Antaisens Pharma Completes First-in-Human Dosing in Domestic Pivotal IIT of FY015A, China’s First ASO Therapy for Spinocerebellar Ataxia Type 3 (SCA3)

Jun 22, 2024 12:15 CST Updated 12:15

ASOcura

Small Nucleic Acid Drug Developer

By ASOcura(hereinafter referred to as: ASOcura)The First Antisense Oligonucleotide Developed in China（ASO）Drug FY015A is used for the treatment of Spinocerebellar Ataxia Type 3.（SCA3）The IIT clinical study has successfully completed the first subject dosing recently.

Spinocerebellar Ataxia（spinocerebellar ataxia，SCA）It is a group of hereditary neurodegenerative diseases caused by genetic mutations, with over 100,000 patients in China. The degeneration and atrophy of the cerebellum, brainstem, and spinal cord are common features, with main clinical manifestations including cerebellar ataxia, reduced motor coordination, speech disorders, and swallowing difficulties. Currently, there are no effective therapeutic drugs available domestically or internationally, and clinical management primarily involves rehabilitation training and symptomatic treatment.

Among them, Spinocerebellar Ataxia Type 3（SCA3）It is the most common SCA subtype, accounting for about two-thirds of Chinese SCA patients. SCA3 is caused by an abnormal expansion mutation of the CAG trinucleotide repeat within the coding sequence of the AXTN3 gene, which generates a toxic protein containing polyglutamine.

FY015A addresses the root cause of diseases by correcting genetic defects and eliminates toxic fragments at the RNA processing level to prevent the production of toxic proteins. In transgenic animal experiments, FY015A efficiently inhibits toxic mRNA and demonstrates better safety compared to similar ASOs, showing strong drug-like properties and market development potential.

The primary purpose of this study is to evaluate the safety and tolerability of FY015A in patients with mild to moderate SCA3. With the dedicated collaboration and close coordination of the researchers at the clinical research center, the first subject was recently enrolled.Completed the first intrathecal injection.Currently, the clinical observation of the first subject after drug administration and related laboratory tests and other evaluation indicators all show that the drug has good safety and tolerability, with no serious adverse events occurring.（SAE）And drug-related adverse events（AE）; In terms of efficacy evaluation, the indicators such as the subject's and their family's self-reports and SARA scores all show that the subject's individual condition remains stable after medication. The researchers will continue to provide subsequent treatment and long-term follow-up observations for the subjects according to the trial protocol.

The successful completion of the first SCA3 subject dosing marks an important advancement in ASOcura's ASO technology; it represents a significant milestone for all SCA patients. In the future, ASOcura is expected to provide SCA patients with a safe and effective clinical treatment.

Beijing Penguin Home Cerebellar Atrophy Patient Care Center(Hereinafter referred to as: Penguin Home)It is caused by spinocerebellar ataxia.（SCA）Penguin House, a private non-enterprise organization registered with the Beijing Civil Affairs Bureau by patients and their families in 2018. Penguin House has been committed to advocating for China's spinocerebellar ataxia (SCA) community and providing services to SCA patients and their families in various aspects such as medical guidance, medical education, doctor-patient communication, and improving the quality of life for patients and their families. Since July 2023, ASOcura has started a comprehensive cooperation with Penguin House, leveraging the advantages of patient organizations through Penguin House. Adhering to the core principle of "patient needs" and clinical value orientation, ASOcura relies on its unique patented technology in the field of small nucleic acid drug development, internationally leading drug research technology, and a robust investment team to jointly promote the rapid translation of SCA3 drug development and explore clinical benefits for patients.

Founder of ASOcura and Director of FY015A, Professor Yimin Hua"This marks a qualitative leap for FY015A from animal experiments to clinical trials with the first subject dosed in this study, and we hope it will bring disease improvement and therapeutic benefits to more SCA3 patients." Nusinersen, a small nucleic acid drug that has saved the lives of tens of thousands of SMA patients worldwide.(Nusinersen, brand name Spinraza)The inventor, Professor Hua Yimin, stated that he and his team will fully promote the research on drugs for treating SCA, aiming to benefit more patients as soon as possible and fill the significant unmet clinical needs in the global SCA field.

Penguin Home, as the only non-profit organization in China dedicated to supporting patients with spinocerebellar ataxia, has been committed to the physical and mental health of patients with spinocerebellar ataxia. It also focuses on disseminating medical knowledge and the latest scientific research advancements related to spinocerebellar ataxia. This research was initiated by the investigators and Penguin Home, with support and funding from He Rui Venture Capital's SCA3 Industry Fund and Professor Hua Yimin's research team. We look forward to this IIT study benefiting more SCA3 patients while promoting the development of orphan drug research and clinical treatment. Additionally, it aims to provide societal value by exploring pathways for the expedited market entry of drugs that treat rare diseases, benefiting patients with spinocerebellar ataxia.

It is hoped that in the near future, the first drug in China for treating spinocerebellar ataxia can be launched as soon as possible. This will not only be a significant achievement in medical research but also the earnest hope of countless patients and their families. With the continuous advancement of clinical research, the future holds promise for opening a new chapter in the treatment of neurological diseases.

E.N.D

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