IntraBio Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for IB1001 for the treatment of Niemann-Pick disease Type C (NPC). The FDA granted the application Priority Review, setting a Prescription Drug User Fee Act (PDUFA) target action date of September 24, 2024.
The NDA is based on data from a pivotal Phase 3 trial (IB1001-301) involving adult and pediatric patients with NPC. The trial met all primary and key secondary endpoints, demonstrating that IB1001 significantly improved neurological signs and symptoms, functioning, and quality of life compared to placebo. These findings were published in the New England Journal of Medicine on February 1, 2024.
The NDA also included data from a positive multinational Phase IIb trial of IB1001 for NPC, which also met its primary and secondary endpoints, showing improvement in symptoms, functioning, and quality of life in pediatric and adult patients with NPC with the drug being well-tolerated.
Clinical Trial Success and Endpoints
The Phase 3 trial (IB1001-301) evaluated the efficacy and safety of IB1001 in both adult and pediatric NPC patients. The primary endpoint focused on improvements in neurological manifestations of the disease. Key secondary endpoints included assessments of overall functioning and quality of life. The results indicated a statistically significant benefit in favor of IB1001 across these measures.
Expert Commentary
Sean Kassen, Director of the Ara Parseghian Medical Research Fund, expressed enthusiasm about the NDA filing acceptance, highlighting the high unmet medical need in NPC and the potential of IB1001 to improve the quality of life for affected individuals.
Mallory Factor, IntraBio’s Executive Chairman, stated that the FDA’s acceptance and priority review bring the company closer to delivering effective treatments to patient communities with high unmet needs. He expressed optimism that IB1001 will be approved and rapidly available for all NPC patients.
Professor Elizabeth Berry-Kravis from Rush University Medical Center commented, “We are tremendously excited to have potential new treatment options for our NPC patient community. Once approved, we intend to treat all our NPC patients who meet the labelling criteria with IB1001 and to carefully track long-term improvement relative to baseline with whatever therapies they are on to help elucidate the long-term effects.”
About IB1001
IB1001 (N-acetyl-L-leucine) is an orally administered therapy designed to be taken up by monocarboxylate transporters, which are expressed ubiquitously and deliver IB1001 to all tissues, including across the blood-brain barrier. Inside cells, IB1001's underlying mechanism of action is multi-modal and targets the major drivers of disease pathophysiology in rare and common neurological disorders.
Financial Support for Commercialization
IntraBio also announced the recent closing of an equity financing round, raising over $40 million to support the commercialization and launch of IB1001, contingent upon FDA approval.
