Company: NCEL
Filing Date: 2025-09-10
Form Type: 424B3
Source: 0001213900-25-086600
Chunk: 406

Company: NewcelX Ltd.
Filing Date: 2025-09-10
Form: 424B3
Chunk 406
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 Safety, Tolerability, And Effectiveness of Repeated Administration of Cryopreserved AstroRx ®in Patients with Amyotrophic Lateral Sclerosis (ALS).” This study will be conducted in participants with a sporadic or familial ALS, who meet the El Escorial criteria of probable, or definite criteria for a diagnosis of ALS, with disease onset of 18months or less, from first symptoms until screening. At screening, eligible participants must be between ages 18 -80years old, have an ALSFRS -Rscore ≥35, and a SVC ≥70% of predicted capacity for age, height and gender. Participants on a stable dose of riluzole and/or edaravone or PB (sodium phenylbutyrate)/TURSO (taurursodiol), and those not taking riluzole and/or edaravone or PB/TURSO at screening are eligible for inclusion as long as they meet specific protocol requirements. Approximately 30 adult participants will be enrolled into the double -blindstudy with a randomization ratio of 2:1 (20 participants in the AstroRx ®arm and 10 participants in the placebo arm). Kadimastem 209 began preparations for the clinical trial including extensive contact with key opinion leader and clinical centers in the United States and signing a tech -transferagreement with qualified Contract Development and Manufacturing Organization for the manufacturing of clinical grade AstroRx ®product. Kadimastem’s AstroRx ®has received orphan drug status from the FDA, a status given to certain drugs called orphan drugs, which show promise in the treatment, prevention, or diagnosis of orphan diseases. Kadimastem believes that the granting of orphan drug status for the AstroRx ®product is an acknowledgment of the uniqueness and medical potential of the product and will give a significant boost to its entry into the market once it’s approved for marketing. The FDA grants orphan drug status in order to provide an incentive for the development of drugs and medical treatments. Companies whose drugs have been awarded this status enjoy exclusive marketing rights for the drugs for seven years after marketing approval is obtained. This recognition is also useful in obtaining grants and financial concessions in development and registration of drugs for marketing. The FDA has authority to grant orphan drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition. Orphan drug designation qualifies sponsors for incentives including tax credits for qualified clinical trials, exemption from user fees, and potential seven years of market exclusivity after approval. Orphan drug status provides