Sentynl Therapeutics Announces Presentation of Study Evaluating the Efficacy and Safety of NULIBRY® (fosdenopterin) for the Treatment of MoCD Type A at 2023 SSIEM Annual Symposium

07 Sep 2023
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Drug ApprovalClinical ResultAcquisition
Risk of death was 5.1 times higher in untreated patients versus patients treated with NULIBRY
Treatment also led to improvements in urinary biomarker levels and development outcomes
NULIBRY is the first therapy to reduce the risk of mortality in patients with known or presumed MoCD Type A
SOLANA BEACH, Calif., Sept. 7, 2023 /PRNewswire/ -- Sentynl Therapeutics, Inc. (Sentynl), a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare diseases, announced that data evaluating NULIBRY® (fosdenopterin) for the treatment of patients with molybdenum cofactor deficiency (MoCD) Type A were presented at the 2023 Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Meeting on Aug. 30 in Jerusalem. NULIBRY is indicated to reduce the risk of mortality in patients with MoCD Type A, an ultra-rare, autosomal recessive, inborn error of metabolism causing sulfite-induced neurodegeneration and early death.
"Clinicians must consider MoCD in infants with encephalopathy or intractable seizures," said Dr. Guenter Schwarz, Professor for Biochemistry at the Department of Chemistry & Center for Molecular Medicine, Cologne University, Germany. "Early treatment with fosdenopterin (NULIBRY) improves survival and neurodevelopmental outcomes, reducing burden on patients and caregivers."
In comparison of three non-randomized retrospective and prospective open-label studies versus a retrospective and prospective natural history study, the authors reported the following survival and clinical outcomes of patients treated with recombinant cPMP/NULIBRY:
Risk of death was 5.1 times higher in untreated MoCD Type A patients compared to those treated with rcPMP/NULIBRY (Cox proportional hazards [95% CI]=5.1 [1.32, 19.36], P=0.0144).
Treatment with rcPMP/NULIBRY led to a normalization of MoCD Type A-associated urinary biomarker levels.
Treated patients were more likely than untreated patients to feed orally and meet growth and developmental milestones.
"These results were pooled from a series of carefully designed studies and corroborate previous anecdotal findings. Early treatment of children with typical neonatal disease manifestation has a dramatic effect on their survival and overall development," said Dr. Bernd C. Schwahn, Consultant in Paediatric Metabolic Medicine, Manchester Centre for Genomic Medicine, St. Mary's Hospital, in Manchester, United Kingdom. "Increased disease awareness and the use of new technologies, including improved biochemical testing and rapid whole genome sequencing, will help to shorten the diagnostic gap and allow timely access to effective treatment for a higher proportion of affected children."
Based on these data, NULIBRY was approved by the U.S. Food and Drug Administration, the European Medicines Agency and the Israeli Ministry of Health. To further evaluate the safety and efficacy of NULIBRY, a post-approval non-interventional study is being initiated in Europe.
"Given Sentynl's focus on meaningful treatments for serious rare diseases, we are grateful to the authors for presenting these data and increasing awareness of MoCD Type A in the medical community," said Matt Heck, CEO of Sentynl. "We thank the patients, their families and caregivers, and all investigators involved in this study. We remain focused on continued evaluation of NULIBRY to reduce the risk of mortality and progression of this devastating disease."
Treatment with NULIBRY/rcPMP was generally well-tolerated in patients with MoCD Type A. Most patients experienced a treatment-emergent adverse event (TEAE), the majority of which were catheter-related or infections and were mild to moderate in severity. In the retrospective study, two serious TEAEs of death occurred, one from RSV pneumonia (unrelated to treatment), and one from necrotizing enterocolitis (possibly related to treatment).
Please click here for full Prescribing Information of NULIBRY.
About Molybdenum Cofactor Deficiency (MoCD) Type A
MoCD Type A is an autosomal recessive, inborn error of metabolism caused by mutations in the molybdenum cofactor synthesis 1 gene and characterized by a deficiency in molybdenum cofactor production, leading to a lack of molybdenum-dependent enzyme activity.1,2 The lack of activity leads to decreased sulfite oxidase activity with buildup of sulfite and secondary metabolites (such as S-sulfocysteine) in the brain, which causes irreversible neurological damage.2
MoCD Type A is an ultra-rare disease. The estimated prevalence of MoCD Type A in the European Union is 0.005 per 10,000. Based on these estimates, MoCD Type A is likely to be underdiagnosed.
The most common presenting symptoms of MoCD Type A are seizures, feeding difficulties and encephalopathy. Patients with MoCD Type A who survive beyond infancy typically suffer from progressive brain damage, which presents in characteristic patterns on magnetic resonance imaging (MRI). This damage leads to severe psychomotor impairment and an inability to make coordinated movements or communicate with their environment.
About NULIBRY® (fosdenopterin) for Injection
NULIBRY® (fosdenopterin) for Injection is a substrate replacement therapy that provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulfite oxidase, an enzyme that reduces levels of neurotoxic sulfites. NULIBRY is approved by the U.S. Food and Drug Administration, the European Medicines Agency and the Israeli Ministry of Health for the treatment of patients with known or presumed MoCD Type A (discontinued if MoCD Type A is not confirmed), and it is the first and only FDA-approved therapy indicated to reduce the risk of mortality in patients with MoCD Type A. Clinical trials have demonstrated that patients treated with NULIBRY or rcPMP had an improvement in overall survival compared to the untreated, genotype-matched, historical control group.
References
1 Mechler K et al. Genet Med. 2015;17(12):965-970.
2 Schwarz G. Cur Op in Che Bio. 2016;31:179-187.
Sentynl Therapeutics is a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare diseases. The company was acquired by the Zydus Group in 2017. Sentynl's experienced management team has previously built multiple successful pharmaceutical companies. With a focus on commercialization, Sentynl looks to source effective and well-differentiated products across a broad spectrum of therapeutic areas to address unmet needs. Sentynl is committed to the highest ethical standards and compliance with all applicable laws, regulations and industry guidelines. For more information, visit https://sentynl.com.
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