Ocular Diseases Drug Discovery

GTCbio | November 29th, 2017

By 2050, it is projected that vision loss will grow to affect more than 10 million people, from the current statistic of 4.4 million. The total prevalence of cataract, diabetic retinopathy, glaucoma and AMD is expected to increase to nearly 70 million adults by 2050 which urges to development of drugs for ocular diseases. In the late 19th and early 20th century numerous optical instruments were developed by prominent opticians. In Berlin, Albrecht von Graefe introduced iridectomy as a treatment for glaucoma and improved cataract surgery. Further, Pilocarpine, a medication used to treat increased pressure inside the eye and dry mouth was isolated in 1874. In 2005, a chinese-born swedish scientist Dr. Cao founded a company “Clanotech” that develops novel antiangiogenic drugs for the treatment of ocular disease and fibrosis disease. Aflibercept, a drug used for cancer was co-developed for eye diseases by Bayer HealthCare and Regeneron under a deal signed in 2006. In 2014, FDA gave a total of 41 approvals for NME and biological products.

Currently, OCU300 (brimonidine tartrate) is a re-purposed drug being developed through the FDA’s 505(b) (2) pathway for the treatment of OGVHD (ocular graft versus host disease). Also, Bevacizumab is approved for medical use in the United States and is listed for its use in treating eye disease. FDA has also approved cetirizine ophthalmic solution 0.24% (Zerviate), which is the first topical ocular formulation of the antihistamine cetirizine, which is already approved for oral use to treat ocular itching associated with allergic conjunctivitis. Further, various topical and intravitreal routes are widely used to deliver therapeutics to retinal tissue with the help of ocular inserts and implants that are still under development and may improve drug delivery drastically in the years to come. Some of the controlled delivery systems, including ocular inserts and implants currently in market use are Surodex™, Iluvien, Retisert, Durasert, Vitrasert, Ozurdex, Cortiject and I-Vation. Moreover, the landscape for ocular drug therapy has substantially changed and our knowledge of the pathogenesis of ophthalmic diseases has grown considerably. As anti-angiogenic drugs emerged as the most effective form of therapy for age-related macular degeneration and retinal vascular diseases and decreasing the intraocular pressure still remained the mainstay for glaucoma treatment but neuroprotective drugs emerged as a promising next-generation therapy.

With a glimpse towards the future of drug discovery for ocular diseases, the revolution of induced pluripotent stem cell (iPSC) technology provides a platform for development of cell therapy, disease modeling and drug discovery for ocular diseases. The galectin-3 (gal-3) protein is an intriguing new drug target to treat a variety of human disorders. Galectin has a discovery program for identification of small molecule inhibitors of gal-3 and such oral inhibitors will likely open many possibilities for galectin-directed therapies for a broad range of human diseases including ocular diseases. Recent research also shows that Luxturna, developed for vision loss due to confirmed Biallelic RPE-mediated inherited retinal disease is close to approval by the FDA and its treatment will be the first ever gene therapy to hit the US market. It is expected that ROCK inhibitors will become new molecular target drugs for vitreoretinal diseases in the coming future. Thus, the future holds great promise for safe and effective therapies for ocular disease but this depends on ongoing investment in ophthalmic drug development covering the whole translational spectrum from ‘bench to bedside’.

Ocular2018 1000x186 1 - Ocular Diseases Drug DiscoveryMany of these new advances will be discussed at GTCbio’s 10th Ocular Diseases Drug Discovery Conference, which will be held on March 20-21, 2018, in San Diego, CA. The sessions include the following:

  • Opening Keynote Session: Historical Perspective and State of the Industry
  • Preclinical Development of Novel Technologies
  • Translational Studies and Lessons Learned
  • Partnering between Industry, Academia, and CROs to Advance Research
  • Panel: Ophthalmology Funding Environment
  • Panel: Regulatory Challenges
  • Start-Up Showcase (Lunch Session)
  • Closing Keynote Session: Predictions for Future

Please review the agenda at