Eyes on New Product Development

Abstract

In this reporting period, several ophthalmic gene therapy products are in clinical development for the treatment of degenerative and neovascular retinal diseases. In glaucoma, clinical development of long-duration ocular hypotensive implants continues as well as that of minimally invasive glaucoma stents. Better therapeutics for postoperative inflammation and its associated pain as well as for external eye disease continue to be developed. Enrollment for a large prophylactic study of a biological prophylactic therapy for geographic atrophy was completed, with anticipation for results. As well, combination products for ophthalmology (e.g., concomitant use of a biological and a laser) continue to be developed. The new U.S. Congress is considering several issues surrounding the costs of executing the 21st Century Cures Act and encouraging development of generic medications and competition to manage pharmaceutical prices.

Ophthalmic Pharmaceuticals and Biologics

• Aura Biosciences received clearance from the U.S. FDA for its investigational new drug exemption (IND) for AU-011, an intravitreal injection of viral nanoparticle conjugates, intended for activation with a laser. AU-011 is in a phase 1b clinical trial for the treatment of ocular melanoma (February 2017).

• Bausch + Lomb resubmitted its new drug application (NDA) for latanoprostene bunod ophthalmic solution, 0.024% for lowering intraocular pressure in patients with open angle glaucoma or ocular hypertension to the FDA (February 2017).

• BioTime obtained a world-wide license to assets from the University of Pittsburgh for methods to develop 3D retinal images (February 2017).

• Clearside Biomedical started enrollment in a phase 3 clinical trial “SAPPHIRE” of Zuprata™, its proprietary suspension formulation of the corticosteroid triamcinolone for the treatment of macular edema associated with retinal vein occlusion (February 2017).

• Envisia Therapeutics presented results from the second cohort of its ENV515 (travoprost XR) phase 2 trial in patients with glaucoma (February 2017).

• EyeGate^® Pharmaceuticals presented results from a pilot trial of its EyeGate Ocular Bandage Gel (“EyeGate OBG”) in patients having undergone photorefractive keratectomy (January 2017). EyeGate and Valeant have expanded their licensing arrangement for the EyeGate II Delivery System and EGP-437 combination product candidate (dexamethasone) for the treatment of postoperative pain and inflammation in ocular surgery patients (February 2017).

• Genentech launched its Lucentis^® (ranibizumab) prefilled syringe in the United States (January 2017). As well, it completed enrollment in 2 large phase 3 clinical trials, CHROMA and SPECTRI, investigating intravitreal lampalizumab in reducing the progression of geographical atrophy (February 2017).

• GenSight Biologics was granted orphan status by the FDA for its GS030, a gene therapy product encoding a photoactivatable channelrhodopsin protein and biomimetic goggles, for the treatment of retinitis pigmentosa. (January 2017).

• Nicox S.A. is planning clinical studies in 2017 of NCX 4251, its novel ophthalmic suspension of fluticasone propionate nanocrystals, for the topical treatment of acute exacerbation of blepharitis (January 2017).

• Ocular Therapeutix announced its resubmission of a NDA for its DEXTENZA™ (dexamethasone insert) for intracanalicular use for the treatment of ocular pain occurring after ophthalmic surgery and has been accepted for review (February 2017).

• Regenexbio announced that its IND in the United States for RGX-314, a gene transfer product, for the treatment of wet age-related macular degeneration (wet AMD) is active (February 2017).

• Spark Therapeutics submitted the clinical portion of its biological license application for its RPE-65 gene transfer product for the treatment of retinitis pigmentosa to the FDA (February 2017).

Ophthalmic Devices

• Glaukos completed enrollment in its iStent Supra trial (February 2017).

Regulatory, Government, and Pharmaceutical Industry

• Marathon Pharmaceuticals received FDA approval for Efmalza^® (deflazacort), a drug to treat Duchenne muscular dystrophy. The late stage development and approval was based on neurologists' and patients' demands to reinvigorate research from previous decades (February 2017).¹

• The U.S. Centers for Disease Control reported ∼5,000 cases of mumps infections in 2016, up from ∼500–1,200 cases in recent years (January 2017).

• The U.S. Congress is considering the “Lower Drug Costs through Competition Act” (HR 4784, sponsored by Rep. Kurt Schrader (D-Ore.) and Gus Billrakis (R-Fla). The measure plans to promote generic drug development in situations of a drug shortage or for drugs that lack competitors (February 2017). As well, the U.S. Congress is considering the latest version of the Prescription Drug User Fee Act VI, the current version of which expires in late 2017. There is consideration for additional fees to support the additional actions in the 21st Century Cures Act of 2016 (February 2017).² Finally, there is consideration of revision of the Orphan Drug Act vis-à-vis the high product revenue for some approved products.³

Footnotes

Author Disclosure Statement

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

References

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