Amalex at Jefferies Conference: Strategic Pipeline Updates

On Thursday, 05 June 2025, Amalex (NYSE:AMLX) presented at the Jefferies Global Healthcare Conference 2025, showcasing its robust drug pipeline. The company highlighted significant advancements in treatments for post-bariatric hypoglycemia (PBH), Progressive Supranuclear Palsy (PSP), Wolfram syndrome, and ALS. The conference was a platform for both optimism and caution, reflecting on promising data and the challenges ahead.

Key Takeaways

• Amalex’s Avexatide, targeting PBH, is in a Phase III trial with data expected by 2026, aiming for a 2027 launch.
• AMX35 shows potential for treating Wolfram syndrome and PSP, with ongoing trials and data anticipated in Q3 2025.
• AMX114 for ALS is advancing with data expected by the end of the year.
• The PBH market is growing, driven by the high number of bariatric surgeries in the U.S.
• Pricing strategies for Avexatide will be determined closer to its commercial launch.

Financial Results

• Avexatide’s pricing will be aligned with rare endocrine disorder drugs, pending Phase III results.
• The PBH market in the U.S. affects approximately 160,000 people, with 270,000 bariatric procedures annually contributing to this population.

Operational Updates

• Avexatide (PBH):

- Phase III trial recruitment is expected to complete by year-end, with data in early 2026.

- Phase IIb trial showed significant reductions in hypoglycemic events, earning FDA breakthrough therapy designation.

• AMX35 (Wolfram Syndrome):

- 48-week data indicates improvement across multiple measures. Phase III trial plans are underway.

• AMX35 (PSP):

- Phase II trial data expected in Q3 2025. The trial has a high bar for advancing to Phase III.

• AMX114 (ALS):

- Ongoing clinical trial with data anticipated by year-end. The drug has received a fast track designation.

Future Outlook

• Avexatide (PBH):

- Aiming for a 2027 launch, with NDA submission planned post-Phase III results.

• AMX35 (Wolfram Syndrome):

- Collaborative efforts with the FDA continue for Phase III trial design.

• AMX35 (PSP):

- Results from Phase II will determine the feasibility of a Phase III trial.

Q&A Highlights

• Avexatide (PBH):

- The Phase III trial is placebo-controlled over 16 weeks, with no expected attenuation of effects.

• AMX35 (Wolfram Syndrome):

- A Phase III study is in planning, following positive outcomes in earlier trials.

• AMX35 (PSP):

- The ongoing Phase II trial involves 139 participants over a minimum of 24 weeks.

Amalex’s strategic focus on addressing unmet medical needs is evident in its comprehensive pipeline. For more detailed insights, refer to the full transcript below.

Full transcript - Jefferies Global Healthcare Conference 2025:

Andrew Tsai, Senior Biotech Analyst, Jefferies: We’re going to get started with our next session. I’m Andrew Tsai, senior biotech analyst at Jefferies. Thanks for tuning in. And it’s my pleasure to have the Amalex team with me today. To my direct left, Justin Klee, co CEO I’m sorry, Josh Cohen, co CEO, and then to his left, Justin Klee, co CEO, as well.

Welcome both of you.

Justin Klee, Co-CEO, Amalex: Thank you.

Josh Cohen, Co-CEO, Amalex: Thanks, Emily.

Andrew Tsai, Senior Biotech Analyst, Jefferies: So for those in the audience who may be less familiar with the Amelix story, spend a couple of minutes talking about what you’re working on, what you’re trying to achieve, what kind of milestones we can expect for the next six or twelve months.

Josh Cohen, Co-CEO, Amalex: Sure, absolutely. So at Amelix, we’re developing therapies for significant unmet needs, particularly rare all rare diseases that we’re focused on as well. Our lead asset is a compound called Avexatide, which is a competitive inhibitor of GLP-one. And we are using that in diseases of hypoglycemia, particularly focused in post bariatric hypoglycemia. This is a rare condition that people get in the years following bariatric surgery that results in them having persistent, sudden blood sugar drops, often in response to a meal, but sometimes in response to exercise, stress, other triggers.

And having that chronically means that people end up living a fairly sheltered life. They may be unable drive a car. They may be unable to they may have lapses in challenges with a job or daily living. So a disease that really, really has a substantial unmet need. Avexatide has been studied in five past trials in PBH, all of which showed significant benefits on glucose and insulin measures, as well as in the phase II and phase IIb, significant reductions in hypoglycemic events, including at the dose that we’re taking into phase III, a 53 reduction in level two hypoglycemic events with a p value of point zero zero four, and a 66% reduction in level three hypoglycemic events with a p value of point zero zero zero three.

So based on that, we have breakthrough therapy designation. We’re conducting a phase III study that we expect to complete recruitment by the end of the year with data in the first half of ’twenty six and pending good data, plan to submit and hopefully get approval and launch in PBH. In addition to that, and I’ll talk very quickly, we have two other assets across three trials, AMX35, which we’re studying in PSP and Wolfram syndrome. In Wolfram syndrome, we just released forty eight week data from an ongoing study, which showed whereas Wolfram is a progressive disease, you expect multiple measures to get worse over time. We’re actually seeing an improvement across the measures that we’ve been studying.

So based on that data, we’re you know, defining a potential phase three trial. And we expect to share plans for that later this year. We also have data coming out from a clinical trial in PSP with AMX thirty five in Q3 of this year based on the science and pharmacology that we’ve seen with tau reduction with AMX-thirty five. That’s what’s made us excited about the potential in PSP. And finally, we have AMX-one hundred fourteen, an antisense oligonucleotide targeting calpane two, which is one of the critical proteins in axonal degeneration, also one of the critical proteins in processing of neurofilament.

We’ve seen pre clinically significant effects on axonal degeneration and on neurofilament with our ASO, AMX114. So that’s ongoing in a clinical trial in ALS, placebo controlled ascending dose study. And we expect to have some data before the end of the year. And we also just got a fast track on that program as well. So I’ll stop there.

Lead assets of vexatide and, you know, multiple other assets in progress as well behind that.

Andrew Tsai, Senior Biotech Analyst, Jefferies: Thanks. That’s a great overview. So, I’d like to start by digging into the PBH program. Can you help us frame the market opportunity for Avexatite ultimately? What how big is a market?

If approved, would it be used in level two, level three? How big is that market? Just help us frame or give us context how many patients are out there in The U. S.

Justin Klee, Co-CEO, Amalex: Yeah, thank you, and that’s critical. So post bariatric hypoglycemia, or PBH, is a condition that affects an estimated one hundred and sixty thousand people in The United States, and that number will only continue to go up. It’s a rare complication that can happen in the years following bariatric surgery, but the reason you get to quite substantial numbers is that there have been millions of bariatric procedures just in the past ten years alone. The most recent data on bariatric procedures is that there have been about 270,000 procedures annually. So even with the GLP-one agonists in 2023, there are about 270,000 procedures.

And the reason is because bariatric surgery is still the best option we have for people who are morbidly obese, people who need to lose 100, one hundred and 50 pounds. So we think bariatric surgery will continue to be used. It has great data on improving all cause mortality outcomes, but that also means that we’re going to continue to have post bariatric hypoglycemia, which also is a condition when someone has it, unfortunately it doesn’t go away. In terms of level two and level three events, PBH is characterized by very persistent hypoglycemic events. So whereas, for example, in type two diabetes, oftentimes people are worried if there’s a single hypoglycemic event because it’s so dangerous.

People with PBH may have an event a week. They may have more than one event per week. So when we talk to adult endocrinologists, they describe people with PBH as some of the most fragile patients they have under their care. Currently, the mainstay treatment is medical nutrition therapy. So there are no treatments specifically approved for PBH.

Essentially, people with PBH are told to try to keep their glycemic control as best as possible by eating very frequently and avoiding simple carbs, and even despite that, they’re having these significant hypoglycemic events. So, as you might imagine, when people’s blood sugars drop this way, they may suddenly lose consciousness. That may happen when they’re driving. So, the majority of people have their licenses taken away, they’re in the hospital, in the ER very frequently, people may have seizures. So this is a really significant unmet need.

This is the program is the furthest in development. We’re very optimistic that we may be able to make a big impact in this unmet need.

Andrew Tsai, Senior Biotech Analyst, Jefferies: Hence your breakthrough designation on good data. So, then as we think about the peak sales opportunity, what is the price point you’re trying to achieve? And are there any analogs to justify that price range?

Josh Cohen, Co-CEO, Amalex: Yeah. So, you know, we haven’t priced the drug yet. You know, I think we will wait until we have our phase three data and get closer to launch to ultimately price the drug. But I think we’ve been one, this is an orphan disease. We have orphan drug designation.

I think we’ve been encouraged to see a number of different products coming through the pipeline in other rare endocrine disorders. You know, whether you look at some of the recent drugs from, you know, Seleno or from Kinetics or from Ascendis. There have been a number of recent rare endocrine drugs that have helped develop this kind of rare endocrine space. So again, we haven’t selected a specific price, but we’re definitely looking at this as rare disease.

Andrew Tsai, Senior Biotech Analyst, Jefferies: That’s helpful. And then remind us you did mention it in the prepared remarks, just remind us one more time is just the efficacy summary that you saw and the safety summary that you saw that led to this breakthrough designation. That’d Yeah,

Justin Klee, Co-CEO, Amalex: absolutely. So we are now in the sixth and pivotal study in post bariatric hypoglycemia with Avexatide. And we were excited just a month and a half ago to dose the first participant in that study. And then as Josh said, we expect to complete recruitment by the end of this year and have data in the first half of next year. What supported the FDA breakthrough therapy designation were those five prior trials in people with PBH, and particularly the two Phase IIs.

In the Phase IIb trial, which used the ninety milligram dose, which is what we’re now using in the Phase three, there was a fifty three percent reduction in level two events with a p value of point zero zero three or point zero zero four, and then a 66% reduction in level three events with a p value of point zero zero zero three. So very significant reductions and very meaningful reductions in what are considered quite significant events. So now going into this Phase III, our goal is to try to keep the patient population as consistent as possible with what we saw in the Phase II. We’re looking at the same outcomes, the same ways of measuring those outcomes, and I think if we see a recapitulation or anything close, I think this would be a really meaningful opportunity or really meaningful therapy for people with PVH.

Andrew Tsai, Senior Biotech Analyst, Jefferies: And do you need to see a recapitulation for this to be approved? Or is there a cushion where you can show maybe less efficacy, yet still get approved? Why did you agree with the FDA?

Josh Cohen, Co-CEO, Amalex: Yeah. First I’d say one advantage of having breakthrough therapy designation is you do get very frequent and kind of extra collaboration with the FDA as well. So we did submit our protocol to FDA. We got feedback from them. And we do believe the study we’re running is pivotal.

Would be the study to support approval. In terms of you know effect size, that’s not you know, I think as we’ve talked to physicians and as we’ve talked to FDA, I think the view is that if you are showing a significant reduction in these events, that is very meaningful for patients. Particularly maybe going through the two types of events. A level three event is when you need a rescue from somebody else, which means you are so incapacitated that you cannot self rescue your blood sugar and things like that. So you can picture that as losing consciousness, becoming so weak, becoming so confused that you’re no longer able to self rescue.

Level two is when you’re in the range that’s considered neuroglycopenia. It’s the range where your blood sugar’s less than 54 mgs per deciliter. And when you’re at that level, you don’t think clearly, you can be very shaky, you really don’t feel good at all. So seeing a reduction in those events would be quite meaningful. From a powering perspective, definitely going into a phase three trial, you want to ensure that you’ve given yourself the absolute best chance for success.

So we powered the study. Whereas we saw a 53% effect on level two and a 66% effect on level three in the phase 2B, we’ve powered this study so that we have at least 90% power if it’s only a 35% effect. And we even put some conservative assumptions about placebo effect and otherwise in that powering analysis as well. So we view this study to be very well powered even under conservative assumptions.

Andrew Tsai, Senior Biotech Analyst, Jefferies: And speaking of placebo, how do we is there a placebo risk in an indication like this? And what are you assuming for placebo?

Justin Klee, Co-CEO, Amalex: So we don’t believe there’s a placebo risk, as you said, in this study. The reason is we didn’t see it in the Phase II. So in the Phase II, which was a crossover design but had a screen in portion, then a placebo portion, and then two different dose groups, there was no difference in event rates between what we saw in the run-in and what we saw in the placebo period. That being said, it’s always prudent to model conservatively. So in our modeling, we modeled up to a 50% placebo effect and 35% effect size, and we’re still well powered to see that.

So we’re taking a conservative approach with our statistical powering, but ultimately we don’t expect much of a placebo effect. And I think the reason is that people are already doing everything they can to try to control their blood glucose. So they stick very closely to their diet, and very unfortunately, if they have excursions, you know, they have quite significant events. So I think people really, really try to maintain their lifestyles as best as they can.

Andrew Tsai, Senior Biotech Analyst, Jefferies: Male Understood. And can you remind me how long the phase two studies were, and is the phase three different in trial length, and does that change things by much?

Josh Cohen, Co-CEO, Amalex: Yeah. So the phase two and phase 2b were four weeks of active treatment. The phase three will be sixteen weeks of active treatment. So it is longer. I’d add though, we don’t have any reason to believe that the treatment effect is going to be different weeks one to four compared to weeks five through sixteen.

You know, this mechanism is a well studied mechanism. GLP-one doesn’t seem to have some sort of counter regulatory response. If you look at the on the agonist side, many of those trials are eighteen months and you continue to see the benefits accrue through eighteen months. And we also haven’t seen anything with neutralizing antibodies or anti drug antibodies that would suggest any tachyphylaxis. So we believe that the effect will just continue to accrue.

And we have dose for longer in animals as well. And similarly, no expectation that there’d be any attenuation over time.

Andrew Tsai, Senior Biotech Analyst, Jefferies: Interesting. And speaking of the open label data that you shared, is that part of another set of updates you could share later this year? Or what are you sharing from the prior phase two studies later this year, if you are?

Justin Klee, Co-CEO, Amalex: So the prior Phase II studies have completed, so the data is what it is. And again, they were very strong, which supported breakthrough therapy designation. The Phase II data are published. The Phase IIb were presented at the ENDO Conference a couple of years ago, but that was just in a presentation. So at this year’s upcoming ENDO conference in July, we will have several presentations, including more on the PKPD, particularly supporting the ninety milligram dose, which is what we are studying in the Phase III trial, as well as the composite outcome of Level II and Level III events, which is the primary outcome.

So the data from the prior Phase 2b trial. Actually, are other presentations or posters from other researchers on PBH I’d point out, including the inventors of vevexatide from Stanford, have a presentation on the prevalence estimates of PBH, and so they use different methodology than we did, but actually came to quite similar overall numbers, in fact a little higher than our estimates. But that was nice to see that independent work is coming to similar market size is what our research has shown. Okay.

Josh Cohen, Co-CEO, Amalex: And just one comment to add to. The past studies were controlled studies, not open label studies as well. Even back to the phase one studies, which were single dose and multi dose crossover studies, but crossover between a placebo and a active.

Andrew Tsai, Senior Biotech Analyst, Jefferies: Male Understood. Thanks for clarifying. And going back to the phase three study, in terms of baseline characteristics, are there any differences compared to the phase two, such as like surgery type and so forth that you mentioned that are different?

Justin Klee, Co-CEO, Amalex: So our goal, again, is to be as consistent as possible. So probably the key criteria, the first is the number of events per week that someone needs to qualify for the study. As you might imagine, the event rate is what ultimately leads to the powering. So in the prior Phase II studies, it was required to have an or at least one event per week. We’re doing the same in the Phase III trial.

Now, they had a two week run-in. We’re having a three week run-in, so we’re requiring, you know, three events in three weeks. They had two events in two weeks, but the event rate is the same. In terms of surgery type, while we believe PBH is PBH, doesn’t matter what surgery led to it, we have the most data on Roux en Y gastric bypass leading to post bariatric hypoglycemia, and that’s what was studied in the Phase II. The Phase IIb, they allowed people with a variety of different surgeries.

So in the Phase III, we are allowing only Roux en Y gastric bypass PBH, which again, as we go forward, as we have discussions, future discussions with FDA on label, we think there’s a strong case to say PBH is PBH. We have data on people from a variety of different surgeries leading to PBH and that if exotide is effective and the pathophysiology is the same. But for the Phase III, we want to make sure we’re trying to control all the variables we can.

Andrew Tsai, Senior Biotech Analyst, Jefferies: Male Makes sense. And I’d imagine reducing level two, level three events are meaningful already, but presumably you’re capturing other endpoints, outcome measures, I don’t know, symptoms, quality of life measures. Maybe talk us through what you’re capturing to eventually strengthen the totality of data to convince payers to pay for this drug.

Josh Cohen, Co-CEO, Amalex: Yeah. So I mean, first I’d say, you know, maybe reiterating your first point, level two and level three hypoglycemic events are already a very big deal for people. Level three, you know, in particular to highlight, this is people becoming completely incapacitated. You’re preventing people becoming completely incapacitated. That’s a big deal.

But we are also guided in part, too, by some of the past work that had been done on the asset, including structured patient interviews. So there had been work to do structured interviews with patients after they had finished the trials to assess what their perception was of being on Avexotide, of being on the drug. And patients reported when they were asked to rate the drug on a scale of one to 10, all but one of the interviewed patients rated it a 10. The other one rated it a nine was a tough grader. But when asked why, why did you give it that rating, a lot of them kind of described that it was like a fog had lifted.

That they had a lot more energy, that fatigue was quite a lot less. Of course, that they felt protected by the fact that they were having fewer events. So as we thought about quality of life in the phase three, we thought about incorporating some measures that might be helpful for that as well. So we do have some measures related to hypoglycemia fear. We have some measures related to fatigue.

We are also looking at kind of generalized quality of life measures. But I’d come back to the start, you know, level two and level three are already very robust for seeing a, you know, clinically meaningful treatment effect.

Andrew Tsai, Senior Biotech Analyst, Jefferies: Right. And so again, you’re enrolled you started you’ve actually dosed a patient, it sounded like. And then you complete enrollment later this year. Is that right?

Justin Klee, Co-CEO, Amalex: That’s exactly right.

Andrew Tsai, Senior Biotech Analyst, Jefferies: And data in first Yeah.

Justin Klee, Co-CEO, Amalex: Yeah. Are exactly the milestones.

Andrew Tsai, Senior Biotech Analyst, Jefferies: And so do you need ICH six month, one year long term follow-up data before you file?

Justin Klee, Co-CEO, Amalex: Male You’re saying for clinical?

Andrew Tsai, Senior Biotech Analyst, Jefferies: Male

Justin Klee, Co-CEO, Amalex: This is the pivotal study. So the FDA reviewed the protocol, sixteen week placebo controlled trial, and this is what would support approval.

Andrew Tsai, Senior Biotech Analyst, Jefferies: Male Oh, wow. That’s great. And so you can file right after, in the marketplace, 2027?

Justin Klee, Co-CEO, Amalex: Male Or

Andrew Tsai, Senior Biotech Analyst, Jefferies: late twenty twenty seven.

Justin Klee, Co-CEO, Amalex: Male Yeah. So top line results, first half of twenty twenty six. Our team will work as fast as we can with positive data of course to submit and then that would mean commercialization 2027.

Andrew Tsai, Senior Biotech Analyst, Jefferies: And are there any other peripheral studies you need to do in the meantime to support this submission?

Josh Cohen, Co-CEO, Amalex: I’d say nothing material. You know, there’s always work that you do and kind of boxes you check leading up to a potential NDA submission. But there’s nothing that you know, the clinical trial is the main element.

Justin Klee, Co-CEO, Amalex: Yeah. I’d add, too. We’re very proud of our team, and this was a team that got an orphan drug approved. We had a very successful orphan drug launch. So this is a very experienced team.

They’re already working towards what would be required for the NDA submission next year. We’re starting to look at things like you were mentioning market access, which is critical to support access for people if the drug is approved. So the engine’s going, and we’re you know, but first things first, you know, we’re focused on execution on the trial.

Andrew Tsai, Senior Biotech Analyst, Jefferies: Yep. And maybe a couple of questions on competition, then talk about why you think you’re differentiated from others working on the same indication.

Josh Cohen, Co-CEO, Amalex: Yeah, so we’re not aware of any other drug that’s shown any efficacy in this space. So, you know, I think our view is, you know, there’s a lot of hurdles before we have competition.

Andrew Tsai, Senior Biotech Analyst, Jefferies: Yep. I don’t know if this is misinformed, but when I think about GLPs, is that something that can shrink the market for you?

Justin Klee, Co-CEO, Amalex: You know, we don’t believe so. And I think the reason is a few fold. So first, the people who are looking to get a bariatric surgery as opposed to use a GLP-one agonist for weight loss, it’s a very different population. People who get bariatric surgery are morbidly obese. There are people who need to lose 100, one hundred and 50 pounds.

And so getting a surgery is, you know, that’s quite a significant undertaking, but people are doing it for their health. That’s still a very substantial population in The United States. It’s about nine percent of people who have a BMI of forty or greater. And so, so far, you know, we haven’t seen the number of bariatric procedures decrease. And as I mentioned with PBH, once someone has PBH it does not appear to go away, and so we think that those numbers will only increase.

You know, I’d say on the efficacy side, you know, GLP-one agonist, one of the contraindications or risks is hypoglycemia. So again, this is the other side of the equation. This is raising the glucose nadir and preventing hypoglycemia, which is why we think it’s a very exciting mechanism. Too much glucose is bad, too little glucose is bad. We really want good glycemic control.

Andrew Tsai, Senior Biotech Analyst, Jefferies: Okay. Well, it sounds like you’re executing, and good luck on that data set. It’s coming up shortly. So maybe in the last five minutes we can talk about your other programs, starting with Wolfram. What data have you shown so far?

It seems like you reported some long term data set. And when do you start the phase three trial, how is this going to be powered? What’s the primary endpoint?

Josh Cohen, Co-CEO, Amalex: Yeah, great questions. So we conducted, you know, maybe starting what got us excited about Wolfram. Wolfram is considered a prototypical disease of ER stress. It’s a monogenic disease caused by mutations in the WFS1 gene, which is believed to be a protein that helps the cell to resolve ER stress. And AMX35, ER stress is one of the main things it’s thought to target.

So we did several years of preclinical work, showed highly significant differences in both cell models of the disease as well as the mouse model of the disease, which led to the clinical trial. Wolfram syndrome itself is kind of a half endocrine, half neurodegenerative disease. So people initially look like type one diabetics. They get progressive diabetes. But as time goes on, they get vision loss, hearing loss, walking and movement loss, swallowing loss, and ultimately usually death through respiratory failure and breathing challenges, usually in their early 30s.

So when we designed the trial, we were looking at endocrine and neurodegenerative outcomes. Most specifically, we were looking at various markers of glycemic control, C peptide, hemoglobin A1C, and CGM markers for blood glucose control, as well as vision, visual acuity, we were looking at as well. And going into the trial, what we’d expect and what’s been seen in the natural history of Wolferam is that all these measures get worse over time. It’s progressive disease. What we saw in the trial initially over twenty four weeks, which we presented late last year, and then most recently over forty eight weeks, which we just presented, we saw an improvement or stabilization across all those outcomes.

And you know, the separation continued to increase as we got out to forty eight weeks as compared to twenty four weeks. For many of the outcomes, nearly twice as large an effect at forty eight weeks compared to twenty four. So we’re very excited about that. But it is a single center, open label study. And so I think, you know, our kind of base case of the next step is that we’ll be running a Phase III study.

We’re right now working with FDA on the design of that. And I’d add, as we go into any Phase III study, and Wolfram is no exception, we try to stay as similar to the Phase II as we possibly can. So while we have no final design, I’d say as much as we can stay in terms of endpoints, in terms of population, etcetera, similar to the phase two, I think that’s to the advantage for the phase three.

Andrew Tsai, Senior Biotech Analyst, Jefferies: Okay. So that’s just pending. And then turning to PSP, then talk about the PSP market, why you’re excited about it. It sounds like there’s a data update in Q3 coming up. So what is the gono go threshold?

Justin Klee, Co-CEO, Amalex: Yeah, so starting with PSP. So PSP, or progressive supranuclear palsy, is a movement disorder. It’s often characterized as an atypical Parkinson’s. It’s a really tough neurodegenerative disease, so it’s characterized by gait disturbances, challenges with eye movements. People then progressively lose movement functioning and get to a locked in state, and then the disease is usually fatal in about six to eight years.

So it’s a really tough, progressive, fatal neurodegenerative disease. There are an estimated about twenty three thousand people with PSP in The United States, but that’s definitely underdiagnosed. There have been many autopsy reports of people who had not so typical or atypical Parkinson’s, and they in fact had PSP. And you can see that because there’s significant tau buildup in the brain, as well as a characteristic midbrain brain degeneration that occurs and what causes the clinical manifestations. So currently there are no treatments for PSP, not even symptomatic treatments, which is very tough on people and their families.

We are studying AMX35 in PSP, both because of the mechanism and because AMX-thirty five in a prior trial showed very significant tau lowering, and tau is what drives the degeneration in PSP. So next quarter we will have the top line results from the Phase II portion of our trial. That will be one hundred and thirty nine people randomized through at least twenty four weeks of treatment, and we’ll be looking at the PSP rating scale as well as a number of other clinical and biomarker outcomes. We’re going to have a high bar if we want to go into Phase III. You know, we want to have confidence that we may see a real effect in a Phase III trial.

But that being said, there’s also nothing for PSP. So I think there’s a real unmet need and we’ll be excited to have those results in the third quarter.

Andrew Tsai, Senior Biotech Analyst, Jefferies: Okay. And presumably you’ll try to go for accelerated approval, I’m assuming, if you pass the go no go threshold maybe through a tau biomarker approach, pair it with a PSP score, I don’t know.

Justin Klee, Co-CEO, Amalex: I think the results will dictate the path forward. And we always try to be very transparent about the results. I will say, though, there is nothing for PSP. So we will certainly, as I know others will take that into account. That being said, nothing has worked in PSP.

So it is a tough field. But we hope we’ll change that, but we’ll see the results in the third quarter.

Andrew Tsai, Senior Biotech Analyst, Jefferies: All right. Very good. Thank you so much for taking the time to chat with me today. And thanks, everyone, for listening.

Justin Klee, Co-CEO, Amalex: Excellent. Thank you for having us.

Josh Cohen, Co-CEO, Amalex: Thanks so much.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.