Eyes on New Product Development

Adverum Biotechnologies received fast track designation from the U.S. FDA for ADVM-022, a novel gene therapy candidate for the treatment of wet age-related macular degeneration (September 2018).

Aldeyra announced results from its phase 2b clinical trial of topical ocular reproxalap in patients with dry eye disease (September 2018).

Apelis started 2 phase 3 clinical trials for APL-2 for geographic atrophy (“DERBY” and “OAKS”; September 2018).

Astellas acquired Quethera, a novel gene therapy program for glaucoma (August 2018).

Eyevance Pharmaceuticals acquired the FreshKote line of lubricant eye drops from Focus Laboratories (September 2018). The firm also entered into a licensing agreement with OcuNexus Therapeutics for NEXAGON, a 30-base antisense oligomer being developed for the orphan indication of persistent corneal epithelial defect nonresponsive to standard of care (October 2018).

Nightstar Therapeutics announced the treatment of a U.S. patient with its gene therapy treatment for X-linked retinitis pigmentosa as part of a clinical trial (September 2018).

Novartis announced results of its RAINBOW phase 3 study of ranibizumab for the treatment of retinopathy of prematurity. They also announced plans to submit marketing authorizations for this as a new indication for Lucentis^® (September 2018).

Oxurion announced enrollment in its phase 2 open-label multicenter study to evaluate intravitreal THR-317, an anti-PIGF antibody, for the treatment of macular telangiectasia type 1 (September 2018).

Presbyopia Therapies announced results of its phase 2b study of its PRX ophthalmic solution for the treatment of presbyopia (July 2018).

SiteOne Therapeutics received an award from the U.S. NIH to initiate IND-enabling studies for its Ocular Na 1.7 Program for pain associated with ocular surgery, injury, and disease (September 2018).

Spark Therapeutics received recommendation for approval of its Luxturna^® (voretigene neparvovec) for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy (September 2018).

Sun Pharmaceutical received approval from the U.S. FDA for its Xelpros™ (latanoprost ophthalmic emulsion) 0.005% for the reduction of elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension (September 2018).

VEO Ophthalmics received U.S. FDA approval for its “Customflex ArtificialIris,” a surgically implanted artificial iris (September 2018).

NIH is conducting clinical trials of DM1157, a modified form of chloroquine, as a possible antimalarial agent (September 2018).

The British Medical Journal has begun to publish clinical trials conducted in the European Union that did not report results within the 1 year time frame as required by regulations. Compliance rates were 68% for commercial sponsors, and 11% for noncommercial sponsors (September 2018). ¹

The U.S. FDA reports an increase in meeting requests for firms developing biosimilars, an indicator of increased development activity in this area (October 2018).

The U.S. Justice department is evaluating whether major pharmaceutical firms potentially violated laws by providing free services to doctors and patients (September 2018).

The U.S. National Institute of Health is expanding its Undiagnosed Diseases Network (September 2018).