Closely-held IntraBio reported positive topline results from a Phase 3 clinical trial, showing IB1001 significantly improved symptoms, functioning, quality of life, and cognition in pediatric and adult patients with Niemann-Pick disease Type C (NPC).
The primary endpoint of the trial evaluated the impact of IB1001 on the Scale for the Assessment and Rating of Ataxia, compared with placebo after 12 weeks.
The positive Phase 3 results are consistent with an earlier Phase 2b multinational clinical trial with IB1001 for NPC, which also showed a statistically significant and clinically meaningful improvement on the SARA, mSARA, and Investigator’s CGI-I endpoints and a benign safety profile.
Based on these positive trials, IntraBio will proceed with global regulatory submissions to the FDA, the European Medicines Agency, and other global regulators.
NPC is a very rare, inherited disease that causes damage to the nervous system over time and is always fatal. There is currently no approved therapy for NPC in the U.S.
“This is a critical breakthrough for the NPC patient community,” Dr. Marc Patterson, Prof. of Neurology, Pediatrics, and Medical Genetics at Mayo Clinic, said in a statement.
He said the trial of IB1001 shows clinically meaningful effects at a high level of statistical significance in patients with NPC. “I am hopeful that IB1001 will be rapidly approved to ensure that every NPC patient will be able to access safe and effective treatment for this devastating disease.”
