Krystal Biotech Announces First Patient Dosed in Phase 3 Clinical Trial of KB803 for the Treatment and Prevention of Corneal Abrasions in Patients with Dystrophic Epidermolysis Bullosa
Krystal Biotech (NASDAQ: KRYS) has initiated dosing in the Phase 3 IOLITE clinical trial of KB803, evaluating its effectiveness in treating and preventing corneal abrasions in dystrophic epidermolysis bullosa (DEB) patients. The trial is designed as an intra-patient, double-blind, multicenter, placebo-controlled study with crossover design.
The study will enroll approximately 16 subjects, targeting DEB patients aged 6 months or older. Previous compassionate use of the treatment showed promising results, with one patient achieving full corneal healing at 3 months and improved visual acuity from hand motion to 20/25 at 8 months. The condition affects over 750 patients in the United States and 2,000 worldwide, with no current corrective therapies available.
The trial's primary endpoint will measure the change in average days per month with corneal abrasion symptoms while receiving KB803 versus placebo. The treatment is administered as a weekly eye drop at a concentration of 109 PFU/mL.
Krystal Biotech (NASDAQ: KRYS) ha iniziato la somministrazione nella sperimentazione clinica di Fase 3 IOLITE di KB803, volta a valutarne l'efficacia nel trattamento e nella prevenzione delle abrasioni corneali nei pazienti affetti da epidermolisi bollosa distrofica (DEB). Lo studio è progettato come uno studio intra-paziente, in doppio cieco, multicentrico, controllato con placebo e con disegno crossover.
Lo studio arruolerà circa 16 soggetti, puntando a pazienti DEB di età pari o superiore a 6 mesi. L'uso compassionevole precedente del trattamento ha mostrato risultati promettenti, con un paziente che ha raggiunto una completa guarigione corneale a 3 mesi e un miglioramento dell'acuità visiva da percezione di movimento della mano a 20/25 a 8 mesi. La condizione colpisce oltre 750 pazienti negli Stati Uniti e 2.000 nel mondo, senza terapie correttive attualmente disponibili.
L'endpoint primario dello studio misurerà la variazione del numero medio di giorni al mese con sintomi di abrasione corneale durante il trattamento con KB803 rispetto al placebo. Il trattamento viene somministrato come collirio settimanale a una concentrazione di 109 PFU/mL.
Krystal Biotech (NASDAQ: KRYS) ha iniciado la dosificación en el ensayo clínico de Fase 3 IOLITE de KB803, que evalúa su efectividad para tratar y prevenir abrasiones corneales en pacientes con epidermólisis bullosa distrófica (DEB). El estudio está diseñado como un ensayo intra-paciente, doble ciego, multicéntrico, controlado con placebo y con diseño cruzado.
El estudio inscribirá a aproximadamente 16 sujetos, con pacientes DEB de 6 meses o más. El uso compasivo previo del tratamiento mostró resultados prometedores, con un paciente que logró la curación completa de la córnea a los 3 meses y una mejora en la agudeza visual de percepción de movimiento manual a 20/25 a los 8 meses. La condición afecta a más de 750 pacientes en Estados Unidos y 2,000 en todo el mundo, sin terapias correctivas disponibles actualmente.
El objetivo principal del ensayo medirá el cambio en el promedio de días por mes con síntomas de abrasión corneal durante el tratamiento con KB803 en comparación con el placebo. El tratamiento se administra como gotas oculares semanales a una concentración de 109 PFU/mL.
Krystal Biotech (NASDAQ: KRYS)는 KB803의 3상 IOLITE 임상시험에서 투약을 시작했으며, 이 시험은 퇴행성 표피박리증(DEB) 환자의 각막 찰과상 치료 및 예방 효과를 평가합니다. 이 시험은 환자 내, 이중 맹검, 다기관, 위약 대조, 교차 설계로 진행됩니다.
본 연구는 약 16명의 DEB 환자(6개월 이상)를 모집할 예정입니다. 이전의 치료 목적 사용에서는 한 환자가 3개월 만에 각막 완전 치유를 이루었고, 8개월 만에 시력이 손 움직임 인지에서 20/25로 개선되는 등 유망한 결과를 보였습니다. 이 질환은 미국 내 750명 이상, 전 세계적으로 2,000명 이상에게 영향을 미치며 현재 교정 치료법은 없습니다.
시험의 주요 평가 지표는 KB803 투여 시와 위약 투여 시 각막 찰과상 증상으로 고통받는 월평균 일수 변화를 측정합니다. 치료는 109 PFU/mL 농도의 주 1회 점안제로 투여됩니다.
Krystal Biotech (NASDAQ : KRYS) a commencé l'administration dans l'essai clinique de phase 3 IOLITE de KB803, évaluant son efficacité pour traiter et prévenir les abrasions cornéennes chez les patients atteints d'épidermolyse bulleuse dystrophique (DEB). L'essai est conçu comme une étude intra-patient, en double aveugle, multicentrique, contrôlée par placebo avec un plan croisé.
L'étude recrutera environ 16 sujets, ciblant des patients DEB âgés de 6 mois ou plus. L'utilisation compassionnelle antérieure du traitement a montré des résultats prometteurs, avec un patient ayant obtenu une guérison complète de la cornée à 3 mois et une amélioration de l'acuité visuelle de la perception des mouvements de la main à 20/25 à 8 mois. La maladie touche plus de 750 patients aux États-Unis et 2 000 dans le monde, sans thérapies correctives actuellement disponibles.
Le critère principal de l'essai mesurera la variation du nombre moyen de jours par mois avec des symptômes d'abrasion cornéenne sous KB803 par rapport au placebo. Le traitement est administré sous forme de collyre hebdomadaire à une concentration de 109 PFU/mL.
Krystal Biotech (NASDAQ: KRYS) hat mit der Dosierung in der Phase-3-Studie IOLITE von KB803 begonnen, die dessen Wirksamkeit bei der Behandlung und Prävention von Hornhautabschürfungen bei Patienten mit dystropher Epidermolysis bullosa (DEB) untersucht. Die Studie ist als intraindividuelle, doppelblinde, multizentrische, placebokontrollierte Studie mit Crossover-Design konzipiert.
Die Studie wird etwa 16 Probanden einschließen, wobei DEB-Patienten ab 6 Monaten eingeschlossen werden. Die bisherige einfühlsame Anwendung der Behandlung zeigte vielversprechende Ergebnisse, wobei ein Patient nach 3 Monaten eine vollständige Hornhautheilung und nach 8 Monaten eine Verbesserung der Sehschärfe von Handbewegung auf 20/25 erreichte. Die Erkrankung betrifft über 750 Patienten in den USA und 2.000 weltweit, wobei derzeit keine korrigierenden Therapien verfügbar sind.
Der primäre Endpunkt der Studie misst die Veränderung der durchschnittlichen Anzahl der Tage pro Monat mit Symptomen von Hornhautabschürfungen unter KB803 im Vergleich zu Placebo. Die Behandlung wird als wöchentlicher Augentropfen mit einer Konzentration von 109 PFU/mL verabreicht.
- Previous compassionate use showed full corneal healing and significant visual improvement
- Large addressable market with over 2,000 patients worldwide and no current corrective therapies
- Decentralized study design allowing home administration
- Strong statistical power of 90% to detect treatment effect
- Small trial size of only 16 subjects
- Requires 12-week run-in period before treatment can begin
- Limited previous data with only one compassionate use case
Insights
Krystal Biotech advances potential first-ever treatment for DEB corneal abrasions with promising early data in Phase 3 trial.
Krystal Biotech has initiated a critical Phase 3 trial for KB803, positioning the company to potentially address a significant unmet need in dystrophic epidermolysis bullosa (DEB) patients suffering from corneal abrasions. The trial design demonstrates robust statistical planning with a sample size of approximately 16 subjects providing 90% power to detect an effect size of at least 25% - notably strong for a rare disease study. The crossover design is particularly sophisticated, enabling each patient to serve as their own control, which is ideal for conditions with high inter-patient variability.
The earlier compassionate use case provides compelling preliminary evidence - a patient improved from hand motion vision to 20/25 visual acuity at 8 months, representing a dramatic functional improvement. This suggests KB803's COL7A1 gene delivery mechanism effectively enables local type VII collagen production in the corneal epithelium.
The market opportunity, while modest in absolute numbers (~750 US patients and ~2,000 worldwide), represents a concentrated patient population with no existing corrective therapies. The decentralized study design with at-home drug administration demonstrates patient-centric trial execution, likely enhancing recruitment and retention in this rare disease population.
If successful, KB803 would complement Krystal's existing DEB portfolio and reinforce their strategy of comprehensive disease management. The primary endpoint of reducing symptomatic days provides a clinically meaningful and patient-relevant outcome measure that directly addresses the burden of disease.
Intra-patient, double-blind, multicenter, placebo-controlled study with crossover design
Repeat administration under compassionate use was previously shown to be well tolerated and associated with full corneal healing by 3 months as well as significant visual acuity improvement from hand motion to 20/25 at 8 months
PITTSBURGH, June 24, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) announced today that the first patient has been dosed in its Phase 3 clinical trial (“IOLITE”), an intra-patient, double-blind, multicenter, placebo-controlled study with crossover design evaluating KB803 for the treatment and prevention of corneal abrasions in dystrophic epidermolysis bullosa (DEB) patients. KB803 is designed to deliver two copies of the COL7A1 transgene to the corneal epithelium and enable local type VII collagen production in the front of the eye.
“The initiation of IOLITE is another important step for Krystal as we work tirelessly to treat DEB as comprehensively as possible,” said Suma Krishnan, President, Research & Development, Krystal Biotech, Inc. “With dramatic and durable improvements already reported for the patient treated under compassionate use, we are excited by the potential of KB803 to restore full eye function and reduce or eliminate the otherwise persistent threat of vision loss imposed by these recurring corneal abrasions.”
Over
IOLITE Phase 3 Study
IOLITE is an intra-patient, double-blind, placebo-controlled, multicenter Phase 3 study with a crossover design to evaluate KB803, administered as an eye drop, for the treatment and prevention of corneal abrasions in DEB patients, 6 months of age or older. Patients seeking to participate in IOLITE must first enroll in the ongoing natural history study and complete a 12-week run-in period, during which they report the number of days that they experience symptoms of corneal abrasions. Subjects meeting the inclusion criteria following the 12-week run-in are eligible to participate in the IOLITE trial.
Approximately 16 subjects will be enrolled in the IOLITE study. The sample size was calculated based on the average symptomatic days per month and standard deviation data from subjects enrolled in the natural history study who would be otherwise eligible for IOLITE and provides
The primary study endpoint will be the change in the average number of days per month with corneal abrasion symptoms while receiving KB803 versus placebo. Statistical significance will be analyzed via intra-patient paired measurements to account for potentially expected high inter-patient variability. Safety and secondary efficacy data, including weekly assessments of eye pain and monthly Epidermolysis Bullosa Eye Disease Index (EB-EDI) questionnaires, will be collected through to the end of the 24-week study period. More details of the IOLITE study can be found at www.clinicaltrials.gov under NCT identifier NCT07016750.
Ongoing Natural History Study
48 DEB patients have been enrolled in the ongoing natural history study. 39 of the 48 subjects (
Previous Compassionate Use Case
Beremagene geperpavec-svdt (B-VEC) had previously been applied to the eye of one DEB patient under a compassionate use protocol. The clinical observations of this compassionate use case were published in the New England Journal of Medicine in February 2024. The patient presented with severe cicatrizing conjunctivitis secondary to DEB. Surgical symblepharon lysis of the patient’s right eye with pannus removal was conducted and regular B-VEC administration as an eye drop directly to the eye, at a concentration of ~109 PFU/mL, were added to routine post-surgical care. Treatment was well tolerated with no drug-related adverse events noted. Full corneal healing was observed at three months, as well as significant visual acuity improvement from hand motion to 20/25 by eight months.
About KB803
KB803 is a redosable, eye drop gene therapy designed to deliver two copies of the COL7A1 transgene to the epithelial cells in a patient’s eye for the treatment and prevention of corneal abrasions in DEB patients. The goal of therapy with KB803 is to address the fundamental disease-causing mechanism at the molecular level, by providing the patient’s epithelial cells of the eye with the template to make normal type VII collagen locally. In a DEB patient treated under compassionate use with clinical observations reported the New England Journal of Medicine, regular eye drop administration was well tolerated with full corneal healing observed at three months, as well as significant visual acuity improvement from hand motion to 20/25 by eight months.
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., including statements about the potential of KB803 to restore full eye function and reduce or eliminate the otherwise persistent threat of vision loss imposed by recurring corneal abrasions associated with DEB; the Company’s expectations (i) that over
CONTACT
Investors and Media:
Stéphane Paquette, PhD
Krystal Biotech
spaquette@krystalbio.com
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