Nobelpharma Attends Rare Disease Symposium 'SSIEM 2023'... "Presentation of Non-Clinical Results"

Rare drug specialist bio-venture Novelpharma announced on the 1st that it attended the SSIEM Annual Symposium 2023, hosted by the Society for the Study of Inborn Errors of Metabolism (SSIEM), and presented preclinical results of the Sanfilippo Syndrome Type A (MPS IIIA) treatment jointly developed with GC Green Cross.

The SSIEM Annual Symposium 2023 is the annual symposium held by SSIEM, a society dedicated to rare genetic metabolic diseases. This year, it was held from August 29 to September 1 at the International Convention Center (ICC) in Jerusalem, Israel.

The event was well attended by medical professionals and experts in the rare disease field from around the world. It provided an opportunity to share the latest treatments and clinical status related to rare diseases and to build networks for research and development.

At this symposium, Professor Yang Aram of the Department of Pediatrics at Kangbuk Samsung Hospital presented preclinical results of NP3011 (substance name GC1130A), an MPS IIIA treatment. The preclinical study using mice confirmed excellent efficacy of recombinant human heparan-N-sulfatase enzyme replacement therapy administered intracerebroventricularly for treating MPS IIIA.

A Novelpharma representative said, “Attending the world’s largest rare disease symposium hosted by SSIEM and presenting the preclinical results of the MPS IIIA treatment attracted industry attention. It was a great opportunity to highlight the excellence and innovation of our MPS IIIA treatment, which directly replenishes the deficient enzyme in the central nervous system through intracerebroventricular (ICV) administration.”

Meanwhile, Novelpharma is accelerating preparations for global clinical trials of the innovative MPS IIIA treatment, aiming to apply for clinical approval from the U.S. Food and Drug Administration (FDA) early next year.