Phase III traumatic brain injury trial does not meet primary endpoint

pharmafile | June 3, 2021 | News story | |   

vasopharm GmbH has announced that its NOSTRA III traumatic brain injury (TBI) Phase III clinical trial of ronopterin did not meet its primary endpoint.

However, hypothesis driven post-hoc analysis reveals a statistically significant and clinically meaningful increase in eGOS over time in patients with moderate and severe TBI when ronopterin is infused within 12 hours after trauma.

The NOSTRA III trial is a pivotal European trial assessing the efficacy and safety of ronopterin for the treatment of patients with moderate and severe TBI.

Current treatments for acute TBI are predominantly supportive measures, initiated reactively, to decrease elevated intracranial pressure with the aim of reducing mortality and morbidity. To date, no specific pharmacologic intervention has been demonstrated to improve long-term physical and cognitive recovery.

Ronopterin is the first drug which simultaneously targets blood vessels and tissue of the injured brain by reducing the excessive production of nitric oxide via upregulated inducible NO synthase (iNOS).

Professor Dr John Stover, Chief Medical Officer of vasopharm, said: “While NOSTRA III did not achieve statistical significance in the pre-specified primary endpoint of eGOS at six months after trauma, we performed a detailed hypothesis driven post-hoc analysis guided by pathophysiology combined with biochemistry and pharmacology.

“This analysis clearly reveals the potential of ronopterin to benefit patients when infused within the initial 12 hours after TBI.

“This efficacy, in the opinion of the Data Monitoring Committee, clearly outweighs the known adverse event profile, resulting in a positive benefit-risk assessment. We consider these results robust and clinically relevant, and vasopharm will seek scientific advice from Competent Authorities on the further regulatory pathway for ronopterin.”

Frank Tegtmeier, Chief Scientific Officer of vasopharm, commented: “We are pleased that we have identified a clinically coherent explanation for the results of the pre-specified analysis and that we can provide a clear rationale for the beneficial use of ronopterin in patients with moderate and severe TBI.

“The early infusion and trajectory of improvement over time are supported by recent publications of observational studies and randomised controlled Phase III trials.”

Lilly Subbotin

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