Cuộc họp công bố kết quả kinh doanh Quý 3/2026 của Zevra (ZVRA): MIPLYFFA thúc đẩy tăng trưởng doanh thu 45%

Thông tin trọng tâm

• Tổng doanh thu thuần quý 3/2026 tăng 45% so với cùng kỳ năm ngoái lên 37,8 triệu USD, chủ yếu nhờ 34,0 triệu USD doanh thu thuần từ MIPLYFFA tại Mỹ.
• Doanh thu MIPLYFFA tăng 12,6% so với quý 2/2026 và tăng 52% so với quý 3/2025. Zevra đã nhận được 13 đơn đăng ký kê đơn trong quý, đưa tổng số lượng đăng ký tích lũy từ khi ra mắt lên 197.
• Lợi nhuận thuần đạt 19,2 triệu USD, tương đương 0,31 USD trên mỗi cổ phiếu cơ bản và 0,26 USD trên mỗi cổ phiếu pha loãng, so với khoản lỗ thuần 0,5 triệu USD của cùng kỳ năm trước. Kết quả bao gồm khoản tín dụng giá trị hợp lý phi tiền mặt 5,5 triệu USD liên quan đến chứng quyền.
• Tiền, các khoản tương đương tiền và các khoản đầu tư đạt 270,9 triệu USD tính đến ngày 30 tháng 9 năm 2026, tăng khoảng 10,7 triệu USD so với ngày 30 tháng 6.
• Zevra dự kiến sẽ nộp Đơn đăng ký thuốc mới bổ sung cho MIPLYFFA đối với bệnh nhân dưới 24 tháng tuổi trong quý 1/2027. Ban quản lý ước tính nhóm này chiếm khoảng 5% tổng số bệnh nhân có thể tiếp cận điều trị.
• Công ty dự kiến nhận được ý kiến của CHMP về arimoclomol trong quý 4/2026 và lên kế hoạch thảo luận thêm với FDA trong nửa đầu năm 2027 về chiến lược nộp hồ sơ tiềm năng cho celiprolol có kết hợp dữ liệu thực tế.

Dữ liệu tài chính cốt lõi

Kết quả hoạt động kinh doanh và vận hành

Động lực thương mại của MIPLYFFA

Zevra đã báo cáo 13 đơn đăng ký kê đơn mới trong quý 3, đưa tổng số kể từ khi ra mắt lên 197. Quý này bao gồm cả bệnh nhân người lớn và trẻ em, cũng như tỷ lệ đáng kể các bệnh nhân mới được chẩn đoán. Ban quản lý cho biết số lượng đăng ký tiếp tục cân bằng tương đối giữa người lớn và trẻ em.

Tỷ lệ chi trả của bên bảo hiểm đạt 69%, trong khi hầu hết các bệnh nhân còn lại đạt được chi trả thông qua các quy trình ngoại lệ y tế. Công ty cũng báo cáo tỷ lệ duy trì điều trị và tái kê đơn cao, phù hợp với trải nghiệm chương trình mở rộng khả năng tiếp cận trước đó.

Zevra tin rằng số lượng bệnh nhân NPC đã được chẩn đoán tại Mỹ có thể lớn hơn ước tính trước đây là 300 đến 350 bệnh nhân. Ước tính tỷ lệ lưu hành trong quá khứ là khoảng 900 bệnh nhân, dựa trên một nghiên cứu năm 2021 được thực hiện trước khi có liệu pháp điều trị NPC được phê duyệt. Phân tích yêu cầu bồi thường bảo hiểm, hợp tác xét nghiệm di truyền và các ca đăng ký mới được chẩn đoán liên tục đang củng cố quan điểm của ban quản lý rằng cơ hội thương mại nằm ở khoảng giữa các con số đó.

Công ty đã mở rộng nhẹ các đội ngũ tiếp xúc khách hàng để tiếp cận các cơ sở y tế cộng đồng ngoài các trung tâm chuyên khoa hàng đầu. Trang web nâng cao nhận thức về bệnh NPC của công ty đã ghi nhận gần 56.000 lượt truy cập trong quý 3, tăng 123% so với khoảng 25.000 lượt trong quý 2.

Mở rộng chỉ định của MIPLYFFA và sở hữu trí tuệ

Sau cuộc họp Loại B với FDA, Zevra dự kiến nộp sNDA trong quý 1/2027 nhằm mở rộng chỉ định của MIPLYFFA cho trẻ sơ sinh dưới 24 tháng tuổi. Ban quản lý cho biết nhóm tuổi này chiếm khoảng 5% tổng số bệnh nhân có thể tiếp cận, khiến quy mô mở rộng về con số tương đối nhỏ nhưng mang lại ý nghĩa lớn cho khả năng tiếp cận điều trị.

MIPLYFFA có các bằng sáng chế được niêm yết trong Sách Cam (Orange Book) kéo dài đến năm 2041 và quyền độc quyền thuốc hiếm tại Mỹ đối với bệnh NPC cho đến năm 2031. Zevra cũng đang theo đuổi việc gia hạn thời hạn bằng sáng chế và hiện dự kiến USPTO sẽ ra quyết định vào năm 2027.

Châu Âu và khả năng tiếp cận toàn cầu

Việc CHMP xem xét lại Đơn xin cấp phép lưu hành của arimoclomol đang được tiến hành với một báo cáo viên và đồng báo cáo viên mới. Một nhóm chuyên gia đặc biệt cũng sẽ xem xét các bằng chứng lâm sàng. Ban quản lý tiếp tục kỳ vọng CHMP sẽ đưa ra ý kiến trong quý 4/2026.

Chương trình Mở rộng Khả năng Tiếp cận toàn cầu của Zevra đã ghi nhận 134 bệnh nhân tham gia thông qua các chương trình sử dụng vì mục đích nhân đạo và hoàn trả cho bệnh nhân đích danh. Công ty đã bổ sung một vùng lãnh thổ trong quý và tiếp tục đánh giá các thị trường bổ sung.

Phát triển celiprolol

Thử nghiệm DiSCOVER Giai đoạn 3 của celiprolol đối với hội chứng Ehlers-Danlos thể mạch máu đã thu nhận thêm 6 bệnh nhân trong quý 3, đưa tổng số bệnh nhân tham gia lên 72. Tính đến cuối quý đã có 4 biến cố được xác nhận, trong khi phân tích giữa kỳ của thử nghiệm được kích hoạt khi đạt 28 biến cố.

Sau cuộc họp Loại C thứ hai với FDA, Zevra đang xây dựng kế hoạch phân tích thống kê kết hợp các bộ dữ liệu thực tế hồi cứu. Tùy thuộc vào biên bản cuộc họp chính thức, công ty có kế hoạch tổ chức một cuộc thảo luận tiếp theo với FDA trong nửa đầu năm 2027.

Dự báo của Ban quản lý

• Zevra dự kiến sẽ nộp sNDA cho chỉ định sử dụng MIPLYFFA ở trẻ sơ sinh trong quý 1/2027.
• Ban quản lý dự kiến CHMP sẽ đưa ra ý kiến về arimoclomol trong quý 4/2026.
• Công ty dự kiến USPTO sẽ ra quyết định về khả năng gia hạn thời hạn bằng sáng chế của MIPLYFFA vào năm 2027.
• Zevra có kế hoạch thảo luận lại với FDA về chiến lược nộp hồ sơ cho celiprolol trong nửa đầu năm 2027.
• Dù có mở rộng nhẹ đội ngũ thương mại, ban quản lý không dự kiến chi phí năm 2027 sẽ tăng đáng kể so với năm 2026 và hiện không dự báo có thay đổi lớn đối với chi phí tiếp thị.

Rủi ro và các điểm cần theo dõi

• Góc nhìn đã được sửa đổi về cơ hội thị trường NPC tại Mỹ dựa trên dữ liệu yêu cầu bồi thường bảo hiểm mới xuất hiện và kinh nghiệm thương mại; Zevra chưa đưa ra ước tính chính thức mới về số lượng bệnh nhân đã được chẩn đoán.
• Hồ sơ xin cấp phép của arimoclomol tại Châu Âu vẫn đang được CHMP xem xét lại, và kết quả pháp lý cuối cùng vẫn chưa chắc chắn.
• Thử nghiệm DiSCOVER đã ghi nhận 4 biến cố được xác nhận so với 28 biến cố cần thiết để kích hoạt phân tích giữa kỳ, khiến mốc thời gian phụ thuộc vào sự xuất hiện của biến cố.
• Các khoản hoàn trả EAP toàn cầu có thể biến động giữa các quý tùy thuộc vào thời điểm và nhịp độ đặt hàng.
• Lợi nhuận thuần quý 3 được hưởng lợi từ khoản tín dụng giá trị hợp lý phi tiền mặt 5,5 triệu USD liên quan đến chứng quyền.
• Việc Shionogi thâu tóm IntraBio có thể làm thay đổi cục diện cạnh tranh trong mảng NPC, mặc dù ban quản lý Zevra kỳ vọng việc đầu tư rộng hơn của toàn ngành sẽ hỗ trợ việc nâng cao nhận thức và chẩn đoán bệnh.

Nội dung nổi bật từ phần Hỏi & Đáp với chuyên gia phân tích

Các chuyên gia phân tích tập trung vào quy mô thị trường NPC, tăng trưởng số lượng đăng ký trong tương lai và tác động của những bệnh nhân mới được chẩn đoán. Ban quản lý cho biết các ca chẩn đoán mới duy trì ở mức đáng kể trong khoảng 5 quý liên tiếp và bao gồm cả nhóm người lớn lẫn trẻ em. Một số bệnh nhân được phát hiện thông qua hợp tác xét nghiệm di truyền, trong khi những trường hợp khác có vẻ liên quan đến các nỗ lực nâng cao nhận thức về bệnh rộng rãi hơn.

Về đầu tư thương mại, Zevra cho biết đội ngũ nhân sự thị trường được mở rộng có quy mô phù hợp với điều kiện thị trường hiện tại và có thể mở rộng quy mô khi dữ liệu yêu cầu bồi thường bảo hiểm giúp xác định thêm bệnh nhân. Ban quản lý không dự kiến các thay đổi về cạnh tranh sẽ đòi hỏi tăng mạnh chi phí tiếp thị trong năm 2027.

Về mặt cạnh tranh, Zevra đánh giá việc đầu tư thêm vào mảng NPC sẽ mang lại tác dụng hỗ trợ nhận thức và chẩn đoán bệnh. Công ty nhấn mạnh vị thế của MIPLYFFA là liệu pháp duy nhất có dữ liệu chứng minh việc sử dụng kết hợp với miglustat.

Ban quản lý cũng làm rõ rằng không nên mặc định bệnh nhân NPC người lớn chỉ mắc bệnh ở thể nhẹ. Người lớn có thể bị suy giảm thần kinh và trải qua các giai đoạn tiến triển nhanh hơn, qua đó củng cố tầm quan trọng của việc phát hiện bệnh nhân ở mọi lứa tuổi.

Về quản lý vòng đời sản phẩm trong dài hạn, Zevra cho biết công ty tiếp tục nghiên cứu các chỉ định tiềm năng bổ sung và các cơ hội khác cho dòng sản phẩm MIPLYFFA, nhưng chưa công bố các chương trình cụ thể.

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 afternoon, and thank you for joining Zevra's Third Quarter 2026 Financial Results and Corporate Update Conference Call. [Operator Instructions]

Today's call is being recorded and will be available via the Investor Relations section of the company's website later today. The host for today's call is Nichol Ochsner, Zevra's Vice President of Investor Relations and Corporate Communications.

Nichol Ochsner

Thank you, and welcome to those who are joining us. Today, we will provide an overview of our recent accomplishments, followed by a review of our third quarter 2026 financial results. I encourage you to read our financial results news release, which was distributed this afternoon and is available in the Investors section of our website.

Before we begin the call, please note that certain information shared today will include forward-looking statements. Actual results may differ materially from those stated or implied by any forward-looking statements due to risks and uncertainties associated with Zevra's business.

Forward-looking statements are not promises or guarantees and are inherently subject to risks, uncertainties and other important factors that may lead to actual results differing materially from the projections made and should be evaluated together with the Risk Factors section in our most recent quarterly report on Form 10-Q, our annual report on Form 10-K and our other filings with the SEC.

I'm pleased to welcome Zevra's management team members participating in today's call. Neil McFarlane, Zevra's President and Chief Executive Officer; Joshua Schafer, our Chief Commercial Officer; and Justin Renz, our Chief Financial Officer. Now it's my pleasure to hand the call over to Neil.

Neil McFarlane

Thank you, Nichol, and welcome to everyone joining our quarterly call this afternoon. Zevra is a purpose-driven company, redefining what is possible and bringing life-changing therapies to people living with rare diseases. In rare disease, the path is complex. The needs are urgent and the responsibility is significant. The people and families behind these needs at the heart of what we do every day.

Progress with the U.S. launch of MIPLYFFA the first approved therapy for Niemann-Pick disease Type C, or NPC reflects the disciplined execution of our strategic plan and the determination, expertise and commitment of our people. Together, we are building a trusted company with the capabilities and resolve to make a lasting difference.

I'm pleased to share that total net revenue for the third quarter was $37.8 million, an increase of 45% compared to the third quarter of 2025, and net U.S. product revenue for MIPLYFFA was $34 million. What we're seeing in the market suggests that it may be just the beginning, particularly as we strengthen our understanding of the NPC community in the United States, where an estimated 900 people are living with NPC, of whom 300 to 350 have been diagnosed. These estimates are derived from a study published in 2021 prior to any approved treatment for NPC.

In the third quarter, we received 13 prescription enrollment forms, bringing the cumulative since launch to 197. In rare disease, the number of identified patients often grows over time as awareness increases, more patients receive a diagnosis and new therapies are approved. Josh will share more details on how our initiatives are driving patient identification, diagnosis and treatment later in the call.

Our commitment to realizing MIPLYFFA's full potential and supporting the NPC community is driving our investment to expand patient access. In the second quarter, we published data from a multicenter open-label pediatric substudy evaluating treatment of NPC with arimoclomol in combination with miglustat in infants 6 months to less than 24 months of age. The substudy assessed safety and tolerability for up to 36 months of treatment and found that arimoclomol was well tolerated with no new observed safety signals.

On the heels of this, in the third quarter, we engaged with the FDA in a Type B meeting to explore the potential of expanding the label to include infants less than 24 months of age. Based on the feedback, we expect to file an sNDA in the first quarter of 2027.

Importantly, we believe MIPLYFFA is supported by a strong intellectual property portfolio. MIPLYFFA is covered by patents listed in the Orange Book with expiration dates extending to 2041 and has U.S. orphan drug exclusivity for the treatment of NPC through 2031. These provide distinct layers of protection, regulatory-based exclusivity and listed patent coverage.

As mentioned on previous calls, we are also pursuing a potential patent term extension through the U.S. Patent and Trademark Office. And based on the current processing times, we now anticipate a decision from the USPTO in 2027.

In the EU, the reexamination process for our Marketing Authorisation Application, or MAA for arimoclomol is underway by the Committee for Medicinal Products for Human Use, or CHMP. As part of the reexamination, a new rapporteur and co-rapporteur have been appointed to lead the CHMP's evaluation. Additionally, an ad hoc expert group or AHEG, will meet to review the data and provide their specialized scientific and clinical expertise. This is an important forum for experts in the field to offer perspectives that will contribute to the CHMP's procedural discussions.

We believe the totality of the clinical and real-world evidence supports the clinically meaningful benefits observed in patients treated with arimoclomol. The significant unmet need in Europe motivates us to keep working on behalf of people living with NPC, and we expect the CHMP's opinion in the fourth quarter.

Due to the proximity of the AHEG and CHMP meetings, we will be brief in addressing questions regarding the ongoing procedure. Turning to our Global Expanded Access Program, or EAP. We remain steadfast in our commitment to the NPC community and supporting access to arimoclomol for eligible patients.

Our global EAP has enrolled 134 patients and is comprised of compassionate use and named patient reimbursement programs. As previously discussed, quarterly reimbursement amounts fluctuate based on the timing and cadence of orders. Of note, this past quarter, we expanded the program to include an additional territory and continue to evaluate more opportunities to expand access to patients globally.

Moving to our late-stage pipeline. Celiprolol is being evaluated for the treatment of vascular Ehlers-Danlos syndrome, or vEDS. vEDS is a rare inherited connective tissue disorder caused by a change in the COL3A1 gene, leading to fragile walls in blood vessels and follow organs, which can cause arterial rupture or dissection among other complications. With our Phase 3 DiSCOVER trial, we are confirming the ability of celiprolol, our selective adrenoceptor modulator to reduce the mechanical stress on these tissues, thereby lowering the risk of major event.

We enrolled six new patients in the third quarter for a total of 72. As a reminder, the trial was designed with an interim analysis triggered at 28 events, and there have been four confirmed events through the end of Q3. We've discussed previously that we're focused on advancing the ongoing DiSCOVER trial and are exploring options to accelerate the program by engaging with the FDA.

Following a Type C meeting in the first quarter, we recently held a second Type C meeting to discuss a potential submission strategy with the addition of real-world data sets. While we await formal meeting minutes to confirm next steps, we are developing a statistical analysis plan to include the retrospective data sets with the intent to have a follow-up discussion with the agency in the first half of 2027.

Across our business, we remain focused on advancing key opportunities while maintaining a strong financial position. With more than $270 million in cash, cash equivalents and investments as of the end of Q3, we believe we have the flexibility to pursue near-term opportunities with MIPLYFFA in the U.S. and broadening access for patients in global markets as well as advancing our development pipeline.

I'll now ask Josh to review our commercial momentum. Josh?

Joshua Schafer

Thank you, Neil. We continue to build the foundation for sustained MIPLYFFA growth through patient identification, disease awareness and stakeholder engagement. Our progress is reflected not only in the 197 prescription enrollment forms submitted since launch, but also in our broader activity and growing awareness across the NPC community. In the third quarter, the 13 prescription enrollment forms we received included a balance of adult and pediatric patients as well as a meaningful proportion of newly diagnosed patients.

As a reminder, NPC is a fatal lysosomal storage disorder leading to abnormal lipid accumulation, which over time causes progressive dysfunction of the nerves, brain and other organs. This results in neurodegenerative and visceral symptoms, including negative impacts on speech, cognitive function, fine motor skills and the ability to swallow. The symptoms are variable across patients and many people with NPC experience a prolonged period before diagnosis due to the lack of disease awareness.

Disease progression is irreversible and varies based on the age of symptom onset. MIPLYFFA in combination with miglustat has been established as a foundational treatment for NPC and has demonstrated a halting of disease progression at 12 months and rapid improvement by the first assessment at week 12 based on NPC clinical severity scale. This is further reinforced by the recently published clinical practice guidelines as well as real-world evidence confirming MIPLYFFA's long-term efficacy and safety profile.

We published new data in the third quarter and at the recent SSIEM Annual Symposium, findings were presented from our Early Access programs showing sustained disease stabilization across age groups, including adults. Since launch, we have observed high patient retention and refill rates, consistent with what we observed in our EAP study. This data helps support our market access position and payer coverage, which is currently 69%.

The majority of remaining patients receive coverage through medical exception pathways. This broad coverage is facilitated through our ongoing dialogues and engagement with payers, including regular presentations and conference participation.

Our integrated commercial strategy is focused on identifying new patients, accelerating their diagnosis and expanding access to treatment. Because NPC symptoms vary widely, the disease can be difficult to diagnose. We support overall disease awareness with our campaign called Learn NPC, Read Between the Signs.

We also support early diagnosis through collaborations with genetic testing providers. Additionally, we identify healthcare professionals who may be treating NPC patients through our proprietary analytics model, and we engage these professionals in discussions focused on disease awareness and relevant treatment options. Many of these clinicians may be treating their first and perhaps only NPC patient.

To facilitate education and strengthen the understanding of treatment options, our Expert Connect program provides clinicians less experienced with NPC, direct access to experts in the field. We are encouraged by the impact of these efforts. Our digital presence spans multiple platforms, including the disease awareness website, the MIPLYFFA branded website and our patient and caregiver channels.

In the third quarter, we saw increased traffic across all digital channels with growth in display impressions, search clicks and click-through rates among HCP and patient audiences.

Specifically, our disease awareness website delivered strong results with an increase of 123% in site visits quarter-over-quarter from 25,000 in Q2 to nearly 56,000 in Q3. This was in part driven by the launch of our new NPC Knowledge Hub, which provides educational resources for patients and caregivers.

These results demonstrate some of the impact we are making in expanding disease awareness and supporting Zevra's focus on accelerating time to NPC diagnosis, activities which have directly led to enrollments.

In addition, we expanded our engagement with the NPC patient advocacy community and strengthened our scientific leadership through participation in national and regional congresses. During the quarter, we also held scientific steering committee meetings with NPC experts to gather input on diagnosis, treatment decision-making and long-term disease management. Based on analysis of medical claims data, we believe that the number of individuals with NPC in the U.S. may be higher than previous estimates.

With our flexible commercial model, we are able to scale quickly to respond to new information. As a result, we modestly expanded our customer-facing teams to broaden our reach in community practices outside of the centers of excellence, where we continue to focus. Together, these efforts reflect a comprehensive approach to connecting patients with appropriate treatments and access. We're grateful for our partnerships across the NPC community and proud to support those living with this devastating disease.

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

Justin Renz

Thank you, Josh. Before I begin, I want to encourage everyone to refer to our quarterly report on Form 10-Q, which we intend to file later today for more detailed financial information.

Our revenue growth and the strength of our balance sheet driven by MIPLYFFA, we believe we have a solid financial foundation to support ongoing operations to execute our corporate priorities. In the third quarter of 2026, we generated total net revenue of $37.8 million, which was an increase of $11.7 million or 45% compared to $26.1 million in Q3 2025. This is comprised of $34 million from MIPLYFFA net sales in the U.S., $0.2 million from OLPRUVA and $3.6 million in net reimbursements from the global EAP.

Revenue from MIPLYFFA in the U.S. grew 12.6% compared to the second quarter of this year and 52% compared to the third quarter of last year. During the third quarter of 2026, our operating expenses were $24.1 million, which was an increase of $3.7 million compared to the same quarter a year ago. R&D expense was $4.9 million for Q3 2026, which was an increase of $1.5 million compared to Q3 2025 due primarily to our ongoing investment in arimoclomol and in the celiprolol development program.

SG&A expense was $19.1 million for Q3 2026, which was an increase of $2.2 million compared to Q3 2025, primarily due to commercial and infrastructure-related costs. We recorded a $0.5 million income tax provision in Q3 2026.

Net income for the third quarter of 2026 was $19.2 million or $0.31 per basic and $0.26 per diluted share compared to a net loss of $0.5 million or $0.01 per basic and diluted share for the same quarter a year ago.

In the third quarter, we recorded a favorable mark-to-market fair value noncash credit of $5.5 million related to our outstanding common stock warrants, which is included in the other income section in our consolidated statements of operations.

As of September 30, 2026, total cash, cash equivalents and investments were $270.9 million, which was an increase of approximately $10.7 million compared to June 30, 2026. In summary, we are in a strong financial position with the capacity and flexibility to advance our strategic priorities.

Now I'll turn the call back to Neil for his closing remarks. Neil?

Neil McFarlane

Thanks, Justin. We believe we're well positioned to build on our momentum with MIPLYFFA in the U.S., expanding access to arimoclomol globally and the path to potentially accelerate the development of celiprolol. With a solid corporate foundation underpinning our strategic plan and a shared commitment to putting patients first, we are dedicated to making a meaningful impact for all Zevra stakeholders. Operator, please open the line for questions.

Operator

[Operator Instructions]

We'll take our first question from Kristen Kluska with Cantor.

Phần hỏi đáp

Kristen Kluska

I had two of them. The first question is during your prepared remarks, you noted that you are looking at near-term opportunities for MIPLYFFA in the U.S. given your strong cash balance. I was hoping you could further elaborate on that. Is that driven by the -- your comments about the sNDA, excuse me?

Neil McFarlane

Kristen, I'm not sure if I understood the question, but let me see if I can answer it two ways. One, yes, we are looking at near-term opportunities for MIPLYFFA and the fact that we published our pediatric substudy earlier this year. And after that, we went to the agency to explore a Type B meeting and the opportunity to potentially expand the label to include patients less than 24 months of age.

That feedback really informed our strategy to move forward with the planned sNDA in Q1 of '27. So we do think that, that is a great opportunity, especially now that we've got this intellectual property Orange Book listing, as we mentioned in our last quarter out to 2041.

Kristen Kluska

And then just one question on the U.S. commercial launch. It seems like with the new numbers today that collectively, you have about 2/3 of the U.S. patient population, which is really impressive in slightly over a year. Just looking forward, I'm curious like how we should be setting expectations. Do you still expect there's more to unlock for the already diagnosed patients? Or should we be thinking that more of the near-term new enrollment forms are really going to be driven by any new potential patient adds?

Neil McFarlane

Yes. Thanks, Kristen. So yes, you're right. We have come out of the gate, and in the first seven quarters, full quarters post launch, been able to get to a large portion, you said 2/3 of the diagnosed patient population. The good part about what we see today, and I think what's providing us a lot of confidence that we're somewhere between this 350 and 900 patients in the United States is because we're learning a lot. We're seeing newly diagnosed patients. We're seeing the efforts that we have, and I'll ask Josh to talk a little bit more about some of the specifics, the learning that we're taking around adult patients as well as the pediatric patient split around 50% each that we really have now seen that the market we expect is estimated to be bigger than what we had originally anticipated between that 350 and 900. So we've got a long way to go, but I'll let Josh talk a little bit about some of these learnings and quite frankly, why we did this modest expansion.

Joshua Schafer

Yes. Thanks, Kristen, for the question. As we noted, we have 197 enrollments since launch, and we've been really pleased to see that it's this great balance between pediatric patients and adult patients. In addition, we're seeing a pretty stable number of new patients coming in every quarter. All of that, supplemented then by this analysis that we're doing of the claims data, which we've really been digging into, suggests that there are more patients out there than we had originally estimated.

We've consequently expanded our sales team to be able to find those patients. It's early days, but we do have confidence that those patients are out there and that the market potential may be larger than we had originally estimated.

Operator

We'll move on now to Sumant Kulkarni with Canaccord.

Sumant Kulkarni

I have three. You made some qualitative comments in your prepared remarks, but given the numbers in diagnosed and treated patients and prevalence data were as of 2021, what's your latest understanding on how much higher those numbers could be now that MIPLYFFA has been approved for a little more than two years in the U.S.? And what fraction of the patient is less than 24 months of age?

Neil McFarlane

I'll see if I can start that off and ask Josh to answer some of the specifics. Sumant, we've mentioned this a number of times throughout the last 1.5 years. And a lot of times, what you see when you get a product launched first time in a rare disease, you start to see the kind of the tide rising and you see newly diagnosed patients come in, which we've now seen for about the last five quarters in a row, a meaningful amount of newly diagnosed patients in addition to children and adults. And you start to see a little bit of the disease state awareness and the integrated strategy kind of lifting the awareness of the disease area and you start to get more patients diagnosed.

I think this is all -- and a lot of these learnings are what's really driving our confidence that the market of what we thought were the 300 to 350 diagnosed patients, but a prevalent number around 900, we're now starting to see that kind of tide rising and allow us to see between that 350 and 900. So I'll ask Josh to talk a little bit about what the 2021 publication, which is what we quote versus what some of the learnings we've seen over the last seven quarters or so.

Joshua Schafer

Yes. So the early estimates were based off of a publication in 2021 that was looking retrospectively at claims data and looking at patients with the diagnosis of NPC. And subsequently, now fast forward five years, we're seeing -- well, first of all, the data set is much more comprehensive than it was previously.

But also, as Neil has mentioned, with the launch of MIPLYFFA with greater disease awareness. And as we noted, our disease awareness campaign has nearly doubled in terms of the number of visits to the site, both with clinicians and patients. I think all of that is really helping to build a greater awareness of NPC. We're also seeing diagnosis rates increasing. And so all of that gives us confidence that the number is greater than the original estimate.

Neil McFarlane

I think you also asked a question. Sorry, Sumant, I think you were also asking a question in regards to the pediatric population and what our thoughts were around that of the 900 patients, with that pediatric population. Looks like Josh will talk a little bit about that.

Joshua Schafer

Yes. In terms of total addressable market and an increase with that expanded label potentially of 24 and under, it's not a significant number of additional patients. It's probably in the ballpark of 5%. But it's very meaningful for us because it shows a continued increase in our expansion of the label, our ability to bring access to more and more patients. So we're very excited about that. But in terms of absolute numbers, it does help grow the market, but it's a relatively small patient size.

Sumant Kulkarni

Got it. And then how do you expect competitive dynamics to change in the NPC market now that Shionogi is acquiring IntraBio? And in your opinion, what might hold the key to the EMA potentially reversing its earlier decision on arimoclomol as part of this reexamination process?

Neil McFarlane

Well, maybe I'll start with the last question, then I'll ask Josh to talk a little bit about the addition of additional companies coming into the marketplace, which we see is great. I'm going to be measured in my comments, Sumant, in regards to the activities ongoing with the AHEG that I discussed as well as the CHMP opinion. Much more to say that we continue to be confident in the totality of our data package. The fact that there's a high level of unmet need and the fact that we now have these experts that are coming to be able to actually opine on the clinical meaningfulness and statistical relevance of our data. So with that, I'll ask you to stay tuned, and we do still expect our opinion in Q4. Josh, do you want to talk a little bit about the additional companies coming into the NPC space?

Joshua Schafer

Yes. So we view this as a really good thing for a number of reasons. One, I think it validates what we're saying that the market opportunity is larger and really validates that there is room to continue to grow within the NPC marketplace. We have a great deal of respect for Shionogi, and we think that this is great for patients in general, and this is going to help to increase awareness and diagnosis. And importantly, this helps reinforce MIPLYFFA's strong foundational position given that we are the only drug that has data showing that we work in combination with miglustat. And we know that combination therapies are the fastest-growing part of the market and are supported by the consensus guidelines. So overall, I see this as a really positive thing for the market.

Neil McFarlane

Maybe I'll add a little bit to that because we do think that part of the overall expansion of the market and the increase in the newly diagnosed patient population is because of the fact that we have a number of companies that are investing into Disease State Awareness. And we've seen that in the year-over-year numbers just out of the claims data alone and the number of patients in 2026 that have been newly diagnosed versus those patients in previous years.

So we're really fortunate to be able to have the investment that is rising the NPC marketplace. And again, as Josh mentioned, the estimated number that we think the market of those diagnosed patients, we believe now is higher than what we've seen from the previous numbers. We are going to go test it and get it out there. But now with the other folks that are coming in the marketplace, we think we can address it quicker with the strength of our data.

Operator

We'll move on now to Lachlan Hanbury-Brown with William Blair.

Truman Dunkley

This is Truman on for Lachlan. Congrats on the quarter. And you spoke on the SD&A for my that you'll be filing. But do you foresee any additional data will be needed for that? Or will that be ready to go?

Neil McFarlane

So the feedback that we received post our pediatric substudy is informing our submission strategy. I won't get into the specific data, but we feel confident that we can file an sNDA in Q1 of 2027.

Operator

We'll move on now to Brandon Folkes with H.C. Wainwright.

Brandon Folkes

Congrats on another good quarter. A few for me, if I may. I know this one is probably tricky answer, but understanding you don't contract and a lot goes through medical exception. Can you just talk about the trend you're seeing on net realized price per patient on therapy? Has it increased in the year? Has it been relatively stable? And does that change if a patient is on multimodal therapy?

Neil McFarlane

I might start that one off and just let you know that we continue to have very strong reimbursement rates through both our covered lives as well as with the medical exception and the strength of our data driving a lot of those decisions. But I'll ask Josh to talk about some of the specifics that you asked about.

Joshua Schafer

Yes. So we continue to see really strong reimbursement rates. We continue to see really strong refill rates, very similar to what we saw in the expanded access programs. So I think that's largely driven based on the strength of our clinical data and the work that our team is doing to get in front of payers. We have data showing that we work in combination with miglustat. So certainly, that helps to support that reimbursement as well as the long-term efficacy data that we've been able to present publicly. So all that really lends itself to continued high reimbursement and retention and refill rates. And yes, we don't see any change in that.

Neil McFarlane

Yes. And then, just to be specific, we see the patient enrollment forms that come in from MIPLYFFA, and we see what enrollments are actually and the high level of those enrollments that go on to commercial product. What we cannot answer for you is what happens with anybody else.

Brandon Folkes

And maybe just following on from an earlier question. Just given the changing dynamic in the market with Shionogi and potential additional competitors, does that change how you think about sales and marketing spend in 2027? I know you talked about sort of the collective investments being a rising tide. But just how do you view your spend in 2027?

Neil McFarlane

I'll maybe kick that over to Justin in a moment. But for us, we've been very disciplined in regards to our execution, and I consider that we'll continue to do that. But we may not provide the full guidance for 2027, Brandon, on the SG&A. But Justin, do you want to talk a little bit about that?

Justin Renz

Thank you. With the modest expansion next year, we don't foresee any meaningful increases from this year. So again, as Neil mentioned, we're in a solid financial position. We have the capacity and flexibility. But I would not look to make major changes to our marketing spend going forward.

Operator

We'll move on now to Jason Butler with Citizens.

Jason Butler

Congrats on the quarter. Can you speak to the characteristics of the newly diagnosed patients that you're seeing enrollments for in terms of age and disease severity? And is there any evidence that you're actually seeing an acceleration of diagnosis in the population? And specifically, are any of these patients coming directly from your efforts to expand genetic testing?

Neil McFarlane

That's great. These sound like a lot of really good specific questions exactly for Josh. But overall, I think we're quite -- back to the learning, we're learning every day about what this market looks like. But really, the newly diagnosed patients is what's providing us a lot of confidence that this market is bigger than the 350 and somewhere between 350 and 900.

Joshua Schafer

Yes. And I wish I could give you some sort of generalized characterization of these newly diagnosed patients, but they're really across the entire marketplace. So we see some older patients. We see some younger patients. Many of them have come through either genetic testing, through our collaboration with some of those genetic testing providers. I think some of it is just coming from our efforts to really increase the disease awareness. So there is no real sort of overarching characteristic that I can provide to you.

Neil McFarlane

I think a lot of this, Jason, like anything else, there's no silver bullet. This is an integrated strategy that we have to be able to unlock earlier diagnosis through disease state awareness, being able to provide treatment options for physicians and make that education really readily available. And we see the opportunity to educate more people now because we've -- hence, the reason we did this modest expansion because we see a larger number of potential patients out there in terms of what we've uncovered so far.

And then lastly, we want to make sure that we continue to provide access. Those three things we've invested in, and I think the team is doing an outstanding job in that. But it's hard when you have such small numbers to be able to give you characteristics that might be meaningful, except what I've said on previous calls. We always thought that this disease was a little bit more of a childhood level disease. And the fact that we still see a fairly consistent 50% kids, 50% adults and newly diagnosed in the same kind of ratios. Those are learnings for us that we're now piggybacking on to be able to go out and make sure we can do more disease state awareness, get patients diagnosed earlier and then provide access to them.

Operator

[Operator Instructions] We'll move next to Eddie Hickman with Guggenheim Securities.

Eddie Hickman

You noted about half of the enrollments are adults who are milder. For the 13 forms this quarter, how did the adult share in the community practice share compare with the first half? And because the constraints seems to be like getting new clinicians rather than finding patients, do you think you would need a bigger field team or a medical affairs spend to reach those milder adult patients? And then on the sNDA, how many patients do you think are under 24 months that you could add to the TAM?

Neil McFarlane

All right. I'm going to take the first question and the last question. I'll ask Josh to opine in the middle. Eddie, thank you for your question. And I want to make sure that I make something clear. Just because you're an adult patient doesn't mean that you have milder disease. The trajectory and the natural history shows that all patients will continue to progress but it is not unusual for an adult patient who has progressed very slowly to then actually have a change that then will allow them to start progressing at a much more rapid pace and be more impacted.

So I don't want this to -- and I hope that this need to come across that an adult patient means that they're milder. That's not always the case. And I think that we all need to be cognizant of the fact that these patients that are adults also in order to be treated with MIPLYFFA, you actually need neurological impact, and these patients are seeing neurological impact. So these adults that we're seeing that are about 50% of our current prescription enrollment forms, we're learning from. Some of them misdiagnosed with other diseases. We've had somebody recently who was misdiagnosed with multiple sclerosis that then finally ended up getting an NPC diagnosis many, many years later, genetically diagnosed. And that patient was progressing. So we see a lot of these -- this is just one anecdote of many because as you know, one patient, you know one patient. So that was one.

Adults can have very aggressive disease as well as children. So clarify that. The next thing I wanted to talk to you about was the sNDA. Our sNDA is an opportunity to be able to expand the label, but these patients, we think, are in the 900-patient prevalent numbers today. And as we continue to see newly diagnosed patients, we wanted to be able to provide both the physicians with the understanding of how to utilize the products, as well as the payer community to be able to know how to be able to make it easier for them to gain access to the product. So it's not increasing the TAM per se or the prevalence per se. It's part of this increased confidence that we're seeing of the number of diagnosed patients that we believe is estimated to be larger than our original 300 to 350.

Joshua Schafer

Yes. And then in terms of the size of the team to be able to reach these new patients. This is based on our analysis and increasing understanding of the claims data. And what I'm really pleased with was that we've been able to identify that there are more patients out there with NPC diagnosis. And very quickly, we were able to expand the team modestly, but we were able to expand the team very, very quickly in order to respond to that new understanding of the marketplace. I think this is a model that we're going to adopt that one that allows us to scale up or down as we learn more about the market, as we identify these patients. So I think we're rightsized for where we are today, but I'm really pleased with our ability to pivot quickly if need be based on the data.

Operator

We'll move on now to Kambiz Yazdi with U.S. Bancorp BTIG.

Kambiz Yazdi

Beyond the intellectual property strengthening and potential label expansion in your 24 months, can you give us any color on what the next layer of life cycle management looks like for the MIPLYFFA franchise? Are there any formulation, combination or next-generation compound strategies in the pipeline that we should be thinking about over the longer-term horizon?

Neil McFarlane

Yes. Thanks, Kambiz, and I appreciate the question. As I mentioned, I believe, on one of the earlier calls this year, we looked at our Orange Book listing going out to 2041, along with strengthening the moat of our patent portfolio with the patent term extension and the continued investments that we're looking at. We also see the opportunity to be able to explore what additional indications or other areas we may want to look at. It's really too early for us to come back to you and tell you anything at this point. But I do want to say that we are always exploring opportunities to maximize the franchise and even provide optionality for patients with NPC.

Operator

And it appears that we have no further questions at this time. I'm happy to return the call back over to our hosts for any concluding remarks.

Neil McFarlane

Thank you for joining our call today, and we look forward to keeping you appraised of our future progress.

Operator

Thank you. This brings us to the end of today's meeting. We appreciate your time and participation. You may now disconnect.