Company: NCEL
Filing Date: 2025-02-05
Form Type: F-3
Source: 0001213900-25-010223
Chunk: 17

Company: NewcelX Ltd.
Filing Date: 2025-02-05
Form: F-3
Chunk 17
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urodegenerative diseases are degenerative diseases of the CNS. These diseases are characterized by massive mortality of neurons. The prevention of neurodegeneration represents one of today’s most significant unmet medical needs. The development of therapies that preserve neuron health present unique challenges, including an imperfect understanding of underlying biology and a lack of translation of activity observed in preclinical studies to results in clinical trials. Kadimastem believes that currently approved therapies for many neurodegenerative diseases are generally only symptom modifying and have demonstrated limited efficacy. Accordingly, Kadimastem believes there remains an urgent need for novel approaches to address most neurodegenerative diseases, especially for progressive and severe conditions such as ALS.

Currently, there are no United States Federal Drug Administration, or FDA, approved-treatments to stop the disease’s progression or prevent onset. There are two FDA-approved treatments which attempt to slow ALS progression; Riluzole and Radicava. There are a number of ongoing trials examining potential treatments for ALS.

ALS
is a multifactorial disease and therapeutic approaches should consider the multiplicity of pathophysiological mechanisms that underlie
motor neuron, or MN, degeneration in this disease. Though MNs are the main affected cells in the disease, there is involvement of malfunctioning
astrocytes in the pathogenesis and progression of ALS. This notion supports the rationale that transplantation of healthy human astrocytes
into the CNS of ALS patients may compensate for the malfunctioning astrocytes and rescue remaining MNs by supply supportive factors, clues
and functions. The product AstroRx® is composed of healthy human astrocytes derived from hESCs which allows ready scale-up
of large numbers of donor cells. AstroRx® cells are manufactured in compliance with GMP guidelines. Once manufactured, the cells are
resuspended in PlasmaLyte A (a physiological solution) to formulate the AstroRx® product. The product is loaded into a syringe and
delivered to the physician at point-of-care for intrathecal injection, or IT injection, into the CSF of ALS patient following minimally
invasive lumbar puncture, or LP.

Kadimastem’s Solution

The cell therapy Kadimastem
is developing to treat ALS, AstroRx®, is based on the transplantation of allogeneic glial cells (mainly cells that support the CNS,
or astrocytes) that have differentiated from pluripotent stem cells, thereby returning to the recipient the motoneuron-supporting environment.
It is