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Goldman Sachs 45th Annual Global Healthcare Conference

Jun 12, 2024

Moderator

Welcome again to the 45th Annual Goldman Sachs Healthcare Conference. We're thrilled to be joined today by the team from Athira Pharma. And maybe we'll just start, 'cause I know there's also been some, like, leadership changes in the past year, etc. , with an introduction of each of you and an overview of the company and pipeline. How about that?

Andrew Gengos
CFO, Athira Pharma

Andrew Gengos, I'm Chief Financial.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

I'm Rachel Lenington. I'm Chief Operating Officer and Chief Development Officer.

Javier San Martin
CMO, Athira Pharma

I'm Javier San Martin, Chief Medical Officer, and I am the new kid on the block. I had.

Moderator

Perfect.

Javier San Martin
CMO, Athira Pharma

I joined a couple of weeks ago.

Moderator

Yeah, perfect. Who wants to provide an overview of the pipeline?

Andrew Gengos
CFO, Athira Pharma

Rachel, why don't you go?

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

Yeah, I can provide a brief overview. So at Athira, we're really taking a different approach for neurodegeneration. We're focused on a natural repair pathway that your body uses to take care of your nerve cells and to help injury within. And we're really focused on repair and protection and targeting that. In neurodegenerative diseases such as Alzheimer's or ALS, that shifts, the balance shifts to neurotoxicity, and our small molecules target that and help to restore the balance and improve neuronal health and protect the neurons.

Moderator

Maybe you could go a little bit more explicit in terms of the exact mechanism of action for Fosgonimeton, just to kind of, like, get us started here.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

Yeah, no. Great, great, great question. Fosgonimeton is our lead molecule. It's in phase II, III Alzheimer's disease study, and it is focused on positively modulating the HGF neurotrophic signaling system, which really is something that helps to decrease inflammation, improve mitochondrial function, and maintain neurons. And so, this target we've shown in both preclinical and phase I and phase II studies, to have both effects on pathology, the disease pathology, but also on clinical outcomes.

Javier San Martin
CMO, Athira Pharma

Do you want me to add a few things?

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

Yes.

Javier San Martin
CMO, Athira Pharma

Again, I'm new to this field, and I'm fascinated about this. People ask, "Hey, this is not known. What's the mechanism of function?" And when I started to read the history of this, HGF may have been known for many, many years. It's really critical in prenatal development of the CNS and postnatal. It's located in many areas of the CNS, definitely in the hippocampus, definitely in the neuromuscular junction, which I think is critical for ALS. So the idea of this concept working in neurodegenerative disease, I think is been around for some time. The problem is being how to drug this pathway, and I think a lot of people tried in the past and couldn't make it.

So I think the clever idea here is to create a small molecule that actually modulate positively this pathway, and the good news is you can measure that, and you can quantify that. And that's one of the methodology that the team at Athira used to say that was the right molecule, and I think they did that specifically for each indication, and how to really quantify the magnitude of phosphorylation of the MET and how that translate in the cascade down of the other cytokines or tyrosine kinase subtypes that are related to neuron survival, and survival in other tissues as well.

So when you think about that and all the work that has been done, and I've been focusing more on the in vivo work, but the model is well recognized, and injecting amyloid beta in the brain of the rats and observe the animals in many different aspects. So they saw a clear decrease in inflammation with a typical pro-inflammatory cytokines, IL-6, IL-1B, TNF-alpha, but importantly, the one that is specific in the CNS, like GFAP, which we saw it preclinically and clinically.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

Mm.

Javier San Martin
CMO, Athira Pharma

The neuroprotection is also seen on the mitochondrial dysfunction. There are very good assays to estimate the level of oxidative stress and mitochondrial function. In the end, mitochondrial dysfunction is the way that these cells die. So preserve function is the key mechanism by which those neurons will survive longer. Survival study, also on the hippocampal level, it's been demonstrated that this drug, in the context of the insult that triggers the disease, it also prolongs survival and improve or increase the growth and length of the axons and the neurites. So that, again, it's all about what is this disease all about? It's dysfunction and death of neurons in the CNS. And I think back to, I think, Rachel's point is, again, as a newcomer, I see the landscape in Alzheimer's disease, and you have the symptomatic and stage of disease approach.

You have the amyloid beta antibodies, which definitely the concept is addressed, the underlying trigger of the disease. But nothing is working, nobody's working in the actual target tissue, which are the neurons, and that's how I got excited and joined the team and said, like: "Well, this is a gap." Mechanistically, it's attractive, and when I think about the disease and the clinical care and the health system, the antibodies are really interesting. But when you fast-forward and say: "Well, how are you gonna identify all these people with mild cognitive impairment, with mild disease? How are you gonna screen, you know, across the board? Well, that would be easy to do in those who you can predict. So the homozygote for you, for.

Well, you can use this." So when I put all that together, it's like: Okay, we need to think about clinical practice. And clinical practices, you identify the patients most of the time, but they already have some level of disease, call it mild or moderate, and that's where we want to play, where the unmet medical need is greater, where I think the sense of urgency is also different, and with the mechanism of function that at this point, we have quite a robust preclinical level of evidence, and we're building the clinical piece. So the ACT study, if eventually you want to talk about that, I think is part of the evidence that support our conviction that this.

Moderator

Sure.

Javier San Martin
CMO, Athira Pharma

This has a future.

Moderator

Sure. So beyond the mechanism of action, and you mentioned like that it's a small molecule, but in terms of other design features that are then optimized for Alzheimer's disease patients, anything else in particular that you'd call out with respect to this, the design of Fosgo?

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

Well, well, Fosgo was really designed to go to the CNS, and I think that is what makes it unique from our other pipeline molecules. That, for instance, ATH-1105, which we just got into the clinic this week. We're very excited about that, and that is more targeted to both the CNS and the periphery, which is important in ALS. So, Fosgo is really targeted to, you know, get past the blood-brain barrier-

Javier San Martin
CMO, Athira Pharma

Right.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

And get to the target tissue.

Moderator

Great. So to date, you've primarily evaluated, obviously, in Alzheimer's disease. Maybe just refresh us on the clinical data sets that you have talked about to date.

Javier San Martin
CMO, Athira Pharma

Sure.

Moderator

Okay.

Javier San Martin
CMO, Athira Pharma

So I'll start with the ACT trial because that's the largest study we did so far, and it's in the target patient population, so patients with mild to moderate Alzheimer's disease. In that study, we enrolled 77 patients. About 40% of them were on cholinesterase inhibitor, the other 50-60 were not, randomized to three treatment groups. Key endpoints, clinical endpoints, Cog11, ADL-23, and then all the biomarkers. And what we observed, particularly in those patients, specifically in those patients who were not on cholinesterase inhibitors, a significant and clear change and improvement in cognition, in Cog11. And we also saw across essentially all the biomarkers, a positive trend all in the right direction, the inflammatory, the neurodegenerative, NfL specifically, and the protein burden, like p-Tau and amyloid beta 42/40.

So we're seeing all four biomarkers going in the right direction, cognition going in the right direction. This is six months and in a small sample size. You probably know, because this was presented publicly, that the totality of the data in this study, when we don't exclude those patients on cholinesterase inhibitors, we didn't see quite this magnitude of response. So that triggered a lot of work to really understand where that interaction happened, and I think that the team at Athira came out with a number of evidence to say, "You know, there is reason to believe that this interaction is possibly real." Now, we have a large experiment ongoing to confirm that, which is the LIFT trial. That will be very soon.

Based on the results of that study, we decided to amend the LIFT trial and, you know, include patients that were not on cholinesterase inhibitors moving forward. And so that's, I think, the key component of the more recent clinical data, I guess.

Moderator

Yeah. You alluded to it, but maybe you could expand a little bit more on the specific mechanism of action that you think probably drives the lack of activity in patients on background cholinergics?

Javier San Martin
CMO, Athira Pharma

So, you know, there are a component of this that is, preclinical evidence, and there is a component of this that I think in a small sample size, may be a confounding factor clinically as well. So what happened? Who are those patients who are on therapy to begin with? How differ from those who are not? And second, the concomitant background therapy of this treatment may somehow mask the improvement in the clinical benefit observed with a new intervention. And, you know, like I said, we're talking about maybe 20 patients in that group, so we're talking about a relatively small group. Now, with regard to the underlying mechanism, by which the interaction can happen, do you remember any of the key features there?

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

Yeah. I think the key features, the things that we learned, is that it's a pharmacodynamic interaction, so it's not a typical PK interaction.

Javier San Martin
CMO, Athira Pharma

Right.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

is what we're hypothesizing. And really, because this is a signaling pathway and you want to re-regulate that, it could be that what we've discovered is that when you are hitting some of these pathways at the same time, that you're compensating by shutting it down. And so that's what we've been able to uncover in a few of our preclinical experiments. And as Javier said, in the second half of this year, we're going to have a really good answer about what it means clinically, because we have the LIFT-AD trial, which we're very excited about, is coming.

Moderator

Mm-hmm.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

And we're aiming for the fall to have those results. And you know, it's a large study. It's much larger than what we have in the ACT study, and we'll have about 315 patients in the primary analysis population. So this is the group of patients who are not on background cholinergics. But we'll also have. You know, there'll be you know, upwards of 180 to 200 patients who are on the background therapy that were included in the study and completed the study before we made that change.

Moderator

Mm-hmm.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

So we'll have really good information about how this drug works and in what settings, and who it works best in.

Moderator

Okay. Obviously, the phase II ACT data didn't hit on the primary endpoint, which is P300, but you've since done a number of analyses, including kind of taking a look at the data early. Maybe if you walk through the most important things that gave you confidence to continue moving forward with the LIFT, LIFT study.

Javier San Martin
CMO, Athira Pharma

Well, so I think it's the totality of the data when you see the ACT trial, including the cholinesterase inhibitor subset, in which almost consistently all endpoints when the regulation, I'm now referring to the P300, but specifically to cognition, and to all four biomarkers. So I think it's a totality of data. Say, how many times you see every single endpoint going in the right direction, and this is about 17 patients on treatment, so it's a small sample size. But I think that was what we felt very confident about it. And, you know, I don't know how much the SHAPE study is being presented, but that's a different type of dementia, and the cognition level of improvement was very similar despite, like I said, it was Parkinson's disease and Lewy body dementia, but the severity was similar and the magnitude of improvement.

There you have two studies that show a movement in cognition and in biomarkers of the similar type.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

The interim analysis of LIFT was also a key component because, you know, we did see all of that in the ACT study, but obviously we wanted to understand if we should continue. And so we enabled an interim analysis, an unblinded interim analysis by an independent data monitoring committee. And we basically asked both a futility and an efficacy question to determine if it was futile to continue or if we should. And if we did continue, what would our sample size need to be to have a statistically significant and robust result? And so we performed that.

We passed both futility, and we were told, you know, to add, you know, up to 150 patients, additional patients, to be able to be well-powered to see the Global Statistical Test, which is a combination of both ADAS-Cog11 and ADCS-ADL23, which are the, you know, FDA.

Moderator

Yeah

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

M andated endpoints. So the combination of those two things, statistically significant, is what we're aiming for with LIFT. And that interim analysis gave us a great deal of confidence.

Moderator

Okay. Maybe we can talk a little bit then on the phase III trial design, kind of as it stands now, and I know you're gonna spend some time on this in, like, a fuller rundown next week. Maybe let's start with the endpoint. Global Statistical Test. At a high level, what is that, and how is it measured?

Javier San Martin
CMO, Athira Pharma

Sure. So, first of all, this is one way to do something that many people have done before. So as an example, on both antibody therapeutic development programs on phase II, they use a version of a GST. They call it integrated, but essentially what they did is integrate both major clinical outcome, the cognition or Cog 11 or Cog 13, I think, was in that case, and ADL 23. So we're doing something very similar. What I think is very interesting and important about this is, in order for you to hit that endpoint, you should have positive results in both components of that composite endpoint, Cog 11 and ADL.

So it's not possible that you will have good result in one and the opposite in the other, and you will hit a statistical power value on the GST. So I think that's important because I think it's important to recognize that. On a high level, the way they do that is they normalize the data to Z-S cores, and then, because one score, you know, improvement is increased and the other improvement is decreased on the value, so they need to normalize to come back, to come out with one single value. So I think it's very simple. You do that with the individual patient, and then you aggregate the data, and Suzanne Hendrix will explain this in a lot more detail next week.

But what's the relevance of this is that you have one number with a statistical power and p value that represent the global aspect of this disease. And I think that's what get people excited. What Suzanne is excited about this is a way, in her mind, and she would describe this better, how this is a way to assess disease modifying both by the results and by the concept of time to decline. And so, she's very excited about that. I think most people in the field are excited about that. Even, I think, Lilly used that concept at the advisory committee a couple of days ago. So, so GST, again, is a way to aggregate both psychometric tests. They measure different things, but they should go in the right direction in order for you to have a successful drug.

Moderator

Okay. I guess, can you remind us now that you've got kind of the increased size, et cetera, what some of the powering assumptions are or, like, kind of what you're powered to show in terms of difference on GST?

Javier San Martin
CMO, Athira Pharma

Yeah. So the value number in GST is about three, right? The unit. But I think what matters is the effect size within each.

Moderator

Yeah

Javier San Martin
CMO, Athira Pharma

Psychometric test, and that's, again, as a newcomer for me, like, the GST is a great way to aggregate 2 psychometric scales. But when I look at the data and I explain to clinicians, I think I would like to say this is.

Moderator

You still don't know what's clinically meaningful.

Javier San Martin
CMO, Athira Pharma

Yeah.

Moderator

Yeah.

Javier San Martin
CMO, Athira Pharma

This is the effect size in Cog 11. You have a point of reference based on the antibodies. I think we think that we may have more than three points, I think, was what we were. Four points difference is what we were thinking.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

Well, we saw about three-point difference.

Javier San Martin
CMO, Athira Pharma

Three points, yeah.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

In the ACT on ADAS-Cog, and we almost two on ADL. So, you know, so.

Javier San Martin
CMO, Athira Pharma

So.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

We 're powered, but also.

Javier San Martin
CMO, Athira Pharma

For that

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

W hen you think about what sort of standard of care, about 2.5 on ADAS-Cog is pretty common with the cholinergics. And, you know, this study is in a different patient population, so I think it's really important to think about this in the context of the patient population. That, you know, these are patients who are more advanced in their disease than what we've been seeing with the antibodies. They're progressing more quickly. The study's only 6 months long. You know, if that's enough to see a decline and a change. But we also have, you know, pretty strong belief that given the biology and given the way that we expect our mechanism to work, that this could be a very important medicine longer term.

One thing we also have going, that we haven't talked about yet, is an open label extension.

Moderator

Mm-hmm.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

That open label extension was actually recently extended by another 12 months, basically, because a lot of the patients and physicians, caregivers were saying, "Hey, you know, we like this. We wanna stay on it.

Moderator

Mm-hmm.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

And also, you know, this is a long-term disease. It's a disease you live with for a long time, and, being able to understand the effects over the long, long haul.

Moderator

Yeah

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

I s really important.

Moderator

Can you remind us the cadence of patients that chose to go onto the open label extension now, and, like, maybe how many are still on at one year or two years, or whatever kind of data you can provide quantitatively?

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

Yes. So we have.

Javier San Martin
CMO, Athira Pharma

Well, about in total, about 80% of patients are ongoing in the extension, which I think is huge for a daily subcutaneous injection. So that give us a really good sense of something is moving, people are feeling something. This study started enrollment in 2020?

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

2020 .

Javier San Martin
CMO, Athira Pharma

2020. So the distribution of time in, on open label is very, you know, wide, and we need to quantify that better. And actually, next week, we will talk about that, but it's an important question. I think the point there is, we're not seeing any obvious safety finding, because otherwise, it will show up. We're seeing people that are comfortable receiving these drugs subcutaneously daily for a very long period of time. That also give me a sense of, you know, it's feasible clinically in a real life, because an open label looks like a real life situation. And maybe there is there is really a motivation to do that. There's something going on.

So we will show in more detail the exposure by year to get a sense of how much data we have to talk about this.

Moderator

Yeah

Javier San Martin
CMO, Athira Pharma

L ong-term sustain. And we will have that data by the end of the year, right? The next cut of the open label. Because I think we're excited also to see what will happen with those patients. We're measuring cognition, we're measuring biomarkers. So of course, you don't have the control group, but I think there are many ways to put in context.

Moderator

Yeah

Javier San Martin
CMO, Athira Pharma

T hat kind of data.

Moderator

You talked about a number of the biomarkers, but maybe you could expand a little bit on the blood-based biomarkers that you're using, that you think are most predictive, and the role that they'll play in terms of understanding and interpreting the data we get later this year?

Javier San Martin
CMO, Athira Pharma

Yeah. Well, the first one I wanted to mention is the GFAP, which, b ecause I think it's a very unique situation, that this is a biomarker related to brain inflammation. And again, as I was searching about it, there is a disease that is caused by the excess of this biomarker or this cytokine, sorry. Not a biomarker, in that case, it's a cytokine. So super interesting. That's a pro-inflammatory disease, and it responds well to steroids, but the hallmark of that disease is the elevation of GFAP. So that would be the first one I think is easy to put in context. It's easy to connect it to the underlying pathophysiology of Alzheimer's disease, and we have preclinical data to the translation. So that, I think, is one of the key one.

The NfL is exciting because it's been recognized in another disease, of course, ALS, but I think it's a common biomarker of any neurodegenerative disease. Of course, it's not fully validated in Alzheimer's, but I think many of us and other companies and other academic group are very interesting to go there and eventually be useful. So that speaks specifically to, are neurons dying or are they surviving? And then the two others or three around protein pathology, are the other one I think would be critical to say, well, is this intervention that is not directly to the amyloid therapy by improving cell function and autophagy can decrease the protein burden? So we're measuring the p-Tau217, 181.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

One.

Javier San Martin
CMO, Athira Pharma

One, and then amyloid beta 42/40. So the three more classic one we're measuring, and I think if we see positive result in that, it is a unique feature, with not an antibody that sort of the amyloid protein.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

Mm.

Javier San Martin
CMO, Athira Pharma

We're doing something within the neuron and the glia tissue that can help deal with the burden of protein, and by that. And that is one of the mechanisms by which we increase survival and function of the neurons. So I, I think that's what get me excited about, and the.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

Yeah. I think the biomarkers are gonna be very relevant. These serum biomarkers are what everyone is testing. You know, I think, you know, they're moving towards diagnosis using those biomarkers. We're not there quite yet, but, but I think these are all going to be able to help explain the treatment effect and how and why we are improving cognition and function is really working underneath on the underlying disease.

Moderator

Okay.

Javier San Martin
CMO, Athira Pharma

Sorry, that's the key point, I guess, that I didn't quite say. When I think about disease-modifying, yeah, I know people like to think about that with the antibody, and I think what the antibody do is remove the insult. What happen here is try to help the brain to survive and function better. So I think that it really define what disease modifying is, in my mind.

Moderator

So as you think about potentially positive data at the end of this year, what are the next steps that have to come after that in terms of meeting this with regulators, potentially registrational path? How are you thinking through kind of the multiple options that will be ahead of you if the data look good?

Javier San Martin
CMO, Athira Pharma

Sure. Yeah. So we're planning for success. As you say, we're gonna have data within the next few months. We're gonna be ready to kick off the next regulatory interaction, for which we will need to present all this data, make the case. We're working on really hitting every single aspect that would be necessary to enable the registration phase III study and go to the agency, define the population, all the features of the study design, define the sample size, whether we have any finding that help us to, for example, the cholinesterase inhibitor, yes or no? How about the APOE E4 mutations, homozygous or carriers? So I think all that will help us to define the phase III program. What I feel comfortable now is that the study as is, and it will enable that.

So we have the full tox package to do that. We have the CMC component to kick off a phase III, and we're gonna have a 540 phase II, III study that I think will answer all of these questions. And I compare, I did the exercise to go back to the antibody phase II program, and as a therapeutic dose, we have almost twice as many patients. So I'm very comfortable that this study will answer the question, and by doing that, I think it will serve as the foundation for the phase III program.

Moderator

Okay. So in the past, I think we talked about, like, optionality of going straight to regulators and asking for approval versus, like, having to run additional phase IIIs. Do you have more visibility than on, on that at this stage, or is that still?

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

I think there's... Look, it's still data dependent, but you've got to think about the safety requirements in a population like this, right?

Moderator

Yeah.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

And, you know, we've seen this with the antibodies with Lilly and Eisai, and those recent approvals, I mean, they have thousands of patients.

Moderator

Okay.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

Yeah, I think we're being realistic about that, but.

Moderator

Okay.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

But we are, you know, we're measuring all the right things, so could this be part of the registrational package?

Javier San Martin
CMO, Athira Pharma

But the population- for the population as a whole, I think what Rachel said is absolutely right, but the data will tell us, how about if we see a differentiate effect, more rapid differentiation between placebo and treatment in the APOE 4? That's where the net need is incredible because there are no treatment for that. And I'm- this is hypothetical, right? If something like that happened, I'll be the first one, I'm coming from the rare disease field, to say: Can we make this a subgroup and have a clinical program in parallel to the large indication.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

Mm-hmm

Javier San Martin
CMO, Athira Pharma

That could be shorter, faster, and so forth? So I think we haven't gotten into the details about that, but it gets us excited to think about, well, wait a minute, if something like that happened, that you can narrow the population initially while you're doing the big picture in parallel, can take us to the market earlier-

Moderator

Yeah

Javier San Martin
CMO, Athira Pharma

T hat's something that I think the FDA will always be open to think about it.

Moderator

Sure. In terms of just market sizing, obviously, Alzheimer's is large, we all know that. But, like, as you think about stratifying the market for patients that would be most appropriate for this kind of therapy, I guess, like, what are the numbers around that, that you can provide?

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

Well, so it's really interesting. I mean, 2.1 million people in the United States are getting prescriptions for in the mild to moderate space or, or treatment eligible for that. I mean, six million overall, there's earlier stage disease. But in mild to moderate, it's where 81% of the patients are being diagnosed.

Moderator

Mm-hmm.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

It is a large, large market.

Moderator

Mm-hmm.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

I know if you say, "Well, okay, if they can't take cholinergics, that's the standard of care. What's that gonna do to your market?" Well, there's not a lot of options in this disease. I mean, people are taking those medicines earlier. They're still declining. They're still dying.

Moderator

Mm-hmm.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

Having something new, offering something new that works very differently.

Moderator

Mm-hmm

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

C an really fill a gap in the, in the therapeutic space.

Moderator

Mm-hmm

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

A nd in the treatment, you know, in the treatment options for patients and their caregivers and for doctors.

Moderator

Yeah

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

B ecause they really have had limited tools.

Moderator

I know you've seen what you've discussed in terms of the combination with cholinergics, but in terms of post-cholinergic patient populations, have you seen the same kind of negative effect on the drug's activity?

Javier San Martin
CMO, Athira Pharma

People who discontinue?

Moderator

Yeah, if you've had prior cholinergics, does that impact your ability to be treated with Fosgonimeton?

Javier San Martin
CMO, Athira Pharma

Well, I think we're gonna learn this on the LIFT. The data is collected to answer the question whether people who used in the past, how they would respond.

Moderator

Yeah.

Javier San Martin
CMO, Athira Pharma

And so.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

They have a very short half-life, so I would.

Moderator

You would expect that.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

There's no reason to expect that there-

Moderator

Okay

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

W ould be any effect there.

Moderator

Okay. Maybe moving on briefly to the ALS program, because I know you announced first patient in this week. In that, I guess, first, the drug has got the similar pathway that it's targeting to fosgonimeton, but there are some differences. What are the key differences between the two programs, and why does it matter for ALS patients?

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

Well, one of the key differences is it's oral.

Moderator

Yeah.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

And it's our next generation HGF/MET positive modulator, so we're excited about that. Some, you know, more advanced chemistry, but also it is more targeted to periphery and really getting at that neuromuscular junction.

Moderator

Sure

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

W hich is an area where that is a really important part of the ALS pathology.

Moderator

Sure.

Javier San Martin
CMO, Athira Pharma

And I think what is interesting, just one comment about that preclinical program is, you're right. Most of the experiments provide similar results or neurodegeneration, neuroprotection, anti-inflammatory. But what I think is impressive, at least for me, as a newcomer, is that we have the survival studies in the ALS model, where they clearly show a benefit in survival. And when we present this to the ALS Association about a month ago, asking for feedback and insights, and we didn't present that as a prime moment. It was like, this, this is the presentation. And when we passed on that slide, they were like: "Oh, this is not the usual picture, so this is really relevant, and we're excited about it." It changed the dynamic of that conversation, I thought, when we presented that data.

Moderator

Yeah.

Javier San Martin
CMO, Athira Pharma

The level of evidence technically, I think, is.

I t's in good place.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

Very strong, and also we have NfL data.

Moderator

Yeah.

Javier San Martin
CMO, Athira Pharma

And then we know in ALS.

Moderator

Mm-hmm

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

That, NfL, at least in the SOD1 construct, is, you know, was able to support approval.

Moderator

Yeah.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

We think there's a really good opportunity for ATH-1105 to be, have an accelerated development pathway, given the rare disease, given how challenging it is. Now, we know the bar is high.

Moderator

Mm-hmm

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

But the unmet need is also high.

Moderator

Sure.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

Our mechanism might actually be quite, you know, well-tuned.

Moderator

Right

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

For the needs in ALS.

Moderator

Okay. In terms of then, now that the trial is up and running, you've got patients in, can you talk to us about the trial design?

Javier San Martin
CMO, Athira Pharma

Well, so we have normal healthy volunteers right now, that's, that's what we're doing, the SAD and MAD. And we're already drafting the amendment, I guess, of this study to include patients next year.

Moderator

Okay.

Javier San Martin
CMO, Athira Pharma

So that's gonna happen once we are clear on the, you know, SAD, MAD, and clear.

Moderator

Mm-hmm.

Javier San Martin
CMO, Athira Pharma

We're using, you know, a combination of the NfL data with our preclinical data, and try to model that to say, "Let's try to do a phase I-B study on patients large enough to at least really have a signal on the NfLs.

Moderator

Mm-hmm.

Javier San Martin
CMO, Athira Pharma

I think that's what the value will be, to say if you got to that point, now you will be ready for a phase II/3-type of program.

Moderator

Yeah.

Javier San Martin
CMO, Athira Pharma

And so it's one of the moment that if you do the investment to, and ask patients to take the risk to get into an early program, make sure that you do it right and large enough that you will answer the question.

Moderator

Yeah. In terms of the endpoint and time that it will take to show a change on things like neurofilament, like change, I guess, how long will that phase one be in terms of patients staying on therapy?

Javier San Martin
CMO, Athira Pharma

We think that about three months.

Moderator

Okay

Javier San Martin
CMO, Athira Pharma

I s probably about right. When you look at the SOD1, then maybe, maybe it's three to four or five months, but it's in that range that we expect to see difference. But, you know, talking to the ALS Association and so forth, it won't be like a three-month study. The study will continue, patient will transition. So I, I will envision a six-month placebo control and then crossover, you know, I guess the Biogen/Ionis sample. And SOD1, it, it's a good point of reference to start the program.

Moderator

Okay, understood. So maybe we can start to see kind of data from patients as early as towards the end of next year?

Javier San Martin
CMO, Athira Pharma

End of next year.

Moderator

Is that right? Okay. Great. Today, on the cash runway, I'll get you in here, Andrew. What's embedded in that guidance?

Andrew Gengos
CFO, Athira Pharma

Yeah. So we ended the first quarter with $122 million, and that's certainly more than enough to get to the other side of the LIFT results that we're expecting, we're targeting in the fall of this year. The more exciting question is, how we—'cause we're bullish. We think we're gonna have a positive trial.

Moderator

Sure.

Andrew Gengos
CFO, Athira Pharma

We think what a positive trial will do is give proof of concept, not only in the Alzheimer's setting, but in the mechanism of modulating HGF.

Moderator

Mm-hmm.

Andrew Gengos
CFO, Athira Pharma

If that were to occur, we want to, in parallel rather than one drug at a time, try to address multiple indications, whether that's Parkinson's, you know, FTD, ALS, there are others. So to do that, we're gonna need a lot more money.

Moderator

Yeah.

Andrew Gengos
CFO, Athira Pharma

We want to wait and raise capital on the LIFT outcome.

Moderator

Okay.

Andrew Gengos
CFO, Athira Pharma

So we don't need capital now. Our runway is more than enough to get beyond lift, but we are, you know, thinking through financing scenarios.

Moderator

Yeah

Andrew Gengos
CFO, Athira Pharma

In order to pursue that larger vision.

Moderator

Right. Recognizing that on this side of the table, we have to think about both sets of outcomes. I guess, pending that it doesn't look as you would like to see in terms of success, can you also get through the patient data that you're talking about with ALS towards the end of 2025?

Andrew Gengos
CFO, Athira Pharma

Yeah, I mean, we've thought through scenarios like that.

Moderator

Yeah.

Andrew Gengos
CFO, Athira Pharma

I'd argue that they're less likely than the positive ones, but I'll answer your question. We don't have a very good idea of what the design of the patient ALS.

Moderator

Yeah

Andrew Gengos
CFO, Athira Pharma

T rial component will look like after the single ascending and multiple ascending dose. So if you just kind of take a typical design, we would probably need more capital to get to the other side.

Moderator

Okay

Andrew Gengos
CFO, Athira Pharma

O f patients, but certainly, we have enough capital to get through the SAD, MAD, and to contemplate starting. The other thing that I would point out is, there's a lot of pharmaceutical company interest in that ALS program.

Moderator

Sure.

Andrew Gengos
CFO, Athira Pharma

So we've been promoting it in the sense of, "Hey, do you wanna learn about this program?" We haven't been actively looking for a partner. From those conversations, that we could partner it if we needed to do that as a way to access the capital to complete the patient trial.

Moderator

Okay.

Andrew Gengos
CFO, Athira Pharma

I don't anticipate doing that, but it's an option for us.

Moderator

Okay, great. Well, with that, I think we're at time. Thank you so much for joining us today, everyone. Thanks to everyone who joined us both here and on the webcast.

Andrew Gengos
CFO, Athira Pharma

Thank you.

Rachel Lenington
COO and Chief Development Officer, Athira Pharma

Thank you.

Javier San Martin
CMO, Athira Pharma

Thank you, and I.

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