Q1 2025 Amylyx Pharmaceuticals Inc Earnings Call
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Min: Good morning. My name is Meen and I will be your conference operator today. At this time, I would like to welcome everyone to the analytics pharmaceuticals first quarter earnings conference call. All participants will be in vision only mode.
Min: After to this presentation, there will be an opportunity to ask questions. To ask a question, please express is part one on your telephone keypad.
Min: To withdraw your question, please press part two. Please limit your questions to one with one follow up. If you have additional questions, you may rejoin the queue.
Min: Please be advised that this call is being recorded at the company's request. I would now like to turn the call over to Lindsay Allen, Vice President, Investor Relations and Communications. Please proceed.
Lindsey Allen: Good morning, and thank you for joining us today to discuss our first quarter 2025 financial results in business updates.
Speaker Change: With me on the call today, our Josh Cohen and Justin Klee, our co-CEOs, Dr. Camille Bedrosian, our Chief Medical Officer, and Jim Frates, our Chief Financial Officer.
Speaker Change: Before we begin, I would like to remind everyone that any statements we make or information presented on this call that are not historical facts are forward-looking statements that are based on our current police.
Speaker Change: Plans, and expectations, and are may pursuant to the safe harbor provisions of the Private Security's Litigation Reform Act of 1995.
Speaker Change: The impact thereof, and the expecting timing thereof, and statements regarding our cash runway.
Speaker Change: Actual events and results could differ materially from those expressed or implied by any forward-looking statement.
Speaker Change: You are cautioned not to place any undue reliance on these forward-looking statements, and Amalus disclaims any obligation to update such statements unless required by law. Now I will turn the call over to Justin.
Justin: Good morning, and thank you all for joining us. 2025 is an important year of execution at Amlix, as we advance three potential therapies across four clinical trials, each targeting diseases with high unmet need.
Justin: Already this year, we've achieved several meaningful milestones. Last month, we dose the first participant in our pivotal phase three lucidity clinical trial of the Vexatide in post-bariatric hypoglycemia
Justin: We also dose the first participant in our Phase 1 Luminical Trial of AMX 114 in ALS.
Justin: In addition, we strengthened our financial position by raising approximately $65.5 million at the start of the first quarter, which extends our anticipated cash runway through the end of the 2026.
Justin: Now, I'd like to briefly walk through each of our programs.
Justin: Starting with our lead asset, Avexatide, an investigational GLP-1 receptor antagonist with FDA-Break Sucerepidesignation for post-bariatric hypoglycemia.
Justin: PBH is a chronic and often progressive condition that affects approximately 160,000 people in the United States. However, there are no approved treatment options.
We believe that Exitide has the potential to fill that gap.
Justin: We are encouraged by the level of engagement from the clinical trial sites participating in our Pivotal Phase 3 Lucidity trial.
Justin: We continue to expect enrollment completion in 2025 and top-line data in the first half of 2026.
Justin: In addition, we are preparing diligently to be launch ready, and if approved, we anticipate a commercial launch in 2027.
Justin: Later during the call, Camille will share more details about a vexatide and lucidity trial.
Justin: Turning to AMX 35, which is an oral small molecule therapy designed to target endoplasmic reticulum or ER stress and might accondrial dysfunction.
Justin: AMX 35 is currently being evaluated in Wolfram Syndrome and Progressive Supernuclear Policy for PSP.
This disorder is caused by mutations in the WFS-1 gene.
Justin: The WFS-1 gene encodes a protein called Wolfram and that spans the membrane of the endoplasmic in particular, and mutations in Wolfram and directly cause ER stress and mitochondrial dysfunction.
Justin: We believe AMX 35 has the potential to address the urgent unmet need for approximately 3,000 people living with all from syndrome in the United States.
Justin: Last year, we reported positive top line data from the 12th person, Faith II, Open Label Helios Child, and Adults with both from Syndrome.
Justin: Participants showed improvement or stabilization across all measured outcomes at week 24.
Justin: In addition, longer-term data for the subset of participants who had reached treatment through week 48 showed sustained improvement over time.
Justin: We continue to follow participants in the Helios trial and plan to present full week 48 data at the upcoming Joint Congress of the European Society for Pediatric Endocrinology and the European Society of Endocrinology, which is this coming weekend.
Justin: The poster will be made available on the presentation's page of our website next Monday, and those findings, along with our ongoing discussions with the FDA, will inform the design of a phased retrial.
Justin: Now, I'd like to turn to AMX 35 as a potential treatment for progressive super nuclear palsy.
Justin: PSP is a rare, progressive and fatal neurodegenerative disease that affects and estimated 23,000 people in the US and is no currently approved treatment.
Justin: PSP is a tallopathy, which is defined by the build-up of cow protein in the brain.
Justin: Based on its prior effect in reducing cow and cerebral spinal fluid in people with Alzheimer's disease, we believe AMX-35 is the first brain and cell penetrant agent that has demonstrated a significant cow reduction in CSS to be tested in PSP.
Justin: We completed enrollment in the phase-to-be portion of the Orion trial in January of this year, with the total of 139 participants randomized.
Justin: We expect to report data in the third quarter of this year.
Justin: Those results will guide our decision about whether to advance into the phase 3 portion of the trial.
Justin: Next in our pipeline, this AMF114, our investigational anti-sense oligonucleotide targeting knockdown of CalPain II for the potential treatment of ALS.
Justin: This is a novel program built on decades of academic research linking the Proteus Calpain 2 to axonal degeneration, an early and destructive driver of ALS progression.
Justin: In pre-clinical studies, AMX 114 showed potent and durable reductions in calpane 2 levels, improved neuron survival, and reduced nerve filament light chain levels, a well-established biomarker of axonal degeneration.
Justin: We were excited to have those the first participant in our Phase One Luminatrial last month.
Justin: Lumina is a multinational, randomized, double-blind, placebo-controlled, multiple ascending-dose trial, evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of AMX 114 in people living with DLS.
Justin: We look forward to early cohort data from Lumina later this year.
Justin: With strong scientific rationale, clinical momentum and a clear path ahead, we believe we're well positioned to execute across our clinical programs.
Justin: With that, I'll now turn the call over to Camille to share more about the lucidity trial and our work with the Vexatide.
Camille: Thanks, Justin. We are very excited about the potential of a vexatide, our Investigational DLP-1 Receptor and Tagonist.
Speaker Change: The GLP-1 receptor, a mediator of important, well-characterized biology, is an effective target to modulate the biology.
Speaker Change: The GLP-1 receptor is one of the key regulators of insulin and glucose.
Speaker Change: Unlike GLP1 agonist, which increase insulin secretion, a GLP1 receptor antagonist decreases increases insulin secretion and therefore stabilizes to a glucose levels.
Speaker Change: A vexatizer shown promised to treat postbariatric hypoglycemia and as a result, has FDA breakthrough therapy designation.
Speaker Change: Last month, we dosed the first participant in the 16-week randomized double-blind placebo controlled phase 3 lucidity clinical trial, evaluating a vexatide in approximately 75 individuals with P.B.H. following rule on why gastric bypass surgery.
Speaker Change: Lucidity is designed to have similar inclusion and exclusion criteria to the previous successful Phase 2 prevent and Phase 2 B trials of a Vexatide in PDH.
Speaker Change: In addition, lucidity is evaluating the FDA agreed upon primary outcome of reduction in the composite of level 2 and level 3 hypoglycemic events through week 16.
Speaker Change: P.B.H. is a debilitating condition, believed to result from an excessive GLP1 response following bariatric surgery.
Speaker Change: P.B.H. Manifest as persistent, recurrent, and debilitating hypoglycemic events that can impose a life altering and enduring burden on a person's health, independence, and ability to engage
Speaker Change: On average, the symptoms appear approximately one to three years fall in bariatric surgery.
Speaker Change: Once people have TBH, the condition is chronic and often progressive.
Speaker Change: We estimate based on our projections from data and published literature and claims base work that there are about 160,000 people in the U.S. who are living with PVH today.
Speaker Change: Additionally, bariatric surgery remains a standard of care for addressing obesity particularly for people who require substantial and sustained weight loss.
Speaker Change: Therefore, we believe the unmet need and PBAs will continue to grow.
Speaker Change: A few weeks ago, the American Society for Metabolic and Bariatric Surgery, or ASMDS, published this new 2023 surgery data.
Speaker Change: The results estimate that approximately 270,000 new bariatric surgery procedures occurred in 2023 in the US, including 220,000 of the two most common surgical types, Ruan-Wai, Guest who bypassed surgery and freed Guest direct to me.
Speaker Change: There was a slight uptick in Rwanda, I guess, to bypass, and a slight downtick in sleep.
Speaker Change: Overall, there was no significant change in the procedure trends from the prior year.
Thank you for tuning in. We'll see you next time.
Speaker Change: Importantly, we'll believe that the biology of PDH is the same regardless of the type of bioretics surgery patients received.
and Despite Dietary Modification
Speaker Change: Rescue measures such as Glucogon, and off-label drugs, many people with P.B.H. continued to experience persistent symptoms and hypoglycemic events with no sustainable management options.
Speaker Change: Furthermore, there are no approved treatments for PB-8 and the current options used off-label generally are inadequate for this condition.
Speaker Change: The Lucidity trial is intended to build on the robust body of data generated to date for a bed-excited, which includes five clinical trials demonstrating consistent.
Speaker Change: Dose Dependent Effects, including Statistically Significant and Clinically Meaningful Reductions and Hypoglycemic Events.
Speaker Change: and a 66% reduction in level 3 hypoglycemic event with a p-value of 003.
Speaker Change: A vexatile is generally well tolerated with a favorable safety profile replicated across the clinical trial.
Speaker Change: 90 milligrams once daily of a vexatide, the dose who are evaluating a lucidity, also demonstrated a favorable pharmacokinetic profile, maintaining exposure in the therapeutic range to 24 hours
Speaker Change: This characteristic translated to similar meaningful improvements in native glucose levels as measured by continuous glucose monitoring, both during the day and overnight.
Speaker Change: We are excited to present additional analyses of the Avexatide Phase 2 and Phase 2 B studies at End of 2025 in July .
Speaker Change: These presentations will include new population PK and PD data supporting sustained effects at the 90 milligram once daily dose regimen.
Speaker Change: as well as a composite rate of level 2 and level 3 hypoglycemic event.
Speaker Change: We are encouraged by the engagement from the clinical trial sites and continue to expect to complete recruitment by the end of 2025.
Speaker Change: We are grateful to our trial participants, investigators and collaborators who inspire and guide us, guide our work each day.
Speaker Change: Now, I'll turn over the call to Jim to discuss the financial highlights from the corridor. Jim.
Jim: Thanks, Camille. We believe we're well positioned to achieve our goals. We ended the first quarter with a cast position of $204.1 million, which includes approximately $65.5 million in net proceeds from our public offering, which closed in January of this year.
Jim: We believe we have the necessary cash to deliver our planned clinical milestones through the end of 2026.
Jim: These milestones are top line data from the Phase III lucidity trial of the Exitide and PBH.
Jim: Week 48 data from the ongoing Helios trial in Wolfram Syndrome
Jim: Topline data from the Phase 2V portion of the Ryan Trial in PSP
Jim: and phased one data from our Luminatrial of AMX 114 and ALS.
Jim: In addition, our cash supports the advancement of our commercial preparations for the potential first-to-market launch of Exitide and PVH.
Jim: So, let's turn to our results. Total operating expenses for the quarter were $37.8 million, down 82% from the same period in 2024.
Jim: Research and development expenses were $22.1 million, compared to $36.6 million in Q1, 2024.
Jim: Primarily due to a decrease in spending on AMX 35 for the treatment of ALS, a decrease in payroll on personnel related costs and in preclinical development activities.
Jim: Selling General and Administrative Expenses were $15.7 million, compared to $57.8 million in Q1 2024.
Jim: Primarily due to a decrease in payroll and personal related costs and a decrease in consulting, professional and other services.
Speaker Change: Thank you for watching. I hope you enjoyed the video. If you did, please give it a thumbs up and subscribe. I'll see you next time.
Jim: We recognize $6.8 million of non-cash stock-based composition expense for the quarter.
Speaker Change: Compared to $9.9 million of non-cash duck-based compensation expense in Q1 2024.
Speaker Change: We also use roughly $6 million in cash, related to product rebates and the settlement of purchase commitments for AMX 35, who were established prior to the voluntary discontinuation of sales of Relivrio and Albrioza in April of 2024.
Speaker Change: We recorded $1.4 million of expense in the first quarter of 2025 related to these payments.
with the remaining expense recorded in prior periods.
Speaker Change: Going forward, the residual cash obligations related to the discontinuation of Relivrio and Albrioza are $3.1 million, which we expectably paid through the remainder of 2025.
Speaker Change: We're pleased with our cash position as we progress through the year, and we're reiterating our expected cash runway through the end of 2026.
Josh: With that, I'll turn the call over to Josh for some closing remarks.
Josh: Thank you, Jim. As we look ahead, we remain grounded in our mission to develop novel therapies for diseases with high unmet needs.
Josh: We are focused right now on strong execution across the four clinical trials, each targeting serious, neurodegenerative or endocrine disorders.
Josh: In the coming days, we are excited to share week 48 data from our Helios trial and Wolfram syndrome. And in the third quarter, we look forward to sharing unblinded phase 2B data from the Orion trial and PSP.
Josh: By the end of the year, we also expect early cohort data from the Phase I Luminatrial of AMX 114 and people living with ALS. And in the first half of next year, we expect top-line data from the Phase III Lucidity trial of the Vexatide and PBH.
Josh: We continue to believe we have the necessary cash to advance our pipeline and to support commercial preparations for the potential first-market launch of the Vesatide and PBH.
Josh: Thank you for your continued interest and we look forward to keeping you updated on our progress. Now I would like to open the call up for questions.
Thank you for watching. We'll see you next time.
Speaker Change: We will now begin the Q&A session to ask a question, this press is tar one on your telephone keypad. To withdraw your question, this press is tar two. Please limit your questions to one with one follow up. If you have additional questions, you may rejoin the queue. At this time, we will post momentarily to assemble our roster.
Speaker Change: Your first question comes from Michael DiFiore of every core IFI. Please go ahead.
Michael DeFiore: Thank you for taking my question and congrats on all the progress. Just two for me.
Michael DeFiore: One, Ananda Vectatide. Just your recent commentary on a Vectatide for PB8 suggests that a large...
Michael DeFiore: Patient Education Campaign will be required. My question is, is this because the 8% of symptomatic patients don't necessarily know that P.B.H. or, or will these educational efforts...
Attempt to shore up in penetrate into less symptomatic patients. Have a fun.
Michael DeFiore: Yeah, thanks Mike. Good, good, an important question. So I would say PBH, certainly among adult endocrinologists and sadly among people who are suffering with PBH is pretty well known, including the signs and symptoms.
Michael DeFiore: I think sometimes it can be a bit of a connecting the dots because oftentimes PVH takes years on average one to three years, falling in bariatric surgery to manifest.
Michael DeFiore: But adult endocrinologists when they can pretty clearly recognize the signs and symptoms of hypoglycemia, and then of course once they actually do testing in the clinic on blood glucose then it becomes very clear as well as at home measures as like finger stick blood glucose and CGM.
Michael DeFiore: So I think it's certainly well recognized, and unfortunately it's quite a severe condition as well. People can have such severe events as sudden loss of consciousness or even seizures.
and these are happening on a reasonably frequent basis.
Michael DeFiore: I think when we talk about the education it's because this is kind of classic rare disease. There have not been treatments before for PBH and I think as we find with many rare diseases there are many unmet needs throughout the community. Thank you.
Michael DeFiore: So we see it as really our job to make sure that we're educating the medical community, educating advocacy and people living with TBH and then hopefully if a backside is approved, educating them on the potential benefits of the backside as well.
Michael DeFiore: Got it, very helpful. My second question is on PSP. I just want to, as we head into the interim data, I just want to confirm the efficacy bogey on the PSP rating scale that we should be looking for in the interim. I just given that the placebo group in many prior PSB trials.
Declan by 10 or 11 points over 52 weeks.
Should we expect roughly half of that empecibo?
Michael DeFiore: And furthermore, I sources say that the minimally clinically meaningful difference on the PSP rating scale over six months is around six points. So taking together, should we expect maybe a flat to one point improvement in the drug treated arm? Thank you.
Michael DeFiore: So, you know, maybe first on the efficacy bogie, so this study has about 80% power to detect a 30% effect.
Michael DeFiore: on the PSB rating scale. And I'd say the PSB rating scales are primary end points, certainly going to be one of the main things we look at. But we do have other secondary end points and markers as well. So ultimately our decision on the next steps for the program will be driven by all the data.
you know, not just the PSPRS.
Michael DeFiore: I'd say, you know, PSP has only had so much work in terms of clinical meaningfulness.
Michael DeFiore: We have assembled some doctors and spoken to them as well and we've heard everything from a single point difference could be clinically meaningful. We've heard differences such as 20 or 30% change and I think it maybe bears in mind to discuss what is actually happening in this disease. We've heard differences such as 20 or 30% change and I think it maybe bears in mind to discuss what is actually happening in this disease.
Um...
Michael DeFiore: This is a disease that often has survival of six years, sometimes even less.
Michael DeFiore: and during that time these patients almost in a way reminiscent of ALS become eventually locked in. They'll have progressive motor impairment, progressive walking impairment, those speech and swallowing difficulties, so the ability to make that go a meaningful percentage slower.
Michael DeFiore: You know, I think our view and certainly many of the KOL's we've spoken to would be highly meaningful so you know won't put a specific line on it we're gonna look at all the data as it comes but I'd you know maybe just remind we're 80% powered to see a 30% effect. [inaudible]
That is very helpful. Thank you.
Speaker Change: Your next question comes from Mark Goodman of Learing. Please go ahead.
Speaker Change: Hi, good morning. Thank you for taking our question. This is Basma on for Mark.
Speaker Change: We had a question up on TBH regarding the prevalent population of 160,000 patients.
Speaker Change: Remind me this again, how many are seeking treatments? So this is basically a follow-up question, the previous one. So are all of these patients again are symptomatic? And also if you can provide any color on new incidents, so we know they're like 200 to 300 thousand surgery per year.
Speaker Change: Should we still assume that 8% of this surgery of the patient who undergo this surgery, you will eventually develop a deviation like three to four years time, framework, or should we assume something else? Thank you very much.
Speaker Change: Thank you for the great questions on the PVH population. So starting with your first question on the 160,000 by those people seeking treatment.
I'd actually say there's a larger group seeking treatment.
and I can walk you through that.
Speaker Change: If you look at the number of people who have hypoglycemia following bariatric surgery in any form and any frequency, it's as high as 30, 40, 50 percent of the population depending on the methodology you use. [inaudible]
Speaker Change: Now, people with hypoglycemia-falling bariatric surgery are counseled to use medical nutrition therapy, sometimes off-label prescriptions like a carbose or tried.
Speaker Change: So that, if you look at just even over the past decade of people, you're looking at a population of half a million to a million people in that group. When we're talking about P.B.H., we're talking about the people who had tried those things and yet still have persistent hypoglycemia.
Speaker Change: That's how we get to about 8% or about 160,000 people. So this is pretty rare. We're talking single visit percentage.
Speaker Change: of people who get bariatric surgery and, again, years to manifest.
Speaker Change: But it's a single digit percentage of a population of millions of people who have had a bariatric surgery, so in terms of seeking treatment as you might imagine people who have such a debilitating condition where they're having sudden and from what they can tell unexplained drops in blood glucose that leads to neuro glycopenia, which means their brains aren't functioning as they're supposed to are certainly seeking medical attention. Back to the first answer, you know,
Speaker Change: I think with rare disease what often happens, if you don't have a treatment then hopefully we can deliver one with a vexatide then I think suddenly you have options that weren't there before and I think as we were able to do with ALS.
Speaker Change: Access is certainly important and education is very important as well.
Speaker Change: Your second question should we continue to model 8% in the population? We certainly think so.
Speaker Change: I think as Camille mentioned, the new, the 2023 numbers from the ASMBS on bariatric procedures came out. I'm still well in 270,000 procedures in the year. And we know that with these upper GI surgeries, that there is a portion of people, again, single digit percentage, but a portion of people who will develop persistent hypoglycemia. All right, so.
Speaker Change: So we think that one of the major drivers of that is that the body seems to have a potentially accelerated GLP1 response.
Speaker Change: We think that's why a GLP-1 receptor antagonist makes a lot of sense in this condition and you know we see that continuing but again I'd remind this is a single digit percentage so it's not everyone but it's a very large population of people who are getting very average surgery.
Speaker Change: And I just have one smaller detail. What we've observed in literature and talking to KOLs is usually PBH appears one to three years following surgery. So it's you know possible a little sooner than that than the three to four years you mentioned.
Garrett, thank you so much for your help.
Speaker Change: Your next question comes from Tim Anderson with Back-up America Securities. Please go ahead.
Good morning, this is Susan on for Tim Anderson.
Speaker Change: We're really looking forward to the upcoming QAIRs data next week. My question is on the ongoing discussions with the FDA on trial design.
Speaker Change: What are some of the remaining questions or debates that you guys are having with the FGA on trial design and what are we expecting to learn from the 48 week data that will help inform one way or the other some of the decisions that will need to be made on trial design. Thank you.
Speaker Change: Thank you for the question. This is Camille. We are looking forward to presenting the week 48 data next week.
Speaker Change: I asked you to recall the ISPAD data that were presented for the week 24 data for Wolfram in the Wolfram study last year and now we have twice as much time for those participants in the study.
Speaker Change: So, we will be looking at similar endpoints and change in AUC in the CPEP type response to a mixed male tolerance as well as other glycemic measures such as hemoglobin A1C as well as visual acuity.
Speaker Change: and overall global impressions of change. So, I invite you to look at those data and see how things have evolved over an additional 24 weeks.
Speaker Change: We're calling that these are adults with this genetic disorder and it is a neurodegenerative and beta-cellegenerative disorder where progression is expected in these folks.
Speaker Change: Your first question was regarding the Phase III and the FDA.
Speaker Change: So, we really do not go into the details of our interactions and discussions with the FDA, having said that for sure the week 48 data will...
Speaker Change: Inform and is informing our Phase 3 program and when we have additional information and alignment with the agency on the Phase 3 protocol.
to be able to share that information.
Speaker Change: Just to remind, also, this is the very first clinical trial of an agent to potentially impact
Speaker Change: So the design, there's no template for the design of a trial in Wolfram Syndrome and so we're having those discussions for the FDA.
Speaker Change: Good to be here. Thank you Good to be here. Bye. Take care. Bye. Bye. Bye.
Hello.
Speaker Change: Alright, our next question comes from Graig Suvannavejh of Musibur. Please go ahead.
Speaker Change: Hi, this is Sam on for Greg, thanks for taking a question. Maybe a couple on thevexatide. First, will there be any kind of subgroup analysis?
Speaker Change: And then also assuming approval, do you anticipate any kind of step up or restrictions from a payer perspective that would limit access based on either severity of the disease or type of surgery or anything like that?
Thank you for tuning in. We'll see you next time.
Speaker Change: Good questions. So I'd say maybe first, you know, our kind of focus on the study is, you know, in the full study population, we are in rolling a population all of which are required to be having during, you know, a run in period, we have a three-week run in period, and people have to be having at least one level two or level three event per week.
Speaker Change: So we are enrolling a population that is having frequent events [inaudible]
Speaker Change: So all of the patients we have will have that characteristic as well.
Speaker Change: In terms of step therapy, there are no currently approved therapies for PBH, so I would say we do not anticipate step therapy in this indication.
Speaker Change: and certainly as we get closer to launch, we'll spend time interacting and educating on pairs but we do believe we have a quite exciting and differentiated approach here. We have FDA Breakthrough Therapy Designation, Firefire Trials showing differences in patients who are already trying the very best. We have FDA Breakthrough Therapy Designation, Firefire Trials showing differences in patients who are already trying the very best.
Speaker Change: to reduce these symptoms, and we're still seeing those differences even in that context.
Speaker Change: Yeah, and I would say that, you know, access is very important to us. If we believe that you have a treatment that can help people, you need to make sure that people can access it efficiently. So our team's already working on that. And I just remind, you know, for what I was saying earlier, that we're already talking about people who have been on medical nutrition therapy, which is really standard of care right now. And yet still have
of these persistent hypoglycemic events which are very, very debilitating.
and I think that's important because...
We're talking about a population that really needs help.
and from a physician and payer perspective.
Speaker Change: These are people who have to regularly seek medical attention for very dangerous events. So I think those will be very important messages as we do our market access education.
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That's a helpful caller. Thank you so much.
Speaker Change: Your next question comes from Joel Beatty of Baird. Just go ahead.
Joel Bietti: Thanks. My question relates to a Vexatide. GLP1 Agnes seemed to have many favorable short-term and long-term health effects.
Speaker Change: So, you know, with that in mind in that context, you know, what I guess you can't fit into that GLP1 entaginous like a backside won't cause some type of safety issue, and then I have a follow-up.
One That You Have Indoginously
Speaker Change: And so I dad with that, we don't really see or we haven't seen Wade Gain, Hyperglycemia, any of the things you might think about in that contact. So maybe I'd say that overall we've seen good safety profile to date and just kind of highlighting as well in the animal studies we even dozed.
Speaker Change: Many fold above from a human equivalent dose perspective, many fold above what we're dosing in the human clinical trials, and even there, we don't see particular adverse events of concern.
Speaker Change: Great, that makes sense. And then as a follow-up, how are you currently thinking about potential business development activities?
Speaker Change: So I would say one, we're very excited about our pipeline. We have major milestones across each of our four clinical trials.
Speaker Change: over the next 12 to 15 months, which is very exciting, and each is in a disease where there's no treatment or a substantial inadequate treatment.
Speaker Change: So we're very excited that the potential in each one of these programs, we, you know, are always. Thank you very much.
Speaker Change: Our mission is to help people who have unmet medical needs and so with that you know we're always making sure that we stay on top of what's promising but our focus right now as I was mentioning my remarks is really on execution because we have some very exciting milestones ahead. Thank you very much.
Thank you
Speaker Change: Your last question comes from Ananda Ghosh of H.C. Wainerite and Company. Please go ahead.
Ananda Ghosh: Hey, hi, comrades on the quarter. Maybe one question with respect to the ALS trial. Given the importance of NFL in this trial and especially with the pre-tinical data showing the effect of knocking on CalPain 2 on NFL.
Thank you.
Speaker Change: Can you remind me if the inclusion criteria for the trial, like during the inclusion criteria of the trial, did you consider the NFL levels of the LS patients, which might be higher compared to the
Ananda Ghosh: Yeah, Ananda that's a great question. It's actually something we discussed a lot as we were planning and designing the study as well. We do not have a inclusion criteria that requires particularly high NFL levels going into the trial. I think a couple things went into that.
One, just to, um...
Ananda Ghosh: Put context on it as well. In the CSF and ALS, NFL levels are often 10 times as high as normal, and it's quite a stark separation as well. So you can generally expect an ALS that particularly when you're measuring CSF.
You're going to see pretty high levels.
Ananda Ghosh: And then additionally, given that this is our first, you know, in human with the drug, we didn't want to oversubset and potentially miss some populations of signals that could be quite important. So we definitely will be looking at that, you know, looking at those patients who maybe come in with.
Ananda Ghosh: Higher as compared to lower neurofilament but we didn't want to exclude them from the trial in the initial trial.
You got it. Makes sense. Thanks.
Speaker Change: Thank you, we still have one question from that absurpee of Pareto Securities. Please go ahead.
Dan Apshupi: Hello everyone, and thank you for taking my questions, congrats on the progress and the excited for the hellish readout next week. Just a more general question, how you compare it, a bit to other drugs that could enter the space that are more inhibiting into lean and CHILPY wants a creation like Sumata Set in Analogue.
Speaker Change: and how you see the placebo response risk in post-bariatric hypoglycemia for the patients. Thank you.
Yeah, good good questions.
Ananda Ghosh: So maybe first I'd remind, you know, we're in phase three with Vexatide, we have breakthrough therapy designation, built on five prior successful trials.
Ananda Ghosh: So we think the profile of exotite is quite strong, both in terms of what we've seen thus far, in terms of safety and efficacy.
Ananda Ghosh: I think all you know any other programs in the space are quite a bit earlier and you know have a number of hurdles. [inaudible]
Ananda Ghosh: I would say to overcome and so you know we're focused on a backside at this time and do believe it as the best profile we've seen thus far.
Ananda Ghosh: Breakthrough Therapy means benefit over existing treatments and of course right now there are no existing treatments for approved treatments for pvh. So we're very excited about the potential and that's why we're focused on rigorous execution in this study. Yeah and I realized you also asked about placebo rate.
Ananda Ghosh: So, you know, in the phase two study, there was a placebo period as well and we did not see a meaningful difference between the run-in and the placebo. So I'd say it purically, we haven't really seen much of a placebo effect in this indication. That being said, when we did the power analysis for the study, we certainly thought about that and we do believe our power is robust that even if there is some degree of placebo effect we should have enough power regardless of the study.
Camille: Yeah, and this is Camille. I would just also comment that these individuals have tried and are trying everything to manage this very debilitating condition. And that really isn't going to change whether they're in a clinical trial or not. So there.
Their You
Camille: Bailey Life will not be changing because of being in a clinical trial.
Camille: There is no question at this time I'll turn the call back to Mr. Klee
Camille: Thank you operator and thank you all for your time. If you have any follow-up questions please reach out to Lindsey. We hope you have a great rest of your day.