Innostellar Biotherapeutics Presents One-Year Clinical Data of LX102 Gene Therapy for AMD at Angiogenesis 2023

On February 11, Beijing time, Dr. Li Tong from the National Clinical Research Center for Ocular Diseases was invited by Dr. Philip Rosenfeld, an internationally authoritative ophthalmology expert, to deliver a special report at the Angiogenesis, Exudation, and Degeneration 2023 conference on the one-year clinical research results of LX102 gene therapy for AMD independently developed by Innostellar Biotherapeutics.

The conference provided a detailed introduction of the 1-year follow-up results of the IIT (Investigator-Initiated Trial) study for Innostellar Biotherapeutics' LX102. All subjects no longer required additional anti-VEGF treatments, and the visual acuity of the studied eyes improved compared to baseline. The improvement in vision was similar to that of existing antibodies or fusion proteins. Disease activity in all studied eyes was effectively suppressed, achieving the significant milestone of single-dose administration with zero rescue therapy! The average age of this group of patients was 71 years. Before receiving LX102 treatment, they had undergone an average of 15 anti-VEGF treatments, with an annualized frequency of 3-7 anti-VEGF treatments.

Age-related Macular Degeneration (AMD) is one of the most common blinding eye diseases in the elderly. Neovascular AMD, also known as wet Age-related Macular Degeneration (wAMD), can cause severe acute vision loss. Although it accounts for only 10%-20% of AMD cases, it is responsible for 90% of AMD-related blindness. The current standard treatment typically involves intravitreal injections of anti-VEGF drugs at intervals of one or two months, with patients requiring an average of 6-12 injections per year, some needing lifelong treatment. Interruptions or insufficient treatment lead to reduced long-term efficacy and irreversible vision loss. As a technological breakthrough, gene therapy is expected to significantly reduce the injection frequency required for wAMD patients and may become a first-line maintenance therapy for wAMD in the future.

Since 2021, Innostellar Biotherapeutics has collaborated with the National Clinical Research Center for Ocular Diseases to conduct an Investigator-Initiated Trial (IIT) on the use of LX102 in gene therapy for wAMD, making it the first gene therapy drug in China for this indication to be used in humans. Although the number of cases in this one-year follow-up data is small, all patients have shown good and stable anti-VEGF efficacy lasting up to one year after a single dose. Compared to similar drugs abroad, this product has demonstrated good safety and efficacy at lower doses. On January 18, the Phase I/II clinical study of LX102 held its kick-off meeting at the National Clinical Research Center for Ocular Diseases, and patient enrollment was initiated. Innostellar Biotherapeutics looks forward to enabling patients to benefit more from anti-VEGF treatment through gene therapy, achieving long-term stability and maintaining a normal quality of life with a single injection.

The Angiogenesis Conference is an authoritative meeting in the field of international ophthalmology and retinal disease new drug development. Hosted by the Bascom Palmer Eye Institute of the University of Miami Miller School of Medicine, the top-ranked ophthalmology hospital in the U.S. for 15 consecutive years, since 2004, it gathers the latest clinical research results through multiple rounds of selection by experts in the field to deliver keynote reports. The conference focuses on the diagnosis and development of new therapies for ocular neovascularization, exudative, and degenerative diseases. Attendees include outstanding scientists and clinical experts from various fields of ophthalmic new drug development and research, such as the National Eye Institute, the top ten ophthalmology centers in the U.S., and globally renowned new drug development companies. With the groundbreaking results of several major ophthalmic products being released first, including Regeneron's Aflibercept, Novartis' Brolucizumab, Roche's Faricimab, and Apellis’ APL-2, its scale and influence have continued to expand globally, making it a core forum for the release and discussion of pre-market ophthalmic new drug research findings.

Professor Philip Rosenfeld, as an authoritative expert in retinal diseases in the United States, is the principal investigator and research project leader for multiple clinical trials on age-related macular degeneration (AMD). He has played a decisive role in clinical trials of anti-vascular endothelial growth factor (VEGF) therapies, as well as in the diagnosis and disease management of macular diseases using OCT. He is considered one of the leaders in new drug development and translational clinical research in the field of ophthalmology. Throughout his career, Dr. Philip Rosenfeld has received numerous awards, including the Senior Achievement Award from the American Academy of Ophthalmology (AAO), the Richard and Hinda Foundation Award from the Macula Society, and the J. Donald Gass Award from the Retina Society.

Ophthalmic gene therapy has developed rapidly on the international stage in recent years. Innostellar Biotherapeutics' invitation to participate in the Angiogenesis conference is yet another testament to its leading position in the field of ophthalmic gene therapy. Innostellar Biotherapeutics has long been reputed for its deep understanding and accumulated expertise in clinical-grade AAV production processes, as well as its extensive clinical experience in treating ophthalmic diseases.

About LX102 Injection

LX102 Injection, independently developed and manufactured by Innostellar Biotherapeutics, is a gene therapy with a clear mechanism of action and proven efficacy for nAMD. It delivers DNA expressing an anti-VEGF fusion protein into retinal cells via an AAV vector, ensuring long-term expression of anti-VEGF protein within the patient’s eye. In investigator-initiated clinical studies conducted previously, LX102 demonstrated good tolerability and long-term efficacy in improving vision in multiple patients. LX102 received its IND approval on December 23, 2022.

About Innostellar Biotherapeutics

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

Since 2021, the company has put more than 5,000 square meters of space into operation, establishing R&D and clinical research centers in Zhangjiang, Pudong, Shanghai; a pilot and production base located in Suzhou Biomedical Industry Park has built an internationally leading serum-free suspension culture process production platform.

Innostellar Biotherapeutics has chosen ocular disease products as the breakthrough point for gene therapy, developing safe and precise gene replacement and gene editing technologies to achieve clinical applications of gene therapy. The company has established a research and development team and platform production technology for gene therapy across multiple systemic diseases. Gene therapy products for treating Leber Congenital Amaurosis (LCA), LX101, and for treating wet Age-related Macular Degeneration (wAMD), LX102, have both demonstrated good safety in clinical studies, with efficacy showing improved vision in patients. Currently, Innostellar's team has completed drug molecule screening and validation for multiple pipelines, two of which have successfully obtained clinical trial approvals from the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA). LX101 is the first gene therapy product for hereditary retinal degeneration in China to receive a clinical trial approval. The company focuses on the field of gene therapy for hereditary and chronic ocular diseases, possessing a foundation for the development and industrialization of various pipeline products. It closely collaborates with clinical medical scientists from renowned hospitals both domestically and internationally, enhancing innovation capabilities to develop gene therapy products more efficiently, ultimately applying them in clinical settings.