Intellia Therapeutics Q1 2025 Earnings Call Transcript

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Provided by Quartr
May 8, 2025
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Operator

Good morning, and welcome to the Intellia First Quarter twenty twenty five Financial Results Conference Call. My name is Drew, and I will be your conference operator today. Following formal remarks, we will open the call up for a question and answer session. This conference is being recorded at the company's request and will be available on the company's website following the end of the call. As a reminder, all participants are currently in listen only mode.

Operator

I will now turn the conference over to Brittany Chavez, Senior Manager of Investor Relations at Intellia. Please proceed.

Brittany Chaves
Brittany Chaves
Senior Manager, Investor Relations at Intellia Therapeutics

Thank you, operator, and good morning, everyone. Welcome to Intellia Therapeutics first quarter twenty twenty five 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.

Brittany Chaves
Brittany Chaves
Senior Manager, Investor Relations at Intellia Therapeutics

At this time, I would like to take a minute to remind listeners that during this call, Intellia management may make certain forward looking statements and ask that you refer to our SEC filings available at sec.gov for 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 from Intellia are John Lettard, Chief Executive Officer David Lebwal, Chief Medical Officer Ed Dulak, Chief Financial Officer and Birgen Schultz, our Chief Scientific Officer, who will join for Q and A. John will begin with recent business highlights. David will then provide updates on our clinical pipeline progress and Ed will review our financials before we open the call for questions.

Brittany Chaves
Brittany Chaves
Senior Manager, Investor Relations at Intellia Therapeutics

With that, I will now turn the call over to John, our Chief Executive Officer.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

Thank you, Brittany. Good morning, everyone, and thank you all for joining us today. We entered the year with clear priorities and a plan for operational excellence, and we've already made tremendous progress in the first quarter. We're on a mission to offer life changing benefits with one time therapies for people living with severe diseases. Our progress is fueled by the core values of the company.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

One team exploring possibilities, delivering results and disrupting the status quo. We are committed to changing the treatment paradigm for patients suffering from hereditary angioedema and ATTR amyloidosis. Of the six milestones we outlined for 2025, we've accomplished two critical ones in the first three months of the year, dosing the first patient in our Phase three study for HAE and dosing the first patient in our Phase three study for hereditary ATTR with polyneuropathy. We continue to see significant interest from both investigators and patients across our programs. Enrollment in our global Phase three HALO study for HAE is progressing rapidly and reinforces our market research that the unmet need remains high despite existing treatment options.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

Patients are eager to pursue more convenient and more effective therapies. The transformational potential from a single infusion of NTLA-two thousand and two resonates strongly with patients and physicians. Our global Phase three magnitude study for ATTR with cardiomyopathy continues to be ahead of schedule. We now have over 90 sites actively enrolling and we continue to benefit from interest in our emerging profile for NexGurin and Cyclumerin, which we also refer to as NexSee from our Phase one data presented last November. In the first quarter, the FDA granted Intellia the RMAT designation for NexSee for the treatment of ATTR with cardiomyopathy, which follows prior RMET designations received for Nexe for ATTR with polyneuropathy and for NTLA-two thousand and two in HAE.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

In parallel to the great execution of our Phase three studies, we've been building critical commercial foundations in order to bring our promising therapies to patients as quickly as possible. Through the past few months, our commercial team has broadened its leadership capabilities and includes extensive experience with one time therapies and in disease areas of interest. We're increasingly confident in our ability to evolve into a strong commercially ready company. We're excited to share multiple clinical updates throughout the year. We expect longer follow-up to further solidify the emerging and highly differentiated safety and efficacy profiles of our lead programs.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

In the case of HAE, we'll present new data from patients who have crossed over in our Phase two portion of our Phase onetwo study later this year. This expansion of patients receiving the fifty milligram dose will provide a more robust perspective with more than 30 patients in total on unique and valuable profile afforded by a one time therapy like NTLA-two thousand and two. More immediately in June, we'll have two year follow-up data from our ongoing Phase one study of NTLA-two thousand and two at the European Academy of Allergy and Clinical Immunology Congress. For ATTR with polyneuropathy, we'll extend the durability window out to at least three years further extending our leadership position in in vivo gene editing. We're confident in our plans, diligent in our execution and excited by the value creating opportunities that lie ahead.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

Before I hand the call over to David Levwall, our CMO, I want to take a moment to address how we're thinking about the regulatory environment given leadership changes and developments at the FDA. Like everyone else, we will monitor the situation closely. And at this point, we've experienced no tangible changes to our interactions with the agency or timelines associated with our programs. We remain on track to meet or exceed our stated regulatory timeline and objectives. We remain in close communication with our review teams and continue to move our programs toward approval as per our original plan.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

We have a strong active relationship with the FDA as exemplified by the two prior RMAT designations and our most recent NATTRCM. We remain on course to file our first BLA in 2026. Similarly, we continue to monitor potential implications of pending pharmaceutical tariffs. We have well established manufacturing and distribution capabilities and are confident in our ability to manufacture and deliver supply for our clinical trials and eventually commercial product upon approval. Beyond that, we're convinced our products will yield significant value for patients and the healthcare system.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

We're continuing to monitor the environment, but amidst all the changes, there's one thing that remains to say and that's our dedication to bringing highly differentiated therapies that have the ability to reset the treatment standards for patients with HAE and ATTR. I'll now hand the call over to David Levwald, who will provide an update on our clinical programs. David?

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

Thanks, John. I'll begin with two thousand and two in development for HAE. As John noted, we dosed the first patient with 2,002 in our HALO Phase three study in the first quarter. HALO is a 60 patient global randomized double blind placebo controlled study. Patients are randomized two:one to either a single fifty milligram infusion of two thousand and two or placebo after washing out their long term prophylaxis therapy.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

They are then followed for a twenty eight week primary observation period and for a total of one hundred and four weeks in the study. Enrollment is going very well and progressing ahead of our projections. We are motivated by the early progress and excited by this patient and investigator interest. The team is executing well and we are in a position to go from the first patient to last patient dosed in less than nine months. This speed of enrollment confirms our market research and speaks to the high unmet need and demand in the HAE community as well as the significant room for improvement.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

We expect to complete enrollment by the end of the third quarter of this year. We are pleased to share that new 2,002 data were accepted for an oral presentation at the European Academy of Allergy and Clinical Immunology Congress on Sunday, June 15 in Glasgow. This update will include at least two years of follow-up in patients in the Phase one portion of the Phase onetwo study. Later this year, we plan to present longer term data from patients in the Phase two portion of the study, including those who initially received a twenty five milligram dose or placebo and were subsequently given the fifty milligram dose of two thousand and two selected for the Phase three study. As John mentioned, this Phase two update will more than double the total patients who have received the fifty milligram Phase three dose to more than 30 patients.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

Intellia is committed to ending the burden of HAE attacks and chronic treatment for HAE. The emerging profile of 2,002 from our Phase onetwo study suggests that many HAE patients can be free from attacks and free from the medications that are currently used to treat this disease. We believe and our market research shows that 02/2002 will bring significant value to patients, physicians and payers. The value proposition for 02/2002 is comprehensive and compelling, offering patients freedom from HAE attacks and chronic treatment, physicians freedom from persistent administrative burdens in managing chronic HAE therapies and material pharmacoeconomic benefits for payers. 02/2002 is poised to be the first ever one time treatment for people living with HAE and the first approved therapy using in vivo CRISPR gene editing.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

Let's move on to NextZ in developments for the treatment of ATTR amyloidosis. In March, the first patient was dosed in the global Phase three MAGNETUDE II study for the treatment of hereditary ATTR amyloidosis with polyneuropathy. This pivotal study is a placebo controlled study with expected enrollment of 50 patients. Patients are randomized to either a single fifty five milligram infusion of NexV or placebo. We plan to measure MNIST plus seven at eighteen months and serum TTR levels as key endpoints in the study.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

Full study enrollment is expected to be completed in 2026 to enable our second BLA filing by early twenty twenty eight. Also in March, we announced the FDA granted RMAT designation to NEXT Z for the treatment of ATTR amyloidosis with cardiomyopathy. As John mentioned, with the granting of a third RMAT designation, all of our lead programs and indications will benefit from earlier and more frequent engagement with the FDA. This is a testament to the potential of our therapies to reset the standard of care and the impact they can have on patients. We continue to be very pleased by the enrollment of the global Phase three MAGNETUDE study in ATTR amyloidosis with cardiomyopathy, which is ahead of our projections.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

We expect cumulative enrollment to exceed five fifty patients by year end. Later this year, we will present longer term data from patients with either ATTR polyneuropathy or cardiomyopathy in the Phase one study, which will include updated measures of clinical efficacy and safety. We will have a median follow-up of two years in cardiomyopathy and three years in polyneuropathy. We look forward to sharing these updates in the second half of twenty twenty five. I'll now hand over the call to Ed, our Chief Financial Officer, who will provide an update on our financial results as of first quarter twenty twenty five.

Edward Dulac
Edward Dulac
EVP & CFO at Intellia Therapeutics

Thank you, David. Good morning, everyone. Intellia continues to maintain a solid balance sheet that allows us to execute on our pipeline and platform. Our cash, cash equivalents and marketable securities were approximately $707,100,000 as of 03/31/2025, compared to $861,700,000 as of 12/31/2024. Our balance sheet evolution reflects normal expenses from operations during the first quarter and nonrecurring costs associated with decisions we took to prioritize our portfolio and reduce our real estate footprint and workforce, all of which diminished the medium and long term capital needs for the company.

Edward Dulac
Edward Dulac
EVP & CFO at Intellia Therapeutics

These outcomes represent positive developments and allow our current balance sheet to bridge to our expected launch for NTLA-two thousand and two in HAE during the first half of twenty twenty seven. During this time, Intellia will achieve several important value creating clinical development and regulatory milestones, which we expect will help us further capitalize the company and aggressively pursue our plans for Next Z in ATTR with polyneuropathy and cardiomyopathy. Our collaboration revenue was $16,600,000 during the first quarter of twenty twenty five compared to $28,900,000 during the first quarter of twenty twenty four. The $12,300,000 decrease was mainly driven by a decrease in collaboration revenue under the Avansel license and collaboration agreement. Recall that during the first quarter of twenty twenty four, there was a transition to equity method accounting for Avancell, which resulted in a onetime recognition of revenue of approximately $21,000,000 R and D expenses were $108,400,000 during the first quarter of twenty twenty five compared to $111,800,000 during the first quarter of twenty twenty four.

Edward Dulac
Edward Dulac
EVP & CFO at Intellia Therapeutics

The $3,400,000 decrease was primarily driven by employee related expenses, stock based compensation, research materials and contract services, offset by an increase in the advancement of our lead programs. Stock based compensation included in R and D expenses was $12,600,000 for the first quarter. G and A expenses were $29,000,000 during the first quarter of twenty twenty five compared to $31,100,000 during the first quarter of twenty twenty four. The $2,100,000 decrease was primarily related to lower employee related expenses due to a workforce reduction in January 2025 and lower stock based compensation, partially offset by increases related to severance expenses recorded in the first quarter. Stock based compensation included in G and A expense was $9,200,000 for the first quarter.

Edward Dulac
Edward Dulac
EVP & CFO at Intellia Therapeutics

As guided previously, we continue to expect a year over year decline in GAAP operating expenses of between 510% this year and that our cash balance is sufficient to fund our operating plans into the first half of twenty twenty seven.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

Thanks, Ed. In conclusion, Intellia continues to meet and even exceed our goals in all programs, and we're excited to report on our progress in the months ahead. With that, we'll now open the call for your questions. To do our best to address as many questions as possible, we will only be able to take one question per caller. Operator, you may now open the call for Q and A.

Operator

The first question comes from Gena Wang with Barclays. Please go ahead.

Gena Wang
Gena Wang
Analyst at Barclays Capital

Thank you. You have so many updates on different progress, but I will limit my questions to one. So since that's the most pressing questions, so I will ask about the magnitude Phase three trial enrollment seems like ongoing very well. And if you can give your updated metrics regarding the patient baseline characteristics, which includes like percentage of patients who are on baseline stabilizer and also the silencer dropping rate. What are these rates?

Gena Wang
Gena Wang
Analyst at Barclays Capital

And are these rates and, are these rates in line with your internal expectation?

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

David, do you want to speak to the evolving characteristics at baseline of patients?

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

Yes, thank you for that Gina. Yes, the exciting thing is the rate at which this is enrolling. And, in terms of the patients around the world, tafamidis is becoming, more commonly used, including The UK recently. So as we've said really from the beginning, we do expect more than fifty percent of the patients, to be on tafamidis in the study and that we're monitoring that. We do think it's important to show a benefit over tafamidis that hasn't been shown yet with the silencers and this is also valuable then to have a large group of tafamidis patients on the study.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

In terms of silencer, of course, has just been approved in The U. S. Recently. We don't expect many patients to crossover though at this point there are no patients. But over time, we do anticipate in our statistics that a percentage of the patients will be going over to Silencer and we're ready for that in terms of the results.

Gena Wang
Gena Wang
Analyst at Barclays Capital

Thank you.

Operator

The next question comes from Mani Bhurrahar with Leerink Partners. Please go ahead.

Lili Nsongo
VP - Equity Research, Biotechnology at Leerink Partners

Hi, this is Lillian Songo on for Mani. Thank you for taking our question. I just had a question regarding cash burn and OpEx. So you just mentioned that you were expecting a 5% to 10% year over year decrease in OpEx. But could you maybe give us a little more in terms of what we should expect in terms of cash burn in the next twelve to twenty four months, especially as the restructuring progresses?

Lili Nsongo
VP - Equity Research, Biotechnology at Leerink Partners

So are there any notable nonrecurring costs or event that we should be taking into consideration?

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

Thanks for the question. Ed, do you want to walk through? There's a lot of details, but I think it's really important to understand what's going to be the baseline running rate going forward and Ed can take you through that. Ed?

Edward Dulac
Edward Dulac
EVP & CFO at Intellia Therapeutics

Yes. Thank you. Thanks for the question. This is an important focus for the company. I think the key point I want to make for investors that we estimate that our average cash use over 2025 and 2026 will be about $95,000,000 per quarter.

Edward Dulac
Edward Dulac
EVP & CFO at Intellia Therapeutics

And so this is consistent with the guidance that we reiterated today this morning that our current cash will fund our operating plans into the first half of 'twenty seven. As I indicated during the fourth quarter call a few months ago, we expected the first quarter results today to be pretty noisy, just given the broad restructuring decisions that we made at the company earlier in the year. So I wanted to just unpack a little bit, the cash, what drove our cash use during the quarter. The first key driver was our normal company operations. We spent $86,000,000 to run the business, which, again, is very consistent with sort of that $95,000,000 per average cash used per quarter that I mentioned previously.

Edward Dulac
Edward Dulac
EVP & CFO at Intellia Therapeutics

So normal operations, we spent $86,000,000 in the quarter. Employee bonuses, of course, this is something we routinely do, but that was $18,000,000 and we did use that to pay in bonuses to existing employees but also those that were impacted by the restructuring that we announced in January. And the last driver of cash use for the quarter was sort of these nonrecurring costs. That was about $51,000,000 in the quarter, and we used some of this to pay employee severance and related costs. But primarily, we used this cash to enter into payments associated with real estate transactions that we disclosed in February as part of our 10 ks filing.

Edward Dulac
Edward Dulac
EVP & CFO at Intellia Therapeutics

And I'll talk maybe a little bit about our real estate transactions that I just referenced. We're actually very excited about the development and the evolution of our real estate portfolio. And just to remind folks, in February, we entered into a cash neutral transaction to reduce our portfolio, simplify our operations and identify additional and significant savings. The cash neutrality to that transaction is really important for us. So we essentially took cash that we had budgeted for the company's real estate portfolio through 2026, and we used that to pay agreed upon lease modification payments.

Edward Dulac
Edward Dulac
EVP & CFO at Intellia Therapeutics

So if I say that a little bit differently, the near term cash outlays associated with the real estate transaction will be fully recouped from the absence of cash payments that we had formally expected to pay for the real estate portfolio. So the bottom line, our real estate portfolio really better aligns with the focus and the needs of the company, and it does bring us a few important benefits. The first one is we'll simply have a new corporate headquarters that the company is excited about. So by the end of twenty twenty six, we'll have a new headquarters located in Cambridge, where we plan to consolidate most, if not the entire company. And we expect this will support the growth and the support and grow our collaboration and innovation and culture at the company.

Edward Dulac
Edward Dulac
EVP & CFO at Intellia Therapeutics

So we're very excited about those prospects. From an operational perspective, we're just going to have to run a very smaller, simpler portfolio. So we'll have about a 30% reduction in the real estate capacity over the next couple of years, including the release of all the obligations from a long term lease that we had in Waltham, Massachusetts for more than 140,000 square feet. And the last thing I'll say on the real estate portfolio, we according to our estimates, we'll expect nearly $50,000,000 in cash savings from operating our smaller footprint. There'll be other synergies and cost savings associated with the management of our portfolio, and there's potential sublease income from the smaller buildings that will remain in the portfolio.

Edward Dulac
Edward Dulac
EVP & CFO at Intellia Therapeutics

So I covered a lot of ground there, but I think it's such an important topic. I wanted to spend a little bit of time on it. And just to reiterate, we will use an average of $95,000,000 of cash per quarter through this year in 2025 and in 2026, and this importantly will allow us to do three very critical things. First one, fully invest in our three Phase III studies that we provided an update this morning. It has, and we will continue to build the commercial infrastructure in The U.

Edward Dulac
Edward Dulac
EVP & CFO at Intellia Therapeutics

S. To capture the significant value that we see across both of our lead programs. And importantly, we created a financial bridge to our first anticipated launch in the first half of twenty twenty seven for NTLA-two thousand two and HAE.

Operator

The next question comes from Andy Chen with Wolfe Research. Please go ahead.

Analyst

This is Hannah Tran calling. Thanks for taking our question. Just wondering in on a question previously asked. We see that you guide to a cash runway into the first half of twenty twenty seven, but post 2027, have you considered non dilutive financing? And if so, what options and how feasible would they be to obtain?

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

Ed, do you want to keep on going from the prior question, just talking about how we look further down the road and some of the options that we're actively thinking through?

Edward Dulac
Edward Dulac
EVP & CFO at Intellia Therapeutics

Yes. I appreciate the question. I'll just start with what we kind of indicated this morning. In January, we made some difficult decisions on the restructuring. We've already seen, I think, early and encouraging signs in the first quarter.

Edward Dulac
Edward Dulac
EVP & CFO at Intellia Therapeutics

Our operating expenses were down 4% versus the year ago quarter and down 7% already from the fourth quarter of last year. So for the things that are immediately in our control, we're definitely focused on making sure that we are operating very efficiently, and that will continue over the next few years. As it relates to capital raising going forward, I think we have clearly built an opportunity for the company to click through a number of important milestones. We think they will be value creating for shareholders, and we would think about how do we raise additional capital on the back of some of those catalysts that the company has over the next twelve to twenty four months. We are big believers in building the company, and so we're going to continue the evolution to a commercial stage company.

Edward Dulac
Edward Dulac
EVP & CFO at Intellia Therapeutics

We also don't talk much about our research pipeline, but we do have one, and we're continuing to invest there. So we're looking to build the company over the long term. And I think a number of different levers are on the table for us. One would be collaborations. We currently have a collaboration with Regeneron on our TTR asset.

Edward Dulac
Edward Dulac
EVP & CFO at Intellia Therapeutics

There are opportunities to think about that collaboration potentially differently. But we do have wholly owned assets like 2,002 that we can consider partnership. And then we have pipeline assets that are also open to potential collaboration. I would say another option that becomes increasingly more available to a company like Intellia, as we approach commercialization, funding options like royalty transactions could make sense. The other option that you mentioned would be sort of a term debt or venture debt sort of a structure that also could make sense as we think about commercialization, the revenue generation and the profitability that this company could have on a three year view.

Edward Dulac
Edward Dulac
EVP & CFO at Intellia Therapeutics

And in reality, we may consider one or more of those over the next two to three years. So we've been talking about this just given the current macroeconomic situation and geopolitical situation for quite some time. I think we have some clear plans in place, and stay tuned. But we feel really good about the balance sheet, where we're heading, and I think we have multiple levers to capitalize the company over the next next couple of years.

Operator

The next question comes from Costas Belouris with BMO Capital Markets. Please go ahead.

Kostas Biliouris
Kostas Biliouris
Director - Biotech Equity Analyst at BMO Capital Markets

Good morning, everyone. Congrats on the progress and thanks for taking our question. A question from us on 8A given that you plan to file in 2026 and this will potentially be the first ever commercial in vivo gene editing therapy. Can you help us understand how should we be thinking about the launch dynamics there in terms of activating sites for patients, securing coverage and potentially time required from patient decision to receive the therapy, to infusion time. Should we expect similar timeline dynamics to in vivo gene therapies that we have today in the market?

Kostas Biliouris
Kostas Biliouris
Director - Biotech Equity Analyst at BMO Capital Markets

Thank you.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

Justus, thank you for the question. It's think really a very important one. As you pointed out HAE will be not only our first in vivo gene editing launch, but the world's first CRISPR in vivo gene editing launch and something that we're paying great attention to. In our Phase three program, which as David commented earlier, things have been progressing extremely well. We're ahead of our timeline and learning much about how to deal with sites, how to make the drug available, etcetera.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

In parallel,

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

we've

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

been building our commercial organization and have staffed it with people with deep insights into prior one time therapies from which we can certainly learn a lot. Many of those one time therapies, however, are not good analogs to what we're doing. And the reason for that is ours is a very straightforward outpatient infusion where patients receive a dose of dexamethasone the day before therapy, come and sit in the clinic for two to four hours with another repeat dose of dexamethasone and some antihistamines and they go home. And so how to provide that versus some of the prior examples that have been made available is day and night. With respect to switching from therapies that patients may already be taking, as you might imagine, as we carry out our Phase three trial, we're learning a lot about that.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

And in many instances, this is a simple matter of looking at the pharmacokinetics of the drugs that patients are currently taking and compensating for how those will wash out over time while the effect of the gene edit takes place. So we think we're in really excellent position from a drug profile point of view and we presented some of that data. You'll see more this year. We're very excited about how patients do and how the profile evolves over time and are really looking forward to sharing that soon. And from a standpoint of getting into the marketplace, once the drug is approved, we think that we can progress extremely efficiently and bring the drug to many, many patients, which has been what we've been learning from our market research.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

So, I think we're in a very, very good position at this point.

Operator

The next question comes from Luca Isi with RBC. Please go ahead.

Luca Issi
Luca Issi
Senior Biotechnology Analyst at RBC Capital Markets

Great. Thanks so much for taking my question and congrats on all the progress. Maybe a quick one on pricing. What was your reaction when you saw Alnylam actually not lowering their price and they're going to label expanded from TTR polyneuropathy to TTR cardiomyopathy. Were you surprised by it?

Luca Issi
Luca Issi
Senior Biotechnology Analyst at RBC Capital Markets

And just maybe bigger picture, how you're thinking about pricing for your molecules more broadly given the one and done nature of them? Thanks so much.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

Thank you. Obviously, we're paying attention to the TTR market broadly. It's not just Alnylam. There's as you know other oral participants including a recent entrant and we watch the uptake and the performance of those different drugs. What we see is an increasingly large and rapidly growing marketplace.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

Diagnostic procedures are improving. The disease is more widely recognized and across the board this translates into an opportunity for all entrants. But we think with the profile that we've seen thus far from our drug, this is going to be a very exciting market for us to participate in. With respect to Alnylam's price, obviously they're responding to what they see in the marketplace, the dynamics that they've observed already polyneuropathy and the prior experience with ONPATTRO. We think that translates into very significant opportunity for us.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

And as we get down the road, we'll further hone how we think about that. In the meanwhile, it's all about getting clinical trials enrolled, which we're doing very, very aggressively. And as David has said, especially with the progress of those programs, we're well ahead of schedule. And if anything, we see enrollment accelerating. So, we're looking forward to participating in that marketplace and we're quite confident that we will do very, very well.

Luca Issi
Luca Issi
Senior Biotechnology Analyst at RBC Capital Markets

Got it. Thanks so much.

Operator

The next question comes from Maury Raycroft with Jefferies. Please go ahead.

Maury Raycroft
Maury Raycroft
Equity Research Analyst at Jefferies

Hi, good morning. Congrats on the progress and thanks for taking my question. Going back to enrollment for the HALO Phase three, in late March you changed the minimum age from 18 years old to 16 years old on ct.gov. Just wondering if that's driven by patient interest or demand or was it to accelerate enrollment or for other reasons? And then based on cardiomyopathy enrollment continuing to track better than expected, can you say more on where you expect enrollment to land by the end of the year?

Maury Raycroft
Maury Raycroft
Equity Research Analyst at Jefferies

Could it be greater than 100 patients on study or 600 patients on study?

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

I guarantee you it will be more than 100 patients by the end of the year. Thank you for the question Mark. Yes, let's start with the HALO study first. Basically our interest is in having the broadest possible label, whether it's from age or disease severity. And we've designed a program that should permit that.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

Patients coming into the Phase three study resemble to a great extent those that come into the Phase two and Phase one studies and we see a range of disease severity and we think that that's very, very helpful in terms of assessing how the drug performs and should auger well for the label that we expect to get. With respect to enrollment of that study, we've been extremely gratified by interest across the board, United States, outside The United States, where we've had patients essentially lined up and that is not an exaggeration at all sites and we are well ahead of what we projected. So we'll have some opportunity to further refine exactly what that looks like. But as we look down the road into how we think uptake will go in the marketplace, Many of the comments that we've seen others offer where this is a well satisfied space, we see that that's not correct. Patients and physicians tell us that there's significant remaining need, desire to get to a state of no attacks and no further therapy, which we've seen in the majority of patients thus far is very, very compelling to patients.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

And we actively see people very, very actively seeking that out. With respect to the cardiomyopathy enrollment, similarly, we've been very gratified by the very rapid enrollment there. As we've guided, we expect to have cumulative enrollment beyond five fifty patients. Typically, we under promise and over deliver and that may be a further instance of that. And as we get down through the year, we'll give some additional details there.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

But we like where we are. And as we look at comparators from prior experience, we've been very pleased because we're well ahead of those projections.

Luca Issi
Luca Issi
Senior Biotechnology Analyst at RBC Capital Markets

Got it. Thank you.

Operator

The next question comes from Alec Strenihan with Bank of America. Please go ahead.

Matthew Guggenbiller
Matthew Guggenbiller
Equity Research Analyst at Bank of America

Hey guys, this is Matthew on for Alec. Thanks for taking our question. Maybe just one looking forward from us. I know that you said you're still developing other in vivo and ex vivo candidates. Maybe just some color on the timeline of these, whether they're likely to come after the potential approval in HAEATTR?

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

You're referring to our pipeline, is that? We've talked about some things in the past that we've been working on, but what we've been focusing on since the end of last year and throughout this year is very much the clinical programs. And that will be what we spend most of our time talking about because that's what's going to be the near term driver of significant value. I think to separate us from other companies in the space, we're well beyond the proof of concept phase. We've demonstrated clinical activity with these drugs and what we're doing now is building a label with pivotal trials and gaining approval.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

So that's where the focus is going. We do have very significant efforts underway for additional in vivo candidates. We're not talking about most of those at this point. In the past, we've spoken some about alpha-one and you may hear about that as time goes on. We're very excited about progress we're making with our GeneWriter.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

We can imagine areas where that can bring real utility. And from a Nextiva point, we think we have some insights that can significantly open up that space. And as time goes on, we'll be talking more about that, but these are competitive areas. So we'll focus on the clinical work.

Operator

The

Operator

next question comes from Mitchell Kapoor with H. C. Wainwright. Please go ahead.

Mitchell Kapoor
Director, Senior Biotechnology Analyst at H.C. Wainwright & Co., LLC

Hey, everyone. Thanks for taking the question. Can you just talk a bit about the payer perception of potentially having to cover both tafamidis and MEKZ and how that changes the way we should interpret data from ATTR studies?

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

David, do you in the clinical trials, have you seen any information that bears on how payers are looking at Tefamidis in the well, actually you asked about Nexe. I'm sorry, was misinterpreting. I'll say this. We're building a database with respect to payers where we have increasing insight. Much of the early insight we're getting is for HEE, which has been the bulk of our work.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

We expect that at the time of launch for Nexe that tafamidis will be a generic drug. Most estimates suggest that that will be the case. And if that is in fact the situation, I would expect from a payer point of view that that will not be a significant point of discussion. I think we'll get some early insights in terms of how payers behave in the most stringent circumstances with the Invupra launch and look for examples where tafamidis may or may not be used together. Again, the clinical circumstances are a little different in that case where the clinical benefit was not shown in a statistically meaningful way in the HELIOS study.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

As David said, we are designing our trial in such a way and have sufficient patients coming in on Tafamidis to in fact demonstrate an expected benefit when the two drugs are used on top of Tafamidis alone. So regardless of payers, we'll have the clinical evidence and we would certainly intend to have it in the label. So that's where the situation stands as we speak today.

Operator

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

Jay Olson
Research Analyst at Oppenheimer & Co. Inc.

Hey, thanks for taking the question. Maybe just to follow-up on the previous question about the patient baseline characteristics ignoring in magnitude. Can you talk about how these characteristics will impact your estimate for the time to reach the acquired number of events for the primary endpoint? And whether that would happen before or after the first half of twenty twenty seven? Thank you.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

Yes. I don't know if we're going to be talking about timelines, but you want to talk about baseline characteristics and how you're thinking about that David with respect to the study progression?

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

Yes. So the patients in this study are looking very similar to the other Phase three studies, other than what I mentioned that there's more and more use of tafamidis. So if you look at the number of patients with variant disease, which is a more aggressive disease, proportion is in same range ten percent to fifteen percent as it is in the other studies. Class III patients who also progress at a more rapid rate are also in the same range in the ten percent to fifteen percent range. So we do think the events to evolve fairly similar to the recent studies based on patients who obviously more patients on tafamidis.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

And again, the timing as we get obviously get closer to analyses, we'll be able to tell you more about the timing of that.

Operator

The next question comes from Rick Winkowski with Cantor Fitzgerald. Please go ahead.

Rick Bienkowski
Analyst at Cantor Fitzgerald

Hey, good morning and thanks for taking the question. For 02/2002, I was hoping you could expand on the value proposition in HAE and thoughts on the degree of flexibility you'll have for pricing. Just given the competitive landscape here, I'd like to know your thoughts on how a one time treatment should be valued against chronic treatments and what potential cost offsets could be realized by payers over time?

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

Yeah, it's a very important question. I think it's important to start with the clinical profile that we've seen thus far where in addition to attack rate reduction, which we see across the board in patients, the vast majority of patients reach a point of no attacks off therapy. I repeat, no attacks without any other therapy. And that is a very important distinction and it's a category that of outcomes that's unique to 02/2002. So that clearly brings value to patients.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

If they can behave in a way where they don't have to think about their disease, that's what they want. They don't want faster demand therapy or longer term prophylaxis. They want to get rid of their disease if they can. That's what's driving interest and that's true for the physicians as well who in many cases struggle with the time demands to reauthorize patients for their very expensive therapy year in and year out. Remember that many of these patients are diagnosed in adolescence.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

So the value that the drug brings when you look at from a purely pharmacoeconomic point of view and a payer's perspective is substantial and very, very significant. These patients in The United States start at over a quarter of a million dollars a year with many of them costing over a million dollars a year in drug therapy alone. That provides a substantial window for us to price the drug in a way that can be very competitive with any other existing therapies, that can be very, very resource sparing for the healthcare system generally and performed very well for the shareholders of our company. And we're refining that work today where prior precedents are taken into consideration and relationships between annual costs and the price of one time therapy gives us some guideposts. We will not be setting any new records for high priced drugs here at Intellia.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

What we're trying to do is address all of our stakeholders in the best possible way and as that story unfolds, we'll provide more insights.

Rick Bienkowski
Analyst at Cantor Fitzgerald

Great. Thank you.

Operator

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

Yanan Zhu
Yanan Zhu
Analyst at Wells Fargo

Hi, thanks for taking our question. Our question is also around Nexi in ATTR Centimeters. So we know overall the Phase III study enrollment is progressing well. Can you specifically talk about the enrollment in The U. S?

Yanan Zhu
Yanan Zhu
Analyst at Wells Fargo

And are you seeing any impact from the approval of Atrube and Invutra? And are you allowing drop in of those two drugs?

Yanan Zhu
Yanan Zhu
Analyst at Wells Fargo

Thank

Yanan Zhu
Yanan Zhu
Analyst at Wells Fargo

you.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

David, do want to speak to have you seen any impact from the new drugs and the magnitude enrollment in The United States?

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

Sure. Yes, just first speaking to the enrollment being brisk. What we saw, I think what you saw at last year is that the drugs is doing something different from what's been seen with other drugs for this disease. We've seen that the progression is really stopped and patients even improve. We've seen a very low event rate in this group and this seems to have touched the investigators looking at this and really push forward the enrollment.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

That includes The U. S. And really all over the world. We have really have sites pretty much everywhere where the disease where there are specialists for this disease. What we've seen in terms of Vutrisiran, you've heard from Alnylam, they don't expect there to be a significant combination of tafamidis with vutrisiran.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

In The U. S. Where vutrisiran is approved, virtually every patient is on tafamidis right now. So, though there is a chance to go on there is a possibility of patients being able to get through trisoran, it's not expected to be a common event based on the data that Alnylam has provided. So far we have been able to keep up, obviously it's early for Vutris, so we don't know a lot, but we so far have been able to maintain the enrollment and even accelerate in recent months despite the fact that vutrisiran is coming out.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

And the way we see it is that physician may choose vutrisiran for a patient or tafamidis initially. And with our trial, it gives them the chance not only to get tafamidis, if they're on tafamidis, but also to get a drug that may add substantially to the tafamidis effect. So that's what we're seeing so far. And obviously, we'll be keeping close tabs on that as going forward. In terms of being able to crossover the protocol, we're telling physicians if they intend to use reticerine, they should not enroll in the study right now, obviously, that would be their decision and the patient's decision.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

But if they are enrolling in the study, we don't expect them to go over to vutrisiran during the initial year or two.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

I think it's also the case that most physicians in our experience don't see much of a difference between tafamidis or vutrisiran at least with the data.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

Yes. That's what we're hearing from them. So we're still getting very brisk enrollment despite the availability of vutrisiran.

Operator

It. Next question

Operator

comes from Troy Langford with T. B. Cowen. Please go ahead.

Troy Langford
Biotechnology Equity Research Vice President at TD Cowen

Hi, there. Congrats on all the progress this quarter and thanks for taking our questions. I just want to follow-up on one of the comments that you actually just made about the recent 2,001 Phase one data for functional data from last November. So when you all show that data to physicians since you presented it, can you talk a little bit more about maybe like what one data point stands out most of them or seems to resonate most with them?

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

David, you speak to the physicians, what are they like about 2,001?

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

Sure. The first point is the profiles we showed on reduction. If you recall, reach our nadir in one month and reach about 90% reduction to levels of nineteen micrograms per ml. What vutrisiran has shown in a recent New England Journal is that they take about nine months to get to the nadir. So it's a very delayed reduction in TTR and they reach a level about 50.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

So it's a very big difference in terms of what's happening with TTR. That's what physicians believe many of them that that's what drives efficacy and all the results are consistent with that from last November. We don't see increases in proBNP, we don't see decreases in six minute walk and all those things are seen in populations of patients receiving either silencer or stabilizer drug in recent Phase three studies that are available in a similar group of patients. The other thing they see is we've recently presented the time to first event and that also looks very different from what we've seen in those Phase three studies. So we think when we do talk to physicians, most of them are quite impressed by the data we're seeing with Nexe.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

And as I said, I think this is driving enrollment and also obviously will be important in our trial results that this will drive a successful Phase three trial.

Troy Langford
Biotechnology Equity Research Vice President at TD Cowen

Great. Thanks for the color.

Operator

The next question comes from Myles Mitra with William Blair. Please go ahead.

Jake Roberge
Equity Research Analyst at William Blair & Company, L.L.C

Hi, this is Jake on for Myles. Thank you so much for taking our question. We had a question about some changes that have recently happened at CBER and whether this is influencing your plans for timing of your BLA submission or plans for hiring on a sales force potentially in relation to 02/2002?

Jake Roberge
Equity Research Analyst at William Blair & Company, L.L.C

Thank you.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

It's, well, generally speaking changes at the FDA have been much commented on by others and for good reason, we all have to work with the FDA. In our experience thus far, none of those changes have directly affected us. We've established strong working relationships with our review teams. As David mentioned in his comments during the earlier part of the call, just recently received our third of three instances of RMAT designation. So we know people are working hard on looking at the merits of the drug, etcetera.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

Team has been review team has been engaged. We have meetings with the FDA and we think we're in good shape. With respect to the most recent change at CBER, what we see is a strong interest in actual clinical data as opposed to surrogate markers, which we think affects areas where we're not active, whether it's vaccines or single arm studies, surrogate markers in oncology. All of the programs that we're running are randomized comparative trials that are controlled and they have clinical endpoints that are unambiguous and well standardized in the field. So we think that we're speaking the same language as

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

some of

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

the new participants at the FDA and we look forward to sharing our data on time or ahead of time given the enrollment and the RMAT designation with all the indications.

Jake Roberge
Equity Research Analyst at William Blair & Company, L.L.C

Thanks.

Operator

The next question comes from Brian Cheng with JPMorgan. Please go ahead.

Brian Cheng
Brian Cheng
Senior Biotech Analyst at J.P. Morgan

Hey guys, thanks for taking our question this morning. I'm curious if you can elaborate a little bit more about your latest thinking on just the timeline for ATTR cardiomyopathy. Since that during the call, you said that the enrollment is progressing ahead of projection. What should be our base case for the time to get to events to have the first interim look for this Phase three? And just given that the recent trials have been taking a little bit longer than expected, yes, so can you elaborate a little bit more on the timeline?

Brian Cheng
Brian Cheng
Senior Biotech Analyst at J.P. Morgan

Thanks.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

I'll just maybe lay the groundwork and David can deal with the details. But you're right, enrollment as we've said is going extremely well and we're very pleased with where we are and the progression of the study around the world, U. S, ex U. S. That's all progressing very, very quickly.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

Interim analyses are a function of enrollment and event rates and that's something that we'll be watching here. But David, do you want to talk about any enrollment for the study regardless, we think will be done by when and how are you thinking about interim analysis?

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

Yes. So what we've said and we'll keep to is that the enrollment fees finished by the beginning of twenty twenty seven and the '26. So we do think that interim analysis, the first timing would be after enrollment is complete. So in 2027 as you say, the idea of an interim analysis is that you have a drug with outstanding efficacy that you can stop a trial early because you see something at that early point. As I just talked about, we do think we have outstanding efficacy with this drug.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

We will continue to follow closely the long term results in the Phase one. We'll be looking at the event rate obviously and magnitude as well, as well as looking back at other trials. But looking at all that, we do think, it's certainly possible that this trial could stop at an interim analysis, based on the efficacy we're seeing, the exact timing, as I said, you need to be following these other things events to know when that is, wouldn't rule out 2027 based on what we're seeing. But obviously as we get closer, we'll give

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

you more information on that.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

Would say in contrast with prior studies more likely to be early than later.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

I think we're pretty confident

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

about that.

Operator

The next question comes from William Pickering with Bernstein. Please go ahead.

William Pickering
Analyst at Bernstein

Hi, thank you for taking my question. For the MAGNATUDE II PN study, could you talk about your expectations for the enrollment rate? I know you said, completion in 2026, which is understandably a pretty wide range just given it's fairly early in the study. But do you think the Alnylam and Ionis PN studies are reasonable benchmarks? And just any color you can share on early interest in the study?

William Pickering
Analyst at Bernstein

Thanks so much.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

Thanks David.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

Yes. So this is different from the earlier studies and that of course the widespread use of vutrisiran has required us to go to countries that don't yet have vutrisiran available. However, because those studies don't have the modern therapies, there is a very large interest from the investigators in this therapy. It will enroll briskly and as said into 2026 as we get closer, we could give you more details on when that enrollment ends. But it does give us the possibility of a submission in 2028 using standard timelines.

David Lebwohl
David Lebwohl
EVP & Chief Medical Officer at Intellia Therapeutics

Again, because of the high level of efficacy we're seeing in PN and we'll be talking more about this just in June at PNS, because of that, this also could stop at an interim analysis or even accelerated approval type situation. So, keep in touch, look for the data in June and you'll get more on where this timeline may go.

William Pickering
Analyst at Bernstein

Thank you.

Operator

And the last question today will come from David Lebowitz with Citi. Please go ahead.

David Lebowitz
David Lebowitz
Analyst at Citigroup

Thank you very much for taking my question. In terms of the primary endpoint of the MAGNETUDE trial, given the range and for the events from I think eighteen months to eighteen weeks to eighteen months to forty eight months, what are the dynamics relative to I guess cost? Number one, if the events were to come in slower, does it change at all your thoughts on what type of cash you have and need? And additionally, when looking at the Alnylam label because they had shuffled their primary endpoint, one of their endpoints from thirty to thirty six weeks to thirty six to forty two, but in the labeling, it ultimately was pulled to being thirty to thirty six weeks for all the endpoints. How do you think the FDA would look at your primary endpoint with such a wide range on timing?

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

Two points. First of all, from a runway point of view, we've taken a conservative view that considers the scenario that you touched on and even adding additional patients should that be the case. So we think from a runway point of view, we're in good shape. With respect to prior work that was done by Alnylam, a contrast in the design of our study with HELIOS V is that from the get go, our study magnitude has been an endpoint study. It's not dictated by time.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

It's dictated by when patients experience any of the various endpoints that are included in the composite list. So we don't see ourselves patching it up at the end of the study to get to some sort of average duration to get to endpoints by its design, it should accomplish that. So we think that overall we're in good shape. We have the funding to complete the enrollment and the trial and as Ed said in his earlier comments, the bridge that we've built takes us into the launch of 02/2002. So in contrast with other companies in the space, we're talking about commercialization and revenues coming into the company and we're well beyond the proof of concept stage.

John Leonard
John Leonard
President and Chief Executive Officer at Intellia Therapeutics

So we're on well on our way to being a fully integrated pharmaceutical company that we've always intended to be. Thank you very much. Appreciate it.

Operator

This concludes the question and answer session and Intelli Therapeutics' first quarter twenty twenty five financial results conference call. Thank you for attending today's conference. You may now disconnect your line.

Executives
    • Brittany Chaves
      Brittany Chaves
      Senior Manager, Investor Relations
    • John Leonard
      John Leonard
      President and Chief Executive Officer
    • David Lebwohl
      David Lebwohl
      EVP & Chief Medical Officer
    • Edward Dulac
      Edward Dulac
      EVP & CFO
Analysts
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Key Takeaways

  • Intellia hit two of its six 2025 milestones by dosing the first patients in its Phase 3 trials for hereditary angioedema (HAE) and ATTR amyloidosis with polyneuropathy.
  • Enrollment in the global HALO Phase 3 study for HAE is ahead of projections and set to complete by Q3 2025, while the MAGNITUDE II cardiomyopathy study has over 90 active sites enrolling patients.
  • The FDA granted RMAT designations to NTLA-2002 in HAE and NEXE for both ATTR indications, supporting frequent agency engagement and keeping Intellia on track for its first BLA filing in 2026.
  • With $707 million in cash at the end of Q1 2025 and an expected average cash burn of $95 million per quarter, Intellia’s runway extends into the first half of 2027—covering its planned launch of NTLA-2002 in HAE.
  • Intellia has strengthened its commercial team with leaders experienced in one-time therapies and is building infrastructure to secure site activation, payer coverage and physician adoption ahead of market launch.
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Earnings Conference Call
Intellia Therapeutics Q1 2025
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