ViGeneron Gains FDA Rare Pediatric Disease Status for VG901 and DSMB OK to Progress Phase 1b Retinitis Pigmentosa Trial

ViGeneron GmbH, a trailblazer in next-generation gene therapy, has announced significant progress for its therapy candidate, VG901, designed to combat retinitis pigmentosa (RP) caused by CNGA1 gene mutations. This innovative treatment has been awarded the Rare Pediatric Disease Designation (RPDD) by the U.S. Food and Drug Administration (FDA), and the independent Data Safety Monitoring Board (DSMB) has given the green light for dose escalation in its ongoing Phase 1b clinical trial.

The FDA's RPDD acknowledges the urgent need for treatments targeting rare pediatric conditions like RP due to CNGA1 mutations. This designation provides ViGeneron with the opportunity to qualify for a Priority Review Voucher (PRV) once VG901 secures marketing approval. A PRV allows for a faster FDA review process for any drug candidate within the company's pipeline, cutting down the review timeframe to six months, and can be transferred for use or sale.

Dr. Caroline Man Xu, Co-founder and CEO of ViGeneron, expressed that the FDA's recognition acknowledges the pressing need for medical solutions in the field of retinitis pigmentosa. It highlights VG901's potential as the first and only clinical-stage therapy aiming to address CNGA1-associated retinitis pigmentosa. This RPDD, alongside the previously granted FDA Orphan Drug Designation, helps propel the advancement of VG901.

The ongoing Phase 1b clinical trial is designed to assess VG901’s safety and initial effectiveness. Utilizing ViGeneron's next-generation vgAAV capsid, the therapy delivers the functional CNGA1 gene directly to retinal photoreceptor cells through intravitreal injections. The DSMB, comprised of independent experts, rigorously evaluates trial data after each patient cohort.

Dr. Bart P Leroy, Ophthalmologist & Clinical Geneticist, and DSMB Chair for the trial, shared that the DSMB has unanimously recommended proceeding with dose escalation. No dose-limiting adverse events linked to VG901 have been observed in the initial dose cohort, marking a pivotal step towards advancing to a higher dose and representing a crucial milestone in VG901's clinical development.

Retinitis pigmentosa (RP) encompasses a group of related eye conditions leading to progressive vision loss. It typically begins with night blindness during childhood or early adulthood, followed by peripheral vision loss, "tunnel vision," central visual impairment, decreased visual acuity, and eventually, total blindness. RP stands as the most prevalent type of inherited retinal diseases, affecting approximately 1 in 3,500 to 1 in 4,000 individuals in the United States and Europe. Mutations in the CNGA1 gene, which encodes a subunit of CNG channels in rod photoreceptors, are responsible for about 2%-8% of autosomal recessive retinitis pigmentosa (arRP).

VG901 is distinguished as a first-in-class and sole clinical-stage gene therapy targeting CNGA1-associated Retinitis Pigmentosa. It employs ViGeneron's proprietary vgAAV vector to deliver the CNGA1 gene via intravitreal injection. The therapy has shown in vivo functionality in mouse models and passed a GLP safety study with a six-month observation period verifying its safety, durability, and tolerability. VG901 is currently undergoing a Phase 1b clinical trial, which is an open-label, single-arm, dose-escalation study evaluating the safety, tolerability, and initial effectiveness of a one-time intravitreal administration.

ViGeneron GmbH is committed to pioneering gene therapy innovations to meet unmet medical needs. The company is advancing its proprietary gene therapy pipeline to treat ophthalmic diseases while partnering with prominent biopharmaceutical entities to address retinal diseases, CNS, cardiovascular, and other conditions. ViGeneron was founded in 2017 by a team with extensive expertise in AAV vector technology and clinical ophthalmic gene therapy programs and is based in Munich, Germany.