Eyes on New Product Development

Abstract

In this reporting period, several drug delivery firms reported results on Phase 2 and Phase 3 trials with glaucoma and anti-inflammatory therapies, as did a firm using RNAi technology. Cell and gene therapies are making progress in the treatment of retinal diseases. One firm has resubmitted a request for approval of a combination product for the treatment of corneal disease. A novel biologic treatment is being evaluated in the giant cell arteritis, a disease with no approved therapy.

Ophthalmic Pharmaceuticals and Biologics

• Ampio Pharmaceuticals is continuing to develop Optina™ (danazol) for the treatment of diabetic macular edema in patients refractory to currently available drugs (October 2015). www.prnewswire.com/news-releases/ampio-pharmaceuticals-inc-announces-updates-on-the-regulatory-path-for-fda-approval-of-optina-an-oral-treatment-for-diabetic-macular-edema-dme-300159361.html

• Avedro, Inc. resubmitted its New Drug Application (NDA) for riboflavin ophthalmic solution/KXL^® System to the US Food and Drug Administration (FDA) for the treatment of progressive keratoconus and corneal ectasia following refractive surgery (October 2015). http://avedro.com/press-releases/avedro-announces-resubmission-of-new-drug-application-to-fda-for-corneal-cross-linking/

• BioTime, Inc. and its subsidiary Cell Cure Neurosciences Ltd. received fast track designation from the US FDA for OpRegen^®, a cell-based therapeutic product consisting of retinal pigment epithelial (RPE) cells designed to block the progression of the severe dry form of age-related macular degeneration (September 2015). http://investor.biotimeinc.com/phoenix.zhtml?c=83805&p=irol-newsArticle_print&I0=2090854

• Envisia Therapeutics reported results from a Phase 2a trial of ENV515 (travoprost XR) in patients with glaucoma and ocular hypertension (October 2015). www.envisiatherapeutics.com/category/announcements/

• Gene therapy based on a mouse model of Leber hereditary optic neuropathy at the National Institutes of Health¹ is now in a clinical trial (October 2015). www.nih.gov/news/health/oct2015/nei-05.htm

• Genentech is conducting a Phase 3 trial of tocilizumab for the treatment of giant cell arteritis (October 2015). www.healio.com/rheumatology/vasculitis-and-giant-cell-arteritis/news/online/%7Ba4147dee-3341-42c5-aef8-df5ce7a144b2%7D/phase-3-actemra-trial-underway-for-patients-with-giant-cell-arteritis

• Ocular Therapeutix submitted an NDA for Dextenza^® (sustained release of dexamethasone in punctal plugs) for the treatment of postsurgical ocular pain. It also started a third Phase 3 study for this product in this indication, as well as reported results for this product in this study for the treatment of allergic conjunctivitis. Finally, the firm reported results for a Phase 2b study of travoprost for the treatment of glaucoma and ocular hypertension (September and October 2015). https://beta.finance.yahoo.com/news/ocular-therapeutixtm-reports-topline-results-203300345.html; www.streetinsider.com/Corporate+News/Ocular+Therapeutix+(OCUL)+Announces+Statistically+Significant+Topline+Data+from+DEXTENZA+Phase +3/10995694.html; http://investors.ocutx.com/phoenix.zhtml?c=253650&p=irol-newsArticle&ID=2090846; http://investors.ocutx.com/phoenix.zhtml?c=253650&p=irol-newsArticle&ID=2097159

• Shire received a complete response letter from the US FDA for their NDA for lifitegrast for dry eye. Additional clinical data were requested (October 2015). www.benzinga.com/news/15/10/5921835/shire-receives-fda-crl-for-lifitegrast-nda-and-plans-to-respond-with-opus-3-trial

• Spark Therapeutics announced results from a Phase 3 trial of its gene therapy product candidate, SPK-RPE65, for the treatment of RP65-mediated inherited retinal dystrophies (October 2015). http://ir.sparktx.com/phoenix.zhtml?c=253900&p=irol-newsArticle&ID=2093863

• Sylentis announced results of a dose-finding Phase 2 study for the novel RNAi therapeutic bamosiran, which blocks the production of β₂-adrenergic receptors in patients with open-angle glaucoma or ocular hypertension (October 2015). www.eurekalert.org/pub_releases/2015-10/p-sat100215.php

Regulatory, Government, and Pharmaceutical Industry

• The American Society of Cataract and Refractive Surgery (ASCRS) has released an official position statement opposing directives that restrict 2 well-established presurgical ophthalmic practices: the use of diluted povidone–iodine solution for topical infection prophylaxis and the use of multidose topical eye drops on multiple patients. ASCRS finds dilution of marketed 10% povidone–iodine solution with saline to prepare a 5% solution to be “safe and effective.” They also support the use of multidose eye drops on multiple patients as part of the preoperative surgical protocol (October 2015). www.prweb.com/releases/2015/08/prweb12907908.htm

• Supporting Initiatives to Redistribute Unused Medicine (SIRUM) is providing unused, unexpired prescription medications to low-income patients (October 2015). www.wsj.com/articles/match-com-for-unused-medicine-1443798827*

Footnotes

Author Disclosure Statement

The author consults for numerous ophthalmic, pharmaceutical, and medical device firms.

References

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