(Sharecast News) - Commercial-stage biopharmaceutical company Amryt Pharma released efficacy and safety data from the 'Lochnes' investigator-sponsored study of lomitapide in familial chylomicronemia syndrome (FCS) on Tuesday, which was conducted by expert lipidologists in Italy.
The AIM-traded firm said the study enrolled 18 adult patients across three sites, with all patients having a genetic diagnosis of FCS and a history of pancreatitis.
It said the median baseline fasting triglyceride level of patients was 1884 mg/dL, adding that patients were administered the open-label treatment with escalating doses of lomitapide for 26 weeks.
The median fasting triglyceride level at 26 weeks was 672 mg/dL representing a 70.5% median reduction versus the baseline.
Amryt said 14 patients achieved a reduction in triglycerides to less than 1000 mg/dL, and 13 of them achieved triglycerides of 750 mg/dL or below at 26 weeks.
Treatment with lomitapide was "generally well-tolerated", and all patients completed 26 weeks of treatment.
It said adverse events were mild to moderate, and mostly related to gastrointestinal tolerability and liver enzyme elevations.
Liver MRI imaging revealed an increase in hepatic fat content, which was between 30% and 50% at week 26 in three patients.
The company said it would now evaluate and analyse the full data set from the study, and planned to discuss the potential development path forward with both the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
"We are encouraged by the data from the study, and look forward to our discussions with the respective health authorities to progress lomitapide as a potential treatment for FCS," said chief medical officer Dr Mark Sumeray.
Professor Maurizio Averna, lead investigator, and Professor Angelo Cefalù, study co-investigator, said the results of the study supported the hypothesis that lomitapide could be a potential treatment for reducing triglycerides in FCS.
"Interestingly, at the end of the study, 14 out of 18 patients achieved triglyceride levels below 1000 mg/dL , which is considered the pancreatitis risk threshold," they said in a joint statement.
At 0934 BST, shares in Amryt Pharma were up 0.49% at 207p.
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