IntraBio Reports Positive Data from NPC Study

Rare Daily Staff

IntraBio reported positive data from its phase 2 multinational clinical trial of its experimental therapy IB1001 for the treatment of Niemann-Pick disease type C, a rare, metabolic disease.

Niemann-Pick disease Type C (NPC) is a lysosomal storage disorder. The disease begins in early childhood and presents with systemic, psychiatric, and neurological symptoms, including cerebellar ataxia. NPC is chronic and progressive in nature and is characterized by rapid degeneration of the cerebellum and major organ systems which severely impacts the quality of life. There is no approved treatment for NPC in the United States. Treatment of NPC in the European Union and several other countries is limited to substrate reduction therapy drug miglustat.

The study evaluated IB1001 for both the symptomatic and neuroprotective, disease-modifying treatment for adult and pediatric patients with NPC.  Patients aged 6 years and older were enrolled at trial sites in the United States, the United Kingdom, and the European Union.

The company said IB1001 demonstrated a statistically significant and clinically meaningful improvement in symptoms, functioning, and quality of life in both primary and topline secondary endpoints for both pediatric and adult patients with NPC. The trial met its primary endpoint, the Clinical Impression of Change in Severity (CI-CS), which was assessed by blinded, centralized raters.

The trial also met topline secondary endpoints, including the Scale for the Assessment and Rating of Ataxia (SARA) and the Investigators’ Clinical Global Impression of Change (CGI-C) assessment.

IB1001 was observed to be safe and well-tolerated, with no drug-related serious adverse events.

“The statistically significant and clinically meaningful response in primary and topline secondary endpoints with IB1001, together with its compelling safety profile and easy oral administration [sachet mixed with water], affirm the very favorable risk/benefit profile of IB1001 as a treatment for this devastating disease,” said Susanne Schneider, principal investigator, Ludwig Maximilian University of Munich. 

IB1001 is part of IntraBio’s platform of novel treatments to provide neuroprotection, disease modification, and symptomatic relief from multiple neurodegenerative and lysosomal storage diseases. The company is studying IB1001 in GM2 (Tay-Sachs and Sandhoff disease) and Ataxia-Telangiectasia (AT). The company is also preparing to initiate clinical trials with IB1001 for other rare and neurodegenerative diseases.

Photo: Susanne Schneider, principal investigator, Ludwig Maximilian University of Munich