Krystal Biotech Announces First Patient Dosed in Phase 1/2 Trial of KB801 for the Treatment of Neurotrophic Keratitis
Krystal Biotech (NASDAQ: KRYS) has initiated its Phase 1/2 clinical trial EMERALD-1 with the first patient dosed using KB801, an innovative eye drop gene therapy for treating neurotrophic keratitis (NK). The trial is a randomized, double-masked, multicenter, placebo-controlled study that will enroll up to 27 adults with Stage 2 or Stage 3 NK.
The study will evaluate KB801's safety and efficacy, administered twice weekly for 8 weeks. NK is a degenerative corneal disease affecting an estimated 68,000 patients in the United States as of 2024, marking a significant 115% increase from 31,000 patients in 2020. The therapy aims to overcome current limitations in treating corneal epithelial defects through sustained protein expression via simple eye drop application.
Krystal Biotech (NASDAQ: KRYS) ha avviato il suo studio clinico di Fase 1/2 EMERALD-1, con il primo paziente trattato con KB801, una terapia genica innovativa in gocce oculari per il trattamento della cheratite neurotrofica (NK). Lo studio è randomizzato, in doppio cieco, multicentrico e controllato con placebo, e prevede l'arruolamento di fino a 27 adulti con NK di Stadio 2 o Stadio 3.
La ricerca valuterà la sicurezza e l'efficacia di KB801, somministrato due volte a settimana per 8 settimane. La NK è una malattia degenerativa della cornea che colpisce circa 68.000 pazienti negli Stati Uniti nel 2024, segnando un significativo aumento del 115% rispetto ai 31.000 pazienti del 2020. La terapia mira a superare le limitazioni attuali nel trattamento dei difetti epiteliali corneali attraverso un'espressione proteica sostenuta tramite la semplice applicazione di gocce oculari.
Krystal Biotech (NASDAQ: KRYS) ha iniciado su ensayo clínico de Fase 1/2 EMERALD-1, con el primer paciente tratado con KB801, una innovadora terapia génica en gotas oculares para el tratamiento de la queratitis neurotrófica (NK). El estudio es aleatorizado, doble ciego, multicéntrico y controlado con placebo, y reclutará hasta 27 adultos con NK en etapa 2 o etapa 3.
El estudio evaluará la seguridad y eficacia de KB801, administrado dos veces por semana durante 8 semanas. La NK es una enfermedad degenerativa de la córnea que afecta a aproximadamente 68,000 pacientes en Estados Unidos en 2024, representando un aumento significativo del 115% respecto a los 31,000 pacientes en 2020. La terapia busca superar las limitaciones actuales en el tratamiento de defectos epiteliales corneales mediante una expresión proteica sostenida a través de la sencilla aplicación en gotas oculares.
Krystal Biotech (NASDAQ: KRYS)가 신경영양 각막염(NK) 치료를 위한 혁신적인 점안 유전자 치료제 KB801를 사용하여 첫 환자 투여를 시작하며 1/2상 임상시험 EMERALD-1을 개시했습니다. 이 임상시험은 무작위 배정, 이중 눈가림, 다기관, 위약 대조 연구로, 2기 또는 3기 NK 성인 최대 27명을 등록할 예정입니다.
본 연구는 8주 동안 주 2회 투여되는 KB801의 안전성과 효능을 평가합니다. NK는 2024년 미국에서 약 68,000명의 환자에게 영향을 미치는 퇴행성 각막 질환으로, 2020년 31,000명 대비 115%의 상당한 증가를 보였습니다. 이 치료법은 간단한 점안제 적용을 통해 지속적인 단백질 발현으로 각막 상피 결손 치료의 기존 한계를 극복하는 것을 목표로 합니다.
Krystal Biotech (NASDAQ : KRYS) a lancé son essai clinique de phase 1/2 EMERALD-1 avec le premier patient traité par KB801, une thérapie génique innovante sous forme de gouttes oculaires pour le traitement de la kératite neurotrophique (NK). L'étude est randomisée, en double aveugle, multicentrique et contrôlée par placebo, et prévoit d'inclure jusqu'à 27 adultes atteints de NK de stade 2 ou 3.
L'étude évaluera la sécurité et l'efficacité de KB801, administré deux fois par semaine pendant 8 semaines. La NK est une maladie dégénérative de la cornée qui touche environ 68 000 patients aux États-Unis en 2024, soit une augmentation significative de 115 % par rapport aux 31 000 patients en 2020. Cette thérapie vise à dépasser les limites actuelles du traitement des défauts épithéliaux cornéens grâce à une expression protéique soutenue via une simple application de gouttes oculaires.
Krystal Biotech (NASDAQ: KRYS) hat seine Phase-1/2-Studie EMERALD-1 gestartet und den ersten Patienten mit KB801 behandelt, einer innovativen Gentherapie in Augentropfenform zur Behandlung der neurotrophen Keratitis (NK). Die Studie ist randomisiert, doppelblind, multizentrisch und placebokontrolliert und wird bis zu 27 Erwachsene mit NK im Stadium 2 oder 3 einschließen.
Die Studie wird die Sicherheit und Wirksamkeit von KB801 bewerten, das zweimal wöchentlich über 8 Wochen verabreicht wird. NK ist eine degenerative Hornhauterkrankung, die in den USA im Jahr 2024 schätzungsweise 68.000 Patienten betrifft – ein signifikanter Anstieg von 115% gegenüber 31.000 Patienten im Jahr 2020. Die Therapie zielt darauf ab, die derzeitigen Einschränkungen bei der Behandlung von Hornhautepitheldefekten durch eine anhaltende Proteinexpression mittels einfacher Augentropfenanwendung zu überwinden.
- Novel eye drop gene therapy approach for sustained protein expression
- Significant market opportunity with NK patient claims increasing 115% from 2020 to 2024
- Addresses an unmet medical need in a degenerative corneal disease
- Early-stage clinical trial with uncertain outcomes
- Small trial size of only 27 patients may limit statistical significance
Insights
Krystal Biotech's first patient dosed in NK gene therapy trial represents potentially breakthrough approach using redosable eye drops for sustained protein expression.
Krystal Biotech has reached a significant clinical milestone with the dosing of its first patient in the EMERALD-1 Phase 1/2 trial for KB801, their gene therapy candidate for neurotrophic keratitis (NK). This study is structured as a 2:1 randomized, double-masked, multicenter, placebo-controlled trial - the gold standard design for clinical evaluation.
What makes KB801 scientifically notable is its novel delivery mechanism: a redosable eye drop gene therapy designed to overcome two major limitations that have historically hindered biologic treatments for corneal conditions: rapid protein clearance rates and high cell turnover in the anterior eye. By enabling sustained expression of nerve growth factor (NGF) directly at the disease site through simple eye drop administration, this approach could potentially offer significant advantages over current treatments that require frequent application or invasive procedures.
The clinical significance is substantial considering NK's progressive nature - where damage to corneal nerves leads to epithelial defects, ulcers, and potentially severe vision loss if untreated. The prevalence data cited (10-50 cases per 100,000) confirms this is an orphan indication, yet the company reports diagnosis rates are rising sharply, with 68,000 US patients having NK claims in 2024 - a 115% increase from 2020's 31,000 patients. This growth trajectory suggests both improved disease awareness and potentially expanding market opportunity.
The trial's primary endpoint appropriately focuses on safety and tolerability, while secondary endpoints measuring complete durable healing of corneal epithelium will provide initial efficacy signals. The twice-weekly dosing schedule, if successful with minimal adverse effects, would represent a significant treatment burden reduction compared to daily applications required by some existing therapies.
Eye drop administration of KB801 designed to enable sustained expression of NGF in the front of the eye
Investor call and webcast to be held July 9 at 8:30 am ET to discuss program and trial design
PITTSBURGH, July 09, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) announced today that the first patient has been dosed in its Phase 1/2 clinical trial (“EMERALD-1”), a 2:1 randomized, double-masked, multicenter, placebo-controlled study evaluating KB801 for the treatment of neurotrophic keratitis (NK).
“Our ophthalmology pipeline is well under way with the dosing of our first patient in EMERALD-1, along with initiation of our Phase 3 IOLITE study in DEB,” said Suma Krishnan, President, Research & Development, Krystal Biotech, Inc. “Until now, rapid protein clearance rates and high cell turnover in the front of the eye have severely limited the therapeutic potential of biologics and gene therapies for the treatment of corneal epithelial defects and other front of the eye diseases. We now have an opportunity to drive sustained expression and repeat administration of therapeutic biologic payloads with a simple eye drop application and look forward to delivering meaningful benefit to NK patients in need.”
KB801 is a redosable eye drop gene therapy in development for the treatment of NK, a degenerative corneal disease characterized by damage or loss of function in the neurons innervating the eye leading to corneal epithelial defects, ulcers, and perforation. Left untreated, NK can result in severe vision loss. Although NK is a rare disease with an estimated prevalence in the range of 10 to 50 cases per 100,000, claims data analyses suggest awareness and diagnosis rates are on the rise in the United States. Based on available claims data, an estimated 68,000 patients in the United States had a NK claim in 2024, up over
EMERALD-1 is a randomized, double-masked, multicenter, placebo-controlled study evaluating KB801, administered as an eye drop, for the treatment of NK. Up to 27 adults with Stage 2 or Stage 3 NK, as defined by the Mackie criteria, will be enrolled and randomized 2:1 to receive either KB801, at a concentration of 1010 PFU/mL, or placebo topically to the study eye twice weekly for 8 weeks.
The primary objective of EMERALD-1 is to evaluate the safety and tolerability of topical ocular administration of KB801 in patients with NK. The secondary objective is evaluation of efficacy based on the proportion of patients with complete durable healing of corneal epithelium at 8 weeks, defined as 0 mm corneal fluorescein staining in the area of the corneal lesion at both week 8 and 0 mm corneal fluorescein staining in the same area at week 10, as assessed by a masked reader. Additional exploratory efficacy measures will include change in corneal lesion size from baseline, each assessed at weeks 4, 6, 8, 10, and 20, as well as evaluations of corneal sensation and patient-reported symptom burden. More details of the EMERALD-1 study can be found at www.clinicaltrials.gov under NCT identifier NCT06999733.
The Company will host an investor conference call and webcast today, Wednesday, July 9, 2025, at 8:30 am ET, to discuss NK, the KB801 program, and clinical study design. Investors and the general public can access the live webcast at: https://www.webcaster4.com/Webcast/Page/3018/52704. For those unable to listen to the live webcast, an archived version will also be available on the Investors section of the Company’s website for at least 30 days.
About KB801
KB801 is a redosable eye drop gene therapy designed to enable sustained, localized expression and secretion of nerve growth factor (NGF) by epithelial cells in the front of the eye for the treatment of neurotrophic keratitis (NK), a rare, degenerative corneal disease that leads to corneal epithelial defects, ulcers, and perforation. Recombinant NGF eye drops have been shown to significantly improve corneal healing and are approved for the treatment of NK in multiple jurisdictions worldwide, but rapid clearance from the eye requires intensive administration six times a day, limiting therapeutic utility. By enabling the cells of the front of the eye to produce NGF locally, KB801 has the potential to significantly reduce the treatment burden for patients while also maintaining more consistent NGF levels in the front of the eye.
About Krystal Biotech, Inc.
Krystal Biotech, Inc. (NASDAQ: KRYS) is a fully integrated, commercial-stage, global biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs. VYJUVEK®, the Company’s first commercial product, is the first-ever redosable gene therapy and the first genetic medicine approved by the FDA and EMA for the treatment of dystrophic epidermolysis bullosa. The Company is rapidly advancing a robust preclinical and clinical pipeline of investigational genetic medicines in respiratory, oncology, dermatology, ophthalmology, and aesthetics. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania. For more information, please visit http://www.krystalbio.com, and follow @KrystalBiotech on LinkedIn and X (formerly Twitter).
Forward-Looking Statements
Any statements in this press release about future expectations, plans and prospects for Krystal Biotech, Inc.’s product candidate, KB801, and its EMERALD-1 clinical trial evaluating KB801 for the treatment of neurotrophic keratitis (NK), including statements about the potential of KB801 to significantly reduce the treatment burden for NK patients while also maintaining more consistent NGF levels in the front of the eye resulting in meaningful benefits to NK patients; the estimated prevalence of NK and the expectation, based on claims data analyses, that awareness and diagnosis rates are on the rise in the United States; the EMERALD-1 trial design, including the expected number of patients that will be participating in the study and the study objectives; and other statements containing the words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “likely,” “will,” “would,” “could,” “should,” “continue,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including uncertainties inherent in the initiation and conduct of clinical trials, as well as regulatory review of clinical trials and applications for marketing approvals; and such other important factors as are set forth under the caption “Risk Factors” in the Company’s annual and quarterly reports on file with the U.S. Securities and Exchange Commission. The forward-looking statements included in this press release represent the Company’s views as of the date of this press release. The Company anticipates that subsequent events and developments will cause its views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company’s views as of any date subsequent to the date of this press release.
CONTACT
Investors and Media:
Stéphane Paquette, PhD
Krystal Biotech
spaquette@krystalbio.com
FAQ
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