How Large Is Prothena's Market Opportunity?

Source: Thinkstock Parkinson’s disease is a degenerative disorder that affects 50,000 to 60,000 new cases every year. There may even be 7 million to 10 million people living with this disease worldwide. In fact we have probably seen a few in the spotlight, such as Michael J. Fox or Muhammad Ali. A new potential breakthrough in treatment has come to light in the fight against this disease. Prothena Corp. PLC (NASDAQ: PRTA) announced positive results from a Phase 1 single ascending dose study of PRX002, a monoclonal antibody for the potential treatment of Parkinson’s disease and other related synucleinopathies.

A little background on Prothena: it is a late-stage clinical biotechnology company focused on the discovery, development and commercialization of novel protein immunotherapy programs.

Now, according to the Phase 1 study data, PRX002 was safe and well-tolerated, meeting the primary objective of the study. Results from this study showed that administration of PRX002 leads to mean reduction of free serum alpha-synuclein levels of up to 96%.

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To put it in slightly simpler terms, Gene Kinney, PhD, Chief Scientific Officer and Head of Research and Development at Prothena, said:

Importantly and for the first time in humans, we demonstrated that this robust, rapid and dose-dependent reduction of free serum alpha-synuclein was safe and well-tolerated. Thus, this approach may translate into a clinically meaningful delay or reversal of disease progression in patients with Parkinson`s disease. We look forward to building upon these data with results from the on-going, multiple ascending dose study in patients with Parkinson`s disease expected in the first half of 2016.

In December 2013, Prothena and Roche entered into a worldwide collaboration to develop and commercialize antibodies that target alpha-synuclein, including PRX002. So far, Prothena has received roughly $45 million of the potential $600 million of total milestones from its collaboration with Roche. In the United States, Prothena has an option to co-promote PRX002 and both the companies will share all profits and costs, on a 30/70 basis (30% Prothena and 70% Roche).

The truth of the matter is that if any company were to come up with a truly effective treatment for Parkinson’s, it would be an immediate blockbuster drug and would be incredibly profitable. Still, 24/7 Wall St. would remind readers that this was a Phase 1 study rather than a late-stage study, and it was a double-blind, placebo-controlled, single ascending dose study, which enrolled 40 healthy volunteers. There is still a long road ahead for Prothena.

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Shares of Prothena soared 40% at $40.80 in the premarket session on Friday. Its 52-week trading range is $16.71 to $48.46, and the stock has consensus analyst price target of $40.29.