• IVERIC bio submitted to the FDA the third and final part of its new drug application (NDA) for rolling review of avacincaptad pegol, a novel investigational complement C5 inhibitor for the treatment of geographic atrophy secondary to AMD. Per the company’s agreement with the FDA for rolling NDA review, part 3 of the NDA included chemistry, manufacturing and controls data. 

• Viridian Therapeutics announced the first patient has been enrolled in its THRIVE Phase 3 trial evaluating the efficacy and safety of the investigational agent VRDN-001 in patients with active thyroid eye disease. The primary efficacy endpoint for THRIVE is proptosis (eye bulging) responder rate, defined as the proportion of patients who achieve a reduction of proptosis of at least 2 millimeters compared to baseline at week 24. 

• Aldeyra Therapeutics submitted an NDA to the FDA for ADX‑2191 (methotrexate injection, USP), an investigational drug candidate, for the treatment of primary vitreoretinal lymphoma, a rare but potentially fatal cancer with no FDA-approved therapy. The NDA submission is supported by a combination of published literature on the safety and efficacy of methotrexate for the treatment of primary vitreoretinal lymphoma and safety data from the recently completed Phase 3 GUARD Trial of ADX‑2191 for the prevention of proliferative vitreoretinopathy.

• The FDA granted orphan drug designations to Ocugen Inc.’s OCU400 — human nuclear hormone receptor subfamily 2 group E member 3 (hNR2E3) — for the treatment of retinitis pigmentosa and Leber congenital amaurosis. OCU400 represents Ocugen’s modifier gene therapy approach, which is based on Nuclear Hormone Receptors that regulate diverse physiological functions, such as homeostasis, reproduction, development and metabolism to potentially improve retinal health and function.

• Melt Pharmaceuticals reported top-line results of its Phase 2 pivotal efficacy and safety study for its lead product candidate, MELT-300, a sublingual, needle- and opioid-free patented formulation for procedural sedation during cataract surgery. The trial compared MELT-300 against placebo alone, sublingually delivered midazolam alone, and sublingually delivered ketamine alone, with two independent primary efficacy endpoints: appropriate cataract surgery sedation using a validated sedation scale or the management of intraoperative pain. 

Quick Notes is published weekly. Unless otherwise noted, the information presented is based on press releases. Find earlier editions here. To submit a press release to be considered for publication, click here.