Q2 2021 bluebird bio Inc Earnings Call
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Yeah.
Operator: Good day, and thank you for standing by. Welcome to the bluebird bio second quarter 2021.
Good day, and thank you for standing by and welcome to the Bluebird Bio second quarter plenty plenty of 1 earnings conference call at the.
Operator: At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question-and-answer session. To ask a question during the session, you will need to press star 1 on your telephone keypad. We request that you please limit your question to one question only. Please be advised that today's conference is being recorded. Now, I would like to hand the conference over to your first speaker for today, Ms. Lesley Pinkett. Please go ahead.
This time all participants are in a listen only mode. After just speaker's presentation. There will be a question and answer session to ask the question Gerry the fashion you will need the press star 1 on the telephone keypad, we request. The please limit your question to 1 question on.
Please be advised that the day conference is being recorded.
Now I would like to hand, the conference over to your first speaker for today and there's less being pet. Please go ahead.
Lesley Pinkett: Good morning everyone, and thank you for joining today's call to discuss our second quarter results and associated business updates. The press release announcing these updates can be found in the investor relations section of our website. 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 financial projections in addition to statements of the company's plans, expectations, or intentions regarding the development of our clinical program.
Good morning, everyone and thank you for joining today's call to discuss our second quarter results and associated business update the.
The announced they need updates can be found on the Investor Relations section of our website.
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 financial projections and addition to statements of the Companys plans expectations and intentions regarding development of our clinical program.
Lesley Pinkett: 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. While we may elect to update forward-looking statements in the future, we specifically disclaim any obligation to do so, even if our expectations change, except as required by law. You should not rely on these forward-looking statements as representing our expectations for any date subsequent to today.
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 and description of these risks is contained in our filings with the S. E C, which are available on the Investor Relations section of our website www.
W that bluebird bio dotcom and while we may elect to update forward looking statements and the future. We specifically disclaim any obligation to do so even if our expectations change except as required by law you should not rely on these forward looking statements as representing our expectations of any date subsequent to today today's agenda is that.
Lesley Pinkett: Today's agenda is as follows. Nick will make opening remarks, and then Andrew himself will review the updates associated with our severe genetic disease business. Then Chip will review oncology news and share an update on the planned business separation. They will then be joined for Q&A by several additional members of the management team. With that, I'll turn it over to Nick. Thanks.
All of those Nick will make opening remarks, and then Andrew and felt overview of the updates of associated with our severe genetic disease and that.
And then chip will review oncology news and share and update on the planned business separation and they will then be joined for Q&A by separate and several additional members of the management team with that I'll turn it over to Nick.
Nick: Thanks Liz and thank you everyone for joining the call this morning. As we outlined in the press release this morning, we look forward to sharing the incredible progress we've made over the last seven months as we prepare to separate and launch two exciting companies early in the fourth quarter, Bluebird and 270. We'll also talk with you more about the clinical hold the FDA has placed on our CLD studies and the safety event associated with that hold. One thing I'll say about the split is that one of my biggest worries with the decision was the potential impact on our people and the unique bluebird culture.
Thanks, Liz and thank you everyone for joining the call this morning and.
As we outlined in the press release. This morning, we look forward to sharing the incredible progress we've made over the last 7 months as repair the separate and launched 2 exciting companies early in the fourth quarter Bluebird and 270 will also talk with you more about the clinical hold the F. D. A has placed on our Seattle D studies and the safety of event associated with that hold.
1 thing I'll say on the split is 1 of my biggest worries with the decision was the potential impact on our people and the unique Bluebird culture.
Nick: I'm happy to share that my worry has turned to excitement as the passion and commitment to the Y have only gotten stronger as we focus and channel the teams into the Bluebird and 270 missions. We may use different words going forward, but the deep blue DNA will continue to be a strong, humbling, and inspiring force. Our intent with the separation is to ensure that we optimize each business from top to bottom in order to deliver on the respective mission. In the next few minutes, I will share the progress we've made to ready ourselves for the approaching day one. There are a few items of particular note.
I'm happy to share. The my worry is turned to excitement is the passion and commitment to the why is the only gotten stronger as we focus on channel of the teams into the Bluebird and $2.70 missions. We may use different words going forward, but the deep blue DNA will continue to be a strong humbling and inspiring force.
Our intent with the separation is to ensure that we optimize each business from top to bottom in order to deliver on the respective missions.
And the next few minutes I will share of the progress we've made to ready ourselves for the approaching day 1.
There are a few items of particular note.
Nick: During today's call, you will hear from Andrew, our post-split Bluebird CEO, that we made the hard but right call to focus our severe genetic disease business on the U.S. The current EU access and reimbursement environment is simply not ready to support and adopt one-time potentially curative innovative medicine. Andrew will share more. Next, the team will share the very recent emergence of an MDS diagnosis for a boy in our ALD study. Our hearts go out to this young man and his family. The FDA, out of an abundance of caution, placed the CLD program on clinical hold.
During today's call you will hear from Andrew are post split Bluebird seal that we made the hard but right call to focus our severe genetic disease business on the U S.
And the current EU access and reimbursement environment is simply not ready to support and adopt onetime potentially cured of innovative medicines and Andrew will share more.
Next the team will share of the very recent emergence of and M. D. S diagnosis for a boy and our E. L. D study our Hearts go out to this young man and his family.
On the F D. A out of abundance of caution placed the C. L. D program on a clinical hold we.
Nick: We have discussed this new information on the CLD program at length with the investigators, external experts, and thought leaders. We all believe that the risk-benefit for patients with CLD remains positive. Pending resolution of the hold, we continue to plan to pursue a U.S. regulatory submission this year. Importantly, we believe that this event does not reflect upon the field of lentiviral-based gene therapy broadly and will have no impact on our THAL, SICKLE, and oncology programs, including our planned THAL and SICKLE BLA submissions.
We have discussed this new information on the C. L. D program at length with the investigators external experts and thought leaders. We all believe that the risk benefit for patients with C. L. D remains positive.
Pending resolution of the hold and we continue to plan to pursue of U S regulatory submission this year.
Importantly, we believe that this event does not reflect upon the field of length of viral based gene therapy broadly and will have no impact on our cell sickle and oncology programs, including our planned cell and sickle BLA submissions.
Shifting gears, we're now 7 months into our separation efforts and our confidence has grown significantly that it was the right strategic and operational move.
Nick: Shifting gears, we are now seven months into our separation efforts, and our confidence has grown significantly that it was the right strategic and operational move. Most importantly, and to the credit of an unbelievably resilient and committed team, our foundation has only gotten stronger as we execute our plan. Our efforts have focused on four key areas that we know to be important to the task ahead. First, establish in tune the vision and strategy for each business.
Most importantly, and to the credit of and unbelievably resilient and committed team. Our foundation has only gotten stronger as we execute our plans.
Our efforts are focused on 4 key areas that we noted the importance to the task ahead.
First established and tuned the vision and strategy for each business.
Nick: Second, double down in every way on the products and R&D capabilities that will create impact for patients and, over time, create value for all stakeholders. Third, get the best people, leaders, and culture in place to execute with conviction.
Second double down in every way on the products and R&D capabilities that will create the impact for patients and over time create value for all stakeholders.
Third get the best people leaders and culture and place to execute with conviction.
Nick: Finally, enable businesses with a healthy financial runway and balance sheet. I'm pleased to say we've made huge progress on all four fronts, and we'll be ready to launch two highly innovative companies at the beginning of the fourth quarter. On the vision and product front, we have made clear plans. We have clear plans in place with several near-term milestones. The SGD business, under Andrew's leadership, has made great progress staying on track with the FAO U.S. VLA filing and continuing to move forward with the CLD filing while making a tough but necessary call to focus the business in the U.S. 270 is shaping up even better than expected.
And finally enable the businesses with a healthy financial runway and balance sheet.
I'm pleased to say, we've made huge progress on all 4 fronts and we'll be ready to launch 2 highly innovative companies at the beginning of the fourth quarter.
On the vision and product front, we have made clear plans, we have clear plans in place with several near term milestones the.
STD business under Andrew's leadership has made great progress staying on track with the Val U S BLA filing and continuing to move forward <unk> filing, while making the tough but necessary call to focus of the business and the U S.
270 is shaping up even better than expected the uptake of of Beckman has been tremendous and our product engine efforts run deep with the first 2 Nextgen Indies coming soon.
Nick: The uptake of BECMA has been tremendous, and our product engine efforts run deep, with the first two next-gen IMDs coming soon. People on the culture front. Teams are in place, fired up, and ready to go. All 1000 birds have been sorted into their future homes in oncology and the S. G. D. Business. We continue to add to the team that has unprecedented bench-to-bedside experience in an incredibly complicated field. The leadership teams are operating independently, with several more key senior executives and board members joining soon, so stay tuned to hear more news on that front. Lastly, on the financial picture, we've been busy ensuring we have the right-sized operational plans, as well as a strong starting cash position. Chip will share more, but here is the take-home.
People and culture from teams are in place fired up and ready to go.
<unk> thousand and birds have been sort of to their future homes and oncology and the S. G D businesses.
We continue to add to the team that has unprecedented bench to bedside experience and an incredibly complicated field.
The leadership teams are operating independently with several more key senior executives and board members. Joining soon so stay tuned to hear more news on that front.
Lastly on the financial picture, we've been busy ensuring we have the right sized operational plants as well as the strong starting cash positions.
Chip will share more but here on the take home.
Nick: We have systematically reshaped our cost structure over the last 12 months to eliminate approximately $400 million in cumulative expenses, unlocking runways. In the past two weeks, we've pulled some levers which further reshape and streamline our structures. Collectively, we expect these efforts and existing and emerging sources of revenue will allow us to launch each business within approximately 24 months, two years of runway following the separation. In short, we're ready and excited for the approaching launch.
We have systematically reshaped our cost structure over the last 12 months to eliminate approximately $400 million and cumulative expenses unlocking runway and.
And the past 2 weeks, we pulled some levers, which further reshape and streamlined our cost structure.
Collectively we expect these efforts and existing and emerging sources of revenue will allow us to launch each business with approximately 24 months to years of runway following the separation.
In short, we're ready and excited for the approaching launch despite the challenges of the pandemic and the extra work and navigating separation each business is not only delivering consistently on the respective products and pipelines, but also now supported by a strong and enabling financial foundation.
Nick: Despite the challenges of the pandemic and the extra work in navigating separation, each business is not only delivering consistently on their respective products and pipelines but is also now supported by a strong and enabling financial foundation. With that, I will now pass it over to Andrew to share some more thoughts on the bluebird slash SGD front. Andrew. Thank you, Nick.
With that I will now pass it over to Andrew to share some more thoughts on Bluebird Slash SGD front Andrew.
Thank you Nick.
Andrew: This morning we announced that we will focus the SGD business on a significant opportunity within the U.S., and we plan to initiate an orderly wind-down. Meanwhile, you're actively seeking strategic alternatives to provide access to our therapies for patients outside of. We are super proud of the bluebird teams who were able to successfully navigate the European regulatory process and prepare for commercialization. Governments and payers have not yet recognized and appropriately priced the innovative value of our transformative gene therapy, despite strong support from patient and physician. This unfortunately makes it untenable for us to bring Tentaglo and Skysona to patients in Europe who cannot afford to wait it out.
And this morning, we announced that we will focus the yesterday business and the significant opportunity within the U S market and then we plan to initiate an orderly wind down of our activities in Europe.
Meanwhile, we are actively seeking strategic alternatives to provide access to our therapies for patients outside of the United States.
While we are super proud of the Bluebird teams were able to successfully navigate the European regulatory path and prepare for commercialization governments and payers have not yet recognized and appropriately priced the innovative value of our transformative gene therapies. Despite strong support for the patient and physician communities.
This unfortunately mixed untenable for us to brings and take low and spice toner to patients in Europe at this time.
As a small innovative company, we cannot afford to wait it out.
Andrew: Turning to the U.S., there are a significant number
Turning to the U S for a significant number of patients in need and a clearer path for reimbursement we.
Andrew: There are a significant number of patients in need and a clear path to reimbursement. We are excited about directing our commercialization efforts to serve these patients. We believe that there are over 20,000 patients with sickle cell disease, beta thalassemia, and cerebral adrenal leukodystrophy in the US that may be eligible for our therapies at launch. And over time, we plan to reach additional patients with product improvements. The majority of these US patients have sickle cell, which is a devastating disease characterized by debilitating pain and frequent hospitalization.
And we're excited about directing our commercialization efforts to serve these patients in need and.
We believe that the over 20000 patients with sickle cell disease beta thalassemia, the cerebral adrenoleukodystrophy and the U S. The.
Of the eligible for our therapies at launch and over time, we plan to reach additional patients with product improvements.
The majority of these U S patients have sickle cell, which is a devastating disease characterized by debilitating pain frequent hospitalization.
Andrew: Through our clinical trials and extensive market preparation, we have a deep understanding of how to deliver our gene therapies to these patients. We know where the patients are and have three-plus years of partnering with Qualified Treatment, which will serve the patients we plan to treat. We are actively building our U.S. commercial infrastructure to deliver a seamless customer experience for both physicians and patients. Additionally, we have prioritized patient access and are pleased with our ongoing engagement with U.S. payers.
Through our clinical trials and extensive market preparation, we have a deep understanding of how to deliver our gene therapies to these patients.
We know where the patients are and have 3 plus years partnering with qualified treatment centers that will serve the patients we plan to treat.
We are actively building our U S commercial infrastructure to deliver a seamless customer experience for both physicians and patients.
And we of prioritize patient access and are pleased with our ongoing engagements with the U S. Players.
Moving.
Andrew: Moving forward, we are on track to submit our data package for approval of our thalassemia therapy to the FDA in the next couple months, and we will continue to invest in product improvements and next-generation technologies that we hope to bring to patients globally in the future. To summarize, we've had to make some difficult decisions, but we have a laser focus on our major milestones ahead and have streamlined our business to tackle our largest opportunities to impact patients' lives.
A word we are on track to submit our data package for approval of our thalassemia therapy for the FDA and the next couple of months and we will continue to invest and product improvements and next generation technologies that we hope to address patients globally and the future.
To summarize we've had to make some difficult decisions.
Of a laser focus on our major milestones ahead, and a streamlined our business to tackle on largest opportunities to impact patients' lives.
Andrew: In addition, we have an important LSL safety update. Today, we announce that a patient treated with LSL approximately one year ago in the phase three ALD 104 study was diagnosed with myelodysplastic syndrome with single lineage dysplasia. Our thoughts are with this boy and his family.
Separately, even and important <unk> safety update.
Today, we announced that the patient treated with allo cell approximately 1 year ago and the phase III a L. D 100 for study with diagnosed with Myelodysplastic syndrome with single lineage dysplasia.
Our thoughts are with this for you and his family.
Philip: He remains under the care of his treating physician and continues to be followed in a bluebird bio-sponsored clinical trial of LSD. It has been determined that this case of MDS is likely mediated by lentid lentiviral vector insertion. As Nick mentioned, this information has been shared with the relevant stakeholders, and out of an abundance of caution, the FDA has placed this program on clinical hold. Pending resolution of the hold, we continue to push forward on the regulatory front, anticipating that we will complete the rolling BLA for LSL in 2021.
So out of the care of the treating physician and continues to be followed and of Bluebird bio sponsored clinical trial of <unk> cell.
It has been determined that this case of Mds is likely mediated by Lindsay D lengthy viral vector and the assertion.
As Nick mentioned this information has been of shared with the relevant stakeholders and out of abundance of caution. The FDA has placed this program on clinical hold.
I think and resolution of the hold and we continue to push forward on the regulatory front and anticipating and we will complete the rolling BLA for <unk> and 2021.
Philip: We believe that this safety event and the clinical hold on the CALD studies do not have an impact on LVV gene therapy programs broadly or specifically on our LVV programs in sickle cell and thalassemia. I'd like to turn it over to Philip to share some additional details regarding the safety update.
We believe that this safety event and the clinical hold on the <unk> studies do not have an impact on LTV of gene therapy programs broadly or specifically on our <unk> programs and sickle cell and thalassemia.
Like to turn it over to Filip and share some additional details regarding the safety updates and Philip.
As Andrew stated are patient and are a L. D..1 of them for study treated with at least on all of approximately 1 year ago has been diagnosed with M. D. S.
Philip: As Andrew stated, a patient in our ALD-104 study, treated with Elysol approximately one year ago, has been diagnosed with MDS. This patient is one of 67 treated with Alizel and one of three we have previously reported to regulatory authorities with a predominant clone, which is triggered when a given insertion site has a greater than 50% clonal contribution. Two of these three patients presented with thrombocytopenia and dysplastic megakaryocytes in the bone marrow, and as announced today, one of these boys has developed MDS.
This patient is 1 of 67 treated with all the cell and 1 of 3 we have previously reported to regulatory authorities with the predominant claim.
And just triggered 1 of given insertion scientists of graces with 50% clonal contribution.
2 of these 3 patients who presented with thrombocytopenia and dysplastic, Mike carrier science, and the bone marrow and as announced today 1 of these boys has developed Mds.
All 3 patients of being closely monitored by the treating physicians.
Philip: All three patients are being closely monitored by their treating physicians. While MDS can transform into leukemia, importantly, this patient does not have tumor cells known as blast cells, indicating that he does not presently have leukemia. Without BLAST cells to evaluate, we cannot confirm the exact role Lenti-D-LVV played in the emergence of MDS. However, due to the presence of this predominant clone, the site of integration in the MECOM locus associated with MDS, and due to the observed misregulation of gene expression at the MECOM locus in the peripheral blood of this patient.
While the MTS can transform into leukemia importantly.
Importantly, this patient does not have tumor cells known as blast cells, indicating that he does not presently have leukemia.
Without blast cells to evaluate we cannot confirm the exact role <unk> played and the emergence of M. D S.
However, due.
Due to the presence of the predominant clone the science of integration and the Metcom Lucas.
And as previously associated with M D S.
And due to the observed mis regulation of gene expression at the May come Lucas and the peripheral blood of this patient.
Philip: We believe this case of MDS is likely mediated by lentid LDZ insertion. Nonetheless, in discussion with the investigators, with our external advisors, and with several experts in gene and cell therapy, we believe the benefit-risk profile of Le Cell remains favorable in the context of the devastating consequences of CALD, the efficacy of LACell in preventing CLD progression, as well as the limitations, morbidity, and mortality associated with Indeed, across our LSL studies, we treated 67 patients with CALD, 90% of whom are alive and free of major functional disabilities or MFDs at the two-year mark post-treatment. Without treatment, this devastating neurological disease usually results in progressive neurological decline and death within five years.
We believe this case of M. D. S is likely mediated by <unk> D LTC insertion.
Yeah.
Nonetheless, and discussion with the investigators.
With our external advisors and with several experts and gene and cell therapy. We believe the benefit risk profile of early cell remains favorable and the context of the devastating consequences of C. E. L D.
The efficacy of at least cell and preventing C. L D progression.
And as well as the limitations morbidity and mortality associated with allogeneic stem cell transplant.
Indeed across our early cell studies, we treat the 67 patients with CA on the 90% of whom are alive and free of major functional disabilities or M. Ftes at the 2 year Mark post treatment.
Without treatment the step with a devastating neurological disease, usually results and progressive neurological decline and death within 5 years.
Philip: For comparison, treatment with an allogeneic stem cell transplant, the only other medical option, has approximately 76% MFD-free survival at 24 months and significant risks of debilitating graft-versus-host disease, including transplant-related mortality. Based on all the available information and pending resolution of the clinical hold, we believe the safety and efficacy profile of this program supports our plan to complete the LSL BLA this year. Furthermore, we believe that the safety event is isolated to the CLD program and that our other programs are not implicated.
For comparison treatment with an allogeneic stem cell transplant the.
Only other medical option has approximately 76% MF D free survival at 24 months and significant rest of debilitating graft versus host disease, including transplant related mortality.
Based on all of the available information and pending resolution of the clinical hold we believe the safety and efficacy profile of this program supports our plan to complete the submission of the <unk> BLA this year.
Furthermore, we believe that the safety event is isolated to the <unk> program and the true.
Other programs are not implicated.
Philip: This is because each treatment is custom designed to address a specific severe genetic disease. Because TLD is a neurodegenerative disease, expression of the therapeutic ALD protein was therefore placed under the control of a viral promoter known as MNDU3, which was selected for its broad tissue activity that results in high expression of the therapeutic transgene in all cell types. To our knowledge, no other LVV HST program employs this design.
This is because each treatment is custom designed to address the specific severe genetic disease.
T L D is a neurodegenerative disease.
<unk> of the therapeutic protein was therefore placed under the control of a viral promoter known as <unk>, 3 which was selected for its broad tissue of activity that results and high expression of the therapeutic transgene and all cell types.
To our knowledge no other LTV HFC program employees. This design.
Philip: For Bluebird, this means our foul and sickle clinical studies, which use a different vector called BB 305, are open, and our plan to file the BLA for foul remains on track for Q3 2021. Now, let me turn the call back over to Andrew. Thank you, Philip.
For Bluebird this means our thal and sickle clinical studies, which use of different vector called <unk> 3 of 5 are open.
And our plan to file the BLA for <unk> remains on track for Q3.2021.
Now, let me turn the callback of every bit of Andrew.
Thank you Philip important updates and we look forward to discussing more in depth and the Q&A and in the coming weeks as we engage with investors.
Andrew: Important updates. And we look forward to discussing them more in depth in the Q&A and in the coming weeks as we engage with investors. In closing, the SGD side of the house is in a strong position to deliver gene therapy to patients in the U.S. We didn't talk about it much in today's call, but our clinical data continues to show the tremendous potential benefit of these therapies. We plan to share an update on our sickle cell data, in particular, at the end of the year.
And closing yesterday side of the house is it excuse me the SGD side of the house and a strong position to deliver gene therapy to patients and the U S.
And we didn't talk about it much on today's call and our clinical data continues to show the tremendous potential benefit. These therapies, we plan to share an update on our sickle cell data and particularly at the end of the year.
Andrew: Our regulatory filings continue with our TDT BLA expected to be completed in Q3 of this year. The CALD BLA planned to be completed by year end, by year end 2021, pending resolution of the clinical hold, and a planned update on the sickle cell regulatory path by end of year.
Our regulatory filings continue with our TVT BLA expected to be completed in Q3 of this year the Caa.
L D BLA and planned to be completed by the year end of year end 2021 pending resolution of the clinical hold.
The planned update on the sickle cell regulatory path by end of the year.
Chip: And our business is focused. We have a strong management team in place with key hires to be disclosed in the coming weeks and a laser focus on getting our therapies to patients in the US and working to expand our reach over time through scientific advancements of our product. I look forward to sharing more detail on this strategy in partnership with the SGD management team in the coming weeks and months. Now, I'll turn to Chip to talk about oncology. Thanks, Andrew.
And our business is focused we have a strong management team in place the key hires to be disclosed on the coming weeks and a laser focus on getting our therapies to patients and the U S and working to expand our reach over time through scientific advancements of our products and.
Look forward to sharing more detail on the strategy and partnership with the S Gene SCD management team and the coming weeks and months.
And now I'll turn to chip to talk about oncology and 270.
Thanks, Andrew.
Chip: Switching gears, I'll highlight key aspects of VEXMA's first quarter of commercial launch. Recall, we announced FDA approval on March 27th of this year, and from the start, we've been extremely pleased with how quickly the team was able to get treatment sites onboarded and up and running, which speaks both to the excellent launch preparation as well as strong interest in EPECMA among physicians and patients. Demand has significantly exceeded our expectations and highlights the tremendous unmet need in patients with multiple myeloma, particularly in the later line setting.
And gears I'll highlight key aspects of the veterans first quarter of commercial launch.
Recall, we announced FDA approval on March 27th of this year and from the start we've been extremely pleased with how quickly the team was able to get treatment sites on board and up and running which speaks both of the excellent launch prep of as well as strong interest and impact.
On physicians and patients.
Demand has significantly exceeded our expectations and highlights the tremendous unmet need and patients with multiple myeloma, particularly in the later line settings.
Chip: We're encouraged to see the unprecedented results from the CARMA clinical study being translated into such strong demand, and we're working with our partners at BMS to deploy every available resource to continue to bring additional capacity online as quickly as possible. On the reimbursement side, we've been able to get significant reimbursement coverage for patients and have not seen that as a barrier to treatment. We still have a lot of work to do to bring abecma to as many patients as possible, including advancing this therapy through our ongoing clinical studies and earlier lines.
We've been encouraged to see the unprecedented results from the Karma clinical study and translated into such strong demand.
And we're working with our partners at BMS and the point every available resource to continue to bring additional capacity online as quickly as possible.
On the reimbursement side, we've been able to get significant reimbursement coverage for patients and of not seeing that is bearing the treatment.
And we still have a lot of work to do to bring it back but to as many patients as possible, including advancing this patient this therapy through our ongoing clinical studies and earlier lines and look forward to continuing that work is the back when it becomes a foundational piece of $2.70 bio.
Chip: I look forward to continuing that work as the background becomes a foundational piece of 270 Bio. Pivoting from the EPECMA launch, I wanted to share some of the strategic background to the resilience collaboration we recently announced. This deal is a true win-win outcome.
Pivoting from the effect on the launch I wanted to share some of the strategic background to the resilience of collaboration we recently announced.
This deal is a true win win outcome. We think the deal provides clear external validation of the suspension of <unk> expertise we've developed the blue.
Chip: We think the deal provides clear external validation of the suspension LED expertise we've developed at Blue. Resilience will be able to leverage the infrastructure and the highly skilled employee base we established in North Carolina to continue to support current and future vector needs for both Bluebird and 270. Resilience plans to retain all of its North Carolina employees. Financially, we will receive $110 million at closing, which we anticipate occurring in the third quarter. The future cost savings is approximately $25 million per year and increases over time; we will have preferred access to the facility at a preferential rate.
Resilience will be able to leverage the infrastructure and the highly skilled employee base, we established and North Carolina to continue to support current and future vector and needs for both Bluebird and $2.70.
The resilience plans to retain all of our North Carolina and place.
Financially, we received $110 million of closing, which we anticipate occurring and the third quarter the.
The future cost savings is approximately $25 million per year and increases over time.
We will have preferred access to the facility at preferential rates.
Importantly, we remain committed to manufacturing innovation for $2.70, the strategic collaboration with the resilience is about much more than an asset sale of the <unk> facility.
Chip: Importantly, we remain committed to manufacturing innovation for 27. The strategic collaboration with the resilience company is about much more than an asset sale of the BRT facility. We will be collaborating on the manufacturing of future pipeline products together, which we expect will increase the number of programs and the pace of development. We also plan to invest in internal drug product manufacturing capability and capacities to support clinical trials and expect to share more details on that as we approach the separation.
And we'll be collaborating on the manufacturing of future pipeline products, together, which we expect will increase the number of programs and the pace of development.
We also plan to invest and internal drug product manufacturing capability and capacity to support clinical trials and expect to share more details on that as we approach the separation.
We believe that the ability to rapidly iterate and early clinical development will be a vital component of our ask and answer engine for design of new product candidates and oncology.
Chip: We believe that the ability to rapidly iterate in early clinical development will be a vital component of our ask and answer engine for designing new product candidates in oncology. As we approach the plan separation in the fourth quarter and look towards the end of the year, we plan to continue to share information about each business. Meanwhile, internally, we remain focused, as always, on the important clinical and regulatory models.
As we approach the planned separation and the fourth quarter and look towards the end of the year, we plan to continue to share information about each business.
Model internally, we remain focused as always on the important clinical and regulatory milestones.
Andrew highlighted the upcoming SGD milestones and we are equally excited about the future of the $2.70 side we.
We plan to file of 1 to 2 new <unk> by the end of this year and and anticipate delving into those and is more deeply on and upcoming investor call.
Nick: Andrew highlighted the upcoming SGD milestones, and we are equally excited about the future on the 270 side. We plan to file one to two new INDs by the end of this year and anticipate delving into those INDs more deeply on an upcoming investor call. We're excited about the continued launch trajectory of Vecma and the increased financial flexibility that it will provide over time. On both sides, we're also pleased at the progress that's been made on building out top care management teams and boards, and we'll share more about those key hires in the coming weeks and months.
Excited about the continued launch trajectory of the Beckman and the increased financial flexibility of that.
It will provide over time.
On both sides. We're also pleased of the progress that's been made on building on top tier management teams and boards and we'll share more about those key hires and the coming weeks and months.
So more to come on a lot of fronts, and we look forward to share and some to some increased investor engagement as we head towards the separation.
A lot of moving parts of today, but we wanted to step back and summarize what it all means financially.
We've been focused on.
Nick: So more to come on a lot of fronts, and we look forward to sharing some, just some increased investor engagement as we head towards the separation. A lot of moving parts today, but we wanted to step back and summarize what it all means financially. We've been focused on building both businesses, having strong balance sheets and reshaped and right-sized operating plans to achieve value-creating milestones. And we've made important progress on this front.
And on building both businesses.
Having strong balance sheets, and reshaped and rightsize, the operating and plans to achieve value creating milestones.
And we've made important progress on this front since the beginning of 2020 as Nick highlighted we have reduced our expenses by approximately $400 million from our original operating plan.
And with the resilience of partnership and the planned wind down of the European operations, We announced today, we further streamlined the future capital needs for each business and short was taken the tough but necessary measures to ensure these companies launch from the position of strength.
Nick: Since the beginning of 2020, as Nick highlighted, we've reduced our expenses by approximately $400 million from our original operating plan. And with the resilience partnership and the planned wind-down of the European operations we announced today, we further streamline future capital needs. In short, we've taken the tough but necessary measures to ensure these companies launch from a position of strength. We ended the second quarter with approximately $942 million in cash, based on our current cash position and the expected $110 million upfront payment from the Resilience Collaboration.
We ended the second quarter with approximately $942 million and cash.
Based on current cash position and the expected 110 million upfront payment from the resilience of collaboration the company anticipates, having a cash balance of approximately $900 million at the time of separation together.
Together with the existing and emerging sources of revenue, we expect our cash balance will be sufficient to fund approximately 24 months of operations for Bluebird and for $2.70 under their respective current business plans.
And with this runway, we expect that we will carry of both organizations through significant value creating milestones.
Nick: The company anticipates having a cash balance of approximately $900 million at the time of separation. Together with the existing and emerging sources of revenue, we expect our cash balance will be sufficient to fund approximately 24 months of operations for bluebird and for 270 under their respective current business plans. And with this runway, we expect that it will carry both organizations through significant value creation. We'll have much more to say about the starting cash position and forward-looking financial guidance closer to the split, but we're reiterating the foundational principle that both businesses will start with equally strong balance sheets. With that, I'll turn it back to Nick for a closer. Thanks Chip, Phillip, and Andrew for walking us through these updates. There was a lot to digest on this call.
We'll have much more to say about the starting cash position and forward looking financial guidance closer to the split but we're reiterating the foundational principles of both businesses, we'll start with equally strong balance sheet positions. So the.
With that I'll turn it back to Nick for closing remarks.
Thanks, Chip, Philip and Andrew for walking us through these updates of lots of digest on this call. So we'll be happy to take Q&A and have several members of the management team on the line as well to field any questions. So with that let's go to the operator and see what questions. We can field.
Thank you as a reminder to ask the question you need to press Star 1 on your telephone keypad to withdraw your question perhaps of the balance sheet. We also requests the please limit your questions to 1 question the only your.
Your first question comes from <unk> reached their Goldman Sachs. Your line is now open.
Hi, Thank you so much for taking our questions the Sonya on for Celgene.
Nick: So we'll be happy to take Q&A and have several members of the management team on the line as well to field any questions. So with that, let's go to the operator and see what questions we can field. Thank you. As a reminder, to ask a question, you need to press star one on your telephone keypad. To withdraw your question, press the pound. We also request that you limit your question to one only.
For the case of Mds and the patient treated with Alice how can you leverage and learnings from earlier this year to better understand it and what design features of L. D D.
Are likely to have contributed to it and then I have on my follow up question. Thank you.
Thanks, Tony and for the question I think that's probably the best field of by Phil Yeah. Thanks for the question.
Yes short answer is yes, the the prior work up that we did and the case of sickle cell disease that as you will recall exonerated the LTV in that case.
Operator: Your first question comes from...
Operator: Salveen Richter of Goldman Sachs. Your line is now open.
Essentially we will try and do the same thing I'd like to remind you that at this point, we don't have a tumor cells or leukemia too.
Sonia: Hi. Thank you so much for taking our question. This is Sonia from Salveen. For the case of MDS in the patient treated with LSL, can you leverage learnings from earlier this year to better understand it? And what design features of LVV are likely to have contributed to it? And then I have one more follow-up question. Thank you.
To analyze at this point that said several features that I went through on the call, namely the site of integration and the Mis regulation of gene expression of that site and peripheral blood.
Make us believe that this is likely a lengthy D <unk> mediated event.
Nick: Thanks, Sonia, for the question. I think that's probably best answered by Philip.
You sort of you also asked about.
The other are the features of the of the Victor that.
Philip: Yeah, thanks for the question. The short answer is yes. The prior workup that we did in the case of sickle cell disease, which, as you will recall, exonerated LVD in that case. Essentially, we will try and do the same thing.
We can particularly point to.
The they're actually many differences between the sickle programs and the.
And the ILD program, but from a vector perspective the.
The promoter of usage is quite distinct and thanks.
On the call the promoter used and the ILD program was designed for broad tissue expression and high levels of expression across all cell types.
Philip: I'd like to remind you that at this point, we don't have tumor cells or leukemia to analyze at this point. That said, several features that I went through on the call, namely the site of integration and the misregulation of gene expression of that site in peripheral blood, make us believe that this is likely an LNTD LVV insertion-mediated event. You also asked about, you know, are there features of the vector that... you know, we can particularly point to.
By contrast, the DB 3 of 5 back the designed for tissue specific expression only and those cells that will drive towards red blood cells of the erythroid languishes and that's a very important distinction between the 2 programs.
Thanks, a lot.
Thanks for question.
Oh, sorry.
And I ask another quick 1 of ours.
We're trying to limit to 1 but if it's a quick follow up on yes, just a quick follow up on the what experiments of assays would you be doing to better understand that.
Philip: I just, you know, there are actually many differences between the CICL programs and the ALD program, but from a vector perspective, the promoter usage is quite distinct. As I said on the call, the promoter used in the ALD program was designed for broad tissue expression and high levels of expression across all cell types. By contrast, the DB305 vector is designed for tissue-specific expression only in those cells that will drive towards red blood cells, so the erythroid lineages, and that's a very important distinction between the two.
Yes, so at this point without tumor cells to analyze we've completed the experiments that we can do at this time.
Thank you.
Your next question comes from money flow Har of NAV.
The b.
Your line is now open.
Thanks for taking the question.
And we've seen a couple of tough for you guys Nick commercially in the <unk>.
Europe some some.
The challenge of the come with doing novels by developing complex therapies for pretty severe diseases.
Philip: I'm sorry, could I ask another quick one, or we're trying to limit it to one, but if it's a quick follow-up and yes, just a quick follow-up on the what experiments or assays would you be doing to better understand that? Yeah, so at this point,
Talk a little about the commercial situations and the U S for our rare disease franchise.
And what and where the investors look for confidence.
And that execution and realize the reimbursement will be better and the U S. Given the challenges you've had you've had in the EU and to what extent will you be able to overcome any trepidation around fees.
Philip: Yeah, so at this point, without tumor cells to analyze, we've completed the experiments that we can do at this time.
The signals that may reasonably or not the read across into the signal of beta cell program.
Operator: Your next question comes from Mani Foroohar of SVB.
Mani Foroohar: Your line is now open. Thanks for taking the question. So we've seen a couple of tough outs for you guys commercially in Europe, some challenges that come with doing novel science and developing complex therapies for pretty severe diseases. Now, let's talk a little about the commercial situation in the U.S. for the rare disease franchise. What can investors look for confidence that execution and realized reimbursement will be better in the U.S. given the challenges you've had in the EU, and to what extent will you be able to overcome any trepidation around these safety signals that may reasonably or not be read across into the sickle and beta-thal program? This is Nick.
This is Nick I'll pass it over here to the Andrew and Tom Real quick just on your last little bit there.
It's actually quite reasonable and then there's no read through to cell to sickle awards and the broader community and we believe the regulators.
Certainly have signaled that same with the approach that they're taking and the engagement. So that said 1 of I pass it on because we certainly have studied the U S vs. European dynamics extensively and have a very strong belief there but andrew.
So just as a reminder, very different systems in Europe, and the U S. European systems have each have a single consolidated payer with the governments and negotiate prices for the entire country.
Nick: I'll pass it over here to Andrew and Tom real quick. Just on your last little bit there, I think it's actually quite reasonable that there's no read-through to thal, to the broader community, and we believe the regulators certainly have signaled that same with the approach that they're taking and the engagement. So that said, why don't I pass it over, because we certainly have studied the U.S. versus European dynamics extensively and have a very strong belief there. But Andrew?
The U S has free pricing overall in general and it's many different parties private payers and government payers that are negotiated with so very very different systems, but to come and more I'd like to hand, the Tom Klema, who is the chief commercial officer for the spirit of genetic disease and Tom just for you go Manny can you just mute your line real quick there's some background noise. If you don't mind.
Sure Yeah. Thanks for the question Manny I think.
Andrew: So just as a reminder, very different systems in Europe than in the US. European systems each have a single consolidated payer with the government and negotiate prices for the entire country. The US has free pricing overall, in general, and it's many different parties, private payers, that are negotiated with. So very, very different systems.
And we've been actively working both with our qualified treatment centers and <unk> and our U S payers for over 3 years now and continue to get very positive feedback and dialogue about our path for securing both coverage and reimbursement access for our patients is 1 of our nimble and priorities and we're working closely with.
All of the stakeholders to make sure that we can bring these potentially curative onetime therapies to patients.
Thomas J. Klima: But to comment more, I'd like to hand it to Tom Klima, who is the chief commercial officer for Spear Genetics. Tom, just before you go, Mani, can you just mute your line real quick? There's some background noise, if you don't mind. Yeah, thanks for the question, Mani. I think we've been actively working both with our qualified treatment centers and our US payers for over three years now and continue to get very positive feedback and dialogue about our path forward, both coverage and access for our patients is one of our number one priorities, closely with all of the stakeholders, curative one-time therapies for patients.
Thanks, guys.
Thank you Matt.
Next question comes from Yaron Werber of Cowen. Your line is now open.
Yes, good morning.
The question on upstream of basketball.
Off to a great launch in the quarter the.
Question is more about supply can you give us a little bit of a sense for I understand is the.
<unk>.
The rate limiting step here is actually the vector production and I think there's been some confusion at some point I believe Bristol said, they can get through as many of the 100 patients per month in August, but it's not clear that that's really going to be the run rate from this point onwards. The could you just give us a sense. So we can better understand what the supply could be and we can model that appropriately. Thank you.
Yaron Werber: Yaron Werber of Colin. Your line is now open. Yes, good morning. I've got a question on Psema, and Vecma, which are off to a great launch in the quarter. The question is more about supply. Can you give us a little bit of a sense to understand there's a rate limiting step here is actually vector production?
Yes. Ron. This is this is Nick I think it's a fair comment I think both BMS and US. The 1 thing I can say is we are completely.
We focused on this because of the importance of this medicine clearly is to the patient community as evidenced by the by the uptake and so both on the vector and the drug product side of the equation, we're running on the exact numbers and the evolution of that as something that we are working on with the FDA and actually have made great progress since since approval and so forth and exactly where it will.
Nick: And I think there's been some confusion at some point. I believe Bristol said they could get to as many as a hundred patients per month in August, but it's not clear that that really is going to be the run rate from this point onwards. So could you just give us a sense of what the supply could be, and we can model it appropriately? Thank you. Yeah, Ron, this is this is Nick. I think it's a fair comment.
By when is something that we're working on with BMS and haven't fully disclosed right. We're certainly optimistic that we're going to be able to meet as much of this demand between now and end of year as we continue to ramp capabilities on both sides of the house, meaning drug product and vector I don't know chip. If you wanted to add any more details of that.
Nick: I think both BMS and us, one thing I can say is we are completely focused on this because of the importance of this medicine clearly to the patient community, as evidenced by the uptake. And so both on the vector and the drug product side of the equation, we're running at the exact numbers, and the evolution of that is something that we are working on with the FDA and actually have made great progress since approval, and so forth.
No Nick I think that was well said.
Minder vector today is produced to actually add a third party CMO and and adhere the setting we're also pushing towards bringing suspension vector online at a future date.
But today the <unk>.
Focus amongst the teams is on increase in vector production and <unk>.
Nick: Exactly where we'll be by when it's something that we're working on with BMS. And having fully disclosed, right, we're certainly optimistic that we're going to be able to meet as much of this demand between now and the end of the year as we continue to ramp capabilities on both sides of the house, meaning drug product and vector. I don't know, Chip, if you want to add any more details. No, Nick, I think that was well said.
Increasing the the weekly and monthly number of drug products, we can produce.
Downstream testing and release testing.
Amit there is focused on every aspect of that supply chain to deliver for as many patients as quickly as we can and we certainly expect.
Between here and the end of the year debt capacity will be increasing but.
And I think we're in a stop short of kind of give me and month by month numbers in terms of the expectation just yet and you.
Nick: Reminder, Vector today is produced at a third-party CMO, and here in the setting, we're also pushing towards bringing suspension Vector online at a future date, but today, the focus amongst the teams is on increasing Vector production, increasing the weekly and monthly number of drug products, downstream testing and release testing, you name it, there's focus on every aspect of that supply chain, liver, for as many patients as possible. And we certainly expect between here and the end of the year that...
On the exciting part here is sort of.
And certainly don't seem to be demand constrained right..1 1 question was what is the interest in this type of therapy for the later lines and that clearly has been answered and so whether it's giovanni of Chris of the various folks over of BMS I can tell you on a weekly basis are completely engaged.
Great.
Next question.
Next question comes from the Fei Yang Mizuho Securities.
And I will open.
Yeah, Hi, good morning, and thanks for taking my question so on.
Operator: The next question comes from Di-Fei Yang of Mizuno Securities.
Back.
How quickly do you think you can move into early early airline huh.
Operator: Your line is now open.
Di-Fei Yang: Hi. Good morning, and thanks for taking my question. So, on ABACMA, how quickly do you think we can move into earlier lines of treatment, and when should we be expecting to see initial data? And then a quick question on the CALD part. I'm just curious if you are still thinking about adding additional pipeline on the rare disease side, and if that is the case with what's happening with CALD, would that limit your choices?
And the treatment.
And we.
When should we be expecting to see initial data and a quick.
Yeah.
On the account part.
And I guess curious if you are still thinking about adding additional pipeline.
And on the rare disease side and if.
And that is the pace with what's happening with Cowen.
And would that limit the like tweaked.
Nick: This is Nick.
And it.
Nick: This is Nick. I'll take the earlier lines question, and Andrew, maybe you can speak to the CLD question. Earlier lines, there continues to be a broad and comprehensive development plan in collaboration with BMS, everything from third line to second line and so forth. So that is something that we are working on with them. The data will continue to emerge throughout this year and, more likely, next year and the year after, as one would expect with those studies.
This is Nick I'll I'll take the earlier lines of question and the Andrew maybe you can speak to the CL D question earlier lines of continues to be abroad, and comprehensive and it.
Development plan and collaboration with BMS and everything from third line second line and so forth. So that is something that we are working on with them. The data will continue to emerge throughout sort of this.
Of this year more likely and the next year and the year. After as 1 would expect with those studies, so still a very broad spectrum commitment to that as we have certainly seen the interest in the back of amount and the therapy and we believe that over time and it'll be shown that this medicine also can work as well if not better and earlier lines of therapy, so that commitment and investment.
Andrew: So still a very broad spectrum commitment to that, as we have certainly seen the interest in Becma and the therapy, and we believe that over time it will be shown that this medicine also can work as well, if not better, in earlier lines of therapy. So that commitment and investment continue. Now, let me just pass it over for the ALD question. Yeah, absolutely. So the short answer is we're focused right now on our three products that we're going to bring to patients in the U.S., including CALD, for which we do believe the risk-benefit there is positive. We do have active research into in vivo LVV right now, and we remain confident that we know how to design, something that we have the capability to do, so it will not limit our approach.
<unk> now.
Now let me just pass it over for the ALJ question, Yeah, absolutely. So the the short answer is we're focused right now on our 3 product and we're going to bring the patients in the U S, including CIL D. Because we do believe the risk benefit there is positive.
We do have and active research into in vivo and <unk> right now and we remain confident that we know how to design the doctor safely and that is something that we have the capability to do so we will not limit our approach to using LTV for future indications.
Nick: And part of the benefit also, I think, here in that question is that the move here to focus on the US allows you to invest not only in those products but also in their improvement and then also in the future by limiting the sleeve, if you will, where we can invest and deploy capital, which is, I think, a benefit of the decision taken today. Good question, thank you. The next question comes from Mr. Matthew Harrison of Morgan Stanley. Sir, your line is now open. Thank you for taking my question. This is Charlie for Matthew.
And part of the benefit also I think here and that question is the move here to focus on the U S allows you to invest not only on those products, but also on the improvement of those products and then also the future by limiting the sleeve. If you will where we can invest and deploy capital which is I think the benefits of the decision taken today.
Good question. Thank you.
Next question comes from Matthew Harrison of.
Morgan Stanley Sir Your line is now open.
Thank you for taking my question. This is Charlie on for Matthew.
Operator: Do you have a timeline for the European operations winding down, and what kind of savings can we expect from that winding down? And maybe just another one is, would you expect to be able to find a marketing partner this year? Or is it more like a separate kind of item?
Do you have a timeline for the European operations, and so we're winding down and what kind of say the can.
And we expect this on.
And now wind down.
And maybe just another 1 is noteworthy and would you expect to be able to find the luck of the partner this year.
And what is the more of like a similar kind of items. Thank you.
Andrew: Well, why don't we start Andrew, you take the first part of the question about sort of the timing and details of the wind down, maybe Chip, you can comment on the financial implications. We expect the majority of the wind down to be completed by the end of the year. We will be treating patients that are in the pipeline through the end of the year, but after that, the operation will either be pushed by or will be taken over by a partner or, Yeah, in terms of the cost side, Europe was a meaningful piece of our overall SG&A cost structure.
Well why don't you start and then you take the first part of the question of us sort of the timing and details of the wind down and maybe chip you can comment on the implications financially.
We expect the majority of the wind down to be completed by the end of the year and we will be treating patients that are in the pipeline through the end of the year, but after that and the operations pool.
Either be pushed or will be taken over by a partner for them.
Yes, and in terms of the cost side.
Europe was a meaningful piece of our overall SG&A and cost structure.
Andrew: We are moving through a consultation period with European employees as mandated by governments in Europe, and so that'll take some time. So I think we're a little short of giving precise guidance there. I think importantly, too, we will maintain the value of those assets, https://www.kenhub.com regulatory submissions or whatever, but the infrastructure will largely be coming down. We think it'll be meaningful savings as we look to the future. And I think we'll have more to say as we get through the process. And the other part of your question, which I'll just mention was that on the partnering side, there are no specific comments on that.
We are moving through a consultation period with.
European employees of mandate as mandated by the.
Governments in Europe, and so that'll take some time, so I think we are.
A little short of giving a precise.
Precise guidance, there and importantly, too we will maintain the value of those assets in terms of.
On longer term extension studies.
And regulatory submissions of whatever but the infrastructure will largely be coming down we think it will be meaningful savings as we looked for the future.
And I think we'll have more.
As we get through the process of winding it down.
And the other part of your question. There I'll just mentioned was the on the partnering size of Theres no specific comments on that but we certainly are optimistic that there are a number of different players that will engage with us on that front and are engaging with us on that front exactly where that lands, we'll have to see because we do think it is and important set of products to get to patients.
Andrew: But we certainly are optimistic that there are a number of different players that will engage with us on that front and are engaging with us on that front. Exactly where that lands, we'll have to see because we do think it is an important set of products to get to patients, if not right now, but certainly as we lean towards the sickle product, including in Europe.
Right now over time, and certainly as we lean towards the sickle.
Product.
Moving to Europe.
Thank you.
Your next question comes from Dane Leone of <unk>.
Chip: The next question comes from Dane Leone of Raymond James.
England genes. Your line is now open.
Dane Vincent Leone: Dane Leone of Raymond James, your line is now open. Hi, thank you for taking the questions. In your disclosure, and I'm not sure in the script you addressed it, but you mentioned that the hope is, post split, you have about 24 months of runway for both businesses on a cash basis for operations. I know you might get into it a little bit later, but can you just clarify a little bit more for our models, how that's going to work versus the current operating burn that we see on a consolidated basis, maybe with an adjustment for less spend in Europe, but there's going to be a degree of disenergy post the split.
Hi, Thank you for taking the question.
And in your disclosure and.
And I'm not sure on the script you addressed it but.
You mentioned that the the hope is post split you have about 24 months of runway for both businesses on a cash basis for operations.
And I know you might get into a little bit later, but can you just clarify a little bit more for our models.
How that's going to work versus the.
The current operating burn that we see on a consolidated basis, maybe with an adjustment for for less spend in Europe, but.
There is gonna be a degree of the synergy post the split.
Dane Vincent Leone: So any color you can give us just in terms of what we might see in additional rationalization on R&D or SG&A for the post-split businesses would be helpful. Thank you. Good question. Why don't I pass that right on over to Chip?
So any color you can give us just in terms of what we might see and additional rationalization on R&D or SG&A.
For the post split businesses.
I think would be helpful. Thank you.
Good question of why don't I pass that right on over to chip.
Chip: Yeah, no, thanks. I would say yes, we are continuing to rationalize the cost structure, and those effects won't be fully realized until next year between the transition of the ERT manufacturing plant to resilience and then this decision in Europe. So those are two pretty big levers.
Yes, no. Thanks.
I would say, yes, we are continuing to rationalize the cost structure and those effects.
Won't be fully realized until next year between.
The transition of the BRT manufacturing plants, the resilience and then this decision and Europe. So those are 2 pretty big levers at.
Chip: At the same time, we see Becma continuing to ramp, which provides an important tailwind for the 270 or the oncology business. In terms of the pipeline for SGD, the focus is going to be squarely on bringing those three late stage products to patients in the United States. And then for 270, it's going to be the focus on the IND engine. So, as we alluded to, there's a cost side of that, which is largely in our control, and a commitment to managing the business within that 24-month runway.
And at the same time, we see back from of continuing to ramp which provides an important tailwind.
For the $2.70 of the oncology business in terms of the pipeline for SCD. The focus is going to be squarely on bringing those 3 late stage products to patients.
In terms of those patients and the United States.
And then for $2.70.
It's going to be the focus on the <unk> engine. So.
As we alluded to.
There is the cost side of that which is largely in our control and our commitment to managing the business to within that 24 months.
Chip: And there's also the revenue side of the equation, which is existing sources of revenue with the BACMA, emerging sources of revenue on the SGD side as products are approved here in the United States next year. More to say on the revenue side, but we feel good about our ability to operate for the next two years. Thank you. Thank you. The next question comes from Gina Wang of Barclays. Your line is now open.
Our runway and.
And there's also revenue side of the equation, which is existing sources of revenue and the backbone of emerging sources of revenue on the SCD side as products are approved here in United States next year, and then I think we see a number of strategic levers that we can continue to exercise on on the revenue side. So more to say there as we move for it but we.
Feel good about our ability to operate these businesses for the next 2 years.
Thank you.
The question. Thank you.
Next question comes from Gena Wang of Barclays. Your line is now open.
Huidong Wang: Thank you for taking my questions. I have one question regarding the Lenti-D. I'm just wondering what kind of experiments or data you need to submit to the FDA to show that the reason for this MDS is because of the MD-DU3 promoter, not due to the other components. I just want to make sure that the backbone for Lenti-D versus Lenti-Globin has no other differences, including the locus control region and also, you know, the three prime ions that you remove the insulator element
Thank you for taking my question just 1 question regarding the <unk>.
Lengthy D.
Just wondering what kind of experiments on data.
And to submit to the FDA to show that.
The reason for the Mds because of the M D D.
The 3 promoter.
Due to the other components just wanted to make sure the bank.
<unk> for lengthy D. The.
<unk> there is no other differences, including low cost control raging and also the.
3 timeline that you removed the insulating.
Element.
I think I'll pass that 1 over to Philip.
Philip: I think I'll pass that one over to Philip. Thanks, Gina. So it's difficult, obviously, to do these experiments in retrospect. That said, there are substantial differences between the vectors that you're alluding to.
Thanks Gena.
And so.
So it's.
It's difficult obviously to do these experiments and.
In retrospect that said there are substantial differences between the vectors that you are alluding to.
Philip: However, we believe that the critical component is the choice of promoter, and specifically the choice of a promoter in the case of LentD, which was necessary for broad tissue expression versus one that's much more restricted and tissue specific. We think that's the major difference that's playing a role here. I want to make it clear that by itself, an integration into a particular locus isn't sufficient.
However, we believe that the critical component is the choice of promoter.
And specifically of the choice of the promoter and the case of lengthy D, which was necessary for broad tissue expression versus 1 that's much more restricted and tissue specific.
We think that's that's the that's the major difference that's playing a role here I.
I want to make it clear that.
The by itself and integration and to a particular Lucas is we don't think thats sufficient right. So that by itself is not enough that said, we think that's sort of the the first hit if you will and there may be other things that are necessary.
Philip: So that by itself is not enough. That said, we think that's sort of the first hit, if you will. And there may be other things that are necessary. So that's an important component. But from a difference between the globin promoter that's used in BP-005 and the CLD-Lentid with the MNDUC promoter, we think that's the significance.
And that's an important component, but from a difference between the.
The what the globe and promotes of <unk>, that's used and BB sort of 5 and the CLO the let T D.
And with the <unk>.
<unk> 3 promote and we think that's the significant difference.
Philip: Okay, thank you. One thing that we do expect and appreciate, and certainly this will come down to an engagement with the agency as well, is that we'll go through this process fairly expeditiously given the risk benefit assessment that people believe around this program to make sure that we can continue on our path toward filing. But we'll provide more updates on that front as we have them. Thank you, Gina. Your next question comes from Jason Gerberry of Bank of America. Your line is now open.
Okay. Thank you.
1 thing that we do expect and appreciate and certainly this will come down to and engagement with the agency as well that will go through this process fairly expeditiously given the risk benefit assessment that people believe around this program to make sure that we can.
Continue on our path towards filing, but we'll provide more updates on that front as we have.
Thank you Gina.
Your next question comes from Jason Youre very of.
Bank of America.
Your line is now open.
Jason Gerberry: Hey guys, good morning. Thanks for taking my questions. Just wanted to come back to the European decision and just curious, how much of this was really predicated on one of your key markets being Southern, based in Southern Europe, for Lennig-Wilburn, at least with Betafowl, and, you know, not a market that you typically can capture great pricing in versus, say, Germany. So wondering if there was anything specific to the market opportunities for Lennig-Wilburn in Europe that sort of drove this decision. Because I imagine, you know, investors would look at a product like Zolgensma, which captured pretty good value as a one-time therapy in Europe, and perhaps maybe that comes down to the availability of different alternatives and prognosis for the patient, ultimately, that can drive value. But just wondering, you know Thanks.
Hey, Hey, guys. Good morning, Thanks for taking my questions.
Just wanted to come back to the European decision and and just curious how much of this is really predicated on 1 of your key markets was southern and southern Europe for line of Goldman and at least the beta Thal and.
Not a market that you typically can capture great pricing and versus say of Germany. So wondering if there was anything specific to the market opportunities for line of Goldman in Europe, the sort of drove this decision because I imagine the investors would look at a product like so again, some of which captured pretty good value.
<unk>.
As the onetime therapy in Europe, and perhaps maybe that comes down to.
Availability of different alternatives and prognosis for the patient ultimately the if that can drive value, but just wondering.
And the post script gets written here on E. Letting go of Ben how some of these factors played into the decision. Thanks.
Andrew: Yeah, so there wasn't. I can take that. This is Andrew. Then, there wasn't one smoking gun from one country, right? There were multiple conversations going on across Europe. And we had gotten far enough in multiple countries to see that the value that we were achieving and record value recognition that we're achieving in those countries was not going to be sufficient overall. So Italy, it wasn't particularly Italy, it wasn't particularly Germany, it wasn't particularly any one, single country.
Yeah. So there wasn't I can take that this is Andrew.
The.
There wasn't 1 smoking gun from 1 country right there was multiple conversations going on across Europe.
And we had gotten far enough in multiple countries to see that the value that we were achieving and record value of recognition. We are achieving and those countries is not going to be sufficient overall, so it'll it wasn't particularly Italy, it wasn't particularly Germany wasn't particularly on the 1.1 country.
Andrew: I think you make a good point about the comparator in terms of what the costs that are being relieved. Unfortunately, European governments have really not recognized the unmet need in thalassemia; some did, some did not. In other cases, they were not willing to provide a premium over the standard of care; in some cases, they were, and in some cases, they were not recognizing the duration of therapy, so it was a mixed bag of issues. So, it is so very difficult to parse out a single one.
So you can make a good point about the comparator in terms of what the costs that are being relieved on unfortunately the.
On the European governments have really and some cases and not recognize the unmet need and.
<unk> some did some of the knot and other cases.
We're not willing to provide a premium over standard of care and some cases and they were and in some cases were not recognized and duration of therapy. So it was a mixed bag of.
Issues, so very difficult to parse out of the single 1 the net result, though was that the price levels that we were seeing were not sufficient for us to move forward and.
Nick: The net result was that the price of the product was [inaudible]. The one thing I'd add to that, which is an important question, is that there is an element of the European system that's frankly broken for situations like this. And so the question is, over time, will this get sorted? I think Andrew, myself, Tom, and others are actually quite optimistic about that. But as we look back on the experience that Andrew just described and what we have at this moment in time, we are not ready to be able to think from a value basis for medicine, Nature.
And 1 of the thing I'd add to that and think it is an important question is that there is an element of the European system Thats frankly broken for situations like this and so the question is over time, we'll just get sort of and I think Andrew and myself, Tom and others are actually quite optimistic on that but as we look back on the experience and Andrew just described and what we have at this moment in time, it's not ready to.
And be able to think from of value basis for of medicine of this nature. So that's unfortunate, but it's the reality of where we live and today and Thats something that we hope to fix over time together with the European system, but at this point, we have to make this move.
Nick: So that's unfortunate, but it's a reality of what we live in today. And that's something that we hope to fix over time together with the European system. But at this point, we had to make this move for the company.
For the company.
Operator: The next question comes from
Got it thank you.
Next question comes from Matthew Luchini of BMO Capital. Your line is now open.
Operator: This call comes from Matti Luchini of BMO Capital. Your line is now open.
Matti Luchini: Hi, good morning. Thank you for taking the question. Um, so on the U.S.
Hi, Good morning, Thank you for taking the questions.
So on the U S for severe genetic disease the U S reimbursement.
Matti Luchini: for severe genetic disease, the U.S. reimbursement.
Matti Luchini: Is this the goal or the company's strategy still aiming for?
And the goal or the company's strategy still aiming for the.
Matti Luchini: Five-year value-based reimbursement. Is that still the strategy, and does your kind of current discussion support that approach, or has the philosophy or the strategy shifted a little bit?
5 year value based reimbursement is that some of the strategy and does your kind of current discussions and support.
Our approach for at this point.
And as a philosophy of the strategy shifted a little bit and now it's really just about kind of get the price paid for even as the 1 time I guess sort of recognizing the fragmented nature of the U S market relative to the consolidated nature of them here.
Matti Luchini: And now it's really just about trying to get the product paid for even as a one-time deal, I guess, you know, sort of recognizing the fragmented nature of the US market relative to the consolidated nature in Europe.
Thomas J. Klima: Great question. Why don't we have Tom, and why don't you comment on that, and Andrew, if you have something to add. Yeah, sure. It's a good question.
The question why don't we have Tom 1 of your comment on that and Andrew If you have something that yeah sure and it's a good question on.
Andrew: Our main priority, as I said, is securing access for patients, and we're looking at all different options to achieve access. And so in the case of payment over time, certainly, if a payer was willing to consider that and adopt that, then we would implement payment over time. So, in short, I think we're going to offer numerous different strategic tactics to secure access, and it will depend on the kind of willingness and the flexibility for each payer and how they adopt them. Yeah, thank you, Tom. I think that's right. We're taking an approach that's trying to make sure we take into consideration what the payers want, and we want to be flexible.
Our main priority as I said is securing access for patients and we're looking at all different options to achieve access and so and the case of payment over time, certainly if the if the payer was willing to consider that and adopt that then we would implement payment overtime.
And so it's short I think we're going to offer numerous different strategic tactics to secure access.
And it will depend on kind of the willingness and the flexibility for for each payer and and how they adopt it.
Okay, Yes. Thank you Tom I think that's right. We're taking an approach that is trying to.
Make sure we take into consideration and what the payers point right and we're willing to be flexible around that those issues.
Matti Luchini: Okay, great. Thanks.
Andrew: And quickly, just on Cal D recognizing that there's the absence of blast cells, sounds like the possible tests that can be done without said blast cells have been done. So how do we think about basically the path to hold resolution from here? It seems like sort of progress is stalled in the absence of, you know, sort of meeting these black cells. So where do we go?
Okay, great. Thanks, and quickly just on count the recognizing that there is the absence of blast cells. It sounds like the possible test the can be done without lifestyles hasn't done so.
How do we think about basically the past couple of the resolution from here it seems like.
Sort of progress of stalled and absence of.
I'm sort of meetings as Blackstone, So where do we go from here.
Got it.
Andrew: Yeah, I wouldn't, I don't think that's how we view it. We have 67 patients, as I said, treated with Elycel. We have extensive durability data in that trial, and I think that data set is sufficient for the agency to understand the risk benefit. As I said, in our view and the view of the experts we've spoken to, the treating physicians, you know, we all view that the risk of an insertional event in CALD is low based on the data that we have and actually a much better outcome than would be obtained without a transplant. We think, based on that data set, we should be able to progress forward to filing the LSL application. Thank you for taking the time to answer.
I don't think that's how we view it.
We have 67 patients as I said treated with Ali cell, we have extensive durability data and that trial I think that data set of sufficient for the agency to understand the risk benefit.
As I said and in our view and the view of the experts we've spoken to the the treating physicians.
And we all view that the risk of it.
Of and insertion of event.
And <unk> is low based on the data that we have and actually a much better outcome.
And then we will be obtained with allo transplant.
We think based on that data set we should be able to progress for 2 filing.
The early cell application.
Thank you for taking the questions.
Operator: The next question comes from Mark Brittenbatch of Oppenheimer. Your line is now open. Hey, good morning, guys. Thanks for taking the time to answer the question. Just wanted to clarify the CALD patient who develops mild dysplastic syndrome is distinct from the one who previously showed evidence of nonmalignant clonal expansion at EBMT last year. And can you just remind us if we've seen any evidence of nonmalignant clonal expansion in any of the sickle cell or thalassemia trials to date? Thank you. I'll pass that over to Rich.
Next question comes from Mark weighted and batch of the hope.
And hymer.
Your line is now open.
Hey, good morning, guys. Thanks for taking the question just wanted to clarify the.
Seattle D patient, who developed Myelodysplastic syndrome is distinct from the 1 who previously showed evidence of non malignant clinical expansion.
At <unk> last year and can you just remind us if we've seen any evidence of non malignant clonal expansion and any of the sickle cell or thalassemia trials.
Good day, thank you.
Pass that over to rich, yes, great. Thank you.
Rich: Yeah, great. Thank you. Yes, this is one of two patients that we've talked about previously who had clonal expansion. There have been no other patients in Tickle Cell or Salve who've had any nonmalignant clonal expansion at all. Thank you. Next question, go.
Yes. This is 1 of 2 patients that we had talked about previously who had kind of expansion there have been no other patients and sickle cell or who've had any non malignant or any clonal expansion of at all.
Thank you.
Operator: The next question comes from Raju Prasad of William Blair. Your line is now open.
Next question comes from Rajeev <unk> of William Blair. Your line is now open.
Raju Prasad: Thanks for taking the question. I just wanted to revisit the vector production for a BECMA. Can you just remind us how it's currently being produced? And is it under an adherent process? And then as we think about the resilience partnership and moving to suspension, I know initially it was to meet the skill cell demand, but maybe talk a little bit about how the suspension process may help support the oncology pipeline as you move forward. Thanks. Yes, you're correct. This is Nick.
Thanks for taking the question.
Just wanted to revisit the vector production for our backlog.
Can you just remind us how it is currently being produced and is it under a adherent process and then as we think about the resilience of <unk>.
<unk> and moving the suspension I know initially it was to meet the sickle cell demand.
But maybe talk a little bit about how the process may help support the oncology pipeline as we move forward.
Yes. So you are correct. This is Nick that is made through and adherent process. At this time and we do plan through the suspension of production process that is at BRT facility that is now over time going to be part of the resilience of network that certainly is the <unk>.
Nick: It is made through an adherent process at this time, and we do plan, through the suspension production process that is at the BRT facility that is now, over time, going to be part of the resilience network. That certainly is a commitment they have, and BMS is very much engaged in that as well. So that's something we're moving to as expeditiously as we can on the vector front, which should help on a bunch of fronts. One is the capacity to also cost of goods, and so forth.
And they have and BMS is very much engaged as that as well. So that's something we're moving to as expeditiously as we can on the vector front, which should help on a bunch of fronts..1 is capacity to also cost of goods and so forth and we plan to through the relationship with resilience over time is as they.
Chip: And we plan to through the relationship with resilience over time as they better utilize the breadth of that facility down there and, most likely, expand it for other clients. That's something that they also would be able to make use of is resilience. And that's certainly something that bluebird, in this context, would participate in. So I don't know, Chip, if you had anything else to add to that. No Nick, I think you hit that well.
Better utilize the breadth of that facility down there and most likely expanded for other clients and that's something that also would be able to make use of is the resilience and thats certainly is something that bluebird and this context would participate and so I don't know of chip. If you had anything else to add on that.
No Nick I think you hit that well I think so.
Operator: I think suspension will be key for the long-term continued ramp of PECMA, and access at BRT will be key for both systems on the pipeline side.
Fashion will be key for the long term continued ramp of the Beckman and.
And access at the party will be key for both businesses on the pipeline side and the next and we've got preferred access there. So that was important part of the structure of that deal.
Operator: The next question comes from Luca Issi of RBC.
Great. Thanks.
Lisa: of RBC. Your line is now open. Thank you.
Next question comes from Luca <unk> of RBC.
Your line is now open.
Philip: Hey, perfect. Thanks for taking the question. This is Lisa on behalf of Luca, just more broadly. Given you now have some direct evidence suggesting LVV insertion caused oncogenesis in Cal D, could we maybe expect to see some more focus on developing therapies with your other pipeline technologies like the Megatel gene editing platform?
Okay perfect. Thanks for taking the question. This is Lisa on for Luka.
And just more broadly.
Given you now have some direct evidence, suggesting lv reinsertion of clause oncogenesis and Kathy.
And could we maybe expect to see some more focus on developing therapies with you on.
The pipeline technologies like the Mega <unk> gene editing platform.
Nick: This is Nick. I'm not sure you can make that connection there, but I'll kick it over to Philip to provide a contextualized answer. Yeah, I mean, the first thing I'd say is that what we've seen is, we believe, restricted to CILD, right? So this is a Lenti-D-LBV insertion, and we think that it's critical that it was the Lenti-D vector involved in this particular case. We don't think that's more broadly applicable, in large part, as we've described, because of vector differences, not just between our own programs, such as TAL and SICKL, but also more broadly in the field.
And.
This is Nick I'm not sure you can make that connection there, but I'll kick it over to Phil and to provide a contextualized answering yes, I mean, it's.
The first thing I'd say is that as the what we've seen is we believe restricted the CIL D. Right. So this is a lengthy D LTV insertion and when you.
I think that's it's critical that it was the launch of D. A.
Victor involved and this particular case.
We don't think that's more broadly applicable and large part, especially as we've described because of the vector of differences between not just between our own.
Our own programs and the talent sickle, but also more broadly on the field.
Nick: In terms of the Megatel technology, yep, we have, obviously, gene editing technology. We're applying that. So we're excited about the application of that. We have, as you know, we have collaborations in vivo with Novo around hemophilia. And we have a T cell product that's heading towards IND as well. So, we are using that technology, and we'll continue to look for opportunities to use that technology and other products in the future.
On.
In terms of the Mega cell technology, yet we have obviously, we have of gene editing technology for applying that and so we're excited about the application of that and we have as you know we have collaborations and vivo with Nova around hemophilia and we have we have a T cell product, that's heading towards and as well so.
So we are using that technology, and we will continue to look for opportunities to use that technology and other products and the future.
Yeah.
The question Lisa and thank you.
Philip: The next question comes from Michael Smith of Guggenheim.
The next.
Next question comes from Michael Smith of Guggenheim. Your line is now open.
Operator: Michael Schmidt of Guggenheim. Your line is now open. Hey guys, thanks for taking my question. I had another one regarding the upcoming business split. Just from a balance sheet perspective, will the current cash balance be split evenly between 270 and legacy Bluebird? Or will it be, you know, a different ratio and perhaps relative to street consensus? I suppose what a back my ramp you have built into this two year cash runway guidance? Thanks so much.
Hey, guys. Thanks for taking my question I had another 1 regarding the upcoming business split just from a balance sheet perspective.
Will the debt.
And cash balance.
Evenly between our 270 and legacy of Bluebird or will it be.
A different ratio and.
And perhaps relative to street consensus for both what our back from a ramp have you built into the 2 year cash runway guidance. Thanks, so much.
Chip: Good question here, Chip. Would you want to jump in on it? Yeah, happy to. In terms of the split, we would expect the cash to be apportioned fairly evenly between the two, because the businesses don't have the exact same burn rate.
Good question here and chip you want of jumping on it.
Happy to.
In terms of the split we would expect the cash to be a portion of fairly evenly between the 2 the businesses don't have the kind of exact same burn rate. So again, the salt is for an equal amount of runway on both sides and again as we look at the math.
Chip: So again, the Salve is for an equal amount of runway on both sides. And again, as we look at the math, we see roughly two years or 24 months of runway for each with a balance of $900 million approximately to be split between them. And yes, PECMA is an important piece of the runway component on the oncology side of the house. Again, $24 million of total U.S. revenue here in the first quarter of commercial launch and the very strong demand that we're seeing.
And we see.
And roughly 2 years of 24 months of runway for each with a balance of $900 million approximately to split between the 2 businesses.
And yes. The <unk> is an important piece of the run rate component on the.
And the college side of the house again $24 million of total U S revenue here and the first.
Quarter of commercial launch and.
And very strong demand that we're seeing so at that debt.
Nick: So that that provides a tail end for 270 and helps support investment, the earlier aspects of the pipeline. So again, we'll provide more detailed guidance with the public filing of our Form 10 and other documents as we move forward. The fourth quarter moment of separation. The only thing I might add there, because I think also, just without being specific on the numbers because I think it's difficult, but we do anticipate this year and next year to be really a capacity game as it relates to BECMA, right?
Provides a tailwind for $2.70, and and help support and investment in a.
The earlier aspects of the pipeline. So again, we will provide more detailed guidance with the public filing of our form 10, and other documents as we move into <unk>.
On the fourth quarter moments of separation of.
The only thing I might add there because I think also just without being specific on the number of existing it's difficult, but we do anticipate this year and next year to be really of capacity game as it relates to our back given the interest there to be able to maximize out and treat as many patients as we can even with the advancement of the other possible approvals and that same timeframe we are.
Nick: Given the interest there to be able to maximize out and treat as many patients as we can, even with the advancements of other possible approvals in that same time frame, we are expecting that to be more about sort of meeting demand as opposed to sharing demand in that regard. I think that's good news.
<unk> that to be more about.
And sort of meeting demand as opposed to sharing demand in that regard. So thats I think of good news and that's also certainly baked into our thinking as we think about the forecast and how to best and most appropriately launch both businesses with the right runway.
Chip: That's also certainly baked into our thinking as we think about the forecast and how to best and most appropriately launch both businesses with the right runway. Greg, thanks. Your next question comes from Yanan Zhu of Wells Fargo. Your line is now open.
Greg Thanks.
Good.
Your next question comes from Yanan Zhu of Wells Fargo. Your line is now open.
Yanan Zhu: Thanks for taking my questions. So I have a question on the LentD and MDS cases and read through to other programs. I think I heard Philip mention that the integration site is the Mecum locus, and I also think there might seem to be some gene dysregulation at this locus due to the vector and probably the promoter. So what gene is affected and what percentage of the clones or overall clones is due to it happening at the MECOM locus?
Hi, Thanks for taking my question.
So I have a question on the land and 90 D and M.
M D S case and read through to the other programs I think I've heard of Philip promotion that the integration site is the need from Lucas.
And I also think there.
Might seem to be something of that dysregulation.
And this regulation at this low cost due to the vector.
And perhaps the probably the promoter.
So for what Jean is.
<unk> and what percentage of the clones or overall crohn's.
Is the due to it happened at the let me come low cost and more importantly for the landscape for.
Yanan Zhu: And more importantly, for the lentiglobin program, what is the rate of integration into this locus? And could there be any chance that integration alone could disrupt the gene? I'll pass this over to Philip here in a second.
For the Atlantic Globe and.
Program, what is the rate of the integration into this.
Lucas.
And could there be any chance that the integration of alone could disrupt the gene that's the strength related.
So I'll pass it over to Philip here on our second the 1 thing I'll just say on the on the regulatory side of the equation is that this is a hold on the E. L. D program and that's an intentional decision made by the regulators and so forth much like in the prior situation. We have fairly recently it was hold specifically on the balance sickle program not on the <unk> program. So it's the.
Philip: The one thing I'll just say on the regulatory side of the equation is that this is a hold on the ALD program and that that's an intentional decision made by the regulators and so forth. But much like in the prior situation we had fairly recently, it was a hold specifically on the Thal and Sickle program, not on the ALD program. So the read-through from a scientific and medical point of view and a regulatory point of view is very clear.
Read through from a scientific and medical point of view on the regulatory point of view is very clear there is no read through and Thats how were planning and that's why we believe we are moving forward with all the other programs and actually not just us but also the field and that's the ecosystem has evolved I think and a very healthy way to understand the nuances of how vectors are different so with that as context of 1 of my of pass it over the.
Philip: There is no read-through, and that's how we plan, and that's why we believe we're moving forward with all the other programs and actually, not just us, but also the field. And that the ecosystem has evolved, I think, in a very healthy way to understand the nuances of how vectors are different. So with that as context, why don't I pass it? Yeah, it's a good question.
For all of them.
Yes, it's a good question.
The.
And the CIL D.
Boy that we've been analyzing this is.
Philip: The In the CLD buoy that we've been analyzing, this is, The integration of the MECOM locus, at least at one point, reached our predominant clone criteria, which is 50% or so. And as I've said, we don't have blasts or tumor cells to analyze, so the proxy has been the peripheral blood. And in peripheral blood, we've seen a misregulation of the MECOM locus. What that means is an increase in the expression of a gene called ED1.
The integration of the <unk> locus.
And.
The 1 point and reached our predominant clone the criteria, which is 50%.
Or so.
And as I've said, we don't have blasts or tumor cells to analyze though of the proxy has been the peripheral blood and the peripheral blood we've seen the misread relation of the Metcom Lucas what that means is an increase and the expression of a gene called E D..1.
Philip: So that's the gene that gets misregulated. Integrations into MECOM are quite common in the ALD program, and they're quite common in general, and we see this across all antiviral therapies, independent of the indication. And that's why it's important to recognize that the design of the vector is critical in what happens after that integration. So, but, as we've described in the ALD case, we have a MECOM integration that's become a dominant clone, and as Rich said earlier, in the context of Sal and Sickle, we don't see dominant clones in those studies at all.
So that's the that's the gene that gets the mis regulated.
On.
Integrations and to me come are quite common and the ILD program and the quite common and general and we see this across all of anti viral therapies.
Independent of of of the.
And indication.
And that's why it is important to recognize that the design of the vector is critical and what happens after that integration.
So.
But and.
As we've described and the <unk>.
<unk> case, we have we have on Mi com integration has become a dominant clio and and as rich said earlier and.
And the context of file and sickle, we don't see dominant claims.
And those studies at all.
Philip: Got it, thank you. So with that, I think we are going to close. We certainly anticipate speaking more with you, and we're excited to continue to keep you updated as we lean towards the launch of these two businesses, both Bluebird and 270 here. And very importantly, the substance behind it, which is we have the US filings as well as the oncology INDs and others that we look forward to sharing with you in more detail as these companies prepare themselves by early in the fourth quarter. So with that, thank you for your time, and we'll speak with you soon again.
Got it thank you.
So with that I think we are going to close and we certainly anticipate speaking more with you and we're excited to continue to keep you updated as we lean towards the launch of these 2 businesses, both Bluebird and 270 here and very importantly, the substance behind it which is we got the U S filings as well as the oncology <unk> and others that we look forward to.
Sharing with you in more detail as these companies prepare themselves by early.
The fourth quarter, so with that thank you for your time and we'll speak with you soon again.
Operator: This concludes today's conference call. Thank you for participating. You may now disconnect.
This concludes today's conference call. Thank you for participating you may now disconnect.
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And then.
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