크리스탈 바이오텍(KRYS) 2026년 3분기 실적 발표 컨퍼런스 콜: VYJUVEK 매출 30% 증가

핵심 요약

• 크리스탈 바이오텍이 발표한 2026년 3분기 비주벡 순매출은 1억 2,730만 달러로 전년 동기 대비 30% 증가했으며, 여기에는 9,530만 달러의 미국 매출과 3,200만 달러의 해외 매출이 포함됩니다.
• 당기순이익은 6,310만 달러, 희석 EPS는 2.05달러를 기록했습니다. 매출총이익률은 94%로 기존 96%에서 하락했는데, 이는 미국에서 승인된 제조 공정 최적화의 혜택이 유럽 및 일본에 아직 반영되지 않은 점도 일부 원인입니다.
• 회사는 9월 말 기준 12억 달러 이상의 현금 및 투자 자산을 보유하며 분기를 마감했습니다. 이사회는 최대 4억 달러 규모의 자사주 매입을 승인했으며, 이는 2년에 걸쳐 집행될 예정입니다.
• 미국 상업화 성과로는 이번 분기 동안 760건 이상의 보험 급여 승인과 80명 이상의 신규 처방의 확보가 포함됩니다. 현재 환자의 약 80%가 비주벡을 자가 투여하고 있습니다.
• 경영진은 연간 비GAAP R&D 및 SG&A 가이던스를 기존 1억 7,500만~1억 9,500만 달러에서 1억 5,500만~1억 6,500만 달러로 하향 조정했습니다.
• 몇몇 임상 일정이 변경되었습니다. 안구 이영양성 수포성 표피박리증(DEB) 치료제 KB803의 결과는 2027년 2월 마지막 환자 방문 직후 발표될 것으로 예상되며, 신경영양성 각막염 치료제 KB801의 환자 등록 및 탑라인 결과는 2027년에 발표될 것으로 예상됩니다.

핵심 재무 실적

크리스탈 측은 출시 이후 비주벡의 누적 매출이 약 11억 달러에 달했다고 밝혔습니다.

사업 및 운영 실적

비주벡 미국 시장 성장

크리스탈은 3분기 동안 미국 전역에서 760건 이상의 보험 급여 승인을 확보하고 80명 이상의 처방의를 새롭게 추가했습니다. 경영진은 진단받았으나 치료를 시작하지 않은 환자들과 미진단 또는 오진 상태로 남아 있는 환자들 사이에서 추가적인 기회가 있을 것으로 보고 있습니다.

재택 치료도 더욱 정착되어 현재 환자의 약 80%가 비주벡을 자가 투여하고 있습니다. 회사는 환자의 상처 관리 요구 변화에 맞춘 지속적인 투여를 지원하는 동시에, 신규 치료 개시 확대에 집중하고 있습니다.

해외 시장 확장

비주벡의 해외 매출은 3,200만 달러에 달했습니다. 독일에서는 환자 투여를 시작한 병원이 20개 이상으로 늘어나 이전 분기 대비 약 15% 증가했습니다. 독일에서의 약가 협상은 완료되었습니다.

프랑스에서는 원외처방(retrocession) 경로를 통해 첫 재택 투여를 완료했습니다. 국가 약가 협상이 진행 중이며, 경영진은 이 절차가 통상적인 일정에 따라 진행되고 있다고 설명했습니다.

일본에서 크리스탈은 환자 및 처방의 수를 지속적으로 늘려가고 있습니다. 경영진은 첫해 처방 제한이 완화되면 제품 도입이 더욱 빨라질 것으로 기대하고 있습니다.

이탈리아에서의 약가 및 급여 협상이 타결되어, 회사는 2027년 초 이탈리아에서 비주벡을 출시할 수 있을 것으로 예상하고 있습니다. 스페인의 약가 결정은 2026년 말 이전에 이뤄질 것으로 보이며, 일부 환자들은 이미 예외적인 지역 기금 지원 제도를 통해 치료를 받고 있습니다.

파이프라인 진행 상황

골린 증후군 성인 환자 3명을 대상으로 한 KB113의 초기 결과에서 약물이 투여된 기저세포암과 투여되지 않은 기저세포암 모두에서 반응이 나타났습니다. 평가된 병변의 절반 이상이 50% 이상 관해되었으며, 종양의 약 10%는 90% 이상 축소되었습니다. 조직학적 평가 결과, 각 환자의 투여 병변 생검 1건에서 완치가 확인되었습니다. 크리스탈은 FDA와 향후 개발 경로를 논의하고 있습니다.

헤일리-헤일리병 치료제 KB111의 경우, HALITE-1 반복 투여 임상시험에 환자 5명의 등록이 완료되었습니다. 경영진은 2026년 말 이전에 데이터 업데이트를 진행한 후 FDA와 논의를 이어갈 예정입니다.

안구 DEB 임상과 관련해 크리스탈은 안전성과 무관한 사유로 이탈한 환자 2명의 대체 환자 및 1세 소아 환자를 포함해 KB803 환자 3명을 추가로 등록했습니다. 마지막 환자 방문은 2027년 2월로 예정되어 있으며, 직후 탑라인 결과가 발표될 예정입니다.

낭성 섬유증 환자 3명을 대상으로 KB407의 반복 투여가 진행 중이며, 연말까지 3명의 환자가 추가로 등록되어 투여받을 예정입니다. 1초간 강제 호기량(FEV1) 데이터를 포함한 중간 결과는 2027년 상반기에 발표될 것으로 예상됩니다. 또한 회사는 허가용 임상시험 디자인에 대해 FDA와 협의하고 있습니다.

진행성 비소세포폐암 치료를 위한 흡입형 KB707의 경우, 크리스탈은 환자 10명을 대상으로 용량 최적화를 완료했습니다. 회사는 2027년 중반까지 선정된 화학요법 용법에 따라 70명의 환자를 등록하고 투여할 계획입니다.

경영진 가이던스

• 연간 비GAAP R&D 및 SG&A 가이던스는 기존 1억 7,500만~1억 9,500만 달러에서 1억 5,500만~1억 6,500만 달러로 하향 조정되었습니다.
• 시장 접근 계획 실행에 따라 비주벡은 2027년 초 이탈리아에서 출시될 예정입니다.
• 스페인의 약가 결정은 2026년 말 이전에 이뤄질 것으로 예상됩니다.
• KB111 헤일리-헤일리병 임상 데이터는 2026년 말 이전에 발표될 것으로 예상됩니다.
• KB803 안구 DEB 탑라인 결과는 2027년 2월 마지막 환자 방문 직후 발표될 것으로 예상됩니다.
• KB801 신경영양성 각막염 환자 등록 완료 및 탑라인 결과 발표는 2027년에 이뤄질 것으로 예상됩니다.
• KB407 낭성 섬유증 중간 결과는 2027년 상반기에 발표될 것으로 예상됩니다.
• 회사는 승인된 4억 달러 규모의 자사주 매입 프로그램을 2년에 걸쳐 집행하는 한편, 상업화 확대 및 임상 개발 자금을 지속적으로 지원할 계획입니다.

리스크 및 주요 관전 포인트

몇몇 임상 결과 발표가 당초 예상보다 더 오래 걸리고 있습니다. KB801의 환자 등록은 좁은 자격 기준, 선별 탈락, 학술 기관에서의 계약 지연 및 미국 내 이미 승인된 기존 치료제와의 경쟁 등으로 영향을 받았습니다.

KB803의 일정은 환자 2명이 매주 투여받는 것에 부담을 느껴 임상을 철회하면서 연기되었습니다. 경영진은 이들의 철회가 안전성과는 무관하다고 밝혔으나, 해당 환자들은 치료의도(intent-to-treat) 분석 대상군에 계속 포함될 예정입니다.

KB407의 환자 등록은 참여 예정 환자들이 병에 걸리면서 지연되었습니다. 경영진은 이번 추가 등록이 용량 문제 때문이 아니라는 점을 강조하며, 현재 환자들이 반복 투여를 잘 견디고 있다고 밝혔습니다.

해외 국가의 보험 급여 적용은 아직 완료되지 않았습니다. 프랑스는 여전히 약가 협상을 진행 중이며, 다른 유럽 국가들도 시장 접근 승인이 필요한 상황입니다. 또한 미국에서 승인된 제조 공정 최적화가 적용되지 않은 상태로 유럽과 일본 사업이 운영되는 동안 매출총이익률이 영향을 받을 수 있습니다.

애널리스트 Q&A 주요 내용

• 자본 배분: 경영진은 4억 달러 규모의 자사주 매입 승인이 비주벡 매출과 회사의 현금 잔고로 파이프라인 개발과 주주 환원을 모두 지원할 수 있다는 자신감을 반영한다고 설명했습니다. 크리스탈은 추가 지출이 환자 등록이나 임상 기관 활성화를 가속화할 수 있는 경우 투자를 늘릴 용의가 있다고 덧붙였습니다.
• KB801 환자 등록: 회사는 미국 내 10~15개 임상 기관을 추가하고 프랑스, 스페인, 영국 및 기타 유럽 시장 전역의 기관을 활성화할 계획입니다. 크리스탈은 각막 병변이 자연적으로 개선되는 환자가 등록되는 리스크를 줄이기 위해 엄격한 병변 지속성 기준을 유지하고 있습니다.
• KB803 신청 준비 상태: 경영진은 제조품질관리(CMC) 요건이 갖추어졌으며, 긍정적인 결과가 나올 경우 데이터베이스 락(lock) 및 분석 직후 생물학적 제제 품목허가 신청(BLA)을 제출할 수 있을 것으로 예상한다고 밝혔습니다.
• 골린 증후군 개발: 환자 3명을 대상으로 한 파일럿 연구에서 관찰된 반응을 바탕으로, 경영진은 FDA와의 협의에 따라 향후 중추적(pivotal) 임상시험에 100명 미만의 환자가 참여할 수 있을 것으로 보고 있습니다.
• 결과 발표 순서: 경영진은 안구 DEB 결과가 KB801 신경영양성 각막염 결과보다 먼저 발표될 것으로 예상된다고 확인했습니다.

실적 발표 컨퍼런스 콜 전문

전체 실적 발표 컨퍼런스 콜 녹취록

경영진 발표

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.

질의응답

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.