Breaking News
September 21, 2018 - Young children’s oral microbiota could serve as early indicator for obesity
September 21, 2018 - Older individuals with multiple sclerosis report higher quality of life than younger counterparts
September 21, 2018 - LineaRx signs agreement with Takis/Evvivax to develop linear-DNA based anti-cancer vaccines
September 21, 2018 - AbbVie Presents Upadacitinib Longer-Term (32-Week) and Patient-Reported Outcomes Data from Phase 2b Atopic Dermatitis Study at 27th European Academy of Dermatology and Venereology (EADV) Congress
September 21, 2018 - Alzheimer’s drug may stop disease if used before symptoms develop
September 21, 2018 - Human skeletal stem cell can generate cartilage, bone
September 21, 2018 - UK and European research collaborations receive vote of confidence by three major cancer charities
September 21, 2018 - Microbiota in the intestines and cell stress cause colon cancer
September 20, 2018 - Arizona EMTs have 39% higher risk for suicide than general public
September 20, 2018 - Driving and older adults: MedlinePlus Medical Encyclopedia
September 20, 2018 - Researchers test autobiographical memory for early Alzheimer’s detection
September 20, 2018 - Organizations join forces to help teens with severe mental health challenges | News Center
September 20, 2018 - Neurons in the human brain can encode numerical information
September 20, 2018 - Potential drugs to treat neurodegenerative diseases garner $3 million grant
September 20, 2018 - Processing speed important to higher order cognitive function in multiple sclerosis patients
September 20, 2018 - Helping a patient survive a hurricane
September 20, 2018 - Tafamidis Treats Transthyretin Amyloid Cardiomyopathy
September 20, 2018 - Low academic achievement can lead to drug abuse decades later, research finds
September 20, 2018 - Study identifies stem cell that gives rise to new bone, cartilage in humans | News Center
September 20, 2018 - Celltrion and Emory University sign ‘Incubation’ agreement to develop new drug candidates for atherosclerosis
September 20, 2018 - TGen and PNOC take part in launch of NIH-supported Kids First Data Resource Portal
September 20, 2018 - Could Household Cleaners Make Your Kid Fat?
September 20, 2018 - Addiction nonprofit makes searching for services simple
September 20, 2018 - We are bombarded by thousands of diverse species and chemicals | News Center
September 20, 2018 - Experts to Present Prostate Cancer Advances at Patient Summit
September 20, 2018 - Alector announces initiation of Phase 1 trial of AL001 for treating frontotemporal dementia
September 20, 2018 - Pfizer’s 20vPnC vaccine receives Breakthrough Therapy designation from FDA
September 20, 2018 - Study could allow doctors to screen patients at risk from Aspergillus
September 20, 2018 - Emergex signs MoU with Brazil’s Fiocruz for development of viral vaccines
September 20, 2018 - The ‘real you’ is a myth – we constantly create false memories to achieve the identity we want
September 20, 2018 - Researchers describe cell mechanism that optimizes proteins production in stressful situations
September 20, 2018 - Cell Medica successfully doses first patient with CMD-501 targeting pediatric neuroblastoma
September 20, 2018 - Sesen Bio to present its three-month Phase 3 VISTA Trial data at Global Congress
September 20, 2018 - Senators unveil legislation to protect patients against surprise medical bills
September 20, 2018 - Study provides insights into development of special-purpose cosmetic products
September 20, 2018 - Research shows enlarged genotype-phenotype correlation for three-base pair deletion in NF1
September 20, 2018 - 91% of people around the world believe medical research will result in dementia cure
September 20, 2018 - DePuy Synthes introduces CONCORDE LIFT Expandable Interbody Device at EUROSPINE 2018
September 20, 2018 - Manx Telecom unveils MT clearSound that improves clarity of mobile phone calls
September 20, 2018 - Mediterranean-style diet appears to reduce stroke risk in women
September 20, 2018 - AbbVie Announces Patient-Reported Outcomes Data from Three Pivotal Phase 3 Studies of Risankizumab, Showing Significant Improvements in Health-Related Quality of Life for Patients with Psoriasis
September 20, 2018 - Characterization of pregnancy microbiome reveals variations in bacterial diversity
September 20, 2018 - New guidance for treatment of bone loss in hematologic stem cell transplant Recipients
September 20, 2018 - Experts to present research on prevention, management of dysphagia at international conference
September 20, 2018 - New study focuses on two-way gene switches controlling gene activity
September 20, 2018 - Zika virus could become a weapon against brain cancer
September 20, 2018 - Home-based video game exercises can reduce chronic low back pain in older people, study finds
September 20, 2018 - Investigators find that bile acids reduce cocaine reward
September 20, 2018 - Cannabinoid drugs reduce perceived unpleasantness of painful stimuli and increase tolerance
September 20, 2018 - Health care companies’ data could enable more accurate flu season forecasts
September 20, 2018 - Geroscience takes center stage in Journal of the American Medical Association
September 20, 2018 - Ambient Particulate Matter Linked to Emergency Asthma Care
September 20, 2018 - Patient satisfaction with plastic surgery—it’s the surgeon, not the practice
September 20, 2018 - Medicine is a team sport – and that’s exactly how it should be
September 20, 2018 - Logos Biosystems releases new electrophoretic tissue clearing system with twice the features in half the space
September 20, 2018 - Novel micro-platform reveals never-before-seen behaviors of cancer cells
September 20, 2018 - PAREXEL partners with Datavant to enhance clinical study design and generate real-world evidence
September 20, 2018 - Robert Koch Institute publishes new data on allergies, mental health problems, and accident injuries
September 20, 2018 - Study finds higher readmission rates in for-profit hospitals
September 20, 2018 - Encouraging youth to do strength-based exercises could help tackle child obesity
September 20, 2018 - Sleep apnea, congenital heart disease in hospitalized infants strongly associated with death
September 20, 2018 - Researchers find way to map mysterious content of non-coding RNA
September 20, 2018 - Air Pollutants Reach Placenta, Might Harm Fetus: Study
September 20, 2018 - Sleep apnea, congenital heart disease may be deadly mix for hospitalized infants
September 20, 2018 - My relative has cancer, should I worry? Encouraging cascade genetic testing
September 20, 2018 - Investigators determine specific treatable traits that can predict future asthma attacks
September 20, 2018 - More doctor visits can lower risk of suicide attempts in fibromyalgia patients
September 20, 2018 - Computer avatars play role in diagnosis of dementia
September 20, 2018 - Addition of CTLA4 targeted therapy to PD-1 targeted therapy may benefit patients with ovarian cancer
September 20, 2018 - ASPREE trial explores whether low dose aspirin can prolong good health in elderly people
September 20, 2018 - ATS publishes new guideline focused on weight loss strategies for sleep apnea patients
September 20, 2018 - The Current issue of “The view from here” is concerned with Drug Delivery
September 19, 2018 - Sleep apnea could favour tumor growth at young ages
September 19, 2018 - Stealth vaping fad hidden from parents, teachers
September 19, 2018 - Witnessing school violence linked to later risk of psycho-social and academic impairment
September 19, 2018 - Common household cleaners could make children overweight by changing gut microbiota
September 19, 2018 - Salk research in yeast leads to serendipitous finding about hypomyelinating leukodystrophy
September 19, 2018 - Study: Overweight or obese women may have increased risk of urinary incontinence
September 19, 2018 - Study shows how cellular waste disposal processes also promote inflammation
September 19, 2018 - New multidisciplinary microsurgery microscope, PROVIDO, introduced by Leica
Ocugen Initiates Phase 3 Clinical Trial of OCU300 for the Treatment of Ocular Graft Versus Host Disease

Ocugen Initiates Phase 3 Clinical Trial of OCU300 for the Treatment of Ocular Graft Versus Host Disease

image_pdfDownload PDFimage_print

MALVERN, PA, July 2, 2018 — Ocugen, Inc., a rapidly growing ophthalmology company developing a rich clinical pipeline of innovative therapies that address rare and underserved ocular diseases, today announced that it has initiated the first of two pivotal, Phase 3 clinical trials of OCU300 for the treatment of ocular discomfort and ocular redness in patients with ocular graft versus host disease (oGVHD) following recent acceptance of Ocugen’s New Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA). OCU300 consists of FDA-approved brimonidine tartrate formulated in a proprietary nanoemulsion based on Ocugen’s patented OcuNanoE™ technology. Ocugen is the first and only company to advance a therapy into a Phase 3 clinical trial for treating patients with oGVHD and OCU300 is the only product candidate to be granted Orphan Drug Designation for oGVHD by the FDA.

oGVHD is a severe ophthalmic comorbidity that affects about 60% of patients who undergo allogeneic bone marrow transplant, yet there is currently no approved treatment. It is a chronic and debilitating autoimmune condition characterized by painful, dry, tearless eyes, vision issues and potential ocular scarring. The proof of concept for using brimonidine to treat oGVHD was established by two previous investigator-led clinical studies. In an exploratory observational study, approximately 90% of oGVHD patients treated for six months with brimonidine eye drops reported an improvement in their symptoms without any significant side effects. In a randomized, placebo-controlled, double-masked Phase 1/2 study, oGVHD patients using brimonidine eye drops had a significant reduction in ocular redness scores (p

Daniel Jorgensen, MD, MPH, Chief Medical Officer of Ocugen, said, “We are excited to advance OCU300 into pivotal-stage clinical development and to potentially deliver to oGVHD patients the first FDA-approved therapy to address this condition. The preclinical efficacy data we presented last month at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting further highlight the ability of OCU300, in particular its novel nanoemulsion formulation, to inhibit the underlying pathophysiological processes associated with oGVHD. We believe OCU300 has significant potential to improve the quality of life of patients living with this debilitating disease.”

Shankar Musunuri, PhD, MBA, Chairman, CEO and Co-Founder of Ocugen, commented, “This is a significant milestone for Ocugen and I would like to thank our dedicated team and our participating clinical sites for their work, which has enabled us to reach this stage and commence Phase 3 development in a highly efficient manner. As we begin enrolling oGVHD patients in this study, we look forward to continuing that efficiency and reporting top-line data as soon as it is available, which we currently expect to be in the second half of 2019. We also expect to launch pivotal studies for our second lead candidate, OCU310 for the treatment of dry eye disease, later this year.”

About the OCU300 Phase 3 Clinical Program

The pivotal clinical program consists of two randomized, double-masked, placebo-controlled Phase 3 studies designed to examine the efficacy of OCU300 in treating ocular discomfort and redness as a result of oGVHD. The studies will be staggered, and each will evaluate approximately 60 patients randomized 2:1 to receive OCU300 or placebo (ophthalmic saline). Patients will receive daily eye drops of their assigned treatment for a period of 84 days. The primary efficacy endpoints will measure ocular discomfort using the visual analog scale (VAS) and ocular redness using the Validated Bulbar Redness (VBR) grading scale.

About OCU300

OCU300 is in pivotal stage clinical development for treating ocular discomfort and ocular redness in patients with the debilitating autoimmune condition called ocular graft versus host disease (oGVHD), which develops in many patients following an allogeneic bone marrow transplant. It is the only product to be granted Orphan Drug Designation for this indication from the U.S. FDA, and it consists of an improved 0.18% ophthalmic nanoemulsion of brimonidine tartrate, an FDA-approved drug with established safety for ocular use, enabling Ocugen to develop OCU300 under the accelerated 505(b)(2) regulatory pathway. Ocugen’s patented OcuNanoE™ technology is designed to enhance efficacy by prolonging retention of this potent anti-inflammatory drug on the eye surface. In addition, it allows OCU300 to be sterile filtered into single-use vials as preservative-free nanoemulsion, thereby eliminating potentially irritating effects of preservatives.

About Ocugen, Inc.

Ocugen, Inc., is a rapidly growing ophthalmology company developing a rich clinical pipeline of innovative therapies that address rare and underserved ocular disorders. The Company’s lead clinical candidate (OCU300) is currently in Phase 3 for treating ocular graft versus host disease (oGVHD) and is the first and only therapeutic with orphan drug designation for oGVHD, providing certain regulatory and economic benefits. The Company’s second lead candidate for treating dry eye disease (OCU310) is also expected to enter pivotal clinical trials in 2018. Both OCU300 and OCU310 leverage Ocugen’s patented OcuNanoE – Ocugen’s ONE Platform™ technology to enhance the efficacy of topical ophthalmic therapeutics. Ocugen is also developing novel biologic therapies for retinitis pigmentosa (OCU100) and wet AMD (OCU200), as well as a groundbreaking modifier gene therapy platform with potential to address a broad spectrum of inherited retinal disorders (OCU400). For more information, please visit www.ocugen.com.

Source: Ocugen, Inc.

Posted: July 2018

Tagged with:

About author

Related Articles