Full 12-month data from Ocugen’s phase 2 eye disease gene therapy show the therapy significantly reduces the size of lesions, though the treatment’s smaller effect compared to an earlier preliminary analysis sent the biotech’s stock down a tad.
One year after an injection of OCU410, patients with geographic atrophy given the now-planned phase 3 dose had a 31% average reduction in their lesion size, Ocugen reported March 24. That winning dose is 500 billion vector genomes per milliliter, the medium dose used in the study.
While that 31% drop was enough to meet the trial’s primary endpoint—and surpassed the lesion-reduction ability of two approved therapies—it wasn’t as high as the 54% seen in a January preliminary analysis.
Ocugen reported “no serious adverse events and no adverse events of special interest” in the phase 2 trial. The Pennsylvania-based biotech now plans to launch a phase 3 trial of the candidate later this year, according to the release.
The phase 3 trial is planned to enroll up to 300 patients and have “an adaptive design powered at over 95%,” Ocugen Chief Medical Officer Huma Qamar, M.D., said in the release. OCU410’s phase 2 trial enrolled 51 patients.
Investors seemed miffed that OCU410 failed to maintain the efficacy it delivered in the earlier interim analysis, sending Ocugen’s share price down from $2.10 at yesterday’s close to $1.89 at 10:15 a.m. ET.
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OCU410 is an AAV-based therapy that delivers the gene for retinoid-related orphan receptor alpha, a key stress-regulating receptor. Geographic atrophy is a serious condition that occurs in patients with dry age-related macular degeneration (AMD), causing damage to the retina over time that steadily leads to vision loss.
Apellis Pharmaceuticals was the first to snag FDA approval for a geographic atrophy drug in 2023 with C3 inhibitor Syfovre, followed closely by Astellas’ Izervay. These two meds are given as frequent injections, in contrast to OCU410’s planned one-time administration.
“In addition to the strong efficacy and safety data, OCU410 has the potential to eliminate the chronic treatment burden associated with monthly or every-other-month intravitreal injections and to reduce treatment attrition driven by patient fatigue,” Lejla Vajzovic, M.D., an ophthalmologist at Duke University and chair of Ocugen’s retina scientific advisory board, said in the release.
Ocugen is hardly the only company pursuing gene therapies for AMD. Other candidates include AbbVie and Regenxbio’s sura-vec, now in two pivotal trials, and Eli Lilly’s phase 3 ixo-vec, picked up from the acquisition of Adverum Biotechnologies last year.
In January, U.K. biotech Ikarovec teamed up with delivery specialist VectorBuilder to develop a gene therapy meant to be given right in a doctor’s office.
