Amylyx (AMLX) Cuộc họp công bố kết quả kinh doanh quý 3/2026: Hồ sơ NDA cho Avexitide đúng tiến độ

Amylyx Pharmaceuticals (NASDAQ: AMLX) đã dành trọng tâm cuộc họp báo cáo kết quả kinh doanh quý 3/2026 cho avexitide sau khi thử nghiệm Giai đoạn III LUCIDITY đạt tiêu chí đánh giá chính và toàn bộ tiêu chí đánh giá phụ trong điều trị hạ đường huyết sau phẫu thuật giảm béo (PBH). Công ty đang chuẩn bị nộp hồ sơ lên FDA và mở rộng năng lực triển khai nhằm hướng tới khả năng thương mại hóa vào năm 2027.

Những điểm chính

• Thử nghiệm Giai đoạn III LUCIDITY cho thấy tỷ lệ kết hợp của các biến cố hạ đường huyết Cấp độ 2 và Cấp độ 3 giảm 55% so với giả dược, với giá trị p là 0,000003. Thử nghiệm này cũng đạt được toàn bộ tiêu chí đánh giá phụ.
• Amylyx vẫn đang đi đúng lộ trình để nộp Hồ sơ Đăng ký Thuốc mới (NDA) cho avexitide lên FDA vào cuối năm 2026. Ban lãnh đạo đang tích cực chuẩn bị cho khả năng được phê duyệt và ra mắt sản phẩm vào năm 2027.
• Tiền và các khoản tương đương tiền đạt 768 triệu USD vào cuối tháng 9, tăng từ mức 251 triệu USD vào cuối quý 2, sau đợt chào bán cổ phần mang lại 545 triệu USD số tiền thu ròng.
• Tổng chi phí hoạt động tăng 38% so với cùng kỳ năm trước lên 49,7 triệu USD, phản ánh chi tiêu cho LUCIDITY, các chương trình phát triển khác trong danh mục và những hoạt động chuẩn bị trước thương mại hóa.
• Ban lãnh đạo ước tính có khoảng 160.000 người tại Mỹ mắc chứng PBH, trong đó khoảng 118.000 người sau phẫu thuật nối tắt dạ dày Roux-en-Y và khoảng 48.000 người sau phẫu thuật cắt dạ dày hình ống.
• Amylyx cũng đang thúc đẩy phát triển AMX0114 trong điều trị xơ cứng teo cơ một bên (ALS) và đặt mục tiêu nộp hồ sơ IND vào năm 2027 cho AMX0318, một chất đối kháng thụ thể GLP-1 tác dụng kéo dài.

Dữ liệu tài chính quan trọng

Ban lãnh đạo cho biết vị thế tiền mặt được mở rộng sẽ cho phép Amylyx bước vào giai đoạn tiềm năng thương mại hóa vào năm 2027 với đầy đủ nguồn vốn cho các ưu tiên ra mắt, chuỗi cung ứng dài hạn và tiếp tục phát triển danh mục sản phẩm.

Hiệu quả kinh doanh và vận hành

Avexitide và thử nghiệm LUCIDITY

LUCIDITY đã đánh giá avexitide, một chất đối kháng thụ thể GLP-1 đang trong giai đoạn thử nghiệm, ở những người mắc PBH sau phẫu thuật nối tắt dạ dày Roux-en-Y. Thử nghiệm đã đạt mức giảm 55% tỷ lệ kết hợp của các biến cố hạ đường huyết Cấp độ 2 và Cấp độ 3 so với giả dược.

Toàn bộ tiêu chí đánh giá phụ đều đạt được, bao gồm các biến cố Cấp độ 2 đo bằng phương pháp tự theo dõi đường huyết, các biến cố Cấp độ 2 đo bằng phương pháp theo dõi đường huyết liên tục và các biến cố Cấp độ 3 được hội đồng độc lập thẩm định.

Hơn 90% người tham gia đã hoàn thành giai đoạn mù đôi kéo dài 16 tuần, và toàn bộ người tham gia đủ điều kiện đều đã bước vào giai đoạn mở rộng nhãn mở đang diễn ra. Phần lớn các biến cố bất lợi đều ở mức độ nhẹ đến trung bình, không có biến cố bất lợi nghiêm trọng nào được coi là liên quan đến việc điều trị bằng avexitide.

Đây là thử nghiệm thứ sáu của avexitide trên bệnh nhân PBH mang lại kết quả có ý nghĩa thống kê. Toàn bộ kết quả của LUCIDITY dự kiến sẽ được trình bày tại hội nghị ObesityWeek 2026 vào ngày 15 tháng 11.

Chuẩn bị ra mắt thương mại

Amylyx đã chuyển từ khâu lập kế hoạch sang triển khai sẵn sàng ra mắt thị trường. Công ty đang tuyển dụng nhân sự ở các mảng tiếp thị, tiếp cận thị trường, dịch vụ bệnh nhân và vận hành thương mại, với kỳ vọng các bác sĩ nội tiết sẽ là nhóm kê đơn chính. Một số trung tâm phẫu thuật giảm béo được chọn cũng sẽ là mục tiêu tiếp cận tại những nơi họ tiếp tục điều trị cho bệnh nhân PBH.

Ban lãnh đạo cho biết các phân tích dữ liệu yêu cầu bồi hoàn bảo hiểm, khảo sát bác sĩ và tiếp cận thực địa tiếp tục củng cố ước tính khoảng 160.000 bệnh nhân PBH tại Mỹ. Một số cơ sở nội tiết được ghi nhận đang điều trị từ 40 đến 70 bệnh nhân, trong khi một số bác sĩ cho biết đang chăm sóc cho hơn 100 bệnh nhân PBH tiềm năng.

Mã ICD-10 dành riêng cho PBH đã chính thức có hiệu lực vào tháng 10/2026. Ban lãnh đạo kỳ vọng mã này sẽ giúp nâng cao nhận thức về bệnh và hỗ trợ nhận diện bệnh nhân theo thời gian, dù quá trình áp dụng trong toàn hệ thống y tế có thể diễn ra từng bước.

Phát triển danh mục sản phẩm

Amylyx đang tiến hành các nghiên cứu tiền đề nộp hồ sơ IND cho AMX0318, một chất đối kháng thụ thể GLP-1 tác dụng kéo dài. Ban lãnh đạo đặt mục tiêu nộp hồ sơ IND vào năm 2027 sau khi các nghiên cứu tiền lâm sàng cho thấy hiệu lực in vitro, hoạt tính in vivo, khả năng dung nạp và đặc tính dược động học phù hợp với một peptide tác dụng kéo dài.

Đối với AMX0114 trong điều trị ALS, công ty đã hoàn tất việc tuyển bệnh nhân cho Nhóm 3 của thử nghiệm tăng liều dần Giai đoạn I LUMINA và bắt đầu tuyển bệnh nhân cho Nhóm 4 ở mức liều 100 miligam. Theo ban lãnh đạo, dữ liệu ban đầu từ Nhóm 1 và Nhóm 2 cho thấy oligonucleotide đối mã này được dung nạp tốt.

Kế hoạch định hướng của ban lãnh đạo

• Amylyx dự kiến sẽ nộp NDA cho avexitide lên FDA vào cuối năm 2026.
• Công ty đang chuẩn bị cho khả năng avexitide được phê duyệt và ra mắt thương mại vào năm 2027, tùy thuộc vào kết quả thẩm định của cơ quan quản lý.
• Ban lãnh đạo đặt mục tiêu nộp hồ sơ IND cho AMX0318 vào năm 2027.
• Amylyx dự kiến tiếp tục đầu tư vào hạ tầng thương mại, tiếp cận thị trường, dịch vụ bệnh nhân và nguồn cung dài hạn trước thềm khả năng ra mắt avexitide.

Rủi ro và các yếu tố cần theo dõi

• Avexitide vẫn đang trong giai đoạn thử nghiệm, thời gian và kết quả thẩm định của FDA vẫn chưa thể chắc chắn.
• Thử nghiệm Giai đoạn III LUCIDITY thu nhận các bệnh nhân mắc PBH sau phẫu thuật nối tắt dạ dày Roux-en-Y. Ban lãnh đạo cho biết chỉ định và nhãn thuốc cuối cùng vẫn phụ thuộc vào quá trình xem xét của FDA.
• Hoạt động trao đổi với các bên chi trả bảo hiểm vẫn đang ở giai đoạn đầu. Amylyx dự kiến bắt đầu các buổi trao đổi thông tin từ quý 1, trong khi các yêu cầu quản lý sử dụng thuốc và định giá vẫn đang trong quá trình xây dựng.
• Ban lãnh đạo dự báo quá trình ra mắt sẽ tăng dần quy mô theo thời gian khi công tác phổ cập kiến thức về PBH, chẩn đoán và việc áp dụng mã ICD-10 được mở rộng ra ngoài các trung tâm điều trị lớn.
• Các nhóm ban đầu của thử nghiệm LUMINA sử dụng hai mức liều AMX0114 thấp nhất, nên còn quá sớm để đánh giá tác động lên dấu ấn sinh học. Ban lãnh đạo đang chờ đợi thêm dữ liệu từ Nhóm 3 và Nhóm 4.

Điểm nhấn phiên hỏi đáp với chuyên gia phân tích

Quy mô bệnh nhân PBH có thể tiếp cận: Amylyx đưa ra ước tính khoảng 160.000 bệnh nhân từ nhiều cơ sở dữ liệu yêu cầu bồi hoàn bảo hiểm, xác định những người từng phẫu thuật giảm béo và mắc chứng hạ đường huyết không do tiểu đường, kết hợp với các yếu tố như số lượt cấp cứu và điều trị nội tiết. Ban lãnh đạo cho biết khảo sát bác sĩ và tương tác thực địa cho kết quả nhất quán với phân tích dữ liệu bảo hiểm.

Lộ trình ra mắt: Ban lãnh đạo dự kiến ban đầu sẽ tập trung vào các bác sĩ nội tiết và các phòng khám có lượng lớn bệnh nhân PBH. Tăng trưởng trên diện rộng hơn sẽ phụ thuộc vào việc nâng cao kiến thức về bệnh, nhận diện bệnh nhân và nhận thức chung trong toàn hệ thống y tế.

Định giá và tiếp cận bên chi trả: Công ty đã tuyển dụng các giám đốc quản lý khách hàng trọng điểm cấp quốc gia và lên kế hoạch mở rộng trao đổi với các bên chi trả. Ban lãnh đạo nhận định rằng việc áp dụng điều trị bậc thang sẽ khó xảy ra do hiện chưa có liệu pháp nào được phê duyệt cho PBH, dù các tiêu chí tiếp cận cụ thể vẫn chưa được thiết lập.

Phạm vi chỉ định tiềm năng: Phần lớn các nghiên cứu về avexitide, bao gồm cả LUCIDITY, đều tập trung vào PBH sau phẫu thuật nối tắt dạ dày Roux-en-Y. Một nghiên cứu Giai đoạn IIb cũng bao gồm các phẫu thuật đường tiêu hóa trên và phẫu thuật giảm béo khác và đã mang lại kết quả tích cực. Ban lãnh đạo cho biết chỉ định cuối cùng không thể xác định trước khi có kết luận thẩm định từ FDA.

Bối cảnh điều trị hiện tại: Ban lãnh đạo đã trích dẫn ba thử nghiệm ngẫu nhiên cho thấy các chất chủ vận GLP-1 không mang lại hiệu quả cải thiện hạ đường huyết ở bệnh nhân PBH. Công ty kỳ vọng nhu cầu điều trị sẽ tập trung chủ yếu vào việc ngăn ngừa các biến cố hạ đường huyết.

Toàn văn biên bản cuộc họp báo cáo kết quả kinh doanh

Toàn văn cuộc gọi công bố kết quả kinh doanh

Phần trình bày của ban lãnh đạo

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.

Phần hỏi đáp

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.