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Orchard Therapeutics plc (ORTX) Q2 2019 Earnings Call Transcript

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Orchard Therapeutics, Plc. (NASDAQ: ORTX)
Q2 2019 Earnings Call
Aug. 8, 2019, 8:00 a.m. ET

Contents:

  • Prepared Remarks
  • Questions and Answers
  • Call Participants

Prepared Remarks:

Operator

Good day, ladies and gentlemen, and welcome to the Orchard Therapeutics Second Quarter 2019 Earnings Conference Call. At this time, all participants are in a listen-only mode. Later, there will be a question and answer session and instructions will follow at that time. If you require any assistance during today's call, please press * then 0 on your touchtone telephone. As a reminder, this conference call is being recorded. I would now like to turn the conference over to Renee Leck, Director of Investor Relations. Ma'am, you may begin.

Renee Leck -- Director of Investor Relations

Thanks, operator. Good morning, everyone, and welcome to Orchard's Second Quarter 2019 Investor Update. You can access slides for today's call by going to the Investor's Sections of our website, Orchard-TX.com.

Before we get started, I'd like to remind everyone that statements that we make on this call will include forward-looking statements. Actual events and results could differ materially from those expressed or implied by any forward-looking statements as a result of various risk factors and uncertainties, including those set forth in the most recent form 20F, filed with the SEC and any other filings that we may make. In addition, any forward-looking statements made on this call represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update or revise any forward-looking statements.

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And with that, I'll turn the call over to our President and CEO, Mark Rothera.

Mark Rothera -- President and Chief Executive Officer

Thanks, Renee. Welcome, and I hope everyone is enjoying their summer. After a review of our recent activities, we'll be using today's call to spend some time on our programs in Wiskott-Aldrich Syndrome, or WAS, and X-Linked Chronic Granulomatous Disease, or X-CGD. These are two primary immune deficiencies that follow our lead programs in ADA-SCID and metachromatic leukodystrophy, or MLD.

Before we get started, I'd like to spend a few minutes reminding you about Orchard's vision and our use of hematopoietic stem cell, or HSC, gene therapy as our scientific platform. We are a pure-play gene therapy company with a focused approach and a singular vision to deliver potentially curative therapies to patients suffering from life-threatening, rare, genetic diseases around the world. Our expertise lies in developing ex-vivo autologous HSC gene therapies, a platform approach that has demonstrated proof of concept in five different diseases across three physiological systems.

To expand upon the curative potential of our therapies, once sustained engraftment of gene-modified cells and therapeutic gene expression are achieved, we see the potential for lifelong benefit in children treated with our therapies. We are well on our way toward delivering on this vision with two regulatory filings for ADA-SCID and MLD on track for the first half of next year.

To remind you, our programs cover three franchises: neurometabolic disorders, primary immune deficiencies, and hemoglobinopathies. With over 150 patients treated across our portfolio and follow-ups showing durability of response going out to eight years or more, we believe we have one of the most robust and comprehensive clinical data sets in the field of gene therapy.

Now, turning to our recent news, last week, we were proud to announce that the FDA granted RMAT designation for our third registrational program in WAS, which is on track for both FDA and EMA filings in 2021. RMAT designation enables us to have a close and frequent dialogue with the FDA and the opportunity for an expedited review process for this program. This designation also recognizes the unmet medical need and the strength of the clinical evidence we've generated in this disease, so far.

Within the neurometabolic franchise, we continue to build our momentum from our news in May announcing the end-licensing of a clinical-stage MPS-1 program from our partners at Ospedale San Raffaele and Tiget. We are pleased to announce that two abstracts from our programs in MPS-1 and in MLD have been accepted for oral presentations at the Society for the Study of Inborn Errors of Metabolism Meeting, known as SSIEM, in early September.

For MPS-1, Tiget will be presenting updated results from the ongoing proof-of-concept trial. Investigators are continuing to identify and treat patients and enrollment in this program is on track to complete in the first half of 2020.

For MLD, the presentation will go beyond the pivotal data set presented in March to cover an integrated set of results from the 20-patient registrational trial, as well as mined patients from expanded access programs.

The clinical results for 02L-200, which extend out to eight years, have shown the cessation of neurodegeneration in patients treated early enough in the progression of their disease. This is a condition, to remind you, that's characterized by a devastating loss of motor and cognitive function, resulting in death often by the age of five in the most severe form. Our goal is to get this therapy approved for patients around the world as rapidly as possible. And our initial regulatory filing is on track in Europe for the first half of 2020.

Now, preparing to commercialize on a global scale is crucial to the success of our potential launches in MLD, ADA-SCID, and our future gene therapies. This work is well under way with operations established in North America and a growing presence in Europe where we have set up commercial leadership and are in the process of establishing Orchard in France, Germany, and Italy. Within the next few years, we also plan to expand into Latin America, Turkey, the Middle East, and Asia.

We have also made key hires in such global functions as market access, diagnostics, and patient advocacy, to promote knowledge of and access to our therapies. Some of our gene therapies represent the first potential treatments for the diseases they are targeting. Therefore, we see new impetus in patient communities and with healthcare professionals to ensure that effective diagnostic pathways for these diseases are in place. We are also establishing treatment centers in the first countries where we expect to launch and are putting in place a well-controlled supply chain.

So alluding to the earlier stage pipeline, beyond our lead indications, we have a deep pipeline that includes clinical-stage programs in X-CGD and transfusion-dependent beta-thalassemia, which Bates recently achieved proof-of-concept. We are now turning our attention to the work of designing registrational trials for these programs, engaging with regulators and undertaking commercially focused CMC process development work.

So 2019 has been a year of tremendous execution and I am really proud of the team as we continue to deliver on our strategy and remaining milestones for the year.

For the rest of our time together, we'll use the following agenda. Bobby Gaspar, our Chief Scientific Officer, will spend some time on manufacturing, in addition to WAS and S-CGD. Frank Thomas, our Chief Financial Officer and Chief Business Officer, will then review our second-quarter financial results and upcoming milestones. I'll return to close with some commentary on how we are thinking about value in relation to our investigational gene therapy. So Bobby, over to you.

Bobby Gaspar -- Chief Scientific Officer

Thank you, Mark. We're very excited about our lead programs in ADA-SCID and MLD that are approaching their initial regulatory filings in the first half of next year and is the result of a tremendous amount of work by our team.

However, today, I would like to focus the majority of my time on WAS and X-CGD, two primary immunodeficiencies where we have previously spent a little less time, but where we are equally excited by the opportunity to really benefit patients. I also want to touch on the topic of manufacturing, which we see as a very important area as we plan for our investigational gene therapies to move into the commercial arena.

So let's start with manufacturing. Our three most advanced programs, MLD, ADA-SCID, and WAS will use the CMO network in place for the ongoing clinical trials for commercial manufacturing, as well. Oxford BioMedica handles vector manufacturing and Lonza, the drug product manufacturing for ADA-SCID. For MLD and WAS, Molmed manages both of these functions.

Looking ahead to programs such as X-CGD and transfusion-dependent beta-thalassemia, the ability to optimize the manufacturing process will help reduce the cost of goods and improve drug product characteristics and consistency. We are making good progress in this space with ongoing work using the transduction enhancers for the manufacturer of the drug product. This was exemplified by our agreement with SIRION late last year to license the use of LentiBOOST technology for certain programs. We are also looking at improving the way we make lentiviral vectors and we are automating many different aspects of the transduction process.

I'm proud to note that at Orchard, we have been expanding our research and discovery efforts to support these innovations with the goal of implementing these developments and future trials and programs. We are also planning the buildout of our Fremont facility in California, which is intended for both vector and drug product manufacture. Design work is well under way and we remain on track to open in 2021.

All of this highlights our commitment to develop optimal processes for our products so they can be manufactured at scale and serve global patient populations across multiple diseases. I look forward to discussing these developments with you in greater detail at a future time point.

Now, to talk about Wiskott-Aldreth Syndrome. I now want to provide some additional information on the recent press release we issued about the RMAT designation, which we received for OTL-103 for the treatment of WAS. In addition to facilitating a close dialogue with the FDA, this designation recognizes the life-threatening nature of WAS, a severe immunodeficiency and bleeding disorder, as well as the existing unmet need for this population. The current standard of care is an allogeneic hematopoietic stem cell transplant, which can carry risks of mortality and significant morbidity, such as graft versus host disease. The RMAT designation also indicates the clinical evidence generated of OTL-103, so far, has the potential to address these needs.

WAS presents with severe platelet and immune system abnormalities. So the two major clinical aspects of the disease, and consequently the highest cause of mortality are severe bleeding episodes and severe infections. All eight patients treated in the registrational trial for OTL-103 are alive and are showing significant clinical improvements in these areas. We measured the frequency of bleeding episodes and severe infections pre- and post-treatments and patients treated with OTL-103 had no life-threatening severe bleeding episodes due to an increase in their platelet counts. This is in patients with up to eight years of follow-up.

The patients also showed a significant reduction in the frequency of severe infections with negligible episode post-gene therapy. This is related to an improvement in immune function, including the correction of lymphocyte proliferation, immunoglobulin production, and improved vaccine-specific responses. We will be reporting the three-year primary end-point data on all patients in this trial by the end of the year.

In line with our commercial strategy, we have initiated a cryopreserved formulation study in this indication, which is on track to enroll six patients by the first half of 2020. In summary, the OTL-103 study is moving forward extremely well. Molmed, who manages vector and drug product manufacturing for the cryo study will also be the commercial manufacturer for this product, without the need for additional bridging studies.

The RMAT designation gives us a real opportunity to engage with the FDA and submit the BLA application consistent with our 2021 guidance. We also plan to submit an MMA application in Europe within this timeframe.

I now want to turn to the design of our registrational study in x-linked chronic granulomatous disease (X-CGD), an indication where we demonstrated proof-of-concept at the end of last year. This was shown in a clinical trial where six out of seven evaluable patients had greater than 10% functional neutrophils for 12 months or more, leading to clinical improvements such as decreased antibiotic usage, hospitalizations, and infections. Best results to date have been seen in late adolescence and adults. There are many individuals living with CGD who have a lifetime of chronic infections and as a result, have sustained damage to their lungs and liver. These patients, in turn, become high-risk candidates for an allogeneic transplant, which is the current standard of care. Indeed, all six, all the patients who benefited from OTL-102 fit the description.

We are designing the upcoming registrational study with all of this in mind and will likely focus on late adolescent and adult patients, initially. This coincides with the highest proportion of the large prevalent population. Registry data, which is often an underestimate, suggests there are well over 2,500 patients in the US, EU, and Japan. Overall, we estimate there as many as 6,500 patients living with this disease in countries that reimburse orphan drugs, based on published data.

As I mentioned in my previous section, we plan to introduce improvements in the manufacturing process for OTL-102, including the use of transduction enhancers and the use of automated processes. The use of transduction enhancers, especially for older patients, will all us to use less vector per patient, while consistently achieving target VCNs, which will be an important factor in this disease.

The next step is to discuss the planned registrational trial design with the FDA and obtain their guidance. Once we have everything in place, including the final commercial manufacturing process, we will be able to initiate the registrational study. The teams are working hard across our deep pipeline of exciting programs and I look forward to updating you as the year carries on. Frank, over to you.

Frank Thomas -- Chief Financial Officer and Chief Business Officer

Thanks, Bobby. You can find the full financial results for the second quarter in this morning's press release, which I will briefly highlight here. We ended the second quarter with more than $420 million after the recent follow-on offering and credit facility. This puts us in a very strong financial position to execute on the many important milestones we have upcoming without the need to access the capital markets. With three potential filings in the US and Europe, the cash on the balance sheet will allow us to accelerate our transition to accompany commercializing multiple products around the globe. We expect that our existing cash and investments will fund our anticipated operating and capital expenditures into the second half of 2021.

For the second quarter of 2019, R&D expenses were approximately $40 million, compared to $151 million in the same period in 2018. Last year's R&D expenses included a one-time charge associated with the GSK agreement, which we signed in April 2018, totaling about $134 million. So excluding this charge, research and development expenses would have increased $23 million compared to the same period in 2018. This increase was primarily driven by the upfront consideration for the license of our new clinical-stage MPS-1 program, as well as higher program and personnel-related costs from a larger portfolio compared to last year.

SGNA expenses were $14 million for the second quarter of 2019 compared to $7 million in the same period last year. The increase was primarily due to higher personnel costs to support public company operations, as well as costs to prepare for the potential commercialization of our three late-stage development programs. We used about $73 million of cash to fund operations in the first half of 2019, which included a significant pay-down of 2018 accrued expenses, some of which related to deferred payments on the GSK transaction for inventory and transition services.

I'll end my prepared remarks with a summary of our remaining 2019 milestones. We've released two of three registrational data sets for our lead programs. This positions us well for the initiation of the rolling BLA for ADA-SCID in the US and the submission of an MAA for MLD in Europe in the first half of 2020. Later this year, we will be releasing data from patients who received the cryopreserved formulations of these therapies, providing us with additional supportive clinical evidence for these filings. We're also on track to present a third registrational data set for WAS by the end of the year. In our X-CGD program, we have made substantial progress on the design of the registrational trial for OTL-102. In terms of next steps, we will walk down our proposed protocol, meet with regulators to discuss the registrational trial design and complete the remaining CMC work.

For OTL-203, our newest clinical program treating patients with MPS-1, enrollment is ongoing in a proof-of-concept trial and we have additional data releases planned for later this year and next. Finally, the submission of a CTA and initiation of a clinical trial for MPS-3A in Europe remains on track for this year, highlighting the potential for a seventh clinical-stage program in our portfolio.

So we believe the second half of 2019 offers a number of important milestones across our programs that will continue to build on the already exciting clinical data generated to date. Let me now hand the call back to Mark.

Mark Rothera -- President and Chief Executive Officer

Thanks, Frank. As you can tell, we're excited about the clinical and regulatory progress we are making across the business. And now that we're approaching commercialization, we have given great thought to how we characterize the value of our therapies. Ex vivo autologous hematopoietic stem cell gene therapy is fundamentally different from anything we have previously experienced in medicine. Instead of chronic treatments that address symptoms or slow disease progression, we are developing therapies that have the potential to correct the underlying genetic defect of some terrible, life-threatening pediatric diseases. When sustained engraftment has been achieved with adequate gene expression, we are potentially collapsing a lifetime of benefit into a single intervention.

So when we think about value, we begin by thinking about the child and what our therapies, if approved, could do, especially given the sobering reality that today 30% of children living with a rare disease do not live to see their fifth birthday. So let's start with the child. Today, a child with one of these conditions will miss many birthdays, holidays, and other life milestones due to frequent hospitalization and other interventions.

And for the children we are seeking to treat, their lifespans are severely limited. On the other hand, a child treated with gene therapy could have the opportunity to live a completely different life, a life potentially free from the symptoms and related suffering caused by disease. But it's about more than the child. Think about the effects on the family, as well. The financial toll that comes from caring for a very ill child is quite real. Parents may have to quit or scale back their employment. And it takes an emotional toll, as well, with great stress placed on family relationships. As any parent knows, particularly a parent who has cared for a child with a serious disease, the opportunity to transform the life of a child creates the opportunity to also transform the lives of everyone in that family.

Now, think of the community. Our schools, civic organizations, and employers, they all step in to support the family as they care for a sick child. This requires significant infrastructure, including people and equipment within homes and schools. Making a child better means that everyone around that child is then able to contribute to the community in other ways.

Let's also think about the healthcare system. Specialist physicians and other healthcare providers now have the opportunity to deliver a potentially curative therapy, where previously, they would have had few or no treatment options for their patients. In some cases, the healthcare system would also save vast resources that otherwise may have been spent on chronic and palliative care. Costly interventions such as ER visits, long-term hospitalizations, and surgical procedures will no longer be required.

And finally, think of society. Each of us within broader society benefits, no matter who we are. We would all be better off living in a world where dreaded diseases that impact children could potentially be stopped in their tracks.

So that's how we are considering value, as it branches out to touch the lives of not just the child, and the family, but also the community, the healthcare system, and society. We recognize our responsibility and are committed to working in partnership with others to help ensure a sustainable path forward for potentially curative gene therapies. And we are undertaking the work necessary to evaluate all of our programs in this light.

Thank you for your time and attention today. Operator, you may now open the line for questions.

Questions and Answers:

Operator

Thank you. Ladies and gentlemen, if you wish to ask a question at this time, please press * then 1 on your touchtone telephone. If your question has been answered or you wish to remove yourself from the queue, please press the # key. To prevent any background noise, we ask that you please press your line on mute once your question has been stated.

Our first question comes from Graig Suvannavejh with Goldman Sachs. Your line is open.

Graig Suvannavejh -- Goldman Sachs -- Analyst

Great, thank you. Good afternoon or good morning to you guys, wherever you are. Congrats on the progress on the quarter. I've just got two questions, please. The first is on your newest pipeline opportunity, OTL-203. I know that you'll have data coming up in early September, but could you, perhaps, frame for us what kind of data we should expect to see at that conference? I probably won't be able to attend. And then when might you expect that next key set of critical data for that program? And then the second question really has to do with the pricing environment. There has been a lot that's been written about Zolgensma and some of the challenges they're seeing given the pricing that they've established for that product. I just wanted to know as we continue down this path of having exciting new gene therapies on the market, how you are thinking around what you're seeing with competitors and their pricing, how that might impact or not your thinking. Thank you.

Mark Rothera -- President and Chief Executive Officer

Graig, thank you very much for those two questions. So I will begin, then, with your MPS-1 question and maybe, Bobby, you could speak to the SSIEM forthcoming abstract.

Bobby Gaspar -- Chief Scientific Officer

Yeah, thanks Mark and thanks for your question, Graig. The presentation at the SSIEM meeting will really be, primarily, on longer follow-up on the patients that have been recruited into this MPS-1 study. And if you remember, we presented data at ASGCT earlier this year. That was on four patients and in patients who had sufficient follow-up, we were able to demonstrate super-normal expression of the IDUA enzyme and also significant reduction in the accumulation of substrates, both in the periphery and also in the CSF, as well. And given that we know that by chemical correction well with clinical outcome, this is obviously very exciting and encouraging data.

And at the meeting in September, we will be presenting further and longer follow-up with more data on those patients.

Mark Rothera -- President and Chief Executive Officer

And Graig, maybe, I could then move on to the second question about the pricing environment. As you know, we have two filings coming up in the first half of next year and so thereafter we're looking at launching those two first gene therapy programs from our pipeline. It's clearly too early to be guiding you to our specific pricing philosophy or specific guidance on pricing, but we do feel that it's very important to initiate the conversation around the value or benefit of these one-off potentially curative therapies, which as I addressed in the earlier remarks, can have an enormous impact, not just on the child, but on the families and society, and the healthcare system. And we can go into that in more detail, but we think that right now, it's very important to bring to life, if you like, the value of these, or the benefit of these medicines, which could be lifelong but collapsed into one single administration.

So I think that's our focus today. And in time, as we articulate the value for each of our therapies, we'll be working also with the stakeholders, with payers, others in the healthcare system, to talk about how we pay for these. And I think there we can be very flexible and think about what's the best methodology working with them in partnership. But it starts with really what is the value of being able to so dramatically change the course of the child's life? To give them their potential, their full potential, to think about eliminating substantial healthcare resources through not needing chronic care, hospitalizations, ER visits. We're also thinking about surgical interventions. So there are many, many aspects to this that we're going to work on for each and every one of our programs.

Graig Suvannavejh -- Goldman Sachs -- Analyst

Thank you very much and good luck.

Mark Rothera -- President and Chief Executive Officer

Thank you, Graig.

Operator

Our next question comes from Anupam Rama with JP Morgan. Your line is open.

Matt -- JP Morgan -- Analyst

Hey, good morning, guys. Thanks so much for taking our questions. This is Matt on for Anupam. So just a couple from us. First, on 201 and MPS-3A, I think last quarter you guys highlighted some three-month data from one patient that your colleagues at Manchester had treated. So just wondering if any additional follow-up has been made available to you, and if so, how does that look? Secondly, just at a higher level, and not to get too far ahead of myself, but beyond the slate of varied diseases that your team is currently pursuing, what is your appetite for expanding into some broader indications? It's something we're starting to see a little bit more in gene therapy, so just curious to get your thoughts on that. Thanks so much.

Mark Rothera -- President and Chief Executive Officer

Great, thank you very much for those questions. Maybe I'll hand it over to Bobby in a second on the MPS-3A question. But just to remind you, we're really excited about the whole neurometabolic field because, as you know, we've generated substantial clinical evidence going out to eight years in MLD demonstrating that hematopoietic stem cell gene therapy can address a neurodegenerative condition. We're also encouraged by the data we're beginning to see coming out of the MPS-1 program. And so to have a third program going into the clinic by the end of this year, I think, is a really great place to be. Bobby, I don't know if you want to add any points around that specific patient that you were mentioning in the question.

Bobby Gaspar -- Chief Scientific Officer

Yes, so if you remember, [inaudible], that was a patient who was treated outside of the formal clinical trial child who had no other treatment options and then was, therefore, treated at Manchester under a special license, which is available for treatment to the patients in the UK. So that child underwent the gene therapy procedure and actually went through a process that will be used in the upcoming clinical trial, but the encouraging data at three months showed super-normal expression of the enzyme and engraftment of gene-modified cells. So that was at three months. And I know that the child will be coming up for further follow-up soon, but we just haven't had any data shared with us as far as that is concerned, but clearly, that is something that the physicians in Manchester will do and we look forward to communicating with them about that. And they may share that more publicly. But as I say, we'll wait and see as far as that is concerned.

And just to remind you, that the CCA for the formal trial has been submitted and we're looking forward to opening that trial and starting to enroll patients later this year. So we'll look forward to updating you as far as that is concerned.

Mark Rothera -- President and Chief Executive Officer

Now, to the other question you asked about the application of ex vivo hematopoietic stem cell gene therapy to broader indications. Let me just first start by saying we're in a remarkable position, by the end of this year, we're going to have seven programs in the clinic for a variety of rare disease indications, many of which have little or no treatment options today. And as you've seen, we're showing some really remarkable data and potential for this gene therapy approach in these indications. And we have a significant pre-clinical pipeline that goes beyond that. And so we really do see a sustainable and significant business model in building out the rare disease arena and launching these medicines around the world and continuing to bring through more indications for patients with rare diseases.

Now, could this platform also be applied to broader indications? I think the answer is yes. And there will be, I suspect at some point, a view as to whether we should go in that direction. It's too early to speculate around that today. But if and when we do move in that direction, we'll be letting people know.

Matt -- JP Morgan -- Analyst

Great. Thanks so much, guys and congrats on some really solid progress.

Mark Rothera -- President and Chief Executive Officer

Thank you.

Operator

Our next question comes from David Nierengarten with Wedbush Securities. Your line is open.

David Nierengarten -- Wedbush Securities -- Analyst

Thanks for taking the question. I just had one on X-CGD. When you think about talking to the regulators and a clinical trial design, I guess, what would your ideal trial look like in terms of endpoints, duration, number of patients, to set some dialogue? Thanks.

Mark Rothera -- President and Chief Executive Officer

Sure, thank you, David, for the question. Clearly, we're excited by our development in this indication. It's an area of high unmet need. It's a relatively large population that's living with X-CGD around the world. Perhaps, Bobby, would you like to just share some of our thinking as we look at the design and the execution of this trial?

Bobby Gaspar -- Chief Scientific Officer

Sure. Thanks, Mark, and thanks, David, for your question. So as we think about the registrational study in CGD, we really want to start that study in the population where we've seen the strongest efficacy. And that has actually been in the late adolescent and adult population. So if I remind you of the strong proof-of-concept data that we've previously shared, in six out of seven evaluable patients there was greater than 10% functional neutrophils sustained for a year or later.

So long-term reconstitution oddly on the line defect as a result of that one-off gene therapy. Now, in fact, all of the patients that benefited -- those six patients were all in that late adolescent and adult population. So it makes sense to start the registrational study there.

Now, in addition, that is where there is the largest proportion of the large prevalent population that we've talked about. So we've estimated anything up to 6,500 patients. So that is a large population. There is an unmet need there. There are patients there that have a lifetime of living with CGD with chronic lung disease or liver disease as a result of recurrent infections and they're often poor candidates for an allogeneic transplant, which is the current standard of care.

So that is the population that would be best addressed by the registrational study. And of course, as far as endpoints are concerned, we'll be looking at not just by chemical endpoints, but also clinically meaningful endpoints and we're confirming that. Once we have the design in place, we'll go to the regulators to talk about the design of the study.

David Nierengarten -- Wedbush Securities -- Analyst

Maybe a quick follow-up. We've seen in multiple instances where, obviously, in grafts and in transplants, are a little bit better tolerated by younger patients. Would there be any plans to expand to a younger patient population, understanding that the prevalent population is the late adolescent and young adult?

Bobby Gaspar -- Chief Scientific Officer

Sure, so to again recap, the number of patients who are evaluable in the younger age group is very limited in the study, so far. We had one patient with a 12-month follow-up that had poor engraftment for reasons that we've talked about previously as to why that might be the case. But there's no biological reason why this treatment shouldn't work in this condition, we just haven't been able to evaluate enough patients to be able to do that. And of course, what we'll do is to look at that pediatric population in more detail, evaluate further patients, and hope we will be able to move into that at a later stage.

David Nierengarten -- Wedbush Securities -- Analyst

Okay, thank you.

Operator

Thank you and I'm showing no further questions at this time. I would like to turn the call back over Mark Rothera for closing remarks.

Mark Rothera -- President and Chief Executive Officer

Thank you all for joining the call today. As I hope we demonstrated, we're building a great deal of momentum as we gear up for two regulatory filings next year and one the year after, while also delivering our milestones across our rich portfolio of clinical and pre-clinical programs. I'm very proud of what the team has accomplished in this first half of the year. We're making tremendous progress toward our mission of bringing potentially curative therapies to patients around the world. And if approved, we believe that our therapies have the potential to provide a lifetime of benefit to patients through a single administration. We look forward to sharing additional progress in the second half of this year. Thank you all again for joining us and have a great day.

Operator

Ladies and gentlemen, concludes today's conference. Thank you for joining and have a wonderful day.

Duration: 42 minutes

Call participants:

Renee Leck -- Director of Investor Relations

Mark Rothera -- President and Chief Executive Officer

Bobby Gaspar -- Chief Scientific Officer

Frank Thomas -- Chief Financial Officer and Chief Business Officer

Graig Suvannavejh -- Goldman Sachs -- Analyst

Anupam Rama -- JP Morgan -- Analyst

David Nierengarten -- Wedbush Securities -- Analyst

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