Pharma, Startups

Knopp Biosciences raises $5M, pivots flailing ALS drug to target new indication

A lot of hopes were pinned on using dexpramipexole to treat those with ALS. But it […]

A lot of hopes were pinned on using dexpramipexole to treat those with ALS. But it the small molecule drug, thought to maintain mitochondrial function, flopped in Biogen Idec’s Phase 3 trials last year: Though fast-tracked in 2009 for the indication, it failed to show any statistical efficacy in improving the symptoms or lifespan of patients with Lou Gehrig’s disease.

That said, Pittsburgh startup Knopp Biosciences is still actively studying dexpramipexole: It just raised about $5 million in a recent fundraise from 18 investors, and has the go-ahead from the NIH to research the compound in a host of other diseases – including ALS.

Of course, this is small potatoes for a company that raised $140 million in 2010 – in the days when dexpramipexole was promising for ALS – but this recent funding round shows that investors still have some faith in the molecule’s effifacy.

Here’s Knopp’s pipeline:

The NIH’s National Institute of Allergy and Infectious Diseases is working with Knopp to conduct a clinical trial of dexpramipexole in hypereosinophilic syndrome, or HES, which causes multiple organ damage.

Dexpramipexole is closely related to pramipexole, a dopamine agonist commonly used in treating Parkinson’s disease and restless legs syndrome under trade names Mirapex, Mirapexin and Sifrol.

 

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