CLINUVEL released positive final results of the open label pilot study (CUV801) in arterial ischemic strokereleased positive final results of the open label pilot study (CUV801) in arterial ischemic stroke (AIS), evaluating multiple doses of CLINUVEL's drug afamelanotide in six adult patients. Afamelanotide was shown to be well tolerated, with five of the six patients showing considerable clinical and functional recovery up to 42 days after treatment.
STUDY RESULTS CUV801: CUV801 is the first clinical study assessing afamelanotide as a treatment for a life-threatening brain injury. All six patients enrolled in the study carried an increased risk of stroke due to their history of cardiovascular disease, elevated blood pressure or diabetes type II, and all suffered a stroke (blood clot) in the left half of the brain. The study was conducted at the specialist stroke unit of the Alfred Hospital in Melbourne, Australia. Safety was the primary endpoint of the pilot study with afamelanotide administered up to four times over ten days following the stroke. This frequency of dosing seemed not to affect patient safety, with no drug-related adverse events reported during or after the study completion. One patient with a complex cardiovascular history passed away following a second stroke on day 5, which was assessed as unrelated to afamelanotide treatment.
Treatment efficacy was measured using computer imaging to assess the volume of the area affected by the stroke, and validated clinical assessments of function, neurological impairment, and disability. Analyses of the brain scans (MRI-FLAIR2) performed at days 3 and 9 showed a reduction in size of the affected area in five of the six patients. Analyses of the NIHSS scores1 up to day 42 indicated that all five surviving patients showed an improvement in neurological functions and reduction in overall impairment (p=0.0625). Four out of five surviving patients showed an improvement of 4 points or more on the scale, regarded as significant, and all five patients reported a clinically meaningful reduction of 3 points. Two patients were symptom free at day 42.