NovelPharma, a bio-venture specializing in rare medicines, is set to officially begin clinical trials for a treatment for Sanfilippo Syndrome Type A (MPS IIIA).
On the 20th, NovelPharma announced that it received approval from the U.S. Food and Drug Administration (FDA) for the Phase 1 clinical trial application (IND) of ‘NP3011 (compound name GC1130A)’, a treatment for MPS IIIA co-developed with GC Green Cross.
NovelPharma is currently preparing for global clinical trials of NP3011. Starting with the FDA approval in the U.S., the company plans to initiate multinational clinical trials within the year in countries including Korea and Japan.
Sanfilippo Syndrome Type A is a hereditary lysosomal storage disease (LSD) characterized by the accumulation of heparan sulfate in the central nervous system, including the brain and spinal cord, causing progressive damage to the CNS. NP3011 is a therapeutic agent that directly delivers the heparan N-sulfatase enzyme, which is not expressed in patients, to the central nervous system using intracerebroventricular (ICV) administration. By breaking down and removing the accumulated heparan sulfate in the CNS, it is expected to improve brain lesions.
In Phase 1 clinical trials, the safety and tolerability of NP3011 will be evaluated in patients with Sanfilippo Syndrome Type A. Given the rarity of the disease, the target number of participants is small, which is expected to allow for quicker confirmation of Phase 1 trial results.
A company representative stated, “We have already identified candidate patients at clinical sites in the U.S., Japan, and Korea where participation has been discussed, so we expect to proceed with Phase 1 trials more rapidly. We will accelerate efforts aiming to enter Phase 2 one year after the start of Phase 1.”
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