Opus Genetics Announces Updates on OPGx-LCA5 Clinical Program
Opus Genetics (Nasdaq: IRD) has announced significant progress in its Phase 1/2 clinical trial for OPGx-LCA5, a gene therapy treatment for Leber congenital amaurosis (LCA). The company has successfully dosed its first pediatric patient and plans to share initial data from this cohort by Q3 2025.
The trial has already shown promising results in adult patients, with meaningful visual improvements observed as early as one month after treatment in the first three adult patients. New 12-month data on these adult patients will be presented at a major medical conference in Q2 2025.
An FDA Type D meeting is scheduled for March 2025 to discuss Phase 3 trial design and registrational endpoints. The ongoing Phase 1/2 trial evaluates safety and preliminary efficacy through multiple endpoints, including the Multi-Luminance orientation and Mobility Test, Full-Field Stimulus Testing, and microperimetry.
Opus Genetics (Nasdaq: IRD) ha annunciato progressi significativi nel suo trial clinico di Fase 1/2 per OPGx-LCA5, un trattamento di terapia genica per l'amaurosi congenita di Leber (LCA). L'azienda ha somministrato con successo la dose al suo primo paziente pediatrico e prevede di condividere i dati iniziali da questo gruppo entro il terzo trimestre del 2025.
Il trial ha già mostrato risultati promettenti nei pazienti adulti, con miglioramenti visivi significativi osservati già un mese dopo il trattamento nei primi tre pazienti adulti. Nuovi dati a 12 mesi su questi pazienti adulti saranno presentati a una importante conferenza medica nel secondo trimestre del 2025.
Un incontro di tipo D con la FDA è programmato per marzo 2025 per discutere la progettazione del trial di Fase 3 e i parametri di registrazione. L'attuale trial di Fase 1/2 valuta la sicurezza e l'efficacia preliminare attraverso molteplici parametri, inclusi il Test di Orientamento e Mobilità Multi-Luminance, il Test di Stimolo a Campo Completo e la microperimetria.
Opus Genetics (Nasdaq: IRD) ha anunciado avances significativos en su ensayo clínico de Fase 1/2 para OPGx-LCA5, un tratamiento de terapia génica para la amaurosis congénita de Leber (LCA). La compañía ha administrado con éxito la dosis a su primer paciente pediátrico y planea compartir datos iniciales de este grupo para el tercer trimestre de 2025.
El ensayo ya ha mostrado resultados prometedores en pacientes adultos, con mejoras visuales significativas observadas tan pronto como un mes después del tratamiento en los primeros tres pacientes adultos. Nuevos datos a 12 meses sobre estos pacientes adultos se presentarán en una importante conferencia médica en el segundo trimestre de 2025.
Una reunión de tipo D de la FDA está programada para marzo de 2025 para discutir el diseño del ensayo de Fase 3 y los puntos finales de registro. El ensayo de Fase 1/2 en curso evalúa la seguridad y la eficacia preliminar a través de múltiples puntos finales, incluidos el Test de Orientación y Movilidad Multi-Luminance, el Test de Estímulo de Campo Completo y la microperimetría.
Opus Genetics (Nasdaq: IRD)는 레버 선천성 흑암증(LCA)을 위한 유전자 치료제 OPGx-LCA5의 1/2상 임상 시험에서 중요한 진전을 발표했습니다. 이 회사는 첫 번째 소아 환자에게 성공적으로 약물을 투여했으며, 2025년 3분기까지 이 집단의 초기 데이터를 공유할 계획입니다.
이 임상 시험은 이미 성인 환자에서 유망한 결과를 보여주었으며, 첫 세 명의 성인 환자에서 치료 한 달 만에 의미 있는 시각 개선이 관찰되었습니다. 이 성인 환자들에 대한 12개월 새로운 데이터는 2025년 2분기에 열리는 주요 의료 회의에서 발표될 예정입니다.
FDA의 타입 D 미팅이 2025년 3월로 예정되어 있으며, 3상 시험 설계 및 등록 목표에 대해 논의할 예정입니다. 진행 중인 1/2상 임상 시험은 다수의 지표를 통해 안전성 및 초기 효능을 평가하며, 여기에는 다중 밝기 방향 및 이동 테스트, 전체 자극 테스트, 미세 망막측정이 포함됩니다.
Opus Genetics (Nasdaq: IRD) a annoncé des progrès significatifs dans son essai clinique de Phase 1/2 pour OPGx-LCA5, un traitement par thérapie génique pour l'amaurose congénitale de Leber (LCA). L'entreprise a réussi à administrer la première dose à son premier patient pédiatrique et prévoit de partager les premières données de cette cohorte d'ici le troisième trimestre 2025.
L'essai a déjà montré des résultats prometteurs chez les patients adultes, avec des améliorations visuelles significatives observées dès un mois après le traitement chez les trois premiers patients adultes. De nouvelles données à 12 mois sur ces patients adultes seront présentées lors d'une importante conférence médicale au deuxième trimestre 2025.
Une réunion de type D avec la FDA est prévue pour mars 2025 afin de discuter de la conception de l'essai de Phase 3 et des points de terminaison d'enregistrement. L'essai de Phase 1/2 en cours évalue la sécurité et l'efficacité préliminaire à travers plusieurs points de terminaison, y compris le Test d'Orientation et de Mobilité Multi-Luminance, le Test de Stimulation de Champ Complet et la microperimétrie.
Opus Genetics (Nasdaq: IRD) hat bedeutende Fortschritte in seiner klinischen Studie der Phase 1/2 für OPGx-LCA5, eine Gentherapie zur Behandlung der Leber kongenitalen Amaurose (LCA), bekannt gegeben. Das Unternehmen hat erfolgreich seinen ersten pädiatrischen Patienten behandelt und plant, bis zum dritten Quartal 2025 erste Daten aus dieser Kohorte zu teilen.
Die Studie hat bereits vielversprechende Ergebnisse bei erwachsenen Patienten gezeigt, mit signifikanten visuellen Verbesserungen, die bereits einen Monat nach der Behandlung bei den ersten drei erwachsenen Patienten beobachtet wurden. Neue 12-Monats-Daten zu diesen erwachsenen Patienten werden auf einer wichtigen medizinischen Konferenz im zweiten Quartal 2025 präsentiert.
Ein FDA-Typ-D-Meeting ist für März 2025 angesetzt, um das Design der Phase-3-Studie und die Registrierungsziele zu besprechen. Die laufende Phase-1/2-Studie bewertet die Sicherheit und vorläufige Wirksamkeit durch mehrere Endpunkte, einschließlich des Multi-Luminance-Orientierungs- und Mobilitätstests, des Full-Field-Stimulationstests und der Mikroperimetrie.
- Early clinical proof of concept achieved with meaningful visual improvement in adult patients
- Successful expansion of trial to pediatric patients
- Positive 6-month data confirmed by new 12-month results showing durability of response
- FDA meeting scheduled to discuss Phase 3 trial design, indicating program advancement
- None.
Insights
The latest clinical developments for Opus Genetics' OPGx-LCA5 gene therapy program represent several critical value-creating milestones. The expansion into pediatric patients is particularly significant as early intervention in LCA5 could maximize therapeutic benefit before disease progression causes irreversible vision loss. The rapid onset of efficacy - showing improvements within one month - is remarkable for gene therapy, where benefits typically take longer to manifest.
The upcoming 12-month data presentation is important for three reasons:
- It will validate the durability of treatment effects, a key consideration for gene therapy commercialization
- It will support discussions with the FDA regarding Phase 3 trial design
- It could help establish OPGx-LCA5 as a potential first-in-class therapy for LCA5
The trial's sophisticated endpoint selection, including the Multi-Luminance orientation and Mobility Test (MLoMT), Full-Field Stimulus Testing, and microperimetry, provides a comprehensive assessment of functional vision improvement. These endpoints are particularly relevant for regulatory approval as they measure real-world visual function rather than just anatomical changes.
The scheduled FDA Type D meeting in March 2025 could accelerate the program's development timeline. Type D meetings typically focus on specific questions about development programs that don't require extensive data review, suggesting Opus is seeking alignment on Phase 3 trial design and endpoints before proceeding with the pivotal study. This proactive regulatory strategy, combined with the University of Pennsylvania partnership, positions Opus strongly in the competitive landscape of genetic ophthalmology therapeutics.
First patient dosed in the pediatric cohort of the Phase 1/2 trial of OPGx-LCA5; initial data on the cohort anticipated by Q3 2025
New 12-month data on the first three adult OPGx-LCA5 patients to be presented at a major medical conference in Q2 2025
FDA meeting scheduled in March 2025 to discuss Phase 3 trial design and registrational endpoints for OPGx-LCA5
DURHAM, N.C., Feb. 18, 2025 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage ophthalmic biotechnology company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and therapies to treat other ophthalmic disorders, today announced that the first pediatric patient was dosed in its ongoing Phase 1/2 clinical trial evaluating OPGx-LCA5, its investigational gene therapy for the treatment of Leber congenital amaurosis (LCA). Opus plans to share initial data from the pediatric cohort by Q3 2025.
The ongoing trial has achieved early clinical proof of concept, demonstrating meaningful visual improvement starting as early as one month following treatment in the first three adult patients, as was evident in the 6-month data first released in October 2024 and reviewed at a company-sponsored KOL event in December 2024 (HERE). Opus now plans to share the new 12-month data on these three adult LCA5 patients at a major medical conference during the second quarter of 2025. An FDA Type D meeting is scheduled in March to discuss the pivotal trial design and endpoints.
“We are proud of the progress of our ongoing OPGx-LCA5 clinical trial and pleased to reach this critical next step as we expand the trial to pediatric patients. Early intervention in pediatric LCA5 patients is particularly important, as it offers the best chance to preserve or restore visual function before the disease progresses,” said George Magrath, M.D., Chief Executive Officer of Opus Genetics. “We are encouraged by the new 12-month results that confirm the durability of the positive response observed at 6 months. This data will be shared in our scheduled meeting with the FDA in March to discuss a proposed Phase 3 trial design and registrational endpoints for the OPGx-LCA5 program. We are hopeful that this may offer a potentially life-changing therapeutic option for individuals living with LCA and will continue to work closely with the medical and patient communities to advance this important program. We’re grateful to our partners at the University of Pennsylvania for their hard work and dedication to our program.”
Phase 1/2 Trial Design
This clinical trial was designed to evaluate the safety and preliminary efficacy of subretinal gene therapy with OPGx-LCA5 in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene. It is an open-label, Phase 1/2 trial evaluating OPGx-LCA5. Efficacy endpoints include measurement of functional vision using: 1) the Multi-Luminance orientation and Mobility Test (MLoMT); 2) Full-Field Stimulus Testing (FST), which measures the retina's sensitivity to light; and 3) microperimetry, which measures point-wise sensitivity to light. For more information, visit clinicaltrials.gov (NCT05616793).
The six-month results on adult patients treated with OPGx-LCA5 were presented in a Key Opinion Leader (KOL) webinar, hosted by Opus on December 11, 2024. A replay of the webinar can be accessed here.
About OPGx-LCA5
OPGx-LCA5 is designed to address a form of Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene (LCA5), which encodes the lebercilin protein. LCA5-associated inherited retinal disease is an early-onset severe inherited retinal dystrophy. Studies in patients with this mutation have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic intervention through gene augmentation. OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. OPGx-LCA5 is currently being evaluated in a Phase 1/2 clinical trial at the University of Pennsylvania designed to evaluate its safety and preliminary efficacy in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene.
About Opus Genetics
Opus Genetics is a clinical-stage ophthalmic biotechnology company developing gene therapies to treat patients with IRDs and other treatments for ophthalmic disorders. The pipeline includes adeno-associated virus (AAV)-based investigational gene therapies that address mutations in genes that cause different forms of bestrophinopathy, Leber congenital amaurosis (LCA) and retinitis pigmentosa. Our most advanced investigational gene therapy program is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein, and is currently being evaluated in a Phase 1/2 open-label trial, with encouraging early data. BEST1 investigational gene therapy is designed to address mutations in the BEST1 gene, which is associated with retinal degeneration; we expect that a Phase 1/2 trial to be initiated in 2025. The pipeline also includes Phentolamine Ophthalmic Solution
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| Nirav Jhaveri CFO ir@opusgtx.com | Corey Davis, Ph.D. LifeSci Advisors cdavis@lifesciadvisors.com |
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