Aldeyra Therapeutics Announces Improvement from Baseline in Retinal Function in Phase 2 Clinical Trial of ADX‑2191 in Patients with Retinitis Pigmentosa
- Best Corrected and Low‑Light Visual Acuity Statistically Significantly Improved
- As Assessed by Electroretinography, Time to Retinal Response Statistically Significantly Improved
- As Assessed by Macular and Dark-Adapted Perimetry, Retinal Sensitivity Statistically Significantly Improved
- ADX‑2191 Was Well Tolerated and No Safety Concerns Were Identified
- Planned Phase 2/3 Clinical Trial to be Discussed with Regulatory Authorities
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“The improvement in retinal function relative to baseline observed in this retinitis pigmentosa clinical trial of ADX-2191 may offer hope to patients that today have no therapeutic options,” stated
Based on preclinical evidence suggesting that methotrexate may facilitate the clearance of mutated rhodopsin1, a protein critical for visual cycle function, an open-label, single-center Phase 2 clinical trial of ADX‑2191 was performed in eight retinitis pigmentosa patients with rhodopsin misfolding mutations. Over three months of treatment with ADX-2191, four patients received monthly injections and four patients received twice-monthly injections. The primary endpoint of the clinical trial was safety. Secondary endpoints included change from baseline in visual acuity; retinal function, as assessed by macular and dark-adapted chromatic perimetry and electroretinography; and retinal morphology, as assessed by optical coherence tomography. Visual acuity, perimetry, and morphology assessments were performed monthly for four months from initiation of therapy. Electroretinography was performed at baseline and at 90 days from initiation of therapy.
All enrolled patients completed the trial per protocol. Relative to baseline, across all patients, statistical significance was achieved for improvement in best corrected visual acuity (P<0.0001), low-light visual acuity (P=0.0001), time to electroretinographic response to light (P=0.02), macular sensitivity to light (P<0.0001), and dark-adapted peripheral sensitivity to light (P<0.0001). ADX‑2191 was well tolerated, and no safety concerns were identified. No treatment-related adverse events associated with retinal morphology were observed. No serious adverse events were reported, and no patients discontinued due to adverse events.
“Retinitis pigmentosa is a relentlessly progressive disease that inevitably leads to loss of vision,” stated
ADX-2191 is a novel intravitreal formulation of methotrexate in clinical development for proliferative vitreoretinopathy and retinitis pigmentosa, both of which are rare, sight-threatening retinal diseases with no approved therapies. The prevalence of retinitis pigmentosa is more than one million people worldwide, and genetic mutations leading to rhodopsin misfolding account for approximately one-third of cases. ADX-2191 has been granted orphan drug designation by the
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1FASEB J. 34(8): 10146-10167, 2020.
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