vasopharm Announces Enrolment of First Patient In Phase III Traumatic Brain Injury (TBI) Trial
NOSTRA III trial to assess efficacy and safety of Ronopterin (VAS203) in the treatment of moderately to severely injured traumatic brain injury patients
vasopharm GmbH, a privately held biopharmaceutical company focusing on novel therapeutics for the treatment of cerebrovascular diseases, today announced that the first patient has been enrolled in to the NOSTRA III (NO Synthase in TRAumatic Brain Injury) trial – a phase III clinical trial assessing efficacy and safety of Ronopternin (VAS203) for the treatment of moderately to severely injured closed head traumatic brain (TBI) injury patients.
Traumatic brain injury (TBI) is the leading cause of death and disability among young adults and occurs when a sudden trauma causes damage to the brain. Every year, over 1,600,000 patients sustain a traumatic brain injury in the EU, and 70,000 of these die, with a further 100,000 being left disabled.
Christian Wandersee, Chief Executive Officer of vasopharm, commented:
“We welcome the recruitment of the first patient in to this pivotal phase III trial. The phase III trial is a key test to confirm our belief in the clinical efficacy of Ronopternin (VAS203) and its role in the treatment of moderately to severely injured closed head traumatic brain injury patients and leads us another step closer to bringing a drug for a highly unmet need to market. We believe VAS203 will provide physicians with a real opportunity to improve long-term outcomes for patients with this devastating condition.”
About the NOSTRA III Trial
VAS203 is an investigational Nitric Oxide Synthase inhibitor, which demonstrated statistically significant improvements to both short term (Therapy Intensity Level) and long term (extended Glasgow Outcome Scale, 6 months and 12 months) measures of treatment efficacy in a clinical phase II trial (1).
The NOSTRA III European confirmatory multicentre, randomised, double-blind, placebo-controlled trial is planning to enrol 232 patients suffering from a moderate to severe TBI who are hospitalised and have received an intra-cranial pressure probe. The study is designed to evaluate efficacy and safety of VAS203. In total, 35 European neuro-trauma centres in Germany, Austria, France, UK and Spain will participate in the trial. The intravenous administration of VAS203 will be applied between six and 18 hours after the injury and the infusion period will last for 48 hours. The primary endpoint will be the extended Glasgow Outcome Scale evaluated at six months after the injury. Secondary efficacy assessments include Quality of Life (QOLIBRI) as well as Therapy Intensity Level (TIL) over 14 days after brain injury. Data read-out (final clinical report) of the study is estimated for mid-2019.
For further information, please contact:
Christian Wandersee, CEO
Mary Clark, Eva Haas, Hollie Vile
Tel: +44 2034405654
Notes to editors:
About Traumatic Brain Injury:
Traumatic brain injury (TBI) occurs when a sudden trauma causes damage to the brain. Every year, over 1,600,000 patients sustain a traumatic brain injury in the EU, and 70,000 of these die, with a further 100,000 being left disabled. Significantly, 75% of the victims are children and young adults, and TBI is the leading cause of disability in people under 40 years of age. Traumatic brain injury results in more lost working years than cancers, stroke and HIV/AIDS together. On a global scale, the number of life years lost due to traumatic brain injury is four times that of diabetes-related loss. Recent statistics show a steep increase in the incidence of TBIs, with an increase of 21% over the last five years – threefold greater than the rate of increase in population, at an annual cost of over Euros 100 billion. Despite this, TBI has been seriously underrepresented in medical R&D efforts compared to many other, less significant health problems. (2)
- Olesen, J. et al,, "The economic cost of brain disorders in Europe.", Eur J. Neurol. 2012 Jan (19) 1: 155 – 62
VAS203 is an analogue of the natural co-factor biopterin, which is involved in the generation of nitric oxide by the Nitric Oxide Synthase (NOS) family of enzymes. The mechanism of action of VAS203 is believed to confer selective down regulation of inducible NOS (iNOS) without significantly inhibiting the function of other NOS enzymes. It is believed that iNOS has a significant involvement in the cascade of damaging sequellae following a traumatic brain injury. Technical : VAS203 is (4-amino-(6R,S)-5,6,7,8-tetrahydro-L-biopterin dihydrochloride dehydrate) a structural analogue of (6R)-5,6,7,8-tetrahydro-L-biopterin, the natural endogenous cofactor of NOS and phenylalanine hydroxylase. Data for pharmacokinetics, pharmacodynamics, metabolites and surrogate markers measured in the brain during VAS203 studies is a key differentiator to all other previous clinical trials in TBI which provides strong support for the potential of a positive outcome in this trial.
About vasopharm GmbH:
vasopharm is focused on the development of small molecule therapeutics which modulate the bioavailability of biological NO, by addressing the entire NO/cGMP signal cascade and its functional counterpart NOX. vasopharm's drug candidate VAS203 represents a completely new class of NOS modulators targeting cerebral vessels and cerebral tissue. For VAS203, vasopharm received orphan drug designation for the treatment of moderate and severe TBI in Europe.back