What Will Retrophin Inc (RTRX) Get For Its Money On A Raptor Pharmaceutical Corp. (RPTP) Deal?

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It works in three different ways to target Huntington’s. First, it increases the synthesis of an amino acid called cysteine. The science behind this is pretty hazy right now, but low levels of cysteine have been linked to Huntington’s as well as things like Alzheimer’s, cancer and a few other diseases. The theory is that by increasing cysteine, severity can halt or reverse. The second is the inhibition of what’s called transglutaminase, which translates to a reduction in a process called proteolysis. The proteolysis of mutant huntingtin (the protein created by the mutant gene that cases the condition) is believed to contribute to the progression of the disease. Reducing proteolysis should, theoretically, reduce progression rate. Finally, it promotes the expression of BDNF, which in turn promotes neuronal survival and synaptic plasticity.

Trials to date have been promising, and we believe Retrophin Inc (NASDAQ:RTRX) is looking to pick up this late stage asset ahead of the pivotal completion, with the goal of submitting an NDA itself. The 96 patient trial is ongoing, and is pitching RP103 against placebo across an eighteen-month study period. It’s double blind, so we’ve not got any real insight into how the drug is performing as yet, but Retrophin must believe it has a solid chance of success if it is considering taking over the development.

So that’s the primary target, what else will Retrophin pick up? Raptor’s secondary candidate is a cystic fibrosis target called MP-376. This one is an inhalable formulation of fluoroquinolones, designed to inhibit replication of bacterial DNA in CF patients. It’s essentially a reformulation based on an alternative administration (a nebulizer) designed to improve efficacy and increase time to action. While not as neatly as RP103 might, MP-376 also fits into Retrophin Inc (NASDAQ:RTRX)’s mission statement.

The deal is rumored to amount to somewhere in the region of $600 million, but chances are we will see a premium on that price given Raptor Pharmaceutical Corp. (NASDAQ:RPTP)’s current buy-volume-driven market cap of $638 million.

We’ll keep an eye on this as it plays out.

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Note: This article is written by Mark Collins and was originally published at Market Exclusive.

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