intact.
How much overlap do you think there would be between the PTC is that you have you're going to establish this year and then those that would be needed for sickle cell and then just quickly on the ex U S opportunity.
When do you think you would be ready to think about that thank you.
Good morning, I'll, just take the last one first and hand it to Tom.
We are focused on the U S only right now.
We are getting us therapies launched getting therapies to patients in the U S. It's not it's not on our radar screen for our ex U S at the moment.
Tom Yes, sure Im going to start with your <unk> question first.
<unk> the way, we designed our <unk> network was to pick Utc's at high <unk>.
Expertise in cell and gene therapy, but also treat both.
Beta thalassemia and sickle cell disease over indexed those that have high populations of beta thalassemia first.
And then we're going to grow too gtc's that have higher concentrations of patients with sickle cell disease.
But the simple answer is we believe that 100% of our network will be both.
Integra and sky so in Ah patients.
I'm sorry, he does centers when you think about the competition in your competitive question.
I think I said this earlier, but I think the first and foremost thing to point out is that we're already in these treatment centers and so.
Even with.
The slate.
Shifts in our in our timeline, we believe that we have a competitive advantage by being their first it won't be there first of all over a year.
And then.
As we've done market research over the years, we've seen a slight advantage for lower sales. So we feel very confident that the shift in timeline is that going to impact.
There are those things.
Great. Thank you so much.
Right.
Thank you and our next question coming from the line of Matthew Harrison with Morgan Stanley . Your line is open.
Hi, This is John on for Matthew Thanks for taking my question. So can you comment on the cadence of that patient starts. So what happens after a patient starts like how long it take to sell correction.
Do you expect all the patient starts to be converted into dose patients.
In other words should we expect some patients maybe not going in to dosing after his thoughts. Thanks.
Go ahead Tom.
As far as cadence.
Just to give you a high level overview of patient will come in for obviously a consultation.
They start the benefits verification prior authorization process.
As I noted on the call. The prioritization process is taking about two weeks on average which is great news. The payers have recognized the value is in turbulent skies soda.
And then obviously they'll go through their cell collection, and then go back home.
So collection to when we ship the cells back as between 70 and 90 days and then it's really up to the patient and UTC to decide when they come back in for their infusion in their treatment of.
That part is a little bit less clear to us right now.
We'll wait until we treated it.
A lot more patients before we kind of give thoughts on how long that might take on average.
<unk>.
But the.
Oh Your last question was how many patients that we're focusing in right now and it starts because we believe that once the patient goes through the <unk> process Theyre committed to therapy. So we don't anticipate a lot of patients too.
Pullout pullout of therapy or fall out of the funnel once they go through the self collection.
But that's why we're focusing you on patient starts.
Okay. Thank you.
Thank you and I'm showing no further questions at this time I would now like to turn the call back over to Mr. Andrew Robinson for any closing remarks.
Thank you very much.
And thank you everyone for joining this morning, I just wanted to reiterate.
How proud I am of our team in terms of what we've done over the last several years to bring this to bring two therapies to the patients and getting close to a third completing the BLA in and actually pulling the walk forward in a timely manner. So.
And we it is a time, where bluebird is finally delivering on our mission has had for over a decade, so really really proud of the team. So thank you everyone.
Ladies and gentlemen that does conclude our conference for today. Thank you for your participation you may now disconnect.
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