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Ionis says rare disease drug works, but safety concerns linger

Inotersen has been linked to cases of thrombocytopenia

IONISIonis has reported that its rare disease therapy inotersen hit its efficacy objectives in a phase III trial - but also had side effects that could make getting regulatory approval a challenge.

The phase III NEURO-TTR trial in patients with familial amyloid polyneuropathy (FAP) - a painful and rapidly progressive genetic disease that leads to early death - found that the drug was effective in delaying symptom progression and improving patents’ quality of life.

However Ionis' drug was linked to three cases of low platelet levels (thrombocytopenia), one of which resulted in a patient death from stroke, while another patient stopped therapy after platelet levels started to decline. Study investigators also reported two cases of severe kidney damage and another patient stopped treatment due to changes to kidney biomarkers.

It is not the first time that inotersen's potential has been brought into question. Last year the FDA placed a clinical hold on the drug - also known as IONIS-TTRx - because of thrombocytopenia, and this was followed by partner GlaxoSmithKline (GSK) handing back rights to the programme - although it still has an option to buy back in.

FAP is one of a number of conditions linked to the deposition of transthyretin-related (TTR) amyloidosis, for which there are no approved therapies and a desperate need for effective drugs. With Ionis' efficacy data for the drug still only top-line, the big question is whether the safety issues will be outweighed by inotersen's benefits.

Investors were spooked by the risks and Ionis shares declined 11%, while rival Alnylam - which is expecting phase III results for its FAP therapy patisiran in the coming months - rose 13%. Alnylam dropped lead amyloidosis drug revusiran last year after reports that it worsened nerve damage in a late-stage trial, bringing in patisiran as a replacement.

"FAP is a devastating genetic disease that is painful and rapidly progressive leading to early death," said Morie Gertz, a haematology and oncology specialist at the Mayo Clinic who works a lot with amyloidosis patients.

"The positive results from the NEURO-TTR study today are very encouraging for this underserved patient population [and] I believe inotersen has the potential to transform the current standard of care for patients with TTR amyloidosis."

Ionis' chief executive Stanley Crooke said during the firm's first-quarter results call earlier this month that the company was working with GSK towards preparing first regulatory filings for inotersen before the end of the year.

Article by
Phil Taylor

17th May 2017

From: Research

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