vasopharm Announces Last Patient Last Visit in NOSTRA III Traumatic Brain Injury Clinical Trial

Top-line results expected Q4 2020

WÜRZBURG, Germany, July 1, 2020 /PRNewswire/ -- vasopharm GmbH, a privately held biopharmaceutical company focusing on novel therapeutics for the treatment of cerebrovascular diseases, is pleased to announce today that the Last Patient Last Visit (LPLV) in its phase 3 NOSTRA III clinical trial  occurred on June 17, 2020.

The NOSTRA trial (NO Synthase in TRAumatic Brain Injury) is a pivotal European trial assessing the efficacy and safety of Ronopterin (VAS203) for the treatment of moderately to severely injured closed head traumatic brain (TBI) injury patients. Current approaches to the treatment of acute TBI focus on short term patient mortality and have no significant impact on reducing long-term physical and cognitive deficits. The NOSTRA III trial is a placebo-controlled, randomised, double-blind, multi-centre study in five countries at 29 investigational sites in Austria, France, Germany, Spain and the UK. 223 patients have been enrolled on the trial. The company remains on schedule to deliver headline data in Q4 2020.

Frank Tegtmeier, PhD, Chief Scientific Officer of vasopharm, commented; "We are very pleased to have reached another significant milestone for our lead drug candidate Ronopterin. It is the first drug which targets both blood vessels and tissue of the brain and represents a completely novel pharmacological approach. We are deeply grateful to the investigators and their teams who continued their trial related work in an outstanding manner, especially during these challenging times of COVID-19."

Prof. Dr. Erich Schmutzhard, Medical University of Innsbruck, Austria and Chief Investigator for the trial said: "vasopharm's innovative and differentiated approach has the potential to offer new, advanced treatment options to acute TBI patients and we are looking forward to evaluating this promising and unique target. It is very exciting to see the completion of the NOSTRA III trial and we believe VAS203 will provide physicians with a real opportunity to reduce long-term sequelae of TBI, thereby improving long-term outcomes of TBI patients."

NOSTRA III's primary endpoint is the extended Glasgow Outcome Scale (eGOS) at six months post-trauma; secondary endpoints include Quality of Life (QOLIBRI) at six months post-trauma, QOLIBRI overall scale (QOLIBRI-OS) at three and six months post trauma, eGOS at three months post-trauma, Therapy Intensity Level (TIL) over 14 days after start of treatment and the number of craniectomies.

About vasopharm GmbH

vasopharm is a drug development company focused on small molecule therapeutics for treatment of Traumatic Brain Injury (TBI). vasopharm's lead drug candidate VAS203 (INN Ronopterin) is currently in the Phase III NOSTRA trial assessing efficacy and safety of VAS203 for the treatment of moderately to severely injured closed head traumatic brain injury patients.

Current approaches to the treatment of acute TBI focus on short term patient mortality and have no significant impact on reducing long-term physical and cognitive deficits. VAS203 avoids unwanted side effects, combined with unprecedented clinical efficacy. The European regulator, EMA has granted the drug orphan drug designation for moderate to severe brain injury.

vasopharm was founded in July 1998 as a spin-off from the University of Wuerzburg Medical School by Harald Schmidt, MD, PhD, Professor of Pharmacology and Toxicology, and Ulrich Walter, MD, Professor of Clinical Biochemistry and Pathobiochemistry. To date, the company has raised about Euros 50 million through several financing rounds from various Family Investment Offices and Venture Investors.

See https://www.vasopharm.com/ for further details.

About Traumatic Brain Injury

Traumatic brain injury (TBI) occurs when a sudden trauma, such as a road traffic accident, or a fall, causes damage to the brain. Every year, over 2,500,000 patients sustain a traumatic brain injury in Europe, of that 1,000,000 are admitted to hospital, and 75,000 of these will die, with a further 100,000 being left disabled. TBI is the leading cause of death and disability in young adults. 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. Moderate and severe head injury (respectively) are associated with a 2.3 and 4.5 times increased risk of Alzheimer's disease. See https://www.center-tbi.eu/ for more information. 

About VAS203 (Ronopterin)

VAS203 is an analogue of the natural co-factor tetrahydrobiopterin, 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 sequelae following a traumatic brain injury.  VAS203 is (4-amino-(6R,S)-5,6,7,8-tetrahydro-L-biopterin dihydrochloride dihydrate) a structural analogue of (6R)-5,6,7,8-tetrahydro-L-biopterin, the endogenous cofactor of NOS and phenylalanine hydroxylase.

About NOSTRA III

NOSTRA III is a randomised, double-blind, placebo-controlled trial. The study has enrolled 223 patients who have suffered a moderate to severe TBI resulting in hospitalisation and who have received an intra-cranial pressure probe. 29 European neuro-trauma centres in Germany, Austria, France, UK and Spain are participating. The primary endpoint is the eGOS 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. NOSTRA III* is a registration trial which seeks to validate the data from the highly significant NOSTRA II trial. The study aims to confirm the data from a Phase II trial in which VAS203 has shown a (2 point) improvement in the extended Glasgow Outcomes Score.

*Efficacy of Ronopterin (VAS203) in patients with moderate and severe traumatic brain injury (NOSTRA phase III trial): Study protocol of a confirmatory, placebo controlled, randomised, double-blind multi-centre study. https://trialsjournal.biomedcentral.com/articles/10.1186/s13063-019-3965-4