Eyes on New Product Development

Abstract

In the most recent period, several ophthalmic drug delivery systems are in development—ranging from an initial investigational new drug (IND) to early and late stage clinical data to supplemental clinical data for an approved product. Progress on novel formulations and new molecular entities for anterior segment inflammation (postoperative and dry eye disease) is reported. In addition, novel antibody and gene therapy treatments for posterior segment disease are also in various stages of development. In terms of regulatory activity, the U.S. Food and Drug Administration (FDA) continues to enforce laws against promotion of unapproved cell therapy. Finally, concerns on opioid abuse in the United States result in unique actions. News items complementary to articles in this issue are noted with an asterisk.

Ophthalmic Pharmaceuticals and Biologics

Aerie filed an IND exemption with the U.S. FDA for AR-1105, an intravitreal implant of dexamethasone, to the treatment of macular edema due to retinal vein occlusion (January 2019). The firm also presented results of a phase 2 study of its netarsudil ophthalmic solution 0.02% in Japanese American subjects (January 2019).

Aldeyra completed dosing in a phase 3 trial of reproxalap for allergic conjunctivitis (December 2018).

Allergan presented results for a second phase 3 trial of its bimatoprost sustained-related intracameral implant (January 2019).*

Aurinia presented results for its phase 2 trial of its topical voclosporin in the treatment of dry eye disease (January 2019).

Graybug presented results from a phase 1/2a study of its GB-102 (sunitinib) encapsulated intravitreal delivery system for the treatment of wet age-related macular degeneration (January 2019).

Kala Pharmaceuticals' New Drug Application (NDA) for its KPI-121 0.25% (loteprednol etabonate) for the treatment of dry eye disease was accepted for review by the U.S. FDA (December 2018).

Kodiak Sciences presented phase 1 results on its intravitreal KSI-301, an antibody biopolymer conjugate for patients with diabetic macule edema (December 2018).

Ocular Therapeutix submitted a supplemental NDA for its Dextenza^® (dexamethasone ophthalmic insert) for the treatment of ocular inflammation after ophthalmic surgery (January 2019). The product is currently approved for the treatment of pain.

Oculis entered into an agreement with Novartis to in-license a novel topical anti-TNF alpha antibody, LME 636, for the treatment of ocular inflammatory conditions of the anterior segment of the eye, including dry eye disease (January 2019).

Gene and Cell Therapy

ProQR received fast track status from the U.S. FDA for its QR-421a, an investigational treatment for vision loss associated with Usher syndrome type 2 and nonsyndromic retinitis pigmentosa due to mutations in exon 13 of the USH2A gene (January 2019).

The U.S. FDA continues to be concerned with the promotion of unapproved stem cell products for therapeutic use (December 2018).

Medical Devices

Oculogica received clearance from the U.S. FDA for its eye tracking device to test concussion (January 2019).

Regulatory, Government, and Pharmaceutical Industry

Costco, a membership discount department store, no longer makes its loperamide, an over-the-counter mu-opioid agonist with very low abuse potential used to treat diarrhea, in a 200 count bottle. The firm makes only a 36-count bottle, which is available only upon request to the pharmacist. They cite potential abuse issues. Note that other firms continue to sell large count bottles without pharmacist interaction (January 2019).

Footnotes

Author Disclosure Statement

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