Good morning, and thank you so much for joining us today. I'm Kat Lange, Chief Business Officer at Avidity Biosciences. Today, we are excited to share several updates relating to our Delvotax program for people living with FSHD. Before we get started, I would like to share that this presentation contains forward-looking statements as defined under applicable law. Forward-looking statements involve risks and uncertainties, both known and unknown, which may cause actual results to differ from the forward-looking statements contained in this presentation. You are cautioned to not place undue reliance on these forward-looking statements and to refer to the more detailed cautionary language in this slide and in the risk factor sections of our recent reports filed with the SEC. With that, I'd like to hand it over to our CEO, Sarah Boyce. Thank you, Kate, and good morning, everybody. As you know, our vision at Avidity is to profoundly improve people's lives by revolutionizing the delivery of RNA therapeutics. I want to talk to you a little bit today about Josh. The Josh pictured here was formerly a D1 volleyball player. He is an incredibly talented musician, and we were very privileged, actually, to have him bring his band to play for us at Avidity. One of the things that you will notice if you look closely at Josh is the loss of muscle in his arm, and in particular, the brace that he is wearing on his arm in order to still be able to play in his band. People living with FSHD have been told their entire lives that any muscle they lose will never return. They've also been told that the disease is relentless in its progression. Living with FSHD was described to me recently by a patient talking about it's like Alice in Wonderland falling down the rabbit hole, but never knowing where the bottom is. We're looking to change that. Today's update, we're going to talk about three things. Firstly, we're going to show FDA confirmation of the accelerated approval pathway. The accelerated approval pathway for FSHD is open in the U.S. That is huge news for our Delvotax program, for FSHD, and most importantly, for the FSHD community and the people we are looking to serve. Our primary endpoint for the Fortitude biomarker study is CDUX, the circulating biomarker. We're also going to be presenting to you 12-month top-line data from the Fortitude study, as well as announcing the initiation of our global confirmatory phase III study. With the accelerated approval pathway for FSHD, that now puts us in a position to file three successive BLAs in a 12-month period, starting at the end of this year with Delvotax. We are accelerating Delvotax towards approval as quickly as possible. We have written confirmation from the FDA on the pathway being open, along with detailed feedback on some of the work that we need to do, things like validating the assay for CDUX, as an example. The primary endpoint is reduction of the circulating biomarker, or CDUX, and we'll get into that more today. We are now anticipating top-line data from Fortitude biomark Q2 of 2026 and a BLA submission in the second half of 2026. The second area we're going to focus on today is the data from the Fortitude study. What you're seeing is consistent and reproducible top-line data, the type of data set that actually you've come to expect from Avidity. We're going to be sharing information on CDUX, as well as also the improved functional mobility and muscle strength, along with a favorable safety and tolerability profile. The third thing we're going to be talking about today is that the global confirmatory phase three study has initiated. This study is called Forward, and it's a 200-part effort, 18-month trial with a one-to-one randomization, double-blind, placebo-controlled. Joining me today are my colleagues, Steve and Mike, along with Kat. We are also very honored to have Dr. Jeffrey Stafford join us once again. Dr. Stafford is one of the world experts in FSHD, and we always benefit from the insights that he brings. The agenda for today, Steve will begin, and then we'll hand to Dr. Stafford over to Mike to focus on the biomarkers, and then back to Steve to share about the regulatory study As always, we have time for questions and answers at the end. With that, I'm going to hand over to Steve. Thank you, Sarah. The one-year top-line data we'll present today is from the Fortitude dose escalation cohorts. Before I get into the data, I'd like to take a few moments to recap on the design and objective of those cohorts. Fortitude dose escalation was a double-blinded, placebo-controlled study randomized two- to- one. It was one year in duration. Participants received the first three doses at an every six-week dose interval, which is the interval we're using for the biomarker cohort and our phase three study, and all subsequent doses at a longer, every 13-week dose interval. Consistent with this being the first experience in humans, the primary and secondary endpoints focused on safety and PK, and exploratory endpoints looked at biomarkers. Clinical endpoints included 10-meter walk run, timed up and go, reachable workspace, QMT, and we also looked at patient-reported outcomes. Now we're going to move to the data, starting with the baseline characteristics. Next slide, please. All treatment groups had a diagnosis of FSHD in their twenties, which is typical, and the diagnosis was confirmed genetically among participants. Of note, at baseline, clinical severity score is between 8.5-9.5 out of 15, so this is right in the mid-range, which is where we know patients are most likely to have detectable disease progression over a one-year period. Additionally, not on the slide, the muscle strength at baseline was between 30-35% of predicted normal, again illustrating how severely affected these patients are. Next slide, please. Now turning to safety. As with our other two AOC programs for which we have shared data, Delvotax continues to be very well tolerated and is showing an extremely favorable long-term safety profile. Thirty-nine patients were enrolled in the dose escalation cohorts, and all thirty-nine of those have now rolled over into the open label extension and remain on study, although at the time of the data cut, which was in early May, only thirty-eight have rolled over. There have been no serious or severe adverse events related to study drug, no discontinuations, all adverse events except for two participants have been mild or moderate, and for the most common related adverse events, those occurring in greater than three participants, there were no surprises. Fatigue is an extremely common symptom in FSHD, and the AEs relating to hemoglobin changes are predictable, transient, and are expected based upon targeting the transferrin receptor. There has been one participant with an unrelated severe adverse event that was non-serious. This was a case of herpes zoster or shingles, and there's been another participant with unrelated severe but serious adverse events comprising multiple fractures following a fall. Next slide, please. In a moment, I'll hand over to Dr. Stafford, who will walk you through the exciting and unprecedented clinical endpoint data from the Fortitude dose escalation cohorts. However, before that, a couple of points of orientation as you look at the clinical data slides. We showed you last year that suppression of DUX4-regulated circulating biomarker was identical at the two and the four-mg/kg doses. And since DUX4 expression is the underlying cause of the disease, we would therefore not expect four-mg/kg to be any better than two-mg/kg, and that's exactly what we saw. So we've pooled the Delvotax cohorts for the main presentation of the data. However, we are going to show you the data broken out by dose a little later in the deck, and there are no surprises. As I mentioned just now, they're the same as expected. Also, as we go through the data, you'll see in the footnotes that we excluded one participant from all of the analysis since they had an unreliable baseline, and a second participant was excluded from the 10-meter walk run since they had an injury on study requiring the need for a walker, which confirms the test. The data you'll see over the next several slides is truly remarkable. The [Guess] treated participants have improvements in muscle function, mobility, and muscle strength at one year as compared to placebos, and they've also showed improvements in multiple patient-reported outcomes. Perhaps even more remarkable than the data is the fact that this is at a longer dose interval of every 13 weeks than we selected for the biomarker cohort and our phase three study, where we're giving Delvotax at a shorter dose interval of every six weeks in order to ensure that DUX4 is silenced over the entire dose interval. Without further ado, I'll introduce Dr. Stafford. Dr. Jeffrey M. Stafford is a distinguished professor of neurology at the University of Kansas Medical Center. Dr. Stafford is a leading expert in the field of neuromuscular diseases, including FSHD, Duchenne muscular dystrophy, spinal muscular atrophy, and myotonic dystrophy. His research interests are focused on developing new disease-relevant outcome measures to assess patient-reported disease burden, functional impairment, and physiological changes in muscle. Dr. Stafford is also the co-PI for the FSHD Clinical Trial Research Network, a global network of clinical science that is advancing the understanding and treatment of neuromuscular diseases. Dr. Stafford, over to you. Great. Thank you very much. I want to thank Avidity for giving me the opportunity to share this top-line data on strength, motor function, and patient-reported disease impact from the Fortitude trial. I do want to take a moment and just reflect. This is our first molecularly designed gene-targeted drug for FSHD, and I hope you'll agree with me that at the end of these presentations, we're really seeing Delvotax engaging its target and showing a response at multiple levels of clinical response, including the molecular, the circulating biomarker of muscle damage, and what I'm going to show you here, which is really at the whole person level. When we talk about FSHD, we're talking about one of our most common muscular dystrophies with about 27 impacted individuals in the United States. The onset typically occurs in the teenage or early adult years, but it can impact people throughout the course of their lifetime. It causes chronic and progressive weakness that can lead to loss of the ability to walk or maintain a job, with about 20% of people losing that ability to walk and requiring a wheelchair. There are no approved therapies for FSHD, so it represents a really large unmet medical need. There are two genetically distinct causes for FSHD, type- 1 and type-2, but they converge on a common downstream pathway, which is the aberrant expression of the transcription factor, DUX4, which is believed to cause the disease through a toxic gain of function, and DUX4 is the target for Delvotax therapy. Next slide. When we think about measuring a response to therapy, it's essential that we really put this into the context of what the patient experiences. What we're showing here is the data from a very large survey of people with FSHD called the Voice of the Patient Report. The most prevalent impactful symptom categories that patients identified were related to the loss of strength, function in the upper extremities, mobility, gait, core function, and the results of that, things like pain and fatigue. When we ask people short of a cure, what would be important for you for a treatment for FSHD, the vast majority of people mention slowing or stopping the loss of muscle function or regaining strength or their motor function. Indeed, when we think about what people are worried about as they think about their future, these are also related to the impact of that loss in strength, losing independence, losing the mobility or the ability to walk, and becoming a burden on their families. Next slide. When we're thinking about functional motor ability, we're going to start with an outcome measure related to mobility, the 10-meter walk run. This is the most sensitive motor functional task we have in an average adult population to the natural history of the disease. It's the time it takes to go 10 meters or about 30 feet as fast as they can safely do. All of the slides are orientated in the same way. The x-axis is going to be the change from baseline, with the dotted line representing no change. The orange bar is Delvotax treated participants. The gray are the placebo, and the bars are the standard error bars. You can see here improved functional performance on the 10-meter walk run for Delvotax treated participants compared to the placebo group. Putting this into some context of what we know about the disease, performance on the 10-meter walk run from zero to four seconds would be essentially normal. From five secs- 12 secs, people are losing function, but they're still ambulatory. At around 12 secs, they're starting to need things to help them get around, AFOs, canes, or walkers. At 15 secs, you may see people requiring wheelchairs for distance or to get around. Preservation of time on the 10-meter walk run is likely to predict a delay in the need for those assistive devices over time. Can we go to the next slide? The other mobility measure that we're showing you here is called the timed up and go. This is traditionally a measure of balance. It's the time it takes for someone to get up from a chair, walk with their casual gait for 3 meters, then they turn 180 degrees, walk back to the chair, and sit down. You can see here improved performance on the timed up and go compared to the placebo group. Again, like the 10-meter walk run, that preservation in time to perform this task likely is related for the need for these other mobility aids or assistance later in time. Can we go to the next slide? Switching gears now and looking at a measure of strength here, it's quantitative myometry. This is measured with a handheld digital force transducer in standardized positions to isolate different motor tasks at the elbow and knees for flexion and extension, at the ankle for dorsiflexion, and at the shoulders for abduction and rotation. They're then summed together to create a composite score. These are muscles that are of particular interest in FSHD and are often impacted. You can see here preserved strength compared to the placebo group in patients treated with Delvotax. To put this into some sort of context, that 3% loss that we're seeing in the placebo group here is roughly similar to what we see in our natural history study in an average population of adults. Next slide. Switching gears now and thinking about function in the upper extremities, we're using an outcome measure called the reachable workspace. This uses a 3D stereographic camera in standardized positions to detail the volume of space that someone can reach into in different quadrants, as illustrated here. What we're presenting here are those upper quadrants, quadrant one and three. This would be related to activities like taking a shirt on and off or taking a gallon of paint and putting it up on a shelf. You can see that the Delvotax treated patients improve in their reachable workspace area compared to the placebo patients who are decreasing in that reachable workspace area over a similar period of time. When we think about what happens in our natural history study, at a year to 18 months, we see people losing around 2%-3% of their reachable workspace area. Can we go to the next slide? The slides that we are showing for the next couple of slides are Forrest plots detailed to show you the dose response. What we are seeing here is the difference between the treated patients and the placebo group. In orange is for the 2 milligram per kilogram treated cohort. In blue is for the 4 milligram per kilogram treated cohort. This is just showing there are improvements in performance compared to placebo for the 10-meter walk run, as well as the timed up and go for both of the dose cohorts. As had been mentioned earlier, that response size is roughly equivalent between the two doses. The dotted line that you see on these graphs is the minimally clinically important difference as estimated in our natural history studies. How we get at that value of what would be a meaningful change, we use statistical approaches. We relate the change in the 10-meter walk run or the timed up and go to a change on a functional measure where each of those changes of one would be the loss of some key function, like getting up from a chair or the ability to run. You can see here already we're seeing an effect size for the two dose groups that is slightly above that size that we would think would be minimally clinically important. Next slide. Here you can see the dose response for the 2 and 4 milligram groups compared to placebo for the quantitative myometry strength testing, as well as the reachable workspace area. For both, you can see that the dose favors treatment over placebo and is similar whether we're talking about the 2 or 4 milligram group. This is part of the reason why the dose of 2 milligrams per kilogram was chosen for the key registration in phase three studies that will be described later. Next slide. We're switching gears now, and here we're going to start looking at the patient-reported disease impact. Again, we're showing the change from baseline, the dotted line representing no change. Improvement on these outcomes is to the right, as the green arrow illustrates. Delvotax treated participants are in orange, and placebo are in the gray. You can see improvements in physical function and fatigue for the treated participants compared to the placebo group. You can see similar improvements using the NorAQUAL, looking at the upper extremity score, as well as, interestingly, a score for sleep disturbance, which really favored treatment. Can we go to the next slide? That summarized and then moved forward, you can see equates to improvements in pain and overall health on the left side of the graph here. On the right, you can see that corresponding to the patient's own report of change with global improvements in their change and their sense of the severity of FSHD over the course of their participation in the trial. Can we go to the next slide? In summary, I think we're seeing Delvotax improving strength and function across multiple modalities. Upper extremity function is measured by the reachable workspace. Mobility is measured by the 10-meter walk run and the timed up and go, as well as underpinning both of these a sum score of key muscles in FSHD showing strength. This is then reflected in the patient-reported disease impact and in the setting of what is a favorable safety and tolerability profile so far in the development program. Indeed, I think this is reflected in one of the patient interviews that I had the privilege to be able to hear. These are exit interviews for people who participated in the trial after their participation was complete for the first year. This participant pointed out that the key thing that he felt he gained from his participation in this trial was the ability to physically do activities with his children. In particular, he mentioned there was a park near his house with a quarter-mile track. Before entering the clinical trial, he was unable to go around that track with his children. After his participation in the trial, he could go around that track two times without getting too fatigued. This had really changed this person's outlook for the future. Where before they were concerned that they would be a burden on their family and be unable to participate in physical activities like this, they now had some hope that there would be a drug that could enable them to be physically present in their children's lives. I'm going to turn it over now to Michael Flanagan, who will be talking about a circulating biomarker, I think very important for this program, as you will hear, but really for the overall field of FSHD research. Thank you. Thank you, Dr. Stafford, and good morning, everyone. Having seen how Delvotax drives meaningful improvements across mobility, strength, and quality of life, I'll now focus exclusively on our biomarker, KHDC1L, or CDUX, which stands for circulating DUX4-regulated biomarker. We believe CDUX is a breakthrough for the FSHD community and for the field as a whole. Next slide. CDUX is a circulating biomarker that's significantly elevated in people living with FSHD, typically six- to nine-fold higher than healthy individuals. The elevated levels in FSHD are directly linked to worsening of disease and loss of muscle function. In our 42 trials, Delvotax treatment led to rapid and statistically significant reductions in CDUX. This effect was mirrored by reductions in creatine kinase, or CK, a marker of muscle damage. Perhaps most importantly, placebo patients who crossed over to Delvotax in the open label extension showed rapid decrease in CK levels, demonstrating the consistency and reproducibility of Delvotax. The effect on CDUX and CK following Delvotax treatment, along with the functional data just presented by Dr. Statlin, established CDUX as a promising marker of clinical activity and a viable path towards accelerated approval. Let's take a closer look at CDUX and its connection to DUX4 for the underlying cause of FSHD. Next slide. Through a rigorous multi-year discovery process in collaboration with Dr. Tapscott's lab at the Fred Hutchinson Cancer Research Center, a world-leading lab in FSHD, we leveraged muscle biopsies, patient-derived cell lines, RNA sequencing, plasma samples to identify KHDC1L as a circulating protein we call CDUX that is implicated in germ cell biology and apoptosis. The CDUX transcript is directly regulated by DUX4 and contains DUX4 binding sites in the promoter region. We call DUX4 a transcription factor, and it binds to this promoter. We first identified CDUX as a highly expressed transcript in FSHD muscle biopsies. In fact, CDUX for KHDC1L is a member of our Avidity 4-gene panel and, of course, the 41-gene panel. The real eureka moment was finding that CDUX was secreted from FSHD patient-derived myoblastin cell culture. Most importantly, CDUX was found to be significantly elevated in plasma samples from our FSHD participants that enrolled in the resolved natural history study and our 42 trial. What makes CDUX truly valuable is the translational bridge. CDUX levels reflect DUX4 activity across all the muscles in the body, which means it tracks not only the presence of disease, but its progression and therapeutic response. This makes CDUX a powerful bridge between DUX4 activity and clinical outcomes. Next, let's examine CDUX levels, how they change over time in response to Delvotax treatment. Next slide. Here we show the longitudinal biomarker data from the 42 trial. On the left, you'll see CDUX levels. On the right, CK. Blue arrows indicate dosing of Delvotax. Gray colored represents placebo cohort, and orange represents the combined Delvotax treated participants at both 2 and 4 milligrams per kilogram. From the early time points and through 12 months, Delvotax induced rapid and consistent reductions in both CDUX and CK. Nearly every time point achieves statistical significance with p-values less than 0.005. I'd like to focus your attention on the dose schedule and the associated change in DUX4 levels. These data were essential to informing our dose schedule of 2 mg per kg every six weeks for the 42 biomarker cohort and the forward phase 3 study. As you can see, during the first three months, we dosed every six weeks, allowing us to quickly get to steady-state levels of silencing RNA. As you can see, between day zero and three months, we see a rapid reduction in CDUX. Interestingly, and many of you probably have noticed, as we transition FSHD participants to longer dose intervals of every 13 weeks at month three to month 12, we see that CDUX begins to return to baseline, suggesting that DUX4 is leaking through the suppression and activating CDUX. You can also see the rise in CK levels. Now, remember, you have to think about treating FSHD as a prevention model. What that means is that you always have to have silencing RNA in the muscle cell to prevent DUX4 expression. If DUX4 gets expressed, it's too late since it activates a cascade of events that poisons the muscle. This connection between DUX4, CDUX, CK, and Delvotax treatment strongly supports the potential for CDUX to reasonably predict clinical activity. It is this strong connection that makes CDUX our primary endpoint for the 42 biomarker cohort. Finally, I want to give you a sneak peek of our open label extension data and what happens after placebo patients transition to Delvotax. Please focus on the light gray panel. Here you can see CK levels in FSHD participants who initially received placebo and then crossed over to our Delvotax treatment in the open label extension. As soon as treatment began, the CK levels dropped rapidly, consistent with the pattern seen in those who received Delvotax from the start. Interestingly, you can also see that patients that were on Delvotax that had gone to a longer dose interval, when they started receiving the six-week dose interval, you can see they dropped immediately again on CK levels, indicating that the PK of Delvotax is good at the longer or the shorter intervals. These data not only confirm the biological activity of Delvotax, but also further strengthen the link between the therapeutic exposure and the biomarker response. To wrap up my section, let's summarize how all of this supports the case for CDUX as a compelling candidate for accelerated approval. CDUX meets two key criteria needed for validation as a potential surrogate biomarker endpoint. First, there is a clear mechanistic link to FSHD. CDUX is a direct transcriptional target of DUX4, the root cause of disease. Elevated CDUX levels correlate with disease severity and disease progression. Second, CDUX is linked to clinical activity. We have shown statistically significant reductions in CDUX and CK in response to Delvotax. These changes correspond to improvements in functional mobility, strength, and quality of life. Finally, and most importantly, the FDA has provided written confirmation that an accelerated approval path is open. CDUX is our primary endpoint in our 42 biomarker accelerated cohort. The cohort is completely enrolled with anticipated top-line data in Q2 2026. Now I'll pass it on to Steve, who will present our accelerated and global confirmatory phase three trial. Thanks, Mike. If we can move to the next slide, please. Over the next few slides, I'm just going to present the outline, clinical trial designs, and the main objectives and endpoints for both the 42 biomarker cohort, which of course is targeting accelerated approval, and in a moment for the Forward confirmatory global phase three study. The 42 biomarker cohort is double-blinded, randomized, placebo-controlled. The dose for Delvotax is 2 mg per kg every six weeks. That's our go forward registrational dose. Actually, the phase three study also uses the same dose regimen. It's randomized two to one, active to placebo, and it's actually already fully enrolled. We completed enrollment back in March of this year with 51 patients. The study is 12 months long, and the data readout for the top-line data will be at the end of that 12-month period. There are no interim analyses. Given that the study is already completely enrolled, we now just need to follow the patients up, analyze the data, and we're expecting the top-line readout in Q2 of next year, followed by a BLA submission anticipated in the second half of next year. Not very long now. All participants, when they complete the end of the placebo control period, are able to enroll in an open label extension study where everyone will receive active drug at the go forward phase three regimen of 2 mg per kg every six weeks. Can we move to the next slide, please? This slide now just looking at some of the key objectives and endpoints for the study. We're going to move left to right. Most of what's in the left-hand panel I've already covered. Just to point out maybe one thing that for this study, and also in fact for the Forward phase three study, we've extended the age limit down through to 16. We're pulling in pediatric participants in the upper adolescent range there. We're now moving to the middle panel. Our key biomarker endpoints primary is CDUX, and creatine kinase is a secondary endpoint for this study. Then, of course, we'll be looking at the clinical endpoints that we've already shown you today, 10-meter walk run, timed up and go, and quantitative muscle testing. Of course, we've already shown very strong data across all of these endpoints showing that Delvotax is having a profound effect, and we would firmly expect to reproduce those effects in the biomarker cohort. Finally, we'll also be looking at patient-reported outcomes, again, where we've already seen very strong effects of Delvotax treatment. Next slide, please. Now moving to the Forward global confirmatory phase 3 study. This study is actually already underway. We started it very recently. Again, it's a double-blinded, randomized placebo-controlled study. The dose, as I already mentioned, is 2 mg per kg every six weeks, the same as the biomarker cohort. The duration of placebo control is 18 months. The key endpoints will read out at the end of that 18-month period. There is no interim analysis. All patients are able to roll over to an open label extension study. Randomization for this study is one-to-one, and we're targeting about 200 participants in total. It's a global study, so we'll be recruiting in North America, Europe, and Japan. Just to remind you, the FDA is in alignment with the study design that we're presenting today. Next slide. This is my last slide, just hitting on some of the key objectives and endpoints for the Forward study, starting again in the left-hand side. Most of this I've already covered, but just to point out that, again, we're going down into the upper adolescent age range of 16, and we're going to have about 45 sites globally in order to enroll the study. Moving to the center panel, the key registrational endpoints, the same endpoints as you've seen, remarkable data for today, 10-meter walk run, timed up and go, and quantitative muscle testing. At this point in time, quantitative muscle testing is assigned in the protocol as the primary endpoint. We had to select something in order to submit the protocol, so we've put that in essentially as a placeholder. We have designed the study with a huge amount of flexibility, and our plan is to actually confirm the primary endpoint and the order of the subsequent hierarchy of testing closer to the completion of the study. In order to do that, we are going to be having a couple of key additional data readouts. One is the final 12-month data from the biomarker cohort, where, of course, we are testing these endpoints in a larger number of participants, and we can pool that data with 39 participant data that we already have from that study for a total of about 90 Delvotax treated participants. That will be very informative in terms of how these endpoints are moving over time. The other data points are from the natural history studies. The MOVE and the RESOLVE natural history studies are actually ongoing, and the data from those will be maturing over the subsequent year or so. We will look at both of those additional sources of data before we finalize the primary endpoint, and then we will update the statistical analysis plan ahead of unblinding the phase three study in order to select our final primary endpoint and the order of testing for the key sectors. Additional endpoints that we will be testing in the study, again, PROs, which we have shown you data for today. In this study, we are not taking any muscle biopsies. That makes the study much easier to conduct. It makes more centers eligible and is easier on the patients. Of course, our circulating biomarker CDUX and creatine kinase, these are blood tests. We're taking blood in the study anyway, so we'll be collecting the CDUX and CK at multiple intervals during the study. That's my last slide, and I'll now hand over to Sarah for closing remarks. Thank you, Steve. Today we've covered three things. Firstly, the opening of the accelerated approval pathway for FSHD. We've also shared with you more details on the cyclase and biomarker CDUX that is our primary endpoint, now tracking for three BLA submissions in a 12-month period. We also shared the consistent and reproducible data from the 42 study, as well as the initiation of Forward, our global confirmatory phase three study. Today is a big day. It's a big day for the team here at Avidity that work on our Delvotax programs. It's a big day for all of us at Avidity. It's also an important day and a big day for all of the investigators in our study, the sites, and the participants who volunteered to be part of the 42 study. We are so grateful for their time and their commitment. Most importantly, for people living with FSHD and for the FSHD community, this is a huge day. This is the opening of an accelerated approval pathway for this patient community. It's been enabled by the discovery of the CDUX biomarker, and it now being the primary endpoint for the 42 biomarker study. At Avidity, our vision is to profoundly improve people's lives by revolutionizing the delivery of RNA therapeutics. We understand the deep responsibility that we have in delivering on that vision. One of the patient interviews from participants in the 42 study, and one that struck me, was a young woman who talked about how she'd largely stepped back from going to events, from actually leaving her house for fear of falling, for fear of having an incident in being outside her house, and was recently actually able to attend a friend's wedding. What she described was, "I'm now able to say yes to life." It has not lost on the Avidity team for one moment the urgency that we bring to our work, and the urgency of science, and the importance of delivering on our vision. With that, I'd like to ask my colleagues to rejoin me, along with Dr. Stafford, and we can move into Q&A. Excellent. Well done to the team, and thank you, Dr. Stafford. We've got quite a few questions coming in here. We will start off with the first question from Eric Schmidt at Cantor. "Congratulations on the terrific progress," he says, "and I have two questions here. The first one will go to Dr. Stafford. You showed data across several clinical and biomarker endpoints. What would you say is the most convincing evidence in support of Delvotax's activity?" Yeah, I mean, I think that's a really good question, but you know from my point of view, what's most convincing about the program so far isn't any one outcome, but rather the consistency that we're seeing across these different outcomes. The evidence that it's hitting its molecular target and having an effect with the circulating biomarker. This is reflected in these very strong early signs that we're starting to see some separation occurring for things like strength and function, and then the corresponding patient reports of disease impact. It is that consistency that is most encouraging. Excellent. Thank you. The second part of the question I will pose to Steve. "How do you envision the CDUX assay being used in future drug development programs or clinical practice?" The CDUX assay is really game-changing. It allows for tracking of DUX4 activity over time. For us, hitting the target, it was really instrumental in allowing us to do a dose selection and dose measurement selection and selecting the 2 mg per kg every six weeks moving forward to our registrational studies. I think the data that you have seen today really tells us that we have got the right dose of measurement for the go forward studies. I would envisage CDUX being equally important for any future phase three studies. Now that we've shared what CDUX is, that allows the whole community working in this disease to benefit from this breakthrough. Excellent. Thank you. Okay, our second question comes from Jo Wards at Leerink Partners, who says, "Congratulations on the continued great progress." The first question to Michael Flanagan. "Where do you expect CDUX to end up relative to baseline when Delvotax is administered more frequently? So how deep of a reduction in CDUX do you think you can achieve in the phase three study?" Yeah, CDUX has been a huge discovery for us, and we've been really excited to be able to share it now. We realize that there's a responsibility to share it. We hope that everyone will be able to start using it. Now, thinking about CDUX and KHDC1L, how does it look? I guess I'd go back initially to the preclinical data that we showed, I think, last year or maybe the year before, where we have a mouse model of overexpressing DUX4. What you see is just small changes, actually, of about 20-30% changes completely bring these mice back to essentially a wild type phenotype. They look exactly like wild type mice. They have the same strength, they run the same speed, have the same force. It really doesn't take very much changes. Looking at what we've shown is that we see somewhere around 30-40% change right now at a 2 mg or 4 mg per kg every 13 weeks. What you're seeing is that translates to increased mobility, improvements in strength, improvements in quality of life. Really, it doesn't take very much of a change for these gain of function like FSHD to see an effect. We expect that we may drive it down further, but at the same time, we're seeing effects already Excellent. Thank you. The second part of the question goes to Steve. "How are you thinking about the choice of primary endpoint in Forward? Can you help us think about the relative importance of improvements in strength versus function in FSHD, and why QMT is a place to start?" I'd first say that all three of the key endpoints, QMT, 10-meter walk run, timed up and go, are all very well powered. We have great power across all of them. In selecting what we were going to have as our basically placeholder for the phase three study, we felt that at the time we were writing the protocol, QMT was an appropriate choice. As I said, we've kept things open so that we can refine that choice and further de-risk the phase three program prior to unblinding of the study. There's no single endpoint that's going to completely capture the disease for all patients. Strength, of course, affects mobility. Both strength and mobility are important in terms of falls and other things. We've heard from Dr. Stafford about just how important to the patient that was in the park with his family being able to have increased mobility. For another patient, the most important thing might be large improvements in muscle strength. The important thing is that across the endpoints that we're looking at, we're trying to capture many different aspects of the disease. Across the patient-reported outcomes, that collects additional, perhaps less tangible in terms of ability to measure with a physical test, elements of the disease. We also do exit interviews from all of our studies where we allow the patients to describe the impact of the drug in their own voice. All of those things together are really what's required to properly appreciate the impact of Delvotax on the patient's lives. Great. Thank you. Our next set of questions comes from Ritu Barooah at TD Cowen. I'll ask the first question to Sarah. "Can you give us the status of your current commercial efforts across DMD and how you plan to layer on DM1 and FSHD in 2027? What are the timelines for hiring, disease awareness campaigns, and deployment? Thanks, Ritu. Great question. We are well underway with regards to our preparations and building our commercial organization. In actual fact, we have some parts of it already fully built, in particular focusing around supply chain, patient services, site of care. We also have an MSL team that is fully built and in place as well. One of the big elements actually around preparation and around preparation now for three successive launches is also our work in tech ops. We're making a lot of drug, and that is also very much underway and ramped up. One of the other elements I would also add, this is three launches in the same therapeutic category. What that enables is a very efficient infrastructure build in that everything, for example, that we do to build our patient services organization and all the piping that goes around that, then we're able to layer on each launch on top of the other. It is actually a very efficient commercial operating model that we're building. Thank you. Second part of the question will go to Dr. Stafford, please. "Is preservation of function a compelling treatment effect for FSHD patients? What percentage of the diagnosed population do you think is so progressed that they wouldn't consider Delvotax treatment? And conversely, who is the ideal patient that you estimate right now with just the data in hand?" Right. This is a question we get frequently. I think preservation of function is vitally important to people when you're talking about their lives. Patients will tell you at any stage of the disease when they come in that the function they have is really important to them, and preserving that is a worthwhile goal. I think when we think about a treatment like this, there's really a pretty full spectrum of patients for this disease that are going to be amenable to the treatment. Even someone who's no longer able to walk and they're having to use a wheelchair, they're still using their arms. They're still breathing. There are things that you can preserve that would be really important to the patients. I do think there's a large percentage of the population that would benefit from the treatment, but I think the individual decision for each patient will need to be made by their individual providers who know them best. Great. Thank you. Our next question comes from Yannon Ju with Wells Fargo. And he says, "Congrats on the regulatory and 42 data updates." Thank you, Yannon. So question to Steve. "Could you talk about the powering assumption for the biomarker cohort's 12-month CDUX primary endpoint?" At a very high level, extremely well powered. We do not tend to give large details out about our powering assumptions other than to say we make conservative estimates so that we do not have any surprises at the end of the day. As we do the analysis, adjusting for things like baseline characteristics and things like that allows you to dial in a bit more power. We are extremely well powered for the biomarker cohort and actually for the Forward study as well. All three of those key endpoints are extremely well powered. Thank you. Our next question comes from Gina Wang at Barclays. Also to Steve. "Since you already completed pivotal cohort enrollment back in March of 2025, did FDA endorse the 51-patient two-to-one randomization, assuming you have met with the FDA since then?" Yes. We have spoken to FDA about the design of the biomarker cohort, and FDA has indicated there is a very clear path forward for the accelerated approval. Actually, what we were looking for in that interaction was to get FDA's feedback on the things that we needed to do to really validate the biomarker and do the technical validation. They have essentially given us test questions. We are working our way through that, and we will have completed those validations well ahead of the time to file. Thank you. We have a question that came in for Sarah. "So three BLAs in 12 months is a lot. How will your team achieve that?" It is a lot. Most importantly, it's really important from an aspect of the patient community. I think one of the areas that comes in here is the efficiency that we have as a team in that all three are in the same therapeutic area space. We talk to Neurodivision One at the FDA a lot across three programs. It also is where you start to see some of the beauties and power of a platform technology. As from one submission to another, it's the same antibody. In some cases, in the case of Delvisra and Delvotax, we're using an siRNA approach, obviously for Delvotax, a PMO approach. There are a lot of synergies from one file to the other that we can essentially lift and put into the next submission. It is. I think actually three BLAs in 12 months is something that you do not see very often in our industry. I do not want to use the word unprecedented because we were trying to find examples of other biotechs who have done this, and we could not immediately find any. Certainly, this is something that is really game-changing and I think really separates the work that we are doing here and the importance of it for the patient community. There is a big element where one of the keys as to why you can do it, well, there are three things. Firstly, drugs work. Secondly, it is around the efficiency that you get from a platform technology. Thirdly, in that they are all in the same therapeutic category. Great. Thank you. Our next question comes from Louise Chen at Scotiabank, and I will pose this one to Mike. Why do you think the four milligram per kilogram dose didn't show any meaningful additional benefit?" Yeah. This is a question that we get often. You need to think about FSHD different than other diseases. Other diseases, you think about it like, "I need to knock down a protein and knock it down".FSHD is different in the sense that it's a prevention model. You need to have drug on board to prevent DUX4 from being expressed. If you have more drug on board, it doesn't suppress DUX4 any more than having it on board. What you need to do is reach a drug concentration in muscle cells that blocks DUX4 expression. Remember, anytime DUX4 gets expressed, it activates a downstream cascade of events that then poisons muscle. If you can at all times knock it down or keep it silenced, that's all you need. That's really what informed our 2 mg per kg dose every six weeks. If you remember, we went from 2 mg per kg and 4 mg per kg every six weeks, then we transitioned to a longer interval. We found that with the longer interval, you started again seeing that rise in CDUX levels, which indicated to us and really reinforced our decision to go with more frequent dosing, again, to completely suppress DUX4. Great. Thank you. We are getting towards the end of our time, so I think we just have one final question here that I will pose to Steve. This one comes from Kostas at Beamo. "Steve, could you comment on what FDA would require for approval beyond meeting the primary endpoint?" Yeah. The bar for an accelerated approval is showing that your biomarker is reasonably likely to predict clinical benefit. There are a number of steps that you take to do this. We've already made great headway in many of these. One, the biomarker showing that it's actually related to the disease. We've looked at CDUX in healthy volunteers. We've looked at CDUX in patients with FSHD in the natural history. We've looked at CDUX in patients with FSHD in the Fortitude study. We see that the patients are about six- to nine-fold higher than healthy volunteers. In healthy volunteers, it's barely detectable. For patients, both in the natural history and in the Fortitude study, there's a clear separation that's statistically significant from the healthies. Actually, the Fortitude levels and the natural history levels were basically overlapping. The next thing is to show that actually higher levels of DUX4 have a faster disease progression than lower levels of DUX4. We have already shown correlations there. As we go through the cohort, we go through the biomarker cohort, we will be able to draw the links again between the CDUX expression and changes in the clinical endpoints. As we knock down CDUX, we will be able to see changes in clinical endpoints as we have already shown. That further reinforces the reasonable likelihood to predict clinical benefit. Excellent. Thank you, everyone. That was our final question. I would like to hand it over to Sarah for closing remarks. Thank you, Kat. Thank you, everyone, for joining us this morning. There we go. The video started. Thank you, everyone. Thank you for joining us this morning and for being on the journey with us and the journey around delivering on our vision to be able to make a profound impact in people's lives by revolutionizing the delivery of RNA therapeutics. The urgency of science and the urgency to deliver for the patient communities that we're looking to serve is not for one second lost on the team here at Avidity. We look forward to sharing with you more future progress over the course of the years. Thanks so much and have a great day.