Amlex at BofA Conference: Strategic Insights on Clinical Pipeline

On Tuesday, 13 May 2025, Amlex Pharmaceuticals (NYSE:AMLX) presented at the BofA Securities 2025 Healthcare Conference, offering a strategic overview of its clinical pipeline. The company highlighted both promising advancements and ongoing challenges in its efforts to address unmet medical needs. Key updates included progress in trials for vexatide and AMX-35, as well as discussions with the FDA regarding Wolfram syndrome.

Key Takeaways

• Vexatide, targeting post bariatric hypoglycemia (PBH), is in a Phase 3 trial with recruitment expected to complete by year-end.
• AMX-35 showed sustained improvement in C-peptide levels for Wolfram syndrome, with Phase 3 trial design underway.
• Early cohort data for AMX-114 in ALS is anticipated by the end of the year.
• Discussions with the FDA continue for the Wolfram syndrome trial design.
• Vexatide Phase 2 trials showed significant reductions in hypoglycemic events.

Operational Updates

Amlex Pharmaceuticals emphasized its commitment to addressing significant unmet needs in the medical community. The company’s lead asset, vexatide, is undergoing a Phase 3 trial for post bariatric hypoglycemia. Recruitment for this trial is expected to conclude by the end of the year, with data anticipated in the first half of 2026. In Phase 2b trials, vexatide showed a 53% reduction in Level II hypoglycemic events and a 66% reduction in Level III events, demonstrating its potential efficacy.

For AMX-35, the company reported positive outcomes in a 12-person open-label clinical study for Wolfram syndrome, showing sustained improvement in C-peptide levels, a marker of beta cell function. The Phase 3 trial design is currently in progress. Meanwhile, data for progressive supranuclear palsy (PSP) is expected in the third quarter of 2025.

Future Outlook

Amlex is optimistic about its pipeline’s potential impact. The company is particularly focused on vexatide, which has received FDA breakthrough therapy designation. Amlex aims to replicate the positive results from Phase 2 trials in the ongoing Phase 3 study. Additionally, early cohort data for AMX-114 in ALS is expected by the end of the year, with preclinical studies showing promising effects on biomarkers like neurofilament.

Q&A Highlights

During the Q&A session, executives addressed questions about the pharmacokinetics of vexatide, noting that its effects last through 24 hours, which is crucial for therapeutic benefit. They also discussed the estimated 160,000 individuals affected by PBH in the United States and the approximately 3,000 people with Wolfram syndrome. The company expressed confidence in completing recruitment for ongoing trials by the end of the year, with a focus on delivering impactful data in the coming years.

In conclusion, Amlex Pharmaceuticals remains committed to advancing its clinical pipeline to address significant medical needs. Readers interested in more detailed insights are encouraged to refer to the full transcript.

Full transcript - BofA Securities 2025 Healthcare Conference:

Josh: Reduces, you know, insulin and raises the blood sugar nadir, which is particularly important in diseases of hypoglycemia. It is FDA breakthrough therapy designation in both post bariatric hypoglycemia and congenital hyperinsulinism. And our focus right now is on post bariatric hypoglycemia, where there’s been five prior trials, all of which showed significant effects on glucose and insulin. We’re now conducting the phase three trial. We’ve started recruitment.

We expect to have recruitment complete by the end of the year with data in the first half of twenty twenty six. So that’s our lead program, and we can talk a lot more about that as we go through and including kind of the unmet need that these patients face, which is quite significant. And then, you know, very briefly on the on the rest of the pipeline, we have AMX thirty five, which targets endoplasmic reticulum stress and mitochondrial dysfunction. We just reported data in Wolfram syndrome, Phase two data that showed benefit across all the measures that we were measuring in Wolfram syndrome, which is an ultra rare kind of endocrine and neurodegenerative disease. We also have data coming in the third quarter in progressive supranuclear palsy, which we’ve been excited about based on tau lowering that we’ve seen with Amex thirty five and our view Amex thirty five is the only drug that both crosses the blood brain barrier and gets intracellular and reduces tau and PSP is a tauopathy.

So that’s where that kind of makes a lot of sense. And then finally, we’ve started recruiting with Amex one fourteen, our antisense oligonucleotide targeting calpin two in ALS. And we expect to have some early cohort data by the end of the year. And with that drug, we’ve seen effects in the pre clinic, including on biomarkers like neurofilament. So it’ll be interesting to see if those translate into the clinical data as we get later into the year.

But back over to you.

Unidentified speaker: Great introduction, Josh. So I actually want to take a step back. Just yesterday, you guys presented the long term data from the HELIOS trial in Wolfram’s syndrome. Can you just get the room up to speed on what Wolfram syndrome is? And what has the data told you?

Justin: Yes, very happy to. Thank you. So Wolfram syndrome is a monogenic disease caused by mutations in WFS1. Those mutations, WFS1 encodes for the Wolframin protein. Wolframin is an ER transmembrane protein.

So when it’s dysfunctional, it directly causes ER stress and mitochondrial dysfunction. That genetic cause causes degeneration and dysfunction first in the beta cells of the pancreas and then in different neuron types. So as you might imagine, from how I’m describing it, the disease presents as early onset diabetes, kids six, seven, eight. And then what sort of cues physicians on that maybe this isn’t just early onset diabetes is that kids get progressive vision loss and progressive hearing loss and early mortality. So it’s really a tough disease.

We estimate there are about three thousand people with Wolfram syndrome in The United States. It’s very much a global disease as well. We’ve been working in Wolfram syndrome now for almost eight years. As you might imagine, with the mechanism of sodium phenylbuterate and Tversadile targeting ER stress and mitochondrial dysfunction is very much a match of the mechanism of the drug to the mechanism of disease. So we’ve been evaluating AMX-thirty five in a 12 person open label clinical study in people in adults with Wolfram syndrome.

When we went into the trial, our hope in a progressive disease, especially in adults who have had the disease for some time, was to slow progression. We’ve actually seen stabilization or even improvement over time. What we just presented yesterday was the full cohort out to week forty eight, so about a year of dosing now. And what we’ve seen is actually the sustained improvement over time, particularly in C peptide. C peptide is a direct measure of insulin production.

So that’s how much the body is producing secreting insulin, which means the beta cells are functioning better. To our knowledge, this is the first time in any diabetic condition that C peptide has increased because, again, it means that the beta cells are functioning better. So we’re very excited about the data, what it might mean for people with Wolfram syndrome. So now we’re working on the design of a Phase III study, and we’ll update on that this year.

Unidentified speaker: So one of the really exciting things about this most recent data update is it’s supposed to inform your discussions with the FDA. What can you tell us about what they’re looking for, trial design, anything you can share?

Josh: Yeah. I’d say probably at the highest level, stay tuned. We’re still having those discussions. And, you know, certainly, when we have the, you know, final design of a of a phase three, you know, we will share that. I’d say, though, you know, we are quite excited with the data as as just now.

We you know, improvement numerically on the c peptide. All the other measures move in lockstep with that. So you see on the hemoglobin a one c on the time and target range by CGM, All those are moving in lockstep together with the c peptide, which is what you hope to see. It kinda gives you added confidence in what you’re seeing. Additionally, we saw stabilization or some improvement on visual acuity.

And these are patients who generally, you know, go blind over time. So to see the the vision pretty flat or even possibly improving was quite exciting. And then patients were also asked and the clinician was asked, you know, do you you know, are is the patient stable, improving, worsening? And every patient was described as at least stable or improving, you know, by both the clinician and by the patient. So as, you know, it’s a strong initial result, but it’s also the first you know, the first company to be developing an interventional therapy in Wolfram syndrome.

And so I think that’s and it’s a multisystem. There’s a lot of things you could measure. So I think those are kind of what drive the discussions with FDA of, you know, what is the best measure, hopefully, to, you know, bring this, you know, ultimately to patients.

Unidentified speaker: So maybe just in the interest of time, let’s turn to vexatide, which is the company’s lead asset. Again, can you just set the stage for the audience? What have you seen in the Phase II data? And how do you expect the data to show up in Phase III?

Justin: Yes. Well, I certainly wish I knew the answer to that now. But I’d say our hope is really if we can even replicate what we saw in Phase II, I think that would be a really meaningful advance for people with post bariatric hypoglycemia. The Phase II trials showed very significant reductions in level II and level III hypoglycemic events. Level II is defined by blood glucose.

Level III means that the person is so incapacitated that they need independent help. That can be severe confusion, it can be loss of consciousness, it can be seizures. So these are really dangerous episodes. And in both Phase two trials, vexatide showed very significant reductions in both of these types of events. Furthermore, showed dose dependency as well in the Phase III trial, we’re testing the highest daily dose tested ninety mg once daily.

So these are what supported FDA breakthrough therapy designation. The fact that PBH is a really significant unmet need. We estimate about one hundred and sixty thousand people in The United States currently have PBH. Unfortunately, it doesn’t go away. So the population will only grow.

There are no treatments currently for approved for PBH. So as Josh was saying, we go into this trial with a lot of excitement at the potential of what this could mean for people with PBH. And we’ll look forward to having those results in the first half of next year.

Josh: And maybe just to highlight as well, probably one of the key results from the past trials, it’s five past trials, so maybe this is still one of the key results. In the phase 2b, the ninety mg dose, which is the dose we’re taking forward in phase three, sort of 53% reduction in level two hypoglycemic events with a p value of 0.004 and a 66% reduction in level three events with a p value of 0.0003. And, you know, in the phase three trial, we’re measuring level two and level three hypoglycemic events. Our primary outcome is the composite of level two and level three hypoglycemic events. And we’ll share the data on the composite as well at endo upcoming in July.

But as Justin said, strong evidence coming from the past trials and the hope to again see that benefit on the level two and level three, which is the agreed upon primary outcome with FDA. Again, this program is breakthrough therapy status. So there’s been a lot of FDA interaction and that’s the agreed upon endpoint, you know, we believe to support a potential approval. So

Unidentified speaker: just to make it really clear in your discussions with the FDA and physicians, what’s considered a clinically meaningful, you know, number of PBH event reductions?

Justin: Yeah, it’s a really important point in question. So I start with currently, as I said, there’s no approved treatments for PBH. And so unfortunately, the bar is low, especially given what a severe condition this is. So when in terms of FDA, as Josh was saying, I think the composite endpoint that we’re looking at as a primary outcome is particularly meaningful. The level two and level three hypoglycemic events have been defined now for several decades by the American Diabetes Association and other endocrinology fields, as well as in FDA guidance.

So when we talk with physicians, what we really hear from them is that they want to keep their patients safe. If you think any one of these level three events can be life altering, we’ve heard many stories about someone driving a car, their blood glucose crashes, they pass out, they crash their car, they end up in the hospital. As we started to do market research, the number of times people end up in the hospital because of post bariatric hypoglycemia is just astonishing. So you understand from the physician’s perspective, really being able to reduce any number of these events is really meaningful. For people with PBH, what we hear consistently is, gosh, I just wish I had something.

I mean, it’s just I think it’s so frustrating for them that they finally get the diagnosis and then only to find out that, well, try to stick to this very draconian diet being avoid all carbs, eat small meals very, very frequently. And besides that, there are no approved therapies for PVH. So I think just highlights what an unmet need there is. And again, our hope that we can change that.

Josh: And one thing to touch on very, very quick too. In the past studies, patients were also interviewed in kind of a structured you know, interview that that was conducted with the patients. And they were asked, you know, now that you’ve completed this trial, been on this therapy, how would you rate it on a scale of one to 10? And every patient but one that was interviewed rated it a 10. The one other patient rated at a nine.

And their reasoning was, yes, the reduction in hypoglycemic events, but they also described just being able to feel a difference, you know, in terms of, you know, energy, fatigue, you know, things like that. Because if you imagine constantly having hypoglycemia, sometimes severe enough to cause, you know, loss of consciousness or otherwise. But even when you have hypoglycemia that’s just severe enough to make you lethargic, low energy, etcetera, to suddenly be out of that fog, you know, is a dramatic thing for patients. So, you know, we’re certainly, you know, with the as it relates to the events, as Justin said, you know, even one level three event, which could be losing consciousness or otherwise is a big deal preventing even one of those. But what we hope is kind of a more holistic ability for patients to kind of return to their normal life and their normal activities.

Unidentified speaker: Okay. Maybe just to wrap up on Avexatide. We know that you guys will have top line pivotal data in first half of twenty six. But just coming back to something you mentioned, you’ll be presenting some data at Endo very soon. Can you just highlight what the main takeaways investors should have from this data?

Sure. So two abstracts that we’ll be presenting, one on pharmacokinetics,

Josh: basically showing that vexatides pharmacokinetics last through twenty four hours, stay above the level where we believe there’s therapeutic benefit through twenty four hours. So we have one abstract kind of on PKPD modeling. An additional one is running kind of the phase three model on the phase two data so that, you know, you can kind of see how that performs. I’ll say, you know, neither of these do we expect to be, you know, particularly surprising, but nice kind of confirmatory and further evidence as we continue going along. And I’d say we also noticed at the conference, there is an abstract on the prevalence of PVH.

It’s a question we get frequently about, since it’s a new market, exactly how many patients have this, how severe are they, etcetera. And it was, you know, quite an interesting abstract arrived at pretty similar numbers to our hundred sixty thousand, but arrived from a very different angle and different methods. So it’s an interesting abstract to look at as well.

Unidentified speaker: With that, I think we’re coming to the end of our presentation time. We have another company presenting. I just wanted to add that, as you mentioned earlier, you will have data in PSV and three q twenty five. You know, look for Josh and Justin to maybe follow-up on that. With that, let’s stop there.

Justin: Excellent. Thank you.

Josh: Thank you so much.

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