Californian pharma company Ionis has release Phase III trial results showing its rare disease candidate inotersen hit its efficacy targets.

The NEURO-TTR trial involved patients with familial amyloid polyneuropathy (FAP) - a painful and rapidly progressive genetic disease that leads to early death.

The study found inotersen was effective when it came to delaying symptom progression as well as improving patents’ quality of life.

But the study also showed the candidate was linked to three cases of thrombocytopenia (low platelet levels), one of which resulted in a patient death from stroke.

It’s the latest in inotersen's chequered development history, which last year saw the FDA place a clinical hold on it – because of thrombocytopenia.

Brett Monia, senior vice president of drug discovery and franchise leader for oncology and rare diseases at Ionis Pharmaceuticals, said the firm was excited about the top-line NEURO-TTR results.

“We observed a benefit in disease progression in patients treated with inotersen, regardless of disease stage (Stage 1 and Stage 2) or TTR mutation (V30M and non-V30M). We believe these preliminary results suggest a favourable benefit-risk profile for inotersen in patients with FAP."