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Santhera closer to filing for muscular dystrophy drug

Catena/Raxone slows loss of lung function in late stage trial
Santhera Pharmaceuticals logo

New late stage data has put Santhera Pharmaceuticals  on track to be file its drug to treat respiratory complications in patients with muscle weakness.

Previous results from the DELOS trial in 64 patients with Duchenne muscular dystrophy (DMD) showed that after one year of treatment with Santhera's Catena/Raxone (idebenone), the drug slowed the progressive loss of lung function - measured using hospital-based spirometry assessments - by 66 per cent compared to placebo.

Now, secondary outcome data from the study indicates that when lung function is measured by a patient themself using a handheld peak flow meter, the difference between the active drug and placebo was even more profound.

After one year peak expiratory flow (PEF) rates were 80 per cent better in patients on Catena/Raxone compared to the placebo group, while forced expiratory volume (FEV1) measurements were 78 per cent better.

The supportive data are "clearly supportive and establish the clinical meaningfulness of Catena/Raxone treatment," commented Santhera's head of development Nick Coppard.

"We believe we are well positioned to discuss regulatory approval with the authorities," he added. Santhera had previously filed for approval of the drug as a treatment for the rare eye condition Leber's hereditary optic neuropathy (LHON) but had its application turned down by the European Medicines Agency (EMA).

News of the positive results is a welcome boost for patients with DMD, who were deeply disappointed by Prosensa's antisense candidate drisapersen failure to show efficacy in a phase III trial last year.

Rather than tackling the complications of DMD, drisapersen is designed to tackle the underlying genetic defect in the disease by producing a truncated but semi-functional form of the dystrophin protein that is abnormal in DMD, restoring some muscle function.

Prosensa has said it is still committed to developing drisapersen for DMD despite a decision by former partner GlaxoSmithKline (GSK) to hand back rights to the programme.

Meanwhile, there was further encouraging news this week with the announcement of positive phase Ia results for Summit's utrophin modulator SMT C1100, with the drug found to be safe and able to dampen down levels of a market for muscle fibre damage. Summit says it intends to start phase II trials of the drug later this year.

23rd May 2014

From: Research

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