MeiraGTx Submits Prospectus Following Phase III Failure of Ophthalmic AAV Gene Therapy Bota-vec

May 07, 2025 12:01 CST Updated 12:01

Johnson & Johnson

Medical Device R&D and Manufacturer

Ophthalmic AAV Gene Therapy: A Major Focus in Innovative Drug Development in Recent Years

Unfortunately, as the project continues to advance, no more products have received regulatory approval, and globally, there remains only one.Luxturna, although a few pipelines have advanced to phase 3, many more have disappeared due to failures and various reasons.

Recently, Johnson & Johnson also announced that one of its ophthalmic AAV gene therapies did not meet the primary endpoint in Phase III clinical trials.

Purchased for $415 million

Johnson & Johnson's Ophthalmic Gene Therapybotaretigene sparoparvovec（bota-vec，AAV5-hRKp.RPGR), derived from Johnson & Johnson's subsidiary Janssen andMeiraGTx's collaborative assets achieved in 2019.

Later due tobota-vec shows potential in early clinical trials andClinical Progress of Bota-Vec,Johnson & Johnson invested up to $415 million in December 2013（$130 million upfront and near-term milestone payments + up to $285 million in additional funds after the first commercial sales of bota-vec in the U.S. and EU, as well as manufacturing technology transfer.The amount ofMeiraGTx Reaches Agreement, obtainedExclusive rights of bota-vec.

bota-vec is an AAV gene therapy that utilizesAAV5 CarryinghRKp.RPGR, throughSubretinalInjectionDelivered to target cells to treat causesX-linked Retinitis Pigmentosa (XLRP) Caused by RPGR Gene Mutation.It is worth noting that,Bota-Vec has obtained clinical approval in China and has beenIncluded in the list of breakthrough therapies.

Bota-vec announced in 2020The interim data from the Phase I/II trial showed that the low and medium dosesBota-vec treatment was well-tolerated and resulted in significant improvement in patients' vision.

Phase III Study of Bota-VecLumeos was launched in December 2020 and completed patient recruitment in 2023.Lumeos recruited a total of 95 patients, of which 58 patients received a single low dose or high dose.Bota-vec infusion.

Phase III Failure

According to the results published on May 2, 2025,The primary endpoint of the Lumeos study was not met and failed to show improvement.Visual Navigation Ability of XLRP Patients.

ButSeveral secondary endpoints of Lumeos showed improvement, includingPatient-reported vision and eye chart vision test scores. Although the p-values for these results are less than 0.05, Johnson & Johnson did not claim statistical significance but instead used the values for descriptive purposes only. According to data released by Johnson & Johnson, 22 out of 55 treated patients showed improvement in two or more endpoints, while no patients in the control group demonstrated improvement.

In terms of safety data, all recipientsAll patients receiving bota-vec experienced at least one adverse event, of which 86% were considered mild or moderate in severity, and 53% of patients experienced at least one event related toBota-vec-related adverse events.

Currently,The study is shown to be completed on clinicaltrials.gov.As of April 25th, Johnson & JohnsonPhase III follow-up study is still ongoing for patients involved in the initial late-stage trial.

A spokesperson for Johnson & Johnson stated that they are working to understand all the data, including the clinically relevant improvements shown in most secondary endpoints, as Johnson & Johnson is evaluating.Bota-Vec's Strategic Choices and Next Steps.

Summary

In fact, the development of ophthalmic AAV gene therapy is not easy.

Previously, Biogen purchasedTwo products acquired from Nightstar TherapeuticsOphthalmology AAV pipelines have also failed in later-stage clinical trials.

References:

1.https://www.fiercebiotech.com/biotech/jj-gene-therapy-fails-improve-visual-navigation-late-stage-rare-eye-disease-trial

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