Ocugen Announces Completion of Dosing in Subjects with Stargardt Disease in High Dose Cohort of Phase 1/2 GARDian Clinical Trial of OCU410ST—A Modifier Gene Therapy
Ocugen (NASDAQ: OCGN) has completed dosing in the third cohort of its Phase 1/2 GARDian clinical trial for OCU410ST, a modifier gene therapy for Stargardt disease. The trial, conducted at six U.S. retinal surgery centers, has finished Phase 1 of the dose-escalation portion. Three subjects received the highest dose (2.25×1011 vg/mL) via subretinal injection. The GARDian trial aims to assess safety and efficacy of OCU410ST in Stargardt patients, who currently lack FDA-approved treatments. Dr. Charles Wykoff, lead investigator, noted an encouraging safety and tolerability profile. The trial includes three dose levels, and Ocugen plans to provide periodic clinical updates as it advances this potential one-time treatment option.
Ocugen (NASDAQ: OCGN) ha completato la somministrazione nel terzo gruppo della sua sperimentazione clinica di fase 1/2 GARDian per OCU410ST, una terapia genica modificatrice per la malattia di Stargardt. Lo studio, condotto in sei centri chirurgici retinici negli Stati Uniti, ha terminato la fase 1 della porzione di incremento della dose. Tre soggetti hanno ricevuto la dose più alta (2,25×1011 vg/mL) tramite iniezione subretinica. La sperimentazione GARDian mira a valutare la sicurezza e l'efficacia di OCU410ST nei pazienti affetti da Stargardt, che attualmente non dispongono di trattamenti approvati dalla FDA. Il Dr. Charles Wykoff, responsabile dello studio, ha sottolineato un profilo di sicurezza e tollerabilità incoraggiante. Lo studio include tre livelli di dosaggio e Ocugen prevede di fornire aggiornamenti clinici periodici man mano che avanza in questa potenziale opzione di trattamento una tantum.
Ocugen (NASDAQ: OCGN) ha completado la dosificación en la tercera cohorte de su ensayo clínico de fase 1/2 GARDian para OCU410ST, una terapia génica modificadora para la enfermedad de Stargardt. El ensayo, realizado en seis centros de cirugía retinal en EE. UU., ha finalizado la fase 1 de la porción de escalado de dosis. Tres sujetos recibieron la dosis más alta (2,25×1011 vg/mL) a través de una inyección subretiniana. El ensayo GARDian tiene como objetivo evaluar la seguridad y efectividad de OCU410ST en pacientes con Stargardt, quienes actualmente carecen de tratamientos aprobados por la FDA. El Dr. Charles Wykoff, investigador principal, señaló un perfil de seguridad y tolerabilidad alentador. El ensayo incluye tres niveles de dosis, y Ocugen planea proporcionar actualizaciones clínicas periódicas a medida que avanza en esta potencial opción de tratamiento de una sola vez.
Ocugen (NASDAQ: OCGN)은 Stargardt병에 대한 유전자 치료제인 OCU410ST의 1/2상 GARDian 임상 시험에서 세 번째 그룹의 투약을 완료했습니다. 이 시험은 미국의 여섯 개 망막 수술 센터에서 수행되었으며, 용량 증가 단계의 1단계를 완료했습니다. 세 명의 피험자는 가장 높은 용량(2.25×1011 vg/mL)을 망막 아래 주사로 받았습니다. GARDian 임상 시험은 현재 FDA 승인을 받은 치료법이 없는 Stargardt 환자에서 OCU410ST의 안전성과 효능을 평가하는 것을 목표로 합니다. 수석 연구원인 Charles Wykoff 박사는 고무적인 안전성 및 내약성 프로필을 언급했습니다. 이 시험은 세 가지 용량 단계로 구성되어 있으며, Ocugen은 이 잠재적인 일회성 치료 옵션을 추진하면서 정기적인 임상 업데이트를 제공할 계획입니다.
Ocugen (NASDAQ: OCGN) a terminé l'administration dans la troisième cohorte de son essai clinique de phase 1/2 GARDian pour OCU410ST, une thérapie génique modifiée pour la maladie de Stargardt. L'essai, mené dans six centres de chirurgie rétinienne aux États-Unis, a terminé la phase 1 de la portion d'escalade de dose. Trois sujets ont reçu la dose la plus élevée (2,25×1011 vg/mL) par injection sous-rétinienne. L'essai GARDian vise à évaluer la sécurité et l'efficacité de OCU410ST chez les patients atteints de Stargardt, qui ne disposent actuellement d'aucun traitement approuvé par la FDA. Le Dr Charles Wykoff, investigateur principal, a noté un profil de sécurité et de tolérabilité encourageant. L'essai comprend trois niveaux de dose, et Ocugen prévoit de fournir des mises à jour cliniques périodiques à mesure qu'il fait progresser cette option de traitement potentiellement unique.
Ocugen (NASDAQ: OCGN) hat die Dosierung in der dritten Kohorte seiner Phase 1/2 GARDian-Studie für OCU410ST, eine modifizierende Gentherapie für Stargardt-Krankheit, abgeschlossen. Die Studie, die an sechs retinalen Operationszentren in den USA durchgeführt wird, hat die Phase 1 des Dosissteigerungsabschnitts abgeschlossen. Drei Probanden erhielten die höchste Dosis (2,25×1011 vg/mL) durch subretinale Injektion. Die GARDian-Studie zielt darauf ab, die Sicherheit und Wirksamkeit von OCU410ST bei Stargardt-Patienten zu bewerten, die derzeit keine von der FDA zugelassenen Behandlungen haben. Dr. Charles Wykoff, leitender Prüfer, merkte ein ermutigendes Sicherheits- und Verträglichkeitsprofil an. Die Studie umfasst drei Dosislevels, und Ocugen plant, regelmäßige klinische Updates bereitzustellen, während es diese potenzielle einmalige Behandlungsoption voranbringt.
- Completion of dosing in the high-dose cohort of Phase 1/2 trial for OCU410ST
- Encouraging safety and tolerability profile reported by lead investigator
- Potential to address an unmet medical need for approximately 100,000 Stargardt disease patients in the U.S. and Europe
- OCU410ST could potentially be a one-time treatment option
- None.
Insights
The completion of dosing in the high-dose cohort of Ocugen's Phase 1/2 GARDian clinical trial for OCU410ST marks a significant milestone in the development of a potential treatment for Stargardt disease. This gene therapy approach targets a condition affecting approximately
The trial's progress to the highest dose level (2.25×1011 vg/mL) without reported safety concerns is encouraging. However, it's important to note that while safety and tolerability appear promising, efficacy data are yet to be released. The subretinal injection method could offer a one-time treatment option, potentially revolutionizing Stargardt disease management if proven effective in later trial phases.
From an investment perspective, Ocugen's progress in the OCU410ST clinical trial is a positive development. The completion of Phase 1 dosing brings the company closer to potential commercialization, which could significantly impact its market value. However, investors should remain cautious as the path to FDA approval is still long and uncertain.
The addressable market of
MALVERN, Pa., Aug. 28, 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 GARDian clinical trial for OCU410ST (AAV-hRORA)—a modifier gene therapy candidate being developed for Stargardt disease. Stargardt disease affects approximately 100,000 people in the United States (U.S.) and Europe.
"With all patients dosed in cohort 3 (high dose), Phase 1 of the dose-escalation portion of the trial is complete,” said Dr. Huma Qamar, Chief Medical Officer of Ocugen. “We will continue to advance the trial as efficiently as possible, and work toward fulfilling an unmet medical need for Stargardt patients.”
Three subjects received a single subretinal injection of the highest dose (2.25×1011 vg/mL) being tested. The GARDian clinical trial is being performed at six leading retinal surgery centers across the U.S.
“OCU410ST is a novel modifier gene therapy that has the potential to be a one-time treatment given by subretinal injection in the operating room,” said Charles Wykoff, MD, PhD, Director of Research, Retina Consultants of Texas, lead investigator in the study. “The safety and tolerability profile of OCU410ST remains encouraging as the clinical trial progresses and brings hope to patients with Stargardt disease, who have no FDA-approved treatment options.”
The GARDian clinical trial will assess the safety and efficacy of unilateral subretinal administration of OCU410ST in subjects with Stargardt disease and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose-ranging study consisting of three dose levels [low dose (3.75×1010 vg/mL), medium dose (7.5×1010 vg/mL), and high dose (2.25×1011 vg/mL)].
Ocugen remains committed to advancing treatments for blindness, focusing on innovative gene therapy solutions that aim to provide lasting benefits to patients.
The Company will continue to provide clinical updates on a periodic basis.
About Stargardt Disease
Stargardt disease is a genetic eye disorder that causes retinal degeneration and vision loss. Stargardt disease is the most common form of inherited macular degeneration. The progressive vision loss associated with Stargardt disease is caused by the degeneration of photoreceptor cells in the central portion of the retina called the macula.
Decreased central vision due to loss of photoreceptors in the macula is the hallmark of Stargardt disease. Some peripheral vision is usually preserved. Stargardt disease typically develops during childhood or adolescence, but the age of onset and rate of progression can vary. The retinal pigment epithelium (RPE), a layer of cells supporting photoreceptors, is also affected in people with Stargardt disease.
About OCU410ST
OCU410ST utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. It represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptor (NHR) RORA that regulates pathway links to Stargardt disease such as lipofuscin formation, oxidative stress, compliment formation, inflammation, and cell survival networks.
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 OCU410ST clinical trial for Stargardt disease?
What is the highest dose of OCU410ST tested in the OCGN clinical trial?
How many patients are affected by Stargardt disease in the U.S. and Europe?
What are the dose levels being tested in Ocugen's OCU410ST clinical trial?
Are there any FDA-approved treatments for Stargardt disease currently available?
