Good morning, everyone. Welcome to Day 3 of Needham's 24th Annual Conference. My name is Poorna Kannan, and I'm an associate here at Needham. I'm really excited to have with us here today Josh Cohen and Justin Klee. They're the co-founders and co-CEOs of Amylyx Pharmaceuticals. They'll be providing us with a corporate presentation. Following that, we'll open up the floor for any questions. Just as a reminder, please send any questions that you may have on the dashboard. Josh and Justin, if you're ready, please take it away. Thank you.
Excellent. Thank you so much, Poorna. Thank you, Needham, for hosting us. As Poorna mentioned, I'm Justin. With me is Josh. We're the two co-CEOs and co-founders at Amylyx. We're very excited to share a bit more about Amylyx and our exciting clinical pipeline today. Before we get into the presentation, just a brief disclaimer. We will be making forward-looking statements. I would advise you to please review this disclaimer, as well as look for more information on our investor website. Brief overview of our clinical pipeline. We have three assets in four ongoing clinical trials. Our lead asset is avexitide. Avexitide is a first-in-class GLP-1 receptor antagonist. It lowers insulin and raises glucose. Avexitide has FDA breakthrough therapy and orphan drug designation. We are enrolling in a pivotal study in post-bariatric hypoglycemia, or PBH, right now.
We expect top-line results from that study in the first half of next year. PBH is really a significant unmet need. There are no treatments available for people with PBH today. We estimate there's about 160,000 people in the United States who have PBH. I'll share a bit more on that in a moment. We're very excited about the prospects of avexitide to help people with PBH. Next is our combination small molecule, AMX0035, targeting ER stress and mitochondrial dysfunction. We have AMX0035 in two clinical trials, both in diseases characterized by ER stress and mitochondrial dysfunction. The first is Wolfram syndrome. We announced the first results from our first clinical trial last year in Wolfram syndrome. Quite exciting. We are continuing to follow those participants now longer term, as well as planning for our pivotal study in Wolfram syndrome.
We also have an ongoing study of AMX0035 in PSP. PSP is a really devastating neurodegenerative disease affecting about 20,000 people in the United States. There are no treatments available. We are running a phase IIb/III clinical trial right now. We will have an unblinded interim efficacy analysis of that trial in the Q3 . That will be the go-no-go point for if we then enroll for a phase III trial. Last, AMX0114 is our first in-house developed antisense oligonucleotide targeting calpain-2. Calpain-2 is a key effector in axon degeneration. We think that that has potential promise in multiple disease areas. We're first evaluating in ALS. We just announced this morning that we have dosed the first participant in our study in people with ALS with 114. We should have our first early cohort data by the end of this year.
Across our clinical assets, we have milestones in each program over the next 12 to 15 months. Very excited about each of them. We have other preclinical programs as well, such as our potential long-acting GLP-1 antagonist we're working on with Gubra. Today, we'll primarily focus on our clinical pipeline. Sharing a bit more about our lead asset, avexitide. As I mentioned, avexitide is a GLP-1 receptor antagonist. GLP-1 receptor antagonist is potentially important in conditions of hyperinsulinemic hypoglycemia, when the body secretes too much insulin, which causes very rapid drops in blood glucose. The primary indication we're studying avexitide in is called PBH, or post-bariatric hypoglycemia. PBH occurs in a rare group of people in the years following bariatric surgery. What happens is the body seems to potentiate the GLP-1 response.
Our bodies will secrete GLP-1 in response to a meal, sometimes in response to other triggers. In people with PBH, we do not totally know why, but it seems that the body overproduces and secretes GLP-1, sometimes up to 10 times normal levels. This causes a very rapid and high secretion of insulin, which then causes a very precipitous drop in blood glucose. What happens when our bodies do not have enough blood glucose is essentially our brains are starved. The medical term is called neuroglycopenia. People experience bouts of severe confusion, even loss of consciousness, even seizures sometimes. This is really an unmet medical need. There are no treatments available for PBH right now. Given that we think GLP-1 has such a prominent role in PBH, it is why avexitide, a receptor antagonist, makes so much sense in the condition.
As I mentioned, this is a rare complication that happens in the years following bariatric surgery. On average, it takes one to three years after the surgery. It can happen even longer before symptoms manifest. There have been millions of bariatric procedures that have been performed in the U.S. We estimate about 8% of people who get a bariatric surgery will develop PBH. With the well over 2 million bariatric procedures that have been performed, this gets to an overall prevalence in the U.S. of about 160,000 people. It is also a persistent condition. Once people have PBH, it unfortunately does not go away. This population will only continue to grow. This population has actually been quite well studied in the literature.
I would point you to some of the publications that we've listed to in our presentation, both prospective and retrospective studies looking at people in the years following bariatric surgery. This is quite a challenge and a problem. We've started to look at claims-based data as well. It corroborates these numbers. I think this population will only continue to grow. As I mentioned, it really significantly impacts people's lives. We think we have a real opportunity to hopefully change that with avexitide. Now, you know what also makes us very excited about the prospects of avexitide to potentially help people with PBH is the consistency we've seen in all the prior trials. There have been five prior trials of avexitide in people with PBH. In each one, avexitide did what we would expect, which is it raises glucose.
In particular, it raises post-prandial or after a meal glucose nadir, doing quite a significant job at preventing those very significant drops in blood glucose. We saw that again and again and again from the phase I to the ascending dose studies and then to the phase IIs. I'll walk through the phase II data in a little more detail as that is what is substantially informing our phase III pivotal clinical trial design. In the phase IIs, what was particularly looked at was what's called level 2 or level 3 hypoglycemic events. These have been well-agreed-upon definitions by the American Diabetes Association and other groups for quite a few decades now. Level 2 means that the person's blood glucose has dropped below 54 mg/dL. The reason that's important is because that's when people start to enter the range that is neuroglycopenia.
It's when the range where the brain is no longer getting enough blood glucose, and people are at very significant risk for these associated symptoms. Level 3 means that the person has been so severely impacted, regardless of whatever the blood glucose level is, they need independent rescue. This is the severe confusion I was talking about, as well as a number of other symptoms. These are very clinically meaningful outcomes. As I hope you can appreciate, in both the phase II and phase IIb, there were substantial reductions in the level 2 and level 3 events. Going to the phase IIb, there's 57%, 68%, 53%, and 66% reductions in level 2 and level 3 events at the two-dose group studied. As we now go into the phase III study, we went with the single daily dose, the highest dose studied.
That's 90 mg once per day. Importantly, the composite of level 2 and level 3 hypoglycemic events, so that's that combination of these two here, is an agreed-upon primary outcome from FDA. That's both in guidance. FDA has also reviewed our phase III protocol. We are starting enrolling in that study now. A little more on the pivotal study design. Given the very strong results in phase II and the unmet need, that's what supported the FDA breakthrough therapy designation. Our goal really with the phase III trial is to try to have as substantially similar population as possible and keep the design as similar as we can. A few things of note. In the phase II, what was studied was Roux-en-Y gastric bypass, PBH. In the phase IIb, a number of different surgeries that can cause PBH were studied.
Now, we do not think there is anything different about the physiology of these different surgeries causing PBH. We think PBH is PBH. We have the most data in people with Roux-en-Y gastric bypass causing PBH. That is the population we will be studying in the phase III. Also important in terms of inclusion criteria in the phase II studies, very key were that people had to have a run-in period where there was at least one event, one hypoglycemic event per week in order to qualify for the study. We are doing the same thing in the phase III. That is really to make sure that we are evaluating people who have this very persistent hypoglycemia before entering the study. The dose we are studying, again, is the highest daily dose studied, which was in the phase IIb trial.
It'll be a 16-week placebo-controlled study, with an open label extension. As I mentioned, the FDA has reviewed the protocol. We are actively enrolling for this study. We are very excited, as I mentioned, for the prospects and what this might mean for people with post-bariatric hypoglycemia. Just very important as well is the safety profile. I mentioned the efficacy. Importantly, avexitide was quite safe and well tolerated in both phase II trials as well. Injection site reactions were quite comparable to placebo. When they occurred, the vast majority were mild, very occasionally moderate, and seemed to resolve quickly. Strong efficacy from the phase II trials and a good accompanying safety profile as well. Reviewing the overall timelines, we're very excited about the prospects of this program and what it might mean for people with PBH.
We are actively recruiting now. We expect a complete recruitment of this pivotal study by the end of the year and then have data in the first half of next year. Given breakthrough status, which would mean a priority review, this would mean a commercial launch in 2027. Again, as I mentioned, for a condition where there are no treatments, and we estimate an overall prevalence of 160,000 and really a substantial unmet need. Our IP rights go out to 2037. That is before patent term extension. We have orphan drug designation as well. We are very excited about avexitide as our lead program. As I mentioned, we also have two additional assets and three ongoing clinical trials. For more on that, I will happily pass to Josh.
Thanks, Justin. I will talk today about the rest of our pipeline. Starting with AMX0035, this is our combination of two small molecules targeting ER stress and mitochondrial dysfunction. Maybe just an important step back to that part of the reason why we were so interested from the start in sodium phenylbutyrate and taurursodiol is really decades of literature where these are often used as tool compounds to target stress and mitochondrial dysfunction. That is an effect of these compounds that has been replicated again and again across the literature. Maybe starting with Wolfram syndrome, we started working in this space about eight years ago. I'll describe the disease a little bit. Wolfram syndrome is a monogenic disease. It is caused by mutations in the WFS1 gene. What the WFS1 gene is believed to do is stop ER stress in cells when it gets excessive.
What's observed is when patients have mutations and dysfunctional WFS1, the ER stress response can become overactive, leading to cell death and dysfunction. Where that particularly happens is in the beta cell, the pancreatic beta cell, and in neurons. That translates into what you see from the clinical phenotype, which namely people with Wolfram syndrome will start by looking like type 1 diabetes, usually at about six years old. That will progress to full-blown insulin-dependent diabetes. They will also see neurodegeneration, such as blindness, deafness, eventually speech and swallowing difficulties, and breathing difficulties. Usually, people unfortunately pass away in their early 30s. It's a progressive disease. All these symptoms are getting worse over time. We estimate there are about 3,000 people living with Wolfram in the United States today. There are no approved therapies for Wolfram syndrome.
Touching a bit too on the mechanism, as I said, this is a monogenic disease caused by mutations in WFS1. It's often called in the literature the prototypical disease of ER stress. That's where our compounds come in, having shown evidence against ER stress repeatedly across many different models. You can also see on the QR code on the right a link to some of our preclinical data. As I mentioned, we had been studying this combination of compounds in Wolfram syndrome for about eight years. In that work, we studied cellular models as well as the mouse model of the condition, showing highly statistically significant results in slowing the progression in the mouse model, as well as ameliorating the phenotype that we see in the cellular models. It was that work which ultimately drove us towards the clinical trial, which we have presented some data on.
Diving into that a little bit, maybe first, what is expected in people with Wolfram syndrome? As I said, Wolfram syndrome is a progressive disease. Over time, the diabetic phenotype gets worse and worse. Vision gets worse and worse until patients become completely blind. They have progression across all the other symptoms that I described earlier that you can measure with a clinician global impression of change or a patient global impression of change as well. Again, coming into this study, we would have expected patients to show progressive decline across these measures. There are some natural history studies. There are published studies out of the WashU registry under Fumihiko Urano. They are consistent with this finding that people progressively decline who have Wolfram syndrome. We conducted a 12-patient open label study, phase II study with AMX0035.
What was so exciting to us about that study is we saw stabilization or improvement across these measures. Particularly in the diabetic phenotype, we saw consistent changes on C-peptide, which is a measure of beta cell function, hemoglobin A1c, and time in target glucose range measured by continuous glucose monitoring. We saw stable or improved vision. Patients reported that their symptoms, patients and clinicians reported that the symptoms were improving. By and large, really promising data in our view in this initial study. In terms of safety as well, just to recall, AMX0035 through its work in ALS has been in thousands of patients. I'd say the safety profile we saw in Wolfram was very consistent with that. The most common AE was mild diarrhea, which again was consistent with what we've seen in the past. What's next for Wolfram?
We're continuing to follow participants in the phase II HELIOS trial that I just described. We expect to share week 48 data in the coming months. We're also interacting with regulators and working on our phase III program. We intend to provide an update on that in this year as well. Maybe moving next to our program in progressive supranuclear palsy or PSP, also with AMX0035. Similar to Wolfram, I'll start by describing the disease just a little bit. PSP is often considered one of the purest tauopathies. Why is that? There's strong genetic data linking variants in tau, in the gene for tau, to the disease. There's clear pathology both in postmortem samples and imaging studies suggesting that tau is instrumental to the disease. Generally considered a disease driven by abnormal tau protein. How does the disease manifest?
People present initially looking a little like Parkinson's with gait disturbance. What makes the disease distinct? Patients will have trouble moving their eyes up and down. Their arms will be abducted to the side, kind of out to the side. They also have balance difficulties. Ultimately, this disease progresses to a point where patients are fairly locked in, having difficulty moving, speaking, breathing. Usually through respiratory complications, people pass away six to eight years after symptom onset. In the US, we estimate about 23,000 people living with this disease and no approved therapies to date. I mentioned that PSP is a tauopathy, possibly one of the purest tauopathies. We have seen with AMX0035 in a randomized trial in Alzheimer's disease a highly significant reduction in both total tau and phosphorylated tau-181 in the cerebrospinal fluid.
This was one of the elements that got us quite excited about studying AMX0035 in tauopathy and probably particularly in PSP being one of the purest of tauopathies. When we think about our work in PSP, we really look at it as we're testing the hypothesis of whether a small molecule that can get across the blood-brain barrier, can get intracellular and reduce tau, will have an attendant clinical effect. To our knowledge, this is the first time that a compound that can get intracellular, can get across the blood-brain barrier and reduce tau is being studied in PSP. That study randomized 139 people living with PSP to active or placebo. We expect data in Q3 2025, just around the corner. That data will be a full unblinded analysis, including the outcomes I show on the right here.
Primary outcome is the PSP rating scale, which is a functional rating scale assessing people's progression with PSP. We estimate that with the 139 sample size, we have about 80% power to detect a 30% effect. We also have additional secondary outcomes and biomarkers, which should provide additional data as well. We are using this analysis as a go, no go. If the data are strong, we will proceed to phase 3. If the data are weak, we believe we have many exciting programs in Amylyx, and we'll reprioritize resources to those. Maybe lastly, and maybe excitingly today, because we just announced first patient dosed in this program, I'll describe our AMX0114 program, which is our antisense oligonucleotide targeting calpain-2.
Calpain-2, over 10 years that we've been working in ALS, we've spent a lot of time thinking about targets that might be particularly interesting to impact the disease. The target that really came to the top of the list for us was calpain-2. Why is that? It's well known in ALS that one of the primary pathologies is axonal degeneration, basically this idea that the axon of the neuron is retracting and degenerating and dying. There's been a lot of research into the pathway called Wallerian degeneration, which is how axons are believed to self-degenerate and die. One of the proteins that's been most implicated in Wallerian degeneration is calpain and calpain-2. Additionally, looking at ALS specifically, there is evidence of calpain-2 being upregulated. Inhibition has shown benefit in mouse models. It has linkage to various ALS substrates.
I'll touch on too the link that it has to neurofilament. As folks may know, in ALS, there's been a lot of evidence that neurofilament is significantly higher than in healthy control and potentially correlates with disease progression. Interestingly, if you look at the Western blot on the right, what we see in ALS is actually cleaved neurofilament. Neurofilament is a 68 kDa protein, which you'll notice is absent. What you're actually seeing is a cleavage product of neurofilament. What the literature suggests is that the primary protease cleaving neurofilament is calpain and calpain-2. We believe that it's possible that that neurofilament signal you're seeing in ALS is really a result of excess calpain activity. In terms of our preclinical work as well, we've seen highly significant reductions in neurofilament in different insult models designed to induce axonal degeneration.
We've seen rescues in models of oxidative stress. We've also studied in genetic ALS-specific models and shown rescues in mortality. More of that data is published and presented and can be found on our website and through the QR code here. We are now enrolling in our first clinical trial with AMX0114. This is a randomized placebo-controlled multiple ascending dose study in people living with ALS. I know that was a mouthful, but to kind of go through it, it will study four cohorts. In each cohort, nine people will receive active drug, three people will receive placebo. These are people living with ALS. In this study, we are going to be very interested in safety and tolerability as it is our first in-human study with 114.
Additionally, we're quite interested in the biomarker results, probably with a particular focus on neurofilament given the linkage to calpain-2 and to ALS. We expect that we'll have early cohort data this year. Maybe kind of zooming out through all of our programs through the company. We raised money at the start of this year. One of the main intentions in raising that money was to ensure that we would be able to get through our lead asset readout, the avexitide readout, without an explicit need to re-tap the capital markets. That is how we're managing the business. We expect cash through the end of 2026. The cash we had on hand at the end of last year was $176.5 million.
We did also do the offering right at the start of the year, which brought us to about pro forma $240 million. Maybe just to highlight some of the upcoming events for Amylyx with avexitide, we are actively recruiting our phase III trial. We expect to complete recruitment by the end of the year, data readout in the first half of 2026. That's our lead program. With AMX0035, we expect to release week 48 data from the HELIOS trial in the coming months. We're also interacting with regulators and designing our phase III trial, which we'll share this year as well. With the ORION trial and PSP, we expect data in Q3, just around the corner. With AMX0114, we just dosed our first participant. We expect early cohort data this year.
With that, we're really excited about our programs, all of which we believe have the potential to help patients who have significant unmet needs. Thank you so much for having us. I'll pass it over for questions.
Thank you so much, Josh and Justin, for the wonderful presentation. We have a few questions here, so I can just start. Can we start with the press release that came out today? Could you talk a little bit about the LUMINA trial and what can we expect from the early cohort data this year?
Sure. As we shared this morning, we dosed our first participant in the LUMINA trial. As a reminder, this is an antisense oligonucleotide delivered intrathecally in people living with ALS. It's a randomized placebo-controlled multiple ascending dose study. As I said, a bit of a mouthful, but that is the design that we're working on. For the early cohort data this year, that will be data both on safety, and we expect to also have biomarkers. We're looking both at calpain-specific biomarkers as well as biomarkers such as neurofilament, which have garnered a lot of interest in the field. That is kind of the data that we expect to have later this year.
Okay. Thank you so much. Could you walk us through the phase III LUCIDITY design and your confidence in what you observed in the prior phase II trials? In addition, what are your powering assumptions for this phase III design? The design is slightly different from your phase II study design, right? I just want to confirm that.
Yeah, very happy to walk through that. Maybe I'll go a bit in reverse order. The phase II trials were both crossover designs. The phase II had a screening period, a placebo period, and then crossover to one of two dose groups, same total daily dose, but one was once a day, one was twice a day. The phase IIb study had a screening period and then two different dose groups, again, same total daily dose, once a day versus twice a day, although the phase IIb dose was 50% higher than in the phase II.
I'd say that now going into the phase III, most of the details are substantially similar. The way that we're collecting data is the same. We're getting blood glucose measures via fingerstick. All study participants will also have a continuous glucose monitor. Level 3 events are being adjudicated by an independent committee, as was done in the phase II trials.
The dose that we are studying is the same as was studied in the phase IIb. The run-in period too is the same. One event per week. In the phase IIs, we had a two-week run-in period. In the phase III, we have a three-week run-in period. It is basically just to allow for a little more training, but same event rate. It is still at least one per week. The difference really is that the phase III is a parallel group, placebo-controlled trial instead of a crossover, and also that it is 16-week instead of 28-day dosing. That being said, we do not have any reason to believe that the efficacy should wane over time. There is nothing in the non-clinical studies that suggests neutralizing antibodies or anything of the sort. That is the key difference as we go into the phase III trial.
In terms of powering, I'd go back to the phase II trials. As you can see, the results on lowering level 2 and level 3 hypoglycemic events were very statistically significant and with far fewer participants. In the phase III trial, we have more participants and more time, which means that we only have more power than we did in the phase II trials. Even if we show half of the effect size that was observed in the phase II trial, we still will be over 90% powered. Even using quite conservative assumptions going into the phase III study, this is still greater than 90% power. I think that's because of the very highly significant results that were observed in the phase II.
Okay. That's really helpful. You mentioned that it's a slightly longer duration study, right, in phase III. Are there any potential safety concerns that might be a risk with the longer-term study versus what you saw in phase II?
Yeah, I'd say by and large, we've seen a very strong safety profile with avexitide, both clinically thus far as well as non-clinically. In our preclinical studies in animals, we've dosed to many-fold higher than the human equivalent dose that we're studying here. We've seen, frankly, a very, very strong safety profile. In all of our clinical studies, a strong safety profile as well. There are no particular concerns. It is the first time we'll be studying the drug out to 16 weeks, but we have no particular reason to be concerned.
Okay. That makes sense. Could you go a little bit into detail on your prevalence estimates for PBH? Also, within the PBH, is there a particular patient segment you see benefiting the most from a therapy like avexitide? Lastly, what are your expectations from the duration of treatment? Would it be given chronically?
Yeah, those are great questions. Maybe starting with the population a bit. If we look at first the number of people who've had a bariatric procedure over the past decade, it's well over 2 million people. Of that group, actually quite a high number will have some hypoglycemia, some hypoglycemic events. That's more like 30% or 40% of the overall population. The population that we're talking about with what we call PBH are the people who have particularly persistent symptomatic treatment-resistant hypoglycemia. So that 160,000, that is the so-called more severe group, the group who really have persistent hypoglycemic events.
When we're talking about where do we think avexitide could be a meaningful treatment, it's particularly in that population. That being said, this year, we're doing a lot of commercial preparations, particularly market insights, to really understand where should we focus our efforts first. I think that 160,000 is quite a substantial prevalence. We want to make sure that we're very targeted and thoughtful in our commercial approach. Again, we think it's a large unmet medical need, but I think often in rare disease, particularly in areas where there haven't been prior treatments, it's very prudent to have a targeted approach. That's our plans. We'll get more of those market insights as we go forward this year.
Okay. That makes sense. Like you mentioned, you're preparing for the commercialization. I guess, can you talk a little bit more about that? This would be the first drug that you're taking to market. What are you doing to build the commercialization team and everything?
Yeah, great question. Actually, as you may recall as well, this actually will be our second drug that we're taking to market.
I'm sorry.
Sorry to correct. We are, one, taking all the experiences we took from our Relyvrio launch, where we did about just under $400 million in year one sales. I think we took a lot of learnings from that as well and certainly are applying them as we move into this launch. We're hiring a great team. We just announced at the start of this year, Dan Monahan as our Chief Commercial Officer, who has tremendous experience and is really strong in the role. We've been hiring the leadership underneath him as well.
I think we have some great people there also. I'd say as we think about this market, as Justin said, it is a new market. We will be the first, potentially be the first drug that is launched in PBH. We do think that education will be important. We think there's a lot. There's certainly a set of KOLs and people who are very aware of this disease and will speak broadly about how significant of an unmet need it is. There's also a large cohort of people who will require education and otherwise kind of as we get out there. As Justin said, this year is really a year where we're doing a tremendous amount of market research and insights so that as we get into 2026, we're ready to start putting those into action and then launch in 2027.
Okay. That makes a lot of sense. I guess for the last few minutes, we can move on to the next therapies. For AMX0035, what can we expect to see from the interim analysis? Could you give us additional color on the specifics behind the go and no-go decision that you might have?
Yeah, very, very happy to. In our progressive supranuclear palsy trial, we enrolled, randomized 139 participants. We will be looking at everyone who through at least 24 weeks of treatment. For the people who have been on treatment longer, we'll use all available data. When we do the interim analysis in the Q3 , it will be unblinded. We'll be looking not just at safety, but efficacy as well. Probably the most important outcome we'll be looking at is the PSP rating scale. That's the most widely used scale in PSP.
It's been quite consistent trial -to -trial. Unfortunately, no treatments have worked in PSP. Looking trial -to-t rial, the progression rate has been quite consistent on the PSPRS. We'll be looking at a number of other clinical scales as well as biomarkers as well. When we do that analysis, we'll really be looking at the totality of the data. For the go/no-go, I think it's very important as we go into a potential phase III, we want to have high confidence, right? That's a significant undertaking. We want to really make sure that we have a high probability of success before we enroll in that study. I think I'd really orient people towards the PSPRS, the associated outcomes, the totality of the data, but we're going to have a pretty high bar to enroll for that phase III part of the trial.
Okay. Thank you. That makes a lot of sense. In terms of your Wolfram syndrome study, the Helios study, what insights have you gained from that study and how are you leveraging that to inform your potential phase III design?
Yeah, it's an excellent question. I think we've learned, one, we've certainly learned a lot just in conducting the study about some of the outcome assessments. Some of them can be somewhat burdensome as well. We're definitely taking those learnings as we go into phase III. I think the other thing is we saw something very surprising here. We did not expect, we were hoping for slowing the disease or stabilizing the disease. What we actually saw on many of our outcomes was improvement.
On the C-peptide particularly, which was our primary outcome, we showed a numerical improvement, which has not really been seen all that often in diabetic conditions and really suggests some degree of rescue of the pancreatic beta cells. As we go into the phase III, I think we have a lot of, I think, really well-understood, well-validated type outcomes that we can use. We are trying to run effectively the most efficient study. I think we believe that this is potentially a very important drug for Wolfram. We want to cut the most rapid path we can to people in need.
Great. Awesome. Thank you for providing all of that additional color. With that, we are actually at time. I just want to thank everyone for joining us today. Thank you to Josh and Justin for providing this wonderful presentation. With that, hope you all have a good rest of your day. Thank you.
Excellent. Thank you, Poorna. Thank you all.
Thank you.