AskBio Advances Gene Therapy Trial for Limb-Girdle Muscular Dystrophy Type 2I/R9
AskBio Inc., a leading gene therapy company wholly owned by Bayer AG, has announced a significant milestone in its Phase 1/Phase 2 LION-CS101 clinical trial for the investigational gene therapy AB-1003. The trial, which aims to treat patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), has progressed with the dosing of the first participant in the second cohort. This advancement follows a favorable review by the Data Safety Monitoring Board (DSMB), which evaluated safety data from the first cohort.
Overview of the LION-CS101 Clinical Trial
The LION-CS101 trial is a double-blind, randomized, placebo-controlled, dose-escalation study designed to evaluate the safety of AB-1003 gene therapy in adult participants diagnosed with LGMD2I/R9. The trial includes two sequential dose-level cohorts, with adult participants between the ages of 18 and 65 who have a confirmed genetic diagnosis of LGMD2I/R9.
AB-1003, an investigational gene therapy, is administered via a single intravenous (IV) infusion. In the first cohort, participants were given either the gene therapy or a placebo. Following the successful safety review of this group, the DSMB recommended advancing to the second cohort, allowing the dosing of the first participant.
A Step Closer to Providing Hope for LGMD2I/R9 Patients
LGMD2I/R9 is a rare and debilitating form of muscular dystrophy that primarily affects the muscles of the shoulders, hips, and upper arms. There are currently no approved therapies for this condition, and its impact on patients and families is profound. This makes the advancement of the LION-CS101 clinical trial even more significant.
Dr. Nicholas Johnson, Principal Investigator and Vice Chair of Research at the Department of Neurology at Virginia Commonwealth University School of Medicine, expressed his optimism regarding the trial’s progress: “Dosing the first participant in the second cohort of the trial is positive news for people living with LGMD2I/R9. This is a rare and debilitating type of muscular dystrophy, and this advancement brings the LION-CS101 trial another step closer to completion.”
Milestone Achieved with DSMB Review
The DSMB, an independent group of experts responsible for monitoring the safety and progress of clinical trials, conducted a thorough review of the safety data from the first cohort of participants. Based on their evaluation, they recommended advancing to the second cohort, where participants will receive the same investigational treatment. This positive recommendation is a key milestone in the trial and reflects the confidence in the safety of AB-1003.
AskBio’s Chief Medical Officer, Dr. Canwen Jiang, expressed enthusiasm about the DSMB’s recommendation: “The dosing of the first participant in cohort two marks an important milestone for the trial as enrollment continues for LION-CS101. We are encouraged by the DSMB’s recommendation to advance our study, following their thorough assessment of AB-1003 in cohort one, and are excited to proceed with the second cohort.”
Rare Disease Designations and Unmet Need
AB-1003 has received several important designations from the U.S. Food and Drug Administration (FDA) due to the significant unmet medical need in LGMD2I/R9. These include Rare Pediatric Disease Designation (RPDD), Orphan Drug Designation (ODD), and Fast Track Designation (FTD). These designations provide AskBio with various benefits in the drug development process and highlight the urgency of finding a treatment for this rare condition.
Despite the progress being made, there is currently no approved therapy for LGMD2I/R9, further emphasizing the importance of this clinical trial. The trial not only aims to evaluate the safety of AB-1003 but also offers hope to those suffering from a disease that significantly impacts their quality of life.
What is AB-1003?
AB-1003 is an investigational gene therapy based on a recombinant adeno-associated virus (AAV) vector. It is designed to restore FKRP enzyme activity within muscle cells to treat LGMD2I/R9. The gene therapy aims to address the root cause of the disease by providing the necessary enzyme for muscle cells to function properly. AB-1003 is delivered as a one-time intravenous infusion and has shown promise in early-stage trials.
Although the gene therapy is still under investigation and has not been approved by any regulatory authority, the ongoing clinical trials are designed to evaluate its efficacy and safety, which will determine its potential as a treatment for LGMD2I/R9.
Looking Ahead: The Future of LGMD2I/R9 Treatment
The successful dosing of the first participant in the second cohort marks a critical step forward in the development of AB-1003 as a potential treatment for LGMD2I/R9. As the trial continues to enroll participants, AskBio remains committed to advancing its gene therapy research for this rare disease.
With its promising gene therapy and regulatory designations, AskBio is optimistic about the potential to provide a breakthrough treatment for individuals living with LGMD2I/R9. As the trial progresses, more data will be gathered, and the hope for a new, effective treatment for this debilitating disease grows stronger.
About Limb-Girdle Muscular Dystrophy Type 2I/R9
LGMD2I/R9 is a rare form of LGMD caused by mutations in the FKRP gene and is associated with weakness and wasting of arm and leg muscles.6 Those with LGMD2I/R9 notice symptoms in late childhood, typically around 11 years of age, and may experience difficulty running and walking. The symptoms gradually worsen over time, and affected people generally rely on wheelchairs for mobility and may experience impaired heart and lung function.5-7 LGMD2I/R9 is estimated to affect fewer than 5,000 people in the United States.6 Currently, there is no approved treatment, and management is based on the signs and symptoms present in each individual.5
About AskBio
AskBio Inc., a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications with a clinical-stage pipeline that includes investigational therapeutics for congestive heart failure, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease. AskBio’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive array of capsids and promoters. With global headquarters in Research Triangle Park, North Carolina, the company has generated hundreds of proprietary capsids and promoters, several of which have entered pre-clinical and clinical testing. An early innovator in the gene therapy field, with over 900 employees in five countries, the company holds more than 600 patents and patent applications in areas such as AAV production and chimeric capsids. Learn more at www.askbio.com or follow us on LinkedIn.
About Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2024, the Group employed around 93,000 people and had sales of 46.6 billion euros. R&D expenses amounted to 6.2 billion euros. For more information, go to www.bayer.com.
