REGENXBIO Inc. has unveiled plans to launch a pivotal phase IIb/III clinical trial focusing on surabgene lomparvovec (sura-vec, ABBV-RGX-314) for the treatment of diabetic retinopathy (DR), using suprachoroidal delivery. This significant development comes on the heels of promising two-year data from the phase II ALTITUDE trial and its follow-up study, paving the way for a global clinical program targeting DR. The initiative aims to introduce a potentially groundbreaking treatment for the vast number of individuals grappling with DR, as highlighted by Dr. Steve Pakola, the company’s Chief Medical Officer.
Diabetic retinopathy is a progressive ailment that often culminates in vision-threatening events, making it a major cause of blindness among working-age adults worldwide. By venturing into late-stage development, REGENXBIO is striving to unlock the transformative potential of sura-vec for DR patients. The company’s collaboration with AbbVie has been instrumental in propelling this clinical advancement forward, with a focus on addressing the unmet needs of individuals dealing with the complexities of DR. The upcoming phase IIb/III trial, bolstered by positive outcomes from the ALTITUDE trial, will have a primary endpoint of achieving a >2-step DRSS improvement at the one-year mark.
An integral part of this progression is the amendment to the collaboration and license agreement between REGENXBIO and AbbVie, which includes a revamped milestone structure for the DR program. AbbVie has committed to significant financial investments, with an immediate payment of $100 million to REGENXBIO upon the dosing of the first subject in the phase IIb/III trial, followed by an additional $100 million after the initiation of a second phase III clinical trial. Furthermore, AbbVie will independently fund and manage a new phase III ACHIEVE trial focusing on wet age-related macular degeneration (AMD), aimed at assessing the potential benefits of sura-vec over standard care in reducing injection frequency and preserving long-term vision.
Sura-vec, based on the NAV AAV8 vector, is designed to inhibit vascular endothelial growth factor (VEGF) and thereby impede the growth of leaky blood vessels that contribute to retinal fluid accumulation. This investigational therapy holds promise not only for DR but also for chronic retinal conditions like wet AMD, positioning it as a potential one-time treatment option. DR’s significant impact on global vision health underscores the urgency of advancing innovative therapies like sura-vec, particularly in a landscape where treatment options are limited and often involve ongoing monitoring or invasive procedures.
In conclusion, REGENXBIO’s strategic move towards the pivotal phase IIb/III clinical trial of surabgene lomparvovec for diabetic retinopathy marks a crucial step in the quest for novel and effective treatment modalities for this visually debilitating condition. By leveraging gene therapy and innovative delivery mechanisms, the company aims to offer new hope to millions of individuals grappling with DR worldwide. The collaboration with AbbVie and the comprehensive clinical development plan underscore the commitment to addressing the unmet medical needs of patients while pushing the boundaries of therapeutic innovation in ophthalmology.
Key Takeaways:
– REGENXBIO’s initiation of a pivotal phase IIb/III clinical trial for surabgene lomparvovec in diabetic retinopathy signifies a significant step towards addressing the unmet medical needs of patients worldwide.
– The collaboration between REGENXBIO and AbbVie, coupled with significant financial investments, highlights the commitment to advancing innovative therapies for retinal conditions like DR and wet AMD.
– Sura-vec, an investigational therapy based on the NAV AAV8 vector, shows promise in inhibiting VEGF and addressing the underlying mechanisms contributing to retinal complications in diabetic retinopathy and other chronic retinal conditions.
– The strategic focus on late-stage development and global clinical programs underscores REGENXBIO’s dedication to maximizing the value and impact of surabgene lomparvovec for patients living with diabetic retinopathy.
Tags: gene therapy
Read more on pharmabiz.com