Amylyx (AMLX) 2026年第三季度业绩电话会：Avexitide NDA按计划推进

在avexitide治疗减重术后低血糖症（PBH）的III期LUCIDITY试验达到主要终点和所有次要终点后，Amylyx Pharmaceuticals（NASDAQ: AMLX）将2026年第三季度财报电话会议的重点放在了该药物上。该公司目前正准备向FDA提交申请，并扩大商业化上市能力，以备在2027年实现潜在的商业化。

核心要点

• III期LUCIDITY试验显示，与安慰剂相比，2级和3级低血糖事件的复合发生率降低了55%，p值为0.000003。该试验还达到了所有次要终点。
• Amylyx仍按计划将于2026年底前向FDA提交avexitide的新药上市申请（NDA）。管理层正在为2027年可能获得的批准及商业化上市做准备。
• 在完成带来5.45亿美元净收益的股权融资后，截至9月底，现金及现金等价物达到7.68亿美元，高于第二季度末的2.51亿美元。
• 总运营费用同比增长38%至4970万美元，反映出在LUCIDITY试验、其他管线项目以及商业化前准备活动上的支出。
• 管理层估计，美国约有16万人患有PBH，其中约11.8万人接受了Roux-en-Y胃旁路术，约4.8万人接受了袖状胃切除术。
• Amylyx还在推进治疗肌萎缩侧索硬化症（ALS）的AMX0114，并计划于2027年提交长效GLP-1受体拮抗剂AMX0318的新药临床试验申请（IND）。

关键财务数据

管理层表示，充裕的现金储备将使Amylyx在2027年进入潜在商业化阶段时，拥有充足资金支持重点上市工作、长期供应以及持续的管线开发。

业务与运营表现

Avexitide与LUCIDITY试验

LUCIDITY试验评估了在研GLP-1受体拮抗剂avexitide在接受Roux-en-Y胃旁路术后出现PBH的患者中的疗效。与安慰剂相比，该试验使2级和3级低血糖事件的复合发生率降低了55%。

所有次要终点均已达到，包括通过自我血糖监测评估的2级事件、通过持续葡萄糖监测评估的2级事件，以及经独立裁定的3级事件。

超过90%的受试者完成了为期16周的双盲期，且所有符合条件的受试者均进入了正在进行的开放标签扩展期。大多数不良事件为轻度至中度，未发生被认为与avexitide治疗相关的严重不良事件。

该研究是avexitide在PBH领域取得统计学显著结果的第六项试验。LUCIDITY试验的全部结果计划于11月15日在2026年肥胖周（ObesityWeek 2026）上公布。

商业化上市准备

Amylyx已从上市规划阶段转向上市筹备执行阶段。公司正在市场营销、市场准入、患者服务和商业运营等部门招聘人员，预计内分泌科医生将是主要的处方开具者。部分仍继续管理PBH患者的特定减重手术中心也将成为目标。

管理层表示，理赔数据分析、医生问卷调查和一线实地沟通持续支持其对美国约16万名PBH患者的估算。经确认，部分内分泌诊所管理着40至70名患者，而某些医生报告其诊治的潜在PBH患者超过100名。

针对PBH的专属ICD-10编码已于2026年10月生效。管理层预计，随着时间的推移，该编码将提高疾病认知度和患者识别率，不过医疗系统对该编码的采纳可能是一个渐进的过程。

管线研发进展

Amylyx正在为长效GLP-1受体拮抗剂AMX0318开展支持IND申报的临床前研究。临床前研究显示该药物具有体外效力、体内活性、良好的耐受性以及符合长效多肽特性的药代动力学特征，管理层计划于2027年提交IND申请。

针对治疗ALS的AMX0114，公司已完成I期LUMINA多剂量递增试验队列3的入组，并开始在100毫克剂量组入组队列4。据管理层介绍，队列1和队列2的早期数据显示，该反义寡核苷酸耐受性良好。

管理层指引

• Amylyx预计将于2026年底前向FDA提交avexitide的NDA申请。
• 在监管机构审查的前提下，公司正为avexitide在2027年可能获得批准并实现商业化上市做准备。
• 管理层计划于2027年为AMX0318提交IND申请。
• Amylyx计划在avexitide可能获批上市前，继续投资于商业基础设施、市场准入、患者服务和长期供应。

风险与关注要点

• Avexitide仍处于在研阶段，FDA审查的时间表和最终结果存在不确定性。
• III期LUCIDITY试验入组的是Roux-en-Y胃旁路术后的PBH患者。管理层表示，最终的适应症和药品说明书仍取决于FDA的审查。
• 与支付方的沟通仍处于早期阶段。Amylyx计划从第一季度开始与支付方进行信息交流，而用药管理要求和定价仍在制定中。
• 管理层预计，随着PBH的疾病普及教育、诊断和ICD-10编码的采纳从大型诊疗中心向外拓展，上市放量将随着时间推移逐步显现。
• LUMINA试验的前期队列采用了两个最低剂量的AMX0114，因此目前评估生物标志物效应还为时过早。管理层正寄望于从队列3和队列4中获取更多数据。

分析师问答环节亮点

PBH目标患病人群：Amylyx对约16万名患者的估算是基于多个医保理赔数据库得出的，筛选条件包括有减重手术史且出现非糖尿病性低血糖的人群，同时结合了急诊就诊和内分泌诊疗等因素。管理层表示，医生问卷调查和实地沟通得出的结果与理赔数据分析相符。

上市放量节奏：管理层预计初期将重点聚焦内分泌科医生及PBH患者集中的诊所。更广泛的增长将取决于疾病普及教育、患者识别以及整个医疗系统对该病的认知提高。

定价与支付方准入：公司已聘请全国大客户总监，并计划开启更广泛的支付方沟通。管理层认为，由于目前尚无获批用于治疗PBH的疗法，推行阶梯疗法限制可能会比较困难，但具体的准入标准尚未确立。

潜在说明书适用范围：包括LUCIDITY在内的大多数avexitide研究均侧重于Roux-en-Y胃旁路术后的PBH。一项IIb期研究还纳入了接受其他上消化道及减重手术的患者，并取得了积极结果。管理层表示，在FDA完成审查前尚无法确定最终适应症。

当前治疗现状：管理层援引了三项随机试验，这些试验显示GLP-1受体激动剂在PBH治疗中未能带来低血糖获益。公司预计，治疗需求将主要集中在预防低血糖事件上。

财报电话会议完整纪要

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完整财报电话会议逐字稿

管理层陈述

Operator

Good morning. My name is Reya, and I will be your conference operator today. At this time, I would like to welcome everyone to the Amylyx Pharmaceuticals' Third Quarter 2026 Earnings Call. [Operator Instructions] Please be advised that this call is being recorded at the company's request.

I would now like to turn the call over to Courtney O'Leary, Director of Investor Relations. Thank you. Please go ahead.

Courtney O'Leary

Good morning, and thank you all for joining us today to discuss our third quarter 2026 financial results and business update. 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, AMX0035, AMX0114, and AMX0318, 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 expected 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 Klee

Thank you, and good morning, everyone. It has been a momentous period for Amylyx. In the third quarter, we reported positive top line results from the Phase III LUCIDITY trial of avexitide, an investigational first-in-class GLP-1 receptor antagonist in participants with Post-Bariatric Hypoglycemia or PBH, following Roux-en-Y gastric bypass surgery. Fueled by the strength of this data, we completed an equity offering to support our launch readiness efforts to potentially deliver the first ever approved therapy for PBH in 2027. Our pipeline development efforts with avexitide and our other programs remain strong as well, which Josh will talk about later in the call.

As we reflect back on what has already been a milestone year for Amylyx, we are proud of our team's strong execution across the 3 key objectives for avexitide that we outlined in the first quarter. In the third quarter, we continued to make substantial progress toward achieving each of them, and we now look ahead to another potential milestone year in 2027.

Our first objective for 2026 was to execute on the Phase III LUCIDITY trial of avexitide in people with PBH, following Roux-en-Y gastric bypass surgery. In August, we reported positive top line results from the pivotal Phase III LUCIDITY trial. Avexitide demonstrated consistent, highly statistically significant, clinically meaningful reductions in hypoglycemic events across all endpoints with a favorable safety profile.

We were pleased to learn that these data were accepted as a late-breaking oral presentation at the upcoming ObesityWeek 2026 Annual Meeting on November 15. As we have spent more time engaging with the PBH community and sharing the top line data, our conviction in the significant unmet need and market opportunity has grown with many physicians reacting positively to the strength of the LUCIDITY data.

Our second objective was to execute on our NDA submission efforts. Our team has been actively preparing the submission, and we remain on track to submit our NDA to the FDA by the end of this year.

Our third objective was to execute on our launch readiness efforts for avexitide, anticipating a potential 2027 approval and launch as the first ever approved treatment for PBH. We have an experienced commercial and medical affairs leadership team in place, and our efforts this year have given us a strong understanding of the patient journey for people with PBH and the healthcare professionals who care for them.

Building on the momentum generated from the LUCIDITY results and supported by the additional capital we recently raised, we're investing in the capabilities, infrastructure and team needed to support a successful launch. We believe we have the opportunity to potentially change practice in PBH, a debilitating disease which, to date, has had no approved therapies. It is this opportunity informed by the stories of the many people with PBH we have heard from that will continue to drive our team to execute on our objectives with precision and urgency. We look forward to an exciting year-end and another potential transformational year in 2027.

With that, I'll turn the call over to Camille to discuss LUCIDITY results in more detail, our NDA submission, and our field medical team efforts.

Camille Bedrosian

Thank you, Justin, and good morning, all. PBH is a chronic metabolic condition characterized by recurrent and often debilitating hypoglycemia that can lead to cognitive impairment, confusion and in some cases, seizures or loss of consciousness. The condition can have a profound impact on daily life with patients often structuring their lives around the constant risk of a hypoglycemic event and facing challenges maintaining their independence, employment, and family responsibility.

In one study, more than 90% of individuals with PBH describe themselves as living with a disability, underscoring the profound impact the condition can have on quality of life. Endocrinologists consistently describe PBH as one of the most challenging conditions they manage. Despite this burden, there are currently no FDA-approved therapies.

LUCIDITY met its prespecified primary endpoint, demonstrating a 55% reduction in the composite rate of Level 2 and Level 3 hypoglycemic events compared to placebo with a p-value of 0.000003.

The study also met all secondary endpoints with consistent, highly statistically significant and clinically meaningful reductions across Level 2 events measured by self-monitoring of blood glucose, Level 2 events measured by continuous glucose monitoring, and independently adjudicated Level 3 events.

Importantly, for people living with PBH, preventing even one Level 2 or Level 3 hypoglycemic event can have a meaningful impact on daily life. Furthermore, the American Diabetes Association recognizes hypoglycemia as a potential medical emergency. Against this backdrop, we believe the consistency, statistical strength and clinical meaningfulness of the LUCIDITY results show its potential.

LUCIDITY is the sixth clinical trial of avexitide in PBH, and all of those trials generated statistically significant results. In addition, LUCIDITY is the largest and longest study of avexitide conducted to date. Alongside the efficacy results, avexitide demonstrated a favorable safety profile, consistent with that observed across the clinical development program to date.

Avexitide was generally well tolerated. The majority of adverse events were mild to moderate, and there were no serious adverse events deemed related to avexitide treatment. More than 90% of the participants completed the 16-week double-blind treatment period and all eligible participants entered the ongoing open-label extension portion of the trial. Additionally, our expanded access program is ongoing.

Our field medical team has been focused on actively facilitating scientific exchange around the LUCIDITY data. They have established a strong presence, engaging directly with a substantial number of healthcare professionals to answer questions about the data and gain insights regarding the unmet need and patient journey in PBH.

Across these interactions, they continue to hear that awareness of PBH among specialists is high, recognition of the serious consequences in PBH is growing and endocrinologists see a significant need for an FDA-approved treatment option given that there are currently no approved therapies. These discussions reinforce our belief that avexitide has the potential to be the first FDA-approved treatment to address a significant unmet need in PBH.

In addition, we are pleased that the LUCIDITY results will be presented to the medical community on November 15 at ObesityWeek, results that we believe will continue to demonstrate the consistency and strength of the data set and the overall potential of avexitide to change practice in PBH. Our team remains focused on executing our planned NDA submission by year-end. And as a reminder, avexitide has both breakthrough therapy and orphan drug designations from the FDA.

With that, I'll now turn over the call to Dan.

Dan Monahan

Thank you, Camille, and good morning, everyone. Bolstered by the strength of the Phase III LUCIDITY data and with additional capital, we have transitioned from launch planning to executing on our launch readiness road map. Our continued engagement with the PBH community has reinforced our belief that this indication represents a significant market opportunity with a substantial unmet need. We are investing in the team and capabilities needed to realize the long-term potential of avexitide if approved.

As we prepare for a potential launch in 2027, our efforts are focused on 3 key areas. First, we are applying our growing understanding of the PBH market and the patient journey to inform our go-to-market plans. This includes our ongoing claims analysis work, additional market research, and direct engagement with healthcare professionals as well as patients. These efforts help validate the patient population size and provide valuable insights into where patients with PBH are being treated.

Second, we are expanding our disease state education campaign, uncoverpbh.com, with the goal of increasing recognition and understanding of PBH across the broader clinical community. This campaign has been well received and reached a broad number of endocrinologists, bariatric surgeons, and people living with PBH.

Third, we are scaling our commercial organization and infrastructure. The full commercial leadership team is now in place, and we are making key hires across marketing, market access, patient services, and commercial operations. We are also investing in capabilities needed for a successful launch. More broadly, we continue to see increasing recognition of PBH across the medical community and healthcare system. Just last month, a PBH-specific ICD-10 code went into effect, which reflects the growing awareness of PBH within the medical community.

We believe PBH represents a significant and underserved market opportunity. We estimate there are approximately 160,000 people living with PBH in the U.S. Of this population, approximately 118,000 people were estimated to have received Roux-en-Y gastric bypass surgery and approximately 48,000 people are estimated to have received sleeve gastrectomy. Our independent claims analysis and ongoing field engagements to understand the market opportunity continue to support these estimates.

In conclusion, with compelling LUCIDITY results and key hires in place, the team is focused on executing launch preparations to potentially commercialize the first approved therapy for PBH in 2027.

And with that, I will now turn the call over to Jim to review our financials.

James Frates

Thanks, Dan. Good morning, everyone. We had an excellent quarter from a financial point of view. The success of the LUCIDITY trial has allowed us to move to the next phase of growth, executing on our NDA preparation, scaling our commercial organization and building long-term supply by investing in both our current and a new supplier. We're preparing for a large potential market, and we're well on our way.

Financially, our plans were enhanced by our recent oversubscribed equity offering that gave us net proceeds of $545 million. That left us with $768 million in cash and cash equivalents at the end of September compared to $251 million at the end of the second quarter. We were thrilled to bring in anchor orders from a number of leading experienced investors in commercial biotech companies. As we've highlighted on this call, the more work we do in this area, the more excited we get. And we believe we're well funded to deliver on our goals and importantly, enter potential commercialization in 2027 from a position of financial strength with the capital necessary to execute on our launch priorities.

So turning now to our results for the quarter. Total operating expenses for the quarter were $49.7 million, up 38% from the same period in 2025. Research and development expenses for the quarter were $28.4 million compared to $19.9 million in Q3 2025. The increase was primarily due to an increase in spending related to the pivotal Phase III LUCIDITY clinical trial and other costs related to avexitide and our other programs. The increase was partially offset by a decrease in spending related to AMX0035.

Selling, general and administrative expenses for the quarter were $21.3 million compared to $16.2 million in Q3 2025. This increase was primarily due to an increase in personnel-related costs to support our pre-commercialization activities and the vesting of PSU awards during the quarter.

We also recognized $10.4 million of non-cash stock-based compensation expense for the quarter compared to $7.1 million of non-cash stock-based compensation expense in Q3 2025. We're focused on a disciplined capital allocation, prioritizing investments that support the advancement of avexitide in both the short and the long term, preparation for a potential commercial launch and continued development of our pipeline. We believe we have the team, the data and the capital in place to execute on our ambitious plans.

With that, I'll turn the call over to Josh.

Joshua Cohen

Thanks, Jim. As you heard from our remarks today, with positive Phase III results in hand, our first priority remains clear: to advance avexitide towards potential approval and commercialization. For many people living with PBH, the condition has a debilitating impact on day-to-day life, underscoring the urgent need for an approved treatment option. Later this month, we are excited to share that we will be hosting an in-person and virtual investor event at ObesityWeek with experts who will discuss the current burden of PBH and the data on avexitide generated to date. We look forward to seeing many of you there.

Looking beyond our initial launch opportunity, we are also investing in a long-acting GLP-1 receptor antagonist, AMX0318. In preclinical studies, AMX0318 demonstrated strong in vitro potency, evidence of in vivo efficacy and tolerability, and a favorable pharmacokinetic profile consistent with a long-acting peptide. IND-enabling studies are underway and progressing well, and we are targeting a 2027 IND filing. We see opportunities to broaden avexitide's reach into additional indications and to pursue geographic expansion in markets where there is a significant unmet need.

Taken together, we believe these efforts position us not only to bring a potential first approved treatment to people living with PBH, but also to drive multiple long-term opportunities for future growth. We also continue to make progress across our broader pipeline.

For AMX0114 in ALS, we continue to advance the Phase I LUMINA multiple ascending dose clinical trial in people living with ALS. We are progressing through the dose cohorts and recently completed enrollment in Cohort 3 and began enrolling participants in Cohort 4.

Just last week, we presented data from the LUMINA trial at the Annual NEALS Meeting from the initial 2 dose cohorts. These data are available on the Amylyx Publications page.

Additionally, over the summer, we entered into a second research collaboration with Gubra to screen and develop peptide candidates for another rare endocrine disease of high unmet need. We continue to make progress on this program and look forward to sharing updates as it advances.

Reflecting on the year, the positive LUCIDITY results were a defining milestone for Amylyx. And now our focus shifts to commercial execution, an area that we have deep experience in as we prepare for a potential launch of avexitide and pursue what we believe is a significant long-term opportunity ahead.

With that, operator, we are now ready to take questions.

Operator

[Operator Instructions] Your first question comes from the line of Joseph Thome of TD Cowen.

分析师问答

Jacob Ormes

This is Jacob on the line for Joe. I just wanted to ask on the sales force strategy for avexitide and, sort of, which kind of physicians you'll be targeting?

Dan Monahan

Sure. Thanks, Jacob. So from a sales force perspective, endocrinologists are going to be the primary prescribers here. Just on a broader perspective, we have initiated our go-to-market efforts. So that project is well underway. But from an overall approach, you can envision this as -- it's certainly a rare endocrine launch, and I think there's a few that you can reference to look at from a sizing perspective. But endocrinologists will be the primary prescribers here. We are aware of some bariatric surgery centers that also maintain connectivity with these patients. So we're aware of those, and we'll focus on some of them as well.

Operator

Next question comes from the line of Seamus Fernandez of Guggenheim.

Seamus Fernandez

Great. Thanks for the question. So a couple of quick ones here. In terms of LUCIDITY and what we're going to see this weekend, can you just help us understand what it teaches about disease severity and the impact of avexitide on patients with very severe disease? And I was just kind of hoping that you might be able to extend that into our learnings about potential low-hanging fruit and the patients that doctors are really going to be seeking to treat urgently or those patients that are going to be seeking to get into the endocrinologist's office relatively quickly.

And just the second question is on the EAP, where do we stand just in terms of the kind of inclusion of OLE patients into the early access program? How far along and how many patients have been recruited, if you're willing to update that? Or if not, what does it take to kind of activate those patients? And do you see an opportunity to further expand the EAP? Or are there potential limitations to that, whether it be supply, the 3 lots that you have to kind of confirm with FDA, et cetera, et cetera. So those are the questions.

Joshua Cohen

Thanks. I think I caught all of them. And thanks, Seamus as well. So maybe starting on LUCIDITY and the disease severity. I mean, I think first, it's important to note that even a single Level 2 or Level 3 hypoglycemic event can be a medical emergency. So people who are experiencing those, and we believe that's the 160,000 people living with PBH are -- have a need for medical therapy in our view. When we've done ad boards, LUCIDITY required people to be having at least one event of Level 2 or Level 3 severity per week during the run-in period. When we've done advisory boards with different physicians, they described that it's fairly typical of their PBH patients. Certainly, some have it more frequently, some less, but it's not uncommon to see people having an event a week or more.

In terms of -- I think you kind of mentioned low-hanging fruit from kind of a commercial perspective. I think it's a little early to say. But I will say, as we've been to different conferences, ENDO and others, we do see a high degree of interest from physicians, kind of, proactively coming to our booth or otherwise asking for more information, kind of noting that this is a really difficult condition for them to treat and that they're really looking for, kind of, new options and have excitement about avexitide.

And then, I think in terms of the EAP, probably unsurprisingly, there's a lot of excitement and interest in the EAP. We probably won't give, sort of, interim numbers as it goes on, especially because the main point of the program is access. But it is a clinical trial. So you do have to activate sites. There is the similar kind of contracting and otherwise as you might see in clinical trials, but we've had quite a lot of interest for that program.

Operator

Next question comes from the line of Geoffrey Meacham from Citibank.

Geoffrey Meacham

I just have a couple. The first is, when you think about the 160,000 U.S. PBH patients, maybe just help us with the math that you've done that informs how many are officially diagnosed and in medical management that are the truly addressable populations in the, call it, the first year or so?

And the second question, just again on the EAP. Can you talk at least about what you can learn that would inform the patient identification, persistent rates, just related to the launch execution?

Joshua Cohen

Sure. I'm happy to start, and maybe I'll pass to Dan as well. So in terms of the math on diagnosis, I think we start from the claims data. And when we've done our claims-based analysis, we've been able to identify approximately 160,000 people in claims who we believe have PBH. And PBH only recently got an ICD-10 code. So we did that work initially looking at those people who had hypoglycemic events following bariatric surgery that couldn't be explained by other means. But if they're having those codes, we sort of look at that population as certainly one that's being looked at by the medical profession, possibly diagnosed as well.

What I'd add to that, sort of, helped confirm this, we went out and did survey-based work where we kind of query sites about how many patients are under their care. And what we found was that their responses were very concordant with our claims-based data, which again, to us confirm that our claims-based data is successfully identifying those patients that the physicians view as sort of diagnosed and under their care. And then, I'd say in terms of EAP, it's probably early to talk about persistency rates. I think we'll probably hold off on that until we're further along and probably until we're hopefully into commercialization as well.

I don't know if you want to add anything, Dan, as well.

Dan Monahan

I think you covered it really well. I would say from the claims analysis that we've done, and we've done it multiple times looking at several different databases, we continue to feel more and more confident in the size and the representation of the 160,000. We start with the known patient who's had bariatric surgery, and then we apply the hypoglycemic -- hypoglycemia diagnosis, non-diabetes hypoglycemia. And then we add in other factors, whether it was a trip to the emergency room, a visit to an endocrinologist, back to their bariatric surgeon. So that's how we're tracking. We feel every time we look at it, we feel more and more confident in the size of that and the representation of the 160,000.

Operator

Next question comes from the line of Marc Goodman from Leerink Partners.

Marc Goodman

A different type of question. I was thinking about business development and just how much time you guys are spending on finding the next avexitide? And are we looking at CNS, are we looking at endocrinology? Like what areas are you looking in? And how likely is it that over the course of the next 12 months, you might bring in another product like this one?

Justin Klee

Yes. Thanks, Marc. I appreciate the question. So I'd say, first and foremost, we have a lot to do around here, which we're very excited about submitting the NDA by the end of this year and then preparing for launch in an unmet need with a rare but substantial population in the United States. I'd say even beyond that, there's a substantial unmet need of PBH internationally. There are other surgeries that lead to the same condition. There -- and then, of course, we have our ongoing work with AMX0318 as well with the goal to submit the IND for that next year as well.

So -- and then, of course, we also have AMX0035 in Wolfram Syndrome and AMX0114 in ALS. So we have a lot going on here. That being said, I think that we have a really excellent team here who has shown great ability to work in areas of high unmet need, execute on clinical development, and we are now building our commercial capabilities as well. So we always, sort of, keep our eyes open for unmet need and opportunities for the company, but we have some very strong programs we're working on right now. So that's really our focus for the -- certainly for the next 12 months that you asked about.

Operator

Next question comes from the line of Michael DiFiore from Evercore ISI.

Michael DiFiore

Two quick ones for me. In terms of any payer discussions or feedback you may have gotten so far, any feedback have you received on various DUR requirements for therapy, whether it be documented low glucose, nutritional therapy failure, off-label medication step edits, et cetera?

And my second question is on pricing. I know it's early, but how are you balancing rare endocrine pricing with broad access?

Dan Monahan

Sure. Thanks, Mike, for the question. The first one on the payer front. So we're in the process of building out our payer team right now. So we've hired our national account directors. They're currently on board and they're getting ready to engage with the broader payer community, and those conversations will take place pretty soon.

From a utilization management criteria, those are all the conversations that we'll begin to have with the payers as we start to have the payer information exchanges in the first quarter of the year. I think, from a utilization management, again, I think the first thing I'd say, there are no approved medications for patients with post-bariatric hypoglycemia. So I think it'd be hard for a payer to put a step or a criteria in place where a product is not approved. But that's all the education and the conversations that we'll have with the community in the coming months to learn more and educate the payers about post-bariatric hypoglycemia.

From a pricing perspective, the second question, it's work again that we're going to start and kick off now that we have our LUCIDITY results. First thing I'd say on the pricing from the commercial perspective, we're extremely excited about the LUCIDITY results. I think the clinical impact and the opportunity to potentially become the first approved treatment for patients with PBH, that's quite significant and not something we will take lightly. A little bit early to discuss the specifics on pricing. I think there's certainly some rare products with similar sizing, similar prevalence populations and even similar in the rare endocrinology space. So more to come as we get more and more specific within the pricing segment.

Justin Klee

I'll add just briefly on the payer point, too. So while we're doing that work now, as Dan indicated, I'll highlight that what I think is helpful here is that while PBH is new for payers, hypoglycemia is certainly not given their coverage of insulin and other drugs that can cause severe hypoglycemia. And severe hypoglycemia has been known for a long, long time to be really a medical emergency. So I think we have a body of literature to draw on to help educate payers as to the severity of this condition when you have people who are having such frequent medical emergencies. So I think that's really the first objective for our team is to highlight just what a critical condition PBH is. And then, of course, we believe we have the opportunity to potentially change practice with the first treatment that can treat those hypoglycemia events.

Operator

Next question comes from the line of Rami Katkhuda from LifeSci Capital.

Rami Katkhuda

I guess, do you expect PBH patients to be concentrated at high-volume bariatric surgery centers or key endocrinology practices? And how does that influence your commercial launch strategy? And then I know it's early, but how should we think about the initial launch trajectory for avexitide? How large of a bolus of patients do you anticipate at launch given the EAP and open-label extension?

Dan Monahan

Excellent. I can start. Yes. So on the concentration of the marketplace and the patients with post-bariatric hypoglycemia, again, that's work that we've been doing. We've mentioned the claims analysis to validate the 160. Within those -- that analysis, we can also see where these patients are. So we can see whether there's a setting of care or an endocrinologist that has 40 to 60 patients, some have upwards of 70, and we've even spoken with endocrinologists that have greater than 100 patients or potential patients with post-bariatric hypoglycemia in their care. So that's certainly where we'll focus.

We also know that there's a broader educational effort that's needed to raise awareness of, one, the severity of post-bariatric hypoglycemia, but also the signs and symptoms. So as our disease state education efforts take shape and as our field medical colleagues get out there and continue to educate the marketplace, I think you'll see the treatment expand beyond the top centers. And I wouldn't even say it's within the top centers. It's just we know that's where a lot of these patients are because these are extremely severe patients. And oftentimes, they are then channeled towards a particular expert in the local marketplace.

So from a launch perspective and the launch trajectory, again, the first thing I'd say is, there's no approved therapies for post-bariatric hypoglycemia. It's certainly a sizable market. It's 160,000 size that's quite significant for a rare disease. But it's also a market that we continue to see and we believe that will build over time.

With the endocrinologists that we've done research with that we continue to engage with, there's a very high intent to treat these patients with post-bariatric hypoglycemia. So again, as we know where these -- a lot of these patients are. We certainly don't know where all of them are. So I think we'll see a good concentration upfront. But then as our educational efforts take shape and more and more awareness of post-bariatric hypoglycemia occurs across the healthcare setting, we'll see that launch continue to build over time.

Operator

Next question comes from the line of James Condulis from Stifel.

James Condulis

Congrats on all the progress. Maybe just one on, sort of, the current treatment landscape. I think one thing we've heard from some doctors is just anecdotally that GLP-1 agonists have been used in this space. And just like, kind of, like curious your latest perspectives there, and if that has sort of like any implications, if at all, on, sort of like, how the PBH treatment landscape will evolve over time.

Joshua Cohen

Sure. I think as we've talked to physicians in the space, we've heard kind of 2 concepts there. One, the concept of could a GLP-1 agonist be helpful for hypoglycemia and then another one of just if people want to lose more weight. Talking on the hypoglycemia, there's actually been 3 randomized trials, all of which showed no benefit of GLP-1 agonist for hypoglycemia in PBH, which is -- makes a lot of sense when you consider these drugs were originally approved for diabetes to lower your A1c, basically to lower your blood sugar. It would be quite surprising for them to raise your blood sugar. So we do not believe, based on the current evidence that these are effective for hypoglycemia.

In terms of weight loss, yes, certainly, they can be effective for weight loss, but most people who have PBH have already lost significant weight. They've had a bariatric surgery. So we don't anticipate that to be a particularly large pool of utilization either. So it's something we'll definitely watch, but I don't think -- I think what people are really looking for in this disease, given the severity, given the frequent medical emergencies that these patients are experiencing is treatment of hypoglycemia. And I think that's what we hope to offer with avexitide.

Operator

Next question comes from the line of Graig Suvannavejh of Mizuho.

Samuel Lee

This is Samuel, on for Greg. Maybe two quick ones for me. First, in terms of the NDA for avexitide, will that be only Roux-en-Y? And so how are you guys thinking about the gastric sleeve patients? And do you guys have a study planned there? And then maybe on the pipeline for LUMINA study in Cohorts 2, 3, any guidance on when we can expect data? And then in terms of expectations of what would be a good signal in terms of the biomarker responses given the Cohort 1 kind of stayed near baseline?

Camille Bedrosian

Great. Thank you very much. With regard to the NDA, we'll be submitting the data from the overall avexitide program, featuring prominently, of course, the LUCIDITY trial, which was conducted in people with PBH following Roux-en-Y gastric bypass surgery. And most of the studies conducted were in people having had Roux-en-Y. Having said that, we also had a Phase IIb study in which individuals also received other upper GI surgeries and other upper bariatric surgeries as well. And those data were also -- those results were also positive. So we're submitting the data, and we're very excited about the data, as you've heard about the LUCIDITY results, highly statistically significant and clinically meaningful. And beyond that, the data will speak for themselves, I would say.

And then you had another question about the pipeline, LUMINA, and what we might see there. So first, we're very excited about the progress of the LUMINA study. As we noted, we've completed enrollment in Cohort 3, which is our third of 4 dose levels, 50 milligrams, and we're currently enrolling Cohort 4 at 100-milligram dose level. We also recently presented results of Cohorts 1 and 2 at the NEALS Congress. And we were very pleased that the data show a very well-tolerated ASO in people with ALS. And those were the first lowest doses, the Cohort 1, the lowest and then the next lowest dose. So early days yet to see biomarker results. And therefore, we're very excited to see how the data unfolds in Cohorts 3 and 4. Importantly, we're able to go to Cohorts 3 and 4 because the drug is well tolerated. So we're very excited about the potential here.

Justin Klee

And Sam, just to the first question as well. So obviously, we have yet to submit the NDA, and so we're a ways off from the label indication statement. We believe that PBH is PBH, the pathophysiology is the same. But as Camille indicated, the Phase III trial was in PBH following Roux-en-Y gastric bypass surgery. So then, as we look forward, first, I'd say, the current existing prevalent population in the United States, our estimates are that about 120,000 of the 160,000 currently have Roux-en-Y gastric bypass that have led to their PBH. And we'll be prepared for whatever scenario.

Avexitide, you can see the effect of it in a single dose. And as Camille indicated, we had strong data from the Phase IIb of different surgeries leading to the same hypoglycemic condition and avexitide appears to work regardless of the surgical intervention that led to the condition. So we'll be prepared for any scenario and both in terms of our launch preparations and our development preparation.

Operator

Next question comes from the line of Jason Gerberry from Bank of America.

Dina Ramadane

This is Dina on for Jason. Two quick ones from us. Does the dedicated ICD-10 code provide any sort of a launch tailwind or change your ability to identify high-volume treaters heading into launch? And then at the LUCIDITY top line, you said that the treatment and placebo distributions were essentially not overlapping once the patients were on drug. When you present the full data set, should we expect that benefit to look relatively uniform across patients? Or is there a subset of super responders? And specifically, what proportion of patients became completely free of Level 2, 3 events?

Dan Monahan

Sure. I can start with the first question on the ICD-10 code. So yes, an ICD-10 code for -- specifically for post-bariatric hypoglycemia did go into effect in the beginning of October. We definitely view the availability of a dedicated code as an important milestone for this community. It's also a reflection of the growing awareness of PBH. So that's something we are looking forward to.

With the ICD-10 code, they're often used for diagnosis as well as tracking of patients within the healthcare system. The code itself, though, it's not needed for reimbursement, but it does represent an important step towards improving disease awareness as well as patient identification over the long term. What I would say on the code, specifically a new one, with the new diagnosis code, adoption as well as implementation of a specific code for PBH, it's going to take some time to be absorbed across the broader medical community.

Joshua Cohen

Yes. And then on maybe the LUCIDITY distribution of patients question. Maybe first, I'd say, just to remind, we're incredibly excited about the LUCIDITY results. I think the more we've dug into them, just the more excited we've continued to get. We met our prespecified primary outcome. We met every secondary outcome. We also had a favorable safety profile. So getting to the presentation, I'll probably let Marilyn Tan in her presentation to give, kind of, all the details. But our hope there is certainly just to kind of give more detail across the secondaries, across the safety, et cetera, all of which has been quite favorable. And given the strength of the p-value, what that essentially means is the error bars are pretty small here. So the distributions are pretty tight in terms of how the active group looks and in terms of how the placebo group looks.

Operator

Next question comes from the line of Ananda Ghosh from H.C. Wainwright.

Ananda Ghosh

Congrats on the quarter. I have two questions from the commercial front. Of the patients identified in your claims work, how many have been validated as actively managed clinically confirmed PBH patients at your initial target centers? And now that the PBH code is effective, what will you measure to distinguish existing patients being recorded from newly diagnosed patients entering care?

Dan Monahan

So I can start with the first question just on the -- specifically on the patients. And of the patients that we've used within the -- we've identified within the claims analyses, one, we've done it 4 times now, multiple times and every time we look at it. So we continue to feel more and more comfortable in the sizing of the particular marketplace. But specifically, as Josh mentioned earlier in his comments, we've also referenced that with additional market research. So we've done additional market research survey work to validate the 160,000 specifically within very specific settings of care. But then we also have our field medical colleagues who are out there engaging and talking with key opinion leaders as well as large practices and some of the experts that continue to validate the size of the marketplace within the local geographies.

Joshua Cohen

Yes. And then maybe on your question around the code as well. Again, probably a little early as codes take a little bit of time to kind of get implemented into people's medical record systems and all those sorts of things. But yes, we'll certainly continue looking at that. But probably the most important thing is what it represents in terms of this disease growing in terms of awareness and kind of being a key disease that hopefully will be tracked and managed well by endocrinologists and otherwise.

Operator

Next question comes from the line of Christopher Chen of Baird.

Christopher Chen

Congrats on a successful quarter. Just regarding patient eligibility and avexitide's potential label, I believe patients in your trials needed to have been at least a year removed from bariatric surgery. Just curious as to why that criterion was in place? And is it possible that avexitide's potential label could include patients less further removed from bariatric surgery?

Camille Bedrosian

Yes. Thanks, Chris. So we did -- that was the eligibility, individuals had to be at least 1 year out. And it really is in part historical, we want to be as consistent as possible with how the prior trials were conducted. And why 1 year? Because that is generally the time frame in which symptoms and signs of PBH develop in people post-bariatric surgery. So that's the context there.

With regard to the label, well, right now, we're focusing on submitting our NDA, and it is too early yet to be contemplating what the label or the indication will be.

Joshua Cohen

And maybe only other comment, just also important to note that if you think about the time immediately after bariatric surgery, there's a lot of changes happening for the individual. It's not uncommon over the first 6 months for people to lose 100-plus pounds following bariatric surgery. So you do also, in the trial setting, want to run in a case where they've achieved some degree of stability before entering the trial as well.

Justin Klee

Yes. And I'll just add one more, I think, important note of context, prevalent population. Once PBH occurs, it does not appear to go away. We very regularly work with people who've had PBH for 15, 20 years. So I'd say it's very unfortunate for people living with PBH. But again, we think it represents both the severity, the unmet need of the condition, but the opportunity in front of us as well.

Operator

Next question, or last question, comes from the line of Rudy Li of Wolfe Research.

Guofang Li

Can you maybe provide more color on the uncovered mystery of PBH campaign? Were you able to locate more patients in your database and how many healthcare professionals have signed up? Like what's the specialty mix?

Dan Monahan

Great. Thanks for the question on the disease state education campaign. And it's a campaign that, again, we launched it in June and the response has been quite significant. And the feedback we received from both bariatric surgeons, from endocrinologists, we're aware of primary care physicians who have engaged for education. So the response has been quite high. But we've also received specific feedback on the site, too, just that there's a recognition of the unmet need, the need for more education across the broader healthcare system. So we're excited about the results.

Again, we've put it out there in many channels, but that's something as we continue to expand and prepare for a potential launch in '27, we are bringing that and bringing uncovered PBH to a broader community within the healthcare ecosystem. From a specific metrics perspective, I don't think we're going to get into which -- the numbers of endocrinologists or bariatric surgeons, just to say that we're excited about the results, and we're very happy with the engagement from both patients as well as healthcare professionals.

Operator

There are no further questions at this time. I will turn the call back to Mr. Klee.

Justin Klee

Thank you, operator, and thank you all for your time. If you have any follow-up questions or want to connect, please reach out to our Investor Relations team. We hope you have a great rest of your day.

Operator

Ladies and gentlemen, that concludes today's call. Thank you all for joining. You may now disconnect.