Top 4 Eye Disease Treatments on the Way

Promising treatments for dry eye disease (DED), diabetic retinopathy, and presbyopia are expected to be available in the near future, as manufacturers vie for FDA approval. Find the top 4 treatments in the pipeline below.

1. Bausch + Lomb and Novaliq GmbH submitted a New Drug Application (NDA) to the FDA at the end of June, seeking approval for NOV03 (perfluorohexyloctane) for DED associated with Meibomian gland dysfunction (MGD).

If approved by the FDA, NOV03 will be the first available therapy indicated to address the signs and symptoms of DED for this patient population, said Joseph C. Papa, chairman and CEO of Bausch + Lomb in a news release.¹

"This New Drug Application submission is a critical milestone and the first drug submission of a water-free therapy. We are excited to unfold the full potential of NOV03 by bringing this novel drug option for this condition to the United States.” said Christian Roesky, Ph.D., CEO of Novaliq.

2. Regeneron Pharmaceuticals said the FDA accepted its supplemental Biologics License Application (sBLA) to review for Eyelea (aflibercept) injectionin patients with diabetic retinopathy (DR).

The sBLA is for an every 16-week 2 mg dosing regimen (after initial monthly doses). The target action date for the FDA decision is February 28, 2023.

In 2019, Eyelea was approved for the treatment of all stages of DR with a dosing regimen of every 4 or 8 weeks after 5 initial monthly doses, Regeneron said in a news release.²

“If approved, the 16-week dosing regimen could offer certain patients a potentially longer treatment interval and doctors with greater flexibility to individualize treatment,” Regeneron said.

The sBLA is supported by data from the Phase 3 PANORAMA trial investigating every 8- and 16-week EYLEA dosing regimens, versus sham, in patients with severe non-proliferative diabetic retinopathy (NPDR) without diabetic macular edema (DME). The submission was further supported by data from the NIH-sponsored Protocol W trial investigating an EYLEA every 16-week dosing regimen in patients with moderate to severe NPDR without center-involved DME versus sham.

At 1 year, PANORAMA met its primary endpoint of proportion of patients with ≥2-step improvement in Diabetic Retinopathy Severity Scale (DRSS) score. At 2 years, in both the PANORAMA and Protocol W trials, a greater proportion of patients receiving EYLEA every 16-weeks experienced a ≥2-step improvement in DRSS score, along with greater reductions in the risk of developing vision-threatening complications, versus sham.

In addition to DR with a 4- or 8-week dosing regimen, Eylea is approved to treat neovascular (Wet) age-related macular degeneration, macular edema following retinal vein occlusion and DME.

3. Orasis Pharmaceuticals’ CSF-1, a novel eye drop candidate to improve presbyopia, the gradual loss of the eyes' ability to focus on nearby objects, met its key endpoints in Phase 3 trials.

In both trials, CSF-1 met its primary and key secondary endpoints on Day 8, achieving statistically significant 3-line or more gain in distance-corrected near visual acuity (DCNVA), and no loss of 1-line or more in distance visual acuity, Orasis said in a news release.³ Across the two studies, 40% and 50% of participants demonstrated these gains 1-hour post-dose 1 and 1-hour post-dose 2, respectively.

Additional details of the trials will be presented at future medical meetings and will serve as the basis for the company’s NDA submission to the FDA in the second half of this year.

“These statistically significant and clinically meaningful results are promising as eye care providers are eager to find alternate treatment options to improve quality-of-life for their patients with presbyopia, many of whom rely solely on reading glasses, which can be cumbersome,” said Edward Holland, M.D., professor of ophthalmology at the University of Cincinnati and Director of Cornea at the Cincinnati Eye Institute.

“The potential of CSF-1 to provide a first-line treatment option for people living with presbyopia is promising and we look forward to working with regulatory authorities to advance CSF-1 toward commercialization,” said Elad Kedar, CEO of Orasis Pharmaceuticals.

4. Aldeyra Therapeutics achieved the primary endpoints in a crossover clinical trial of 0.25% reproxalap ophthalmic solution, an investigational new drug candidate to treat DED.

Reproxalap was statistically superior to vehicle for each of the two prespecified primary endpoints, ocular redness in a dry eye chamber, a measure of tear production, after a single day of dosing, the company said in a news release.⁴ The secondary endpoint of Schirmer test ≥10 mm responder analysis, which was multiplicity-controlled, was also achieved.

“Statistical significance in favor of reproxalap over vehicle for all three dry eye disease signs that we intend to submit to a New Drug Application, in addition to observed rapid symptomatic improvement, favorably positions reproxalap, if approved for sale, as a potentially differentiated therapeutic option for the treatment of dry eye disease,” said Todd C. Brady, M.D., Ph.D., president and CEO of Aldeyra.

References

1. Bausch + Lomb and Novaliq announce submission of new drug application for investigational treatment NOV03 (Perfluorohexyloctane). News release. Basuch + Lomb. July 7, 2022. Accessed July 15, 2022. https://www.bausch.com/news/releases/?id=98.
2. Eylea (Aflibercept) injection sBLA for every 16-week dosing regimen in patient with diabetic retinopathy accepted for FDA review. News release. Regeneron. June 29, 2022. Accessed July 15, 2022. investor.regeneron.com/news-releases/news-release-details/eylear-aflibercept-injection-sbla-every-16-week-dosing-regimen.
3. Orasis Pharmaceuticals announces positive Phase 3 toppling results of novel dye drop candidate, CSF-1, for the treatment of presbyopia. News release. Orasis Pharmaceuticals. April 21, 2022. Accessed July 15, 2022. https://www.orasis-pharma.com/orasis-pharmaceuticals-announces-positive-phase-3-topline-results-of-novel-eye-drop-candidate-csf-1-for-the-treatment-of-presbyopia/.
4. Aldeyra Therapeutics achieves primary endpoints in dry eye disease chamber crossover clinical trial. News release. Aldeyra Therapeutics. July 12, 2022. Accessed July 15, 2022. https://ir.aldeyra.com/news-releases/news-release-details/aldeyra-therapeutics-achieves-primary-endpoints-dry-eye-disease.