Company: NCEL
Filing Date: 2025-05-16
Form Type: 20-F
Source: 0001213900-25-044868
Chunk: 210

Company: NewcelX Ltd.
Filing Date: 2025-05-16
Form: 20-F
Item: Item 4
Chunk 210
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S)”. 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 18 months or less, from first symptoms until screening.
At screening, eligible participants must be between ages 18-80 years old, have an ALSFRS-R score ≥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-blind study with a randomization ratio of 2:1 (20 participants in the AstroRx®arm and 10 participants in the
placebo arm). Kadimastem began preparations for the clinical trial including extensive contact with key opinion leader and clinical
centers in the United States and signing a tech-transfer agreement 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 faster testing tracks and responses from the FDA and other regulatory agencies. Orphan drug