Intellia Therapeutics, Inc.

Hello, and welcome to Intellia Therapeutics' third quarter 2025 financial results conference call. My name is Rocco, and I will be your conference operator today. Please be advised that today's conference is being recorded. I will now turn the conference over to Jason Fredette, Vice President of Investment Relations and Corporate Communications at Intellia. Please proceed, sir.

Thank you, Rocco, and hello, everyone. Earlier this afternoon, we issued a press release and filed our 10-Q, outlining our recent business updates and our third quarter financial results. These documents can be found on the Investors and Media section of Intellia's website at intelliatx.com. At this time, I would like to take a moment to remind listeners that during this call, Intellia management may make certain forward-looking statements. We ask that you refer to our SEC filings, available at scc.gov for a discussion of potential risks and uncertainties. All information presented on this call is current as of today, and Intelia undertakes no duty to update this information unless required by law. Joining me on the call are John Leonard, our Chief Executive Officer, and Ed Dulac, our Chief Financial Officer. With that, I'll turn the call over to John. Thanks, Jason.

And thanks to all of you who have tuned in for today's call. In terms of the agenda for today, we'll begin with the status of our NEXE program in ATTR amyloidosis, since that is obviously top of mind for all of you. We then will provide an update on the significant progress we have made with LanvoZ, which is being developed as a potential one-time treatment for patients with hereditary angioedema, or HAE. And we will close with Ed's financial review. First, for NEXE. Since the start of 2024, we've been enrolling patients in magnitude or phase three clinical trial for ATTR amyloidosis with cardiomyopathy. And we've been enrolling magnitude two for patients with hereditary ATTR amyloidosis with polyneuropathy since the start of 2025. Both trials have advanced rapidly, which we believe demonstrates patients' interest in a potential one-time treatment option. On October 24th, a patient was admitted to the hospital after reporting abdominal pain to his principal investigator. This is a patient with ATTR cardiomyopathy in his early 80s who enrolled in magnitude and received a dose of Nexi on September 30th. The patient's labs showed that his AST and ALT levels exceeded three times the upper limit of normal, and that his bilirubin exceeded two times the upper limit of normal. These levels triggered a protocol-specified pause on patient dosing and screening for magnitude in the interest of patient safety. We decided to also pause patient dosing and screening in magnitude 2 as a precaution. On October 29th, the FDA notified us verbally that it had placed a clinical hold on magnitude and magnitude 2. We are now awaiting the FDA's formal clinical hold letter. We were very saddened to learn that the patient passed away last night. We have been advised by the treating physician that this is a case with complicating comorbidities and the case is being further evaluated. Since learning of this case, we've taken a number of actions in the interest of patient safety. For instance, we've mandated that clinical sites collect additional labs from patients in the initial weeks following dosing to detect potential liver elevation sooner. An internal team has been closely reviewing the blinded safety data, baseline characteristics, among other factors to identify potential contributors to the liver-related events seen in magnitude. We've been working with the trial's independent data safety monitoring committees as we consider other potential monitoring and risk mitigation strategies. And of course, we are engaging with global regulatory authorities and other stakeholders to understand their perspectives, concerns, and requirements so we can develop a plan that would allow us to resume enrollment as soon as appropriate. Not surprisingly, given the clinical hold, we are unable to maintain our milestone guidance for NEXE, and we expect to provide an update once we finalize a plan with regulators. Simply put, a lot has transpired over the past couple weeks and in recent hours, and there is still much work ahead. There's a lot of focus on the safety profile of Nexi at this stage, as there should be. That said, we continue to believe in this product candidate's potential to address important omit needs for patients with ATTR amyloidosis. This is based on a few key factors. First, ATTR amyloidosis is a disease with high mortality. While undeniable progress has been made in its treatment, Current therapies only slow its advance, and most patients continue to face progressive morbidity and mortality. Second, we've enrolled more than 650 patients in magnitude and 47 patients in magnitude 2. To date, grade 4 liver transaminase elevations have been reported in less than 1% of all patients enrolled in magnitude. Each of these cases has been observed approximately three to five weeks following randomization and dosing. There have been no grade four liver transaminase elevations in magnitude two. And third, we are highly encouraged by the data from our ongoing phase one clinical trial of Nexi. On Monday, in a late breaker oral session at the 2025 AHA scientific sessions in New Orleans, we will have the opportunity to share longer term data for Nexi that we believe demonstrates its potential to improve various disease measures and mortality Let's move on to WAMBO-Z, which is being investigated in our ongoing Halo Phase III clinical trial for HAE. Over the course of 2025, we've made considerable progress in this trial. Enrollment was completed in September, less than nine months after we dosed our first patient. This puts us on track to share top-line data by mid-2026 submit a BLA to the FDA in the second half of 2026, and prepare for an anticipated commercial launch in the U.S. in first half of 2027. We believe La Vozzi could completely redefine the HAE treatment landscape. We aim to reset expectations and the standard of care for patients living with a stabilitating disease by completely eliminating attacks and the need for other HAE medications for a majority of patients all with one dose. This Saturday, at the American College of Allergy, Asthma, and Immunology Annual Scientific Meeting in Orlando, we will be presenting longer-term safety and efficacy data from all of the patients who received a 50-milligram dose of LomboZ in our Phase 1-2 clinical trial. I'll now hand over the call to Ed, our Chief Financial Officer, who will provide an update on our financial results for the third quarter 2025.