Amylyx Pharma Q2 2026 Earnings Call Transcript

Amylyx Pharma (NASDAQ: AMLX ) held its second-quarter earnings conference call on Thursday. Below is the complete transcript from the call. This transcript is brought to you APIs. For real-time access to our entire catalog, please visit for a consultation.

8 million in Q1 2026, ensuring a cash runway into 2028. The company is preparing for the pivotal Phase 3 LUCIDITY top-line data readout for Avexitide, aiming for an NDA submission and potential commercialization in 2027. 7 million, driven by investments in Avexitide and strategic initiatives, despite a decrease in R&D expenses related to AMX35. The company launched a disease state education campaign, 'Uncover the Mystery of Post-Bariatric Hypoglycemia,' and is enhancing commercial and market access teams.

Management is optimistic about Avexitide's potential, emphasizing the unmet need in post-bariatric hypoglycemia and preparing for a 2027 launch if approved. Full Transcript OPERATOR Good morning, my name is Carrie and I will be your conference operator today. At this time I would like to welcome everyone to the Amylyx Pharma second quarter 2026 earnings conference call. All participants will be in a listen-only mode.

After today's presentation, there will be an opportunity to ask questions. To ask a question, please press star one on your telephone keypad. To withdraw your question, please press. Please be advised that this call is being recorded at the company's request.

I would now like to turn the call over to Lindsey Allen, Vice President, Investor Relations and Communications. Please proceed. Lindsey Allen, Senior Vice President, Investor Relations & Communications Good morning and thank you all for joining us today to discuss our second quarter 2026 financial results and business updates. With me on the call today are Josh Cohen and Justin Klee, our co-CEOs, Dr.

Camille Bedrosian, our Chief Medical Officer, Dan Monahan, our Chief Commercial Officer, and Jim Frates, our Chief Financial Officer. 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 beliefs, plans and expectations, and are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995.

These statements include, but are not limited to, our expectations with respect to Avexitide, AMX35, AMX114 and AMX318, statements regarding regulatory and clinical developments, the impact thereof and the expected timing thereof, and statements regarding our cash runway. Actual events and results could differ materially from those expressed or implied by any forward-looking statements. You are cautioned not to place any undue reliance on these forward-looking statements and Amylyx disclaims any obligation to update such statements unless required by law. Now I will turn the call over to Justin.

Justin B. Klee, Co-Founder & Co-CEO Good morning everyone and thank you for joining us. This is an exciting time for Amylyx and the post-bariatric hypoglycemia community as we approach the pivotal Phase 3 LUCIDITY top-line data readout. At the beginning of the year we outlined three strategic priorities for Avexitide, our investigational first-in-class GLP-1 receptor antagonist with FDA Breakthrough Therapy designation in post-bariatric hypoglycemia, or PBH.

First, advancing the pivotal Phase 3 LUCIDITY trial toward top-line data. The last participant has recently completed their last visit in the trial. We are on track and eagerly anticipating the top-line data readout in late August or early September. The database has yet to be locked at this time and we remain blinded to the data.

Second, in parallel, advancing NDA readiness and regulatory preparations. We are actively preparing all applicable sections of the NDA to support a potential submission; and third, strengthening our launch readiness to prepare for the potential commercialization of Avexitide in 2027, if approved. We have deployed our field medical affairs team to facilitate on-the-ground HCP scientific exchange and we have continued to build out our teams across marketing, market access and commercial operations.

At Endo 2026 in June, we activated our disease state education campaign, Uncover the Mystery of Post-Bariatric Hypoglycemia, designed to increase awareness, understanding and action around PBH. Dan will provide more details on our launch readiness efforts later in the call. We are fully focused on execution as we work toward the potential of delivering the first FDA-approved therapy for people living with PBH. With that, Camille, I'll turn the call over to you.

Camille Bedrosian, Chief Medical Officer Thank you, Justin. PBH is characterized by recurrent and often debilitating hypoglycemia thought to be caused by an exaggerated GLP-1 response, primarily after food intake. The American Diabetes Association recognizes hypoglycemia as a potential medical emergency because low blood glucose levels can compromise the body's ability to maintain essential physiologic processes and can result in cognitive dysfunction, seizures or loss of consciousness. PBH is a chronic condition with no FDA-approved therapies and many people with PBH live with the ongoing fear of hypoglycemia.

Our pivotal Phase 3 LUCIDITY trial is a randomized, double-blind, placebo-controlled trial evaluating Avexitide 90 milligrams once daily in adults with PBH following Roux-en-Y gastric bypass surgery. The LUCIDITY trial is anchored in the robust data generated to date from the five prior Avexitide clinical trials in PBH that demonstrated statistically significant reductions in hypoglycemic events. The primary endpoint is the FDA-agreed-upon outcome of reduction in the composite of level 2 and level 3 hypoglycemic events through week 16.

At the Endo 2026 annual meeting in June, several case reports describing many aspects of PBH and hypoglycemia were presented, highlighting a growing recognition of PBH and the seriousness of hypoglycemia across the medical community. Dr. Colleen Craig presented a poster characterizing the clinical, economic and humanistic burden of PBH in the US and evaluating how level 2 and level 3 hypoglycemic events in PBH impact healthcare utilization, productivity and overall cost at the patient level. Patients face direct medical costs, productivity loss such as missed work, diminished quality of life, caregiver burden and out-of-pocket non-medical costs.

At the systemic level, societies face resource utilization and downstream clinical consequences in addition to direct healthcare costs, non-medical system costs and societal productivity loss. Also at Endo 2026, we had meaningful scientific dialogue about PBH with endocrinologists that underscored the tremendous unmet need for people living with this condition. We met many endocrinologists with whom we had not previously engaged and many of them already were aware of PBH. They described the challenges in managing these individuals given the limited options for treatment and had strong interest in learning more about this condition.

This interest is inspiring as our field medical affairs team furthers PBH scientific exchange and engagement with endocrinologists. In addition, in June, CMS and CDC published their 2027 ICD-10 code files which represent the official code set to be used for patient encounters beginning October 1, 2026. This code set includes an ICD-10 code specific to PBH, also demonstrating the growing recognition of this condition by the medical community. In closing, we are encouraged by the increasing awareness and understanding of PBH along with our informative engagements with healthcare professionals.

We continue to be focused on strong execution as we approach our anticipated LUCIDITY top-line data readout. With that, Dan, I'll now turn the call over to you. Dan Monahan, Chief Commercial Officer Thank you, Camille, and good morning everyone. We continue to make significant progress in our preparations for the potential commercialization of Avexitide next year.

We remain encouraged by the growing recognition of PBH among healthcare professionals. Importantly, our understanding of the PBH population continues to strengthen. We estimate that approximately 160,000 people in the US are living with PBH following the two most common bariatric procedures, Roux-en-Y gastric bypass and sleeve gastrectomy. This estimate remains supported by our independent claims analysis, ongoing field insights, and a growing body of published prospective and retrospective literature.

Together, these data continue to reinforce both the significant unmet need and the opportunity to identify and reach appropriate patients. Our market research also continues to demonstrate a high intent among endocrinologists to treat PBH with an approved therapy, providing further confidence in the commercial opportunity should Avexitide be approved. We are excited to share that we recently activated our disease state education campaign, Uncover the Mystery of Post-Bariatric Hypoglycemia, to address the educational need among HCPs and the PBH community. com.

This platform provides educational resources for both healthcare professionals and the PBH community. The HCP website is intended to help clinicians better recognize and understand PBH. The community website empowers people with PBH with information to support discussions with their healthcare teams. As Camille mentioned, engagement at Endo 2026 was high, where we also introduced an interactive disease state education experience that generated strong engagement and positive feedback.

One of our most encouraging early observations was the level of familiarity many healthcare professionals already had with PBH, reinforcing our view that awareness of the condition continues to grow within the endocrinology community. In parallel, we continue to add key talent across the organization and advance our commercial readiness efforts as we prepare for the potential launch of Avexitide in 2027, should it be approved. And with that, I will now turn the call over to Jim to review our financials. Jim Frates, Chief Financial Officer Thanks, Dan.

Good morning everyone. Our financial results for the second quarter reflect our continued disciplined execution of the Phase 3 LUCIDITY trial and targeted investment in advancing our broader pipeline. 8 million at the end of the first quarter. This capital provides us with an anticipated cash runway into 2028 to fund our operations through expected milestones including our key focus, the LUCIDITY top-line readout, potential FDA approval and potential commercial launch of Avexitide in 2027.

7 million, up 7% from the same period in 2025. 2 million in Q2 2025. The decrease was primarily due to a decrease in spending related to AMX35 for the treatment of progressive supranuclear palsy, offset primarily by an increase in spending related to the clinical development of Avexitide in PBH and other costs related to Avexitide. 6 million in Q2 2025.

This increase was primarily due to increased legal expenses, including a one-time legal charge, and investment in commercial strategic initiatives. 4 million of non-cash stock-based compensation expense in Q2 2025. With the LUCIDITY top-line data readout later this month or early September, we're entering into an exciting phase. We're investing in a disciplined manner as we prepare for the potential launch of Avexitide and we believe we're well funded to execute.

With that, I'll turn the call over to Josh. Joshua N. Cohen, CEO and Founder Thanks, Jim. In addition to Avexitide, we continue to advance our broader pipeline and our commitment to the communities we serve with high unmet needs.

For AMX35 in Wolfram syndrome, we presented week 96 data from the Phase 2 open-label HELIOS clinical trial in the spring which continued to show stabilization or improvement in measures of glycemic control and vision, consistent with week 24 and week 48 data. For AMX114 in ALS, we continue to advance the Phase 1 LUMINA multiple ascending dose clinical trial in people living with ALS. We are progressing through the dose cohorts given the favorable safety profile AMX114 has exhibited in LUMINA so far.

Camille Bedrosian, Chief Medical Officer We are currently enrolling Cohort 3, and for AMX318, our long-acting GLP-1 receptor antagonist, IND-enabling studies are underway and we are targeting a 2027 IND filing. Additionally, we are pleased to share we entered into a second research collaboration with Gubra in July following the collaboration that identified AMX318. This new collaboration will screen and develop peptide candidates for another rare endocrine disease of high unmet need. Joshua N.

Cohen, CEO and Founder We are looking forward to sharing additional updates on this program as it advances. In closing, we are executing against our three strategic priorities for avexitide, including delivering top-line data from Lucidity, advancing our NDA readiness, and strengthening our commercial launch preparations. Guided by the profound unmet need of the PBH community, we are working with urgency to bring this potential treatment to people living with PBH. Before we turn it over to the operator for Q&A, I want to reiterate that this is an exciting time for Amylyx Pharma as we are eagerly awaiting Phase 3 top-line data.

Because we are getting close to locking the Lucidity trial database, we will be entering a quiet period after this call. With that, operator, we are now ready to take questions. OPERATOR We'll now begin the Q&A session. To ask a question, please press star, then the number one on your telephone keypad.

To withdraw your question, please press star one again. At this time we'll pause momentarily to assemble our roster. Your first question will come from Simon Fernandez with Guggenheim. Simon Fernandez, Analyst at Guggenheim Oh great.

Thanks for the question. So I just have a couple here. You know, I believe maybe you guys could just update us on when the last patient visit was and, you know, what are the kind of key factors that kind of need to be completed to lock the database—where are the areas of kind of core focus? Or is it, you know, the sort of need to get all of the sites kind of fully aligned to be able to lock the database?

And then the second question is, you know, I'm guessing you won't be able to update us on specific numbers, but maybe you can provide us a little bit of color on how the early access program is progressing at this point. Are patients actively being recruited into the EAP? And, you know, how would you kind of characterize the demand for that program at this point? It's obviously an opportunity to really put the company commercially on a very strong footing going forward.

Thanks. Camille Bedrosian, Chief Medical Officer Great. Seamus. Hi.

Thanks for the questions. So your first question revolved around last patient visit and what efforts need to continue to be ready for the database lock and then top-line data. If we start with when our last patient first visit was, we announced we completed enrollment in late March, or mid-March, I would say. And so it's a 16-week double-blind period.

So advance toward that we get to about mid-late July. And there are, as you point out, rightly so, a number of activities that the team is working on, and they're continuing to execute beautifully. And the enthusiasm continues very strong by the trial participants as well. There is data cleaning, making sure that everything matches up, dotting all the I's, crossing all the T's, because we only lock a database once in this instance for the core study, the 16-week double-blind period.

So the team is being very diligent in that regard. And as we said, we expect top-line data late August, early September. With regard to the early access program, yes, we're so excited to have an early access program for people living with PBH. The initial phase, if you will, of the EAP, as we abbreviate it, is for those individuals in Lucidity who have completed the study double-blind period, of course, and then the open-label extension, and then they have the opportunity to roll over into the EAP.

And it also, at this period of time, is open for those individuals who have been in prior avexitide trials. Many of those individuals are at other centers, so they have to be activated as well in order to participate in the EAP. It's a separate study, separate protocol, so we're early days yet. The enthusiasm is great about being able to participate in the EAP, and we'll continue to give you updates along the way.

Simon Fernandez, Analyst at Guggenheim Great, thank you. OPERATOR Your next question will come from Joseph Toomb, TD Cowen. Joseph Toomb, Analyst at TD Cowen Hi there. Good morning and thank you for taking my questions.

I'm not sure if you can answer this, but I guess in terms of the patient population that you did enroll for the phase, I guess, does it overall look similar to the prior Phase 2 experience or any sort of comments you can make on that?