Eyes on New Product Development

Abstract

In this issue, two firms announced clinical trials in the treatment of graft-versus-host disease, and novel drug delivery technologies are at several stages of development. For retina, numerous therapies are in development, including an oral small molecule, a novel drug delivery system, and gene therapy. For cornea, an improvement on an approved therapy is in late-stage development as is a novel formulation of a small molecule. In regulatory and government actions, the Food and Drug Administration (FDA) is hopeful about gene therapy, the availability of biosimilar products increases, and concern about the prices of prescription pharmaceuticals continues, as does that of compliance at cell therapy clinics. The Right to Try Act of 2017 (S 204) was signed into law in the United States. While seemingly a good way the make investigational products available for patients with otherwise terminal disease, it is not without risks, and it does not force a company to provide such drugs to patients.¹ Before approval of the law, the FDA commissioner stated that there are already programs that cover this need.

Ophthalmic Pharmaceuticals and Biologics

Acucela released data on a phase 2 study of its oral emixustat in a proliferative diabetic retinopathy population (June 2018).

Avedro enrolled the first patient in a pivotal phase 3 trial of its epi-on corneal collagen cross-linking procedure for progressive keratoconus (June 2018).

Allergan announced results of a phase 3 trial of its bimatoprost sustained-release implant for the treatment of elevated intraocular pressure (IOP) (June 2018).

Cambium Medical Technologies enrolled the first patient in a phase 1/2 study of its enriched platelet-rich-plasma lysate serum treatment for dry eye secondary to graft-versus-host disease (June 2018).

Clearside announced results of a phase 2 clinical trial of suprachoroidal triamcinolone acetonide with aflibercept in the treatment of patients with diabetic macular edema (May 2018).

Eyenovia received Notices of Allowance from the U.S. Patent and Trademark Office for patent applications providing coverage on physiologic microdroplet ejection diameters and velocities (June 2018).

GenSight's presented additional data of its REVERSE phase 3 clinical trial of intravitreal GS010 for the treatment of Leber hereditary optic neuropathy (June 2018).

Kala Pharmaceuticals stated that it plans to submit a new drug application (NDA) to the FDA this year for KPI-121 (0.25% loteprednol etabonate) as a short-term treatment for dry eye disease (June 2018).

Lin BioScience received orphan designation from the European Medicines Agency for its LBS-008, an oral therapy designed to prevent the accumulation of lipofuscin, for Stargardt disease and atrophic age-related macular degeneration (June 2018).

Medicines for Global Health received U.S. FDA approval for its moxidectin (8 mg oral) for the treatment of onchocerciasis (river blindness). The sponsor also received a transferrable “tropical disease priority review voucher” (June 2018).

Mylan received U.S. FDA approval for a biosimilar drug to Amgen's Neulasta^® (pegfilgrastim, indicated for decrease of the incidence of infection in patients receiving oncology therapeutics, June 2018).

Nightstar received advanced therapy designation from the U.S. FDA for its choroideremia treatment NSR-REP1 (June 2018).

Ocugen started two pivotal phase 3 clinical trials of its OCU300 (brimonidine tartrate formulated in a proprietary nanoemulsion) for the treatment of ocular discomfort and ocular redness in patients with ocular graft-versus-host disease under a U.S. Investigational NDA (July 2018).

Ocular Therapeutix resubmitted its NDA to the U.S. FDA for its Dextenza™ (dexamethasone insert) for the treatment of ocular pain after ophthalmic surgery (June 2018).

Sun Pharmaceuticals stated that it has addressed FDA's issues at its Halol facility in Gujarat, India. Note that this factory is the site where its pending novel formulation of latanoprost is being made (June 2018).

Medical Devices

EyeGate Pharmaceuticals filed a supplement to its investigational device exemption for punctate epitheliopathy using its Ocular Bandage Gel (May 2018).

Glaukos' iStent inject trabecular microbypass system received premarket approval from the FDA for the reduction of IOP (June 2018).

Regulatory, Government, and Pharmaceutical Industry

The U.S. FDA has announced plans to reorganize the Center for Drug Evaluation and Research. In a publicly available chart, Ophthalmology (current part of a division with transplant drugs) would be in its own reviewing division (June 2018).

The U.S. FDA filed two complaints in federal court seeking permanent injunctions to stop two stem cell clinics from marketing stem cell products without FDA approval and for significant deviations from current good manufacturing practice requirements (May 2018).

U.S. FDA Commissioner Scott Gottlieb, MD, said he expects the FDA to approve 40 gene therapies by 2022 and might see cures for diseases such as sickle cell anemia within 10 years (June 2018).

After an investigation found senior officials at the National Institutes of Health (NIH) improperly communicated with the alcoholic beverage industry (including Anheuser-Busch) to fund a study of the potential heart benefits of moderate drinking, NIH Director Francis Collins last week shut down the $100 million clinical trial just 4 months into its expected 10-year span.²

U.S. Health and Human Services Secretary Alex Azar testified before the U.S Congress on prescription drug prices (June 2018).

The U.S. Supreme Court refused to consider a case against ophthalmic pharmaceutical manufacturers regarding drop size and drug prices (May 2018).

Novartis announced its intention to spin off of their Alcon eye care devices business (June 2018).

NTC announced the acquisition of some Novartis ocular antibiotic products (May 2018).

The Right to Try Act of 2017 (S 204) was signed into law in the United States. This law seeks to make potentially beneficial medications available to patients with life-threatening diseases (May 2018).¹

Footnotes

Author Disclosure Statement

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

References

Novack

G.D

. Pipeline: the right to try. Ocul. Surf., 13:88–89, 2015.

Wadman

. NIH kills alcohol trial, starts hunt for other suspect studies. Science. 360:1286, 2018.