Ocugen, Inc. Announces Removal of Clinical Hold on Investigational New Drug Application for OCU200 Phase 1 Clinical Trial
Ocugen, Inc. (NASDAQ: OCGN) announced that the FDA has lifted the clinical hold on the investigational new drug application for the Phase 1 clinical trial of OCU200, a recombinant fusion protein designed to treat diabetic macular edema (DME). OCU200 targets the integrin pathway and could potentially benefit all DME patients, including those unresponsive to current anti-VEGF therapies.
The trial is a multicenter, open-label, dose-ranging study with three cohorts in the dose-escalation portion and a fourth cohort combining OCU200 with anti-VEGF therapy. DME affects approximately 746,000 people in the United States. Ocugen plans to explore additional indications for OCU200, including diabetic retinopathy and wet age-related macular degeneration, which collectively impact nearly nine million Americans.
Ocugen, Inc. (NASDAQ: OCGN) ha annunciato che la FDA ha sollevato il blocco clinico sulla domanda di nuovo farmaco investigativo per il trial clinico di Fase 1 di OCU200, una proteina ricombinante progettata per trattare l'edema maculare diabetico (DME). OCU200 agisce sul percorso integrinico e potrebbe potenzialmente beneficiare tutti i pazienti affetti da DME, compresi quelli non responsivi alle attuali terapie anti-VEGF.
Lo studio è un trial multicentrico, aperto, a dose variabile con tre coorti nella fase di incremento della dose e una quarta coorte che combina OCU200 con terapia anti-VEGF. Il DME colpisce circa 746.000 persone negli Stati Uniti. Ocugen prevede di esplorare ulteriori indicazioni per OCU200, inclusa la retinopatia diabetica e la degenerazione maculare senile umida, che colpiscono collettivamente quasi nove milioni di americani.
Ocugen, Inc. (NASDAQ: OCGN) anunció que la FDA ha levantado la suspensión clínica de la solicitud de nuevo fármaco en investigación para el ensayo clínico de Fase 1 de OCU200, una proteína de fusión recombinante diseñada para tratar el edema macular diabético (DME). OCU200 se dirige a la vía de las integrinas y podría beneficiar potencialmente a todos los pacientes con DME, incluidos aquellos que no responden a las terapias anti-VEGF actuales.
El ensayo es un estudio multicéntrico, abierto y de rango de dosis con tres cohortes en la parte de escalado de dosis y una cuarta cohorte que combina OCU200 con terapia anti-VEGF. El DME afecta a aproximadamente 746,000 personas en los Estados Unidos. Ocugen planea explorar indicaciones adicionales para OCU200, incluyendo la retinopatía diabética y la degeneración macular relacionada con la edad húmeda, que en conjunto impactan a casi nueve millones de estadounidenses.
Ocugen, Inc. (NASDAQ: OCGN)는 FDA가 OCU200의 1상 임상 시험을 위한 새로운 의약품 신청에 대해 임상 보류를 해제했다고 발표했습니다. OCU200은 당뇨 황반 부종(DME) 치료를 위해 설계된 재조합 융합 단백질입니다. OCU200은 인테그린 경로를 표적하며 현재의 항-VEGF 치료제에 반응하지 않는 환자들을 포함하여 모든 DME 환자에게 혜택을 줄 가능성이 있습니다.
이 시험은 다기관 공개 용량 범위 연구로서, 용적 세분화에 세 가지 집단과 OCU200을 항-VEGF 요법과 결합한 네 번째 집단이 포함됩니다. DME는 미국에서 약 746,000명에게 영향을 미칩니다. Ocugen은 OCU200의 추가 적응증, 즉 당뇨망막병증 및 습성 노인성 황반변성을 탐색할 계획을 세우고 있으며, 이는 총 900만 명의 미국인에게 영향을 미칩니다.
Ocugen, Inc. (NASDAQ: OCGN) a annoncé que la FDA a levé la suspension clinique de la demande de nouveau médicament expérimental pour l'essai clinique de Phase 1 d'OCU200, une protéine recombinante conçue pour traiter l'œdème maculaire diabétique (DME). OCU200 cible la voie des intégrines et pourrait potentiellement bénéficier à tous les patients atteints de DME, y compris ceux qui ne répondent pas aux thérapies anti-VEGF actuelles.
L'essai est une étude multicentrique, en ouvert et à doses progressives avec trois cohortes dans la phase d'escalade de dose et une quatrième cohorte combinant OCU200 avec une thérapie anti-VEGF. Le DME affecte environ 746 000 personnes aux États-Unis. Ocugen prévoit d'explorer d'autres indications pour OCU200, y compris la rétinopathie diabétique et la dégénérescence maculaire liée à l'âge humide, qui touchent collectivement près de neuf millions d'Américains.
Ocugen, Inc. (NASDAQ: OCGN) kündigte an, dass die FDA die klinische Sperre aufgehoben hat für den Antrag auf ein neues, investigatives Arzneimittel für die Phase-1-Studie von OCU200, einem rekombinanten Fusionsprotein zur Behandlung von diabetischem Makulaödem (DME). OCU200 zielt auf den Integrinweg ab und könnte potenziell allen DME-Patienten, einschließlich derjenigen, die auf aktuelle Anti-VEGF-Therapien nicht ansprechen, zugutekommen.
Die Studie ist eine multizentrische, offene Dosisfindungsstudie mit drei Kohorten in der Dosissteigerung und einer vierten Kohorte, die OCU200 mit einer Anti-VEGF-Therapie kombiniert. DME betrifft in den Vereinigten Staaten etwa 746.000 Menschen. Ocugen plant, weitere Indikationen für OCU200 zu untersuchen, einschließlich diabetischer Retinopathie und feuchter altersbedingter Makuladegeneration, die zusammen fast neun Millionen Amerikaner betreffen.
- FDA lifted the clinical hold on OCU200's Phase 1 clinical trial for DME
- OCU200 targets underlying disease mechanisms through the integrin pathway
- Potential to benefit all DME patients, including non-responders to current therapies
- Plans to explore additional indications for OCU200, expanding market potential
- None.
Insights
The FDA's removal of the clinical hold on OCU200's Phase 1 trial for diabetic macular edema (DME) is a significant milestone for Ocugen. This novel recombinant fusion protein targets the integrin pathway, potentially offering a solution for the
The trial design, involving multiple cohorts and dose-ranging, is robust. It includes a combination therapy arm with anti-VEGF, which could expand OCU200's market potential. With 746,000 DME patients in the U.S. and plans to expand into diabetic retinopathy and wet AMD (affecting 9 million Americans), OCU200 has a substantial addressable market.
However, investors should note that this is still an early-stage trial and success is not guaranteed. The path to market approval is long and costly, which may impact Ocugen's financials in the short term. The company's ability to fund the development through to completion will be important for long-term success.
This news represents a positive development for Ocugen, potentially unlocking value in its pipeline. The removal of the clinical hold allows the company to progress OCU200, which could become a significant revenue driver if successful. However, it's important to note that Ocugen, with a market cap of
Investors should consider the following financial implications:
- R&D expenses are likely to increase as the trial progresses
- Cash burn rate may accelerate, potentially leading to future capital raises
- Successful trial results could attract partnership opportunities or increase the company's acquisition appeal
While positive, this news doesn't immediately impact revenues. The company's financial stability and ability to fund development through potential approval will be critical factors to monitor. Investors should weigh the long-term potential against the near-term financial challenges typical of clinical-stage biotech companies.
MALVERN, Pa., Oct. 09, 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 U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the investigational new drug application for the Phase 1 clinical trial evaluating OCU200, a recombinant fusion protein consisting of tumstatin and transferrin, for treating diabetic macular edema (DME).
“We are excited to launch the Phase 1 clinical trial for OCU200, which is designed to treat patients with DME,” said Dr. Arun Upadhyay, Chief Scientific Officer and Head of Research & Development at Ocugen. “Approximately 30 to
OCU200 possesses unique features that potentially enable it to treat vascular complications of DME. Tumstatin is the active component of OCU200 and binds to integrin receptors, which play a crucial role in disease pathogenesis. Transferrin is expected to facilitate the targeted delivery of tumstatin into the retina and choroid and potentially help increase the interaction between tumstatin and integrin receptors.
“I look forward to bringing OCU200 into the clinic and advancing this important candidate in Ocugen’s portfolio for treating blindness diseases,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “DME is becoming more prevalent as the number of people with diabetes in the U.S. rises, making the condition even more imperative to address.”
This is a multicenter, open-label, dose-ranging study with 3 cohorts in the dose-escalation portion assessing safety of OCU200 and in the fourth cohort a combination of OCU200 (MTD) with anti-VEGF following sequential intravitreal administration.
DME is one of the most common vision-threatening diseases occurring in people with diabetes and includes blurriness in vision and progressive vision loss as the disease progresses. Approximately 746,000 people in the United States are affected with DME.
The Company intends to pursue additional indications for OCU200 to potentially treat diabetic retinopathy and wet age-related macular degeneration, which combined affect nearly nine million Americans.
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 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.
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
What is the latest update on Ocugen's OCU200 clinical trial for DME?
How does OCU200 (OCGN) differ from current DME treatments?
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