Intellia Therapeutics, inc (NTLA) Q2 2021 Earnings Call Transcript

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Intellia Therapeutics, inc (NTLA 1.56%)
Q2 2021 Earnings Call
Aug 6, 2021, 10:00 p.m. ET

Contents:

• Prepared Remarks
• Questions and Answers
• Call Participants

Prepared Remarks:

Operator

Good morning, and welcome to the Intellia Therapeutics Second Quarter 2021 Financial Results Conference Call. [Operator Instructions]

I will now turn the conference over to Ian Karp, Senior Vice President of Investor Relations and Corporate Communications at Intellia. Please proceed.

Ian Karp -- Senior Vice President, Investor Relations And Corporate Communications

Thank you, operator, and good morning, everyone. Welcome to Intellia's Second Quarter 2021 Earnings Call. Earlier this morning, Intellia issued a press release outlining the company's progress this quarter as well as topics for discussion on today's call. This release can be found on the Investors and Media section of Intellia's website at intelliatx.com. This call is being broadcast live, and a replay will be archived on the company's website. At this time, I would like to take a minute to remind listeners that during the call, Intellia management may make certain forward-looking statements and ask that you refer to our SEC filings available at sec.gov for a discussion of potential risks and uncertainties. All information presented on this call is current as of today, and Intellia undertakes no duty to update this information unless required by law.

Joining me on the call today are Dr. John Leonard, Chief Executive Officer; Dr. David Lebwohl, Chief Medical Officer; Dr. Laura Sepp-Lorenzino, Chief Scientific Officer; and Glenn Goddard, Chief Financial Officer. On today's call, John will lead with the company's second quarter and recent business highlights, followed by David, who will provide an update on clinical efforts surrounding NTLA-2001. Lara will then recap the company's R&D progress, followed by Glenn, who will review Intellia's financial results for the quarter. John will then make some closing remarks, and we'll open up the call for Q&A.

With that, let me turn the call over to our Chief Executive Officer. John?

John Leonard -- President And Chief Executive Officer

Thank you, Ian, and thank you all for joining us today. The past few months have been incredibly exciting for both Intellia as well as for patients battling diseases that may be addressable with our genome editing therapies. Most notably, in June, we became the first company to report clinical data for a systemically delivered CRISPR-based therapy in patients. This milestone represents a significant advance in our mission to develop curative genome editing treatments. At Intellia, we've set out to build the broadest and deepest toolbox to fully realize the promise of revolutionary CRISPR-based medicines. To that end, our platform deploys differentiated modular solutions across in vivo and ex vivo therapeutic applications of our Nobel Prize-winning technology. For genetic disease, we're leveraging a lipid nanoparticle or LNP-based delivery system to selectively inactivate disease-causing genes or precisely insertions to produce desired proteins. Our ex vivo approach is designed to produce a homogeneous robust cell product that epitomizes the patient's natural immune system to eliminate cancer cells. Across these efforts, we've made significant progress advancing our full spectrum strategy since last quarter's call.

Our landmark data presented in June, highlighted the interim clinical data from the first two cohorts of the dose escalation portion of our ongoing Phase I trial for NTLA-2001. These data indicate an encouraging safety profile in dose-dependent protein knockdown response. In the second dose level, treatment with NTLA-2001 in the three patients in the cohort led to a mean serum TTR reduction of 87% at day 28 with a maximum 96% production. Notably, these results exceed the current standard of care for ATTR amyloidosis patients with polyneuropathy. At both dose levels, NTLA-2001 was generally well tolerated by the six patients included in the interim analysis with no serious adverse events by day 28. We view this as a profoundly important outcome for patients, for Intellia and for medicine. These positive results not only support NTLA-2001's therapeutic potential as a onetime treatment for ATTR amyloidosis but also offer proof of concept for our nonviral delivery platform. It gives us added confidence to pursue other targets to treat diseases that originate in the liver via reproducible development path. More broadly, we believe these results open a new era in medicine in which curing genetic disease may be within reach. Shortly, David will elaborate more detail on the landmark data and make steps for this program.

Beyond the NTLA-2001 readout, NTLA-2002, our in vivo knockout candidate for hereditary angioedema, or HAE, and NTLA-5001, our lead engineered TCR T-cell therapy for acute myeloid leukemia or AML continue marching toward the clinic. In June, we shared that we submitted our first CTA for NTLA-2002 to regulatory authorities. We're pleased to announce this morning that we have submitted our first CTA for NTLA-5001 as well. Pending regulatory authorization, both first-in-human trials are on track to begin by the end of this year. In addition, together with Cellex Cell Professionals and Blackstone Life Sciences, we launched a new universal CAR-T company. This new company combines Intellia's allogeneic cell engineering platform with a clinically validated universal CAR-T construct from Jamo a. Additionally, this company, which aims to develop differentiated immunooncology and autoimmune therapies is resourced with up to a $250 million financing commitment from Blackstone.

In exchange for a license to our technology, Intellia will receive a significant equity stake of this new company along with two options to co-develop and co-commercialize two allogeneic universal CAR-T candidates in the U.S. and key European countries. Collaborations like this one are a key instrument of our broader corporate strategy to enable us to realize the full scope and potential of our technology for patients that can extend the reach of our technology beyond our core areas of strategic focus while retaining rights across a diverse ex vivo landscape. And this collaboration brings the additional benefit of a concurrent manufacturing agreement with Cellex, which Laura will expand on a bit later. Finally, before passing the call on, I'd like to extend a warm welcome to two new members of our leadership team, Jim Basta, Executive Vice President and General Counsel; and Ian Karp, Senior Vice President of Investor Relations and Corporate Communications. We're thrilled to have them join Intellia. And I have no doubt that their expertise and support will prove to be of great value as our company continues to mature.

I'll now hand over the call to our Chief Medical Officer, David Lebwohl, who will recap our NTLA-2001 data and provide a more in-depth update on our progress in the clinic. David?

David Lebwohl -- Executive Vice President, Chief Medical Officer

Thank you, John. I'll start with a bit of background on ATTR amyloidosis and our approach with 2001. ATTR amyloidosis is a rare, progressive and fatal disease caused by the buildup of TTR protein in multiple organs. People living with the disease can have either the hereditary or wild-type forms, resulting in a diverse range of disease manifestations, the most frequent being polyneuropathy and cardiomyopathy. Our lead candidate 2001 is the first systemically delivered CRISPR-based therapy dosed in a patient and a potentially curative treatment for ATTR amyloidosis. 2001 applies our in vivo LNP delivery technology to knock out the TTR gene in the liver, which is a source of circulating TTR protein therefore, permanently reducing amyloid deposition after a single dose. Although the current standard of care has achieved meaningful therapeutic impact with roughly 80% knockdown of serum TTR, there remains unmet medical need in this population. Moreover, as Dr. Gilmore shared at our June investor event, beyond the benefits of a onetime treatment as demonstrated in ATTR amyloidosis as well as other types of amyloidosis, the radar TTR knockdown is anticipated to improve clinical outcomes.

We therefore believe a single-dose therapy that most importantly produces greater and durable TTR reduction will offer a significant benefit to patients and value to our healthcare system over the current standard of care. The 2021 Peripheral Nerve Society's annual meeting and concurrently published in the New England Journal of Medicine, we shared positive interim data from a Phase I study evaluating 2001 in patients living with hereditary ATTR amyloidosis with polyneuropathy that support this hypothesis. Data representing six patients from the first two dose cohorts of Part one of the study with single ascending dose portion were highly encouraging showing that 201 induced a dose-dependent reduction in serum TTR. In our second dose level, 0.3 milligrams per kilogram, we achieved an average TTR reduction of 87% at day 28, reaching a maximum TTR reduction of 96%. 2001 was generally well tolerated at both doses with adverse events all being Grade one and mostly unrelated to the treatment. Altogether, the data suggests that we can achieve targeted in vivo editing for the desired pharmacological effect supporting 2001 as a first-in-class single-dose treatment for ATTR amyloidosis, validating our LNP technology and unlocking treatment of diseases that originate in the liver. In terms of next steps for this program, we are continuing enrollment in the dose escalation portion of the Phase I study.

We plan to provide additional data from Part one of the study at a scientific or medical conference later this year, including additional durability data beyond 28 days from the initial cohort. For Cohort three, each subject will receive a single dose of 2001 at one milligram per kilogram. For Cohort four, we have flexibility in the protocol to proceed to a higher dose or exploring intermediate dose. Once we identify the recommended dose, we plan to initiate Part two of the trial, a single dose expansion cohort. In this portion of the study, eight patients will be administered the recommended dose identified in Part one with the objective to further characterize the activity of 2001, including an initial assessment on clinical measures of neuropathy and neurologic function. We expect to advance into Part two of the study later this year.

In addition, we plan to engage with regulators, including the FDA to move into later phase clinical trials in which we aim to include both patients with polyneuropathy and cardiomyopathy. While we recognize these are interim Phase I results with much more to learn, observations so far are quite meaningful, offering support that substantially derisks our in vivo efforts. We look forward to keeping you updated on our progress.

With that, I'll now turn over the call to our Chief Scientific Officer, Laura Sepp-Lorenzino, to provide updates on our two additional development candidates 2002 and 5001 and across our R&D efforts.

Laura Sepp-Lorenzino -- Executive Vice President, Chief Scientific Officer

Thanks, David. Given the modularity of our platform, we're equally excited about what the interim chemical data imply for our efforts beyond NTLA-2001. Now that we have demonstrated we can deliver to hepatocyte segments, we believe this accelerates the development of additional in vivo programs and increases the probability of technical success. This applies to several programs and especially to our in vivo not programs, such as NTLA-2002, our wholly owned candidate in development for HAE. NTLA-2002 leverages the same LNP delivery system as NTLA-2001, but targets the KLKB1 gene in the liver to permanently reduce plasma kallikrein protein and thereby effectivity a mediator of disease. This approach is expected to provide continued suppression of calacrine activity and eliminate HAE tanks. In June, alongside results from NTLA-2001, we announced the submission of our first clinical trial application to the New Zealand Medicines and Medical Devices Safety Authority to initiate a first-in-human study for NTLA-2002. We're meeting additional applications for this program and expect to enroll the first patient by year-end.

The first in-human study will evaluate NTLA-2002 for safety, tolerability and activity, including levels of kallikrein in patients with HAE. This program has leveraged insights from NTLA-2001 from its inception throughout its development and on the basis of our preclinical data and clinical results from NTLA-2001. We expect to return in the recommended dose with a very limited number of dosing cohorts. Now turning to our ex-vivo efforts. Here, we're using CRISPR/Cas9 as a tool to create engineered cell therapies. Similar to our efforts in vivo, our proprietary approach to cell engineering serves as a foundation to our modular platform. This approach allows us to mix and match cell type, targeting modality and edits necessary for eliciting the desired pharmacology. Regardless of therapeutic format, we expect to achieve highly efficient sequential editing high yields, optimal cell performance and scalable manufacturing. Our lead program, NTLA-5001 is a potential best-in-class engineered T-cell therapy designed to treat all genetic subtypes of AML. This investigation candidate is an autologous T-cell receptor, or TCR, T-cell therapy targeting the Wilms' Tumor one antigen and restricted to the HLA-0201 allele.

Importantly, WT1 is overexpressed in more than 90% of AML patients regardless of mutation subtype. And HLA-0201 is one of the most common series. Furthermore, NTLA-5001 utilizes our proprietary cell engineering process to replace patients in endogenous TCRs with WT1 targeting TCR, creating a homogeneous product with high intrinsic potency and improved safety by eliminating TCR mispairing. By utilizing our sequential editing process, the resulting for product has the higher attributes of high viability robust levels of expansion, desirable memory phenotype, increased functional activity and reduced translocations. Despite recent therapeutic advances delivering improved response rates in its subsets of AML, long-term outcomes continue to be poor, with overall five-year survival below 30%. With our proprietary cell engineering process and the prevalence of WTI, we believe NTLA-5001 will be a highly active and well-tolerated agent to improve outcomes for AML patients. As John mentioned, today, we're excited to share that we have submitted our first CTA to United Kingdom's Medicines and Healthcare Products Regulatory Agency for a Phase I study of NTLA-5001 in the United Kingdom. This first-in-human trial will evaluate the safety and activity of NTLA-5001 in patients with persistent or recurrent AML who have previously received first-line therapies. We expect to be screening patients for the Phase I study by year-end. Beyond NTLA-5001, we plan to share more information on our allogeneic solution later this year. We believe our approach is differentiated from others currently in the clinic that typically address only graph versus host injection.

We have focused on creating allogeneic cells that not only avoid graft-versus-host disease but also persist by avoiding elimination by host T-cells and NK cells. This approach should avoid the aggressive need for depletion and the reduced performantleficiency current employed to address a significant challenge in adoptive cell therapy. In June, concurrent to launching a new universal CAR-T cell therapy company, we entered into a preferred partnership agreement with Cellex. This agreement provides Intellia access to dedicated GMP manufacturing capacity and high-quality donor cells for our allogeneic products. Our access to both will be of great value in supporting and accelerating the development of our wholly owned ex vivo programs. All the R&D progress along with demonstrations earlier this year, such as preclinical data introducing our proprietary base editor and our first demonstration of in vivo editing outside the liver reflects both the breadth of our technology's reach and our steadfast commitment to drive our pipeline forward through continued platform innovation. To that end, we plan to present additional in vivo and ex vivo update for scientific conferences later this year. We remain on track to nominate at least one new allogeneic candidate this year, and we continue to work toward nominating the company's first allogeneic development candidate by the first half of 2022.

With that, I would like to hand over the call to our Chief Financial Officer, Glenn Goddard, who will provide an overview of our second quarter financial results.

Glenn Goddard -- Executive Vice President, Chief Financial Officer

Thank you, Laura, and good morning. Our cash, cash equivalents and marketable securities were $551.3 million as of June 30, 2021, compared to $597.4 million as of December 31, 2020. The decrease was driven by cash used to fund operations of approximately $115.1 million, which was offset in part by $45.3 million of net proceeds from the company's at-the-market agreement, $20.5 million in proceeds from employee-based stock plans and $3.2 million of funding from cost-sharing agreements with Regeneron. Following the disclosure of our NTLA-2001 clinical data, we completed a successful follow-on offering in July, raising $648.1 million in net proceeds, putting Intellia in a strong position to aggressively advance and expand our pipeline. Our collaboration revenue decreased by $9.7 million to $6.6 million during the second quarter of 2021 compared to $16.3 million during the second quarter of 2020. The decrease was driven by $8.4 million of revenue recognized for a onetime catch-up adjustment related to the amendment and expansion of our 2016 Regeneron collaboration agreement in Q2 of last year.

Our R&D expenses increased by $21.1 million to $58.9 million during the second quarter of 2021 compared to $37.8 million during the second quarter of 2020. This increase was driven by a $10 million onetime payment related to the amendment of our 2014 Novartis agreement as well as employee-related expenses due to the continued expansion of our development organization. Our G&A expenses increased by $5.2 million to $16.7 million during the second quarter of 2021 compared to $11.5 million during the second quarter of 2020. This increase was mainly due to employee-related expenses, including stock-based compensation of $2.1 million. And finally, we expect our current cash balance to fund our operating plans beyond the next 24 months, as Intellia is well positioned to drive long-term growth and execute on our upcoming milestones.

And now I will turn the call back over to John for closing remarks.

John Leonard -- President And Chief Executive Officer

Thank you, Glenn. As you can see, we've made significant progress executing against the ambitious goals and strategic priorities set forth at the beginning of this year. Our presentation of proof-of-concept clinical data from the first ever study of a systemically administered CRISPR genome editing therapy in humans was clearly a highlight. Not only is this data important for the ATTR amyloidosis community, but it helps us advance our growing full-spectrum pipeline. Moreover, this milestone represents just one key staff in unlocking the full therapeutic potential of our CRISPR platform. Following the positive interim data from our Phase I study of NTLA-2001, we look forward to sharing additional interim results into initiating part two of the study, a single dose expansion cohort later this year.

We've continued to progress our pipeline with CTA submissions for our second in vivo candidate, NTLA-2002 and/or lead ex vivo candidate, NTLA-5001 and we anticipate initiating both first in human trials by the end of this year. With validation of our CRISPR/Cas9 platform, we remain on track to nominate at least one new development candidate by the end of the year. Additionally, we plan to share preclinical data on our allogeneic solution this year and expect to nominate the company's first allogeneic development candidate by the first half of next year. And across our R&D efforts, we look forward to sharing additional in vivo and ex vivo updates at are coming scientific and medical conferences. We're eager to carry this momentum forward as our recent progress offers a springboard from which to advance our long-term vision for Intellia.

Operator, you may now open the call for questions.

Questions and Answers:

Operator

[Operator Instructions] Our first question comes from Maury Raycroft from Jefferies. Please go ahead.

Maury Raycroft -- Jefferies -- Analyst

Hi, Good morning everyone. Congrats on the progress. To start off, I was wondering if you can talk more about when you aim to engage with FDA about next step trials? Would that be after the eight-patient expansion data? Or can you have those meetings sooner? And for the eight patient expansion, any new thoughts on how many mixed patients will be included in that? And if you will assess for cardiovascular measures and biomarkers like NT-proBNP?

John Leonard -- President And Chief Executive Officer

Thanks, Maury. Morning. Nice to hear from you. Maybe I'll turn to David, maybe you can just tell us a little bit about your plans for progressing the work.

David Lebwohl -- Executive Vice President, Chief Medical Officer

Thank you, John. Thank you, Maury. So with the data that we have from Phase one so far. We're seeing that we've achieved an 87% reduction in TTR. So this result is, as you know, is better than with available therapy. So in the trial, we think we are getting close to where we need to be with dosing. We will be reporting on that before the end of the year. We are, at the same time, planning on the next studies at this time. We don't think we need any particular additional data to be able to engage with regulators, both the FDA and other regulators around the world about our next plans. In terms of the expansion, what is in the trial as described it is patients with polyneuropathy. We -- if you look at clinicaltrials.gov, we talked about some of the measures. There are measures of cardiac function because, as you know, many of these patients do as well have cardiac involvement, but they don't have heart failure. Their main involvement is neuropathy.

Maury Raycroft -- Jefferies -- Analyst

Got it. That's all helpful. And so it sounds like you could potentially engage with regulators sooner than later to get some additional feedback on what next step trials could look like?

David Lebwohl -- Executive Vice President, Chief Medical Officer

That is a reasonable assumption.

Maury Raycroft -- Jefferies -- Analyst

Okay. And then a follow-up question just on cohort for -- what are gating factors to determine if you decide to assess an intermediate dose at dose level in that cohort? And what could that dose look like?

David Lebwohl -- Executive Vice President, Chief Medical Officer

We're going to look at the data from Cohort three. We mentioned Cohort four, but it's not required within the study. If we think we have the right dose to go forward based on the first three doses. We can do that. But we also do have the flexibility to go to either an intermediate dose that's higher or lower than that one milligram per kilogram. So we'll be looking at all the data that comes in from the third cohort and then decide what to do after that.

Maury Raycroft -- Jefferies -- Analyst

Ok, Thanks john for taking my question. Thank you.

Operator

The next question comes from Salveen Richter from Goldman Sachs. Please go ahead

Sonya Bhatia -- Goldman Sachs -- Analyst

Hi, Thanks for taking my question. This is Sonya on for Salveen. We just had two questions. The first is on NTLA-2001. How should we think about the upcoming data for Cohorts three. Just if you could help us frame expectations there? And then the second one is on the trial design for WT1 AML. Just if you could remind us what that is, again, that would be helpful. Thank you.

John Leonard -- President And Chief Executive Officer

I think I'll turn to David again. David, just say a little bit about our expectations for the end of the year. And I know it's early for WT1 trial designs, but maybe you can just speak to where we are in the regulatory process, just to remind our listeners

David Lebwohl -- Executive Vice President, Chief Medical Officer

Okay. Thank you, John. The -- so what we'll be reporting before the end of the year is additional data from the dose escalation portion of the trial. What you know from our reports so far is that we will have data from patients receiving one milligram per kilogram. And we've also said that we'll be talking more about the durability in the cohort in the existing patients in the trial. Of course, we'll bring forward as much information as we can from the trial and let people know where we are with that. Looking at 5001, we have announced that we've made our first CTA submission or a regulatory submission that is. And with that, we will be talking about the trial design after we are really getting started with the trial, after we have our first approval to move forward in the trial.

Operator

The next question comes from Gena Wang from Barclays.Please go ahead.

Sheldon -- Barclays -- Analyst

Hi, This is Sheldon on for Gena.Thank you for taking my question. We have two. One on 2001. So for the second half update pretty narrowed down a little bit for us, whether third quarter or fourth quarter? And how could you update us also on the enrollment progress.

John Leonard -- President And Chief Executive Officer

Yes. This is John. Thanks, Sheldon. We're really not going to be able to give more specificity other than we'll give you an update by the end of the year. And we will apply the principles that we've been applying throughout the study today. We want those updates to be interpretable, meaningful and consistent and that's been -- what's been guiding us throughout the work. So it's certainly our expectation to share more information, but as we did with the first go around, we'll give people advanced notice on where we're going to do that and how we're going to do that. So stay tuned for that. We're not going to catch anybody by surprise.

Sheldon -- Barclays -- Analyst

Great. Very helpful. My second question is on HAE. So when you share the data, how should we think about the gating factors about when you will be able to share is like to achieve certain follow-up or to achieve certain lockdown level?

John Leonard -- President And Chief Executive Officer

Yes. I think that's premature at this time. I understand what's behind the question. We've begun the regulatory process as we laid out, and we're excited about moving that forward very, very expeditiously. As Laura commented, we think we're in a very good position to benefit from the work that's been done with 5001, and that will find its way into the clinical program. Once we've cleared on the regulatory side, we'll talk about the specifics of the program and the protocol. And at that point, we can start to think ahead about what we'll share and when we'll share it. But I think, as you know, in these first-in-human studies, it's all very much a function of the protocol design and the rate of progression of enrollment that dictates what you can talk about. So stay tuned again. I think you should just expect that we'll do it in a fashion is quite similar to what we did with 2001.

Sheldon -- Barclays -- Analyst

That's helpful. Thank you.

Operator

The next question comes from Joon Lee from Truist Securities. Please go ahead.

Joon Lee -- Truist Securities -- Analyst

Hi, Thanks for taking my question. For the patients in the Cohort one, if you look at the data, they didn't look like they plateaued. Have you collected more data beyond day 28? And if so, as you see a further knockdown in TTR? And related to that, when will you be able to share some clinical base which could be particularly interesting given the completely define PK/PD profile compared to ASO? And I have a follow-up.

John Leonard -- President And Chief Executive Officer

I'll take that. As David said in his comments, we'll provide more information as the study progresses, and we've committed to doing that before the end of this year. We presented the data that we had. It's an ongoing study and what we tried to do is present information that we think is meaningful. And generally speaking, we expect the TTR effects to settle out by day 28. But subsequent work and data collection, I think, will corroborate that, at least based on preclinical work. But with the upcoming update, we'll share the information that we have. So stay tuned. It's an ongoing process. With respect to the clinical information, that's something that we'll collect more robustly in the second stage of the study, although there's some descriptive work in this first phase, which, as you know, will speak to the particular doses that the patients have received. So as we shared with our update a few weeks ago, the Cohort two, we think will -- has very, very meaningful knockdown, but I don't think that presents the drug with the maximal effect that one would hope to see from a clinical point of view. So that better, more robust description really is going to come on stage two.

Joon Lee -- Truist Securities -- Analyst

Got it. And then as you advance 2001 toward commercialization, can you discuss your freedom to operate in the U.S. specifically to commercialize that in the U.S. unencumbered? Or do you foresee a need to negotiate with another company or pay royalty? Is that something James spots that will be tackling? Thank you.

John Leonard -- President And Chief Executive Officer

I think we're in a really good position to commercialize the product. And at this point, anything to share specifically with respect to dealings with other companies.

Joon Lee -- Truist Securities -- Analyst

Thank you.

Operator

The next question comes from Luca Issi from RBC Capital Markets. Please go ahead.

Luca Issi -- RBC Capital Markets -- Analyst

Great, Thank you so much. Congrats on all the progress here. I have a philosophical question on durability. I know gene therapy is an imperfect comp, but we have seen the FDA asking for at least two years durability before considering approval at least for liver. And if you look at the BioMarin data in hemophilia A as a comp, conceptually, do you anticipate the FDA to ask shorter time line for getting given its permanent nature? Or maybe do you expect them to ask you a longer time line given we don't really understand the long-term consequences of potential off-target effects? Thanks, so much.

John Leonard -- President And Chief Executive Officer

That is not only philosophical, but also speculative. And I think look in the spirit of your question, the specifics of what it's going to take for this particular therapy to be approved by regulators or something that will be subjected to ongoing future regulatory studies. I think that in the case of gene therapy in terms of how we view it, the particular approach has inherent tendencies to at least be susceptible to waning over time. And that's, I think, as you know, tied to the notion of having an episome that is not integrated into the genetic material of the cell. It's our expectation, and we've certainly shared a lot of preclinical work to support this that the effects should be, in the case of gene editing, lifelong over the life of the patient. And we have no evidence to the contrary at this point. So we'll see what the FDA is looking for, but I think that certain principles that are analogous to gene therapy are likely to be applied, but a lot of that is just looking for the clinical outcomes that come with it and sufficient information to indicate that the assumptions are correct.

Luca Issi -- RBC Capital Markets -- Analyst

Great, Thanks so much john.

Operator

The next question comes from Mani Foroohar from SVB Leerink. Please go ahead.

Dae Gon -- SVB Leerink -- Analyst

Good morning. This is Dae Gon for Mani.Thanks for taking my question. What is your current thinking around the regulatory path for 2001 in cardiomyopathy and polyneuropathy phenotypes in the context of existing treatment options like stabilizers and silencers in these phenotypes that are becoming standard of care. Are you guys expecting to do non-inferiority studies for one of both phenotypes?

John Leonard -- President And Chief Executive Officer

I'd say at this time, it's far from a foregone conclusion that the cardiomyopathy space, in particular, will be required to do comparative studies. And obviously, we watch the space carefully to learn whatever we can from the progress of other agents. I don't think that most KOLs would agree that there's a standard of care. And certainly, from a knockdown point of view, there's not even an improved product yet for cardiomyopathy. So that basis of thinking, I think, for regulatory studies for us is probably quite premature. So with respect to the polyneuropathy space, that we're thinking through at this point. But where the program stands today is we're especially interested in getting information in the cardiomyopathy patients. And after David selects his optimal biological dose, and we collect that information, then we'll progress that as efficiently as we can.

Dae Gon -- SVB Leerink -- Analyst

Awesome. My second question is that so, so far, we've seen HP data from three different programs, I think ATTR, HAE and AATD, but the tested doses were not disclosed. But in terms of knockout efficiency, are you seeing similar knockout across different genes at similar dose levels? Or are you seeing some meaningful differences and knockout efficiency depending on the gene of interest. And how does this in combination with one clinical data inform both the trial design and knockout expectations for 2002 in HAE?

John Leonard -- President And Chief Executive Officer

Well, one of the benefits of the modular approach is that we use the same lipid shell that contains essentially the same cargo. And by that, I mean, specifically the mRNA that encodes the Cas9 enzyme. And for that matter, the guide RNA is virtually identical with the exception of last 20 nucleotides. What we found is that from a product efficiency point of view in our preclinical models, there is great consistency across all of the different targets that we've pursued. So we're excited that the logic that we employed at the very beginning about modularity appears to be holding true. And I think the first real test of that is going to be as we move to 2002. And ideally, hopefully, corroborate the results that we've already seen with 2001. But our preclinical studies, which have been a very good guide thus far is -- have been very, very helpful in terms of predicting where we go in humans.

Dae Gon -- SVB Leerink -- Analyst

Thanks again for taking my question.

Operator

The next question comes from Jay Olson from Oppenheimer. Please go ahead.

Jay Olson -- Oppenheimer -- Analyst

Thank you for taking the question. Maybe just a follow-up on 2001 in cardiomyopathy. Do you think that the greater TTR knockdown can lead to improved clinical benefits in both cardiomyopathy and polyneuropathy? And then maybe just a quick question on 5001 for AML. Since you're planning to do your early clinical work in the U.K., can you remind us sort of the pros and cons of doing network in the U.K. versus submitting an IND in the U.S.? Thank you.

John Leonard -- President And Chief Executive Officer

Let me speak to the whole knockdown question. Our central thesis, and I think it's a thesis that's broadly held in the amyloidosis field, is that you want to reduce the offending protein to the greatest extent possible. And by that, I mean, to the greatest extent possible. And there's clinical evidence when you look broadly across different proteins that cause different forms of amyloidosis to indicate that that's the case. And there's even existing clinical data for the agents that currently exists that suggests that deeper responses lead to better effects. There's no reason that we can think of and no good reason that we've heard and we certainly discussed this extensively with people who are experts in the field to indicate that, that's going to be any less true for cardiomyopathy than it is for polyneuropathy. And so the goal of our program is to achieve those greatest responses in TTR that we possibly can. And we believe clinical trials then will bear that out. And that is, as I said, the central thesis of the overall program. With respect to 5001 or the TCR program, I would point out that the regulatory submissions are in their early stages, and I would not view this as limited in any way and certainly not limited to the U.K. However, our experience in the U.K., and we anticipate that we'll continue this way has been excellent with standards of care that are essentially indistinguishable from those that would be expected here in the U.S. So from a limitation point of view, we don't see any.

Jay Olson -- Oppenheimer -- Analyst

Great, Thanks for taking the question.

John Leonard -- President And Chief Executive Officer

Sure.

Operator

The next question comes from Steve Seedhouse from Raymond James. Please go ahead.

Ryan Deschner -- Raymond James -- Analyst

This is Ryan Deschner on for Steve Seedhouse. I just wanted to ask, you guys expect to report mNIS+7 data in the ATTR trial by end of year in patients with clinically relevant dose? And then I have a follow-up question.

John Leonard -- President And Chief Executive Officer

David, the question is do you expect any mNIS data from this trial by the end of the year.

David Lebwohl -- Executive Vice President, Chief Medical Officer

We are measuring mNIS as part of the not mNIS+7 is part of the early patients. Of course, this is a dose escalation, so there will be very limited follow-up. So if there were sufficient follow-up to be meaningful, we present it, but we're not sure we'll have that yet.

Ryan Deschner -- Raymond James -- Analyst

Okay. And then also, have you -- do you plan to assess the 2001 propensity for chromates ex vivo? And is there a way to monitor this in vivo?

John Leonard -- President And Chief Executive Officer

This work has been done extensively in the preclinical setting. And that has been characterized and even reported on with respect to any off-target analysis in the New England Journal paper. I'd refer you to the supplement where it's far longer than the paper itself, that speaks to how this work is done, the data that's resulting, the testing at concentrations that far exceed those that are expected to be achieved in the clinic. And from the standpoint of chromotypsus. I don't think the word even applies actually. What we're looking for is any evidence of off targets. And what we find is that there are none at the doses and concentrations that we're administering to patients.

Ryan Deschner -- Raymond James -- Analyst

Ok, Thank you very much.

Operator

The next question comes from Silvan Tuerkcan from JMP. Please go ahead.

Silvan Tuerkcan -- JMP -- Analyst

Good morning and Thanks for taking my question. I have two questions, please. Just on the NTLA-5001, I know you can't tell us much about the trial, but would there be any reason to believe that there will be major differences from the Fred Hunterson trial that included pre- and post-transplant patients. Were there any reason why you would exclude one of them or not? And then I have a follow-up.

John Leonard -- President And Chief Executive Officer

I want to make sure I understand the question. Are you asking from an outcome point of view? Or are you asking about from a design point of view.

Silvan Tuerkcan -- JMP -- Analyst

From a design point of view.

John Leonard -- President And Chief Executive Officer

Yes. I'd say it's still a little early to speak specifically about the design of what we're going to be doing with 5001. I think there are general principles that people broadly apply that you should anticipate in the early studies. But once we get regulatory clearance, we'll go into greater detail as to exactly what we're going to do. We're certainly looking ahead when -- as you referred to the University of Washington, Fred Hutchison results. We look to that as a helpful guide to set some expectations in terms of what might work and some of that logic supports our thinking about WT1. But there's differences between our particular approach and the one that they applied.

Silvan Tuerkcan -- JMP -- Analyst

Great. And maybe if you can speak to the high-level strategy for the new company that you've created with GMP and Blackstone. Do you think these new CAR-T cells will they explore new targets? Or do you plan to just produce better sales with existing well-established CAR-T targets? If you can just speak to that.

John Leonard -- President And Chief Executive Officer

So the NewCo will have its own story to tell as it moves along. It's an independent company, which we have a significant ownership position in. We're very, very excited about that. I'll remind you that we're supplying the allogeneic platform that we have not broadly disclosed. It is available to them, and you should look forward to hearing a little bit more about that as we go forward here. but it's our expectation that they'll pursue whatever they think the modality best addresses in that could apply to existing CAR-T targets. And with the switchable protein that actually targets these lymphocytes, they're certainly in a position where they could potentially explore other targets that I think that technology uniquely helps them to do. But that will be their story to tell as they lay out their plans going forward.

Silvan Tuerkcan -- JMP -- Analyst

Great. Thank you for taking my question.

Operator

The next question comes from Jack Allen from Baird.

Jack Allen -- Baird -- Analyst

Hi, Thanks for taking my question. Congratulations on all the progress this quarter. We wanted to ask about your most recent thoughts surrounding the potential for redosing with your LNP platform. How are you thinking about the potential need to redose NTLA-2001? Is this something that's on the radar? Or are you very confident that one dose is going to be enough to drive meaningful clinical benefit? And then a quick follow-up question was, how frequently are you receiving data from the study of NTLA-2001. I know it's a single-arm study, and I imagine you're receiving data on some sort of rolling basis. Any color you can provide there would be great as well. Thank you so much.

John Leonard -- President And Chief Executive Officer

I'll take that. We certainly look very, very carefully at the data as it comes in and it's an ongoing interaction with the investigation sites and our development team is in daily contact with them as that study progresses. It's not a blinded study. So we have the benefit of getting information and interacting with the investigators as we go. And the -- you're going to have to remind me the first question that you asked me, I just lost, I'm sorry -- redosing, I'm sorry. So yes. The way we think about redosing is the LNPs certainly enable that, and that's by their very nature. And one of the reasons that we chose the way back at the very beginning. They're not inherently immunogenic, and they do not raise neutralizing antibodies, which means that if we need to redose, we can redose. The dosing, in the case of 2001 for the patients participating in our first-in-human study has been to say to patients that once we determine an optimal biological dose, we will make that available to patients who did not receive that. That's not formerly a part of this particular protocol, but that is something that will be made available to the patients as the program proceeds. And going forward, as we think about dosing patients and generally, once we have the final format and final dosing. Again, it's our expectation, and this is based on the preclinical results that we have that the likelihood of the require redose, assuming an optimal dose given in the first place is remote, probably not necessary.

Jack Allen -- Baird -- Analyst

Thank you so much.

Operator

The next question comes from Yanan Zhu from Wells Fargo. Please go ahead.

Yanan Zhu -- Wells Fargo -- Analyst

Hi, Thanks for taking my question. So I was wondering for AML patients for the 5001 program, is there anything that might be different from the T cells from AML patients compared with B-cell lymphoma patients that might affect your -- the ability to grow out and manufacturing CAR-Ts in those -- from those patients.

John Leonard -- President And Chief Executive Officer

David, do you want to try to address that? Is there any reason that we should be aware of underlying differences in sales based on what we're doing relative to other cell types and other programs?

David Lebwohl -- Executive Vice President, Chief Medical Officer

We don't think that patients with AML have any intrinsic issue, let's say, with their T cells if they can't be manufactured well. Of course, we'll get more experience in the trial when we go forward. But so far, we have no evidence of that.

Yanan Zhu -- Wells Fargo -- Analyst

Got it. And a quick follow-up on your transduction method. I think in the past, you mentioned that you have a gentler approach to transduction and manufacturing the -- either the TCR or the CAR-T cells. Could you elaborate a little more on that? And what might be the advantage to traditional CAR-T manufacturing?

John Leonard -- President And Chief Executive Officer

Yes. Thanks for the question. It's something that we're really proud of that our scientific team has worked on and developed. We don't share the specifics of how we do that. We think that, that is an area of competitive advantage for us for the simple reason that standard out-of-the-box electroporation damage itself. Certainly, an effective way of getting material in cells and it's widely used. But the approach that we've taken does that at least as well, maintains cell viability, allow cells to behave as if they had never been transduced in the first place from a division point of view. We think that's going to translate into dosing advantages for patients. And importantly, when you look at the genetic architecture of those cells once they've been transduced. It's maintained. I mean, there's no inherent damage done to the genetic architecture by our transduction technique, irrespective of the editing itself, which is certainly not the case for standard electroporation. So it's -- our approach is that the editing is part of the story. How you do the editing is also critically important. And what you want to do ultimately is have cells that are as normal as possible an advantage to the greatest extent so that they'll behave well once the patient is dosed.

Yanan Zhu -- Wells Fargo -- Analyst

Great, Thanks for taking the question.

Operator

The next question comes from Tony Butler from ROTH Capital. Please go ahead.

Tony Butler -- ROTH Capital -- Analyst

John, 5001 or maybe the first program in cell therapy. I wanted to address what may be a very simplistic question. And that is, back when you and the team sat down to think about what would be your first target, you arrived at AML and WT1, which is great. The question is, was there any specific reason for that particular program that is a blood tumor target versus maybe, say, a solid tumor target? And I'm asking not because it's WT1 or because it's AML. But I would -- just based upon the way that I perceive you've laid out all the other programs, there may be some learnings you want to take from a particular set of experiments. And this may give you -- or this target and this blood tumor may give you the answer to those particular questions. And I'm curious if that's correct. And if you may share that.

John Leonard -- President And Chief Executive Officer

I think it's an astute observation of how we think about our work. We try very hard to isolate a key variable, get the clearest answer that we can with respect to that variable and then move on from there. So in the case of engineered cells, we believe, as we presented extensively in other venues that a T-cell receptor-based approach brings with it tremendous opportunities moves outside the fairly limited space that CAR-T cells are active in. What we wanted to determine was, could we with our approach, isolate healthy donor TCRs, develop an editing paradigm that introduce those TCRs into cells and then we could show that they could work. And what we tried to do is strip away all of the other potential confounding factors, solid tumors have some, liquid tumors tend to be less -- they have their own complexity for sure, but there's other factors that characterize solid tumors that don't apply in liquid tumors. And we thought with AML and the high levels of WT1 expression that characterize this that would be a wonderful test case for the overall thinking. And we're excited to see if our preclinical work is on target. And as David shared, we're at the point where we're going to find those things out because we're moving to the clinic by the end of this year.

Tony Butler -- ROTH Capital -- Analyst

Im grateful for you comment.

Operator

There are no more questions in the queue. This concludes our question-and-answer session. I'd like to turn the conference back over to Ian Karp for any closing remarks.

Ian Karp -- Senior Vice President, Investor Relations And Corporate Communications

Great. Thank you so much, and thank you, everyone, for joining us today and for your continued interest and support in Intellia. We look forward to updating you on our progress, and we hope everyone has a great day. Bye now.

Operator

[Operator Closing Remarks]

Duration: 60 minutes

Call participants:

Ian Karp -- Senior Vice President, Investor Relations And Corporate Communications

John Leonard -- President And Chief Executive Officer

David Lebwohl -- Executive Vice President, Chief Medical Officer

Laura Sepp-Lorenzino -- Executive Vice President, Chief Scientific Officer

Glenn Goddard -- Executive Vice President, Chief Financial Officer

Maury Raycroft -- Jefferies -- Analyst

Sonya Bhatia -- Goldman Sachs -- Analyst

Sheldon -- Barclays -- Analyst

Joon Lee -- Truist Securities -- Analyst

Luca Issi -- RBC Capital Markets -- Analyst

Dae Gon -- SVB Leerink -- Analyst

Jay Olson -- Oppenheimer -- Analyst

Ryan Deschner -- Raymond James -- Analyst

Silvan Tuerkcan -- JMP -- Analyst

Jack Allen -- Baird -- Analyst

Yanan Zhu -- Wells Fargo -- Analyst

Tony Butler -- ROTH Capital -- Analyst

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