Opus Genetics Announces One-Month Clinical Data from Pediatric Patient in Phase 1/2 Trial of OPGx-LCA5 Gene Therapy in Inherited Retinal Diseases
Opus Genetics (Nasdaq: IRD) has reported encouraging one-month clinical data from its first pediatric patient treated with OPGx-LCA5 gene therapy in a Phase 1/2 trial for LCA5-related inherited retinal disease. The 16-year-old patient showed meaningful vision improvement and no drug-related adverse events after a single subretinal injection.
A second pediatric patient has been dosed, with the three-patient pediatric cohort expected to complete enrollment in Q2 2025. Initial data from all pediatric patients is anticipated in Q3 2025. The company previously reported positive six-month results in adult patients, with visual improvements observed in all three adult participants.
Following an FDA Type D meeting, Opus received feedback on its proposed registrational trial design. The company plans to conduct a single-arm, adaptive pivotal study with approximately 19 patients, using the multi-luminance orientation and mobility test (MLoMT) as the primary endpoint. The pivotal trial could potentially begin in Q1 2026.
Opus Genetics (Nasdaq: IRD) ha riportato dati clinici incoraggianti dopo un mese dal trattamento del suo primo paziente pediatrico con la terapia genica OPGx-LCA5 in uno studio di Fase 1/2 per la malattia retinica ereditaria legata a LCA5. Il paziente di 16 anni ha mostrato un significativo miglioramento della vista e non ha presentato eventi avversi correlati al farmaco dopo una singola iniezione sottoretinica.
Un secondo paziente pediatrico è stato trattato, con il gruppo pediatrico di tre pazienti che dovrebbe completare l'arruolamento nel secondo trimestre del 2025. I dati iniziali da tutti i pazienti pediatrici sono attesi nel terzo trimestre del 2025. L'azienda aveva precedentemente riportato risultati positivi a sei mesi nei pazienti adulti, con miglioramenti visivi osservati in tutti e tre i partecipanti adulti.
Dopo un incontro di tipo D con la FDA, Opus ha ricevuto feedback sul design del suo studio registrativo proposto. L'azienda prevede di condurre uno studio pivotale a braccio singolo e adattivo con circa 19 pazienti, utilizzando il test di orientamento e mobilità a multi-luminanza (MLoMT) come endpoint primario. Lo studio pivotale potrebbe potenzialmente iniziare nel primo trimestre del 2026.
Opus Genetics (Nasdaq: IRD) ha informado datos clínicos alentadores de un mes de su primer paciente pediátrico tratado con la terapia génica OPGx-LCA5 en un ensayo de Fase 1/2 para la enfermedad retinal hereditaria relacionada con LCA5. El paciente de 16 años mostró una mejora significativa en la visión y no presentó eventos adversos relacionados con el fármaco después de una única inyección subretiniana.
Se ha administrado tratamiento a un segundo paciente pediátrico, y se espera que el grupo pediátrico de tres pacientes complete la inscripción en el segundo trimestre de 2025. Se anticipan datos iniciales de todos los pacientes pediátricos en el tercer trimestre de 2025. La empresa había informado previamente resultados positivos a seis meses en pacientes adultos, con mejoras visuales observadas en los tres participantes adultos.
Tras una reunión de tipo D con la FDA, Opus recibió comentarios sobre el diseño de su ensayo registrativo propuesto. La empresa planea llevar a cabo un estudio pivotal adaptativo de un solo brazo con aproximadamente 19 pacientes, utilizando la prueba de orientación y movilidad de multi-luminancia (MLoMT) como el objetivo primario. El ensayo pivotal podría comenzar potencialmente en el primer trimestre de 2026.
Opus Genetics (Nasdaq: IRD)는 LCA5 관련 유전 망막 질환에 대한 1/2상 시험에서 OPGx-LCA5 유전자 치료로 치료받은 첫 번째 소아 환자에 대한 한 달간의 고무적인 임상 데이터를 보고했습니다. 16세 환자는 단일 망막하 주사 후 의미 있는 시력 개선을 보였고 약물과 관련된 부작용은 없었습니다.
두 번째 소아 환자가 치료를 받았으며, 세 명의 소아 환자 그룹은 2025년 2분기까지 등록을 완료할 것으로 예상됩니다. 모든 소아 환자로부터의 초기 데이터는 2025년 3분기에 예상됩니다. 회사는 이전에 성인 환자에서 6개월 긍정적인 결과를 보고했으며, 세 명의 성인 참가자 모두에서 시력 개선이 관찰되었습니다.
FDA와의 D형 회의 후, Opus는 제안된 등록 시험 설계에 대한 피드백을 받았습니다. 회사는 약 19명의 환자를 대상으로 단일 팔 적응형 주요 연구를 수행할 계획이며, 다중 조명 방향 및 이동성 테스트(MLoMT)를 주요 목표로 사용할 예정입니다. 주요 시험은 2026년 1분기에 시작될 가능성이 있습니다.
Opus Genetics (Nasdaq: IRD) a rapporté des données cliniques encourageantes d'un mois pour son premier patient pédiatrique traité avec la thérapie génique OPGx-LCA5 dans le cadre d'un essai de Phase 1/2 pour la maladie rétinienne héréditaire liée à LCA5. Le patient de 16 ans a montré une amélioration significative de la vision et aucun événement indésirable lié au médicament après une seule injection sous-rétinienne.
Un deuxième patient pédiatrique a été traité, et le groupe pédiatrique de trois patients devrait compléter son inscription au deuxième trimestre 2025. Les données initiales de tous les patients pédiatriques sont attendues au troisième trimestre 2025. L'entreprise avait précédemment rapporté des résultats positifs à six mois chez des patients adultes, avec des améliorations visuelles observées chez les trois participants adultes.
Suite à une réunion de type D avec la FDA, Opus a reçu des commentaires sur le design de son essai d'enregistrement proposé. L'entreprise prévoit de réaliser une étude pivotale adaptative à bras unique avec environ 19 patients, utilisant le test d'orientation et de mobilité à multi-luminance (MLoMT) comme critère principal. L'essai pivot pourrait potentiellement commencer au premier trimestre 2026.
Opus Genetics (Nasdaq: IRD) hat ermutigende klinische Daten von einem Monat für seinen ersten pädiatrischen Patienten veröffentlicht, der im Rahmen einer Phase-1/2-Studie mit der OPGx-LCA5 Gentherapie gegen LCA5-bedingte erbliche Netzhauterkrankungen behandelt wurde. Der 16-jährige Patient zeigte eine signifikante Verbesserung des Sehvermögens und keine arzneimittelbedingten unerwünschten Ereignisse nach einer einzigen subretinalen Injektion.
Ein zweiter pädiatrischer Patient wurde behandelt, und die dreiköpfige pädiatrische Kohorte wird voraussichtlich im 2. Quartal 2025 abgeschlossen sein. Erste Daten von allen pädiatrischen Patienten werden im 3. Quartal 2025 erwartet. Das Unternehmen hatte zuvor positive Ergebnisse nach sechs Monaten bei erwachsenen Patienten berichtet, wobei bei allen drei erwachsenen Teilnehmern Sehverbesserungen beobachtet wurden.
Nach einem FDA-Typ-D-Meeting erhielt Opus Feedback zu seinem vorgeschlagenen Registrierungsstudien-Design. Das Unternehmen plant, eine einarmige, adaptive Pivot-Studie mit etwa 19 Patienten durchzuführen, wobei der Multi-Luminanz-Orientierungs- und Mobilitätstest (MLoMT) als primärer Endpunkt verwendet wird. Die Pivot-Studie könnte potenziell im 1. Quartal 2026 beginnen.
- First pediatric patient showed meaningful vision improvement with no drug-related adverse events
- All three adult patients demonstrated visual improvements at 6 months, with efficacy lasting up to one year
- FDA provided constructive feedback for registrational trial design
- Potential for streamlined pivotal trial with only 19 patients
- Additional information requested by FDA before trial approval
- Pivotal trial initiation not expected until Q1 2026
- Small patient sample size in current trial phases
Insights
Opus Genetics' one-month data from their first pediatric patient provides encouraging early signals for their OPGx-LCA5 gene therapy program. The reported visual improvements - notably the patient's ability to distinguish letters and navigate independently - represent potentially meaningful functional benefits in this rare genetic condition that currently has no approved treatments.
The consistency between pediatric and adult responses suggests a reproducible therapeutic effect across age groups, which is critical for genetic disorders like LCA5. Particularly notable is the preliminary evidence that efficacy in adult patients has persisted for a full year, indicating potential durability of treatment effect.
The FDA Type D meeting outcomes appear constructive, with the agency engaging on Opus's proposed streamlined registration pathway using just 19 patients. The MLoMT primary endpoint (a VR version of the previously FDA-accepted MLMT) represents a pragmatic functional outcome measure for a vision restoration therapy. However, the FDA's request for additional statistical and CMC information signals some regulatory hurdles remain before the planned 2026 pivotal trial.
While these early results from a single pediatric patient are promising, the upcoming Q3 2025 readout from all three pediatric subjects will provide a more substantive efficacy signal. This is still an early-stage program with significant development milestones ahead before potential commercialization.
The clinical improvements reported in this LCA5 patient represent potentially significant therapeutic benefits in a devastating genetic blindness condition. The subjective report that "objects were significantly brighter" suggests improved photoreceptor function, while the ability to "distinguish letters and navigate with independence" points to meaningful functional vision restoration.
LCA5 mutations affect the lebercilin protein, essential for photoreceptor maintenance. The disease's progressive nature makes the early intervention in pediatric patients particularly important, as suggested by Dr. Magrath. Treating before extensive photoreceptor loss occurs theoretically provides the best chance for functional improvement.
The safety profile appears favorable with no drug-related adverse events reported. This is crucial for subretinal gene therapies, where procedure-related complications are always a concern.
The use of multiple complementary assessment methods - MLoMT for functional vision/mobility, FST for light sensitivity, and microperimetry for point sensitivity - creates a comprehensive efficacy evaluation framework. The planned virtual reality MLoMT endpoint for the pivotal trial represents an evolution of methodology previously accepted by regulators.
While these early results merit cautious optimism, longer follow-up across more patients is essential to determine if the therapy can truly modify disease progression in LCA5, particularly given the neurodegenerative component of many inherited retinal diseases.
First pediatric patient shows encouraging early safety profile and meaningful improvement in visual function at one month
A second pediatric patient was recently dosed, and the pediatric cohort is expected to complete enrollment in Q2 2025; initial data from all three patients anticipated in Q3 2025
FDA Type D meeting provided clarity on next steps for a registrational trial design to support BLA submission, with potential trial initiation in 2026
DURHAM, N.C., April 08, 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 to treat other ophthalmic disorders (“Opus” or the “Company”), today announced one-month clinical data from the first pediatric patient treated with its investigational gene therapy, OPGx-LCA5, in a Phase 1/2 open-label trial for LCA5-related inherited retinal disease (IRD). The new data builds upon previously reported positive results from adult patients treated in the same study.
“The preliminary results observed in our pediatric patient are encouraging and are consistent with the improvements we previously observed in adults,” said Tomas Aleman, M.D., Scheie Eye Institute Perelman, the study Principal Investigator. “She noticed that objects were significantly brighter and was able to distinguish letters and navigate with an independence she had never had before, after only one-month following treatment. We’re excited to continue enrolling patients and studying improvement over longer periods of time in this important study.”
George Magrath, M.D., Chief Executive Officer of Opus Genetics added “We believe these findings provided further evidence supporting the potential of OPGx-LCA5 to restore meaningful vision in patients affected by LCA5. The meeting with the FDA regarding the design of our program may lead to the start of a trial in 2026. Early intervention may be particularly beneficial in pediatric patients, given the progressive nature of this disease.”
OPGx-LCA5 is currently being evaluated in a Phase 1/2 trial in adult and pediatric patients with inherited retinal degeneration due to mutations in the LCA5 gene. The trial is progressing and began enrolling a cohort of three pediatric patients in February 2025. Initial data from this pediatric cohort are expected in the third quarter of 2025. LCA5 is a rare and severe genetic disorder that leads to early-onset vision loss due to mutations in the LCA5 gene, which encodes lebercilin, a protein essential for photoreceptor function. There are no approved therapies for LCA5-related IRD to date, making gene therapy a potentially transformative approach.
The first pediatric participant, aged 16 (at time of consent), received a single subretinal injection of OPGx-LCA5 and clinically meaningful improvement in vision was observed at one-month post treatment. In addition to these promising early efficacy signals, there have been no observed drug related adverse events reported to date. These data are preliminary, and we expect to be able to read out efficacy data for all three pediatric patients in the third quarter of 2025.
In the ongoing Phase 1/2 trial, we have observed early clinical proof of concept in adult patients. In previously announced results for OPGx-LCA5, we observed visual improvement in all 3 of the adult patients at six months. New one-year data from the study, to be presented at the Association for Research in Vision and Ophthalmology (ARVO) 2025 Meeting, on May 4, 2025 provide preliminary evidence that both subjective and objective signs of efficacy in these adult patients persisted for a year.
Next Steps for OPGx-LCA5 Following FDA Type D Meeting
An FDA meeting was held to discuss the potential regulatory path for OPGx LCA5, including the design of a potential registrational study. Opus proposed a single arm, adaptive pivotal study, to enroll as few as 19 patients, with a primary endpoint utilizing the multi-luminance orientation and mobility test (MLoMT), which is a functional vision and patient mobility test. MLoMT is a virtual-reality version of the multi-luminance mobility test (MLMT), which provided evidence to support a prior FDA approval. The company also received constructive feedback on its proposed statistical analysis plan (SAP) as well as chemistry, manufacturing, and controls (CMC). The FDA requested additional information on these topics, and Opus plans to submit further materials and continue discussions with the FDA. Opus anticipates the pivotal trial could initiate in Q1 2026.
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. The trial has been enrolling both adult and pediatric patients. Dosing of the first pediatric patients began in February 2025. 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 copy of the presentation from 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 biopharmaceutical company developing therapies to treat patients with inherited retinal diseases (IRDs) and other treatments for ophthalmic disorders. Our 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, dose-escalation trial, with encouraging early data. Our pipeline also includes BEST1 investigational gene therapy, designed to address mutations in the BEST1 gene, which is associated with retinal degeneration. The pipeline also includes Phentolamine Ophthalmic Solution
Forward Looking Statements
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- Data reported in this press release is preliminary and related to one patient, and, as a result, data that initially appears promising may be revised, updated, or invalidated at a later data readout and/or may ultimately not be capable of duplication in additional patients;
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Contacts
| Corporate | Investor Relations |
| Nirav Jhaveri CFO ir@opusgtx.com | Corey Davis, Ph.D. LifeSci Advisors cdavis@lifesciadvisors.com |
FAQ
What are the early results of Opus Genetics' OPGx-LCA5 gene therapy trial in pediatric patients?
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How effective was OPGx-LCA5 in adult patients during the Phase 1/2 trial?
