Aldeyra's Dry-Eye Drug Rebounds with New Phase III Results

Aldeyra Therapeutics has announced that its small-molecule RASP modulator, reproxalap, has demonstrated success in a new Phase III clinical trial for dry-eye disease. This development comes as the company prepares to make a second attempt at securing FDA approval, following a complete response letter (CRL) from the agency last November. The positive trial results led to an 11% increase in the company's stock price on Friday, with plans to refile an application later this year.

CEO Todd Brady commented, "To our knowledge, the results announced today represent the first positive Phase III clinical trial in a dry-eye chamber with a symptom as a primary endpoint. We believe that the results are supportive of the potential rapid clinical effect of reproxalap on reducing the ocular discomfort associated with dry-eye disease."

The trial involved 132 patients who were initially given a placebo version of the treatment before and during exposure to a controlled dry-eye environment. Aldeyra stated that this method aligns with FDA guidelines for studying dry-eye disease. Patients who met the necessary criteria were then randomly assigned to receive either the active drug, reproxalap, or the placebo again during a second round of exposure to the dry conditions.

This trial was specifically designed to meet the FDA's resubmission requirements outlined in the CRL, which had previously indicated that Aldeyra had failed to demonstrate the drug's efficacy. The CRL called for "at least one additional adequate and well-controlled study to demonstrate a positive effect on the treatment of ocular symptoms of dry eye."

In the new study, reproxalap achieved the primary endpoint of reducing ocular discomfort within 80 to 100 minutes of exposure to the dry-eye chamber, with a statistical significance of p=0.004. No safety concerns were observed, with the most commonly reported adverse event being "mild and transient instillation-site discomfort." Importantly, there were no treatment-related discontinuations.

Aldeyra highlighted that reproxalap is the first investigational drug to show pivotal data supportive of both acute and chronic activity in reducing symptoms, as well as ocular redness, in the context of chronic administration. This sets reproxalap apart as a potentially groundbreaking treatment for dry-eye disease.

Before receiving the CRL last year, Aldeyra had entered into a licensing agreement with AbbVie. This agreement grants AbbVie the rights to develop and commercialize reproxalap both within and outside the United States. Under the terms of this arrangement, Aldeyra stands to receive up to $300 million in regulatory and commercial milestone payments, including a $100-million payment contingent upon FDA approval of the drug for dry-eye disease.

With the positive results from the new Phase III trial, Aldeyra is optimistic about reproxalap's potential to meet the FDA's requirements and secure approval, ultimately offering a new treatment option for patients suffering from dry-eye disease.