On December 7, 2023, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (“ODD”) to NXL-004, an investigational AAV gene therapy product being developed by NeuExcell Therapeutics Inc. (“NeuExcell”), for the treatment of malignant glioma. This is the world’s first AAV gene therapy product for malignant glioma to receive FDA ODD designation.

Malignant gliomas are the most common primary malignant brain tumors in adults, about 60% of which are glioblastoma (GBM). Characterized by high malignancy and mortality, the current standard-of-care of GBM, encompassing surgery, radiotherapy, and chemotherapy, results in a median overall survival of merely 15-18 months and a 5-year survival rate below 10%. Recurrence is virtually inevitable for malignant glioma and there is no clear standard salvage therapy.

NXL-004 is an innovative treatment based on the groundbreaking astrocyte-to-neuron (“AtN”) conversion platform developed by Professor Gong Chen. It has shown good efficacy and safety in preclinical studies and will enter first-in-human clinical trials in early 2024. According to the U.S. Orphan Drug Act, drugs that have received ODD in the U.S. can enjoy a series of regulatory support, including but not limited to tax credits for clinical trial expenses, exemption of new drug application fees, and 7-year market exclusivity after product approval. ODD can help accelerate development of NLX-004 and provide new hope for many GBM patients around the world.

NeuExcell is committed to restore health and improve the quality of life for millions of patients worldwide suffering from neurodegenerative diseases and neurological injuries. NeuExcell’s disruptive AtN technology has the potential to regenerate neuronal tissues and restore neurological functions, with robust preclinical efficacy data in a wide variety of disease models including non-human primates. NeuExcell’s lead clinical programs include Alzheimer’s disease, ischemic stroke and GBM, all expected to enter the clinic in 2024, with pipeline expansion opportunities in numerous indications such as Huntington’s disease and Amyotrophic Lateral Sclerosis.

Source: https://www.prnewswire.com/news-releases/neuexcells-aav-gene-therapy-for-malignant-glioma-granted-orphan-drug-designation-by-fda-302017315.html

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