Sentynl Therapeutics, Inc., a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences, Ltd., is pleased to announce the publication of NULIBRY’s three clinical studies comparing the treatment effect of NULIBRY versus a natural history study in the Journal of Inherited Metabolic Disease. Fosdenopterin gained its approval by the United States Food and Drug Administration (FDA) in February 2021, the Israeli Ministry of Health (MoH) in July 2022, the European Medicines Agency (EMA) in September 2022, and the Medicines and Healthcare products Regulatory Agency (MHRA) in April 2024 for the treatment of MoCD Type A.
These data represent the first comprehensive body of clinical research in the only approved treatment for MoCD Type A patients, as well as the most abundant findings on the natural progression of the disease. The results showed statistically significant prolonged survival in treated versus untreated patients, as well as genotype-match controls with the risk of death at 5.1 times higher in the untreated patients (Cox proportional hazards 5.1; 95% CI 1.32-19.36; p=0.01). Additionally, significant improvements were seen in cognitive and motor functioning in the treated versus the untreated patients.
Fosdenopterin-treated patients experienced mild to moderate treatment emergent adverse events with the most common adverse events being catheter-related complications and infections. There were no discontinuations or dose modifications due to adverse events.
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MoCD Type A is an ultra-rare, autosomal recessive disease caused by variants in the MOCS1 gene. It typically presents at birth or shortly thereafter and if left untreated causes rapid, irreversible neurogeneration due to a build up of toxic sulfite levels in the brain, and ultimately a premature death. Fosdenopterin, a synthetic form of cPMP, restores normal sulfite levels thereby reducing the toxic sulfite concentration.
“This publication marks the end of a truly collaborative and enthusiastic path from basic research to the bedside that started 21 years ago, when we reported the successful treatment of a mouse model of MoCD type A with cPMP (cyclic pyranopterin monophosphate, fodenopterin),” said Guenter Schwarz, Professor, Institute of Biochemistry, Department of Chemistry and Biochemistry & Center for Molecular Medicine, Cologne University, Germany. “Increased awareness and expeditious testing upon suspicion of MoCD are critical to improve neurological outcomes and overall survival in patients with MoCD Type A who can be treated early with fosdenopterin.”
“It is important that clinicians consider the diagnosis of MoCD type A in neonates with intractable seizures, encephalopathy, feeding difficulties or increased startle response as earlier treatment appeared to improve achievement of developmental milestones,” said Liza Squires, MD, Sentynl Medical Consultant.
Sentynl Therapeutics is committed to treating rare genetic diseases, which often present in infancy and early childhood. “The publication of these data in JIMD is a huge milestone for MoCD Type A patients, caregivers, and the medical community,” said Matt Heck, CEO, Sentynl. “It is the culmination of four companies’ commitment in NULIBRY’s clinical development program spanning over two decades.”
Source: Businesswire