REGENXBIO Launches Pivotal Phase IIb/III Trial for Diabetic Retinopathy Treatment

REGENXBIO Announces Pivotal Program for Surabgene Lomparvovec in Diabetic Retinopathy

REGENXBIO Inc., a leading biotech company in gene therapy, has declared a major advancement in the treatment of diabetic retinopathy (DR) by launching a pivotal Phase IIb/III clinical trial for surabgene lomparvovec (sura-vec, ABBV-RGX-314). This investigational treatment will employ a novel suprachoroidal delivery method, building on the promising results observed in previous trials.

Clinical Background

Diabetic retinopathy is a significant public health issue, being the leading cause of blindness among adults aged 24 to 75 worldwide. In the United States, nearly 10 million people suffer from this condition, leading to various complications that can ultimately threaten vision. Existing treatment options have primarily involved anti-VEGF therapies or retinal lasers, but these options may not be sufficient for all patients, indicating a pressing need for new interventions.

The recent announcement follows encouraging findings from the Phase II ALTITUDE® trial that revealed a durable safety and efficacy profile for sura-vec over a two-year follow-up period. The results suggested that a single in-office injection was effective for those with non-proliferative diabetic retinopathy (NPDR), without any serious drug-related adverse effects over the course of two years. These data lay the groundwork for the initiation of the global clinical program targeting DR.

The Pivotal Trial Details

The upcoming trial is specifically a two-part placebo-controlled study set to assess the effectiveness of sura-vec. The primary goal is to achieve a > 2-step improvement in the Diabetic Retinopathy Severity Scale (DRSS) at the one-year mark. Currently, site selection for the trial is underway. The collaboration with AbbVie, which enhances REGENXBIO's capabilities in advancing this therapy, includes a crucial milestone structure: a $100 million payment will be made to REGENXBIO upon the first subject being dosed in the pivotal Phase IIb/III trial, with an additional $100 million upon dosing in a subsequent Phase III trial.

Commitment to Patients

Dr. Steve Pakola, Chief Medical Officer at REGENXBIO, expressed excitement about the advancement of their drug development program aimed at addressing the unmet needs of millions of individuals living with DR. Dr. Primal Kaur, Senior Vice President at AbbVie, emphasized the necessity of these developments in a field where patients experience progressive disease leading to vision-threatening events.

The Technology Behind Sura-vec

Sura-vec employs the NAV® AAV8 vector, designed to carry an antibody fragment that inhibits vascular endothelial growth factor (VEGF). This action aims to prevent the growth of new and leaky blood vessels that contribute to fluid buildup in the retina, addressing one of the root causes of diabetic retinopathy.

REGENXBIO’s commitment to gene therapy represents a beacon of hope for patients who currently face limited treatment options. As the company progresses toward their pivotal trial, the implications of sura-vec could reshape diabetes management by providing an effective one-time treatment for a degenerative condition that affects so many lives.

Conclusion

In summary, REGENXBIO's initiation of the pivotal clinical trial for surabgene lomparvovec marks a promising turn in the treatment landscape for diabetic retinopathy. The results from the ongoing ALTITUDE trial and increased collaboration with AbbVie showcase the potential for substantial advancements in patient care and outcomes in the near future. As gene therapy continues to evolve, it may pave the way for new hope for countless individuals battling diabetic retinopathy and similar diseases.

For more information about REGENXBIO and its ongoing projects, visit REGENXBIO's official website.