Clinuvel has released positive final results of the open-label pilot study (CUV801) in arterial ischemic stroke (AIS), evaluating multiple doses of afamelanotide in six adult patients.
The company said 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.
CUV801 is the first clinical study assessing afamelanotide as a treatment for a life-threatening brain injury. The study was conducted at the specialist stroke unit of the Alfred Hospital in Melbourne.
"Final analyses from the CUV801 study show that surviving patients who received treatment with afamelanotide all seemed to have recovered well in the six weeks following their brain injury,” said Clinuvel's head of clinical operations Dr Pilar Bilbao.
“Our clinical team often publicly emphasise the significance of afamelanotide as a safe drug in patients, and in this study, we obtained further data that patients with longstanding cardiovascular disease seem to tolerate afamelanotide well. The significance of these findings is of benefit to all our current and future programs.”
The company said 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 in the left half of the brain.
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," said the company, adding analyses "up to day 42 indicated that all five surviving patients showed an improvement in neurological functions and reduction in overall impairment."
"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," it said.
“Despite the considerable impact of strokes on individuals and society, the treatment options available, even at specialist stroke units, are tragically limited,” continued Dr Bilbao.
“We are seeking to prove that afamelanotide can provide a safe, effective treatment option which can improve the overall prognosis post-stroke and reduce patient disability long-term.
“The first steps are to gain comfort that the intervention with afamelanotide poses no harm to patients, while obtaining objective measures of impact of treatment on the course of the patients’ disease. With CUV801 we have achieved both these outcomes and can now pursue further studies and regulatory interactions with a degree of confidence that the drug performs as expected.
“The gain for stroke patients, but also for society as a whole, lies in the improvement in neurological functions, since the ability to resume independent living saves high costs to our healthcare systems,” added Dr Bilbao.