On 30 September, IntraBio announced positive results from their multinational clinical trial testing IB1001 in Niemann Pick Type C (NPC). NPC is a rare lysosomal storage disorder, which has ataxia as a symptom. IB1001 resulted in a clinically meaningful improvement of symptoms, including in the Scale for the Assessment and Rating of Ataxia (SARA) score. The treatment was observed to be safe and well-tolerated, with no drug-related serious adverse events. The trial took place in the USA, European Union and the UK.
IntraBio are currently testing IB1001 in a clinical trial for other neurological conditions, including ataxia-telangiectasia, and are planning trials in other neurodegenerative conditions.
“The positive outcome of the IB1001-201 clinical trial is an important step in bringing this promising treatment to patients as quickly as possible,” said Taylor Fields, Senior Vice-President, IntraBio.
Posted on 01/10/2020