Eyes on New Product Development

Acucela, Inc., a subsidiary of Kubota Pharmaceutical Holdings, started enrollment in a study to evaluate emixustat in subjects with macular atrophy secondary to Stargardt disease (November 2018).

Aerie reported that the European Medicine Association accepted for review the Marketing Authorization Application for Rhokiinsa^® (netarsudil ophthalmic solution) 0.02%.

BioTime presented results from a phase 1/2a study on OpRegen, a retinal pigment epithelium cell therapy transplant for dry AMD (October 2018).

Clearside Biomedical announced results from its Xipere trial of its suprachoroidal triamcinolone acetonide given with aflibercept in patients with retinal vein occlusion (November 2018).

EyePoint Pharmaceuticals received U.S. FDA approval for Yutiq™ (fluocinolone acetonide intravitreal implant) for the treatment of chronic noninfectious posterior segment uveitis (October 2018).

Genentech announced results from its phase 2 STAIRWAY trial using the bispecific anti-VEGF/anti-Ang2 faricimab (formerly called RG7716) in wet AMD with 16-week dosing intervals (September 2018). Together with Roche, they subsequently are starting 3 large phase 3 clinical trials evaluating the Port Delivery System with ranibizumab to treat wet AMD and faricimab to treat diabetic macular edema (October 2018).

Ionis Pharmaceuticals announced a collaboration with Roche to develop IONIS-FB-L (Rx) for the treatment of complement-mediated diseases such as dry AMD (October 2018).

Kala submitted a NDA to the U.S. FDA for KPI-121 (loteprednol etabonate 0.25%) for the treatment of dry eye disease (October 2018).

Kodiak Sciences completed enrollment of patients in the company's phase 1 safety and tolerability study of KSI-301, an anti-VEGF biological, for the treatment of patients with retinal vascular diseases (August 2018).

Mati Therapeutics presented results from a planned interim analysis of an ongoing phase 2 clinical trial evaluating the safety and efficacy of nepafenac delivered in Mati's proprietary Evolute ocular drug delivery platform in patients undergoing cataract surgery (September 2018).

MC2 Therapeutics announced results of a phase 2 study of its MC2-03 treatment for dry eye patients with keratitis (October 2018).

Mitotech started enrollment in VISTA-1, a phase 3 clinical trial of SkQ1, a cardiolipin peroxidation inhibitor, in patients with moderate to severe dry eye disease (October 2018).

Novaliq announced results from a phase 2/3 study of CyclASol (0.1% cyclosporine) in patients with dry eye disease (October 2018).

Ocugen enrolled patients into its phase 3 clinical trial of its dry eye disease treatment OCU310 [brimonidine tartrate 0.2% (October 2018)].

Ophthotech announced results from its phase 2a safety trial of Zimura™ (avacincaptad pegol), a complement factor C5 inhibitor, in patients with wet AMD in combination with Lucentis^® [ranibizumab, (November 2018)].

Ophthotech Corp. acquired Inception 4, Inc. and its small molecule inhibitors of HtrA1, which are under development for the treatment of a range of retinal diseases (November 2018).

Oyster Point Pharma announced results from the ONS RAINIER studies, 2 separate phase 2b clinical trials evaluating the company's novel therapies, OC-01 and OC-02—both nicotinic acetylcholine receptor agonists administered as a nasal spray, for the treatment of dry eye disease (October 2018).

ProQR received an exclusive worldwide license from Ionis for IONIS-RHO-2.5 (now QR-1123, is a gapmer with a mutant allele-specific knockdown) for autosomal dominant retinitis pigmentosa (October 2018).

Santen, Inc. started a phase 3 development program (SPECTRUM) with omidenepag isopropyl (DE-117), a selective agonist for the prostanoid receptor EP2, for the treatment of glaucoma or ocular hypertension (October 2018).

Nightstar announced results from a phase 1/2 study of NSR-RPGR, a gene therapy treatment for X-linked retinitis pigmentosa (October 2018).

Regemxbio presented 6-month results of an ongoing phase 1 study evaluating its RGX-314 gene therapy using an NAB-AAV8 vector for patients with wet AMD (October 2018).

Spark Therapeutics received European Commission marketing authorization for Luxturna^® (voretigene neparvovec-rzyl), a treatment for inherited retinal disease (November 2018).

EyeGate announced results from a study of its Ocular Bandage Gel for the management of corneal epithelial defects in patients after photorefractive keratectomy surgery (November 2018).

Carl Zeiss Meditec will acquire IanTECH, developer of a lens fragmentation device (October 2018).

Eyevance Pharmaceuticals acquired Flarex^® (fluorometholone acetate ophthalmic suspension) from Novartis (October 2018).

The Foundation Fighting Blindness created the Retinal Degeneration Fund (RD Fund), a not-for-profit subsidiary focused on making venture philanthropy investments to further the foundation's mission (October 2018).

The U.S. executive branch announced a plan to reduce U.S. pharmaceutical prices by adjusting to European prices (October 2018).

U.S. FDA commissioner Scott Gottlieb, MD announced that FDA would give more attention to the use of real-world evidence as part of its postmarketing surveillance system (November 2018). In other actions, the U.S. FDA approved Retacrit^® (epoetin alfa-epbx) as a biosimilar to Epogen/Procrit (epoetin alfa) for the treatment of selected anemias (May 2018); it released a draft guidance on adaptive clinical trial designs (September 2018). The FDA also released additional product-specific guidance on generic drug development and proposed modernization of predicate devices used in the 510(k) pathway.

The United State Pharmacopeia (USP) is updating its compounding standards including USP 795 (nonsterile compounds) and USP 797 (sterile compounds; November 2018).