Innostellar Biotherapeutics Announces Completion of Phase III Patient Enrollment for LX101 Gene Therapy in Inherited Retinal Dystrophy

July 30, 2024, Shanghai —— Innostellar Biotherapeutics Co., Ltd. announced that its gene therapy candidate drug LX101 for the treatment of inherited retinal dystrophy (IRD) has successfully completed patient enrollment for the entire clinical Phase III trial.This important milestone marks a critical step forward for LX101 on its path to market entry.

LX101 is an adeno-associated virus (AAV) vector-basedGene Therapy DrugsThe drug, through adeno-associated virus type 2 (AAV2) carrying the normal RPE65 gene, can specifically recognize and transfect the diseased retina.Membrane pigment epithelial cells, and the continuous high-efficiency expression of the RPE65 protein, which the patient lacks, restores the normal visual cycle to achieve the therapeutic goal of improving visual function.Since entering clinical trials for the first time in 2021LX101 has demonstrated good safety and efficacy in early clinical trials, laying a solid foundation for subsequent Phase III clinical trials.。

LX101 Phase III Clinical Trial (STAR) is a multicenter, randomized controlled clinical trial., aiming to evaluate the efficacy and safety of LX101 in patients with RPE65 biallelic mutation-associated inherited retinal dystrophy (IRD).. The trial was conducted at five clinical research centers in China. Study endpoints included visual function improvement, safety, etc.

Completion of Patient Enrollment in the Registrational Clinical Trial Marks an Important Milestone in the Development of LX101. This not only reflects the high recognition from the ophthalmology community but also lays a solid foundation for subsequent data analysis and new drug application. According to the trial design, all patients will undergo at least one year of follow-up to comprehensively evaluate the long-term efficacy and safety of LX101.

Innostellar Biotherapeutics Founder and CEO Fenghua Wang stated: "The completion of patient enrollment in the LX101-III trial is the result of years of effort and perseverance by the team. We are one step closer to bringing a revolutionary treatment to patients with congenital retinal dystrophy.". As developers, we sincerely thank all the patients and researchers for their trust, and have also witnessed significant improvements in patients after receiving treatment.We look forward to the early market launch of LX101, benefiting more patients worldwide."

With the successful completion of the Phase III clinical trial, Innostellar Biotherapeutics will continue to focus on the subsequent development and commercialization of LX101. At the same time, the company will also accelerate the progress of other gene therapy projects under research, striving to achieve breakthroughs in more fields of genetic and chronic disease treatment, providing innovative therapeutic solutions for patients worldwide.

About Innostellar Biotherapeutics

Innostellar Biotherapeutics Co., Ltd. is dedicated to the research, development, and manufacturing of innovative gene therapy drugs. Established in 2020, the company focuses on addressing critical unmet needs in the treatment of genetic disorders and chronic diseases, building a robust foundation for the development of a multi-pipeline product portfolio.

The company focuses on ocular disease products as a breakthrough point for gene therapy, developing safe and precise gene replacement and gene editing technologies to achieve the clinical application of gene therapy. It has established a research and development team and platform production technology for gene therapy across multiple disease systems. Two products have officially received IND approval, both showing good safety and efficacy trends in current clinical trials. These are used for treating RPE65 mutation-associated inherited retinal dystrophy (IRD) LX101, developed by Innostellar Biotherapeutics, is the first IRD gene therapy product in China to have received clinical trial approval, with over 1 year of follow-up completed for 15 patients. The company focuses on gene therapy for hereditary and chronic eye diseases, with a strong foundation in the development and industrialization of multiple pipeline products. Collaborating closely with renowned clinical medical scientists both domestically and internationally, the company aims to enhance innovation capabilities and develop gene therapy products with greater efficiency and superior quality for clinical application.