Positive Results from Repligen Corporation

Repligen Corporation introduced positive outcomes from a Phase 1 survey to assess the pharmacokinetic (PK) and security profile of RG3039, a brand new small molecule medication nominee for potential remedy for spinal muscular atrophy (SMA). SMA is truly an inherited neurodegenerative disease wherein indicators of massive damage to motor neurons including loss of muscular perform generally appear very early in life and often improvement to acute physical disability and early on loss of life.

The Phase 1 trial has been a blinded, ascending, single dose study of RG3039 taken to 32 healthy people. The investigation results show that in fact RG3039 was well accepted at all doses administered, without serious adverse effects confirmed. The data also indicated evidence of a dose-related drug answer leading to 90% impediment of a typical target enzyme. Each of these outcomes could help to set up appropriate RG3039 dosing regimens for future research, such as potential efficacy studies in SMA affected individuals.