Eyes on New Product Development

Acucela continues development of emixustat for the treatment of retinal degeneration based upon a recent publication ¹ (December 2019), and press release regarding continued enrollment in a phase 3 trial in patients with Stargardt disease (February 2020).

Aerie's submission of a marketing authorization application for Roclanda^® (netarsudil and latanoprost ophthalmic solution, Rocklatan^® was accepted for review by the European Medicines Agency (January 2020).

Aerpio announced results from the fifth cohort of subjects from a phase 1b trial of a topical ocular formulation of AKB-9778 in patients with ocular hypertension or primary open angle glaucoma (January 2020).

Alcon received US FDA approval for switching Pataday^® (olopatadine) from prescription to over-the-counter (February 2020).

Aldeyra started enrollment in its phase 3 INVIGORATE Trial of topical ocular reproxalap in patients with allergic conjunctivitis. The firm also started enrollment in a phase trial of ADX-2191 (intravitreal methotrexate) for the prevention of recurrent retinal detachment due to proliferative vitreoretinopathy (January 2020).

Allysta Pharmaceuticals started enrollment in a phase 1/2a trial of ALY688, a novel peptide agonist that binds to and activates adiponectin receptors, in patients with dry eye disease (January 2020).

Genentech and Roche started enrolled in a phase 3 trial of its Port Delivery System with ranibizumab in people with diabetic macular edema (DME, January 2020).

HanAll Biopharma and Daewoong Pharmaceutical announced topline results from the first US phase 3 study of HL036 for treatment of dry eye (VELOS-2 study, January 2020).

Iveric announced results for its Zimura (avacincaptad pegol), a novel C5 inhibitor, in a phase 2b trial for the treatment of patients with geographic atrophy secondary to age-related macular degeneration (AMD). They also announced the design for a second trial (January 2020).

Novartis received approval from the European Commission for its Beovu^® (brolucizumab) for the treatment of wet AMD (February 2020).

Oculis reported results from a phase 2 study of its OCS-01 (a novel eye drop formulation of dexamethasone) in patients with DME (February 2020).

Santen cleared a regulatory hurdle to make its Verkazia^® (cyclosporine) available for patients with severe vernal keratoconjunctivitis in Quebec (January 2020).

Applied Genetic Technologies Corporation reported interim 6-month data from its ongoing phase 1/2 clinical program in X-linked retinitis pigmentosa (January 2020).

Lineage Cell Therapeutics provided an update on its ongoing phase 3 study for the treatment of dry AMD of OpRegen, a retinal pigment epithelium transplant therapy delivered subretinally using a delivery system from Gyroscope Therapeutics (February 2020).

Alexion completed its acquisition of Achillion (January 2020).

Bartlett et al. ² conducted a cross-sectional analysis on the potential to use real-world data ³ to replicate clinical trial evidence. They found that only 15% of the US-based clinical trials published in high-impact journals in 2017 could be feasibly replicated through analysis of administrative claims or electronic health records data. These findings suggest the potential for observational data to complement but not completely replace randomized clinical trials (January 2020). ²

Björk et al. found that there are few citations to articles in “predatory” journals, suggesting that they have little impact.^4,5 On a personal note, this author received an unsolicited e-mail from a predatory journal promising to waive publication costs if I cited an article from their journal.

Santen Pharmaceutical and the Alphabet subsidiary verily announced the establishment of a joint venture focused on applying microelectronics and scalable digital technologies to ophthalmology (February 2020).

A growing number of states are proposing laws to ban cosmetics the safety of which is testing on animals before marketing. Animal-tested cosmetics already are banned in Europe, India, and elsewhere (January 2020).

The challenges in developing novel treatment for infectious diseases (including antibiotics and antivirals) from scientific, regulatory, and business perspectives continue to be debated (December and January 2020). ⁶

The practice of dispensing 90 days of medication, efficient for health care delivery, was questioned for patients with depressive disorders, as it may provide them an easily available method to commit suicide. ⁷

There is a growing realization that sponsors of clinical trials are not complying with the law regarding posting of results on clinicaltrials.gov. The apparent noncompliers include academic, government, and industry-sponsored studies (January 2020). ⁸

In this period, activities from the FDA include the following:

○ Release of a guidance for Human Gene Therapy for Retinal Disorders (January 2020)

○ Conduct of a study on brand names of drugs with respect to how they influence consumers' and health care providers' perceptions, and may overstate efficacy (January 2020) ⁹

○ Apparent evidence of policy on orphan designation and approval [21 CFR 316.3(b)(3)], in FDA approval of a nasal spray of diazepam to treat frequent seizure activity (Valtoco™, Neurelis), and denial of a citizen's petition by Aquestive investigating a buccal film formulation of diazepam, in which they requested staying of the approval pending additional clinical studies. It also appears that this new product will receive 7 years of marketing exclusivity.

○ Concern regarding therapeutic products that are derived from human cells, tissues, or cellular or tissue-based products related to the ongoing COVID-19 (corona virus) outbreak (February 2020).