Phase 2 trial: ADX-2191 may improve retinal function in patients with retinitis pigmentosa
Key takeaways:
- ADX-2191 is an intravitreal formulation of methotrexate.
- Patients experienced improvement in visual acuity and other retinal functions.
Aldeyra Therapeutics announced positive results from a phase 2 trial of ADX-2191 for the treatment of retinitis pigmentosa.
According to a press release, the trial demonstrated improvement in retinal function across several physiological and psychological assessments compared with baseline.
“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,” Todd C. Brady, MD, PhD, president and CEO of Aldeyra, said in the release. “Based on compelling proof-of-concept clinical activity that is consistent with a well-defined mechanism of action supported by preclinical evidence, we are excited to meet with regulatory authorities to discuss initiation of a potentially pivotal phase 2/3 clinical trial, as we enthusiastically advance ADX-2191 to the next stage of development.”
ADX-2191 is an intravitreal formulation of methotrexate in clinical development for the treatment of retinitis pigmentosa, as well as proliferative vitreoretinopathy.
In the study, four patients with retinitis pigmentosa received a monthly injection of ADX-2191 and four received twice-monthly injections. Both groups received 3 months of treatment.
Relative to baseline, patients experienced improvement in best corrected visual acuity (P < .0001), low-light visual acuity (P = .0001), time to electroretinographic response to light (P = .02), macular sensitivity to light (P < .0001) and dark-adapted peripheral sensitivity to light (P < .0001).
The drug was well-tolerated and researchers identified no safety concerns, according to the release.
“Retinitis pigmentosa is a relentlessly progressive disease that inevitably leads to loss of vision,” Ramiro S. Maldonado, MD, principal investigator of the trial, said in the release. “The promising results presented today are supportive of a potential novel approach for the treatment of retinitis pigmentosa patients with rhodopsin mutations.”
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