Aldeyra Therapeutics (ALDX) Status Update summary

Regulatory update and trial status

• FDA issued a Complete Response Letter for reproxalap, citing insufficient efficacy data in treating ocular symptoms of dry eye disease and requesting at least one more well-controlled symptom trial.

• No manufacturing or safety issues were identified by the FDA; mild and transient instillation site discomfort remains the most common adverse event.

• A Type A meeting with the FDA is anticipated within 30 days to clarify deficiencies and discuss next steps.

• Top-line results from ongoing field and chamber trials are expected in Q2 2025, with a potential NDA resubmission targeted for mid-2025, pending positive outcomes and FDA discussions.

• Regulatory review timelines and clinical trial schedules remain flexible and subject to change based on regulatory workload and trial logistics.

Clinical trial design and data

• The ongoing field trial is a randomized, double-masked, vehicle-controlled study with 421 patients, focusing on subject-reported ocular discomfort over six weeks.

• The chamber trial is a randomized, double-masked, vehicle-controlled study with 116 patients, assessing ocular discomfort after controlled exposure.

• Previous field and chamber trials showed statistically significant improvements in ocular discomfort for reproxalap compared to vehicle, with pooled data suggesting consistent symptom control and rapid activity.

• Baseline imbalances in prior trials were highlighted by the FDA, but such differences are considered rare and due to chance; ongoing trials are not expected to have similar issues.

Financial position and future plans

• Cash, cash equivalents, and marketable securities totaled $101 million as of December 31, 2024, expected to support operations into 2027, including multiple clinical programs.

• Most costs for the additional trials were incurred in 2024, with 2025 trial costs estimated at $6 million.

• Pending positive trial results and FDA discussions, NDA resubmission for reproxalap is anticipated in mid-2025, with a potential six-month review period.

• Additional clinical milestones include phase II and phase 2/3 trials in atopic dermatitis and retinitis pigmentosa, and IND submissions for other indications.