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Retrophin rockets away as rare disease trial hits targets

Sparsentan has potential to become a blockbuster FSG drug, according to analysts

RetrophinWhat a difference a year makes. Under pressure in 2015, US firm Retrophin now has a positive trial in hand for a rare disease drug that some analysts say could approach $1bn in annual sales.

Last year, Retrophin was indirectly caught up in the scandal surrounding its founder Martin Shkreli - who was eventually fired by the board and faced allegations of using company stock to pay off debts incurred in his other business dealings.

Now, the company says a phase II trial of sparsentan in focal segmental glomerulosclerosis (FSG), a rare disease characterised by progressive scarring of the kidneys that often leads to kidney failure, has met its primary objective.

Shares in Retrophin shot up after the company announced it would now start talking to the FDA about accelerated approval for the drug, which could become the first licensed treatment for the estimated 40,000 people with FSG in the US. Leerink analyst Joseph Schwartz has suggested the drug could make $950m in sales from use in FSG.

Sparsentan was brought into Retrophin's pipeline by Shkreli in 2012 - who licensed it from Ligand Pharmaceuticals - and combines angiotensin receptor blockade with endothelin receptor blockade, both mechanisms already exploited by drugs for high blood pressure.

In the 109-patient DUET trial it was shown to reduce levels of protein in the urine, a biomarker for kidney damage, more effectively than the control drug irbesartan.

Shkreli took to Twitter to trumpet the success of "the first drug I ever did research on", with a couple of side-swipes at the management of Retrophin, who are suing him in a $65m lawsuit. He also suggested there could be plenty of scope for follow-up indications for the drug.

He added that after taking on the drug a decision had to be taken on developing it for FSG or another rare kidney disease called IgA nephropathy, as there was only budget for one programme.

Article by
Phil Taylor

8th September 2016

From: Research, Sales

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