Intellia Therapeutics, Inc.

Good morning and welcome to IntelliA Therapeutics' second quarter 2025 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. If anyone requires operator assistance during the conference, please press star zero on your telephone keypad. I will now turn the conference over to Brittany Chavez, Senior Manager of Invest Relations at IntelliA. Please proceed.

Thank you, operator, and good morning, everyone. Welcome to IntelliA Therapeutics' second quarter 2025 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 in 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 this call, IntelliA management may make certain forward-looking statements and ask you to refer to our SEC filings available 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 Leonard, Chief Executive Officer, David Lebwald, Chief Medical Officer, Ed Dulac, Chief Financial Officer, and Birgit Schultz, our Chief Scientific Officer, who will join for Q&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. With that, I will now turn the call over to John, our Chief Executive Officer.

Thanks, Brittany. Good morning, everyone, and thank you all for joining us today. 2025 is proving to be a year of excellent execution and exciting clinical updates. Thus far, we're meeting or exceeding all the objectives we set for ourselves, which sets us up well for the second half of the year. Financially, our restructuring is delivering the benefits that we expected, which support a runway through several major milestones and into the first half of 2027, when we expect to be launching Long Vosie for HAE. Clinically, presentations of the longer-term follow-up data presented from our ongoing trials suggest our lead programs have the potential to set new standards for the treatment of HAE and for both the polyneuropathy and cardiomyopathy manifestations of ATTR amyloidosis. Also, from an operational perspective, all three Phase III studies across Long Vosie and MEXIE are enrolling faster than we expected. We're benefiting from strong interest from patients and physicians. That interest, coupled with our team's excellent execution, positions us to accelerate guidance we set at the beginning of the year. We now anticipate completing enrollment earlier in our HAE and ATTR polyneuropathy programs than previously thought, and we expect that we will enroll more patients this year in our cardiomyopathy program than originally planned. Among the many favorable updates we provided across our programs today is our decision to increase enrollment to approximately 1,200 patients in magnitude, our Phase III study evaluating MEXIE in ATTR cardiomyopathy, subject to health authority review. Expanding the patient number in the study will provide a more robust data set, particularly in the stabilizer stratum, which we know will be very important to patients, clinicians, and payers. We believe MEXIE, in combination with the stabilizer, will provide meaningful clinical benefits beyond treating with only a stabilizer, which will be a key differentiator in the commercial setting. It's also important to note that the improvements gained from a larger study size do not impact either our previously projected enrollment or our cash runway. When we initially designed our study, we recognized that the TTR treatment landscape could change as new agents became available during our Phase III program. We also knew that we were well positioned to adapt to changes in TTR treatments because of the timing of our program. Now, with the benefit of recent clinical readouts, we know how to best capitalize on the rapid, deep, and consistent TTR reduction achieved with MEXIE to make it into a formidable and differentiated competitor in this large and growing market. Based on the strong enrollment magnitude, we also said this morning that we are now targeting at least 650 patients chemilatively by year end. Again, we believe this increase, relative to our prior guidance, for more than 550 patients is made possible by the operational excellence of our team. But importantly, it also reflects the enthusiasm from investigators and significant demand from patients to participate in the trial. Let's turn to magnitude two for the treatment of hereditary ATTR polyneuropathy. We've seen the same high level engagement from patients and physicians in response to the promising data and potential of MEXIE. Enrollment continues to track ahead of our initial projections, and we've refined our guidance now expecting the complete enrollment of the trial in the phase three study of LAMBOZI, formerly known as NTA 2002. Today, we announced we have ended recruitment and expect a complete randomization during the third quarter. This milestone, consistent with our market research, reflects the high need in the HAAE community despite existing treatment options. We believe LAMBOZI's maturing data and profile as a one-time therapy administered in an outpatient setting resonates with patients and physicians. Building further on our strong momentum, we shared positive interim data throughout the quarter that continues to support the growing body of evidence for both LAMBOZI and MEXIE. David will expand on that in a moment. We also look forward to sharing more clinical and operational milestones from our lead programs later this year. The positive developments within our studies have been matched by the progress we have made in building our commercial and medical teams required for a successful launch. Senior leadership positions hired within the commercial and medical affairs organizations during the first half of the year include head of US sales and head of commercial operations, as well as several additional senior leaders with responsibilities for commercial data and field operations, marketing, pricing, patient services, market access, forecasting, and medical communications.