SYGNIS successfully completes clinical study IIa for AX200 in stroke (AXIS)

Primary and secondary endpoints reached

HEIDELBERG, Germany | July 23, 2007 |
NIS Pharma AG, focused on the exploration, development and commercialisation of innovative drugs for the treatment of neurodegenerative disorders successfully completed the clinical phase IIa study for AX200 in stroke (AXIS). After a comprehensive evaluation of the study data, the use of AX200 in stroke patients can be considered to be safe. This evaluation has also been supported by the opinion of the independent Data Safety Monitoring Board, which has not expressed any concerns regarding the use of AX200 in stroke patients. Thus, the path has been cleared for the further clinical development of AX200.

AX200 with dual therapeutic approach
AX200 for the treatment of acute stroke is the most advanced drug candidate in SYGNIS’ product pipeline. The AX200 protein is produced at highly elevated levels in the brain, as part of an endogenous protective mechanism after brain damage. Thus, if this drug administered in the acute phase of stroke, the brain’s own protective action is further boosted. This offers a highly beneficial, dual neurotherapeutic approach which has been discovered by SYGNIS. AX200 stops neuronal cell death in the acute phase of stroke, and in addition, it stimulates the regeneration of the already damaged nervous tissue through the induction of new nerve cells and blood vessels and the reorganisation of neuronal networks. Besides stroke, AX200 is currently in preclinical testing for further neurodegenerative indications such as Amyotrophic Lateral Sclerosis (ALS).

AXIS-study IIa - AX200 is safe and well tolerated
AXIS stands for "AX200 in Acute Ischemic Stroke", a national, multicenter, randomised, doubleblind, placebo-controlled dose escalation study. 4 different doses of AX200 were tested in 6 centers in Germany (Münster, Leipzig, Heidelberg, Hamburg, Wiesbaden, Erlangen). Altogether 44 patients were enrolled in the trial, 14 patients were administered placebo, and 30 received AX200. The primary aim of the study was to show safety and tolerability of AX200 in acute stroke patients. A particular demand was to clarify whether the increase of leucocyte (white blood cell) production might have a negative influence on the stroke process. The study has clearly shown that this is not the case and that the increase in leucocytes is only short-term and rapidly decreases after completion of the three-day therapy. None of the other measured blood parameters showed any clinically relevant modifications. The study also demonstrated that during the AX200 treatment there was no increase of thromboembolic incidence or bleeding complications, an important condition for the further development of the stroke drug. The overall study results have demonstrated the tolerability of AX200 in stroke patients. This is also supported by the fact that no patient aborted the study.

Increased probability of success for further clinical development
Although the focus of the AXIS-study was the collection of data regarding safety and tolerability, data regarding the efficacy of AX200 were also monitored (clinical outcome, infarct growth). The primary goal was to gain insights for the optimization of future efficacy studies such as inclusion criteria or endpoints.

A detailed statistical evaluation of the AXIS-data provided hints that certain stroke patients may benefit from treatment with AX200. Since AXIS has been designed as safety study with a small number of patients, a significant difference in the overall consideration of the usual clinical endpoints could accordingly not be establish. These insights will serve to help guide the design of the following phase IIb trials.

"The study has achieved all of its aims" emphasized the Principal Investigator PD Dr. Wolf-Rüdiger Schäbitz, University Hospital Münster. "Beyond the demonstration of safety, the escalating doses of the compound in stroke patients has also provided valuable hints for the planning and implementation of further clinical development. For subsequent statistical analyses of the data, as well as in the design of the next clinical development phase (IIb), we will be able to build on the possibility that certain types of stroke might show an exceptionally beneficial reaction to AX200".

"We are highly satisfied with the excellent quality and quantity of the data produced by he participating study centers", stressed Dr. Alfred Bach, CEO of SYGNIS Pharma G: "The study results provide us with great encouragement to proceed with the xpanded clinical development of AX200. We are committed to conduct an efficacy tudy as soon as possible and hope to demonstrate that AX200 delivers the promise nd the drug will provide an effective and safe treatment option for stroke patients."Stroke affects over 5 million patients worldwide every year and is the third leading ause of death, presenting a major socio-economic burden.


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