Shanghai, China, November 06, 2024 — Belief BioMed Inc. (hereinafter, "BBM"), a globally innovative biotech company focusing on cutting-edge gene therapies, announced today that the U.S. Food and Drug Administration (FDA) has recently granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to the company’s gene therapy drug BBM-D101 for the treatment of Duchenne Muscular Dystrophy (DMD).

This dual designation indicates that BBM-D101 has been positively acknowledged by U.S. regulatory authority for its potential clinical value.

By meeting both the ODD and RPDD criteria, BBM-D101 has become the company's third gene therapy to receive ODD and RPDD dual designation, following BBM-H901 and BBM-H803.

DMD is an X-linked recessive muscle disorder caused by mutations in the dystrophin gene. Globally, approximately 1 in every 5,000 boys is affected by DMD[1], and approximately 30% of cases are due to new mutations[2]. In the United States, the estimated patient population is fewer than 50,000[3]. Symptoms typically manifest before age 6, often during infancy, and most patients progressively lose their ability to walk between ages 10 and 12, ultimately succumbing to heart and/or respiratory failure around age 30[4]. This highlights a critical need for innovative treatment solutions.

BBM-D101, independently developed and produced by Belief BioMed, is a recombinant adeno-associated virus (rAAV) gene therapy, which uses engineered AAV as a vector to deliver the optimized gene expression cassette to muscle tissues throughout the body, in order to effectively treat DMD. The Investigator Initiated Trial (IIT) of this drug has officially started in July 2024.

[1] https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10330733/

[2] https://www.ncbi.nlm.nih.gov/books/NBK482346/

[3] https://rarediseases.info.nih.gov/diseases/6291/duchenne-muscular-dystrophy

[4] https://www.genome.gov/Genetic-Disorders/Duchenne-Muscular-Dystrophy

The FDA's Office of Orphan Products Development grants ODD to eligible rare disease drugs, including biologics. The main criteria for evaluation were: the disease associated with the drug affects fewer than 200,000 patients in the United States; the potential of the drugs for the prevention, diagnosis or treatment of rare diseases or conditions; there is a scientific basis for the drug's potential benefits. ODD qualifies sponsors for incentives including tax credits for qualified clinical trials, waiver of NDA or BLA application fees, and potential seven years of market exclusivity after approval.

RPDD is designed to incentivize the development of new drugs and biological products for serious and life-threatening rare pediatric diseases or conditions, and the FDA grants RPDD to drug treatments for serious or life-threatening diseases that affect fewer than 200,000 people in the United States and with manifestations primarily affecting individuals aged from birth to 18 years. The sponsor of an RPDD product who receives an NDA or BLA approval may qualify for a voucher that can be redeemed to receive priority review for a different product. The sponsor may also transfer or sell the voucher to another sponsor.

About Belief BioMed

Belief BioMed Inc. (BBM) is a global biotech company that integrates the research and development, manufacturing and clinical application of gene therapy products. The company is committed to providing innovative and more effective gene therapies for severe genetic and chronic diseases through safe and efficient viral vector technology. BBM has developed hundreds of key vector technologies, including HEK293 cell suspension serum-free culture process and full-scale chromatography purification process, and has established a commercial production platform for gene therapy drugs. The company has been building up its capabilities in a variety of fields including novel AAV capsids targeting different tissues, efficient transgene expression cassette design, and advanced clinically applicable vector manufacturing process. It has also established an extensive R&D pipeline covering a wide range of unmet clinical needs in different therapeutic areas such as hemophilia, DMD, Parkinson's disease, osteoarthritis, etc. Several product pipelines have entered clinical studies or submitted IND filings. The NDA of a gene therapy for the treatment of adult patients with hemophilia B, has been accepted by the NMPA of China. For more information, visit www.beliefbiomed.com.

Statement

This information is only for the purpose of introducing the company's event and information on that date, and is not intended to promote any company's products and/or services, nor should it be construed as providing any advice or recommendation on the selection of any drugs, medical devices and treatment options.

For information about any company products, diseases and/or treatments, please consult a healthcare professional.

BBM-D101 described herein has not been approved for marketing.