Ocugen, Inc. Announces Completion of Dosing in Subjects with Geographic Atrophy Secondary to dAMD in High-Dose Cohort of Phase 1/2 ArMaDa Clinical Trial of OCU410—A Novel Modifier Gene Therapy
Ocugen, Inc. (NASDAQ: OCGN) has completed dosing in the high-dose cohort of its Phase 1/2 ArMaDa clinical trial for OCU410, a novel modifier gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD). The trial, conducted at 14 leading retinal surgery centers in the US, has finished the Phase 1 stage with three subjects receiving the highest dose. OCU410 aims to provide a one-time treatment option for GA, which affects approximately 1 million people in the United States.
The company has now initiated a Phase 2 clinical trial to assess the safety and efficacy of OCU410 in a larger patient group, randomized into medium-dose, high-dose, or control groups. This novel approach could potentially transform GA treatment, addressing limitations of current FDA-approved therapies that require continuous intravitreal injections over several years.
Ocugen, Inc. (NASDAQ: OCGN) ha completato la somministrazione nel gruppo ad alta dose del suo studio clinico di Fase 1/2 ArMaDa per OCU410, una nuova terapia genica modificatrice per l'atrofia geografica (GA) secondaria alla degenerazione maculare senile secca (dAMD). Lo studio, condotto in 14 centri chirurgici retinici leader negli USA, ha terminato la fase 1 con tre soggetti che hanno ricevuto la dose più alta. OCU410 mira a fornire un'opzione di trattamento una tantum per la GA, che colpisce circa 1 milione di persone negli Stati Uniti.
L'azienda ha ora intrapreso uno studio clinico di Fase 2 per valutare la sicurezza e l'efficacia di OCU410 in un gruppo di pazienti più ampio, randomizzato in gruppi a dose media, alta dose o gruppo di controllo. Questo approccio innovativo potrebbe trasformare il trattamento della GA, affrontando le limitazioni delle attuali terapie approvate dalla FDA che richiedono iniezioni intravitreali continue per diversi anni.
Ocugen, Inc. (NASDAQ: OCGN) ha completado la administración en el grupo de alta dosis de su ensayo clínico de fase 1/2 ArMaDa para OCU410, una nueva terapia génica modificadora para la atrofia geográfica (GA) secundaria a la degeneración macular relacionada con la edad seca (dAMD). El ensayo, realizado en 14 centros líderes de cirugía retiniana en EE. UU., ha finalizado la fase 1 con tres sujetos que recibieron la dosis más alta. OCU410 busca proporcionar una opción de tratamiento única para la GA, que afecta a aproximadamente 1 millón de personas en los Estados Unidos.
La compañía ha iniciado un ensayo clínico de fase 2 para evaluar la seguridad y eficacia de OCU410 en un grupo de pacientes más grande, randomizado en grupos de dosis media, alta dosis o control. Este enfoque novedoso podría transformar el tratamiento de la GA, abordando las limitaciones de las terapias actualmente aprobadas por la FDA que requieren inyecciones intravítreas continuas durante varios años.
Ocugen, Inc. (NASDAQ: OCGN)는 OCU410의 임상 1/2단계 ArMaDa 임상 시험에서 고용량 집단의 투약을 완료했습니다. OCU410은 건성 연령 관련 황반 변성을 통한 이차적 지리적 위축(GA)에 대한 새로운 조정 유전자 치료법입니다. 이 시험은 미국의 14개 주요 망막 수술 센터에서 진행되었으며, 가장 높은 용량을 받은 3명의 피험자가 포함된 1단계가 완료되었습니다. OCU410은 GA에 대한 일회성 치료 옵션을 제공하는 것을 목표로 합니다. GA는 미국에서 약 100만 명에게 영향을 미칩니다.
회사는 이제 OCU410의 안전성과 유효성을 평가하기 위한 2단계 임상 시험을 시작했습니다. 이 임상 시험은 중간 용량, 고용량 또는 대조군으로 무작위 배정된 더 큰 환자 집단에서 진행됩니다. 이 혁신적인 접근법은 GA 치료를 변화시킬 수 있으며, 몇 년에 걸쳐 지속적인 유리체 내 주사를 필요로 하는 현재 FDA 승인 치료법의 한계를 극복할 수 있습니다.
Ocugen, Inc. (NASDAQ: OCGN) a achevé l'administration dans le groupe à forte dose de son essai clinique de Phase 1/2 ArMaDa pour OCU410, une nouvelle thérapie génique modificateur pour l'atrophie géographique (GA) secondaire à la dégénérescence maculaire liée à l'âge sèche (dAMD). L'essai, mené dans 14 centres de chirurgie rétinienne de premier plan aux États-Unis, a terminé la phase 1 avec trois sujets ayant reçu la dose la plus élevée. OCU410 vise à fournir une option de traitement unique pour la GA, qui touche environ 1 million de personnes aux États-Unis.
L'entreprise a maintenant initié un essai clinique de Phase 2 pour évaluer la sécurité et l'efficacité d'OCU410 dans un groupe de patients plus large, randomisé en groupes à dose moyenne, à forte dose ou en groupe témoin. Cette approche novatrice pourrait transformer le traitement de la GA, en s'attaquant aux limites des thérapies actuellement approuvées par la FDA qui nécessitent des injections intravitréennes continues pendant plusieurs années.
Ocugen, Inc. (NASDAQ: OCGN) hat die Dosierung in der Hochdosisgruppe seiner Phase 1/2 ArMaDa-Studie für OCU410 abgeschlossen, eine neuartige modifizierende Gentherapie zur Behandlung der geografischen Atrophie (GA), die sekundär zur trockenen altersbedingten Makuladegeneration (dAMD) auftritt. Die Studie wurde an 14 führenden retinalen chirurgischen Zentren in den USA durchgeführt und hat die Phase-1-Phase mit drei Probanden abgeschlossen, die die höchste Dosis erhalten haben. OCU410 zielt darauf ab, eine einmalige Behandlungsoption für GA bereitzustellen, die etwa 1 Million Menschen in den Vereinigten Staaten betrifft.
Das Unternehmen hat nun eine Phase-2-Studie gestartet, um die Sicherheit und Wirksamkeit von OCU410 in einer größeren Patientenpopulation zu bewerten, die in Gruppen mit mittlerer Dosis, hoher Dosis oder Kontrollgruppen randomisiert wurde. Dieser neuartige Ansatz könnte die Behandlung von GA revolutionieren, indem er die Begrenzungen der derzeit von der FDA zugelassenen Therapien angeht, die über mehrere Jahre hinweg kontinuierliche intravitrealen Injektionen erfordern.
- Completion of Phase 1 dosing for OCU410 gene therapy
- Initiation of Phase 2 clinical trial for OCU410
- Potential for OCU410 to be a one-time treatment for geographic atrophy
- Addressing limitations of current GA treatments that require multiple injections
- None.
Insights
The completion of dosing in the high-dose cohort of Ocugen's Phase 1/2 ArMaDa clinical trial for OCU410 marks a significant milestone in the development of a novel gene therapy for geographic atrophy (GA). This news is particularly noteworthy for several reasons:
- OCU410 is targeting a large market, with GA affecting approximately
1 million people in the United States. - The therapy aims to address limitations of current FDA-approved treatments, which provide benefits and require frequent injections.
- As a one-time treatment, OCU410 could potentially revolutionize GA management if proven effective.
The initiation of the Phase 2 trial is a important next step, as it will provide more robust data on safety and efficacy. However, investors should remain cautious, as gene therapies often face challenges in later-stage trials and regulatory approvals. The company's commitment to periodic updates will be vital for tracking progress and assessing the therapy's potential market impact.
From a financial perspective, Ocugen's progress with OCU410 presents a potentially significant opportunity:
- The GA market is substantial, with current treatments showing limitations. A successful one-time gene therapy could command premium pricing and capture significant market share.
- Advancing to Phase 2 suggests confidence in the therapy's safety profile, potentially de-risking the development process somewhat.
- However, it's important to note that Ocugen is still in the clinical stage, without approved products generating revenue. The company will likely continue to incur substantial R&D expenses.
Investors should consider:
- Ocugen's cash position and burn rate to ensure sufficient funding for completing clinical trials.
- The competitive landscape, including other emerging therapies for GA.
- The potential timeline to market, considering the typically lengthy development and approval process for gene therapies.
• Initiated Phase 2 clinical trial
MALVERN, Pa., July 25, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced that dosing is complete in the third cohort of its Phase 1/2 ArMaDa clinical trial for OCU410 (AAV-hRORA)—a novel modifier gene therapy candidate being developed for geographic atrophy (GA), an advanced stage of dry age-related macular degeneration (dAMD). GA affects approximately 1 million people in the United States.
“Currently, there are two FDA approved anti-complement therapies for GA, which targets only one pathway for a disease that has a multifactorial and complex etiology,” said Majda Hadziahmetovic, MD, Associate Professor of Ophthalmology, Vitreoretinal Diseases and Surgery, Duke University Eye Center, and the lead investigator for the OCU410 Phase 1/2 trial. “Though these treatments are a significant milestone, they provide limited benefits and involve a continuous regimen of multiple intravitreal injections over several years. This modifier gene therapy could potentially transform the landscape of GA treatment.”
Three subjects received a single subretinal administration of the highest dose (200 µL of 1.5x1011 vg/mL) being tested, which completed the dosing for the Phase 1 stage of the trial. The ArMaDa clinical trial for OCU410 is being performed at 14 leading retinal surgery centers across the United States.
“We are very encouraged about the potential of OCU410 as a one-time treatment option for GA,” said Dr. Huma Qamar, Chief Medical Officer of Ocugen.
We have initiated a Phase 2 clinical trial that will assess the safety and efficacy of OCU410 in a larger group of patients who will be randomized into either of two treatment groups (medium- or high-dose) or a control group.
The Company will continue to provide clinical updates on a periodic basis.
About the Phase 1/2 ArMaDa clinical trial
The ArMaDa Phase 1/2 clinical trial will assess the safety of unilateral subretinal administration of OCU410 in subjects with geographic atrophy and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose-escalation study consisting of three dose levels [low dose (2.5×1010 vg/mL), medium dose (5×1010 vg/mL), and high dose (1.5 ×1011 vg/mL)]. Phase 2 is a randomized, outcome accessor-blinded, dose-expansion study in which subjects will be randomized in a 1:1:1 ratio to either one of two OCU410 treatment groups or to an untreated control group.
About dry age-related macular degeneration (dAMD) and geographic atrophy (GA)
Dry age-related macular degeneration (dAMD) affects approximately 10 million Americans and more than 266 million people worldwide. It is characterized by the thinning of the macula, the portion of the retina responsible for clear vision in one’s direct line of sight. dAMD involves the slow deterioration of the retina with submacular drusen (small white or yellow dots on the retina), atrophy, loss of macular function, and central vision impairment. dAMD accounts for 85
About OCU410
OCU410 utilizes an adeno-associated virus (AAV) platform for the retinal delivery of the RORA (ROR Related Orphan Receptor A) gene. The RORA protein plays an important role in lipid metabolism, reducing lipofuscin deposits and oxidative stress, and demonstrates an anti-inflammatory role as well as inhibiting the complement system in both in vitro and in vivo (animal model) studies. These results demonstrate the ability of OCU410 to target multiple pathways linked with dAMD pathophysiology. Ocugen is developing AAV-RORA as a one-time gene therapy for the treatment of geographic atrophy.
About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patients’ lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.
Contact:
Tiffany Hamilton
Head of Communications
Tiffany.Hamilton@ocugen.com
FAQ
What is the current status of Ocugen's OCU410 clinical trial for geographic atrophy (OCGN)?
How many subjects received the highest dose of OCU410 in Ocugen's Phase 1 trial (OCGN)?
What is the potential advantage of Ocugen's OCU410 over current GA treatments (OCGN)?
How many people in the United States are affected by geographic atrophy, the condition targeted by Ocugen's OCU410 (OCGN)?
