Data and Safety Monitoring Board Approves Initiation of Phase 2 of OCU410ST GARDian Clinical Trial for Stargardt Disease
Ocugen, Inc. (NASDAQ: OCGN) announced that the Data and Safety Monitoring Board (DSMB) has approved enrollment for the second phase of the Phase 1/2 clinical trial for OCU410ST, a novel modifier gene therapy candidate for Stargardt disease. The DSMB determined the high dose of OCU410ST to be the maximum tolerated dose, with no serious adverse events reported. The trial will proceed to Phase 2 using high and medium doses.
The first phase enrolled nine patients receiving low, medium, or high doses of OCU410ST via subretinal injection. Dr. Charles Wykoff, a lead study investigator, noted that OCU410ST appears to be safe and well-tolerated. Ocugen's Chief Medical Officer, Dr. Huma Qamar, expressed enthusiasm about OCU410ST's potential to be the first one-time novel modifier gene therapy for Stargardt disease, addressing an unmet medical need for an estimated 100,000 patients in the U.S. and Europe.
Ocugen, Inc. (NASDAQ: OCGN) ha annunciato che il Comitato per il Monitoraggio dei Dati e della Sicurezza (DSMB) ha approvato l'arruolamento per la seconda fase della sperimentazione clinica di Fase 1/2 per OCU410ST, un candidato innovativo di terapia genica modificatrice per la malattia di Stargardt. Il DSMB ha stabilito che la dosi elevata di OCU410ST è la massima dose tollerata, senza eventi avversi gravi segnalati. Lo studio procederà alla Fase 2 utilizzando dosi elevate e medie.
La prima fase ha arruolato nove pazienti che ricevevano dosi basse, medie o elevate di OCU410ST tramite iniezione subretinica. Il Dr. Charles Wykoff, un investigatore principale dello studio, ha osservato che OCU410ST sembra essere sicuro e ben tollerato. Il Direttore Medico di Ocugen, Dr. Huma Qamar, ha espresso entusiasmo per il potenziale di OCU410ST di essere la prima terapia genica modificatrice innovativa somministrata una sola volta per la malattia di Stargardt, rispondendo a un bisogno medico non soddisfatto per un numero stimato di 100.000 pazienti negli Stati Uniti e in Europa.
Ocugen, Inc. (NASDAQ: OCGN) anunció que el Comité de Monitoreo de Datos y Seguridad (DSMB) ha aprobado la inscripción para la segunda fase del ensayo clínico de Fase 1/2 para OCU410ST, un candidato de terapia génica modificadora novedosa para la enfermedad de Stargardt. El DSMB determinó que la dosis alta de OCU410ST es la dosis máxima tolerada, sin eventos adversos graves reportados. El ensayo avanzará a la Fase 2 utilizando dosis altas y medias.
La primera fase inscribió a nueve pacientes que recibieron dosis bajas, medias o altas de OCU410ST mediante inyección subretiniana. El Dr. Charles Wykoff, un investigador principal del estudio, señaló que OCU410ST parece ser seguro y bien tolerado. La Directora Médica de Ocugen, Dra. Huma Qamar, expresó entusiasmo por el potencial de OCU410ST de ser la primera terapia génica modificadora novedosa administrada una sola vez para la enfermedad de Stargardt, abordando una necesidad médica no satisfecha para un estimado de 100,000 pacientes en EE. UU. y Europa.
Ocugen, Inc. (NASDAQ: OCGN)는 데이터 및 안전 모니터링 위원회(DSMB)가 OCU410ST에 대한 제1/2상 임상 시험의 두 번째 단계에 대한 참여를 승인했다고 발표했습니다. OCU410ST는 스타가르트병에 대한 새로운 조절 유전자 치료 후보입니다. DSMB는 OCU410ST의 고용량이 최대 허용 용량이라고 판단했으며 심각한 부작용은 보고되지 않았습니다. 이 시험은 고용량 및 중간 용량을 사용하여 2상으로 진행됩니다.
첫 번째 단계에서는 아홉 명의 환자가 피하주사를 통해 OCU410ST의 저용량, 중용량 또는 고용량을 받았습니다. 주 연구자인 찰스 와이코프 박사는 OCU410ST가 안전하고 잘 견딜 수 있는 것으로 보인다고 언급했습니다. Ocugen의 최고 의학 책임자인 휴마 카마르 박사는 OCU410ST가 스타가르트병을 위한 최초의 일회성 신약 조절 유전자 치료제가 될 잠재력에 대해 열정을 표명했습니다고 하며, 이는 미개선 의료 요구를 충족시켜 줄 것입니다. 미국과 유럽에서 약 100,000명의 환자를 위한 것입니다.
Ocugen, Inc. (NASDAQ: OCGN) a annoncé que le Comité de Surveillance des Données et de la Sécurité (DSMB) a approuvé l'inscription pour la deuxième phase de l'essai clinique de Phase 1/2 pour OCU410ST, un candidat de thérapie génique modératrice novateur pour la maladie de Stargardt. Le DSMB a déterminé que la dose élevée d'OCU410ST est la dose maximale tolérée, sans événements indésirables graves signalés. L'essai passera à la Phase 2 en utilisant des doses élevées et moyennes.
La première phase a inscrit neuf patients recevant des doses faibles, moyennes ou élevées d'OCU410ST par injection subrétinienne. Le Dr Charles Wykoff, un enquêteur principal de l'étude, a noté qu'OCU410ST semble être sûr et bien toléré. Le Directeur Médical d'Ocugen, Dr Huma Qamar, a exprimé son enthousiasme quant au potentiel d'OCU410ST d'être la première thérapie génique modératrice novatrice administrée une seule fois pour la maladie de Stargardt, répondant à un besoin médical non satisfait pour environ 100 000 patients aux États-Unis et en Europe.
Ocugen, Inc. (NASDAQ: OCGN) gab bekannt, dass die Data and Safety Monitoring Board (DSMB) die Einschreibung für die zweite Phase der Phase 1/2 klinischen Studie für OCU410ST genehmigt hat, einen neuartigen modifizierenden Gentherapie-Kandidaten für die Stargardt-Krankheit. Die DSMB stellte fest, dass die hohe Dosis von OCU410ST die maximal tolerierte Dosis ist, ohne dass schwerwiegende unerwünschte Ereignisse gemeldet wurden. Die Studie wird mit hohen und mittleren Dosen in die Phase 2 übergehen.
In der ersten Phase wurden neun Patienten eingeschrieben, die niedrige, mittlere oder hohe Dosen von OCU410ST durch subretinale Injektion erhielten. Dr. Charles Wykoff, ein leitender Studieninvestigator, bemerkte, dass OCU410ST sicher und gut verträglich zu sein scheint. Ocugens Chief Medical Officer, Dr. Huma Qamar, äußerte Begeisterung über das Potenzial von OCU410ST, die erste einmalig verabreichte neuartige modifizierende Gentherapie für die Stargardt-Krankheit zu sein, und deckt einen unerfüllten medizinischen Bedarf für schätzungsweise 100.000 Patienten in den USA und Europa.
- DSMB approved proceeding to Phase 2 of the clinical trial
- No serious adverse events reported in Phase 1
- High dose of OCU410ST determined to be the maximum tolerated dose
- OCU410ST shows promising safety and tolerability profile
- None.
Insights
The DSMB's approval to initiate Phase 2 of the OCU410ST GARDian clinical trial for Stargardt disease is a significant milestone for Ocugen. Key points:
- No serious adverse events reported, indicating good safety profile
- High dose (2.25 x
10^11 vg/mL) determined as maximum tolerated dose - Phase 2 to proceed with high and medium doses
- Potential to be the first FDA-approved treatment for Stargardt disease
This progress is crucial for Ocugen's pipeline and addresses an unmet medical need for an estimated
This clinical progress is positive for Ocugen's financial outlook, albeit with caveats:
- Advancing to Phase 2 reduces development risk and potentially shortens time to market
- Addresses a sizeable market of
100,000 patients in the U.S. and Europe - As a potential first-in-class therapy, OCU410ST could command premium pricing if approved
- No mention of partnerships or funding, suggesting Ocugen bears full development costs and risks
With a market cap of
- Determined the high dose of OCU410ST to be the maximum tolerated dose
- No serious adverse events have been reported
- Approved proceeding to Phase 2 using high and medium doses of OCU410ST
MALVERN, Pa., Oct. 22, 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, biologics, and vaccines, today announced that the Data and Safety Monitoring Board (DSMB) for the OCU410ST GARDian clinical trial recently convened and approved enrollment for the second phase of the Phase 1/2 clinical trial. OCU410ST (AAV5-hRORA) is a novel modifier gene therapy candidate being developed for Stargardt disease.
“The DSMB has recommended moving forward with Phase 2 enrollment, as safety data indicates that OCU410ST appears to be safe and well-tolerated to date,” said Charles Wykoff, MD, PhD, Director of Research, Retina Consultants of Texas & Retina Consultants of America, and a lead study investigator. “The safety and tolerability profile of OCU410ST remains encouraging as the clinical trial has progressed and continues to bring hope to patients with Stargardt disease, which still has no FDA-approved treatments.”
The first phase of the Phase 1/2 clinical trial was an open-label, dose-ranging study that enrolled nine patients to receive either a low (3.75 x 1010 vg/mL), medium (7.5 x 1010 vg/mL), or high (2.25 x 1011 vg/mL) dose of OCU410ST administered via subretinal injection. No serious adverse events (SAEs) have been reported, and the DSMB determined the high dose to be the maximum tolerated dose (MTD).
Stargardt disease is the most common form of inherited macular dystrophy. Symptoms of bilateral central vision loss typically begin in childhood and gradually worsen over time.
“We are enthusiastic about the potential of OCU410ST to be the first one-time novel modifier gene therapy for Stargardt disease,” said Huma Qamar, MD, MPH, Chief Medical Officer of Ocugen. “We are encouraged by the prospect of addressing a substantial unmet medical need for the estimated 100,000 Stargardt patients in the U.S. and Europe.”
The GARDian clinical trial is currently being performed at 6 leading retinal surgery centers across the U.S.
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, complement 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, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patient’s 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.
Cautionary Note on 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, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, the ability to initiate new clinical programs; 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 annual and 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
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