Israeli stroke treatment developer BrainsGate, (in which Elron Electronic Industries (TASE: ELRN) holds a 30% stake), has today published the complete results from its leading trial of a device designed to reduce damage from stroke. The trial did not find that using the device was beneficial in comparison with the control group. In a group composed solely of cortex stroke patients (patients suffering damage to the cerebral cortex, the front area of the brain, which is more vulnerable), however, an effect was found that barely missed the requirements set for the trial.
BrainsGate set two main targets for the trial: improvement in the state of the stroke in the entire group and improvement in the cortex patients (whose state was more serious). Since the analysis of cortex patients only is a second analysis on the same set of data, however, the company received a statistical "penalty" - the burden of proof was made more difficult. It had to achieve significant results at a 0.025 level of significance, instead of 0.05. The company just missed this target; the p-value for the trial was 0.0258.
BrainsGate now plans to consult with the US Food and Drug Administration (FDA) in the hope of submitting its product for approval on the basis of these results, even though they were marginal. It is believed that the company's aim is to nevertheless register the product for all stroke victims, not just cortex patients, because it is frequently difficult to distinguish in real time exactly what stroke a patient has suffered. Significant use of the product in actual conditions will therefore require approval for general use.
Partial results from this trial were published last August. The company did not publish numerical data at the time, but stated then that it had not met the statistical target in either of the two tested groups. At the same time, it explained that "benefit for stroke patients" had been observed in the trial, although the results were not significant. BrainsGate published the results on two occasions in order to keep some of the data for an exclusive presentation at a conference of stroke doctors currently taking place.
The ball is now in the court of the FDA, which must decide whether the results published by BrainsGate are convincing, even though they did not meet the official target. There are precedents for such approvals in the past (for example, a product made by Given Imaging, which was approved on a limited scale on the basis of results that met the target exactly; the company had to conduct a follow-on trial of the product after it was already on the market). There are also negative precedents, however, in which a company believed that the FDA would approve its product on the basis on the basis of retroactive analysis of results that did not meet the official targets, but was proved wrong (this happened with Kamada's AAT inhalation product, for example).
BrainsGate's trial, which included 1,000 patients, was one of the largest ever trials of a medical device for stroke. Due to its size, it was a very long trial, lasting seven years. The trial was originally intended to be smaller, but was expanded after the committee saw signs of effectiveness that were not statistically significant. The idea was that enlarging the trial would make it possible to attain statistically significant results. The results reported today show that the expansion of the trial was only partially successful.
Published by Globes, Israel business news - en.globes.co.il - on October 21, 2018
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