Conferencia de resultados del T3 de 2026 de Krystal Biotech (KRYS): Los ingresos de VYJUVEK aumentan un 30%

Puntos clave

• Krystal Biotech informó de unos ingresos netos por VYJUVEK en el tercer trimestre de 2026 de 127,3 millones de dólares, un 30% más interanual, incluidos 95,3 millones de dólares de Estados Unidos y 32,0 millones de dólares a nivel internacional.
• El beneficio neto fue de 63,1 millones de dólares, con un BPA diluido de 2,05 dólares. El margen bruto disminuyó al 94% desde el 96%, en parte debido a que Europa y Japón aún no se han beneficiado de las optimizaciones en el proceso de fabricación aprobadas en EE. UU.
• La empresa cerró septiembre con más de 1.200 millones de dólares en efectivo e inversiones. Su consejo de administración autorizó hasta 400 millones de dólares en recompra de acciones, que se prevé ejecutar a lo largo de dos años.
• El impulso comercial en EE. UU. incluyó más de 760 aprobaciones de reembolso y más de 80 nuevos médicos prescriptores durante el trimestre. Aproximadamente el 80% de los pacientes se autoadministra actualmente VYJUVEK.
• La dirección revisó a la baja la previsión de gastos no GAAP en I+D y SG&A para todo el año a 155-165 millones de dólares, desde los 175-195 millones de dólares.
• Se modificaron los plazos de varios ensayos clínicos. Los resultados de KB803 para DEB ocular se esperan poco después de la última visita del paciente en febrero de 2027, mientras que la inscripción y los resultados principales de KB801 para la queratitis neurotrófica se proyectan ahora para 2027.

Resultados financieros principales

Krystal señaló que los ingresos acumulados de VYJUVEK desde su lanzamiento han alcanzado aproximadamente 1.100 millones de dólares.

Rendimiento comercial y operativo

Crecimiento de VYJUVEK en EE. UU.

Krystal obtuvo más de 760 aprobaciones de reembolso en todo el país y sumó más de 80 prescriptores durante el tercer trimestre. La dirección ve nuevas oportunidades entre los pacientes diagnosticados que aún no han iniciado el tratamiento y aquellos no diagnosticados o con un diagnóstico erróneo.

El tratamiento domiciliario también se está afianzando: aproximadamente el 80% de los pacientes se autoadministra actualmente VYJUVEK. La empresa está centrada en ampliar el inicio del tratamiento al tiempo que apoya su uso continuado a medida que cambian las necesidades de cura de heridas de los pacientes.

Expansión internacional

Los ingresos internacionales de VYJUVEK alcanzaron los 32 millones de dólares. Alemania cuenta ahora con más de 20 centros que han iniciado a pacientes, aproximadamente un 15% más que en el trimestre anterior. Las negociaciones de precios en Alemania han concluido.

Francia completó su primera administración domiciliaria mediante la vía de retrocesión. Las negociaciones nacionales sobre la fijación de precios siguen su curso, y la dirección destaca que el proceso avanza dentro de los plazos habituales.

En Japón, Krystal continúa añadiendo pacientes y médicos prescriptores. La dirección prevé que la flexibilización de las restricciones de prescripción durante el primer año favorezca una adopción más rápida.

Las negociaciones de precio y reembolso en Italia han concluido, y la compañía prevé lanzar VYJUVEK en dicho país a principios de 2027. Se espera una decisión de fijación de precios en España antes de finales de 2026, mientras que algunos pacientes ya reciben tratamiento mediante acuerdos de financiación regional de carácter excepcional.

Avances en la cartera de desarrollo

Los resultados iniciales de KB113 en tres adultos con síndrome de Gorlin mostraron respuesta en carcinomas basocelulares inyectados y no inyectados. Más de la mitad de las lesiones evaluadas remitieron en más de un 50%, mientras que aproximadamente el 10% de los tumores se redujo al menos un 90%. La evaluación histológica confirmó la remisión completa en un tumor inyectado analizado mediante biopsia en cada paciente. Krystal está en conversaciones con la FDA sobre posibles vías de desarrollo.

En cuanto a KB111 para la enfermedad de Hailey-Hailey, cinco pacientes han completado la inscripción en el estudio de dosis repetidas HALITE-1. La dirección prevé publicar una actualización de los datos antes de que finalice 2026, a la que seguirán conversaciones con la FDA.

En DEB ocular, Krystal inscribió a tres pacientes adicionales para KB803, incluidos los reemplazos de dos retiradas no relacionadas con la seguridad y un niño de un año. Se espera la última visita del paciente en febrero de 2027 y los resultados principales poco después.

La administración de dosis repetidas de KB407 está en marcha en tres pacientes con fibrosis quística, y se prevé la inscripción y dosificación de otros tres pacientes antes de fin de año. Se esperan resultados preliminares, incluidos los datos de FEV1, en el primer semestre de 2027. La compañía también mantiene contactos con la FDA sobre el posible diseño de un estudio de registro.

En el caso del KB707 inhalado para el cáncer de pulmón no microcítico avanzado, Krystal completó la optimización de la dosis en 10 pacientes. Se prevé inscribir y tratar a 70 pacientes con el régimen de quimioterapia seleccionado para mediados de 2027.

Previsiones de la dirección

• La previsión de gastos no GAAP en I+D y SG&A para todo el año se revisó a 155-165 millones de dólares, frente a la previsión anterior de 175-195 millones de dólares.
• Se prevé lanzar VYJUVEK en Italia a principios de 2027, sujeto a la ejecución de los planes de acceso al mercado.
• Se espera una decisión sobre el precio en España antes de finales de 2026.
• Se esperan datos sobre KB111 para la enfermedad de Hailey-Hailey antes de finales de 2026.
• Se aguardan los resultados principales de KB803 para DEB ocular poco después de la última visita del paciente en febrero de 2027.
• Se espera la finalización de la inscripción y los resultados principales de KB801 para la queratitis neurotrófica en 2027.
• Se esperan los resultados preliminares de KB407 para la fibrosis quística en la primera mitad de 2027.
• La empresa prevé ejecutar su programa autorizado de recompra de acciones por 400 millones de dólares a lo largo de dos años, mientras continúa financiando la expansión comercial y el desarrollo clínico.

Riesgos y factores a seguir

La publicación de varios datos clínicos está demorándose más de lo previsto inicialmente. La inscripción para KB801 se ha visto afectada por unos criterios de elegibilidad muy estrictos, fallos de cribado, retrasos en la contratación en centros académicos y la competencia de una terapia aprobada y disponible en EE. UU.

El calendario de KB803 se modificó después de que dos pacientes se retiraran al no desear comprometerse con una administración semanal. La dirección aclaró que las retiradas no estuvieron relacionadas con la seguridad, pero los pacientes permanecerán en la población por intención de tratar.

La inscripción para KB407 se retrasó tras la enfermedad de algunos posibles pacientes. La dirección hizo hincapié en que la necesidad de una inscripción adicional no respondió a un problema de dosis y afirmó que los pacientes actuales están tolerando la administración repetida.

El reembolso internacional aún no está completado. Francia continúa negociando el precio y otros países europeos requieren aprobaciones de acceso al mercado. El margen bruto también podría verse afectado mientras Europa y Japón operen sin las optimizaciones del proceso de fabricación ya aprobadas en EE. UU.

Puntos clave del turno de preguntas de los analistas

• Asignación de capital: La dirección señaló que la autorización de recompra por 400 millones de dólares refleja la confianza en que los ingresos por VYJUVEK y la posición de efectivo de la compañía permiten respaldar tanto el desarrollo del pipeline como la retribución al accionista. Krystal mantiene su disposición a aumentar la inversión allí donde un gasto adicional pueda acelerar la inscripción o la activación de centros.
• Inscripción para KB801: La empresa planea añadir entre 10 y 15 centros más en EE. UU. y activar centros en Francia, España, el Reino Unido y otros mercados europeos. Krystal mantiene criterios estrictos sobre la persistencia de las lesiones con el fin de reducir el riesgo de inscribir a pacientes cuyas lesiones corneales mejoren de forma espontánea.
• Preparación de la solicitud de KB803: La dirección indicó que los requisitos de CMC están cumplidos y que la compañía prevé solicitar la autorización de comercialización biológica (BLA) poco después del cierre y análisis de la base de datos, suponiendo que los resultados sean positivos.
• Desarrollo en el síndrome de Gorlin: Sobre la base de la respuesta observada en el estudio piloto de tres pacientes, la dirección considera que un eventual ensayo pivotal podría contar con menos de 100 pacientes, a la espera del acuerdo con la FDA.
• Orden de publicación de resultados: La dirección confirmó que se prevén los resultados para DEB ocular antes de la presentación de los datos del KB801 en queratitis neurotrófica.

Transcripción completa de la conferencia de resultados

Transcripción completa de la conferencia de resultados

Comentarios de la dirección

Operator

Good morning, and thank you for standing by, and welcome to Krystal Biotech's Third Quarter 2026 Earnings Conference Call. [Operator Instructions] As a reminder, today's conference is being recorded. I would now like to hand the conference over to your host, Stephane Paquette, Chief -- sorry, Senior Vice President of Corporate Development. Please begin.

Stephane Paquette

Good morning, and thank you all for joining today's call. Earlier today, we released our financial results for the third quarter of 2026. The press release is available on our website at www.krystalbio.com. We also filed our earnings 8-K and 10-Q with the SEC earlier today. Joining me today will be Krish Krishnan, Chairman and Chief Executive Officer; Suma Krishnan, President of Research and Development; Laurent Goux, Executive Vice President and General Manager for Europe; Christine Wilson, Senior Vice President and Head of U.S. Commercial; and Kate Romano, Chief Accounting Officer. This conference call will and our responses to questions may contain forward-looking statements.

You are cautioned not to rely on these forward-looking statements, which are based on current expectations using the information available as of the date of this call and are subject to certain risks and uncertainties that may cause the company's actual results to differ materially from those projected. A description of these risks, uncertainties and other factors can be found in our SEC filings. With that, I'll turn the call over to Krish.

Krish Krishnan

Good morning, and thank you for joining us. Q3 was another strong Q for Krystal. We delivered $127.3 million in VYJUVEK revenue, an increase of 30% from the same period last year and reported diluted earnings of $2.05 a share and ended September with $1.2 billion in cash and investments. Those results, they reflect a business that's growing, that's profitable and generating the resources to advance our pipeline. They also reflect the work our teams have done to build Krystal into a global company with commercial operations, manufacturing capabilities, clinical development programs that we own and control. And to date, we have generated approximately $1.1 billion in VYJUVEK revenue since launch, and that is an important milestone for a medicine developed here at Krystal.

Behind that number are so many patients receiving treatment, physicians incorporating a new paradigm of genetic medicines into their practices and families getting access to a medicine that addresses the underlying cause of DEB. In the U.S., demand continues to grow as does the prescribing physician base. And internationally, we continue to translate physician interest into patient starts across Germany, France and Japan. The pricing secured by VYJUVEK in Japan and Germany and Italy to date reflects recognition of the meaningful benefits that VYJUVEK provides patients and families.

I'm proud of our team's thoughtful work to demonstrate that value and establish a strong foundation for patient access internationally. We'll continue to build on this progress in ensuing pricing discussions across additional markets, and Laurent will discuss those markets in more detail. We also made meaningful progress in development. We were really encouraged by the recent Gorlin Syndrome clinical results that demonstrated progression in both injected and non-injected lesions together with the histologically confirmed clearance of an injected lesion in each patient. So that gives us a very strong foundation to have conversations with the FDA on next steps. I want to address the updated timing of some of our clinical readouts. And while we had hoped to share these readouts sooner, I want to be clear that the programs continue to progress well, and we look forward to sharing results in the upcoming months. Suma will elaborate more in a few minutes.

Alongside these developments, our Board authorized a share repurchase program of up to $400 million that we expect to deploy over the next 2 years. We have reached a position where our financial resources allow us to support global commercial expansion, pipeline and clinical development and have the flexibility to return capital to shareholders. And the authorization reflects our confidence in Krystal's long-term prospects and our responsibility to allocate this capital thoughtfully. So taken all together, this Q demonstrated the strength of the business we've built. It demonstrated continued commercial growth, substantial profitability and progress across the pipeline. For that, I'd like to thank our employees, our investigators and the patients and families participating in our studies for helping make that progress possible. And with that, I'll turn it over to our team to walk through commercial, clinical and financial updates.

Laurent Goux

Thank you, Krish. VYJUVEK international revenue reached $32 million in the third quarter. We continue to translate physician interest into patient starts while strengthening the growth foundation through reimbursement progress and greater access to treatment at home. In Germany, pricing negotiations have concluded, providing greater visibility as we expand our reach across the country. More than 20 centers have now initiated patients on VYJUVEK, up circa 15% from last quarter. Doing so, we continue to broaden the treatment network to reach patients who are not routinely seen at established expert centers.

In France, we achieved an important milestone with the first home administration through the retrocession pathway. This creates an opportunity to reduce the practical burden of treatment on patients and families and support treatment continuity. We are now working to expand this approach to additional eligible patients and centers while national pricing discussions continue. And in Japan, our team is making steady progress, bringing new patients to treatment and expanding our prescriber network. We are also advancing support initiatives to help the EB community maximize the potential benefits of VYJUVEK therapy and navigate the complexity of VYJUVEK's GMO status.

Looking ahead, pricing and reimbursement negotiations in Italy have also concluded, and we now expect to launch VYJUVEK in early 2027. In Spain, pricing discussions are progressing with a decision expected before the end of the year. Some patients already have access through exceptional regional funding arrangements, providing a foundation for a potential launch next year. We are also advancing regulatory and market access activities in additional countries to expand VYJUVEK's reach over time. Taken together, these developments give us confidence in the international opportunity.

Health systems are recognizing VYJUVEK's value, physicians are gaining treatment experience and barriers to administration are beginning to come down. Our focus is on turning that progress into more patients starting and continuing treatment. With that, I'll hand the call over to Christine.

Christine Wilson

Thank you, Laurent. U.S. net revenue for the third quarter was $95.3 million, reflecting continued demand for VYJUVEK and our efforts to reach more patients. We have now secured more than 760 reimbursement approvals nationwide. During the quarter, we added over 80 new prescribers. This expanding physician base supports continued adoption, and we believe substantial opportunity remains among diagnosed patients who have not yet started treatment as well as those who remain undiagnosed or misdiagnosed. We are also seeing the benefits of greater treatment flexibility. Approximately 80% of patients now self-administer VYJUVEK, helping patients and caregivers incorporate treatment into their wound care routines at home.

As patients gain experience, our focus extends beyond treatment initiation to supporting continued use as their wound care needs evolve. Our VYJUVEK voices and VYJUVEK connection programs provide education, practical support and opportunities for patients and caregivers to learn from one another. Engagement with physicians, advocacy organizations and the broader DEB community also helps us understand barriers to treatment and improve the support we provide. Looking ahead, our priorities remain clear: reach more patients, expand the prescribing network and support long-term treatment. With continued demand, a growing physician base and greater flexibility in administration, we believe VYJUVEK is well positioned for sustained U.S. growth.

We are proud of the progress we have made and excited about the opportunities ahead as we continue working to establish VYJUVEK as the long-term standard of care for patients living with DEB. With that, I'll turn the call over to Suma.

Suma Krishnan

Thank you, Christine, and good morning, everyone. We have encouraging progress to share this morning, including our initial Gorlin syndrome results, the upcoming Hailey-Hailey readout and the ongoing dosing in cystic fibrosis. But before I get into the update, I want to acknowledge that some clinical results are going to take a bit longer than we initially expected. We recognize the importance of these milestones. Our teams are working diligently to advance each study, and we look forward to sharing the data as they become available. Importantly, the studies themselves are progressing well, and we remain encouraged by their eventual outcome. I'll walk through our rationale and expectations from here shortly.

With that, I would like to start today's update with the latest addition to our pipeline, KB-113 for Gorlin syndrome. Gorlin syndrome is a rare genetic disease estimated to affect more than 10,000 Americans. Patients can develop hundreds of basal cell carcinomas or BCCs over their lifetime, requiring repeated surgeries that can lead to tissue loss, disfigurement and significant psychological burden. With no therapy specifically approved for Gorlin syndrome, there is a substantial need for a safe, effective treatment to reduce BCC burden. KB707 is our HSV1-based immunotherapy designed to drive sustained localized expression of human IL-2 and IL-12 in the tumor microenvironment and promote systemic immune-mediated tumor clearance.

We recently completed a pilot study evaluating a low dose of intratumoral KD707 which we now refer to as KB113 in 3 adult patients with Gorlin syndrome. Each received 6 doses over 14 weeks. Investigators assess regression in both injected and non-injected landmark BCCs throughout treatment and 30 days after the final dose using a prespecified 5-point scale. Profound treatment benefits were observed following KB113 treatment, including broad antitumor responses in all 3 Gorlin syndrome patients. As summarized in the table on the left, over 80% of the assessed patients regressed over the course of the study and more than half of all lesions regressed by over 50%. Critically, substantial regression was observed in lesions that were never directly injected, providing preliminary evidence of systemic immune activation and an abscopal effect.

Investigators also reported that a meaningful proportion, roughly 10% of all assessed tumors had shrunk by at least 90%, indicating potential for tumor eradication with KB113 treatment. To further assess depth of response, 1 injected tumor from each patient was biopsied for histological assessment. In all 3 cases, complete clearance was confirmed with no evidence of residual tumor tissue in the biopsy. Images shown on the right highlight the rapid improvement achieved in Gorlin patients with KB113 and add to the body of evidence suggesting significant potential for KB113 to dramatically reduce PCC burden in patients with Gorlin syndrome. With the addition of KB113 to our pipeline, we are now building out a robust development effort in rare dermatology diseases, an area we know well and where we can move fast.

In the case of KB113, we have already started engaging with FDA regarding potential clinical development pathways in Gorlin syndrome. We will share an update on our plans once aligned with the FDA. We are also making good progress on our KB111 program for HHD. We have completed enrollment of 5 patients in our repeat dose study for HALITE-1 and expect to share a data update before year-end. We then expect to meet with the FDA to discuss next steps. Turning to our ophthalmology pipeline. I want to start by saying that both NK program and the ophthalmic VJUVEK programs are progressing well. In the case of KB803, we enrolled 3 additional patients in our registrational IOLITE study. Two of these additional patients have been enrolled to replace 2 patients who withdrew for reasons unrelated to safety. The third patient, a child 1 year of age, was enrolled to generate evidence to support potential treatment of corneal abrations in DEB patients from birth.

We now expect that the last patient last visit in February 2027 with top line results to be reported shortly thereafter. In KB801 for NK patients, enrollment has progressed more slowly than we initially anticipated, reflecting the study's narrowly defined eligibility criteria and the challenges of enrolling patients in a placebo-controlled trial in an indication with an available approved therapy. We are also working diligently to expand our clinical trial infrastructure. We have activated 22 sites in the United States, and we are close to activating sites in France, Spain and United Kingdom. With continued investment and expansion of our trial network, we now expect to complete the enrollment and report top line results in 2027.

In the lung, we are steadily advancing our program in CF and AATD. Starting with KB407 in CF, we are currently dosing 3 patients in our repeat dose study and expect to enroll and dose 3 additional patients before the year-end. Based on this enrollment timeline, we now anticipate interim study results in the first half of 2027. We are progressing in parallel our discussion with the FDA regarding a potential registrational study design, having recently been selected for FDA's Complex Innovative Design Meeting Program. This is enabling intensive FDA engagement and has us on track for a 27 registrational study start. We also continue to enroll in our KB408 repeat dose study and are on track to report interim data next year. The successful gene delivery already confirmed by biopsies for both KB407 and KB408, we believe we are uniquely positioned to fill urgent treatment gaps for CF and AATD patients. We look forward to sharing more in 2027.

Finally, we are making great progress on our inhaled KB707 program, enrolling patients with advanced NSCLC in the global KYANITE-1 expansion cohort evaluating inhaled KB707 plus chemotherapy. We have completed dose optimization in 10 patients to inform chemotherapy dose selection and expect to enroll and dose 70 patients at the selected regimen by mid-2027. Results from this cohort will help us finalize our plans for subsequent registrational study of KB707. Each disease that we are targeting with our pipeline is an urgent unmet need. We recognize the importance of progressing deliberately and quickly to fill these treatment gaps. With our expanded R&D infrastructure, I'm confident that we can deliver on our ambitions and dramatically grow our impact. With that, I'll hand the call over to Kate.

Kathryn Romano

Thank you, Suma. I'll now provide a few additional highlights from our third quarter financial results as reported in our press release and 10-Q filing earlier today. Net revenue from global sales of VYJUVEK was $127.3 million for the quarter, driven by the continued growth from our Europe and Japan launches and representing a 30% increase over the prior year's third quarter of $97.8 million, which included only a partial quarter of sales from Europe and excludes sales from Japan. Cost of goods sold for the quarter was $7.6 million compared to $4.3 million in the prior year's third quarter. Gross margin for the quarter was 94% as compared to 96% in the third quarter of 2025. The 2% decrease in gross margin was due in part to the increase in sales of products in Europe and Japan, which do not yet benefit from manufacturing process optimizations approved in the U.S. as well as the sale of lower cost R&D batches in the prior year quarter following FDA approval of the optimized process.

R&D expenses for the quarter were $14.9 million compared to the prior year of $14.6 million. G&A expenses were $33.7 million compared to $37.6 million in the prior year's third quarter. This $3.8 million decrease was primarily due to the receipt of insurance proceeds as reimbursement of legal costs previously incurred along with a reduction in period-over-period legal costs, professional service fees and marketing costs, offset by increased payroll costs. Operating expenses for the quarter included noncash stock-based compensation of $13.9 million compared to $13.2 million in the third quarter of last year. Net income for the quarter was $63.1 million, which represented $2.13 per basic and $2.05 per diluted share.

In the third quarter of 2025, net income was $79.4 million and EPS was $2.74 per basic and $2.66 per diluted share. In the third quarter of last year, we had released a majority of the valuation allowance that was historically recorded against our deferred tax assets, and we also benefited from the reversal of the Section 174 R&D capitalization requirement under the One Big Beautiful Bill legislation. Both of these items were nonrecurring. Looking ahead to the remainder of the year, we are also revising our full year non-GAAP R&D and SG&A guidance to $155 million to $165 million from our prior guidance of $175 million to $195 -- and finally, we continue to build on our solid financial position, now exceeding $1.2 billion in overall cash and investments. With this foundation, we remain focused on disciplined capital allocation to support our pipeline and global commercial strategy while maintaining the ability to return capital to shareholders through our newly authorized share repurchase program. And now I'll turn the call back over to Krish.

Krish Krishnan

What excites me most is the opportunity we have to build a company whose impact extends well beyond its first medicine. And with each program, we can apply what we've learned, deepen our capabilities and pursue additional diseases where patients need better options. With pricing negotiations behind us in Japan, Germany and Italy, we can now more clearly see meaningful room for VYJUVEK growth internationally. We have a rich pipeline with several important readouts in the upcoming months. And as I said before, the initial Gorlin findings are very encouraging, and we look forward to the upcoming Hailey-Hailey results along with CF and NK. Each program will generate evidence that helps us determine how best to bring additional medicine to patients.

Financially, we've put ourselves in a good place. The growing commercial business allows us to invest not just in global expansion, but also in development and buy back shares opportunistically. And so we'll allocate capital with a clear focus on building lasting value per share. I'm confident in Krystal's future. We have a strong foundation. We have the resources to invest and a team that's super committed to turning scientific potential into meaningful medicines. -- and our responsibility is to translate all those strengths into more patients treated. So thank you to the employees, the patients, investigators and our shareholders for your continued support, and I'll open the call up for questions.

Operator

Thank you. [Operator Instructions] Our first question today is coming from Lachlan Hanbury-Brown with William Blair.

Preguntas y respuestas

Lachlan Hanbury-Brown

On all the progress. I guess first question maybe on KB407. You said you're enrolling an additional 3 patients. I was wondering on the rationale for that. I mean, are you going to test a different dose? Is there something that you've seen in the first few patients that makes you a larger sample here? And then on the second question, wondering about Japan. Krish, you've talked in the past about how the sort of first year restrictions can limit adoption. So as we kind of approach that 1-year mark, wondering how we should think about the sort of acceleration of growth in Japan moving forward?

Krish Krishnan

Thanks Lachan for the question, Suma?

Suma Krishnan

Yes, I can address the KB803, sorry, I can address the KB407 question, sorry, KB407. Yes, I mean, we have 3 patients right now. They're ongoing on the repeat dosing. It has nothing to do with the dose. I mean we are very excited. I mean these patients are tolerating repeat dose. That was one of the biggest questions we had to address the -- with regarding to safety. And obviously, we're collecting FEV1 data.

The enrollment for these additional 3 patients are going to happen this year. Unfortunately, it was a little slow because some of the patients that were supposed to get on the drug fell sick. And remember, these are patients that are novel and with no other options. So it is their life events that actually pushed out. But I think we feel confident all the patients will get on the study, and we expect to have this data by middle of next year.

Krish Krishnan

On the Japan question, I mean that's valid like that in the next, I think, upcoming -- I don't remember the timing, which should be November, December, the prescription kind of lifts off and it goes from maybe twice a month to once a month, and we definitely believe that, that would incentivize a faster adoption, especially in a country where people commute long distances to get to the physician office.

Operator

Our next question is coming from Ritu Baral with TD Cowen.

Ritu Baral

I wanted to keep my 2 questions to KB801 in NK. Can you talk a little bit about how much you're expanding sites beyond the 22 U.S. sites? Do you have a number of additional sites and how you expect that to impact potential study conduct? And also, can you talk to sort of like the exclusion criteria where that's driving most of the screen failures for enrollment of the study? And anything else you can give us on potential timing in 2027?

Suma Krishnan

Thank you. So I'll address this question. We are expanding additional sites in the U.S. We have another 10 to 15 sites. Unfortunately, the academic sites big ones that have the patients have been delayed because of contracts. And remember, this is a GMO and it's going to be home administered. So we ran into some glitches with them, but we are overcoming all these sites. So we expect many of these academic sites with a large number of patient pools to come online very shortly. I think we have overcome the bump.

So we continue to expand the U.S. pool, but we are going to go beyond U.S. because there are -- Oxervate is available to these patients. As you know, Oxervate has been aggressively marketed in the U.S. So I think we have an opportunity in the EU where Oxervate is not available. We are -- we have talked to sites. They have a lot of patients. We are in the process of actually activating all of the sites. So we are not -- I mean, we are going pretty aggressively and getting multiple sites across the EU region and even Eastern Europe to get -- to actually really improve our enrollment. So I think with what we are seeing, I'm hopeful that enrollment will pick up.

But to your second point also, we do have some screen failures that we run into because of our stringent inclusion/exclusion criteria. We want to make sure that the lesion that we are seeing is persistent. So if we see any decrease in the lesion size between our first and screening -- second screening, we do not enroll that patient because, obviously, as you know, with this disease that there is a lot of waning and ebbing of the corneal lesion. And we want to make sure we know our drug is therapeutically effective. So we want to make sure we are really enrolling the chronic patients because it's a placebo-controlled study and our chances of winning against the placebo gets is robust. So these 2 factors has delayed NK, but we feel confident that we can get back on track and get this -- the enrollment going early next year with all these multiple sites.

Operator

Our next question is coming from Yigal Nochomovitz with Citigroup.

Yigal Nochomovitz

I just sort of have a broad one on capital allocation. Obviously, you have a lot of cash, $1.2 billion. You lowered R&D a little bit to $155 million to $165 million and your quarterly R&D spend is obviously very, very low at $15 million. So I just was kind of wondering in terms of how you think about spend, I'm just wondering, would you perhaps consider expanding and increasing on R&D, especially since some of these studies like DEB and NK and CF are taking a little bit longer. And if you invested more in each of them, perhaps you could enroll more patients and get the data a little faster. I'm just curious if you could speak to that just general thinking in terms of the way you're deploying capital.

Suma Krishnan

Thanks for the question. I would say the following. if we -- the whole point of initiating a buyback is a high level of comfort that we have enough both in terms of the revenues from VYJUVEK and in terms of supporting our pipeline before we embark on such a things such as cash buyback. And you have to -- I mean, we definitely -- in any place, whether it's NK or CF or oncology or Gorlin, if additional investment would immediately translate to either increasing enrollment or conducting a faster study or opening a site, of course, we are very open to doing that, and we have done some of that. That seems like something we should be doing and we are doing.

So I think the whole timing of the buyback was based on, look, with pricing in Germany and Italy behind us, we have much more clarity on what VJUVEK revenues could be globally and really pleased with how that is trending. We have a really good idea of the amount it would take to bring some of these pipeline products to the market in a timely manner. We also internally believe that the intrinsic value of the -- what the stock value is and what we believe is intrinsic value are dislocated at the moment. And a balance of like $1.2 billion and the $400 million gives us plenty of cushion to do all of the above.

Operator

Our next question is coming from Gavin Clark-Gartner with Evercore.

Gavin Clark-Gartner

Can I just ask for the ocular DEB, for those patients that withdrew, what exactly was the reason? Suma, I think you mentioned the patients may have gotten sick. And then a similar topic for NK, acknowledging it's been slower enrollment. Have you seen the trial completion rates, at least thus far in line with your expectations?

Suma Krishnan

Yes. I'll address the KB803 first. I mean on the DEB eye, I mean, these are patients. Remember, they're difficult patients. I mean -- so these 2 just dropped out because they were traveling and they didn't want to commit to the weekly dosing. So it was becoming -- they don't want to be -- didn't want to be compliant, so they dropped out. And as we mentioned, the third subject is because there were patients that were in a natural history study, and they were expecting to jump on to the study. Some of them came in later. And we had to stop at some point because there were more patients that were wanted to participate.

We did open up for a 1-year-old child, again, because they were very interested in this child at constant abrasion in the eye. We figured this is a younger population that we can add on the label and with request from the investigator and the patient family, we decided to enroll that patient. So just truly because we wanted to get that patient in. And we thought even if we didn't add these patients, we had enough powering for the study. But again, it made sense to add them because it didn't affect the time -- it just pushed the timeline by a few months. So that was the logic -- that was the rationale for including these additional patients in KB803. So nothing with regard to NK.

NK, again, as I mentioned, we have 21 sites up and running. We do have a bunch of screen failures. It's not because of lack of enrolling these patients because of our stringent inclusion/exclusion criteria. As you know, when you have sites open, they do want to put patients in and they put in milder patients or patients that tend to spontaneously close their lesions. So we wanted to ensure that we didn't want to enroll these patients. But now we understand with enrollment, what kind of patients, and we are actively getting additional sites in the U.S. As I mentioned, there were some academic sites that were delayed because, unfortunately, as I said, because of some of the viral safety requirements. I think we have just crossed those hurdles. So we expect with all of these academic sites up and running and the European sites up and running, we are very confident that we should be able to get back on track with enrolling NK patients.

Krish Krishnan

Gavin, just to a couple of things. You mentioned some kind of sickness, but that was a comment on KB407, not on KB803. The second point I'll make on NK is to date, the study is progressing really well, and we feel good about the limited number of patients on the trial.

Operator

Our next question is coming from Lisa Walter with RBC.

Unknown Analyst

Maybe first one for me on the NK program. Can you share how many patients have been enrolled so far or give us the benchmark? Like, for instance, are we at the halfway mark in terms of enrollment? And also on ocular DEB, should we expect to see the results ahead of the NK readout? Any color here would be helpful.

Suma Krishnan

With regarding to NK, we are not giving any guidance. But as I said, we are very confident that we should be able to get these patients -- get the study enrolled 2027. So we are doing all efforts. We don't want to give you any indication -- I mean, any numbers right now. But again, we are -- as I said, from what we see and the sites up and coming, we feel confident we -- we should be able to enroll the patients we need for that trial. On the ocular, again, as I said, we have 3 patients. The last patient out should be end of February. So again, this will be -- we'll -- once we have the last patient in, we should be shortly announcing data on KB803 first half of next year.

Krish Krishnan

Lisa, to answer your question directly, the ocular DEB will come ahead of NK at the moment.

Operator

Our next question is coming from Josh Schimmer with Cantor Fitzgerald.

Joshua Schimmer

On the quarter. This is Alexa on for Josh. So for the Hailey-Hailey disease program, maybe you can clarify a bit what you're looking for in those first 5 patients that would give you confidence to move this program forward into a registrational study?

Suma Krishnan

Hailey-Hailey is, again, this is an indication that there is no prior studies or there's no validated clinical endpoints. As I mentioned earlier, we did have -- we discussed with the FDA. We have a natural history that's ongoing that has enrolled a pretty decent number of patients. We developed a scale based on, again, we have discussions with the agency. We are -- we validated the first part of the scale. The second part of the scale should complete validation. So we're using the scale. It's an investigator-based evaluation.

So the patients come in and the investigator uses the scale to look at improvement in these patients. And we feel pretty good about the scale. As I said, Part A of the validation is completed. The Part B section should be almost done. So it will be an investigator-based evaluation. We show improvement from baseline and after treatment at the end of 3 months. We'll also be biopsying these patients at baseline and at the end of the treatment to show molybdenum correction and also expression of ---sorry, [indiscernible]. So we should have that data by end of this year.

_:p id="534984067" name="Krish Krishnan" type="E" /> And one last point. We also intend to show -- they're not easy to see, but before and after images of the area that we have been treated along with the biopsy and the scale validation improvement.

Operator

Our next question is coming from Roger Song with Jefferies.

Jiale Song

This is Fiona on for Roger. Congrats on the quarter. Just 2 quick ones from us. One is on the ex-U.S. revenue. Now with our Germany and Italy pricing negotiations completed, should we expect a more stabilized ex-U.S. revenue moving forward? Or any other factors that you expect to still contribute to the volatility? And the second one is on o DEB ocular DEB. Assuming the February completion goes smoothly and data is positive, anything else that is outstanding before the filing?

Krish Krishnan

On the ex-U.S., EU revenue, as Laurent mentioned, demand continues to grow really nicely. We're really pleased that pricing in some of the leading countries like Germany and Italy are behind us, and we're not too far away from Spain. And because there are a lot of countries and pricing hasn't been done in many of those countries, approvals will continue to be part of the story until many of those countries, at least the 5 like Italy, Spain, U.K. are behind us. Spain and U.K. are behind us. I wouldn't use the word volatility because demand continues to go up in some of the big countries. Pricing is already done, but we are excited and looking forward to bringing more and more patients in Europe on the drug as soon as possible. Germany was the big one with the approval, and that's behind us at the moment.

Suma Krishnan

Yes. On ocular DEB, I think we are all set. From a CMC perspective, we have all the requirements. I think from a BLA, we should be ready to go as soon as we have the last patient enrolled, and we have locked the database and we analyze the data. So obviously, we are working very diligently to get the BLA started. So it will be just once we have the data, we should be able to file the application shortly after that.

Operator

Our next question is coming from Alec Stranahan with Bank of America.

Alec Stranahan

Two from us, one on commercial and one on the type. I appreciate the color on the pricing negotiations. Maybe just in France, I think the press release said no earlier than 2027. Can you just remind us kind of the nuances around pricing in France specifically? And what sort of gating factors are at this point to get that completed? And then maybe one actually on the Gorlin syndrome program, which I'm surprised no one's actually asked you about. But I guess, what are you sort of working through with the FDA? What kind of size of the study do you think would be feasible in this population? And I guess since you've observed an abscopal effect, do you expect a lesion level or maybe a patient level registration endpoint for the primary?

Krish Krishnan

I'll let Laurent answer the France question.

Laurent Goux

Thank you, Krish. In France, the new President of the committee negotiating the price in terms of negotiating the price has been made clear that they usually expect a 13-month negotiation timelines. And for us, these 13 months, I mean, we are 6 months in this negotiation framework. So we are completely within the boundaries of the negotiations. Usually, the negotiations are in 2 steps. The first step is to agree on what the framework of the reimbursement will be and then entering into the more granular aspect of the pricing itself. So we are well engaged in the negotiations and it's following its normal course.

Suma Krishnan

I'll take the Gorlin question. I mean, as you can see from the 3-patient study where we looked at it, the data was pretty remarkable. We can see response in all 3 patients. I think we were also surprised to see the response. It was consistent across the 3 patients. And we were also able to confirm this by histology to show that the injected tumors that showed complete response had no BCCs in them. So again, very exciting. I mean, as you know, with Gorlin lesions do not heal on its own unlike the other. So whatever we see is a drug effect. So even if you assume placebo as a 0% response and for us to see a pretty good response, we don't expect to see this to be a large study. So again, it should be well within less than 100 patients that we should be able to do a pivotal trial if we have to, based on the response rate that we see in these 3 patients.

Operator

Our next question is coming from Andrea Newkirk with Goldman Sachs.

Andrea Tan

Suma, maybe here to follow up on NK. Could you just remind us on the inclusion criteria with respect to the expected proportion of Oxervate experienced versus naive patients? And then also just given the availability of commercial Oxervate, especially in the U.S., would you consider any protocol adjustments such as adjusted lesion windows to accelerate the accrual?

Suma Krishnan

I mean if you look at our inclusion/exclusion criteria, again, there's 2 screenings. The first screening, we look at the lesion size and obviously, it's random -- it is stratified for lesion surface area. And we -- it's pretty stringent. So even if you see like a 10% decrease between the first and the second screening, we don't enroll those patients. With regards to Oxervate, we have our inclusion criteria where the Oxervate patients have to wash out if they are in Oxervate or if they failed Oxervate and they want to get on our study. There is a 30-day washout for these patients before they can enroll on the study.

Operator

Sorry, did you have another question?

Andrea Tan

Maybe I could just ask one other question on KB407. Could you just confirm if we'll see FEV1 data in the first half of next year when that data set comes? And what threshold of lung function improvement or disease stabilization would you use the benchmark to support the plan for the pivotal program?

Suma Krishnan

Yes. We will have FEV1 data in these patients. Obviously, we are also selecting some of the natural history of these patients. So I think we will have -- I mean, it's not like a number. Hopefully, we will have it holistically. We will look at these patients, what was their lung function over the period of time and the lung function of these patients with -- on KB407. So that's what we are expecting to announce.

Operator

next question is coming from Morgan Lamberti with Goldman Sachs.

Morgan Lamberti

Can you please speak to the rate of recurrence for patients taking Oxervate? And then how that informs your view on the expected TAM for KB801 in terms of if the TAM is actually patients who have not been treated with Oxervate. Just how to think about that.

Suma Krishnan

Yes. I mean we do not really ask those questions. we are -- we do not look at the history, the sales on Oxervate or there's recurrence. We do expect some medical history at this point. It's too early to tell for us. We have not looked at all the data. But all of that, we will have collected as a medical history. We tried to calculate. But we have not -- I mean it's too early to look at that data population to make that determination.

Operator

Our final question today is coming from Kalpit Patel with Wolfe Research.

Kalpit Patel

For ocular DEB, for those 2 patients that withdrew, would those be counted in the ITT efficacy population? And any color on how you might handle incomplete crossover data in the primary analysis, that would be useful. And then for the NK program, you mentioned that you're trying to essentially trying to reduce spontaneous lesion decreases. Do we know -- is that criteria similar to how it was in the Oxervate studies?

Suma Krishnan

I'll take I'll answer the KB803 first. Yes, the 2 patients, yes, they will meet the ITT because that's the definition of ITT. Obviously, we have sensitivity analysis to capture that. And also, remember, we have the natural history of each of these patients. So in our analysis, I'm not going to get into depth of it, but we will be able to use some of that natural history study, even though with the crossover design to help with if there is any carryover effect. So all of that is taken into consideration for the statistical analysis plan for us to improve the chance of success.

Krish Krishnan

Some other question was the spontaneous lesion decrease, did Oxervate also observe that in their study?

Suma Krishnan

We don't know, but they did have an inclusion criteria for some persistence, but I can't comment on the Oxervate what was that level. But I think they did have something in the inclusion criteria. But I don't know the level of decrease for them to enroll that patient, not sure.

Operator

Thank you. Ladies and gentlemen, this does conclude today's question-and-answer session and will also conclude today's conference. You may disconnect your lines at this time, and we thank you for your participation.