About Neurophyxia

Neurophyxia B.V. is an innovative drug development company, focusing on disorders affecting the newborn child. The lead product, 2-Iminobiotin (2-IB) is a biotin (Vitamin H or B7) analogue and is presently in development for the treatment and prevention of the devastating effects of perinatal asphyxia (oxygen shortage at birth).

The product is currently not on the market, and can therefore not be made available for use in patients.

Despite major improvements in obstetric and perinatal care, perinatal asphyxia remains one of the major causes of neonatal morbidity and mortality. Depending on the literature cited, 4% to 20% of those affected die and 25% are severely handicapped due to long-term complications such as cerebral palsy, mental retardation with learning difficulties, cerebral visual impairments and/or epilepsy. Also globally, asphyxia is responsible for about one fourth of the neonatal death percentage.

It is increasingly being recognized that newborns with rather mild asphyxia, who initially seem to recover without complications, have cognitive problems, which can eventually be traced back to the perinatal period. Until now, no specific pharmacological therapy is available to treat or prevent perinatal asphyxia. 2-Iminobiotin could be the first registered pharmaceutical therapy for this devastating condition.

The annual number of patients in the Western world is estimated at 50.000 to 100.000, and the product is an Orphan Product according to US and EU guidelines.

2-Iminobiotin is a selective nitric oxide synthase (NOS) inhibitor. Nitric oxide (NO) plays a critical role in the development of perinatal asphyxia. Its synthesis occurs through three nitric oxide synthase (NOS) iso-enzymes, two of which can be inhibited selectively by 2-IB.

Phase I clinical testing started December 2009, and preliminary results obtained during a halfway interim analysis have shown an excellent safety profile. The study will be completed in the second quarter of 2010, and subsequently, a Phase II clinical study will be started to provide clinical proof of principle for 2-IB in term infants. In this study, patients will receive treatment within a few hours after birth.

Once the efficacy of 2-IB has been established in these patients, the company will attempt to broaden the target population to preterm infants. Also mothers who are at risk of giving birth to children with perinatal asphyxia may be eligible for treatment, in which case the drug will be given just before parturition.

The company collaborates with established contract research organizations in the areas of:

  • Manufacturing of drug substance: Partner in the UK
  • Formulation of drug product: Partners in the Netherlands, Israel and the USA
  • Storage and distribution of pharmaceutical products: Partner in the Netherlands
  • Preclinical safety studies: Partner in the US and in the Netherlands
  • Preclinical efficacy studies: University groups in the Netherlands and Australia
  • Phase clinical I study: PRA International, Zuid Laren, the Netherlands