AskBio Announces First Patient Dosed in Phase 1 / Phase 2 Trial of AB-1003 Gene Therapy for Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)

• Gene therapy developed to restore FKRP enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9.

RESEARCH TRIANGLE PARK, N.C. , Aug. 3, 2023 /PRNewswire/ -- Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the first patient has been dosed in the Phase 1 / Phase 2 LION-CS101 clinical trial of patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).¹ AB-1003 (also known as LION-101) is a novel investigational FKRP gene replacement therapy.

"Hearing that the first patient has been dosed in this study evaluating AB-1003 is an exciting moment for the limb-girdle muscular dystrophy community and individuals living with this debilitating disease," said Kelly Brazzo, Co-Founder & CEO, CureLGMD2i. "Given the current lack of disease modifying treatments for LGMD, many in the community know of the potential of gene therapy. The initiation of this trial offers hope that patients with this condition may, in the future, have a significantly improved quality of life."

LGMD2I/R9 is a form of LGMD caused by changes in the FKRP gene and is associated with weakness and wasting of arm and leg muscles.² Those with LGMD2I/R9 notice symptoms in late childhood, around 11 years of age, which may include loss of mobility and impaired heart and lung function.^2,3 As symptoms worsen, individuals generally require wheelchairs.² LGMD2I/R9 is a rare disease, estimated to affect more than 5,000 people in the US and EU.⁴

"While the inherited nature of limb-girdle muscular dystrophy means those with the FKRP gene mutation can't produce a normal FKRP protein for physiological muscle function, AB-1003 is designed to introduce the normal FKRP gene into the muscle and express a normal protein, and it has shown promise in restoring normal FKRP protein function in muscle in preclinical studies performed in mouse models of LGMD," said Nicholas Johnson, MD, Principal Investigator and Vice Chair of Research at the Department of Neurology, Virginia Commonwealth University School of Medicine. "This trial is the first step toward evaluating the safety of AB-1003 and assessing the potential that AB-1003 has to improve the lives of patients with this serious, inherited ultra rare condition."

"The first limb-girdle muscular dystrophy 2I/R9 patient dosed in the LION-CS101 clinical trial is another example of AskBio's success in taking gene therapy candidates, such as AB-1003, from the bench into clinical development," said Jude Samulski, PhD, Co-Founder and Chief Scientific Officer of AskBio. "With our knowledge and expertise, we are working to harness the power of gene therapies as potential treatments for many of the world's most devastating diseases. Our investigational therapy for limb-girdle muscular dystrophy is one of these and an important part of our portfolio, which also includes clinical stage investigational therapies for congestive heart failure, Huntington's disease, multiple system atrophy, Parkinson's disease, and Pompe disease."

AB-1003, which is manufactured by AskBio's wholly owned and independently operated subsidiary Viralgen, was granted fast track designation by the FDA and orphan drug designation by the European Commission.^5,6

Recruitment for the LION-CS101 clinical trial is ongoing.

About the LION-CS101 Clinical Trial

The LION-CS101 clinical trial is a Phase 1 / Phase 2, double-blind, randomized, placebo-controlled, dose-escalation trial to evaluate the safety of AB-1003 gene therapy in adult subjects (18-65 Years) who have genetic confirmation of LGMD2I/R9. Adults diagnosed with LGMD2I/R9 will be given a single intravenous infusion of AB-1003 or placebo. Participants will be treated in sequential, dose-level cohorts with two arms of different doses and one placebo arm. The trial will include up to 14 participants and was initiated in the US in Q2 2023.¹ For more information about the LION-CS101 clinical trial, visit clinicaltrials.gov (NCT05230459), or visit askbio.com.

About AskBio

Asklepios BioPharmaceutical, Inc. (AskBio), 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 therapeutics for congestive heart failure, Huntington's disease, 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 capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, Scotland, the company has generated hundreds of proprietary capsids and promoters, several of which have entered clinical testing. An early innovator in the gene therapy field, with over 900 employees in five countries, the company holds more than 800 patents and patent applications in areas such as AAV production and chimeric and self-complementary 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. Its 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 2022, the Group employed around 101,000 people and had sales of 50.7 billion euros. R&D expenses before special items amounted to 6.2 billion euros. For more information, go to www.bayer.com.

About Viralgen

Viralgen, an independently operated Contract Development and Manufacturing Organization (CDMO), was founded in 2017 as a joint venture between AskBio and Columbus Venture Partners (a venture capital firm based in Spain). As a leading manufacturer of cGMP certified recombinant AAV, Viralgen uses the Pro10^TM based suspension manufacturing platform, a technology licensed from AskBio and developed by Co-Founder R. Jude Samulski, PhD, at University of North Carolina. Located in Spain, in the Gipuzkoa Science and Technology Park, Viralgen produces AAV gene therapy treatments with the aim of accelerating the delivery of new medicines that can improve patients' lives.

AskBio Forward-Looking Statements

This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include without limitation statements regarding AskBio's clinical trials. These forward-looking statements involve risks and uncertainties, many of which are beyond AskBio's control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned clinical and regulatory milestones and timelines, its reliance on third-parties, clinical development plans, manufacturing processes and plans, and bringing its product candidates to market, due to a variety of reasons, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office. Any of the foregoing risks could materially and adversely affect AskBio's business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

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¹ A Study to Evaluate the Safety of LION-101 in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1). Available at: https://clinicaltrials.gov/ct2/show/NCT05230459. Accessed August 2023.
² National Institutes of Health — National Center of Advancing Translational Sciences: Genetic and Rare Diseases Information Center. Limb-Girdle Muscular Dystrophy Type 2I – Summary. Available at: https://rarediseases.info.nih.gov/diseases/12533/limb-girdle-muscular-dystrophy-type-2i. Accessed August 2023.
³ Chu ML, Moran E. The Limb-Girdle Muscular Dystrophies: Is Treatment on the Horizon? Neurotherapeutics. 2018;15(4):849-862.
⁴ Liu W et al. Estimating prevalence for limb-girdle muscular dystrophy based on public sequencing databases. Genet Med. 2019;21(11):2512-2520.
⁵ AskBio Receives FDA Fast Track Designation for LION-101, a Novel Investigational AAV Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9). Available at: https://www.askbio.com/askbio-receives-fda-fast-track-designation-for-lion-101-a-novel-investigational-aav-gene-therapy-for-the-treatment-of-limb-girdle-muscular-dystrophy-type-2i-r9-lgmd2i-r9/. Accessed August 2023.
⁶ AskBio Receives European Commission Orphan Drug Designation through its EU-Based subsidiary BrainVectis for AB-1003, a Novel Investigational AAV Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy (LGMD). Available at:  https://www.askbio.com/askbio-receives-european-commission-orphan-drug-designation-through-its-eu-based-subsidiary-brainvectis-for-ab-1003-a-novel-investigational-aav-gene-therapy-for-the-treatment-of-limb-girdle-muscular/. Accessed August 2023.

SOURCE AskBio